Emerging treatments in cystic fibrosis

Drugs. 2009 Oct 1;69(14):1903-10. doi: 10.2165/11318500-000000000-00000.


There are a number of potential drugs for the treatment of cystic fibrosis (CF) currently undergoing clinical studies. A number of antibacterials formulated for delivery by inhalation are at various stages of study; these include dry-powder inhaler versions of colistin, tobramycin and ciprofloxacin, and formulations of azteonam, amikacin, levofloxacin, ciprofloxacin and fosfomycin/tobramycin for nebulization. Clinical trials of anti-inflammatory agents, including glutathione, phosphodiesterase-5 inhibitors such as sildenafil, oral acetylcysteine, simvastatin, methotrexate, docosahexaenoic acid, hydroxychloroquine, pioglitazone and alpha1-antitrypsin, are ongoing. Ion channel modulating agents, such as lancovutide (Moli1901, duramycin) and denufosol, which activate alternate (non-CF transmembrane regulator [CFTR]) chloride channels, and GS 9411, a sodium channel antagonist, are now at the stages of clinical study and if successful, will offer a new category of therapeutic agent for the treatment of CF. Correction of the underlying gene effect, either by agents that help to correct the dysfunctional CFTR, such as ataluren, VX-770 and VX-809, or by gene transfer (gene therapy), is a particularly exciting prospect as a new therapy for CF and clinical studies are ongoing. This article reviews the exciting potential drug treatments for CF currently being evaluated in clinical studies, and also highlights some of the challenges faced by research and clinical teams in assessing the efficacy of potential new therapies for CF.

MeSH terms

  • Anti-Infective Agents / therapeutic use*
  • Anti-Inflammatory Agents / therapeutic use*
  • Chloride Channels / metabolism
  • Cystic Fibrosis / complications
  • Cystic Fibrosis / drug therapy*
  • Cystic Fibrosis / metabolism
  • Cystic Fibrosis / therapy*
  • Cystic Fibrosis Transmembrane Conductance Regulator / genetics
  • Cystic Fibrosis Transmembrane Conductance Regulator / metabolism
  • Exocrine Pancreatic Insufficiency / complications
  • Exocrine Pancreatic Insufficiency / drug therapy
  • Expectorants / therapeutic use*
  • Genetic Therapy*
  • Humans
  • Respiratory Mucosa / drug effects
  • Respiratory Mucosa / metabolism


  • Anti-Infective Agents
  • Anti-Inflammatory Agents
  • Chloride Channels
  • Expectorants
  • Cystic Fibrosis Transmembrane Conductance Regulator