Neuroblastoma Research

Tremendous advances have been made in the treatment of childhood cancer. Research into new drugs for the treatment of cancer in children at UCSF Benioff Children's Hospital is conducted through the Pediatric Clinical Research Center, one of six state-of the-art units for children in the United States funded by the National Institutes of Health. We have an active research program that tests new therapies in a range of pediatric cancers.

It is important to understand the long-term health effects associated with chemotherapy and radiation in the areas of reproduction, learning, socialization and common adult diseases, such as stroke and heart failure. We are participating in a national study to closely monitor the health of a group of 20,000 survivors of childhood cancer to answer these important questions.

Novel Radiotherapy in Drug Form for Neuroblastoma

Dr. Kathryn Matthay, chief of pediatric hematology and oncology at UCSF, is leading clinical trials through the nationwide consortium New Approaches to Neuroblastoma Therapy studying novel radiotherapies for the disease.

Matthay has pioneered studies of a treatment called 131I-MIBG in high-risk patients with drug-resistant, metastatic or recurrent neuroblastoma. A component of 131I-MIBG is a gradually decaying radioisotope of iodine, which acts as a radiotherapy. The injected drug specifically targets neuroblastoma and related cells, and also can be used to visualize tumor tissue with scans.

Matthay and her consortium collaborators have shown that 131I-MIBG can effectively be combined with standard chemotherapy to improve the duration of survival without neuroblastoma regrowth, even in patients whose cancers have grown and spread despite prior treatments.

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Katherine Matthay
Dr. Katherine Matthay,
pediatric hematologist-oncologist