A powerful network of biological regulators in the cell controls the fate of nearly every protein, targeting them for destruction when needed. For years, drug developers have sought to hijack this waste-disposal system to treat disease by pursuing small molecules that work as ‘molecular glues’. These small molecules bring therapeutically relevant targets into the proximity of the proteins that tag them for degradation. Writing in Nature, Yeo et al.1 reveal that a small-molecule drug under active clinical investigation, UM171, works through this mechanism. Xie et al.2 further show that UM171 structurally and functionally mimics mutations linked to brain cancer, providing a striking example of drug design unexpectedly imitating nature.
Competing Interests
J.W.B. and P.R.P. are employees of Amgen, a biotechnology company.
