An allosteric modulator of HIV-1 protease shows equipotent inhibition of wild-type and drug-resistant proteases

J Med Chem. 2014 Aug 14;57(15):6468-78. doi: 10.1021/jm5008352. Epub 2014 Aug 1.

Abstract

NMR and MD simulations have demonstrated that the flaps of HIV-1 protease (HIV-1p) adopt a range of conformations that are coupled with its enzymatic activity. Previously, a model was created for an allosteric site located between the flap and the core of HIV-1p, called the Eye site (Biopolymers 2008, 89, 643-652). Here, results from our first study were combined with a ligand-based, lead-hopping method to identify a novel compound (NIT). NIT inhibits HIV-1p, independent of the presence of an active-site inhibitor such as pepstatin A. Assays showed that NIT acts on an allosteric site other than the dimerization interface. MD simulations of the ligand-protein complex show that NIT stably binds in the Eye site and restricts the flaps. That bound state of NIT is consistent with a crystal structure of similar fragments bound in the Eye site (Chem. Biol. Drug Des. 2010, 75, 257-268). Most importantly, NIT is equally potent against wild-type and a multidrug-resistant mutant of HIV-1p, which highlights the promise of allosteric inhibitors circumventing existing clinical resistance.

Publication types

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

MeSH terms

  • Allosteric Regulation
  • Allosteric Site
  • Benzothiazoles / chemical synthesis
  • Benzothiazoles / chemistry*
  • Drug Resistance, Multiple, Viral
  • HIV Protease / chemistry
  • HIV Protease / genetics*
  • HIV Protease Inhibitors / chemical synthesis
  • HIV Protease Inhibitors / chemistry*
  • Kinetics
  • Molecular Dynamics Simulation
  • Mutation
  • Pepstatins / chemistry
  • Phthalimides / chemical synthesis
  • Phthalimides / chemistry*
  • Small Molecule Libraries / chemistry
  • Structure-Activity Relationship

Substances

  • 4-nitro-2-(2-thioxo-2,3-dihydrobenzothiazol-6-yl)isoindoline-1,3-dione
  • Benzothiazoles
  • HIV Protease Inhibitors
  • Pepstatins
  • Phthalimides
  • Small Molecule Libraries
  • HIV Protease
  • p16 protease, Human immunodeficiency virus 1
  • pepstatin