Research ArticleCystic Fibrosis

Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis

Science Translational Medicine  23 Jul 2014:
Vol. 6, Issue 246, pp. 246ra96
DOI: 10.1126/scitranslmed.3008680

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Potentiating Trouble

Cystic fibrosis (CF) is a genetic disease caused by mutations of the CF transmembrane conductance regulator (CFTR) ion channel, resulting in pulmonary and other complications. Ivacaftor is the only targeted drug approved for CF, but it is not effective enough to treat the severest and most common form of this disease. Ivacaftor is a “potentiator,” which means that it improves the activity of mutant CFTR but cannot work if there is no CFTR on the cell surface. Other drugs, called “correctors,” help bring mutant CFTR to the cell surface, but two manuscripts by Cholon and Veit and coauthors now show that combining the two types of drugs does not work effectively, because potentiators make CFTR less stable, accelerating the removal of this channel from the cell membrane.