Treating AML with IDH1/2 mutations by targeting DNA repair problems
Targeting Defective DNA Damage Response Pathways in IDH1/2-mutant AML
This project looks at whether drugs that block DNA repair (PARP inhibitors) can help people with acute myeloid leukemia who have IDH1 or IDH2 mutations.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Yale University NIH-funded |
| Lab location | 1 site (New Haven, United States) |
| Project ID | NIH-11258988 on NIH RePORTER |
What this research studies
If I had AML with an IDH1 or IDH2 mutation, this work would try to exploit a weakness in my cancer's DNA repair caused by that mutation. Researchers will use lab-grown cells, animal models, and patient leukemia samples to see whether PARP inhibitors kill the cancer or make other treatments work better. They plan to test combinations with existing IDH inhibitors or other therapies to overcome resistance and reduce leftover mutant cells. The goal is to develop approaches that could move into clinical testing for people like me.
Who could benefit from this research
Good fit: Ideal candidates would be people with acute myeloid leukemia whose tumors carry IDH1 or IDH2 mutations, especially those who have not had durable responses to current IDH-targeting drugs.
Not a fit: Patients without IDH1/2 mutations, those with other types of leukemia, or those unable to tolerate PARP inhibitors are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, this could lead to new treatment combinations that more effectively eliminate IDH1/2-mutant AML cells and reduce relapses.
How similar studies have performed: Preclinical work has shown that PARP inhibitors can be effective against IDH-mutant cancers, but clinical evidence in AML is limited and combining PARP and IDH-targeted therapies is a newer strategy.
Where this research is happening
New Haven, United States
- Yale University — New Haven, United States (Active)
Researchers
- Principal investigator: Bindra, Ranjit — Yale University
- Study coordinator: Bindra, Ranjit
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.