Improving treatments for acute myeloid leukemia with IDH mutations
Official title: Project 1: Increasing therapeutic efficacy in isocitrate dehydrongenase (IDH)–mutant acute myeloid leukemia (AML)
Funded at Sloan-Kettering Inst Can Research · NIH-11124242 · Data updated 2026-11-12
This research aims to find better ways to treat acute myeloid leukemia (AML) in patients whose cancer has specific IDH gene changes.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Sloan-Kettering Inst Can Research NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | August 24, 2021 |
| Project end | June 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $235,096 |
| NIH project number | 5P50CA254838-05 |
| Project ID | NIH-11124242 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with acute myeloid leukemia (AML) who have specific IDH1 or IDH2 gene mutations, particularly those who have not responded to current IDH inhibitors or have relapsed, would be ideal candidates.
✕Less likely to help
Patients whose acute myeloid leukemia does not have IDH1 or IDH2 mutations would likely not benefit directly from this specific research.
What this research studies
Current treatments for acute myeloid leukemia (AML) with IDH gene mutations work for some patients, but not all, and some patients experience a return of their cancer. This project will look closely at patient samples and conduct laboratory studies to understand why some treatments are more effective than others. Researchers will also test new combinations of therapies to improve outcomes for patients with IDH-mutant AML. This includes conducting clinical trials for patients with specific genetic profiles, such as those with both FLT3 and IDH mutations.
Why it matters
Potential benefit: If successful, this work could lead to more effective and lasting treatments for patients with IDH-mutant AML, especially those who don't respond well to current therapies or who relapse.
How similar studies have gone: Existing IDH1 and IDH2 inhibitors are already approved and have shown success in some patients with IDH-mutant AML, but this project seeks to overcome limitations like resistance.
Where this research is happening
-
Sloan-Kettering Inst Can Research
New York, United States Active
Researchers
- Principal investigator: Levine, Ross L — Sloan-Kettering Inst Can Research
- Study coordinator: Levine, Ross L
Related research
Other NIH-funded projects on the same topics.
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Acute Myeloid Leukemia: Improving Targeted Therapies through Genetically Defined PDX Models
Researchers are using patient-like leukemia models to develop therapies that target specific genetic features of AML to improve survival.
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A broader genomic risk picture to guide AML treatment
AIM to refine how genetic information guides treatment decisions for adults with acute myeloid leukemia by looking at how inherited and recurrent mutations affect risk.
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RNA Polymerase II as a Therapeutic Target in AML with RAS Activation
This project explores a new way to treat a common type of blood cancer (AML) by blocking a key machinery (RNA Polymerase II) that cancer cells use when the RAS signaling pathway is overactive.
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SHP2 and BCL2 inhibitors together for AML growth and resistance
A patient-focused look at whether combining SHP2 inhibitors with a BCL2 inhibitor can slow or stop AML, especially in people whose disease has FLT3 or KIT mutations.
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Targeting RNA polymerase II in AML with RAS pathway activation
This project tests drugs that block RNA polymerase II to help people whose acute myeloid leukemia has RAS pathway activation and is resistant to FLT3 inhibitors.
-
New treatment approach for myelodysplastic syndrome and acute myeloid leukemia
This project aims to develop a new antibody-based therapy to help people with MDS or AML, especially older or frail patients who cannot tolerate high-dose chemotherapy.
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.