Unusual binding mode of an HIV-1 protease inhibitor explains its potency against multi-drug-resistant virus strains

J Mol Biol. 2002 Dec 6;324(4):739-54. doi: 10.1016/s0022-2836(02)01139-7.

Abstract

Protease inhibitors (PIs) are an important class of drugs for the treatment of HIV infection. However, in the course of treatment, resistant viral variants with reduced sensitivity to PIs often emerge and become a major obstacle to successful control of viral load. On the basis of a compound equipotently inhibiting HIV-1 and 2 proteases (PR), we have designed a pseudopeptide inhibitor, QF34, that efficiently inhibits a wide variety of PR variants. In order to analyze the potency of the inhibitor, we constructed PR species harboring the typical (signature) mutations that confer resistance to commercially available PIs. Kinetic analyses showed that these mutated PRs were inhibited up to 1,000-fold less efficiently by the clinically approved PIs. In contrast, all PR species were effectively inhibited by QF34. In a clinical study, we have monitored 30 HIV-positive patients in the Czech Republic undergoing highly active antiretroviral therapy, and have identified highly PI resistant variants. Kinetic analyses revealed that QF34 retained its subnanomolar potency against multi-drug resistant PR variants. X-ray crystallographic analysis and molecular modeling experiments explained the wide specificity of QF34: this inhibitor binds to the PR in an unusual manner, thus avoiding contact sites that are mutated upon resistance development, and the unusual binding mode and consequently the binding energy is therefore preserved in the complex with a resistant variant. These results suggest a promising route for the design of second-generation PIs that are active against a variety of resistant PR variants.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Substitution
  • Antiretroviral Therapy, Highly Active
  • Binding Sites
  • Crystallography, X-Ray
  • Drug Design
  • Drug Resistance, Microbial / genetics
  • Drug Resistance, Multiple / genetics
  • Female
  • Genotype
  • HIV Infections / drug therapy*
  • HIV Infections / virology
  • HIV Protease Inhibitors / chemistry
  • HIV Protease Inhibitors / metabolism*
  • HIV Protease Inhibitors / pharmacology*
  • HIV-1 / drug effects*
  • HIV-1 / genetics
  • HIV-1 / metabolism
  • Humans
  • Kinetics
  • Male
  • Models, Molecular
  • Molecular Conformation
  • Molecular Structure
  • Mutation
  • Oligopeptides / chemistry
  • Oligopeptides / metabolism*
  • Oligopeptides / pharmacology*
  • Protein Conformation
  • Protein Engineering
  • Recombinant Proteins / chemistry
  • Recombinant Proteins / isolation & purification
  • Recombinant Proteins / metabolism
  • Reverse Transcriptase Inhibitors / pharmacology

Substances

  • HIV Protease Inhibitors
  • Oligopeptides
  • QF34 pseudopeptide
  • Recombinant Proteins
  • Reverse Transcriptase Inhibitors