ENaC inhibitors and airway re-hydration in cystic fibrosis: state of the art

Curr Mol Pharmacol. 2013 Mar;6(1):3-12. doi: 10.2174/18744672112059990025.

Abstract

Cystic fibrosis (CF) is a hereditary disease caused by mutations in the gene encoding the chloride channel "cystic fibrosis transmembrane conductance regulator" (CFTR). The lack of functional CFTR in CF airways leads to impaired ion and fluid homeostasis of the fluid layer which lines the airway surfaces (ASL). The ASL is important for proper ciliary beat and clearance of mucus from the airways. According to the "low volume hypothesis", CF airway epithelia hyperabsorb sodium via the epithelial sodium channel (ENaC). Although the contribution of ENaC to CF pathogenesis is still under debate, there is convincing data demonstrating that re-hydration of the ASL might improve mucociliary clearance in CF patients. ASL re-hydration might, amongst other things, be achieved by a block of airway transepithelial sodium absorption with inhibitors of ENaC. This mini-review article describes the role of ENaC in ASL fluid homeostasis and rehydration, and summarizes the current state of the art in the discovery and establishment of compounds which inhibit ENaC activity and may represent pharmacological tools for the treatment of CF.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Amiloride / chemistry
  • Amiloride / pharmacology
  • Amiloride / therapeutic use
  • Cystic Fibrosis / metabolism*
  • Cystic Fibrosis / pathology
  • Cystic Fibrosis / therapy
  • Cystic Fibrosis Transmembrane Conductance Regulator / metabolism
  • Epithelial Sodium Channel Blockers / chemistry
  • Epithelial Sodium Channel Blockers / pharmacology
  • Epithelial Sodium Channel Blockers / therapeutic use
  • Epithelial Sodium Channels / chemistry
  • Epithelial Sodium Channels / metabolism*
  • Humans
  • Mucociliary Clearance / drug effects
  • Protease Inhibitors / chemistry
  • Protease Inhibitors / pharmacology
  • Protease Inhibitors / therapeutic use
  • Purinergic Agonists / chemistry
  • Purinergic Agonists / pharmacology
  • Purinergic Agonists / therapeutic use
  • Sodium / metabolism

Substances

  • Epithelial Sodium Channel Blockers
  • Epithelial Sodium Channels
  • Protease Inhibitors
  • Purinergic Agonists
  • Cystic Fibrosis Transmembrane Conductance Regulator
  • Amiloride
  • Sodium