The goal of the Vitanza Lab is to find new treatments that are both safe and curative for high-grade, aggressive pediatric brain and spinal cord tumors, such as diffuse intrinsic pontine glioma (DIPG), diffuse midline glioma H3 K27M-altered (DMG), and atypical teratoid rhabdoid tumor (ATRT). Our central efforts include:
- Generating new central nervous system (CNS) tumor laboratory models, including treatment-naïve DIPG/DMG models derived from patient biopsies and autopsy-derived models made possible through generous donations from patients and families across the country.
- Identifying biologic and epigenetic vulnerabilities in DIPG/DMG so that we can advance the best molecularly-targeted drugs to kill tumor cells.
- Designing and deploying chimeric antigen receptor (CAR) T cells, an immunotherapy that can be delivered directly into the brain for highly-specific targeted tumor killing – such as on our BrainChild clinical trials.
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Recent Key Publications
- January 7, 2025 Intracerebroventricular B7-H3-targeting CAR T cells for diffuse intrinsic pontine glioma: a phase 1 trial
- May 17, 2023 ONC201 in Combination with Paxalisib for the Treatment of H3K27-Altered Diffuse Midline Glioma
- January 9, 2023 Intraventricular B7-H3 CAR T Cells for Diffuse Intrinsic Pontine Glioma: Preliminary First-in-Human Bioactivity and Safety
- January 3, 2023 Locoregional CAR T cells for children with CNS tumors: Clinical procedure and catheter safety
- July 12, 2021 Locoregional infusion of HER2-specific CAR T cells in children and young adults with recurrent or refractory CNS tumors: an interim analysis
- March 25, 2021 Optimal therapeutic targeting by HDAC inhibition in biopsy-derived treatment-naïve diffuse midline glioma models