Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Market - Global Industry Analysis, Size, Share, Growth, Trends and Forecast 2017 - 2025

This press release was orginally distributed by SBWire

Albany, NY -- (SBWIRE) -- 08/04/2017 -- Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare blood disease of bone marrow stem cells, which are genetically characterized by the somatic mutation in the phosphatidylinositol glycan protein A (PIG-A) gene. PNH generally occurs in the early 30s. Around 10% patients develop PNH symptoms at 21 years of age or earlier. Around 1 to 5 individuals per million people in the U.S. are estimated to suffer from PNH. This is much lower than the incidence rate of bone marrow aplasia. PNH often goes unrecognized; delay in diagnosis may range from one year to more than 10 years.

The global PNH treatment market is anticipated to expand at a rapid pace during the forecast period. It is a niche market, with many pharmaceutical and biotech companies investing in research of bone marrow stem cells. According to current studies, the ideal treatment available is to replace all the hematopoietic stem cells with normal stem cells via stem cells transplantation. However, this treatment is not ideal in some cases as stem cell transplantation requires a stable histocompatible donor. Complete stem cells transplantation is usually considered in severe cases of PNH, for instance aplastic anemia and transformation to leukemia, as these can be life threatening complications.

Factors driving the PNH treatment market include rise in number of blood & bone marrow related disorders, increase in aging population, and technological advancements in stem cells transplantation. However, increase in cost of medical equipment, specifically surgical equipment required for stem cell transformation; lack of reimbursement policies in developing regions; and occurrence of side effects in related current available treatments may hamper the PNH treatment market.

The global PNH treatment market can be segmented based on diagnosis test, type of treatment, drugs, and end-user. In terms of diagnosis test, the market can be divided into complete blood count test (CBC), lactate dehydrogenase test (LDH), bilirubin test, bone marrow examination, urine test for hemosiderin, flow cytometry, and others. Based on the type of treatment, the PNH treatment market can be segregated into treatment of PNH patients associated with hemolysis, treatment of PNH patients associated with thrombosis, treatment of PNH patients associated with non-hemolytic anemia, allogeneic stem cell transplant (SCT)/bone marrow transplant (BMT), treatment of pregnant PNH patients, treatment of pediatric PNH patients, and others. In terms of drugs, the market can be classified into eculizumab (Soliris), ALXN1210, and others. Based on end-user, the PNH treatment market can be split into hospitals, pharmaceutical & biotech companies, clinics, academic & research institutes, and others.

Make an Enquiry @ http://www.transparencymarketresearch.com/sample/sample.php?flag=B&rep_id=28220

Geographically, the market for PNH treatment can be divided into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa (MEA). North America dominates the global PNH treatment market due to the rise in the number of blood & bone marrow related diseases, availability of satisfactory reimbursement policies, and increase in awareness about the early diagnosis of the disease in the region. The market in Europe is also expected to expand rapidly, as key players are collaborating with research institutions and labs to develop new innovative products. The PNH treatment market in Asia Pacific is anticipated to expand at a fast pace owing to the unmet needs regarding PNH treatment of the growing population. Additionally, factors such as development of the health care network, rise in disposable income, increase in health care awareness, and availability of reimbursement facilities are boosting the PNH treatment market in Asia Pacific.

Key players operating in the PNH treatment market include Alexion Pharmaceuticals, Inc., Thermo Fisher Scientific Inc., GE Healthcare, and Johnson & Johnson.

About Transparency Market ResearchTransparency Market Research (TMR) is a global market intelligence company providing business information reports and services. The company's exclusive blend of quantitative forecasting and trend analysis provides forward-looking insight for thousands of decision makers. TMR's experienced team of analysts, researchers, and consultants use proprietary data sources and various tools and techniques to gather and analyze information. Our business offerings represent the latest and the most reliable information indispensable for businesses to sustain a competitive edge.

For more information on this press release visit: http://www.sbwire.com/press-releases/paroxysmal-nocturnal/release-843409.htm

See the original post:
Rise in Number of Blood & Bone Marrow Related Disorders Drives the Global Paroxysmal Nocturnal Hemoglobinuria ... - Digital Journal

Recommendation and review posted by simmons

WASHINGTON & HACKENSACK, N.J.--(BUSINESS WIRE)--MedStar Georgetown University Hospital in Washington, D.C. in collaboration with John Theurer Cancer Center, part ofHackensack Meridian Health, Hackensack University Medical Center in Hackensack, N.J., announce the 100th blood stem cell transplant performed since the BMT programs first patient was treated in September, 2013.

The patient, a woman from Arlington, Virginia, received her blood stem cell transplant at MedStar Georgetown as a treatment for multiple myeloma diagnosed in December 2016.

The BMT program at MedStar Georgetown is a joint effort with specialists from Hackensack John Theurer Cancer Center and a key component of the Lombardi Comprehensive Cancer Center, the only cancer program in the Washington, D.C. region designated by the National Cancer Institute (NCI) as a comprehensive cancer center.

Once considered experimental, BMT is todays established gold standard for treating patients with a number of malignant and other non-malignant diseases of the immune system, blood, and bone marrow, including multiple myeloma, lymphoma, and acute and chronic leukemia. For some conditions, blood stem cell transplant can provide a cure in patients who have failed conventional therapies, says Scott Rowley, MD, chief of the BMT program at MedStar Georgetown as well as a member of the John Theurer Cancer Centers Blood and Marrow Stem Cell Transplantation. For some conditions, it can actually be a cure; for others, it prolongs survival and improves quality of life. Having performed 100 BMTs at MedStar Georgetown including allogenic transplantation illustrates the strength and maturity of our program achieved in rather short time.

MedStar Georgetowns program is also the only comprehensive BMT center within Washington, D.C. and southern Maryland with accreditation from the Foundation for the Accreditation of Cellular Therapy (FACT) for adult autologous procedures, where the patient donates his or her own cells.

The BMT program at JTCC is one of the top 10 transplant programs in the United States, with more than 400 transplants performed annually.

A BMT involves a two-step process: first, collecting bone marrow stem cells from the patient and storing them for future use. Then, a week or so later, patients receive high dose chemotherapy to eliminate their disease. The previously stored cells are reinfused back into the bloodstream, where after reaching the bone marrow, they begin repopulating and allow the patient to recover their blood counts over the following 2 weeks.

Even though BMT is considered standard therapy for myeloma worldwide, in the United States fewer than 50 percent of the patients who could benefit from BMT are referred for evaluation, says David H. Vesole, MD, PhD, Co- Chief and Director of Research of John Theurer Cancer Centers Multiple Myeloma division and director of MedStar Georgetowns Multiple Myeloma Program.

Thats mostly due to physicians concerns that a patient is too old or compromised from other health conditions like diabetes, cardiac disease or renal failure. But new techniques and better supportive care have improved both patient outcomes and the entire transplant process, extending BMT to more patients than ever before.

The MedStar Georgetown/Georgetown Lombardi Blood and Marrow Stem Cell Transplant Program is part of a collaborative cancer research agenda and multi-year plan to form an NCI-recognized cancer consortium. This recognition would support the scientific excellence of the two centers and highlight their capability to integrate multi-disciplinary, collaborative research approaches to focus on all the aspects of cancer.

The research areas include expansion of clinical bone marrow transplant research; clinical study of haplo transplants use of half-matched stem cell donor cells; re-engineering the function and focus of key immune cells; and the investigation of immune checkpoint blocking antibodies that unleash a sustained immune response against cancer cells.

In this partnership, weve combined John Theurers strength in clinical care with Georgetown Lombardis strong research base that significantly contributes to clinical excellence at MedStar Georgetown. By working together, we have broadened our cancer research to offer more effective treatment options for tomorrows patients, says Andrew Pecora, MD, FACP, CPE, president of the Physician Enterprise and chief innovations officer, Hackensack Meridian Health. This is one of many clinical and research areas that have been enhanced by this affiliation.

Our teams are pursuing specific joint research projects we feel are of the utmost importance and significance in oncology particularly around immuno-oncology as well as precision medicine, says Andr Goy, MD, MS, chairman of the John Theurer Cancer Center and director of the division chief of Lymphoma; chief science officer and director of Research and Innovation, RCCA; professor of medicine, Georgetown University. Together our institutions have a tremendous opportunity to transform the delivery of cancer care for our patient populations and beyond.

ABOUT THE JOHN THEURER CANCER CENTER AT HACKENSACK UNIVERSITY MEDICAL CENTER

John Theurer Cancer Center at Hackensack University Medical Center is New Jerseys largest and most comprehensive center dedicated to the diagnosis, treatment, management, research, screenings, and preventive care as well as survivorship of patients with all types of cancers. The 14 specialized divisions covering the complete spectrum of cancer care have developed a close-knit team of medical, research, nursing, and support staff with specialized expertise that translates into more advanced, focused care for all patients. Each year, more people in the New Jersey/New York metropolitan area turn to the John Theurer Cancer Center for cancer care than to any other facility in New Jersey. Housed within a 775-bed not-for-profit teaching, tertiary care, and research hospital, the John Theurer Cancer Center provides state-of-the-art technological advances, compassionate care, research innovations, medical expertise, and a full range of aftercare services that distinguish the John Theurer Cancer Center from other facilities.www.jtcancercenter.org.

MedStar Georgetown University Hospital is a not-for-profit, acute-care teaching and research hospital with 609 beds located in Northwest Washington, D.C. Founded in the Jesuit principle of cura personaliscaring for the whole personMedStar Georgetown is committed to offering a variety of innovative diagnostic and treatment options within a trusting and compassionate environment.

MedStar Georgetowns centers of excellence include neurosciences, transplant, cancer and gastroenterology. Along with Magnet nurses, internationally recognized physicians, advanced research and cutting-edge technologies, MedStar Georgetowns healthcare professionals have a reputation for medical excellence and leadership.

For more information please visit: medstargeorgetown.org/bmsct

About Hackensack Meridian Health Hackensack University Medical Center

Hackensack Meridian Health Hackensack University Medical Center, a 775-bed nonprofit teaching and research hospital located in Bergen County, NJ, is the largest provider of inpatient and outpatient services in the state. Founded in 1888 as the countys first hospital, it is now part of one of the largest networks in the state comprised of 28,000 team members and more than 6,000 physicians. Hackensack University Medical Center was listed as the number one hospital in New Jersey in U.S. News & World Reports 2016-17 Best Hospital rankings - maintaining its place atop the NJ rankings since the rating system was introduced. It was also named one of the top four New York Metro Area hospitals. Hackensack University Medical Center is one of only five major academic medical centers in the nation to receive Healthgrades Americas 50 Best Hospitals Award for five or more years in a row. Beckers Hospital Review recognized Hackensack University Medical Center as one of the 100 Great Hospitals in America 2017. The medical center is one of the top 25 green hospitals in the country according to Practice Greenhealth, and received 25 Gold Seals of Approval by The Joint Commission more than any other hospital in the country. It was the first hospital in New Jersey and second in the nation to become a Magnet recognized hospital for nursing excellence; receiving its fifth consecutive designation in 2014. Hackensack University Medical Center has created an entire campus of award-winning care, including: the John Theurer Cancer Center; the Heart & Vascular Hospital; and the Sarkis and Siran Gabrellian Womens and Childrens Pavilion, which houses the Joseph M. Sanzari Childrens Hospital and Donna A. Sanzari Womens Hospital, which was designed with The Deirdre Imus Environmental Health Center and listed on the Green Guides list of Top 10 Green Hospitals in the U.S. Hackensack University Medical Center is the Hometown Hospital of the New York Giants and the New York Red Bulls and is Official Medical Services Provider to The Northern Trust PGA Golf Tournament. It remains committed to its community through fundraising and community events especially the Tackle Kids Cancer Campaign providing much needed research at the Childrens Cancer Institute housed at the Joseph M. Sanzari Childrens Hospital. To learn more, visit http://www.HackensackUMC.org.

