Structure-activity relationships of cyanoquinolines with corrector-potentiator activity in ΔF508 cystic fibrosis transmembrane conductance regulator protein

J Med Chem. 2012 Feb 9;55(3):1242-51. doi: 10.1021/jm201372q. Epub 2012 Jan 23.

Abstract

Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel. The most common CF-causing mutation, ΔF508-CFTR, produces CFTR loss-of-function by impairing its cellular targeting to the plasma membrane and its chloride channel gating. We recently identified cyanoquinolines with both corrector ("Co", normalizing ΔF508-CFTR targeting) and potentiator ("Po", normalizing ΔF508-CFTR channel gating) activities. Here, we synthesized and characterized 24 targeted cyanoquinoline analogues to elucidate the conformational requirements for corrector and potentiator activities. Compounds with potentiator-only, corrector-only, and dual potentiator-corrector activities were found. Molecular modeling studies (conformational search ⇒ force-field lowest energy assessment ⇒ geometry optimization) suggest that (1) a flexible tether and (2) a relatively short bridge between the cyanoquinoline and arylamide moieties are important cyanoquinoline-based CoPo features. Further, these CoPo's may adopt two distinct π-stacking conformations to elicit corrector and potentiator activities.

Publication types

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

MeSH terms

  • Animals
  • Cells, Cultured
  • Cystic Fibrosis Transmembrane Conductance Regulator / chemistry*
  • Epithelial Cells / drug effects
  • Epithelial Cells / metabolism
  • Humans
  • Models, Molecular*
  • Molecular Conformation
  • Nitriles / chemical synthesis
  • Nitriles / chemistry*
  • Nitriles / pharmacology
  • Quinolines / chemical synthesis
  • Quinolines / chemistry*
  • Quinolines / pharmacology
  • Rats
  • Rats, Inbred F344
  • Structure-Activity Relationship

Substances

  • Nitriles
  • Quinolines
  • Cystic Fibrosis Transmembrane Conductance Regulator