NEW YORK, Feb. 24, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB) today announced that aggregated results from 309 children treated with Ryoncil (remestemcel-L) were presented atthe American Society for Transplantation Cellular Therapy and the Center for International Blood & Bone Marrow Transplant Research (TCT) meeting in Orlando, Florida on February 22. The data showed that treatment with RYONCIL across three separate trials resulted inconsistent treatment responses and survival outcomesinchildren with steroid-refractory acute graft versus host disease (SR-aGVHD).

Key findings and conclusions were:

Mesoblast Chief Medical Officer Dr Fred Grossman said: These aggregated data from three studies demonstrate consistent efficacy and safety of RYONCIL in children suffering from steroid refractory acute graft versus host disease. If approved, RYONCIL has the potential to be an effective and safe therapy to improve survival outcomes in the most vulnerable population of children with severe forms of this disease who can have mortality rates as high as 90 percent.

In January, Mesoblast filed a Biologics License Application (BLA) to the United States Food and Drug Administration (FDA) for RYONCIL for the treatment of children with steroid-refractory aGVHD. The Company has requested Priority Review of the BLA by the FDA under the product candidates existing Fast Track designation. If approved, RYONCIL is expected to be launched in the US in 2020.

About Acute GVHDAcute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT). Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, and these numbers are increasing.1 In patients with the most severe form of acute GVHD (Grade C/D or III/IV) mortality is as high as 90% despite optimal institutional standard of care.2,3. There are currently no FDA-approved treatments in the US for children under 12 with SR-aGVHD.

About Ryoncil Mesoblasts lead product candidate, RYONCIL, is an investigational therapy comprising culture- expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is administered to patients in a series of intravenous infusions. RYONCIL is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in SR- aGVHD by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.

References1. Niederwieser D, Baldomero H, Szer J. (2016) Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey.2. Westin, J., Saliba, RM., Lima, M. (2011) Steroid-refractory acute GVHD: predictors and outcomes. Advances in Hematology.3. Axt L, Naumann A, Toennies J (2019) Retrospective single center analysis of outcome, risk factors and therapy in steroid refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation.

About MesoblastMesoblast Limited (Nasdaq: MESO; ASX: MSB) is a world leader in developing allogeneic (off-the-shelf) cellular medicines. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platforms to establish a broad portfolio of commercial products and late-stage product candidates. Mesoblasts proprietary manufacturing process yields industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast has filed a Biologics License Application to the United States Food and Drug Administration (FDA) to seek approval of its product candidate Ryoncil (remestemcel-L) for steroid-refractory acute graft versus host disease (acute GvHD). Remestemcel-L is also being developed for other rare diseases. Mesoblast is completing Phase 3 trials for its rexlemestrocel product candidates for advanced heart failure and chronic low back pain. If approved, RYONCIL is expected to be launched in the United States in 2020 for pediatric steroid-refractory acute GVHD. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

Story continues

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

Mesoblasts Forward-Looking StatementsThis announcement includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about the timing, progress and results of Mesoblasts preclinical and clinical studies; Mesoblasts ability to advance product candidates into, enroll and successfully complete, clinical studies; the timing or likelihood of regulatory filings and approvals; and the pricing and reimbursement of Mesoblasts product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblasts actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

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Consistent Outcomes Using Ryoncil as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Acute GVHD - Yahoo Finance

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Alia (2nd from left) and her father (left) with Dr Arif Khan (3rd from left), paediatric ophthalmologist and ocular geneticist at Cleveland Clinic Abu Dhabi, and other hospital doctors at a press conference in Abu Dhabi. Alia and her sister, Hessa, were the first to receive gene therapy in the UAE. Picture by Samihah Zaman, Image Credit:

Abu Dhabi: Two Emirati sisters from Abu Dhabi have become the first to receive gene therapy in the UAE, it was announced in the capital today.

The therapy was conducted with the aim of saving the vision of Alia, 13, and Hessa, 11, who suffered from retinal damage due to a defective gene.

The vision-saving surgery was performed at Cleveland Clinic Abu Dhabi in January (2020), and doctors said at a press conference that they are hopeful it will slowly improve the vision of both patients.

"The prospect of slowly losing one's vision from an untreatable condition is traumatic for both children and their parents. This genetic therapy means we can now replace the faulty gene in the eye, saving and even improving the vision of an individual who would otherwise have eventual irreversible blindness," said Dr Arif Khan, paediatric ophthalmologist and ocular geneticist at the hospital.

Only centre in region with procedure

Cleveland Clinic Abu Dhabi is only one of 10 centres worldwide, and the only one in the region, that is qualified to offer this procedure - the first gene therapy that was approved by the Food and Drug Administration in the United States, doctors said. But its availability opens the doors for more gene therapies to be offered to treat conditions as varied as diabetes and spinal muscular atrophy, they added.

Weve travelled abroad multiple times to find some treatment for our daughters, and this procedure has been a godsend. It has only been a month since the therapy however, and we are hoping for much more improvement to their vision over time, Fatima, the girls mother, told Gulf News.

The procedure

Alia and Hessa were born with RPE65-related retinal dystrophy, a genetic dystrophy in which the RPE65 protein is lacking because a child inherits two copies of the defective RPE65 from both parents. It is a recessive disease and therefore rare, and known to affect one in 200,000 people worldwide. But the condition is also more common in the region because of the limited genetic pool and cultural preferences for consanguineous marriages.

Patients with the condition face gradual damage to their retinas, the photosensitive layer at the back of the eye, and could eventually end up with irreversible blindness.

The gene therapy to treat the condition uses a vector - a bioengineered non-pathogenic virus to deliver normal copies of the RPE65 gene to the eye.

The procedure takes only about an hour, but it is very delicate. The retina can be considered an offshoot of the brain because its cells are very thin, like brain tissue. We use advanced microscopes to create openings in the white of the eye, then use a specialized canula to reach under the retina and inject the vector into the specific area, which can be as small as one-tenth of a millimeter, explained Dr Emad Abboud, chief of the department of the posterior segment at the hospitals Eye Institute.

Post-surgical recovery takes only about a week, but outcomes take a while to become obvious.

As Dr Khan explained, the main benefits for patients receiving the therapy is an improvement of vision in low-light conditions, and an improvement of the visual field.

Vision is a complex sense, involving photosensitivity, visual field, visual acuity, colour sensitivity, motion sensitivity, and the ability to navigate. But [at the least], this therapy prevents the progressive deterioration of the retina, he explained.

What the family said

My handwriting has definitely gotten better, Alia told Gulf News when asked about how the therapy has helped.

I cant tell what else will get better but I already feel that this has been life-changing, she added.

According to Fatima, she noticed her daughters had visual impairment when they were as young as two months old.

The doctor pointed out to me that they were not focusing or making eye contact. So we were aware of the condition. It definitely made life very challenging, especially as their development was delayed, the mother explained.

Alia and Hessa both crawled and walked late, and have needed assistance getting around. While they kept pace with their peers at school, it took Hercualean efforts from both girls, their parents, shadow teachers and at-home tutors, and required the use of Braille and visual aid resources.

Fatima and her husband, Mubarak, kept looking for treatments for both girls, travelling to Germany, India and Spain.

In 2016, we visited the Cleveland Clinic Abu Dhabi, and it was then that we heard of this revolutionary treatment. It was still not approved by the FDA, but we kept dreaming, she said.

It has been a month since the surgery and the doctors said they are more photosensitive now and that this is a good sign. Of course, as a mother, I would love for them to eventually have complete vision, Fatima added.

What is the therapy?

The UAE Ministry of Health and Prevention (MoHAP) approved the gene therapy for RPE65-related retinal dystrophy, Luxturna, in June 2019.

In a statement released at the time, the MoHAP said its registration of Luxturna was only the third global registration of the drug at the health authority level, and was aimed at paving the way for future gene-based therapies for complex conditions.

The United States Food and Drug Administration only approved Luxturna, developed by gene therapy developer, Spark Therapeutics, and the Childrens Hospital of Philadelphia, in late 2017.

The gene therapy to treat the condition uses a vector - a bioengineered non-pathogenic virus to deliver normal copies of the RPE65 gene to the eye with the defective gene.

While the Cleveland Clinic Abu Dhabi did not provide the cost of treatment, international media reports put it at $425,000 (Dh1.56 million) per eye.

Benefits of gene therapy

Dr Khan said at the press conference that us familiar with a few more families with the RPE65-related retinal disorder.

Over time, gene therapy could be used to treat many conditions. The eye is uniquely suited for these therapies because it is a self-contained organ. But at least five other genetic therapies are in development, some at the human trial level, he explained.

Consider gene delivery to be like providing a protein factory to the body. So for instance, some diabetic patients require regular insulin injections. If a gene could be engineered to make insulin, and it could be delivered to the patient, he wouldnt need these regular injections, Dr Khan added.

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UAE's first gene therapy improves eyesight of two Emirati sisters - Gulf News

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When you first meet her, you wont be able to tell that Ipek Kuzu suffers from a rare genetic disease. The three-year-old plays happily on her own for hours, driving her toy cars and cooking in her pretend kitchen. But shes not well. Shes a little wobbly on her feet and doesnt say much, and if nothing is done, she may die by her mid-20s. Ipek has ataxia-telangiectasia, or A-T, a disease caused by an error in her DNA. It causes the loss of brain cells, along with a high risk of infection and cancer.

Its the sort of problem that makes doctors shake their heads. But Ipeks father, Mehmet, and mother, Tugba, hope shell escape that fate. Thanks in part to the persistence of Mehmet, a programmer at Google, in January she became one of the first handful of US patients to receive a hyper-personalized gene medicine, tailored to treat a unique mutation. The one-person drug, designed for her by a Boston doctor, Timothy Yu, is being called atipeksen, for A-T and Ipek.

To create atipeksen, Yu borrowed from recent biotech successes like gene therapy. Some new drugs, including cancer therapies, treat disease by directly manipulating genetic information inside a patients cells. Now doctors like Yu find they can alter those treatments as if they were digital programs. Change the code, reprogram the drug, and theres a chance of treating many genetic diseases, even those as unusual as Ipeks.

The new strategy could in theory help millions of people living with rare diseases, the vast majority of which are caused by genetic typos and have no treatment. US regulators say last year they fielded more than 80 requests to allow genetic treatments for individuals or very small groups, and that they may take steps to make tailor-made medicines easier to try. New technologies, including custom gene-editing treatments using CRISPR, are coming next.

Where it had taken decades for Ionis to perfect its drug, Yu now set a record: it took only eight months for Yu to make milasen, try it on animals, and convince the US Food and Drug Administration to let him inject it into Milas spine.

I never thought we would be in a position to even contemplate trying to help these patients, says Stanley Crooke, a biotechnology entrepreneur and founder of Ionis Pharmaceuticals, based in Carlsbad, California. Its an astonishing moment.

Antisense drug

Right now, though, insurance companies wont pay for individualized gene drugs, and no company is making them (though some plan to). Only a few patients have ever gotten them, usually after heroic feats of arm-twisting and fundraising. And its no mistake that programmers like Mehmet Kuzu, who works on data privacy, are among the first to pursue individualized drugs. As computer scientists, they get it. This is all code, says Ethan Perlstein, chief scientific officer at the Christopher and Dana Reeve Foundation.

A nonprofit, the A-T Childrens Project, funded most of the cost of designing and making Ipeks drug. For Brad Margus, who created the foundation in 1993 after his two sons were diagnosed with A-T, the change between then and now couldnt be more dramatic. Weve raised so much money, weve funded so much research, but its so frustrating that the biology just kept getting more and more complex, he says. Now, were suddenly presented with this opportunity to just fix the problem at its source.

Ipek was only a few months old when her father began looking for a cure. A geneticist friend sent him a paper describing a possible treatment for her exact form of A-T, and Kuzu flew from Sunnyvale, California, to Los Angeles to meet the scientists behind the research. But they said no one had tried the drug in people: We need many more years to make this happen, they told him.

Courtesy Photo (Yu)

Kuzu didnt have years. After he returned from Los Angeles, Margus handed him a thumb drive with a video of a talk by Yu, a doctor at Boston Childrens Hospital, who described how he planned to treat a young girl with Batten disease (a different neurodegenerative condition) in what press reports would later dub a stunning illustration of personalized genomic medicine. Kuzu realized Yu was using the very same gene technology the Los Angeles scientists had dismissed as a pipe dream.

That technology is called antisense. Inside a cell, DNA encodes information to make proteins. Between the DNA and the protein, though, come messenger molecules called RNA that ferry the gene information out of the nucleus. Think of antisense as mirror-image molecules that stick to specific RNA messages, letter for letter, blocking them from being made into proteins. Its possible to silence a gene this way, and sometimes to overcome errors, too.

Though the first antisense drugs appeared 20 years ago, the concept achieved its first blockbuster success only in 2016. Thats when a drug called nusinersen, made by Ionis, was approved to treat children with spinal muscular atrophy, a genetic disease that would otherwise kill them by their second birthday.

Yu, a specialist in gene sequencing, had not worked with antisense before, but once hed identified the genetic error causing Batten disease in his young patient, Mila Makovec, it became apparent to him he didnt have to stop there. If he knew the gene error, why not create a gene drug? All of a sudden a lightbulb went off, Yu says. Couldnt one try to reverse this? It was such an appealing idea, and such a simple idea, that we basically just found ourselves unable to let that go.

Yu admits it was bold to suggest his idea to Milas mother, Julia Vitarello. But he was not starting from scratch. In a demonstration of how modular biotech drugs may become, he based milasen on the same chemistry backbone as the Ionis drug, except he made Milas particular mutation the genetic target. Where it had taken decades for Ionis to perfect a drug, Yu now set a record: it took only eight months for him to make milasen, try it on animals, and convince the US Food and Drug Administration to let him inject it into Milas spine.

Whats different now is that someone like Tim Yu can develop a drug with no prior familiarity with this technology, says Art Krieg, chief scientific officer at Checkmate Pharmaceuticals, based in Cambridge, Massachusetts.

Source code

As word got out about milasen, Yu heard from more than a hundred families asking for his help. Thats put the Boston doctor in a tough position. Yu has plans to try antisense to treat a dozen kids with different diseases, but he knows its not the right approach for everyone, and hes still learning which diseases might be most amenable. And nothing is ever simpleor cheap. Each new version of a drug can behave differently and requires costly safety tests in animals.

Kuzu had the advantage that the Los Angeles researchers had already shown antisense might work. Whats more, Margus agreed that the A-T Childrens Project would help fund the research. But it wouldnt be fair to make the treatment just for Ipek if the foundation was paying for it. So Margus and Yu decided to test antisense drugs in the cells of three young A-T patients, including Ipek. Whichever kids cells responded best would get picked.

Matthew Monteith

While he waited for the test results, Kuzu raised about $200,000 from friends and coworkers at Google. One day, an email landed in his in-box from another Google employee who was fundraising to help a sick child. As he read it, Kuzu felt a jolt of recognition: his coworker, Jennifer Seth, was also working with Yu.

Seths daughter Lydia was born in December 2018. The baby, with beautiful chubby cheeks, carries a mutation that causes seizures and may lead to severe disabilities. Seths husband Rohan, a well-connected Silicon Valley entrepreneur, refers to the problem as a tiny random mutation in her source code. The Seths have raised more than $2 million, much of it from co-workers.

Custom drug

By then, Yu was ready to give Kuzu the good news: Ipeks cells had responded the best. So last September the family packed up and moved from California to Cambridge, Massachusetts, so Ipek could start getting atipeksen. The toddler got her first dose this January, under general anesthesia, through a lumbar puncture into her spine.

After a year, the Kuzus hope to learn whether or not the drug is helping. Doctors will track her brain volume and measure biomarkers in Ipeks cerebrospinal fluid as a readout of how her disease is progressing. And a team at Johns Hopkins will help compare her movements with those of other kids, both with and without A-T, to observe whether the expected disease symptoms are delayed.

One serious challenge facing gene drugs for individuals is that short of a healing miracle, it may ultimately be impossible to be sure they really work. Thats because the speed with which diseases like A-T progress can vary widely from person to person. Proving a drug is effective, or revealing that its a dud, almost always requires collecting data from many patients, not just one. Its important for parents who are ready to pay anything, try anything, to appreciate that experimental treatments often dont work, says Holly Fernandez Lynch, a lawyer and ethicist at the University of Pennsylvania. There are risks. Trying one could foreclose other options and even hasten death.

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Kuzu says his family weighed the risks and benefits. Since this is the first time for this kind of drug, we were a little scared, he says. But, he concluded, theres nothing else to do. This is the only thing that might give hope to us and the other families.

Another obstacle to ultra-personal drugs is that insurance wont pay for them. And so far, pharmaceutical companies arent interested either. They prioritize drugs that can be sold thousands of times, but as far as anyone knows, Ipek is the only person alive with her exact mutation. That leaves families facing extraordinary financial demands that only the wealthy, lucky, or well connected can meet. Developing Ipeks treatment has already cost $1.9 million, Margus estimates.

Some scientists think agencies such as the US National Institutes of Health should help fund the research, and will press their case at a meeting in Bethesda, Maryland, in April. Help could also come from the Food and Drug Administration, which is developing guidelines that may speed the work of doctors like Yu. The agency will receive updates on Mila and other patients if any of them experience severe side effects.

