Category Archives: Gene Medicine

Chris Gard and Connie Yates, the parents of terminally ill baby Charlie Gard, speak to the media on Monday in London.

Carl Court/Getty Images

The parents of Charlie Gard announced on Monday that theyd given up on treating their 11-month-old child, who suffers from a rare and deadly gene mutation affecting his mitochondrial DNA. The roller-coaster case began in February when physicians at the London hospital treating the infant said it was time to remove Charlies life support. They refused to let the British couple fly him to New York City for a last-ditch, experimental treatment that, according to its inventor, had a small but significant chance of reversing his brain damage. Over the past five months of legal battles, the hospital never wavered from its claims that every reasonable means of saving Charlie had been tried already and that he should be spared any further suffering that might come with a form of therapy that has never been tested on a patient with exactly his condition, and which isnt part of any clinical trial.

Daniel Engberis a columnist for Slate.

Charlies parents now say that its too late for any intervention and that its time to let him go. But for several months now, the #CharlieGard saga has served as the focus for a broader push for patients rights in Washington. Conservative politicians were quick to champion Charlies parents causePresident Trump tweeted his support and the House tried to grant the couple permanent residencyin keeping with the GOPs strong endorsement of so-called right-to-try laws. These measureslately passed in 33 statesare meant to guarantee very sick people access to experimental or nonconventional medical treatments that havent yet passed muster with the Food and Drug Administration. In practice, that means the parents of a dying patient such as Charlie wouldnt need to ask permission from the FDA to move ahead with therapy; they could just request it directly from the manufacturer.

Its hard to argue with vocal patient-advocates who say their lives were saved by gaining access to experimental treatments. The right to try sounds like common sense: It should be up to patients to decide whether the potential upside of a treatment (surviving a terminal illness) seems worth any risk of painful side effects. Why not let them give it hell and go down swinging? But stepping back from anecdotes, the spread of experimental access laws (like the calls for Charlies puddle-jumping medevac) suggests that critical decisions about the final months of peoples lives are often based on biased judgments of reality. Patients seem to overvalue innovation, as a rule, and assume that newer drugs have a better chance of working than any other treatment, just because theyre new. Not only does this sanguine view of scientific progress fail to fit the facts; it also leads patients to the converse, false impression that nonconventional treatments arent likely to be harmful in themselves. A more sober view suggests that the hope that often moves people to seek out these types of treatmentsand the ever-present pressure to fight until the endis not as useful as we think.

The spread of experimental access laws suggests that critical decisions about the final months of peoples lives are often based on biased judgments of reality.

Unfounded optimism tends to be the rule in medicine. A 2015 review of several dozen studies of peoples expectations from treatment, comprising data from more than 27,000 subjects, found systematic evidence of a Pollyanna Patient problem: We overestimate the value of the care that we receive and underestimate its harm. That work is cited in an excellent article by Liz Szabo of Kaiser Health News, on the surprising ineffectiveness of cancer drugs that have been FDA approved. Its not just that these treatments do little to prolong survival, Szabo says; according to one study, many patients never grasp this fact. In a sample of several thousand adults, 39 percent said they believe the FDA only approves prescription drugs that are extremely effective; 1 in 6 asserted that drugs that have serious side effects cannot be advertised to consumers. Neither statement is even close to being true. According to Vinay Prasad, an oncologist and expert in evidence-based medicine at Oregon Health and Sciences University, we dont have any hard evidence of benefitin terms of patients living longer livesfor the majority of cancer drugs approved in recent years.

If FDA-approved drugs often fail to offer substantive benefits, then experimental onesthose that havent even passed the suspect bar for agency signoffare even less likely to be helpful. In fact, about 90 percent of experimental treatments flunk out during clinical trials, either because they arent shown to be any more effective than the standard treatment or because their side effects are too severe. In some cases, experimental treatments once thought to be miraculouslike the use of bone-marrow transplants as a cure for breast cancer, which started in the 1980shave turned out to be worse than ineffective in clinical trials. In the bone marrow case, the procedure could be deadly on its own. This abysmal failure rate persists in spite of the enormous cost of running trials and researchers clear incentive (read: bias) to produce positive results.

Such dire stats have done little to discourage eager patients, though. When it comes to clinical trials, we seem to harbor a version of the favoritelong shot biasthe tendency of horse-track gamblers to overvalue the underdog at the expense of the odds-on favorite. In medicine, this translates to fixation on the value of experimental treatmentsand the remote possibility that they might turn out to be wonder cures. Indeed, for those who are faced with imminent death, the desire to bet ones health on long-shot drugs (and the right to do so, when all other options have been tried) is so insistent that patients even deride clinical trials as another structure blocking access to potentially life-saving treatments. The trials randomized treatment groups and stringent inclusion criteria mean the majority of patients never get the chance to serve as guinea pigs at all.

