FDA panel rejects BrainStorm Cell Therapeutics’ ALS treatment.
A Setback for ALS Treatment: FDA Panel Votes Against BrainStorm Cell Therapeutics’ Therapy
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).
The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee stated that BrainStorm’s stem cell-based therapy, NurOwn, has not demonstrated effectiveness for ALS patients, also known as Lou Gehrig’s disease. They also highlighted significant gaps in the data provided.
ALS, a debilitating disease that affects nerve cells controlling voluntary muscle movements, leads to paralysis as these cells deteriorate and die.
“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.
According to Ms. Lee, although BrainStorm Cell Therapeutics highlighted the drug’s efficacy in multiple clinical trials, she remains unconvinced by the available data.
Ms. Lee also criticized comments made by Dr. Anthony Windebank, a Mayo Clinic neurologist who presented on behalf of BrainStorm during the meeting.
Dr. Windebank had emphasized the urgency of the situation, stating that thousands of ALS patients would likely succumb to the disease within the next five years if they had 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.”
Expressing disappointment, Stacy Lindborg, co-CEO of BrainStorm, stated that many of the FDA’s questions remained unanswered due 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. “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 added.
According to the Centers for Disease Control and Prevention, approximately 31,000 people in the United States are living with ALS.
About 90 percent of ALS cases occur without any known family history or genetic cause, while the remaining 10 percent are inherited through a mutated gene associated with the disease, as reported by the ALS Association.
Last year, the FDA approved Amylyx Pharmaceuticals’ drug, Relyvrio, to slow the progression of ALS in adults. In April, the agency approved Biogen’s Qalsody for the treatment of this neurological disease.
Reuters contributed to this report.
How did Dr. Anthony Windebank argue for the urgency of approving BrainStorm’s treatment despite the concerns raised by the panel, and what was Lisa Lee’s criticism of his comments
The Food and Drug Administration (FDA) panel recently voted against BrainStorm Cell Therapeutics’ treatment for amyotrophic lateral sclerosis (ALS), a rare and fatal form of the neurodegenerative disease. The FDA’s advisory committee, which focuses on cellular, tissue, and gene therapies, expressed concerns about the treatment’s effectiveness and highlighted significant gaps in the data provided by BrainStorm.
ALS is a debilitating disease that affects the nerve cells controlling voluntary muscle movements. As these cells deteriorate and die, patients experience paralysis. Sadly, there is currently no cure for ALS, and treatments that can effectively halt its progression are desperately needed.
Lisa Lee, a renowned bioethics and research integrity expert from Virginia Tech, was one of the experts on the panel who voted against BrainStorm’s treatment. Lee expressed her concerns about false hope and the use of statistical manipulation to provide misleading expectations. She stated, “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.”
Despite BrainStorm Cell Therapeutics’ insistence on the drug’s efficacy in multiple clinical trials, Lee remains unconvinced by the available data. She believes that post hoc and biomarker analyses, when added to the totality of the evidence, can easily generate positive outcomes that are overestimated, leading to misleading conclusions.
During the panel meeting, Dr. Anthony Windebank, a neurologist from the Mayo Clinic, spoke on behalf of BrainStorm. He emphasized the urgency of the situation, stating that thousands of ALS patients would likely succumb to the disease within the next five years if they had to wait for another trial of the drug. However, Lee criticized these comments as misleading and unconvincing. She argued, “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.”
The outcome of the FDA panel’s vote is undoubtedly a setback for ALS treatment. ALS patients and their families had hoped that BrainStorm’s stem cell-based therapy, NurOwn, would provide a glimmer of hope in the fight against this devastating disease. However, the panel’s decision raises significant concerns about the treatment’s effectiveness and the validity of the data supporting it
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