BrainStorm Cell Therapeutics Hail Mary with FDA on Nurown for ALS

BrainStorm Cell Therapeutics is developing Nurown, an investigational therapy intended for treating ALS.

I recently posted about BrainStorm and its prospects for providing real hope on stem cells for ALS.

I see it as a long shot. The ALS community needs real hope, but one challenge is determining how good clinical data have to be to allow FDA approval.

BrainStorm Cell Therapeutics, NurOwn
MSF-NTF cells also known as NurOwn stained to show production of GDNF. BrainStorm image.

BrainStorm Cell Therapeutics pushes back on FDA decision

There are many factors impacting the firm’s efforts to get Nurown approved including working with the FDA. This week an agency advisory committee meeting will be held on Nurown. 

The September 27th FDA meeting is the result of BrainStorm Cell Therapeutics taking an unusual tactic. To try to get around what seems to be a general lack of enthusiasm at the FDA, the company has done something called “file over protest.”

I hadn’t heard of this before so looked it up. On the FDA file over protest info page you can get all the details on this mechanism. In a nutshell, the FDA has refused to file an action on Nurown. BrainStorm disagrees with this refusal so is taking a shot at protesting it.

Given all we know about the FDA’s view of Nurown and the data about it, this protest seems like a Hail Mary. The FDA documents released yesterday for this upcoming ALS advisory meeting are consistent with that. E.g. there is this devastating verbiage from the agency (emphasis mine):

“FDA conducted a filing review and determined that a substantive review could not be performed, because the BLA submission was scientifically incomplete and grossly deficient. Critical clinical and manufacturing deficiencies were identified.”

I asked around about this file under protest mechanism and it seems somewhat obscure. I wonder how often that approach  succeeds with the FDA?

Nurown data

How did Nurown, also known as Debamestrocel, get to this point?

Their data weren’t very strong or consistent. They turned to post-hoc analysis. I’m generally skeptical of ad-hoc analyses when primary and secondary endpoints have failed. Such analyses can sometimes pick up real stuff, but often they focus on outcomes that end up never being reproducible. That happens a lot in the cell therapy and stem cell arena.

Based on the negative trial data an the FDA’s language, overall I’m fairly pessimistic about the future of Nurown for ALS. If it consistently and significantly delayed disease progression in specific subgroups of ALS patients, even if not in all, then that could be a worthwhile victory for patients and the field.

It’s just very uncertain if this is a concrete possibility moving forward or more like a blip in post-hoc analysis. Investors have concerns as BrainStorm Cell Therapeutics stock is down about 90% in the past twelve months.

FDA standards and patients

Still, Nurown could end up approved despite all of this. On that front, there’s also concern about the FDA lowering drug standards.

Matt Perrone at the AP has a new piece on these recent Nurown developments as well. From the piece:

“If the standards fall too low, that sends a message to industry that you don’t have to prove your drug works,” said 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.”

FDA advisory meetings have sometimes yielded surprises so it’ll be interesting to see how the Nurown one goes this week. The company has thousands of patients backing it in this effort. Patient advocates can have a powerful influence on the FDA.

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