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U.S. National Institutes of Health
Cancer Diagnosis Program Cancer Imaging Program Cancer Therapy Evaluation Program Developmental Therapeutics Program Radiation Research Program Translational Research Program Biometric Research Branch Office of Cancer Complementary and Alternative Medicine
Last Updated: 04/25/2012


Improving Outcome for Children with High-Risk Neuroblastoma

Neuroblastoma is the most common cancer diagnosis in the first year of life and is the most common solid tumor of childhood. Children with biologically aggressive neuroblastoma that has disseminated throughout the body have poor outcome, with long-term survival rates below 50 percent.

To improve outcome for children with high-risk neuroblastoma, NCI supported the Children’s Oncology Group (COG) to conduct the ANBL0032 phase 3 clinical trial. The trial’s main objective was to determine the role in the treatment of high-risk neuroblastoma of the ch14.18 monoclonal antibody. The antibody stimulates an immune response to the tumor by binding to a glycolipid (a fat molecule attached to a carbohydrate) known as GD2 that sits on the surface of neuroblastoma cells.

All patients on the ANBL0032 clinical trial had responded to standard therapy, which includes intensive chemotherapy, surgery, and a stem cell transplant, followed by radiation therapy. Half were then randomized to receive standard treatment with 13-cis-retinoic acid (RA) alone, while the others received RA plus the experimental treatment, consisting of the ch14.18 monoclonal antibody plus an alternating regimen of two immune-stimulating cytokines, GM-CSF and IL-2. Progression-free survival at 2 years was 66 percent in the ch14.18 plus cytokines arm and 46 percent in the standard treatment arm, and overall survival at 2 years was 86 percent and 75 percent, respectively. These outstanding results define ch14.18 plus cytokines as a new component of standard of therapy for children with high-risk neuroblastoma.

Yu AL, Gilman AL, Ozkaynak MF, et al. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for Neuroblastoma. N Engl J Med 2010:363;1324-34.

CTEP continues to make ch14.18 available to children with neuroblastoma who have completed their stem cell transplant through either the ANBL0032 ( study or through the ANBL0931 ( study. The latter study is collecting detailed toxicity data to support licensing of ch14.18 by FDA for the treatment of neuroblastoma. The Biopharmaceutical Development Program (BDP) at NCI-Frederick manufactures ch14.18 for CTEP-sponsored clinical trials. CTEP has identified a pharmaceutical collaborator (United Therapeutics Corporation) that will assume responsibility for manufacturing ch14.18 and that will be responsible for licensing ch14.18 for the treatment of neuroblastoma.