Use of Targeted Therapies in the Management of High-risk Neuroblastoma

Researchers at the Center for Childhood Cancer Research (CCCR), led by Rochelle Bagatell, MD, are enrolling children with high-risk neuroblastoma onto a Phase 3 clinical trial to assess the use of targeted therapies in newly diagnosed patients. As a result of the preclinical and clinical work led by physician scientist Yael P. Mossé, MD, children with alterations in the anaplastic lymphoma kinase (ALK) gene will be assigned to receive standard therapy combined with the ALK-targeted agent crizotinib. Patients whose tumors do not have alterations in ALK will be eligible for a portion of the study designed to determine whether the addition of the radiopharmaceutical MIBG improves outcome. Some of the patients will be assigned to receive standard therapy while some will be assigned to receive MIBG as a component of therapy. 

MIBG is a molecule that is readily taken up by neuroblastoma cells. Coupling a radioactive agent to the molecule allows for delivery of radiation directly to the neuroblastoma cells, rather than irradiating the body (or part of it) with photon or proton therapy. MIBG is currently available at a limited number of centers, but it may become more widely available if improved outcomes are seen.

Our Collaborators

Collaborators from outside of the Center for Childhood Cancer Research include: Steven G. DuBois, MD, MS; Julie R. Park, MD; Brian D. Weiss, MD; Meaghan P. Granger, MD; Meredith S. Irwin, MD; Arlene Naranjo; Emily Greengard, MD; and Julia L. Glade Bender, MD.