Hexahydroquinolines are antimalarial candidates with potent blood-stage and transmission-blocking activity

Nat Microbiol. 2017 Oct;2(10):1403-1414. doi: 10.1038/s41564-017-0007-4. Epub 2017 Aug 14.

Abstract

Antimalarial compounds with dual therapeutic and transmission-blocking activity are desired as high-value partners for combination therapies. Here, we report the identification and characterization of hexahydroquinolines (HHQs) that show low nanomolar potency against both pathogenic and transmissible intra-erythrocytic forms of the malaria parasite Plasmodium falciparum. This activity translates into potent transmission-blocking potential, as shown by in vitro male gamete formation assays and reduced oocyst infection and prevalence in Anopheles mosquitoes. In vivo studies illustrated the ability of lead HHQs to suppress Plasmodium berghei blood-stage parasite proliferation. Resistance selection studies, confirmed by CRISPR-Cas9-based gene editing, identified the digestive vacuole membrane-spanning transporter PfMDR1 (P. falciparum multidrug resistance gene-1) as a determinant of parasite resistance to HHQs. Haemoglobin and haem fractionation assays suggest a mode of action that results in reduced haemozoin levels and might involve inhibition of host haemoglobin uptake into intra-erythrocytic parasites. Furthermore, parasites resistant to HHQs displayed increased susceptibility to several first-line antimalarial drugs, including lumefantrine, confirming that HHQs have a different mode of action to other antimalarials drugs for which PfMDR1 is known to confer resistance. This work evokes therapeutic strategies that combine opposing selective pressures on this parasite transporter as an approach to countering the emergence and transmission of multidrug-resistant P. falciparum malaria.

MeSH terms

  • Amino Acid Sequence
  • Animals
  • Anopheles
  • Antimalarials / pharmacology*
  • CRISPR-Cas Systems / genetics
  • DNA, Protozoan / genetics
  • DNA, Protozoan / metabolism
  • Drug Combinations
  • Drug Resistance
  • Endocytosis / drug effects
  • Ethanolamines / pharmacology
  • Fluorenes / pharmacology
  • Gene Editing
  • HEK293 Cells
  • Heme
  • Hemoglobins / drug effects
  • High-Throughput Screening Assays
  • Humans
  • Lumefantrine
  • Malaria / drug therapy*
  • Malaria / transmission
  • Malaria, Falciparum / blood
  • Malaria, Falciparum / drug therapy*
  • Malaria, Falciparum / transmission
  • Male
  • Membrane Transport Proteins / genetics
  • Membrane Transport Proteins / metabolism
  • Multidrug Resistance-Associated Proteins / drug effects
  • Multidrug Resistance-Associated Proteins / genetics
  • Mutation
  • Oocysts / drug effects
  • Plasmodium berghei / drug effects*
  • Plasmodium berghei / pathogenicity
  • Plasmodium falciparum / drug effects
  • Plasmodium falciparum / genetics
  • Quinolines / chemistry
  • Quinolines / pharmacology*

Substances

  • Antimalarials
  • DNA, Protozoan
  • Drug Combinations
  • Ethanolamines
  • Fluorenes
  • Hemoglobins
  • Mdr1 protein, Plasmodium falciparum
  • Membrane Transport Proteins
  • Multidrug Resistance-Associated Proteins
  • Quinolines
  • Heme
  • Lumefantrine