AB Science has released a medRxiv preprint detailing a significant analysis of long-term survivors of amyotrophic lateral sclerosis (ALS) treated with masitinib at a dosage of 4.5 mg/kg/day in the phase 2b/3 AB10015 study. The analysis, which included 130 masitinib-treated patients, revealed a 5-year survival rate of 42.3% from disease onset, with this figure increasing to 52.9% in patients who had not experienced a complete loss of functionality prior to treatment. These findings contrast sharply with historical benchmarks of approximately 23.5%, highlighting the potential of masitinib in extending survival for ALS patients.

The significance of these findings lies in the reported median overall survival of 121 months among 55 long-term survivors, which exceeds the 42 months predicted by the ENCALS model, resulting in a 79-month residual median survival gain. Furthermore, nearly 49% of these survivors were able to maintain a satisfactory quality of life without the need for mechanical assistance. Masitinib acts as a tyrosine kinase inhibitor, specifically targeting microglial and mast cell activity, which may play a crucial role in the neuroinflammatory processes associated with ALS.

These results underscore the therapeutic potential of masitinib and pave the way for a prospective confirmatory trial, AB23005, which aims to randomize 408 patients to receive either masitinib in combination with riluzole or riluzole plus placebo. This approach could significantly shift current research paradigms by validating the efficacy of targeted therapies in ALS and potentially accelerating drug development timelines for similar neurodegenerative conditions.

Source: longevity.technology