Basic Research Data Pivotal for First FDA Approval of Immunotherapy for Pediatric Cancer

The equivalency of an immunotherapy that until now had only been available to patients enrolled in research studies and a product that has been manufactured for commercial use was recently published in Cancer Chemotherapy and Pharmacology.1 This data was pivotal to the U.S. Food and Drug Administration (FDA) approval of the first immunotherapy for pediatric patients and is now available to all patients.

The immunotherapy dinutuximab is a monoclonal antibody against disialoganglioside, and it was developed to harness a patient's immune system to battle neuroblastoma. Neuroblastoma is a nervous system cancer that typically occurs in children age 5 years or younger. It results in death within 5 years of diagnosis for about half of the children with this disease.

Dinutuximab improved event-free survival and overall survival in a randomized, phase 3 trial that compared results with dinutuximab treatment to those of patients who did not receive the immunotherapy.

The dinutuximab used in the clinical trials was manufactured by the National Cancer Institute (NCI) in relatively small quantities, so the production of dinutuximab was assumed by United Therapeutics Corporation so larger quantities could be made. The commercially produced version had to be tested in patients and shown to be equivalent to the NCI-produced version.

"After a 2 year study that compared commercially manufactured dinutuximab to the material produced by NCI, we see no difference in how patients metabolize or respond to the treatment," said Araz Marachelian, MD, principal investigator at Children's Hospital of Los Angeles in California. The study found no notable safety or tolerability differences.

"I, along with my colleagues across the country, are proud of the work that has been done to make additional therapies available for children with this devastating disease."

Dr Marachelian is also an assistant professor of Clinical Pediatrics at the Keck School of Medicine of USC and director of the New Approaches to Neuroblastoma Therapy (NANT) consortium. Through NANT, she is currently leading 2 active trials investigating the use of dinutuximab in combination with other agents.


1.    1. Marachelian A1, Desai A2, Balis F, et al. Comparative pharmacokinetics, safety, and tolerability of two sources of ch14.18 in pediatric patients with high-risk neuroblastoma following myeloablative therapy [published online ahead of print January 20, 2016]. Cancer Chemother Pharmacol. doi: 10.1007/s00280-015-2955-9.

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