Understanding MYC: Why a Key Cancer Gene Is Hard to Stop

Researchers have long studied the MYC gene because it is linked to many cancers. MYC has been implicated in about 70% of human cancers and is described as a master regulator of cell growth, metabolism, and division. Although it drives cancer, MYC has been called 'undruggable' because the protein is intrinsically disordered, lacks a clear binding pocket, and works in the cell nucleus where many drugs cannot reach. While small molecules have failed to bind MYC directly, scientists have developed alternative approaches that are being tested.

One strategy is to target DNA enhancers that increase MYC expression; in pediatric medulloblastoma, researchers have found an enhancer that drives MYC and can be disrupted. Other teams have shown that chromatin regulators control MYC amplification, offering epigenetic targets that could limit copy-number gains. New drug platforms such as PROTACs and engineered peptides (for example, Omomyc) have shown activity against MYC in lab models, and a biotech company recently treated the first patient in a Phase 2a trial of a MYC inhibitor. These efforts are early, but they have opened several paths to try to neutralize a gene long seen as untouchable.