University of California San Francisco
Helen Diller Family Comprehensive Cancer Center

Less chemotherapy works well for some childhood cancer

By Kate Vidinsky | | September 29, 2010

Less chemotherapy works well for some childhood cancer

Katherine Matthay, MD

A particularly aggressive childhood cancer can be fought successfully with far less chemotherapy than previously believed, avoiding harmful side effects caused by cancer drugs.

The 96 percent survival rate found in an eight-year clinical trial at the University of California, San Francisco, stands to change the approach toward fighting intermediate-risk –  stage three and stage four – neuroblastoma. Neuroblastoma attacks the network of cells that control the body’s response to stress, known as the sympathetic nervous system, and affects 650 children in the United States each year.

Findings from the phase III trial conducted by the national research collaborative Children’s Oncology Group (COG) are being reported today in the September 30, 2010, issue of the New England Journal of Medicine, and are available online at

“Our goal was to reduce the level of chemotherapy needed to effectively treat intermediate-risk neuroblastoma patients while maintaining an excellent rate of survival, and that is exactly what we did,” said senior author Katherine Matthay, MD, chief of pediatric oncology at UCSF Benioff Children’s Hospital. “This trial will lead to permanent treatment reductions in our protocol for treating this disease and will have a significant impact on the hundreds of children who are diagnosed with neuroblastoma each year.”

The study involved 479 patients, the largest trial focused on reducing the amount of chemotherapy needed to treat intermediate-risk neuroblastoma. The researchers aimed to reduce exposure to chemotherapy by at least 40 percent while maintaining the same 90 percent survival rate achieved in earlier trials using higher doses. Reducing the amount of chemotherapy is particularly important for young children whose developing bodies are extremely sensitive to the toxic cancer drugs, known to cause a host of side effects and increase the risk for later diseases unrelated to the initial cancer.

“It is my hope that we will be able to continue reducing the amount of chemotherapy we give to certain groups of neuroblastoma patients, so we can improve the long-term quality of life for these children and not have to worry about them having any secondary cancers,” Matthay said. “It is gratifying to see our patients many years after they have undergone treatment, when they are on their way to have kids of their own.”