CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Verastem, Inc., (VSTM) focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, announced that lead cancer stem cell inhibitor, VS-6063 (defactinib), has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for use in the treatment of mesothelioma, a rare form of lung cancer. The designation is designed to encourage the development of drugs which may provide significant benefit to patients suffering from rare diseases. Mesothelioma is among the most aggressive and lethal cancers but has limited treatment options, said Robert Forrester, Verastem President and Chief Executive Officer. We are pleased that the FDA recognizes the significant unmet medical need in mesothelioma. We previously received orphan medicinal product status for defactinib in Europe and these two designations are an important component of our development strategy. Verastem recently outlined details of the registration-directed clinical study of defactinib in patients with malignant pleural mesothelioma. This study is designed as a double-blind, placebo-controlled trial with an expected enrollment of approximately 350-400 patients at clinical sites in 11 countries. We are in discussions with the regulatory agencies and clinical investigators worldwide, said Dr. Joanna Horobin, Verastem Chief Medical Officer. We … Continue reading →

The project involves researching, manufacturing and transplanting adult, human stem cells Certain stem cell-based therapies could be available to Irish patients within a couple of years, as part of a new 2.5m research partnership between the Irish Blood Transfusion Service and research centres in Galway, Dublin and Cork. The project will involve the researching, manufacturing and for the first time transplanting of adult human stem cells for the treatment of arthritis, burns and diabetic foot ulcers. For decades, blood transfusions have been used as a basic, temporary form of cell therapy. But time and technology has moved on and now the targeted delivery of cells to injured or diseased tissue is seen as a promising future method of treating a whole variety of illnesses and conditions. As part of its objective to be involved in research, the Irish Blood Transfusion Service (IBTS) put out a call for ideas for research into cell-based therapies. The winning proposal came from the Regenerative Medicine Institute (REMEDI) in NUI Galway, supported by the National Adult Stem Cell Transplant Centre in St James's Hospital and the Centre for Research in Vascular Biology in University College Cork. The IBTS will provide 2.5m over the next five … Continue reading →

The three blind mice might get the chance to see because of groundbreaking stem cell research being done in Britain. Scientists in the UK took stem cells from mice embryos, put them in a petri dish and coaxed them into becoming photoreceptors, the cells in the retina that catch light. After collecting 200,000 of the stem cells turned photoreceptors the scientists then injected the cells into the eyes of blind mice, and some of the cells integrated into the host retina and restored sight. The rodents were then run through a maze and examined by optometry to confirm that they did indeed respond to light. Shutterstock The groundbreaking work was published by Britain's Medical Research Council and in the science journal "Nature Biotechnology." While the work is still years away from helping humans, it is an extremely promising start to curing blindness caused by photoreceptor loss like retinitis pigmentosa and age-related macular degeneration. The biggest breakthrough that the team in England was able to achieve was turning the finicky stem cells into stable photoreceptors, instead of deadly cancer cells. Additionally, researchers in the past could turn stem cells into rod cells which are a component of the complex network of … Continue reading →

Featured Article Academic Journal Main Category: Epilepsy Also Included In: Neurology / Neuroscience;Psychology / Psychiatry Article Date: 23 Jul 2013 - 0:00 PDT Current ratings for: Early death 11 times more likely with epilepsy The study was carried out by researchers from the University of Oxford and University College London in the UK, who analyzed 69,995 patients in Sweden suffering from epilepsy over a 41-year period. The researchers looked at the risks and causes of premature mortality in people with epilepsy, including suicide, accidents and assaults. These circumstances were assessed and compared with 660,869 age-matched and sex-matched individuals without epilepsy from the general population. In addition, 81,396 healthy siblings of the epilepsy sufferers were assessed for the influence of genetic or early environmental risk factors. Throughout the study, 6,155 (9%) people with epilepsy died compared with 4,892 (0.7%) of the general population who do not suffer from the disorder. The most common cause of death in epilepsy sufferers was found to be what the researchers call "external causes", with 15.8% dying from suicides, accidents and assaults. Three-quarters of these 15.8% had been diagnosed with a mental disorder, and substance misuse and depression were the biggest mental health risk factors. Seena … Continue reading →

