MEPicides: potent antimalarial prodrugs targeting isoprenoid biosynthesis

Sci Rep. 2017 Aug 21;7(1):8400. doi: 10.1038/s41598-017-07159-y.

Abstract

The emergence of Plasmodium falciparum resistant to frontline therapeutics has prompted efforts to identify and validate agents with novel mechanisms of action. MEPicides represent a new class of antimalarials that inhibit enzymes of the methylerythritol phosphate (MEP) pathway of isoprenoid biosynthesis, including the clinically validated target, deoxyxylulose phosphate reductoisomerase (Dxr). Here we describe RCB-185, a lipophilic prodrug with nanomolar activity against asexual parasites. Growth of P. falciparum treated with RCB-185 was rescued by isoprenoid precursor supplementation, and treatment substantially reduced metabolite levels downstream of the Dxr enzyme. In addition, parasites that produced higher levels of the Dxr substrate were resistant to RCB-185. Notably, environmental isolates resistant to current therapies remained sensitive to RCB-185, the compound effectively treated sexually-committed parasites, and was both safe and efficacious in malaria-infected mice. Collectively, our data demonstrate that RCB-185 potently and selectively inhibits Dxr in P. falciparum, and represents a promising lead compound for further drug development.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aldose-Ketose Isomerases / antagonists & inhibitors
  • Animals
  • Antimalarials / administration & dosage
  • Antimalarials / pharmacology*
  • Disease Models, Animal
  • Enzyme Inhibitors / administration & dosage
  • Enzyme Inhibitors / pharmacology*
  • Malaria, Falciparum / drug therapy
  • Mice
  • Plasmodium falciparum / drug effects*
  • Plasmodium falciparum / growth & development
  • Prodrugs / administration & dosage
  • Prodrugs / pharmacology*
  • Terpenes / antagonists & inhibitors*
  • Treatment Outcome

Substances

  • Antimalarials
  • Enzyme Inhibitors
  • Prodrugs
  • Terpenes
  • 1-deoxy-D-xylulose 5-phosphate reductoisomerase
  • Aldose-Ketose Isomerases