St. Baldrick’s Research Outcome in Neuroblastoma

by St. Baldrick's Foundation
March 27, 2012

St. Baldrick’s researcher Dr. Alice Yu is developing a new immunotherapy treatment for kids with neuroblastoma.

For many years, doctors had only three ways to treat most cancers: surgery, radiation, and chemotherapy. A newer weapon in the fight against cancer is immunotherapy, harnessing the power of the immune system to attack cancer cells.

St. Baldrick’s donations helped fund research that has resulted in the first successful immunotherapy treatment for a childhood cancer, high-risk neuroblastoma.

Neuroblastoma is a cancer of the sympathetic nervous system. The average age of diagnosis is 2, and it’s rare in children over 10 years old.

Most patients have the high-risk form of the disease, and for years, only 1 in 3 of these children survived. With this new treatment, almost half may survive!

The treatment, developed by Dr. Alice Yu, a St. Baldrick’s researcher at the University of California San Diego, tested the effects of immunotherapy on the relapse rate of neuroblastoma patients.

Immunotherapy boosts the immune system to attack cancer cells by introducing man-made agents, like an antibody, to fight off the cancer. In the case of neuroblastoma, Dr. Yu and her team of researchers introduced an antibody called CH14.18, which targets a molecule on the surface of tumor cells called GD2.

Immunotherapy Patient Celebrates One Year of Remission
The study has now shown that 66 percent of the patients receiving the immunotherapy became cancer free, compared with 46 percent of those who did not receive the treatment. Because of the positive life-saving results, the study was stopped early and all high-risk neuroblastoma patients were given the option to switch to ch14.18 immunotherapy.

“This trial was an important milestone for a terrible disease,” Dr. Yu said. “It offers new hope to many patients and families.”

This treatment is currently a phase III clinical trial of the Children’s Oncology Group (COG). The St. Baldrick’s Foundation has given more than $33 million to the Children’s Oncology Group, as well as two grants directly to Dr. Yu for this work.

“Without St. Baldrick’s support, we wouldn’t be able to develop this treatment. It’s a lot of work that requires the laboratory staff to process the samples and to do the proper assays to provide answers,” Dr. Yu explained. “St. Baldrick’s is also helping us with the next steps of our project. To further improve the cure rate, we plan to take the antibody, ch14.18, to the front line of standard treatment and combine it with chemotherapy. Thanks to the donors and volunteers of the St. Baldrick’s Foundation, I am optimistic that we will realize this for the future of kids with cancer.”

UPDATE: On March 10, 2015, the FDA approved this treatment for kids with neuroblastoma. Learn more about this exciting news and how YOU helped make it possible.

Help make new treatments like this available to all kids with cancer. Get involved today.

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