Archive for the ‘Gene Therapy Doctor’ Category

Timothy Ray Brown, dubbed the Berlin Patient, the first ever to be cured of HIV, died from cancer on September 29.

Brown was cured of HIV in 2007. He was diagnosed in 1995, and about a decade later was diagnosed with leukemia. A physician at the Free University of Berlin used a stem cell transplant from a donor with a rare genetic mutation that provided natural resistance to HIV in hopes of curing both diseases. It took two procedures but was successful, and in 2008 Brown was announced free of both HIV and leukemia.

Two years later he went public with the announcement.

I am living proof that there could be a cure for AIDS, he told Agence France-Presse (AFP) in 2012. Its very wonderful, being cured of HIV.

In 2019, a second HIV patient, Adam Castillejo, underwent a similar procedure. He was dubbed the London Patient. A UK resident diagnosed with HIV in 2003, Castillejo began antiretroviral therapy in 2012. He was later diagnosed with advanced Hodgkins lymphoma. He was treated with a stem cell transplant in 2016 after he received chemotherapy. He then continued to receive antiretroviral therapy for 16 months.

To evaluate whether the HIV-1 infection was actually in remission, he went off the usual antiretroviral therapy. After he had been in remission for 18 months, testing confirmed that his HIV viral load was undetectable.

The donors for both men carried a rare genetic mutation called CCR5-delta 32. This made these patients resistant to HIV. Castillejo is currently living HIV-free.

Brown, 54, was born in the U.S. He was diagnosed with HIV in 1995 while living in Berlin. He developed acute myeloid leukemia in 2007.

The leukemia that eventually led to his HIV cure returned this year, where it metastasized to his brain and spinal cord.

Browns partner, Tim Hoeffgen, posted on Facebook, It is with great sadness that I announce that Timothy passed away surrounded by myself and friends, after a five-month battle with leukemia. Tim committed his lifes work to telling his story about his HIV cure and became an ambassador of hope.

The procedure itself is not routinely used to treat HIV because it is both too risky and aggressive. It is primarily used to treat certain types of cancer. In the case of both Brown and Castillejo, it was the combination of HIV and resultant cancers that are effectively treated with stem cell transplants, that made it feasible. Nonetheless, it gave patients hope that there may someday be a cure.

We owe Timothy and his doctor, Gero Hutter, a great deal of gratitude for opening the door for scientists to explore the concept that a cure for HIV is possible, stated Adeeba Kamarulzaman, president of the International Aids Society (IAS).

Sharon Lewin, director of the Doherty Institute in Melbourne, Australia, noted, Although the cases of Timothy and Adam are not a viable large-scale strategy for a cure, they do represent a critical moment in the search for an HIV cure. Timothy was a champion and advocate for keeping an HIV cure on the political and scientific agenda. It is the hope of the scientific community that one day we can honor his legacy with a safe, cost-effective and widely accessible strategy to achieve HIV remission and cure using gene editing or techniques that boost immune control.

Although largely a treatable disease, HIV/AIDS affects about 37 million people globally, and about 1 million people die from HIV-related causes each year. Treatment typically involves a cocktail of antiretroviral therapy, which HIV patients take their entire lives.

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Timothy Ray Brown, First Patient to Be Cured of HIV, Dies of Leukemia - BioSpace

If youre a parent, you know the thoughts, feelings and emotions that come with protecting your child. Youd do anything for them to ensure they live a long, healthy life. But what will you do when there are times when health conditions, such as childhood cancer, stand in the way?

Cancer in children is more common than you may think, as it accounts for the second-leading cause of death behind accidents. While a cancer diagnosis, or even the thought of your child developing cancer one day, is overwhelming, modern medicine has improved survival rates. As with any cancer, early detection is key. To help you familiarize yourself with childhood cancer, we asked Michael Confer, M.D., a radiation oncologist at the INTEGRIS Cancer Institute, about the different types of cancers, which signs to look for and how they can be treated.

Unlike adult cancers, which can result from the environment or exposure to certain things, childhood cancers result from genetic mutations that occur early on in life or before birth.

It all comes down to changes in genes. Your DNA contains information to make different types of cells in your body. In other words, your skin cells contain information to be brain cells, while your kidney cells contain information to be heart muscle cells. As cells mature, they become specialized, turning on and off certain genes to allow them to perform specific duties. Cells need to be able to replicate to replace damaged cells of the same category. They grow with help from genes called proto-oncogenes.

When your DNA changes, it leads to genetic mutations, and cells can become permanently activated. This can lead to cells duplicating uncontrollably, known as cancer.

Tumor suppressor genes slow down cell division. They repair DNA mistakes before cells divide and control cells internal death process (apoptosis or programmed cell death), Dr. Confer says. DNA mutations within tumor suppressor genes can also allow cells to duplicate uncontrollably. Children can be born with mutated proto-oncogenes or tumor suppressor genes in certain cells. These abnormally programmed cells lead to most childhood cancers.

So, what causes DNA changes? Your child can inherit genes from a parent that increases their risk of cancer or they can acquire these genes. Cancers from acquired, sporadic gene mutations are more common than those from inherited gene mutations 5% of all childhood cancers come from inherited mutations.

Breast cancer and ovarian cancer are the most common types of cancer caused by inherited DNA changes from BRCA1 or BRCA2 gene mutations. Even with how well-known these are, only 5 to 10% of breast cancer cases come from BRCA1/BRCA2 inherited mutations. Plus, breast cancer and ovarian cancer are more common in adults than children. Talk to your doctor or visit a genetic counselor if you have specific questions about inherited mutations.

Cancer can impact any part of your body, ranging from your bones and blood cells to your brain, spinal cord and other internal organs. You may be most familiar with leukemia, lymphoma, and brain and spinal tumors, since they are the most common. But, here is a full overview of cancers that commonly affect children, according to the American Cancer Society.

Leukemia: This is the most common type of cancer in children, accounting for 28% of cases. It generally starts in white blood cells and becomes fast growing (acute). Acute lymphocytic leukemia (ALL) and acute myeloid leukemia (AML) are the two most common types of leukemia. Three out of every four children with leukemia have ALL. This type of cancer starts in the lymphoid cells, called lymphocytes, whereas AML starts in myeloid cells. Chronic myeloid leukemia (CML) and chronic lymphocytic leukemia (CLL) are two types of rare cancers

Brain and spinal cord tumors: These types of cancers make up 26% of all cases. Brain tumors are more common than spinal cord tumors. The cancer generally starts in the lower part of the brain.

Neuroblastoma: This type of cancer affects infants and young children. Neuroblastoma starts in nerve cells during pregnancy and accounts for 6% of childhood cancers. Abdomen swelling is a common sign of neuroblastoma.

Wilms tumor: This type of cancer starts in the kidneys and impacts children who are 3 or 4 years old. It accounts for 5% of childhood cancers.

Lymphoma: Although lymphoma isnt as common as other types of cancers, youve likely heard of Hodgkin lymphoma and non-Hodgkin lymphoma, the two main types of cancer that show up in the lymphocytes. Non-Hodgkin lymphoma (accounts for 5% of childhood cancers) appears in younger children and is more common than Hodgkin lymphoma (accounts for 3%), which is more common in younger adults. You may notice a swollen lymph node under your childs arm or near their throat.

Rhabdomyosarcoma: This type of cancer develops in areas that your child uses to move their body, such as the head, pelvis, arms or legs. It accounts for 3% of childhood cancer cases.

Retinoblastoma: This cancer develops in the eyes, and your child is most at risk around the age of 2 until the age of 6. It accounts for 2% of childhood cancers.

Bone cancer: Bone cancer is more prominent in teens, but it still accounts for 3% of childhood cancers. There are two types, osteosarcoma and Ewing sarcoma, that show up via swelling around the bones. Ewing sarcoma is a less common type of cancer that is more prominent in older children and younger teens. Osteosarcoma, meanwhile, is more common in teens and in areas where bones grow quickly.

There isn't a one-size-fits-all guide to know if your child has cancer. In general, Dr. Confer says to keep an eye on any changes in your childs behavior, such as walking, eating, playing or sleeping. If theyre older, listen to any complaints they may have. Some cancers may produce a lump or swelling and pain in certain areas. Other symptoms include a loss of energy, weight loss, sudden eye or vision changes, frequent headaches with vomiting or a persistent fever that signifies the body is fighting an infection.

For example, leukemia, the most common type of childhood cancer, affects most children between the ages of 2 and 4. Typical symptoms include fever, bleeding, deep pain in the bones, small red spots on the skin called petechiae, bruises and enlarged lymph nodes.

Notify your childs pediatrician if any of these concerns arise. Aside from that, you should schedule your child for routine checkups and wellness visits.

Routine checkups and wellness visits help monitor normal growth and development. A good pediatrician-patient relationship helps the physician better identify subtle signs of cancer and gives parents a trusted sounding board for the concerns parents or children may have, Dr. Confer says.

Many childhood cancers have become increasingly treatable, leading to longer survival rates. Dr. Confer says acute lymphoblastic leukemia, lymphoma or kidney tumors known as Wilms tumor all have more than a 90% five-year survival rate (the percentage of patients who are alive five years after receiving treatment or a diagnosis).

In fact, the overall five-year survival rate for childhood cancers has improved from 58% in the mid-1970s to 84%, according to the American Cancer Society. But, certain types of aggressive cancer still exist. Diffuse intrinsic pontine glioma (DIPG), a rare brain tumor, is often cited as the childhood cancer with the poorest survival rate (less than 1% for five years).

No matter the diagnosis, continual hope and quality, proven therapies are the most important factors for children and families facing childhood cancers, Dr. Confer says.

Here are some of the most common forms of therapies to treat childhood cancer.

Surgery can help many patients, whether you need an entire tumor removed or a procedure to ease pain caused by a tumor. Your childs surgeon can also debulk a tumor, meaning they remove part of it and treat the rest with another method. Surgery has the highest success rate when its contained to one area, before the cancer has an opportunity to metastasize (spread to other parts of the body).

High doses of radiation help reduce cancer by either killing the cells or damaging their DNA to slow growth. Over time, these cells die and your body removes them. You can either receive internal or external radiation. External radiation comes from a beam that treats a specific body part, whereas internal radiation is in solid or liquid form. More specifically, brachytherapy is the medical term for solid internal radiation. Your doctor will place capsules, seeds or ribbons near the tumor. Systemic therapy is the medical term for liquid internal radiation. With this method, the radiation travels through your blood via a pill, injection or IV to kill cancer cells.

Chemotherapy comes in many methods of application, such as IV, oral, injection, topical or through a catheter, port or pump. Chemotherapy also kills healthy cells, which is one of the downsides. This is why many chemotherapy patients lose their hair and experience other side effects. Depending on the type and progression of the cancer, chemotherapy can help shrink a tumor to increase the success rate of surgery or radiation. Chemotherapy can also fight against any lingering cancer cells following surgery or radiation. Its also used to treat cancer that returns or metastasizes.

The immune system is your bodys way of defending itself against harmful germs, bacteria and viruses. When it comes to cancer, the immune system can have trouble recognizing and fighting off harmful cells because cancer starts in healthy cells. Immunotherapy helps your body pinpoint cancer kills to better defend against them. There are many types of immunotherapy treatments to boost your immune system. One type, chimeric antigen receptor (CAR) T-cell therapy, mixes your own T-cells with a virus that teaches the T-cells how to kill cancer cells.

Targeted therapy is a form of chemotherapy. But, as the name suggests, these drugs zero in on a specific area of the cancer cells. Depending on the drug, targeted therapy can change the protein levels in cancer cells or block chemical signals that help cancer cells grow. Other targeted therapy drugs can limit blood vessel production to cut off the cancer cells or distribute toxins to specifically kill the cancer while sparing healthy cells.

Stem cells, which originate in the bone marrow, make red blood cells, white blood cells and platelets. Leukemia and lymphoma start in the blood cells, causing damage to the cells your body needs to function. A stem cell transplant involves destroying cancer cells via chemotherapy and/or radiation before replacing them with new, healthy cells. This allows doctors to use stronger doses of chemotherapy or radiation knowing new cells, via a transplant, will replace old, damaged cells. Stem cell transplants can come from your own cells or the cells of another person. Donated cells are often more effective since they can help kill off cancer cells.

While you cant do anything to prevent your child from developing cancer, you can be proactive by scheduling regular checkups and looking out for warning signs and symptoms. Contact an INTEGRIS pediatrician if you have any concerns, and they can refer you to an oncologist at the INTEGRIS Cancer Institute.

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Different Types of Childhood Cancer | INTEGRIS - Integris

Dive Brief:

Gene therapies have graduated from the laboratory bench to doctor's offices. The first wave of agents, Roche's Luxturna and Novartis' Zolgensma, are already altering the course of disease in two conditions, respectively an inherited form of blindness and the degenerative, often fatal disease spinal muscular atrophy.

In both cases those treatments were developed by companies that had relatively smaller pipelines, Spark Therapeutics for Luxturna and AveXis for Zolgensma.

Enter Taysha, which is headed by a former AveXis business development vice president, R.A. Session II. The company has highly ambitious hopes to launch a new product every two to three years, with the goal of building a durable business around adapting its technology across many diseases driven by defects in single genes.

The company also plans to build a commercial-scale manufacturing plant from the start, aiming to avoid some of the setbacks that can occur when production moves from facilities built to supply clinical trials.

The linchpin of the company's business is an agreement with UT-Southwestern, under which Taysha funds research and can obtain exclusive rights to experimental therapies for central nervous system disorders, through the end of 2021. Neurodegenerative disorders have proven challenging for some gene therapies because of the difficulties in delivering the viral vectors that carry gene replacements to brain tissue.

In spinning out Taysha, UT-Southwestern took an ownership stake in Taysha, amounting to 2.2 million shares, which is now worth more than $40 million. These ownership stakes have become more common with gene therapies in particular, as big pharma companies have been reluctant to license intellectual property straight out of university laboratories.

Taysha's lead project is called TSHA-101, which seeks to treat a condition called GM2 gangliosidosis, a disorder in which lipid accumulation destroys nerves in the brain and spinal cord. The first clinical trial is scheduled to begin in Canada by the end of 2020.

The company's shares rose following their first trades on the NASDAQ exchange, gaining 20% to close the day at $24.06.

Editor's note: This story was updated to reflect the share price at the close of trading.

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Gene therapy company Taysha completes sprint from first funding to IPO - BioPharma Dive

New Jersey students browsing a course catalog will find many programs of study thatwerentthere when their parents went to college.

Some programs seem to be exactly appropriate for the time we are in.

Theres cyberpsychology at New Jersey Institute of Technology (NJIT), environmental studies at Rutgers, financial technology at Seton Hall and cannabis studies at Stockton University.

As perhaps the largest mass demonstrations in American history have drawn attention to prejudice against minorities, including police brutality and inequity in the impact and treatment of the COVID-19 pandemic, The College of New Jersey (TCNJ) offers a minor in social justice.

Its so interesting that it fits the moment, but it is not a new idea for us, said Dr. PiperKendrixWilliams, a professor in the English and African American Studies departments at TCNJ.

The idea for the minor is three or four years old, and we worked on it for about two years, she said.

New Jersey tends to have segregated areas white suburbs and minorities in the cities, she said. Even in college, students experience can be really segregated.

Our students need an education in social justice, she said. Every student in the college needs some understanding of race, gender and inclusion. I can see a lot of students wanting this.

Williams said the program is a natural outgrowth of TCNJs Bonner Scholars program in which participants are required to do 300 hours of community service in the Greater Trenton area.

The minor requires five courses: Introduction to Social Justice, Introduction to Womens and Gender Studies, Introduction to the Sociology of Race and two junior-level electives.

Activism cant come until youre educated, until you know what youre fighting for, Williams said. Higher education has to step up to what is relevant in the world.

Dr. JulieAnciswill be the director of NJITs new program in cyberpsychology, offering a Bachelor of Science degree. It is the first such major in New Jersey and NJITs first foray into the behavioral sciences.

Im very excited about this program because its truly innovative, saidAncis, who has been a professor of psychology and associate vice president of diversity at Georgia Tech.

It is designed for students with an interest in psychology and human behavior, she said, noting that projections call for at least an 11% job growth in the field in the next 10 years.

The program will use existing research and literature to prepare students to solve real-world problems and apply their skills to a variety of domains,Ancissaid.

Potential career fields will include cyber security, games We want games to be a force for good, she said telepsychology, virtual reality, app development, digital health and any positions related to online engagement.

We will be exploring all aspects of technologically interconnected human behavior through the lens of psychology,Ancissaid. We will look at ways to enrich online experiences and decrease anxiety, explore the psychology of cyber threats both for perpetrators and victims factors that influence privacy precautions such as perception of risks, cyberbullying, ethics and many other areas.

The ways in which we learn, socialize and communicate have been transformed, she added. Were in a totally new worldapproximately 60% of the worlds population is using the internet.

Anciswill teach Foundations of Cyberpsychology, which has a waiting list for registration.

I expect this really to grow, she said.

NJIT also is offering a new undergraduate major in forensic science, and certificates in big data, data mining, and cell and gene therapy.

Cannabis has been on college campuses for decades, but with medical marijuana now legal in 33 states and recreational marijuana legal in 11, a new industry has spawned.

The university was interested in having a scientist involved because there might be perceptions that illegal activity was involved, said Dr. EkaterinaSedia, a plant biologist and coordinator of the cannabis studies minor at Stockton University. The program is very much based in science.

OK, we have a new industry, its coming, its here and theyre going to be hiring. We want to get those jobs.

About 50 students are enrolled in the program, which started a year ago.

I anticipated the demand,Sediasaid. I was a little surprised how quickly it became known. We have students from a variety of majors, including business, environment and sustainability, and communications.

The program also looks at hemp and CBD products, and includes such aspects as banking, cybersecurityand regulatory compliance. It studies the non-psychoactive aspects of cannabis, such as use in cosmetics, too.

Its not necessary that youd be growing it yourself, but you may be involved with a business that grows it,Sediasaid. I have a student who says she wants to be the Johnson & Johnson of cannabis.

Elven Riley of Seton Halls Stillman School of Business saidthe financial technology major FinTech for short is aimed at preparing students for jobs where finance and the digital world intersect.

In the finance department we see that banking and brokerages and, to some extent, insurance are reducing their traditional workforces and moving to more technology savvy employees, he said. There are just thousands of jobs out there. New York and London are hotbeds.

The major is designed for business students to become literate in the language of technology.

We think it will put our students at the head of the queue to get those jobs, said Riley, who had a 30-year career in investment banking. Were getting attention from the industry that they want these students.

Seton Hall now has students who pursue dual majors in finance and information technology. Forty to 50 students are expected to switch to the FinTech major.

This is a mash-up of the two concentrations, Riley said. Looking around higher education, thereare not a lot of these designed programs.Theresnot a lot of faculty in higher education that has experience in this space.

Theres big interest, lots of jobs, very exciting, but also very challenging, he said.

The possible careers are in big data, algorithmictradingand edge computing.

Theres no part of the financial industry that doesnt involve technology, Riley said. You need people who are trained and literate inboth of these.

We do think incoming students will seek this out. We think this will attract high-quality students to our program. We would be remiss not to go in this direction.

Rutgers University-New Brunswicks School of Arts and Sciences has launched a new major in environmental studies that brings together 17 science and humanities programs.

This appeals to up-and-coming generations who have grown up with the environment being at the forefront of their world, said Dr. Paul OKeefe, whose specialty is geography.

Different in focus from environmentalscience,the environmental studies major will prepare liberal arts students to be professionals on environmental issues, OKeefe said.

We currently have 26 students enrolled in the environmental studies minor and expect several to transition to the major along with new entrants to the program, he said.

Students in all majors must take Environmental Techniques courses, which give them skills applicable to many workplaces. There also is an optional internship component for students.

Rutgers also offers a minor in agroecology in the School of Environmental and Biological Sciences in New Brunswick. It appeals to a small number of students from a wide variety of majors.

I have had landscape architects and nutrition majors who are interested in how we grow food and food systems, said Dr. Mark Robson, Board of Governors Distinguished Service Professor in the Department of Plant Biology. I have had kids from the business school who want to know about the costs of growing food. I have had kids in our traditional ag(riculture)and food systems major that used to be ag(riculture) science, and then I have students who are just interested in ecology in general and this caught their eye.

So, given its general nature and the fact that we all eat, it seems to interest a lot of students, he said.

Students have gone on to work for state environmental agencies and the U.S. Department of Agriculture, and some have gone into farming as well, Robson said.

Other new programs at Rutgers include majors in cinema studies, Japanese and Korean, and minors in African languages, archaeology, Holocaust studies, and medical ethics and health policy.

Kean University has started a new major in exercise science in its School of Health and Human Performance. It can lead to careers in physical and occupational therapy, chiropractic, cardiac rehabilitation, and sports medicine, and lead to work in the fitness industry.

Some of our students become entrepreneurs and start their own fitness businesses, said Dr. Adam Eckart, program coordinator, who has worked as a full-time trainer and whose bachelors and masters degrees are from Kean.

It can be a feeder for graduate health programs, including medical school, he said.

Eckart said there are 215 exercise science majors, and the program is expected to grow by 40 to 50 students a year.

Health care organizations will be looking in the future to refer patients to exercise professionals, he said. Students need an insight into where the field is headed.

He said most of health care spending results from chronic conditions that can be avoided with intervention through diet and exercise, pointing to the motto of the American College of Sports Medicine: Exercise is medicine.

Seton Hall also has launched a dual major in religion and law, which will allow students to graduate in six years with a bachelors degree in religion and a juris doctor in law.

Were very excited about it, said Dr. Brian Sheppard of Seton Hall Law School in Newark, who said the religion major requires a rigorous study of texts, which overlaps with law school rigor. He said it appeals to students who are interested in working in the nonprofit sector.

We like the idea of building this bridge, Sheppard said. We get some incredible students from Seton Hall undergraduate. This just strengthens that.

It is one ofseveralthree-plus-three programs offered by Seton Hall, leading to both undergraduate and graduate degrees in six years.

You use your first year of studies in law school as your fourth year of college, Sheppard said. It really offers a premium to students who want both degrees. It allows you graduate with less debt. Debt can be a real obstacle to careers in social justice.

And Seton Halls School of Diplomacy and International Relations is offering non-majors a certificate in global affairs.

Acting Dean Courtney Smith said many diplomacy majors minor in languages and other disciplines in Seton Halls other schools.

This is an opportunity for us to offer a reciprocal service, he said, to better serve students already here.

The certificate will require four classes that will give students a better grasp of global affairs, Smith said, since many of the issues facing society in the future require an international approach, including climate change.

He said students can take a broad or narrow approach in choosing their classes. It requires only 12 credits, rather than the 18 required for a minor.

Kean also has started a new dual admission program Pathway to Kean that offers students whodontinitially meet university admissions standards a path toward earning a bachelors degree.

Qualified students are given conditional acceptance to the universityas long asthey complete their associate degree at one of Keans partner county colleges. The university will provide counseling and support services while the students are attending county colleges.

The program begins this fall in partnership with Essex County College, Middlesex County College, the County College of Morris, Ocean County College, Union County College and Warren County Community College. It is expected to include more New Jersey county colleges over the next two years.

Students in the program will have their applications sent to their respective county colleges. Kean will work with each county college admissions office to ease their enrollment into the program.

Kean will provide support services on the county college campuses and remotely for students in the program.

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Focused on the future: Innovative college programs in N.J. pave way for the real world - Jersey's Best

A recently approved agent, burosumab (Crysvita), was better than placebo across a range of efficacy outcomes for 14 predefined subgroups of adults with X-linked hypophosphatemia (XLH), new research shows.

The authors analyzed data from the initial 24-week randomized blinded phase of the pivotal phase 3 trial that led to regulatory approval of this drug in the United States in 2018 for XLH, a rare form of rickets characterized by low serum phosphorus levels, skeletal defects, pain, and stiffness.

As in the main analysis, in the subgroups, among patients who received burosumab, serum phosphorus levels were improved, and outcomes were better on the following measures: Western Ontario and McMaster Universities Arthritis Index (WOMAC) stiffness scale, the WOMAC physical function measure, and the Brief Pain Inventory (BPI), which were the main efficacy outcomes. Improvements were seen for many other outcomes as well.

Maria-Luisa Brandi, MD, Careggi University Hospital, Florence, Italy, presented the new subanalysis during the virtual American Society of Bone and Mineral Research (ASBMR) 2020 annual meeting.

The subgroup results were consistent with the overall trial findings, "showing a favorable direction of effect of burosumab relative to placebo" except for results in patients recruited in Asia and non-White patients; those results were considered inconclusive because there were too few participants in those categories, she told Medscape Medical News,.

