Genomic analysis identifies acamprosate as a candidate treatment for C9orf72-related ALS and FTD
Original source
Mechanism-free repurposing of drugs for C9orf72-related ALS/FTD using large-scale genomic data. (opens in a new tab)Compass summarised this from the study's abstract.
Study details
- Studied in
- Human
Related topics
A genomic drug-repurposing analysis identified acamprosate as a potential treatment for people carrying C9orf72 repeat expansions linked to amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). The drug showed neuroprotective effects in cell models, with effects comparable to riluzole in those tests.
Why this matters
This finding is relevant to people with C9orf72-related ALS or FTD because acamprosate is an existing drug that could potentially be studied for a new use. However, the evidence reported here comes from genomic analysis and cell models, not treatment in people, so it does not currently change clinical care.
Limitations and context
The study used genomic data from 41,273 people with ALS or without ALS and 1,516 C9orf72 carriers, then tested the candidate in cell models. The source does not report a clinical trial or evidence of benefit or safety in people with ALS or FTD. Further clinical studies would be needed to determine whether acamprosate helps patients.