Sterol 14α-Demethylase Structure-Based Optimization of Drug Candidates for Human Infections with the Protozoan Trypanosomatidae

J Med Chem. 2018 Dec 13;61(23):10910-10921. doi: 10.1021/acs.jmedchem.8b01671. Epub 2018 Nov 30.

Abstract

Sterol 14α-demethylases (CYP51) are cytochrome P450 enzymes essential for sterol biosynthesis in eukaryotes and therapeutic targets for antifungal azoles. Multiple attempts to repurpose antifungals for treatment of human infections with protozoa (Trypanosomatidae) have been undertaken, yet so far none of them have revealed sufficient efficacy. VNI and its derivative VFV are two potent experimental inhibitors of Trypanosomatidae CYP51, effective in vivo against Chagas disease, visceral leishmaniasis, and sleeping sickness and currently under consideration as antiprotozoal drug candidates. However, VNI is less potent against Leishmania and drug-resistant strains of Trypanosoma cruzi and VFV, while displaying a broader spectrum of antiprotozoal activity, and is metabolically less stable. In this work we have designed, synthesized, and characterized a set of close analogues and identified two new compounds (7 and 9) that exceed VNI/VFV in their spectra of antiprotozoal activity, microsomal stability, and pharmacokinetics (tissue distribution in particular) and, like VNI/VFV, reveal no acute toxicity.

Publication types

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

MeSH terms

  • 14-alpha Demethylase Inhibitors / chemistry*
  • 14-alpha Demethylase Inhibitors / metabolism
  • 14-alpha Demethylase Inhibitors / pharmacology*
  • 14-alpha Demethylase Inhibitors / therapeutic use
  • Antiprotozoal Agents / chemistry
  • Antiprotozoal Agents / metabolism
  • Antiprotozoal Agents / pharmacology
  • Antiprotozoal Agents / therapeutic use
  • Chagas Disease / drug therapy*
  • Drug Design*
  • Drug Stability
  • Humans
  • Microsomes / metabolism
  • Models, Molecular
  • Protein Conformation
  • Sterol 14-Demethylase / chemistry
  • Sterol 14-Demethylase / metabolism*
  • Trypanosoma cruzi / drug effects*
  • Trypanosoma cruzi / physiology*

Substances

  • 14-alpha Demethylase Inhibitors
  • Antiprotozoal Agents
  • Sterol 14-Demethylase