Technology ID

Anti-Glypican 2 Chimeric Antigen Receptor (CAR) Containing CD28 Hinge And Transmembrane Domains For Treating Neuroblastoma

Lead Inventor
Ho, Mitchell (NCI)
Thiele Galetto, Carol (NCI)
Li, Nan (NCI)
Nguyen, Rosa (NCI)
Kaplan, Rosandra (NCI)
Therapeutic Areas
Development Stages
Pre-clinical (in vivo)
Lead IC

Neuroblastomas are the most common extracranial solid tumors in pediatric patients, with 700-800 new cases annually in the United States. Metastatic neuroblastomas have a five-year survival rate of 50% and account for 15% of all pediatric cancer deaths. As such, more effective treatments against high-risk neuroblastomas are urgently needed. Glypican-2 (GPC2) is a cell surface protein that is highly expressed in neuroblastomas and other cancers, including medulloblastoma, retinoblastoma, small-cell lung cancers, uterine carcinosarcomas and high-grade gliomas, which makes GPC2 an attractive candidate for targeted therapy in solid tumors. 

Researchers at the National Cancer Institute’s (NCI) Center for Cancer Research have developed a novel Chimeric Antigen Receptor (CAR) specific for GPC2 that includes a potent anti-GPC2 antibody CT3 and a CD28 hinge and transmembrane domains.  CT3 has been shown to specifically target GPC2-expressing neuroblastoma, medulloblastoma, and retinoblastoma cell lines. CT3.28H.BBζ CAR T cells were shown to be more potent against neuroblastoma cells than the previous anti-GPC2 CAR T cells in vitro and in vivo. These preclinical data suggest that CT3.28H.BBζ CAR T cells may be further developed as therapeutics for patients with neuroblastoma and other GPC2-positive cancers. Incorporation of the CD28 hinge and transmembrane domains increases the potency of the CT3 CARs against neuroblastoma cells.

Competitive Advantages:

  • CT3 antibody with high GPC2 binding specificity leading to  successful targeting
  • CT3 antibody with high GPC2 binding specificity leading to lower potential side-effects
  • Increased potency against neuroblastoma 
  • Potential immunotherapy for several GPC2-positive cancer types with few treatment options 


Commercial Applications:

  • Immunotherapeutic treatments of GPC2-positive pediatric malignancies, including neuroblastoma, medulloblastoma, retinoblastoma, and a subset of acute lymphocytic leukemias
  • Immunotherapeutic treatments of GPC2-positive adult cancers, including small-cell lung cancers, uterine carcinosarcomas and high-grade gliomas
  • CT3.28H.BBζ CAR available for immediate testing


Licensing Contact:
Lambertson, David