Category Archives: FDA Stem Cell Trials

CPT welcomes original articles in the emerging areas of translational, predictive and personalized medicine; new therapeutic modalities including gene and cell therapies; pharmacogenomics, proteomics and metabolomics; bioinformation and applied systems biology complementing areas of pharmacokinetics and pharmacodynamics, human investigation and clinical trials, pharmacovigilance, pharmacoepidemiology, and population pharmacology. Contact the editorial office at for deadlines and further information. Listen here. This episode features Bernard Vrijens of WestRock Healthcare discussing quantifying the influence of adherence and dose individualization. Read his Commentary which published in the April issue here. FIND MORE CLINPHARMPOD EPISODES HERE This Annual Issue highlights the translation of molecular insights into novel management paradigms in pulmonary hypertension; inflammatory bowel disease; asthma, and viral infections, emerging nucleic acid-based technologies that are poised to transform human therapeutics. Plus, the evolution of fundamental clinical pharmacology platforms that optimize the efficiency of bench to bedside translation of therapeutic discoveries across the continuum of development, regulation, and utilization. Read these outstanding contributions in the January issue Enjoy FREE access to key collections of articles from CPT selected by the Editor-in-Chief. Read the virtual issues: CPIC Guidelines & Updates - from the Clinical Pharmacogenetics Implementation Consortium (CPIC) Clinical Trials Excerpt from: Clinical Pharmacology & Therapeutics - … Continue reading →

Neuralstem is seeking to treat the symptoms of ALS via transplantation of its NSI-566 human spinal cord stem cells (HSSCs) directly into the gray matter of the patients spinal cord. In ALS, motor neurons die, leading to paralysis. In preclinical animal work, Neuralstem cells both made synaptic contact with the host motor neurons and expressed neurotrophic growth factors, which are protective of cells. View published papers here: 1, 2, 3. Neuralstem initiated the first FDA-approved stem cell trial for ALS in January 2010, at Emory University. This Phase I safety trial, to evaluate the safety of the NSI-566 cells and surgical technique, was designed to enroll up to 18 patients. The Principal Investigator is Eva Feldman, MD, PhD, Director of the A. Alfred Taubman Medical Research Institute, and Director of Research of the ALS Clinic at the University of Michigan Health System. The Site Investigator is Jonathan Glass, MD, Professor of Neurology, Emory School of Medicine and Director of the Emory ALS Center. The trial was awarded an Orphan Drug Designation by the FDA in February 2011. In humans, Neuralstem expects that the transplanted cells will: In a review of the safety data from the initial nine patients, Neuralstem cells … Continue reading →

Human Embryonic Stem Cell Clinical Trials[edit] Introduction[edit] The Food and Drug Administration (FDA) approved the first clinical trial in the United States involving human embryonic stem cells on January 23, 2009. Geron Corporation, a biotechnology firm located in Menlo Park, California, originally planned to enroll ten patients suffering from spinal cord injuries to participate in the trial. The company hoped that GRNOPC1, a product derived from human embryonic stem cells, would stimulate nerve growth in patients with debilitating damage to the spinal cord.[1] The trial began in 2010 after being delayed by the FDA because cysts were found on mice injected with these cells, and safety concerns were raised.[2] In the United States, the FDA must approve all clinical trials involving newly developed pharmaceuticals. Researchers must complete an Investigational New Drug (IND) application in order to earn the FDAs approval. IND applications typically include data from animal and toxicology studies in which the drugs safety is tested, drug manufacturing information explaining how and where the drug will be produced, and a detailed research protocol stating who will be included in the study, how the drug will be administered and how participants will be consented.[3] Testing for new drugs must successfully … Continue reading →

ANN ARBOR, Mich. - The U.S. Food and Drug Administration gave the green light Friday for a clinical trial of a new stem cell treatment for amyotrophic lateral sclerosis (ALS). University Michigan neurologist, Eva Feldman, M.D., Ph.D., will be the overall principal investigator for the first human clinical trial of a stem cell treatment for ALS, a fatal neurodegenerative disease. The FDA approved an Investigational New Drug application from Neuralstem, Inc., a Rockville, Md.-based biotech company, to test the safety of a treatment in which patients will receive injections of the company's patented neural stem cells at multiple sites along the spinal cord. Director of the U-M ALS Clinic and the U-M Program for Neurology Research & Discovery, Feldman worked with a team of neurologists and with Neuralstem Inc. to develop the protocol for delivering the stem cells into the spinal cord of patients. The Phase 1 trial to determine the safety of the treatment is expected to take place exclusively at Emory University in Atlanta, Ga., subject to approval by its Internal Review Board. "We are very excited about this clinical trial," said Feldman, the DeJong Professor of Neurology at the U-M Medical School. "This is a major stride … Continue reading →

I invited the Chief Scientific Officer (CSO) of Bioheart, Kristin Comella, to do an interview after hearing some buzzthat this couldbe a critical time for the company and that it might have been recently visited by the FDA. Note that Comella not just Bioheart CSO, but also the primary instructor for physician training in stem cell methods offered by the company US Stem Cell Training. Can you update us on how Bioheart is doing with its clinical trials/INDs? Comella: Our completed clinical trials of MyoCell to date have been primarily targeted to patients with severe, chronic damage to the heart who are in Class II or Class III heart failure according to the New York Heart Association, or NYHA, heart failure classification system. We have completed various clinical trials for MyoCell including the SEISMIC Trial, a 40patient, randomized, multicenter, controlled, Phase IIa study conducted in Europe and the MYOHEART Trial, a 20patient, multicenter, Phase I doseescalation trial conducted in the United States. We were approved by the FDA, to proceed with a 330patient, multicenter Phase II/III trial of MyoCell in North America and Europe, or the MARVEL Trial. Thus far, 20 patients, including 6 control patients, have been treated. Initial … Continue reading →

