Chest
Ahead of the CurveCystic Fibrosis Therapeutics: The Road Ahead
Section snippets
What Lies Ahead
The pathophysiology of CF lung disease is known to involve self-perpetuating cycles of airway obstruction, infection, and inflammation,6 as illustrated at the bottom of Figure 1. Accordingly, many current and candidate treatments directly target one or more of these pathophysiologic elements; for example, there are multiple therapies currently addressing airway infections, inflammation, and airway mucus rheology. More encouraging are novel therapies, including CFTR modulators and airway cell
Conclusion
The past 20 years have transformed the field of CF treatment so that changing the course of this fatal illness is becoming a reality. The view of the road ahead has never been so encouraging in CF therapeutics, and there is cause for unprecedented optimism for anyone who cares for people with this devastating disease. In the next decade, it is likely that both a CFTR potentiator (Kalydeco) and at least one CFTR corrector may be available for the majority of patients with CF. These drugs have a
Acknowledgments
Financial/nonfinancial disclosures: The authors have reported to CHEST the following conflicts of interest: Dr Hoffman receives grant funding from the Cystic Fibrosis Foundation and the National Institutes of Health. Dr Ramsey, in her capacity as director of the Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, has received grants from the following companies in the past 3 years: AlgiPharma AS; Amgen, Inc; Aradigm Corporation; Axcan Pharma, Inc; Bayer Healthcare
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