Plasticity in binding confers selectivity in ligand-induced protein degradation

Nat Chem Biol. 2018 Jul;14(7):706-714. doi: 10.1038/s41589-018-0055-y. Epub 2018 Jun 11.

Abstract

Heterobifunctional small-molecule degraders that induce protein degradation through ligase-mediated ubiquitination have shown considerable promise as a new pharmacological modality. However, we currently lack a detailed understanding of the molecular basis for target recruitment and selectivity, which is critically required to enable rational design of degraders. Here we utilize a comprehensive characterization of the ligand-dependent CRBN-BRD4 interaction to demonstrate that binding between proteins that have not evolved to interact is plastic. Multiple X-ray crystal structures show that plasticity results in several distinct low-energy binding conformations that are selectively bound by ligands. We demonstrate that computational protein-protein docking can reveal the underlying interprotein contacts and inform the design of a BRD4 selective degrader that can discriminate between highly homologous BET bromodomains. Our findings that plastic interprotein contacts confer selectivity for ligand-induced protein dimerization provide a conceptual framework for the development of heterobifunctional ligands.

Publication types

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

MeSH terms

  • Acetamides / chemistry
  • Acetamides / pharmacology*
  • Adaptor Proteins, Signal Transducing
  • Binding Sites / drug effects
  • Cell Cycle Proteins
  • Crystallography, X-Ray
  • Dose-Response Relationship, Drug
  • Humans
  • Ligands
  • Models, Molecular
  • Molecular Conformation
  • Nuclear Proteins / chemistry
  • Nuclear Proteins / metabolism*
  • Peptide Hydrolases / chemistry
  • Peptide Hydrolases / metabolism*
  • Thalidomide / chemistry
  • Thalidomide / pharmacology*
  • Thiophenes / chemistry
  • Thiophenes / pharmacology*
  • Transcription Factors / chemistry
  • Transcription Factors / metabolism*
  • Ubiquitin-Protein Ligases

Substances

  • Acetamides
  • Adaptor Proteins, Signal Transducing
  • BRD4 protein, human
  • CRBN protein, human
  • Cell Cycle Proteins
  • Ligands
  • Nuclear Proteins
  • Thiophenes
  • Transcription Factors
  • Thalidomide
  • Ubiquitin-Protein Ligases
  • Peptide Hydrolases