Identification of multiple structurally distinct, nonpeptidic small molecule inhibitors of protein arginine deiminase 3 using a substrate-based fragment method

J Am Chem Soc. 2015 Mar 18;137(10):3616-21. doi: 10.1021/jacs.5b00095. Epub 2015 Mar 5.

Abstract

The protein arginine deiminases (PADs) are a family of enzymes that catalyze the post-translational hydrolytic deimination of arginine residues. Four different enzymologically active PAD subtypes have been characterized and exhibit tissue-specific expression and association with a number of different diseases. In this Article we describe the development of an approach for the reliable discovery of low molecular weight, nonpeptidic fragment substrates of the PADs that then can be optimized and converted to mechanism-based irreversible PAD inhibitors. The approach is demonstrated by the development of potent and selective inhibitors of PAD3, a PAD subtype implicated in the neurodegenerative response to spinal cord injury. Multiple structurally distinct inhibitors were identified with the most potent inhibitors having >10,000 min(-1) M(-1) k(inact)/K(I) values and ≥10-fold selectivity for PAD3 over PADs 1, 2, and 4.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Drug Discovery*
  • Enzyme Inhibitors / chemistry*
  • Enzyme Inhibitors / pharmacology*
  • Humans
  • Hydantoins / chemistry
  • Hydantoins / pharmacology
  • Hydrolases / antagonists & inhibitors*
  • Isoenzymes / antagonists & inhibitors
  • Protein-Arginine Deiminases
  • Small Molecule Libraries / chemistry*
  • Small Molecule Libraries / pharmacology*
  • Substrate Specificity

Substances

  • Enzyme Inhibitors
  • Hydantoins
  • Isoenzymes
  • Small Molecule Libraries
  • Hydrolases
  • Protein-Arginine Deiminases