Targeting a unique weakness in a rare liver cancer to improve treatment outcomes
Official title: Exploiting A Critical Vulnerability To Glutamine Antimetabolite Therapy in Fibrolamellar Hepatocellular Carcinoma (FLC)
Funded at Johns Hopkins University · NIH-11045064 · Data updated 2026-11-12
This study is looking at a rare type of liver cancer called fibrolamellar hepatocellular carcinoma, mainly in kids and young adults, and it’s testing a new treatment that combines special medicines to block a nutrient the cancer needs to grow with immune boosters to help the body fight the cancer better.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Johns Hopkins University NIH-funded |
| Lab location | 1 site (Baltimore, United States) |
| Project start | March 1, 2024 |
| Project end | February 28, 2029 |
| Funding year | 2025 |
| Award (this year) | $643,101 |
| NIH project number | 5R01CA289041-02 |
| Project ID | NIH-11045064 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with fibrolamellar hepatocellular carcinoma, particularly children and young adults.
✕Less likely to help
Patients with other types of liver cancer or those who do not have the specific genetic alteration associated with FLC may not benefit from this research.
What this research studies
This research focuses on fibrolamellar hepatocellular carcinoma (FLC), a rare and aggressive liver cancer affecting mainly children and young adults. The study investigates a specific genetic alteration that leads to the cancer's dependence on glutamine, a nutrient that fuels tumor growth. By using a combination of glutamine antimetabolite therapy and immune checkpoint inhibitors, the research aims to enhance the immune response against the tumor and improve treatment efficacy. Patients may have the opportunity to participate in clinical trials that explore this innovative therapeutic approach.
Why it matters
Potential benefit: If successful, this research could provide a new treatment option for patients with fibrolamellar hepatocellular carcinoma, potentially improving survival rates and quality of life.
How similar studies have gone: Preliminary data from preclinical models suggest that similar approaches targeting glutamine dependency have shown promise, indicating potential for success in clinical settings.
Where this research is happening
-
Johns Hopkins University
Baltimore, United States Active
Researchers
- Principal investigator: Yarchoan, Mark — Johns Hopkins University
- Study coordinator: Yarchoan, Mark
Related research
Other NIH-funded projects on the same topics.
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A funded project at a medical university aims to understand how the liver’s immune cells interact with a specific type of liver cancer by looking at metabolism, especially glutamine and ammonia.
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This project tests a new PET imaging approach to help diagnose and guide treatment for liver cancer in veterans.
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Early blood markers to find hepatitis B–related liver cancer in Hispanics
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-
Towards a blood-based test using nanophotonic exosomes to screen and diagnose liver cancer
A new blood test using tiny particles shed by liver cancer cells aims to screen for hepatocellular carcinoma earlier and more accurately.
-
Metabolic imaging to guide targeted therapy for liver cancer
A clinician-scientist at UPenn is pursuing training to use metabolic imaging to tailor therapy for hepatocellular carcinoma.
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.