Improving complex medical care while awaiting next-generation CFTR potentiators and correctors: The current pipeline of therapeutics.

Abstract

While a major target in cystic fibrosis (CF) research in recent years has been the development of corrector and potentiator drugs targeting the cystic fibrosis transmembrane conductance regulator (CFTR) protein, these therapies have not yet proven robust enough to replace or eliminate other therapies that have demonstrated improved health outcomes and quality of life in patients with CF. Further, ivacaftor is only indicated for approximately 5% of the US CF population, although the FDA has recently approved lumacaftor/ivacaftor, a combination therapy intended for those homozygous for Phe508del, which should reach a much larger number of patients. This review appraises therapeutics currently available or being studied while we await the next generation of CFTR potentiators and correctors.

• Authors: Jennifer L Goralski, Stephanie D Davis
• Journal: Pediatric pulmonology (Pediatr Pulmonol) Vol. 50 Suppl 40 Pg. S66-73 (Oct 2015) ISSN: 1099-0496 [Electronic] United States
• PMID: 26335956 (Publication Type: Journal Article, Review)
• Copyright: © 2015 Wiley Periodicals, Inc.

Chemical References

• Aminophenols
• Anti-Bacterial Agents
• Anti-Inflammatory Agents
• CFTR protein, human
• Quinolones
• Cystic Fibrosis Transmembrane Conductance Regulator

Topics

• Aminophenols
• Anti-Bacterial Agents (therapeutic use)
• Anti-Inflammatory Agents (therapeutic use)
• Cystic Fibrosis (drug therapy, therapy)
• Cystic Fibrosis Transmembrane Conductance Regulator (genetics)
• Drug Therapy (methods)
• Gene Deletion
• Homozygote
• Humans
• Quality of Life
• Quinolones
• Treatment Outcome