In certain casesthink of early AIDS drugs or Ebola vaccinesthis rigidity can indeed have tragic consequences. But how much rigor should be sacrificed, and how many rules should be suspended, on behalf of patients whose expectations may be substantially inflated? In late June, that question served as the backdrop for a two-day symposium of doctors, bioethicists, patient-advocates, and public-health officials on the future of randomized controlled trials. The problems with RCTs are legion, speakers said: Theyre not well-suited to emerging threats; theyre too expensive; theyre too slow.

But it seemed just as clear from the proceedings that patients should think twice before they clamor for inclusion in these trials and for greater flexibility in their administration. The randomized trial is the single greatest medical innovation of the 20th century, said Prasad, who was in New York City for the meeting. But he warned against the use of massive studies of experimental treatments that may have only very tiny benefits in the end. Its unethical, he said, to put so many desperate patients on a drug unless you have good reason to believe in its effectiveness.

Even in this era of informed consent, patients may not understand exactly what they stand to gain (or lose) by entering a trial. Research going back to 1982 has found that many suffer from a therapeutic misconception: They assume theyll benefit personally from being in a clinical trial, though in fact they may not get the tested treatmentand even if they did, chances are it wouldnt help. (In fairness, some researchers now say this problem has been overstated.) My advice is, youre better off in the control group, warned former FDA chief Robert Califf in his keynote lecture at the symposium, speaking to prospective patients in the audience who had been arguing for greater access to experimental drugs. Most things dont work or theyre dangerous.

This creates an intoxicating atmosphere of progressa sense that new and better treatments are always on the verge of coming out.

The fact that an experimental drug is usually a bad bet isnt likely to dull our instinct to gamble on untested treatments, though. The idolatry of experimentation has even spawned a sinister, for-profit industry, lurking in the shadows of the FDA approval process. In a disturbing paper published last week, bioethicist Leigh Turner describes how the government website ClinicalTrials.gova registry established in 1997 to improve the reliability of formal research on potential treatmentsis being used to market sketchy medical practices. Patients who are looking for a way to break into a clinical trial may scan the registry for opportunities to volunteer; now, instead of finding only legitimate, government-sanctioned research trials, they could land on so-called patient-funded or patient-sponsored ones. In these, they have to pay for access to a therapy that isnt necessarily based on any peer-reviewed, preclinical data, and which may lack any evidence of safety or effectiveness. (Already there have been reports of patients suffering severe complications from their participation in these ersatz trials.)

What makes us so gung-ho for things that arent fully tested? It may in part be human nature, but aspects of the bias seem to be conditioned, too. Even honest science coverage tends to focus on putative medical breakthroughs that have either just occurred or may be coming soon; less scrupulous media figures hawk salves or potions with little basis whatsoever. Taken altogether this creates an intoxicating atmosphere of progressa sense that new and better treatments are always on the verge of coming out.

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We're also too quick to pretend that a dead child isn't dead; Charlie Gard died months ago. Nothing that was offered would have changed that. I mention this because the other prob with have is an infantile faith in miracles. More...

Yet the excitement in the air rarely matches up to reality: Actual medical advancement tends to be incremental and excruciatingly slow. The discord this createsbetween the feeling of innovation inspired by the media and the real options that were offered in the clinicmay distort our view of experimental treatments. It could make us think there must be some reason why our cancers havent yet been cured; there must be some external factors preventing us from getting access to the new and better drugs weve heard so much about. If only regulators werent so overcautious and uptight, we end up thinking, it would be possible to tap this cache of innovation.

Right-to-try laws indulge the fear that unbending bureaucrats in Washington have kept patients from medical cures with an excess of red tape. In fact, these laws have little real effect. Thats because the FDA already offers access to experimental treatments with very modest oversightand in recent years the agency has done away with a few unnecessary rules that slowed the process down. The problem isnt that patients (or their parents) have insufficient freedom to decide how theyd like to balance out potential risks and benefits from experimental treatments. Its that our bias often makes them victims of false hope.

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Our Unfounded Medical Optimism - Slate Magazine

July 25, 2017

Researchers from Genethon, the AFM-Telethon laboratory, Inserm (UMR) and the Royal Holloway University of London demonstrated the efficacy of an innovative gene therapy in the treatment of Duchenne muscular dystrophy. Indeed, after injecting microdystrophin (a "shortened" version of the dystrophin gene) via a drug vector, the researchers managed to restore muscle strength and stabilise the clinical symptoms in dogs naturally affected by Duchenne muscular dystrophy. A first. This work, published today in Nature Communications, has been achieved thanks to donations from the French Telethon.

Duchenne muscular dystrophy is a rare progressive genetic disorder involving all the muscles of the body, and affects 1 in 5,000 boys. It is the most common neuromuscular disorder in children. It is associated with abnormalities in the DMD gene, which encodes dystrophin, a protein that is essential for proper muscle function. This gene is one of the largest in our genome (2.3 million base pairs, of which over 11,000 are coding). Because of this size, it is technically impossible to insert the entire DNA for dystrophin into a viral vector (or even the 11,000 coding base pairs alone), as is usually done for gene therapy.

