Basic Research Led to First FDA-Approved Immunotherapy for Pediatric Cancer

(EurekAlert!) – Building upon more than two decades of basic research conducted at Children’s Hospital Los Angeles, Araz Marachelian, MD, of CHLA, and her colleagues at pediatric academic centers across the U. S., have shown that an immunotherapy that until now has only been available to patients enrolled in research studies, is equivalent to the product that has been manufactured for commercial use and can be made available to all patients. The data, published in the journal Cancer Chemotherapy and Pharmacology on January 20, was pivotal to the FDA approval of the first immunotherapy for the treatment of a pediatric cancer. (article: http://www.ncbi.nlm.nih.gov/pubmed/26791869)

Neuroblastoma is a cancer of the nervous system that exists outside the brain and typically occurs in children 5 years of age or younger. Despite very aggressive treatment, many patients fail to respond to therapy or experience recurrence of their disease. Approximately half of all children with this disease die within 5 years of diagnosis.

A treatment, called dinutuximab, was developed to harness a patient’s own immune system to battle neuroblastoma. The Children’s Oncology Group conducted a randomized, phase 3 trial of dinutuximab and found that children treated with the monoclonal antibody had improved event-free survival and improved overall survival compared to patients who did not receive the immunotherapy.

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