The FDA is also considering giving doctors more leeway to modify genetic drugs to try in new patients without securing new permissions each time. Peter Marks, director of the FDAs Center for Biologics Evaluation and Research, likens traditional drug manufacturing to factories that mass-produce identical T-shirts. But, he points out, its now possible to order an individual basic T-shirt embroidered with a company logo. So drug manufacturing could become more customized too, Marks believes.

Custom drugs carrying exactly the message a sick kids body needs? If we get there, credit will go to companies like Ionis that developed the new types of gene medicine. But it should also go to the Kuzusand to Brad Margus, Rohan Seth, Julia Vitarello, and all the other parents who are trying save their kids. In doing so, they are turning hyper-personalized medicine into reality.

Erika Check Hayden is director of the science communication program at the University of California, Santa Cruz.

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If DNA is like software, can we just fix the code? - MIT Technology Review

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Several years ago, I was working in the White House Office of Science and Technology Policy where I had the privilege of helping lead the effort to develop President Obamas Precision Medicine Initiativean effort that aimed to catalyze a new era of medicine where patients receive the right treatments at the right time.

On the day of the launch, a young man named Bill Elder came to visit me in my office. He had bright eyes, brown hair, and a big grin on his faceall of which was remarkable, because Bill also had a rare, deadly disease called cystic fibrosis. And at 27 years old, he was about to hit the median age of survival.

Cystic fibrosis is a progressive, genetic disease that causes frequent and difficult to treat infections, and over time, limits the ability to breathe. Growing up, Bill had been able to manage some of his symptoms with therapy and medication, but he also knew that most CF patients didnt live past their twenties. While steady advances were extending life expectancy by a few years at a time, there was no treatment that would address the root cause of his disease, and little hope that one would arrive in time to save his life.

And yet, it didbecause around the time that Bill was born, a critical alignment was emerging between CF patients and researchers. The Cystic Fibrosis Foundation, founded in 1955, had spent decades organizing the patient community and building a national patient registry. That paved the way for new researchand in 1989, a team of scientists led by now-NIH Director Francis Collins identified the gene responsible for cystic fibrosis.

One breakthrough led to the next. The Cystic Fibrosis Foundation set up a clinical trial network and funded therapeutics development and drug screening efforts to search for treatments. And it worked. In 2012, the FDA approved a new, first-of-its kind treatment that addressed the underlying cause of the disease for a small subset of CF patients.

Bill was among the first five patients to receive the drug. He took his first pill before he went to bed one night in February 2012and at about 2 a.m., he woke up with a sensation he hadnt felt in 15 years. He could breathe through both nostrils. Not only that, he could actually smell. The drug was working, and Bill realized that his future had changed overnight.

Years later, that story still floors me. Bill now expects to live a full and healthy life. While the drug that helped Bill was initially approved for only 4% of CF patients, it opened the door to a new treatment approach that is now poised to help 90% of the community.

For me, the central lesson here is that a highly organized group of patients can play a pivotal role in accelerating medical research. Today, Im on a quest to see how we can empower more patient communities to lead the fight against their own rare diseases.

The need is immense. Like cystic fibrosis, many rare diseases are deeply debilitating, if not deadly. And although they are individually rare, they are remarkably common. Experts believe there are at least 25 to 30 million Americans living with a rare disease, and roughly 400 million worldwide.

Unlike cystic fibrosis, though, for the vast majority of the worlds rare diseases treatments are not yet in sight. In fact, 95% of diseases do not have an approved treatment. The reasons are multifold: from difficulty identifying patients for studies to difficulty collecting patient data, from a lack of awareness to a lack of funding to support research.

What this means is that there are hundreds of millions of rare disease patients, half of them children, who are left with little hope. Their disorders might be every bit as threatening as a common cancer or heart diseaseor more. But for the vast majority of these diseases, the science isnt moving forward fast enough. Thirty percent of children with a rare disease die before their fifth birthday.

The good news is that we dont need to accept that kind of inequity. When patient communities have the tools and resources to organize themselves, they can dismantle the barriers to medical researchand drive progress toward treating even the rarest diseases.

In fact, this is already happening. David Fajgenbaum is a medical professor who has Multicentric Castleman disease, a rare and deadly immune disorder. Little was known about Castleman disease when Fajgenbaum was diagnosed in 2010. Patients tended to be isolated, and the research community was fractured. Fajgenbaum decided that the best hope for saving his own life would be to get the Castleman community working together.

[Image: iStock]Today, more than 1,400 patients, clinicians, and researchers are doing exactly that. Together, the Castleman Disease Collaborative Network has defined research objectives, funded projects, shared dataand deepened our understanding of the disorder. Fajgenbaum has applied those insights to his own treatment. And he has driven his disease into remission.

Other rare disease patients are building research networks and infrastructure to study their diseases. In 2007, Josh Sommer created the Chordoma Foundation, which has enabled the identification of more than 20 drug targets and launched seven clinical trials. The Tuberous Sclerosis Alliance, led by Kari Rosbeck, has enrolled more than 2,000 patients in its network and driven groundbreaking research into the disease. The list goes on.

One thing these groups have in common is the belief that they can lower the barriers to studying their diseases. Our goal at the Chan Zuckerberg Initiative is to help them.

As a first step in this project, called Rare As One, weve made grants to 30 rare disease organizations to support them in building patient-led research networks. In addition to funding, we will work to develop the internal capacity of these organizations to achieve their missions. We will encourage these groups to collaborate with one another, and develop a common body of knowledge about how best to build, sustain, and direct their networks. By working in partnership, we aim to develop a set of tools and resources that can be applied to the fight against many rare diseases.

Weve also started working directly with rare disease leaders like Brian Wallach, who is building a movement to fight amyotrophic lateral sclerosis (ALS). Together, were identifying software tools and infrastructure that could help other rare disease communities expand their reach and achieve their priorities.

Of course, there are more than 7,000 different rare diseases, and a strategy that works for one community may not always transfer to another. But in the coming years, we hope to get to the point where any group of patients will have a basic road map they can followto organize their communities, define their priorities, work directly with researchers, and advance the science of their disease.

Thats a future where fewer people are losing everything to a little-known disease for which there was no hopeful path to follow. And its a world where there are more people like Bill Elderwho, now a young doctor himself, has dedicated his life to saving others.

Tania Simoncelli, CZI Science Policy director and Rare As One project lead. For the past 20 years, Tania Simoncelli has designed advocacy strategies and policy solutions to address complex issues at the intersection of science, technology, law, and ethics.

Currently, she leads Science Policy at the Chan Zuckerberg Initiative. Previous roles include positions at the Broad Institute of MIT and Harvard, the White House Office of Science and Technology Policy, the U.S. Food and Drug Administration, and the American Civil Liberties Union. In 2013, Simoncelli was named by the journal Nature as one of 10 people who mattered for her work in spearheading the ACLUs successful Supreme Court case challenging the patenting of human genes.

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Patients are leading the way in the fight against rare diseases - Fast Company

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A Manchester dad who was diagnosed with breast cancer says he was left "isolated and lonely" due to the lack of support for people with the condition.

David McCallion, 55, is one of just around 390 men a year diagnosed with the condition in the UK.

He was forced to have mastectomy and undergo both chemotherapy and radiotherapy as part of his treatment.

However he says he was left feeling lonely and isolated after being "declined membership" for support groups for people with the condition as he was a man.

He says he is trying to break down taboos and increase understanding of it to help other people in his position.

David, who has two sons, builder, Ryan, 27, and communications professional, Liam, 25, with his carer wife, Julie, 54, said: "When I tell other blokes about my diagnosis, half say, 'Men don't get breast cancer,' and I lift up my shirt and say, 'Yes they do.'

"Some ask why I've had it, as if to say, 'Are you really a man?'

"I don't care what people say about that sort of thing, though. I've been married for 30 years, I have two sons and two grandkids."

After his diagnosis, he says he did not know any other male sufferers to share his experience with, so looked online.

"I tried to join a support group on Facebook," he says.

"But they sort of politely told me that because I was a man it might prevent members from opening up - so I thought it was best that I didn't join. I was effectively declined membership.

"I was made to feel like I was muscling in, but the last thing I wanted to do was jump up and down saying, 'Look at me I've got breast cancer too.'

"But if the other 389 men feel anything like I did, something needs to be done for men with breast cancer."

"I will never be the same person I was before my diagnosis because, politely speaking, I didn't realise how lonely this disease is," he continued.

"Cancer is lonely full stop. But being a man in what I call the 'pink world' of breast cancer - that's even lonelier."

David was diagnosed with gynaecomastia in 2015, a common condition causing men's breasts to become larger than normal, in April 2019. He noticed his right nipple was inverted but thought it must be related to the condition.

"I'd been seeing my GP for about a year, as I'd been struggling with anxiety after losing my mum, Joan at 91, in June 2018, after a long battle with dementia and breaking her hip," he said.

"Then, in May 2019, I was just leaving his surgery, when I lifted my jumper and said, 'What's this?'

"The doctor looked at my nipple and told me not to panic, but said that we had to rule a few things out."

He was referred to the Royal Oldham Hospital on July 10, 2019, however David said at that point he was not "overly concerned."

"I just assumed it was something to with my man boobs," he said.

Given a physical examination, a mammogram and an ultrasound scan, doctors then told him he needed a biopsy, where a sample of tissue is taken and examined more closely.

"They were able to rule out a cyst right away and 20 mins after the biopsy the doctor said that, in his opinion, there was a 99 per cent chance it was going to be cancer," said David.

"The first thing I thought was, 'How the hell am I going to tell my family?'

"My second thought was, 'How am I going to tell everybody else I have breast cancer - as a man?'"

"I didn't know whether I was coming or going - my head was completely gone," he added.

Returning to hospital with his wife on July 24, a consultant explained that David had grade two invasive ductal carcinoma, which is found in the breast duct and surrounding tissue.

According to Prevent Breast Cancer - which says 80 men die each year of the disease in the UK - it is the most common form of breast cancer in men and women.

"For the next eight minutes everything the doctor said was addressed to my wife and, in the end, I had to tell him to talk to me and not to her.

"I got quite angry, but later apologised and explained that I've got to own the breast cancer - it's mine.

"He explained that he breaks this news to women day in day out, which is why he didn't address me at first. He's been brilliant ever since, though."

He was told a full mastectomy on his right breast was the best course of action, although David asked for the surgery to be delayed until after his 30th wedding anniversary, on August 19.

"The doctor told me the goal was to cure the cancer, but they wanted to do everything possible to make sure it didn't come back," he continued.

"But it's not your 30th wedding anniversary every day and I wanted to celebrate that before the operation.

"We had a bit of a road trip, heading down to Birmingham to meet family, before spending a weekend in London."

David, who also suffers with an abdominal aortic aneurysm (AAA) - a bulge in the aorta, the main blood vessel that runs from the heart down through the chest and tummy - had a mastectomy at North Manchester General Hospital in Setpember.

He also had a lymph node removed to test for traces of cancer.

David said: "The doctors told me that having anaesthetic could raise my blood pressure and cause complications, but vascular surgeons were there as a back-up during the mastectomy.

"Julie was with me until I had to put my gown on and I gave her a quick hug and said, 'See you later.' I didn't want her to think I was worried, but with the added complication of the AAA, of course I was."

Surrounded by smiling faces when he came to from the two-hour operation, David was told his surgery had been a success.

He was not offered reconstructive surgery and said he does not want it.

After the lymph node tested positive for cancer, on October 4, David had a further 24 removed.

Luckily, none of the other lymph nodes were cancerous, but on December 5, he began a gruelling regime of chemotherapy, having treatment every week, which will finish at the end of next month.

He will then have 15 days of radiotherapy in April, before taking Tamoxifen, a hormone therapy that helps prevent the disease returning, for five to 10 years.

"The side effects have been crap," he said.

"I've had infection after infection, upset bowels and a sinus infection that has lasted for nine weeks.

"It's been a bit of a rocky ride to say the least, but people have it a lot worse than me, so I thank my lucky stars."

David also faces the prospect of his breast cancer being hereditary, as his mother had it in her eighties.

Once his treatment has finished, he will be tested for the BRCA1 and BRCA2 gene mutations, which run in families and increase the chances of developing breast, ovarian, colorectal and prostate cancer, according to the NHS.

David said: "My mum's cancer was similar to mine, so it is a concern that it could be hereditary.

"The doctors haven't given any suggestion that it is and we don't know for definite what kind of cancer Mum's was - it was so long ago - so all we can do is hope that it's not hereditary.

"I've not been tested for the BRCA1 of BRCA2 genes yet, they're waiting until after treatment.

"If I do have them, my boys will have to be checked, too, but I've told them they don't need to worry until I've been tested.

"I just hope my breast cancer is some fluke or case of bad luck and not hereditary."

As David went through his treatment, he has found out about #bluegetittoo - an awareness campaign about male breast cancer started by a woman whose partner was diagnosed.

"Not long after I was effectively declined membership of a support group, I was on the internet looking for places to talk about male breast cancer and found a Facebook page set up by Lorraine Milligan, who wanted to offer support after her partner was diagnosed.

"At the time, there were only about 11 people that had liked it, but it was great to see people talking about male breast cancer with the hashtag #bluegetittoo."

Now David is keen to make men aware that breast cancer can affect them, too, and is urging them to check themselves for tell-tale symptoms.

"Men need to talk about their health more," he said. "By the time they finally do it's often too late.

"There's a culture of checking down below for testicular cancer and that needs to be broadened to include other areas, including the chest area."

"Pick a day of the month - it could be a birthday or pay day - but pick a number and check your body once a month on that day," he continued.

"Feel your chest, look at your body and examine it properly.

"Men always turn up at the doctors late. I should have gone as soon as my inverted nipple appeared.

"A lot of people don't believe this happens, but 390 men are diagnosed with this disease every year, so male breast cancer is definitely real."

Follow this link:
Brave dad on 'loneliness' that comes with having breast cancer as a man - Manchester Evening News

Recommendation and review posted by Bethany Smith

Some of the most notorious low testosterone myths center around male pattern baldness.

Some myths say, on the one hand, that too much testosterone causes balding. On the other hand, some myths say that low testosterone leads to balding.

Is testosterone really causing men to lose the hair on their head?

What the evidence shows is pretty interesting. If youre concerned about hair loss, you should be looking into the genetics of male pattern baldness.

Androgenic alopecia (the scientific term for male pattern baldness) is often wrongly attributed to hormones.

Its true that those experiencing male pattern baldness have particular hormonal profiles, and hormones certainly play a role.

Men experiencing male pattern baldness usually have a high level of dihydrotestosterone (DHT), which is a byproduct of testosterone that has been broken down for use by the body.

DHT has been proven to shrink hair follicles, which makes it impossible for healthy hair to survive. Its then easy to incorrectly conclude that its the amount of DHT circulating in the system thats causing the shrinkage of hair follicles and subsequent hair loss.

More DHT doesnt necessarily mean youre going to lose your hair.

Male (and female) pattern baldness is actually the result of DHT acting under a specific set of genetic conditions.

Some men are genetically predisposed to developing hair follicles that react strongly and negatively to the presence of DHT. Their follicles have an increased number of receptors that allow DHT to bind to them and cause the follicles to constrict.

Hair loss is almost entirely determined by this genetic increase in the number of receptors, not on the amount of DHT in mens systems.

The catch is that only men with this specific genetic increase in receptors have this reaction and lose their hair.

The gene that determines DHT sensitivity is found on the female X chromosome.

Men have one X chromosome, and one Y chromosome. If that single X chromosome contains the sensitivity gene, they inherit the sensitivity.

On the other hand, women have two X chromosomes and no Y chromosome. That means that they get a second chance. Both X chromosomes would have to possess the sensitivity gene, which is a rare occurrence.

Its that most basic genetic makeup, XY for men and XX for women, that sets up a relatively high statistical occurrence of baldness in men, but a low occurrence in women.

Most testosterone myths have developed as a result of observing differences between men and women, and people assumed the natural differences in testosterone levels was the determining factor.

Thats simply not true in the case of male pattern baldness.

Male pattern baldness is primarily a hereditary trait, and some genetic predictions can be made: If you have a high occurrence of male pattern baldness in your family, the chances are high that you, as a man in that genetic line, have the genetic factor, too.

After having said all of that about genetics determining male hair loss, there is a different kind of hair loss you should keep an eye on: Thinning body and facial hair (one of the more common signs of low testosterone).

If youve found that your beard is thinning or that youre losing body hair, it could be a symptom of low testosterone.

(Read more about the many symptoms of low testosterone here.)

Theres a definite positive conclusion to be drawn from all of this information: If youre suffering from low testosterone and currently have a full head of hair, the supplemental testosterone injections used for testosterone replacement therapy (TRT) wont suddenly cause you to develop male pattern baldness.

That means you can utilize the benefits of a regimented TRT plan that has the potential to restore your quality of life and help you feel like yourself again, all without the fear of suddenly losing your full head of hair.

If you still have questions regarding the genetics of male pattern baldness, or if you would like to learn more about the role of hormones in hair loss, we invite you to check out some further information we have available on the subject click the button to learn more.

