Category Archives: FDA Stem Cell Trials

Maybe youve already dumped chilled water atop your head and encouraged friends and loved ones to do likewise by taking the ALS Ice Bucket Challenge. Or, perhaps inspired by seeing the innumerable videos of people doing the Ice Bucket Challenge, youve made a donation to the cause, thereby contributing to the unprecedented tens of millions of dollars that this grassroots initiative has raised. Regardless, its likely youve heard more about amyotrophic lateral sclerosis aka Lou Gehrigs disease in the past month than the world had in the 75 years combined since the Yankees Hall of Famer made his legendary luckiest man on the face of the earth retirement speech. Of course, we here at The Post are all too familiar with ALS. Our colleague, former award-winning courts reporter and best-selling author Susan Spencer-Wendel, succumbed at age 47 to the neurodegenerative condition earlier this summer, three years after being diagnosed. What is ALS? ALS is a progressive, invariably fatal disease that disables the bodys motor neurons which are specialized nerve cells in the brain and spine. Experts believe that, in 90 percent of ALS cases, the cause is sporadic meaning there is no identifiable origin. Though the symptoms are initially subtle … Continue reading →

By: Steve Bauer, Ph.D. One of FDAs primary missions is to make sure that the products we approve are safe and effective. There is tremendous interest in the development of regenerative medicine, including numerous proposed products that rely on stem cells. Stem cells have the ability to generate more stem cells or to turn into more mature cell types such as nerve- or bone-producing cells. These properties make stem cells potentially well suited for use in regenerative medicine. They might be used in repairing heart, nerve, and brain damage or in treating diabetes and other diseases by repairing or replacing cells and tissues. Steve Bauer, Ph.D., chief of the Cellular and Tissues Therapy Branch, Division of Cellular and Gene Therapies, in the Office of Cellular, Tissue and Gene Therapy at CBER. Because stem cells can change based on their surroundings, whether during growth outside of the body or following injection into the body, ensuring the safety of effective regenerative medicine products can be challenging. One type of adult stem cell, the multipotent marrow stromal cell (MSC) more popularly called the mesenchymal stem cell is the subject of a great deal of research in regenerative medicine. These cells can divide repeatedly, … Continue reading →

US FDA researchers are developing tools to help cell therapy developers improve how they manufacture stem cells in a new lab consortium unveiled by the agency this week. In a blog post this week Food and Drug Administration (FDA) cellular and tissue therapy chief, Steve Bauer, outlined the potential of mesenchymal stem cell, the scientific hurdles the developers and regulators face and the agencys efforts to address them. Stem cells have the ability to generate more stem cells or to turn into more mature cell types such as nerve- or bone-producing cells. These properties make stem cells potentially well suited for use in regenerative medicine. But there are still scientific questions to answer about MSCs. A particularly important set of questions is how the manufacturing of these cells outside of the body could affect their potential healing properties and their safety. Research focus To address these scientific questions the CBER has formed a seven-strong laboratory consortium to create tests and techniques to help developers improve their stem cell manufacturing processes, using cells from eight different donors. Work ranges from the identification of 84 cell surface proteins that manufacturers can use to track the growth and differentiation of stem cells in … Continue reading →

What the heck happened to the FDA when it comes to regulating stem cell clinics? Has it given up? Or does it just seem that way because it moves in slow motion? The FDA might as well have thrown in the towelbecause, as I like to say, the stem cell field moves in dog years. Often times 7 years worth of stem cell happenings take place in just onehuman calendar year. An example of this warp speed is the approximate doubling of the number of stem cell clinics in the US in the last year or so. There was a time when I was perhaps naive enough thatI thought that regulatory agencies legally tasked with oversight of biologics would take swift action against violators, particularly if the products or procedures in question were not proven to be safe or effective. Patients are at serious risk. Throw in some evidence of predatory behavior and dubious public claims and lack of training at some clinics, and the FDA in the US, for example, surely would do something about it as thousands of patients get these dubious interventions. Right? Im no so sure anymore. I still believe in appropriate regulation of stem cell-based … Continue reading →

Albany Molecular Research Inc. (AMRI: Quote), which reported better-than expected Q2 results, has also upped its full year contract revenue guidance, sending the stock up by more than 7 percent. The company now expects full year contract revenue to range between $275 million and $283 million, up from its prior outlook of $243 million to $253 million. AMRI closed Tuesday's trading at $20.29, up 7.98%. Cytori Therapeutics (CYTX: Quote) plummeted in after-hours trading Tuesday following the company's decision to halt its cell therapy heart failure trials. According to the company, the multi-center, prospective, randomized, double-blind PMA/IDE safety and feasibility (phase I/II) trials investigating adipose-derived stem and regenerative cells in patients who suffer from a severe form of refractory (untreatable) heart failure due to chronic myocardial ischemia, dubbed ATHENA and ATHENA II, were placed on hold based on a safety review of reported cerebrovascular events. CYTX closed Tuesday's trading at $2.10, down 0.47%. In after-hours, the stock was down 22.38% to $1.63. Globus Medical Inc. (GMED: Quote) plunged 19 percent in extended trading on Tuesday as investors were disappointed with downward revision of the company's full-year revenue outlook. The company now expects full year 2014 net sales to be in the … Continue reading →