Lorenz Hofbauer, MD, scientific chair of the ASBMR meeting, said that the take-away message is that the drug "works to reduce pain and disability" in adults with XLH with more severe/less severe symptoms, and "it provides new hope for many patients suffering from this disease," he told Medscape Medical News.

Burosemab also appears superior to what has previously been considered standard therapy for XLH, phosphate/calcitriol, the experts say.

"The disease prevalence is 1 to 9 in a million," Brandi said. "Undiagnosed adults are treated by the doctor that makes the diagnosis, usually a nephrologist or a rheumatologist or a bone doctor; this depends on the prevalent complications in a given patient. The endocrinologist who treats this patient is the one expert in bone disorders."

Hofbauer noted, however, that "[r]are is relative. If you run a bone clinic, you will see four to five patients with XLH; if you are a regional center, 20 to 30 patients. People with rare disease travel more than 1000 miles to see experts."

The US Food and Drug Administration approved burosumab for use in children and adults with XLH 2 years ago. The European Medicines Agency (EMA) approved it for use in children.

The drug is expected to be approved by the EMA for adults with XLH some time this year, said Hofbauer, who is from Dresden Technical University, Dresden, Germany.

Burosumab is a "game changer" with respect to previous treatments, he stressed.

This study is one of the top five clinical abstracts of the ASBMR meeting, which are selected on the basis of "scientific content/novelty, making a difference in clinical practice," Hofbauer explained. He noted that "new drugs that work are always in the top ranks."

Craig Munns, PhD, who was senior author of a recent review about burosumab, agrees.

"Burosumab is transformative, as it is a paradigm shift in the way we manage XLH," he told Medscape Medical News.

"Standard therapy for children is with oral phosphate and calcitriol, and many adults do not receive any therapy," said Munns, from the University of Sydney, Sydney, Australia.

"Phosphate and calcitriol need to be taken multiple times per day, is an incomplete therapy, and has many complications. Burosumab offers a 2-weekly (children) or 4-weekly (adult) dosing regime with superior outcomes compared to no treatment or phosphate/calcitriol," he emphasized.

"Burosumab is an anti-FGF-23 [antifibroblast growth factor-23] antibody for a rare genetic disease, XLH, in which the gene for PHEX is defective," Hofbauer explained.

"PHEX is an enzyme that clears FGF-23; if it does not work, then FGF-23 accumulates in the body and causes phosphate wasting with wide consequences for bone, muscle, and joints. Burosumab is a smart approach, since it blocks these excessive FGF-23 effects."

Children with XLH have rickets, deformities in the lower skeleton, and short stature, Brandi noted, whereas adults have fractures, pseudofractures, enthesopathy (calcification of joint capsule, tendon insertions, and ligaments), pain, stiffness, and impaired physical function.

However, "treatment with oral phosphate and vitamin D is associated with nephrocalcinosis and hyperparathyroidism," she said.

In the phase 3 trial, 134 adults (aged 18 to 65 years) with XLH were randomly assigned in a double-blind manner to receive either burosumab or placebo for 24 weeks, followed by 24 weeks of open-label burosumab. The patients' serum phosphorus levels were <2.5 mg/dL, and they were experiencing measurable bone/joint pain.

Baseline characteristics were similar for the patients who received placebo (66) and those who received burosumab (68). The mean age of the patients was 40 years; 65% were women; and 81% were White.

The current exploratory analysis examined efficacy outcomes in patients grouped according to the following factors and characteristics: sex; age (41 years or >41 years); race (non-White, White); region (Asia, North America/Europe); baseline WOMAC pain score; WOMAC total pain; WOMAC stiffness; WOMAC physical function; BPI worst pain; BPI average pain; opioid use; pain medication use; active fractures and pseudofractures; and 6-minute walking test distance.

The efficacy outcomes were as follows: serum phosphorus level (primary outcome), BPI worst pain, WOMAC stiffness, and WOMAC physical function (key secondary outcomes); and WOMAC pain, WOMAC total score, BPI average pain, BPI pain interference, BPI worst fatigue, BPI global score, patient global impression (PGI), and 6-minute walking distance.

In the overall cohort, at 24 weeks, in comparison with patients who received placebo, patients who received burosumab had favorable responses with respect to serum phosphorus level, WOMAC stiffness (P =. 012),WOMAC physical function (P = .048), and BPI worst pain (P = .092, not significant), as well as significant improvements in WOMAC total score and the 6-minute walk test. There were nonsignificant improvements in WOMAC pain and BPI average pain.

In the subgroup analysis, burosumab was superior to placebo for the primary outcome (serum phosphorus) in all subgroups. It was also superior to placebo for the key secondary outcomes (worst pain, stiffness, and physical function) across all subgroups except for patients from Asia (18 patients) and non-White patients (26).

The study was funded by Kyowa Kirin in partnership with Ultragenyx. Brandi receives consultancy and speaker fees as well as research grants from Kyowa Kirin and other pharmaceutical companies. Munns has received research funding from Kyowa Kirin.

The American Society of Bone and Mineral Research (ASBMR) 2020 Annual Meeting Virtual Event: Presented September 12, 2020.

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Burosumab Is a 'Game Changer,' Effective in All Subgroups of XLH - Medscape

Spider-Man might not be a superhero typically known for his sidekicks, but he's definitely had some over the years. And these deserve a comeback.

It took Spider-Man a long time to finally relent and join a superhero team. Even then, it took the combined effort of Captain America and Iron Man to convince him. Before this, he had only been in the Avengers for a paltry four issues and had flirtations with the Defenders and the Fantastic Four. Even his Avengers membership had been as a reserve member.

RELATED: The Top Ten Spiderman And Deadpool Team Ups, Ranked

That does not mean the wall-crawler was exclusively a solo act. In fact, the long-running Marvel Team-Up featured Spider-Man in almost every issue of its thirteen-year run. Spider-Man was not averse to working with others, even if he did annoy most of his partners with his awkward gags. In fact, Spider-Man has nurtured and partnered with a lot of othercharacters in his time. From teenage runaways to reformed criminals, the web-slinger has had his fair share of sidekicks.

Robert Farrell has turned up on both sides of the law in his brief appearances. As well as being an adversary and sidekick of Spider-Man, he has also assisted the Avengers and worked undercover for S.H.I.E.L.D. As a child, realizing that the odds were stacked against him as a poor, black youth from the slums, he developed a rocket-powered skateboard. He used this to commit minor robberies to pay rent and bills for his mother.

After a stint on Ryker's Island, he reformed and began to assist Spider-Man. His cool, eighties look is very current. With his background that touches on themes inherent in the BLM movement, the world needs a new Rocket Racer story.

In her short stint in comics, Jackpot has developed quite a backstory. She began as an employee at Oscorp and was exposed to experimental gene therapy. This put her in a coma for four months, after which she awoke, gave birth, and realized she had developed super strength.

She was only a superhero briefly beforequitting to raise her daughter. In a very modern twist, she then sold her identity and brand. The new Jackpot teamed up with Spider-Man until her death when the outfit returned to its original occupant. The single mother superhero narrative has been approached in comics recently, but fans would be impressed to see a story involving a working superhero motherwho doesn't have a big team behind her.

Cloak and Dagger are two of the most recognizable of Spider-Man's sidekicks. They are so iconic, they even got their own television series. Since their debut in 1982, Tyrone and Tandy have been members of the Runaways, X-Men, and also fought alongside Captain America in the Civil War. They have even fought against heavyweights such as Doctor Doom and the Beyonder.

Fans adore Cloak and Dagger most when they are fighting street-level villains alongside Spider-Man. They were last seen guarding Hong Kong against the influence of Mr. Negative. Fans eagerly await their return home to New York.

Puma is a very complex ally to Spider-Man, and deserves to be more of a partner to Spidey than a sidekick. He is of Native American descent, and fiercely loyal to his heritage. Though a man of morals and good standing, he has also clashed with the web-slinger on some occasions.

RELATED: 5 Spiderman Villain Who Are Actually Sympathetic (& 5 Who Are Pure Evil)

His powers are a mix of martial arts training, technology, and Native American mysticism. As a successful businessman, it is surprising that his company, Fireheart Enterprises,never made deals with Parker industries. In a world polarized between the values of tradition and technology, fans would delight in a renewed version of the Puma.

Silver Sable has everything. She is a businesswoman, bounty hunter, a princess, and the owner of an international company. Despite lacking any actual super powers, she has access to a wealth of technology and is a fierce martial artist.

While not forgotten, Silver Sable is longoverdue a permanent comeback in the world of the wall-crawler. Her antagonistic nature and single-mindedness could either be a blessing or curse for Spider-Man. The idea of her as a private security consultant, as explored in the recent Spider-Man videogame, could see her tip the wall crawlers world upside down.

Cardiac's last appearance was during the Superior Spider-Man era when Doctor Octopus was inhabiting the body of Peter Parker. Despite friction, they decided they would still work together on medical research projects in the future. In fact, Cardiac even made Otto Octavius feel guilty about his past schemes, which is just the kind of character Cardiac is.

Elias Wirtham is a physician and owner of a biomedical research company who became Cardiac to right the wrongs ofgreedy pharmaceutical companies after the death of his brother. He is a medical Robin Hood, robbing drugs and equipment for those in need. While this can get him into trouble, his intent is always noble and he has partnered Spider-Man on many occasions.

Though he partnered with Spider-Man during the '90s, Nightwatch was anything but a good guy. In fact, in a bid to erase his former existence from history, he condemned a whole town to genocide. His motivation to be a hero was founded on the premise that they had the best retirement plans.

Nightwatch was last seen in the pages of She-Hulk, who brought him to justice using the law. Now atoning for his crimes in jail, a return from Nightwatch would finally allow him to make amends and reconcile his past sins. Who would be better equipped to help him do that than Spider-Man?

Of all the sidekicks that Spider-Man has had in his tenure, the most memorable come from a kooky TV series. Spider-Man and his Amazing Friends might not make much sense in terms of Spidey's comic book canon, but in the early '80s, this team-up cartoon was huge.

RELATED: Spider-Man: Ten New Characters Introduced During The Clone Saga

The team formed in episode one, movinginto Aunt May's apartment with a small dog and deciding to fight crime. For a modern update, the backstory may need reworking. They did have a brief reunion inanIceman mini-series.There's no doubt that fans would relish the chance to read a retro Amazing Friends comic in the vein of X-Men '92.

The White Tiger belongs to a legacy and has been a moniker adopted by five different heroes in the history of Marvel. The first White Tiger, Hector Ayala, was the hero most affiliated with Spider-Man. Hector carried a jade amulet that would let him transform into a White Tiger. He teamed up with Spider-Man many times in the'70s until he was accused of a crime he did not commit.

Despite the best efforts of his lawyer, Matt Murdock (Daredevil), he was found guilty and later assassinated. Ava Ayala, his younger sister, was the most recent to take uphis mantle of White Tiger. Whether she still has the White Tiger amulet or another has taken up the moniker remains to be seen.

Solo is a work for hire mercenary, similar in his outlook and abilities to The Punisher. He will use lethal means to get what he wants. Though this often brings him into conflict with the web-slinger, it has also meant that they have worked side by side on many occasions.

Solo's adventures have since taken him to some wild and wonderful situations. He has worked for Deadpoolas one of the "mercs for money"and even with S.H.I.E.L.D. in the past, though he is always best when trying to tow the line alongside Spider-Man.

NEXT: Spider-Man: The 10 Worst Things Black Cat Has Ever Done

Next Iron Fist Vs. Elektra: Who Would Win?

Carl Jackson is a typical English gentleman currently residing in Eastern Europe. He lives with his two cats, wife, and Neo Geo arcade machine. He is an avid retro gamer, collector of the eighties toy line M.A.S.K. and enjoys getting new ink on his Marvel vs Capcom sleeve.

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10 Forgotten Spiderman Sidekicks They Need To Bring Back | CBR - CBR - Comic Book Resources

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that it presented the latest results from three Phase III studies from the Tecentriq (atezolizumab) clinical development program in triple-negative breast cancer (TNBC) at the European Society for Medical Oncology (ESMO) Virtual Congress 2020.

While we have made great progress in the treatment of many forms of breast cancer, TNBC remains an aggressive and difficult-to-treat disease, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. We are proud of our work to address challenges and advance scientific understanding of cancer immunotherapy in the context of distinct chemotherapy regimens and in various TNBC treatment settings. Although the IMpassion131 study did not reach its endpoint, we are pleased to bring new treatment options for some TNBC patients, and remain committed to improving the lives of all women with early and advanced stages of this disease.

Results from the Phase III IMpassion031 study, evaluating Tecentriq in combination with chemotherapy (Abraxane [albumin-bound paclitaxel; nab-paclitaxel]; followed by doxorubicin and cyclophosphamide) in comparison to placebo plus chemotherapy (including nab-paclitaxel) demonstrated a statistically significant and clinically meaningful improvement in pathological complete response (pCR) for the treatment of people with early TNBC, regardless of PD-L1 expression. pCR was observed in 57.6% (95% CI: 49.7-65.2) of patients treated with Tecentriq in combination with chemotherapy, an increase of 16.5% from 41.1% (95% CI: 33.6-48.9) in patients treated with placebo plus chemotherapy (one-sided p=0.0044, significance boundary = 0.0184) in the intention-to-treat (ITT) population. The safety profile was consistent with the established profile of the individual medicines and no new safety concerns were identified.

The IMpassion031 study is the second positive Phase III study from Genentech demonstrating the benefit of Tecentriq in TNBC, and the first Tecentriq study to demonstrate benefit in early TNBC. Tecentriq in combination with nab-paclitaxel is currently approved in more than 70 countries worldwide, including the United States and across Europe, for the treatment of adults with unresectable locally advanced or metastatic TNBC in people whose tumors express PD-L1 (IC1%).

The final overall survival (OS) analysis of the Phase III IMpassion130 study, evaluating Tecentriq in combination with nab-paclitaxel compared with placebo plus nab-paclitaxel as a first-line treatment for patients with metastatic TNBC, was consistent with the first and second interim analyses. There was no significant difference in OS between the treatment groups in the ITT population. Clinically meaningful improvements in OS were seen with Tecentriq plus nab-paclitaxel in PD-L1-positive patients. The magnitude of OS improvements with Tecentriq in PD-L1-positive patients remained clinically meaningful, with an increase of 7.5 months in median OS with Tecentriq plus nab-paclitaxel compared with placebo plus nab-paclitaxel (hazard ratio [HR]=0.67 [95% CI: 0.530.86]). However, this result could not be formally tested due to the prespecified statistical testing hierarchy. The cumulative safety of the Tecentriq plus nab-paclitaxel combination remains consistent with the previously reported safety data for this study and the known risks of individual study medicines. No new safety concerns were identified with longer follow-up.

Finally, results from the Phase III IMpassion131 study, evaluating Tecentriq in combination with paclitaxel compared with placebo plus paclitaxel as a first-line treatment for patients with metastatic TNBC, did not show significant improvement for progression-free survival (PFS) in the PD-L1-positive population (HR=0.82 [95% CI: 0.60-1.12]). The OS data showed a negative trend; however, the study was not powered for the secondary endpoint of OS, and OS data were immature at the time of analysis (initial HR=1.55 [95% CI: 0.86-2.80] in the PD-L1-positive population, based on 21% of patients with an event; updated HR=1.12 [95% CI: 0.76-1.65], updated analysis based on 41% of patients with an event). The safety profile of Tecentriq plus paclitaxel was consistent with the established safety profile of the individual study medicines and no new safety concerns were identified.

Genentech has an extensive development program for Tecentriq, including multiple ongoing and planned Phase III studies across several types of lung, genitourinary, skin, breast, gastrointestinal, gynecological, and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

About the IMpassion031 study

The IMpassion031 study is a Phase III, multi-center, randomized, double-blind study evaluating the efficacy and safety of Tecentriq (atezolizumab) in combination with chemotherapy (Abraxane [albumin-bound paclitaxel; nab-paclitaxel]; followed by doxorubicin and cyclophosphamide) in comparison to placebo plus chemotherapy, in people with previously untreated, early TNBC. The primary endpoint is pCR using the American Joint Committee on Cancer (AJCC) staging system in the intention-to-treat (ITT) population and in the PD-L1-positive population. Secondary endpoints include overall survival (OS), event-free survival, disease-free survival and quality of life measures.

About the IMpassion130 study

The IMpassion130 study is a Phase III, multicenter, randomized, double-blind study evaluating the efficacy, safety and pharmacokinetics of Tecentriq plus nab-paclitaxel compared with placebo plus nab-paclitaxel in people with unresectable locally advanced or metastatic TNBC who have not received prior systemic therapy for metastatic breast cancer. The study enrolled 902 people who were randomized equally (1:1). The co-primary endpoints are PFS per investigator assessment (RECIST 1.1), and OS in the ITT population and PD-L1-positive population. Secondary endpoints include objective response rate and duration of response.

About the IMpassion131 study

The IMpassion131 study is a Phase III, multi-center, randomized, double-blind study evaluating the efficacy and safety of Tecentriq in combination with paclitaxel, in comparison to placebo plus paclitaxel, in people with previously untreated, inoperable, locally advanced or metastatic TNBC. The study enrolled 651 people who were randomized in a 2:1 ratio to receive Tecentriq or placebo plus paclitaxel. The primary endpoint is PFS per investigator assessment (RECIST 1.1) in the PD-L1-positive population, followed by intention-to-treat (ITT) population. Secondary endpoints include OS, objective response rate, and duration of response in the PD-L1-positive and ITT populations.

About triple-negative breast cancer

Breast cancer is the most common cancer among women worldwide. According to the American Cancer Society, close to 280,000 people in the United States will be diagnosed with invasive breast cancer, and more than 42,000 will die from the disease in 2020. Breast cancer is not one, but many diseases based on the biology of each tumor. In triple-negative breast cancer, tumor cells lack hormone receptors and do not have excess HER2 protein. Approximately 15 percent of breast cancers are triple-negative based on the results of diagnostic tests. It is an aggressive form of the disease with few treatment options.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

Tecentriq U.S. Indications (pronounced t-SEN-trik)

Tecentriq is a prescription medicine used to treat adults with:

A type of bladder and urinary tract cancer called urothelial carcinoma.

Tecentriq may be used in patients with urothelial carcinoma if their bladder cancer has spread or cannot be removed by surgery, and if they have any one of the following conditions:

The approval of Tecentriq in these patients is based on a study that measured the amount of time until patients disease worsened. Continued approval for this use may depend on the results of an ongoing study to confirm benefit.

A type of lung cancer called non-small cell lung cancer (NSCLC).

Tecentriq may be used alone as the first treatment in patients with lung cancer if:

Tecentriq may be used with the medicines bevacizumab, paclitaxel, and carboplatin as the first treatment in patients with lung cancer if:

Tecentriq may be used with the medicines paclitaxel protein-bound and carboplatin as the first treatment in patients with lung cancer if:

Tecentriq may be used alone in patients with lung cancer if:

A type of breast cancer called triple-negative breast cancer (TNBC).

Tecentriq may be used with the medicine paclitaxel protein-bound in patients with TNBC when their breast cancer:

The approval of Tecentriq in these patients is based on a study that measured the amount of time until patients disease worsened. Continued approval for this use may depend on the results of an ongoing study to confirm benefit.

A type of lung cancer called small cell lung cancer (SCLC).

A type of liver cancer called hepatocellular carcinoma (HCC).

Tecentriq may be used with the medicine bevacizumab when a patients liver cancer:

A type of skin cancer called melanoma.

Tecentriq may be used with the medicines cobimetinib and vemurafenib when a patients melanoma:

It is not known if Tecentriq is safe and effective in children.

Important Safety Information

The most important information about Tecentriq is:

Tecentriq can cause the immune system to attack normal organs and tissues and can affect the way they work. These problems can sometimes become serious or life-threatening and can lead to death.

Patients should call or see their healthcare provider right away if they get any symptoms of the following problems or these symptoms get worse.

Tecentriq can cause serious side effects, including:

Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider may treat patients with corticosteroid or hormone replacement medicines. A healthcare provider may delay or completely stop treatment with Tecentriq if patients have severe side effects.

Before receiving Tecentriq, patients should tell their healthcare provider about all of their medical conditions, including if they:

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Tecentriq when used alone include:

The most common side effects of Tecentriq when used in lung cancer with other anti-cancer medicines include:

The most common side effects of Tecentriq when used in TNBC with paclitaxel protein-bound include:

The most common side effects of Tecentriq when used in hepatocellular carcinoma with bevacizumab include:

The most common side effects of Tecentriq when used in melanoma with cobimetinib and vemurafenib include:

Tecentriq may cause fertility problems in females, which may affect their ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of Tecentriq. Patients should ask their healthcare provider or pharmacist for more information. Patients should call their doctor for medical advice about side effects of Tecentriq.

Report side effects to the FDA at (800) FDA-1088 or http://www.fda.gov/medwatch. Report side effects to Genentech at (888) 835-2555.

Please visit http://www.Tecentriq.com for the full Tecentriq Prescribing Information for additional Important Safety Information.

About Genentech in cancer immunotherapy

Genentech has been developing medicines to redefine treatment in oncology for more than 35 years, and today, realizing the full potential of cancer immunotherapy is a major area of focus. With more than 20 immunotherapy molecules in development, Genentech is investigating the potential benefits of immunotherapy alone, and in combination with various chemotherapies, targeted therapies and other immunotherapies with the goal of providing each person with a treatment tailored to harness their own unique immune system.

In addition to Genentechs approved PD-L1 checkpoint inhibitor, the companys broad cancer immunotherapy pipeline includes other checkpoint inhibitors, individualized neoantigen therapies and T cell bispecific antibodies. For more information visit http://www.gene.com/cancer-immunotherapy.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

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Genentech Presents New Data From Multiple Phase III Studies of Tecentriq in Triple-Negative Breast Cancer at ESMO Virtual Congress 2020 - Business...

CHICAGO, Sept. 16, 2020 /PRNewswire/ -- BCS Financial (BCS) announces today it has partnered with 2nd.MD to provide top-tier medical navigation, expert consultation, and personalized second opinions to four million policyholders. Under the agreement, members covered under BCS Stop Loss and Excess of Loss policies can work with highly skilled, experienced nurses to help them navigate diagnoses, virtually connect with elite doctors who specialize in their specific condition, and receive assistance finding a local doctor or specialist in their area at no cost to them.

As the number one reinsurer of Blue Cross Blue Shield plans, BCS offers a wide range of products and services to help insurance carriers and their members navigate healthcare costs, particularly those exceeding $500,000 annually. The partnership is one of several features included in BCS Financial's Risk Navigator solutions, a highly predictive and actionable approach to managing claims risk.

"Second opinions can play a vital part in ensuring healthcare costs are not wasted on unnecessary or incorrect care," said Peter Costello, presidentand chief executive officer, BCS Financial. "BCS is committed to helping our policyholders and their members manage their risks while improving healthcare outcomes."

Members see direct benefit from the second-opinion consultation. "Members facing a complex diagnosis, surgeryor healthcare decision often find it difficult to navigate the healthcare system and manage costs," said Chris Kurtenbach, vice president, Services & Operations, BCS Financial. "With this partnership, we can help members get the appropriate care they need by connecting them with board-certified, elite specialists from top medical institutions, and reducing their costs along the way."

In many cases, the second opinion can change the treatment trajectory 85% of 2nd.MD's consultations result in improved treatment plans, 35% lead to alternative diagnoses, and nearly 30% of surgery consults result in surgery cancellation.

This is an especially important service as the market for gene therapy and cellular immunotherapy continues to ramp up. An estimated 50-75 therapies will be approved for the U.S. market by 2030, with costs for cellular immunotherapies averaging around $400,000, and gene therapy costs estimated in the $2-$4 million range. For those with complex cancers, rare or orphan conditions, and aggressive recommendations, access to top specialists can make all the difference.

"Better care and outcomes for members is always our priority," said Costello. "This partnership is just one of the ways we are delivering value to our Blue Cross and Blue Shield partners and their members by helping them continue to manage healthcare costs and quality."

This service is currently available to members covered by BCS Stop Loss and Excess of Loss policies.

About BCS Financial Corporation (BCS)

BCS Financial Corporation has more than 70 years of experience delivering a wide-range of insurance and financial solutions for Blue Cross and Blue Shield organizations nationwide. Through its subsidiaries, BCS Insurance Company and 4 Ever Life Insurance Company, BCS is licensed in all 50 states and is rated A- (Excellent) by A.M. Best. BCS Financial is headquartered in Oakbrook Terrace, Illinois. Learn more at bcsf.com.

About 2nd.MD

2nd.MD offers medical certainty by connecting you with board-certified, leading specialists from across the country for an expert second opinion via video or phone within three to five days, from the comfort of your own home. From the minute you reach out, 2nd.MD's Care Team of nurses will put you at the center of your medical care by coordinating all the details. This includes understanding your health goals, gathering all of your medical records, and connecting you with a doctor who specializes in your specific medical condition, so that you can focus on what matters most getting the best care possible. No travel. No hassle. No cost. If you haven't used 2nd.MD before, it can be life-changing. In fact, 98% of people who use 2nd.MD recommend it to their friends and family.

