Treatment efficacies of drugs depend on patient-specific pharmacokinetic and pharmacodynamic properties. Here, we developed an assay to measure functional levels of the CFTR potentiator VX-770 in human plasma and observed that VX-770 in plasma from different donors induced variable CFTR function in intestinal organoids. This assay can help to understand variability in treatment response to CFTR potentiators by functionally modeling individual pharmacokinetics.

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Journal of Cystic Fibrosis
Department of Pediatrics

Dekkers, R., Vijftigschild, L. A. W., Vonk, A. M., Kruisselbrink, E., De Winter-De Groot, K., Janssens, H., … Beekman, J. (2015). A bioassay using intestinal organoids to measure CFTR modulators in human plasma. Journal of Cystic Fibrosis, 14(2), 178–181. doi:10.1016/j.jcf.2014.10.007