Our Position on NurOwn

We are urgently working to find new treatments and cures for ALS and are currently funding over 168 research projects in 12 countries. We work with many pharmaceutical companies that have attempted to bring new treatments to market – some successful and some unsuccessful. We work with drug companies by providing them grant funding or collaborating with them on public-private partnerships around specific ALS initiatives

We invited BrainStorm Cell Therapeutics in 2020 to submit a proposal for biomarkers measurement as part of the phase 3 trial for NurOwn. The proposal was reviewed by independent experts, and we worked with the BrainStorm team to incorporate the advice of these experts into their experimental plan. Based on the recommendation from this review and approval from our research committee, we funded BrainStorm for a total of $500,000.

At the end of this review and funding decision, I AM ALS partnered with us and provided $100,000 while we contributed $400,000 towards the grant. As far as we know, we are the largest patient advocacy funder of Brainstorm’s ALS program on NurOwn.

On June 6, 2023, BrainStorm announced that its advisory committee meeting (AdComm) for NurOwn will take place on September 27, 2023. It also announced that its PDUFA date, which is the date by which the FDA must decide whether to approve a treatment, is December 8, 2023

We only make recommendations on drug approvals after seeking independent peer review (as described here). Specifically, we ask sponsors to share data that will be submitted to the FDA and engage the recommendations of external experts who are not conflicted in any way with the program or the sponsor.

(Consistent with that policy, we consulted with independent experts and worked with Amylyx and Biogen to review FDA package data for AMX0035 (Relyvrio) and tofersen (Qalsody) before taking any stance on advocating at the AdComm and urging for their approval.)

UPDATE – APRIL 2024: Relyvrio was voluntarily withdrawn from the market by Amylyx following a phase 3 trial that failed to show it was effective. The ALS Association stands by its decision to push for early approval of Relyvrio given the promising phase 2 trial data and the safety of the treatment. At the time, we said that if it turns out to be ineffective, at worst, people living with ALS would have taken an ineffective therapy without risk of harm. If it was indeed effective, delaying access would have meant that people living with ALS would have lost two years of being able to take a life-extending therapy. In the interests of transparency and education, we are leaving this information up for future reference. People living with ALS need life-saving treatments and we are working as urgently as possible to advance the many more potential treatments in clinical trials.

After BrainStorm shared that its Phase 3 trial of NurOwn did not meet its primary or secondary endpoints, we have consistently requested access to the full data package so we could try to better understand its effect on people living with ALS. The amazing testimonials we have seen online do not align with the data that BrainStorm has shared with us or has been published in peer-reviewed publications.

We have an obligation to the community we serve to be vigilant and data-driven, and our approach has served the ALS community well in recent FDA reviews. Until we have the opportunity to conduct an independent review, we cannot take a position for or against approval of NurOwn. If Brainstorm wishes us to make any statements to the FDA, we encourage them to go through this independent review process

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