Repurposing antiviral drugs to block mobile DNA in high‑risk neuroblastoma
Official title: Targeting retrotransposons for improved treatment of refractory childhood cancer
Funded at Roswell Park Cancer Institute Corp · NIH-11106018 · Data updated 2026-11-12
Researchers are looking at whether antiviral drugs called NRTIs can stop active mobile DNA elements in tumors to help children whose high‑risk neuroblastoma has come back or not responded to treatment.
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) | $360,013 |
| NIH project number | 5R01CA292639-02 |
| Project ID | NIH-11106018 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with relapsed or treatment‑refractory high‑risk neuroblastoma, especially those whose tumors have failed standard therapies and whose families can provide tumor samples or follow up with the research team.
✕Less likely to help
Children with newly diagnosed low‑risk neuroblastoma, patients with other cancer types, or those unable or unwilling to provide tumor samples are unlikely to benefit directly from this program.
What this research studies
Researchers will study tumor samples and laboratory models to see if active retrotransposons (mobile DNA elements) drive treatment resistance in pediatric neuroblastoma. They will test existing nucleoside reverse transcriptase inhibitors (NRTIs) in cell and animal models and analyze human tumor tissue to confirm the same mechanisms occur in children. The team will also explore combining NRTIs with immune‑based approaches to boost anti‑tumor effects. Because these antivirals are already used in patients for other reasons, promising results could speed the path toward clinical use for children with refractory disease.
Why it matters
Potential benefit: Could provide a new, faster-to-deploy treatment option for children with relapsed or refractory high‑risk neuroblastoma by reversing a mechanism of drug resistance.
How similar studies have gone: Early lab studies and work in some adult cancers suggest retrotransposons can be targeted with NRTIs, but applying this approach to pediatric neuroblastoma is novel and not yet proven in children.
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.