DIPG remains a fatal disease and new treatment approaches are necessary. A novel Cancer Systems Biology approach identifies master regulator (MR) proteins that are necessary for tumor growth and predicts and prioritizes drugs that reverse their effects. This study will apply such an approach to evaluate new treatment strategies in DIPG.
What are the goals of this project?
We will analyze the PBTA-CBTN and other DIPG gene expression data using innovative computational algorithms to describe the landscape of MR proteins in DIPG tumors and prioritize drugs by their ability to shut down the most aberrant MR proteins both in individual patient samples and across the disease as a whole. We will evaluate the efficacy of treatments predicted by the MR approach in patient-derived DIPG cell lines and animal models.
What is the impact of this project?
This will advance drug development and suggest novel therapy approaches in DIPG to improve outcomes in this difficult-to-treat disease.
The Children's Brain Tumor Network contributed to this project by providing access to the Pediatric Brain Tumor Atlas.
Paul Kirk Reardon
Javad Nazarian, PhD, MSc
I am an investigator at the Center for Genetic Medicine in Children’s National Hospital in Washington, D.C., and an assistant professor in Integrative Systems Biology at the George Washington University. I received my PhD from the George Washington University in Genetics in 2005. My dissertation
Children’s National Hospital
Brainstem glioma- Diffuse intrinsic pontine glioma
A presumptive diagnosis of DIPG based on classic imaging features, in the absence of a histologic diagnosis, has been routinely employed. Increasingly however, histologic confirmation is obtained for both entry into research studies and molecular characterization of the tumor.[