AI Identifies Cancer Drug Vorinostat for Liver Fibrosis Treatment

DeepMind's AI-powered platform, Co-Scientist, has identified vorinostat, a cancer drug, as a promising candidate for treating liver fibrosis, a chronic condition that leads to scarring of the liver and causes over 1.4 million deaths annually. This breakthrough comes from a collaboration led by geneticist Gary Peltz at Stanford University, whose findings were published in Advanced Science on May 19, 2026.

Using Co-Scientist, Peltz tested five drug candidates on live human liver cells. Notably, two of the three AI-selected drugs showed significant efficacy, including vorinostat, which blocked 91% of a damage response pathway linked to liver fibrosis. In contrast, the two drugs identified manually by Peltz's team failed to show any benefit. This result highlights the potential of AI in sifting through vast medical literature to uncover overlooked treatment options.

Liver fibrosis, often a precursor to cirrhosis, has limited therapeutic options. Current standard care focuses on addressing underlying causes like viral hepatitis or metabolic dysfunction, but no broadly approved drugs exist to directly reverse fibrosis across all etiologies. While resmetirom (approved in 2024) and efimosfermin (granted FDA breakthrough designation in April 2026) have emerged for metabolic-associated steatohepatitis (MASH)-related fibrosis, they target specific pathways. Vorinostat’s ability to reshape gene activity suggests a new direction in treatment strategies.

DeepMind's approach aligns with the growing trend of leveraging AI in drug discovery. Earlier this year, Johns Hopkins researchers demonstrated an AI-driven liquid biopsy for detecting early-stage liver fibrosis, while preclinical studies in January 2026 showcased promising results using combination therapies to reverse fibrosis. These innovations reflect the industry's shift toward mechanism-based treatments that could fundamentally alter the progression of fibrotic diseases.

Vorinostat's potential as an anti-fibrotic agent could reshape the drug development landscape for liver diseases. With established safety profiles from its approved use in oncology, the drug may accelerate through clinical trials to address unmet needs in fibrosis treatment. If successful, it could join a new generation of therapies aimed at not just halting, but reversing, liver damage.

Looking forward, the field remains competitive with multiple Phase 2 and Phase 3 trials underway for other fibrosis-targeting agents, including efruxifermin and FXR agonists. As AI tools like Co-Scientist gain traction, the timeline for repurposing existing drugs could shorten, providing faster relief for millions of patients worldwide.