TOPSHOT - Valentyn Mozgovy, paralysed by amyotrophic lateral sclerosis (ALS), breathes with a respiratory unit in his flat in Kyiv on January 21, 2023. - Adapting to power cuts triggered by Russian strikes on Ukrainian energy infrastructures was a matter of life or death for Valentyn Mozgovy, who cannot breathe without a ventilator.
A meeting of federal health experts was called on Wednesday as a result of years of patient attempts to obtain access to an unproven medication for Lou Gehrig's disease. The decision was decisively against the experimental treatment.
The Food and Drug Administration (FDA) expert panel ruled 17-1 that Brainstorm's stem cell-based therapy has not been proven beneficial for people with ALS, also known as amyotrophic lateral sclerosis, a deadly condition that destroys muscles. A panelist didn't participate in the vote.
The vote does not obligate the FDA, but it substantially supports the agency's startlingly critical analysis published earlier this week. Staff scientists therein referred to Brainstorm's application as "scientifically incomplete" and "grossly deficient."
A Last-Ditch Effort
The purpose of the public gathering on Wednesday was essentially to make a last-ditch effort by Brainstorm and the ALS community to influence FDA policy regarding the NurOwn treatment.
NurOwn did not demonstrate an improvement in patient mobility, a prolongation of life, or a slowing of disease in Brainstorm's single 200-patient study. However, after receiving a petition with 30,000 signatures from ALS patients and advocates calling for a public meeting, the FDA decided to hold the panel of outside experts meeting.
After almost 20 years without new treatments, the FDA authorized two new ALS medications. Following vigorous lobbying by advocacy groups, the clearances were given.
However, the FDA doesn't seem willing to disregard the falsified study findings and missing data from Brainstorm's submission, which include crucial information on production and quality control necessary to prove the product's safety.
The disease ALS kills the nerve cells in the brain and spinal cord necessary for breathing, walking, talking, and swallowing. Most deaths occur three to five years after the onset of symptoms.
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The Plea For Approval
More than a dozen people, including people living with ALS, their family members, and doctors, spoke during a public comment period on Wednesday and pleaded with the FDA to approve.
Videos of patients who took part in Brainstorm's study walking, climbing stairs, and accomplishing other things that they attributed to NurOwn were shown in several speakers' before-and-after presentations.
By December 8, the FDA is anticipated to make a decision regarding the therapy.
The stock price of Israel-based Brainstorm Cell Therapeutics has dropped more than 90% in the past year, reaching a low of 39 cents per share before being suspended before Wednesday's FDA meeting.
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