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 forward in what still could be a long road to a new and improved treatment for ALS.
"ALS is a terrible disease that ultimately kills by paralysis. In work with animals, these spinal cord stem cells both protected at-risk motor neurons and made connections to the neurons controlling muscles. We don't want to raise expectations unduly, but we believe these stem cells could produce similar results in patients with ALS," Feldman said.
ALS, also known as Lou Gehrig's disease, affects about 30,000 Americans. It progressively destroys the neurons that control voluntary muscles, leaving affected people unable to move or speak. There are no known treatments for the disease that slow its progression.
The trial will ultimately consist of 18 ALS patients with varying degrees of the disease. The FDA has approved the first stage of the trial, which consists of 12 patients who will receive five-to-ten stem cell injections in the lumbar area of the spinal cord. The patients will be examined at regular intervals post-surgery, with final review of the data to come about 24 months later.
Jonathan Glass, M.D., director of the Emory Neuromuscular Laboratory, is expected to be the site principal investigator for the trial.
Individuals interested in further information on the trial should contact Emory Health Connection, 404-778-7777, or 1-800 75EMORY, or go to www.neurology.emory.edu/als
Institutional review boards at U-M and Emory University must first approve the protocol.
If Phase I results are favorable, the treatment will need to prove effective in Phase II and III trials and win final FDA approval before it can be available for public use.
Funding: Neuralstem, Inc. plans to conduct and fund the Phase I trial of its patented technology.
Patents/conflict disclosures: Dr. Feldman has no financial interest in or financial arrangement with Neuralstem.