Neuroblastoma Developmental Therapeutics

Neuroblastoma is a solid tumor that can form in many places of the body including the adrenal glands, chest, neck, and pelvis. This disease also has the propensity to spread to the bones, bone marrow, liver, lungs and brain. Neuroblastomas are thought to develop when normal neuroblasts (immature cells of the sympathetic nervous system) fail to mature into normal nerve cells.

Treatment for neuroblastoma upfront may include surgery, chemotherapy including stem cell/bone marrow transplantation, radiation therapy and immunotherapy.

Through an integrated environment of basic, translational, and clinical research, investigators at the Center for Childhood Cancer Research are catalyzing the incorporation of research advancements into routine clinical care for children with relapsed or refractory neuroblastoma. The key goals of the program are focused on:

• Elucidating the molecular mechanisms of neuroblastoma development and progression by comprehensively understanding the genetic basis of the disease at diagnosis and relapse.
• Translating laboratory discoveries to novel therapeutics and biomarkers of response and outcome.

The efforts of investigators at the CCCR have resulted in:

• Identification of mutations in genes involved in critical pathways of tumor growth that are often enriched for at time of relapse, and developing specific combination therapies to block these pathways.
• Discovery and validation of ALK as tractable molecular target in the inherited and sporadic forms of neuroblastoma, which has led to several new clinical trials for patients with ALK-driven tumors.
• The implementation of dynamic clinical trial designs that integrate real-time molecular profiling of each patient’s tumor to select more targeted and precise treatment options.
• Novel immunotherapeutic strategies being developed based on identification of cell surface targets for both antibody and cell-based immunotherapies.