Selective inhibition of BET bromodomains

Nature. 2010 Dec 23;468(7327):1067-73. doi: 10.1038/nature09504. Epub 2010 Sep 24.

Abstract

Epigenetic proteins are intently pursued targets in ligand discovery. So far, successful efforts have been limited to chromatin modifying enzymes, or so-called epigenetic 'writers' and 'erasers'. Potent inhibitors of histone binding modules have not yet been described. Here we report a cell-permeable small molecule (JQ1) that binds competitively to acetyl-lysine recognition motifs, or bromodomains. High potency and specificity towards a subset of human bromodomains is explained by co-crystal structures with bromodomain and extra-terminal (BET) family member BRD4, revealing excellent shape complementarity with the acetyl-lysine binding cavity. Recurrent translocation of BRD4 is observed in a genetically-defined, incurable subtype of human squamous carcinoma. Competitive binding by JQ1 displaces the BRD4 fusion oncoprotein from chromatin, prompting squamous differentiation and specific antiproliferative effects in BRD4-dependent cell lines and patient-derived xenograft models. These data establish proof-of-concept for targeting protein-protein interactions of epigenetic 'readers', and provide a versatile chemical scaffold for the development of chemical probes more broadly throughout the bromodomain family.

Publication types

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

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Azirines / chemical synthesis
  • Azirines / chemistry
  • Azirines / pharmacology*
  • Binding Sites
  • Carcinoma, Squamous Cell / physiopathology
  • Cell Cycle Proteins
  • Cell Differentiation / drug effects
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Chromatin / metabolism
  • Dihydropyridines / chemical synthesis
  • Dihydropyridines / chemistry
  • Dihydropyridines / pharmacology*
  • Female
  • Humans
  • Mice
  • Mice, Nude
  • Models, Molecular*
  • Molecular Sequence Data
  • Nuclear Proteins / antagonists & inhibitors*
  • Nuclear Proteins / metabolism*
  • Protein Binding / drug effects
  • Protein Structure, Tertiary
  • Recombinant Proteins / metabolism
  • Sequence Alignment
  • Skin Neoplasms / physiopathology
  • Stereoisomerism
  • Transcription Factors / antagonists & inhibitors*
  • Transcription Factors / metabolism*

Substances

  • Azirines
  • BRD4 protein, human
  • Cell Cycle Proteins
  • Chromatin
  • Dihydropyridines
  • Nuclear Proteins
  • Recombinant Proteins
  • Transcription Factors
  • diazipine

Associated data

  • PDB/2OSS
  • PDB/3MXF
  • PDB/3ONI