Funded by the 2016 Vitale Gala in memory of Julia Mounts.
Rhabdomyosarcoma (RMS) is the most common soft tissue cancer in children. Treatment and survival for children with RMS has not changed for thirty years. Aggressive treatments result in deformities and other health issues in survivors. This highlights a need for more understanding of RMS. Identifying the key features of RMS would guide the search of new treatments. The Pediatric Cancer Genome Project (PCGP) at St. Jude Children’s Research Hospital identified many genetic changes in RMS. The most common subtype of RMS is embryonal rhabdomyosarcoma (ERMS). ERMS had many different genetic changes.
It remains unclear which identified genetic changes drive ERMS formation. Identifying the drivers will expose new targets for treatment. Our lab proposes to identify the essential drivers of ERMS. We will utilize a cell-based approach to explore the genetic changes identified by the PCGP. This system allows us to quickly test the effect of each of the potential driving changes.
We will develop new model systems that more closely resemble our patient’s tumors. Next, we will use these simple models to test new treatments. It is our hope that models with the minimal gene changes necessary for ERMS will simplify our search for new treatments. The result will enable us to pinpoint methods to kill tumor cells. The long-term goal is to couple the specific genetic changes to new therapies.