WASHINGTON (AP) — 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.
A panel of Food and Drug Administration experts voted 17-1 that drugmaker Brainstorm’s stem cell-based treatment has not been shown to be effective for patients with a fatal, muscle-wasting disease called ALS or amyotrophic lateral sclerosis. One member of the panel abstained from voting.
Although the FDA is not tied to the vote, surprisingly it is largely tied to the agency. negative reviews Released earlier this week, staff scientists described Brainstorm’s application as “scientifically incomplete” and “grossly inferior.”
“Creating false hope could be considered an ethical injury and the use of statistical magic or manipulation to provide false hope is problematic,” said Lisa Lee, a bioethics and research integrity expert at Virginia Tech, who voted against the treatment. The only positive vote came from the panel member representing patients.
Wednesday’s public meeting was essentially a long-term effort by Brainstorm and the ALS community to influence the FDA’s thinking on the treatment, called NeuroNon.
Brainstorm’s single 200-patient study failed to show that Neuron extended life, slowed the disease or improved patient mobility. But the FDA agreed to convene a panel of outside advisers after ALS patients and advocates submitted a 30,000-signature petition demanding a public meeting.
FDA approved last year Two New Drugs For ALS, after nearly a 20-year drought of new options. approval followed intense lobbying By advocacy groups.
FDA leaders recently pushed for a new level of “regulatory flexibility” when reviewing experimental treatments for deadly, difficult-to-treat conditions, including ALS. Alzheimer’s And muscular dystrophy,
But the agency is unwilling to ignore the failed study results and the missing information in Brainstorm’s submission, which includes critical details on manufacturing and quality controls needed to establish the product’s safety.
“This is really a disease that needs a safe and effective treatment and there are many other possibilities that we need to be encouraged about. Giving approval to someone like this will come in the way of that,” said the doctor. Kenneth Fischbach of the National Institutes of Health.
ALS destroys nerve cells in the brain and spinal cord needed for walking, talking, swallowing and eventually breathing. Most people die within three to five years of their first symptoms.
More than a dozen people spoke during a public comment session Wednesday, including ALS patients, their family members and people seeking FDA approval. Several speakers presented before-and-after videos of patients participating in Brainstorm’s studies walking, climbing stairs, and performing other tasks that they attributed to NeuroN.
“When Matt is on Neuron it helps him, when he goes off it it makes his condition worse,” Mitz Klingenberg said, speaking on behalf of his son Matt Klingenberg, who was diagnosed with ALS in 2018. walked.
The FDA is expected to issue a decision on the therapy by December. 8.
Israel-based Brainstorm Cell Therapeutics’ share price has fallen more than 90% over the past year, falling to 39 cents a share before being halted ahead of Wednesday’s FDA meeting.
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