NEW HAVEN, CT — A new study integrates genetic and electronic health record (EHR) data to help identify and evaluate drugs that can be repurposed to treat alcohol-use disorder (AUD).
Even though alcohol-use disorder (AUD) is a chronic, relapsing condition and a major public health problem, few medications are approved to treat AUD, and those available show limited efficacy. A study led by the Yale University School of Medicine pointed out that drug repurposing is a cost-effective strategy to identify novel therapeutic uses for existing medications.
The Uniformed Services University of the Health Sciences and the Henry M. Jackson Foundation for the Advancement of Military Medicine Inc., both in Bethesda, MD, as well as VAMCs in West Haven, CT, and Philadelphia participated in the study, along with other research institutions.
The study team said its approach included the following:
- alcohol-associated gene identification and biological network generation;
- mapping drugs to target proteins;
- filtering promising repurposing candidates; and
- an exemplar pharmacoepidemiologic analysis of the effect of an identified drug (i.e., baclofen) on alcohol consumption.
Results published in Alcohol, Clinical and Experimental Research indicated that linking loci to genes from a genome-wide association study (GWAS) of problematic alcohol use identified 94 genes, which were expanded to 327 alcohol-related genes through network-based analyses. Across those analyses, 52 genes were linked to 195 FDA-approved drugs, including four already approved or used off-label to treat AUD. After filtering for safety, relevance and data availability, 26 candidate drugs, including baclofen, were selected for further evaluation.1
The researchers added that an evaluation of the real-world effectiveness of baclofen using national EHR data from the VA provided evidence that baclofen-exposed patients reduced alcohol consumption more than propensity-score-matched unexposed patients.
“This approach, which aligns genomic findings with real-world clinical data, provides an efficient method for identifying promising drug repurposing candidates and prioritizing those that merit evaluation in randomized trials to ultimately advance pharmacotherapies for AUD,” the authors concluded.
- Rentsch CT, Malone SG, Shi M, Setzer MR, Piserchia Z, Winterlind EL, Farokhnia M, Tazare J, Justice AC, Fiellin DA, Leggio L, Kranzler HR, Gray JC. Bridging genomics and pharmacoepidemiology to expand treatment options for alcohol use disorder. Alcohol Clin Exp Res (Hoboken). 2026 Mar;50(3):e70247. doi: 10.1111/acer.70247. PMID: 41811207.
