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Gene That Causes Childhood Cancer Neuroblastoma Is Found
Date:8/25/2008

--May Lead to New Prevention and Treatment Strategies--

PHILADELPHIA, Aug. 25 /PRNewswire-USNewswire/ -- Scientists have discovered gene mutations that are the main cause of the inherited version of the childhood cancer neuroblastoma. In addition, the researchers found that the same mutations play a significant role in high-risk forms of non-inherited neuroblastoma, the more common form of the disease.

"This discovery enables us to offer the first genetic tests to families affected by the inherited form of this disease," said pediatric oncologist Yael P. Mosse, M.D., of The Children's Hospital of Philadelphia, the first author of the study, published online Aug. 24 in the journal Nature. "Furthermore, because there already are drugs in development that target the same gene in adult cancers, we can soon begin testing those drugs in children with neuroblastoma."

Neuroblastoma is the most common solid cancer of early childhood. It accounts for 7 percent of all childhood cancers, but due to its often aggressive nature, causes 15 percent of all childhood cancer deaths. It arises in the developing nerves of a child, often appearing as a tumor in the chest or abdomen.

Because only about 600 new cases of all forms of neuroblastoma occur annually in the U.S., familial (inherited) neuroblastoma is a very rare subset of a relatively uncommon disease. Scientists at Children's Hospital have studied familial neuroblastoma for the past 15 years, and the current study drew on family data collected from throughout the world.

John M. Maris, M.D., senior author of the current study and director of the Center for Childhood Cancer Research at Children's Hospital, leads a laboratory with the world's largest collection of neuroblastoma tissue samples, gathered through the multicenter Children's Oncology Group in the U.S. and through multiple international collaborations. Maris said, "This is a very important discovery, as it not only helps us unde
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SOURCE The Children's Hospital of Philadelphia
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