Inhibition of RAS function through targeting an allosteric regulatory site

Nat Chem Biol. 2017 Jan;13(1):62-68. doi: 10.1038/nchembio.2231. Epub 2016 Nov 7.

Abstract

RAS GTPases are important mediators of oncogenesis in humans. However, pharmacological inhibition of RAS has proved challenging. Here we describe a functionally critical region, located outside the effector lobe of RAS, that can be targeted for inhibition. We developed NS1, a synthetic binding protein (monobody) that bound with high affinity to both GTP- and GDP-bound states of H-RAS and K-RAS but not N-RAS. NS1 potently inhibited growth factor signaling and oncogenic H-RAS- and K-RAS-mediated signaling and transformation but did not block oncogenic N-RAS, BRAF or MEK1. NS1 bound the α4-β6-α5 region of RAS, which disrupted RAS dimerization and nanoclustering and led to blocking of CRAF-BRAF heterodimerization and activation. These results establish the importance of the α4-β6-α5 interface in RAS-mediated signaling and define a previously unrecognized site in RAS for inhibiting RAS function.

MeSH terms

  • Allosteric Site / drug effects*
  • Animals
  • Antibodies, Monoclonal / chemistry
  • Antibodies, Monoclonal / metabolism*
  • Antibodies, Monoclonal / pharmacology*
  • COS Cells
  • Cells, Cultured
  • Chlorocebus aethiops
  • HEK293 Cells
  • Humans
  • Mice
  • NIH 3T3 Cells
  • ras Proteins / antagonists & inhibitors*
  • ras Proteins / chemistry*
  • ras Proteins / metabolism

Substances

  • Antibodies, Monoclonal
  • ras Proteins