A Molecular Hybridization Approach for the Design of Potent, Highly Selective, and Brain-Penetrant N-Myristoyltransferase Inhibitors

J Med Chem. 2018 Sep 27;61(18):8374-8389. doi: 10.1021/acs.jmedchem.8b00884. Epub 2018 Sep 12.

Abstract

Crystallography has guided the hybridization of two series of Trypanosoma brucei N-myristoyltransferase (NMT) inhibitors, leading to a novel highly selective series. The effect of combining the selectivity enhancing elements from two pharmacophores is shown to be additive and has led to compounds that have greater than 1000-fold selectivity for TbNMT vs HsNMT. Further optimization of the hybrid series has identified compounds with significant trypanocidal activity capable of crossing the blood-brain barrier. By using CF-1 mdr1a deficient mice, we were able to demonstrate full cures in vivo in a mouse model of stage 2 African sleeping sickness. This and previous work provides very strong validation for NMT as a drug target for human African trypanosomiasis in both the peripheral and central nervous system stages of disease.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Acyltransferases / antagonists & inhibitors*
  • Animals
  • Blood-Brain Barrier / drug effects*
  • Cell Survival
  • Drug Design*
  • Female
  • Humans
  • Mice
  • Mice, Inbred Strains
  • Models, Molecular
  • Molecular Structure
  • Protein Conformation
  • Structure-Activity Relationship
  • Trypanocidal Agents / chemistry*
  • Trypanocidal Agents / pharmacology*
  • Trypanosoma brucei brucei / drug effects*
  • Trypanosomiasis, African / drug therapy*
  • Trypanosomiasis, African / microbiology

Substances

  • Trypanocidal Agents
  • Acyltransferases
  • glycylpeptide N-tetradecanoyltransferase