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Updated: Jun 14, 2026
Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
Published on: February 11, 2017
Patient-Derived Intestinal Organoids in the Global Cystic Fibrosis Landscape
Suzanne Kroes1,2, Jennifer L Taylor-Cousar3, Marco Zampoli4
1Department of Paediatric Pulmonology, Wilhelmina Children's Hospital - University Medical Centre Utrecht, Utrecht University, Utrecht, The Netherlands.
Insights
Patient-derived intestinal organoids (PDIOs) can improve cystic fibrosis (CF) diagnosis and care in low- and middle-income countries (LMICs). These organoids help overcome barriers like limited screening and access to CFTR modulators, bridging global health disparities.
Area of Science:
- Biomedical Science
- Genetics
- Global Health
Background:
- Cystic fibrosis (CF) diagnosis and care are significantly limited in low- and middle-income countries (LMICs).
- Barriers include restricted newborn screening, limited diagnostic testing, underrepresentation of diverse CFTR variants, and inadequate access to basic therapies.
- Sparse patient registries hinder epidemiological understanding and resource allocation in LMICs.
Purpose of the Study:
- To explore the potential of patient-derived intestinal organoids (PDIOs) in addressing diagnostic and therapeutic gaps in CF care within LMICs.
- To evaluate PDIOs as a platform for functional diagnosis, genotype-phenotype correlation, and drug testing, particularly for rare CFTR variants.
- To identify strategies for implementing PDIO technology in LMICs to mitigate global health disparities in CF.
Main Methods:
- Utilizing patient-derived intestinal organoids (PDIOs) for functional CFTR analysis.
- Characterizing genotype-phenotype correlations using PDIO models.
- Assessing the feasibility of comprehensive CFTR sequencing and drug screening with PDIOs, including for rare variants.
Main Results:
- PDIOs provide a versatile platform for functional diagnosis and characterization of CF, including rare CFTR variants.
- PDIOs can generate renewable material for advanced genetic analysis and drug testing.
- Implementation challenges in LMICs exist but can be addressed through mentorship, collaboration, and funding.
Conclusions:
- Patient-derived intestinal organoids (PDIOs) offer a promising approach to improve CF diagnosis and care in resource-limited settings.
- Integrating PDIOs can help bridge global disparities in CF treatment by enabling personalized medicine and addressing variant-specific challenges.
- Successful implementation requires coordinated scientific, clinical, and policy efforts to overcome cost and access barriers.
Abstract:
Cystic fibrosis (CF) care has advanced rapidly, yet diagnosis and inclusion in patient registries remain severely limited in low- and middle-income countries (LMICs). Barriers include restricted newborn screening, limited availability of sweat chloride testing, and underrepresentation of non-European CFTR variants in standard panels. Sparse registries further impede epidemiological insight and resource planning. Also, access to comprehensive CF care remains a major challenge in LMICs, where limitations in basic therapies, including pancreatic enzyme replacement, airway clearance strategies, and infection control, continue to result in markedly reduced life expectancy. Within this context, access to CFTR modulators (CFTRm) represents an additional and critical barrier, constrained by prohibitive costs, restricted variant eligibility, and few tiered pricing mechanisms. Patient-derived intestinal organoids (PDIOs) offer a versatile platform to help address some of these gaps. PDIOs enable functional diagnosis, genotype-phenotype characterization, and renewable material for comprehensive CFTR sequencing and drug testing, including for rare variants. While implementation in LMICs is challenging, targeted mentorship, collaborative screening pipelines, and sustained funding could expand access. Integrating PDIOs into CF care could help bridge some global disparities, but success will require coordinated scientific, clinical, and policy efforts.
