The ALS Buzz Around Brainstorm and the Israeli Study

The ALS community looks hopefully at the results of a clinical trial conducted in Israel at Hadassah Medical Center.  The results were published last week in JAMA Neurology, with the paper titled, “Safety and Clinical Effects of Mesenchymal Stem Cells Secreting Neurotrophic Factor Transplantation in Patients With Amyotrophic Lateral Sclerosis – Results of Phase1/2 and 2a Clinical Trials.”  Dr. Dimitrios Karussis, who conducted the clinical trial, was quoted as seeing the results as “very encouraging.”

Bone marrow samples were taken from each of the 26 patients.  In a lab, the bone marrow cells were grown into a type of stem cell that produces NTFs.  These NTFs are nutrients for neuron cells and can have protective effects.  The newly formed stem cells were then injected back into the patients, in their muscles, their spinal cord, or both.  Investigators hoped the neurons would be protected by absorbing the NTFs and less likely to deteriorate and stop functioning, as seen in ALS.  There were some encouraging results that shows this kind of treatment slowed the progression of ALS symptoms.  

So what does it really mean?  There are questions from friends on social media, and news stories showing patients being injected in the spine.  Is this a cure for ALS?

Not yet.  We can be encouraged, but not satisfied.  In truth, it really brings more questions left to be answered.

All eyes will be on the results of the clinical trials at the Mayo Clinic, Massachusetts General Hospital and University of Massachusetts Memorial Medical Center that will use the same treatment with the exact same protocol.  The more patients involved, the more accurate the results will be.  These phase 2 clinical trials will show the safety of the treatment, what type of patients might use this treatment, what dosage and how frequent it should be given.  If those treatments show positive outcomes, the treatment will proceed to Phase 3 clinical trials.  

The news is an important step forward in the right direction.  This kind of step infuses the whole scientific and medical community with energy to keep going.  All those other investigators, who are looking at ALS treatments using different methods, will not stop their work either.  A multi-faceted approach to dealing with ALS is coming.

Details on the NEALS-sponsored BrainStorm trial of implanted NTF-MSCs can be found here:https://clinicaltrials.gov/ct2/show/study/NCT02017912

Information about clinical trials, can be found at http://www.alsconsortium.org/browse.php or  https://clinicaltrials.gov/.