Current treatments for high-risk neuroblastoma patients include chemotherapy, radiation therapy, surgery, stem cell transplant, and immunotherapy. However, less than half of the children survive because the standard therapy is intensive and toxic. Long-term survival after a relapse is less than 5%. For patients with relapsed neuroblastoma, there are few promising treatment options in clinical trials.

Cancer treatment vaccines differ from other vaccines in that they treat active cancers or help to prevent recurrence. In a case study reported in Pediatrics (2013; doi:10.1542/peds.2012-0376), complete remission was achieved in a 6-year-old boy with recurrent neuroblastoma. One year after his last treatment, the boy shows no evidence of disease.

Previous clinical trials in adult solid tumors have successfully used cancer-specific targets (NY-ESO-1, MAGE-A1, and MAGE-A3) to kill cancer cells. Now, scientists at the University of Louisville, led by Kenneth Lucas, MD, have used these same targets for neuroblastoma by creating a vaccine that causes the body’s own immune system to attack the tumor cells. The study includes children with sarcomas as well as neuroblastoma, and will be completed in 2013.


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Dendritic cells are immune cells collected from the patient and grown in cultures after they are exposed to specific antigens. The dendritic cells are returned to the patient’s body via a series of injections; the dendritic cells then teach the patient’s T-cells to seek out and kill the cancer cells.

“This research builds on 5 years of preclinical research, which identified three new immunological targets that are specific to this pediatric cancer,” said Scott Kennedy, the Executive Director of Solving Kids’ Cancer, which provided funding for the research. “The case study highlights the potential therapeutic progress that can be made against neuroblastoma, and brings hope to patients and their families in finding a lasting cure.”