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WASHINGTON (AFNS) -- A congressionally mandated pilot program set to launch July 25 will enhance an existing Defense Department program that provides care and treatment for military children with autism, a senior DOD official said. Dr. Jonathan Woodson, assistant secretary of defense for health affairs and director of the TRICARE Management Activity met with reporters yesterday to explain the new program. An estimated 8,500 children of active-duty military families have a form of autism, Woodson said. He sought to dispel military parents' concerns about rumors of a potential loss in benefits for their children with autism and autism spectrum disorder. "We understand that there's a lot of anxiety in the community of interest around autism about suspected changes that would adversely affect care," he said. "Providing care to children who have autism spectrum disorder and making sure they get the full range of care they need is a priority to us." "All care will be continued," Woodson added, noting that active-duty service members' children's autism care benefits in the applied behavior analysis administered through TRICARE would not change. "Anyone who's receiving care under the (Enhanced Access to Autism Services Demonstration) -- there will be no change," he said. There's also … Continue reading →

Online New program helps more than 250 autistic children, but more than 18,000 need services in Utah. A Utah pilot program offers free autism treatment for more than 250 children, but it only helps kids who hit the jackpot in a state lottery. With more than 18,000 Utah children with autism, the Beehive State needs insurance reform to require coverage of such treatment for more children, said Jon Owen, the Utah Autism Coalitions new president, during Tuesdays live online forum Trib Talk, offered by The Salt Lake Tribune. About the pilot programs Thirty-five states require health insurance companies to cover autism services. Utahs autism community has been pushing for a similar mandate, but instead got HB272, which created three pilot programs now underway. The Medicaid pilot will serve up to 300 children through June 2014. Utah Department of Health is managing an Autism Treatment Account, which supported care for 29 kids in June with money from the state, Zions Bank and Intermountain Healthcare. The Public Employees Benefit and Insurance Program will cover up to 50 children whose parents work for state or local government. Administrators will be tracking the cost and effectiveness of the treatments and report their results by … Continue reading →

Newswise More than 300 parents, healthcare providers and educators from across the U.S. and around the world, will come together July 26 through July 27, 2013 at the second annual national conference for families and professionals, Treating the Whole Person with Autism: Care Across the Lifespan. The conference sponsored by Autism Speaks, the worlds leading autism and advocacy organization, with our educational partners at Nationwide Childrens Hospital, Ohio State University Wexner Medical Center, the U.S. Health Resources and Services Administration and the American Academy of Pediatrics will be held at The Hilton Columbus at Easton in Columbus, Ohio. The program includes scientific sessions and hands-on workshops featuring new approaches to providing a lifetime of supports and services for individuals with autism spectrum disorder (ASD), whether self-directed by individuals with ASD or provided by their families to help children, adolescents and adults with autism lead healthy and successful lives. This conference is a hallmark of the Autism Speaks Autism Treatment Network (AS-ATN), an initiative aimed at improving the health and well-being of individuals with ASD, remarked Daniel Coury, M.D., ATN medical director and chief of Developmental & Behavioral Pediatrics at Nationwide Children's Hospital. Utilizing a whole person and family-centered approach, this … Continue reading →

Public release date: 22-Jul-2013 [ | E-mail | Share ] Contact: Jane E. Rubinstein jrubinstein@rubenstein.com 212-843-8287 Autism Speaks COLUMBUS, O.H. (July 22, 2013) More than 300 parents, healthcare providers and educators from across the U.S. and around the world, will come together July 26 through July 27, 2013 at the second annual national conference for families and professionals, "Treating the Whole Person with Autism: Care Across the Lifespan." The conference sponsored by Autism Speaks, the world's leading autism and advocacy organization, with our educational partners at Nationwide Children's Hospital, Ohio State University Wexner Medical Center, the U.S. Health Resources and Services Administration and the American Academy of Pediatrics will be held at The Hilton Columbus at Easton in Columbus, Ohio. The program includes scientific sessions and hands-on workshops featuring new approaches to providing a lifetime of supports and services for individuals with autism spectrum disorder (ASD), whether self-directed by individuals with ASD or provided by their families to help children, adolescents and adults with autism lead healthy and successful lives. "This conference is a hallmark of the Autism Speaks' Autism Treatment Network (AS-ATN), an initiative aimed at improving the health and well-being of individuals with ASD," remarked Daniel Coury, M.D., … Continue reading →