Targeting retrotransposons to help kids with hard-to-treat cancers like neuroblastoma
Official title: Targeting retrotransposons for improved treatment of refractory childhood cancer
Funded at Roswell Park Cancer Institute Corp · NIH-11535292 · Data updated 2026-11-12
Aims to repurpose safe antiviral drugs to block retrotransposons in children with relapsed or refractory neuroblastoma to improve outcomes.
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) | $224,100 |
| NIH project number | 3R01CA292639-03S1 |
| Project ID | NIH-11535292 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children and young people with relapsed or refractory neuroblastoma or other pediatric cancers where retrotransposon activation is relevant.
✕Less likely to help
Patients without retrotransposon-driven resistance or those who cannot tolerate nucleoside reverse transcriptase inhibitors may not benefit.
What this research studies
This grant focuses on pediatric cancer, especially high-risk neuroblastoma, a leading cause of cancer death in children. Researchers plan to test whether already approved antiviral medicines can silence LINE1 retrotransposons that drive cancer cell resistance, potentially making tumors more treatable. The approach builds on prior findings and explores how to combine retrotransposon targeting with possible immunotherapy to enhance effectiveness. The work emphasizes feasibility in pediatric patients and seeks to translate discoveries into new treatment options for kids with few alternatives.
Why it matters
Potential benefit: If successful, this work could lead to new, orally available drugs that help children with relapsed neuroblastoma respond better to therapy.
How similar studies have gone: Preliminary data suggest retrotransposon activation plays a role in cancer resistance and may be targeted by NRTIs, with early signals in pediatric contexts.
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.