ITCC-P4 International Workshop: Improving pediatric oncology drug development through preclinical research Amsterdam, The Netherlands

Cancer remains the leading cause of disease-related death in children. For the ~25% of children who experience relapses of their malignant tumors, usually after very intensive first-line therapy, curative treatment options are scarce.

Over the last few years, regulatory initiatives in the US and Europe have significantly changed the landscape of new oncology drug development for children. However, there is a major need to improve the field to bring effective new therapies to patients more rapidly and better prioritise drugs to be developed in the pediatric population among the numerous anticancer drugs currently developed in adults as well as drugs specifically developed for children.

Preclinical drug testing can contribute to identify promising treatment options that match the molecular and immunology makeup of the patient’s tumor and should be more systematically part of science-driven drug development plans based on the compounds molecular mechanism of action and the biology of pediatric malignancies.

Two preclinical projects have been recently launched to better address preclinical drug testing: the NCI Pediatric Preclinical Testing Consortium (PPTC) in the US and the IMI2 ITCC Paediatric Preclinical Proof of Concept Platform (ITCC-P4) in Europe.

There is a need to define an international scientific consensus on preclinical evaluation to facilitate and improve new anticancer drug development for children and adolescents in the currently changing international regulatory environment.