New Therapeutic Approaches to Modulate and Correct Cystic Fibrosis Transmembrane Conductance Regulator

Pediatr Clin North Am. 2016 Aug;63(4):751-64. doi: 10.1016/j.pcl.2016.04.006.

Abstract

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators are clinically available personalized medicines approved for some individuals with cystic fibrosis (CF) to target the underlying defect of disease. This review summarizes strategies used to develop CFTR modulators as therapies that improve function and availability of CFTR protein. Lessons learned from dissemination of ivacaftor across the CF population responsive to this therapy and future approaches to predict and monitor treatment response of CFTR modulators are discussed. The goal remains to expand patient-centered and personalized therapy to all patients with CF, ultimately improving life expectancy and quality of life for this disease.

Keywords: CFTR modulator; Corrector; Personalized medicine; Potentiator; Therapeutics.

Publication types

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

MeSH terms

  • Aminophenols / adverse effects
  • Aminophenols / therapeutic use*
  • Aminopyridines / adverse effects
  • Aminopyridines / therapeutic use*
  • Benzodioxoles / adverse effects
  • Benzodioxoles / therapeutic use*
  • Chloride Channel Agonists / adverse effects
  • Chloride Channel Agonists / therapeutic use*
  • Cystic Fibrosis / drug therapy*
  • Cystic Fibrosis / genetics
  • Cystic Fibrosis Transmembrane Conductance Regulator / drug effects*
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics
  • Humans
  • Molecular Targeted Therapy / adverse effects
  • Molecular Targeted Therapy / methods*
  • Mutation
  • Quinolones / adverse effects
  • Quinolones / therapeutic use*

Substances

  • Aminophenols
  • Aminopyridines
  • Benzodioxoles
  • Chloride Channel Agonists
  • Quinolones
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • ivacaftor
  • lumacaftor