Pediatric Sarcomas: Sarcomas are among the most difficult-to-treat cancers in pediatric oncology, with metastatic forms having the highest mortality. We have established genetically defined human cell-based models and genetically engineered murine models for the pediatric skeletal muscle cancer known as rhabdomyosarcoma. Using these models, we can study the causative role of certain genetic changes (e.g. chromosomal translocations and oncogenic RAS) in rhabdomyosarcoma formation and treatment resistance. Specific goals of this research program include the identification of signaling pathways corrupted in rhabdomyosarcoma, with focus on the PAX3-FOXO1 mutation and its downstream effectors and oncogenic RAS, and identification of new therapeutic targets for treatment of this childhood cancer.
