Second phase of ALS stem-cell therapy trial completed

A patient with amyotrophic lateral sclerosis, or ALS, received 16 million stem cells directly to the spinal cord this month in the final leg of the FDA-approved Phase II trial of this novel therapy.

That brings to 30 the number of ALS patients receiving the groundbreaking stem cell implantations over the course of Phases I and II of the trial, which was designed by University of Michigan neurologist Eva L. Feldman, M.D., Ph.D.

The aim of the trial is to show that stem cells can safely be delivered to the spinal cords of patients with neurodegenerative diseases, and it is hoped that the presence of the neural stem cells will slow the progression of ALS symptoms.

The Phase II trial is approved by the U.S. Food and Drug Administration and funded by Neuralstem, Inc., the Maryland-based company whose stem-cell product the trial is testing. It seeks to study any effect that injected stem cells might have on motor neurons — muscle-controlling nerve cells that die in ALS patients, eventually robbing them of the ability to walk, speak and breathe.

Feldman, the Russell N. DeJong Professor of Neurology in the U-M Medical School and director of the Program for Neurology Research and Discovery, is the principal investigator for the trial. Feldman serves as an unpaid consultant to the company, and has led the analysis of results from the Phase I trial, which concluded in 2012.  Researchers have reported possible signs that in one subgroup of participants, ALS progression may have been interrupted.

Feldman said she now will begin evaluating patient data from Phase II and preparing the application for a wider Phase IIb trial of the landmark therapy.

“We look forward to seeing what the data tell us about the safety and efficacy of this approach,” said Feldman.  The Phase II trial began in September 2012 with the first surgery taking place at the University of Michigan Health System.  Parag Patil, M.D., Ph.D., and a member of the U-M Medical School faculty, performed the surgeries at U-M.  Other trial sites include Emory University in Atlanta and Massachusetts General Hospital in Boston.

The first 12 patients in the Phase 2 trial received doses of stem cells ranging from five injections of 200,000 cells per injections to 20 injections of 400,000 cells each, all to the cervical region of the spine where the nerves that control breathing reside.  The final three patients underwent two separate surgeries each, with cells being implanted to both the cervical and lumbar (lower) areas of the spinal cord.  Those patients received a total of 40 injections of 400,000 cells per injection for a total of 16 million cells.

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