---
title: Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis
description: Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis
image: https://resources.huborganoids.nl/hubfs/publication-thumb-cell-rep-feb-2019.png
---

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# Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis

Cell Rep | February 2019

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## Abstract

*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.

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