To meet this challenge, teams at Genethon developed, in collaboration with a team at Royal Holloway University of London led by Pr. Dickson, and produced, a gene therapy drug combining an AAV-type viral vector with a shortened version of the dystrophin gene (approximately 4,000 base pairs), allowing the production of a functional protein. Dr Le Guiner's team tested this innovative treatment in 12 dogs naturally affected by Duchenne muscular dystrophy. By injecting this microdystrophin intravenously, and hence into the whole body of the dogs, the researchers observed that dystrophin expression returned to a high level, and muscle function was significantly restored, with stabilisation of the clinical symptoms observed for over 2 years following injection of the drug (see video). No immunosuppressive treatment was administered beforehand, and no side-effects were observed.

Some Golden Retrievers develop Duchenne muscular dystrophy naturally. The successful treatment of these dogs, which show the same clinical symptoms as children with this disease, and are of a similar weight, is a decisive step toward developing the same treatment in children."This preclinical study demonstrates the safety and efficacy of microdystrophin, and makes it possible to consider developing a clinical trial in patients. Indeed, this is the first time that it has been possible to treat the whole body of a large-sized animal with this protein. Moreover, this innovative approach allows treatment of all patients with Duchenne muscular dystrophy, regardless of the genetic mutation responsible," says Caroline Le Guiner, the main author of this study.

"This is tremendously exciting progress towards a gene therapy for DMD. The studies in GRMD dogs have been spectacular and exceeded our expectations. My team has worked for many years to optimise a gene therapy medicine for DMD, and now the quite outstanding work of colleagues in France, in Genethon, in Nantes and in Paris has taken us close to clinical trials in DMD patients. I pay thanks also to the amazing and steadfast support of this research by AFM-Telethon and MDUK (Muscular Dystrophy UK) which has been essential to this achievement." commented George Dickson.

For Frederic Revah, Chief Executive Officer of Genethon: "For the first time, researchers obtained a systemic therapeutic effect on a neuromuscular disease in dogs using microdystrophin, and without immunosuppressive treatment. This highly complex cutting edge technology has been developed as part of an exceptional collaborative effort between Genethon and academic teams from Britain and France. Now our bioproduction experts have the task of producing a sufficient quantity of these new drug vectors, under GMP conditions, for the clinical trial."

"This new evidence of the efficacy of gene therapy in Duchenne muscular dystrophy strengthens the therapeutic arsenal developed (exon skipping, CRISPR Cas-9, pharmacogenetics, etc.), and the first results are there. We need to forge ahead to complete the final phase and transform these scientific advances into drugs for children," emphasises Serge Braun, Scientific Director of AFM-Telethon.

Explore further: Gene therapy treats all muscles in the body in muscular dystrophy dogs

More information: Caroline Le Guiner et al, Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy, Nature Communications (2017). DOI: 10.1038/ncomms16105

Journal reference: Nature Communications

Provided by: AFM-Telethon laboratory

(HealthDay) Emflaza (deflazacort) has been approved by the U.S. Food and Drug Administration to treat Duchenne muscular dystrophy in people five years and older, the agency said Thursday in a news release.

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Microdystrophin restores muscle strength in Duchenne muscular dystrophy - Medical Xpress

It is a privilege to lead the center and to leverage my previous experience to build Stanfords preeminence in stem cell and gene therapies, said Roncarolo, who is also chief of pediatric stem cell transplantation and regenerative medicine, co-director of the Bass Center for Childhood Cancer and Blood Diseases and co-director of the Stanford Institute for Stem Cell Biology and Regenerative Medicine. Stanford Medicines unique environment brings together scientific discovery, translational medicine and clinical treatment. We will accelerate Stanfords fundamental discoveries toward novel stem cell and gene therapies to transform the field and to bring cures to hundreds of diseases affecting millions of children worldwide.

The center consists of several innovative pieces designed to allow the rapid development of early scientific discoveries into the clinic that in the past have languished. This includes an interdisciplinary team of basic and clinical scientists to shepherd nascent therapies developed at Stanford. The team will be headed by associate directors Matthew Porteus, PhD, associate professor of pediatrics, and Anthony Oro, MD, the Eugene and Gloria Bauer Professor and professor of dermatology.

To help with clinical development, the center boasts a dedicated stem cell clinical trial office with Sandeep Soni, MD, clinical associate professor of pediatrics, as medical director. In addition, the center has dedicated clinical trial hospital beds in the Bass Center for Childhood Cancer and Blood Diseases located on the top floor of the soon-to-open LucilePackardChildrensHospital. From work performed by scientists over the past decade, the center already has a backlog of nearly two dozen early stage therapies whose development the center will accelerate.

The center will provide novel therapies that can prevent irreversible damage in children, and allow them to live normal, healthy lives, said Mary Leonard, MD, professor and chair of pediatrics and physician-in-chief at Stanford Childrens Health. The stem cell and gene therapy efforts within the center are aligned with the strategic vision of the Department of Pediatrics and Stanfords precision health vision, where we go beyond simply providing treatment for children to instead cure them definitively for their entire lives.