For more information, please contact Nathan Post at 630-472-7860.

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BCS Announces Partnership With 2nd.MD to Improve Member Healthcare Cost, Quality, and Outcomes - PRNewswire

IRVINE, Calif. & BOSTON--(BUSINESS WIRE)--Novus Therapeutics, Inc. (Novus) (NASDAQ: NVUS) today announced it has completed the acquisition of Anelixis Therapeutics, Inc. (Anelixis), a privately held clinical stage biotechnology company developing a next generation anti-CD40 Ligand (CD40L) antibody as a potential treatment for organ and cellular transplantation, autoimmune diseases, and neurodegenerative diseases. Concurrent with the acquisition of Anelixis, Novus entered into a definitive agreement for the sale of non-voting convertible preferred stock (the Preferred Stock) in a private placement to a group of institutional accredited investors led by BVF Partners L.P., with participation from Cormorant Asset Management, Ecor1 Capital, Logos Capital, Fidelity Management and Research Company, Adage Capital Partners L.P., Woodline Partners LP, Ridgeback Capital, Janus Henderson Investors, and Samsara BioCapital, as well as additional investors. The private placement is expected to result in gross proceeds to Novus of approximately $108 million before deducting placement agent and other offering expenses. The proceeds from the private placement will be used to fund the Companys operations, including to advance Phase 2 clinical trials of AT-1501, a humanized IgG1 anti-CD40L antibody with high affinity for CD40L, in renal transplantation, islet cell transplantation, autoimmune nephritis, and amyotrophic lateral sclerosis (ALS).

We are excited about AT-1501 and the potential to develop and commercialize the next generation anti-CD40L antibody, a well-validated target with broad therapeutic possibilities, said Keith A. Katkin, Chairman of the Board of Directors of Novus. After exploring a range of strategic options to maximize shareholder value, we believe this acquisition represents the greatest value creation opportunity for Novus stockholders, and we are confident that we have the management and scientific leadership team to fully realize this opportunity for patients in need of new treatment options.

Leadership & Organization

In addition to the strategic acquisition and private placement, Novus announced its Board of Directors has previously appointed David-Alexandre DA C. Gros, M.D. to serve as Chief Executive Officer and Director. Dr. Gros joins Novus from Imbria Pharmaceuticals Inc., where he served as Co-Founder, Chief Executive Officer and Director. Prior to Imbria, Dr. Gros was President and Chief Operating Officer of Neurocrine Biosciences, Inc., Chief Business and Principal Financial Officer of Alnylam Pharmaceuticals, Inc., and Chief Strategy Officer of Sanofi, S.A. Before Sanofi, Dr. Gros held leadership positions in healthcare investment banking at Centerview Partners, LLC, and Merrill Lynch, Pierce, Fenner & Smith, Inc., and in healthcare consulting at McKinsey & Company. Dr. Gros earned a Doctor of Medicine from Johns Hopkins University School of Medicine, a Master of Business Administration from Harvard Business School, and a Bachelor of Arts from Dartmouth College.

I am both thrilled and humbled to join the Novus management team and Board during this new phase of the companys evolution, as we prepare to initiate multiple Phase 2 trials for AT-1501 said Dr. Gros. Through this acquisition and financing, we now have the scientific, organizational and financial resources to build upon a deep historical understanding of the CD40/CD40L pathway, as well as Anelixis preclinical and Phase 1 data, to address the needs of people undergoing organ or cellular transplantation, or living with autoimmune and neurodegenerative diseases.

Joining Dr. Gros on the Novus management team and Board of Directors is Steven Perrin, Ph.D., Founder and Chief Executive Officer of Anelixis, who will take on the role of President and Chief Scientific Officer. Dr. Perrin brings 20 years of drug development experience to Novus, having held R&D positions at the Hoechst-Ariad Genomics Center, Aventis Pharmaceuticals, Inc., and Biogen Idec, Inc. Over the past decade, Dr. Perrin has worked with the ALS Therapy Development Institute to develop the worlds largest ALS drug development program, bridging preclinical and clinical programs. Dr. Perrin received a Ph.D. in biochemistry from Boston University Medical Center, where he also started his career as Associate Professor of Medicine, and a Bachelor of Science from Boston College.

The activation of CD40/CD40L signaling is critical to mediating antibody and cellular inflammatory response. We are developing antibodies to inhibit the activation of this pathway with the hope of offering new treatment modalities for people living with conditions such as autoimmune nephritis and ALS, or those requiring a potentially life-saving transplant, said Dr. Perrin. I have dedicated my career to developing better medicines for these patients and their families, and I look forward to working with the team to advance these clinical programs.

Concurrent with the acquisition, former Anelixis Chairman of the Board Walter Ogier has been appointed to the Novus Board of Directors. Mr. Ogier has more than 30 years of experience developing therapeutic medical products ranging from pharmaceuticals to medical devices, stem and immune cell therapies, and gene therapies. He has served in multiple CEO roles including Genetix Pharmaceuticals, Inc. (now bluebird bio, Inc.) and Acetylon Pharmaceuticals, Inc., which Celgene Corporation acquired in 2016. In addition to Novus, he serves as a director of Biothera Pharmaceuticals, Inc., Thetis Pharmaceuticals, LLC, and Nemucore Medical Innovations, Inc., and as Board advisor to Kodikaz Therapeutic Solutions, Inc., and ME Therapeutics, Inc.

Novus Board members will also include Keith A. Katkin, Chairman of the Board; Gary A. Lyons; and John S. McBride. The company will continue to maintain its executive offices in Irvine, Calif. and will have research and development facilities in Boston, Mass.

About the Transactions

The acquisition of Anelixis was structured as a stock-for-stock transaction whereby all of Anelixis outstanding equity interests were exchanged in a merger for a combination of shares of Novus common stock and shares of Preferred Stock. Concurrently with the acquisition of Anelixis, Novus entered into definitive agreements for a PIPE investment with existing and new investors to raise approximately $108 million in which the investors will be issued shares of Preferred Stock at a price of approximately $500 per share (or, $0.50 per share on an as-converted-to-common basis). The PIPE offering is expected to close on September 14, 2020. Subject to stockholder approval, each share of Preferred Stock will, at the option of the holder, be convertible into 1,000 shares of common stock, subject to certain beneficial ownership limitations set by each holder. The acquisition was approved by the Board of Directors of Novus and the equity holders of Anelixis.

Ladenburg Thalmann & Co. Inc. is serving as exclusive financial advisor and Gibson, Dunn & Crutcher LLP is serving as legal counsel to Novus. Goodwin Procter LLP is serving as legal counsel to Anelixis. SVB Leerink is serving as financial advisor and lead placement agent for the private placement, and Noble Life Science Partners, a division of Noble Capital Markets, Inc., is acting as co-placement agent.

Additional details are available in an updated corporate presentation that can be found online at http://www.novustherapeutics.com.

Webcast Details

Novus will host an audio webcast on Tuesday, September 15, 2020, at 8:30 a.m. EDT to discuss the acquisition. The live audio webcast will be accessible through a direct link and the investor section of http://www.novustherapeutics.com. To access via phone, please dial (833) 614-1390 (toll-free) or (914) 987-7111 (international) and provide the conference ID 4046285. Please visit the investor section of the Novus website at http://www.novustherapeutics.com for the archived webcast and for more information on the acquisition.

About AT-1501

AT-1501 is a humanized IgG1 anti-CD40L antibody with high affinity for CD40L, a well-validated target with broad therapeutic potential. The CD40/CD40L pathway plays a central role in generating pro-inflammatory responses in autoimmune disease, allograft transplant rejection, and neuroinflammation. In a Phase 1 safety study of healthy volunteers and patients with ALS, AT-1501 was well tolerated at all doses tested.

About Novus Therapeutics

Novus Therapeutics, Inc. is a clinical stage biotechnology company using its expertise in targeting the CD40L pathway to develop potential treatments for people requiring an organ or cell-based transplant, and for people with autoimmune and neurodegenerative disease. Novus is headquartered in Irvine, Calif. For more information, please visit the companys website at http://www.novustherapeutics.com.

Follow Novus Therapeutics on social media: @Novus_Thera and LinkedIn.

Notice of Issuance of Inducement Grants

Pursuant to their employment agreements, Drs. Gros and Perrin have been awarded options to purchase a total of 18,279 and 7,857 shares of Preferred Stock, respectively, subject to time-based vesting (the "Inducement Grants"). The Inducement Grants have an exercise price of $500 per share of Preferred Stock, which is equal to the price at which the Preferred Stock is being offered and sold in the PIPE financing and represents (on an as-converted basis) a premium of approximately 30% over the last reported closing price of the Novus common stock prior to grant. The Inducement Grants have been approved by the Novus Board of Directors and the Compensation Committee of the Board of Directors. The Inducement Grants will be issued outside of the Company's stockholder-approved equity incentive plans as an inducement grant in accordance with Nasdaq Listing Rule 5635(c)(4).

Forward-Looking Statements

This press release contains forward-looking statements that involves substantial risks and uncertainties. Any statements about the companys future expectations, plans and prospects, including statements about its strategy, future operations, development of its product candidates, and other statements containing the words believes, anticipates, plans, expects, estimates, intends, predicts, projects, targets, looks forward, could, may, and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, although not all forward-looking statements include such identifying words. Forward-looking statements include, but are not limited to statements regarding: risks related to market conditions; expectations regarding the timing for the commencement of future clinical trials; expectations regarding the success of clinical trials; the rate and degree of market acceptance and clinical utility of the companys products; the companys estimates regarding expenses and cash runway; and the impact of the ongoing coronavirus pandemic. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors. These risks and uncertainties, as well as other risks and uncertainties that could cause the companys actual results to differ significantly from the forward-looking statements contained herein, are discussed in our quarterly 10-Q, annual 10-K, and other filings with the SEC, which can be found at http://www.sec.gov. Any forward-looking statements contained in this press release speak only as of the date hereof and not of any future date, and the company expressly disclaims any intent to update any forward-looking statements, whether as a result of new information, future events or otherwise.

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Novus Therapeutics Announces Acquisition of Anelixis Therapeutics - Business Wire

Seeing a child debilitated by illness is never easy.

When doctors tell youthere is nothing they nor you can do to help ease yourbaby's suffering, well, parents who know that type of helplessness often find it hard to describe.

We were told to take my son home and love him, becausehe probably wouldnt live past his second birthday, said Pace father Todd Hamrick. But, were way past that birthday now.

Hamricks son, Alek, was diagnosed at six months old with spinal muscular atrophy and not expected to live long enough to toddle. ButAlek, now 3, has beaten the odds and outlived that initial, bleak prognosis.

His parents attribute much of his success to his doctor, Richard Finkel, who entered Alek into a clinical trial for what they believe has been a wonder drug for their little boy, Evrysdi, which was recently approved by the Food and Drug Administration.

Whats hard to even get around is that people even bothered to research it, Todd Hamrick said. Its just a small amount of the population that has SMA. Its not like researching a blood pressure medication.

Spinal muscular atrophy, or SMA,is a genetic disorder caused by a loss of nerve cells that effect human motor function.

Essentially, those afflicted by SMA are made weak. Their muscleswaste away. In many cases, eventually, a person loses their ability to walk, to eat andevento breathe, and they die.

Aleks mother, Iwona Hamrick, is a nurse at a local hospital and wellremembers the moment she heardher sons diagnosis.

It was unimaginable. His pregnancy was normal, she said. We did genetic testing and it was negative. Unfortunately, at that time, they were not screening for his disease. So, from a healthy baby to a dying baby, you know?

The parents felt they had to travel, in more ways than one, to find the places and help that they could for their Alek.

Todd Hamrick said that he and wife decided to move from Gulf Breeze to Pace after Aleks diagnosis after feeling ostracized by many of their former acquaintances. The parents felt like some people who they used to know were made uncomfortable by their son's illness.

The area is very in-the-dark when it comes to children with issues, Todd Hamrick said. If your kids are healthy and you'rehealthy, its a great area. But, it doesnt attract the greatest talent or best and biggest facilities or endowments.

So, Alek traveled with his family to meet his future doctor, Finkel, at the Nemours Children's Hospital in Orlando.

Alekwas first treated with a gene therapy drug, and he made some response with that, Finkel told the News Journal. But more recently, he started on a second drug, which seems to be having an enhanced effect, I must say.

Finkel, an expert in the field of pediatric neurologic disorders, left Florida in March for a position leading the new Center for Experimental Neurotherapeutics at St. Jude Childrens Research Hospital in Memphis, Tennessee. Buthe has continued to monitor Aleks progress via video.

Both of the drugs he has received, the gene therapy and this new drug, Evrysdi, are designed to increase a certain protein in Aleks body that is deficient because of his genetic disorder, he explained. But they do it in different ways. The potential advantage of his new drug, Evrysdi because its an oral drug; you take it by mouth it goes into the stomach and into the bloodstream, and from there, it goes to all the tissues of the body.

And, we think that there is an enhanced effect, becauseit gets into the muscle tissue, Finkel continued. "These are very, very, early daysin trying to make assessments, soI dont want to say that we can come to any kind of conclusions yet."

However the cutting-edge drug works doesnt matter to a mother, whos just glad that it isworking.

It gave us hope. Thats for sure, Iwona Hamrick said. Becausewe felt helpless.

Since Alek started his new treatment last November, his strength has increased tremendously.

His muscle tone got better, Iwona Hamrick said. He is much stronger in the upper body, so much so, he is pushing his little wheelchair.

Alek can now cruise around his Pace home in an extraordinarily lite-weight wheelchair designed by a Swedish inventor who alsohas a child with SMA.

Alek had a lot of trouble before starting this medication even pushing it, Iwona Hamrick said. Sonowhe is just rolling around the house. Also, he is barring more weight on his legs.

Recently, Alek has started to be able to walk in a pool a huge milestone for the toddler.

But some worries remain the same.

Every day, Alek must use a type of breathing machine.

Its a cough assist machine, Todd Hamrick explained. We use it two times a day, when hes healthy. Becausewhere we can just clear our throats when we cough, he doesnt have that strength no lung strength.

Alek attends physical therapy, aqua-therapy,hippotherapy, occupational therapy and speech therapy sessions every week to try and ensure he remains healthy and continues to properly develop.

My worries have changed a lot, Todd Hamrick said. I used to worry my child was going to die. Now, Im worrying about if other kids will bully him at school.

"But that'sa great worry to have," he continued. "Compared to how it use to be, getting picked on is a great thing to worry about.

Colin Warren-Hicks can be reached at colinwarrenhicks@pnj.com or 850-435-8680.

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Pace toddler wasn't supposed to survive. Thanks to wonder drug, parents say he's thriving - Pensacola News Journal

One of the major challenges facing gene therapy - a way to treat disease by replacing a patients defective genes with healthy ones - is that it is difficult to safely deliver therapeutic genes to patients without the immune system destroying the gene, and the vehicle carrying it, which can trigger life-threatening widespread inflammation.

Three decades ago researchers thought that gene therapy would be the ultimate treatment for genetically inherited diseases like hemophilia, sickle cell anemia and genetic diseases of metabolism. But the technology couldnt dodge the immune response.

Since then, researchers have been looking for ways to perfect the technology and control immune responses to the gene or the vehicle. However, many of the strategies tested so far have not been completely successful in overcoming this hurdle.

Drugs that suppress the whole immune system, such as steroids, have been used to dampen the immune response when administering gene therapy. But its difficult to control when and where steroids work in the body, and they create unwanted side effects. My colleague Mo Ebrahimkhani and I wanted to tackle gene therapy with immune-suppressing tools that were easier to control.

I am a medical doctor and synthetic biologist interested in gene therapy because six years ago my father was diagnosed with pancreatic cancer. Pancreatic cancer is one of the deadliest forms of cancer, and the current available therapeutics usually fail to save patients. As a result, novel treatments such as gene therapy might be the only hope.

Yet, many gene therapies fail because patients either already have pre-existing immunity to the vehicle used to introduce the gene or develop one in the course of therapy. This problem has plagued the field for decades, preventing the widespread application of the technology.

Traditionally scientists use viruses - from which dangerous disease-causing genes have been removed - as vehicles to transport new genes to specific organs. These genes then produce a product that can compensate for the faulty genes that are inherited genetically. This is how gene therapy works.

Though there have been examples showing that gene therapy was helpful in some genetic diseases, they are still not perfect. Sometimes, a faulty gene is so big that you cant simply fit the healthy replacement in the viruses commonly used in gene therapy.

Another problem is that when the immune system sees a virus, it assumes that it is a disease-causing pathogen and launches an attack to fight it off by producing antibodies and immune response just as happens when people catch any other infectious viruses, like SARS-CoV-2 or the common cold.

Recently, though, with the rise of a gene editing technology called CRISPR, scientists can do gene therapy differently.

CRISPR can be used in many ways. In its primary role, it acts like a genetic surgeon with a sharp scalpel, enabling scientists to find a genetic defect and correct it within the native genome in desired cells of the organism. It can also repair more than one gene at a time.

Scientists can also use CRISPR to turn off a gene for a short period of time and then turn it back on, or vice versa, without permanently changing the letters of DNA that makes up or genome. This means that researchers like me can leverage CRISPR technology to revolutionize gene therapies in the coming decades.

But to use CRISPR for either of these functions, it still needs to be packaged into a virus to get it into the body. So some challenges, such as preventing the immune response to the gene therapy viruses, still need to be solved for CRISPR-based gene therapies.

Being trained as a synthetic biologist, I teamed up with Ebrahimkhani to use CRISPR to test whether we could shut down a gene that is responsible for immune response that destroys the gene therapy viruses. Then we investigated whether lowering the activity of the gene, and dulling the immune response, would allow the gene therapy viruses to be more effective.

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A gene called Myd88 is a key gene in the immune system and controls the response to bacteria and viruses, including the common gene therapy viruses. We decided to temporarily turn off this gene in the whole body of lab animals.

We injected animals with a collection of the CRISPR molecules that targeted the Myd88 gene and looked to see whether this reduced the quantity of antibodies that were produced to specifically fight our gene therapy viruses. We were excited to see that the animals that received our treatment using CRISPR produced less antibody against the virus.

This prompted us to ask what happens if we give the animal a second dose of the gene therapy virus. Usually the immune response against a gene therapy virus prevents the therapy from being administered multiple times. Thats because after the first dose, the immune system has seen the virus, and on the second dose, antibodies swiftly attack and destroy the virus before it can deliver its cargo.

We saw that animals receiving more than one dose did not show an increase in antibodies against the virus. And, in some cases, the effect of gene therapy improved compared with the animals in which we had not paused the Myd88 gene.

We also did a number of other experiments that proved that tweaking the Myd88 gene can be useful in fighting off other sources of inflammation. That could be useful in diseases like sepsis and even COVID-19.

While we are now beginning to improve this strategy in terms of controlling the activity of the Myd88 gene. Our results, now published in Nature Cell Biology,provide a path forward to program our immune system during gene therapies and other inflammatory responses using the CRISPR technology.

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CRISPR can help combat the troubling immune response against gene therapy - The Conversation US

Ovarian cancer affects several women these days. Early diagnosis can help in successful treatment of the cancer. Read here to know everything about its diagnosis.

Ovarian cancer is more common in post-menopausal women

Ovarian cancer stands to be the third most common cause of cancer in Indian women. It is also the second most commonly occurring gynaecological cancers in the country today, as per reports. By the present year, 2020, India is supposed to have over 36,000 new ovarian cancer cases while around a lakh case would be prevalent. The cancer constitutes to be 6% of all cancer cases in India.

There are multiple types of ovarian cancer. The most common ones are those which develop on the ovarian surface lining. This usually affects post-menopausal women. A female reproductive system contains two ovaries, one on each side of the uterus.

The exact cause of ovarian cancer is not known; however, some of the common risk factors associated with the disease include post-menopausal status, genetic predisposition, hormonal replacement therapy, overweight and smoking.

Early detection is critical for curing ovarian cancer. Treatment and cure will be highly effective when the cancer is still confined to the ovary. The disease is largely asymptomatic in its early stages. Consequently, patients do not seek any medical advice which makes the condition worse. Diagnoses is the first step to confirm or rule out the disease.

Early detection can help treat ovarian cancer on timePhoto Credit: iStock

Unlike breast and cervical cancer, there is no standardised mass level screening technique associated with ovarian cancer. Only through a laparoscopic biopsy the epithelial precursor lesion STIC can be detected early, which cannot be applied at a mass level. More useful methods include trans vaginal USG (specially in post-menopausal women) and serial and annual USG evaluation. Another effective method can be to genetically screen the BRCA gene, which has a significant role in the disease process of hereditary ovarian cancer.

Also read:Ovarian Cancer: Causes, Symptoms, Prevention And Treatment

There are several tests used in the cases of ovarian cancer. The CA125 is the most common tested biomarker for ovarian cancer, especially for post-menopausal women. It is highly important to know the disease stage at the time of presentation, as it is crucial for evaluating the diagnostic utility of this test. The test is not sufficient as a diagnostic marker to differentiate between benign and malignant tumours. However, a new found test which is the combination of human epididymis secretory protein 4 (HE4) is considered as more competent (sensitive) in cancer detection.

Another method is to use imaging or radiology in full blown cases of cancerous ovary. The disease stage and viability of surgical resection is determined by computed tomography (CT) scan of the abdomen and pelvis with oral and intravenous contrast, and chest imaging. However, the most reliable method is to conduct a biopsy which includes cutting a small piece or surgical removal of tumour from suspected tissue and getting it examined for malignancy. A microscopic examination reveals the final diagnosis of ovarian cancer and its type.

In early stages when the tumor is operable, surgical resection of the tumor is the treatment of choice. However in advanced cases where the tumor has spread, chemotherapy is the preferred treatment modality.

Also read:Ovarian Cancer Is Deadly, But New Tests, Treatments Start To Emerge

Ovarian cancer hasn't received equivalent focus in comparison with breast and cervical cancers. The biggest challenge of the top three women cancers which include breast, cervical, and ovarian cancers is the stigma associated with it. Owing to numerous social pressures and stigmas associated with check-up of breasts and gynaecology, women don't come forward for diagnosis and worse delay treatment on being diagnosed. Consequently, they are significantly under-reported and under-investigated. It therefore becomes imperative that much awareness is imparted about the second most common gynaecological malignancy in Indian women.

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(Dr. Prabal Deb, Director Lab Operations & Chief Histopathologist, SRL Diagnostics)

Disclaimer: The opinions expressed within this article are the personal opinions of the author. NDTV is not responsible for the accuracy, completeness, suitability, or validity of any information on this article. All information is provided on an as-is basis. The information, facts or opinions appearing in the article do not reflect the views of NDTV and NDTV does not assume any responsibility or liability for the same.

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How Is Ovarian Cancer Diagnosed? Everything You Need To Know - NDTV Doctor

DetailsCategory: Small MoleculesPublished on Saturday, 05 September 2020 18:01Hits: 346

Gavreto is a once-daily, oral precision therapy that selectively inhibits RET-altered cancers

Genentech and Blueprint Medicines will co-commercialize Gavreto in the United States

FDA also granted Priority Review to Gavreto for the treatment of people with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer

South San Francisco, CA, USA I September 4, 2020 I Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has approved Gavreto (pralsetinib) for the treatment of adults with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test. This indication was approved under the FDAs accelerated approval program based on data from the Phase I/II ARROW study. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Gavreto is a once-daily, oral precision therapy designed to selectively target RET alterations, including fusions and mutations.

The FDA approval of Gavreto for RET fusion-positive non-small cell lung cancer is an important step towards our goal of providing an effective treatment option for every person diagnosed with lung cancer, no matter how rare or hard-to-treat their type of disease, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. We remain committed to finding personalized treatment options for people with cancer based on specific genomic or molecular alterations, and we look forward to partnering with Blueprint Medicines to further explore the potential of Gavreto across multiple RET-altered tumor types.

RET-activating fusions and mutations are key disease drivers in many cancer types, including NSCLC and medullary thyroid cancer (MTC), and treatment options that selectively target these genetic alterations are limited. In NSCLC, RET fusions represent approximately 1-2% of patients. Biomarker testing for these fusions is the most effective way to identify people who are eligible for treatment with Gavreto.

The approval is based on the results from the Phase I/II ARROW study, in which Gavreto produced durable clinical responses in people with RET fusion-positive NSCLC with or without prior therapy, and regardless of RET fusion partner or central nervous system involvement. Gavreto demonstrated an overall response rate (ORR) of 57% (95% CI: 46%, 68%) and complete response (CR) rate of 5.7% in the 87 people with NSCLC previously treated with platinum-based chemotherapy, and the median duration of response (DoR) was not reached (95% CI: 15.2 months, not reached). In the 27 people with treatment-nave NSCLC, the ORR was 70% (95% CI: 50%, 86%) with an 11% CR rate. The most common adverse reactions (25%) were fatigue, constipation, musculoskeletal pain and increased blood pressure (hypertension).

