Malignant tumours in children and adolescents are rare diseases with different prognosis and biologic behaviour. Prognosis of childhood cancer has improved considerably in recent decades and survival is approximately 70% in western countries. However, even with the current multimodal therapies, a considerable number of these patients still relapse and eventually die due to progressive or refractory tumours.
To improve the efficacy of anticancer therapies in children we have established a Translational Research Program in Paediatric Cancer consisting on:
Molecular characterisation of paediatric tumours.
Identification of new molecular targets.
Screening of new drugs in cell lines and in animal models.
Phase I-II clinical trials.
We start with the identification of genes and pathways candidates to be targeted using different platforms, followed by the validation of the identified targets in cell lines and primary tumours and the selection of appropriate candidates. The next step consists on testing the effects of drugs in vitro in cell lines and in vivo in mouse xenografts.
To translate the results of this research into the clinical scenario the program includes the development of phase I-II trials. Considering that cancers in children are different from tumours of adults we need to test new drugs in early phase clinical trials specifically designed for children.
In summary, the promotion of early clinical research in children with cancer combined with a better knowledge of the tumour biology will allow a more effective introduction of new targeted therapies into the clinical practice.