AI Drug Repurposing Discovers Potential New ALS Therapy: Approved Antidepressant Shows Neuroprotective Effects
UK AI drug discovery company BenevolentAI identified the approved antidepressant vortioxetine as a potential ALS treatment through knowledge graph analysis, with Phase II trial data showing 31% slowdown in disease progression.
On April 2, UK AI drug discovery company BenevolentAI published a paper in Nature Medicine reporting that its AI knowledge graph analysis identified the approved antidepressant vortioxetine as a potential treatment for amyotrophic lateral sclerosis (ALS). In a six-month Phase II clinical trial, 86 ALS patients taking vortioxetine showed disease progression (measured by ALSFRS-R scores) slowing by 31% compared to placebo.
BenevolentAI's AI system analyzed over 2 billion biomedical literature entries and genomic data, discovering a previously unknown intersection between vortioxetine's 5-HT receptor modulation mechanism and the BDNF signaling pathway related to motor neuron survival. This discovery is purely "drug repurposing" — vortioxetine has been globally marketed since its 2013 approval with extensive safety data, and if Phase III trials confirm efficacy, time to market could be dramatically shortened.
Globally, approximately 350,000 people have ALS, with only two currently approved drugs showing limited efficacy. BenevolentAI CEO stated the company plans to initiate Phase III clinical trials by the end of 2028.
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