Excerpt from:
John Theurer Cancer Center and MedStar Georgetown University ... - Business Wire (press release)

Recommendation and review posted by Bethany Smith

Are YOU acandidate for thisrevolutionary newtreatment?Find out at a FREEEDUCATIONALSEMINAR!Stop LIVING withCHRONIC PAINExperience healing with regenerativeStem Cell Therapy.Stop living with chronic pain and start living the more activelifestyle you love. Todays regenerative medicine offers arevolutionary treatment that helps to heal your injured tissue.If you suffer from injured or degenerative conditions in yourback, knees, hips, shoulders or have arthritic joints or sufferfrom neuropathy or respiratory diseases like COPD. StemCell Therapy can help get you out of pain! Thousands ofpatients have found relief for these conditions and manyothers with the natural healing of stem cells. Stem CellCenters is a national leader in the treatment of theseconditions and our advanced treatment protocols get ourpatients back to the quality of life they desire and expect.Get back to a better quality of life.Stop missing out on life, live more pain free and with moremobility and start enjoying the activities you miss: yourfamily, work, gardening, sports and travel. Stem CellTherapy is a viable option to get you back to loving life.Scientists have discovered amazing thingsStem Cells can do:BACK PAINNEUROPATHYJOINTSCOPD Turn into and create cells that grow cartilage, tendons,discs, and virtually any other tissue in your body. Prevent your bodys own cells from dying prematurely Communicate with the abnormal cells of your body andSwitch them into behaving normally Prevent and break down Fibrotic/Scar tissue Regulate your immune system, with the potential toturn off Auto-Immune DiseasesWhat this means for You.When you come to any one of our many Stem Cell Centersacross the country, you can expect to receive the propertreatment with the right type of regenerative cell, AND thecorrect dosage, giving you the highest chance of gettingback to the quality of life you deserve. Call today to schedule a complimentary consultation for you and/or your lovedone. We will answer all of your questions, providing ampleinformation so that you can make an educated decision onwhether this is the right treatment for you.The Stem Cell Centers Difference.Stem Cell Centers is a national network of surgeons and topdoctors who all work together in collaboration on importantIRB monitored research studies on numerous conditions.The results from these ongoing studies help us to determinethe best protocols to use to treat patients throughout ournetwork. Our doctors are all trained in using either your ownAdipose or Bone Marrow-derived stem cells, as well asethical Cord and Amnion-derived stem cells.Are you acandidate for thisgame-changingtreatment?COPDOsteoarthritis - Back, Knee,Shoulder, Hips, Ankles,Wrist, Thumb Joint, HandsBursitisDegenerative Disc DiseaseHerniated/Bulging DiscPeripheral Neuropathy- R. SpurgeonAdvanced Spine & Pain Clinicsof MinnesotaRegenerative Medicine & Stem Cell ExpertTendonitis612-255-4677stemcellcenters.comSeating Limited - Register Online or Call Today!LEARN MOREAT ONE OF OURONALASKA, WII am no longeroxygen dependent!Meet youR provider:MATT THORSON, MDConditions we treat include:General ArthritisMy friend had a bad hipwhich was fixed with stemcells at Stem Cell Centersin Spokane.I drove from Roseburg,Oregon to see if stemcells could help my COPDthat I have had for severalyears. Since my treatments, my blood oxygenlevels have risen from60% to 90% andFREE EDUCATIONAL SEMINARSThursday, August 17 - 10a, 2p and 6pStoney Creek Hotel and Conference Center3060 S. Kinney Coulee Rd.Onalaska, WIWINONA, MNFriday, August 18 - 10a, 2p and 6pRiverport Inn Conference and Event Center900 Bruski DriveWinona, MNLimited Seating.Reserve yourspot today!

Read the rest here:
30568008.pdf - Chippewa Herald

Recommendation and review posted by Bethany Smith

Scientists have created genetically altered skin cells that may control type 2 diabetes in lab mice. And they believe the general concept could someday be used to treat various diseases.

Using a combination of stem cells and "gene editing," the researchers created patches of skin cells that were able to release a hormone called GLP1 in a controlled manner.

The hormone, which is normally produced in the digestive tract, spurs the production of insulin -- the body's key regulator of blood sugar levels.

The scientists found that transplanting the engineered skin patches onto diabetic lab mice helped regulate their blood sugar levels over four months.

Xiaoyang Wu, a stem cell biologist at the University of Chicago, led the "proof of concept" study. He said it raises the possibility that "therapeutic skin grafts" could be used to treat a range of diseases -- from hemophilia to drug dependence.

Wu's team focused on type 2 diabetes in these initial experiments because it's a common condition.

However, a researcher not involved in the study doubted the usefulness of the approach for diabetes specifically.

People with type 2 diabetes already manage the disease with diet, exercise and medications -- including ones that target GLP1, said Juan Dominguez-Bendala.

Using high-tech gene therapy to get the same result seems unlikely, said Dominguez-Bendala, an associate professor at the University of Miami's Diabetes Research Institute.

"I don't see something like this coming to the clinic for diabetes," he said.

But Dominguez-Bendala also pointed to what's "cool" about the experiments.

Wu's team used a recently developed technology called CRISPR (pronounced "crisper") to create the skin patches. The technique, heralded as a major breakthrough in genetic engineering, allows scientists to make precision "edits" in DNA -- such as clipping a particular defect or inserting a gene at a specific location.

Before CRISPR, scientists could not control where an inserted gene would be integrated into the genome. It might end up in a "bad" location, Dominguez-Bendala explained, where it could, for example, "awaken" a tumor-promoting gene.

Wu and colleauges used CRISPR to make specific edits in GLP1, including one that allowed the gene to be turned "on" or "off" as needed, by using the antibiotic doxycycline.

The modified gene was inserted into mouse stem cells, which were then cultured into skin grafts in the lab. Finally, those grafts were transplanted onto lab mice.

The researchers found that when the mice were fed food with tiny amounts of doxycycline, the transplanted skin released GLP1 into the bloodstream. In turn, the animals' insulin levels rose and their blood sugar dipped.

The engineered skin also seemed to protect the mice from the ravages of a high-fat diet. When the mice were fed a fat-laden diet, along with doxycycline, they gained less weight versus normal mice given the same diet. They also showed less resistance to the effects of insulin, and lower blood sugar levels.

According to Wu, the study lays the groundwork for more research into using skin cells as a way to deliver "therapeutic proteins."

For instance, he said, skin cells could be engineered to provide an essential protein that is missing because of a genetic defect. As an example, he cited hemophilia -- a genetic disorder in which people lack a protein that allows the blood to clot properly.

Skin cells could be an ideal way to deliver such therapies, Wu said.

For one, the safety of skin grafts in humans is well-established, he pointed out. Since the 1970s, doctors have known how to harvest skin stem cells from burn victims, then use those cells to create lab-grown skin tissue.

Because the skin is generated from a patient's own stem cells, that minimizes the issue of an immune system attack on the tissue.

Dominguez-Bendala agreed that using skin cells has advantages. For one, he noted, the skin graft can be easily removed if something goes awry.

But a lot of work remains before therapeutic skin grafts could become a reality for any human disease. And research in animals doesn't always pan out in humans.

A next step, Wu said, is to see whether the skin grafts maintain their effects in lab mice over a longer period. The researchers will also monitor the animals for any immune system reactions against the GLP1 protein itself.

The findings were published online Aug. 3 in Cell Stem Cell.

Excerpt from:
Engineered Skin Cells Control Diabetes in Mice - Newsmax

Recommendation and review posted by simmons

MedicalResearch.com Interview with:

Dr. Xiaoyang Wu

Dr. Xiaoyang Wu PhDBen May Department for Cancer ResearchThe University of Chicago, Chicago, IL

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: We have been working on skin somatic stem cells for many years. As one of the most studies adult stem cell systems, skin stem cells have several unique advantages as the novel vehicle for somatic gene therapy (summarized also in the paper). The system is well established. Human skin transplantation using CEA device developed from skin stem cells have been clinically used for decades for burn wound treatment, and been proven to be safe the effective.

In this study, we developed a skin 3D organoid culture model to induce stratification and maturation of mouse epidermal stem cells in vitro, which allows us to efficiently transfer engineered mouse skin to isogenic host animals. In the proof of concept study, we showed that we can achieve systematic release of GLP1 at therapeutic concentration by engineered skin grafts.

MedicalResearch.com: What should clinicians and patients take away from your report?Response: Engineered skin transplant may provide a safe and long term delivery system for treatment of many human diseases.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: Before clinical translation, we will further characterize our mouse model of skin therapy, for the potential immune reaction, stability of skin grafts, and duration of the therapeutic effects in vivo. We are also interested in using our mouse model to test other potential applications of skin gene therapy, such as human genetic diseases, including hemophilia, urea cycle disorders.

There is no conflict-of-interest.

MedicalResearch.com: Thank you for your contribution to the MedicalResearch.com community.

Citation:Jiping Yue, Xuewen Gou, Yuanyuan Li, Barton Wicksteed, Xiaoyang Wu. Engineered Epidermal Progenitor Cells Can Correct Diet-Induced Obesity and Diabetes. Cell Stem Cell, 2017 DOI: 10.1016/j.stem.2017.06.016

http://www.cell.com/cell-stem-cell/fulltext/S1934-5909(17)30274-6?_returnURL=http%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS1934590917302746%3Fshowall%3Dtrue

Note: Content is Not intended as medical advice. Please consult your health care provider regarding your specific medical condition and questions.

See the article here:
Skin-Grafted Stem Cells May Treat Obesity and Diabetes - MedicalResearch.com (blog)

Recommendation and review posted by simmons

A contagious face cancer has killed off a majority of an absolutely unique animal, the legendary Tasmanian devil.

The Devil Facial Tumor Disease has wiped out 80 percent of the marsupials over the last two decades, and there is no cure or vaccine.

A team at La Trobe University in Australia has developed big plans to derive induced pluripotent stem cells from the devils, which could help turn the tide that has endangered the remaining population of the animals.

The team, lacking funding, has turned to crowdfunding to continue the work, already underway for three years.

The campaign is called See No Devil, Hear No Devil and its goal is to benefit both the ferocious marsupials and humans alike.

The derivation of iPS cells from the Tasmanian Devil is just the first step in a long line of potential applications, Ismael Aguirre Maclennan, a doctoral student behind the work, told Laboratory Equipment. Ultimately, all these goals will increase our understanding of transmissible cancers and potentially uncover unknown mechanisms of cancer transmission that could be applicable to humans.

The stem cells are made by collecting a tiny fragment of the skin through an ear punch. The skin cells are then grown in the laboratory and coaxed into an embryonic-like state by an introduction of a specific set of genes.

Multiple objectives are identified from the team, from applications that would save the devils (currently endangered), to applications human medicine.

The team has so far been the first to generate stem cell lines from any marsupials, they said.

The devils iPS cells could be used to make gametes that would bring the species back from extinction, Aguirre Maclennan explains. The cells could also be genetically edited through CRISPR to provide resistance to the tumors. Additionally, the creation of the cancer cells could provide an in-depth look, allowing scientists to crack its genetic and pathological code.

Proposed in the $15,000 (Australian) campaign is a series of trips to collect samples from healthy Tasmanian devils in the wild and at a sanctuary in Melbourne. The price tag included buying an Engel portable fridge to ensure integrity of the samples, and a laptop and software to helm the campaign in the field. But some 67 percent of the costs would be for laboratory supplies needed to culture the cells and cryogenically preserve them, Aguirre Maclennan explained.

Exceeding the goal could mean working toward production of a stem cell vaccine against the deadly face cancer, according to the team.

We have special interest in using these marsupial stem cells to help save the highly endangered Tasmanian devil from possible extinction but we also seek to exploit the power of marsupial stem cells in biomedical research, said Aguirre Maclennan. We cannot, however, continue with our work due to the lack of funding and therefore this exciting progress may need to be halted permanently.

If you want to fight cancer while saving an endangered species, we wholeheartedly welcome your support, he added.

The campaign has so far raised about 10 percent of its goal through nine backers. But it continues through the weekend.

Amid tighter competition for funds, more and more laboratories and academic programs have turned to crowdfunding.

Read more:
Crowdfunding Campaign Seeks to Save Tasmanian Critter, Use iPS Cells to Research Cancer - Laboratory Equipment

Recommendation and review posted by simmons

The skin grafts that genetically modified have then given to mice that fed high-fat diets to induce obesity.

A new form of gene therapy administered through skin transplants can help improve treatments for Type-2 diabetes and obesity, researchers have claimed. These mice saw a reverse in insulin resistance and gained around half as much weight as those not given the grafts, Engadget said.

Using high-tech gene therapy to get the same result seems unlikely, said Dominguez-Bendala, an associate professor at the University of Miami's Diabetes Research Institute.

Xiaoyang Wu: We have been working on skin somatic stem cells for a long time.

In the study, Wu and colleagues worked with skin because it is a large organ and easily accessible.

In fact, skin cell transplants look to be an ideal way to deliver gene therapy. Clinical translation of our findings will be relatively easy, as skin transplantation in human patients has been well established and clinically used for treatment of burn wounds for many years.

Using CRISPR, researchers from the University of Chicago edited the skin stem cells from newborn mice which prompted the cells to secrete glucagon-like peptide 1 (GLP1) - a hormone that stimulates the pancreas to secrete insulin and regulates blood sugar.

The extra insulin removes excessive glucose from the bloodstream, preventing the complications of diabetes. The hormone can also decrease appetite. This switch turns the gene on, as needed, to make more GLP-1. Using the genetic engineering tool CRISPR, the team inserted a mutation, adding an antibody fragment to the gene that would make the GLP-1 last longer in the blood and an additional modification to the targeting vector that would also attach an inducible promoter.

Wu and colleauges used CRISPR to make specific edits in GLP1, including one that allowed the gene to be turned "on" or "off" as needed, by using the antibiotic doxycycline. As one of the most studied adult stem cell systems, skin stem cells have several unique advantages as the novel vehicle for somatic gene therapy.

The hormone, which is normally produced in the digestive tract, spurs the production of insulin - the body's key regulator of blood sugar levels. About 80% of the engineered skin grafts successfully transplanted onto a small spot on each mouse host's back and began secreting GLP-1 upon the appropriate induction cue. Animals fed on a high-fat diet also did not put on any weight. "Or it could function as a metabolic sink, removing various toxins". He said it raises the possibility that "therapeutic skin grafts" could be used to treat a range of diseases - from hemophilia to drug dependence. The team has demonstrated that skin transplants are not only an efficient way to deliver gene therapy, but that the process can be effectively triggered by an external chemical source.

