The ongoing phase 3 clinical trial of autologous neurotrophic factor-secreting mesenchymal stem cell (MSC-NTF) (NurOwn; BrainStorm Cell Therapeutics, Paramus, NJ) treatment has received funding through a grant. The grant was awarded by the ALS Association and I AM ALS to BrainStorm Cell Therapeutics’ biomarker research study. With the assistance of the grant, further understanding of critical biomarkers associated with treatment response for individuals with amyotrophic lateral sclerosis (ALS) is possible.
The study (NCT03280056) evaluates the efficacy and safety of MSC-NTF cells compared with placebo as measured by the amyotrophic lateral sclerosis functional rating scale (ALSFRS-R). To evaluate biomarkers (such as cell-secreted neurotrophic factors, inflammatory factors, and cytokines in pg/mL) in the cerebrospinal fluid (CSF) and blood throughout the study to evaluate their relationship to treatment with MSC-NTF cells.
If successful, this study will help confirm that the ALS treatment works, which is MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.
"This grant to BrainStorm marks an important step forward in establishing how exactly NurOwn works in the body," said Calaneet Balas, president and chief executive officer of the ALS Association. "This research is also important to our overall pursuit of identification and validation of ALS biomarkers. We hope NurOwn is ultimately proven effective in treating ALS and we stand ready to support BrainStorm in its plan to apply for a biologics license for NurOwn."
Danielle Carnival, chief executive officer of I AM ALS commented, "We need to move with urgency in all of our efforts to deliver treatments and cures for ALS. This biomarker research will help us more expeditiously understand the effectiveness of NurOwn, while possibly unlocking discoveries that provide clues for other promising treatments. We are at a pivotal time for ALS research in pursuit of treatment solutions and will do whatever we can, together, to drive new answers and new hope for patients."
BrainStorm has agreed to share data and samples with the ALS community so that the results can be independently validated and to advance other ALS research.
Jakai D. Nolan, DO, MPH, and Jacqueline A. Nicholas, MD, MPH
David Z. Rose, MD
Danielle S. Shpiner, MD; Crystal Dixon, MD; Melissa R. Ortega, MD; and Henry Moore, MD