FDA Panel Rejects Experimental ALS Drug
FDA Panel Rejects Experimental ALS Drug
Stem Cell Treatment for ALS Rejected by FDA Advisory Panel
In a disappointing announcement, an advisory panel to the U.S. Food and Drug Administration (FDA) has voted overwhelmingly against recommending a stem cell-based experimental treatment for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. While the FDA is not bound by the panel’s vote, agency scientists have already issued a scathing review of the treatment, called NuOwn, labeling it as “scientifically incomplete” and “grossly deficient.”
ALS is a devastating disease that typically proves fatal within three to five years of the onset of symptoms. The condition destroys nerve cells in the brain and spinal cord, gradually robbing patients of their ability to walk, talk, swallow, and breathe. The urgent need for effective treatments prompted Brainstorm, the company behind NuOwn, to develop this stem cell therapy. However, the recent developments have dashed hopes for ALS patients eagerly awaiting a breakthrough.
The advisory panel vote against NuOwn was decisive, with 17 out of 18 members opposing the drug, and only one panelist representing patients voting in favor of it. Additionally, one adviser abstained from voting, underscoring the concerns raised by FDA staff scientists in their review. Lisa Lee, a bioethics and research integrity expert from Virginia Tech, emphasized the potential moral harm of creating false hope and expressed reservations about statistical manipulation used to provide false optimism.
The FDA’s decision to convene the advisory panel was in response to a 30,000-signature petition from ALS patients and advocates. However, the results of a study conducted by Brainstorm, involving 200 patients, failed to demonstrate that NurOwn extended life, slowed disease progression, or improved patient mobility. Furthermore, the company’s application lacked essential information on manufacturing and quality control, further undermining its case for approval.
Medical professionals and experts agree that ALS desperately needs a safe and effective treatment. However, many believe that approving a treatment like NuOwn would only hinder the development of other promising therapies in the pipeline. Dr. Kenneth Fischbeck, a distinguished investigator in the Hereditary Neurological Diseases branch of the National Institutes of Neurological Disorders and Stroke, expressed this sentiment, stating, “It really is a disease that needs a safe and effective treatment, and there are a lot of other prospects out there that we need to encourage.”
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During the public comment period, ALS patients, family members, and physicians had the opportunity to share their perspectives. Several individuals presented compelling before-and-after videos of ALS patients from Brainstorm’s study, highlighting the potential benefits of NuOwn. One such example was Matt Klingenberg, diagnosed with ALS in 2018, whose mother, Mitze Klingenberg, spoke on his behalf, asserting, “When Matt is on NuOwn, it helps him, when he’s off of it, he gets worse.” These personal accounts underscore the desperate search for effective treatments and the complexities faced by both patients and their loved ones.
It is worth noting that the FDA has approved two other ALS medications, Qalsody and Reylvrio, in the past year, signifying a significant breakthrough after a twenty-year drought of new options. These approvals offer a glimmer of hope for ALS patients and demonstrate the continued commitment of medical researchers and the FDA to address this debilitating disease.
Conclusion
The rejection of stem cell-based experimental treatment for ALS by the FDA advisory panel highlights the challenges faced in finding effective therapies for this devastating disease. While the panel’s vote is not binding, the concerns raised by FDA staff scientists regarding the incomplete nature of the application and the lack of key information on manufacturing and quality control cast doubt on the viability of the treatment. Patients, their families, and the medical community continue to advocate for safe and efficacious treatments for ALS, as witnessed during the public comment period. Despite this setback, recent approvals of ALS medications offer hope and reinforce the ongoing efforts to combat this debilitating condition.
