Category Archives: FDA Stem Cell Trials

The National Multiple Sclerosis Society (NMSS), by far the largest MS nonprofit organization in the US, has three times rejected grant applications from the Tisch Multiple Sclerosis Research Center of New York, which were submitted in an effort to procure funding for what is now the only FDA approved regenerative stem cell trial being conducted on MS patients in the nation. The trial in question uses a very sophisticated approach to this experimental therapy, arrived at after over a decade of development in the laboratory, in an attempt to repair nervous system tissues damaged by multiple sclerosis (click here). The failure of the NMSS to help fund this trial is, at the very least, extremely disappointing, and should be of great concern to all those who support the organization. The goal of nervous system repair and regeneration has long been a Holy Grail of MS research, and investigations into using stem cells to achieve this end hold terrific potential. MS patients around the world are eager to see stem cell research accelerated, as dissatisfaction with current treatment paradigms runs rampant in many segments of the MS population. Patients with the progressive subtypes of the disease are especially desperate for innovative … Continue reading →

No, that skin cream cannot reverse aging. It cant repair DNA damage and it cant cure rosacea. RunPhoto / Getty Images The FDA's taken on five cosmetic companies in the past five months, telling them to stop makingmedical claims for wrinkle creams. One product at a time, the Food and Drug Administration is taking on the beauty industry and some of the over-the-top claims being made for some of the products. Five warnings have gone out since November. The latest warning letter went out to Strivectin, whose wrinkle creams are sold at retailers that range from Costco to Nordstrom. The language FDA objects to? The claims on your website indicate that the products are intended to affect the structure or any function of the human body, rendering them drugs under the (Federal Food, Drug, and Cosmetic) Act, FDA says in a warning letter sent to the company. And when Strivectin says its Advanced Tightening Neck Cream, can restore the elastin fiber architecture, providing noticeable lift and improving resistance to gravity, its actually claiming that product is a drug and its one that hasnt been through the FDAs review process, the agency said in its letter. In response, Strivectin has changed … Continue reading →

No, that skin cream cannot reverse aging. It cant repair DNA damage and it cant cure rosacea. RunPhoto / Getty Images The FDA's taken on five cosmetic companies in the past five months, telling them to stop makingmedical claims for wrinkle creams. One product at a time, the Food and Drug Administration is taking on the beauty industry and some of the over-the-top claims being made for some of the products. Five warnings have gone out since November. The latest warning letter went out to Strivectin, whose wrinkle creams are sold at retailers that range from Costco to Nordstrom. The language FDA objects to? The claims on your website indicate that the products are intended to affect the structure or any function of the human body, rendering them drugs under the (Federal Food, Drug, and Cosmetic) Act, FDA says in a warning letter sent to the company. And when Strivectin says its Advanced Tightening Neck Cream, can restore the elastin fiber architecture, providing noticeable lift and improving resistance to gravity, its actually claiming that product is a drug and its one that hasnt been through the FDAs review process, the agency said in its letter. In response, Strivectin has changed … Continue reading →

NEW YORK, Aug. 14, 2013 /PRNewswire/ --The Tisch MS Research Center of New York announced today that it has received Investigational New Drug (IND) approval from the Food and Drug Administration (FDA) to commence a Phase 1 trial using autologous neural stem cells in the treatment of multiple sclerosis (MS). MS is a chronic human autoimmune disease of the central nervous system that leads to myelin damage and neurodegeneration and affects approximately 2.1 million people worldwide. "To my knowledge, this is the first FDA-approved stem cell trial in the United States to investigate direct injection of stem cells into the cerebrospinal fluid of MS patients, and represents an exciting advance in MS research and treatment," said Dr. Saud A. Sadiq, Senior Research Scientist at Tisch MS Research Center of New York and the study's principal investigator. The groundbreaking study will investigate a regenerative strategy using stem cells harvested from the patient's own bone marrow. These stem cells will be injected intrathecally (into the cerebrospinal fluid surrounding the spinal cord) in 20 participants who meet the inclusion criteria for the trial. This will be an open label safety and tolerability study. All study activities will be conducted at the Tisch MS … Continue reading →

By Anna Rose Welch, associate editor What FDA decisions have you missed in the last week? Bristol-Myers Squibb Immunotherapy Gets Green Light Bristol-Myers Squibb beat Merck in the race to approval for its immunotherapy targeting non-small cell lung cancer (NSCLC). In December 2014, Opdivo (nivolumab) was approved to treat melanoma, following in the footsteps of Mercks Keytruda. However, three months ahead of the expected action date, the FDA approved Opdivo to treat those with NSCLC who saw their disease progress following platinum-based chemotherapy. In a Phase 3 study, the PD-1 inhibitor proved its ability to extend survival compared to docetaxel. Analysts expect the drug to bring in upwards of $1.7 billion next year, with the promise of earning $7.3 billion by 2020. Bayer BLA Accepted For Review Bayers BLA for its hemophilia A candidate, BAY 81-8973 (Kovaltry), has been accepted for review by the FDA. The recombinant Factor VIII compound has been investigated in children and adults in the LEOPOLD clinical trial program. Patients enrolled in the trial were treated with the drug two and three times a week in order to determine the drugs pharmacokinetics, efficacy, and safety. FDA To Review Actelion NDA Actelion expects to receive an answer … Continue reading →

