Drugs aimed at the BPTF protein to treat neuroblastoma
Targeting the chromatin binding domains of BPTF for neuroblastoma epigenetic therapy
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 ID | NIH-11252307 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: Patients whose tumors are not driven by BPTF-related biology or who have other cancer types are unlikely to benefit from these agents.
Why it matters
Potential benefit: Could lead to new targeted treatments that slow or stop high-risk neuroblastoma growth.
How similar studies have performed: 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
Memphis, United States
- 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
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.