Targeting mutant IDH1 to treat liver cancer and other solid tumors
Official title: Harnessing mutant IDH1 as a therapeutic target in liver cancer and other solid malignancies
Funded at Massachusetts General Hospital · NIH-11131471 · Data updated 2026-11-12
This study is looking at how to better treat liver cancer and brain tumors that have a specific gene mutation (IDH1) by using special medicines, and it aims to find ways to overcome any resistance to these treatments so that patients can live longer and healthier lives.
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 | 2025 |
| Award (this year) | $693,781 |
| NIH project number | 1R01CA292508-01A1 |
| Project ID | NIH-11131471 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients diagnosed with liver cancer, particularly cholangiocarcinoma, or brain tumors such as glioma, who have mutations in the IDH1 gene.
✕Less likely to help
Patients without IDH1 mutations or those with cancers not related to liver or brain tumors may not benefit from this research.
What this research studies
This research investigates the treatment of cancers that have mutations in the IDH1 gene, particularly focusing on liver cancer and brain tumors. The approach involves using medicines that inhibit mutant IDH1, which have been shown to extend patient lifespans, but resistance to these treatments can develop over time. The research aims to understand the mechanisms behind this resistance and to develop combination therapies that can enhance treatment effectiveness and prevent relapse. By studying how the metabolite 2-hydroxyglutarate (2HG) produced by mutant IDH1 affects cancer growth and immune response, the researchers hope to improve patient outcomes.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for patients with liver cancer and other solid tumors that harbor IDH1 mutations.
How similar studies have gone: Previous research has shown promise in targeting mutant IDH1, indicating that this approach could lead to significant advancements in treatment.
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.