Follow this link:
Artificial skin transplants could help treat diabetes - ExpressNewsline

Recommendation and review posted by simmons

Agilis Biotherapeutics, Inc. (Agilis), a biotechnology company advancing innovative DNA therapeutics for rare genetic diseases that affect the central nervous system (CNS), and Gene Therapy Research Institution Co, Ltd. (GTRI), a corporation with the mission of developing and delivering of the safest and most efficient gene therapies, announced that the companies have completed a manufacturing and collaboration partnership joint venture (JV) to advance adeno-associated virus (AAV) gene therapies. The JV was initiated earlier this year in connection with a grant from the Japanese Ministry of Trade, Economics and Industry (METI) and Japan External Trade Organization (JETRO) for the development of a state-of-the-art AAV manufacturing facility in Japan. GTRI was co-founded by Professor Shin-ichi Muramatsu, M.D., a leading pioneer in gene therapy who has performed basic science and clinical research in the field for over two decades.

The JV, headquartered in Japan, will initially focus on developing and manufacturing AAV gene therapy vectors using Sf9 baculovirus and HEK293 mammalian cell systems and operate a process development and production facility located in the Tokyo area designed to meet international manufacturing standards, including cGMP, GCTP and PIC/S GMP requirements. Agilis and GTRI will also collaborate to expedite the development, approval and commercialization of select gene therapies in specific CNS diseases. Terms of the joint venture were not disclosed.

We are pleased to collaborate with Agilis to leverage each organizations capabilities and know-how, advance the manufacturing state-of-the art for gene therapy, and develop novel gene therapies, commented Katsuhito Asai, Chief Executive Officer of GTRI and a Director of the joint venture. Our partnership will seek to capitalize on the strong recent progress in the field of gene therapy and expedite the development of innovative gene therapies for patients in need, with a major emphasis on the quality production of safe, effective therapeutics.

We are thrilled to partner with GTRI, said Mark Pykett, Agilis CEO and a Director of the joint venture. We believe that our partnership will enhance the efforts of both organizations, build important shared production capabilities, and accelerate development and commercialization of important gene therapies. We look forward to working with GTRI on a range of initiatives.

View post:
Agilis Biotherapeutics, Gene Therapy Research Institution Enter Strategic Partnership - Drug Discovery & Development

Recommendation and review posted by sam

An international team of researchers recently published, in the journal Nature, their study using genome editing to correct a heterozygous mutation in human preimplantation embryos using a technique called CRISPR-Cas9. This bench research, while far from bedside use, raises questions about the medical ethics of what could be considered genetic engineering. The AMA Code of Medical Ethics has guidance for physicians conducting research in this area.

In Opinion 7.3.6, Research in Gene Therapy and Genetic Engineering, the Code explains:

Gene therapy involves the replacement or modification of a genetic variant to restore or enhance cellular function or the improve response to nongenetic therapies. Genetic engineering involves the use of recombinant DNA techniques to introduce new characteristics or traits. In medicine, the goal of gene therapy and genetic engineering is to alleviate human suffering and disease. As with all therapies, this goal should be pursued only within the ethical traditions of the profession, which gives primacy to the welfare of the patient.

In general, genetic manipulation should be reserved for therapeutic purposes. Efforts to enhance desirable characteristics or to improve complex human traits are contrary to the ethical tradition of medicine. Because of the potential for abuse, genetic manipulation of nondisease traits or the eugenic development of offspring may never be justifiable.

Moreover, genetic manipulation can carry risks to both the individuals into whom modified genetic material is introduced and to future generations. Somatic cell gene therapy targets nongerm cells and thus does not carry risk to future generations. Germ-line therapy, in which a genetic modification is introduced into the genome of human gametes or their precursors, is intended to result in the expression of the modified gene in the recipients offspring and subsequent generations. Germ-line therapy thus may be associated with increased risk and the possibility of unpredictable and irreversible results that adversely affect the welfare of subsequent generations.

Thus, in addition to fundamental ethical requirements for the appropriate conduct of research with human participants, research in gene therapy or genetic engineering must put in place additional safeguards to vigorously protect the safety and well-being of participants and future generations.

Physicians should not engage in research involving gene therapy or genetic engineering with human participants unless the following conditions are met:

(a) Participate only in those studies for which they have relevant expertise.

(b) Ensure that voluntary consent has been obtained from each participant or from the participants legally authorized representative if the participant lacks the capacity to consent, in keeping with ethics guidance. This requires that:

(i) prospective participants receive the information they need to make well-considered decisions, including informing them about the nature of the research and potential harms involved;

(ii) physicians make all reasonable efforts to ensure that participants understand the research is not intended to benefit them individually;

(iii) physicians also make clear that the individual may refuse to participate or may withdraw from the protocol at any time.

(c) Assure themselves that the research protocol is scientifically sound and meets ethical guidelines for research with human participants. Informed consent can never be invoked to justify an unethical study design.

(d) Demonstrate the same care and concern for the well-being of research participants that they would for patients to whom they provide clinical care in a therapeutic relationship. Physician researchers should advocate for access to experimental interventions that have proven effectiveness for patients.

(e) Be mindful of conflicts of interest and assure themselves that appropriate safeguards are in place to protect the integrity of the research and the welfare of human participants.

(f) Adhere to rigorous scientific and ethical standards in conducting, supervising, and disseminating results of the research.

AMA Principles of Medical Ethics: I,II,III,V

At the 2016 AMA Interim Meeting, the AMA House of Delegates adopted policy on genome editing and its potential clinical use. In the policy, the AMA encourages continued research into the therapeutic use of genome editing and also urges continued development of consensus international principles, grounded in science and ethics, to determine permissible therapeutic applications of germline genome editing.

Chapter 7 of the Code, Opinions on Research & Innovation, also features guidance on other research-related subjects, including informed consent, conflicts of interest, use of placebo controls, and the use of DNA databanks.

The Code of Medical Ethics is updated periodically to address the changing conditions of medicine. The new edition, adopted in June 2016, is the culmination of an eight-year project to comprehensively review, update and reorganize guidance to ensure that the Code remains timely and easy to use for physicians in teaching and in practice.

Here is the original post:
Genome editing and the AMA Code of Medical Ethics - American Medical Association (blog)

Recommendation and review posted by sam

In BriefThis is truly the golden age of anti-aging research, with extended telemores, senescent cell therapies, and young-blood transfusions being three of the most promising treatment avenues. However, even if one of these therapies proves to be the proverbial "fountain of youth," the financial cost of a long, healthy life is still well out of reach for most people.

The idea of never growing old is seductive, but it has remained a pipe-dream throughout history. However, that may not be the case for much longer as the scientific community has seen a surge in anti-aging research in recent years.All across the globe, researchers are now exploring different methods to combat aging and extend human health span (the number of years of good health a person experiences).

The avenue that is arguably generating the most support involves telemores. These are the caps that sit on the ends of chromosomes. They provide protection for the DNA molecules, and their length has been linked to good health. Unfortunately, they shrinkwith every division until they can no longer protect the cell and it dies or damages surrounding cells through senescence.

So far, the research on telemores has been promising.Maria Blasco of the Spanish National Cancer Research Centre used gene therapy to extendthe telemores in mice, which led to a 40 percent increase in lifespan.

Meanwhile Helen Blau, Director of the Baxter Laboratory for Stem Cell Biology at Stanford, modified the RNA of skin cells to increase telemore length. This caused the cells to divide up to 40 moretimes than their untreated counterparts did before dying or stagnating.

Another promising avenue of anti-aging research involved targeting senescent cells. These cells pump out chemicals as they deteriorate that are damaging to their neighboring cells, causing many of the diseases associated with aging, so researchers have been looking for ways to either inhibit their development or periodically purge them.

At the Mayo Clinic in Rochester, Minnesota,Darren Bakerand his colleagues found that giving mice a drug that destroyed these cells delayed the development of the diseases of aging, as well as made the mice look plumper and younger.

At the slightly more unsettlingend of the anti-aging treatment spectrum is the process of transfusing the blood of the young into the old. Despite the vampiric and macabre nature of the treatment, researchers have found evidence that it is effective. Individuals who receive blood from younger donors report health benefits, such as lowered cholesterol levels, while older mice have been shown to be rejuvenated by injections of blood from younger mice or evenhuman teenagers.

While science is movingquickly toward a future in which aging and its consequences are obsolete, the few commercial means of receiving the treatments above are, at present, extremely expensive.

Liz Parrishis not a biologist by training, but she did enlist the help of scientists to develop the telemore-based treatment offered by her company,BioViva. Ostensibly, Parrish has developed an injection based on Blancos principles, andshe herself is patient zero, having already injected herself with that telomere-extending treatment as well as one designed to preserve muscle mass. While BioVivahasnt gone to market yet, Parrish told New Humanist that eachinjection costs between $200,000 to $400,000 to produce.

While no commercial means of senescent cell therapy exists as of yet, individuals can buy young blood transfusions. Jesse Karmazins company Ambrosia offers blood plasma transfusions for anyone willing to pay $8,000.

However, Stanford University neuroscientist Tony Wyss-Coray, who has conducted numerous experiments on mices reaction to young blood, thinks youd be better off saving your money. He dismisses the science behind the treatment,telling MIT Technology Review that people want to believe that young blood restores youth, even though we dont have evidence that it works in humans.

For the moment, anti-aging therapies are attainable in theory, but well out of financial reach for all except a wealthy few. Once the science is crystallized, however, the treatments should become exponentially cheaper, and a long, healthy life will be neither a pipe dream nor a hideously expensive commodity.

Read this article:
Researchers Are Finding Remarkable Ways to Combat Aging and Extend Human Health - Futurism

Recommendation and review posted by simmons

Affimed Therapeutics (NASDAQ:AFMD)

Q2 2017 Earnings Conference Call

August 1, 2017 8:30 AM ET

Executives

Anca Alexandru Head of Communications

Adi Hoess Chief Executive Officer

Florian Fischer Chief Financial Officer

Analysts

Maury Raycroft Jefferies

Do Kim BMO Capital Markets

Michael Schmidt Leerink

Peter Lawson SunTrust

Operator

Good day and welcome to the Affimed Second Quarter 2017 Financial Results and Corporate Update Conference Call. Todays conference is being recorded. At this time, I would like to turn the conference over to Anca Alexandru. Please go ahead.

Anca Alexandru

Thanks. I would like to welcome you to our investor and analyst call on the results for the second quarter of 2017. On the call with me today are Adi Hoess, CEO of Affimed, who will present the corporate update; and Florian Fischer, Affimeds CFO, who will walk you through the financials.

Slide 2, before we start, please note that this call and the Q&A session contains forward-looking statements, including statements regarding our future financial condition, business strategy, and our plans and objectives for our future operations.

These statements represent our beliefs and assumptions only as of the date of this discussion. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

These forward-looking statements are subject to risks and uncertainties and actual results may differ materially from those expressed or implied in the forward-looking statements due to various factors including, but not limited to those identified under the section entitled risk Factors in our filings with the SEC and those identified under the section entitled cautionary statements regarding forward-looking statements in our Form 6-K filed with the SEC earlier today.

Thank you for your understanding. I will now hand the call over to our CEO, Adi Hoess, who will provide the corporate update.

Adi Hoess

Thanks a lot, Anca. Affimed has developed an immune cell engager and our clinical and preclinical pipeline based on tetravalent bi and trispecificantibody formats. Were an industry leader in NK cell engagement and our lead product candidate AFM13 is to our knowledge, the most advanced NK-cell engager in clinical development.

We also have a well-differentiated T-cell based approach, which includes our clinical candidate AFM11 and well provide an update on these clinical programs as well as our pre-clinical programs today. We employ about 75 full time equivalents with our headquarter located in Heidelberg, Germany, and affiliate offices in the U.S., that is Affimed Inc., as well as our subsidiary AbCheck in Plze, in the Czech Republic.

Slide 4. We have an unencumbered clinical and pre-clinical pipeline of NK and T-cell engagers, with our NK-cell engagers being developed in hematological diseases and solid tumors. Based on our NK-cell platform, we have one clinical and two pre-clinical programs in developments. And based on our T-cell platform, we have one program in our own clinical development. And second T-cell engager program based on our platform called AMV564 is being developed by Amphivena, a company of which we own about 18.5% fully diluted. AMV564 has recently entered clinical development.

Slide 5 summarizes our second quarter updates for our NK cell engager program. For AFM13, we have completed the dose escalation part of our Phase 1b combination study with Mercks Keytruda in Hodgkin Lymphoma and initiated the expansion phase. The AFM13 Phase 2a monotherapy trial in Hodgkin Lymphoma sponsored by the German Hodgkin Study Group is open to recruit under new study design, which includes patients pre-treated with both brentuximab vedotin and anti-PD1.

Columbia University has recently initiated a translational study of AFM13 in CD30-positive lymphoma with cutaneous manifestation and I will provide more detail later. We made further progress in our collaboration with MD Anderson Cancer Center to evaluate AFM13 in combination with MD Andersons NK cell product. In June, we presented new data for our NK cell engagers AFM24 and AFM26 at two conferences and I will go into detail later on this.

Slide 6 summarizes the progress, we have made with our T-cell engager. Two Phase 1 dose-escalation studies are ongoing with AFM11, which offer a significant opportunity to address the high unmet medical need in diffuse large B-Cell lymphoma and mantle cell lymphoma. We believe that both the properties of AFM11 and the design of our studies can attract, specifically, mutations of other drugs in development.

