WASHINGTON (Reuters) -
Researchers have found a gene that
causes most inherited forms of neuroblastoma, a rare and deadly
form of childhood cancer, and say the discovery points to new
treatments.

Mutations in a gene called ALK were strongly linked to
neuroblastoma, the researchers from the United States, Italy
and Belgium reported on Sunday. They said several companies
already are working on drugs that target this gene, which is
also mutated in some cases of lung cancer and lymphoma.

“This discovery enables us to offer the first genetic tests
to families affected by the inherited form of this disease,”
said Dr. Yael Mosse of The Children's Hospital of Philadelphia,
who worked on the study.

“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 accounts for 15 percent of childhood cancer
deaths, with just a 40 percent survival rate, even though it
only causes about 7 percent of all pediatric cancers.

Writing in the journal Nature, the team at Children's
Hospital, the Scripps Research Institute in La Jolla,
California, and elsewhere said they studied 20 families with
one or more children with neuroblastoma.

They found mutations in the ALK gene that cause it to be
constantly active in helping cells proliferate. Out-of-control
proliferation is the hallmark of cancer.

“This is a very important discovery, as it not only helps
us understand the genetic roots of this terrible disease, but
also has led to dramatically new ideas for curative therapy,”
said Dr. John Maris of Children's Hospital, who directed the
study.

After finding the ALK mutations in familial neuroblastoma,
the researchers looked at sporadic cases — those not occurring
in families with a history of the disease. They found ALK
mutations in 12 percent of 194 tumor samples from children with
the aggressive, high-risk form of the disease.

(Reporting by Maggie Fox, editing by Vicki Allen)