ANN ARBOR ? The U.S. Food and Drug Administration has approved the Phase II clinical trail of the nation’s first clinical trial of stem cell injections in patients with the deadly degenerative disease known as amyotrophic lateral sclerosis, often called ALS or Lou Gehrig’s disease.
Approval paves the way for UM to become the second site in the trial, pending approval of the UM Institutional Review Board. To date, the first phase of the trial has taken place at Emory University, with University of Michigan neurologist Eva Feldman, M.D. serving as principal investigator.
The FDA approval of a Phase II trial was announced by Neuralstem, the company whose product the trial is testing. The Phase II trial will continue to evaluate the safety of the stem cell injections, delivered directly into patients? spinal cords in escalating doses of up to 400,000 cells per injection, with a maximum of 40 injections. It will also assess any signs that the injections might be impacting patients? ALS symptoms or progression.
Feldman serves as an unpaid consultant to the company, and has led the analysis of results from the Phase I trial. In data presented last year, spinal cord injections of up to 100,000 cells were delivered safely and tolerated well ? with possible signs that in one subgroup of participants, ALS progression may have been interrupted.
?In Phase II, we?ll be injecting stem cells into the upper part of the spinal cord, and our goal is to continue to assess whether this approach is safe, and to look at whether this approach offers some benefit to our patients,? said Feldman, Russell N. DeJong Professor of Neurology at the UM Medical School, research director of UM?s ALS Clinic, and director of UM?s A. Alfred Taubman Medical Research Institute. The neurosurgeon for the trial is Parag Patil, M.D.
The approach uses injections of stem cells delivered during an operation performed by a neurosurgeon. The first phase of the trial involved 15 patients; specific plans for Phase II are still being made but information will be available at neuralstem.com.
If the UM site team receives IRB approval to recruit local participants, more information will be available at uofmhealth.org. The study at UM will be funded by the ALS Association, the National Institutes of Health and Neuralstem.