Both dose escalation studies, which are conducted in ALL and in NHL respectively are designed with accelerated titration followed by a classical 3+3 design. In both studies, AFM11 was overall well tolerated with no dose limits and toxicity observed to date. In the AFM11 study in relapsed refractory ALL, which was initiated in September 2016, patients are currently being recruited into the fourth dose cohort. 12 sites are open and recruiting in the Czech Republic, Poland, Russia, Austria and Israel.

As mentioned, no DLTs were observed in particular no AFM11 related grade 3 or grade 4 neurotoxicity or side effects are frequently observed with T-cell engaging antibody agents observed. In our study of AFM11 in relapsed refractory NHL, patients are currently being recruited into the third dose cohort. Recall that this study has been amended in the past to enroll patients under a new revised study design.

We believe that we have addressed this lower than effective recruitment by opening further trial sites. A total of 10 sites are now opened in the Czech Republic, Poland, Germany as well as the U.S. Like in our ALL trial, no AFM11 related grade 3 or grade 4 neurotoxicity was observed to date under their revised study design. We intend to provide regular update on both the AFM11 studies in the future.

A second T-cell engager program based on our platform is AMV564, a bispecific tetravalent CD33/CD3 antibody developed by Amphivena in AML. A Phase 1 study is recruiting. However, no further updates have been provided by Amphivena.

Slide 7 shows our platform, which is very distinguished from others as, in contrast, the most comparatives were developing tetravalent bispecific molecules. The bivalent binding of two receptors on two different cells enables high-affinity binding through the avidity effect, which is advantageous to maintain high specificity at very high affinity.

We believe that this is very important in order to obtain a favorable safety profile. Furthermore, our platform allows multi-specificity in the tailored PK. Further differentiating Affimed, while most immune cell engaging approaches to date focus on T cells, our technology platform reliably generates both T and NK-cell engagers.

While increasingly NK-cells are becoming a cornerstone of cancer immunotherapy, and we're excited to be pioneering this development. There are a number of reasons why NK-cell-based approaches are very attractive and one of the reasons is that there seems to be a positive correlation between NK-cell infiltration and clinical outcome in patients.

In this context, it has been described that a low cytotoxicity is associated with higher incidence of cancer. In addition, recent clinical data show improved anti-tumor responses of ex vivo expanded and activated NK-cell populations. NK-cell-based immunotherapy has recently advanced with different treatment approaches, including engagers, check points, cytokines and adoptive cellular transfer.

To-date, it seems that NK-cell-based approaches have this strong advantage of controlling a well manageable favorable safety profile. This creates an opportunity for NK-cell redirection to address the lack of recognition of cancer cells and also allows for potential combination of NK-cells with other approaches to enhance efficacy.

A common theme in all different cancer types is the ability of the tumor cell to evade recognition by the immune system and, specifically, by NK-cells as shown on Slide 9. Normally, NK-cells are capable of killing foreign or aberrant cells, like tumor cells, have acquired mechanisms to escape the so-called immune surveillance.

As a result, such NK-cells cannot recognize tumor cells as foreign or aberrant and, therefore, cannot fight them. We believe that our platform has the potential to overcome these limitations by disabling the tumor evasion mechanisms, and I will explain on the next slide what this belief is based on.

Our expertise and leadership in natural-killer cell-based approaches is one of our key assets. As we can see here, there are a multitude of activating and inhibitory NK-cell receptors being discovered that CD16A, a dominant activating receptor on innate immune cells, is the only activating receptor that triggers the cytotoxic activity of nave human NK cells, even in the absence of costimulatory signals.

Based on these properties and on our preclinical and clinical data generated to date, we believe that targeting CD16A is key for efficient recruitment of and killing by NK cells and macrophages. We have secured a solid IP position around CD16A targets.

Slide 10. We believe that through targeting CD16A with high affinity and specificity, the significant limitations of IgGs can differ. With our tetravalent bispecific immune cell engagers, we can restore NK-cell killing in tumor immune control, and this is depicted here.

Let me explain in more detail why we believe that our approach is superior compared to IgG-based approaches. The human body is not using NK-cell engagement by IgG to eliminate cancer cells. However, this mechanism is used for cells infected by viruses or bacteria.

In this situation, the human immune system generates a collagen antibody response that highly decorates such infected cells or organisms.

Highly decorated means that many different proteins are expressed on the cell surface, which can then be found bound by antibodies. This polyclonal and high-density binding leads to NK-cells killing upon high avidity XP binding, plus antibodies for CD16A on the NK-cell and other XP gamma receptors, for example, CD32 and CD64.

In the setting of targetable cancer cells, however, with IgG, the situation is very different. Firstly, the therapeutic molecule targets a single epitope. Hence, it confers ammonia killing response. And secondly, there are cancer cells which express only very low numbers of the desired target.

The consequence of this very low target density is an insufficient amount of IgG, decorating the cancer cells and thereby not being able to efficiently recruit immune cells. This is shown in the middle picture. Interesting, most therapeutic monoclonal antibodies are target-modulating antibodies, such as cetuximab, polatuzumab, gevokizumab, just to mention a few of them.

We are addressing this limitation by targeting CD16A with high affinity and specificity, as shown. Indeed, our immune cell engagers has the potential to elicit a robust NK-cell killing and immune control due to multivalent and apparent high-affinity binding to CD16A even at limiting antigen densities on the target.

Slide 11, furthermore, CD16A in confers additional superior engager features. The binding of immune cells through CD16A with high affinity and specificity induces NK-cell activation, which triggers an integrated immune response that can be mediated by both innate and adaptive immune cells. In particular, our NK-cell engagers do not bind to CD16B and neutrophils, which avoids the sync effect. Their affinity has been demonstrated to be over 1,000 fold higher than that of monoclonal antibodies and our engagers bind independently of the 158 valine phenylalanine polymorphism.

Most importantly, theres virtually no competition with plasma IgG, which is shown here. In the ground stage, CD16A on innate immune cells is occupied by polyclonal plasma IgG. But there is a huge excess of plasma IgG versus therapeutic antibodies, this creates a significant threshold for FC-based therapeutic antibodies, however, not for CD16A target enhancement.

Our tetravalent and bispecific molecules, which recognize a different epitope from CD16A, are virtually unaffected by plasma IgG. All these unique features result in overall increased potency and efficacy of NK-cell engagers.

Slide 12. Our lead candidate, CD30/CD16A-specific NK-cell engager, AFM13 is a first-in-class antibody suitable for mono and combination therapy. This has demonstrated safety and clinical activity in heavily pretreated Hodgkin lymphoma patients in a Phase 1 study. In this Phase 1 study, tumor shrinkage and potential responses were observed in patients treated with four weekly doses of at least 1.5 mg/kg of AFM13. In 62% of patients, which was eight out of thirteen patients, we observed tumor shrinkage in 23% of patients, which was a total of three out of thirteen experienced partial response. None of the patients experiencing a PR had been previously treated with brentuximab vedotin.

Recall, that in our investigation the Phase 2a trial for AFM13 in relapsed and refractory Hodgkin lymphoma, which is led by the German Hodgkin Study Group, we have previously guided to change the study protocol to ensure a recruitment of a homogeneous patient population pre-treated with both BV and anti-PD1 antibodies. The study is now open to recruit under the new study design.

We had also provided some preliminary data from patients enrolled under the original study protocol, where partial responses were observed in two of seven evaluated patients who had been pre-treated with brentuximab vedotin, but were anti-PD1 naive. This suggests, now, for the first time that AFM13 is active as a single agent in this heavily pre-treated group of patients and, in particular, that AFM13 is active post brentuximab vedotin. We have learned from the study sponsor that both after-responders had failed BV as the most recent treatment prior to AFM13 therapy, with one patient experiencing stable disease and the other one partial in the progressive disease under the BV treatment.

As previously guided, full data from the ongoing study will be presented upon its anticipated completion in 2019. And prior to that, a decision of data publication time points will be made together with the German Hodgkin Study Group.

We are further developing AFM13 as a combination therapy. Preclinical affinity has been demonstrated in combination with anti-PD1 in vivo in a PDX model. This has been the basis of our Phase 1b trial in relapsed refractory Hodgkin lymphoma in combination with Mercks Keytruda. And here, we have completed the dose escalation part of the trial. In detail, three patients were enrolled into dose levels one and two, respectively, and six patients were enrolled into dose level three. While no grade three or four adverse events related to the study treatment were observed, one DLT was observed in cohort 3, which was a repeated grade two infusion-related reaction, leading to discontinuation of AFM13 treatment. This event is classified as a DLT according to the protocol definition. No further DLTs occurred.

The dose expansion cohort has been initiated with the highest dose explored during dose escalation. Data readout is ongoing in the treated cohort and we intend to present data from the dose escalation at a scientific medical conference in the second half of 2017.

Another update this quarter is that Columbia University has initiated a translational Phase 1b/2a study to evaluate the validity of activity of AFM13 in patients with relapsed and refractory CD30-positive lymphoma with cutaneous manifestation. Affirmed is supporting this trial which is designed to allow for serial biopsies, thereby enabling assessment of NK-cell biology and tumor cell killing within the tumor environment. The first patient was enrolled into the study in July 2017. In general, we view CD30-positive lymphoma as an attractive indication that may broaden the potential of AFM13. In terms of further guidance, we will work together with Columbia University to provide update on this study.

Slide 13. Additional opportunities for our NK-cell engagers include combinations with adoptive NK-cell transfer. Patients on NK cells can be stimulated by monotherapy using NK-cell engagers to overcome tumor immune evasion and immunosuppression. Ex vivo expansion and stimulation of autologous NK-cells followed by reinfusion alone or in combination with NK cell engager, is a viable therapeutic approach providing increased numbers of activated NK cells. Alternatively, NK cells can be derived from peripheral blood, cord blood or IPS cells from healthy donors, which is an allogeneic setting, or from immortalized cells. After ex vivo stimulation and expansion, the NK cells are infused into the patients in combination with NK cell engagers.

We are investigating this approach with our partner MD Anderson. Initially, we plan to investigate AFM13 with MDACCs NK-cell product in the transplant setting. Preclinical research activities are on track and these are intended to be followed by Phase 1 clinical trial. Proof-of-concept for this combination would also pave the way for combinations of other pipeline product such as for AFM23.

Affimed holds an option to exclusive worldwide rights to develop and commercialize any product developed under the collaboration. In addition to our clinical product candidates, we have created a strong preclinical pipeline. Over the last quarter we have further characterized our most advanced preclinical candidates, AFM24 and AFM26, which we are developing for three solid tumors and multiple myeloma respectively.

Despite several marketed agents such as cetuximab and tyrosine kinase inhibitor or TKIs, there is a significant medical need for a novel approach to treat EGF receptor-positive tumor. Both efficacy and toxicity can be addressed. EGFR-blocking drugs have been described to have side-effects including serious skin toxicity which might impact physicians willingness to prescribe a drug. In terms of efficacy, there is a need to overcome intrinsic or acquired resistance. For example, there is no clear indication of efficacy of EGFR-blocking antibodies in patients with RAS mutation.

We are developing a first-in-class NK cell engager designed to overcome the limitations of conventional therapy. AFM24 is designed to effectively treat EGFR-expressing solid tumors, such as lung and neck, or colon cancers. It is an EGFR/CD16A targeting tetravalent bispecific antibody that is well differentiated from cetuximab, it is more potent cytotoxicity in vitro and in vivo including a potential to kill RAS-mutant cell lines. There is novel mechanism of action in safety profile and it has the potential to overcome intrinsic or acquired resistance, which is described by many patients with EGFR positive tumors.

AFM24s potent NK cell recruitment may enable the shift of the validated target EGF receptor, primary receptor block toward immuno-oncology. We have identified several development candidates for which we have initiated IND-enabling studies.

Slide 15, there are several factors which differentiate AFM24 from other therapy. Firstly, AFM24 is differentiated through its efficacy. Here you can see that in vitro, our NK cell engager which is highly potent tumor cell killing independent of RAS mutational status. In vivo, we have demonstrated efficacy in tumors resistant to EGFR targeting agents. Importantly, as shown in the graphs on the right hand side, AFM24 was similarly efficacious in a cetuximab-sensitive model.

Secondly, AFM24 is differentiated through safety, Slide 16. We have completed pilot toxicity studies in cynomolgus monkeys with no major safety findings. At the EACR-AACR-SIC Special Conference, we presented data on a dose-range binder study in which AFM24 was dosed up to 93.75 mg/kg and a repeated dose study in which AFM24 was dosed up to 30 mg/kg in 4 weeks.

No AFM24-related macro or microscopic changes were seen in tissues including vital organs, skin and injection site. Importantly, there was no evidence of skin toxicity in those studies. Also no signs of delayed toxicity was observed in the repeated dose study recovery animals. On a molecular level, we learned from in vitro toxicology studies but there was no cytokine release or NK cell proliferation in the absence of target cells. This further substantiates AFM24s potential beneficial safety profile.

Slide 17, like for EGFR targeted tumors, there is a significant need for a novel approach to treat multiple myeloma. Even though, new therapies have significant improved outcomes, cure still remains elusive and the medical need to achieve minimal residual disease negativity is not yet addressed.

MRD positivity is associated with a poorer prognosis, and it has been recorded that persistent MRD by predictive marker of unsustained complete response. A particular hurdle for therapeutics aimed at immune cell engagement are very high M-protein serum levels up to 170mg/mL. Indeed the competition by serum IgG is known to strongly impair antibody-dependent cell-mediated cytotoxicity, the activity of monoclonal antibodies.

