Drugs aimed at the BPTF protein to treat neuroblastoma
Official title: Targeting the chromatin binding domains of BPTF for neuroblastoma epigenetic therapy
Funded at St. Jude Children's Research Hospital · NIH-11252307 · Data updated 2026-11-12
Developing drug-like molecules that block the BPTF protein to help children with aggressive neuroblastoma.
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 1, 2025 |
| Project end | December 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $716,776 |
| NIH project number | 5R01CA290805-02 |
| Project ID | NIH-11252307 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with neuroblastoma, especially those with high BPTF levels or high-risk or recurrent disease, would be the most relevant patients if this work advances to clinical testing.
✕Less likely to help
Patients whose tumors are not driven by BPTF-related biology or who have other cancer types are unlikely to benefit from these agents.
What this research studies
This project will create and refine small molecules that stop the BPTF protein from binding to DNA and regulating genes. Researchers will test these chemical probes in laboratory-grown neuroblastoma cells and in preclinical models to see if blocking BPTF slows tumor growth. They will look at effects on key cancer drivers like MYC/MYCN and on cancer cell survival. Promising probes could serve as starting points for new targeted therapies for children with high-risk disease.
Why it matters
Potential benefit: Could lead to new targeted treatments that slow or stop high-risk neuroblastoma growth.
How similar studies have gone: Other efforts targeting chromatin remodelers have shown promise in the lab, but high-quality drugs specifically targeting BPTF are novel and largely untested in patients.
Where this research is happening
-
St. Jude Children's Research Hospital
Memphis, United States Active
Researchers
- Principal investigator: Fischer, Marcus — St. Jude Children's Research Hospital
- Study coordinator: Fischer, Marcus
Related research
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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.