Current Research Project

Next Steps in the Rapid Preclinical Development of a Targeted Therapy Combination for Diffuse Intrinsic Pontine Glioma (DIPG)

Sponsored by the Lyla Nsouli Foundation

cc-TDI is a leader in the multi-institution consortium dedicated to moving a multi-agent chemotherapy into clinical trial for Diffuse Intrinsic Pontine Glioma (DIPG). DIPG is a rare tumor of the brainstem that occurs almost exclusively in children under 10 years old. It represents one of the most devastating diagnoses among pediatric cancers, with an average survival rate of just nine months.

Remarkably, this is the first time that a group of basic and translational scientists and physicians from institutions throughout North America and Europe have come together as a consortium to focus on a bench-to-bedside approach to rationally target therapy for children with DIPG. Our Scientific Director, Charles Keller, MD, was responsible as co-Chair of the Children’s Oncology Group brain tumor new drugs committee (CNS-DVL) for building this coalition and coordinates activities among the 13 institutions worldwide.

Throughout 2014 and 2015, consortium members made great progress sequencing DNA and RNA samples that will help us move closer to the development of a 2-drug combination trial. One of these agents has already been identified for use (and is anticipated to be made available for a cooperative group trial by a major pharmaceutical partner!). Over the next year, cc-TDI will be dedicated to working with the consortium to define the two-drug combination of choice.

Our Goal

Move the most effective two-drug combination therapy for DIPG into an early phase clinical trial.

Our Timeline

The phase I trial of the first drug we have defined for DIPG begin in 2016 in partnership with the Pediatric Brain Tumor Consortium clinical trials group. The clinical data may provide the basis for moving to a Phase II trial in 2016. We aim to identify the 2-drug combination by 2018.


Consortium Partners:

Charles Keller, MD – Children’s Cancer Therapy Development Institute

Oren Becher, MD – Duke University Medical Center

Michelle Monje, MD, PhD – Stanford University

Maryam Fouladi, MD – Cincinnati Children’s Hospital Medical Center

Cynthia Hawkins, MD, PhD – University of Toronto

Xiao-Nan Li, MD, PhD – Baylor College of Medicine

Dannis G. van Vuurden, MD, MSc, & Esther Hulleman
VU Cancer Center Amsterdam

Jacques Grill – Institut Gustave-Roussy, Villejuif, France

Eric H. Raabe – Johns Hopkins University

Katherine Warren, Paul Meltzer, and Martha Quezado
National Institutes of Health

Mariko DeWire – Nationwide Children’s Hospital,

Dennis Steindler – University of Florida

Marta Alonso – University of Navarra, Madrid, Spain

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