Differences in conformational dynamics between Plasmodium falciparum and human Hsp90 orthologues enable the structure-based discovery of pathogen-selective inhibitors

J Med Chem. 2014 Mar 27;57(6):2524-35. doi: 10.1021/jm401801t. Epub 2014 Mar 3.

Abstract

The high sequence conservation of druggable pockets of closely related proteins can make it challenging to develop selective inhibitors. We designed a new drug discovery approach that exploits both the static and dynamic differences of two orthologues. We applied it, as a proof of concept, to identify compounds that discriminate between the molecular chaperone Hsp90 of the protozoan pathogen Plasmodium falciparum (Pf) and that of its human host. We found that the ATP-binding pocket has a Pf-specific extension, whose sequence lining is identical in human Hsp90 but which differs by tertiary structure and dynamics. Using these insights for a structure-based drug screen, we discovered novel 7-azaindole compounds that exclusively bind the recombinant N-terminal domain of PfHsp90 but not of human Hsp90 nor of a PfHsp90 mutant with "human-like" dynamics. Moreover, these compounds preferentially inhibit the growth of yeast complemented by PfHsp90 and block the growth of Pf in culture.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adenosine Triphosphate / metabolism
  • Amino Acid Sequence
  • Animals
  • Antimalarials / chemical synthesis*
  • Antimalarials / chemistry
  • Antimalarials / pharmacology*
  • Benzoquinones / pharmacology
  • Cell Survival / drug effects
  • Computational Biology
  • Cysteine Proteinase Inhibitors / pharmacology
  • Drug Discovery
  • HSP90 Heat-Shock Proteins / chemistry*
  • High-Throughput Screening Assays
  • Humans
  • Lactams, Macrocyclic / pharmacology
  • Models, Molecular
  • Molecular Sequence Data
  • Plasmids / genetics
  • Plasmodium falciparum / chemistry*
  • Protein Conformation
  • Rats
  • Saccharomyces cerevisiae / drug effects
  • Saccharomyces cerevisiae / growth & development
  • Structure-Activity Relationship

Substances

  • Antimalarials
  • Benzoquinones
  • Cysteine Proteinase Inhibitors
  • HSP90 Heat-Shock Proteins
  • Lactams, Macrocyclic
  • Adenosine Triphosphate
  • geldanamycin