We are developing AFM26 to overcome the limitations of conventional therapies in multiple myeloma. AFM26 is a first-in-class tetravalent bispecific antibody targeting BCMA/CD16A. Targeting BCMA and employing NK cell engagement offers the potential to achieve MRD-negativity. For AFM26, NK cell binding is largely unaffected by circulating IgG, which creates the potential of NK cell activation in the presence of M-protein.

Indeed, the high affinity binding to both target and NK cells leads to a prolonged cell retention. This is shown on the right on the slide on the right bottom. AFM26 shows high cytotoxicity cytotoxic activity towards both low and high BCMA-expressing myeloma cells. AFM26 may be potentially safer than T cell-based approaches, which would allow for faster development timelines. Based on these characteristics, AFM26 might be positioned in first line of combination with adoptive NK-cell transfer during ASCT or in a salvage setting.

AFM26 binds the B-cell maturation antigen, which is an antigen ubiquitously expressed on malignant plasma cells. Its expression on healthy tissues is limited to plasma cells and peripheral dendritic cells. We believe the BCMA is an ideal target for immunotherapy of multiple myeloma.

At ASCO and at the EACR-AACR-SIC, both in June, well present the data on AFM26 NK-cell binding properties and activity. As shown here, these data underscore that compared to native and FC-enhanced IgG. AFM26 shows improved binding and cell surface retention.

Slide 20, we also show that AFM26 is well differentiated through target cell binding in potent NK-cell mediated tumor cell lysis. And this is shown here in comparison with two marketed agents, daratumumab and anti-CD38 antibody and elotuzumab, which targets PS1. Importantly, other than described for daratumumab and elotuzumab, AFM26 did not induce NK-cell mutation.

Slide 21, like our other NK-cell engagers, AFM26 is also well differentiated for other agents [indiscernible] safety. Here you can see that compared to a T-cell engager, AFM26 is similarly potent that shows a reduced cytokine release pattern. This point is going to improve safety profile, making AFM26 uniquely suited to engage NK-cells with multiple myeloma.

I will now hand over the call to our CFO, Florian Fischer, who will provide further details on the financial figures.

Florian Fischer

Thank you, Adi. Affimeds consolidated financial statements have been prepared in accordance with IFRS as issued by the International Accounting Standards Board or IASB. The consolidated financial statements are presented in euro, which is the companys functional and presentation currency. Therefore, all financial numbers that I will present here in this call unless otherwise noted will be in euros. Any numbers referring to Q2 2017 and Q2 2016 are unaudited.

Cash and cash equivalents and financial assets totaled 48.9 million as of June 2017 compared to 44.9 million as of December 31, 2016. The increase was primarily attributable to the net proceeds of 16.4 million from a public offering of common shares in the first quarter, and of 2.5 million from the drawdown of the second tranche of the loan from Silicon Valley Bank, largely offset by operational expenses.

Net cash used in operating activities was 13.1 million for the six months ended June 30, 2017compared to 17 million for the six months ended June 30, 2016. The decrease was primarily related to lower cash expenditure for research and development in connection with Affimeds development and collaboration programs and to the expiration of the Amphivena collaboration.

Affimed expects to have cash to fund our operations at least until the end of 2018. This provides runway for the planned development of our clinical programs, as well as for product discovery and early development activity.

Revenue for the second quarter of 2017 was 0.5 million compared to 2.1 million for the second quarter 2016. Revenue in the 2017 period was primarily derived from AbCheck services, while revenue in 2016 period predominantly to Affimeds collaboration with Amphivena.

R&D expenses for the second quarter of 2017 were 5.4 million compared to 8.6 million for the second quarter of 2016. The decrease was primarily related to lower expenses for AFM13 and our discovery and early stage development activities and the expiration of the Amphivena collaboration.

G&A expenses for the second quarter of 2017 were unchanged at 2.0 million compared to the second quarter of 2016. Net loss for the second quarter of 2017 was 7.9 million, or 0.18 per common share, compared to a net loss of 8 million or 0.24 per common share for the second quarter of 2016.

The decrease of operating expenses was offset by lower revenue. In addition, the result was affected by finance costs of 1.2 million in the second quarter of 2017, whereas finance income of 0.5 million was shown in the second quarter of 2016.

I will now turn the call back over to Adi for a summary of our two clinical programs and our pipeline. Adi?

Adi Hoess

Thanks a lot Florian. Our strategy is to maximize the value of our unencumbered clinical and preclinical pipeline of NK-cell and T-cell engagers, as well as from our platform. Were leveraging our lead product, AFM13, for CD30-positive lymphoma initially focusing on the Hodgkin Lymphoma salvage setting enabling a fast development path and allowing the establishment of a cost efficient marketing and sales structure.

In addition, we believe investigating AFM13, both as monotherapy and in combination with Keytruda, reduces its development. Overall, our preclinical and clinical strategy is designed from the scientific leadership of our NK-cell platform with CD16A as proprietary target. We are expanding the preclinical and clinical activities of our tetravalent and bispecific NK-cell engager platform in solid tumors with our preclinical candidate AFM24 and in hematologic diseases, where we intend to leverage additional opportunities for AFM13 and AFM26, for example, in combination with adoptive NK-cells. We also develop T-cell engagers and our lead T-cell engager, AFM11, is being investigated in two ongoing ALL and NHL trials. BMV564, a T-cell engager derived from our technology platform, is in clinical development through Amphivena to treat AML.

In addition, as mentioned earlier, moving beyond our standard format, we are developing different tetravalent bispecific antibody formats tailored to specific indications and patient populations. And as outlined in previous earnings calls, we have more projects ongoing at the discovery stage and preclinically, including molecules developed from our MHD type complex targeting platform.

Thank you very much for your interest. The call is now open for questions.

Question-and-Answer Session

Operator

Thank you. [Operator Instructions] Our first question now comes from Maury Raycroft from Jefferies. Please go ahead.

Maury Raycroft

Good morning. Thanks for taking my questions. So I was wondering if you can mention what the AFM13 dose was that the DLT patient received in the combo trial? And then what youre going to use in the expansion cohort? And then is this dose higher, lower or in line with your predictions?

Adi Hoess

Hi, Maury, this is Adi. What we have done is we have used or given a PD-1 as its active dose and have dosed up AFM13 under the following strategy, under the following regime. We always are initially giving AFM13 three times per week for two weeks. Then, we give AFM13 once weekly for six weeks and, subsequently, we dose AFM13 every three weeks. The starting dose was 0.15 mg/kg and then switching to 0.5 mg/kg, the next one was 0.5 mg/kg going to 1.5 mg/kg, and the highest dose was 3 mg/kg going then to 7 mg/kg. So the three is always three times per week, and the seven is the weekly or every three weeks. We have seen 1 DLT in the highest dose. So at three times 3 mg/kg and once 7 mg/kg then have included an additional three patients and have not observed another DLT. So thats why we decided to go with the highest dose of 3 mg/kg three times per week subsequently given and then subsequently followed by 7 mg/kg.

Maury Raycroft

Got it, okay. And you also mentioned earlier about the two PRs generated with the monotherapy treatment? And I think you said there is a stable disease, but I missed some of the additional context, and I was just wondering if you can recap that for me?

Adi Hoess

See the original post:
Affimed Therapeutics' (AFMD) CEO Adi Hoess on Q2 2017 Results - Earnings Call Transcript - Seeking Alpha

Recommendation and review posted by simmons

He played his last game more than five decades ago, and has been dead (or at least frozen) for 15 years, but Ted Williams is still very much alive in the minds of baseball fans.

Producers of the American Masters series announced that the Splendid Splinter will be the subject of an upcoming documentary the first baseball player to be so profiled.

Advertisement

A major American cultural figure whose story has never been properly told, Ted Williams is a fitting first, Michael Kantor, American Masters series executive producer, said in a statement. This film will reveal the man behind the legendary .406 batting average: complex, misunderstood and profoundly human.

Its not exactly true that Williamss story has never been properly told. In recent years, the Hall of Famer was the subject of not one but two excellent biographies, both written by former reporters at the Boston Globe: Ted Williams: The Biography of an American Hero by Leigh Montville and Ben Bradlee Jr.s The Kid: The Immortal Life of Ted Williams.

Get The Weekender in your inbox:

The Globe's top picks for what to see and do each weekend, in Boston and beyond.

The American Masters doc, slated to premiere next summer on PBS in honor of Williamss centennial, is being co-produced with Major League Baseball and David Ortizs Big Papi Productions, among others. The film will look at Williamss incredible baseball career and his service as a decorated combat pilot in the Korean War. Its not clear if the doc will discuss at all the bizarre and troubling disposition of Williams after he died, when his head was removed and frozen at the Arizona-based Alcor Life Extension Foundation, which deep-freezes bodies (or just heads) in the hope that scientific advances will allow them to be revived in the future.

In addition to Williams, the new season of American Masters will feature documentaries about filmmaker Richard Linklater, artist Tyrus Wong, writer Edgar Allan Poe, and entertainer Bob Hope.

See the original post here:
Ted Williams will be first baseball player to get 'American Masters' treatment - The Boston Globe

Recommendation and review posted by simmons

The expiration dates on over-the-counter and prescription medications seem pretty black and white, but there's some question about whether drugs last even longer.

Expiration dates typically range from 12 to 60 months after production. But manufacturers aren't required to determine how long they'll remain potent after that, enabling them to set their own expiration dates and possibly shortchange consumers.

Testing reported inJAMA Internal Medicineshowed that eight medications with 15 different active ingredients were still potent decades beyond their expiration dates.

The U.S. government's own Shelf Life Extension Program extends the dates on some drugs in federal stockpiles to save the military from the cost of replacing them. Its own study found that 90 percent of more than 100 drugs were perfectly good even 15 years after expiration.

But what about the meds in your home?

A lot depends on how carefully you store them -- you probably don't do as good a job as the U.S. Army. That's why the U.S. Food and Drug Administration recommends never taking drugs beyond their expiration date -- it's just too risky. In particular, nitroglycerin, insulin and liquid antibiotics shouldn't be used after their expiration dates.

To safeguard all medications, protect them from heat, light and humidity by keeping them in a cool, dry, dark place. A steamy bathroom isn't a good environment.

Know, too, that some drugs can lose their potency more quickly than others, including aspirin. If you take aspirin for heart health, be sure to replace it as needed.

2017 HealthDay. All rights reserved. This material may not be published, broadcast, rewritten, or redistributed.

Visit link:
Can you take medications past their expiration date? - CBS News

Recommendation and review posted by simmons

On Thursday August 3, the Hopkins County extension agents hosted a fun and fast-moving workshop for Extension leaders. About two dozen individuals and committee members were present, representing various aspects of Extension projects including 4-H, Master Gardener, Wellness /Nutrition and Special Events.

The meetings purpose was to dispense information and to gather information. According to a census of attendees, radio and e-mail are the preferred mediums for receiving information about Extension activities. Agent Johanna Hicks asked to suggest future programs that would fill local needs, and to be willing to mentor new 4H programs that may help youth. One of the guidelines of new programs is an 8-task learning requirement. Reports given to leaders showed that over 500 hours in volunteer services were logged during 2016, with that number is expected to increase for 2017. Another report showed the BLT program, or Better Living for Texans, provides $1200 annually for expenses for local nutrition education programs. Another report showed that the relatively new Fee-Based extension programs require attendees to remit $10 which the College Station headquarters uses to offset wages paid to extension employees. Extension Agent Mario Villarino said that the annual Hay Show, which is an Ag Extension program held in the Fall, is in need of support by new producers as its population is aging.

Those present enjoyed a healthy meal prepared by the agents, and entertaining role-play exercise and door prize give-aways based on facts learned at the meeting. Everyone also met Extension intern Aida Ugalde, who will graduate from Texas A and M Commerce with studies in Health Promotions. The public is invited to get involved with Extension office efforts to serve local needs. The agents welcome suggestions, assistance and leadership by volunteers! Pick up a list of upcoming programs and activities already in place that you can join at the Hopkins County Agri-Life Extension office at 1200 West Houston Street in Sulphur Springs or phone 903-885-3443.

Johanna with Intern Aida Ugalde

See the rest here:
Johanna and Mario Host Extension Leadership Workshop - KSST (press release) (registration) (blog)

Recommendation and review posted by simmons

BWX Technologies subsidiary BWXT Nuclear Energy Canada (BWXT NEC) has secured a new contract to supply seven primary heat transport motors for six nuclear reactors operated by electricity company Bruce Power.

The deal isvalued at C$34m ($27m) andis a part of Bruce Powers life-extension programme, which isexpected to ensure continuous supply of clean, low-cost, and reliable electricity to Ontario, Canada.

BWXT NEC will be responsible for project management, engineering, and manufacturing of the seven 11,000hp motors, which are employed to drive the main circulating pumps used to push heavy water through the reactor core into the steam generators.

The first motor of this order is scheduled to be delivered by mid-2018.

"BWXT NEC will be responsible for project management, engineering, and manufacturing of the seven 11,000hp motors."

Bruce Power president and CEO Mike Rencheck said: Partnering with BWXT NECfor this important motor work is critical to ensuring the life extension and operation through 2064.

Planning and preparation is key to our continued on-time and on-budget performance since January 2016 when our life extension programme was started.

With the new extension, Bruce Power aims to create and sustain 22,000 direct and indirect jobs every year. It is also expected to create C$4bn ($3.1bn) in annual economic benefit for Ontario.

