Finally Mayo Rochester has reported results from its mesenchymal stem cell study of multiple system atrophy. Local support group member John Yanez-Pastor participated in the study. This was a phase I/II safety and tolerability study, NOT an efficacy study. Wolfgang Singer, MD, reported at this week’s annual meeting of the American Academy of Neurology that the 24 probable MSA patients who participated in the very small study reported no serious adverse events with the treatment.
I guess with the phase II aspect of the study, a bit about efficacy could be studied. Dr. Singer said that the “efficacy of MSCs on slowing multiple system atrophy progression….appeared to be dependent on the dose, and, in the highest dose individuals, had a painful implantation response.”
Slowed disease progression was measured by the Unified MSA Rating Scale. There was only one point difference between the study group and a “historical placebo group.” I’m not sure a one point difference really leads to better quality of life. (Sorry.) There was no change on on any autonomic scales.
Mayo Rochester is in the late planning stages for a multicenter, double-blind, placebo-controlled phase II/III study.
There’s a four-minute video interview with Dr. Singer here:
And here’s a link to the full article from Clinical Neurology News:
VIDEO: Pilot stem cell trial for multiple system atrophy shows promising results
Publish date: April 25, 2017
By: Jeff Evans, Clinical Neurology News
At AAN 2017