Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis

Gitte Berkers, Peter van Mourik, Annelotte M Vonk, Evelien Kruisselbrink, Johanna F Dekkers, Karin M de Winter-de Groot, Hubertus G M Arets, Rozemarijn E P Marck-van der Wilt, Jasper S Dijkema, Maaike M Vanderschuren, Roderick H J Houwen, Harry G M Heijerman, Eduard A van de Graaf, Sjoerd G Elias, Christof J Majoor, Gerard H Koppelman, Jolt Roukema, Marleen Bakker, Hettie M Janssens, Renske van der MeerRobert G J Vries, Hans C Clevers, Hugo R de Jonge, Jeffrey M Beekman, Cornelis K van der Ent

Research output: Contribution to journal/periodicalArticleScientificpeer-review

168 Citations (Scopus)


In vitro drug tests using patient-derived stem cell cultures offer opportunities to individually select efficacious treatments. Here, we provide a study that demonstrates that in vitro drug responses in rectal organoids from individual patients with cystic fibrosis (CF) correlate with changes in two in vivo therapeutic endpoints. We measured individual in vitro efficaciousness using a functional assay in rectum-derived organoids based on forskolin-induced swelling and studied the correlation with in vivo effects. The in vitro organoid responses correlated with both change in pulmonary response and change in sweat chloride concentration. Receiver operating characteristic curves indicated good-to-excellent accuracy of the organoid-based test for defining clinical responses. This study indicates that an in vitro assay using stem cell cultures can prospectively select efficacious treatments for patients and suggests that biobanked stem cell resources can be used to tailor individual treatments in a cost-effective and patient-friendly manner.

Original languageEnglish
Pages (from-to)1701-1708.e3
JournalCell Reports
Issue number7
Publication statusPublished - 12 Feb 2019


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