Discovery of potent and selective inhibitors of Trypanosoma brucei ornithine decarboxylase

J Biol Chem. 2010 May 28;285(22):16771-81. doi: 10.1074/jbc.M109.081588. Epub 2010 Mar 10.

Abstract

Human African trypanosomiasis, caused by the eukaryotic parasite Trypanosoma brucei, is a serious health problem in much of central Africa. The only validated molecular target for treatment of human African trypanosomiasis is ornithine decarboxylase (ODC), which catalyzes the first step in polyamine metabolism. Here, we describe the use of an enzymatic high throughput screen of 316,114 unique molecules to identify potent and selective inhibitors of ODC. This screen identified four novel families of ODC inhibitors, including the first inhibitors selective for the parasitic enzyme. These compounds display unique binding modes, suggesting the presence of allosteric regulatory sites on the enzyme. Docking of a subset of these inhibitors, coupled with mutagenesis, also supports the existence of these allosteric sites.

Publication types

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

MeSH terms

  • Allosteric Site
  • Animals
  • Automation
  • Chemistry, Pharmaceutical / methods
  • Drug Design
  • Enzyme Inhibitors / chemical synthesis
  • Enzyme Inhibitors / pharmacology*
  • Humans
  • Malate Dehydrogenase / chemistry
  • Mutagenesis, Site-Directed
  • Ornithine Decarboxylase Inhibitors*
  • Phosphoenolpyruvate Carboxylase / chemistry
  • Protein Binding
  • Trypanocidal Agents / pharmacology
  • Trypanosoma brucei brucei / enzymology*
  • Trypanosomiasis, African / drug therapy

Substances

  • Enzyme Inhibitors
  • Ornithine Decarboxylase Inhibitors
  • Trypanocidal Agents
  • Malate Dehydrogenase
  • Phosphoenolpyruvate Carboxylase