Transcriptional Regulation of Heterogeneous Populations in Choroid Plexus Carcinoma

The New York College of Osteopathic Medicine (NYITCOM) earned a federal grant to conduct focused research on choroid plexus carcinoma (CPC), a rare and aggressive pediatric brain tumor that forms in the cerebrospinal fluid–secreting tissue of the brain and responds poorly to current treatments. Supported by a three-year U.S. Department of Defense Rare Cancers Research Program Idea Development Award totaling $499,800, this study will leverage developmental biology and molecular approaches to define the transcriptional regulation and cellular heterogeneity that drive CPC pathogenesis. A central aim of the work is to identify molecular processes and therapeutic targets that could enable safer, more effective interventions that suppress tumor growth without harming children’s developing brains.

 

The team’s research interrogates transcriptional programs and factors—previously presented in international and national venues such as the International Symposium on Pediatric Neuro-Oncology and the Pediatric Neuro-Oncology Research Conference—where others have shared findings on cellular differentiation heterogeneity and the role of factors like Sox2 in CPC.

The translational perspective of this work is reflected in experimental results indicating that restoring multicilia in choroid plexus tumors, referenced in their work, can decrease tumor growth, demonstrating how mechanistic insights may point to novel treatment strategies. Training physician-scientists is also an integral component of the project with DO-PhD students working in the lab helps build on clinical research experience from a neurosurgical-oncology fellowship and working toward predicting tumor evolution and treatment response by leveraging knowledge of tumor heterogeneity.

This dual focus on rigorous basic science and clinical relevance positions the program to contribute both to fundamental understanding of CPC transcriptional regulation and to identification of candidate targets for therapeutic development. The funded project emphasizes collaborative dissemination and peer engagement, with team members presenting internationally and participating in conferences to integrate emerging insights across pediatric neuro-oncology.