Four Organizations Fund International Research Consortium to Improve Lives of Children Suffering From Diffuse Intrinsic Pontine Glioma (DIPG)
January 23, 2012 (Washington, DC, and Bethesda, MD) – Accelerate Brain Cancer Cure (ABC2), CureSearch for Children’s Cancer, The Cure Starts Now Foundation and The Lyla Nsouli Foundation for Children’s Brain Cancer Research today announced their collaborative funding to support groundbreaking research aimed at dramatically improving the lives of children suffering from Diffuse Intrinsic Pontine Glioma (DIPG) – one of the most devastating pediatric cancers. Together, the four organizations have committed $229,000 to support the work of the DIPG Preclinical Consortium, the only international scientific group focused on preclinical development of targeted therapy combinations for DIPG. The goal of the research is to test and then move the most effective therapy forward to early phase clinical trials in the next 18 – 24 months.
Children with DIPG have a uniformly dismal prognosis with a median survival of 9 months. A DIPG tumor grows amidst the nerves in the pons (middle) of the brain stem, and therefore cannot be surgically removed. Radiotherapy provides only temporary improvement of symptoms. No chemotherapy has ever proven effective. Novel therapies are desperately needed. “The scientific community has truly rallied around this cause. The mandate for a novel therapeutic approach was born in the Children’s Oncology Group brain tumor committee under the bold leadership of Dr. Amar Gajjar. With the consortium co-leadership of clinical trialist Maryam Fouladi and the accountability to DIPG patients and their family, this program is moving unexpectedly quickly towards its goal,” says Charles Keller, MD, Associate Professor and leader of the Pediatric Cancer Biology Program, Pape' Family Pediatric Research Institute in the Department of Pediatrics at Oregon Health & Science University.
“If we succeed, it will be because families that have donated their children’s tumor gave us this opportunity. We are reminded every day that the cultures we study are parent-directed Legacy Gifts of the most selfless kind from children who current therapy could not save (the brain stem being vital to life; therefore, tumor donation can only occur at autopsy). What ABC2 , CureSearch for Children’s Cancer, the Lyla Nsouli Foundation, and The Cure Starts Now have done to make our consortium possible, and so quickly, is unprecedented – and greatly appreciated,” adds Dr. Keller.
The research project entitled, "Rapid Preclinical Development of a Targeted Therapy Combination for DIPG" was launched with initial support from The Cure Starts Now Foundation. Two additional European labs were added to the project with funding from The Lyla Nsouli Foundation for Children’s Brain Cancer Research (based in London, UK).
The funding from ABC2 and CureSearch for Children’s Cancer added a cutting-edge functional genomics component that will prioritize potential new drug targets. “We are proud to support this multi-national team of researchers in their efforts to rapidly develop effective drugs to treat children suffering from DIPG,” said Max Wallace, CEO of ABC2. “By combining forces with our non-profit partners, ABC2 looks forward to leveraging the resources and expertise of all the organizations to improve the lives of children with cancer.” John Lehr, president and CEO of CureSearch for Children’s Cancer echoed Wallace’s comments saying that “developing new drug targets is an integral step to providing children with DIPG a better prognosis. CureSearch is committed to funding research in rare cancer types so that one day, all children will be guaranteed a cure.”
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About the DIPG Preclinical Consortium
The multi-national consortium is identifying potentially important biological pathways in DIPGs that are readily targetable with currently available molecularly-targeted agents. In addition, the consortium has successfully grown human DIPG tumors from autopsy materials in the petri dish and has developed mouse models of DIPG – a key resource to functionally testing potential therapies.
Since the number of children with this unfortunate disease is limited, and the number of available targeted agents is quite large, the consortium hypothesizes that it can identify a promising combination of molecularly-targeted agents using functional genomics to prioritize targets. The ultimate goal is to move the most effective single agent or combination therapy forward to early phase clinical trials in the next 18-24 months.
The DIPG Preclinical Consortium team includes:
Charles Keller MD, Kellie Nazemi MD and Nate Selden MD, PhD at Oregon Health & Science University
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
For More Information about the Research Funding Partners:
Accelerate Brain Cancer Cure: www.abc2.org
CureSearch for Children’s Cancer: www.curesearch.org
The Cure Starts Now Foundation: www.curestartsnow.org
The Lyla Nsouli Foundation for Children’s Brain Cancer Research: www.lylansoulifoundation.org
The Lyla
Nsouli Foundation is proud to announce the award of a 67,000 USD pilot grant to
Dr. Sakir ‘Hume’ Gultekin, Neuropathologist and Associate Professor of
Pathology, Oregon Health & Science University. In this pilot study, Dr. Gultekin will
examine a new set of cell surface receptors in diffuse intrinsic pontine glioma (DIPG) to determine whether their
co-expression matches a profile consistent with novel therapeutic targets.
While at the earliest stage of preclinical development, it is hoped that novel
targets and unique therapeutic approaches might lead to a clinical trial for
children with DIPG in the foreseeable future. 
