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Brain Cancer Health Center

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Vastly Better Odds for Kids With Deadly Cancer

New Neuroblastoma Treatments Improve Survival Rate, Lessen Long-Term Harm
WebMD Health News
Reviewed by Laura J. Martin, MD

Sept. 29, 2010 -- An aggressive new treatment offers new hope to kids with high-risk neuroblastoma, while kids at somewhat lower risk benefit from less intense therapy.

The findings come from two breakthrough studies that already are changing treatment for children with this deadly cancer.

The first study shows that for kids with high-risk neuroblastoma, adding a new treatment developed at the National Cancer Institute to standard treatment improves cancer survival from 46% to 66%.

"To improve two-year survival by 20% is just amazing in cancer," study researcher Katherine Matthay, MD, tells WebMD. Matthay, a leading expert in childhood cancer, is chief of pediatric hematology and oncology at the University of California, San Francisco Medical Center.

The second study shows that for kids with intermediate-risk neuroblastoma -- and certain favorable tumor characteristics -- less intensive chemotherapy is just as effective as standard treatment with far fewer short- and long-term side effects.

"There are some tumors where it does not pay to be aggressive, where we need to back off and think of the lifetime to come," Matthay says.

Neuroblastoma Prognosis

Neuroblastoma is a cancer of the nerves outside the brain. It usually starts in the adrenal glands, located just above each kidney, although the cancer often begins in the nerve centers near the spinal cord.

It's a deadly cancer. Although there are only about 650 to 700 cases a year in the U.S., neuroblastoma accounts for 12% of childhood cancer deaths. About 90% of cases occur in children under age 10; some 60% occur before age 2. It's the most common cancer of newborns.

About half of kids with neuroblastoma have tumors with what doctors call an "unfavorable biology." This means the cancer is very aggressive, carries extra copies of the MYCN cancer gene, and spreads rapidly. Only about 30% to 40% of these children survive.

"The other group of patients have more favorable neuroblastoma, and their tumors are either localized and can be taken out without chemo, or cover a larger area and are invasive but have a favorable biology," Matthay says. "Even with metastases, if it happens before age 1 year, some of these tumors are more favorable and can be cured with chemo and without bone-marrow transplant."

All patients with neuroblastoma usually undergo surgery to take a biopsy of the tumor and to remove as much of the cancer as possible.

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