Emerging drug treatments for cystic fibrosis

Author: Zeitlin, Pamela L

Source: Expert Opinion on Emerging Drugs, Volume 12, Number 2, May 2007 , pp. 329-336(8)

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Abstract:

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.

Keywords: absorption; cAMP; chloride; ClC-2; calcium-activated chloride channel; cystic fibrosis transmembrane conductance regulato; endoplasmic reticulum; epithelia; gene therapy; inositol polyphosphate; ion channel; mannitol; mucolytic; plasma membrane; premature termination codon; secretion; sodium

Document Type: Research article

DOI: http://dx.doi.org/10.1517/14728214.12.2.329

Affiliations: 1: The John Hopkins School of Medicine, Department of Pediatrics, Children's Health Building, 200 N. Wolfe St., Baltimore, MD 21287, USA +1 443 287 8981; +1 410 955 1030;, Email: pzeitlin@jhmi.edu

Publication date: 2007-05-01

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