This article discusses the innovative approach of using small molecule drugs to bestow new functions on proteins rather than merely inhibiting them. Specifically, it highlights UM171, a drug in clinical trials that operates via a 'molecular glue' mechanism, linking therapeutic targets with degradation proteins to control their fate. Additionally, research shows that UM171 mimics brain cancer-linked mutations, showcasing a significant advance in drug development that reflects nature's strategies. This discovery may shape future rational drug design, emphasizing functional enhancement over inhibition.
For years, drug developers have sought to hijack this waste-disposal system to treat disease by pursuing small molecules that work as 'molecular glues'.
Writing in Nature, Yeo et al. reveal that a small-molecule drug under active clinical investigation, UM171, works through this mechanism.
This small molecule imbues a protein with a new function, suggesting a novel mechanism that could significantly inform rational drug design.
Xie et al. further show that UM171 structurally and functionally mimics mutations linked to brain cancer, providing a striking example of drug design imitating nature.
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