FDA Advisors Vote Against Experimental Patient-Driven ALS Treatment

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Federal health advisers voted overwhelmingly against an experimental treatment for Lou Gehrig's disease at a Wednesday meeting spurred by years of efforts by patients seeking access to the unproven therapy.

The Food and Drug Administration's expert panel voted 17-1 that drugmaker Brainstorm's stem cell-based treatment has not been shown to be effective for patients with the deadly muscle-wasting disease known as ALS, or amyotrophic lateral sclerosis. One panel member abstained from voting.

While the FDA is not bound by the vote, it largely aligns with the review surprisingly negative of the agency published earlier this week, in which staff scientists described Brainstorm's application as "scientifically incomplete" and "extremely flawed."

“Creating false hope can be considered moral harm, and using statistical magic or manipulation to provide false hope is problematic,” said Lisa Lee, an expert in bioethics and research integrity at Virginia Tech, who voted against the treatment. The only positive vote came from a panel member representing patients.

Wednesday's public meeting was essentially a risky attempt by Brainstorm and the ALS community to influence the FDA's thinking about the treatment, called NurOwn.

Brainstorm's single study of 200 patients failed to show that NurOwn prolonged life, slowed disease, or improved patient mobility. But the FDA agreed to convene the outside advisory panel after ALS patients and advocates submitted a 30,000-signature petition requesting a public meeting.

Last year, the FDA approved two new medicines for ALS, after a nearly 20-year drought of new options. The approvals came after intense pressure by advocacy groups.

FDA leaders have recently emphasized a new level of “regulatory flexibility” in reviewing experimental treatments for fatal and difficult-to-treat conditions, including ALS, Alzheimer's and muscular dystrophy .

But the agency appears unwilling to overlook the study's flawed results and missing information in Brainstorm's filing, including key details about manufacturing and quality control needed to establish the product's safety.

“It really is a disease that needs a safe and effective treatment and there are many other perspectives that we must encourage. Passing one like this would hinder that,” said Dr. Kenneth Fischbeck of the National Institutes of Health.

ALS destroys nerve cells in the brain and spinal cord necessary for walking, talking, swallowing and, eventually, breathing. Most people die within three to five years after the first symptoms.

More than a dozen people spoke during a public comment session Wednesday, including ALS patients, their families and doctors who implored the FDA to grant approval. Several speakers presented before-and-after videos showing patients who participated in the Brainstorm study walking, climbing stairs, and performing other tasks they attributed to NurOwn.

«When Matt takes Nurown, it helps him; "When he doesn't take it, he gets worse," said Mitze Klingenberg, speaking on behalf of her son, Matt Klingenberg, who was diagnosed with ALS in 2018.

The FDA is expected to issue a decision on the therapy by December 8.

The stock price of Israel-based Brainstorm Cell Therapeutics has lost more than 90% of its value over the past year, falling to 39 cents per share before pausing ahead of Wednesday's FDA meeting.

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