FDA Panel Votes Overwhelmingly Against BrainStorm Cell Therapeutics’ Experimental ALS Treatment

FDA Panel Votes Overwhelmingly Against BrainStorm Cell Therapeutics’ Experimental ALS Treatment
The U.S. Food and Drug Administration (FDA) in White Oak, Md., on June 5, 2023. (Madalina Vasiliu/The Epoch Times)
Katabella Roberts

A panel of advisers with the Food and Drug Administration (FDA) on Sept. 27 voted overwhelmingly against BrainStorm Cell Therapeutics’ treatment for a rare and fatal form of the neurodegenerative disease amyotrophic lateral sclerosis (ALS).

In their meeting, the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee said that BrainStorm's stem cell-based therapy, NurOwn, has not been shown to be effective for patients with ALS—also known as Lou Gehrig's disease—noting that there were also large amounts of missing data.

ALS affects nerve cells in the brain and spinal cord that control voluntary muscle movements such as walking, chewing, and breathing, leading to the nerve cells not working and dying. The disease eventually leads to paralysis.

"Creating false hope can be considered a moral injury and the use of statistical magic or manipulation to provide false hope is problematic in this current situation and in the drug approval process at large. I'm worried what was demonstrated today might be false hope for persons with ALS, their caretakers, and loved ones," said Lisa Lee, a bioethics and research integrity expert from Virginia Tech, who was among the experts who voted against the treatment.

Ms. Lee said that while drug efficiency was noted in multiple clinical trials by BrainStorm Cell Therapeutics, she ultimately does not believe the data supports that claim.

Additionally, Ms. Lee took aim at comments made by Dr. Anthony Windebank, a Mayo Clinic neurologist who presented on BrainStorm’s behalf during the meeting.

Dr. Windebank had earlier stressed that thousands of patients with ALS were likely to die from the disease within the next five years if they were forced to wait for another trial of the drug.

"I think post hoc and biomarker analysis, adding that to the totality of the evidence is mixing evidence with anecdote and this generated positive outcomes that I believe are overestimated. I think there's no denying that effective treatments are needed but comments like 30,000 people with ALS will die while awaiting approval are misleading," Ms. Lee said.

Vote Is 'Sad Outcome for ALS Community'

BrainStorm Cell Therapeutics said evidence from its Phase 3 clinical trial shows "clinically meaningful effectiveness in ALS participants who have not progressed to advanced levels of disease progression."

Stacy Lindborg, co-CEO of BrainStorm, shared her disappointment with the outcome of Monday's vote in a statement and said that many of the FDA's questions had gone unanswered owing to time constraints.

"The Committee's vote was a sad outcome for the ALS community, who have too few options to help manage this merciless and deadly disease," she said in a statement. "We firmly believe that the totality of data presented for NurOwn today provides a compelling case for approval, with clinical evidence in those with less advanced disease supported by strong and consistent biomarker data that are predictive of clinical response."

"We truly did our best to make the NurOwn data clear to the FDA Advisory Committee. Unfortunately, had more time and opportunity been allowed, many remaining questions posed by Advisory Committee members could have been sufficiently addressed," she said.

Approximately 31,000 people are living with ALS in the United States, according to the latest data from the Centers for Disease Control and Prevention.

Roughly 90 percent of ALS cases occur without any known family history or genetic cause, while the remaining 10 percent of cases are inherited through a mutated gene with a known connection to the disease, according to the ALS Association.

The FDA last year granted approval of Amylyx Pharmaceuticals drug, Relyvrio, to help slow the disease in adults. In April, the agency approved Biogen's Qalsody for the treatment of the neurological disease.
Reuters contributed to this report.