Read More

The rest is here:
The Genetics of Male Pattern Baldness - tctmed.com

Recommendation and review posted by Bethany Smith

What is a genetic disease?

A genetic disease is any disease caused by an abnormality in the genetic makeup of an individual. The genetic abnormality can range from minuscule to major -- from a discrete mutation in a single base in the DNA of a single gene to a gross chromosomal abnormality involving the addition or subtraction of an entire chromosome or set of chromosomes. Some people inherit genetic disorders from the parents, while acquired changes or mutations in a preexisting gene or group of genes cause other genetic diseases. Genetic mutations can occur either randomly or due to some environmental exposure.

What are the four types of genetic disorders (inherited)?

There are a number of different types of genetic disorders (inherited) and include:

The baby with Down syndrome has a hallmark appearance. However, every aspect of the appearance does not need to be present as the phenotype, the way the genes make the child look, can be markedly different for each patient. Common Down syndrome symptoms are:

7 single gene inheritance disorders

Single gene inheritance is also called Mendelian or monogenetic inheritance. Changes or mutations that occur in the DNA sequence of a single gene cause this type of inheritance. There are thousands of known single-gene disorders. These disorders are known as monogenetic disorders (disorders of a single gene).

Single-gene disorders have different patterns of genetic inheritance, including

Some examples of single-gene disorders include

7 common multifactorial genetic inheritance disorders

Multifactorial inheritance is also called complex or polygenic inheritance. Multifactorial inheritance disorders are caused by a combination of environmental factors and mutations in multiple genes. For example, different genes that influence breast cancer susceptibility have been found on chromosomes 6, 11, 13, 14, 15, 17, and 22. Some common chronic diseases are multifactorial disorders.

Examples of multifactorial inheritance include

Multifactorial inheritance also is associated with heritable traits such as fingerprint patterns, height, eye color, and skin color.

4 chromosomal abnormalities

Chromosomes, distinct structures made up of DNA and protein, are located in the nucleus of each cell. Because chromosomes are the carriers of the genetic material, abnormalities in chromosome number or structure can result in disease. Chromosomal abnormalities typically occur due to a problem with cell division.

For example, Down syndrome (sometimes referred to as "Down's syndrome") or trisomy 21 is a common genetic disorder that occurs when a person has three copies of chromosome 21. There are many other chromosomal abnormalities including:

Diseases may also occur because of chromosomal translocation in which portions of two chromosomes are exchanged.

3 mitochondrial genetic inheritance disorders

This type of genetic disorder is caused by mutations in the non-nuclear DNA of mitochondria. Mitochondria are small round or rod-like organelles that are involved in cellular respiration and found in the cytoplasm of plant and animal cells. Each mitochondrion may contain 5 to 10 circular pieces of DNA. Since egg cells, but not sperm cells, keep their mitochondria during fertilization, mitochondrial DNA is always inherited from the female parent.

Examples of mitochondrial disease include

What is the human genome?

The human genome is the entire "treasury of human inheritance." The sequence of the human genome obtained by the Human Genome Project, completed in April 2003, provides the first holistic view of our genetic heritage. The 46 human chromosomes (22 pairs of autosomal chromosomes and 2 sex chromosomes) between them house almost 3 billion base pairs of DNA that contains about 20,500 protein-coding genes. The coding regions make up less than 5% of the genome (the function of all the remaining DNA is not clear) and some chromosomes have a higher density of genes than others.

Most genetic diseases are the direct result of a mutation in one gene. However, one of the most difficult problems ahead is to further elucidate how genes contribute to diseases that have a complex pattern of inheritance, such as in the cases of diabetes, asthma, cancer, and mental illness. In all these cases, no one gene has the yes/no power to say whether a person will develop the disease or not. It is likely that more than one mutation is required before the disease is manifest, and a number of genes may each make a subtle contribution to a person's susceptibility to a disease; genes may also affect how a person reacts to environmental factors.

Medically Reviewed on 10/17/2019

References

United States. National Heart, Lung, and Blood Institute. "Cystic Fibrosis." <https://www.nhlbi.nih.gov/health-topics/cystic-fibrosis>.

United States. National Human Genome Research Institute. "Frequently Asked Questions About Genetic Disorders." <https://www.genome.gov/19016930/faq-about-genetic-disorders/>.

See the article here:
21 Common Genetic Disorders: Types, Symptoms, Causes ...

Recommendation and review posted by Bethany Smith

The likelihood of conceiving twins is a complex trait. It is probably affected by multiple genetic and environmental factors, depending on the type of twins. The two types of twins are classified as monozygotic and dizygotic.

Monozygotic (MZ) twins, also called identical twins, occur when a single egg cell is fertilized by a single sperm cell. The resulting zygote splits into two very early in development, leading to the formation of two separate embryos. MZ twins occur in 3 to 4 per 1,000 births worldwide. Research suggests that most cases of MZ twinning are not caused by genetic factors. However, a few families with a larger-than-expected number of MZ twins have been reported, which indicates that genetics may play a role. It is possible that genes involved in sticking cells together (cell adhesion) may contribute to MZ twinning, although this hypothesis has not been confirmed. Most of the time, the cause of MZ twinning is unknown.

Dizygotic (DZ) twins, also called fraternal twins, occur when two egg cells are each fertilized by a different sperm cell in the same menstrual cycle. DZ twins are about twice as common as MZ twins, and they are much more likely to run in families. Compared with the general population, women with a mother or sister who have had DZ twins are about twice as likely to have DZ twins themselves.

DZ twinning is thought to be a result of hyperovulation, which is the release of more than one egg in a single menstrual cycle. To explain how DZ twinning can run in families, researchers have looked for genetic factors that increase the chance of hyperovulation. However, studies examining the contributions of specific genes have had mixed and conflicting results. Few specific genes in humans have been definitively linked with hyperovulation or an increased probability of DZ twinning.

Other factors known to influence the chance of having DZ twins include the mothers age, ethnic background, diet, body composition, and number of other children. Assisted reproductive technologies such as in vitro fertilization (IVF) are also associated with an increased frequency of DZ twins.

Hoekstra C, Zhao ZZ, Lambalk CB, Willemsen G, Martin NG, Boomsma DI, Montgomery GW. Dizygotic twinning. Hum Reprod Update. 2008 Jan-Feb;14(1):37-47. Epub 2007 Nov 16. Review. PubMed: 18024802.

Machin G. Familial monozygotic twinning: a report of seven pedigrees. Am J Med Genet C Semin Med Genet. 2009 May 15;151C(2):152-4. doi: 10.1002/ajmg.c.30211. PubMed: 19363801.

Mbarek H, Steinberg S, Nyholt DR, Gordon SD, Miller MB, McRae AF, Hottenga JJ, Day FR, Willemsen G, de Geus EJ, Davies GE, Martin HC, Penninx BW, Jansen R, McAloney K, Vink JM, Kaprio J, Plomin R, Spector TD, Magnusson PK, Reversade B, Harris RA, Aagaard K, Kristjansson RP, Olafsson I, Eyjolfsson GI, Sigurdardottir O, Iacono WG, Lambalk CB, Montgomery GW, McGue M, Ong KK, Perry JR, Martin NG, Stefnsson H, Stefnsson K, Boomsma DI. Identification of Common Genetic Variants Influencing Spontaneous Dizygotic Twinning and Female Fertility. Am J Hum Genet. 2016 May 5;98(5):898-908. doi: 10.1016/j.ajhg.2016.03.008. Epub 2016 Apr 28. Pubmed: 27132594.

Painter JN, Willemsen G, Nyholt D, Hoekstra C, Duffy DL, Henders AK, Wallace L, Healey S, Cannon-Albright LA, Skolnick M, Martin NG, Boomsma DI, Montgomery GW. A genome wide linkage scan for dizygotic twinning in 525 families of mothers of dizygotic twins. Hum Reprod. 2010 Jun;25(6):1569-80. doi: 10.1093/humrep/deq084. Epub 2010 Apr 8. PubMed: 20378614. Free full-text available from PubMed Central: PMC2912534.

Shur N. The genetics of twinning: from splitting eggs to breaking paradigms. Am J Med Genet C Semin Med Genet. 2009 May 15;151C(2):105-9. doi: 10.1002/ajmg.c.30204. PubMed: 19363800.

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Recommendation and review posted by Bethany Smith

Can you guess the single most important tool in preventing neglected, stray and unwanted animals? Yep, its spaying and neutering. This one, fairly simple procedure has made a tremendous impact on controlling the number of animals coming into shelters across the nation.

World Spay Day is Tuesday and its a great time to be reminded of how this one effort can prevent animal homelessness. Yet, some people are still hesitant to get their pets spayed or neutered, often due to misinformation or misunderstanding surrounding the procedure.

Here are some common myths about spay and neuter debunked by Humane Society International.

Myth: My pet needs to have a litter/one heat before sterilization.

Fact: Medical evidence indicates just the opposite. In fact, the evidence shows that females spayed before their first heat are typically healthier.

Myth: Its not natural to spay or neuter and will upset my dog or cat.

Fact: The domestication of animals removed them from the natural order and placed responsibility for their care with humans. Applying human emotions to animals is neither realistic nor applicable when it comes to identifying a need for sterilization.

Myth: I want my dog to be protective.

Fact: It is a dogs natural instinct to protect home and family. A dogs personality is formed more by genetics and environment than by sex hormones.

Myth: I do not want my male dog or cat to feel like less of a male.

Fact: Pets do not have any concept of sexual identity or ego. Neutering will not change a pets basic personality. The pet does not suffer any kind of emotional reaction or identity crisis when neutered.

Myth: My pet will get fat and lazy.

Fact: The truth is that most pets get fat and lazy because their guardians feed them too much and do not give them enough exercise.

Myth: But, my dog (or cat) is so special, I want a puppy (or kitten) just like them.

Fact: Your pets puppies or kittens have little chance of being an exact copy of your pet. Even professional breeders cannot make this guarantee. There are homeless pets waiting for homes who are just as cute, smart, sweet and loving as your own.

Spaying or neutering also helps keep dogs and cats healthy by reducing or eliminating the possibility of uterine infection, mammary tumors, prostate problems and certain types of cancers. Neutering male dogs and cats also eliminates the urge to seek out females in heat (sometimes by creatively escaping from their house or yard), thus reducing the risks associated with free-roaming animals.

Marin Humane offers spay and neuter services for the pets (including rabbits) of low-income Marin County residents. And for pit bulls and pit bull mixes, spay and neuter services are free for Marin County residents. We also collaborate with local organizations like Marin Friends of Ferals to sterilize feral cats and kittens, and provide vouchers to the public to subsidize spay and neuter surgeries for feral cats by local veterinarians.

As the saying goes, prevent a litter and fix your critter.

For more information about Marin Humanes spay and neuter services for low-income residents, call 415-883-3383 or go to marinhumane.org.

Lisa Bloch is the marketing and communications director for Marin Humane, which contributes Tails of Marin articles and welcomes animal-related questions and stories about the people and animals in our community. Go to marinhumane.org, Twitter.com/marinhumaneor email lbloch@marinhumanesociety.org.

Go here to read the rest:
The most important way to prevent neglected, stray and unwanted animals - Marin Independent Journal

Recommendation and review posted by Bethany Smith

It is far easier to say about being content with what we have than actually being content. Many males are discontent with their penis size due to which you can see penis enlargement and viagra searches skyrocketing on search engines across the world. There are thousands of products on the internet which claim to enlarge the penis. But the reality is, most of them dont work, and the ones which work may cause side effects. However, Massive Male Plus is a unique product amongst the heap of penis enlargement products.

Massive Male Plus is helpful for all the males who are suffering from small penis syndrome, low hormone levels or erectile problems. Even males with diabetes can consume this supplement without any worries. You dont have to follow any dietary restrictions for taking this supplement. Massive Plus has no negative implications or side effects, and it guarantees 100% results.

Massive Male Plus claims to be an outstanding male enhancement supplement that increases your peniss length with a minimum of 3 inches within 30 days. It also declared that about 64,000 males had experienced its benefits. The average length of the penis is approximately 5 inches, and it differs from person to person if the length is satisfactory or not. If an individual or his partner is not satisfied with such length, then they can try this product. Theres no reason to just accept 5 inches as the fixed-length if theres a scope of having a few extra inches, right?

Massive Male Plus is a natural supplement with 14 foods and herbs from equatorial countries, which will help you to increase your penis size in a month. As it is a natural male enhancer product, you dont have to worry about any side-effects.

You might be thinking about how exactly this supplement works to fulfill the unsaid desire of having a larger penis. Just like any organ, the penis has tissues with filled-up blood. When there is proper blood flow to the erectile tissue, it enlarges and turns the penis erect like a balloon inflated with air.

Viagra is a popular drug that keeps the penis erect for a longer time, but it doesnt increase the length of the penis. Viagra just ensures that your penis holds more blood to stay erect. On the other hand, Massive Male Plus fills your erectile tissue with more blood flow due to which, the size of the tissue also increases. Hypergenesis or hyperplasia is responsible for this enlargement. It occurs when cell proliferation increases the amount of organic tissue.

The critical ingredients of Massive Male Plus get sourced from equatorial countries, including Congo, Ghana, and Nigeria. These ingredients include distinctive versions of Vitamin B3, and Vitamin E found in Liriosma Ovata (known as the Viagra of the Amazon), unaltered species of Damiana aphrodisiaca, and herbs like Entengo and Mkongoraa. Now you can see why Africans have the most gigantic penis on the planet! These ingredients enable Massive Male Plus to give super abilities for the penis.

The following are the ingredients in Massive Male Plus:

To impact the bodys erectile response, Chinese Hawthorn improves the flow of blood to the penis. This leads to bigger and harder erections which would help you to perform well in bed.

Epimedium is an active component found in horny goat weed. This compound helps in blocking the work of an enzyme that is responsible for the restriction of blood flow to your penis.

Damiana ensures that the small muscles present in the arterial walls of your penis remain relaxed. As a result, there is a higher flow of blood to the penis, and thus, you get a stronger erection.

Also called intensity wood, Muira Puama has properties that enhance libido and reproductive capacity.

Ginkgo ensures that the effects of nitric oxide enhance due to which, artery walls relax, and there is an increase in the flow of blood into your penis.

Asian Ginseng, traditionally used in Chinese medicine, enhances sexual behavior and treats sexual dysfunction.

Tribulus, a recommended ingredient for vitality and virility of male health, has the properties to enhance libido and boost testosterone.

Catuaba helps to enhance sexual excitement and experience powerful orgasms. It is also responsible for stimulating the flow of blood into the genitals to prolong and strengthen an erection.

Saw Palmetto is a herb that enhances the sexual performance of males. It also intensifies male orgasms.

Oat Straw increases the levels of nitric oxide and provides vasodilation for improving circulation. Your erection quality will improve when there is an improvement in the supply of blood to your penis.

Cayenne has an adequate quantity of nutrients that help in healing any damage around the area of the penis. One should treat such damages sooner as they might inhibit the quality of the erection.

The following are the benefits of Massive Male Plus, which you would gain after using it regularly:

Each bottle of Massive Male Plus contains 60 capsules. The Basic package offers one bottle at $69 while the Standard package offers two bottles at $59. With the Premium package, you will get four bottles at $196 (50% discount), only at the official website.

Does Massive Male Plus offer any money-back guarantee?

Massive Male Plus offers a 60-day money-back guarantee. So, you can return this supplement if you arent satisfied with its results. However, we suggest you use this supplement for at least 30 days to get good results.

You can send the bottles back to their shipping address for getting a full refund. You will get a Return and Refund Form with the package on delivery. The bottle has to get filled to avail of a refund. Also, you can even send back the bottles on the 59th day of your guarantee period as only the day of reshipment matters for the company. Its totally fine if the package reaches after the guarantee period. Just ensure that the package gets shipped within 60 days.

Can I intake Massive Male Plus capsules even if I have allergies?

The ingredients of Massive Male Plus are in quantities that are below the allergy-triggering levels. So there are no side-effects on individuals with various allergies.

I currently use some supplements to fulfill my vitamin levels. Will Massive Male Plus interfere with those supplements?

Massive Male Plus will target only the erectile problems in your body, and so, it wont interfere with other supplements.

How soon will I get to see the results?

Theres a high possibility that you will see the results from the 1st day itself. This supplement immediately gives the internal effects, and you will experience the visible results in a maximum of 30 days.

Will Massive Male Plus cause any side-effects?

Massive Male Plus causes Zero side-effects, being it formulated with natural ingredients.

By Alex L.

I heard that Africans have larger penises than other parts of the world. I assumed that genetics was the reason behind the same. Although genetics played some role, I thought there must be some type of herbs that do the work. Like many males out there, I wished for a bigger penis, and so, I started searching online for penis enlargement products. I went through almost a dozen products, but I wasnt sure if it would really work for me. But I had an intuition that out of those products, Massive Male Plus will definitely give me results due to its ingredients and firm assurance of results. I consider myself lucky to get the results from Day 1. Hats off to this amazing supplement!

By Tom A.

Instead of paying thousands of dollars for a penis enlargement surgery, you should try this supplement which has reasonable costs. This supplement helped me to regain my sexual confidence and made me feel powerful in bed.

By Jimmy V.