Gavreto is now the sixth FDA-approved medicine in Genentechs portfolio of treatments for lung cancer. The FDA granted Breakthrough Therapy Designation to Gavreto for the treatment of RET fusion-positive NSCLC that has progressed following platinum-based chemotherapy and for RET mutation-positive MTC that requires systemic treatment and for which there are no acceptable alternative treatments.

The FDA has also granted Priority Review to Gavreto for the treatment of people with advanced or metastatic RET-mutant MTC and RET fusion-positive thyroid cancer, and is expected to make a decision on approval by February 28, 2021. This New Drug Application (NDA) was accepted for review under the FDAs Real-Time Oncology Review (RTOR) pilot program, which aims to explore a more efficient review process to ensure safe and effective treatments are available to patients as early as possible.

For those who qualify, Blueprint Medicines will offer patient assistance programs for people prescribed Gavreto by their doctor through YourBlueprint . Please visit http://www.yourblueprint.com or contact 1-888-BLUPRNT for more information.

About the ARROW study

ARROW (NCT03037385) is a Phase I/II, open-label, first-in-human study designed to evaluate the safety, tolerability and efficacy of Gavreto, administered orally in people with rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer (MTC), RET fusion-positive thyroid cancer and other RET-altered solid tumors. The trial consists of two parts: a dose escalation portion, which is complete, and an expansion portion in people treated with 400 mg of Gavreto, once-daily. ARROW is being conducted at multiple sites across the United States, European Union and Asia.

About lung cancer

According to the American Cancer Society, it is estimated that more than 228,000 Americans will be diagnosed with lung cancer in 2020, and NSCLC accounts for 80-85% of all lung cancers. It is estimated that approximately 85% of lung cancer diagnoses in the United States are made when the disease is in the advanced stages. In NSCLC, RET fusions represent approximately 1-2% of patients.

About Gavreto

Gavreto is a once-daily, oral precision therapy designed to selectively target RET alterations, including fusions and mutations, regardless of the tissue of origin. Preclinical data have shown that Gavreto inhibits primary RET fusions and mutations that cause cancer in subsets of patients, as well as secondary RET mutations predicted to drive resistance to treatment. Blueprint Medicines and Genentech are also co-developing Gavreto for the treatment of patients with various types of RET-altered thyroid cancers and other solid tumors.

Gavreto U.S. Indication

Gavreto (pralsetinib) is indicated for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test.

This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Important Safety Information

Gavreto may cause serious side effects, including:

Lung problems (pneumonitis) occurred in 10% of patients who received Gavreto, including 2.7% with Grade 3/4, and 0.5% with fatal reactions. Monitor for pulmonary symptoms indicative of interstitial lung disease (ILD)/pneumonitis. Withhold Gavreto and promptly investigate for ILD in any patient who presents with acute or worsening of respiratory symptoms (e.g., dyspnea, cough, and fever). Withhold, reduce dose or permanently discontinue Gavreto based on severity of confirmed ILD.

Increased blood pressure (hypertension) occurred in 29% of patients, including Grade 3 hypertension in 14% of patients. Overall, 7% had their dose interrupted and 3.2% had their dose reduced for hypertension. Treatment-emergent hypertension was most commonly managed with anti-hypertension medications. Do not initiate Gavreto in patients with uncontrolled hypertension. Optimize blood pressure prior to initiating Gavreto. Monitor blood pressure after 1 week, at least monthly thereafter and as clinically indicated. Initiate or adjust anti-hypertensive therapy as appropriate. Withhold, reduce dose, or permanently discontinue Gavreto based on the severity.

Liver problems (hepatotoxicity): Serious hepatic adverse reactions occurred in 2.1% of patients treated with Gavreto. Increased AST occurred in 69% of patients, including Grade 3/4 in 5.4% and increased ALT occurred in 46% of patients, including Grade 3/4 in 6%. The median time to first onset for increased AST was 15 days (range: 5 days to 1.5 years) and increased ALT was 22 days (range: 7 days to 1.7 years). Monitor AST and ALT prior to initiating Gavreto, every 2 weeks during the first 3 months, then monthly thereafter and as clinically indicated. Withhold, reduce dose or permanently discontinue Gavreto based on severity.

Grade 3 bleeding (hemorrhagic events) occurred in 2.5% of patients treated with Gavreto including one patient with a fatal hemorrhagic event. Permanently discontinue Gavreto in patients with severe or life-threatening hemorrhage.

Impaired wound healing can occur in patients who receive drugs that inhibit the vascular endothelial growth factor (VEGF) signaling pathway. Therefore, Gavreto has the potential to adversely affect wound healing. Withhold Gavreto for at least 5 days prior to elective surgery. Do not administer for at least 2 weeks following major surgery and until adequate wound healing. The safety of resumption of Gavreto after resolution of wound healing complications has not been established.

Based on findings from animal studies and its mechanism of action, Gavreto can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective non-hormonal contraception during treatment with Gavreto and for 2 weeks after the final dose. Advise males with female partners of reproductive potential to use effective contraception during treatment with Gavreto and for 1 week after the final dose. Advise women not to breastfeed during treatment with Gavreto and for 1 week after the final dose.

Common adverse reactions (25%) were fatigue, constipation, musculoskeletal pain, and hypertension. Common Grade 3-4 laboratory abnormalities (2%) were decreased lymphocytes, decreased neutrophils, decreased phosphate, decreased hemoglobin, decreased sodium, decreased calcium (corrected) and increased alanine aminotransferase (ALT).

Avoid coadministration with strong CYP3A inhibitors. Avoid coadministration of Gavreto with combined P-gp and strong CYP3A inhibitors. If coadministration cannot be avoided, reduce the Gavreto dose. Avoid coadministration of Gavreto with strong CYP3A inducers. If coadministration cannot be avoided, increase the Gavreto dose.

Please click here to see the full Prescribing Information for Gavreto.

Gavreto, Blueprint Medicines, YourBlueprint and associated logos are trademarks of Blueprint Medicines Corporation.

About Genentech in lung cancer

Lung cancer is a major area of focus and investment for Genentech, and we are committed to developing new approaches, medicines and tests that can help people with this deadly disease. Our goal is to provide an effective treatment option for every person diagnosed with lung cancer. We currently have six approved medicines to treat certain kinds of lung cancer and more than 10 medicines being developed to target the most common genetic drivers of lung cancer or to boost the immune system to combat the disease.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

SOURCE: Genentech

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Genentech Announces FDA Approval of Gavreto (pralsetinib) for the Treatment of Adults With Metastatic RET Fusion-Positive Non-Small Cell Lung Cancer |...

Today, in our Gilmore Health Q&A Series we discuss genetic diseases with Dr. Sony Sherpa as they are a biological, and financial burden. They may only affect a single member of the family physically but can be socially and mentally draining for the rest of the family.

Genetic diseases are a group of diseases that can pass from parents to their children, causing disease with varying degrees of severity. Genetic diseases are relatively common, making up to 14% of all pediatric discharges. Furthermore, they cost upwards of 50 billion dollars yearly, putting a financial strain on the families of affected individuals

Here we talk more about genetic diseases with Dr. Sony Sherpa, to get more insight into these diseases with the aim of understanding them further.

What are genetic diseases? Arent all diseases genetic, technically?

Dr. Sony Sherpa: Well, yes. As researchers learn more about diseases and the different components to them, they have concluded that almost all diseases do have a genetic component to them. However, a genetic disease is a disease that occurs as a result of a change in a persons DNA. This change or mutation can cause disease of varying severity depending on the extent of damage done to the DNA.

So, when you say all diseases have a genetic component, that means all diseases arise from this change in the DNA?

The short answer is yes. The long answer is that most genetic diseases are diseases arising from mutations in one single gene. But most diseases, in general, are what we call multifactorial, or complex disease. Now, these diseases can arise due to environmental factors, genetic factors, occupational hazards, or iatrogenic reasons. Hence, the name; multifactorial. The genetic component of these diseases, albeit a small part, is a very important part in the understanding of these diseases.

Could you perhaps elaborate on this with an example?

Yes, sure. Diseases like sickle cell or Cystic fibrosis are genetic diseases. They arise from a mutation in a single gene and have a clear cut inheritance pattern. On the other hand, there are diseases like diabetes mellitus type 2 or cardiovascular diseases that are polygenic in origin. Not just that, the inheritance pattern of these diseases isnt understood much either. While many studies are looking into it, its going to be a while before we understand the hereditability factors of these complex diseases.

Can complex diseases be passed on from parents to their children? Is it seen usually, or is that one of the many things yet to be discovered?

Complex diseases usually cluster in families, but as I mentioned before, there is no clear explanation for that phenomenon yet.

Alright, then how do single gene-genetic diseases pass on from parents to offsprings?

That we have an understanding of. At least the basics. There are five inheritance patterns of single-gene genetic diseases, namely; Autosomal Dominant, Autosomal Recessive, X-linked Dominant, X-linked Recessive, and mitochondrial. However, the pattern of inheritance differs for each disease.

All single-gene genetic disorders follow one of these five inheritance patterns. Whats the difference between them, in simple terms? What is the basis of separation between these patterns?

Well, it is pretty interesting. Inheritance patterns tell us the origin of the genetic disease, the genotype it has and it can mathematically estimate the percentage risk that a child born to two carriers/patients will have the disease. The genotype of the genetic makeup decides the phenotype or physical appearance of the disease for everyone. The genotype can be realized from alleles that are received from both parents. These alleles can recognize the traits and then classify the inheritance into dominant or recessive. Basically, if a disease requires a mutation to be present on both alleles, then it is recessive. But if it can be pathological with just one mutated allele, then it is dominant.

What about the mitochondrial inheritance pattern then?

Some books and studies still do not accept mitochondrial inheritance patterns to be one of the main inheritance patterns. But regardless of that, it is an important topic. Mitochondria of the cell have their own DNA. And this DNA is passed down from mother to offspring. Its always inherited from the mother. Therefore, any mitochondrial disease would also be purely maternal in origin.

Since these diseases are inherited with alleles on genes and are due to mutation in the DNA, how does one diagnose these diseases? I am assuming it would require a lot of genetic karyotyping and testing? That sounds like a very tedious and complicated process.

Dr. Sony Sherpa: Yes and No. Yes, genetic diseases are very much gene-related as the name suggests, these diseases are diagnosed mostly through genetic testing. OR better yet, the diagnosis is usually suspected through clinical presentation and laboratory testing, it is confirmed through genetic testing.

Genetic testing is not tedious, and a very simple procedure. It can be done in one of three ways; cytogenetic genetic testing wherein the entire chromosome is analyzed and studied, biochemical genetic testing which includes testing the proteins and the biochemical reactions associated with them, and lastly, molecular genetic testing for analysis of small DNA mutation.

You mentioned suspicion of a genetic disease. What araises suspicion of a genetic disease, what are the symptoms that can be alarming?

The suspicion of genetic diseases depends on physical examination, family, and personal history. The red flags for a doctor usually include positive family history, history of miscarriages or stillbirths in the mother of the affected individual, presence of clinical signs characteristic of a genetic syndrome, and so on. If the mother admits to being exposed to teratogens, whether occupational, alcohol, or certain medications, they may also alert the physician towards the presence of a genetic disease or syndrome. Usually, there are mandatory screening tests for certain genetic conditions in most countries.

Basically, a genetic disorder can be the differential diagnosis of many symptoms an individual might present to the hospital with?

Dr. Sony S: Well, yes. Pretty much so.

And how would someone affected by a genetic disease get treated for these conditions? It would seem like they would need a gene treatment plan.

Treatment of genetic diseases obviously depends on each disease and what it entails. For genetic syndromes, the plan is usually to prevent its progress with medications all the while treating the symptoms of the disease. Depending on the symptoms, appropriate therapy may be applied.

Genetic diseases are a life-long process, and they require a very healthy lifestyle and strict adherence to medical therapy and surgical therapy if needed, for a chance to have a good quality of life. The main aim is to increase the quality of life and increase the life expectancy of affected individuals. It mostly is symptomatic therapy of sorts.

That explains it. Thank you for your time.

And until our next Q&A session please share us and like us so that we may continue to provide you with the latest in medicine, health, and fitness free of charge. You may also join the Gilmore Health newsletter to receive the latest in health news. If you are interested in any subject that you wish us to cover please share it with us at the comments area below!

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Originally posted here:
Gilmore Health: A Q&A Session on Genetic Diseases With Dr. Sony Sherpa - Gilmore Health News

A TEENAGE boy has been warned he could become entombed in his own body as he battles a rare skin condition.

Jaiden Rogers has Stiff Skin Syndrome, a disease which causes the skin to harden.

7

His Mum Natalie says he is turning to stone due to the condition that has left the 15-year-old confined to a wheelchair and at risk of organ failure as his stone-like skin presses on his lungs and heart.

Jaiden was diagnosed with the condition in 2013 after mum Natalie, 54, found a lump of hard skin on his thigh.

It soon spread to his arms, legs, stomach and neck.

Natalie, who is a stay-at-home mum, said: "Jaiden is basically turning to stone and it's become so hard that he can't really move his legs.

"If he doesn't get treatment, doctors say he'll become entombed in his own body.

"His whole body is turning to stone because this disease starts from the inside and works it way to the skin.

Once it reaches his skin, it means all his body underneath is also hard like stone."

7

7

She said the skin has now started to compress on his heart - leaving him unable to breath without a ventilator.

It's pressing on the other organs so they will start failing.

"His future doesn't look good."

Natalie and her husband Tim, 56, are now raising funds to pay for Jaiden to get to Italy where he will be able to receive treatment which will help stop the disease before it crushes his lungs.

The mum-of-three, from Colorado, US, said: "If we don't get him to Italy, he won't live.

7

What is Stiff skin syndrome (SSS)

Stiff skin syndrome is a rare skin condition and is characterised by hard, thick skin on the body.

The thickening of the skin can make the sufferer immobile and can cause joints to get stuck in a bent position.

It is caused by a gene mutation.

What are the signs?

The condition will often present at birth or in childhood and symptoms include:

"Once he has treatment to stop the progression of the disease, he'll be able to have therapy to help loosen the skin.

"The doctors think he's the only one in the world with the condition and there's only been 41 documented cases in the world.

"It started when we found a hard spot on his thigh.

She said that at first, no one really thought anything of it.

We took him to the doctors and after some tests, the doctor called me and told me to sit down.

"That isn't what you want to hear from a doctor.

7

7

"I'd never heard of the condition before and most doctors haven't heard of it so I didn't think much of it until they continued telling me what it would do.

"Within three months it had really started spreading down his leg and he needed a wheelchair.

"It was horrible and he was in severe pain."

Before being diagnosed Jaiden loved to play football.

Now the teen is destined to spend the rest of his life in a wheelchair despite taking chemotherapy medication to help slow down the spread of the disease.

7

Jaiden has also had to drop out of school and is currently being taught at home by his parents.

Natalie said: "He's very quiet about it, he doesn't like talking about it but he cries because it hurts.

"He used to love going to the park and playing soccer and he's obsessed with Ed Sheeran.

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"He was so active, he was all over the place like any other kid but now he sleeps most of the day because of the pain killers he's on.

"This disease has caused him to have no friends because he is in so much pain.

"He'll always be in a wheelchair and we can't change that but we need to get him to Italy so we can at least prolong his life.

"Basically, if we don't get him there, he won't live."

See original here:
Our teenage son is turning to stone and is becoming entombed in his own body - The Sun

Jay Richards talked recently with Matt Scholz, Founder & CEO of Oisn Biotechnologies, about the challenges and promises of the information theory of biotech, especially as related to medicine:

The panel in which Scholz participated at COSM 2019 focused on how artificial intelligence can make a difference in medicine:

From the interview:

Jay Richards: So how would you distill this panel? It was you and Babak Parviz, formerly of Google Glass and now from Amazon (and formerly Google Glass) and Lindy Fishburne, whos on the funding side of information technology and biology.

Matt Scholz: The panel was put together ranging from the computational side of it to the actual therapeutic side and finance. So I think that made it a pretty interesting conversation really, because, to a great extent were working towards the same goals, but from very different angles.

Scholz spoke about his work in gene therapy:

Matt Scholz: What Im working on is, in some respects, the most literal amalgamation of information in life. Like were actually rewriting information in life; its gene therapy. But for the most part, I think the expertise on information and medicine is more on how do you analyze conditions and symptoms, make diagnoses, predict outcomes, that kind of thing.

Note: From the National Institutes of Heath: Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery. Researchers are testing several approaches to gene therapy, including:

Replacing a mutated gene that causes disease with a healthy copy of the gene.

Inactivating, or knocking out, a mutated gene that is functioning improperly.

Introducing a new gene into the body to help fight a disease.

Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently being tested only for diseases that have no other cures.

Jay Richards: A lot of people ask questions about the problems of the regulatory regime in dealing with some of this new stuff. Whats your opinion in terms of, if there was a single primary impediment to real breakthroughs in this area, is it regulation? Is it just the toughness of the subject? Is it something else?

Matt Scholz: Well, I think the regulation in medicine is considerable both in the time and expense it takes, but I dont think its the primary impediment to anything. In fact, I think particularly in gene therapy, the regulators have been very responsive to changes in technology.

Note: Gene therapy has shown some success with muscular dystrophy (NPR, July 27, 2020), for example, and is an approved treatment for thalassemia (LaBiotech.eu, April 6, 2019). On the other hand, a gene therapy for treatment of hemophilia has been delayed, following questions around how long it lasts (STAT, August 20, 2020) The FDA has been expecting a surge of gene therapy trials (Pharmaceutical Technology, February 5, 2019).

Matt Scholz: Building new treatments in biology is the science itself is hard and the tools we use to build it are clumsy. And so for most of human history, we use mashed up plants to treat diseases, and moving to building viruses and ex vivo cell therapies and manipulating DNA is largely uncharted territory. The regulators have been, Id say, doing an admirable job really, trying to stay on top of those changes and interact with people building them. With that said, obviously, if you want to have a system that takes you a decade to get a drug approved

Jay Richards: Thats what we have.

Matt Scholz: it will cost time and money. And I think theres great conversations that could be had about how we weigh the risk of a drug versus the risk of a disease. And the balance weve struck gives us the system we have today. And its pretty comfortable to think, Okay, well, theres smart people at the FDA who are making sure I dont get things that are dangerous, but if youre dying of a disease

Jay Richards: The cost benefit changes.

Matt Scholz: Yeah, its very different. And I think it may be in some respects, less of what could be done about the regulatory environment as to what can be done to empower the patient. Because right now, the one person who has no say in healthcare is the patient. The bottom of the totem pole.

Jay Richards: Its the same thing with the economics of healthcare. Its a third-party payer problem, but if youre a patient, you actually dont know what the price of the services that youre getting is, thats the problem.

Matt Scholz: Its totally wild, and I think the patient should have the right to be the arbitrator of what goes into their body and not just be subject to the winds of the system. It shouldnt be that what the doctor says, the insurance company says, or even what the FDA says in that respect.

Its there for a reason. We know why these things exist, but its easy to imagine circumstances where you would have a very different perspective on risk and reward. And I would love to see the patients get more power.

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Gene Therapy: IT Meets Medicine, But Who Is In Charge? - Walter Bradley Center for Natural and Artificial Intelligence

Princeton Aug 6, 2020 (Thomson StreetEvents) -- Edited Transcript of Iveric Bio Inc earnings conference call or presentation Wednesday, August 5, 2020 at 12:00:00pm GMT

* David F. Carroll

IVERIC bio, Inc. - Senior VP, CFO & Treasurer

* Glenn P. Sblendorio

IVERIC bio, Inc. - CEO, President & Director

IVERIC bio, Inc. - VP of IR & Corporate Communications

* Kourous A. Rezaei

IVERIC bio, Inc. - Senior VP & Chief Medical Officer

* Pravin U. Dugel

IVERIC bio, Inc. - Executive VP and Chief Strategy & Business Officer

Wedbush Securities Inc., Research Division - MD & Head of Healthcare of Equity Research

Good day, and welcome to the IVERIC bio Second Quarter 2020 Results Conference Call. Today's conference is being recorded. At this time, I would like to turn the conference over to Kathy Galante. Please go ahead.

Kathy Galante, IVERIC bio, Inc. - VP of IR & Corporate Communications [2]

Good morning, and welcome to IVERIC bio's conference call. Representing IVERIC bio today are Mr. Glenn Sblendorio, Chief Executive Officer and President; Dr. David Guyer, Executive Chairman; Mr. Dave Carroll, Chief Financial Officer; Dr. Pravin Dugel, Chief Strategy and Business Officer; Dr. Kourous Rezaei, Chief Medical Officer; Dr. Abraham Scaria, Chief Scientific Officer; and Mr. Keith Westby, Chief Operating Officer.

I would like to remind you that today, we will be making statements relating to IVERIC bio's future expectations regarding operational, financial and research and development matters, including statements regarding the impact of the COVID-19 pandemic on our research and development programs, operations and financial position and on the practices of retinal physicians and the conduct of clinical trials, our expectations to use GATHER1, our previously announced clinical trial for Zimura for the treatment of geographic atrophy as a Phase III clinical trial, our development and regulatory strategy for Zimura and our other product candidates, including our expectations for a second Phase III clinical trial, GATHER2, evaluating Zimura for the treatment of geographic atrophy and our expectations of our Phase IIb screening trial evaluating Zimura for the treatment of autosomal recessive Stargardt disease, our hypothesis regarding complement and the HtrA1 inhibition as a mechanism of action for the treatment of geographic atrophy and potentially other retinal diseases.

Our projected use of cash and cash balances, the timing, progress and results of clinical trials and other research and development activities and regulatory submissions, the potentiality and development potential of our product candidates, the size of the potential market for indications our product candidates are intended to treat and the potential of our business development strategy.

These statements constitute forward-looking statements for the purposes of the safe harbor provision under the Private Securities Litigation Reform Act of 1995. These statements cover many events and matters that are subject to various risks that could cause actual results to differ materially from those expressed in any forward-looking statements, including risks relating to the future progression of the COVID-19 pandemic and its impact on our research and development program, operations and financial position, initiation of the progress of research and development programs and clinical trials; availability of data from these programs; reliance on contract development and manufacturing organization, university collaborators and other third parties; establishment of manufacturing capabilities; expectations for regulatory matters; need for additional financing and negotiation and consummation of business development transactions and other risks.

I refer you to our SEC filings and in particular to the risk factors included in our current report on Form 8-K filed on June 17, 2020, for a detailed description of the risk factors affecting our business. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward-looking statements at some point in the future, we disclaim any obligation to do so, except as required by law.

I will now turn the call over to Glenn.

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Glenn P. Sblendorio, IVERIC bio, Inc. - CEO, President & Director [3]

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Thanks, Kathy, and good morning, everyone. We appreciate and thank you for joining our call this morning. First, I hope you and your families are safe and healthy as we continue to navigate through these very challenging times. Here at IVERIC, we are very pleased with the level of execution we have achieved during the second quarter. We are thrilled to have reached another major milestone with Zimura.

In June, we announced positive 18-month results from GATHER1. Our first Phase III clinical trial for Zimura, a novel complement C5 inhibitor for the treatment of geographic atrophy, or GA, secondary to age-related macular degeneration or AMD. The GATHER acronym for Zimura Phase III clinical trials represents GA therapy. The 18-month results from GATHER1 indicated continuous Zimura treatment benefit with a favorable safety profile in patients with GA secondary to AMD. We think this is an impressive achievement since we believe GATHER1 is currently the only Phase III clinical trial showing suppression of GA growth with continuous treatment for 18 months.

Shortly following the positive results from GATHER1, we initiated patient enrollment in GATHER2, our second Phase III clinical trial for Zimura for the treatment of GA secondary to AMD. Our main priority is to aggressively drive patient recruitment and retention in the GATHER2 clinical trial. If the primary endpoint is achieved at month 12, we intend to file for approval of Zimura with the U.S. Food and Drug Administration and the European Medicines Agency.

The initiation of patient enrollment in GATHER2 brings us another step closer to potentially delivering a clinical meaningful therapy safely to patients with GA, where there is currently no treatment. We continue to work closely with the FDA, and we are pleased to receive FDA fast-track designation for Zimura for the treatment of GA secondary to AMD. In addition to the complement systems' potential role in GA and Stargardt disease, we believe, based on scientific data, that Zimura may have potentially -- may have a potentially impactful role in treating intermediate AMD as well as wet AMD. We believe there is strong scientific rationale to support the development of Zimura in multiple forms of AMD early and advanced, dry and wet.

