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Doubling the effect

Dana-Farber's Lisa Diller, MD, checks up on neuroblastoma patient Kane Goodman with the support of his mother, Tracy.

Dana-Farber's Lisa Diller, MD, checks up on neuroblastoma patient Kane Goodman with the support of his mother, Tracy.

As in about half of neuroblastoma patients, Brent McCreesh's cancer caused no diagnosable symptoms until it had spread to several organs and his bone marrow. The doctors at Yale-New Haven moved quickly. They implanted in his chest a flexible rubber catheter, which for the next year would serve as a port for medications, fluids, and blood transfusions, as well as easy access for blood samples.

The opening salvo of treatment was six rounds of combination chemotherapy to shrink the tumors before they were removed during three long operations by Robert Shamberger, MD, at Children's Hospital Boston. Back at Yale, Brent underwent 14 days of radiation therapy. Next, the Children's team prepared the coup de grace. Brent received a blast of high-dose chemotherapy designed to mop up any surviving cancer cells, and then had an infusion of his own blood stem cells (previously removed and stored) to rebuild his bone marrow. A few weeks later, the chemotherapy and stem cell transplant treatment was repeated, this time accompanied by total-body radiation.

At many U.S. cancer centers, Brent would have received a single stem cell transplant. But the Dana-Farber/Children's Hospital Cancer Care team has been pushing the treatment envelope for several years by adding a second transplant after an additional round of chemotherapy and radiation.

Overall, the "very, very aggressive" double transplant treatment has upped the long-term survival rate to about 50 percent, says Diller. One child died during therapy, highlighting the tightrope that doctors are walking with current treatments for high-risk neuroblastoma patients. "We are at our limit with conventional chemotherapy, surgery, and radiation," she says. "Fortunately, a lot of new agents are being developed for this disease."