Using antiviral drugs to target jumping DNA in hard-to-treat childhood neuroblastoma
Official title: Targeting retrotransposons for improved treatment of refractory childhood cancer
Funded at Roswell Park Cancer Institute Corp · NIH-11401311 · Data updated 2026-11-12
Researchers are looking at whether common antiviral drugs can block jumping DNA elements to help children with relapsed or refractory high-risk neuroblastoma.
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 | 2025 |
| Award (this year) | $224,100 |
| NIH project number | 3R01CA292639-02S1 |
| Project ID | NIH-11401311 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be children with relapsed or refractory high-risk neuroblastoma, particularly those whose tumors show evidence of LINE1 retrotransposon activation.
✕Less likely to help
Patients whose tumors lack retrotransposon activation or who have non-neuroblastoma cancers are less likely to benefit from this approach.
What this research studies
This program focuses on high-risk neuroblastoma in children who have relapsed or not responded to standard treatments. Researchers have found that activation of LINE1 retrotransposons (jumping DNA) may drive drug resistance in tumors. They plan to test whether approved nucleoside reverse transcriptase inhibitors (NRTIs) can block these elements using lab studies, animal models, and analyses of tumor samples, and to explore combining this approach with immune-based strategies. The goal is to see if repurposing well-known antiviral drugs could offer a new way to control resistant pediatric tumors.
Why it matters
Potential benefit: If successful, this could provide a new, potentially less-toxic treatment option by repurposing approved antiviral drugs to slow or stop tumor growth in children with refractory neuroblastoma.
How similar studies have gone: Early laboratory work and studies in some adult cancers suggest blocking retrotransposons can impair tumor cells, but applying NRTIs to pediatric neuroblastoma is largely novel and not yet tested in patients.
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.