Neuroblastoma, a cancer of the peripheral nervous system, accounts for 7 percent of all childhood cancers, but because it frequently occurs in an aggressive form, it causes 15 percent of all childhood cancer deaths. While low-risk forms of neuroblastoma may spontaneously disappear, in high-risk forms, the cancer tends to return after initial treatment, usually with lethal results. A group of pediatric oncologists, including neuroblastoma expert John Maris, MD, used immunotherapy — biologic agents that stimulate the body's immune system — to achieve the first substantial increase in cure rates for neuroblastoma in more than a decade.
The team assigned 226 high-risk patients to receive either the standard therapy or standard therapy plus immunotherapy. Within two years of follow-up, approximately 54 percent of patients receiving standard treatment suffered a disease relapse, which is almost uniformly fatal. In contrast, 34 percent of patients receiving the experimental immunotherapy regimen had their disease return, a much higher cure rate. Patients receiving immunotherapy experienced a higher rate of pain and other toxic side effects, yet there was evidence of clear benefits from immunotherapy.
The Cancer Center at Children's Hospital has been using this immunotherapy regimen to treat children with high-risk neuroblastoma from around the world following preliminary trial results reported in June 2009. The findings of the current study, published in the New England Journal of Medicine, are expected to globally change clinical practice and set a new gold standard of treatment for this often-deadly disease.
Dr. Maris, director of the Hospital's Center for Childhood Cancer Research and chair of the neuroblastoma committee of the Children's Oncology Group (COG), co-authored a second study in the same issue of the journal, reporting on a separate trial that found a substantially reduced dose and duration of chemotherapy achieves a survival rates of 98 percent among children with intermediate-risk neuroblastoma. "Together, these studies report important advances in care for children with this challenging cancer," says Dr. Maris. "We will continue to investigate treatments to further refine the standard of care."