In 2022, 1 in 326 children were diagnosed with cancer in the EU before their 20th birthday. Although there have been improvements in research into childhood cancers, the number of new and effective therapies remains low - only 9 of the 150 cancer medicines approved in the last decade targeted children’s cancer.
Part of the reason for that is that most research tools, including tumour models, are designed for adult cancers, not children’s ones. And childhood cancers often behave very differently to adult versions – they are biologically different, often more aggressive and progress more quickly.
ITCC-P4 was the first in the world to develop preclinical drug tests for children with cancer, producing more than 350 of a type of preclinical model called patient-derived xenografts (PDX), for 20 childhood cancers.
Patient-derived xenografts (PDX) are preclinical models that preserve the main molecular features of tumours and can be used to evaluate new therapies and test treatment resistance. Since the end of the IMI2 project, ITCC-P4 successfully transitioned into a non-profit company, providing access to models, drug testing in collaboration with three contract research organisations (CROs) and scientific expertise for drug development.
A much-needed resource
The services provided by ITCC-P4 have proven popular. To date, the non-profit has received 100 project enquiries from 71 different organisations, and of those, industry accounted for approximately 70%, with the remainder coming from academia.
“The demand is high and it is continuing to grow,” says Eva-Maria Rief, director of operations at ITCC-P4, pointing out that those 100 enquiries came in only two and a half years since the non-profit organisation was set up.
Yet, working with the ITCC-P4 project is not the same as contracting usual research services. ITCC-P4 operates differently – it is not a case of clients simply ordering what they want and ITCC-P4 going away and implementing.
Rather, customers come to ITCC-P4 and explain what they want to achieve. ITCC-P4 then prepares a proposal and plan, backed by evidence and academic experts’ knowledge gained from the original IMI2 project. If the customer wants to proceed, then ITCC-P4, in collaboration with three contract research organisations (CROs), carries out the experiments and provides the client with a final report.
“Working with us is a bit different from commissioning a standard research service,” says Gilles Vassal, the chief operating officer of ITCC-P4. “We combine operational study delivery with paediatric oncology expertise, and clients have told us that this approach adds value.”
The ITCC-P4 IMI2 project combined the expertise and know-how of paediatric oncologists and childhood cancer researchers across Europe, and built up a network of public and private partners that had never existed before. All childhood cancers are rare diseases, meaning experts can be spread out across countries, and the connection to this network has proven highly valuable.
To date, five studies have been completed, and twelve service agreements have been signed. The completed studies provided pharmaceutical companies with important preclinical information that helped them to fulfil their obligations under the EU and US regulatory frameworks. As an example, one completed study evaluated targeted treatment approaches and combinations using models of paediatric high-grade glioma, an aggressive type of brain cancer. The results showed clear differences in treatment response depending on the molecular characteristics of the models and generated relevant evidence to help evaluate the therapeutic approach further. In total, 58 enquiries have progressed beyond the initial assessment stage and are now in the subsequent phases, which include feasibility evaluations, model selection, proposal development or study planning.
The most popular cancers that tumour models were requested for included neuroblastomas (a type of cancer that develops in nerve cells and almost exclusively affects children), high-grade gliomas and medulloblastomas (a fast-growing type of brain cancer that primarily affects children).
The enquiries showed that a vast range of innovative potential therapies are being explored, with clients searching for solutions using targeted therapies such as antibody-drug conjugates (ADCs), T-cell engagers, radiotherapy and biomarkers.
“There is an urgent need to accelerate and enlarge the development of new anticancer medicines for children and adolescents. By bringing together relevant paediatric cancer models, molecular data and scientific expertise, ITCC-P4 enables innovative research and generates the robust preclinical evidence needed to prioritise the most promising therapies for further development within an increasingly science-driven and patient-centred regulatory framework,” explains Vassal.
Dialogue with regulators on evolving standards
One thing that customers have found particularly attractive to date is the ongoing dialogue between ITCC-P4 and the European Medicines Agency (EMA), especially in light of the coming revision to the EU pharmaceutical legislation. During the project, channels of communication were opened with regulators, and ITCC-P4 are continuing to engage with regulators to ensure that their scientific approaches remain relevant to and aligned with paediatric drug development regulatory requirements. ITCC-P4 participates in the EMA’s stakeholder interaction activities in the non-clinical domain.
In February 2026, the EMA developed a concept paper on proof-of-concept data to support the development of anti-cancer medicinal products in children. ITCC-P4 took part in the corresponding public consultation, and contributed to a workshop organised by EMA on advancing proof-of-concept data in the development of anti-cancer drugs for children. This workshop will feed into a soon-to-come EMA reflection paper.
A constantly evolving platform
Within ITCC-P4, research is continually ongoing to develop the original models produced as a result of the original IMI2 project and improve the services on offer. In 2026, brain tumour study capabilities were developed further, and a new central tissue microarray asset was implemented, to help facilitate systematic analyses of protein expression across a breadth of ITCC-P4 models. Support for biomarker-driven model selection and bioinformatic analysis was improved, and models of rare paediatric cancers were developed further. ITCC-P4 is currently exploring how tumour organoids could improve further preclinical testing.
ITCC-P4 is continuing to work with other research consortia and partake in grant proposals. “In the field of paediatric research, we are well recognised,” says Vassal.
One example is the PROTECT project, coordinated by the German Cancer Research Center (DKFZ), which aims to establish a platform for the development and preclinical evaluation of innovative protein degraders – meaning drugs that use the cell’s natural waste disposal system to destroy disease-causing proteins rather than just blocking them. By taking part in these research projects, new scientific advances can be made and then integrated back into the ITCC-P4 offering.
Transitioning from a funded project to an established sustainable operating model still remains an important challenge. While income from commissioned studies supports the delivery of individual client projects, it is challenging to get additional financial support which is needed for the shared infrastructure and central capabilities that make these activities possible.
How can you access ITCC-P4?
If you or your research organisation is interested in using ITCC-P4’s models, you can send a query to the team using this form or call the headquarters at +49 (0)6221 71411140. You can take a look at what cancer models are on offer here: R2 Genomics Analysis and Visualization Platform: ITCC-P4 (gGmbH open)