FDA Advises More Clinical Data is Need for Potential ALS Stem Cell Treatment

The Food and Drug Administration (FDA) gave feedback about autologous neurotrophic factor-secreting mesenchymal stem cells (MSC-NTF)(NurOwn; BrainStorm Cell Therapeutics, New York, NY), in a phase 3 clinical trial (NCT03280056) for potential treatment of amyotrophic lateral sclerosis (ALS). The current level of clinical data was determined not to provide the level of substantial evidence needed to support a Biologics License Application (BLA). The recommendation, however, does not prevent BLA submission by the manufacturer. 

Although clinical trial participants with ALS who were treated with MSC-NTF had clinical benefit, it did not reach statistical significance compared with an unexpectedly high response to placebo treatment. 

"Brainstorm will first consult with principal investigators, ALS experts, expert statisticians, regulatory advisors, and ALS advocacy groups to assess the benefit/risk of a BLA submission before making a final decision," said Chaim Lebovits, chief executive officer Brainstorm Cell Therapeutics. "We want to thank our independent advisors for their candor and unwavering commitment to our ALS program. We also would like to give a heartfelt thank you to all ALS patients and their families that were involved in this clinical research program. The company will continue to build on the strength of its wonderful, professional and dedicated team while being well funded at a current position of over $40M in cash. We will soon announce our next flagship product and program for other diseases with unmet needs."

"Many of us with longstanding experience in ALS therapy development agree that there was evidence of benefit from NurOwn cell therapy and hope that there will be an opportunity for further assessment of this modality in ALS," said Robert Brown, DPhil, MD, director of the Program in Neurotherapeutics at the University of Massachusetts Medical School.

"ALS is a devastating disease with worse outcomes than most forms of cancer," said Anthony Windebank MD, Jean and Judith Pape Adams professor of Neuroscience, Mayo Clinic College of Medicine and Science. "The clear signal in this trial that some patients with ALS respond to treatment with NurOwn is a light at the end of the tunnel. The careful study of biomarkers associated with response will help lead us forward towards a broadly effective therapy. The patients and their families who have brought us to this point are true heroes."