Immunotherapy improves survival of high-risk children’s cancer

Neuroblastoma is a tricky cancer. After chemotherapy, surgery, more chemo, radiation therapy and stem cell transplants, more than half the kids with high-risk neuroblastoma have a recurrence of the cancer. And most of those who relapse do not survive.

A study reports that the addition of immunotherapy to the already intense treatment regimen boosts relapse-free survival by 43 percent, say researchers at Children's Hospital of Philadelphia and a consortium of the nation's top pediatric medical centers. The study appears in the New England Journal of Medicine Thursday and is now available online.

Neuroblastoma causes 15 percent of all pediatric cancer deaths. About 750 children a year are diagnosed with the disease — 300 to 400 with the high-risk form of the cancer.

The study followed 226 children with high-risk neuroblastoma who underwent immunotherapy or the standard treatment. After two years, 66 percent of the immunotherapy patients had survived without a recurrence of the cancer, compared to 46 percent of the patients who hadn't gotten immunotherapy. Researchers say it's the first substantial increase in survival in over a decade.

The immunotherapy uses monoclonal antibodies to target cancer cells. Chemicals are also infused to boost the child's immune system response to the tumor cells. Patients undergo five rounds of the painful treatment. The antibodies target a protein on neuroblastoma cells — as well as on nerve cells — so that the patient's own immune system knows to attack and kill the remaining cancer.

In the New England Journal article, the researchers wrote that 32 of the immunotherapy patients had a relapse of their cancer, and 18 of those patients subsequently died. Another patient in the immunotherapy group died of a medication overdose during treatment.

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