CETSA screening identifies known and novel thymidylate synthase inhibitors and slow intracellular activation of 5-fluorouracil

Nat Commun. 2016 Mar 24:7:11040. doi: 10.1038/ncomms11040.

Abstract

Target engagement is a critical factor for therapeutic efficacy. Assessment of compound binding to native target proteins in live cells is therefore highly desirable in all stages of drug discovery. We report here the first compound library screen based on biophysical measurements of intracellular target binding, exemplified by human thymidylate synthase (TS). The screen selected accurately for all the tested known drugs acting on TS. We also identified TS inhibitors with novel chemistry and marketed drugs that were not previously known to target TS, including the DNA methyltransferase inhibitor decitabine. By following the cellular uptake and enzymatic conversion of known drugs we correlated the appearance of active metabolites over time with intracellular target engagement. These data distinguished a much slower activation of 5-fluorouracil when compared with nucleoside-based drugs. The approach establishes efficient means to associate drug uptake and activation with target binding during drug discovery.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Activation, Metabolic / drug effects
  • Azacitidine / analogs & derivatives
  • Azacitidine / pharmacology
  • Biological Assay
  • Deamination / drug effects
  • Decitabine
  • Drug Evaluation, Preclinical*
  • Enzyme Inhibitors / pharmacology*
  • Fluorouracil / metabolism*
  • Humans
  • Intracellular Space / metabolism*
  • K562 Cells
  • Kinetics
  • Phosphorylation / drug effects
  • Small Molecule Libraries / analysis
  • Small Molecule Libraries / pharmacology
  • Thymidylate Synthase / antagonists & inhibitors*
  • Thymidylate Synthase / metabolism
  • Time Factors

Substances

  • Enzyme Inhibitors
  • Small Molecule Libraries
  • Decitabine
  • Thymidylate Synthase
  • Azacitidine
  • Fluorouracil