A kalihinol analog disrupts apicoplast function and vesicular trafficking in <i>P. falciparum</i> malaria.

Chahine, Z; Abel, S; Hollin, T; Barnes, G L; Chung, J H; Daub, M E; Renard, I; Choi, J Y et al. · Science · 2024

basic_science · Level V

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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 <i>Plasmodium falciparum</i> 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 <i>PfSec13</i>, 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.

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