FDA approves multiple sclerosis stem cell clinical trial
The Tisch MS Research Center of New York has received IND approval from the FDA to commence a phase I 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 U.S. 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 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 Research Center and affiliated International Multiple Sclerosis Management Practice (IMSMP).
The clinical application of autologous neural progenitors in MS is the culmination of a decade of stem cell research conducted by a team of scientists headed by Dr. Sadiq and by Dr. Violaine Harris, research scientist at Tisch MS Research Center.
Preclinical testing found that the injection of these cells may decrease brain inflammation and promote myelin repair and/or neuroprotection.
Participants will undergo a single bone marrow collection procedure, from which mesenchymal stem cell-derived neural progenitor cells (MSC-NPs) will be isolated, expanded and tested prior to injection. Participants will receive three rounds of injections at three month intervals. Safety and efficacy parameters will be evaluated in all participants through regular follow-up visits.