Emerging drugs for cystic fibrosis.
BACKGROUND
Cystic fibrosis is an autosomal recessive disease, which is the result of a genetic defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Pulmonary disease accounts for over 90% of the morbidity and mortality associated with the disease. Conventionally, CF treatment has focused on symptomatic therapy.


METHODS
In the past, the emphasis for the development of CF therapeutics has previously been on addressing complications of the manifestations rather than on the underlying disease process. However, in the past few decades there has been a paradigm shift with new attention on the underlying biological mechanisms and therapies targeted at curing the disease rather than simply controlling it. This review summarizes the current CF therapeutics pipeline. These developing therapies include CFTR gene therapy, CFTR pharmacotherapeutics, osmotically active agents and anti-inflammatory therapies, as well as novel inhaled antibiotics.


CONCLUSIONS
The CF therapeutics pipeline currently holds great promise both for novel therapies directly targeting the underlying biological mechanisms of CFTR dysfunction and new symptomatic therapies. While CFTR-directed therapy has the highest potential to improve patients' outcome, it is important to continue to develop better treatment options for all aspects of CF lung disease.
University of Toronto, The Hospital for Sick Children, Division of Respiratory Medicine, Department of Pediatrics, Physiology and Experimental Medicine , 555 University Avenue, Toronto, ON, M5G 1X8 , Canada +416 813 6346 ; +416 813 6246 ; reshma.amin@sickkids.ca.
Reshma
R
Amin
Felix
F
Ratjen
D000818:Animals; D000893:Anti-Inflammatory Agents / Q000494:pharmacology / Q000627:therapeutic use; D003550:Cystic Fibrosis / Q000188:drug therapy* / Q000235:genetics; D019005:Cystic Fibrosis Transmembrane Conductance Regulator / Q000235:genetics; D015316:Genetic Therapy / Q000379:methods; D006801:Humans
D000893:Anti-Inflammatory Agents; D019005:Cystic Fibrosis Transmembrane Conductance Regulator