How an IDH1 mutation changes bile duct cancer and its response to treatment
Functions of mutant IDH in cholangiocarcinoma
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 ID | NIH-11143811 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: Adults with cholangiocarcinoma (bile duct cancer) whose tumors carry an IDH1 mutation would be the main candidates for related clinical studies.
Not a fit: People whose tumors do not have an IDH1 mutation or who have other cancer types are unlikely to benefit directly from this work.
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 performed: 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
Boston, United States
- Massachusetts General Hospital — Boston, United States (Active)
Researchers
- Principal investigator: El-Bardeesy, Nabeel — Massachusetts General Hospital
- Study coordinator: El-Bardeesy, Nabeel
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.