Drug-targetable epigenetic changes driving aggressive neuroblastoma
Targetable epigenetic modifiers that promote neuroblastoma malignancy and plasticity
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 ID | NIH-11258053 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: Patients with low-risk neuroblastoma or unrelated medical conditions are unlikely to receive direct benefit from this preclinical research in the near term.
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 performed: 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
Memphis, United States
- 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
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 below.