UF Scripps Researcher’s Drug-Discovery Method Shows Promise Against Aggressive Breast Cancer

UF Scripps Biomedical Research scientist Matthew Disney, Ph.D. has found yet another groundbreaking approach to a problem that has long vexed scientists: How to cure diseases by targeting key RNA. Until now, RNA has been an elusive target for drug discovery. Discoveries made by Disney and his collaborators have rewritten that dogma. 

The group’s recent discoveries have important, broader implications for possibly treating many now-incurable diseases. For now, most drugs work by targeting proteins from humans or infectious organisms. Developing the ability to modulate RNA, as Disney’s inventions do, potentially increases the number of diseases that can be targeted with pharmaceuticals. 

By targeting RNA — the middleman between gene and protein — factors that cause disease never get built in the first place. Disney’s latest discovery capitalizes on work by his longtime collaborator, Brian Paegel, Ph.D., of the University of California, Irvine, to bring new efficiency to the drug-discovery process. Their approach makes it possible to screen hundreds of millions of drug-RNA interactions for effectiveness. The results were published recently in the Proceedings of the National Academy of Sciences.

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