Allosteric inhibition of antiapoptotic MCL-1

Nat Struct Mol Biol. 2016 Jun;23(6):600-7. doi: 10.1038/nsmb.3223. Epub 2016 May 9.

Abstract

MCL-1 is an antiapoptotic BCL-2 family protein that has emerged as a major pathogenic factor in human cancer. Like BCL-2, MCL-1 bears a surface groove whose function is to sequester the BH3 killer domains of proapoptotic BCL-2 family members, a mechanism harnessed by cancer cells to establish formidable apoptotic blockades. Although drugging the BH3-binding groove has been achieved for BCL-2, translating this approach to MCL-1 has been challenging. Here, we report an alternative mechanism for MCL-1 inhibition by small-molecule covalent modification of C286 at a new interaction site distant from the BH3-binding groove. Our structure-function analyses revealed that the BH3 binding capacity of MCL-1 and its suppression of BAX are impaired by molecular engagement, a phenomenon recapitulated by C286W mutagenic mimicry in vitro and in mouse cells. Thus, we characterize an allosteric mechanism for disrupting the antiapoptotic BH3 binding activity of MCL-1, informing a new strategy for disarming MCL-1 in cancer.

Publication types

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

MeSH terms

  • Allosteric Regulation / drug effects*
  • Animals
  • Apoptosis / drug effects
  • Cell Line
  • Humans
  • Mice
  • Molecular Dynamics Simulation
  • Mutagenesis
  • Myeloid Cell Leukemia Sequence 1 Protein / chemistry*
  • Myeloid Cell Leukemia Sequence 1 Protein / genetics
  • Myeloid Cell Leukemia Sequence 1 Protein / metabolism*
  • Neoplasms / genetics
  • Neoplasms / metabolism
  • Point Mutation
  • Protein Binding / drug effects
  • Protein Conformation / drug effects
  • Protein Domains
  • Proto-Oncogene Proteins c-bcl-2 / chemistry
  • Proto-Oncogene Proteins c-bcl-2 / metabolism
  • Small Molecule Libraries / pharmacology*
  • bcl-2-Associated X Protein / chemistry
  • bcl-2-Associated X Protein / metabolism

Substances

  • MCL1 protein, human
  • Myeloid Cell Leukemia Sequence 1 Protein
  • Proto-Oncogene Proteins c-bcl-2
  • Small Molecule Libraries
  • bcl-2-Associated X Protein