Document Detail

Cystic fibrosis therapeutics: the road ahead.
MedLine Citation:
PMID:  23276843     Owner:  NLM     Status:  MEDLINE    
A great deal of excitement and hope has followed the successful trials and US Food and Drug Administration approval of the drug ivacaftor (Kalydeco), the first therapy available that targets the underlying defect that causes cystic fibrosis (CF). Although this drug has currently demonstrated a clinical benefit for a small minority of the CF population, the developmental pathway established by ivacaftor paves the way for other CF transmembrane conductance regulator (CFTR) modulators that may benefit many more patients. In addition to investigating CFTR modulators, researchers are actively developing numerous other innovative CF therapies. In this review, we use the catalog of treatments currently under evaluation with the support of the Cystic Fibrosis Foundation, known as the Cystic Fibrosis Foundation Therapeutics Pipeline, as a platform to discuss the variety of candidate treatments for CF lung disease that promise to improve CF care. Many of these approaches target the individual components of the relentless cycle of airway obstruction, inflammation, and infection characteristic of lung disease in CF, whereas others are aimed directly at the gene defect, or the resulting dysfunctional protein, that instigates this cycle. We discuss how new findings from the laboratory have informed not only the development of novel therapeutics, but also the rationales for their use and the outcomes used to measure their effects. By reviewing the breadth of candidate treatments currently in development, as well as the recent progress in CF therapies reflected by the evolution of the therapeutics pipeline over the past few years, we hope to build upon the optimism and anticipation generated by the recent success of Kalydeco.
Lucas R Hoffman; Bonnie W Ramsey
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Publication Detail:
Type:  Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't; Review    
Journal Detail:
Title:  Chest     Volume:  143     ISSN:  1931-3543     ISO Abbreviation:  Chest     Publication Date:  2013 Jan 
Date Detail:
Created Date:  2013-01-01     Completed Date:  2013-04-02     Revised Date:  2014-01-09    
Medline Journal Info:
Nlm Unique ID:  0231335     Medline TA:  Chest     Country:  United States    
Other Details:
Languages:  eng     Pagination:  207-13     Citation Subset:  AIM; IM    
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MeSH Terms
Aminophenols / therapeutic use*
Anti-Bacterial Agents / therapeutic use*
Anti-Inflammatory Agents / therapeutic use*
Cystic Fibrosis / drug therapy*,  physiopathology
Drug Therapy / trends
Quinolones / therapeutic use*
Grant Support
Reg. No./Substance:
0/Aminophenols; 0/Anti-Bacterial Agents; 0/Anti-Inflammatory Agents; 0/N-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide; 0/Quinolones

From MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine

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