By RTT News, August 01, 2014, 12:32:00 AM EDT (RTTNews.com) - Alexza Pharmaceuticals Inc. ( ALXA ) is all set to initiate a phase 2a study of AZ-002 for the management of patients with acute repetitive seizures in the second half of 2014. The company has also identified two product candidates - AZ-008 for the acute treatment of restless legs syndrome (RLS) and AZ-009 for hypomobility, or freezing, during "off periods" in Parkinson's disease patients, which will be taken up for pre-clinical testing. ALXA closed Thursday's trading at $4.83, down 2.23%. BioTime Inc.'s ( BTX ) subsidiary OncoCyte Corp. has expanded the clinical development of its urine-based bladder cancer diagnostic test PanC-Dx by initiating a multi-site clinical trial. The trial is expected to begin enrolling patients within the next week and the goal of the trial is to compare the performance of OncoCyte's proprietary PanC-Dx bladder cancer markers to the performance of cystoscopy. OncoCyte is initially focusing its efforts on developing PanC-Dx diagnostic products for use in detecting breast, bladder, and lung cancers. BTX closed Thursday's trading at $2.54, down 3.79%. Cerus Corp. ( CERS ) expects FDA decision on the premarket approval application for INTERCEPT Platelets as early as … Continue reading →

Contact Information Available for logged-in reporters only Newswise July 29, 2014 Advertising claims for cosmetic procedures using stem cells are running far ahead of the scientific evidence for safety and effectiveness, according to a review in the August issue of Plastic and Reconstructive Surgery, the official medical journal of the American Society of Plastic Surgeons (ASPS). "Stem cells offer tremendous potential, but the marketplace is saturated with unsubstantiated and sometimes fraudulent claims that may place patients at risk," write Dr Michael T. Longaker of Stanford University Medical Center and colleagues. 'Worrying advertisements' for cosmetic stem cell procedures Dr Longaker and coauthors raise concerns about the unregulated use of stem cells for unproven indicationsincluding cosmetic procedures. While stem cell therapy "remains in its infancy," they write, "there are a growing number of cosmetic practitioners that are advertising minimally invasive, stem cell-based rejuvenation procedures." The article was prompted by "worrying advertisements" claiming benefits of stem cell procedures for facelifts, breast augmentationeven "stem cell vaginal rejuvenation." These ads claim benefits from procedures that have not undergone rigorous scientific evaluationincluding potential risks related to stem cell and tissue processing and the effects of aging on stem cells. To gain insight into these claims, Dr … Continue reading →

Verastem (NASDAQ: VSTM) is having its annual research and development day in a few weeks, on July 10. Call it a catalyst to announce catalysts. We might even get some new data from the cancer-stem-cell expert. Verastem's lead drug, VS-6063, targets a protein called focal adhesion kinase, or FAK, which is involved in cell survival, among other things. The biotech also has a backup compound, VS-4718 that targets FAK and a third drug that targets the PI3K/mTOR signaling pathway, which is also involved cell survival. Both pathways are up regulated in cancer cells, making them good potential targets. Verastem's drugs preferentially target cancer stem cells, which are believed to be the root of reoccurrence of cancer. Cancer treatments kill a majority of the cancerous cells, shrinking the tumors, but the cancer stem cells often survive, thereby regenerating the primary tumor. The formation of metastases -- tumors at secondary sites -- may be initiated by these cancer stem cells. When in 2015? Without a doubt, Verastem's biggest catalyst is a clinical trial called Command, testing VS-6063 in patients with mesothelioma, a form of lung cancer. The 350 to 400 patients expected to enroll in the trial have already responded to a … Continue reading →

SOUTH BEND, Ind.--- In the 1970's, researchers discovered that a newborn's umbilical cord blood contained special stem cells that could help fight certain diseases. More than 30 years later doctors are still experimenting and learning more about the use of cord blood. Amanda Canale doesn't take time with her daughter and niece for granted. She's just happy to feel good. "I've been in the hospital, and I've been sick my whole life," said Amanda. Amanda was born with a rare blood disorder that required daily shots. "Basically, I have no white blood cells. I have no immune system at all," said Amanda At 23 she developed Leukemia and was given two weeks to live. She desperately needed a Bone Marrow Transplant, but family members weren't matches. Her doctor suggested an Umbilical Cord Blood Transplant. See more here: Umbilical cord blood helps to save lives … Continue reading →

(Reuters) - Geron Corp said the U.S. Food and Drug Administration had lifted a partial clinical hold on a study testing its sole drug as a treatment for myelofibrosis, a rare form of blood cancer, sending the company's shares soaring in early trading. Enrollment in the early-stage trial, sponsored by the Mayo Clinic, was halted in March over concerns about liver toxicity pending followup data from Mayo's investigator on the possible reversibility of the liver damage. Enrollment for the study ceased in January, and about 20 of the 79 patients dropped out. Geron, whose shares rose as much as 29 percent in early trading on Thursday, did not then disclose the reason behind the dropouts, but said the remaining enrolled patients would continue to receive the drug, imetelstat. However, in March the FDA also imposed a halt on separate company-sponsored trials evaluating the use of the drug in thrombocythemia and multiple myeloma, citing similar concerns. Geron said on Thursday the company-sponsored trials remain suspended. Stifel Nicolaus & Co analyst Brian Klein said that while the FDA's lift on the partial hold was positive, it was unlikely that the Mayo Clinic would enroll new patients. As the partial hold had not … Continue reading →