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Models, Mutations and Therapies

Laboratory Research

Dr. Nicholas Vitanza leads the Vitanza Lab, which aims to develop clinically relevant laboratory models of DIPG, identify key molecular and epigenetic vulnerabilities in DIPG, and test CAR T cells against aggressive CNS tumors.

Building Better DIPG Models

Scientific modeling allows us to more closely observe DIPG or DMG tumors in order to explain their behavior and illuminate new vulnerabilities that we can target. In consultation with our neurosurgery team, every patient with DIPG is considered for biopsy. When a biopsy is performed, samples are sent for clinical testing, genetic testing and to our lab to develop new models of this disease. We obtain samples prior to radiation or chemotherapy, creating a critical resource for our studies and the entire research community. Our models provide greater insight into key genetic and immunologic vulnerabilities that will point the way to new treatments and potential cures.

Key Mutations

Cancers are often caused by changes in key genes that control cell growth and expansion. Different kinds of cancers have different mutations. By identifying which mutations are responsible for DIPG, we are beginning to discover ways in which we may be able to inhibit a tumor’s ability to grow and survive.

DIPG and DMG have mutations in histone-related genes. Histones are proteins that package genetic materials. The most common histone mutations in DIPG or DMG are H3.3 or H3.1.

Using molecularly or epigenetically targeted drugs, Dr. Vitanza has shown key vulnerabilities in these tumors and discovered novel therapies that may one day provide improved outcomes for children. 

New Therapies for DIPG

We also focus on finding new ways to treat our patients diagnosed with DIPG. While radiation and chemotherapy are the standard of care, we are looking for therapies that can provide more targeted, specific effects. Through a series of comprehensive drug-screening tests, we have identified new potential combinations of drugs. We also have identified a chemotherapy regimen, which is not curative but improves quality of life for our patients.

Our work has shown that DIPG-specific immunotherapies should focus on activating certain types of cells that can effectively overcome and destroy tumor cells. Our work provides critical background to investigators currently developing immunotherapies for DIPG.