Drug-targetable epigenetic changes driving aggressive neuroblastoma
Official title: Targetable epigenetic modifiers that promote neuroblastoma malignancy and plasticity
Funded at St. Jude Children's Research Hospital · NIH-11258053 · Data updated 2026-11-12
Researchers are looking at whether drugs that change how genes are packaged (epigenetic drugs) can block aggressive neuroblastoma in children.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project start | January 15, 2025 |
| Project end | December 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $696,863 |
| NIH project number | 5R01CA289881-02 |
| Project ID | NIH-11258053 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with high-risk or relapsed neuroblastoma—especially tumors with MYCN amplification or high MYC activity—would be the most relevant patients for future trials or for donating tumor samples.
✕Less likely to help
Patients with low-risk neuroblastoma or unrelated medical conditions are unlikely to receive direct benefit from this preclinical research in the near term.
What this research studies
The team at St. Jude is studying how MYC oncogenes depend on epigenetic enzymes like HDACs and KDM4 to reinforce developmental blocks that make neuroblastoma cells aggressive and able to change identity. They will use genome-wide CRISPR screens, transcriptomics, and laboratory models of neuroblastoma to identify which epigenetic modifiers are essential for tumor survival and core regulatory circuitry. The project includes testing small-molecule inhibitors in cell and preclinical models to see if blocking these enzymes disrupts tumor growth and plasticity. While this is mainly preclinical laboratory work, the findings could point to targets for future clinical trials or help identify which patients might benefit from epigenetic therapies.
Why it matters
Potential benefit: If successful, this work could reveal new drug targets and lead to better treatments for children with high-risk or relapsed neuroblastoma.
How similar studies have gone: Some epigenetic drugs such as HDAC inhibitors have shown promise in preclinical and early clinical cancer work, but targeting KDM4 and linking these modifiers to MYC/CRC-driven neuroblastoma is a newer, less-tested approach.
Where this research is happening
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St. Jude Children's Research Hospital
Memphis, United States Active
Researchers
- Principal investigator: Yang, Jun — St. Jude Children's Research Hospital
- Study coordinator: Yang, Jun
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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.