Tumours with class 3 BRAF mutants are sensitive to the inhibition of activated RAS

Nature. 2017 Aug 10;548(7666):234-238. doi: 10.1038/nature23291. Epub 2017 Aug 2.

Abstract

Approximately 200 BRAF mutant alleles have been identified in human tumours. Activating BRAF mutants cause feedback inhibition of GTP-bound RAS, are RAS-independent and signal either as active monomers (class 1) or constitutively active dimers (class 2). Here we characterize a third class of BRAF mutants-those that have impaired kinase activity or are kinase-dead. These mutants are sensitive to ERK-mediated feedback and their activation of signalling is RAS-dependent. The mutants bind more tightly than wild-type BRAF to RAS-GTP, and their binding to and activation of wild-type CRAF is enhanced, leading to increased ERK signalling. The model suggests that dysregulation of signalling by these mutants in tumours requires coexistent mechanisms for maintaining RAS activation despite ERK-dependent feedback. Consistent with this hypothesis, melanomas with these class 3 BRAF mutations also harbour RAS mutations or NF1 deletions. By contrast, in lung and colorectal cancers with class 3 BRAF mutants, RAS is typically activated by receptor tyrosine kinase signalling. These tumours are sensitive to the inhibition of RAS activation by inhibitors of receptor tyrosine kinases. We have thus defined three distinct functional classes of BRAF mutants in human tumours. The mutants activate ERK signalling by different mechanisms that dictate their sensitivity to therapeutic inhibitors of the pathway.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Cell Line, Tumor
  • Enzyme Activation / drug effects
  • Extracellular Signal-Regulated MAP Kinases / metabolism
  • Female
  • Humans
  • Indoles / pharmacology
  • MAP Kinase Signaling System / drug effects
  • Melanoma / enzymology*
  • Melanoma / genetics*
  • Mice
  • Mitogen-Activated Protein Kinase Kinases / metabolism
  • Mutation*
  • NIH 3T3 Cells
  • Neurofibromatosis 1 / genetics
  • Oncogene Protein p21(ras) / antagonists & inhibitors*
  • Oncogene Protein p21(ras) / metabolism
  • Protein Multimerization
  • Proto-Oncogene Proteins B-raf / genetics*
  • Pyridones / pharmacology
  • Pyrimidinones / pharmacology
  • Sulfonamides / pharmacology
  • Vemurafenib
  • Xenograft Model Antitumor Assays

Substances

  • Indoles
  • Pyridones
  • Pyrimidinones
  • Sulfonamides
  • Vemurafenib
  • trametinib
  • Proto-Oncogene Proteins B-raf
  • Extracellular Signal-Regulated MAP Kinases
  • Mitogen-Activated Protein Kinase Kinases
  • Oncogene Protein p21(ras)