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Experimental targeted therapy shows early promise against medulloblastomas
Date:6/7/2010

(CHICAGO June 5, 2010) Researchers from St. Jude Children's Research Hospital and the Pediatric Brain Tumor Consortium (PBTC) presented at the American Society of Clinical Oncology the findings of a pediatric brain tumor study using an experimental drug that targets the underlying genetic makeup of the tumor. The research focused on a new way to attack the tumors by blocking the Hedgehog pathway that is linked to approximately 20 percent of medulloblastomas.

The study is the first to report that the drug can be safely administered to children. The study also suggested that the drug is showing early signs of efficacy in this patient population, with some children still on treatment almost a year with no progression of disease. All the children on this study had medulloblastomas that persisted or returned despite standard treatment with radiation and chemotherapy. Recurrent medulloblastoma currently has a cure rate of less than 5 percent.

The researchers found that patients whose tumors had the Hedgehog molecular pathway activated appear to be some of the same patients who have responded to treatment in this trial, based on length of time on study. Investigators have observed tumor responses in similar young adult patients whose tumors had the Hedgehog molecular pathway activated. These early findings have given the green light for pediatric research to advance to a larger Phase II study scheduled to open later this year, and to increase the number of patients on the young adult study.

The Phase I study (PBTC -025) included 13 patients, 12 of them evaluable, ranging in age from 4 to 21 years. All received one of two different doses of GDC-0449 daily for a minimum of 28 days and continued on treatment for as long as their disease remained stable. In addition to determining the safety and dosing of this experimental drug for children, the trial also conducted extensive research into pathologic and genomic methods for better ident
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Contact: Carrie Strehlau
carrie.strehlau@stjude.org
901-297-9875
St. Jude Children's Research Hospital
Source:Eurekalert

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