An experimental drug, available only through a clinical trial, helps prevent relapses in children with a rare cancer called neuroblastoma.
Children with high-risk neuroblastoma, which grows in nerve cells in the neck, chest and abdomen, desperately need better treatments, experts say.
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A new, man-made antibody may save some of them, says Alice Yu, a professor at the Moores University of California-San Diego Cancer Center.
In a study of 226 children, Yu found that combining the antibody with other immune therapies cuts the risk of relapse by 20%.
Two years after getting the new therapy, 66% of kids were relapse-free, compared to 46% of kids randomly assigned to receive a standard therapy called retinoic acid, according to a study released Thursday, in advance of the annual meeting of the American Society of Clinical Oncology, which begins in two weeks in Orlando.
Because most relapses occur in the first two years after a bone marrow transplant, Yu says these kids are likely to have been cured. About 86% of those who got the antibody were alive after two years, compared to 75% who got standard therapy.
That's a big improvement for such a stubborn disease, says Nai-Kong Cheung, head of the neuroblastoma program at New York's Memorial Sloan-Kettering Cancer Center, who was not involved in the new study. Neuroblastoma causes 15% of all deaths from pediatric cancer.
Cheung says the study is a "landmark," not only because the drug seems so helpful, but because of the difficulty of conducting a definitive trial in a disease with so few patients.
"This is a major, major event," says Cheung, who says this is the first new therapy for high-risk neuroblastoma in about 10 years.
Most children with the disease are toddlers under age 5. About 40% of the 650 kids diagnosed each year have aggressive tumors. Of those, only about 30% survive, in spite of intense and painful therapies, that include surgery, heavy chemotherapy, radiation and bone marrow transplants.
"We basically throw the book at them," Yu says.
Now, antibody therapy will become the new standard of care, she says.
Yet Cheung notes that doctors will have to wait many years to know if children were truly cured. And he notes that the antibody therapy is not easy to take.
More than 20% of children treated with the antibody suffered significant pain during the five to 10 hours that it takes to receive the intravenous drug, Yu says. Children receive the drug four days a month for five months.
About 7% of children also developed leaky blood vessels and another 7% developed allergic reactions, the study shows.
"It's a very tough treatment," Yu says. "But if we can achieve a cure for another 20% of kids, then it's worth it."
Older therapies pose their own risks. The heavy chemo that children receive early on their therapy can damage the heart and kidneys and even cause sterility or new cancers, Cheung says.
Cheung notes that doctors have been trying to spare as many children as possible from these toxic side effects, even as they struggle to save them from an aggressive disease. Doctors now try to reserve the harshest therapies for children with genetic markers indicating their tumors put them at high risk, he says. Children with low-risk neuroblastoma get a lighter type of chemotherapy, which causes fewer long-term side effects, Cheung says.
Families who are interested in the antibody for their children can still join the study, Yu says. Researchers will continue to monitor the drug's safety in order to apply for approval with the Food and Drug Administration, Yu says.
Yet the drug's future remains uncertain.
Cancers such as neuroblastoma are considered "orphan" diseases, because drugmakers are reluctant to invest heavily in such a small market, Cheung says. The National Cancer Institute provided the antibodies used in the trial because there were no drug companies willing to manufacture it. Although several companies have shown interest in the drug, none have yet committed to making it, Yu says."Now that we've discovered that this drug is useful, it's cruel not to be able to give it to someone," Cheung says. "Someone will have to maintain the supply."