Pharmacological characterization, structural studies, and in vivo activities of anti-Chagas disease lead compounds derived from tipifarnib

Antimicrob Agents Chemother. 2012 Sep;56(9):4914-21. doi: 10.1128/AAC.06244-11. Epub 2012 Jul 9.

Abstract

Chagas disease, caused by the protozoan pathogen Trypanosoma cruzi, remains a challenging infection due to the unavailability of safe and efficacious drugs. Inhibitors of the trypanosome sterol 14α-demethylase enzyme (CYP51), including azole antifungal drugs, are promising candidates for development as anti-Chagas disease drugs. Posaconazole is under clinical investigation for Chagas disease, although the high cost of this drug may limit its widespread use. We have previously reported that the human protein farnesyltransferase (PFT) inhibitor tipifarnib has potent anti-T. cruzi activity by inhibiting the CYP51 enzyme. Furthermore, we have developed analogs that minimize the PFT-inhibitory activity and enhance the CYP51 inhibition. In this paper, we describe the efficacy of the lead tipifarnib analog compared to that of posaconazole in a murine model of T. cruzi infection. The plasma exposure profiles for each compound following a single oral dose in mice and estimated exposure parameters after repeated twice-daily dosing for 20 days are also presented. The lead tipifarnib analog had potent suppressive activity on parasitemia in mice but was unsuccessful at curing mice, whereas posaconazole as well as benznidazole cured 3 of 5 and 4 of 6 mice, respectively. The efficacy results are consistent with posaconazole having substantially higher predicted exposure than that of the tipifarnib analog after repeat twice-daily administration. Further changes to the tipifarnib analogs to reduce plasma clearance are therefore likely to be important. A crystal structure of a trypanosomal CYP51 bound to a tipifarnib analog is reported here and provides new insights to guide structure-based drug design for further optimized compounds.

Publication types

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

MeSH terms

  • 14-alpha Demethylase Inhibitors / administration & dosage*
  • 14-alpha Demethylase Inhibitors / blood
  • 14-alpha Demethylase Inhibitors / chemical synthesis
  • 14-alpha Demethylase Inhibitors / pharmacokinetics
  • Administration, Oral
  • Alkyl and Aryl Transferases / metabolism
  • Animals
  • Chagas Disease / drug therapy*
  • Chagas Disease / enzymology
  • Chagas Disease / parasitology
  • Crystallography, X-Ray
  • Cytochrome P-450 Enzyme Inhibitors*
  • Cytochrome P-450 Enzyme System / metabolism
  • Drug Administration Schedule
  • Female
  • Humans
  • Mice
  • Models, Molecular
  • Nitroimidazoles / administration & dosage
  • Quinolones / administration & dosage*
  • Quinolones / blood
  • Quinolones / chemical synthesis
  • Quinolones / pharmacokinetics
  • Structure-Activity Relationship
  • Triazoles / administration & dosage
  • Triazoles / blood
  • Triazoles / pharmacokinetics
  • Trypanocidal Agents / administration & dosage*
  • Trypanocidal Agents / blood
  • Trypanocidal Agents / chemical synthesis
  • Trypanocidal Agents / pharmacokinetics
  • Trypanosoma cruzi / drug effects*
  • Trypanosoma cruzi / growth & development

Substances

  • 14-alpha Demethylase Inhibitors
  • CYP51 protein, Trypanosoma cruzi
  • Cytochrome P-450 Enzyme Inhibitors
  • Nitroimidazoles
  • Quinolones
  • Triazoles
  • Trypanocidal Agents
  • posaconazole
  • Cytochrome P-450 Enzyme System
  • Alkyl and Aryl Transferases
  • p21(ras) farnesyl-protein transferase
  • tipifarnib
  • benzonidazole