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Disease models

Intestinal organoid model for cystic fibrosis transmembrane conductance regulator (CFTR) function

An intestinal organoid model for CFTR function could help develop compounds to treat cystic fibrosis. Organoids were cultured from human intestinal stem cells and treated with forskolin, which activates CFTR and induces fluid influx and swelling. In organoids cultured from patients carrying mutant CFTR variants, forskolin-induced swelling was lower than that in patients carrying wild-type CFTR. In DF508 mutant CFTR-expressing organoids, compounds that increased CFTR function also increased forskolin-induced swelling compared with vehicle. Next steps include using the organoids to model patient response to CFTR-targeted drugs.
Vertex Pharmaceuticals Inc.'s VX-809, a CFTR corrector, is in Phase III trials to treat DF508 mutant cystic fibrosis (CF) in combination with the CFTR potentiator Kalydeco ivacaftor (VX-770). Vertex markets Kalydeco to treat CF.

SciBX 6(26); doi:10.1038/scibx.2013.669
Published online July 11, 2013

Patent application filed; available for licensing

Dekkers, J.F. et al. Nat. Med.; published online June 2, 2013;
Contact: Jeffrey M. Beekman, University Medical Center Utrecht, Utrecht, the Netherlands