WASHINGTON, D.C. and BOSTON, MA (Sept. 12, 2016) —

, the largest nonprofit dedicated to the brain tumor community in the United States, with its partner, 

, the largest private funder of childhood cancer research grants, as well as several world renowned experts in the field of pediatric neuro-oncology, has announced a new awareness and fundraising campaign to support a major translational research and drug discovery program. Goals of the program include improving clinical outcomes for pediatric brain tumor patients and informing the development of the first-ever standard of care for treating pediatric high-grade gliomas, including DIPG — the deadliest of pediatric cancers. The campaign, called “Project Impact: A Campaign to Defeat Pediatric Brain Tumors,” was unveiled September 12 at the National Press Club in Washington, D.C. A replay of the live streaming from the event is available at 

“Treatment of pediatric high-grade gliomas has been extremely frustrating with little progress made over the past quarter century,” said Dr. Roger Packer of Children’s National Health System in Washington, D.C., and Scientific Director of the Defeat Pediatric Brain Tumors Research Collaborative. “New molecular insights provide hope that therapies will be dramatically more effective in the very near future. But we need to maintain forward momentum — from discovering the molecular and genetic underpinnings of these tumors, to understanding how these changes drive these tumors, and to ultimately developing effective, biologically precise therapies. This is a major opportunity for the field, patients and their families.”

This year, more than 4,600 children and adolescents (0-19 years) will be diagnosed with a pediatric brain tumor. There has never been a drug developed specifically to treat pediatric brain tumors and few standards of care or treatment options exist for children with brain tumors. In particular, pediatric high-grade gliomas have no standard of care and a survival rate currently approaching zero. Further, as no treatment has been developed that takes into account a child’s developing brain, treatments currently used (mainly radiation and chemotherapies) often inflict neurological, cognitive, developmental, and other deficits on children who survive their tumor. More information on pediatric brain tumors can be found 

David Arons, Chief Executive Officer for the National Brain Tumor Society stated, “Researching and developing new treatments for pediatric brain tumors is a particularly challenging task, which faces multiple — but interrelated – barriers that span the research and development spectrum from small patient populations, lack of effective preclinical models, to complex basic biology, regulatory hurdles and economic disincentives. To overcome these complex challenges, and get better treatments to patients, we needed to create an equally sophisticated intervention. We believe that having groups with complementary skills work together in a coordinated effort, sharing data and expertise, and tackling the problem from multiple angles as one team is the starting point for greater and faster progress.”

Project Impact and the Defeat Pediatric Brain Tumors Research Collaborative

Fundraising proceeds from “Project Impact” will help to fund the 

Defeat Pediatric Brain Tumors Research Collaborative

The Collaborative is an NBTS led-program, with support from partners the St. Baldrick’s Foundation, designed to accelerate research through a platform that fosters collaboration and data, information and material sharing. The platform consists of multiple interrelated “Cores” that work on critical areas of research simultaneously and in concert with one another, encourage sharing of findings real time.

Discovery Core (Molecular Diagnostics & Target Discovery):

 Stefan Pfister, German Cancer Research Center

Biomarker Core (Identification & Validation — Tissue & Serum): 

Nada Jabado, Montreal Children’s Hospital

Preclinical Modeling & Drug Screening Core:

 Suzanne Baker, St. Jude Children’s Research Hospital

 Collaboration with the Pacific Pediatric Neuro-Oncology Consortium

“This deadly disease can only be tackled by a world-wide collaborative effort, such as this one, between dedicated groups focusing on this disease with different expertise, including molecular biology, genetics, preclinical modeling, and clinical trial design,” said Dr. Pfister of the German Cancer Research Center. “The Defeat Pediatric Brain Tumor Research Collaborative brings these groups together in a unique way, thereby strengthening existing, and fostering novel, scientific and clinical synergies for the benefit of our patients and families.”

Given insufficient funding from industry and government sources, the investment of private funding through “Project Impact” is critical to accelerating the discovery of treatments for pediatric brain tumors. For more information about “Project Impact,” please visit 

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