We believe HtrA1, or high-temperature requirement A serine peptidase protein 1, could be another important target in the treatment of AMD. We called this program IC-500. HtrA1 is a small molecule inhibitor that is a promising compound in our pipeline. HtrA1 up-regulation has been implicated as a strong risk factor in the development of dry AMD. We are particularly encouraged by IC-500's ability to engage the target, both extra and intracellularly in early preclinical work.

Based on current time lines, we are planning to submit an IND to the U.S. FDA for IC-500 in GA secondary to AMD in 2021. Although bringing to more, to patients is our top priority, we continue to focus on our gene therapy program in orphan inherited retinal diseases. Kourous will review the details of our gene therapy pipeline in a few moments.

Following the positive GATHER1 18-month data, we strengthened our balance sheet with an underwritten public offering and a concurrent private placement with Vivo Capital and Samsara BioCapital, raising approximately $160 million in gross proceeds. Dave will cover our cash runway later in this call. We believe this fundraising enables us to further execute on our strategy to develop and deliver retinal treatments through our Zimura -- through Zimura, our gene therapy programs and IC-500 with potential to create long-term shareholder value.

At the beginning of the second quarter, we welcomed Dr. Pravin Dugel, who many of you know, to IVERIC bio. Pravin is a globally recognized retinal specialist, who has an extensive network and long-standing relationships with the bio and pharma ophthalmic industry. As our Executive Vice President and Chief Strategy and Business Officer, Pravin's experience and network will be instrumental in helping us build alliances with potential future collaborators, investors and other stakeholders. Pravin is helping to lead the company's strategy as we advance our portfolio of therapeutics and gene therapy R&D programs targeting multiple retinal diseases.

In July, we had the privilege of announcing the addition of Dr. Mark Blumenkranz to our Board of Directors. Mark is a biotech industry leader and internationally known vitreoretinal specialist, with a notable expertise in pharmaceuticals for age-related macular degeneration and ocular gene therapy. Mark has cofounded multiple biotech and medical technology companies. His experience and expertise in leading and building biotech companies, this further strengthens our Board. I'd now like to turn the call over to Pravin.

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Pravin U. Dugel, IVERIC bio, Inc. - Executive VP and Chief Strategy & Business Officer [4]

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Good morning, everyone. I hope you're all well. Thank you, Glenn, for the kind introduction. And this is an exciting time at the company. And it is my great pleasure to work closely with Glenn, David, Kourous and all my new colleagues. We are very excited by these 18-month GATHER1 results. This is a significant milestone for Zimura and potentially a significant advancement for patients with GA secondary to AMD.

We believe the 18-month data that we reported this past June further validates our 12-month results regarding Zimura's continuous positive treatment effect with a favorable safety profile in GA secondary to AMD and highlights the potential role of complement C5 inhibition in this disease. In the GATHER1 clinical trial, Zimura met its prespecified primary efficacy endpoint at 12 months and reached statistical significance in the international multicenter randomized, double-masked, sham-controlled Phase III clinical trial in GA secondary to AMD.

The reduction in the mean rate of GA growth over 12 months was 27.38% with a p-value of 0.0072 for the Zimura 2-milligram group as compared to the corresponding sham control group and 27.81% with a p-value of 0.0051 for the Zimura 4-milligram group as compared to the corresponding sham control group. These data for both dose groups were statistically significant. These positive 12-month data are further supported by the 18-month results, which we reported in June.

Over 18 months, the reduction in the mean rate of GA growth was 28.11% for the Zimura 2-milligram group as compared to its corresponding sham group and 29.97% for the Zimura 4-milligram group as compared to its corresponding sham control group. The primary efficacy endpoint was prespecified at 12 months using all of the power to detect a statistically significant difference. Therefore, the p-values at the 12-month statistical analyses are descriptive in nature. The descriptive p-value for the treatment effects at month 12 were a p-value of 0.0014 for the Zimura 2-milligram group and a p-value of 0.0021 for the Zimura 4-milligram group.

We believe having 18-month positive data with continuous treatment is a key differentiating factor for us when compared to other product candidates being developed for GA. Another key differentiating factor for this trial is that the treatment effect was observed in the very first measurement at 6 months with an increasing absolute difference between the treated group and the sham at each subsequent measurement time point. In other words, if these results are replicated in GATHER2 trials, we believe a doctor would be able to tell his or her patient that this drug has been -- has shown an effect as early as 6 months and then may have an increasing effect with every subsequent injection thereafter.

We do not believe this impactful efficacy profile has been observed in any other GA clinical trial to date. Zimura's favorable safety profile, another potential differentiating factor was maintained throughout the 18-month trial with no investigator-reported Zimura-related adverse events, no cases of endophthalmitis and no Zimura-related inflammation. The reported incidence of CNV in the untreated fellow eye was 11 patients, 3.8%; and in the study eye was 3 patients, 2.7% in the sham control group; 2 patients, 7.7% in the Zimura 1 milligram group; 8 patients, 11.9% in the Zimura 2-milligram group; and 13 patients, 15.7% in the Zimura 4-milligram group.

Note, we believe these rates of CNV were lower than what has been published for C3 inhibition, despite the fact that there were more Zimura injections administered over a longer period of time in a patient population with faster progressing disease. This is particularly the case when you look at the ratios of the incidents in the treated arms versus sham. The most frequently reported ocular adverse events in GATHER1 were related to the injection procedure and not the drug.

These GATHER1 reports are from the pre-COVID era, making the trial what we believe is the only pure pre-COVID positive Phase III clinical trial in GA. We believe there is a new environment for clinical trial execution and that the robust data from GATHER1 may help drive recruitment and retention of patients in GATHER2 and any future trials we may conduct for Zimura.

In the challenging COVID-19 pandemic era, we believe that investigators will be more enthusiastic and comfortable recruiting and retaining patients in a clinical trial with a drug that already has high-quality positive Phase III data. We recognize the challenges that retinal physicians face with their practices and conducting clinical trials in the COVID era, and we're working closely to support collaborating physicians. Kourous will discuss how we plan to leverage the quality of the GATHER1 results to maximize patient recruitment and retention for GATHER2 Phase III clinical trial.

Turning to our business development. We plan to continue our aggressive, but selective efforts as we continue to explore our options for future development and potential commercialization of Zimura, including potential out-license and collaboration opportunities.

Thank you for your time. I will now turn the call over to Kourous.

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Kourous A. Rezaei, IVERIC bio, Inc. - Senior VP & Chief Medical Officer [5]

--------------------------------------------------------------------------------

Thank you, Pravin, and good morning, everyone. We are excited to have the robust Zimura GATHER1 results presented at the association for Research in Vision and Ophthalmology, Arvo Annual Meeting by Dr. Karl Csaky, T. Boone Pickens' Senior Scientist and Director of the Molecular Ophthalmology Laboratory at the Retina Foundation of the Southwest and also presented at the American Society of Retina Specialists, ASRS Annual Meeting run 2 weeks ago by Dr. Baruch Kuppermann, Chairman of the Department of Ophthalmology at the University of California Irvine.

In the coming months, we are planning for our data to be presented by key opinion leaders at the major retina meeting in the U.S. and around the world. The positive GATHER1 clinical trial data has ignited a significant enthusiasm in our investigators to participate and enroll patients in the GATHER2 clinical trial. Further, we believe that the early onset and continuous treatment effect demonstrated in the GATHER1 clinical trial will be a key motivator for retention in the GATHER2 trial.

Our experienced clinical trial team has worked tirelessly in the past few months to support the smooth and expeditious initiation of the GATHER2 clinical trial. Some of these efforts include hosting multiple virtual websites with investigators and study coordinators in the U.S. and around the world. Frequent communications with the clinical trial site coordinators to assess and support their preparedness for COVID-19 and minimizing any potential disruption.

Making arrangements for patients to remotely perform certain trial-related tasks to minimize the time spent by the patients at the clinical trial sites and to reduce their exposure risk and ensure a safe environment for our patients. We thank all our principal investigators and their staff for their enthusiasm and support for the GATHER2 clinical trial.

Now I would like to provide some details regarding the design of the GATHER2 trial. GATHER2 is an international randomized, double-masked, sham-controlled, multicenter Phase III clinical trial, evaluating the safety and efficacy of Zimura 2-milligram in patients with geographic atrophy secondary to age-related macular degeneration. We are planning to enroll approximately 400 patients in this clinical trial.

Patients will be randomized 1:1 into 2 cohorts. The first cohort receiving monthly administration of Zimura 2-milligram for 12 months and the second cohort receiving monthly administration of sham. The prespecified primary efficacy endpoint is the mean rate of change in geographic atrophy growth over 12 months, measured by fundus autofluorescence at 3 time points; baseline, month 6 and month 12, very similar to the GATHER1 clinical trial.

If the primary efficacy endpoint is met at month 12, we are planning to file for marketing approval of Zimura for the treatment of geographic atrophy, secondary to age-related macular degeneration with the FDA and EMA. At month 12, we plan to re-randomize patients in the Zimura 2-milligram arm to receive either monthly or every-other-month administrations of Zimura 2-milligram. The final safety evaluation will be performed at month 24 for all patients.

Turning now to Stargardt disease. We currently have an ongoing Phase IIb screening clinical trial assessing the safety and efficacy of Zimura in patients with autosomal recessive Stargardt disease. Initially, we enrolled 95 patients in this trial. We have reopened the enrollment in this trial to add approximately 25 patients with the goal of enrolling a total of 120 patients as was initially intended in the protocol for this trial. We remain masked for this trial and plan to perform data analysis when all the patients have reached the month-18 time points.

Regarding our gene therapy programs, we continue with our IND-enabling activities and natural history studies for IC-100, our product candidate for rhodopsin-mediated autosomal-dominant retinitis pigmentosa and are planning to initiate the Phase I/II clinical trial for the first half of 2021. We also continue with the IND-enabling activity and natural history studies for IC-200, our product candidate for BEST1-related retinal diseases and are planning to initiate the Phase I/II clinical trial next year in 2021.

Our minigene programs continue to move forward. We are currently optimizing the minigene construct for our miniCEP290 program and plan to select the leads construct later this year. Thank you for your time. And please stay safe. I will now turn the call over to Dave. Dave?

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David F. Carroll, IVERIC bio, Inc. - Senior VP, CFO & Treasurer [6]

--------------------------------------------------------------------------------

Thank you, Kourous, and good morning, everyone. I'd like to highlight a few items from our press release this morning and also update our year-end cash guidance and our expected cash runway.

For the quarter, our net loss totaled $18.6 million or $0.32 per share compared to a net loss of $14.4 million or $0.35 per share for Q2 2019. This increase in net loss was driven primarily by an increase in R&D expenses, offset by a favorable settlement of a state income tax audit. Year-to-date, our net loss totaled $33.7 million or $0.61 per share compared to a net loss of $26.9 million or $0.65 per share for the same period in 2019, again, due to an increase in R&D expenses offset by a favorable settlement of the state tax audits.

Turning to our expected year-end cash balance and cash runway. We raised approximately $150 million in our June 2020 public offering and concurrent private placement. We now expect our year-end cash balance will range between $215 million and $220 million. We estimate that our cash will be sufficient to fund our capital expenditures and operating expenses as currently planned for at least mid-2024, excluding any potential approval or sales milestones payable to the Archimex Corp. or any potential commercialization expenses towards Zimura.

These estimates are based on our current business plan, which includes the continuation of our clinical development programs for Zimura, the progression of IC-100 and 200 gene therapy programs into the clinic and the advancement of our IC-500 development program. Our estimates assume that we will enroll approximately 400 patients for the GATHER2 trial. If facts and circumstances change, we'll adjust our guidance accordingly. Of course, these estimates do not reflect any additional expenditures resulting from the potential in-licensing or acquisition of additional product candidates or technologies or any associated development that the company may pursue.

I'll now turn the call back over to Glenn. Thank you for your time.

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Glenn P. Sblendorio, IVERIC bio, Inc. - CEO, President & Director [7]

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Thanks, Dave, and I appreciate that. So just to recap the second quarter, despite these very challenging times, it was a good quarter for IVERIC bio.

There's a number of key takeaways: we completed our 18-month results for GATHER1. And as you've seen and we discussed today, with continuous effect and a good safety profile; second, we raised $160 million in gross proceeds, further strengthening our balance sheet and adding to our cash run normally; and third, although it was a difficult discussion back in March when we decided to pause the GATHER2 trial, we have now begun that trial and are recruiting patients.

So a good quarter for us. So I'd like to thank all of you for listening today to our call and for your continued support. And now I'll turn the call over to the operator so that we can open up the line for any questions.

================================================================================

Questions and Answers

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Operator [1]

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(Operator Instructions)

We will now take our first question from Stacy Ku of Cowen.

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Stacy Ku, Cowen and Company, LLC, Research Division - Equity Research Associate [2]

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Congratulations on the progress. So first question, given the time spent for sites in GATHER2 for Phase III and the recent trial initiation, how should we be thinking about the pace of enrollment? Would you be willing to give us some guidance on timing?

And my second question is around something that was briefly touched upon during your prepared remarks, but curious if you could elaborate what the team is thinking in terms of potential ex-U. S. partnerships. When would you ramp up these conversations?

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Glenn P. Sblendorio, IVERIC bio, Inc. - CEO, President & Director [3]

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Thank you, Stacy. It's Glenn, and thanks for the questions. First, on the timing for GATHER2, I mentioned a couple of things. It's early days and this is also a very competitive environment. And I think you've seen our execution in the past. So while we are dealing with a unique situation here with COVID, and I think as we've updated you through the second quarter, we've kept a very active and continuous dialogue with the investigators.

So at the current time, we're not going to provide any guidance as to when we finish the trial. So -- but we will continue to move at a pace that's as quick as we can. The key to that success will be the number of sites, the quality of the sites and obviously, the patient enrollment. So I am going to provide you detailed guidance at this point and the fact that we've got a pretty experienced team doing this.

As for the second question as it relates to collaborations. I think Pravin and his prepared statements did talk about that. Again, priority one for us today is the recruitment and retention of patients in GATHER2, but we are going to continue to explore our options for potential commercialization of Zimura, including a potential our-license. As you know, we do not have operations overseas even with the successful capital raise in a small company. So partnership at the right time will be an important part of our efforts to commercialize Zimura. Thanks for the calls -- thanks for the questions, Stacy.

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Operator [4]

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And we will now take our next question from David Nierengarten of Wedbush.

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David Matthew Nierengarten, Wedbush Securities Inc., Research Division - MD & Head of Healthcare of Equity Research [5]

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I'm sorry. I was on mute. Apologies. A couple of questions from me. First off, the...

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Glenn P. Sblendorio, IVERIC bio, Inc. - CEO, President & Director [6]

Originally posted here:
Edited Transcript of ISEE.OQ earnings conference call or presentation 5-Aug-20 12:00pm GMT - Yahoo Finance

By John Authers

The coronavirus pandemic has stress-tested the world. Beyond challenging human fortitude, national health services and international rivalries, it has forced a series of moral choices. Many have provoked impassioned disagreement over whether governments can force businesses and schools to close, over sacrifices for the sake of the elderly and, most bitterly and surprisingly, over whether being asked to wear a simple face mask infringes individual liberty.

The toughest moral test lies ahead. The biomedical industry and research facilities around the world are progressing toward creating a vaccine that would offer the best chance to end the pandemic and return life to normal. But the moral dilemmas provoked by the development and distribution of a vaccine will drive ever deeper debates.

The issues strike at profound divisions between schools of ethics. The newly published The Ethics ofPandemics,an anthology edited by philosophy professor Meredith Schwartz of Ryerson University in Toronto, presents contrasting views of academics, doctors and commentators along with a series of impossibly difficult case studies. The scientific, economic and political choices involve moral issues that have divided ethicists for centuries:

How to develop it?

The U.S government says the Covid-19 vaccine will be developed at warp speed. But vaccines take years to develop, for good reasons, and none of the benefits can be realized if they are released before they are safe. A failed Covid-19 vaccine could even compromise confidence in other vaccinations, threatening a return of measles, polio and other plagues.

Testing shortcuts are available but fraught. The first rule of deciding when theyre justified, explains Arthur Caplan, the head of bioethics at the NYU Langone hospital system in New York, is that risks can be balanced against the prospect of better data. Thus, skipping animal testing may pass muster since the data from testing humans is better.

That leads to the issue that divides teams at Moderna Inc. in Boston and at Oxford University in England who are working on the two most promising attempts to find a vaccine. How much risk of harming humans can they justifiably take? The best way to accelerate the process could fall afoul of the long-established obligations of medical ethics, from the Hippocratic oath to do no harm.

That pledge is as old as ancient Greece, it aligns with Christian teaching, and with the powerful school of rights-based philosophy identified with the 18th-century German philosopher Immanuel Kant, which holds that people should never treat humanity as a means to an end. Whatever the ultimate positive consequences, Kantians argue, there is no right to harm anyone. Virtuous ends do not justify unethical means.

Immanuel Kant

In human challenge trials,which have been used to test cholera and dengue vaccines, volunteers are injected with a vaccine and then deliberately infected with the germ that researchers are hoping to neutralize. The subjects are tightly monitored, and results are available within weeks. Researchers at Oxford are developing strains of the coronavirus in preparation for such a trial alongside a much larger conventional study, as are the National Institutes of Health in the U.S. Such a study will require 150 volunteers at the most.

Moderna opted against human challenge trials, and instead started a conventional trial with 30,000 test subjects in July. Volunteers are given either the vaccine or a placebo, and then go about their daily lives as the pandemic rages. Moderna hopes to have scientifically reliable results by the end of the year. Tal Zaks, Modernas chief medical officer, said he expects this approach to reveal how the vaccine behaves with different groups of people and in different regions. By testing in the real world, he said, results can be superior to the outcome of challenge tests, which are held in laboratory conditions.

But the conventional approach is slower, and leaves much to chance. Oxfords attempt to hold such a study in London and Oxford earlier this year came just as the epidemic was beginning to decline in the U.K., making it hard to draw firm conclusions. A rival research team at Imperial College, London, has the same problem and is looking to hold a trial in another country.

Further, doctors are morally obliged to tell volunteers how to avoid getting infected. They cannot tell them to go maskless, or to seek out crowded spaces, even though from a narrowly scientific point of view this would improve their test results. Its also impossible to monitor so many volunteers closely enough to determine if they are reporting their experiences inaccurately and skewing the results.

Rutgers University bioethicist Nir Eyal says that coronavirus challenge testing in the U.S could simultaneously maximize utility and respect rights. Researchers would use only informed, willing, low-risk volunteers from a population that is already in high-risk areas, he said.

Volunteers are abundant. An advocacy group called 1 Day Sooner has found 32,000 volunteers in 140 countries, mostlybetween the ages of 20 and 30, (old enough to consent but much less exposed to serious harm from Covid-19 than their elders) with no relevant underlying medical conditions. Strongly believing in effective altruism, Josh Morrison, who heads 1 Day Sooner, voluntarily donated one of his kidneys to a stranger, as did others helping with the campaign.

But Kantian objections are serious. Michael Rosenblatt, a Harvard Medical School professor and former chief medical officer of Merck Inc., objects that human challenge studies should only be contemplated when some lifesaving treatment, such as an antiviral medicine, is available for a candidate who gets sick. There is no such cure for Covid-19.

Then there is the problem of the unknown. Vaccines must pass muster with libertarians, descended from figures such as the enlightenment philosopher John Locke and the founding fathers of the U.S., who build morality around individual freedom. To counter libertarian objections, researchers must obtain informed consent.

Brazilian pediatric doctor Monica Levi, one of the volunteers who received the COVID-19 vaccine, works at the Specialized Clinic in Infectious and Parasitic Diseases and Immunizations (CEDIPI), in Sao Paulo, Brazil, on July 24, 2020.NELSON ALMEIDA/AFP via Getty Images

Rosenblatt argues that when it comes to Covid-19, Its pretty hard to have informed consent when we barely know anything about this yet. There are fears that the virus can cause lasting damage even in twentysomethings, for example, but little clear evidence. Can volunteers really consent to expose themselves to such poorly understood risks?

Finally, there is the appalling possibility of a volunteer dying. In 1999, this happened to Jesse Gelsinger, a healthy 18-year-old with a rare metabolic genetic disorder who volunteered for a conventional safety trial (not a challenge trial) of a virus-based gene therapy. His death was both a personal tragedy and a scientific disaster that set the field of gene therapy back by at least two decades, Rosenblatt said. That hiatus deprived a generation of patients with genetic disorders of treatments.

Morrison, of 1 Day Sooner, defends the right to volunteer for testing. Estimates at present are that the risk of death from Covid-19 for people in their 20s with no pre-existing conditions is under one in 10,000 less than the risk of dying in childbirth while soldiers (whether volunteer or conscripted) face a far higher chance of dying on the battlefield.

How to pay for it?

A vaccine is meaningless if people are unable to afford it, said John Young, the chief management officer of Pfizer Inc. Nobody asserts that drug companies should be able to charge whatever the market can bear for a Covid-19 vaccine.

But private companies like Pfizer have a responsibility to shareholders. Moreover, anyone who develops a successful coronavirus vaccine will have performed an immense service to humanity and will deserve to be rewarded. And so Pfizer defends its right to make a profit.

Pfizer has a $2 billion deal with the U.S. government to supply as many as 600 million doses of the vaccine it is developing. Many of its competitors are in collaborations with public universities, or receive state funding. That raises an intensely ideological issue: Should a private company be free to set prices for a public good developed with government aid?

We have to make a profit out of the first product, Moderna chief executive Stephane Bancel told Yahoo Finance. We have invested $2 billion of our shareholder capital since we started the company. We need to get a return. But Moderna has also received some $955 million in government funding to finance its big test. According to the Financial Times, Moderna is planning to price its vaccine at $25-$30 per dose, significantly above the $19.50 at which Pfizer is selling each of 100 million doses to the U.S..

Meanwhile, AstraZeneca PLC says it will sell the vaccine it is developing with Oxford to European governments at no profit, while Johnson & Johnson says it will sell its vaccine at a not-for-profit price for emergency use.

A man walks past a sign at an AstraZeneca site in Macclesfield, central England May 19, 2014.REUTERS/Phil Noble/File Photo

The issue is already very political. Five pharma industry leaders have had to testify on their pricing plans before a committee of the U.S. House of Representatives, and Democratic-sponsored bills are in Congress to stop price gouging. They have some Republican support.

Representative Lloyd Doggett, a Texas Democrat who is sponsoring one such bill, told Politico that a drug companys claim that its providing a vaccine at cost should be viewed with the same skepticism as that by a used car salesperson.

Once governments have bought the vaccine, should they require patients to pay for their own shots? Most people with money would happily pay much more than $30 to free themselves from the coronavirus. But in the many developed countries with nationalized health systems, the question doesnt arise: taxpayers pay, and the vaccine is free for patients.

The U.S., however, has a political issue on its hands. Senator Patty Murray, a Washington Democrat, now backs a bill to ensure that every American has a right to a free vaccine. Meanwhile, the deal with Pfizer will result in free immunizations. Having established the principle of taxpayer-paid vaccines, it could be hard to retreat.

These are issues decided within countries. When it comes to international cooperation, poorer countries complain about vaccine nationalism. In the U.K., Prime Minister Boris Johnson pulled out of the EUs so-called Inclusive Vaccines Alliance in a move attacked for playing to his Brexit-friendly political base.

Wealthy countries have little incentive to collaborate with poor ones. Costa Rica led an effort with the World Health Organization to set up a new Covid-19 Technology Access Pool that would share research and then coordinate production and also share the vaccine once it was ready.

But the list of countries that responded is telling. The U.S., China, Canada and Japan are all absent, while the only European countries to sign up have been Belgium, Luxembourg, the Netherlands and Norway. A group of much smaller developing nations has been left to build a collaboration even though the virus knows no boundaries, and it is in all countries interest to stamp it out everywhere.

The virus originally thought to only attack the lungs ravages almost every part of the body. Stock/Getty

Meanwhile, rich countries are prospectively buying up vaccines before they have even been cleared for use. The U.S.-Pfizer vaccine deal, and a similar deal with Glaxo PLC and Sanofi AG, uses American buying power to avoid excessive prices. Britain has done four separate deals with providers for 250 million doses.

What about the poorer countries who may have to pay more for the vaccine? For now, attempts at vaccine justice have been left to philanthropies such as the Gates Foundations Vaccine Network.

How to ration it?

The pharmaceutical industry cannot produce enough vaccine for the entire global population of almost 8 billion all at once. Therefore, rationing is inevitable. Some people will have to wait. Who gets to make these decisions, and by what criteria?

Within the U.S., various medical bodies and government agencies claim authority to draw up the guidelines. No one seems empowered to adjudicate.

