Developing new diagnostics and treatments for a rare pediatric liver cancer.
Project 1
This study is working to find better ways to diagnose and treat a rare liver cancer in kids called fibrolamellar hepatocellular carcinoma, by looking closely at its unique features and how a specific protein affects it, so that doctors can offer more personalized care for young patients.
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-10935705 on NIH RePORTER |
What this research studies
This research aims to create effective diagnostic tools and therapies for fibrolamellar hepatocellular carcinoma (FLC), a rare and aggressive liver cancer in children. The project focuses on understanding the cancer's unique biological characteristics, particularly the role of the DNAJB1::PRKACA oncoprotein. By utilizing advanced techniques such as genetic analysis and patient-derived models, the researchers hope to identify new biomarkers and treatment options that can improve patient outcomes. The ultimate goal is to provide precision medicine tailored to the specific needs of FLC patients.
Who could benefit from this research
Good fit: Ideal candidates for this research include children diagnosed with fibrolamellar hepatocellular carcinoma or those at high risk for developing this cancer.
Not a fit: Patients with other types of liver cancer or those without a diagnosis of fibrolamellar hepatocellular carcinoma may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could significantly improve early diagnosis and treatment options for children with fibrolamellar hepatocellular carcinoma.
How similar studies have performed: Previous research has shown promise in developing targeted therapies for rare cancers, indicating potential success for this novel approach.
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.