Childhood Cancer

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CAMKV as a Target for Immunotherapy in MYCN-Amplified Neuroblastoma

Institution: 
Children's Hospital of Philadelphia
Researcher(s): 
Robyn Sussman, PhD
Grant Type: 
Young Investigator Grants
Year Awarded: 
2015
Type of Childhood Cancer: 
Neuroblastoma
Project Description: 

Background

Neuroblastoma is a pediatric cancer that typically arises in very young children. Approximately half of neuroblastoma patients present with a clinically aggressive form of the disease; this accounts for only 4% of pediatric cancer diagnoses, yet is responsible for 12% of pediatric cancer deaths.  New treatments are needed to help these high-risk neuroblastoma patients. About half of these children with high-risk neuroblastoma have a genomic alteration that results in multiple copies (>10) of the gene MYCN. We used this as a marker for high-risk neuroblastoma and searched for targets that are concentrated on the surface of cells with amplified MYCN. These targets can get used to design new therapies to specifically kill these neuroblastoma cells in patients with amplified MYCN. One of these targets is called CAMKV and aside from it being concentrated on neuroblastoma with MYCN amplification, very little is known about it.

 

Project Goal

We will study why CAMKV is so concentrated in these cells and what CAMKV is doing in these cells. Furthermore, we will determine if CAMKV resides on the outer surface of the cell to inform us how to best design a therapy targeted to CAMKV. We have a number of other targets from our initial analysis that can be driven through this same pipeline in order to find a target that will result in a successful targeted therapy for high-risk neuroblastoma.

Co-funded by: 
Northwestern Mutual Foundation