Neuroblastoma Immunotherapy Improves Overall Survival

Dinutuximab is the first immunotherapy approved for the treatment of a pediatric cancer.

An immunotherapy for pediatric cancer that was only available in research trials was found to be equivalent to the product manufactured for commercial use, which led to an FDA approval.

The National Cancer Institute (NCI) developed the drug dinutuximab to treat neuroblastoma.

Neuroblastoma is a cancer that resides outside the brain and attacks the nervous system. It typically affects children 5 years of age and younger, with about half of those who develop the disease dying within 5 years of diagnosis. This is because patients frequently do not respond to aggressive therapy or suffer disease recurrence.

Dinutuximab is a monoclonal antibody that utilizes the power of the immune system to fight the disease.

A randomized phase 3 trial published in the journal Cancer Chemotherapy and Pharmacology found that children treated with dinutuximab showed improved event-free survival and overall survival rate compared with patients who did not receive the immunotherapy.

During the trial, dinutuximab was only manufactured in small quantities, therefore it needed to be produced in larger quantities in order to become commercially available. Furthermore, the commercially-produced dinutuximab needed to be tested on patients in order to show that it was equivalent to the version used in the clinical trials.

"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 principal investigator Araz Marachelian. "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."

Marachelian is currently in the process of leading 2 investigative trials using dinutuximab in combination with other agents.