PAINS in the assay: chemical mechanisms of assay interference and promiscuous enzymatic inhibition observed during a sulfhydryl-scavenging HTS

J Med Chem. 2015 Mar 12;58(5):2091-113. doi: 10.1021/jm5019093. Epub 2015 Feb 21.

Abstract

Significant resources in early drug discovery are spent unknowingly pursuing artifacts and promiscuous bioactive compounds, while understanding the chemical basis for these adverse behaviors often goes unexplored in pursuit of lead compounds. Nearly all the hits from our recent sulfhydryl-scavenging high-throughput screen (HTS) targeting the histone acetyltransferase Rtt109 were such compounds. Herein, we characterize the chemical basis for assay interference and promiscuous enzymatic inhibition for several prominent chemotypes identified by this HTS, including some pan-assay interference compounds (PAINS). Protein mass spectrometry and ALARM NMR confirmed these compounds react covalently with cysteines on multiple proteins. Unfortunately, compounds containing these chemotypes have been published as screening actives in reputable journals and even touted as chemical probes or preclinical candidates. Our detailed characterization and identification of such thiol-reactive chemotypes should accelerate triage of nuisance compounds, guide screening library design, and prevent follow-up on undesirable chemical matter.

Publication types

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

MeSH terms

  • Biological Assay / standards*
  • Drug Design
  • Drug Discovery*
  • Drug Evaluation, Preclinical
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacology*
  • False Positive Reactions
  • High-Throughput Screening Assays*
  • Histone Acetyltransferases / antagonists & inhibitors*
  • Humans
  • Molecular Structure
  • Nuclear Magnetic Resonance, Biomolecular
  • Sulfhydryl Compounds / chemistry*

Substances

  • Enzyme Inhibitors
  • Sulfhydryl Compounds
  • Histone Acetyltransferases