AI/ML derivation and experimental validation of ATRT biomarkers of survival and response to treatment

Atypical teratoid rhabdoid tumors (ATRT) are challenging pediatric brain tumors that most often affect children age 3 and younger. While there have been improvements in clinical outcomes with multimodal therapy, there remains significant morbidity and toxicities associated with intensive therapy. The goal of this study is to utilize computer and mathematical algorithms to make predictions of possible biomarkers in ATRT that predict response and survival. We now have very sophisticated computer modeling that allows us to use ATRT tumor data, including mutations, changes in the copy number of genes, changes in the RNA, and changes in methylation to make predictions. Importantly, this project is a collaboration where we combine these computer models and then test the predictions in the laboratory using cells derived from ATRT patients. This is an extremely new method of analyzing already existing genomic data, and ATRT data has never previously been assessed in this way.