Experimental Immunotherapy Markedly Improves Cure Rate for Children With Neuroblastoma

Researchers from the Children’s Oncology Group (“COG”) have demonstrated that a new experimental immunotherapy treatment using the chimeric monoclonal antibody ch14.18, that targets a specific carbohydrate/lipid molecule ganglioside, GD2 which is present on the surface of neuroblastoma tumor cells and cytokines IL-2 and GM-CSF resulted in a 20% improvement in cure rates compared to the standard treatment used in the past.

The findings of the COG Phase III study were published in May 2009 online on the American Society of Clinical Oncology ASCO website http://www.abstract.asco.org.

Neuroblastoma is a cancer of early childhood in which the cancer cells arise from cells of the sympathetic nervous system in the neck, chest, or abdomen. Annually, there are approximately 650 new cases of neuroblastoma diagnosed in the U.S. Neuroblastoma is the most common cancer diagnosed in the first year of life and is responsible for 15% of cancer-related deaths in children. Most patients are diagnosed as toddlers, but neuroblastoma can present in infants and older teenagers as well.

“This is the first clinical trial to document that [the] combination of anti-cancer monoclonal antibodies mAbs with cytokines natural hormones that help the immune system is an effective anti-cancer therapy,” said COG lead study investigator, Alice Yu M.D., Ph.D., Professor of Pediatrics at UCSD. She goes on further to say that: “So far, all FDA approved therapeutic anti-cancer mAbs or vaccines are directed against protein or glycoprotein antigens. More importantly, this is the first study that proves that immunotherapy is effective in improving cure rates for this childhood cancer, and we are now focusing on making sure immunotherapy can be available to all children with this disease, and that we can improve on these results in the future.”

The researchers analyzed data from 226 eligible patients with high-risk neuroblastoma with a high likelihood of treatment failure and relapse. The patients were randomized to standard therapy, which consist of intensive chemotherapy followed by autologous hematipoietic stem cell transplantation and post-transplant isoretinoin, or the experimental immunotherapy moAb ch14.18 plus cytokines IL-2 plus GM-CSF and isoretinoin post transplant. Standard therapy was well tolerated, and as anticipated, the aggressive immunotherapy had significant side effects but these were manageable. The study showed that children receiving the experimental immunotherapy had a 20% better chance of remaining disease-free, and this resulted in a significantly improved cure rates. The study was stopped early due to the benefit seen in early analyses of the trial results.

John Maris, M.D., COG Neuroblastoma Disease Committee Chair, responds, “This is the first clinical trial to show a substantive increase in cure rate in well over a decade for this highly malignant childhood cancer.” Moreover, he states, “The 20% improvement in prevention of relapse for children with neuroblastoma receiving the experimental immunotherapy makes this therapy the new standard of care.”

According to COG Group Chair, Gregory Reaman, M.D., “The clinical benefit of this approach in a notoriously recalcitrant cancer of childhood is extraordinary and COG is anxious to continue its investigation of this approach to better define the toxicities and confirm its satisfactory risk: benefit ratio.” Dr. Reaman continues, “In addition, we are hopeful that this further study will result in commercialization and FDA approval and licensing of this experimental immunotherapeutic agent so that it can be made readily available as standard therapy to all children with this dreaded disease”.

For more information about new research, visit us at www.curechildhoodcancer.org

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