Funded by the Dick Vitale Gala
There are over 170 FDA approved chemotherapeutic medications. These medications have shown benefit to a particular segment of cancer populations, often multiple groups of patients. Because of the rarity of pediatric cancers, very few of the medications that are used to improve the lives of children with cancer are FDA approved for that specific use, so called off-label use. Incorporating new medications into childhood cancer treatment often involves testing one agent at a time across a variety of diagnoses followed by focusing on a subset or a few types of cancer. This process has been slow to identify new agents in a group of tumors known as sarcomas. Recently, there have been a significant number of medications approved and it would be impossible to test them all on patients in the manner described above. Furthermore, studies in models of sarcomas have not always been reliable predictors of the medications because they have been tested in amounts that are not achievable in humans or for durations that cannot be achieved without unacceptable side effects. We propose looking at many FDA approved agents at levels that can be safely achieved in people across a panel of sarcoma models to identify agents and then combinations of agents that can be rapidly incorporated into a disease specific trial. We aim to test these agents, and, in the coming two years, identify promising combinations in the four most common sarcomas: osteosarcoma, Ewing Sarcoma, alveolar rhabdomyosarcoma and embryonal rhabdomyosarcoma.