How an IDH1 mutation changes bile duct cancer and its response to treatment
Official title: Functions of mutant IDH in cholangiocarcinoma
Funded at Massachusetts General Hospital · NIH-11143811 · Data updated 2026-11-12
This work looks at how a common IDH1 mutation changes bile duct cancer and whether blocking that mutation can help the immune system fight the tumor.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts General Hospital NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project start | September 20, 2023 |
| Project end | August 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $653,532 |
| NIH project number | 5R01CA280085-03 |
| Project ID | NIH-11143811 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with cholangiocarcinoma (bile duct cancer) whose tumors carry an IDH1 mutation would be the main candidates for related clinical studies.
✕Less likely to help
People whose tumors do not have an IDH1 mutation or who have other cancer types are unlikely to benefit directly from this work.
What this research studies
I want to know why some bile duct cancers with an IDH1 mutation stop responding to drugs that target that mutation. The researchers use genetically engineered mouse models, tumor samples from patients, and lab-grown cancer models to study how the mutant IDH1 molecule makes tumors hide from immune attack. They test whether drugs that block mutant IDH1 restore immune signals like interferon responses and whether that improves the effect of immune checkpoint therapies. The team is also studying how the mutant oncometabolite (R)-2-hydroxyglutarate and TET2 enzyme changes tumor cell differentiation and T cell recruitment.
Why it matters
Potential benefit: If successful, this could lead to treatments that help the immune system better recognize and kill IDH1-mutant bile duct cancers and make IDH1-targeted drugs work longer.
How similar studies have gone: Drugs that block mutant IDH1 have helped some patients with IDH1-mutant cholangiocarcinoma, but responses are often not durable, and combining IDH1 inhibition with immunotherapy is promising in lab and animal studies though not yet proven in patients.
Where this research is happening
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Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: El-Bardeesy, Nabeel — Massachusetts General Hospital
- Study coordinator: El-Bardeesy, Nabeel
Related research
Other NIH-funded projects on the same topics.
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A new mouse model to study intrahepatic cholangiocarcinoma and its immunotherapy
A new mouse model helps researchers understand intrahepatic cholangiocarcinoma and test immune-based treatments to improve outcomes.
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Radiation plus a patient’s own immune cells to boost cholangiocarcinoma treatment
This project tests a therapy that combines precise radiation and the patient’s own dendritic cells with immune drugs to help fight intrahepatic cholangiocarcinoma in people who cannot have surgery.
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Worldwide genetics of bile duct (cholangiocarcinoma)
Researchers are looking for genetic patterns that raise the risk of bile duct cancer in people from diverse regions worldwide.
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MKK4 and Liver Regeneration after Major Liver Surgery
A study at Mayo Clinic will test a new drug, HRX215, to help people regrow liver tissue after major liver surgery for cancer.
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New mouse model to improve immune therapy for intrahepatic bile duct (cholangiocarcinoma) cancer
This project builds a new mouse model to help make immune-based treatments work better for people with intrahepatic cholangiocarcinoma, a type of bile duct liver cancer.
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Mutant IDH in bile duct cancer and its impact on immune response
A patient-facing look at how mutations in IDH1 drive bile duct cancer and how targeting this mutation could help immune cells fight the tumor.
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.