WASHINGTON (AP) — The Food and Drug Administration is meeting this week to consider approval of an experimental treatment for Lou Gehrig’s disease, the culmination of an effort. lobbying efforts for years By patients with fatal neurodegenerative disease.
Those advocates still face a major hurdle: FDA regulators say the treatment isn’t working.
In document Posted on Monday, the FDA reiterated long standing condition A single study from drugmaker Brainstorm doesn’t provide strong evidence that its stem cell-based therapy helps patients with ALS, or amyotrophic lateral sclerosis.
That’s the same message the FDA gave company executives in early 2021 when they first shared data on the treatment, called Nurone. And then last November, when the FDA declined to accept the company’s application for review.
But with the support of thousands of ALS patients, Brainstorm took the rare step of “filing a protest,” essentially forcing the agency to make a decision.
“The FDA is the 800-pound gorilla here and if they’re convinced a drug doesn’t work it’s very hard to change their mind,” said Mark Sheinson, a former associate FDA commissioner who now consults for drug manufacturers.
In documents posted Monday, FDA reviewers outlined their “major concerns” about the company’s evidence.
Still, ALS patients see reasons for optimism.
under pressure From the ALS community and Congress, FDA officials have recently stressed the “urgent need” new als treatments and pledged to use maximum “regulatory flexibility” when reviewing them. FDA approved Two New ALS medications In the past year, none of them met the agency’s traditional approval standards.
Neuron is the clearest test yet of how far the agency might be willing to bend to approve a new drug for a rare and deadly condition with few treatment options.
ALS gradually destroys the nerve connections needed for basic activities and functions, including breathing. Most people die within five years of diagnosis.
At Wednesday’s meeting, federal advisers will hear from FDA scientists, company researchers and patients before taking a non-binding vote on Nuron’s effectiveness. The FDA will make a final decision on the therapy later this year.
The meeting was scheduled after ALS advocates delivered a 30,000-signature petition demanding a public investigation of the treatment.
Brian Wallach, co-founder of the advocacy group I AM ALS, says that even if NurOwn only provides a small benefit for some patients, it should be made available. Wallach, a former Obama White House staffer, was diagnosed with ALS in 2017.
“We don’t want the perfect to be the enemy of the good,” Wallach said, speaking through an interpreter. “The key is to have treatments that make it possible to turn ALS into a chronic disease and allow all patients to live longer and hopefully be cured.”
Yet, there is little consensus on NeuroN among the generally united ALS community.
The ALS Association, the largest organization in the field, has not supported Brainstorm’s bid for approval, despite giving the company $400,000 in research funding. A spokesperson said that Brainstorm has made progress in making its entire dataset available for external review.
“The amazing testimonials we’ve seen online do not match the data shared by Brainstorm,” the group said in a statement.
At Wednesday’s meeting, appeals from ALS patients and their families will surely outnumber those with such reservations. The FDA received more than 1,900 written comments, many of which expressed displeasure that Nurone was not approved years ago.
Patricia Manhardt, who was diagnosed in 2020, wrote, “If we had gotten approval for Neuron when I was still walking, I believe I would still be walking today.”
Neuron is made from stem cells collected from patients’ bone marrow. The cells are processed in the laboratory with biological proteins designed to promote nerve growth, and then injected into the spinal cord.
In a study of 200 patients, Neuron failed to show a statistically significant difference between patients receiving the drug and patients receiving a sham injection. Brainstorm and the academic researchers who conducted the study say the results were skewed by the unexpectedly large number of patients with advanced disease enrolled in the trial.
ALS is measured using a 48-point questionnaire that tracks functions such as walking, swallowing, and handwriting.
On Wednesday, researchers will tell the FDA that because many patients progressed so quickly on the scale, the study failed to show Neuron’s effect on progression. When the data is extrapolated from a small subgroup of healthy patients, they argue, Neuron slowed the disease substantially.
“Leaders in the field have said there is more to the story than ‘No, it doesn’t work,'” said Dr. Mayo Clinic neurologist Anthony Windebank will present on behalf of Brainstorm.
But FDA reviewers said Monday that the company’s theory does not explain the study’s failed results.
Physicians who were not involved in the research suggest that regulators may be willing to compromise: approving Nurone for some patients while waiting for more definitive results.
“I don’t want to lose a potential treatment for ALS, but I also don’t want to impose on the public an expensive treatment that doesn’t work,” Dr. said. Terry Heiman-Patterson of Temple University.
This agreement would be similar to the FDA’s approach Relyvrio Last year, another ALS drug suspicious data, But in that case, the drug maker had started its follow-up study long before approval.
Brainstorm has not started a second study, saying it has been unable to raise sufficient funding.
Meanwhile, FDA observers worry there will be long-term consequences if regulators continue to accept weak evidence from drugmakers.
“If the standards drop too low, it sends a message to the industry that you don’t have to prove that your drug works,” said Holly Fernandez Lynch, a bioethicist at the University of Pennsylvania. “It is understandable why some patients would accept it. But if this becomes the regulatory standard, it could set the sector back in the long term.”
Follow Matthew Perron on Twitter: @AP_FDAwriter
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