Currently, Bruce Power fulfils 30% ofthe province'selectricity demand.

See the original post here:
BWXT NEC to provide seven heat transport motors for Bruce Power reactors - Power Technology

Recommendation and review posted by sam

Hypopituitarism (dwarfism) is a rare disease that results from the low production of hormones in the pituitary gland. The pituitary gland is located deep within your brain and is an important aspect of the endocrine system.

In children, human growth hormone deficiencies can lead to impaired growth, also known as dwarfism. Early diagnosis and medical intervention can sometimes correct this deficiency, allowing the affected individual to reach a normal or near-normal height.

In addition to stunted growth, hypopituitarism can also cause deficiencies in thyroid or adrenal hormones.

The most common cause of hypopituitarism is a tumor located in or around the pituitary gland. Surgery or radiation therapy near the pituitary gland can also stimulate dwarfism. In some rare cases, illnesses like tuberculosis can cause inflammation that causes hypopituitarism as well.

Besides slowed growth, there are many other symptoms of hypopituitarism:

Diagnosing hypopituitarism can be difficult as it often goes unnoticed early on. During the first two years of life, a child with growth hormone deficiency may grow at a normal rate and may seem perfectly healthy.

As the child becomes older, however, parents may notice that their child does not seem to be growing properly. The child may be smaller when compared to other kids his age and may look younger than them or have different proportions.

A child who grows less than 2 inches per year, or who is only as tall as children two or more years younger, should be evaluated by a physician for growth hormone deficiency after other possible causes, such as hypothyroidism, have been ruled out.

Your child's doctor will look at the patient history, perform a physical exam and may recommend an X-ray to look for the presence of a tumor. He may also perform blood tests to check pituitary, thyroid and adrenal glands.

Testing for growth hormone deficiency is done by stimulating the body to produce the hormone and then measuring how much hormone is actually released.

In most cases, people with hypopituitarism will undergo hormonal therapy throughout their entire lifetimes. Depending on the individual's deficiency, different hormones may be administered.

In order for a more normal height to be achieved, children will need to have human growth hormone replacement therapy. Growth hormone deficiency is treated by injections of a growth hormone preparation such as Humatrope (somatropin). A child may receive daily or weekly injections. The child's growth rate increases soon after the injections are started. The treatment continues over several years until the child's maximum growth potential is achieved. Most children will reach an "acceptable" adult height.

The average adult height for someone with untreated severe growth hormone deficiency is 4 feet, 8 inches in men and 4 feet, 3 inches in women, while those who undergo early treatments will reach more average heights in the range of 5 feet and up.

Sources:

Human Growth Foundation. "Disorders of Growth." 2009.

Levy, Richard. "Growth Hormone Deficiency in Children." The Magic Foundation.2009.

Pituitary Network Association. "Hypopituitarism." 2014.

Go here to see the original:
What Is Hypopituitarism (Dwarfism)? - Verywell

Recommendation and review posted by Bethany Smith

Photo Credit: geralt / Pixabay

Researchers from the Faculty of Health Sciences at Ben-Gurion University of the Negev and the Fertility & In Vitro Fertilization (IVF) Unit at Soroka Medical Center have discovered a new genetic mutation that causes a lack of sperm production.

Five percent of men suffer from infertility, and about one percent suffer from azoospermia, a lack of sperm production, although the reasons for this lack of sperm are still a mystery.

The study was made possible as a result of five men from a single family who were treated at Soroka Medical Center for arrest of sperm in their testes with no obvious cause.

The treatment team at the IVF Unit was led by Prof. Eitan Lunenfeld, chair of the Department of Obstetrics and Gynecology.

Profs. Ruti Parvari and Mahmoud Huleihel from the Shraga Segal Department of Microbiology & Immunology, and the Fertility Research Center discovered a mutation in a gene that is supposed to protect the full DNA sequence in the sperm.

The mutation inactivates the function of the gene and thus the production of sperm is arrested. These results link damage to this gene and infertility for the first time.

As a result of this study, in future, specific scans will be available to test for mutations in this gene, which are important for prognostic and treatment of the couples, the researchers said.

The results of the study, Mutation in TDRD9 causes non-obstructive azoospermia in infertile men, were published recently in the Journal of Medical Genetics. First author on the article is Maram Arafat from Prof. Parvaris research group.

The study was supported in part by The Natural Science Foundation of China (NSFC) Israel Science Foundation (ISF) (NSFC-ISF) (1183/14).

Read more here:
Israeli Scientists Discover Genetic Mutation That Causes Male Infertility - The Jewish Press - JewishPress.com

Recommendation and review posted by Bethany Smith

NEW YORK (Reuters) - While President Donald Trump has thrust transgender people back into the conflict between conservative and liberal values in the United States, geneticists are quietly working on a major research effort to unlock the secrets of gender identity.

A consortium of five research institutions in Europe and the United States, including Vanderbilt University Medical Center, George Washington University and Boston Children's Hospital, is looking to the genome, a person's complete set of DNA, for clues about whether transgender people are born that way.

Two decades of brain research have provided hints of a biological origin to being transgender, but no irrefutable conclusions.

Now scientists in the consortium have embarked on what they call the largest-ever study of its kind, searching for a genetic component to explain why people assigned one gender at birth so persistently identify as the other, often from very early childhood. (reut.rs/2w3Ozg9)

Researchers have extracted DNA from the blood samples of 10,000 people, 3,000 of them transgender and the rest non-transgender, or cisgender. The project is awaiting grant funding to begin the next phase: testing about 3 million markers, or variations, across the genome for all of the samples.

Knowing what variations transgender people have in common, and comparing those patterns to those of cisgender people in the study, may help investigators understand what role the genome plays in everyone's gender identity.

"If the trait is strongly genetic, then people who identify as trans will share more of their genome, not because they are related in nuclear families but because they are more anciently related," said Lea Davis, leader of the study and an assistant professor of medicine at the Vanderbilt Genetics Institute.

The search for the biological underpinnings is taking on new relevance as the battle for transgender rights plays out in the U.S. political arena.

One of the first acts of the new Trump administration was to revoke Obama-era guidelines directing public schools to allow transgender students to use bathrooms of their choice. (reut.rs/2l8pWJe) Last week, the president announced on Twitter he intends to ban transgender people from serving in the military. (reut.rs/2uXF8kG)

Texas lawmakers are debating a bathroom bill that would require people to use the bathroom of the sex listed on their birth certificate. (reut.rs/2tXKbgV) North Carolina in March repealed a similar law after a national boycott cost the state hundreds of millions of dollars in lost business. (reut.rs/2nGg1OH)

Currently, the only way to determine whether people are transgender is for them to self-identify as such. While civil rights activists contend that should be sufficient, scientists have taken their search to the lab.

That quest has made some transgender people nervous. If a "cause" is found it could posit a "cure," potentially opening the door to so-called reparative therapies similar to those that attempt to turn gay people straight, advocates say. Others raise concerns about the rights of those who may identify as trans but lack biological "proof."

"It's an idea that can be wielded against us, depending on the ideology of the user," said Kale Edmiston, a transgender man and postdoctoral scholar at the University of Pittsburgh specializing in neuroimaging.

Dana Bevan, a transgender woman, psychologist and author of three books on transgender topics, acknowledged the potential manipulation of research was a concern but said, "I don't believe that science can or should hold back from trying to understand what's going on."

Davis stressed that her study does not seek to produce a genetic test for being transgender, nor would it be able to. Instead, she said, she hopes the data will lead to better care for transgender people, who experience wide health disparities compared to the general population. (reut.rs/2cyp674)

One-third of transgender people reported a negative healthcare experience in the previous year such as verbal harassment, refusal of treatment or the need to teach their doctors about transgender care, according to a landmark survey of nearly 28,000 people released last year by the National Center for Transgender Equality.

Some 40 percent have attempted suicide, almost nine times the rate for the general population.

"We can use this information to help train doctors and nurses to provide better care to trans patients and to also develop amicus briefs to support equal rights legislation," said Davis, who is also director of research for Vanderbilt's gender health clinic.

The Vanderbilt University Medical Center in Tennessee has one of the world's largest DNA databanks. It also has emerged as a leader in transgender healthcare with initiatives such as the Trans Buddy Program, which pairs every transgender patient with a volunteer to help guide them through their healthcare visits.

The study has applied for a grant from the National Institutes of Health and is exploring other financial sources to provide the $1 million needed to complete the genotyping, expected to take a year to 18 months. Analysis of the data would take about another six months and require more funding, Davis said.

The other consortium members are Vrije University in Amsterdam and the FIMABIS institute in Malaga, Spain.

Until now, the bulk of research into the origins of being transgender has looked at the brain.

Neurologists have spotted clues in the brain structure and activity of transgender people that distinguish them from cisgender subjects.

A seminal 1995 study was led by Dutch neurobiologist Dick Swaab, who was also among the first scientists to discover structural differences between male and female brains. Looking at postmortem brain tissue of transgender subjects, he found that male-to-female transsexuals had clusters of cells, or nuclei, that more closely resembled those of a typical female brain, and vice versa.

Swaab's body of work on postmortem samples was based on just 12 transgender brains that he spent 25 years collecting. But it gave rise to a whole new field of inquiry that today is being explored with advanced brain scan technology on living transgender volunteers.

Among the leaders in brain scan research is Ivanka Savic, a professor of neurology with Sweden's Karolinska Institute and visiting professor at the University of California, Los Angeles.

Her studies suggest that transgender men have a weakened connection between the two areas of the brain that process the perception of self and one's own body. Savic said those connections seem to improve after the person receives cross-hormone treatment.

Her work has been published more than 100 times on various topics in peer-reviewed journals, but she still cannot conclude whether people are born transgender.

"I think that, but I have to prove that," Savic said.

A number of other researchers, including both geneticists and neurologists, presume a biological component that is also influenced by upbringing.

But Paul McHugh, a university professor of psychiatry at the Johns Hopkins School of Medicine, has emerged as the leading voice challenging the "born-this-way" hypothesis.

He encourages psychiatric therapy for transgender people, especially children, so that they accept the gender assigned to them at birth.

McHugh has gained a following among social conservatives, while incensing LGBT advocates with comments such as calling transgender people "counterfeit."

Last year he co-authored a review of the scientific literature published in The New Atlantis journal, asserting there was scant evidence to suggest sexual orientation and gender identity were biologically determined.

The article drew a rebuke from nearly 600 academics and clinicians who called it misleading.

McHugh told Reuters he was "unmoved" by his critics and says he doubts additional research will reveal a biological cause.

"If it were obvious," he said, "they would have found it long ago."

Reporting by Daniel Trotta; Editing by Marla Dickerson

See original here:
Born this way? Researchers explore the science of gender identity - Reuters

Recommendation and review posted by Bethany Smith

Many of us have been curious as to why some of us are straight or gay; "Why are some of us attracted to the opposite sex?" "Why are some men attracted to men?" "Why are some women attracted to women?" Currently, we don't know why we vary in sexual orientation, but science suggests being gay at least is partly genetic, rather than a lifestyle choice.

In AsapSCIENCE's latest video, "Does Everybody Have A Gay Gene," Mitchell Moffit and Gregory Brown explain genetics and epigenetic factors the study of how the environment can chemically modify our genes can be used to prove that being gay is not a choice.

Read More: Study Finds Same Sex Couples Make Better Parents

A 2016 study, published in the Archives of Sexual Behavior, found linkages in a specific region of the X chromosome labelled Xq28 and in another region of chromosome 8, known as 8q12, in over 400 gay siblings. Traits like hair color, height and intelligence varied between each brothers in a pair and between all groups of brothers. In other words, any single nucleotide polymorphisms (SNPs) differences on a single letter in the genetic code found in the same genetic locations across the group would likely be associated with sexual orientation.

The region on the X chromosome, Xq28, was previously identified in 1993 by Dean Hamer of the US National Institutes of Health in Bethesda, Maryland. However, there needs to be more work done to determine the specific genes involved and how they work, including if there are equivalent genes in women. The study provides researchers with the potential to narrow down fewer genes linked to sexual preference.

The belief that homosexuality is genetic can create a paradox. For example, homosexuals have 80 percent fewerchildren than heterosexuals, which suggests the genes would not be passed down and would eventually die out.

Enter epigenetics.

A 2015 study published in Science used epigenetics to propose that everyone has a gay gene, but it's contingent on whether the attachment of a methyl group to specific regions of DNA is triggered and turned on. Upon analyzing gay and straight male twins, researchers found a specific methylation pattern was closely linked to sexual orientation. The model was able to predict the sexuality of men with 70 percent accuracy.

However, a caveat of the study is its small sample size, which means there is not too much power to make such a claim. Evidently, there were certain correlations, but a predicting model may not yet be an actual reality. Larger studies are needed to replicate these findings if valid.

A specific gay gene has not been found, but there's scientific data that suggests sexual orientation is linked to genetics on a molecular level. Previous research has found giving birth to a son increases the odds of homosexuality in the next son by 33 percent. Scientists believe a woman's body adjusts the androgen level in her womb as she has more sons, and the androgens interactwith genes to produce homosexuality.

Read More: Kids Of Same-Sex Parents Have Same Emotional, Physical Health As Those With A Mom And Dad

The scientific community still has a long way to go when it comes to studying the causes of homosexuality. Currently, most research only focuses on gay men and neglect other groups, like lesbians. Further research on the genetic and epigenetic factors of homosexuality could help reduce homophobic laws around the world by proving it's not a lifestyle choice.

