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Stem Cell Trial

January 4, 2017

Stem Cell Trials

We successfully completed our phase 2 trial of stem cell transplantation, and published our results in 2016 (Neurology® 2016;87:392–400). This trial was designed to determine safety only, and not therapeutic efficacy. We conclude that stem cell transplantation into the spinal cord in patients with ALS is safe, though complications of the procedure may occur. We have no conclusive evidence that this procedure either slows or stops disease progression. Determination of whether stem cell transplantation is a viable treatment for ALS awaits a larger trial specifically designed to test whether stem cell therapy improves the course, and the lives, of ALS patients. Indeed, ALL stem cell therapies for ALS are currently in the experimental phase, and there is no conclusive evidence that any of the stem cell procedures now being offered provide benefit for patients with ALS. New trials are being planned both here and at other institutions to test various stem cell interventions, and all of us working in this field hope to provide answers to the ALS community regarding the efficacy of stem cell treatments in the near future.

We at the Emory ALS Center understand the dire need for effective treatments for ALS, and the hope that stem cells will be the answer. The problems we face in developing new therapies for ALS will only be solved by rigorous scientific investigation, which takes team efforts of clinicians and scientists around the world, adequate research funding, and of course time.