By MATTHEW PERRONE (AP Health Writer)

WASHINGTON (AP) — The Food and Drug Administration meets this week to think about approval of an experimental remedy for Lou Gehrig’s illness, the fruits of a yearslong lobbying effort by sufferers with the deadly neurodegenerative illness.

Those advocates nonetheless face one big hurdle: FDA regulators say the remedy hasn’t been proven to work.

In paperwork posted Monday, the FDA reiterated its longstanding place {that a} lone examine by drugmaker Brainstorm doesn’t present convincing proof that its stem cell-based remedy helps sufferers with ALS, or amyotrophic lateral sclerosis.

It’s the identical message the FDA delivered to firm executives in early 2021 once they first shared information on the remedy, dubbed NurOwn. And once more final November, when the FDA refused to just accept the corporate’s utility for overview.

But with the backing of 1000’s of ALS sufferers, Brainstorm took the uncommon step of “filing over protest,” primarily forcing the company to render a choice.

“FDA is the 800-pound gorilla here and if they’re convinced that the drug doesn’t work it’s very hard to change their minds,” stated Marc Scheineson, a former affiliate FDA commissioner who now consults for drugmakers.

In the paperwork posted Monday, FDA reviewers outlined their “major concerns” concerning the firm’s proof.

Still, ALS sufferers see causes for optimism.

Under strain from the ALS group and Congress, FDA officers have not too long ago emphasised the “urgent need” for brand spanking new ALS remedies and pledged to make use of most “regulatory flexibility” when reviewing them. FDA has accredited two new ALS medicine within the final 12 months, neither of which met the company’s conventional approval requirements.

NurOwn is the clearest take a look at but of how far the company could also be keen to bend to approve a brand new medication for a uncommon and lethal situation with few remedy choices.

ALS regularly destroys nerve connections wanted for fundamental actions and features, together with respiration. Most folks die inside 5 years of prognosis.

At Wednesday’s assembly, federal advisers will hear from FDA scientists, firm researchers and sufferers earlier than taking a non-binding vote on NurOwn’s effectiveness. FDA will make the ultimate determination on the remedy later this 12 months.

The assembly was scheduled after ALS advocates delivered a 30,000-signature petition searching for a public vetting of the remedy.

Brian Wallach, co-founder of the advocacy group I AM ALS, says even when NurOwn solely gives a small profit for some sufferers, it ought to be made out there. A former Obama White House staffer, Wallach was recognized with ALS in 2017.

“We do not want the perfect to be the enemy of the good,” stated Wallach, talking by means of an interpreter. “The 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.”

Still, there’s little consensus on NurOwn among the many usually tight-knit ALS group.

The ALS Association, the most important group within the area, has not endorsed Brainstorm’s bid for approval regardless of giving the corporate $400,000 in analysis funding. Brainstorm declined to make its full dataset out there for exterior overview, a spokesperson famous.

“The amazing testimonials we have seen online do not align with the data that Brainstorm has shared,” the group stated in an announcement.

At Wednesday’s assembly, folks with such reservations are sure to be outnumbered by appeals from ALS sufferers and their households. The FDA has obtained greater than 1,900 written feedback, many expressing outrage that NurOwn was not accredited years in the past.

“If we would’ve had NurOwn approved back when I was still walking, I believe I might still be walking today,” wrote Patricia Manhardt, who was recognized in 2020.

NurOwn is constructed from stem cells collected from sufferers’ bone marrow. The cells are processed in a lab with organic proteins designed to advertise nerve progress, after which injected into the spinal column.

In a examine of 200 sufferers, NurOwn failed to indicate a statistically vital distinction between sufferers who obtained the drug and people who obtained sham injections. Brainstorm and the educational researchers who performed the examine say the outcomes have been skewed by an unexpectedly excessive variety of sufferers with superior illness who enrolled within the trial.

ALS is measured utilizing a 48-point questionnaire that tracks features like strolling, swallowing and handwriting.

On Wednesday, researchers will inform FDA that as a result of many sufferers declined so rapidly on the size, the examine failed to indicate Nurown’s impact on development. When information from a small subset of more healthy sufferers is remoted, they argue, NurOwn considerably slowed the illness.

“The leaders in this field have said there’s more to this story than just, ‘No it doesn’t work,’” stated Dr. Anthony Windebank, a Mayo Clinic neurologist who will current on Brainstorm’s behalf.

But FDA reviewers stated Monday the corporate’s principle doesn’t clarify the examine’s failed outcomes.

Physicians who weren’t concerned within the analysis counsel regulators could be keen to make a compromise: approving NurOwn for some sufferers whereas awaiting extra definitive outcomes.

“I don’t want to lose a potential treatment for ALS, but I also don’t want to foist on the public an expensive treatment that doesn’t work,” stated Dr. Terry Heiman-Patterson of Temple University.

That compromise can be just like FDA’s method to Relyvrio final 12 months, one other ALS drug with questionable information. But in that case, the drugmaker had began its follow-up examine lengthy earlier than approval.

Brainstorm has not begun a second examine, saying it’s been unable to lift sufficient cash.

Meanwhile, FDA observers fear concerning the long-term penalties if regulators preserve accepting weaker proof from drugmakers.

“If the standards fall too low, that sends a message to industry that you don’t have to prove your drug works,” stated Holly Fernandez Lynch, a bioethicist at University of Pennsylvania. “It’s understandable why some patients would accept that. But if that becomes the regulatory standard, it can set back a field in the long term.”

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Follow Matthew Perrone on Twitter: @AP_FDAwriter

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The Associated Press Health and Science Department receives help from the Howard Hughes Medical Institute’s Science and Educational Media Group. The AP is solely liable for all content material.