Whenever I hear Size doesnt matter from anyone, I feel like punching them right on their face. Size does matter folks! A few more inches provides more satisfaction and even a bit more admiration from your partner. The bigger your thing is, the better you will be able to escalate things on an ultimate level in your bed. I surfed online to find a product that really makes the penis thicker, longer and more prominent. Whenever I used to land on a product that made too many claims, I started doubting the authenticity of the product. The two things which made me purchase Massive Male Plus were its natural ingredients and 60-day money-back guarantee. Even if the product wouldnt have worked for me, I had nothing to lose due to their money-back guarantee. But it worked well for me, a lot more than I expected. The results are just unbelievable! Take these capsules and witness their magic. I have been taking them for two months now, and I can assure you that these capsules wont disappoint you.

By Michael H.

It used to think that it was impossible to enlarge a penis. Still, with Massive Male Plus, I got to experience the miracle itself!

The individuals consuming Massive Male Plus might experience varied results, but all of them will lead to the same effect! This supplement will help you to enlarge your penis, blossom your sex life and increase your confidence while performing in bed. As this supplement is made up of natural ingredients, you wont face any side-effects. You should give Massive Male Plus a try if you are looking for something which would be a life-changing factor to level up your sex life.

Read more:
Massive Male Plus Review - Is This Supplement Worth Buying? - Jollofnews

Recommendation and review posted by Bethany Smith

Polycystic ovary syndrome, or PCOS, is a hormonal disorder in women that can cause irregular periods, abnormal amounts of facial and body hair, infertility, among other symptoms.

The Office on Women's Health reports that PCOS affects 1 in 10 women of childbearing age from 15 to 44 years old. And "as we understand it, is a lifelong disorder," says Richard Legro, MD, Professor of Obstetrics and Gynecology and Public Health Services at the Penn State College of Medicine and Penn State Health.

Here's what you need to know about the causes, symptoms, and symptom relief from PCOS.

PCOS occurs when women have abnormally large tiny follicles in their ovaries. Marochkina Anastasiia/Shutterstock

The name polycystic ovary syndrome is a slight misnomer because it doesn't involve traditional ovarian cysts. Instead, people affected by PCOS often have a larger-than-average number of tiny follicles in their ovaries that look like small cysts but are not like traditional ovarian cysts. These follicles grow but never fully develop to release eggs. And if no eggs are released, you don't ovulate.

The follicles themselves aren't dangerous but the hormonal imbalance they cause can wreak havoc with the person's menstrual cycle. Your body may not produce enough of the female reproductive hormone progesterone to maintain a normal menstrual cycle. As a result, PCOS is the most common cause of infertility in women, according to the Endocrine Society.

Though the exact causes of PCOS are unknown, genetics seem to play a key role, though more research is needed to understand by how much this increases a person's risk.

There is also some evidence that environmental factors including exposure to toxins in the environment like plasticizers may contribute to the condition in rare instances.

Obesity has long been thought to be a cause of PCOS, but it may be the case that obesity only aggravates the condition, rather than causes it. That could be because PCOS causes insulin resistance in the body. Regardless of BMI, all women with PCOS have a degree of insulin resistance, but obesity seems to make the condition worse.

Many of the symptoms associated with PCOS are the result of an increase in male hormones, such as testosterone.

That's because many people with PCOS have insulin resistance in their body, which inhibits the process of sending glucose to cells. As a result, the pancreas has to produce more insulin, which causes problems for the endocrine system and leads the body to ramp up the production of androgens, aka male hormones.

Consequently, you may start having very irregular periods or even stop having periods altogether. You may also put on some extra weight although not every woman with PCOS gains weight.

This excessive amount of androgens also tend to cause you to develop a few other symptoms, notably extra hair on your body and face. The extra hair is what Dr. Legro calls "male-patterned hair." Meaning you're not developing a fine layer of hair all over your body. It's hair that appears in areas where men grow body hair, like the middle of the chest, the midline, and the back.

"Excess facial hair, hair thinning and balding: that's not normal," says Dr. Legro. "Get investigated."

While there's no cure for PCOS, the symptoms of the condition can be abated with various treatments. These treatments include weight loss, birth control pills, and anti-androgen medications.

Read this article:
What is PCOS? Symptoms and treatments for polycystic ovary syndrome - Insider - INSIDER

Recommendation and review posted by Bethany Smith

The Global Steroids Market is expected to grow at a CAGR of XX% during the forecast period, 2017-2025.Increase in geriatric population drives the androgens and anabolic steroids market, as older men are more prone to hypogonadism. Additionally, rise in obesity in men propels the global androgens and anabolic steroids market.

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The increasing poor health status especially in the developing countries is projected to fuel the growth of the market during the forecast period. Moreover, rise in government initiatives for better health care is attributed to the growth of the global androgens and anabolic steroids market.This report analyses the future prospects of Global Steroids Market.

Development policies and plans are discussed as well as manufacturing processes and cost structures are also analyzed. This report also states import/export consumption, supply and demand Figures, cost, price, revenue and gross margins.

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Top Key Companies Analyzed in Global Steroids Market are PFIZER Novartis Merck Sanofi Johnson And Johnson GSK Astrazeneca Cipla Sumitomo

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Steroids Market Size 2019: Global Industry Share, Types, Applications, Top Key Players, Demand Analysis and 2025 Forecast Report - News Times

Recommendation and review posted by Bethany Smith

Market Overview The barrier films market is expected to register a CAGR of 5. 53%, during the forecast period 2019 - 2024. The increasing demand for barrier films can be attributed to the increased demand from various end-user industries, such as pharmaceutical packaging, food & beverage packaging.

New York, Feb. 24, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Barrier Films Market - Growth, Trends, and Forecast (2019 - 2024)" - https://www.reportlinker.com/p05865772/?utm_source=GNW

- With the rapid growth of large retail chains, it has greatly benefitted the barrier films market. These retail chains are expanding the market for packaged food, as well as electronics and pharmaceutical items, also, companies are increasingly using barrier films owing to an increased focus on cost reduction and shelf-life extension. - Supermarkets/hypermarkets are becoming more favored by consumers across the globe due to their wide range of product offerings and various choice of premium brands, unavailable at individual outlets. Thus, these supermarkets/hypermarkets are witnessing increased adoption in major cities and are also expanding their presence in developing markets, which will further expand barrier films demand in these countries. - Moreover, barrier films are designed to safeguard electronic components such as transistors, electronic circuits, and other electronic products from degradation caused by moisture/oxygen. Flexible barrier films enclose flexible, organic, and printed electronics to save them from corrosion without affecting their performance and functionality.

Scope of the Report Barrier films are usually multilayer films that are being designed to be impenetrable to gas migration to protect the integrity and quality of the product packed inside it. These films are used to protect various types of product ranging from food to pharmaceuticals to electronics.

Key Market Trends Food Industry to Hold a Major Share in the Market

- The changing food habits associated with the busy lifestyle of people creates a demand for packaged food. Also, the increasing demand for packaged food can be attributed to the improving lifestyles of people, owing to the increase in disposable income, which is driving the market studied. - The snacking trend across the globe is driving consumers to purchase on-the-go food items, while also demanding healthy and improved flavor options. This has driven grocery stores, restaurants, and supermarkets to welcome this trend by offering a variety of packaged foods and ready-to-eat meals, and has further created the demand for flexible and convenient packaging with high barrier properties. - Grab-and-go food (GNG) refers to pre-packaged, ready-to-eat food items often sold at a self-service refrigerator or something similar, which is expected to act as a significant driver for the growth of packaging forms with longer shelf life and greater barrier properties. - With the increasing revenue of the food packaging market, it will drive the growth of the barrier films market as they are being increasingly used in the industry for packing various food products.

North America Occupies the Largest Market Share

- The North American region is witnessing a change in the consumer lifestyle trends, who are demanding convenience and portability of products, especially in the food sector, which directly drives the barriers films market. - The food industry accounts for more than 5% of the US GDP and is still growing, owing to the greater demand for packaged foods. With the advantages like ease of use at home and on-the-go flexibility, have become necessary factors which drive the purchase of packaged foods. - The U.S flexible packaging industry is witnessing a better growth as compared to the rigid packaging market, owing to the unique solutions provided for many packaging challenges. While considering the full life cycle of flexible packaging, it has superior sustainability attributes including using less material, less energy and creating less GHG emissions and waste, thus, making it a more suitable form of packaging. All these factors have contributed significantly to the growth of barrier films in this region.

Competitive Landscape The barrier films market is competitive owing to the presence of small and large vendors. The market is moderately concentrated slowly moving towards the fragmented stage. Key strategies adopted by the major players are product innovation and mergers and acquisition. Some of the key players in the market are Amcor Limited, Bemis Company, Berry Global among others.

- April 2019 - Amcor has launched AmLite Ultra Recyclable, its first packaging product made from the companys revolutionary, more sustainable high-barrier polyolefin film. The new high-barrier laminate can package a range of food, home, and personal care, and pharmaceutical products, and be recycled in existing polyolefin recycling streams.

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The barrier films market is expected to register a CAGR of 5.53%, during the forecast period 2019 - Yahoo Finance

Recommendation and review posted by Bethany Smith

Erza, a LARP character, introduces himself to the camera. Im very pleased to meet you all, he says, I hope we have a great time together as a community. I heard a lot of good things about S.M.I2.L.E., lots of nice people, heard you all like music techno. Erza smiles, clicks his tongue, and then says, Lets go!

His brief cameo catalyzes a dive into the rabbit hole that is S.M.I2.L.E.- A trip into Synesthesia, the latest film by Omsk Social Club, an anonymous group of artists who are best known for incorporating Live Action Role Play (LARP) and Real Game Play (RGP) in their crypto-raves. These events take place across Europe in warehouses or at art institutionsand you have to mine cryptocurrencies as a prerequisite to attend. After successfully using some of your computers processing power for mining cryptocurrencies, Omsk Social Club will assign you a fictional character and storyline that you are expected to LARP (role play) throughout the ravewhich sometimes lasts for an entire weekend.

Euphoric traditions in ritual and raving form the basis of Omsk Social Clubs post political expressiona momentary reprieve from our political anxieties. Rave cultures origins are in the open acceptance and expression of underground Black and Latino gay scenes in Chicago and Detroitoften held in illegal venues like abandoned warehouses or in uncharted open fields. The trend later burgeoned into Europe, where the risk of getting shut down by authorities became part of the thrill.

While the rave is an inherently utopic state, Omsk Social Clubwho prefer not to disclose their identitiesreject an ultimately idealistic and unattainable utopic future: The whole modern world is built on people trying to reach utopia. So how do we use that to make these speculative designs? Rather than circumventing political discourse, Omsk Social Club suggests trying to understand how you can participate in new models of politics.

As with all role-playing, establishing trust is crucial before entering the real game play scenario. Thats why Omsk worked closely and carefully with participants for this new LARP design pre-S.M.I2.L.E. ceremony. Instead of requiring all participants to mine cryptocurrencies as a rite of passage, they repurposed crypto-jargon by asking them for a distributed consensus during a preparatory workshop.

For those unaware, a distributed consensus is what makes the technology backing cryptocurrencies a trusted decentralized network. Cryptocurrencies are not centralized and governed by one entity like a bank. Bitcoins, for example, are reliable digital assets because a mutual consensus is reached by all hosts within the network. Omsk reached this distributed consensus with participants before the ceremony based on peoples energies, sensory deprivation, self divination, and the use of touch to find comfortable spaces of reading someone elses body language, which helped outline acceptable boundaries and incorporated individual expectations.

Rooted in ancient European pagan and alchemic traditions, a S.M.I2.L.E. ceremony is heavily guided by earlier encounters with rave culture. Of course warehouse parties and raves were the first large-scale, ritualistic and ceremonial, community-led practices that we were part of, an Omsk Social Club member explained, We have to recognize there is a strong pagan and occultist history on our doorsteps. And in a sense, this ruined culture was never put to bedit was just nestled between our subcultures.

Even though the film is set in the future, it conjures a hauntological grounds for raving as a spiritual actand returns to pagan and occult traditions from centuries before.

Never forget the mind coils back to the birth of time, for instance, is a phrase often recited by the group. Another LARP player in the film makes a dazed proclamation, Rest in peace the 90s, twenty years later, still the best music generation of its time.

Omsk Social Clubs coming of age experiences with raves in Manchester, Glasgow and the Edinburgh quarries are built into the core of their game-play designs. Whats carried on from this formative time is how these remote countryside parties still depended on a close relationship with technology.

You are completely isolated, one member recounts, But even in a quarryin the middle of Scotlandyou still need the generator, the rig system, a laptop, Ableton Live. These systems of technology move bodies, together and in symmetry. The Omsk member explains a sort of symbiosis: [Society] always positions technology as external to the human. But digital technology has always been an extension of the human, a fabrication of reality, and another version, I guess, of spirituality. So, you begin to see the body as economies of ritual and collectivity.

Originally, Technobodies curator Yvonne Seidel invited Omsk Social Club to LARP an imagined political upheaval but the group was more interested in touching on Timothy Learys S.M.I2.L.E. prophecy as a system of play. Leary, a Harvard lecturer, hallucinogenic drug advocate and futurist, championed the introspective potential for psychedelics as a source for hyper-human performance and collective expansion of consciousness. S.M.I2.L.E. is his anagram for Space Migration, Increased Intelligence, Life Extension, and according to Omsk, it proves to be one of Learys prominent contributions to the 60s psychedelic movement as a cornerstone theory for achieving higher states of consciousness.

Lysergic highs may have been the basis for Learys lifework, but that aspect isnt as important to Omsk Social Club as much as the concept of rewiring consciousness. Expanded consciousness, we would argue, can be reached without a drug and achieved collectivelythrough sound and movement. Thats something that we tried to show with the film, another member says. We often do a disservice to the expectations of the body. If we start rooting around in there, you can get the body to do fantastic things.

S.M.I2.L.E.- A trip into Synesthesia works from a set of spiritual, ritual, and techno-paganist devices, alternating between new age expressions, like techno music and dynamic dance meditation, to, as they term it, bleed the self into one communal experience for enjoyment. Its no different to the experience of mysticism at a rave, when an individual is fully immersed within a crowd of bodies capering to a four-four beat.

Omsks impulse towards utopian-adjacent escapism shapes their unique kind of post-political entertainment. By experimenting with play as a way to deal with our disappointing political climate, their work merges crossovers between long-established cultural phenomena and shared historiescreating and gaming the fictional realities inside systems, within systems, within systems Omsk Social Club encourages the possibility to access multiple unlived realities that subvert societys usual accelerating pace. In their simulations, they hope to uphold an idea of techno-human togetherness, fixed in a temporal state of euphoria.

Jazmina Figueroa is a writer based in Berlin and is currently completing a masters in Anglophone Modernities in Literature and Culture. She has consulted projects and published her work on topics relating to the politics of technology, arts and culture and digital media theory.

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Omsk Social Club Blurs the Lines of Reality Through Raving and Spirituality - Electronic Beats

Recommendation and review posted by Bethany Smith

Melbourne Feb 25, 2020 (Thomson StreetEvents) -- Edited Transcript of OceanaGold Corp earnings conference call or presentation Thursday, February 20, 2020 at 9:30:00pm GMT

* Scott A. McQueen

Good morning and afternoon, ladies and gentlemen, and welcome to the OceanaGold 2019 Full Year Financial Results Webcast and Conference Call. (Operator Instructions) Note that this call is being recorded, Thursday, February 20 at 4:30 p.m. Eastern Time.

And I would like to turn the conference over to Sam Pazuki. Please go ahead, sir.

Thank you, operator. Good evening, good morning, and welcome to OceanaGold's Fourth Quarter and Full Year 2019 Results Webcast and Conference Call. I am Sam Pazuki, the Vice President of Investor Relations for OceanaGold. I am joined today by Mick Wilkes, President and CEO of OceanaGold; along with Michael Holmes, Chief Operating Officer; and Scott McQueen, Chief Financial Officer.

For this webcast, we will initially discuss our 2019 operational and financial performance, and we will then shift the focus of the presentation to the upcoming year before turning it to you for questions.

Moving on to Slide #2. Before we proceed, note that the references in this presentation adhere to International Financial Reporting Standards, and all financial figures are denominated in U.S. dollars, unless otherwise stated. Also note that the presentation contains forward-looking statements, which by their very nature, are subject to some degree of uncertainty. There can be no assurances that our forward-looking statements will prove to be accurate as future results and events could differ materially. Please refer to the disclaimer on forward-looking statements in our presentation.

I will now turn it over to Mick Wilkes.

Michael Francis Wilkes, OceanaGold Corporation - President, CEO, MD & Director [3]

Thank you, Sam, and hello, everybody. It's a pleasure to be with you today from New Zealand. As I've noted before, 2019 was a challenging year for us. We did close the year on a positive note with strong operational and financial performance from Haile and our New Zealand operations, which delivered an increase of nearly 20% in gold production quarter-on-quarter, while all-in sustaining costs decreased 13%.

In Haile, production increased 26% from the third quarter while costs continued to trend lower. In fact, since the start of 2019, we've reduced our all-in sustaining costs by approximately 40% over the course of the year. Mining unit cost decreased from $5.55 per tonne mined in the fourth quarter of 2018 to just over $3 per tonne mined in the fourth quarter of 2019. Processing unit costs were $14.81 per tonne milled in Q4 2018, and just over $12 per tonne milled in Q4 2019. We expect the unit cost to continue to trend lower in 2020.

