A pipeline of therapies for cystic fibrosis.
Therapeutics development for cystic fibrosis (CF) involves a coordinated effort among many groups, including individuals with CF and their caregivers, clinical research teams, and those in academia and industry who have discovered and developed the therapeutic strategies. In the United States, the Cystic Fibrosis Foundation (CFF) has devoted over $875 million to facilitate and coordinate this process since 1986, resulting in the clinical development and/or assessment of ~50 drug candidates during that time. The more than 30 compounds currently in the pipeline of Foundation-funded therapeutics are used as a platform to discuss why and how therapeutic strategies are brought into clinical development. Consideration is also given to the funding, management, and infrastructure necessary and practical to support the progression of drug candidates and the availability of therapeutics for use by individuals with CF. The importance of the clinical trial process and relevant outcome measures to assess the efficacy of drug candidates is also discussed. Finally, the potential impact of the pipeline for individuals with CF is summarized.
Ashlock
Melissa A
MA
Cystic Fibrosis Foundation Therapeutics, Inc., Bethesda, Maryland 20814, USA. mashlock@cff.org
Beall
Robert J
RJ
Hamblett
Nicole M
NM
Konstan
Michael W
MW
Penland
Christopher M
CM
Ramsey
Bonnie W
BW
Van Dalfsen
Jill M
JM
Wetmore
Diana R
DR
Campbell
Preston W
PW
D002986:Clinical Trials as Topic; D003299:Cooperative Behavior; D003550:Cystic Fibrosis / Q000628:therapy*; D015195:Drug Design; D055808:Drug Discovery / Q000191:economics / Q000379:methods*; D004345:Drug Industry / Q000191:economics / Q000379:methods; D005582:Foundations; D006801:Humans; D014481:United States