Neuroblastoma, a disease of immature nerve cells, is the most common solid tumor cancer occurring in children. About 700 new cases are diagnosed in the U.S. each year with approximately half of these children developing high-risk disease, which has a 40 to 50 percent chance of survival.
In 2015, the Food and Drug Administration approved a monoclonal antibody called dinutuximab for treatment of high-risk neuroblastoma. The approval followed a ground-breaking study, led by the Children’s Oncology Group and published in the New England Journal of Medicine. This pivotal trial halted enrollment earlier than planned because the therapy showed significant improvement in survival over the standard treatment.
“Although dinutuximab is a game-changing treatment in the battle against neuroblastoma, the fact remains – children are still dying from this disease,” said Eugene S. Kim, MD, surgical oncologist at Children’s Hospital Los Angeles.
A team, led by Kim, designed basic research studies that would go beyond using tumor-shrinkage as a marker of efficacy and instead focus on recurrent disease and overall survival. The team’s approach was to create a model system that more faithfully replicates the patient experience.
Patients with neuroblastoma undergo surgery to remove their tumor and then have subsequent therapy.
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