ITCC-P4 will set up a sustainable paediatric preclinical platform to run the evaluation of new compounds as single agent or in combination in order to support the development of new anticancer medicines for children and adolescents with cancer, as well as better understanding of the biology of paediatric malignancies.
Indeed, the regulatory environment is changing with the recently entered into force US FDA RACE for children act and the ongoing revision of the European Pediatric Regulation. From now on, the paediatric development of anticancer medicines will be driven by science (relevance of drugs targets or mechanisms of action with regard to tumour biology) rather than by the adult cancer indication. In this new regulatory ecosystem, ITCC-P4 will provide access to a saturated panel of well characterized (including molecular characterization) preclinical paediatric tumour models. Both FDA and EMA, as well and other agencies, are calling for biology and preclinical data to best informed decisions.
ITCC-P4 led on the development of an international consensus on what is the minimal preclinical requirements to inform paediatric development of anticancer medicines.
ITTC-P4 in partnership with the NCI funded Pediatric Preclinical Testing Consortium (www.preclinicalpivot.org) developed recommendations on the use of cell-based and patients derived xenografts models for paediatric solid and liquid malignancies, as well as guidance on the scope and content of preclinical proof-of-concept data packages to inform clinical development dependent on clinical urgency. It includes special recommendations on the use orthotopic brain tumor models and immune-oncology models, as well as methodology guidelines for running and analysing experiments.
This initiative was supported by Solving Kids’ Cancer, Neuroblatoma UK, Sohn Conference Foundation and ITCC-P4 grant.
The international consensus paper has been published in Molecular Cancer Therapeutic.
International Consensus on Minimum Preclinical Testing Requirements for the Development of Innovative Therapies For Children and Adolescents with Cancer.
Mol Cancer Ther. 2021 Aug;20(8):1462-1468 (DOI: 10.1158/1535-7163.MCT-20-0394)