SILVER SPRING, Md. and RESEARCH TRIANGLE PARK, N.C., March 10, 2015 /PRNewswire/ --United Therapeutics Corporation (NASDAQ: UTHR) announced today that the United States Food and Drug Administration (FDA) has approved Unituxin (dinutuximab) Injection (formerly called ch14.18), in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2), and 13-cis-retinoic acid (RA), for the treatment of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to prior first-line multiagent, multimodality therapy.Neuroblastoma is the most common extracranial solid cancer in childhood and the most common cancer in infancy, with an annual incidence in the United States of approximately 700 patients, of whom 50% are diagnosed as having high-risk disease. Unituxin is a chimeric biologic antibody that induces cell lysis of GD2-expressing cells through antibody-dependent cell-mediated cytoxicity (ADCC) and complement-dependent cytoxicity (CDC) and is part of an immunotherapeutic regimen to treat pediatric high-risk neuroblastoma. The approval was based on demonstration of improved event-free survival (EFS) and overall survival (OS) in a multicenter, open-label, randomized trial (ANBL0032) conducted by the Children's Oncology Group (COG). The trial randomized (1:1) 226 patients to either the Unituxin/13-cis-retinoic acid (RA) arm or the RA alone arm. Patients in each arm received six cycles of treatment. The Unituxin/RA … Continue reading →

Volume 10, Issue 1 Dr. Andre Terzic explains how heart stem cells were developed. Christian Homsy, M.D., president of Cardio3, shares his perspective on the heart stem cell clinical trial. The Food and Drug Administration (FDA) has cleared the way for the Belgian company and Mayo Clinic collaborator Cardio3 BioSciences to launch a phase III clinical trial of its stem cell therapy based on Mayo Clinic regenerative medicine research. The heart stem cell clinical trial will test the use of cardiopoietic (cardiogenically instructed) stem cells designed to improve heart health in people with heart failure. These modified stem cells, which researchers describe as cardio-smart cells, have been produced according to the technique developed by Andre Terzic, M.D., Ph.D., director of the Mayo Clinic Center for Regenerative Medicine in Rochester, Minn., and his colleagues. The multisite U.S. trial, called Chronic Heart Failure Analysis and Registry in the Tohoku District 2 (CHART-2 Study), hopes to recruit 240 patients with chronic advanced symptomatic heart failure. Cardio3 BioSciences is a bioscience company based in Mont-Saint-Guibert, Belgium. Previous trials with the heart stem cells were held in Belgium, Serbia and Switzerland. A version of the phase III trial is now beginning in Europe. . See … Continue reading →

When I started blogging in 2010 the stem cell arena was a very different place. Back then the hot topic was the battle over the legality of federal funding of embryonic stem cell research. That battle is over, or at least in hibernation, with a2013 federal court rulingallowing such funding to continue. The stem cell debate of today, which in its own way is just as fierce as the old one, is focused on how best to regulate the clinical translation and commercialization of innovative stem cell technologies. The stakes in this new stem cell battle on the regulatory front are very high both for the stem cell field and for patients. Too little regulation could lead to harm to patients and damage to the stem cell field at a crucial juncture in its history, while too much regulation could stifle stem cell and regenerative medicine innovations. The goal of stem cell advocates, including myself, is to find a regulatory sweet spot where science-based, innovative stem cell medicine can advance expeditiously. On the other side we have largely physicians and lawyers along with some patients arguing for drastically-reduced regulation and acceleration of for-profit stem cell interventions to patients, even without … Continue reading →

Published 19 February 2015 Seattle Genetics has submitted a supplemental biologics license application (BLA) to the US Food and Drug Administration (FDA) based on data from Phase III Aethera trial of Adcetris (Brentuximab Vedotin) in post-transplant hodgkin lymphoma (HL) patients at high risk of relapse. Adcetris is an antibody-drug conjugate (ADC) directed to CD30, which is expressed in classical HL and systemic anaplastic large cell lymphoma (sALCL). The Phase III trial of Adcetris was carried out as consolidation therapy immediately following an autologous stem cell transplant (ASCT) in HL patients. The trial, which included 329 HL patients, achieved its primary endpoint and showed a significant increase in progression-free survival (PFS) per independent review facility, with a hazard ratio of 0.57 and a p-value of 0.001. Seattle Genetics president and chief executive officer Clay Siegall said: "With approximately half of all Hodgkin lymphoma patients who undergo an autologous stem cell transplant experiencing disease relapse, there is a significant need to identify regimens that extend progression-free survival. "Results from the Aethera trial demonstrated that treating high risk Hodgkin lymphoma patients with Adcetris in following autologous stem cell transplant resulted in a statistically significant improvement in progression-free survival with a manageable safety profile." … Continue reading →

PBR Staff Writer Published 19 February 2015 The US Food and Drug Administration (FDA) has expanded the existing indication for Celgene's Revlimid (lenalidomide) in combination with dexamethasone to include patients newly diagnosed with multiple myeloma (NDMM). In June 2006, Revlimid plus dexamethasone was approved for use in multiple myeloma patients who have received at least one prior therapy. The approval of Revlimid was based on safety and efficacy data from Phase III trials, including the FIRST trial (MM-020/IFM 07-01), The randomized, open-label, three-arm FIRST trial evaluated continuous Revlimid in combination with dexamethasone (Rd Continuous) until disease progression versus melphalan, prednisone and thalidomide (MPT) for 18 months as the primary analysis, and a fixed duration of 18 cycles of Rd (Rd18) as a secondary analysis, in 1,623 newly diagnosed patients who were not candidates for stem cell transplant. Median progression-free survival (PFS), which is the length of time a patient lives from study randomization to disease progression or death, was the primary endpoint of the study. Currently, the company has an application under review with the European Medicines Agency (EMA) for approval to use Revlimid to treat adult patients with previously untreated multiple myeloma who are not eligible for transplant. In … Continue reading →