One of the unique features of the center is its close association with the recently opened $35 million Stanford Laboratory for Cell and Gene Medicine, a 23,000-square-foot manufacturing facility located on California Avenue in Palo Alto. One of the first of its kind in the world, the laboratory has the ability to produce newly developed cell and gene therapy therapies according to the Good Manufacturing Practice standards as required for patient treatment.

Headed by executive director David DiGiusto, PhD, the lab can produce diverse cellular products for patient use, such as genetically corrected bone marrow cells for sickle cell anemia, genetically-engineered skin grafts for children with the genetic disease epidermolysis bullosa or genetically-engineered lymphocytes to fight rejection and leukemia.

We are fortunate that Stanford researchers have created such a strong portfolio of innovative candidate therapeutics to develop, said DiGusto. The capabilities of the laboratory will bridge the gap between research and clinical investigation so that the curative potential of these exciting cell and gene therapies can be realized.

For more information about the center, or for information about trials associated with the center, please see https://med.stanford.edu/ptrm/faculty.html, or contact Jennifer Howard at jmhoward@stanford.edu.

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Stanford announces new Center for Definitive and Curative Medicine - Stanford Medical Center Report

Dr. Sanford Markowitz, founder of Rodeo Therapeutics. (Case Western Reserve University Photo)

If you could write a medical wish-list of futuristic technologies, regenerating tissue would be pretty high up there. It could do things like treat a variety of inflammatory conditions and even help cancer patients regrow healthy cells.

A new Seattle-based biotech startup, Rodeo Therapeutics, is hoping its technology can make tissue regeneration a reality, and it has just raised a $5.9 million Series A round from Seattle-based biotech fund Accelerator Corporationto make it happen.

The general idea is simple: Rodeo is hoping to use small-molecule therapies a category most drugs fall into that stimulate the bodys natural regeneration process, like when a skinned knee heals.

Its first focus is to develop a treatmentfor inflammatory bowel disease and one that can help cancer patients cells grow quickly following stem cell transplants. But those goals are just the beginning.

The ability to stimulate the bodys natural processes for tissue regeneration and repair has broad therapeutic potential in disease settings such as ulcerative colitis and in hemopoietic recovery following bone marrow transplantation. Rodeo Therapeutics is focused on developing small-molecule therapies that stimulate these processes and enable new approaches to address serious medical conditions that today have a substantial unmet medical need, said Rodeo Therapeutics founder and cancer researcher Dr.Sanford Markowitz.

The company is currently working on drugs that inhibit an enzyme called 15-PGDH, which has been shown to speed up regenerative processes.

The startup was founded by Markowitz and Dr. Stanton Gerson, researchers at Case Western University, along with Dr.Joseph Ready, a researcher at the University of Texas Southwestern Medical Center.

Its technology is based on their work. Markowitz is an expert in gastrointestinal cancers, where inflammation can cause serious problems; Dr. Gerson specializes in stem cell and genetic research along with gene therapies and cancer drug development; and Dr. Ready works in regenerative medicine and cancer, specifically synthetic and medicinal chemistry.

The startups corporate office is currently in Accelerator Corporations facilities in Seattle, with its founders in Dallas and Cleveland. Rodeos early operations will be overseen by Accelerator, and the funds CEO Thong Le is currently servingas Rodeos CEO.

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Regenerative medicine startup Rodeo Therapeutics raises $5.9M for drugs to regenerate tissue - GeekWire

BOSTON, July 24, 2017 /PRNewswire/ --Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced the initiation of TAZPOWER, a Phase 2/3 study evaluating elamipretide in patients with Barth syndrome. Barth syndrome is a rare genetic mitochondrial disease, caused by mutations in the TAZ gene, and characterized by cardiac abnormalities, skeletal muscle weakness, recurrent infections and delayed growth.

"The severe problems experienced by patients with Barth syndrome are caused by misshapen and dysfunctional mitochondria, which reduce the energy production in the affected tissues. The resulting muscle weakness can lead to severe fatigue, heart failure and death," said Stealth Chief Medical Officer Doug Weaver. "In this study, we hope to show that elamipretide may have clinical benefit by improving function in these affected mitochondria."

TAZPOWER is a randomized, double-blind, placebo-controlled crossover study that will evaluate the effects of daily elamipretide treatment in a minimum of 12 patients with genetically confirmed Barth syndrome. Patients will be randomized to one of two sequence groups: 12 weeks of single daily subcutaneous injections of elamipretide in Treatment Period 1, followed by 12 weeks of treatment with placebo in Treatment Period 2, with a four-week wash-out period between periods, or vice versa. The primary endpoint is change in distance walked during the six-minute walk test. Secondary endpoints include functional assessments, patient-reported outcomes and safety.

"Our understanding of Barth syndrome and how it manifests has evolved significantly, but current treatment efforts are still limited to the management of symptoms," said Hilary Vernon, M.D., Ph.D., assistant professor of Pediatrics at McKusick-Nathans Institute of Genetic Medicine at Johns Hopkins University and the primary investigator for the study. "The initiation of TAZPOWER represents an important milestone in the potential development of a disease-specific treatment option."