Science is working on proving the biology of homosexuality, but it also raises the question, does it matter if someone is straight or gay?

See Also:

Why Discrimination Against Gays Is A Global Health Hazard

Heterosexual Providers Found To Hold Bias On Sexual Orientation

Read the original here:
Homosexuality Partly Rooted In Genetics Rather Than Lifestyle Choice, Says Science - Medical Daily

Recommendation and review posted by simmons

Weve always said in the past gene editing shouldnt be done, mostly because it couldnt be done safely, said Richard Hynes, a cancer researcher at the Massachusetts Institute of Technology who co-led the committee. Thats still true, but now it looks like its going to be done safely soon, he said, adding that the research is a big breakthrough.

What our report said was, once the technical hurdles are cleared, then there will be societal issues that have to be considered and discussions that are going to have to happen. Nows the time.

Scientists at Oregon Health and Science University, with colleagues in California, China and South Korea, reported that they repaired dozens of embryos, fixing a mutation that causes a common heart condition that can lead to sudden death later in life.

If embryos with the repaired mutation were allowed to develop into babies, they would not only be disease-free but also would not transmit the disease to descendants.

The researchers averted two important safety problems: They produced embryos in which all cells not just some were mutation-free, and they avoided creating unwanted extra mutations.

It feels a bit like a one small step for (hu)mans, one giant leap for (hu)mankind moment, Jennifer Doudna, a biochemist who helped discover the gene-editing method used, called CRISPR-Cas9, said in an email.

Scientists tried two techniques to remove a dangerous mutation. In the first, genetic scissors were inserted into fertilized eggs. The mutation was repaired in some of the resulting embryos but not always in every cell. The second method worked better: By injecting the scissors along with the sperm into the egg, more embryos emerged with repaired genes in every cell.

When gene-editing components were introduced into a fertilized egg, some embryos contained a patchwork of repaired and unrepaired cells.

Gene-editing

components inserted

after fertilization

Cell with

unrepaired

gene

Mosaicism in

later-stage embryo

When gene-editing components were introduced with sperm to the egg before fertilization, more embryos had repaired mutations in every cell.

Gene-editing components

inserted together with sperm,

before fertilization

In 42 of 58

embryos

tested, all

cells were

repaired

Uniform

later-stage embryo

When gene-editing components were introduced into a fertilized egg, some embryos contained a patchwork of repaired and unrepaired cells.

Gene-editing

components inserted

after fertilization

Cell with

unrepaired

gene

Mosaicism in

later-stage embryo

When gene-editing components were introduced with sperm to the egg before fertilization, more embryos had repaired mutations in every cell.

Gene-editing

components inserted

together with sperm,

before fertilization

In 42 of 58

embryos

tested, all

cells were

repaired

Uniform

later-stage embryo

I expect these results will be encouraging to those who hope to use human embryo editing for either research or eventual clinical purposes, said Dr. Doudna, who was not involved in the study.

Much more research is needed before the method could be tested in clinical trials, currently impermissible under federal law. But if the technique is found to work safely with this and other mutations, it might help some couples who could not otherwise have healthy children.

Potentially, it could apply to any of more than 10,000 conditions caused by specific inherited mutations. Researchers and experts said those might include breast and ovarian cancer linked to BRCA mutations, as well as diseases like Huntingtons, Tay-Sachs, beta thalassemia, and even sickle cell anemia, cystic fibrosis or some cases of early-onset Alzheimers.

You could certainly help families who have been blighted by a horrible genetic disease, said Robin Lovell-Badge, a professor of genetics and embryology at the Francis Crick Institute in London, who was not involved in the study.

You could quite imagine that in the future the demand would increase. Maybe it will still be small, but for those individuals it will be very important.

The researchers also discovered something unexpected: a previously unknown way that embryos repair themselves.

In other cells in the body, the editing process is carried out by genes that copy a DNA template introduced by scientists. In these embryos, the sperm cells mutant gene ignored that template and instead copied the healthy DNA sequence from the egg cell.

We were so surprised that we just couldnt get this template that we made to be used, said Shoukhrat Mitalipov, director of the Center for Embryonic Cell and Gene Therapy at Oregon Health and Science University and senior author of the study. It was very new and unusual.

The research significantly improves upon previous efforts. In three sets of experiments in China since 2015, researchers seldom managed to get the intended change into embryonic genes.

And some embryos had cells that did not get repaired a phenomenon called mosaicism that could result in the mutation being passed on as well as unplanned mutations that could cause other health problems.

In February, a National Academy of Sciences, Engineering and Medicine committee endorsed modifying embryos, but only to correct mutations that cause a serious disease or condition and when no reasonable alternatives exist.

Sheldon Krimsky, a bioethicist at Tufts University, said the main uncertainty about the new technique was whether reasonable alternatives to gene editing already exist.

As the authors themselves noted, many couples use pre-implantation genetic diagnosis to screen embryos at fertility clinics, allowing only healthy ones to be implanted. For these parents, gene editing could help by repairing mutant embryos so that more disease-free embryos would be available for implantation.

Hank Greely, director of the Center for Law and the Biosciences at Stanford, said creating fewer defective embryos also would reduce the number discarded by fertility clinics, which some people oppose.

The larger issue is so-called germline engineering, which refers to changes made to embryo that are inheritable.

If youre in one camp, its a horror to be avoided, and if youre in the other camp, its desirable, Dr. Greely said. Thats going to continue to be the fight, whether its a feature or a bug.

For now, the fight is theoretical. Congress has barred the Food and Drug Administration from considering clinical trials involving germline engineering. And the National Institutes of Health is prohibited from funding gene-editing research in human embryos. (The new study was funded by Oregon Health and Science University, the Institute for Basic Science in South Korea, and several foundations.)

The authors say they hope that once the method is optimized and studied with other mutations, officials in the United States or another country will allow regulated clinical trials.

I think it could be widely used, if its proven safe, said Dr. Paula Amato, a co-author of the study and reproductive endocrinologist at O.H.S.U. Besides creating more healthy embryos for in vitro fertilization, she said, it could be used when screening embryos is not an option or to reduce arduous IVF cycles for women.

Dr. Mitalipov has pushed the scientific envelope before, generating ethical controversy with a so-called three-parent baby procedure that would place the nucleus of the egg of a woman with defective cellular mitochondria into the egg from a healthy woman. The F.D.A. has not approved trials of the method, but Britain may begin one soon.

The new study involves hypertrophic cardiomyopathy, a disease affecting about one in 500 people, which can cause sudden heart failure, often in young athletes.

It is caused by a mutation in a gene called MYBPC3. If one parent has a mutated copy, there is a 50 percent chance of passing the disease to children.

Using sperm from a man with hypertrophic cardiomyopathy and eggs from 12 healthy women, the researchers created fertilized eggs. Injecting CRISPR-Cas9, which works as a genetic scissors, they snipped out the mutated DNA sequence on the male MYBPC3 gene.

They injected a synthetic healthy DNA sequence into the fertilized egg, expecting that the male genome would copy that sequence into the cut portion. That is how this gene-editing process works in other cells in the body, and in mouse embryos, Dr. Mitalipov said.

Instead, the male gene copied the healthy sequence from the female gene. The authors dont know why it happened.

Maybe human sex cells or gametes evolved to repair themselves because they are the only cells that transmit genes to offspring and need special protection, said Juan Carlos Izpisua Belmonte, a co-author and geneticist at the Salk Institute.

Out of 54 embryos, 36 emerged mutation-free, a significant improvement over natural circumstances in which about half would not have the mutation. Another 13 embryos also emerged without the mutation, but not in every cell.

The researchers tried to eliminate the problem by acting at an earlier stage, injecting the egg with the sperm and CRISPR-Cas9 simultaneously, instead of waiting to inject CRISPR-Cas9 into the already fertilized egg.

That resulted in 42 of 58 embryos, 72 percent, with two mutation-free copies of the gene in every cell. They also found no unwanted mutations in the embryos, which were destroyed after about three days.

The method was not perfect. The remaining 16 embryos had unwanted additions or deletions of DNA. Dr. Mitalipov said he believed fine-tuning the process would make at least 90 percent of embryos mutation-free.

And for disease-causing mutations on maternal genes, the same process should occur, with the fathers healthy genetic sequence being copied, he said.

But the technique will not work if both parents have two defective copies. Then, scientists would have to determine how to coax one gene to copy a synthetic DNA sequence, Dr. Mitalipov said.

Otherwise, he said, it should work with many diseases, a variety of different heritable mutations.

R. Alta Charo, a bioethicist at University of Wisconsin at Madison, who led the committee with Dr. Hynes, said the new discovery could also yield more information about causes of infertility and miscarriages.

She doubts a flood of couples will have edited children.

Nobodys going to do this for trivial reasons, Dr. Charo said. Sex is cheaper and its more fun than IVF, so unless youve got a real need, youre not going to use it.

See original here:
In Breakthrough, Scientists Edit a Dangerous Mutation From Genes in Human Embryos - New York Times

Recommendation and review posted by sam

Alliance for Gene Cancer Therapy Executive Director Margaret C. Cianci and President and CEO John E. Walter outside the nonprofits headquarters in Stamford. Photo by Phil Hall.

The development of an experimental gene-targeting therapy in cancer treatment that could be approved for the U.S. market this year was sparked in large part by the research funding support of a Stamford nonprofit.

The chimeric antigen receptor T-cell (CAR-T) drug, labeled tisagenlecleucel by its manufacturer, Novartis, in July was unanimously recommended for approval by the oncologic drugs advisory committee of the U.S. Food and Drug Administration. If the FDA grants final approval as expected this fall, it will be the first drug treatment targeting human genes approved for the U.S. market.

In Stamford, the Alliance for Cancer Gene Therapy since 2004 has provided a total of $1.8 million to Dr. Carl June at the University of Pennsylvania, the lead researcher in developing the CAR-T therapy. John E. Walter, president and CEO of the Stamford organization, said Junes work has helped to redefine perceptions of what gene therapy can accomplish.

Oftentimes, gene therapy is perceived as taking the bad genes out and putting some good genes in, Walter said. In this case, a patients T-cells are being removed and re-engineered with a virus and reintroduced in the body. With this genetic re-engineering, they become killer T-cells they go in and go after and kill the cancer cells.

Cancer cells in your body multiply and dont know how to die, said Alliance for Gene Cancer Therapy Executive Director Margaret C. Cianci. We have cells in our system all of the time that are growing and dying, but cancer cells dont do that. This therapy is for supercharging your own immune system to recognize these cancer cells and kill them.

If approved, the Novartis drug would mark a milestone achievement for the Alliance, whose creation in 2001 was driven by a tragic loss caused by cancer in its co-founders family. Edward Netter, chairman and CEO of Geneve Corp., a financial services holding company in Stamford, and his wife Barbara, a staff therapist at Pelham Family Services in Westchester County, lost their daughter-in-law, Kimberly Lawrence-Netter, to breast cancer. Edward Netter died from cancer in 2011. His wife serves as the nonprofits honorary board chairwoman.

Walter, who served as CEO of the Leukemia & Lymphoma Society before joining the Alliance in May 2016, noted that this organization differed from most because all of its raised funds are used solely to finance research. Our administrative expenses are paid for by our board and by the Netters, he said, and the nonprofits four-person staff works out of Geneve Corp. headquarters. One hundred percent of your contributions go to research.

Since its founding, the Alliance has allocated approximately $29 million in grants to U.S. and Canadian projects. These are grants to two different types of scientists, said Cianci. We started funding young investigators at assistant professor level who have just become independent. It is difficult for them to get funding, especially in an area as innovative as gene therapy, and the government doesnt like to fund what they see as high-risk projects. We also fund clinical investigators, which included Dr. June.

The Alliance puts out two requests for funding applications each year, which are judged through a peer-review process coordinated by a scientific advisory committee.

There is always more research than there are dollars, said Walter. Invariably, we are leaving research on the table because we dont have the dollars to fund those.

The nonprofit itself receives funding through contributions from longtime donors and an annual fundraising event coordinated by Swim Across America that is held in the Long Island Sound directly across from its offices. That raises about $400,000 a year, Walter said.

Dr. Junes Alliance-funded research was published in a medical journal in 2011 in a study of three patients with advanced chronic lymphocytic leukemia. Novartis, the Swiss pharmaceutical company, expressed interest in the results and paid the University of Pennsylvania $20 million to license the technology.

Once we have survival data for these patients in Novartis-sponsored clinical trials, over time the FDA could consider using this as frontline treatment instead of highly toxic chemotherapy, said Walter.

For Cianci, the Alliances mission is crucial in encouraging new generations of researchers to focus on cancer and gene therapy solutions, especially when federal funding is being threatened by budget cuts.

If we dont fund the young scientists, they are going to leave the field, she warned. We dont want to lose some of these incredible minds. The average age for getting your first grant from the National Institute of Health is 42. What do you tell someone who just became a postdoctoral researcher and wants to have their own lab? How are they going to get funding?

One in four people could potentially get cancer in their lifetimes, Cianci said. And who hasnt been touched by cancer in one way or another?

Read more:
Gene therapy cancer treatment funded by Stamford nonprofit awaits FDA approval - Westfair Online

Recommendation and review posted by sam

Agilis Biotherapeutics has formed a joint venture with Japans Gene Therapy Research Institution (GTRI). The alliance gives Agilis a base in Japan and a partnership with a fellow CNS specialist to support its development of adeno-associated virus (AAV) vectors and gene therapies.