Macraes also delivered a strong quarter of production which increased 20% quarter-on-quarter, while Waihi delivered steady production as expected. Fourth quarter adjusted earnings per share on a fully diluted basis was better than previous quarter, in line with analysts' consensus figures.

Despite improved performance at the other sites, fourth quarter earnings were impacted by the continued suspension of operations at Didipio with higher corporate, general and administrative costs, including approximately $10 million in carrying costs to maintain Didipio in a state of operational readiness. Fourth quarter cash flow per share before working capital and on a fully diluted basis was $0.02 higher than the previous quarter at $0.07.

In the fourth quarter, we continued to advance the Martha Underground development with approximately 830 meters of development and completion of the ventilation shaft between the levels 800 and 900 drives. Development over the course of the year is expected to gradually increase, and we are on track for first production in the second quarter of 2021.

Exploration continues to yield positive results, particularly at Waihi, where we focus drilling at the Martha Underground and WKP projects. Just last week, we announced an updated resource for Martha Underground Project, which included a significant increase to indicated resources, which now stand at 824,000 ounces of gold, while inferred resources stood at 614,000 ounces. The average grade for both categories at Martha have also increased from the previous resource.

Moving on to Slide 4 and our ESG update. OceanaGold has operated a sustainable business for the past 30 years by applying robust ESG practices across our business. We are proud of our ability to discover orebodies, build projects, operate mines and rehabilitate depleted mines. This is clearly demonstrated by the work we've been doing at the Reefton mine on the West Coast of the South Island of New Zealand. You can see from the photo on the left that the Globe Pit, which operated for about 9 years, is transforming into a new freshwater lake surrounded by native forest. And since the mine closed in 2016, we have completed over 100 hectares of rehabilitation and planted more than 700,000 native seedlings with another 300,000 seedlings to be planted over the next 3 years.

On the social front, we are grateful for the strong show of support from the company -- for the company for the Didipio mine and the renewal of the FTAA at a large, public rally in Manila, as shown on the middle photograph. Approximately 1,200 members of host communities, including indigenous people, traveled 10 hours each way to rally for 2 hours in front of the Presidential Palace.

ESG is -- OceanaGold is a top ESG performer, as measured by the major ESG rating agencies. Although we are proud of our ESG performance and the programs we have, we can always do better. Driving for improvements in health and safety continue to be a critical component of our business, and our total recordable injury frequency rate improved in 2019.

Moving on to Slide 5 and the discussion about Didipio. The resumption of Didipio operations is our top priority. The FTAA renewal process currently sits with the Office of the President after it was re-endorsed by the Department of Environment and Natural Resources and the Mines and Geosciences Bureau before Christmas. We have -- we've had good engagement with national and local stakeholders and support from different levels of government and the regulatory agencies. We have retained our workforce to continue in a state of operational readiness so we can ramp up following the resolution. We've maintained this state of operational readiness since we were forced to suspend operations in October 2019. However, we cannot continue this state indefinitely. If operations are permitted to recommence, then we believe we can achieve full rates of production of 10,000 ounces of gold plus 1,000 tonnes of copper per month within 4 to 6 weeks. If operational permits are not received and we decide to proceed to full care and maintenance, significant workforce reductions would result, and we may be looking at a ramp-up to full production of up to 12 months.

Moving on to Slide 6 and the voice of our host communities. As I mentioned before, we are thankful for the overwhelming support from our host communities. Since July 2019, thousands of people have come together to organize rallies to support the renewal and the lifting of the barricade imposed by the provincial governor. Community groups have held dozens of meetings with government officials and submitted dozens of letters of support to the Office of the President. The communities want this mine, and they want Oceana's goldwork to continue. The Didipio mine has changed the lives of tens of thousands of people for the better. Families across the provinces of Nueva Vizcaya and Quirino have jobs, access to improved health services, better schools, better roads, access to markets and a better quality of life because of the Didipio mine. We are proud of the work that we have done at Didipio and the partnerships we have built with communities.

I'll now turn it over to Scott to discuss the financial results.

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

Scott A. McQueen, OceanaGold Corporation - Executive VP & CFO [4]

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

Thank you, Mick, and hello, everyone. Over the next few slides, we'll cover some key aspects of our fourth quarter and full year 2019 financial performance.

Moving on to Slide 8. Revenue in the fourth quarter increased approximately 14% from the previous quarter due to higher gold sales from Macraes and Haile, which both demonstrated strong fourth quarter operational performance. Full year revenue of $651 million was lower than the previous year due to the absence of sales from Didipio in the second half of the year. This was partially offset by a higher average gold price received. EBITDA increased 33% quarter-on-quarter on our higher revenues, which was partially offset by higher corporate, general and administrative costs. Note that the costs incurred to maintain Didipio in its state of operational readiness were included as part of corporate D&A costs in the fourth quarter. These totaled approximately $10 million. In addition to this, we had $3.4 million noncash write-off related to prior production taxes in the Philippines. As a result, these 2 factors combined to reduce earnings in the quarter by around $0.02 a share. Lower year-on-year EBITDA reflects the second half suspension of Didipio and the associated cost to maintain the asset in its state of operational readiness. Net profit for the quarter was $8.7 million, while adjusted net profit was a negative $0.7 million after adjusting for unrealized gains on hedges of $13.4 million and a write-off of approximately $4 million of exploration costs which are associated with joint venture agreements terminated during the quarter. Net profit for the year was $14.5 million, and the adjusted net profit for the full year was $32.1 million.

Moving to the cash flow summary on Slide 9. Operating cash flows increased quarter-on-quarter to $46.7 million or $0.07 per share with a minimal change in net working capital. The increase in operating cash flow was a result of higher gold sales in Haile, which is partially offset by the absence of sales from Didipio and the associated carrying costs already noted. Investing and financing cash flows were similar quarter-on-quarter. For the full year, the increase in investing cash flow largely reflected higher exploration costs related to the growth projects in the Waihi district. The year-on-year decrease in financing cash flow relates mainly to the discretionary debt repayment we made in 2018.

Turning to Slide 10, which included some additional detail on our capital expenditure. Full year capital expenditure in 2019 was at the bottom end of the guidance range of $240 million. Our investments in growth capital were mainly at Waihi with the start-up development of Martha and, of course, the continuation of the Haile expansion. Our investment in exploration in the Waihi district continues to yield strong results as resource growth just announced the Martha Underground last week.

Moving on to Slide 11 and a snapshot of our balance sheet. As at the end of 2019, our cash balance was $49 million, giving us immediately available liquidity of $99 million. Our net debt increased slightly to $179 million, primarily as a result of the increase of approximately $32 million in equipment leases mainly at Haile, where the new fleet is being progressively added in support of the expansion and increased production rates.

Our balance sheet continues to have relatively low financial leverage with a net debt-to-EBITDA ratio of 0.84. With the support of our long-term bank group, as previously announced, we agreed amendments to our corporate debt facilities in November, whereby we eliminated the [$50 million] step-down and extended the maturity until the end of 2021.

Before moving to the outlook for 2020, I'll spend a minute just having a look at Slide 12, which shows how we've managed our balance sheet over the past several years. Balance sheet's always been managed in a prudent manner by not carrying excess levels of debt. Leverage has been increased as required to support growth and then reduced during production, in production mode and free cash flow allows. Clearly demonstrated by the graphs where leverage increased through peak construction in Didipio around 2012, and also Haile in 2017, both followed by periods of debt reduction. Most recently, you will recall our discretionary debt repayment of $50 million in late 2018.

The continued suspension of operations at Didipio is impacting cash flows. We are monitoring liquidity levels constantly and have options at our disposal to manage through the uncertainty and continue to support the growth projects in our business. The first step in that process was the amendment made to the debt facilities in late 2019. And we will continue to focus on executing prudent options as required to support the continued delivery of key growth projects, including the expansion of the Haile mine, the development of the underground at Martha, further drilling at WKP, and life of mine extension options at Macraes.

Moving to the outlook on Slide 14. As you know, we published our 2020 guidance a couple of weeks ago. On a consolidated basis, excluding Didipio at this stage, we expect to produce between 360,000 and 380,000 ounces of gold, which is similar to what we delivered in 2019 from those operations. All-in sustaining costs are expected to range between $1,075 and $1,125 per ounce sold. We expected to produce approximately 25% more gold this year, offsetting the decrease in production at Waihi where the mill will complete -- where we will complete mining of Correnso orebody this quarter before production from Martha Underground in Q2 2021.

Our capital investment program for 2020 includes an increase in pre-stripping and site mining infrastructure associated with the Haile expansion, where we are bringing forward 2 open pits originally planned for mining in 2021; capital associated with the development of the Martha Underground Project; and the road realignment and the movement of underground mine infrastructure at Macraes to allow for future production. The exploration spend is approximately half that of the previous year as we have narrowed our focus to support the current mine plans, Martha Underground and WKP.

I'll now turn it over to Michael to discuss in more detail the outlook for each operations.

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

Michael Harvy Lou Holmes, OceanaGold Corporation - Executive VP & COO [5]

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

Thank you, Scott, and hello, everyone. Moving on to Slide 15 and the overview of Haile for 2020. Haile ended 2019 on a high note with 26% higher production and lower costs relative to the third quarter. In fact, Haile improved every quarter through 2019 as productivity increased and unit costs decreased. We expect continued improvement this year through our larger mine program and utilization of the newly built and commissioned mining equipment. Haile is expected to produce between 180,000 and 190,000 ounces of gold, which represents an approximate 25% increase in gold production from 2019. The site all-in sustaining costs are expected to decrease and range between $1,080 and $1,130 per ounce sold, while mining unit costs are expected to decrease as the year progresses.

Haile's production profile is second half-weighted with 2/3 of the year's gold output expected in the second half of the year, which is driven by the mining schedule. Higher recoveries are expected in the second half, consistent with higher grades and embedded process plant improvements. We expect production to increase every quarter of the year, while the all-in sustaining costs are expected to decrease each quarter. Haile's all-in sustaining costs will be significantly higher in the first half of the year then significantly lower in the second half to average out to our guidance range.

Mining rates will increase significantly this year as we plan to mine between 50 million and 55 million tonnes of material, including 3.5 million to 4 million tonnes of ore. This compares to approximately 25 million tonnes of material mined in 2019. The mining activities will be supported by the new 15 -- new Komatsu 730E 200-tonne haulage trucks, of which we currently have 12 in service, and expect all 15 to be online by the end of the first quarter, which is ahead of schedule. In addition to these large trucks, we have upgraded the fleet with 1 Komatsu PC3000 excavator, 2 Komatsu PC4000 shovels and 4 new Sandvik DR140is drill rigs to support the increased mining rates.

The process plant continues to achieve higher throughput rates, and we expect mill -- to mill approximately 14% more ore this year than in 2019. We have recently completed the commissioning of the pre-aeration [pit now], which is the last new component of the upgraded regrind service. Our focus for the processing plant now turns to improving recoveries while continually increasing throughput.

As I mentioned, with the enhancement of the open pit operations, we are revisiting the Horseshoe underground mine plan. This study work is underway and will include further analysis of the appropriate backfill to use, which will dictate the mining sequence. We will also select the mining fleet that will support underground operations and use technology similar to what we implemented at Didipio. We expect to have more details on this work in the second half of the year. However, we will likely defer the start of the portal development to 2021. The capital -- the growth capital for 2020 does not include the Horseshoe underground development.

In the meantime, the permitting of the Horseshoe underground is progressing to plan, and we expect the notice of decision from the U.S. Army Corps of Engineers in the near term.

The growth capital investments at Haile are focused on the expansion of the mining operations, which includes a 3-meter lift of the carbon storage facility, which represents half of the growth capital investment for 2020. In addition to the TSF, we are investing in the construction of a large potential out of an acid-generating or PAG cell, which is currently under construction and cost approximately $20 million.

In addition to these investments, we are upgrading the water treatment plant, expanding our dewatering facilities, and relocating an overhead power line to allow for the mining of Mill Zone phase 2 which we brought forward by 6 months from the original mine plan.

Moving on to Slide 16 in Haile physicals. It has been a challenging start as we ramp up Haile. However, we are feeling much better with how the operation is progressing. We do still consider Haile to be a new mine with a long mine life. We have been ramping it up since it went into operations. Mining was challenged by the extreme weather events in quarter 4 2018 that took us by surprise, and the recovery was much longer than expected. The U.S. economy has been very strong, which has led to a labor shortage and impacted our employee turnover, which we have now stabilized. We have upskilled the workforce by recruiting from hard rock mining states as well. While all this has been going on, we have been expanding our mining operations in the process plant, which is now operating with throughput rates that are 50% higher than the nameplate capacity.

We have the right leadership and team in place to drive further improvements and advance the continued expansion of the operations.

As we mentioned, we expect continued improvement for the operation, particularly on the mining front, where productivity significantly improved over the course of year last year and expect to further improve as this year progresses. Unit costs are also expected to decrease as well.

Moving on to Slide 17 in Macraes. Macraes continues to be the mine that keeps on giving, with consistent positive performance and cash flows. There is plenty of opportunity to convert the large resource base to reserves and extend the mine life for a low capital investment. For 2020, Macraes is expected to produce a similar amount of gold to 2019 while costs are steady. We will mine a lot of material as we do normally at Macraes, however, we will do so with a lower strip ratio this year of approximately 7.5:1. The mining operations will be supported by another new 3,600 Hitachi excavator, which is now in service.

The Golden Point underground study is advancing well, and we expect to complete it in the second half of the year. Our objective with Golden Point underground is to replace the Frasers Underground. And this, together with additional open-pit opportunities, form the basis for a potential mine life extension. I would like to point out that the resource at Golden Point underground, the new discovery following initial drilling at other Round Hill project.

With drill results better than expected, we have decoupled the underground portion of the Golden Point from the Round Hill project. The Round Hill project remains as an option for us. However, this is an end-of-mine-life opportunity. In the meantime, we are advancing several open pits, including new stages of Coronation and Coronation North, [Graben] phase 1, [Deep Den] and Innes Mills. We will also be updating NI 43-101 for Macraes in 2020.

Moving on to Slide 18 in Waihi. We are transitioning from Correnso to the Martha Underground. We will complete mining of the main veins from Correnso this quarter and produce approximately 11,000 to 12,000 ounces of gold. We will continue mining the narrow veins from the underground, which we will stockpile ahead of batch processing in the fourth quarter. And in the fourth quarter, we expect to produce an additional 7,000 to 8,000 ounces of gold. The processing plant will be shut down after the first quarter, then restarted in the fourth quarter before we shut it down again ahead of processing from the Martha Underground in the second quarter of 2021.

The greatest capital at Waihi for 2020 is predominantly for the development of the Martha Underground. It also includes approximately $5 million to $7 million on project overheads, with the reminder on underground development, while some of the capital is for some plant modifications during the downtime. The Waihi District Study, which is expected in the second quarter of this year, will outline additional details associated with the development of the Martha Underground.

On to the next slide, Slide 19. The development of Martha Underground will ramp up over the course of the year from approximately 1,300 meters of development this quarter to a steady-state development rate of between 2,600 and 2,900 meters a quarter by the third quarter of this year. We have completed the ventilation shaft between the 2 drill drives and 830 meters of development in the fourth quarter of last year.

The image you see here on your screen is what Martha is expected to look like in 3 years' time. As I just mentioned, the Waihi District Study will have detailed information on the development of Martha Underground, the capital and operating costs as well as the ramp-up profile.

Just last week, we announced the updated Martha Underground resource. We reported an overall increase of 440,000 ounces of gold from the previous update in March 2019. We increased indicated resources by 150% year-on-year to 824,000 ounces of gold, while grades increased in both categories.

Back in 2017, we told the market that we had a new project called Martha, where through historical data and some drill holes, we believe there was mineralization that could underpin a certainty of mine life. Fast forward to today, we have a growing resource, a permitted mine, construction is well underway, and we are working towards initial targeted mine life of 10 years. Exploration drilling will continue in the Martha Underground this year and for many years to come. Our focus for this drilling is mainly on resource definition drilling. However, we have an exploration target of approximately 6 million to 8 million tonnes, grading [4 to 6] grams per tonne, which we will look to convert into resources over the coming years. This exploration target is incremental to the resources we currently have on our books.

Moving on to Slide 20 in the Waihi District Study. Waihi is more than just the Martha Underground, and the Waihi District Study is well underway. As we've said, the study should be completed in the second quarter of 2020. The main components of the study are the Martha Underground and our game-changing discovery at WKP. Both are underground mines and high-grade, particularly at WKP, where last March, we announced an initial resource of 234,000 ounces of gold in the indicated category and 401,000 ounces of gold in the inferred category. The Waihi District Study will be a preliminary economic assessment of PEA, and our intention is to highlight the future of the Waihi operation while providing high levels of detail on numerous parameters, such as production rates, operating and capital costs, mine plans and designs. The study will include resources beyond what we have just reported for the Martha Underground and WKP. However, there will be important [time to] draw a line in the sand, and what we have for resources from the district will be used for the [star performers]. This point is particularly important when we're considering the potential of the WKP targets. It's still a relatively new discovery with only 25,000 meters drilled since the end of 2017. We expect the resource at both the Martha Underground and WKP to grow, especially at WKP where we have only just begun. There is a lot more drilling to do, and we expect exploration to continue well into the mine lives of both Martha Underground and WKP.

