Cancer is a leading cause of disease-related death in children. In Europe, 25% of children diagnosed with cancer die due to relapse of their disease, usually after intensive treatment. And of the children that are cured, over 70% has serious late effects, ranging from psychologic problems to cardiac failure and secondary cancers. This shows the clinical need for the development of new targeted drugs that are less toxic and more effective.
“About 75% of new drug development fail in early developmental stages and 9 out of 10 clinical trials fail”
Failure in drug development and failure of clinical trails results in waste of money and time being wasted on the investigation of non-promising compounds. The traditional clinical drug development model has largely been unchanged for more than three decades and fails to identify and validate promising compounds, specifically in paediatric cancer.
The scientific challenge
The low success rate of clinical trials in paediatric cancer is caused by multiple factors.
- Cancer in children is rare, making the study groups small and investments unattractive
- There is a lack of paediatric cancer specific biology driven approaches in which target identification and validation is key.
- Data sharing between academia and industry is inefficient and there is a lack of multidisciplinary scientist for paediatric targeted drug development
New investigator-initiated, biology-driven approaches, specific for paediatric cancer are needed to validate novel therapeutic strategies. This approach includes the identification and validation of targets and target-drug combinations that can improve survival and quality of life. The power of next-generation-sequencing techniques and development in immunology research have led to new insights in cancer biology and identification of novel targets. Many new genomic, epigenetic and immunologic tumour driving events have recently been identified. However, the translation of these discoveries into therapeutic strategies remains challenging. Industry, lacks proper model systems for paediatric cancer and it is difficult to make a selection of best new therapeutic targets, while for academia incentives are lacking to take their discoveries to the patient. Therefore, there is a strong need for a new generation of creative, entrepreneurial scientist that will become Europe’s next generation of leading researchers that will develop novel strategies in pre-clinical drug development and bridge the gap between academia and industry.
- Gain knowledge in 8 difficult to treat paediatric cancers to overcome the limitation of their rare nature by efficient knowledge and date-sharing between beneficiaries and partners
- Exchange expertise in the 4 steps of the proof of concept pipeline for pre-clinical drug development of genetic, epigenetic and immunological targets
- Train a new generation of young scientist to translate science to therapeutic applications fuelling innovation and entrepreneurship
To achieve our goal, the efficient validation of new target-drug combinations in paediatric cancer, VAGABOND has defined 3 research work packages. These work packages study different types of targets but all try to generate the same Proof of Concept packages and thereby closely interact.
For efficient implementation of VAGABONDs program 4 generic work packages are integrated in the work plan.