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Immunotherapy effective against neuroblastoma in children
May 15, 2009New therapy improves chances of living disease-free with difficult-to-treat childhood cancer
A phase III study has shown that adding an antibody-based therapy that harnesses the body's immune system resulted in a 20 percent increase in the number of children living disease-free for at least two years with neuroblastoma. Neuroblastoma, a hard-to-treat cancer arising from nervous system cells, is responsible for 15 percent of cancer-related deaths in children. The researchers reported their findings - the first to show that immunotherapy could be effective against childhood cancer - online May 14, 2009 on the American Society of Clinical Oncology website in advance of presentation June 2.
"This establishes a new standard of care for a traditionally very difficult cancer in children," said lead author Alice Yu, MD, PhD, professor of pediatric hematology/oncology at the University of California, San Diego School of Medicine and the Moores UCSD Cancer Center. "High-risk neuroblastoma has always been a frustrating cancer to treat because, despite aggressive therapy, it has a high relapse rate."
The therapy targets a specific glycan (a complex sugar chain found on the surface of cells) on neuroblastoma cells called GD2, which inhibit the immune system from killing cancer cells. The antibody - ch14.18 - binds to this glycan, enabling various types of immune cells to attack the cancer.
Neuroblastoma - in which the cancer cells arise from nerve cells in the neck, chest, or abdomen - is the most common cancer diagnosed in the first year of life. Approximately 650 new cases of neuroblastoma are diagnosed in this country every year, and about 40 percent of patients have high-risk neuroblastoma. These high-risk patients are usually treated with surgery, intensive chemotherapy with stem cell rescue (in which patients' adult stem cells, removed before treatment, are returned after chemotherapy to restore the blood and immune system), and radiation therapy. Still, only 30 percent of patients survive.
Yu and her colleagues compared both the percentage of patients who were still alive without experiencing a recurrence after two years as well as overall survival in two groups of 113 patients each. Patients began the trial when they were newly diagnosed with high-risk neuroblastoma. After conventional treatment with surgery, chemotherapy, stem cell rescue and radiotherapy, one group was given the standard treatment (retinoic acid) plus immunotherapy (the antibody plus immune-boosting substances), while 113 similar patients received the standard treatment alone.
After two years, 66 percent of individuals in the immunotherapy group were living free of cancer compared to 46 percent in the standard treatment group. Overall survival improved significantly as well. The trial patient randomization was halted early because of the benefit seen, and all patients enrolled in the trial will receive immunotherapy plus standard treatment.
Yu noted that the two-year mark is especially important because past trials have shown that those neuroblastoma patients who live without disease for two years after a stem cell transplant will most likely be cured.
"This is the first time in many years that we have been able to improve the 'cure rate' for neuroblastoma patients," she said. "This new therapy can help us improve care and perhaps offer new hope to many patients and families."
Yu and her team conducted the early phase I and phase II trials at the General Clinical Research Center at UC San Diego Medical Center.
University of California - San Diego
The therapy targets a specific glycan (a complex sugar chain found on the surface of cells) on neuroblastoma cells called GD2, which inhibit the immune system from killing cancer cells. The antibody - ch14.18 - binds to this glycan, enabling various types of immune cells to attack the cancer.
Neuroblastoma - in which the cancer cells arise from nerve cells in the neck, chest, or abdomen - is the most common cancer diagnosed in the first year of life. Approximately 650 new cases of neuroblastoma are diagnosed in this country every year, and about 40 percent of patients have high-risk neuroblastoma. These high-risk patients are usually treated with surgery, intensive chemotherapy with stem cell rescue (in which patients' adult stem cells, removed before treatment, are returned after chemotherapy to restore the blood and immune system), and radiation therapy. Still, only 30 percent of patients survive.
Yu and her colleagues compared both the percentage of patients who were still alive without experiencing a recurrence after two years as well as overall survival in two groups of 113 patients each. Patients began the trial when they were newly diagnosed with high-risk neuroblastoma. After conventional treatment with surgery, chemotherapy, stem cell rescue and radiotherapy, one group was given the standard treatment (retinoic acid) plus immunotherapy (the antibody plus immune-boosting substances), while 113 similar patients received the standard treatment alone.
After two years, 66 percent of individuals in the immunotherapy group were living free of cancer compared to 46 percent in the standard treatment group. Overall survival improved significantly as well. The trial patient randomization was halted early because of the benefit seen, and all patients enrolled in the trial will receive immunotherapy plus standard treatment.
Yu noted that the two-year mark is especially important because past trials have shown that those neuroblastoma patients who live without disease for two years after a stem cell transplant will most likely be cured.
"This is the first time in many years that we have been able to improve the 'cure rate' for neuroblastoma patients," she said. "This new therapy can help us improve care and perhaps offer new hope to many patients and families."
Yu and her team conducted the early phase I and phase II trials at the General Clinical Research Center at UC San Diego Medical Center.
University of California - San Diego
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Why are some pediatric cancers able to spontaneously regress? Prof. Michael Fainzilber and his team of the Weizmann Institute's Biological Chemistry Department seem to have unexpectedly found part of the answer.
PET Can Help Guide Treatment Decisions for a Common Pediatric Cancer
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Protein That Promotes Cancer Cell Growth Identified
Scientists at Burnham Institute for Medical Research (Burnham) have found that the Caspase-8 protein, long known to play a major role in promoting programmed cell death (apoptosis), helps relay signals that can cause cancer cells to proliferate, migrate and invade surrounding tissues.
U of M Researchers Find Childhood Cancer Risk Rises with Mother's Age
Research from the Masonic Cancer Center, University of Minnesota indicates that a baby born to an older mother may have a slightly increased risk for many of the cancers that occur during childhood.
Variations in 5 genes raise risk for most common brain tumors
Common genetic variations spread across five genes raise a person's risk of developing the most frequent type of brain tumor, an international research team reports online in Nature Genetics.
Genetic finding could lead to targeted therapy for neuroblastoma
Researchers have identified a genetic glitch that could lead to development of neuroblastoma, a deadly form of cancer that typically strikes children under 2.
Researchers identify gene that regulates tumors in neuroblastoma
Virginia Commonwealth University researchers have identified a gene that may play a key role in regulating tumor progression in neuroblastoma, a form of cancer usually found in young children.
New therapy enlists immune system to boost cure rate in a childhood cancer
A multicenter research team has announced encouraging results for an experimental therapy using elements of the body's immune system to improve cure rates for children with neuroblastoma, a challenging cancer of the nervous system.
Drug therapy reduces neuroblastoma tumor growth in pre-clinical investigation
Researchers from the Children's Cancer Hospital at The University of Texas M. D. Anderson Cancer Center have discovered a new drug combination that significantly hinders tumor growth in neuroblastoma, a childhood cancer.
Drug inhibits neuroblastoma blood supply in pre-clinical tests
Researchers from the Children's Cancer Hospital at The University of Texas M. D. Anderson Cancer Center have found a way to prevent blood vessels from aiding the growth of neuroblastoma, a childhood cancer.
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