I will now turn it over to Mick to wrap up the formal presentation. Thank you.

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

Michael Francis Wilkes, OceanaGold Corporation - President, CEO, MD & Director [6]

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Thanks, Scott -- Michael and Scott. As you've been hearing, we have several opportunities we are focused on to achieve our objective of being one of the best gold mining companies in the industry. Our top priority is to resume operations at Didipio. It is an important asset to our business, and we want to deliver on our commitments to our host communities. We will continue to work constructively with all stakeholders towards the renewal of the FTAA and reestablish the best operation in the Philippines.

We have several exciting opportunities that are designed to deliver significant long-term value to shareholders. The Waihi district represents the largest value-creating opportunities we have in our portfolio. The market ascribes very little value to the Waihi operation despite successful resource expansion and project development that now underpin the 10-year mine life. And as the Waihi resource continues to grow, we see more value being ascribed to this asset in this Tier 1 jurisdiction, especially at WKP, which is an exciting opportunity and one that we're advancing as quickly as we can.

The Haile expansion continues to advance well, and we are feeling good about the progress of the operation, which is shaping up to be a robust, long-life mine again in a Tier 1 jurisdiction. Golden Point is another exciting opportunity, which is currently being studied and could add further mine life to our Macraes operation.

I'll now turn it over to Sam.

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Sam Pazuki, OceanaGold Corporation - VP of IR [7]

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

Thank you, Mick. That concludes the formal presentation segment of the webcast. We will now take some questions over the phone, but you can also ask questions or post questions in the webcast site on your computer screen.

I will now turn it over to the moderator to facilitate the Q&A session.

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Questions and Answers

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

Operator [1]

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

(Operator Instructions) And your first question will be from Reg Spencer at Canaccord.

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Reg Spencer, Canaccord Genuity Corp., Research Division - Mining Analyst [2]

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Just a question on Didipio. Like the language around maintaining that state of operational readiness seems to have softened a little bit such that it appears that you may be considering care and maintenance. At what point, given that you're spending $2 million a quarter at the minute -- sorry, $10 million a quarter at the minute, at what point do you make that decision on care and maintenance?

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Michael Francis Wilkes, OceanaGold Corporation - President, CEO, MD & Director [3]

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Well, Reg, thanks for the question. We are focused on the restart of operations at Didipio, and the completion of the renewal remains our primary objective. We have made progress on the renewal in the quarter, which included continued positive engagement with regulators, and ultimately, the MGB and DENR, endorsing the renewal back to the OP, the Office of the President, in December. We've also seen positive steps with regard to the local blockade, including the authorization by the Office of the President to allow fuel into the site, and the Department of Interior Local Government orders, which have been issued to the Nueva Vizcaya government regarding the removal of the checkpoint. So we're encouraged by those various things.

However, time is marching on, and we are concerned about the cost that we're incurring to maintain the mine in a state of operational readiness, which we can't do forever. We can't do that indefinitely. And the impact of our -- on our workforce and the host communities is a key consideration in that decision.

So that's pretty much where we're at. We'll take into consideration all of those things. As was said in the presentation, if we do go into care and maintenance there is a significant a period to ramp-up, get back into operation, so we don't take any of these decisions lightly.

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Reg Spencer, Canaccord Genuity Corp., Research Division - Mining Analyst [4]

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And just the comment about getting some fuel into sites, you've obviously got approvals and permission to do that. Would that include, or may that include the ability to truck out some of the concentrates you have there just to get a little bit of cash flow out of the asset? Is that being contemplated?

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Michael Francis Wilkes, OceanaGold Corporation - President, CEO, MD & Director [5]

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We've obviously requested for that from the government. And we -- those requests is sitting with the DENR, and we're continuing those discussions. The letter from the Office of the President authorizing the fuel to leave site was specifically related to the fuel.

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

Operator [6]

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

Next question will be from Chris Thompson at PI Financial.

More:
Edited Transcript of OGC.AX earnings conference call or presentation 20-Feb-20 9:30pm GMT - Yahoo Finance

Recommendation and review posted by Bethany Smith

CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells

T cells and B cells are 2 primary cell types in our adaptive immune system, the source of immunological memory protecting us from subsequent pathogen exposure. B cells secrete pathogen-specific antibodies, which neutralize pathogens directly, or tag them for attack by other immune cells. T cells destroy pathogenic cells directly as well as secrete cytokines to attract additional immune cells.

Chimeric antigen receptor (CAR) T cells are patient derived T cells genetically manipulated to express an artificial transmembrane receptor. The artificial receptor is engineered from modular parts to bind to a surface protein (also called an antigen) on malignant cells and activate the T cell via engineered T cell signaling switches on the CAR.

Current FDA-approved CAR-T cell therapies express CARs recognizing CD19, which is expressed on the surface of almost all B cells, making these therapies specific for B-cell malignancies. Following binding with a CD19-expressing cell, the CAR T cell is activated to proliferate, eliminate the CD19-expressing cell, and persist within the patient.

Tisagenlecleucel (Kymriah, Novartis) is approved to treat pediatric and young adult patients (up to age 25) with relapsed or refractory (R/R) B-cell acute lymphoblastic leukemia (R/R ALL, ELIANA trial) and adult patients with R/R diffuse large B-cell lymphoma (R/R DLBCL, JULIET trial) after 2 or more lines of systemic therapy. Axicabtagene ciloleucel (Yescarta, Kite Pharma) is approved for adult patients with R/R large B-cell lymphoma (R/R DLBCL, ZUMA-1 trial, NCT02348216) after 2 or more lines of systemic therapy. There are approximately 700 new cases of pediatric and young adult R/R ALL annually in the United States. New cases of R/R DLBCL are approximately 7000 annually in the United States.

CARs can be engineered to recognize virtually any cell surface antigen and can be expressed in a variety of immune cells, suggesting that product development will result in many modular CAR units with vast application versatility. Many different antigen and cell type combinations are already currently in development to address several cancers, such as CAR T cells for pancreatic cancer (NCT03323944).

The first step in creating these personalized genemodified cell therapies is collecting patient lymphocytes via leukapheresis at a clinic or infusion center. Lymphocytes include T cells, B cells, and natural killer cells. The leukapheresis process lasts up to 4 hours and must be coordinated with the patients continuing care regimen to ensure sufficient T cells. The lymphocytes are cryopreserved and immediately shipped to a centralized manufacturing facility.

T cells are separated from the other cells in the leukapheresis product and genetically manipulated, typically using a lentiviral gene delivery method to carry DNA encoding the CAR protein, resulting in CAR T cells. The CAR T cells are cultured to a patient-specific appropriate dose. As this process is finishing, the manufacturer coordinates with the patients health care team to ensure the patient and team are prepared to infuse the CAR T cells. The manufacturing process from the apheresis process to the clinical CAR-T cell product varies widely from patient to patient, from 14 days up to a few months. The limiting step is typically reaching the appropriate CAR-T cell dose.

About a week before the scheduled CAR-T cell infusion, the patient receives multiple days of low-dose conditioning chemotherapy. This step serves to deplete lymphocytes before administration of the CAR T cells, improving the efficacy and persistence of therapy. The CAR T cells are then administered intravenously, and the patient is monitored for adverse events (AEs). The most common AEs with both currently approved products include cytokine release syndrome (CRS), neurological toxicity (NT), hypersensitivity reactions, serious infection, prolonged cytopenias, and hypogammaglobulinemia.3,4 Both products caution that therapy could cause hepatitis B viral reactivation.

The most severe reactions are CRS and NT, both of which can be life threatening. CRS, a common immune reaction following infusion of monoclonal antibodies and CAR T cells, is characterized by fever, nausea, chills, hypotension, tachycardia, asthenia, headache, rash, and dyspnea.5 Mild cases are easily managed, whereas severe cases require more aggressive and invasive therapy, such as mechanical ventilation and intravenous administration of tocilizumab. NT associated with CAR-T cell therapy is characterized by encephalopathy, headache, aphasia, delirium, insomnia, anxiety, tremor, dizziness, seizures, and peripheral neuropathy.

During the clinical trials of Kymriah and Yescarta, CRS and NT occurred in most patients with more than 10% experiencing severe CRS and more than 20% with severe NT.3,4 Initially, the 2 AEs appeared to be independent, but data are beginning to emerge suggesting a correlation. CRS might be a predictor of neurological events; however, neurological events do not predict CRS.6

Real-world evidence from patients treated with Kymriah, presented at the 2019 Society of Hematologic Oncology annual meeting, reported that slightly more than half experienced either of these conditions and less than 20% had severe cases.7 Two-year followup data regarding Yescarta reported severe CRS cases in 11% of patients and severe NT cases in 32%.8 Due to the high rate of occurrence and severity of CRS and NT, both Yescarta and Kymriah have restricted availability through Risk Evaluation and Mitigation Strategies.3,4 Both medications must be administered at a Risk Evaluation and Mitigation Strategiescertified health care facility with health care providers trained in the management and treatment of CRS and 2 doses of tocilizumab available for each patient before CART cell infusion.

Regardless of the potential for severe adverse events (AEs), the benefit of both Yescarta and Kymriah outweigh the risks, as they are highly effective singleadministration therapies. Despite the aggressive nature of the cancers treated with CAR T-cell therapy, meaningful clinical benefit can be achieved within 1 month. A summary of clinical trial primary response rates as well as 2-year data and published real-world data can be found in Table.

Despite differences between the 2 clinically available products, their safety and efficacy profiles in patients with R/R DLBCL are comparable. Differences between the therapies range from the molecular units comprising the CARs, manufacturing differences, lymphodepletion regimen, number of CAR T cells and volume infused, and whether the infusion and short-term patient monitoring occurs in an inpatient or outpatient setting.

Patient-specific genetically modified cell therapies can present many manufacturing challenges. One stark difference between the available therapies, relevant to the patient and clinician experience, has been the manufacturing time and failure rate. During ZUMA-1, of the 101 patients treated with Yescarta, the median time from leukapheresis to product delivery was 17 days (range, 14-51 days), and a 1% manufacture failure rate was reported.4 The ELIANA trial of Kymriah in patients with R/R ALL reported a 9% manufacture failure rate, whereas the JULIET R/R DLBCL trial reported a failure rate of 6.9%, and of the 106 patients receiving Kymriah, the median manufacture time was 113 days (range, 47-196 days).3

Commercial manufacture of Kymriah for DLBCL has struggled to meet specifications.11 While addressing the production issue, Novartis has initiated a safety study evaluating out-of-specification product (NCT04094311) and a managed-access program (NCT03601442).

Known causes of CAR-T cell therapy failure are T cell exhaustion and antigen escape. T cell exhaustion is characterized by a loss of responsive T cells due to changes in gene expression and can be prevented by immune checkpoint inhibitors PD-1, PD-L1, or CTLA- 4. Antigen escape describes a condition in which some cancer cells do not express the CAR-targeted antigen; therefore, they escape immune activation and survive within the patient. Engineering a secondary CAR to a different antigen, such as CD22 in the case of ALL, increases the likelihood of targeting all malignant cells. Solutions to both of these inhibitory mechanisms are currently under clinical trial investigation.12,13

The therapeutic success of CAR T cells ensures gene-modified immune cell therapy will be refined, optimized, and broadly applied until limits are reached. Many clinical groups are investigating biomarkers associated with severe AEs to provide an additional layer of precision care to the CAR-T cell therapy model.6,14 In addition, clinical trials are underway evaluating combination therapies to enhance the efficacy and improve the safety of CART cell therapy. Early-stage research is evaluating the possibility of off-the-shelf CAR-T cell therapy, not a patient-unique manufactured product, to reduce the time to treatment and achieve manufacturing efficiencies and consistencies.15,16 Gene-modified cell therapy, such as CAR T-cell therapy, is revolutionizing oncology, and this living drug model is breathing life into the hopes of patients with cancer and caregivers.

Originally posted here:
CAR T-Cell Therapy: Genetically Programming the Immune System to Attack Malignant Cells - Pharmacy Times

Recommendation and review posted by Bethany Smith

(KSDK) This holiday season was a hot one for at-home genome testing.

Providers like 23andMe and Ancestry.com promoted their heritage and health testing services as a gift for families who want to learn about their genetic makeup.

It might look like the gift that keeps on giving, with new relatives and health findings popping up all the time, but warn that customers may be giving away something theyll never get back.

The history of your DNA doesnt end when you send it in. It might just be the start of the journey your data can take from testing provider to healthcare companies or the police.

While direct-to-consumer genetic testing providers generally have privacy policies to tell customers how their data will be stored and shared, genetic code is sensitive information on a whole new level.

It can connect you to your relatives, predict your risk of disease and even help identify suspects in criminal investigations all over your family tree.

Read more:http://bit.ly/39Z45wZ

See the original post here:
Your DNA test could end up in the hands of healthcare companies or police - WREX-TV

Recommendation and review posted by Bethany Smith

For Immediate Release: February 20, 2020 Statement From:

Statement Author

Leadership Role

Director - CDRH Offices: Office of the Center Director

Leadership Role

Director - Center for Drug Evaluation and Research

Pharmacogenetic testing is a type of genetic test that in some instances can predict how a person will respond to specific medications. Thus, this type of testing offers promise for informing the selection or dosing of some medications for certain individuals. When there is sufficient scientific evidence demonstrating a relationship between how a persons genes may impact their metabolism of a drug or how they may respond to the drug, this information can be useful for health care providers. However, for many medications, there is not sufficient scientific evidence to support using pharmacogenetic test results to inform prescribing decisions.

In a 2018 safety communication, we warned the public about the FDAs concerns with firms offering genetic tests making claims about how to use the genetic test results to manage medication treatment that are not supported by recommendations in the FDA-approved drug labeling or other scientific evidence. Clinicians order tests to help them and their patients make better informed decisions. Such decisions, however, should be based on solid and reliable science. Decisions based on inaccurate information can result in patient harm because patients may not receive the most appropriate medication, may receive a medication that could be harmful or may receive a prescription for an inappropriate dose. All of these scenarios can create unnecessary delays and prevent patients from receiving the most timely and appropriate treatment. Patients and clinicians deserve better. Unfortunately, in the time since our safety communication was issued, some manufacturers of pharmacogenetic tests with claims not adequately supported by sound science have continued marketing their tests, including some for medications to treat seizures, mental illness and pain, including opioids. The FDA remains concerned with the safe use of these medications based on pharmacogenetic test reports that are not supported by sound science.

Many pharmacogenetic tests are being offered as laboratory developed tests (LDTs). For many years, FDA has, in an exercise of enforcement discretion, generally not enforced applicable regulatory requirements for LDTs, such as premarket review. The FDA developed its existing approach to LDT enforcement decades ago, when the field of LDTs was much different, and often relied on simpler technologies in local settings. While the landscape of LDTs has changed, the agencys oversight framework has remained the same. The FDA is committed to continuing to work with Congress on a broader legislative solution to the oversight of in vitro clinical tests generally (including LDTs), which would modernize our regulation of these tests. In the meantime, the FDA should not and cannot stand idly by when safety issues arise. Consistent with our mission to protect and promote public health, we believe it is important to take steps now to help ensure that claims being made for pharmacogenetic tests offered today are grounded in sound science to avoid inappropriate management of patients medicationsand to do so through approaches that both protect patients and advance the development of analytically and clinically validated pharmacogenetic tests. Indeed, genetic testing can enhance patient management by improving the selection of medications or the dosage of a medication, tailoring the therapy to the patientbut only when it is based on sound science.

Thats why today we are introducing a collaboration between the FDAs Center for Devices and Radiological Health and Center for Drug Evaluation and Research intended to provide the agencys view of the state of the current science in pharmacogenetics. Our new web-based resource includes a table that describes some of the gene-drug interactions for which we believe there is sufficient scientific evidence to support the described associations between certain genetic variants, or genetic variant-inferred phenotypes, and altered drug metabolism, and in certain cases, differential therapeutic effects, including differences in risks of adverse events.

The table posted today includes certain established gene-drug interactions that appear in FDA-approved drug labeling. Recognizing, however, that not all supported gene-drug interactions may be found in current FDA labeling, the table also includes some additional gene-drug interactions that are consistent with the current FDA labeling and for which there is sufficient scientific evidence based on published literature. This literature-based scientific evidence is often used in support of the recommendations found in professional guidelines used by clinicians.

The table is not complete, but we felt it is important to provide clinicians, patients and test developers with information now while we continue to review the scientific evidence, including the scientific evidence underlying various professional guidelines, such as those of the Clinical Pharmacogenetics Implementation Consortium. We anticipate updating this web-based resource periodically as the evidence evolves. Further, weve opened a docket for public comment and encourage all stakeholders to provide their feedback on specific pharmacogenetic associations that should or should not be included as the agency continues to update this table. Feedback should include supporting rationale and underlying evidence that supports the pharmacogenetic association. These data will be evaluated as we consider updates to the table.