The principle is to protect those most likely to be harmed, said Caplan of NYU Langone. That leads to one point of clarity: Medical workers go first. Theyre obviously at risk, and have a duty to put themselves in harms way.

But after this, following his criterion leads to prioritizing some of the least privileged in society not because they are underprivileged and deserve help, but because they are most at risk.

Statistically, prisoners follow doctors and nurses on the list of people most likely to be harmed. As prisons are Covid-19 incubators, Caplan suggests that vaccinating inmates would limit the diseases spread.

Calgarians wear masks as they walk along Stephen Avenue Mall in downtown Calgary on Wednesday, July 29, 2020.Gavin Young/Postmedia

Within the U.S., Native-American communities are grievously affected, and therefore have a case for priority. The same is true of some other ethnic minorities, largely because they tend to live in crowded communities, and because higher rates of poverty make them more likely to suffer the underlying conditions that make Covid-19 more deadly.

People are also more at risk if they cannot work from home. In an email, Anthony Skelton, a philosophy professor at the University of Western Ontario and Lisa Forsberg of the Oxford Uehiro Centre for Practical Ethics, make a case for sending those in work-at-home professions to the back of the line. To the extent that racial minorities might live and/or work in conditions that make them less able to avoid coming into contact with infected individuals, the case for giving them priority over people who can work from their home office seems strong, the scholars wrote.

All of these proposals spring from prioritizing people according to risk, but might in practice look like the kind of redistributionist social-justice crusading that provokes controversy, particularly in the U.S.

Rationing could also be affected by where the vaccine was tested. In the case of AIDS, experimental treatments were assessed in Africa, where testing was cheaper, but the treatments then went to developed countries. Severely affected African countries had to pay prohibitive prices as the disease took hold.

Africa could become a Covid-19 test site if regulators do not permit human challenge tests elsewhere. If large-scale testing does happen there, justice will demand that early supplies of the vaccine are made available to Africans, even at the expense of people in the researchers home country.

How to roll it out?

Vaccinations work best when everyone receives them, since germs that cant infect people tend to wither away.

But all vaccines come with risks. That creates a free-rider problem. The best option from a self-interested point of view is that everybody else has the shot (eliminating your personal risk of catching Covid-19) but that you dont (avoiding any personal risk of side-effects). Taxes have the same problem. Taxes are compulsory. Does that mean vaccination should be compulsory, too?

The public-health case for compulsion is strong. But libertarians have a problem with forcing a potentially harmful vaccine on someone without the informed consent thats hard to procure in societies skeptical of experts and low on social trust.

How can the vaccine reach a critical mass without compulsion? Caplan suggests leaving compulsion to private entities. An employer might demand vaccination as a condition of reporting for work. A university might impose the same requirement on faculty and students. A vaccine might be dangled as a golden ticket to return to theaters, cinemas, night clubs or sports events. Governments or foundations could even pay people to receive a shot.

By this thinking, those who assert their right not to be vaccinated would be free to work from home and home-school. They would be voluntarily narrowing their own freedom of movement and assembly.

Yet societies would pay a price. The virus has divided humans in countless ways already. If many citizens opt to stay unvaccinated, the virus and the messy ethics of compelling vaccination will have helped to create another permanent division.

This column does not necessarily reflect the opinion of the editorial board or Bloomberg LP and its owners.

John Authers is a senior editor for markets. Before Bloomberg, he spent 29 years with the Financial Times, where he was head of the Lex Column and chief markets commentator. He is the author of The Fearful Rise of Markets and other books.

2020 Bloomberg L.P.

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Opinion: With any COVID-19 vaccine, humanity will face its biggest moral test - The Province

Victoria Gray, a 34-year-old mother of four in Forest, Miss., had been going to the hospital seven times a year for transfusions to help with the severe pain brought on by her sickle cell disease. In July 2019, she volunteered for a radical new technology known as gene editing. She has been free of the pain and suffering since.

I chose to participate in this trial because of hopehope that it would change my life, Gray tells Barrons. And it has already in so many ways.

Her successful treatment gives hope to the 100,000 other Americans who suffer from sickle cell. It also illuminates the enormous potential for the companies that are pioneering genetic medicine to cure diseases with a one-time treatment.

Sickle cell is among their first targets. The companies include the sponsors of Victoria Grays clinical trial Crispr Therapeutics (ticker: CRSP) and Vertex Pharmaceuticals (VRTX)as well as biotechs like bluebird bio (BLUE), whose gene therapy for sickle cell is further along in testing and could be introduced by 2022.

For these companies, genetic therapies for sickle cell could fetch annual revenue of several billion dollars apiece. A recent Bank of America Securities report predicts that new treatments for sickle cell will surpass $6 billion in sales by 2028. That is meaningful for a company of any size, but particularly so for these biotechs. Crispr doesnt yet have sales. Bluebirds revenue last year was $45 million, and Vertexs, $4 billion.

More important, the sickle cell therapies will demonstrate how such genetic technology could open the door to curing dozens of other diseases.

Investors seem persuaded. They have lifted Crispr stock threefold since March to a recent high of $97, which values the development-stage company above $6 billion. Hitting a record high of $306, Vertexs value has topped $75 billion, while bluebirdwhose investors have waited nearly a decade for their paydaytrades at a more modest market cap of $4.2 billion. Biotech valuations may be hard to rationalize these days, but that reflects the radical changes that new technologies are bringing to the health-care business. One need look only at this years fourfold rise of the messenger RNA vaccine leader Moderna (MRNA).

The focus on sickle cell is a turnaround for the pharmaceutical industry, which had long ignored the inherited disorder. Sickle cell mainly afflicts Black Americans and residents of poor African nations. It is the most commonly diagnosed genetic disorder among newborn Americans. Yet it has not had as much funding as some less-common inherited conditions. Cystic fibrosis, for example, affects one-third as many Americans, but researchers at Duke University have shown that it has historically received more than seven times the federal and foundation research funding per patient.

This year, two new sickle cell drugs came on the market. And sometime next year, bluebird will ask the U.S. Food and Drug Administration to approve its sickle cell gene therapy. Crispr and Vertex hope that they will not be not far behind.

The starting prices of the new sickle cell treatments are expected to range from $100,000 to over $1 million. It will be a challenge to make them accessible to the millions of people in poor countries who have sickle cell.

The disease is caused by a single variation in a gene for hemoglobin, the protein that carries oxygen in our red blood cells. The genetic trait is prevalent among those whose descent traces to sub-Saharan Africa, because a single copy of the sickle cell gene protects you from developing malaria. Inherit a copy from each parent, however, and you become one of 300,000 babies born in the world each year with sickle cell disease. It causes hemoglobin molecules to form long chains that warp red blood cells into sickle shapes that get stuck in blood vessels and block the flow to vital organs.

The result is terrible pain, organ damage, infections, andwhen left untreateddeath before age 5, on average. In the U.S., where the disease occurs in one of every 365 Black babies, available treatments still leave many with a life of pain, disability, and death before age 50.

The first sickle cell treatment approved by the U.S. Food and Drug Administration was hydroxyurea, in 1998. For the half of sickle cell patients with moderate disease, treatment with hydroxyurea, antibiotics, and transfusions can allow productive lives. Hydroxyurea is off-patent and costs less than a dollar a day. But even that price is beyond reach of those who live in resource-poor countries, says Russell Ware, a pediatric hematology professor at the University of Cincinnati College of Medicine. Ware is working with medical colleagues in Uganda to get more children there on hydroxyurea.

Data as of 7/22/20

Bloomberg; company filings

The beauty is that its off-patent, says Ware, but its also a curse, because no one can make money off it.

Novartis (NVS) is supporting hydroxyurea availability in collaboration with the government of Ghana. The Swiss drug giant is also planning clinical trials in Ghana and Kenya for Adakveo, a monoclonal antibody approved by the FDA last November as one of the first novel treatments in decades for sickle cell disease. Adakveo dampens the inflammatory process that makes sickle cells clog blood vessels in cases that send some 50,000 Americans to the emergency room every year.

The intravenous drugs initial sales in the years first half were $36 million, but analysts hope that by the middle of this decade, Adakveos annual sales could reach $1 billion to $2 billion in the U.S. and Europe.

Just days after approving the Novartis drug, the FDA approved another sickle cell drug called Oxbryta, from Global Blood Therapeutics (GBT). Oxbryta is a pill that prevents defective hemoglobin from forming chains within red blood cells. The product is GBTs first, and it produced $14 million in sales for the March quarter, with a loss of $73 million.

But analysts like Yatin Suneja of Guggenheim Securities say that Oxbryta can hit sales of $1.7 billion in a few years, yielding some $13 a share in earnings for GBT. That makes Suneja think that GBT stock can rise from its current level of $72 to $115.

Successful sickle cell treatments could lift a number of biotech companies.

GBT CEO Ted Love says that Oxbrytas benign safety profile gives the company confidence that it will win approval to market the product for children, and in higher doses for adults. If other sickle cell treatments in the companys pipeline pan out, he imagines that sickle cell might one day become as well managed a disease as HIV.

Love is aware of the potentially curative gene therapies being tested by others, but he thinks that patients with milder cases of sickle cell might say, Just give me a pill.

For several decades, it has been possible to cure sickle cell with a bone-marrow transplant from a related donor. But the scarcity of matched donors and the risk of serious immune reactions have limited the number of such procedures to about 1,000. Recent breakthroughs in genetic technology promise to overcome those limitations by extracting a patients own cells, manipulating the cells genetic code, and then replacing the patients marrow with the amended cells.

The Cambridge, Mass.based bluebird bio is already in Phase 3 clinical trials of its beti-cel gene therapy for another inherited disorder of red blood cells called beta-thalassemia. Less common than sickle cell, thalassemia leaves patients with so few red blood cells that they can need more than a dozen transfusions a year. At a recent online gathering of hematologists, bluebird said that 60 children and adults with thalassemia had gone through its beti-cel procedure. About 90% had gone a year without needing a single transfusion.

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With a similar treatment for sickle cell, which bluebird calls LentiGlobin, more than two dozen patients have been infused with their own modified cells. Among the 14 patients who were six months past their treatment, there has been a 99.5% decrease in the blood vessel jam-ups that the patients previously suffered.

That is fundamentally transformative, says bluebird CEO Nick Leschly, and far exceeds any and all expectations weve ever had, any of our investigators have ever had, or the patients that have been treated.

These potentially curative cell therapies are complex procedures that are expected to be priced at about $1 million a patient. The thalassemia treatment is already approved in Europe. After the patients in bluebirds U.S. trials for thalassemia and sickle cell have been followed up for 18 months, the company will seek FDA approvalhopefully next year.

RBC Capital Markets believes that the companys sickle cell treatment could reach sales of $2 billion a year and help lift bluebird stock from its recent price of $66 to $100.

It took bluebird a decade to get to this point, and its shares have sunk as low as $17 and soared as high as $236, as investors reacted to the companys dramatic successes against refractory cancers. With Buy recommendations all along Wall Street, bluebird will get the capital it needs to cross the finish line.

Hard on the heels of bluebird are companies that believe they have better genetic treatments. One of them is Crispr Therapeutics. The company takes its name from CRISPR, shorthand for a Nobel Prizewinning technology that homes in on a targeted stretch of DNA and snips the double-stranded molecular code with a kind of chemical scissors.

The way I describe it to my patients, says patient Grays doctor Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville, is that you have a book with 500 pages of thousands of words, and we are finding one word and correcting it.

Crispr Therapeutics CEO Sam Kulkarni says that CRISPR technology should provide more durable and uniform results than earlier generation treatments like bluebirds. When Crispr reported Grays healthy progress last month, it said that a second patient with sickle cell has also been treated, as well as five patients with thalassemia. Kulkarni hopes to reach the market with his treatments not too far behind bluebird.

This years surge in Crispr Therapeutics stock allowed it to raise about $450 million in a June stock offering. Jefferies analyst Maury Raycroft has estimated that a successful sickle cell treatment could contribute a third of the $5 billion in annual revenue he projects for Crispr by 2030, with earnings above $30 a share. The stock has blasted through his last price target of $82.

Behind Crispr are still other gene-editing companies working on treatments for thalassemia and sickle cell, including Sangamo Therapeutics (SGMO), Editas Medicine (EDIT), and Intellia Therapeutics (NTLA).

One of this years most successful initial public offerings was the February debut of Beam Therapeutics (BEAM), which is in preclinical testing of sickle and thalassemia treatments that would use a next-generation editing technology that CEO John Evans believes will be even more effective than CRISPR.

If these other companies sickle cell treatments pan out, they may be scrapping for market share after bluebird and Crispr have established products. Still, as the pharmaceutical giants shop for gene-editing know-how, there may be buyouts.

Sickle cell has been a long-neglected illness, but many companies are now competing with treatments that each hopes will be best-in-class. This should be a dogfight, says bluebird CEO Leschly, because thats in the interest of patients.

Write to Bill Alpert at william.alpert@barrons.com

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These Companies Are Seeking a Cure for Sickle Cell. And Its Just the Beginning for Some Gene Therapy Stocks. - Barron's

Officials with the FDA have approved atezolizumab (Tecentriq, Genentech) plus cobimetinib (Cotellic, Genentech and Exelixis) and vemurafenib (Zelboraf, Genentech and Daiichi Sankyo) for the treatment of patients with BRAF V600 mutation-positive advanced melanoma. The safety profile observed in the atezolizumab combination was consistent with the known safety profiles of the individual medicines, according to Genentech.

Atezolizumab is a prescription monoclonal antibody designed to bind with a protein, PD-L1, expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. The therapy is currently utilized for treatment of patients with certain types of cancer, including urothelial carcinoma, non-small cell lung cancer, triple-negative breast cancer, and hepatocellular carcinoma.

The approval of the atezolizumab combination for BRAF V600 mutation-positive advanced melanoma is based on results from the Phase III IMspire150 study, a multi-center, double-blind, placebo-controlled randomized study in individuals with previously untreated BRAF V600 mutation-positive metastatic or unresectable locally advanced melanoma. The study results showed the addition of atezolizumab to cobimetinib and vemurafenib helped individuals live longer without their disease worsening or death (progression-free survival, PFS), compared to placebo plus cobimetinib and vemurafenib (median PFS 15.1 months versus 10.6 months respectively; hazard ratio, HR=0.78; confidence interval: 0.63-0.97; P=0.025).

The most common adverse reactions (rate 20%) in patients who received atezolizumab with cobimetinib and vemurafenib were rash (75%), musculoskeletal pain (62%), fatigue (51%), hepatotoxicity (50%), pyrexia (49%), nausea (30%), pruritus (26%), edema (26%), stomatitis (23%), hypothyroidism (22%), and photosensitivity reaction (21%).

The supplemental Biologics License Application or atezolizumab was granted under Priority Review. The review was also conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence that provides a framework for concurrent submission and review of oncology products among international partners.

According to Genentech, the company has an extensive development program for atezolizumab, including multiple ongoing and planned phase 3 studies across lung, genitourinary, skin, breast, gastrointestinal, gynecological, and head and neck cancers. This includes studies evaluating atezolizumab both alone and in combination with other medicines.

For those who qualify, Genentech will offer patient assistance programs for patients prescribed atezolizumab with cobimetinib and vemurafenib by their doctor through Genentech Access Solutions.

REFERENCE

FDA Approves Genentechs Tecentriq plus Cotellic and Zelboraf for People With Advanced Melanoma [news release]. South San Francisco, CA; July 30, 2020: Genentech website. https://www.gene.com/media/press-releases/14868/2020-07-30/fda-approves-genentechs-tecentriq-plus-c Accessed July 30, 2020.

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FDA Approves Combination Therapy for Treating Advanced Melanoma - Pharmacy Times

As the COVID-19 pandemic continues unfolding, technology solutions and government initiatives are multiplying to help monitor and control the viruss journey. Their aid includes reducing the load on the health system and reinforcing the efforts of overworking and burned-out healthcare workers.

While smart technologies cannot replace or compensate public institution measures, they do play a crucial role in emergency responses. Lets take a look at the promising use cases of how technology can help fight the novel coronavirus outbreak.

People tend to think of technology as a heartless machine, which is true, but only until its used for good. Just look at all the wonderful things weve managed to do with its help.

Telemedicine is gaining traction by offering remote patient monitoring and interactive remote doctors visits. At the same time, 3D printing and open-source solutions are facilitating the production of more affordable face masks, ventilators, and breathing filters as well as optimizing the supply of the medical equipment. Even more, the pandemic has driven scientists to desperate measures. They are now experimenting with gene editing, synthetic biology, and nanotechnology to develop and test vaccines faster than ever in the history of humanity.

Smart technologies like the Internet of things (IoT), big data, and artificial intelligence (AI) are being massively adopted to help track the disease spread and contagion, manage insurance payments, uphold medical supply chains, and enforce restrictive measures. Lets go step by step to see how IoT, AI, big data, and mobile solutions are actually enhancing medical care.

IoT has already found its use among healthcare providers. Today, connected patient imaging, health devices or applications, worker solutions, and ambulance programs are being adopted globally. But COVID-19 made the technology take on new applications to help the world combat the epidemic. Tracking quarantine, pre-screening and diagnosing, cleaning and disinfecting, innovative usage of drones, reducing in-home infections, are all new normals thanks to IoT.

For example, an American health technology companyKinsacreates smart thermometers that screen and aggregate peoples temperature and symptoms data in real-time. Having gathered data from over one million connected thermometers, Kinsa rolled out itsUS HealthWeather Map.

The map is updated daily, highlighting how severely the population is being affected by influenza-like illness (ILI). This real-time information helps health authorities see an increase. In fevers as early indicators of the community spread of COVID-19 to streamline the allocation of health resources. These areas are marked in the Atypical mode of the map.

To slow down the spread of COVID-19, a team of Seattle engineers createdImmutouch, a smart wristband vibrating every time a person wearing it tries to touch their face.

Smart speakers, lights, and security systems are being used to open doors and switch on lights to reduce in-home infections. These gadgets allow people to avoid touching the surfaces of doorknobs, switches, mail, packages, or anything that could easily spread germs.

Tapping into big data is a must to develop real-time forecasts and arm healthcare professionals with a profound database to help with decision-making.

IBMClinical Development system is an advanced Electronic Data Capture (EDC) platform that allows an accelerated delivery of medications to market and reduces the time and cost of clinical trials thanks to cognitive computing, patient data assets, and IoT. Additionally, the U.S. government had been in active talks with Facebook, Google, and others to determine how to use location data to glean insights for combating the COVID-19 pandemic.

The COVID-19 pandemic has become a game-changer for the healthcare continuum. Todays mobile apps are on guard to help patients receive online therapy, at-home testing, conclude self-checks, and improve mental well-being. Thanks to smartphone apps, it is now possible to trace the viruss journey and help limit its spread.

Apple COVID-19, for instance, was created in partnership with the Centers for Disease Control and Prevention (CDC), the White House, and the Federal Emergency Management Agency (FEMA). The application contains vital and relevant information from trusted sources on the coronavirus pandemic: hand hygiene practices, social distancing FAQs, quarantine guidelines, self-checking tutorials, tips on cleaning, and disinfecting surfaces. On top of that, it has a screening tool that advises people on what to do when a person has COVID-19 symptoms, has just returned from abroad, or has come in close contact with someone who might be infected with the disease.

Meanwhile, health authorities in Abu Dhabi have created theTraceCovidapp for Bluetooth-enabled smartphones to minimize the spread of the disease. The service allows tracing individuals who have come into proximity with a person tested positive for COVID-19. Thanks to it, medical professionals an react faster and render the necessary healthcare. Germany, in turn, is going to roll outa smartphone app, which will use Bluetooth to alert people if they are close to someone with the confirmed viral infection.

Telemedicine has also proved to be an efficient tool for flattening the curve.The Sheba Medical Centre, the largest Israeli hospital, launched a telehealth program for remote patient-monitoring to control the pandemic spread. Doctolib, a Franco-German company, Qare (France), Livi (Sweden), Push Doctor (the UK), Compugroup Medical (Germany) are offeringvirtual doctorstoo.

Artificial intelligence-powered by natural language processing (NLP) and location monitoring is crucial for identifying, tracking, and scanning outbreaks, predicting hotspots and helping make better decisions.

For example, Microsoft collaborated with the U.S. Centers for Disease Control and Prevention (CDC) to create an AI-basedCOVID-19 Assessment botto treat patients more effectively and allocate limited resources. The bot, nicknamed Clara, can evaluate symptoms, advice on the next steps to take and track users who need urgent care the most.

The Canadian startup BlueDot has applied AI to spot and track the spread of COVID-19 and predict outbreaks, and the Japanese company Bespoke rolled outBebot, an AI-powered chatbot that was developed specifically for travelers. This mobile app informs and assists them with coronavirus-related questions as they move about.

Theres no doubt that the coronavirus pandemic has become a real-life test for everyone. It has caused tremendous damage, but at the same time, it has forced tech innovators to roll out advanced solutions, and it seems that they dont plan on slowing down anytime soon.

Healthcare providers across the globe are continually switching to smart technologies. So if you are in the smart technology niche, consider the current trends to steer your business in the right direction.

Link:
How Can Technology Help Fight the COVID-19 Pandemic? - IoT For All

At its virtual commencement ceremony Saturday for the Class of 2020, Washington & Jefferson College will be presenting an honorary Doctor of Science degree to Mark L. Tykocinski, a medical doctor at Thomas Jefferson University in Philadelphia.

Tykocinski is the dean of the universitys Sidney Kimmel Medical College, one of the oldest in the nation. He has been in that role since 2008. Tykocinski is also the provost and executive vice president of academic affairs at the university. Before working at Thomas Jefferson University, Tykocinski spent a decade as a professor and chair of the Department of Pathology and Laboratory Medicine at the University of Pennsylvania. His research interests have been in the fields of cellular and molecular immunology, cell surface engineering and biologics innovation.

Tykocinski has contributed to the development of novel cell therapy strategies and the engineering of a novel class of mammalian gene expression vectors that have been distributed around the world. He holds research patents in the fields of molecular and cellular immunology, and is a fellow with the National Academy of Inventors. He received degrees from Yale and New York universities.

The college that Tykocinski oversees is closely linked to W&J. The Class of 2020 at W&J includes the first students to receive degrees after completing a year of study at the medical school. This is happening thanks to a partnership W&J struck up with the Sidney Kimmel Medical College that allows W&J students with majors outside the natural sciences to start studying at Sidney Kimmel after their junior year at W&J. Thomas Jefferson University started the partnership as part of the universitys efforts to bring more liberal arts students into their medical college.

W&J had hoped to hold an in-person commencement ceremony Saturday, but changed it to an online event due to rising COVID-19 numbers in the region. The college is hoping to hold an in-person reunion for members of the Class of 2020 at some point, perhaps as soon as next year.

Read more:
Thomas Jefferson University doctor to be honored at W&J commencement - Observer-Reporter

Today's early morning highlights from the major news organizations.

