Targeting a MYCN-linked weakness in childhood neuroblastoma
Official title: MYCN drives a druggable SUMOylation program in neuroblastoma
Funded at Virginia Commonwealth University · NIH-11250076 · Data updated 2026-11-12
This project tests whether a drug that blocks SUMOylation can weaken MYCN-driven high-risk neuroblastoma in children, alone or added to current maintenance therapy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Virginia Commonwealth University NIH-funded |
| Lab location | 1 site (Richmond, United States) |
| Project start | January 1, 2024 |
| Project end | December 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $425,202 |
| NIH project number | 5R01CA285692-03 |
| Project ID | NIH-11250076 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children with high-risk neuroblastoma whose tumors have MYCN amplification.
✕Less likely to help
Children whose tumors lack MYCN amplification or whose disease is driven by different molecular pathways are unlikely to benefit from this approach.
What this research studies
This project uses tumor samples from children to create patient-derived models and lab tests that mimic MYCN-amplified neuroblastoma. Researchers will treat these models with SUMOylation inhibitors such as TAK-981 (Subasumstat), both alone and combined with standard maintenance therapies like anti-GD2 antibodies and retinoids, to see if tumors are weakened. They will examine how blocking SUMOylation disrupts the adrenergic core regulatory complex that sustains these cancers and will monitor safety in preclinical models. The goal is to identify an approach that could move into clinical trials for children whose tumors depend on MYCN.
Why it matters
Potential benefit: If successful, this work could lead to a new targeted therapy that improves outcomes for children with MYCN-amplified high-risk neuroblastoma.
How similar studies have gone: SUMOylation inhibitors like TAK-981 are already in early cancer trials and preclinical work shows promise, but using them specifically for MYCN-amplified neuroblastoma is a relatively new strategy.
Where this research is happening
-
Virginia Commonwealth University
Richmond, United States Active
Researchers
- Principal investigator: Faber, Anthony Charles — Virginia Commonwealth University
- Study coordinator: Faber, Anthony Charles
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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.