Targeting retrotransposons to help kids with hard-to-treat cancer
Official title: Targeting retrotransposons for improved treatment of refractory childhood cancer
Funded at Roswell Park Cancer Institute Corp · NIH-11415610 · Data updated 2026-11-12
A pediatric cancer research grant explores using existing antiviral drugs to help children with relapsed neuroblastoma by turning off retrotransposons that drive cancer growth.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Roswell Park Cancer Institute Corp NIH-funded |
| Lab location | 1 site (Buffalo, United States) |
| Project start | June 5, 2024 |
| Project end | May 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $360,011 |
| NIH project number | 5R01CA292639-03 |
| Project ID | NIH-11415610 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children with high-risk or relapsed neuroblastoma who have limited treatment options and could participate in early-phase studies.
✕Less likely to help
Very early-phase research may not yet benefit patients who need immediate therapies or who do not fit study criteria.
What this research studies
This project focuses on high-risk neuroblastoma, a tough childhood cancer, and tests whether drugs originally used against viruses (NRTIs) can calm down cancer cells by silencing LINE1 retrotransposons. The team will explore how feasible it is to repurpose these drugs for pediatric patients and may combine this approach with immunotherapy strategies. The research involves studying pediatric tumor biology, testing drug effects in relevant models, and identifying biomarkers to track response. If successful, the work could translate into new options for children who have exhausted standard therapies.
Why it matters
Potential benefit: If successful, this work could lead to new treatment strategies that improve outcomes for children with relapsed or refractory neuroblastoma.
How similar studies have gone: Preliminary data in other cancers suggest retrotransposon activation can be targeted, but pediatric-specific results are still emerging.
Where this research is happening
-
Roswell Park Cancer Institute Corp
Buffalo, United States Active
Researchers
- Principal investigator: Gudkov, Andrei V — Roswell Park Cancer Institute Corp
- Study coordinator: Gudkov, Andrei V
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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.