We are also exploring additional approaches for evaluating the evidence underlying gene-drug associations, such as participation in a community-based collaborative approach to the ongoing evaluation of the evolving science, as part of our commitment to continue engaging with health care professional, guideline setting, patient and test developer groups.

We will continue to communicate about the promise and potential of pharmacogenetic testing, and about when we have concerns regarding claims that are not supported by sufficient scientific evidence. We are committed to engaging with stakeholders as we work to protect patients while fostering the development of new and innovative diagnostics.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

###

Here is the original post:
FDA Announces Collaborative Review of Scientific Evidence to Support Associations Between Genetic Information and Specific Medications - FDA.gov

Recommendation and review posted by Bethany Smith

Companies such as 23andMe have proliferated over the past decade, feeding peoples hunger to know who and where they come from, and what diseases their genes might predispose them to. Over that time, it has gradually become clear that the main source of revenue for at least some of these companies comes from selling the data on to third parties.

DTC companies are far from the only ones collecting sensitive data about you. National health systems, health insurers and, increasingly, social media providers are too. Its already being used in research designed to improve our health and wellbeing, and there is a legitimate question to be asked about compensation. 23andMe, for example, asks its customers to waive all claims to a share of the profits arising from such research. But given those profits could be substantial asevidencedby the interest of big pharma shouldnt the company be paying us for our data, rather than charging us to be tested?

These are the privacy concerns that may be behind layoffs, not only at 23andMe, but also atother DTC companies, and that we need to resolve urgently to avoid the pitfalls of genetic testing while realising its undoubted promise.

Read the original post

Read more:
DNA testing companies are making money off your genetic data. Should they be paying you? - Genetic Literacy Project

Recommendation and review posted by Bethany Smith

What happened

Shares of Invitae (NYSE:NVTA) fell as much as 15.5% today after the company reported fourth-quarter and full-year 2019 operating results. The genetic testing company delivered impressive growth in 2019 compared to the prior year, including year-over-year increases of 47% in revenue and 59% in test volumes.

Despite the solid results, Wall Street analysts were expecting $68.1 million in fourth-quarter 2019 revenue. Invitae reported "only" $66.3 million. While the difference was insignificant in the grand scheme of things, it doesn't take much to derail a volatile growth stock.

As of 1:49 p.m. EST, shares had settled to a 11.2% loss.

Image source: Getty Images.

On the one hand, the harsh reaction to the miss on fourth-quarter revenue is certainly an overreaction. Invitae performed well in 2019 and made important strides to expand its business. It launched new tests, formed new partnerships, and joined major health insurance networks.

On the other hand, the genetic testing company reported an operating loss of $244 million in 2019. Investors have accepted steep losses as the price of owning a high-growth company, but the losses are unsustainable.

Metric

2019

2018

Change

Test volume

482,000

303,000

59%

Revenue

$216.7 million

$147.7 million

47%

Gross profit

$98.7 million

$67.6 million

46%

Operating income

($244.1 million)

($122.5 million)

N/A

Cash flow from operating activities

($145.0 million)

($92.2 million)

N/A

Data source: Company press release.

Investors can only hope that a high rate of growth eventually translates into operating profits. For 2020, Invitae expects to achieve more than $330 million in revenue and process at least 725,000 tests, representing year-over-year increases of over 50% for each metric. That should help to create a path to profitability, assuming operating expenses can be held in check. But for the foreseeable future, investors should expect the stock to remain volatile.

Read the rest here:
Here's Why Invitae Stock Is Tumbling Today - Motley Fool

Recommendation and review posted by Bethany Smith

VALDOSTA, Ga. On an October morning in 2018, Eleanor Holmes and her husband left home to run an errand and found two men inside their front gate. They introduced themselves as detectives from Orlando, Florida, and said they needed the couples help.

Standing in the driveway, the casually dressed detectives said they were trying to identify someone whod been found dead many years earlier, the Holmeses recalled. They were looking for the persons relatives, and were using DNA and genealogical records to stitch together a family tree that they hoped would lead them to a name. Friendly and businesslike, they said theyd already got DNA samples from Eleanor Holmes sister and an aunt. And now they wanted hers.

Holmes already knew about the detectives visit to her sister. It worried her that someone in her family had died without anyone knowing about it. She had relatives in Orlando, including a niece whom she hadnt heard from in more than a decade. So she agreed.

I just did it because that was the only thing on my mind, my niece. That was it, bottom line, Holmes said in a recent interview.

The detectives, still standing in the driveway, swabbed Holmes cheek and put the sample in a container. They thanked her, gave her a business card and drove away.

She thought nothing of it until a few days later, when she got a frantic phone call from the girlfriend of one of her sons, Benjamin Holmes Jr. Orlando police had just arrested him for allegedly fatally shooting a college student, Christine Franke, in her Florida home in 2001. Theyd used DNA and genealogical records to tie him to the crime.

In that panicked moment, it dawned on Holmes that the detectives hadnt told her the truth. Theyd used her DNA to help build a case against her son.

When they arrested him, I knew they were lying, Holmes said. They lied to us.

Police have said that the arrest of Benjamin Holmes Jr., 39, shows their commitment to do everything we can to solve crimes. Frankes family says the arrest has given them long-needed answers about her death and allowed them to stop wondering if the killer was still out there, free to prey on others.

Benjamin Holmes Jr. and his parents, though, say he is innocent. He has pleaded not guilty, and his trial, scheduled for June, may be the first to explore how police conduct investigations using genetic genealogy, a largely unregulated technology that has exploded in popularity in recent years.

Holmes and her husband, who are both in their mid-70s, arent the only ones in their family who feel misled by police. In the months before taking her DNA, Orlando detectives visited more than a dozen of her relatives in Florida and Georgia. Several said they were told a similar story before agreeing to provide DNA samples.

It was just deception, not only to me but all my other family members, because they know what they were looking for when they took the DNA, Holmes said. They weren't looking for someone in our family that had been killed, or that was dead. They were looking totally to find out whether or not our DNA coincided with Benjamin's. That's what they were looking for.

For 17 years, Orlando police detectives had tried to figure out who killed Franke. Although the case had gone cold, each did what they could with the available technology and manpower. But every lead, every potential clue found at the scene, left them, and Frankes family, without answers.

I thought theyd never catch him, Frankes mother, Tina, 70, said.

Franke, 25, was one of four siblings raised in Vero Beach, 100 miles outside Orlando. An aspiring elementary school teacher, she was studying at the University of Central Florida while working as a server at a restaurant near the Universal Orlando theme parks. She lived with her girlfriend about a half hour away, on the north side of town.

Early in the morning of Oct. 21, 2001, after working a double shift, Franke returned home to an empty apartment; her girlfriend was out of town. Later that day, after the girlfriend was unable to reach her, she called a neighbor, who found Franke dead just inside the apartment door.

Shed been shot once in the head, and her wallet, containing no cash, had been discarded on the floor, according to court documents. Her clothing had been partially removed, and investigators found semen on her body. Police surmised that she had resisted the killers attempts to rob and rape her.

Police took a sample of the semen and submitted it to the state crime lab, which developed a profile and entered it into a national criminal database. There was no match. They took DNA from dozens of people potential suspects, as well as friends, relatives, co-workers, neighbors, acquaintances and witnesses and compared their profiles to the DNA found at the scene. Again, no hits.

They tried other forensic methods lifting fingerprints from the apartment, entering a shell casing into a national firearms database and found nothing. Years passed with no progress.

That changed in April 2018, when California authorities announced that theyd used a groundbreaking technique to identify a man they said was the Golden State Killer, a serial rapist and murderer whod terrorized the state in the 1970s and the 1980s. Law enforcement officials said theyd solved the case by entering crime-scene DNA into an online database called GEDmatch, where people shared profiles purchased from direct-to-consumer genetic testing companies such as Ancestry.com and 23andMe.

Orlando Detective Michael Fields, who inherited the Franke case from a retired colleague in 2012, decided to try the same tactic. He reached out to a Virginia company, Parabon NanoLabs, which had just started helping law enforcement identify unknown suspects by using genealogy websites to find their relatives and build family trees. The researchers, led by Parabons top genealogist, CeCe Moore, found two cousins of the suspected killer in GEDmatch and traced their common ancestors to a husband and wife who lived in Valdosta in the first half of the 1900s.

The Valdosta couple had an extremely large family, producing a sprawling family tree. Navigating that thicket left Fields and the researchers at dead ends, unable to go further without getting DNA from more people in the family.

Asking innocent people to voluntarily provide their DNA known as target testing is an unseen but essential, and thorny, component of investigative genetic genealogy. While police are seeking straightforward information about family ties, the process can also reveal secrets, including out-of-wedlock births and adoptions, ethics and privacy experts say. Subjects may not fully understand how their DNA profiles will be used.

While American courts have ruled that police are allowed to mislead people to obtain evidence, theres a debate within law enforcement over how honest police should be in seeking DNA from people who arent suspected of a crime.

Investigator Matt Denlinger works cold cases for the Cedar Rapids Police Department in Iowa. He used target testing to help solve the 1979 murder of a teenage girl. He says the truth, without including many details, usually works.

Let our news meet your inbox. The news and stories that matters, delivered weekday mornings.

You just go up and tell them what youre doing. No sleight of hand, he said. Most people are happy to help. They know theyre not involved. People get excited to help solve a mystery, if you phrase it that way.

Jennifer Spears, a cold case investigator with the Seminole County Sheriffs Office in Florida, said she rarely gets any hesitation from the people she approaches. I tell them their DNA is only being used in this case to help us determine where we need to be on the family tree, she said. Are we on the right branch, or are we way off?

The Department of Justice requires informed consent from nonsuspects before collecting DNA for a genetic genealogy investigation, according to an interim policy published last year. If a law enforcement agency decides that getting such consent would compromise the integrity of the investigation, then investigators may obtain the sample covertly, but must first get approval from a judge.

The policy covers cases involving federal authorities, so it does not apply to the Franke case, which was handled by local police. It also leaves unaddressed the use of a false story to persuade someone to provide a DNA sample. The lack of uniform rules opens the process to ethical risks, said Christi Guerrini, an assistant professor in the Center for Medical Ethics and Health Policy at the Baylor College of Medicine.

Unless circumstances compel us otherwise, we generally want people to be informed as to why theyre being asked to provide DNA to help with the investigation, she said, adding that she didnt know enough about Orlando polices actions to evaluate their handling of the Franke case.

The Orlando Police Department and the State Attorneys Office prosecuting the Franke case declined to comment.

Fields also declined to talk about his work on the case, but in interviews last year, he shared his general approach to asking people for their DNA. He said he has done it about 40 times, either as the lead investigator on a case, or assisting other police departments.

Fields said his approach depended on the person: how closely related they appeared to be to the unidentified suspect, whether theyd worked in law enforcement, whether theyd done any research on their own family trees.

He said he typically told his subjects the truth, without getting too specific: that there was a murderer or rapist in their family and he needed their DNA to figure out who it was. Other times, Fields said, he used a ruse. He declined to say what it was, or why he used it. But its effective, he said.

Holmes was raised in Valdosta, a small city near the Georgia-Florida border, but met her husband at a restaurant in Orlando, more than 200 miles away. They married and raised six children there, including Benjamin Holmes Jr., who they said was athletic, outgoing, popular and a junior deacon at his church. He didnt give his parents too much trouble beyond staying out late, they said.

After their children were grown, Holmes, a former nurses aide, moved back to Valdosta with her husband, a retired chef. They live a quiet life there in a single-story home behind a screen of pine trees and a chain-link gate. The couple are intensely private. The visit from the detectives in October 2018 upended that.

Fields and his partner, Detective Michael Moreschi, arrived without any advance notice. It quickly became clear that they already knew a lot about the Holmes family.

They knew my father, they knew my children, they could name every one of my children, where they lived. Everything that they wanted, they had it right there, Holmes recalled. Except for my DNA.

At that point, Fields and his colleagues had collected more than a dozen voluntary DNA samples that had narrowed the search. They believed the killer was one of Holmes two sons, according to an affidavit Fields would later file in court.

Among those whod already given their DNA was Alvin Davis, who said the detectives showed up at his Valdosta home and told him they were trying to identify a woman whod died in Orlando. They thought she was related to him, and needed his DNA to help figure out who she was.

Davis said he wasnt worried; he liked the police and wanted to help. I got nothing to hide, Davis, 63, recalled telling them. They swabbed his cheek and left.

The detectives told Cynthia Young, who lives in Miami, a similar story when they came to her door for her DNA.

Young, 63, a retired corrections officer, said she agreed because she understood how DNA could help a police investigation.

I didnt have a problem with it, she said. I see the good of using DNA.

Those DNA submissions helped pave a genetic trail that led police to Holmes.

Holmes said she isnt a very trusting person. But the detectives put her at ease. Before submitting to the swab, she said she joked with the detectives that they might find out that she was related to them.

I didn't really think it over, she recalled.

The detectives then asked her husband for his DNA. He declined and walked away.

For me to give my DNA to you, you have to come with some kind of papers from lawyers or something, Benjamin Holmes Sr. recalled. Just going to walk in out of the blue and say, I want to take your DNA, could you give me a sample? No.

Immediately after collecting Holmes DNA, police sent it to the Florida state crime lab, which determined that the suspect was one of her two sons, Reginal Holmes and Benjamin Holmes Jr., who both lived in Orlando, according to the Fields affidavit.

Investigators first focused on Reginal Holmes, following him to work as he installed air conditioning units at a construction site. An undercover officer approached him, got into a conversation with him, and offered him a bottle of Gatorade. Reginal Holmes took it and drove away, with the undercover officers tailing him, according to Fields affidavit. When Reginal Holmes threw the bottle into a dumpster, a detective retrieved it and took it to the state crime lab, which obtained a DNA profile and compared it to the crime scene DNA. There was no match, making Benjamin Holmes Jr. the prime suspect.

He was a Wendys restaurant manager with a record of arrests dating to 2001, mostly low-level drug charges and probation violations, as well as a domestic violence charge, according to Fields affidavit. Officers put him under surveillance. On the second day of following him, officers watched him step outside a friends house with a cigar and a beer, then throw them out. An undercover detective retrieved both, and police sent them to the crime lab, which found a match with the DNA from the crime scene.

Based on that link, and a follow-up sample of DNA Benjamin Holmes Jr. provided under court order, police arrested him on Nov. 2, 2018, charging him with shooting and robbing Franke. They did not offer a motive or a connection between the two.

Benjamin Holmes Jr. denied killing Franke, or ever knowing or meeting her. He has no idea how his DNA ended up at that place, his lawyer, Jerry Girley, said.

Girley said he was exploring ways to prevent the DNA evidence from being used at trial. He noted that the Franke case was among several in which DNA samples at state crime labs were found to have been contaminated. Lab officials have said that the samples have since been reanalyzed under sanitary conditions, providing profiles that can be used in court.

Girley has also discussed the case with the American Civil Liberties Union, which is tracking it as part of a broader plan to challenge the warrantless collection of DNA from property abandoned by potential suspects.

Girley acknowledged that police didnt break any laws when they used a ruse to obtain DNA from Holmes. But, he said, the tactic ought to be restricted.

There should be further evolution of the law to come abreast with the evolution of technology, Girley said.

After hearing about Benjamin Holmes Jr.s arrest, the relatives who gave their DNA to supposedly help identify a dead person realized the truth.

Young figured it out after seeing news coverage, which focused on the use of genetic genealogy.

It bothered me because they came to my house and they lied, she said.

Young, who knows Holmes but not her son, said that shes not sure she would have given her DNA if shed known how it would be used.

Had they been honest, I would have made a decision whether to give them my DNA or not, and if I chose not to, they could have gotten it by other means, Young said. Im OK with them getting it through other means. But to come to me and just lie to get what they want?

Davis said he would have given his DNA if the detectives said they were investigating a murderer in his family. He does not know Benjamin Holmes Jr. but has met Holmes. I just regret that they tricked me to get it, he said.

Holmes and her husband said that hearing what their son was accused of sent them into a long period of grief and anger that left them little time to ponder the detectives story.

I was so hurt, I don't think I could have hurt any more if he was dead, if he had been killed, Holmes said. That's just how much I was hurt inside. My heart hurt. I couldn't sleep at night, I didn't want to see anybody, I didn't want to talk to anybody. I was torn up.

She said that if she knew her DNA was going to be used to investigate a potential murderer in her family, she doesnt think she would have given it. I dont want to get involved thats the first thing I would say, she said.

On the other side of the case is the murder victims mother, Tina Franke, a retired nurses aide, who remembers her daughter as fearless and exuberant and a natural with children. She spent 17 years wondering if anyone would ever be charged with the murder. She got a tattoo on her right arm of a doodle her daughter once drew; it says Mom Dad I love you.

Fields explained to her the basics of how genetic genealogy was used to close the case, but Tina Franke said she didnt know how DNA was taken from Benjamin Holmes Jr.s relatives, including Holmes. I feel bad for her, she said.

Still, she said she had no problem with the tactic. Im glad for the end result, she said.

She wonders if Benjamin Holmes Jr.s relatives would feel differently if the situation were reversed.

If they can imagine their own daughter being murdered and 17 years have gone by and they still dont know who did it and they have DNA and no one to attach it to, she said, I think theyd want them to do what it took to find out who did it.