Kaiser Health News:Its About Love And Solidarity: Mutual Aid Unites NYC Neighbors Facing COVIDNancy Perez, a 45-year-old resident of the Brooklyn neighborhood of Bedford-Stuyvesant, contracted COVID-19 in March. She stayed quarantined in her room for a month to isolate from her two sons and grandson. A few days before she got the virus, shed met a volunteer with Bed-Stuy Strong one of the many mutual aid groups around the country that have rallied to provide help in the face of the pandemic. Bed-Stuy Strong assembled an army of volunteers to help vulnerable neighbors with food deliveries and basic supplies. While Perez was in isolation, volunteers regularly delivered cooked food for her sons, ages 17 and 20, and her 4-year-old grandson. (Lawrence, 7/27)

Kaiser Health News:The Color Of COVID: Will Vaccine Trials Reflect Americas Diversity?When U.S. scientists launch the first large-scale clinical trials for COVID-19 vaccines this summer, Antonio Cisneros wants to make sure people like him are included. Cisneros, who is 34 and Hispanic, is part of the first wave of an expected 1.5 million volunteers willing to get the shots to help determine whether leading vaccine candidates can thwart the virus that sparked a deadly pandemic. If I am asked to participate, I will, said Cisneros, a Los Angeles cinematographer who has signed up for two large vaccine trial registries. It seems part of our duty. (JoNel Aleccia, 7/27)

Kaiser Health News:Employers Require COVID Liability Waivers As Conflict Mounts Over Workplace SafetyAfter spending a May day preparing her classroom to reopen for preschoolers, Ana Aguilar was informed that the tots would not have to wear face masks when they came back. Whats more, she had to sign a form agreeing not to sue the school if she caught COVID-19 or suffered any injury from it while working there. Other teachers signed the form distributed by the Montessori Schools of Irvine, but Aguilar said she felt uncomfortable, although it stipulated that staff members would be masked. At 23, she has a compromised immune system and was also worried that she could pass the coronavirus on to her fianc and other family members. (Meyer, 7/27)

Kaiser Health News:Last Thing Patients Need During Pandemic: Being Last To Know A Doctor Left NetworkAs the coronavirus spread silently through New York City early this year, Deborah Koeppel had an appointment with her cardiologist and two visits with her primary care doctor. Both physicians are members of Concorde Medical Group, a practice in Manhattan with an office conveniently located a few blocks from where Koeppel works. She soon received notices telling her after the fact that those doctors were not in her health plans network of providers. According to the notices, she was on the hook for $849 in out-of-network cost sharing for three visits, which typically would cost her nothing from in-network providers. (Andrews, 7/27)

NPR:Florida Case Count Surpasses That Of New York, The Country's Original EpicenterFlorida has recorded more coronavirus cases than New York. Only California, the most populous state in the country, has more. As of Sunday afternoon, data from Johns Hopkins University shows 423,855 people in Florida have tested positive for the coronavirus, compared to 411,736 in New York. California leads with 450,242 cases. (Treisman, 7/26)

Tampa Bay Times:Florida Coronavirus Cases Show Little Sign Of Slowing As State Surpasses New YorkThe number of infections and deaths tied to the novel coronavirus in Florida showed little sign of slowing Sunday as the state surpassed New York for the second-highest number of confirmed cases in the United States. Only California, with a population nearly twice as high as Floridas, has more cases. Floridas Department of Health reported 9,344 infections and 78 fatalities. The overall caseload is 423,855 since March 1, and the number of deaths tied to the virus is 5,972. (Dawson, 7/26)

The Hill:US Surpasses 1,000 COVID-19 Deaths For Fourth Straight DayThe U.S. tallied over 1,000 coronavirus-related deaths Friday for the fourth straight day this week, yet another sign of the alarming spike in COVID-19 cases across the country.There were 1,178 new deaths Friday alone, according to the COVID Tracking project, compared with 1,038 Tuesday, 1,117 Wednesday, and 1,039 Thursday. Over 137,000 people have died in the U.S. and over 4 million people have contracted the virus in the country since the outbreak began. (Axelrod, 7/25)

NPR:U.S. Coronavirus Hot Spots: Mid-Atlantic And Northeast Could BackslideFor weeks the U.S. coronavirus pandemic has largely been driven by spiraling outbreaks in the South and West. But some forecasters say Mid-Atlantic and Northeast states could soon be in deep trouble again, too. The warning comes from researchers at the PolicyLab at Children's Hospital of Philadelphia, which has built a model to provide four-week forecasts for every U.S. county. NPR spoke to David Rubin, PolicyLab's director, an epidemiologist and professor at the University of Pennsylvania. (Aizenman, 7/24)

Stat:Trump Unveils Four Executive Orders Aimed At Lowering Drug PricesThe Trump administration unveiled four executive orders on Friday aimed at bringing down pharmaceutical prices, a last-ditch effort by the White House to cut drug costs before the November election. It remains unclear whether the Trump administration is capable of finalizing many of the actions by Election Day and whether it intends to do so. (Florko and Facher, 7/24)

Politico:Trump Signs Limited Drug Pricing Orders After Last-Minute Debate"The four orders I'm signing today will completely restructure the prescription drug market," Trump said in a speech, hearkening back to his 2016 campaign promise to slash costs. But the ambitious plans are rife with limitations. The rebate order comes with a caveat that any plan cannot increase seniors' premiums, the unworkable problem that led the adminstration to kill its original rebate rule last year. (Owermohle, Cancryn and Luthi, 7/24)

Politico:Trumps Talking Health Care Again, With 2020 In MindPresident Donald Trump is suddenly talking about health care again. He signed several executive orders on drug pricing on Friday. He vowed to unveil some new health plan by the end of next week, although he hasnt provided specifics or an explanation of how hell do it. His aides are touting a speech in which Trump will lay out his health care vision. White House counselor to the president Kellyanne Conway has been calling Trump the health care president. (McGraw and Ehley, 7/26)

AP:AP-NORC Poll: US Course At Record Low, Trump Sinks On VirusA new poll from The Associated Press-NORC Center for Public Affairs Research also finds Trumps approval for his handling of the COVID-19 pandemic falling to a new low, with just 32% of Americans supportive of his approach. ... Even as he tries to refocus his contest with Biden on divisive cultural issues and an ominous law and order message, Trumps reelection prospects are likely to be inextricably linked to his handling of the pandemic and whether voters believe the country will head back in the right direction under his leadership. The AP-NORC poll makes clear the challenge ahead for Trump on that front: 8 in 10 Americans say the country is heading in the wrong direction. (Pace and Fingerhut, 7/26)

Politico:Make America Normal Again: Trump Backers Plead For A Virus PlanPresident Donald Trump restarted the White House coronavirus briefings. He urged Americans to wear masks. He even scrapped his partys convention. To many of his own allies, its still not enough. Trumps political allies, alarmed by his sinking poll numbers, are warning that the presidents best chance to get reelected is to outline more detailed plans to conquer the coronavirus he keeps trying to wish away. They are advising him to offer people something concrete they can look to as the pandemic surges in dozens of states, eroding months of progress. (Kumar, 7/24)

The Wall Street Journal:GOP To Propose Aid Bill, With Extra Jobless Benefits Set To ExpireAfter days of disagreements between the White House and GOP lawmakers, Republicans are set to release their proposal for the next coronavirus relief bill on Monday, with millions of Americans on the verge of losing expanded unemployment benefits.Lawmakers now have little time before the $600 weekly supplement to jobless benefits ends. In negotiations with Democrats, three months before the election, an agreement on unemployment insurance might prove to be the most difficult to reach. (Ballhaus and Duehren, 7/26)

The Hill:White House, Senate GOP Race To Finalize Coronavirus Package Ahead Of Monday RolloutThe White House and Senate Republicans are workingto finalize a coronavirus relief package ahead of a Monday rollout. Treasury Secretary Steven Mnuchin and White House chief of staff Mark Meadows were back in the Capitol on Sunday for a second day of meetings with GOP staff as they work to lock down the forthcoming proposal. As he left the Capitol, Meadows told reporters that they had"been working through just some of the technical language" and had reached "an agreement in principle." (Carney, 7/26)

The Hill:Mnuchin: It 'Wouldn't Be Fair To Use Taxpayer Dollars To Pay More People To Sit Home'Treasury Secretary Steven Mnuchin took a hard line Sunday against the $600 increase in unemployment benefits that was a part of the last coronavirus relief measure, saying, It just wouldnt be fair to use taxpayer dollars to pay more people to sit home than they would working and get a job. GOP lawmakers have taken a hard line against theenhancement as they negotiate with the White House over a new relief measure. The initial bill won blowback from Republicans who said some people would make more money not working than going to work. (Budryk, 7/26)

The New York Times:Trump Officials Float Idea Of Narrow Bill To Extend Unemployment BenefitsTop Trump administration officials proposed on Sunday potentially short circuiting free-ranging stimulus talks with Democrats to rush through a much narrower bill prioritizing an extension of federal unemployment benefits that are set to expire this week for millions of Americans. Mark Meadows, the White House chief of staff, said he would now like to see lawmakers act this week to extend and alter the unemployment program, give tax credits to businesses to help ease reopening costs and grant employers new liability protections while setting aside a long list of other objectives, including Democrats priorities. (Fandos and Cochrane, 7/26)

AP:White House Pushes Narrow Virus Aid; Pelosi Blasts GOP DelayHouse Speaker Nancy Pelosi on Sunday assailed Republican disarray over a new pandemic relief package as the White House suggested a narrower effort might be necessary, at least for now. The California Democrat panned the Trump administrations desire to trim an expiring temporary federal unemployment benefit from $600 weekly to about 70% of pre-pandemic wages. The reason we had $600 was its simplicity, she said from the Capitol. (Mascaro and Superville, 7/26)

The New York Times:Fires And Pepper Spray In Seattle As Police Protests Widen Across U.S.Weeks of violent clashes between federal agents and protesters in Portland, Ore., galvanized thousands of people to march through the streets of American cities on Saturday, injecting new life into protests that had largely waned in recent weeks. One of the most intense protests was in Seattle, where a day of demonstrations focused on police violence left a trail of broken windows and people flushing pepper spray from their eyes. At least 45 protesters had been arrested as of early evening, and both protesters and police officers suffered injuries. (Baker and Bogel-Burroughs, 7/25)

AP:Police And Protesters Clash In Violent Weekend Across The USProtests took a violent turn in several U.S. cities over the weekend with demonstrators squaring off against federal agents outside a courthouse in Portland, Oregon, forcing police in Seattle to retreat into a station house and setting fire to vehicles in California and Virginia. A protest against police violence in Austin, Texas, turned deadly when a witness says the driver of a car that drove through a crowd of marchers opened fire on an armed demonstrator who approached the vehicle. And someone was shot and wounded in Aurora, Colorado, after a car drove through a protest there, authorities said. (Martin, 7/26)

Reuters:Seattle Black Lives Matter Clashes Spark 45 Arrests, 21 Police InjuredDozens were arrested and many police injured in clashes around Seattles biggest Black Lives Matter protest in weeks on Saturday, police said, with a renewed energy sparked by violent clashes between activists and federal agents in nearby Portland, Oregon. Police said officers used non-lethal weapons in attempts to disperse the thousands of marchers in the late afternoon after some protesters set fire to the construction site for a King County juvenile detention facility and courthouse. By 10 p.m., police had made 45 arrests in connection with todays riot in the East Precinct, the Seattle Police wrote in a Twitter post. Scruggs, 7/25)

The New York Times:Hurricanes Choice For Texans: Shelter From The Virus Or The StormBartt Howes boat was his refuge from the pandemic. Battling diabetes and H.I.V., he knew that catching the coronavirus as well could kill him, so he had been living alone on the docked boat for three months. Then Hurricane Hanna began to slam the Texas coast on Saturday, forcing Mr. Howe to trade one deadly menace for another: To avoid injury or death in the hurricane, he had to risk infection ashore. I had managed to stay safe all this time, but the storm kicked me out of my boat, he said with a hint of resignation. Now here I am, back on land, on borrowed time. (Sandoval, 7/26)

AP:Tropical Storm Hanna Drenches South Texas Amid Virus CrisisA day after roaring ashore as a hurricane, Hanna lashed the Texas Gulf Coast on Sunday with high winds and drenching rains that destroyed boats, flooded streets and knocked out power across a region already reeling from a surge in coronavirus cases. Downgraded to a tropical storm, Hanna passed over the U.S.-Mexico border with winds near 50 mph (85 kph), the National Hurricane Center said. It unloaded more than 12 inches (30 centimeters) of rain on parts of South Texas and northeastern Mexico. Border communities whose health care systems were already strained by COVID-19 cases with some patients being airlifted to larger cities found themselves under siege from the first hurricane of the 2020 Atlantic season. There were no immediate reports of any deaths on either side of the border. (Mone and Merchant, 7/26)

The Washington Post:Tropical Storm Hanna Unloading Flooding Rains In South TexasOn Sunday evening, Hanna continued its march southwest as a tropical depression, slipping into Mexico while still lashing the Rio Grande Valley with prolific rainfall. The direct strike by Hanna comes at a time when the Lone Star State is grappling with a spike in coronavirus cases. (Cappucci, Samenow and Freedman, 7/26)

The New York Times:FEMA Sends Faulty Protective Gear To Nursing Homes Battling VirusExpired surgical masks. Isolation gowns that resemble oversize trash bags. Extra-small gloves that are all but useless for the typical health workers hands. Nursing home employees across the country have been dismayed by what theyve found when theyve opened boxes of protective medical gear sent by the federal government, part of a $134 million effort to provide facilities a 14-day supply of equipment considered critical for shielding their vulnerable residents from the coronavirus. (Jacobs, 7/24)

Politico:Trump Administration Invests $472M More In Moderna Vaccine CandidateThe Trump administration is going to pump another $472 million into expanding Modernas clinical trial to test the safety and efficacy of its coronavirus vaccine candidate. What happened: Moderna announced Sunday that the Biomedical Advanced Research and Development Authority, known as BARDA, is pouring the additional dollars the day before the phase three trial of the vaccine candidate is slated to start. (Roubein, 7/26)

Reuters:Moderna Gets Further $472 Million U.S. Award For Coronavirus Vaccine DevelopmentThe U.S.-based drug maker said the additional funding will support its late-stage clinical development including the expanded Phase 3 study of Modernas vaccine candidate. In April, Moderna had received $483 million from the U.S. federal agency that funds disease-fighting technology, when the experimental vaccine was in an early-stage trial conducted by the U.S. National Institutes of Health. (7/26)

The Hill:Meadows Says White House Is 'Hopeful' It Can Announce New Coronavirus Therapies 'In The Coming Days'White House chief of staff Mark Meadows said Sunday that the administration is hopeful it can announce new therapies to treat the coronavirus in the coming days. Meadows told ABCs This Week that the White House has been working around the clock,with a focuson COVID-19 therapeutics, vaccines and mitigation therapies.The president has been very clear whatever amount of money and whatever amount of time needs to be invested, were doing that, the White House chief of staff said. (Coleman, 7/26)

The Hill:Azar: If We Wear Masks, We Can Avoid Further ShutdownsHealth and Human Services Secretary Alex Azar said Sunday that widespread social distancing and mask usage would eliminate the need for resuming shutdowns due to the coronavirus pandemic. If we wear our masks we can avoid further shutdowns but if we dont that will be the consequence, Azar said Sunday on CBS Face the Nation. (Budryk, 7/26)

Politico:Azar Blames Testing Delays On StatesHHS Secretary Alex Azar Sunday blamed the current delays in coronavirus testing on the states, which he said have been too slow to spend federal dollars to boost the countrys testing amid the virus's spread. The Trump administration has frequently sought to put the responsibility for the coronavirus response on governors and local officials, even as many public health officials as well as governors have called for a coordinated national emergency response. (Roubein, 7/26)

Politico:U.S. Testing Czar: Everyone Who 'Needs' A Covid-19 Test Can Get OneAdmiral Brett Giroir, the Trump administration coronavirus testing czar, said that anyone who needs a coronavirus test can get one but he acknowledged that the average turnaround time for tests is too long as states smash records for numbers of cases. Appearing on CNN's "State of the Union," he pushed back at former Trump chief of staff Mick Mulvaney who earlier this month called his familys difficulties obtaining tests promptly inexcusable" this many months into the pandemic. (Roubein, 7/26)

The Hill:Ex-CDC Director On US, COVID-19: 'We Are A Laggard'Former Centers for Disease Control and Prevention Director Tom Frieden on Sunday said the U.S. had been a laggard in addressing the coronavirus pandemic, specifically pointing to lack of centralized information. Ill be frank, we are a laggard, Frieden said on Fox News Sunday. We are one of the top in the world in terms of the cumulative death rate unlike many other countries that have high death rates, ours is continuing to increase. (Budryk, 7/26)

The Hill:CDC: Even Mild Coronavirus Symptoms Can Persist For WeeksCOVID-19 can result in prolonged illness even in people with mild symptoms, including young adults, according to a newanalysisreleased Friday by the Centers for Disease Control and Prevention (CDC).It has been known that people severely ill after contracting COVID-19 can stay sick for several weeks. But less has been known about theeffects of the disease on people with milder symptoms who dont require hospitalization. (Hellmann, 7/24)

Stat:Covid-19 Vaccines May Cause Mild Side Effects, Experts SayWhile the world awaits the results of large clinical trials of Covid-19 vaccines, experts say the data so far suggest one important possibility: The vaccines may carry a bit of a kick. In vaccine parlance, they appear to be reactogenic, meaning they have induced short-term discomfort in a percentage of the people who have received them in clinical trials. This kind of discomfort includes headache, sore arms, fatigue, chills, and fever. As long as the side effects of eventual Covid-19 vaccines are transient and not severe, these would not be sources of alarm in fact, they may be signals of an immune system lurching into gear. (Branswell, 7/27)

The New York Times:Your Coronavirus Antibodies Are Disappearing. Should You Care?Your blood carries the memory of every pathogen youve ever encountered. If youve been infected with the coronavirus, your body most likely remembers that, too. Antibodies are the legacy of that encounter. Why, then, have so many people stricken by the virus discovered that they dont seem to have antibodies? Blame the tests. (Mandavilli, 7/26)

The Wall Street Journal:A Big Unknown In Covid-19 Vaccine Development: How Long Will Protection Last?If any of the most-advanced Covid-19 vaccines prove to work safely, they may protect people for months or years rather than the rest of their lives, according to emerging science and health experts. Only a handful of vaccines generate lifetime immunity for most people, such as the ones for measles, a viral infection that naturally produces lifelong immunity. Experts caution against expectations of such longevity for Covid-19, citing experience with other respiratory viruses plus emerging data on the longevity of the antibodies that can prevent the virus from entering human cells and replicating. (Hopkins, Hernandez and Loftus, 7/26)

The Washington Post:Coronavirus Vaccine Trials Aim To Include The Black And Hispanic CommunitiesEach fall, the Rev. Rob Newells urges the congregation at Imani Community Church in Oakland, Calif., to get a flu shot. He builds bridges everyday between the countrys most vulnerable, marginalized communities and the medical system, defusing suspicion about HIV prevention treatments and educating people about medical research. He prods health-care leaders to think harder about their messengers: Dont send a white doctor to tell black people what they need to do for their own good. But with the first massive coronavirus vaccine trial in people set to start Monday, Newells finds himself in an unfamiliar place: on the fence about what to tell his colleagues, his community, his cousins. Biomedical research, Newells knows, is a long and painstaking process and he is concerned about a vaccine campaign that seems so narrowly focused on speed. (Johnson, 7/26)

Stat:Covid-19 Surge Helps AI Researchers Amass Lung ScansAt first, the images of lungs infected by the novel coronavirus were hard to come by. It was early in the pandemic, and Joseph Paul Cohen, a researcher at the University of Montreal, was trying to stockpile radiology scans to train an artificial intelligence model to recognize warning signs of severe illness. With so few images available, the work was next to impossible. But in recent weeks, the resurgence of Covid-19 in the U.S. and other hotspots has solved that problem, allowing him to amass hundreds of lung scans from clinical reports published around the world. (Ross, 7/27)

The New York Times:Corporate Insiders Pocket $1 Billion In Rush For Coronavirus VaccineOn June 26, a small South San Francisco company called Vaxart made a surprise announcement: A coronavirus vaccine it was working on had been selected by the U.S. government to be part of Operation Warp Speed, the flagship federal initiative to quickly develop drugs to combat Covid-19. Vaxarts shares soared. Company insiders, who weeks earlier had received stock options worth a few million dollars, saw the value of those awards increase sixfold. And a hedge fund that partly controlled the company walked away with more than $200 million in instant profits. (Gelles and Drucker, 7/25)

The Washington Post:ADHD Video Game Treatment Approved By FDACan a video game help children struggling with ADHD? That question inspired hopeful headlines last month after the Food and Drug Administration permitted marketing of the first digital game that may be prescribed to treat children ages 8 to 12 who have been diagnosed with attention-deficit/hyperactivity disorder. In EndeavorRx, designed for iPhones and iPads, children guide an avatar surfing through molten lava and an icy river, dodging fires and icebergs while grabbing flying objects. The game is not yet available for purchase, nor has a price been released, but its Boston-based developer, Akili Interactive Labs, may now feature its unique status in ads and pursue coverage by insurance plans. (Ellison, 7/26)

CIDRAP:Study Finds No Transmission Of COVID-19 From Moms To NewbornsA study yesterday in The Lancet Child & Adolescent Health found no evidence of COVID-19 transmission between 120 babies born to mothers with COVID-19, even after 2 weeks of breastfeeding with appropriate hygiene precautions. The findings led the American Academy of Pediatrics (AAP) to change its guidance on infants and COVID-19positive mothers. The guidelines now recommend that newborns "room-in" with infected others after delivery if proper hygiene precautions are taken, including wearing a mask when appropriate and practicing hand hygiene. (7/24)

CIDRAP:Pilot Study Evaluates Use Of Dogs For SARS-CoV-2 DetectionA small pilot study suggests trained scent-detection dogs have the potential to be used for mass detection of people infected with SARS-CoV-2, the virus that causes COVID-19, German researchers reported yesterday in BMC Infectious Diseases. In the study, eight dogs were trained for 1 week to detect SARS-CoV-2 from saliva or tracheobronchial secretions of patients infected with the virus. During the training, dogs were presented with positive and negative samples (confirmed by RT-PCR tests) using a device with seven scent holes with tubes leading to metal containers that held the samples. Only one hole had a container with a positive sample, and the other six had containers with control samples. After a week, the researchers conducted a double-blind, randomized controlled trial. (7/24)

AP:US Agency Vows Steps To Address COVID-19 InequalitiesIf Black, Hispanic and Native Americans are hospitalized and killed by the coronavirus at far higher rates than others, shouldnt the government count them as high risk for serious illness? That seemingly simple question has been mulled by federal health officials for months. And so far the answer is no. But federal public health officials have released a new strategy that vows to improve data collection and take steps to address stark inequalities in how the disease is affecting Americans. Officials at the Centers for Disease Control and Prevention stress that the disproportionately high impact on certain minority groups is not driven by genetics. (Stobbe, 7/25)

USA Today:Diabetes And COVID: Coronavirus Highlights America's Health ProblemsDr. Anne Peters splits her mostly virtual work-week between a diabetes clinic on the west side of Los Angeles and one on the east side of the sprawling city.Three days a week she treats people whose diabetes is well controlled. They have insurance, so they can afford the newest medications and blood monitoring devices. They can exercise and eat well. Those generally more affluent West LA patients who've gotten COVID-19 have developed mild to moderate symptoms feeling miserable, she said but treatable, with close follow-up at home. ... On the other two days of her work week, it's a different story. (Weintraub, 7/27)

The New York Times:In Era Of Sickness, Doctors Prescribe Unusual Cure: VotingThe sign is easy to miss in the waiting room of the emergency department at Massachusetts General Hospital, next to the reception desk and a hand sanitizer pump. Register to vote here, it says, above an iPad attached to a podium. The kiosk has stood there since November, before the pandemic began and stayed there through the worst weeks of April, when 12 gasping patients were put on ventilators during a single grueling 12-hour shift. Now, as the number of coronavirus patients has slowed to a trickle, Dr. Alister Martin, the 31-year-old emergency room doctor who built the kiosk, is determined to keep trying to register voters. (Stockman, 7/25)

NPR:Gene Therapy Sees Encouraging Success In Child With Duchenne Muscular DystrophyThis is the story of a fatal genetic disease, a tenacious scientist and a family that never lost hope. Conner Curran was 4 years old when he was diagnosed with Duchenne Muscular Dystrophy, a genetic disease that causes muscles to waste away. Conner's mother, Jessica Curran, remembers some advice she got from the doctor who made that 2015 diagnosis: "Take your son home, love him, take him on trips while he's walking, give him a good life and enjoy him because there are really not many options right now." (Hamilton, 7/27)

NPR:Could A Flu Shot Reduce Your Alzheimer's Risk?For years, public health officials have been trying to dispel the myth that people who get a flu shot are more likely to get Alzheimer's disease. They are not. And now there is evidence that vaccines that protect against the flu and pneumonia may actually protect people from Alzheimer's, too. The evidence comes from two studies presented Monday at this year's Alzheimer's Association International Conference, which is being held as a virtual event. (Hamilton, 7/27)

The Washington Post:References To White Men Still Dominate College Biology Textbooks, Survey SaysCharles Darwin. Carolus Linnaeus. Gregor Mendel. Theyre all men. Theyre all white. And their names appear in every biology book included in a new analysis of college textbooks. According to the survey, mentions of white men still dominate biology textbooks despite growing recognition in other media of the scientific contributions of women and people of color. The good news, the researchers say: Scientists in textbooks are getting more diverse. The bad news: If diversification continues at its current pace, it will take another 500 years for mentions of black/African American scientists to accurately reflect the number of black college biology students. (Brookshire, 7/26)

NPR:ADA At 30: 'We Are Not The Ones That Need To Change'Before the Americans with Disabilities Act granted people with disabilities greater protection and accessibility, a little-known law set the groundwork. In 1977, Judy Heumann helped lead a peaceful protest that forced the government to follow through with Section 504. As part of the 1973 Rehabilitation Act, the law would force hospitals, universities and other public spaces that received federal money, to remove barriers to accessibility for all Americans. But its implementation was long delayed over the costs necessary to retrofit buildings to comply with the law. (Shapiro and Bowman, 7/26)

AP:TV Reporter Credits Viewer With Noticing Cancerous LumpA television news reporter in Florida is crediting an eagle-eyed viewer for noticing a lump on her neck and emailing her that she should get it checked out. Victoria Price, a reporter for WFLA in Tampa, followed the advice and was diagnosed with cancer. Price tweeted that she is undergoing surgery on Monday to remove the tumor, her thyroid and a couple of lymph nodes. (7/26)

