FDA Panel Votes Against ALS Treatment

In a significant move, a panel of experts from the Food and Drug Administration (FDA) has voted against endorsing an experimental treatment for Lou Gehrig’s disease, commonly known as ALS. The treatment, developed by the pharmaceutical company Brainstorm, was under scrutiny due to its stem cell-based approach. 

According to US News, the overwhelming 17-1 vote by the panel indicates skepticism about the treatment’s efficacy. This decision echoes the FDA’s earlier sentiments, where they had raised concerns about the completeness and adequacy of Brainstorm’s application. 

Lisa Lee from Virginia Tech, who specializes in bioethics and research integrity, voiced concerns about the potential of giving patients false hope. She emphasized the ethical implications of promoting a treatment based on potentially misleading data. Notably, the sole vote in favor of the treatment came from a representative who stood for the patients. 

The public meeting was more than just a review; it was an avenue for Brainstorm and the ALS community to present their case and possibly influence the FDA’s final decision on the drug, named NurOwn. Despite the drug’s questionable results in a study involving 200 patients, the FDA convened this panel, largely due to a petition that garnered 30,000 signatures, advocating for a public review. 

Recent times have seen the FDA greenlighting two new treatments for ALS, ending a long period where no new drugs were introduced. These approvals were a testament to the relentless efforts of advocacy groups. The FDA has also been vocal about its willingness to be more flexible when it comes to reviewing treatments for severe diseases, including ALS. However, with Brainstorm’s treatment, the FDA seems to be treading cautiously.

They have expressed reservations about the study’s outcomes and gaps in Brainstorm’s data, especially concerning production and quality assurance. Dr. Kenneth Fischbeck, affiliated with the National Institutes of Health, highlighted the dire need for effective ALS treatments. He cautioned that hastily approving treatments without robust evidence could impede the progress of more promising alternatives. ALS is a debilitating condition that affects nerve cells, leading to severe physical impairments.

The disease’s progression is rapid, with most patients facing mortality within a few years of diagnosis. During the meeting, several stakeholders, including patients and their families, passionately made their case, urging the FDA to consider the treatment. Some even showcased the apparent benefits of NurOwn through videos. One poignant testimony came from Mitze Klingenberg, who spoke about her son’s experience with ALS.

She shared that her son showed improvement when on the treatment but regressed without it. As the medical community and patients await with bated breath, the FDA is slated to announce its final decision on NurOwn by December 8th. Amidst these developments, Brainstorm Cell Therapeutics, based in Israel, has seen a sharp decline in its stock value over the past year.