Neuroblastoma Research

There is an enormous amount of heterogeneity across neuroblastoma types, and pediatric patients are usually stratified into three risk categories: low, intermediate or high risk.

Low-risk disease is most common in infants, and positive clinical outcomes are typically achieved by surgery or spontaneous tumor regression. Intermediate-risk disease is usually managed by combinations of surgery, radiation and chemotherapy.

High-risk disease, though, is difficult to treat, even with the most intensive multi-modal therapies that are available.

Researchers at the Center for Childhood Cancer Research are using genomic, immunotherapeutic and targeted molecular approaches to develop new treatment regimens and optimize those that already exist to treat infants and children with neuroblastoma.

The efforts of investigators at the CCCR have resulted in:

• The discovery of new molecular markers that aid in treatment selection and are predictive of disease progression
• Identification of novel targeted and less toxic inhibitors
• Advances in treatment regimens for children with metastatic, relapsed and treatment-refractory pediatric neuroblastomas

Though many of the research programs at the Center for Childhood Cancer Research are experimental, several have advanced to the clinical trial stage and are being evaluated as treatments for neuroblastoma. For example, clinical trials are underway to evaluate the addition of polyamine inhibitors to conventional chemotherapy regimens to treat children with relapsed neuroblastoma.

Also, a novel anti-tumor immunotherapy called chimeric antigen receptor (CAR) T-cells is being clinically evaluated as a treatment for local and metastatic pediatric neuroblastoma.

One of the unique features of the neuroblastoma program at the Center for Childhood Cancer Research is the ability to quickly translate basic research into clinically-relevant therapies that can be used to treat infants and children with neuroblastoma. The depth, breadth and commitment of researchers at the CCCR will ultimately yield a better understanding of neuroblastoma biology and lead to the development of new treatment regimens for this potentially devastating pediatric cancer.