TAZPOWER builds upon Stealth BioTherapeutics's existing rare disease and cardiorenal programs, including three ongoing Phase 2 studies in adults with heart failure (IDDEA-HF, PROGRESS-HF, RESTORE-HF).

"This study underscores our commitment to develop elamipretide for the treatment of rare genetic mitochondrial diseases," said Stealth Chief Executive Officer Reenie McCarthy. "The cardiovascular and skeletal muscle symptoms affecting this population share a common thread with symptoms experienced in diseases commonly associated with aging, such as heart failure, in which mitochondrial dysfunction contributes to the clinical pathology."

For additional information on the TAZPOWER study or elamipretide, please refer to Stealth's website.

About Barth Syndrome Barth syndrome is a rare genetic condition characterized by muscle weakness, cardiac abnormalities, recurrent infections and delayed growth. Barth syndrome occurs almost exclusively in males and is estimated to affect one in 200,000 to 400,000 individuals worldwide at birth. There are currently no FDA-approved therapies for the disease.

About Stealth BioTherapeutics We are a privately held clinical-stage biotechnology company focused on the development of therapeutics for diseases involving mitochondrial dysfunction. We believe there is a strong rationale for our lead product candidate,elamipretide, in indications in these diseases based on encouraging preclinical and early clinical data. We are investigating elamipretide in three primary mitochondrial diseases primary mitochondrial myopathy (PMM), Barth syndrome and Leber's hereditary optic neuropathy (LHON) as well as in heart failure, Fuchs' corneal dystrophy and dry age-related macular degeneration.We received Fast Track designation for elamipretide for the treatment of PMM from the FDA in December 2015. We are developing our second product candidate, SBT-20, for central nervous system disorders.Our mission is to be the leader in mitochondrial medicine. To learn more information about us and our pipeline, visitwww.stealthbt.com.

Contacts Media Relations dna Communications Kate Contreras, 617-520-7088 rel="nofollow">Media@StealthBT.com

Investor Relations Stern IR Beth DelGiacco, 212-362-1200 rel="nofollow">IR@StealthBT.com

View original content with multimedia:http://www.prnewswire.com/news-releases/stealth-biotherapeutics-initiates-phase-23-study-of-elamipretide-in-patients-with-barth-syndrome-300492467.html

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Stealth BioTherapeutics Initiates Phase 2/3 Study of Elamipretide in Patients With Barth Syndrome - Markets Insider

July 25, 2017 Emmental cheese. Credit: Wikipedia

Researchers at the University of California San Diego have found that microbial species living on cheese have transferred thousands of genes between each other. They also identified regional hotspots where such exchanges take place, including several genomic "islands" that host exchanges across several species of bacteria.

Postdoctoral fellow Kevin Bonham and assistant professor Rachel Dutton of UC San Diego's Division of Biological Sciences, along with Benjamin Wolfe, a former postdoctoral fellow in the Dutton lab now at Tufts University, use the rinds of artisanal cheese varieties as simple model systems to study microbiomes, or communities of microorganisms. Microbiomes are known to play a key function in many areas, including human health, protecting us from some diseases and amplifying others.

Cheese rinds offer a novel way to study how genes in microbial communities are passed from one organism to another in a process known as "horizontal gene transfer." Details of the study were published July 25th in the journal eLife.

"We examined the genomes of over 150 bacteria from cheese, and found more than 4,000 genes that were shared between bacterial species, including several large genomic islands that were shared by many species," said Dutton, an assistant professor in the Molecular Biology Section and part of UC San Diego's Center for Microbiome Innovation, which leverages the university's strengths in clinical medicine, bioengineering, computer science, the biological and physical sciences, data sciences and other areas to coordinate and accelerate microbiome research. "Horizontal gene transfer has been studied for decades, but examining it in a more natural context is challenging because it requires studying an entire community of microbes, rather than studying them in isolation."

Dutton said a large percentage of transferred genes involved functions dealing with acquiring nutrients, especially iron, which is known to be in short supply on the surface of cheese. Competition for iron is an important theme for microbes in many environments, including during infections of humans by pathogenic microbes.

"Horizontal gene transfer could influence competition for iron and possibly enable 'cheating' within a mixed community," said Dutton.

Based on the new results, Dutton and her colleagues are now probing the intricate dynamics of horizontal gene transfer and how the process unfolds on cheese.

"Since horizontal gene transfer is prevalent in many microbial communities, including those important for human health, we're now trying to study how this process impacts microbial life and death in a community," said Dutton.

Explore further: Researchers study cheese to unlock secrets of how microbial communities form

More information: Kevin S Bonham et al, Extensive horizontal gene transfer in cheese-associated bacteria, eLife (2017). DOI: 10.7554/eLife.22144

Go ahead and call Rachel Dutton's research cheesy if you must. As far as she's concerned, it's anything but an insult. A Bauer Fellow at the Faculty of Arts and Sciences' Center for Systems Biology, Dutton and her lab study ...

While many microbiologists build entire research careers around studies of a single microorganism, Rachel Dutton has taken her career in the other directionexamining collections of microbes, but with an unusual twist. ...

