Peer-Reviewed Biomarker Study Results for NurOwn® in ALS Phase 3 Trial

A recent study has shed light on the potential benefits of a novel cell therapy for amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig's disease. The research, conducted by a consortium of medical institutions and published in the journal Muscle and Nerve, focused on the effects of an experimental treatment called debamestrocel, or NurOwn®. The findings indicate that this cellular therapy could significantly influence key biomarkers associated with the progression of ALS, offering new hope for patients suffering from this debilitating neurodegenerative condition.

The research involved a comprehensive analysis of 45 biomarkers, out of which three—neurofilaments light (NfL), LAP/TGFb1, and Galectin-1—were identified as predictors of clinical outcomes in patients treated with debamestrocel. Notably, the treatment induced substantial alterations in 64% of the biomarkers studied, affecting multiple disease pathways relevant to ALS. Specifically, there was an observed increase in biomarkers associated with anti-inflammatory and neuroprotective functions, hinting at the therapy's capacity to mitigate inflammation and safeguard neuronal health. Conversely, there was a decrease in markers linked to inflammation and neurodegeneration, suggesting a possible curtailment of disease progression and neuronal damage.

Dr. Anthony J. Windebank, a leading neurologist, underscored the significance of these results, highlighting the biological mechanism through which modified mesenchymal stem cells, such as debamestrocel, could be beneficial for ALS patients. He also noted the positive implications of BrainStorm Cell Therapeutics Inc.'s collaboration with the FDA via a Special Protocol Assessment (SPA), indicating a shared commitment to thoroughly assess the clinical efficacy of the treatment.

Stacy Lindborg, PhD, Co-CEO of BrainStorm, emphasized the growing importance of biomarkers in the development of ALS treatments. The successful peer review and publication of the study's data in a respected journal represent a pivotal step in the advancement of NurOwn®. The company anticipates corroborating these results in an upcoming Phase 3b trial, which is currently being planned to expedite the therapy's availability to ALS patients.

The study's data were derived from a rigorous, randomized, double-blind, placebo-controlled Phase 3 trial assessing the safety and efficacy of repeated doses of NurOwn®. Chaim Lebovits, President and CEO of BrainStorm, expressed gratitude towards the trial participants and the principal investigators, acknowledging their crucial role in the research's success.

NurOwn® represents an innovative therapeutic approach, utilizing autologous MSC-NTF cells harvested and processed from ALS patients to secrete neurotrophic factors that target neurodegenerative diseases. BrainStorm's dedication to ALS research is evident in their extensive preclinical work and clinical trials, all of which have undergone peer review.

The company holds exclusive rights to the NurOwn® technology platform for the treatment of ALS, with autologous MSC-NTF cells receiving Orphan Drug designation from both the FDA and EMA. BrainStorm has completed a Phase 3 trial in ALS, supported by grants from the California Institute for Regenerative Medicine and the ALS Association, among others.