Stem cells and stroke The past decade has seena rise in the number of stem cell-derived therapies targeting ischemic stroke in preclinical and early clinical studies. Corroborated by numerous scientific reports, the therapeutic benefits of stem cells include an extension of the time window for drug intervention, improvement of neurological deficits, reduction of infarct volume, pro-regenerative cerebral reorganization, mitigation of poststroke neuro-inflammation, and tissue restoration, all of which depend on the time after infarct, cell type used, and route of administration13. The wide range of effects observed for stem cell therapies demonstrates that functional recovery after stroke occurs via multiple mechanisms rather than a single target46. Research indicates that the mode of action may depend on the stem cell type and other key factors, including infarct size and location, mode of intervention, and timing poststroke68. Thus, some understanding of the cellular, molecular, and biochemical events that are involved in the mode of action of a stem cell type is a prerequisite to improving and optimizing its therapeutic benefits. Our 2012 review of cell therapy in stroke showed the wide variety of cell types used preclinically and clinically in stroke treatment research1. Mesenchymal stromal cells (MSCs) of multiple origins and phenotypes … Continue reading →

An FDA greenlight for sickle cell disease therapy Endari has catapulted its developer, Emmaus Medical, into the spotlight, but commercializing the new drug could be challenging. The approval means the privately-held biotech has ended a 20-year drought in new therapies for sickle cell disease (SCD), an inherited disease in which red blood cells are abnormally shaped and can't deliver oxygen as effectively to body tissues. Prior to this, the only other drug approved for the disease in the U.S. was hydroxyurea. Most of the 100,000 people in the U.S. with SCD are covered by Medicare and Medicaid, so the company will need to pitch the price of the drug at a level that will ensure access to patients is not obstructed by payer resistance. Its job is made harder by the fact that the active ingredient is already widely used as a dietary supplement. For now, the Californiabiotech, led by Yutaka Niihara M.D., is playing its cards pretty close to its chest. It said it wasplanning a launch in the fourth quarter of the year, but isn't showing its hand on pricing just yet, limiting itself to a statement that it will price Endari at a "reasonable" level to ensure … Continue reading →

The U.S. Food and Drug Administration will ask a panel of advisors to focus on the safety of Novartis AG's experimental gene therapy drug when it meets to review the product on Wednesday. The keenly anticipated preliminary review of the leukemia treatment, posted on the FDA's website on Monday, comes two days ahead of the advisory panel meeting, which will discuss the drug and vote on whether the benefits exceed the risks. If approved, the drug, tisagenlecleucel, would be the first gene therapy to be approved in the United States. The FDA is not obliged to follow the recommendations of its advisors but typically does so. The panel's decision could have significant implications not only for Novartis but for companies making similar drugs, including Kite Pharma Inc. Juno Therapeutics Inc and bluebird bio Inc. The drugs use a new technology known as CAR-T, or chimeric antigen receptor T-cell therapy, which harnesses the body's own immune cells to recognize and attack malignant cells. If approved they are expected to cost up to $500,000 and generate billions of dollars for their developers. Success would also help advance a cancer-fighting technique that scientists have been trying to perfect for decades. Novartis is applying … Continue reading →

Advertising forstem cell therapies not supported by clinical researchoftenmadedirectly to patients and sometimes promoted as a cure for diseases like multiple sclerosis or Parkinsons is a growing problem that needs to be addressed and regulated, a team of leading experts say, calling suchstem cell tourism potentially unsafe. Stem cell tourism is the unflattering name given to the practice of encouragingpatients totravel outside their home country to undergo suchtreatment, typicaly at a private clinic. The article, titledMarketing of unproven stem cellbased interventions: A call to actionandrecently published inthe journal Science Translational Medicine, was co-authored by scientistswith universities and hospitals in the U.S., Canada, U.K., Belgium, Italy, Japan, and Australia. It focuses on the global problem of thecommercial promotion of stem cell therapies and ongoing resistance to regulatory efforts. Its authors suggest that a coordinated approach, at national and international levels, be focused on engagement, harmonization, and enforcement in order to reduce risks associated with direct-to-consumer marketing of unproven stem cell treatments. Treatments involving stem cell transplants are now being offered by hundreds of medical institutions worldwide, claiming efficacy in repairing tissue damaged by degenerative disorders like MS, even thoughthose claim often lack or are supported bylittle evidence . They alsonoted that … Continue reading →