Intraspinal transplantation of up to 16 million stem cells in patients with amyotrophic lateral sclerosis (ALS) was safe and well-tolerated, and caused no acceleration in disease progression, according to data presented by Dr. Eva Feldman at the American Neurological Association’s 2015 Annual Meeting in Chicago.
Additionally, 70 percent of patients recovered to higher levels than those of the historic control.
“The results of Phase I and Phase II are very encouraging, and we plan to move forward and expand this trial in 2016,” said Dr. Feldman, the Russell N. DeJong Professor of Neurology at the University of Michigan, as well as Director of the Program for Neurology Research & Discovery, Director of the A. Alfred Taubman Medical Research Institute, and Research Director of the University of Michigan Comprehensive ALS Clinic.
The data was collected through Phase I and Phase II of a trial that began in 2010. Phase II included 15 ambulatory patients with ALS. Participants were divided into five dosing cohorts with three patients in each, who received increasing quantities of cells in the cervical (upper) region of the spinal cord, ranging from two million to eight million cells. The fifth cohort received an additional eight million cells in the lumbar (lower) region.
The most common adverse effect of the stem-cell procedure was post-operative pain due to the surgery. One serious adverse event due to the surgical procedure was observed, but was not attributed to the cells themselves. The patient’s motor function was initially weakened, but then recovered to the patient’s ALS baseline.