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The FDA has agreed to a new specialized design for a phase 3b clinical trial of NurOwn, a therapy developed by BrainStorm Cell Therapeutics for amyotrophic lateral sclerosis (ALS). This trial, set to begin in 2024, will involve 200 participants who are in the early stages of ALS and aims to provide critical data to support a biologics license application (BLA) for NurOwn. The investigational therapy uses autologous mesenchymal stromal cells to deliver neurotrophic factors that may protect and support neurons.
The trial will follow a robust 2-part protocol under a Special Protocol Assessment (SPA) with the FDA, including a 24-week randomized treatment period comparing NurOwn to a placebo. Participants will receive three intrathecal injections of NurOwn, created from their own bone marrow stem cells. The primary endpoint will measure changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), while additional biomarkers will be analysed to explore neuroinflammation and neuroprotection. Participants will also have access to standard ALS care during the trial.
This new phase 3b study comes after earlier challenges for NurOwn. In 2023, the FDA's advisory committee raised concerns about the treatment's efficacy and manufacturing quality based on phase 3 data, where the therapy failed to meet key endpoints. However, subsequent analyses showed promise in patients with less severe ALS, suggesting that early intervention might be critical. BrainStorm leaders are optimistic that this upcoming trial will build on prior insights, address the FDA's concerns, and bring a potentially life-changing treatment closer to ALS patients.
