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Updated: Jan 29, 2026

Rectal Organoid Morphology Analysis ROMA: A Diagnostic Assay in Cystic Fibrosis
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Rectal Organoid Morphology Analysis ROMA: A Diagnostic Assay in Cystic Fibrosis

Published on: June 10, 2022

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Rectal Organoids Enable Personalized Treatment of Cystic Fibrosis

Gitte Berkers1, Peter van Mourik1, Annelotte M Vonk2

  • 1Department of Pediatric Pulmonology, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht University, 3584 EA Utrecht, the Netherlands.

Cell Reports
|February 14, 2019
PubMed

Insights

Patient-derived rectal organoids accurately predict cystic fibrosis (CF) treatment effectiveness. This stem cell assay guides personalized medicine by correlating in vitro drug responses with in vivo therapeutic outcomes.

Area of Science:

  • Regenerative Medicine
  • Genetics and Genomics
  • Pharmacology

Background:

  • Cystic Fibrosis (CF) treatment selection is challenging due to individual patient variability.
  • Patient-derived stem cell cultures offer a platform for personalized drug testing.
  • Rectal organoids can model CF pathophysiology and drug responses.

Purpose of the Study:

  • To evaluate the correlation between in vitro drug responses in patient-derived rectal organoids and in vivo therapeutic endpoints in Cystic Fibrosis patients.
  • To determine the accuracy of an organoid-based assay for predicting clinical treatment efficacy.

Main Methods:

  • Utilized patient-derived rectal organoids for in vitro drug sensitivity testing.
  • Assessed organoid response using forskolin-induced swelling assay.
  • Correlated in vitro results with in vivo changes in pulmonary function and sweat chloride concentration.

Main Results:

  • In vitro organoid drug responses showed a significant correlation with in vivo changes in pulmonary response.
  • Organoid assay results also correlated with changes in sweat chloride concentration, a key CF biomarker.
  • Receiver operating characteristic analysis demonstrated good-to-excellent accuracy for the organoid-based test in predicting clinical responses.

Conclusions:

  • In vitro drug testing using patient-derived rectal organoids can prospectively identify efficacious treatments for Cystic Fibrosis.
  • This stem cell-based organoid assay provides a patient-friendly and cost-effective method for tailoring individual CF treatments.
  • Biobanked stem cell resources are valuable for developing personalized therapeutic strategies in CF.
Keywords:
CFTRcystic fibrosisdrug responseorganoidspersonalized treatmentpredictingstem cell cultures

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