Investigating new targets for treating high-risk neuroblastoma in children
Official title: Long-read, single-cell RNA sequencing of high-risk neuroblastoma samples
Funded at Children's Hosp of Philadelphia · NIH-11063464 · Data updated 2026-11-12
This study is looking at a tough childhood cancer called high-risk neuroblastoma to find new ways to help treat it, by examining tumor samples to discover unique proteins that could be targeted by new immunotherapies, like CAR T cells, to improve treatment for kids facing this illness.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Children's Hosp of Philadelphia NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | January 1, 2025 |
| Project end | December 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $178,000 |
| NIH project number | 1R03CA293992-01A1 |
| Project ID | NIH-11063464 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children under 11 years old diagnosed with high-risk neuroblastoma.
✕Less likely to help
Patients with low-risk neuroblastoma or other unrelated cancers may not benefit from this research.
What this research studies
This research focuses on high-risk neuroblastoma, a severe childhood cancer, by analyzing RNA from tumor samples to identify new potential targets for immunotherapy. The team will utilize advanced sequencing techniques to explore variations in mRNA processing, particularly alternative splicing, which may reveal new surface proteins that can be targeted by therapies like CAR T cells. By comparing neuroblastoma samples to normal adrenal gland tissue, the researchers aim to discover unique neo-epitopes that could lead to more effective treatments. This approach is designed to address the limitations of current therapies and improve outcomes for affected children.
Why it matters
Potential benefit: If successful, this research could lead to the development of new immunotherapies that significantly improve survival rates for children with high-risk neuroblastoma.
How similar studies have gone: Previous research has shown promise in using transcriptomic analyses to identify new therapeutic targets in various cancers, suggesting this approach could be effective.
Where this research is happening
-
Children's Hosp of Philadelphia
Philadelphia, United States Active
Researchers
- Principal investigator: Thomas-Tikhonenko, Andrei — Children's Hosp of Philadelphia
- Study coordinator: Thomas-Tikhonenko, Andrei
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Researchers are using advanced computer analysis and lab tests to find new genes and drug targets in neuroblastoma, aiming to improve outcomes for children.
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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.