Novel Immune Mediated-Gene Therapy for Pediatric High-Grade Glioma

Award: $519,530 over three years
Principal Investigators: Maria G. Castro, PhD, R. C. Schneider Collegiate Professor of Neurosurgery, Professor of Cell & Developmental Biology at University of Michigan Medical School and Pedro R. Lowenstein, MD, PhD, Richard C. Schneider Collegiate Professor of Neurosurgery, Professor of Cell & Developmental Biology at University of Michigan Medical School
Co-Investigator: Karin Muraszko, MD, Chair and Julian T. Hoff, MD Professor, Neurological Surgery
Funding Partner: Samson Research Fund with funds raised through Hope for Kids

Pediatric high-grade glioma (pHGG) is a common form of highly aggressive pediatric brain cancer that accounts for the leading cause of death by disease among children in the United States.

The University of Michigan Medical School’s Departments of Pediatrics and Neurosurgery have pioneered a treatment approach that uses gene therapy-mediated delivery of therapeutic genes into the tumor — utilizing a combination of viral vectors that express: (1) a gene that induces tumor cells’ death and (2) another gene that trains the patient’s immune system to recognize and kill any remaining tumor cells. The viral vectors are delivered into the tumor cavity or the remaining tumor mass post-surgery to trigger an effective anti-tumor immune response.

This treatment strategy has been approved by the FDA for adult patients with glioblastoma multiforme, the most aggressive form of brain cancer, and a Phase I clinical trial has recently completed patient enrollment at the University of Michigan.

The Pediatric Brain Tumor Foundation’s grant will fund the needed experimental work in pre-clinical models to study the impact of the H3G34R mutation in reprogramming the glioma immune microenvironment and get FDA approval to implement this therapy in children.