Arginine methylation as a potential therapeutic target in high-risk neuroblastoma

University of Rochester, Rochester, NY

Neuroblastoma, an embryonal malignancy of the developing sympathetic nervous system, remains a leading cause of childhood cancer deaths. The goal of this project is to develop novel targeted therapies for high-risk neuroblastoma based on specific, rational molecular vulnerabilities we have uncovered. We have recently discovered a novel regulatory pathway involved in the cancerous properties of neuroblastoma cells. Our preliminary studies have further confirmed the therapeutic potential of using drugs to inhibit this pathway’s role in neuroblastoma cancer cells. In this project, we propose a paradigm-shift approach to determine the role of this pathway in the development of sympathetic nervous system and neuroblastoma tumorigenesis. By better understanding this regulatory pathway, we ultimately aim to improve the treatment of patients with neuroblastoma and other malignancies.