By Dr. Mercola Adult stem cells (as opposed to embryonic stem cells, which are at the heart of the stem cell controversy) are a truly exciting part of the future of medicine, with seemingly limitless potential for anti-aging, arthritis and far more... But the U.S. Food and Drug Administration (FDA) is attempting to regulate stem cell procedures at a Colorado clinic, stating that stem cells are drugs that fall under FDA jurisdiction. Outrageously, the FDA is making this claim even though the stem cells being used in the procedure come from the patient's own body, which means they are essentially claiming that they can regulate a part of your body. What makes stem cells so special is their potential to develop into many different cell types. When a stem cell divides, it either becomes another type of cell, such as a muscle cell or cartilage, or it remains a stem cell. Furthermore, these cells act as an internal repair system in many types of tissues, dividing a seemingly indefinite number of times to replenish other cells. As you age, your stem cells diminish in quality and quantity, so just when you require strong stem cells the most, you're becoming increasingly … Continue reading →

Last week I wrote about a pending lawsuit by the FDA against Regenerative Sciences,a medical company thatdevelopeda stem cell treatment for orthopedic injuries. Whileadult stem cell therapies have been fraught with concerns about safetyand efficacy, this case is particularly important not only because it is unprecedented the FDA is attempting toclassify non-embryonicstem cellsas drugs but also because itbrings into focusthe disconnect between the pace of scientific progressand the struggle ofregulatorsto keep up withrapidly evolving technologies. I spoke withthree people with aunique perspective onwhats at stake a lawyer who specializes in FDA regulation, a lab director of an in-vitro fertilization clinic, and a bioethicist to geta sense of how thecase is viewed intheir industries. One common threadthat emergedfrom these conversations is that the FDA is conflicted, even confused,about how to regulate stem cell-based products.Theprolonged legal battle with Regenerative(it dates back to at least 2008) is duelargely to the fact that the agency recognizes the absurdity of coming down with a one-size-fits-all regulatory approach thatwould apply to every clinicoffering such treatments. To be sure, stem cell medicine has a terrible reputation.Stem cell therapies thatpurport to treateverything fromshot knees and slipped disks toautism, multiple sclerosis,and Parkinsons diseaseare rightlyregarded by many as modern-day quackery. … Continue reading →

Spencer Platt/Getty Images News/Getty Images Debates over the ethics of embryonic stem cell research continue to divide scientists, politicians and religious groups. However, promising developments in other areas of stem cell research might lead to solutions that bypass these ethical issues. These new developments could help win stem cell research more support from those against embryonic stem cell research, since they don't require the destruction of blastocysts. The most recent research has shown that there are many options available other than working with embryonic stem cells. Stem cells can be obtained from cord blood or derived by manipulating differentiated cells (i.e. skin cells) to revert them to a pluripotent state. These are alternatives that may help broaden the acceptance of stem cell research. In November 1998 the first published research paper reported that stem cells could be taken from human embryos. Subsequent research led to the ability to maintain undifferentiated stem cell lines (pluripotent cells) and techniques for differentiating them into cells specific to various tissues and organs. The debates over the ethics of stem cell research began almost immediately in 1999, despite reports that stem cells cannot grow into complete organisms. In 2000 2001, governments worldwide were beginning to … Continue reading →

The Manhattan Regenerative Medicine Medical Group is involved with the Investigational use of Autologous Adipose Derived Stem Cells (ADSCs) for clinical research and deployment. The Manhattan Regenerative Medicine Medical Group is offering patient-funded research to treat individuals with autologous (their own) fat-derived stem cells and are not involved in the use ormanufacturing of any investigational drugs or devices. The Manhattan Regenerative Medicine Medical Group is not offering stem cell therapy as a cure for any condition, disease, or injury. No statements or treatments on this website have been evaluated or approved by the FDA. This website contains no medical advice. All statements and opinions on this website are provided for educational and informational purposes only. We do not diagnose or treat via this website or via telephone. The Manhattan Regenerative Medicine Medical Group does not claim that any applications, or potential applications, using autologous adult stem cell treatments are approved by the FDA, or are even effective. We do not claim that these treatments work for any listed nor unlisted condition, intended or implied. It is important for potential patients to do their own research based on the options that we present so they can make informed decisions. See the … Continue reading →

Want to stay informed? Sign up for our email newsletter here SAN DIEGO and Epalinges, Switzerland June 9, 2015 Stemedica Cell Technologies, Inc. received the FDAs investigational new drug (IND) approval for a United States-based, Phase IIa clinical study using its allogeneic stem-cell therapy to treat subjects with mild to moderate dementia due to Alzheimers disease (AD), the sixth leading cause of death in the United States. The study is sponsored by Stemedica International, S.A. and will start at the University of California, San Diego (UCSD) under Principle Investigator Douglas Galasko, M.D. and expand to other sites. Stemedica International will provide management and financial support for this clinical trial. The clinical trial is titled "A Phase IIa Multicenter, Randomized, Single-blind, Placebo-controlled, Crossover Study to Assess the Safety, Tolerability, and Preliminary Efficacy of a Single Intravenous Dose of Allogeneic Human Mesenchymal Stem Cells in Subjects with Mild to Moderate Dementia Due to Alzheimer's Disease." This study was approved based on the excellent safety profile of Stemedicas cGMP-manufactured, hypoxically-grown stem cells and on solid pre-clinical data obtained by Stemedica International in cooperation with the cole Polytechnique Fdrale de Lausanne of Switzerland and with a grant from the Swiss government, said Lev Verkh, … Continue reading →