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

act, the current study reports the first example of a childhood cancer caused by mutations in an oncogene. Since the mutations discovered by Mosse trigger an "on" signal for neuroblastoma cells, the abnormality is an outstanding target for therapies that inhibit the ALK protein's activity.

Several pharmaceutical companies are currently developing ALK inhibitors in the laboratory, and one ALK inhibitor is already in early-phase adult clinical trials against lung cancer and lymphoma. Now Mosse and her colleagues at Children's Hospital are planning pediatric clinical trials of ALK inhibitors in children with high-risk neuroblastoma. "It's an advantage to be able to start with agents that have already been shown to be safe in adults," Mosse added.

Neuroblastoma has long been a puzzling disease, partly because of its broad range in outcomes. Some types of the disease strike infants but spontaneously disappear with minimal treatment, while other subtypes in older children may be relentlessly aggressive. Earlier this year, Maris's lab reported that common DNA variations in a region of chromosome 6 raise a child's risk of developing sporadic neuroblastoma. "Together with this current study, we are defining the genetic events that underlie this childhood cancer," said Maris. "Better understanding of these biological pathways will guide our efforts to develop more effective treatments."

The National Institutes of Health supported the study, along with grants from the Alex's Lemonade Stand Foundation, the American Society of Clinical Oncology, the Andrew's Army Foundation, the Italian Neuroblastoma Foundation, Scripps Genomic Medicine, the Scripps Dickinson Scholarship, the Giulio D'Angio Endowed Chair, the Foerderer-Murray Fund and the Carly Hillman Fund. The Center for Applied Genomics at The Children's Hospital of Philadelphia also provided support, as did the Abramson Family Cancer Research Institute of the University of Pennsylvania School of Medicine
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SOURCE The Children's Hospital of Philadelphia
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