Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis.

ABSTRACT: Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR). Newly developed "correctors" such as lumacaftor (VX-809) that improve CFTR maturation and trafficking and "potentiators" such as ivacaftor (VX-770) that enhance channel activity may provide important advances in CF therapy. Although VX-770 has demonstrated substantial clinical efficacy in the small subset of patients with a mutation (G551D) that affects only channel activity, a single compound is not sufficient to treat patients with the more common CFTR mutation, ΔF508. Thus, patients with ΔF508 will likely require treatment with both correctors and potentiators to achieve clinical benefit. However, whereas the effectiveness of acute treatment with this drug combination has been demonstrated

SUBMITTER: Cholon DM 

PROVIDER: S-EPMC4272825 | biostudies-literature | 2014 Jul

REPOSITORIES: biostudies-literature