A New Phase in ALS Treatment: NurOwn Enters a Crucial Stage Following FDA Meeting

BrainStorm Cell Therapeutics announced the conclusion of a significant meeting with the FDA regarding NurOwn, a new innovative treatment for ALS. This critical meeting aimed at securing a protocol for a Phase 3b trial. It marks a key milestone in the development of the MSC-NTF cell-based NurOwn®, offering a new, personalized approach to ALS treatment.

The primary focus of the pivotal meeting between BrainStorm and the US Food and Drug Administration (FDA) was on NurOwn®, the Phase 3b trial for amyotrophic lateral sclerosis (ALS) treatment. BrainStorm plans to submit the necessary documentation to comply with FDA requirements for the Special Protocol Assessment (SPA). This process is crucial in drug development, facilitating consensus between the FDA and pharmaceutical companies on clinical trial design and scale.

This agreement is essential for ensuring that a trial can effectively support a future marketing application. BrainStorm's commitment to submitting the necessary documentation for the SPA underscores their dedication to advancing this promising treatment through the regulatory maze.

Chaim Lebovits, President and CEO of BrainStorm, emphasized the significance of the meeting outcomes, stating: “Our meeting with the FDA provided us with valuable input on the Phase 3b trial design, outlining clear steps forward. We believe that an SPA will serve to help de-risk certain regulatory aspects of the NurOwn clinical program.  We recognize the urgency in providing ALS patients with safe and efficacious treatments and are committed to agreeing on a regulatory path forward for NurOwn.”

NurOwn® represents a cutting-edge approach in tackling the complexities of neurodegenerative disorders. This technology, based on autologous MSC-NTF cells, is tailored to each ALS patient, offering a personalized treatment avenue. These cells, harvested and processed using proprietary techniques, are designed to secrete neurotrophic factors that target the specific pathways involved in neurodegenerative diseases.

Furthermore, the NurOwn clinical program has yielded invaluable insights into ALS pathology, disease progression, and treatment. Noteworthy findings from their Phase 3 trial analysis have been shared through peer-reviewed publications. These include an exploration of the Floor Effect, a comprehensive evaluation of multiple pre-specified biomarkers collected longitudinally, and the pioneering use of genetic data analysis for pharmacogenomic insights into clinical outcomes.

About Author: Tracy Mokwe

With over 3 years of experience as a medical laboratory scientist and writer, I am passionate about translating complex scientific information into clear and accessible content. My expertise lies in medical sciences, laboratory science and research, digital health, AI, and cybersecurity in healthcare.