It was recently announced via press release by BrainStorm Cellular Therapeutics that their proposed treatment for ALS, NurOwn, will continue to be investigated in ALS in the United States through a Phase 2 clinical trial. According to the latest update provided by BrainStorm on the status of the clinical trial, the ALS Therapy Development Institute lists this trial as “currently recruiting” in its global ALS clinical trial database.
The announcement comes as a result of the trial’s Data and Safety Monitoring Board (DSMB) recommendation for the continuation of the study. This is not uncommon, highlighted by a similar announcement from AB Science regarding their compound masitinib. It is one of the jobs of a DSMB to review data continuously throughout the trial they are charged with monitoring, and to make a recommendation for its continuation or cancellation based on that data. This announcement should not be interpreted as any indication this treatment is effecting ALS disease progression in either a positive or negative way. It should only be considered a sign that the DSMB believes that the trial should continue forward. It should be expected that a final report of the trial’s results will be known only after the trial is completed, which shouldn’t be expected for another year based on the timelines outlined by BrainStorm previously.
What is NurOwn?
NurOwn may help keep motor neurons healthy in people with ALS by boosting production of GDNF, a neuroprotective substance which promotes their survival and growth. In this potential treatment strategy, adult stem cells from the patient's own bone marrow are reprogrammed into GDNF-secreting astrocyte-like cells and reintroduced. NurOwn can likely be administered without anti-rejection medicines. Earlier clinical trials have taken place on this approach in Israel. Investigators are exploring both intra-muscular and intra-spinal administration of their stem cells in people diagnosed with ALS in a Phase 2 clinical trial currently enrolling in the United States.