Stewardship Report
The Kortney Rose Foundation
Stewardship Report Winter 2010
Pediatric NeuroOncology Program at The Children’s Hospital of Philadelphia
The Pediatric NeuroOncology Program at The Children's Hospital of Philadelphia is one of the largest brain tumor centers in the nation and a national leader in the development of new pediatric oncology therapies for childhood brain tumors. Our multidisciplinary service diagnoses and treats children and young adults with primary brain tumors, spinal cord tumors, and tumors related to neurofibromatosis. We see about 200 new patients a year.
To meet the special needs of patients with brain tumors, we closely coordinate care between physicians in neurooncology and those in neurosurgery, radiation therapy and neuroradiology.
Our program has an extensive laboratory and clinical research program designed to identify new therapies for medulloblastoma, gliomas and infant brain tumors.
As part of the Children's Oncology Group and the Pediatric Brain Tumor Consortium, we develop and conduct our own institutional clinical trials and participate in cooperative group clinical trials.
Our pediatric oncology diagnostic facilities provide magnetic resonance imaging, magnetic resonance spectroscopy and positron emission tomography. Additional diagnostic and support services include neuropsychology, physical therapy and rehabilitation medicine, social services and home health care.
Peter C. Phillips, M.D.: research update
Peter C. Phillips, M.D., is the director of the Pediatric NeuroOncology Program at CHOP.
At the midpoint of Fiscal Year 2009, Dr. Phillips helped shepherd a potential therapeutic for medulloblastoma into a critical stage as the clinical cochair of a multicenter phase I clinical trial of GDC0449. The drug has shown remarkable success in trials with adult basal cell carcinoma patients, and the compound on which it is based has demonstrated similar promising results with mice with medulloblastoma in previous work at The Children’s Hospital of Philadelphia with Tom Curran, Ph.D., deputy scientific director of The Children’s Hospital of Philadelphia Research Institute.
The trial is being run through the Pediatric Brain Tumor Consortium and treats children whose medulloblastoma was either unresponsive to other treatments or returned following previous treatment. Children in the trial take daily doses of GDC0449 as a pill; the study’s primary goals are to examine how children’s bodies process the drug; effects of the drug, both good and bad; what dose of the medication would be best for further studies; and fluctuations of levels of cells and proteins in children’s blood to see whether these are related to the effect of GDC0449 on the brain tumor.
In Phase I trial of GDC0449 for adult basal cell carcinoma patients, the drug prompted significant clinical improvements in eight of nine patients, including some with multiple, disseminated metastases. This success builds on encouraging results in Children’s Hospital studies of a closely related compound on which GDC0449 was based. The compound is a benzamidazole inhibitor known as HhAntag, shown in previous Children’s Hospital studies to eradicate even large tumors developed by mice with medulloblastoma. Hospital studies showed HhAntag is also disruptive to bone formation in young animals treated with the substance, however. It is unknown whether children would suffer the same side effect. Children’s Hospital researchers are exploring imaging approaches to develop biomarkers for tumor responses and developmental toxicities, since tracking both will be important given the potential side effect.
Children’s Brain Tumor Biorepository (Tissue Bank) at CHOP
Funding from the Kortney Rose Foundation has also supported the Children’s Brain Tumor Biorepository at CHOP over the past few years, laying the groundwork for the future of research in neurooncology.
The goal of the tissue bank is to collect biospecimens reflecting the molecular diversity of all pediatric brain tumors. Ultimately, we will need to characterize up to 2,000 specimens collected from several hospitals nationwide as part of a multiyear program. Samples will be used for genetic studies and to generate new models of brain tumors that reflect the properties of each molecular subset. In this way, the principles of personalized medicine will be applied to the understanding and treatment of pediatric brain tumors.
The ongoing, considerable support of the Kortney Rose Foundation has been vital to the continuing success of the pediatric neurooncology program at CHOP, especially in its research. With your support, Children’s Hospital physicianscientists like Dr. Phillips are bringing us ever closer to better treatments for pediatric brain tumors. Thank you for your dedication to this program and to the children it will one day help.
