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 expert panel ruled 17-1 that Brainstorm’s stem cell-based therapy has not been proven to be 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 FDA is not obligated by the vote, but it substantially supports the agency’s own startlingly critical analysis that was published earlier this week. Staff scientists therein referred to Brainstorm’s application as “scientifically incomplete” and “grossly deficient.”
What were the treatment-related concerns raised by the FDA panel?
“Creating false hope can be considered a moral injury, and the use of statistical magic or manipulation to provide false hope is problematic,” said Lisa Lee, a bioethics and research integrity specialist from Virginia Tech, who voted against the procedure. A panelist who was on the side of the patients cast the lone affirmative vote.
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 meeting of the panel of outside experts.
After almost 20 years without any new treatments, the FDA has authorized two new ALS medications. Following vigorous lobbying by advocacy groups, the clearances were given.
ALS, Alzheimer’s disease, and muscular dystrophy are examples of terminal, difficult-to-treat illnesses that FDA authorities have recently stressed a new level of “regulatory flexibility” while examining experimental treatments.
The agency, however, doesn’t seem willing to overlook the insufficient study results and missing data in Brainstorm’s proposal, including crucial information on manufacturing and quality control required to prove the product’s safety.
There are many other opportunities out there that we need to support. It really is a condition that needs a safe and effective therapy. Dr. Kenneth Fischbeck of the National Institutes of Health stated that approving one like this would prevent that.
What do people with ALS think of the medicine?
More than a dozen people, including ALS sufferers, 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.
“When Matt is on Nurown it helps him, when he’s off of it he gets worse,” Mitze Klingenberg stated on behalf of her son Matt Klingenberg, who was given an ALS diagnosis in 2018.
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 ahead of Wednesday’s FDA meeting.
ALS: What is it?
The disease ALS kills the nerve cells in the brain and spinal cord that are necessary for breathing, walking, talking, and swallowing. Most deaths occur three to five years after the onset of symptoms.
“ALS may be seen as an uncommon condition, but it affects 1 in 300 Americans, making it more common than most people realize. According to Brian Frederick, senior vice president of communication at the ALS Association, it can kill anyone at any time.
According to the Centers for Disease Control and Prevention, more than 30,000 Americans are thought to have ALS, and on average, 5,000 new cases are reported countrywide each year.
The condition is sometimes referred to as Lou Gehrig’s disease after the illustrious New York Yankees star who contracted it in the late 1930s.