Certain antibiotic resistance genes are easily transferred from one bacterial species to another, and can move between farm animals and the human gut. A team led by Chinese researchers has characterized this "mobile resistome," ...

Gene transfers are particularly common in the antibiotic-resistance genes of Streptococcus pneumoniae bacteria.

People sure love their cheeses, but scientists have a lot to learn about the fungi responsible for a blue cheese like Roquefort or a soft Camembert. Now researchers reporting in the Cell Press journal Current Biology on September ...

(PhysOrg.com) -- In a new study, scientists at the University of Maryland and the Institut Pasteur show that bacteria evolve new abilities, such as antibiotic resistance, predominantly by acquiring genes from other bacteria.

Researchers from Monash University's Biomedicine Discovery Institute have helped solve the mystery of how emus became flightless, identifying a gene involved in the development and evolution of bird wings.

Researchers at the University of California San Diego have found that microbial species living on cheese have transferred thousands of genes between each other. They also identified regional hotspots where such exchanges ...

A team of scientists from the Kunming Institute of Botany in China and the Max Planck Institute for Chemical Ecology in Jena has discovered that parasitic plants of the genus Cuscuta (dodder) not only deplete nutrients from ...

Our bodies are composed of trillions of cells, each with its own job. Cells in our stomach help digest our food, while cells in our eyes detect light, and our immune cells kill off bugs. To be able to perform these specific ...

Humpback whales learn songs in segments like the verses of a human song and can remix them, a new study involving University of Queensland research has found.

New research from Australia and Sweden has shown how a dragonfly's brain anticipates the movement of its prey, enabling it to hunt successfully. This knowledge could lead to innovations in fields such as robot vision.

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Thousands of genes exchanged within microbial communities living on cheese - Phys.Org

July 24, 2017 06:00 ET | Source: Sernova Corp

LONDON, Ontario, July 24, 2017 (GLOBE NEWSWIRE) -- Sernova Corp. (TSX-V:SVA) (OTCQB:SEOVF) (FSE:PSH), a clinical stage regenerative medicine company, announced today significant scientific progress achieved in the development of a first in world personalized regenerative medicine therapy for the treatment of Hemophilia A patients by the HemAcure Consortium and confirmation of the second phase of funding of the Consortium by the European Commission. The therapy being developed by international scientific Consortium members consisting of three European academic institutions, an enterprise for quality management and Sernova Corp is to treat severe Hemophilia A, a serious genetic bleeding disorder caused by missing or defective clotting factor VIII in the blood stream. This therapy consists of Sernovas implanted Cell Pouch(TM) device transplanted with therapeutic cells, corrected to produce Factor VIII at a level sufficient to significantly reduce the side effects of the disease and improve patient quality of life.

The international HemAcure Consortium team members are pleased with the ground breaking scientific advances achieved at this point and are on track for this regenerative medicine solution to advance into human clinical evaluation, remarked Dr. Philip Toleikis, Sernova President and CEO. Toleikis added, Sernovas Cell Pouch platform technologies are achieving important world first milestones in both diabetes and now hemophilia, two significant clinical indications which are being disrupted by its regenerative medicine approach aimed at significantly improving patient quality of life.

We are thrilled with the approval by the European Union of the next stage of funding for the HemAcure program based on our quality interim report. This is a strong validation of the Consortiums dedication and teamwork and the importance of this regenerative medicine approach, said Dr. Joris Braspenning, HemAcure Program Coordinator.

In summary, the following ground-breaking developments have been achieved by the Consortium:

This combination of advances by the HemAcure team represents a first in world achievement towards developing a regenerative medicine therapy for the treatment of severe hemophilia A patients. In this regard, these fundamental advancements have set the stage for further optimization and implementation of cell production processes under controlled GMP conditions, stated Martin Zierau, IMS member consortium team leader responsible for coordination of GMP processes. With Factor VIII corrected cells, studies are ongoing to optimize cell dosing within the Cell Pouch and for study of safety and efficacy of hemophilia corrected Factor VIII cells in the hemophilia model. These studies are in support of the current extensive regulatory package already assembled for the Cell Pouch in anticipation of human clinical evaluation of the Cell Pouch with hemophilia corrected Factor VIII producing cells.

Sernova has developed its proprietary highly innovative Cell Pouch technologies for the placement and long-term survival and function of immune protected therapeutic cells. It has proven to be safe and efficacious in multiple small and large animal preclinical models and has demonstrated safety alone and with therapeutic cells in a clinical trial in humans for another therapeutic indication. We believe the Cell Pouch platform is the first such patented technology proven to become incorporated with blood vessel enriched tissue-forming tissue chambers without fibrosis for the placement and long-term survival and function of immune protected therapeutic cells.

