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 for a childhood cancer – high-risk neuroblastoma.

Neuroblastoma is a cancer of the sympathetic nervous system. The average age of diagnosis is two, and it’s rare in children over 10 years old. Most patients have the high-risk form of the disease, and for years, only one in three of these children survived. With this new treatment, almost half may survive!

The treatment, developed by Dr. Alice Yu, a professor of pediatric hematology and oncology at the University Of California San Diego and St. Baldrick’s grant recipient, 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 a type of 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.

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,” said Yu. “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,” states Yu. “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.”