A kalihinol analog disrupts apicoplast function and vesicular trafficking in P. falciparum malaria

Science. 2024 Sep 27;385(6716):eadm7966. doi: 10.1126/science.adm7966. Epub 2024 Sep 27.

Abstract

We report the discovery of MED6-189, an analog of the kalihinol family of isocyanoterpene natural products that is effective against drug-sensitive and drug-resistant Plasmodium falciparum strains, blocking both asexual replication and sexual differentiation. In vivo studies using a humanized mouse model of malaria confirm strong efficacy of the compound in animals with no apparent hemolytic activity or toxicity. Complementary chemical, molecular, and genomics analyses revealed that MED6-189 targets the parasite apicoplast and acts by inhibiting lipid biogenesis and cellular trafficking. Genetic analyses revealed that a mutation in PfSec13, which encodes a component of the parasite secretory machinery, reduced susceptibility to the drug. Its high potency, excellent therapeutic profile, and distinctive mode of action make MED6-189 an excellent addition to the antimalarial drug pipeline.

MeSH terms

  • Animals
  • Antimalarials* / chemistry
  • Antimalarials* / pharmacology
  • Apicoplasts* / drug effects
  • Apicoplasts* / metabolism
  • Disease Models, Animal
  • Diterpenes* / chemistry
  • Diterpenes* / pharmacology
  • Drug Resistance / genetics
  • Humans
  • Malaria, Falciparum* / drug therapy
  • Malaria, Falciparum* / parasitology
  • Mice
  • Mutation
  • Plasmodium falciparum* / drug effects
  • Plasmodium falciparum* / genetics
  • Protozoan Proteins / genetics
  • Protozoan Proteins / metabolism

Substances

  • Antimalarials
  • Protozoan Proteins
  • Diterpenes