Cyanotriazoles are selective topoisomerase II poisons that rapidly cure trypanosome infections

Science. 2023 Jun 30;380(6652):1349-1356. doi: 10.1126/science.adh0614. Epub 2023 Jun 29.

Abstract

Millions who live in Latin America and sub-Saharan Africa are at risk of trypanosomatid infections, which cause Chagas disease and human African trypanosomiasis (HAT). Improved HAT treatments are available, but Chagas disease therapies rely on two nitroheterocycles, which suffer from lengthy drug regimens and safety concerns that cause frequent treatment discontinuation. We performed phenotypic screening against trypanosomes and identified a class of cyanotriazoles (CTs) with potent trypanocidal activity both in vitro and in mouse models of Chagas disease and HAT. Cryo-electron microscopy approaches confirmed that CT compounds acted through selective, irreversible inhibition of trypanosomal topoisomerase II by stabilizing double-stranded DNA:enzyme cleavage complexes. These findings suggest a potential approach toward successful therapeutics for the treatment of Chagas disease.

MeSH terms

  • Animals
  • Chagas Disease* / drug therapy
  • Cryoelectron Microscopy
  • DNA Topoisomerases, Type II / metabolism
  • Drug Evaluation, Preclinical
  • Humans
  • Mice
  • Topoisomerase II Inhibitors* / chemistry
  • Topoisomerase II Inhibitors* / pharmacology
  • Topoisomerase II Inhibitors* / therapeutic use
  • Triazoles* / chemistry
  • Triazoles* / pharmacology
  • Triazoles* / therapeutic use
  • Trypanosoma* / drug effects
  • Trypanosomiasis, African* / drug therapy

Substances

  • DNA Topoisomerases, Type II
  • Topoisomerase II Inhibitors
  • Triazoles