Targeting mutant IDH1 in bile duct (cholangiocarcinoma) and other solid cancers
Official title: Harnessing mutant IDH1 as a therapeutic target in liver cancer and other solid malignancies
Funded at Massachusetts General Hospital · NIH-11321278 · Data updated 2026-11-12
Researchers are trying combinations of medicines that block mutant IDH1 to help people with cholangiocarcinoma and other cancers that carry IDH1 mutations.
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 | April 14, 2025 |
| Project end | March 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $676,151 |
| NIH project number | 5R01CA292508-02 |
| Project ID | NIH-11321278 on NIH RePORTER |
Who could benefit from this research
✓Could help
People whose tumors carry an IDH1 mutation, such as some cases of cholangiocarcinoma (bile duct cancer) or glioma, would be the ideal candidates.
✕Less likely to help
Patients without an IDH1 mutation or those unable to receive targeted combination therapies are unlikely to benefit directly from this work.
What this research studies
If my cancer has an IDH1 mutation, this project studies why IDH1-blocking drugs stop working and looks for better combinations to prevent that resistance. Scientists will examine how the mutant IDH1 protein and its abnormal product (2-hydroxyglutarate) affect cancer cells and the immune system using lab models and patient-derived samples. They will test pairing IDH1 inhibitors with other medicines to find combinations that keep tumors controlled longer. The work builds on prior discoveries that IDH1 inhibitors can reduce tumor-promoting effects and revive immune attack.
Why it matters
Potential benefit: If successful, this could produce combination treatments that delay or prevent resistance to IDH1 inhibitors and extend survival for patients with IDH1-mutant cancers.
How similar studies have gone: Single-agent IDH1 inhibitors have extended survival in some patients, but resistance commonly develops, so combining these drugs with other therapies is a promising but still emerging approach.
Where this research is happening
-
Massachusetts General Hospital
Boston, United States Active
Researchers
- Principal investigator: El-Bardeesy, Nabeel — Massachusetts General Hospital
- Study coordinator: El-Bardeesy, Nabeel
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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.