FDA Reevaluating ALS Stem Cell Data following Citizen Petition – NurOwn from BrainStorm Cell Therapeutics
The FDAs new faces are feeling the pressure from patients and advocates on speeding up potential ALS therapies
What is NurOwn?
ALS is a devastating disease, with essentially no answers from today’s medical system. BrainStorm Cell Therapeutics, an Israel-based biotech company, developed a potential therapy called NurOwn. It’s an autologous (from the patient’s own body) culture-expanded (a process where they multiply the stem cells in a lab) mesenchymal stem cell therapy, grown in the lab to increase secretion of neurotrophic factors (NTFs).
English? They take bone marrow from the patient, grow and multiply the stem cells in a lab, and boost their ability to release healing proteins that may help protect and repair nerve cells damaged by ALS.
Trial History
The therapy looked promising in Phase 1 & 2, which had the ALS communities very excited. Unfortunately, it failed to meet the primary endpoints in Phase 3, and thus did not receive approval.
However, there has been some criticism of the trial design, and the situation is unique in that there aren’t other options for ALS. Not every patient feels this way, but there are plenty on social media who would gladly take a tiny chance at healing, and feel the FDA should act with more urgency, before they’re gone.
There is an expanded access program, in which 90% of patients survived for over five years, compared to the typical 10%. It should be noted that the one patient death was from euthanasia, and it’s a pretty small cohort. Additionally, in some of the ALS Facebook groups, many patients who participated in the NurOwn Phase 3 feel they received NurOwn (not placebo) and anecdotally feel they’ve improved.
What’s going on now?
For those who believe NurOwn may hold an answer, it likely feels like a fight for their life. So much so that they’ve put together a citizens’ petition to the FDA to reevaluate the trial data, in addition to a few articles floating around, increasing the pressure on the FDA (see Navy Fighter Pilot with ALS fights for his access to NurOwn). There are even buttons available for order:
As you know, the FDA has had quite the facelift. With RFK, Dr. Makary, and others at the helm, will they make some sort of exception? There have been calls for a new approval system, similar to Japan’s conditional approval system, which allows therapies into the market before full efficacy data is evaluated. The FDA has also seemingly integrated AI into its approval workflows, is decreasing or eliminating the use of animal models in favor of things like organoid testing, and appears to be pro-stem cell.
In my opinion, these are hefty tailwinds for BrainStorm, and they have a good shot. However, given the stem cell industry’s history, I feel they really need to get this right. Allowing this therapy into the market will almost certainly be ammunition for a wide variety of therapies seeking the same. If they make an exception here, I predict other companies will take a page out of this book and apply pressure similarly. This may be fine, as long as we don’t cut corners on science.
Additionally, if they get it wrong and say NurOwn doesn’t work or harms patients, that would backfire at a critical time for the industry, potentially setting it back another few years as a whole.
Nonetheless, BrainStorm has acknowledged the petition and won’t stop their latest Phase 3b ENDURANCE trial of NurOwn, being conducted under the FDA’s Special Protocol Assessment (SPA). The goal of this trial is to generate further robust data on NurOwn’s efficacy and safety in early-stage ALS patients. Additionally, BrainStorm says it supports exploring potential regulatory pathways that could facilitate appropriate access to NurOwn for patients with ALS.
Bob Dagher, M.D., Chief Medical Officer at BrainStorm, stated, “A new review of the NurOwn data offers an important chance to ensure all available evidence is fully considered. We remain dedicated to rigorous science and to increasing access for ALS patients in need.”
I imagine that the FDA will take a look, but they may wait until there is some sort of early/mid-update on the Phase 3b results before making a decision. This follows Florida’s new stem cell therapy bill, which allows physicians to administer perinatal tissue products (with a few caveats).
