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GPC2 as an Oncogene and Immunotherapeutic Target in High-risk Neuroblastoma

Children’s Hospital of Philadelphia
Kristopher Bosse, MD
Grant Type: 
Young Investigator Grants
Year Awarded: 
Type of Childhood Cancer: 
Project Description: 


Neuroblastoma is an aggressive cancer of the developing nerves that occurs in young children. There has been little improvement in the prognosis of children diagnosed with high-risk neuroblastoma over the last few decades. Recent advances in the field of immunotherapy have resulted in unmatched enthusiasm for the use of this potent treatment to fight against neuroblastoma. However, we desperately need new molecules to safely target on neuroblastoma cells with immunotherapy. We have recently discovered that the gene glypican-2 (GPC2) is selectively found on the neuroblastoma cell surface (but not on most normal cells) and that GPC2 helps neuroblastomas grow aggressively. GPC2 may also act as a signal to attract certain types of immunotherapies to the tumor, such as genetically modified immune cells called chimeric antigen receptor (CAR) T cells. 

Project Goal

This work aims to directly validate a new targeted and potent immunotherapy for children with neuroblastoma. First, the project will focus on understanding why there are high levels of GPC2 on neuroblastomas and how GPC2 helps neuroblastomas grow aggressively. We will also engineer GPC2 redirected CAR T cells to specifically seek out neuroblastoma cells to see if this prevents tumors from growing. In addition, learning how GPC2 helps neuroblastomas proliferate could also provide us with critical information that may also contribute to improved neuroblastoma treatments.

"An Alex's Lemonade Stand Foundation Young Investigator (ALSF YI) Grant will help catalyze our exploration of new methods to most efficiently use the immune system to safely and effectively target neuroblastoma." - Kristopher Bosse, MD