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

ifying tumors that have the Hedgehog pathway activated.

"Medulloblastomas are the most common malignant brain tumors in children," said Amar Gajjar, M.D., co-chair of the St. Jude Department of Oncology and principal investigator of the PBTC trial. "The trend in treating children with these cancers is toward targeted therapies like this one, which block key signaling pathways and disable the cancer's ability to function or reproduce. We know that this Hedgehog pathway is important in the growth of these especially hard-to-treat tumors."

The PBTC has an ongoing Phase II trial in recurrent medulloblastomas in young adults (age 22 and older), with this same agent, which has recently been expanded to include more patients. A Phase II trial of GDC-0449's effectiveness against recurrent medulloblastomas in children (up to age 21) will start later this year based on the results of today's reported Phase I trial.

All of the PBTC trials are being sponsored by the Division of Cancer Treatment and Diagnosis, National Cancer Institute (NCI) under a Cooperative Research and Development Agreement Letter of Intent between NCI and Genentech, Inc.

St. Jude is home to the nation's largest research-based pediatric brain tumor program. St. Jude investigators have played a pivotal role in advancing understanding of the molecular missteps that give rise to medulloblastomas as well as seeking new, more targeted therapies to combat the tumors. St. Jude investigators have published evidence that targeting the Hedgehog pathway eradicated medulloblastomas in laboratory models. During fetal development the Hedgehog pathway plays a central role in normal growth of the cerebellum. The cerebellum is located at the base of the skull and is the structure that helps coordinate movement and plays a role in mastering balance and other motor skills. Medulloblastomas begin in the cerebellum, and uncontrolled activity along this pathway is linked to several cancers,
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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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