Reading RNA in individual cells from high-risk childhood neuroblastoma
Official title: Long-read, single-cell RNA sequencing of high-risk neuroblastoma samples
Funded at Children's Hosp of Philadelphia · NIH-11248384 · Data updated 2026-11-12
This project uses long-read RNA sequencing on single tumor cells to find cancer-specific surface proteins that might become new immune therapy targets for children with high-risk neuroblastoma.
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 | 2026 |
| Award (this year) | $178,000 |
| NIH project number | 5R03CA293992-02 |
| Project ID | NIH-11248384 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children diagnosed with high-risk neuroblastoma or families willing to donate tumor tissue or sequencing data for research would be the ideal participants or sample donors.
✕Less likely to help
Patients with low-risk neuroblastoma, adult patients, or those whose tumors do not express the identified splicing-derived targets may not benefit from these findings.
What this research studies
This work looks at RNA from individual neuroblastoma tumor cells using long-read sequencing to capture complex mRNA changes like alternative splicing. Researchers compare tumor samples to normal adrenal gland tissue and focus on changes that alter the parts of proteins exposed on the cell surface. They prioritize events that create new, in-frame extracellular sequences that could act as tumor-specific targets for antibody-drug conjugates or CAR T cells. The team builds on existing pediatric RNA datasets to discover splicing-derived neo-epitopes, with the goal of expanding options beyond current GD2- and GPC2-targeted therapies.
Why it matters
Potential benefit: If successful, this could identify new tumor-specific surface markers that lead to safer, more effective targeted immunotherapies for children with high-risk neuroblastoma.
How similar studies have gone: Existing immunotherapies like dinutuximab and early GPC2-directed approaches have shown benefit, but using long-read, single-cell splicing analysis to find new surface targets is a newer and relatively untested strategy.
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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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.