Novel therapies are desperately needed for pediatric solid tumors, where more than 30% of patients die from their disease. Immunotherapies offer significant potential to meet this need, but this potential has not yet been realized in children. Immune checkpoint inhibitors (ICIs), a type of immunotherapy, have shown success in many adult cancers but rarely work for children. This is partly because pediatric tumors have fewer mutations than adult tumors.

Diffuse Intrinsic Pontine Glioma (DIPG) is the second most common malignant brain tumor in children and the leading cause of pediatric cancer death. We have developed the first published experimental model system to study DIPG, and using this have discovered some of the molecular factors that drive DIPG tumor growth. We now propose to use our mouse model system to test a therapeutic strategy combining two drugs.

Background