Cambridge, Massachusetts-based Agilis set up the joint venture using a grant from the Japanese government. The agreement will establish an AAV manufacturing facility in Japan, from where Agilis and GTRI will work on vectors using Sf9 baculovirus and HEK293 mammalian cell systems. Agilis and GTRI plan to develop and manufacture AAV gene therapy vectors through the joint venture.

Agilis and GTRI also plan is to collaborate on the development and commercialization of certain CNS gene therapies.

GTRIs background suggests it is well-equipped to contribute to the project. The Japanese company grew out of the work of Shin-ichi Muramatsu, M.D., a scientist who sequenced AAV3 in the 1990s before going on to create AAVs designed to cross the blood-brain barrier. GTRI is working on gene therapies against diseases including Alzheimers, amyotrophic lateral sclerosis and Parkinsons that build on this research into AAVs.

Both biotechs are developing gene therapies to treat aromatic l-amino acid decarboxylase (AADC) deficiency. GTRI aims to get its candidate into the clinic in 2019. Agilispicked up its candidate from a university in Taiwan, which enrolled 18 patients in two clinical trials of the gene therapy. Those trials have taken the candidate toward a pivotal trial.

These programs may benefit from the joint venture. Working out of the Life Science Innovation Center of Kawasaki City, the joint venture intends to develop and produce AAVs for use in gene therapies against AADC deficiency and Parkinson's.

The joint venture marks the second time Agilis has looked outside of its walls for help with AAV vectors. Late in 2013, Agilis struck a deal with Intrexon that gave it access to the latters vector platform. Agilis is using the vectors to develop a treatment for Friedreichs ataxia.

See original here:
Agilis forms joint venture to advance gene therapy vectors - FierceBiotech

Recommendation and review posted by sam

There is new hope for muscular dystrophy patients as researchers, using gene therapy, successfully reversed the disease in dogs.

Gene therapy aims to replace missing or defective genes in the DNA of a given cell.

The technique has evolved over the years to become a viable therapy thats safe and effective, opening new paths in the management of many difficult diseases.

Not only can gene editing be used to treat pathologies, but it can also to prevent them. Only, were not there just yet.

Muscular dystrophy is the term used for a group of diseases in which musculature weakens and progressively degenerates until the patient loses most, ifnot all of their mobility.

Symptoms of muscular dystrophy often include general muscle weakness and degeneration, stiff joints, coordination and mobility troubles, and frequent falls.

In most cases a congenital condition, muscular dystrophy disorders are rare. Each disorder of muscular dystrophy is associated with distinct genetic mutations. The nature and location of the genetic mutation define the form of muscular dystrophy.

Although they can occur at any age, the onset of most MD disorders starts during childhood, and usually, affected persons dont live past 30 years of age, especially with particularly aggressive forms of the disease like Duchenne muscular dystrophy.

The most common and most studied form of muscular dystrophy is Duchenne muscular dystrophy (DMD), which affects 1 in 5,000 children at birth, and especially boys (1/3500).

Theres hope for children and other DMD patients, as a scientific experiment suggests that the disease could be reversed and a cure might be on the way.

An international research team, comprised of scientists from Genethon and Insermin France and the Royal Hollowayat the University of London UK, announced theyd managed to treat Duchenne muscular dystrophy (DMD) with gene therapy in dogs.

Their findings were published in the journal Nature Communications.

The team has shown the efficacy of gene therapy in restoring normal muscle function in 12 dogs (Golden Retrievers) affected by canine DMD, with a stabilization of clinical symptoms.

A video of these dogs before and after treatment can be found here.

Researchers injected highly functional micro-dystrophin genes (a short version of the dystrophin gene) through a drug vector (harmless virus) so that the repaired gene could produce the protein involved in muscle function.

2 years after the injection of the drug, researchers observed that all dogs demonstrated signs of significant restoration of their muscles and regained their motor skills. Not to mention that the same dogs werent expected to live past the age of 6 months.

Now, with the method has been shown to be safe and efficient in animals, the next logical step would human trials.

For the many people affected by this debilitating disease, this is a miraculous development.

The rest is here:
Gene Therapy Could Cure Muscular Dystrophy for Dogs and Humans - Edgy Labs (blog)

Recommendation and review posted by sam

Impropriety among big-pharmaceutical corporations has ranged from multi-billion dollar bribery rackets, to marketing drugs to patients for uses they were never approved for by regulators, to covering up known dangerous side-effects of medications they produce and sell.

More recently, big-pharma has been embroiled in a series of price-gouging controversies over equipment and treatments. This includes the hijacking of and profiteering from a revolutionary new treatment called gene therapy.

Gene therapy, the process of re-engineering human cells to either include missing DNA to cure genetic conditions or to arm the immune system to seek and destroy disease, has been the latest hopeful technology scooped up and plundered by big-pharma.

Gene therapy promises a single shot cure to many of the diseases that have confounded humanity the most everything from diabetes to cancer, to blindness, deafness, and even various effects of aging.

At least two treatments using gene therapy have been approved for European markets.

A third that has proven in clinical trials to provide permanent remission for leukemia patients who were unresponsive to chemotherapy, appears to be close to FDA approval.

The Literal Cure for Cancer, Dangled Over the Dying

While the treatment even under experimental conditions costs approximately $20,000 to produce, pharmaceutical giant Novartis has swooped in and industry experts anticipate a markup leaving the price tag between $300,000-600,000.

The New York Times in a 2012 article titled, In Girls Last Hope, Altered Immune Cells Beat Leukemia, reported that (emphasis added):

Dr. June said that producing engineered T-cells costs about $20,000 per patient far less than the cost of a bone-marrow transplant.Scaling up the procedure should make it even less expensive, he said, but he added, Our costs do not include any profit margin, facility depreciation costs or other clinical care costs, and other research costs.

More recently, in a July 2017 Washington Post article titled, First gene therapy a true living drug on the cusp of FDA approval, its reported that:

Novartis has not disclosed the price for its therapy, but analysts are predicting $300,000 to $600,000 for a one-time infusion. Brad Loncar, whose investment fund focuses on companies that develop immunotherapy treatments, hopes the cost does not prompt a backlash. CAR-T is not the EpiPen, he said. This is truly pushing the envelope and at the cutting edge of science.

But it isnt Novartis thats pushing the envelop, or at the cutting edge of science. Charity-funded university researchers are.

Stealing From Charity

The New York Times and the Washington Post both appear to give Novartis credit for this breakthrough in their article, with NYT claiming that the company invested some $20 million on a research center to bring the treatment to market. However, that appears not to be entirely true.

It was, in fact, the Leukemia & Lymphoma Society (LLS) that funded the initial work toward this breakthrough, beginning in the late 1990s and committing some $21 million to the effort.

Novartis is indeed a partnerof LLS, but according to LLS own annual reports (2016, PDF), it is listed under the second tier of donors providing between $500,000-900,000 out of the total $35.6 million LLS received in direct gifts that year. In some years Novartis has donated even less.

LLS itself,in a 2014 press release, stated:

LLS has invested in the work of June and colleagues since 1998 and has committed to investing a total of $21 million through 2017 to get this first treatment to more patients. LLS first funded Grupp in 1992 through its career development program. LLS has also been funding another member of the team, David Porter, M.D. of University of Pennsylvania since 1994.

Elsewhere, LLS reports cite that this breakthrough in curing leukemia has attracted Novartis as a partner, never mentioning that Novartis is actually a long-term LLS partner.

In reality, it appears pharmaceutical corporations like Novartis are using charities like LLS to fund research and development that corporations themselves should be investing in. Instead, Novartis and others are poaching public and charity-funded research and breakthroughs, profiting from what is often decades of dedicated and difficult work.

Beyond LLS partners, it receives millions of dollars annually from other donors ranging from businesses unrelated to the pharmaceutical industry, to fundraising events held nationwide, to families and individuals who have experienced cancer either themselves or through a family member or friend.

The research and breakthroughs LLS funds belong to all of its donors. How the work it funded has ended up in the hands of a single corporation, facing a mark up of anywhere between 15-30 times its cost during experimental trials demands scrutiny and a detailed explanation.

Why Big-Pharma is Gouging Gene Therapy

Gene therapy overall threatens the fundamental business model pharmaceutical giants are built on that is to perpetually peddle medication that covers up the symptoms of disease rather than outright curing it.

It is a business practice that provides profits easily predicted quarter to quarter, with some medications leading to complications big-pharma also has a pill for. Something that treats a patient permanently with a single, inexpensive shot constitutes big-pharmas worst nightmare.

MIT Technology Review in an article titled, A First-of-a-Kind Gene Therapy Cure Has Struggled to Find a Market, tells the tale of another pharmaceutical corporation GlaxoSmithKline (GSK), of another revolutionary gene therapy it scooped up from research done by others, its $665,000 price tag, and why GSK along with the rest of big-pharma are disinterested in gene therapy.

The article notes:

[Alex] Pasteur [investor with F-Prime Capital Partners and interim CEO of Orchard Therapeutics] also says revenues for a rare-disease gene therapy might only ever add up to $100 million a year. Because GSK brings in $36 billion a year, Pasteur is not surprised the company is looking elsewhere for revenue. These are pimples on the back of a whale, he says. But the assets could be very interesting for someone else.

Indeed, a single shot that costs only a few thousand dollars and permanently cures people of virtually every human health infliction not only isnt profitable, but will likely put these enormous, abusive monopolies out of business for good.

Obamacare vs Turmpcare: Nobody Cares, But Innovation Cures

Education is the first step in combating the hijacking and burying of gene therapy and other innovations.

At a time when people arguing over Obamacare versus Trumpcare are realizing thatno oneactually cares about their health more than they themselves, innovation like gene therapy offers to make healthcare so affordable and effective, insurance schemes and government subsidies would be unnecessary.

But gene therapy will only gain traction if the wider public knows about it, including its implications for not only improving their own health, but improving the healthcare systems of their respective nations.

The public must also understand the true costs behind gene therapy and where money for research has come from often from public funding or charity. This knowledge allows the public to call out pharmaceutical corporations attempting to seize credit and profits entirely for themselves.

While pharmaceutical corporations invest inordinate amounts of money attempting to convince the world that they are indispensable, university researchers funded by public money and charity prove they are more often than not setting breakthroughs back, not moving them forward.

If the good people involved in LLS are capable of raising the money to fund these breakthroughs, they are capable of creating a pharmaceutical trust that can bring these cures to market with greater transparency and oversight.

Healthcare debates focused purely on political solutions and debates are frustrating. Getting behind gene therapy and other tangible healthcare innovations is something people can better invest their time, money, energy, and attention into instead.

All images in this article are from the author.

See original here:
No Matter How Bad You Thought Big-Pharma Was, This Is Worse - Center for Research on Globalization

Recommendation and review posted by Bethany Smith

Stem Cell Skin Treatment

Skin care specialists around the world are turning to non-embryonic stem cells for skincare. The stem cells come from specialized plants that when applied, protect human skin stem cells from damage and deterioration. Derma Divine is a unique topical serum that repairs and protects aging skin at a cellular level. Although you can not avoid getting older, now you can reverse the appearance of aging skin! For wrinkle free, hydrated and youthful looking skin, order online today. Users notice results just after three months of consistent use. Remember, you can never be too old to feel and look young.

The number one cause of aging skin is from sun damage. UV rays and pollution deteriorate skin cells making your skin more prone to damage. Derma Divine restores damaged skin cells creating skin that is wrinkle free, hydrated and youthful. Millions are skin care specialists are turning to stem cell skin treatments instead of injections. Injections can leave scaring and is an unnatural way to repair damage. Stem cell treatments however, repair wrinkled and damaged skin at the source of the problem. This method is much more efficient, natural and effective. Order online today for a confident tomorrow.

Stem cells have the ability to develop into different cell types in the body. For skin treatment, essentially the stem cell turns into a new skin cell. The new cell can then create proteins, carbohydrates and lipids to repair wrinkles and restore firmness and elasticity back into the face. Derma Divine is a light weight serum that is fast absorbent allowing it to penetrate deep into the skin. This allows the active ingredients to repair at a cellular level. Did you know that wrinkles, age spots and dry skin are considered wounds? Stem cells repair skin leaving you with irresistibly young looking skin.

The active ingredients are the most important part of any skin care treatment. Derma Divine Anti Aging Serum is unique to others because it uses stem cell stimulation to restore youthful skin back into the face. We combined botanical extracts into the serum to support and protect new skin growth. Below is a few active ingredients within the serum:

Plant Stem Cell Rebuild damaged skin cells. Reverse appearance of aging skin. Firms, smooths & lifts damaged skin.

Vitamin C Corrects hyper pigmentation (dark spots). Helps fight skin cell damage, protects from sun damage and supports collagen production

We understand that using stem cells may come off as strange to new users. Lots of people are skeptical about new skin treatments on the market and that is okay! It is good to be skeptical in order to avoid scams. That is why we are offering new users a free trial to test the product out for themselves before purchasing. We want you to see the benefits so you are 100% satisfied with our products.

FOR BEST RESULTS PAIR WITH SKINCARE PANAMA

Original post:
Derma Divine Serum - Plant Stem Cell Skin Repair ...

Recommendation and review posted by sam