Bis-6-amidino-benzothiazole Derivative that Cures Experimental Stage 1 African Trypanosomiasis with a Single Dose

J Med Chem. 2023 Sep 28;66(18):13043-13057. doi: 10.1021/acs.jmedchem.3c01051. Epub 2023 Sep 18.

Abstract

We designed and synthesized a series of symmetric bis-6-amidino-benzothiazole derivatives with aliphatic central units and evaluated their efficacy against bloodstream forms of the African trypanosome Trypanosoma brucei. Of these, a dicationic benzothiazole compound (9a) exhibited sub-nanomolar in vitro potency with remarkable selectivity over mammalian cells (>26,000-fold). Unsubstituted 5-amidine groups and a cyclohexyl spacer were the crucial determinants of trypanocidal activity. In all cases, mice treated with a single dose of 20 mg kg-1 were cured of stage 1 trypanosomiasis. The compound displayed a favorable in vitro ADME profile, with the exception of low membrane permeability. However, we found evidence that uptake by T. brucei is mediated by endocytosis, a process that results in lysosomal sequestration. The compound was also active in low nanomolar concentrations against cultured asexual forms of the malaria parasite Plasmodium falciparum. Therefore, 9a has exquisite cross-species efficacy and represents a lead compound with considerable therapeutic potential.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Benzothiazoles / pharmacology
  • Benzothiazoles / therapeutic use
  • Mammals
  • Mice
  • Trypanocidal Agents* / pharmacology
  • Trypanocidal Agents* / therapeutic use
  • Trypanosoma brucei brucei*
  • Trypanosoma*
  • Trypanosomiasis* / drug therapy
  • Trypanosomiasis, African* / drug therapy
  • Trypanosomiasis, African* / parasitology

Substances

  • Benzothiazoles
  • Trypanocidal Agents