Merit Cudkowicz, MD, MSc: Assessing NurOwn in ALS


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.

BrainStorm Announces Topline Results from NurOwn® Phase 3 ALS Study. News release. November 17, 2020. Accessed January 4, 2021.
Related Videos
Dilsher Dhoot, MD, on a Promising Start With Diabetic Retinopathy Gene Therapy RGX-314
J. Andrew Livingston, MD, on Forging Forward With Novel Sarcoma Trials
Shankar Musunuri, PhD, on Gene Modifier Therapy vs Gene Replacement Therapy for IRDs
Pat Furlong, BSN, RN, on the State of Gene Therapy in Muscular Dystrophy
Michael Kelly, PhD, on Continuing Progress With Gene Therapy in Muscular Dystrophy
Thomas McCauley, PhD, on Potential Advantages of Epigenetic Therapy Over Small Molecule, Gene Therapy
© 2023 MJH Life Sciences

All rights reserved.