Ami V. Desai, MD, MSCE
Assistant Professor, Department of Pediatrics, University of Chicago
Determining the active profile of entrectinib (RXDX-101) and evaluating its role in neuroblastoma and other pediatric cancers
determining the active profile of entrectinib (rxdx-101) and evaluating its role in neuroblastoma and other pediatric cancers
Entrectinib (RXDX-101) is a drug that targets certain changes in cancer cells that are seen in many adult and pediatric cancers. This drug is being studied in adults and work done in the laboratory indicates this therapy targets specific pathways in neuroblastoma, a type of childhood cancer. In this proposal, we will perform the first pediatric study of entrectinib, in order to identify a safe dose in children with solid tumors of the body and brain tumors. We will learn more about how the body reacts to this drug and how well this drug inhibits certain changes in cancer cells. Finally, we will determine if children with treatment-resistant neuroblastoma and children with other solid tumors respond to this therapy and have evidence of tumor shrinkage. If children with neuroblastoma respond to this therapy, we will perform other trials to determine the potential utility of incorporating entrectinib in the upfront setting to improve outcomes for patients with high-risk neuroblastoma.