Aims


1. To align current activities around organoid research and set up an international network of organoid-based researchers within the ECFS.

2. To standardize and implement organoid-based assays for research in a diagnostic and academic setting.

3. To optimize implementation of intestinal stem cell (ISC)-based assays as predictive biomarkers, by optimizing ethical and juridical prerequisites for international collaborations & interacting with relevant parties such as the EMA.

4. Organize annual meetings & develop a training program for ISC-based methodologies to disseminate expertise in PDIO culture and CFTR function assessment for researchers across Europe

Deliverables per objective

1.

A. Categorize and align current ongoing activities by ECFS members.

B. Establish partnerships between ECFS members to shape future grant application and research activities

C. Connect with other working groups, in particular “Access to novel therapies”, “Diagnostic Network Working Group” and ‘Nasal Cell Working Group” to align goals and avoid double work.

2.

A. Develop standardized protocols for biobanking/culturing organoids, and performing CFTR-function measurements (ROMA, DIS, FIS, 2D electrophysiology, sensor-based read-outs) and data analysis in an academic context

B. Develop a standardized procedure for producing PDIO-based diagnostic data and interpreting such data into a clinical advise on which therapeutic regimen could work optimally for the corresponding person.

C. Establish and publish multi-center validation studies to validate the reproducibility of organoid based assays & the connecting existing preclinical diagnostic data to clinical follow-up and results across institutes

D. Set up a virtual online repository of which PDIO lines are available in which institute, to enhance sharing of PDIO lines and overall collaborative efforts

E. Study how to optimize collaboration in the context of ethical and juridical documentation that is needed for sharing PDIO-material with one another.

F. Establish a database of PDIO-based drug responses, either novel or in combination with existing databases, in addition to academic publications.

3.

A. Design a strategy to educate and interact with health insurance providers and regulatory offices to discuss reimbursement strategies for organoid-based testing

B. Develop a summarizing opinion piece on the cost-effectiveness of organoid-based testing for CFTR modulators aimed at regulators and insurers

4.

A. Organize an annual meeting, starting from Year 1, to share latest results and strategies on ISC based research. Possibly in combination with the ECFS, as attendants will partially overlap.

B. Organize an annual workshop, starting from Year 2, to train and update researchers and clinicians in PDIO-based methodologies. Here we will start with the PDIO-based FIS assay but can expand to a broader set of culture methods and assays at a later stage.

C. Organize a monthly practical trouble-shoot meeting for PDIO updates.