ICM is sensitive to detect potentiation of CFTR-mediated Cl- secretion in patients with cystic fibrosis and the G551D mutation treated with ivacaftor

Background: Sensitive outcome measures of CFTR function may facilitate the implementation of mutation-specific therapy with CFTR modulators in patients with cystic fibrosis with non-G551D mutations. Intestinal current measurement (ICM) is a sensitive assay for functional assessment of mutant CFTR in rectal biopsies and was recently shown to detect potentiator effects of 1-EBIO ex vivo (Roth E. et al., PLOS ONE 2011). The aim of this study was to determine, if ICM is sensitive to detect potentiation of CFTR-mediated Cl- secretion in rectal epithelia from CF patients with a G551D mutation treated with ivacaftor. M…