Researchers have identified the hereditary gene mutations behind a deadly form of childhood cancer, opening the way to genetic tests in high-risk families, according to study released Sunday.
The same wayward gene has been previously linked to lymphoma and lung cancer in adults, so afflicted children could benefit from experimental drugs designed to suppress its activity, the study says.
"This very important discovery not only helps us understand the genetic roots of this terrible disease, but also has led to dramatically new ideas for curative therapy," said lead researcher John Maris, head of the Center for Childhood Cancer Research at The Children's Hospital of Philadelphia.
Neuroblastoma attacks the nervous system. While fairly rare, it accounts for seven percent of all childhood cancers, and 15 percent of non-adult cancer deaths.
The disease has long puzzled scientists because of its highly variable outcomes: some forms strike infants but then recede without treatment, while other variants, especially in older children, can be relentlessly aggressive.
"This discovery enables us to offer the first genetic tests to families affected by the inherited form of this disease," said Yael Mosse, lead author and a pediatric oncologist at Children's Hospital.
"Because there are already drugs in development that target the same gene in adult cancers, we can soon begin testing those drugs in children with neuroblastoma," she said.
An international team led by Maris scanned genomes -- the DNA library unique to every individual -- within 10 families beset by the disease.