Molecular basis of USP7 inhibition by selective small-molecule inhibitors

Nature. 2017 Oct 26;550(7677):481-486. doi: 10.1038/nature24451. Epub 2017 Oct 18.

Abstract

Ubiquitination controls the stability of most cellular proteins, and its deregulation contributes to human diseases including cancer. Deubiquitinases remove ubiquitin from proteins, and their inhibition can induce the degradation of selected proteins, potentially including otherwise 'undruggable' targets. For example, the inhibition of ubiquitin-specific protease 7 (USP7) results in the degradation of the oncogenic E3 ligase MDM2, and leads to re-activation of the tumour suppressor p53 in various cancers. Here we report that two compounds, FT671 and FT827, inhibit USP7 with high affinity and specificity in vitro and within human cells. Co-crystal structures reveal that both compounds target a dynamic pocket near the catalytic centre of the auto-inhibited apo form of USP7, which differs from other USP deubiquitinases. Consistent with USP7 target engagement in cells, FT671 destabilizes USP7 substrates including MDM2, increases levels of p53, and results in the transcription of p53 target genes, induction of the tumour suppressor p21, and inhibition of tumour growth in mice.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Apoenzymes / antagonists & inhibitors
  • Apoenzymes / chemistry
  • Apoenzymes / metabolism
  • Cell Line, Tumor
  • Cell Proliferation / drug effects
  • Crystallography, X-Ray
  • Female
  • Humans
  • Mice
  • Models, Molecular
  • Neoplasms / drug therapy
  • Neoplasms / enzymology
  • Neoplasms / pathology
  • Piperidines / chemical synthesis
  • Piperidines / pharmacology*
  • Proto-Oncogene Proteins c-mdm2 / chemistry
  • Proto-Oncogene Proteins c-mdm2 / metabolism
  • Pyrazoles / chemical synthesis
  • Pyrazoles / pharmacology*
  • Pyrimidines / chemical synthesis
  • Pyrimidines / pharmacology*
  • Substrate Specificity
  • Transcription, Genetic / drug effects
  • Tumor Suppressor Protein p53 / metabolism
  • Ubiquitin-Specific Peptidase 7 / antagonists & inhibitors*
  • Ubiquitin-Specific Peptidase 7 / chemistry
  • Ubiquitin-Specific Peptidase 7 / metabolism
  • Ubiquitination / drug effects
  • Xenograft Model Antitumor Assays

Substances

  • Apoenzymes
  • Piperidines
  • Pyrazoles
  • Pyrimidines
  • Tumor Suppressor Protein p53
  • MDM2 protein, human
  • Proto-Oncogene Proteins c-mdm2
  • USP7 protein, human
  • Ubiquitin-Specific Peptidase 7