Skip to content Skip to navigation

Developing a Targeted Drug Therapy for a Universally Fatal Pediatric Brainstem Tumor

Seed Grants
Awarded in 2012

Interdisciplinary Initiatives Program Round 6 - 2012

Jennifer Cochran, Bioengineering
Michelle Monje, Neurology

Diffuse Intrinsic Pontine Glioma (DIPG) is a devastating illness that accounts for 10-15% of childhood brain tumors and is nearly always fatal. Few treatment options for DIPG exist, and the median overall survival for patients is only 9 months. To improve treatment for DIPG, there is a critical need to design new drugs and test them in cell and animal models that closely replicate the disease. As a result of the generous donation of tumor tissues from DIPG patients, the Monje lab has recently developed cell and animal models that closely mimic DIPG in humans and is currently working to identify new drugs that can kill DIPG cells. Most of these drugs, however, are unable to access tumor cells in the brain due to natural barriers set up by the body to block the passage of toxic compounds. The Cochran lab is developing drug carriers capable of delivering therapeutic cargo specifically to brain tumors and has demonstrated the ability of these carriers to localize to DIPG tumors following injection into mice. In this collaborative project, we will develop methods to chemically attach drugs to these tumor-targeting carriers for specific delivery to DIPG tumors. We will measure how effectively these new molecules target tumor tissue in DIPG models, and will test their ability to reduce tumor burden and increase survival. This approach has the potential to take unprecedented steps toward developing effective treatments for a currently incurable pediatric brain tumor, and will answer important questions surrounding brain tumor biology.