Chemical remodeling of a cellular chaperone to target the active state of mutant KRAS

Science. 2023 Aug 18;381(6659):794-799. doi: 10.1126/science.adg9652. Epub 2023 Aug 17.

Abstract

The discovery of small-molecule inhibitors requires suitable binding pockets on protein surfaces. Proteins that lack this feature are considered undruggable and require innovative strategies for therapeutic targeting. KRAS is the most frequently activated oncogene in cancer, and the active state of mutant KRAS is such a recalcitrant target. We designed a natural product-inspired small molecule that remodels the surface of cyclophilin A (CYPA) to create a neomorphic interface with high affinity and selectivity for the active state of KRASG12C (in which glycine-12 is mutated to cysteine). The resulting CYPA:drug:KRASG12C tricomplex inactivated oncogenic signaling and led to tumor regressions in multiple human cancer models. This inhibitory strategy can be used to target additional KRAS mutants and other undruggable cancer drivers. Tricomplex inhibitors that selectively target active KRASG12C or multiple RAS mutants are in clinical trials now (NCT05462717 and NCT05379985).

MeSH terms

  • Biological Products* / chemistry
  • Biological Products* / pharmacology
  • Biological Products* / therapeutic use
  • Cyclophilin A* / chemistry
  • Cyclophilin A* / metabolism
  • Cysteine / chemistry
  • Cysteine / genetics
  • Humans
  • Immunophilins* / chemistry
  • Immunophilins* / metabolism
  • Molecular Chaperones* / chemistry
  • Molecular Chaperones* / metabolism
  • Neoplasms* / drug therapy
  • Neoplasms* / genetics
  • Proto-Oncogene Proteins p21(ras)* / antagonists & inhibitors
  • Proto-Oncogene Proteins p21(ras)* / chemistry
  • Proto-Oncogene Proteins p21(ras)* / genetics
  • Signal Transduction

Substances

  • Biological Products
  • Cysteine
  • KRAS protein, human
  • Molecular Chaperones
  • Proto-Oncogene Proteins p21(ras)
  • Cyclophilin A
  • Immunophilins

Associated data

  • ClinicalTrials.gov/NCT05379985
  • ClinicalTrials.gov/NCT05462717