Molecular Targets in Embryonal Tumors

Molecular profiling studies have the potential to describe the complexity of cancer genotypes and phenotypes, and provide an opportunity to link signalling pathway deregulation with potential therapeutic strategies.

High-throughput genomic, proteomic and epigenomic data have expanded our knowledge of oncogenic pathways and their functional roles and interactions in embryonal tumors. We preclinically assessed the suitability of selected targets identified within these signalling pathways for novel therapeutic approaches in vitro and in vivo.

Molecular targets identified in our profiling studies of neuroblastoma and other embryonal tumors include MYC and Trk signalling elements, the anaplastic lymphoma kinase, ALK, as well as relevant factors of epigenetic regulation such as histone demethylases. The functional role of these promising targets has been analyzed in cell culture models and xenograft mouse models.

Molecular profiling provides us with a wealth of data including potential targets for novel therapies. The major task in the next few years will be to harmonize biological datasets and functional experimental data to define algorithms for molecular target prioritization to support therapy choices guided by biology. Innovative approaches such as systems biology and sophisticated transgenic mouse models are needed to achieve this goal.