The relatively small number of children with cancer limits the data available to determine the potential benefits of new anticancer agents for this population. To address this, there is a need for pre-clinical models, such as transgenic or xenograft animal models and cell cultures, to screen and evaluate the effectiveness of new agents.
We can access numerous molecular pathways, targets, and potential small-molecule inhibitors in the post-genomic era. Therefore, it is crucial to have effective screening methods and models to identify the most promising candidates. However, many molecular targets suitable for drug therapy in pediatric cancers remain unidentified. Genetically engineered mouse models, which allow for the targeted manipulation of specific genes, provide valuable opportunities to observe tumor progression, regression, and metastasis in vivo. Advances in small animal imaging offer powerful tools for functional imaging of these models. Additionally, RNAi-based genome-wide screens are promising for validating potential drug targets in childhood cancers. Unlike adult cancers, which are often the result of complex genetic changes, childhood cancers generally have simpler genetic and developmental origins, enhancing the potential for discoveries through RNAi-based methods.
We plan to recruit up to four investigators with expertise in mouse models, genome-wide screening, RNAi, and functional imaging to establish a core experimental therapeutics program. The goal is to utilize pre-clinical cancer models to identify and validate “druggable” targets in pediatric cancers, some of which may also be relevant to cancers common in adults.