Advances in cystic fibrosis therapies.
OBJECTIVE
Over the past four decades, outcomes for patients with cystic fibrosis have improved dramatically. Major contributors to this improvement are a better understanding of disease pathogenesis and the systematic conduct of clinical trials evaluating new therapies designed to address these defects. This review describes recent developments in cystic fibrosis pulmonary therapies intended to treat various facets of the disease, including several treatments currently in development.


RESULTS
The mainstays of therapy for cystic fibrosis, such as nutritional support and mechanical mucus clearance, are now supplemented with aggressive antibiotic regimens intended to suppress or eradicate bacterial colonization, anti-inflammatory agents, and new approaches that improve mucociliary clearance. Therapies in development address the underlying ion transport defect found in cystic fibrosis airways and also include small-molecule agents that restore function to the mutant cystic fibrosis transmembrane conductance regulator.


CONCLUSIONS
Recent advances in therapies for cystic fibrosis offer the promise of improved outcomes and longer lives for patients with cystic fibrosis.
Rowe
Steven M
SM
Department of Medicine, University of Alabama at Birmingham, Birmingham, AL 35294, USA. smrowe@uab.edu
Clancy
John P
JP
D000900:Anti-Bacterial Agents / Q000627:therapeutic use*; D000893:Anti-Inflammatory Agents / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D003550:Cystic Fibrosis / Q000235:genetics / Q000503:physiopathology / Q000628:therapy*; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000187:drug effects / Q000235:genetics; D006801:Humans; D007223:Infant; D007249:Inflammation / Q000188:drug therapy; D017136:Ion Transport / Q000187:drug effects
D000900:Anti-Bacterial Agents; D000893:Anti-Inflammatory Agents; D019005:Cystic Fibrosis Transmembrane Conductance Regulator