Politico:Sinclair To Delay Segment Featuring 'Plandemic' Conspiracy TheorySinclair Broadcasting on Saturday said it will delay its scheduled airing of a news segment featuring a viral conspiracy theory surrounding Anthony Faucis role in the Covid-19 pandemic.America This Week host Eric Bolling was scheduled to air an interview with Judy Mikovits, a medical researcher featured in the Plandemic video that claims Fauci, the nations top infectious disease expert, was responsible for the creation of the coronavirus, Media Matters reported. (Eliza Weaver, 7/25)

The Wall Street Journal:Sinclair Postpones Controversial Show About CoronavirusSinclair Broadcast Group Inc., the owner of 191 television stations across the U.S., delayed the airing of an interview about the alleged origins of the coronavirus pandemic that drew widespread criticism on social media. An episode of America This Week, which was slated to air over the weekend, features an interview with medical researcher Judy Mikovits, who has claimed that Dr. Anthony Fauci, the governments top infectious-disease expert, helped manufacture the coronavirus and spread it to China. Dr. Mikovitss claims have previously attracted attention in the documentary Plandemic, which was earlier pulled from major online platforms including Facebook Inc. and Alphabet Inc.s YouTube. (Rizzo, 7/26)

AP:Workers Praise Disney Virus Safety, But Will Visitors Come?Every week, it seems, Kaila Barker, her husband and their five children change their minds about whether to travel from their home in Connecticut to Floridas Walt Disney World as planned in September. On the one hand, the lack of crowds means more opportunities to go on rides without long waits. On the other hand, Connecticut and Florida have implemented pandemic-related quarantines for each others residents and visitors, and the Barkers worry whether the Disney magic will get lost with mandatory mask-wearing for visitors and workers, temperature checks and no parades, fireworks shows or up-close meet-and-greets with costumed characters. (Schneider, 7/26)

The Wall Street Journal:Major Truck-Stop Chains Will Require Drivers To Use Face MasksThe biggest U.S. truck-stop operators will require customers to wear masks starting next week, joining major retailers, restaurants and airlines in rolling out policies aimed at reducing the spread of coronavirus. Pilot Co., which operates 780 travel centers under the Pilot Flying J and other brands, said its mandate will take effect July 28. Similar policies kick in July 29 at Loves Travel Stops & Country Stores Inc. and TravelCenters of America Inc. sites. (Smith, 7/24)

USA Today:Walmart Mask Incident In Minnesota: Pair With Swastikas Banned A YearA Minnesota man and woman who wore face masks with swastikas on them in an incident captured on video have been banned fromWalmart storesnationwide for at least a year.The video, posted to Facebook on Saturday byRaphaela Mueller, shows a man and woman in a Walmart in Marshall, Minnesota, wearing red face coverings with swastikas. The woman flips off the camera while the man checks out groceries. (Culver, 7/26)

AP:Some US Police Resist Enforcing Coronavirus Mask MandatesLang Holland, the chief of police in tiny Marshall, Arkansas, said he thinks the threat of the coronavirus has been overstated and only wears a face mask if hes inside a business that requires them. He doesnt make his officers wear them either. So the day after Republican Gov. Asa Hutchinson signed an order requiring masks to be worn in public throughout Arkansas, Holland made it clear his department wasnt going to enforce the mandate in the Ozarks town of about 1,300, calling it an unconstitutional overreach. (DeMillo, 7/26)

AP:Amid Virus, Uncertainty, Parents Decide How To School KidsJoshua Claybourn is leaning toward sending his kindergarten daughter to in-person classes at a private school next month. Holly Davis sixth-grade daughter will learn online, though the family has not yet decided what to do for school for a teenage daughter who requires special accommodations for hearing problems and dyslexia and another whos starting college. As they decide how their children will learn this fall amid the coronavirus pandemic, parents are anxiously weighing the benefits of in-person instruction against the risks that schools could shut their doors again or that their children could contract the virus and pass it on. (Webber and Groves, 7/26)

The Hill:McEnany Likens Schools To 'Essential Places Of Business' In Push For ReopeningThe White House would support sending children back to school even if future studies showed kids transmitCOVID-19 at a higher rate than currently known, press secretary Kayleigh McEnany said Friday, arguing schools are "essential places of business." McEnany fielded multiple questions from reporters about President Trump's push for a return to in-person learning this fall even as he cancels some events for the Republican National Convention due to concerns about holding a mass gathering during the pandemic. Deborah Birx, the White House coronavirus response coordinator, also said Friday it's "an open question" how rapidly children under the age of 10 spread the virus. (Samuels, 7/24)

AP:Colleges Plan For Virus Testing, But Strategies Vary WidelyFor students heading to Colby College in Maine this fall, coronavirus testing is expected to be a routine part of campus life. All students will be required to provide a nasal swab every other day for two weeks, and then twice a week after that. All told, the college says it will provide 85,000 tests, nearly as many as the entire state of Maine has since the pandemic started. Colby, a private school of 2,000 students, joins a growing number of colleges announcing aggressive testing plans to catch and isolate COVID-19 cases before they spread. Harvard University says all students living on campus will be tested when they arrive and then three times a week. Boston University plans to test most students at least once a week. (Binkley, 7/26)

The New York Times:Tailors Know New Yorkers Pandemic Secret: Everybody Got Fat!With weddings postponed and offices shut, business was bleak at Woodside Tailor Shop in Queens during the long months of pandemic lockdown. There was no need for party dress alterations, or any pressure for slacks to be hemmed. But about three months in, things started picking back up in June, with one particular service in sudden demand: People needed a bit more breathing room in their clothing. Everybody got fat! said Porfirio Arias, 66, a tailor at Woodside. Its not only in New York. Its all over the world that people got fat. (Maslin Nir, 7/25)

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First Edition: July 27, 2020 - Kaiser Health News

The tech startup ecosystem in France is witnessing a spectacular growth, thanks to the fresh pool of aspirational entrepreneurs and investors. This substantial growth has led to numerous fundraising rounds in the country that take these French tech startups to the next level even during the pandemic crisis. Over the past few months, even while France was one of the worst-hit markets due to the COVID-19 outbreak, the investments in tech startups did not cease.

As per a report by Pitchbook, the most valued French tech startups have a joint value of over 6 billion. These companies operate across various industries including Artificial Intelligence, insurtech, and healthcare. Without further ado, heres the list of most valued tech startups in France so far.

Founder/s: Francis Nappez, Nicolas Brusson, Frederic MazzellaFounded year: 2006Funding: 405 million

Paris-based BlaBlaCar is a long-distance ride-sharing network. The carpooling service lets users connect with drivers who have empty seats and book seats online. As it allows commuters to share the ride, the cost per person is relatively lower than travelling alone.

Recently, the French carpooling platform that recently joined hands with Voi Technology to help make scooters reliable and easily available across big cities in France. As this move was during the COVID-19 crisis, the company had to launch the BlaBlaRide service adhering to the local regulations and guidelines.

Back in November 2018, BlaBlaCar secured nearly 100 million later stage VC funding from Socit Nationale des Chemins de fer Franais, Omnes Capital and Insight Partners, thereby reaching a valuation of 1.43 billion.

Founder/s: Jessy Bernal, Ivan Schneider, Stanislas Niox-ChateauFounded year: 2013Funding: 237 million

Doctolib is a French healthtech startup, which offers a software solution to doctors and hospitals. It is basically a doctor appointment booking app, which comes with an entire range of services that help refine and improve the efficiency of medical operations.

The subscription-based online medical appointment management service attained the unicorn status early in 2019 following the investment from General Atlantic, Bpifrance, Accel and others. Well, Doctolib secured 150 million funding, which increased its valuation to 1.13 billion.

Founders: Guillaume Lestrade, Thomas RebaudFounded year: 2016Funding: 269 million

French AI startup Meero aims to simplify the work of the photographer. From providing revenues and market research to invoicing, post-production, and delivery, Meero takes care of all the complicated, time-consuming tasks that plague the industry. The firm collaborates with hundreds and thousands of photographers all over the world so that they can create images for global brands.

Last year, Meero announced a Series C funding round of nearly 205 million led by Eurazeo Growth and Amsterdam-based VC, Prime Ventures. With this, the valuation of the startup that revolutionised the world of professional photography skyrocketed to 891 million.

Founder/s: Steve Anavi, Alexandre ProtFounded year: 2016Funding: 136 million

Qonto is a neobank for freelancers and SMEs that provides great customer support, transparency, and connection to carry out day-to-day business management. Qonto provides all the necessary tools to master the finances of an organisation. It is designed to make to simple to manage all transfers, debits, card payments, and small details.

Earlier this year, Qonto secured a Series C investment of 104 million from Tencent, Valar Ventures, Alven and DST Global among others, which marks the largest VC round in the French fintech industry to date. With this investment the valuation of the French neobank goes up to 811 million.

Founder/s: Ludovic Le Moan, Christophe FourtetFounded year: 2010Funding: 284 million

Sigfox works with a vision to connect every object in the physical world with the digital universe. It is a platform that provides wireless connectivity services for the IoT devices. The global network of Sigfox complements existing connectivity systems and paves the way for two-way, energy-efficient transmission of small quantities of data over long distances.

Back in 2017, Sigfox announced the closure of an undisclosed amount of funding from Khazanah Nasional Berhad to enhance its deployment in the Asian markets. This venture funding round took its valuation to 600 million.

Founder/s: Acher Criou, Emmanuel Freund, Asher Kagan, Stphane HliotFounded year: 2015Funding: NA

Paris-based Blade is the developer of a dematerialised computer intended to offer cloud gaming servers required to play high-end games through any PC. The companys servers are exploitable on any screen and offer access to a virtual high-end computer along with running thousands of virtual machines on server-grade Intel Xeon processors enabling users to get more power and reactivity.

Earlier this year, Blade, which revolutionises cloud computing, raised funding from LG Electronics. The amount that it raised remains undisclosed but it has secured a position among the most valued French tech startups with a valuation of 335 million.

Founder/s: Charles Gorintin, Jean-Charles SamuelianFounded year: 2016Funding: 125 million

Paris-based digital health insurance platform Alan offers hassle-free digital health insurance solutions to users with an excellent price-quality ratio health plan. This digital insurance platform makes coverage, claims, and reimbursements for both users and medical professionals. Alan also lets users access video calls and appointments.

A few months back, Alan secured 50 million Series C funding from Index Ventures and Temasek. With the latest investment, Alans valuation stands at 314 million and its total funding is 125 million.

Founder/s: Jean-Philippe COMBAL, Gloria Gonzalez-AseguinolazaFounded year: 2016Funding: 84.3 million

Paris-based gene therapy biotech company Vivet Therapeutics is dedicated to developing treatments for liver disorders that have been inherited. The company uses its proprietary treatment for Wilson disease, which is a rare, devastating, chronic and potentially life-threatening liver disorder. This company is building a diversified gene therapy pipeline based on novel adeno-associated virus (AAV) technologies.

In March 2019, Vivet Therapeutics bagged 45 million investment from Pfizer to advance its pipeline of gene therapy programs. This takes the overall funding secured by the French biotech firm to nearly 84 million and its valuation is around 300 million.

Founder/s: David Durrleman, Eric Sibony, Jeremy JawishFounded year: 2014Funding: 89.2 million

Shift Technology is a Paris-based startup providing AI-based fraud detection for the global insurance industry. It offers a fraud detection solution called FORCE, which is used by insurers all over the world across all business sectors. This French AI startup provides fraud handlers with a decision-making platform that will scale their capacity and give them more efficiency in claims processing.

Last year, Shift Technology secured 53 million Series C funding led by Bessemer Venture Partners along with participation from its existing investors such as General Catalyst, Accel and Iris Capital. With this, the valuation of the company is now 277 million.

Main image picture credits: BlaBlaCar

Check out the innovations that took home the Blue Tulip Awards this 2020

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These are the most valued tech startups in France in 2020 - Silicon Canals

Austin was three years old and Max was a newborn when their mother, Jenn McNary, learned they had a rare genetic condition called Duchenne muscular dystrophy. The doctor painted a grim picture: Her boys would stop walking by age 12 or 13 and, shortly thereafter, they would require nighttime ventilation. They would each need a tracheotomy, a feeding tube, or both by their late teens. Death would come a few years later.

It hasnt worked out that way, thanks to two new drugs that became available after the boys 2002 diagnosis. Exondys 51, a medicine that targets their genetic mutation, slows the diseases progression, and Emflaza, a corticosteroid, mitigates some of its symptoms. Thanks to these treatments, Austin now attends college and interns at a biotech company. Max attends his local high school in Newton, Massachusetts. Both are able to get around in wheelchairs, and neither needs ventilation. McNary just rented an apartment for her boys because they can function on their own with the help of an aide.

By all accounts, the drugs have been transformative, McNary said. But, she added, her boys arent going to be cured, and extending and improving their life for an unknown period of time comes at a high price. Emflaza came onto the market in 2017 at an annual cost of $65,000. Exondys 51 appeared in 2016 at $748,500. Neither of the drugs will help the young men walk again and, in the eyes of some U.S. health economists, the drugs are not worth the price.

Thats why McNary hates the quality-adjusted life year (QALY, pronounced qua-lee), an economic calculation that attempts to quantify the value of a medical intervention, based in part on the quality of life it bestows on recipients.

First developed by U.S. economists in the late 1960s and early 1970s, variations of the QALY have been used for years by governments around the world to help determine what treatments citizens can obtain under public health care. In Americas free-market health care system, however, QALY calculations have largely been avoided. As McNary and others like her are finding out, thats starting to change.

As policymakers and insurance companies scramble to get a handle on skyrocketing health care costs, they are promoting the idea of paying for value. In this view, drugs designated as higher-value should be prioritized over lower-value treatments. But this raises a thorny question: Who gets to define value? Health economists and insurance companies who seek to use limited health care dollars judiciously? Or patients, parents, and doctors who want to receive the best health care for their situation?

Because the quality-adjusted life year threatens her sons ability to get the medicine they need, McNary is clear about her answer. To me, the QALY is a measurement that says that keeping my sons alive by providing incremental benefit but not totally curing them is never going to be valuable, McNary said. Just mull that around in your head if you are less than perfect, you are worth less money.

In QALY math, a year of perfect health is equal to 1; death equates to 0. The value of other health states is derived from surveys of patients, caregivers, or the general public. Paralysis might be valued at .35, for example, and mild Alzheimers disease at .52, depending on the survey. Those numbers can then be plugged into a formula that allows the relative cost-effectiveness of treatments to be compared to identify the best buys.

Economists developed the QALY concept more than 40 years ago to address a fundamental question: Where should we spend whose money to undertake what programs to save which lives with what probability? Richard J. Zeckhauser and Donald Shepard asked in a 1976 article describing the basic QALY formula. The next year, as U.S. health care spending topped $120 billion, Harvard health policy professor Milton C. Weinstein and his colleague, cardiologist William B. Stason, sounded an alarm bell. It is now almost universally believed that the resources available to meet the demands for health care are limited, they wrote in the New England Journal of Medicine. We, as a nation, will have to think very carefully about how to allocate the resources we are willing to make available for health care.

Their article cited by other authors more than a thousand times in the past four decades pointed out that resources were already being allocated by millions of individual decisions: hospitals rationing beds where they didnt have room for all patients, for example, and insurers agreeing to pay for some tests and treatments but not for others. Such decisions, they argued, were often inconsistent with the societal objective of deriving the maximum health benefits from the dollars spent, an objective that could be achieved by putting the QALY to work.

In the intervening decades, some countries the United Kingdom, the Netherlands, and Sweden, for example have embraced QALY-based evaluations. In the U.K., cost-effectiveness studies are used, in part, to determine which therapies the National Health Service will provide for residents. The publicly-funded health system does not cover Orkambi, the first cystic fibrosis treatment that targets the cause of the disease, for example, because its cost-per-QALY far exceeds the U.K. cost-effectiveness threshold.

In the United States, however, QALY-based assessments have not gained traction until recently. Perhaps the general reason is that we as patients and our providers dont want to be limited in the treatment options available, said Louis P. Garrison Jr., an economist in the Pharmaceutical Outcomes Research and Policy Program at the University of Washington.

In fact, QALY-based cost-effectiveness reviews are so controversial that the federal government has repeatedly quashed their use. In 1992, the Department of Health and Human Services rejected Oregons attempt to use QALY-based cost-effectiveness assessments to determine what services its Medicaid program would cover. In 2010, as part of the Patient Protection and Affordable Care Act, Congress prohibited the use of QALYs by the Medicare program. It also banned the federal Patient-Centered Outcomes Research Institute from using QALY thresholds in its assessments of comparative treatments.

But more than half of U.S. residents are covered by private insurance companies, which are not prohibited from using QALY-based assessments to decide which medicines they will cover for their members. Traditionally, however, private insurers have generally not used QALYs explicitly in their decisions about what tests and treatments they will pay for, according to a recent report by the National Council on Disability. Instead, when major U.S. insurers decide to limit access to a given medication, they usually cite insufficient data to justify its use in a given situation.

Indeed, until recently, U.S. insurers did not have a source for QALY-based cost-effectiveness reports. That began to change in 2014, when the Institute for Clinical and Economic Review, a nonprofit research organization based in Boston, turned its attention to high-cost drugs. Founded in 2006 as a research project based at Harvard Medical School, ICER initially issued reports on broad topics such as obesity management and palliative care. But when Sovaldi, a drug for deadly hepatitis C, came on the market at the then-shocking price of $84,000 for a 12-week course of treatment, ICER kicked into action. Despite the high price, its assessment found that Sovaldi is cost-effective for some patients. Insurers took notice.

Since then, the organization has been churning out several drug-assessment reports each year. Each report includes its opinion of how much the drug is worth; drugs priced higher than that are deemed not cost-effective. ICER has no authority over anyone, but its reports have become popular reading for U.S. insurers. If there is a drug of note being approved by the FDA, theres also likely going to be an ICER assessment of that drug that can factor into their decision-making, said David Whitrap, the research organizations vice president of communications and outreach.

U.S. health care spending has risen dramatically since Weinstein and Stason expressed concern in the mid-1970s. In 2016, the U.S. spent nearly 18 percent of its gross domestic product on health care, far outstripping the average of 11 percent for 10 other high-income nations. High prices for prescription drugs is one reason. Were seeing price tags now of $1 million, $2 million, said Seema Verma, administrator for the federal Centers for Medicare and Medicaid Services, at a conference recently. Thats completely unsustainable for the system.

Thats why Peter Neumann, director of the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center, said cost-effectiveness analyses are needed more than ever. But there are many reasons for the resistance, Neumann and his co-authors wrote in the Journal of the American Medical Association, including an inclination on the part of many individuals in the United States to minimize the underlying problem of resource scarcity and the consequent need to explicitly ration care.

Further, Ari Neeman, a disability rights activist and consultant to Partnership to Improve Patient Care, a coalition of advocacy groups, said the idea that two health conditions can be numerically compared to one another is simply wrong. Proponents of the QALY will say it is this mathematically perfect measure that gives us a superpower ability to compare depression drugs to cystic fibrosis drugs to cancer drugs even though all of those drugs do different things because it lets you translate them back to this common measure, he said. Our concern is that when you engage in that process of translation, you lose some significant nuance in terms of the amount of benefit thats being delivered.

The Partnership argues the QALY calculation is flawed because it assumes quality of life can be captured by a certain number, despite the fact that different surveys arrive at different numbers. For example, a 2006 quality-of-life survey in the U.S. assigns blindness/low vision as .69 on the 0-to-1 scale, while a 2011 survey in the U.K. gives blindness/low vision a score of .78.

Beyond the methodological issues, Neeman said, there are all kinds of ethical problems with it. People with disabilities and chronic medical conditions may value a treatment that offers an incremental improvement in the quality or length of their lives, even though the QALYs gained are less than those for a treatment that prevents the loss of perfect health.

Former U.S. Representative Tony Coelho, a Democrat from California and a primary author of the Americans with Disabilities Act, is the Partnerships chairman. I worry that more focus is being given to what is most cost-effective for the average patient than creating a system that works for each individual patient, he wrote in 2018. The medication I take for epilepsy isnt high value for every patient. But its the only one that works for me.

Thats why, Neeman said, cost-effectiveness analyses must consider the fact that not all patients respond the same way to a drug. Some patients need drugs that arent deemed cost-effective for the general population. Its important to account for that, he said. Otherwise were giving insurers a tool to deny care to people who need it.

When an insurer decides to cover a specific drug, that decision affects everybody who pays into the insurance pool. Michael Sherman, chief medical officer for the insurer Harvard Pilgrim Health Care, uses the example of a gene therapy that costs $1 million to treat a child who will die without it. Under the ACA, families will hit their out-of-pocket maximum at about $16,000, and many health plans have out-of-pocket maximums far below that. The rest of that million dollars is going to be paid by everyone else thats the way it works in insurance, he said. When insurers see that kind of unanticipated budget impact, they raise premiums or out-of-pocket cost-sharing for everyone.

Like other proponents of the QALY, Neumann sees it as an imperfect but useful tool. Any single number is never going to capture everything, he said.

The problem is, if youre not going to use QALYs, what are you going to use?

Thats an urgent question, particularly now when there is a huge pipeline for rare-disease therapies, often called orphan drugs. By 2024, orphan drug sales are expected to reach $242 billion.

In the U.S., a rare disease is defined as one that affects fewer than 200,000 people. While these conditions are individually rare, in the aggregate, an estimated 25 to 30 million Americans thats about one in 10 live with a rare disease. Most rare diseases affect children, and many are fatal or disabling.

Historically, drugmakers spent little effort developing treatments for rare diseases, but that changed with the passage of the Orphan Drug Act of 1983, which provides tax credits and a seven-year marketing exclusivity to companies that develop rare-disease treatments. Hundreds of such treatments have won FDA approval in recent years, with more than 560 medicines in the works.

Those treatments are generally expensive. On average, the per-patient cost for orphan drugs in the U.S. is almost 4.5 times more than for non-orphan drugs.

In the two decades ending in 2017, the average annual cost for orphan drugs was $123,543, based on the price at the time the drug launched, compared to $4,961 for traditional drugs. For Duchenne alone, more than 30 orphan therapies are in development. None of them are going to cure patients, McNary said. But she hopes new treatments, generally used in combination, will help her sons live longer, healthier lives and completely change the disease trajectory for younger patients whose disease has not yet progressed as far.

The barrier she worries about is cost-effectiveness analysis. In August, the Institute for Clinical and Economic Review published its assessment of treatments for Duchenne, which affects about 400 to 600 boys born in the U.S. each year. Emflaza, the corticosteroid, appears to be as good as or better than prednisone, another corticosteroid approved to treat the disease, but it would need a price cut of at least 73 percent to be considered cost-effective.

Exondys 51 approved by the FDA for about 13 percent of the Duchenne population got a worse review. In the clinical trials used to seek FDA approval, no clinical benefit, including motor function improvement, was demonstrated. (The FDA approved the drug because some of the patients treated with Exondys 51 had a slight increase in dystrophin levels in skeletal muscle.) In light of that, Exondys 51 was not deemed cost-effective at any price.

But Jenn McNary said the drug works for her sons. Austin, who was not eligible for the Exondys 51 clinical trial, stopped walking at age 10. Max got in the trial and started taking the drug at age 9.They have the same mutation, they have been raised by the same mother, so one would expect they would progress similarly, she said. But Max walked until he was 17.

Austin was already in a wheelchair when, at age 15, he started taking Exondys 51. He regained some upper-body strength that changed his life, according to his mother. Hes able to use a urinal on his own, which makes is possible for him to have a job and to go to college without an aide, she said.

The Medicaid program in Massachusetts, where the McNarys live, wont pay for Maxs Duchenne therapies. For the time-being, the drugmakers are giving him the drugs free through a patient-assistance program. Austin, because hes enrolled in college, is eligible for student coverage through Blue Cross Blue Shield of Massachusetts. The insurer, by policy, does not cover Exondys 51 for patients who can no longer walk. His mother appeals the insurance denial. Every six months, she sends a video of Austin in action, along with a letter from his doctor and so far, his medicines have been covered.

The payers made their coverage policies before the quality-adjusted life year analysis was published. Now, insurers who have been covering the Duchenne treatments have an independent analysis with which to rethink that decision.

For now, there is one thing that QALY supporters and critics agree on. Very promising drugs are coming, and theyre going to be very expensive, said Neumann, the health economist at Tufts. Increasingly, the QALY appears poised to influence how American health care money is spent.

Lola Butcher is a health care business and policy writer based in Missouri. Follow her on Twitter @LolaButcher

This article was originally published on Undark and has been republished here with permission. Undark can be found on Twitter @undarkmag

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What's a life worth in dollars and cents? Should that influence who gets treated for expensive disease treatments? - Genetic Literacy Project