Emerging drug treatments for cystic fibrosis.
Although the gene for cystic fibrosis was discovered in 1989, a definitive cure remains elusive for this deadly orphan disease. Advances in nutritional rehabilitation, antibiotics, mucolytics and delivery of care have improved survival to a median age of 37.5 years; however, the psychosocial, personal and financial burdens of this lifelong chronic illness remain considerable. The current portfolio of investigational therapeutics is explored here and placed in a context of therapeutic target and predicted clinical benefit. Partnership between large and small pharma, the Cystic Fibrosis Foundation and academia should be fostered to accelerate therapeutic development.
Zeitlin
Pamela L
PL
The John Hopkins School of Medicine, Department of Pediatrics, Children's Health Building, 200 N. Wolfe St., Baltimore, MD 21287, USA. pzeitlin@jhmi.edu
D000818:Animals; D065101:Chloride Channel Agonists; D018118:Chloride Channels / Q000235:genetics / Q000378:metabolism; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000235:genetics / Q000378:metabolism; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000819:agonists / Q000235:genetics / Q000378:metabolism; D015507:Drugs, Investigational / Q000494:pharmacology / Q000627:therapeutic use*; D006801:Humans; D010069:Oxadiazoles / Q000494:pharmacology / Q000627:therapeutic use
D065101:Chloride Channel Agonists; D018118:Chloride Channels; D015507:Drugs, Investigational; D010069:Oxadiazoles; D019005:Cystic Fibrosis Transmembrane Conductance Regulator; C515878:ataluren