GPC2-targeted CAR T therapy for childhood neuroblastoma
GPC2 CARs in neuroblastoma: Mechanisms of resistance and efficacy of next-generation constructs
['FUNDING_R37'] · CHILDREN'S HOSP OF PHILADELPHIA · NIH-11314486
Developing improved GPC2-targeted CAR T cell treatments to help children with high-risk neuroblastoma avoid relapse.
Quick facts
| Phase | ['FUNDING_R37'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CHILDREN'S HOSP OF PHILADELPHIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-11314486 on ClinicalTrials.gov |
What this research studies
Researchers at Children’s Hospital of Philadelphia are developing CAR T cells that target a tumor protein called GPC2, which is common on neuroblastoma cells but not on most normal tissues. They are studying how tumors escape these therapies, including loss of GPC2, increased activity of the ACE enzyme that can cut GPC2 from the cell surface, and how cancer-released extracellular vesicles change GPC2 levels. The team will use those findings to design and test next-generation CAR constructs in the lab and in preclinical models to make the therapy more durable. This work is linked to an early human trial that began in 2023 and aims to guide better treatments for children.
Who could benefit from this research
Good fit: Children with high-risk or relapsed neuroblastoma whose tumors express GPC2 and who meet the trial’s medical eligibility would be the best candidates.
Not a fit: Patients whose tumors lack GPC2, who have medical conditions that preclude CAR T therapy, or who are not eligible for the trial may not receive benefit from this work.
Why it matters
Potential benefit: Could lead to more durable CAR T treatments that reduce relapse and improve outcomes for children with high-risk neuroblastoma.
How similar studies have performed: Other CAR T therapies have helped some pediatric cancers, and GPC2 CARs showed strong anti-tumor activity in preclinical studies and entered a Phase 1 trial, but antigen loss and relapse remain important hurdles.
Where this research is happening
PHILADELPHIA, UNITED STATES
- CHILDREN'S HOSP OF PHILADELPHIA — PHILADELPHIA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BOSSE, KRISTOPHER R — CHILDREN'S HOSP OF PHILADELPHIA
- Study coordinator: BOSSE, KRISTOPHER R
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.