New tests and targeted treatments for fibrolamellar liver cancer
Project 1
Researchers are developing blood tests and targeted medicines to help children and young adults with fibrolamellar hepatocellular carcinoma (FLC).
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mayo Clinic Rochester NIH-funded |
| Lab location | 1 site (Rochester, United States) |
| Project ID | NIH-11178593 on NIH RePORTER |
What this research studies
If you or a family member have FLC, researchers will study the cancer's driver protein (DNAJB1::PRKACA) to find signals that can be used as diagnostic markers and drug targets. They will use tissue and blood from patients plus lab-grown organoids, patient-derived tumor grafts, and genetically engineered mice to see how tumors respond. The team will search the tumor and blood for biomarkers that could allow earlier, less ambiguous diagnosis and test candidate precision drugs in those model systems. Promising findings would be moved toward clinical testing for people with FLC.
Who could benefit from this research
Good fit: People diagnosed with or suspected to have fibrolamellar hepatocellular carcinoma, and those able to provide tumor tissue or blood samples, are the ideal candidates.
Not a fit: People without FLC (other liver cancers or unrelated conditions) or those unable/unwilling to provide samples or travel for care are unlikely to benefit directly from this project.
Why it matters
Potential benefit: Could produce a blood test for earlier diagnosis and targeted therapies that work better than current chemotherapy, improving survival and treatment options.
How similar studies have performed: Previous work identified the DNAJB1::PRKACA fusion and built patient-derived models, but effective diagnostics and targeted treatments for FLC remain largely unproven.
Where this research is happening
Rochester, United States
- Mayo Clinic Rochester — Rochester, United States (Active)
Researchers
- Principal investigator: Simon, Sanford M — Mayo Clinic Rochester
- Study coordinator: Simon, Sanford M
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.