Immune treatment targeting gangliosides in Ewing sarcoma
Official title: Immunotherapeutic targeting of gangliosides in Ewing Sarcoma
Funded at Dana-Farber Cancer Inst · NIH-11169949 · Data updated 2026-11-12
This project is developing CAR T-cell immunotherapy that targets ganglioside molecules to help children and young adults with Ewing sarcoma.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Dana-Farber Cancer Inst NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 19, 2023 |
| Project end | August 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $767,167 |
| NIH project number | 5U01CA281840-03 |
| Project ID | NIH-11169949 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children, adolescents, and young adults with relapsed or metastatic Ewing sarcoma—particularly those whose tumors express GD2 or the related ganglioside—are the most likely candidates.
✕Less likely to help
Patients whose tumors lack GD2 and the related ganglioside, those with cancers not related to Ewing sarcoma, or individuals who cannot undergo cell-based therapies may not benefit from this approach.
What this research studies
This work focuses on bringing CAR T-cell therapy to people with Ewing sarcoma by targeting gangliosides, sugar molecules on the tumor surface. Researchers will analyze patient tumor samples to map which gangliosides and the enzymes that make them are present. They will test CAR T cells that target GD2 and a related ganglioside, and use drugs that can raise GD2 levels so the CAR T cells can better recognize cancer cells. The work includes laboratory and preclinical testing that could lead to patient treatments at Dana-Farber and partner centers.
Why it matters
Potential benefit: If successful, this could provide a targeted immunotherapy that shrinks tumors and reduces reliance on highly toxic chemotherapy for children and young adults with Ewing sarcoma.
How similar studies have gone: CAR T-cell therapies have transformed childhood leukemias and early GD2-directed CAR T work showed activity in some pediatric brain tumors, but applying GD2 CAR T to Ewing sarcoma is a newer and less-tested application.
Where this research is happening
-
Dana-Farber Cancer Inst
Boston, United States Active
Researchers
- Principal investigator: Majzner, Robbie G. — Dana-Farber Cancer Inst
- Study coordinator: Majzner, Robbie G.
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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.