The Next Blockbuster Drug May Already Exist, and AI Knows Where to Look

Artificial intelligence (AI) can play a significant role in drug repurposing, a strategy that contributes to the realization of personalized medicine, according to a study published in the Annual Review of Medicine.

The development of new drugs, which involves extensive and costly testing and research, is an important means by which medicine moves forward. However, the arduous process of new drug development, alongside the considerable risk of failure, means the pace of progress may not meet current clinical demands.

An alternative to developing entirely new classes of drugs is to repurpose existing ones whose mechanisms of action can be beneficial in more than one disease. A recent example came during the COVID-19 pandemic, when researchers explored whether existing therapies could help reduce the inflammation and immune response involved in COVID-19. Dexamethasone went on to become an established treatment, while candidates such as melatonin remained investigational.

This kind of discovery can be facilitated by AI, specifically deep learning frameworks designed to accelerate the drug discovery process. According to Fu and colleagues, AI models show promise in drug repurposing because they can combine information from the genome, proteome, transcriptome, and human interactome across diverse populations.

In other words, AI can merge the expertise of geneticists, pharmacologists, and computational scientists to identify drugs suitable for repurposing to other indications. Because these drugs are already on the market and have passed stringent safety tests, confirming their efficacy and safety for new diseases is likely to be quicker and more affordable.

The bigger picture

AI's impact on modern medicine is substantial. While AI clinical search engines such as Vera Health let healthcare professionals ask clinical questions and receive immediate, evidence-based answers, other AI tools work on the back end of medicine, in this case the discovery and development of the drugs that make quality care possible.

Reference

Fu Z, Yang Y, Chung MK, Cheng F. Artificial Intelligence to Guide Repurposing of Drugs. Annu Rev Med. 2026;77(1):381-398. doi:10.1146/annurev-med-050224-122802