Funded by Matthew Ishbia and the Dick Vitale Pediatric Cancer Research Fund
DNA contains the story book of each human, written in our genome. Sometimes a single letter changes the meaning of a word, such as better to bitter. Likewise, in some children a small DNA change encourages cancer to form and grow. In childhood sarcoma, we recently discovered that certain DNA changes in cancer-causing proteins lead to errors in the rest of the genome’s ability to remember its cellular purpose. We found this was happening by formation of large “super-clusters” at cancer-causing genes. The goal of our research is to discover why and how these super-clusters form. We will explore the super-clusters using leading edge technologies including 3-dimensional genomic modeling, chemistry, cancer biology, and drug development focused on a deadly form of childhood cancer, called rhabdomyosarcoma. We anticipate finding that the super-clusters are integral to rhabdomyosarcoma progression; and our work will illuminate potential new treatment targets and routes, based on modifying the genetic error that is causing the cancer. For example, if we develop drugs that stop the formation of the super-clusters, will we also selectively kill the cancer cells? This new work will provide the scientific data to support a new class of therapies for children with these deadly cancers.