About Hemophilia A

People with Hemophilia have prolonged abnormal bleeding as a result of trauma. Hemophilia A, also called factor VIII (FVIII) deficiency is the most common form of Hemophilia and is a genetic disorder caused by missing or defective FVIII, a blood clotting protein. Severe hemophilia occurs in about 60% of cases where the deficiency of FVIII is less than 1% of normal blood concentration. While it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous change in the gene. According to the US Centers for Disease Control and Prevention hemophilia occurs in about 1 in 5,000 births. If the prolonged bleeding occurs in the brain of a person with hemophilia, it can be fatal. Prolonged bleeding in joints can cause inflammatory responses and permanent joint damage. Approximately 20,000 people in the United States and 10,000 in Europe have the moderate or severe form of hemophilia A, as well as approximately 2,500 in Canada. All races and ethnic groups are equally affected by hemophilia A. Though there is no cure for the disease, it can be controlled with regular infusions of recombinant clotting FVIII. Annual costs for the treatment of the disease for each patient may range from $60,000 to $260,000 US for a total cost of between $2-5B per year in North America and Europe.

About Horizon 2020 Programme

Horizon 2020 is the biggest EU Research and Innovation program ever with nearly 80 billion of funding available over seven years (2014 to 2020). It promises more breakthroughs, discoveries and world-firsts by taking great ideas from the lab to the market. The project is funded as part of societal challenges personalizing health and care in a specific call about innovative treatments and technologies. New therapies, such as gene or cell therapies, often require technological innovation in the form of development of specific component tools and techniques such as isolation and multiplication of a cell or development of a scaffold, delivery of the therapy to the patient and for following-up the effect of the therapy in the patient. In particular, achieving therapeutic scale production and GMP standards at reasonable cost is often underestimated. The European Union aims to improve the development of advanced methods and devices for targeted and controlled delivery, and to bring these innovative treatments to the patient.

About HemAcure

HemAcure is the name of the consortium developing a product for hemophilia A. This project has received funding from the European Union's Horizon 2020 research and innovation program under grant agreement No 667421. The consortium members include the University Hospital Wuerzburg (Coordinating Institute), Germany, IMS - Integrierte Management, Heppenheim, Germany, Universit del Piemonte Orientale Amedeo Avogadro, Novara, Italy, Loughborough University, Loughborough, United Kingdom, ARTTIC International Management Services, Munich, Germany and Sernova Corp., London, Ontario, Canada. The main objective of the HemAcure project is to develop and refine the tools and technologies for a novel ex vivo prepared cell based therapy within Sernovas prevascularized Cell Pouch to treat this bleeding disorder that should ultimately lead to improved quality of life of the patients. http://www.hemacure.eu; twitter.com/HemAcure_EU

About Sernova

Sernova Corp is a clinical stage regenerative medicine Company developing medical technologies for the treatment of chronic debilitating metabolic diseases such as diabetes, blood disorders including hemophilia and other diseases treated through replacement of proteins or hormones missing or in short supply within the body. Sernova is developing the Cell Pouch, an implantable medical device and therapeutic cells (donor, xenogeneic or stem cell derived therapeutic cells) which then release proteins and/or hormones as required.

Forward-Looking Information

This release may contain forward-looking statements. Forward-looking statements are statements that are not historical facts and are generally, but not always, identified by the words expects, plans, anticipates, believes, intends, estimates, projects, potential and similar expressions, or that events or conditions will, would, may, could or should occur. Although Sernova believes the expectations expressed in such forward-looking statements are based on reasonable assumptions, such statements are not guarantees of future performance and actual results may differ materially from those in forward looking statements. Forward-looking statements, which include our beliefs about the functionality of the Cell Pouch and our expectations of the potential benefits of the consortium and the Horizon 2020 hemophilia project including but not limited to projected timelines and potential for entry into human clinical trials, are based on the beliefs, estimates and opinions of Sernovas management on the date such statements were made. Furthermore, there are no assurances that there will be a clinical evaluation of a product developed from the consortium. Sernova expressly disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events or otherwise.

Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

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Sernova-HemAcure Consortium Announce Significant Progress in Development of 'First in World' Regenerative ... - GlobeNewswire (press release)

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The treatment is for an uncommon type of leukemia, called B-cell acute lymphoblastic leukemia, that affects mainly children and young adults, according to the Times. The success rate of the treatment that was seen in a recent clinical trial was "astonishing," said Lee Greenberger, chief scientific officer of the Leukemia and Lymphoma Society (LLS). Greenberger was not involved directly in the research of the new therapy, but the LLS has contributed significant funding toward the work.

Leukemia is cancer of white blood cells, and it starts in the bone marrow, the soft material found in the center of bones that produces blood cells. [11 Surprising Facts About the Immune System]

Simply put, the new treatment works by rewiring a person's own immune cells to fight cancer. To do this, doctors first remove millions of the immune cells, called T cells, from a patient's blood, Greenberger told Live Science. Normally, T cells help destroy infected or cancerous cells.

These T cells are sent to a lab to be purified, and then are genetically engineered, Greenberger said. Scientists mix the cells with a virus that works as "vector" to insert a bit of genetic material into the cells' DNA. (Viruses commonly insert their DNA into living cells.) In this case, the vector that's used is an inactive form of HIV. After 15 to 25 days during which the cells have started to produce the new protein that is encoded by the DNA, as well as grow and multiply the "gene-altered" T cells are infused back into the patient.

