Comprehensive Evaluation of Immune Function in Patients Receiving Multimodal Therapy for High-Risk Neuroblastoma

Children with high-risk neuroblastoma undergo intensive multimodality treatment. Despite this, only~50% of patients are cured, highlighting a need for improved therapies. Because ~90% of patients with neuroblastoma are diagnosed before the age of 5 years, the impact of improved therapy selection has the potential to not only improve survival, but also impact the quality of the many years of life survivors of this disease may have. Unfortunately, we currently lack accurate methods to identify patients who are most likely to benefit from specific therapeutic modalities and those for whom new treatments are needed. Prior research has revealed that immune cells such as T cells play an essential role in killing cancer cells, but we know very little about how their functions change over the course of various components of neuroblastoma therapy. This proposal will focus on using new and highly detailed techniques to study malignant cells from patients and simultaneously study immune cell function from the same patients. Our twin goals are to identify biomarkers that can be used clinically to predict therapy response to optimize treatment selection, and to understand immune cell response during therapy in order to develop novel therapies.