Navigating a Challenging Journey: BrainStorm’s ALS Treatment Faces Obstacles, but a Beacon of Hope Shines for Future Patients

In the world of medical breakthroughs, this week’s spotlight fell squarely on amyotrophic lateral sclerosis (ALS), often referred to as Lou Gehrig’s disease. A pivotal moment unfolded as an FDA advisory committee gathered to deliberate on BrainStorm Cell Therapeutics’ experimental treatment, NurOwn. The outcome, a resounding 17-1 vote against NurOwn’s effectiveness in mild to moderate ALS, was not entirely unexpected.

Leading up to this critical meeting, the FDA had raised concerns about the therapy’s manufacturing plan and the evidence of its efficacy in the Biologics License Application. The agency even deemed the initial submission “scientifically incomplete” and criticized the manufacturing information as “grossly deficient.”

Despite their empathy for ALS patients, the panelists pointed to conflicting data and a perceived lack of efficacy, with only one vote in favor coming from Kathleen O’Sullivan-Fortin, founder of ALD Connect, who emphasized the unique and desperate circumstances faced by ALS patients.

In the United States, where approximately 25,000 individuals battle ALS, there is no cure, and existing treatments aim to alleviate symptoms. However, a ray of hope emerges from the pipeline of potential candidates. Clene’s experimental drug, for instance, showed a remarkable 49% reduction in the risk of death compared to historical ALS therapy trials.

Seelos Therapeutics is also making strides with SLS-005 (Trehalose), a candidate that aims to stabilize proteins and promote autophagy in the brain, effectively clearing toxic protein aggregations characteristic of ALS.

The eagerly anticipated top-line data from the Phase II/III trial in the HEALEY ALS Platform Trial is expected by the end of 2023, making it one of the four late-stage Neurodegenerative Trials to Watch in Q4.

Takeda, a pharmaceutical giant, has stepped into the ALS arena with a $580 million development and commercialization deal with AcuraStem for the promising PIKFYVE program. This program, which includes AS-202, an investigational antisense oligonucleotide, holds tremendous potential as a therapeutic target in ALS.

Takeda now possesses exclusive worldwide licensing rights to AS-202 and AcuraStem’s other PIKFYVE-directed assets, marking a significant leap forward in the battle against this devastating disease.

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