Potent and selective bivalent inhibitors of BET bromodomains

Nat Chem Biol. 2016 Dec;12(12):1097-1104. doi: 10.1038/nchembio.2210. Epub 2016 Oct 24.

Abstract

Proteins of the bromodomain and extraterminal (BET) family, in particular bromodomain-containing protein 4 (BRD4), are of great interest as biological targets. BET proteins contain two separate bromodomains, and existing inhibitors bind to them monovalently. Here we describe the discovery and characterization of probe compound biBET, capable of engaging both bromodomains simultaneously in a bivalent, in cis binding mode. The evidence provided here was obtained in a variety of biophysical and cellular experiments. The bivalent binding results in very high cellular potency for BRD4 binding and pharmacological responses such as disruption of BRD4-mediator complex subunit 1 foci with an EC50 of 100 pM. These compounds will be of considerable utility as BET/BRD4 chemical probes. This work illustrates a novel concept in ligand design-simultaneous targeting of two separate domains with a drug-like small molecule-providing precedent for a potentially more effective paradigm for developing ligands for other multi-domain proteins.

MeSH terms

  • Apoptosis / drug effects
  • Cell Cycle Proteins
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Crystallography, X-Ray
  • Dose-Response Relationship, Drug
  • Humans
  • Ligands
  • Models, Molecular
  • Molecular Structure
  • Nuclear Proteins / antagonists & inhibitors*
  • Nuclear Proteins / chemistry*
  • Nuclear Proteins / metabolism
  • Protein Domains / drug effects*
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / pharmacology*
  • Structure-Activity Relationship
  • Substrate Specificity
  • Transcription Factors / antagonists & inhibitors*
  • Transcription Factors / chemistry*
  • Transcription Factors / metabolism

Substances

  • BRD4 protein, human
  • Cell Cycle Proteins
  • Ligands
  • Nuclear Proteins
  • Small Molecule Libraries
  • Transcription Factors

Associated data

  • PubChem-Substance/318127336
  • PubChem-Substance/318127337
  • PubChem-Substance/318127338
  • PubChem-Substance/318127339
  • PubChem-Substance/318127340
  • PubChem-Substance/318127341
  • PubChem-Substance/318127342
  • PubChem-Substance/318127343