It is a well known problem that little if any industrial funding is made available for design of diagnostic and prognostic assays for paediatric cancer and even more in case of the development of therapeutic compounds specifically targeted at rare paediatric cancers. The present project aims 1) to identify, validate and apply novel biomarkers for improved outcome prediction and treatment stratification and 2) to minimize toxicity and maximize efficacy by optimizing the dose of chemotherapy for each individual patient.
The identified biomarkers can be worldwide implemented in the diagnostic work-up and therapeutic stratification of neuroblastoma patients. The potential application is two-fold. First, using such prognostic and therapeutic stratifiers, clinicians can avoid a long recognised problem of over- and undertreatment of specific patient groups. Given the high costs of current treatment, unnecessary treatment imposes unneeded costs for society. Most importantly, these multimodal therapies expose the children with neuroblastoma to unwanted treatment risks, toxicity, psychological burden and long term side effects. Undertreatment is even more dramatic, as valuable time will be lost before these children will be recognised as high risk (typically after their first relapse). As a second application, such classifiers will be extremely valuable for identifying patient subgroups for inclusion in clinical trials for new therapeutic compounds.