Collaboration boosts development of new drugs for pediatric cancer

Each year, about 9,000 children and adolescents in Europe are diagnosed with cancer. For several childhood cancers, the chances of a cure are high. However, one in four of these children does not cure with current treatments. To stimulate the development of new, broadly applied drugs in pediatric oncology, researchers and industry are working together within the European Union-funded Innovative Medicines Initiative (IMI).

Broad platform

ITCC-P4 gGmbH originates from an IMI project and stands for Innovative Therapies for Children with Cancer Paediatric Preclinical Proof-of-concept Platform. In addition to several research groups from the Princess Máxima Center, researchers from the German Hopp Children's Cancer Centre (KiTZ/DKFZ) in Heidelberg are also involved, including Dr. Marcel Kool, research group leader at the Máxima Center and KiTZ. He explains: 'Because we are dealing with very many different types of childhood cancer, with different subtypes, it is essential that the platform with preclinical models is as broad as possible. ITCC-P4 then uses these models to test, for example, drugs already used in adults and new drugs. The researchers are also looking for biomarkers, recognition points, with which the participation of children in clinical studies can be more targeted.'

PDX models

Within ITCC-P4, 400 different so-called PDX models are used. In these models, cancer cells derived from patients are transplanted into a mouse. This allows researchers to study the effect of a drug in the lab in a living organism, while already looking at human material.

Prof. Dr. Jan Molenaar, leader of the Molenaar group: 'At the Máxima Center, several tumors from these PDX models have already been cultured in the lab as so-called organoids. These mini-tumors can then be used for large-scale robotic drug screens. This is used to select the best drugs and combinations, which are then tested in PDX models. Several researchers from the Máxima Center are involved to further expand both the PDX and organoid platforms for preclinical studies. It is great that what we have developed in the Máxima Center will be used more widely from this partnership. And that this way we are also contributing to finding better treatments for children with cancer.'

Acceleration

In the future, these types of preclinical models specific to pediatric cancer may play an important role in the approval of new drugs. Already in the United States, all new cancer drugs submitted for approval must also be tested for the treatment of pediatric cancer if the mode of action matches the treatment of children with cancer. It is expected from ITCC-P4 that this will also come into play in Europe and that testing in pediatric cancer-specific preclinical research models will become a prerequisite, as in the US.

Public-private collaboration

Because pediatric cancer is a rare disease, international cooperation is crucial. The Máxima Center is therefore a partner in various collaborative projects in both care and research. Prof. Dr. Olaf Heidenreich, research group leader: 'The collaboration between public research institutions and private industry is a flywheel to stimulate the development of new medicines for pediatric cancer. By making our PDX models available within the ITCC-P4 together with KiTZ in Heidelberg, we are boosting the flywheel even further. We stimulate new developments within Europe and take steps to achieve our mission to cure every child with cancer with optimal quality of life'.

Together with eleven other research institutions and three biotech companies, the Princess Máxima Center is a shareholder of non-profit start-up ITCC-P4.