Identifying Novel Endpoints for Cystic Fibrosis Clinical Trials*

Clinical and translational research in cystic fibrosis (CF) is hampered by a lack of biomarkers that can be used to identify promising new therapies. In particular, validated protein biomarkers are needed to evaluate emerging candidate anti-inflammatory and antimicrobial treatments. Noninvasively obtained biomarkers are especially desirable in children where repeatedly sampling the airway is difficult. To more quickly identify and validate candidate protein biomarkers, it will be essential to pursue a phased approach to biomarker development in a large, well-characterized CF patient population. New high-throughput technologies that can simultaneously examine the expression of thousands of genes and proteins are accelerating the discovery of potential biomarkers. It is hoped that individual biomarkers, or more likely a panel of validated markers, will predict clinical outcomes such as susceptibility to rapid disease progression or response to treatment and therefore, greatly improve stratification for future CF clinical trials.