WASHINGTON (AP) 鈥 The Food and Drug Administration meets this week to consider approval of an experimental treatment for Lou Gehrig鈥檚 disease, the culmination of a yearslong lobbying effort by patients with the fatal neurodegenerative disease.
Those advocates still face one giant hurdle: FDA regulators say the treatment hasn't been shown to work.
In posted Monday, the FDA reiterated its that a lone study by drugmaker Brainstorm doesn't provide convincing evidence that its stem cell-based therapy helps patients with ALS, or amyotrophic lateral sclerosis.
It鈥檚 the same message the FDA delivered to company executives in early 2021 when they first shared data on the treatment, dubbed NurOwn. And again last November, when the FDA refused to accept the company鈥檚 application for review.
But with the backing of thousands of ALS patients, Brainstorm took the rare step of 鈥渇iling over protest,鈥 essentially forcing the agency to render a decision.
鈥淔DA is the 800-pound gorilla here and if they鈥檙e convinced that the drug doesn鈥檛 work it鈥檚 very hard to change their minds,鈥 said Marc Scheineson, a former associate FDA commissioner who now consults for drugmakers.
In the documents posted Monday, FDA reviewers outlined their 鈥渕ajor concerns" about the company's evidence.
Still, ALS patients see reasons for optimism.
from the ALS community and Congress, FDA officials have recently emphasized the 鈥渦rgent need鈥 for and pledged to use maximum 鈥渞egulatory flexibility鈥 when reviewing them. FDA has approved new in the last year, neither of which met the agency鈥檚 traditional approval standards.
NurOwn is the clearest test yet of how far the agency may be willing to bend to approve a new medicine for a rare and deadly condition with few treatment options.
ALS gradually destroys nerve connections needed for basic movements and functions, including breathing. Most people die within five years of diagnosis.
At Wednesday鈥檚 meeting, federal advisers will hear from FDA scientists, company researchers and patients before taking a non-binding vote on NurOwn's effectiveness. FDA will make the final decision on the therapy later this year.
The meeting was scheduled after ALS advocates delivered a 30,000-signature petition seeking a public vetting of the treatment.
Brian Wallach, co-founder of the advocacy group I AM ALS, says even if NurOwn only provides a small benefit for some patients, it should be made available. A former Obama White House staffer, Wallach was diagnosed with ALS in 2017.
鈥淲e do not want the perfect to be the enemy of the good,鈥 said Wallach, speaking through an interpreter. 鈥淭he key is to have treatments that make it possible to turn ALS into more of a chronic disease and to allow all patients to live longer and hopefully see a cure.鈥
Still, there鈥檚 little consensus on NurOwn among the normally tight-knit ALS community.
The ALS Association, the largest organization in the field, has not endorsed Brainstorm鈥檚 bid for approval despite giving the company $400,000 in research funding. Brainstorm declined to make its complete dataset available for external review, a spokesperson noted.
"The amazing testimonials we have seen online do not align with the data that Brainstorm has shared," the group said in a statement.
At Wednesday鈥檚 meeting, people with such reservations are certain to be outnumbered by appeals from ALS patients and their families. The FDA has received more than 1,900 written comments, many expressing outrage that NurOwn was not approved years ago.
鈥淚f we would鈥檝e had NurOwn approved back when I was still walking, I believe I might still be walking today,鈥 wrote Patricia Manhardt, who was diagnosed in 2020.
NurOwn is made from stem cells collected from patients' bone marrow. The cells are processed in a lab with biological proteins designed to promote nerve growth, and then injected into the spinal column.
In a study of 200 patients, NurOwn failed to show a statistically significant difference between patients who received the drug and those who got sham injections. Brainstorm and the academic researchers who conducted the study say the results were skewed by an unexpectedly high number of patients with advanced disease who enrolled in the trial.
ALS is measured using a 48-point questionnaire that tracks functions like walking, swallowing and handwriting.
On Wednesday, researchers will tell FDA that because many patients declined so quickly on the scale, the study failed to show Nurown鈥檚 effect on progression. When data from a small subset of healthier patients is isolated, they argue, NurOwn significantly slowed the disease.
鈥淭he leaders in this field have said there鈥檚 more to this story than just, 鈥楴o it doesn鈥檛 work,'鈥 said Dr. Anthony Windebank, a Mayo Clinic neurologist who will present on Brainstorm鈥檚 behalf.
But FDA reviewers said Monday the company's theory does not explain the study's failed results.
Physicians who weren鈥檛 involved in the research suggest regulators might be willing to make a compromise: approving NurOwn for some patients while awaiting more definitive results.
鈥淚 don鈥檛 want to lose a potential treatment for ALS, but I also don鈥檛 want to foist on the public an expensive treatment that doesn鈥檛 work,鈥 said Dr. Terry Heiman-Patterson of Temple University.
That compromise would be similar to FDA's approach to last year, another ALS drug with . But in that case, the drugmaker had started its follow-up study long before approval.
Brainstorm has not begun a second study, saying it鈥檚 been unable to raise enough money.
Meanwhile, FDA observers worry about the long-term consequences if regulators keep accepting weaker evidence from drugmakers.
鈥淚f the standards fall too low, that sends a message to industry that you don鈥檛 have to prove your drug works,鈥 said Holly Fernandez Lynch, a bioethicist at University of Pennsylvania. 鈥淚t鈥檚 understandable why some patients would accept that. But if that becomes the regulatory standard, it can set back a field in the long term.鈥
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