Experimental drug helps kids with neuroblastoma

Two new studies offer hope for babies and toddlers suffering from neuroblastoma, a rare but often deadly cancer of the nervous system that strikes about 750 children a year.

Both therapies offer better, safer treatments for the disease, and belong to the emerging field of "personalized" cancer therapy, with medications tailored to the specific genetic profiles of a patient's tumor, says co-author John Maris of the Children's Hospital of Philadelphia.

Children diagnosed with neuroblastoma, which grows in nerve cells in the neck, chest and abdomen, are typically only about 17 months old, Maris says. About have an aggressive form of disease that relapses despite the best therapy.

A study of 226 children focused on an experimental, man-made antibody, called ch14.18, given as a cocktail with other immune stimulants. Doctors randomly assigned half of children to get standard care and half to receive the new antibody, according to the study in today's New England Journal of Medicine.

This type of antibody is the "holy grail" of cancer therapy, Maris says, because it targets a protein found only on cancer cells, but rarely on healthy ones. Scientists first identified this protein in the 1980s, he says.

The new therapy cut the risk of relapse from 66% to 46% after two years Because most relapses occur in the first two to three years, these children have likely been cured, Maris says. About 86% of those given the new therapy were alive after two years, compared to 75% of those given standard care.

"This is the biggest improvement we've ever seen in neuroblastoma," Maris says. "It's not a magic bullet, but it's the biggest result we've seen in a long time.. .. This is the culmination of 20 years of work."