Year of Grant: 2015

Location: United States


Neuroblastoma is a neuroendocrine tumor arising in parts of the sympathetic nervous system. It is the most common solid cancer in childhood; 75% occur under 5 years of age. What makes this cancer particularly hard to treat is the fact that recurrent or relapsed tumors alter their genetic code in order to develop resistance to current therapy. 

On average, there are about 650 new cases per year in the United States. While not a frequent cancer, less than 45% with relapsed tumors survive past 5 years. 


Depending on the stage of the disease, the standard of care consists of intensive chemotherapy, surgery, radiation therapy, stem cell transplant, immunotherapies with anti-GD2 mAbs. While new anti-cancer drugs have emerged that target cancer specific genetic mutations, little is known about the genetic mutations in relapsed neuroblastoma tumors. These genetic alterations need to be identified so that new tumor-targeted therapies can be found to increase cure rates.


This study seeks to determine the frequency of actionable therapeutic targets in children with relapsed/recurrent neuroblastoma (rNB), which will later be followed by a precision medicine clinical trial conducted through the New Approaches to Neuroblastoma Therapy (NANT) Consortium, a network of 14 pediatric cancer hospitals. This will provide the means for acquiring specimens from 60 children with rNB and generating clinical reports of actionable genomic targets and immunotherapeutic markers to define the actionable rNB subgroup(s). Clinical reports will be provided to physicians and patients enrolled on this study and linked to clinical trials that match the actionable rNB subgroups.


The study will additionally identify the spectrum of mutations at relapse, as well as expression of TAMS and MYC/MYCN protein expression at relapse. Implementation of tumor enrichments from bone marrow samples will allow a greater number of children to participate in future precision based clinical trials.

CHILDREN'S HOSPITAL LOS ANGELES: New Approaches to Neuroblastoma Therapy (NANT) Precision Clinical Trial