Read the original:
'They lied to us': Mom says police deceived her to get her DNA and charge her son with murder - NBC News

Recommendation and review posted by Bethany Smith

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12 Key Findings

13 Appendix

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Newborn Genetic Testing Market Executive Summary And Analysis By Top Players 2020-2026 - Keep Reading

Recommendation and review posted by Bethany Smith

Governor Andrew M. Cuomo today announced a 30-day amendment to the FY 2021 Executive Budget which will include legislation to establish the SUNY Curing Alzheimer's Health Consortium within the State University of New York. The Consortium will work to identify genes that predict an increased risk for developing Alzheimer's and collaborate with public and private research institutions on projects and studies to identify opportunities to develop new therapeutic treatment and cures for Alzheimer's. The goal of the Consortium will be to map the genetics of 1 million people, suffering from or at-risk of developing Alzheimer's Disease, over 5 years. This new wealth of data will support researchers as they work towards developing newtreatments and cures for the disease.

"Alzheimer's Disease affects hundreds of thousands of New Yorkers each year and takes a devastating toll on both patients and caregivers who lack access to sufficient treatment options due to an insufficient body of research"Governor Cuomo said."Genomics have made significant progress in the diagnosis and treatment of diseases ranging from cancer to cardiovascular disease, and could present major breakthroughs in the fight against Alzheimer's Disease. The Curing Alzheimer's Health Consortium will collect genomic data on a statewide scale and support genetic researchers as they work to slow the deadly progress of this disease."

SUNY will issue a request for proposals in partnership with Empire State Development's Life Sciences Initiative for private providers to partner with the SUNY system and other not-for-profit and private hospitals, and non-profit higher education research institutions to map the genomes of individuals suffering from or at risk of Alzheimer's.The ESD Life Science Initiative will provide $20M in existing funding to the Consortium to identify and recruit 200,000 people for genetic testing as part of phase one of the initiative.

Entities awarded the RFP will partner with SUNY's systems, including SUNYUpstate Medical, SUNY Downstate Medical, Renaissance School of Medicine at Stony Brook University,Jacobs School of Medicine and Biomedical Sciences at University at Buffalo, as well asother medical centers and hospitals,to launch an initial phase of their partnership that will map 1 million people suffering from, or at risk of, Alzheimer's over 5 years.Upon completion of the mapping, the resulting database will be made freely available to advance research on Alzheimer's Disease.

Alzheimer's in New York

According to the Department of Health, in 2017 an estimated 390,000 individuals in New York State suffered from Alzheimer's Disease, a figure that is expected to increase to 460,000 by 2025. Despite its prevalence, there remains a concerning lack of research and available treatment options to address Alzheimer's, which contributes to staggering disability and disease burden for patients, their families and society, and billions in economic costs annually to the State

Continue reading here:
Governor Cuomo Announces 30-Day Amendment to FY 2021 Executive Budget to Establish SUNY Curing Alzheimer's Health Consortium - ny.gov

Recommendation and review posted by Bethany Smith

Genetic Testing Devices market Research Report 2020 offers a comprehensive analysis of the market growth drivers, trends, opportunities, prospects, drivers and restrictions inside the market. The report emphasizes to meet the requirement of customers by providing complete knowledge of the Genetic Testing Devices Industry. This carefully organised report is formulated by industry experts and professional experts, in terms of demand and supply, cost organization, barriers and challenges, product category, crucial market players, technology, regions, and applications.

The Genetic Testing Devices market study is based on historical information and present market requirements. As well as includes different business approaches preferred by the decision-makers. That enhanced the Genetic Testing Devices industry growth and make a phenomenal stand in the industry. The market will raise with a prominentCAGRby 2020 to 2026.

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Myriad GeneticsCepheidLuminexSeegeneBioRad LaboratoriesWaferGen BiosystemsIntegraGenBioMerieuxInterpace DiagnosticsQiagenElitechElitechAbbott LaboratoriesBiocartisPerkinElmerRoche DiagnosticsEKF DiagnosticsQuest Diagnostics

Segmentation by Product Type

Type 1Type 2Type 3

Segmentation by Application/ End uses:

Application 1Application 2Application 3

Regional Analysis for Genetic Testing Devices Market:

North America (the United States, Canada & Rest of the countries)

Europe (Germany, The UK, France, Netherlands, Italy, Spain & the rest of the countries)

Asia-Pacific (China, Japan, Korea, India, & rest of the countries)

Middle East & Africa (South Africa, Israel, UAE & rest of the countries)

South America (Brazil, Colombia, Argentina & the rest of the countries)

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What is the regional structure of the market? Our analysis-

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YEARS CONSIDERED FOR THIS REPORT:

Historical Years:2015-2019

Base Year:2019

Estimated Year:2020

Forecast Period:2020-2026

DEFINITE SEGMENTS OF GLOBAL Genetic Testing Devices INDUSTRY:

The analysis highlights a region-wise as well as a worldwide study of the Genetic Testing Devices market. Proportionately, the regional study of the industry comprisesJapan, South East Asia, India, the USA, Europe, and China.Moreover, the report reviews an in-depth market analysis of distinct manufacturers and suppliers. It explainsindustry chain structure, competitive scenario, and study of Genetic Testing Devices industry costin detail. It evenly analyzes global industry size pursued by forecast period (2020-2026) and environment.

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KEY TOPIC COVERED

Growth Opportunities

Market Growth Drivers

Leading Market Players

Market Size and Growth Rate

Market Trend and Technological

Company Market Share

TOC OF Genetic Testing Devices MARKET REPORT INCLUDES:

1 Industry Overview of Genetic Testing Devices

2 Industry Chain Analysis

3 Manufacturing Technology

4 Major Manufacturers Analysis

5 Global Productions, Revenue and Price Analysis of Genetic Testing Devices by Regions, Creators, Types, and Applications

6 Global and Foremost Regions Capacity, Production, Revenue and Growth Rate by 2013-2019

7 Consumption Volumes, Consumption Value, Import, Export and Sale Price Analysis by Regions

8 Gross and Gross Margin Analysis

9 Marketing Traders or Distributor Analysis

10 Global and Chinese Economic Impacts on the Genetic Testing Devices Industry

11 Development Trend Analysis

12 Contact information

13 New Project Investment Feasibility Analysis

14 Conclusion of the Global Genetic Testing Devices Industry 2019 Market Research Report Continued

Finally, the feasibility of new investment projects is assessed, and overall research conclusions are offered.

Key questions answered by the Genetic Testing Devices Report:

What are some of the most favourable, high-growth prospects for the global Genetic Testing Devices market?

Which products segments will raise at a faster pace throughout the forecast period and why?

What are the foremost factors impacting market prospects?

What are the driving factors, restraints, and challenges in this Genetic Testing Devices market?

What are the competitive threats and challenges to themarket?

What are the evolving trends in this Genetic Testing Devices market and reasons behind their emergence?

What are some of the changing customer demands in the Genetic Testing Devices Industry market?

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Global Genetic Testing Devices Market Research Provides an In-Depth Analysis on the Future Growth Prospects and Market Trends Adopted by the...

Recommendation and review posted by Bethany Smith

Guardant Health (GH) is an $8 billion provider of diagnostic tests for early detection of cancer in high-risk populations and recurrence monitoring in cancer survivors.

With sales expected to break above $270 million this year, representing over 30% annual growth, their commercially available tests provide information that helps advanced cancer patients get the right treatment and helps drug companies get new therapies to market faster.

Conquering Cancer with Data

Examining cancer reappearance is widely expected to be a $15 billion market in the United States in the near term, while detecting patients with the risk of cancer is poised to be an $18 billion annual market.

Using specialized blood tests and liquid biopsy (examination of tissue) to detect cancer, Guardant has grown fast after launching the LUNAR DNA test for early detection. Along the spectrum, the companys Guardant360 and GuardantOMNI products have played a significant role in identifying late-stage cancer.

The LUNAR assay is also used for research and for use in prospective clinical trials, including initial studies related to screening and early detection in asymptomatic individuals.

In pursuing what the company calls "precision oncology," Guardant has made a name for itself in matching cancer patients with customized treatments.

Q3 Earnings Inspire Analysts to Raise Estimates

On November 7, Guardant reported a third-quarter 2019 revenue of $60.8 million, an increase of 181% over the prior year period. The company also surprised investors with a quarterly loss of only 14-cents when the Street consensus was looking for a loss of 39-cents, thus delivering a 64% positive surprise.

Here were some of the highlights of the quarter as reported by the company...

**Reported 13,259 tests to clinical customers and 5,280 tests to biopharmaceutical customers in Q3, representing year-over-year increases of 89% and 111%

**Initiated ECLIPSE, a large-scale registrational study designed to support the performance of the companys LUNAR-2 blood test in colorectal cancer screening in average-risk adults

**Submitted Premarket Approval Application package for Guardant360 to the U.S. Food and Drug Administration

**Launched CLIA-version of the LUNAR Assay for use in prospective clinical trials

During the quarter, the Guardant team continued to make significant progress across our business, said Helmy Eltoukhy, PhD, Chief Executive Officer. We are especially excited by the recent initiation of our ECLIPSE study. If successful, we believe this registrational study will pave the way for a blood-based test to address the significant unmet need of physicians and their patients who seek an alternative to the current tools for colorectal cancer screening.

After the 25-cent EPS surprise, Wall Street analysts raised their full-year 2019 consensus by 39-cents from a loss of $1.27 to a loss of 88-cents.

They also boosted next year's outlook from a loss of $1.29 to a loss of $1.13. On the top line, Guardant is expected to grow revenues by 32% to $272 million.

Race to the Future of Preventive Medicine

Genetic diagnostics is an exciting field full of innovative companies providing everything from full genome sequencing to specialized cancer tests.

Not only are people everywhere becoming more curious about their genetic ancestry, they're also getting more proactive about their genetic predisposition for health issues.

Guardant reports its Q4 earnings next week on 2/24, after two of its top peers: Exact Sciences (EXAS), the $13 billion maker of the Cologuard test, and Invitae (NVTA), the young $2.5 billion upstart in medical-grade genetic testing who reports its Q4 today 2/19, after the market close.

On Feb 11, Exact Sciences announced Q4 2019 results with Screening revenues of $229.4 million, reflecting a year-over-year increase of 60%. Cologuard test volume was 477,000, up 63% year over year.

Precision Oncology -- the name of the new business segment after the completed acquisition of Genomic Health Diagnostics (apparently Guardant doesn't have a trademark on that) -- added revenue of $66 million for the period Nov. 8, 2019 through Dec. 31, 2019. Proforma revenue for the new unit was $119 million, an increase of 14% percent from proforma 2018 revenue when Genomic Health was a stand-alone entity, primarily driven by Oncotype DX test volume of 41,000.

Story continues

Average Cologuard recognized revenue per test was $481. Moreover, average Cologuard cost per test of $123 reflected an improvement of $6 per test.

Exact Sciences gross profit (excluding the amortization of acquired intangibles) rose 114.1% to $225.2 million. Further, gross margin expanded 263 basis points (bps) to 76.2%.

The company anticipates revenues of $1.61-$1.65 billion for 2020. The projection includes Screening revenues and Precision Oncology revenues of $1.13-$1.15 billion and $485-$495 million, respectively.

Exact CEO Kevin Conroy made a presentation at the JPM Healthcare Conference in January which was very bullish in terms of their expansion into other leading cancer diagnostics. The next big test area will be for liver cancer for which they are partnered with Mayo Clinic.

Conroy, with a 32-slide presentation deck, also detailed the growth opportunities for Exact in other vital detection markets like pancreatic, esophageal, bladder, ovarian, cervical, stomach, lung, lymphoma, melanoma, kidney, and uterine cancers.

After earnings, Oppenheimer analysts, with the highest Street price target on EXAS of $130, commented on the company's financial guidance and disclosure of further M&A plans with "the first of what we believe could be a series of bolt-on acquisitions to leverage its newly acquired Precision Oncology sales channel. The acquisitions combined with more aggressive spending on the companys liquid biopsy franchise results in 2020 R&D guidance coming in $60M above our forecast. We view the increased R&D intensity as a structural component of the merged Exact Sciences-Genomic Health infrastructure which offers potential for upside surprise to our revenue forecast beginning in 2021 while also pushing out time to profitability."

At $100 and nearly a $13 billion market cap, the $1.65 billion revenue projection for this year makes EXAS trade at a forward price-to-sales multiple of under 8X.

Guardant Shares Are Not Cheap and Here's Why

Meanwhile, Guardant Health looks very expensive trading at 29.5X sales ($8 billion market cap / $270 million sales). Why the big disparity in valuations?

Because Guardant, since its 2018 IPO, has been viewed in light of a more rapid growth phase by virtue of its 82% climb in 2018 sales and its likely 125% ascent to over $200 million in 2019.

But more so, Guardant is testing liquid biopsy as a detection modality that could offer an important alternative in the 100 million-patient colorectal cancer (CRC) screening market. Exact also has clinical trials ongoing in this area and the data readouts by middle of next year are highly anticipated.

According to SVB Leerink analysts who have a $130 price target on GH shares, they see data readouts by 2H21-1H22 from potentially 3 large 10,000+ patient prospective, registrational trials using liquid biopsy (LBx) in average risk CRC screening indication. This sets the stage for ultimately revealing whether LBx as a modality would succeed in the broader cancer screening market, challenging the longstanding role of colonoscopy as the standard of care in this market.

The analysts recently said "With the current commitments into these large blood-based 10,000+ patients trials costing $70M-$100M, we believe these companies see potential for liquid biopsy to hold a position in this large market ultimately."

They also noted two recent updates that confirm their conviction in GH. Guardant provided two product/pipeline updates around the JPM Healthcare Conference: 1) a collaboration with Amgen (AMGN) to develop a G360 companion diagnostic (CDx) for KRAS inhibitor AMG 510, and 2) Initiation of NRGI005 COBRA study aimed at validating the utility of Guardant's molecular residual disease (MRD) LUNAR-1 assay for selecting patients for adjuvant chemotherapy in stage II colorectal cancer (CRC). "We view these developments favorably and are pleased to see a continued global pharma partnership focus as GH pursues the $50B+ liquid biopsy market."

This Amgen partnership is obviously a big plus for Guardant. And it's possible that after the Exact buyout of Genomic Health that some investors look at GH as a potential acquisition target by a much larger player like Amgen or Biogen (BIIB).

After a big 2019 post-IPO rally, GH shares fell from above $110 to $60. Then Q3 earnings got investors interested again in the story at a better valuation. In fact, the fourth quarter of 2019 saw net accumulation by institutional investors, with one of my favorite stock-pickers, Andreas Halvorsen at Viking Global, adding 960K shares to bring his haul to 3,834,753.

Meet the Young Upstart: Invitae

Invitae, whose sales are also expected to eclipse $200 million for 2019 (we'll know when they report today), will be watched closely by investors and analysts who want to see and understand their 2020 game plan for 725,000 genetic test samples and $330 million in sales, which would represent more than 50% annual growth in both volume and revenue.

This full-year guidance was recently revised downward from their goal of 1 million tests and $500M in revenues.

Challenges for all of these companies include making money when the cost of testing is coming down so quickly and making sure that insurance reimbursement is available. Invitae has a head start in this arena after being selected by UnitedHealth (UNH) as one of only 7 key providers in their Preferred Lab Network, which includes the Mayo Clinic.

Plus, Invitae is creating a bigger brand footprint by offering many tests for free to those in need. In July, the company announced their Detect programs to provide no-charge genetic testing for conditions in which testing is underutilized and can improve diagnosis and treatment.

According to Invitae, research has shown no-charge testing programs result in earlier diagnosis and treatment. Enrollment was opened for Detect programs in four conditions: muscular dystrophy, prostate cancer, cardiomyopathy and arrhythmia and lysosomal storage diseases.

And in September, Invitae expanded the Detect program by announcing the availability of Detect Hereditary Pancreatic Cancer, a testing program that offers no-charge genetic testing and counseling to patients with pancreatic cancer. Genetic testing is recommended by clinicians for all pancreatic cancer patients to guide treatment choices and evaluate eligibility for clinical trials.

While the Detect no-charge programs are a net cost for the company, they create a wider data set for research in addition to fostering good will throughout public and medical communities.

In the neighborhood of innovation, Invitae announced in November the launch of Invitae Discover, a clinical research platform that leverages biometric data available through Apple Watch to provide better understanding of the genetic causes of disease. The first study on the platform will evaluate genetics in cardiovascular disease and was announced in conjunction with the American Heart Association's Scientific Sessions where researchers are presenting data on genetic screening in familial hypercholesterolemia.

Then in January, Invitae announced with BioMarin Pharmaceutical that Biogen, Encoded Therapeutics, Neurogene, Praxis Precision Medicines and PTC Therapeutics joined Behind the Seizure, an innovative, cross-company collaboration that aims to provide faster diagnosis for young children with epilepsy. The program will also be expanded to make no-charge testing available for healthcare providers to order for any child under the age of eight who has an unprovoked seizure.

While Guardant is expensive relative to EXAS and NVTA, it remains a unique leader in its specialties and I would recommend owning any two of these genetic diagnostics companies to remain at the forefront of the genomic revolution.

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Bull of the Day: Guardant Health (GH) - Yahoo Finance

Recommendation and review posted by Bethany Smith