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Scientists have identified a gene that causes inherited forms of neuroblastoma, a fast-growing cancer that mostly affects infants and children. They are hopeful the findings will pave the way for new treatments.
Neuroblastoma, a cancer that attacks the nervous system, is the third most common childhood cancer. It affects nearly 1 in 80,000 children in the United States.
In a new study, researchers from Belgium, Italy and the United States strongly linked neuroblastoma to mutations in an ALK gene (anaplastic lymphoma kinase).
Several pharmaceutical companies are already working on medications that target this gene, which is also a variation in some adult cases of lung cancer and lymphoma. These same drugs can soon begin testing in children with neuroblastoma.
"This discovery will enable us to extend, for the first time, genetic tests to those families that are directly affected by the inherited form of this terrible disease," said Dr. Yael Mosse, a pediatric oncologist at Children's Hospital, in Philadelphia.
After researchers determined the source of familial neruoblastoma, they examined whether ALK mutations also played a role in random, non-inherited cases – those that do not occur in families.
They found ALK mutations in 12 percent of 194 tumor samples that were taken from children with an aggressive form of the neruoblastoma.
“These study findings are very important, as they will help us to further understand the genetic makeup of this disease, while also leading us to new curative therapies,” said Dr. John Maris of Children’s Hospital and director of the study.
The study is published in the British journal Nature. #