Despite the prevalence of nonalcoholic fatty liver disease (NAFLD), the most common liver disease worldwide, there are currently no approved pharmacotherapies for NAFLD. This review explores the potential and challenges of using genetic findings to enhance and expedite the development of effective treatments for NAFLD. Recent advancements in understanding the genetic factors that contribute to individual variations in NAFLD susceptibility, progression, and outcomes have opened new avenues for drug discovery. Leveraging genetic information can help identify and prioritize potential drug candidates, predict drug safety and efficacy, and explore opportunities for drug repurposing.

The drug development process for NAFLD is challenging, with few agents reaching clinical approval. To overcome this, more efficient methods to identify promising therapeutic targets are needed. The study of the human genome in large patient cohorts has significantly advanced knowledge of the genetic mechanisms underlying NAFLD. By integrating genetic insights into the drug discovery pipeline, researchers can better select targets with a higher likelihood of clinical success.

Reference: Eslam M, George J. Genetic Insights for Drug Development in NAFLD. Trends Pharmacol Sci. 2019 Jul;40(7):506-516. doi: 10.1016/j.tips.2019.05.002. Epub 2019 May 31. PMID: 31160124.