As a physician-scientist, my laboratory is devoted to the development of novel molecular therapies for advanced childhood solid tumors associated with high morbidity or mortality. To achieve this goal, my laboratory utilizes physiologically-accurate, genetically-engineered mouse models (GEMMs) of three related pediatric small round blue cell tumors: medulloblastoma, alveolar rhabdomyosarcoma and embryonal rhabdomyosarcoma. Medulloblastoma is a childhood brain tumor with a predilection for leptomeningeal metastasis for which radiation-sparing treatments are needed, whereas alveolar and embryonal rhabdomyosarcoma are aggressive muscle cancers that are difficult to cure if not completely resected or if metastatic. The long-term emphasis of my laboratory’s research is the investigation of tumor progression, since advanced disease remains the greatest challenge in the treatment of children with these cancers. The ultimate goal of my research program is to develop pre-clinically tested, molecularly-targeted therapies to halt progression or induce regression for gross residual disease, metastatic disease and relapsed disease.