"It's basically a one-time therapy," Greenberger said.

The genetic material that the virus inserted into the T cells makes the cells do two things when they are put back into the patient's body, Greenberger said. First, the T cells produce an antibody that sits on the cell's surface, he said. This antibody enables the T cells to recognize the cancer cells. Also, the new genetic material activates the T cells so that, when they arrive at the tumor cells, they not only recognize them but also latch on and destroy them. [10 Do's and Don'ts to Reduce Your Risk of Cancer]

Once in the body, these "hunt and destroy" T cells multiply, so the patient ends up with an "army" of them in the blood, Greenberger said.

The entire process of the T cells killing the cancer cells lasts a couple of weeks, Greenberger said. But when the treatment is working, it can cause some serious side effects, he noted. The side effects include a condition called cytokine release syndrome, which can cause fevers, and another condition called neurotoxicity, which can cause symptoms such as disorientation and an inability to speak.

These side effects start as the T cells begin to kill the tumor cells, and when the tumor cells are depleted, the symptoms calm down, Greenberger said. During the entire process, however, the patient stays in the hospital and is monitored very carefully. In some cases, patients need intensive care.

In addition, some normal, noncancerous cells also carry the protein that the T cells are engineered to recognize. That means that the T cells will also kill these healthy B cells. "But people will survive without those [B cells]," Greenberger said. They do, however, need to get regular infusions of "immune globulins," which help to boost the immune system.

The FDA panel's recommendation was based on the results of a clinical trial run by the drug company Novartis, The New York Times reported. In the trial, 63 patients were given the new therapy, and 52 of them, or 83 percent, went into remission meaning the cancer went away. The other 11 patients died.

Greenberger noted that the treatment is being recommended for leukemia patients who have no other options left. And based on the results of the research, "it works," he said. The FDA panel recommended that it be approved specifically for patients whose cancer has not responded to other treatments or whose cancer returned after treatment.

So far, the patients who were successfully treated with the new therapy sometime between April 2015 and August 2016 haven't had their cancer return and haven't developed any serious side effects, Greenberger said. Still, they'll need to be monitored over the long term to see if anything changes. Novartis plans on monitoring the patients for 15 years.

Originally published on Live Science.

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A New Gene-Altering Cancer Treatment Shows 'Astonishing' Rate of Success - Seeker

July 21, 2017 Credit: CC0 Public Domain

A study by scientists at the Children's Medical Center Research Institute at UT Southwestern (CRI) is providing insight into the genetic basis of neuropsychiatric disorders. In this research, the first mouse model of a mutation in the arid1b gene was created and then used to show that growth hormone treatments reverse some manifestations of the mutation.

The ARID1B gene is one of the most commonly mutated genes in patients with intellectual disability and autism spectrum disorders, but scientists have not yet discerned if and how defects in the ARID1B gene contribute to these clinical manifestations. To understand how reduced levels of the protein product of the gene might cause these disorders, a team of researchers led by Dr. Hao Zhu and including graduate student Cemre Celen genetically modified mice to carry a mutation in one of two copies of the ARID1B gene. This mutation replicates the genetics of Coffin-Siris syndrome, a disorder that some patients with defects in the ARID1B gene have that is characterized by speech and social development problems, intellectual disability, and delayed physical growth.

The hope is that by understanding the molecular basis of Coffin-Siris syndrome, scientists will gain a deeper understanding of more common diseases involving intellectual and social impairment.

Scientists found mice with the mutated ARID1B gene exhibited the same type of physical and social changes seen in children with Coffin-Siris syndrome, such as abnormal brain development, muscle weakness, and increased anxiety and fear. The mice also displayed features consistent with autism spectrum disorder, such as social interaction abnormalities, repetitive behaviors, and abnormal "squeaks" or vocalizations. Further testing found these mice had lower-than-expected growth hormone and insulin-like growth factor (IGF1) levels in the blood, potentially explaining the small stature and delayed development seen in human patients. Treating mutant mice with growth hormones restored body size and muscle function, but did not significantly change the behaviors associated with the syndrome.

"These results suggest that growth hormone treatment could be a useful therapy for ARID1B patients. This is an interesting finding because we know some pediatricians already treat Coffin-Siris patients with growth hormones, although they were unaware that this response might be common to many people with ARID1B mutations," said Dr. Zhu, an Assistant Professor at CRI with joint appointments in Internal Medicine and Pediatrics at UT Southwestern Medical Center and a CPRIT Scholar in Cancer Research.

Dr. Zhu said he believes the study provides the scientific community with an important animal model to further investigate ARID1B's role in human brain disorders and will be a useful tool for therapeutic testing of potential treatments for autism, intellectual disability, and Coffin-Siris syndrome.

Explore further: Mice provide insight into genetics of autism spectrum disorders

More information: Cemre Celen et al. Arid1b haploinsufficient mice reveal neuropsychiatric phenotypes and reversible causes of growth impairment, eLife (2017). DOI: 10.7554/eLife.25730

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Scientists provide insight into genetic basis of neuropsychiatric disorders - Medical Xpress