Discovery of Small-Molecule Inhibitors of Ubiquitin Specific Protease 7 (USP7) Using Integrated NMR and in Silico Techniques

J Med Chem. 2017 Dec 28;60(24):10056-10070. doi: 10.1021/acs.jmedchem.7b01293. Epub 2017 Dec 8.

Abstract

USP7 is a deubiquitinase implicated in destabilizing the tumor suppressor p53, and for this reason it has gained increasing attention as a potential oncology target for small molecule inhibitors. Herein we describe the biophysical, biochemical, and computational approaches that led to the identification of 4-(2-aminopyridin-3-yl)phenol compounds described by Kategaya ( Nature 2017 , 550 , 534 - 538 ) as specific inhibitors of USP7. Fragment based lead discovery (FBLD) by NMR combined with virtual screening and re-mining of biochemical high-throughput screening (HTS) hits led to the discovery of a series of ligands that bind in the "palm" region of the catalytic domain of USP7 and inhibit its catalytic activity. These ligands were then optimized by structure-based design to yield cell-active molecules with reasonable physical properties. This discovery process not only involved multiple techniques working in concert but also illustrated a unique way in which hits from orthogonal screening approaches complemented each other for lead identification.

Publication types

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

MeSH terms

  • Aminopyridines / chemistry
  • Binding Sites
  • Catalytic Domain
  • Cell Line
  • Computer Simulation
  • Crystallography, X-Ray
  • Drug Discovery / methods
  • Drug Evaluation, Preclinical / methods
  • Enzyme Inhibitors / chemistry*
  • Enzyme Inhibitors / pharmacology*
  • Humans
  • Magnetic Resonance Spectroscopy / methods
  • Oxadiazoles / chemistry
  • Small Molecule Libraries / chemical synthesis
  • Small Molecule Libraries / chemistry
  • Small Molecule Libraries / pharmacology*
  • Ubiquitin-Specific Peptidase 7 / antagonists & inhibitors*
  • Ubiquitin-Specific Peptidase 7 / chemistry
  • Ubiquitin-Specific Peptidase 7 / metabolism

Substances

  • Aminopyridines
  • Enzyme Inhibitors
  • Oxadiazoles
  • Small Molecule Libraries
  • USP7 protein, human
  • Ubiquitin-Specific Peptidase 7