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. 2009 Nov;15(6):604-10.
doi: 10.1097/MCP.0b013e328331805b.

Update on New Pulmonary Therapies


Update on New Pulmonary Therapies

George Z Retsch-Bogart. Curr Opin Pulm Med. .


Purpose of review: Understanding the molecular and cellular processes responsible for the development of lung disease in cystic fibrosis (CF) has led to evaluation of a broad range of new therapies within multiple therapeutic classes. For these reasons, clinical research in CF is accelerating, as new agents progress through the early stages of drug development, move into clinical trials and are offered to study patients. This review focuses on the most notable clinical trials of pulmonary therapies reported in the last year.

Recent findings: Progress in gene therapy remains slow, but is offset by significant gains in development of cystic fibrosis transmembrane conductance regulator modulators and drugs that restore airway surface liquid. Although addressing downstream consequences of CF lung pathophysiology, the substantial burden of chronic infection makes both antibiotic and anti-inflammatory therapies a critical component of treatment, such that additional agents to manage sustained inflammation and resistant microorganisms along with improved delivery systems are needed.

Summary: The pace of drug development in CF will require an expanding pool of patients willing to participate in clinical research to test new agents. Although these potential therapies will likely improve quality of life for people with CF and contribute to improved survival, it will be important to avoid adding excessively to the already high burden of treatment. If the demands on patients' time continue to grow, a decrease in adherence to effective therapies may paradoxically lead to worse outcomes and negate the benefits new treatments bring.

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