The director of the Healey & AMG Center for ALS spoke to the recent topline results of a phase 3 study of NurOwn in patients with the neuromuscular disease.
“We selected people who were rapid progressors, and the reason for that was really 2-fold—1 is that’s the group we saw the best response in, in the phase 2 study, and also because in a 6-month period, that’s the group you’re more likely to see an effect in.”
In late 2020, BrainStorm Cell Therapeutics announced the topline results from its randomized, double-blind placebo-controlled phase 3 trial evaluating NurOwn (mesenchymal bone marrow stromal cells secreting neurotrophic factors) as a treatment for amyotrophic lateral sclerosis (ALS). The findings, while suggesting that the therapy was generally well-tolerated, did not achieve statistically significant results. The primary end point was achieved in 34.7% of NurOwn participants versus 27.7% of the placebo group (P = .453).
The treatment was assessed in a population of rapidly progressing patients with ALS, investigator Merit Cudkowicz, MD, MSc, told NeurologyLive, and did display a numerical improvement in the treated group compared to placebo across the primary and key secondary efficacy end points. Notably, the trial met the expected 35% NurOwn treatment group efficacy response assumption, though there was a high placebo response that exceeded placebo responses observed in contemporary ALS trials.
To find out more about the trial and what the clinical community should take away from these findings in light of the lack of statistical significance, NeurologyLive interviewed Cudkowicz, who is the director of the Healey & AMG Center for ALS and chief of the department of neurology at Massachusetts General Hospital.