Novel small molecule inhibitors of botulinum neurotoxin A metalloprotease activity

Biochem Biophys Res Commun. 2003 Oct 10;310(1):84-93. doi: 10.1016/j.bbrc.2003.08.112.

Abstract

Botulinum neurotoxins (BoNTs) are among the most lethal biological substances to have been weaponized and are listed as biodefense category A agents. Currently, no small molecule (non-peptidic) therapeutics exist to counter this threat; hence, identifying and developing compounds that inhibit BoNTs is a high priority. In the present study, a high-throughput assay was used to identify small molecules that inhibit the metalloprotease activity of BoNT serotype A light chain (BoNT/A LC). All inhibitors were further verified using a HPLC-based assay. Conformational analyses of these compounds, in conjunction with molecular docking studies, were used to predict structural features that contribute to inhibitor binding and potency. Based on these results, a common pharmacophore for BoNT/A LC inhibitors is proposed. This is the first study to report small molecules (non-peptidics) that inhibit BoNT/A LC metalloprotease activity in the low microM range.

Publication types

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

MeSH terms

  • Botulinum Toxins / antagonists & inhibitors*
  • Metalloproteases / antagonists & inhibitors*
  • Protease Inhibitors / pharmacology*

Substances

  • Protease Inhibitors
  • Metalloproteases
  • Botulinum Toxins