About the Project
Partner4VRT brings together European and U.S. researchers to improve knowledge and treatment of very rare cancers in children, adolescents, and young adults, focusing on pancreatoblastoma and adrenocortical carcinoma. Because these cancers are extremely uncommon, the project will combine patient data from multiple countries to create a larger dataset for research.
By analysing clinical information, genetic data, treatments, and patient experiences, researchers aim to identify biomarkers for earlier diagnosis, better risk assessment, and more effective therapies. The project will also address ethical, legal, and technical challenges of international data sharing - creating a framework for future global collaboration on rare paediatric cancers.
Project Goals
A pilot collaborative observational study will be established as proof of concept to accelerate progress by joining forces and increasing the accrual of sufficient numbers of patients for meaningful analyses. The target cohorts will be analysed by age, sex, race, ethnicity, and clinical presentation.
Genetic, clinical, and treatment-specific characteristics will be collected to enable the identification of potential biomarkers for possible early detection, diagnosis, prognosis and risk classification, therapeutic response, and drug development.
Patient-centric research will be fostered by involving advocacy groups and patients in designing ethical data-sharing protocols as well as patient-reported outcomes (PROs).
This smaller-scale project will address practical and legal challenges related to data collection, regulatory alignment, and integration of complex data types such as molecular data, imaging, and PROs. The goal is to establish a framework for future expansion to additional rare paediatric tumours, enabling international collaboration.
The direct grant will involve the European EXPERT Group for Very Rare Tumours and include relevant stakeholders (Legal and IT experts, CCI-E, SIOPE, EUROCAN) and will use the “Partner” Registry maintained by ERNPaedCan.
Project Impact & Ambition
The initiative is expected to deliver harmonised EU–US definitions and common data elements for rare paediatric and AYA cancers, along with a functioning pilot observational study on PBL and ACC that demonstrates the feasibility of joint international patient accrual.
It will generate an integrated clinical, genetic, molecular, imaging and PRO dataset to enable the identification of biomarkers for early detection, diagnosis, prognosis, risk classification and treatment response. The project will improve understanding of disease presentation and outcomes across demographic groups and embed patient-centric research practices through ethical data-sharing protocols and inclusion of PROs.
It will also clarify practical, legal and regulatory challenges in cross-border data collection and propose initial solutions for integrating complex data types. Ultimately, the pilot will create a scalable framework for expanding collaboration to additional rare paediatric tumours and strengthen long-term EU–US research partnerships.
Consortium & Kick-off Meeting
List of Project Partners:
- ST. ANNA KINDERKREBSFORSCHUNG GMBH
- EBERHARD KARLS UNIVERSITAET TUEBINGEN
- AZIENDA OSPEDALE UNIVERSITA PADOVA
- INSTITUT CURIE
- KLINIKUM DORTMUND GGMBH
- UNIVERSITATSKLINIKUM AUGSBURG
- GDANSKI UNIWERSYTET MEDYCZNY
- AZIENDA SOCIO-SANITARIA TERRITORIALE DEGLI SPEDALI CIVILI DI BRESCIA
The partners met on 13 January 2026 for the online kick-off meeting of the project. The meeting included a general project overview, presentations of all work packages (WP1–WP6), and discussions with representatives from DG SANTE and HaDEA. More on social media about the success of the kick-off meeting.
Project Coordinators
Ruth LADENSTEIN, Coordinator, Children’s Cancer Research Institute (CCRI)
Ines BRECHT, Past Chair of EXPeRT Group
Daniel Orbach, Current Chair of EXPeRT Group
Project Contact
Zoltan Dobai, Deputy Head of the Research Management Office, Children’s Cancer Research Institute (CCRI)
+43 660 3545753, Partner4VRT@ccri.at
Dissemination Materials & News
More to come - Stay tuned!
