Immunotherapy Improves Outcomes for Patients with High-risk Neuroblastoma

According to the results of a Phase III clinical trial that will be presented at the 2009 annual meeting of the American Society of Clinical Oncology (ASCO), use of an investigational immunotherapy treatment reduced the risk of relapse and improved survival among patients with high-risk neuroblastoma.

Neuroblastoma is a disease in which cancerous cells form in the nerve tissues of the adrenal gland, neck, chest, or spinal cord. Although neuroblastoma is rare (it affects roughly 650 children and adolescents in the United States each year), it is the most common malignancy diagnosed in infants.

Based on the age of the patient and the specific characteristics of the cancer, neuroblastoma is classified as low-, intermediate-, or high-risk. Patients with high-risk neuroblastoma tend to have lower survival rates than other patients with neuroblastoma. 

Conventional treatment of high-risk neuroblastoma often includes surgery, intensive chemotherapy, stem-cell transplant, and radiation therapy. Even with aggressive treatment, however, only 30 percent of patients survive; this highlights the importance of finding new and more effective approaches to treatment.

The current Phase III clinical trial evaluated an investigational immunotherapy treatment. The treatment consists of an antibody targeted against a specific molecule (GD2) on neuroblastoma cells, given in combination with cytokine therapy (GM-CSF and IL2). The goal of therapy is to prompt the body’s immune system to attack the cancer cells.  

The study involved 226 patients with newly diagnosed, high-risk neuroblastoma that responded to chemotherapy and stem-cell transplant. Patients were then assigned to receive standard treatment (retinoic acid) plus immunotherapy or standard treatment alone.

  • After two years, 66% of patients in the immunotherapy group were alive and free of relapse compared with 46% of patients in the standard treatment group.
  • Overall survival at two years was 86% in the immunotherapy group compared with 75% in the standard treatment group.
  • The most common side effects in the immunotherapy group were pain (21%), vascular leak syndrome (7.3%), and allergic reactions (7.2%).

Random assignment to treatment group was halted when the benefit of the immunotherapy treatment became apparent. The study will continue to enroll patients into the immunotherapy group, however, in order to collect additional safety information. This therapy has not yet been approved by the U.S. Food and Drug Administration (FDA), and is currently available only to participants in this study.

Reference: 2009 ASCO Annual Meeting May 14 Presscast. Yu AL et al. A phase III randomized trial of the chimeric anti-GD2 antibody ch14.18 with GM-CSF and IL2 as immunotherapy following dose intensive chemotherapy for high-risk neuroblastoma: Children’s Oncology Group (COG) study ANBL0032. Abstract #10067z.

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