Improving treatment 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-10862892 · Data updated 2026-11-12
This study is looking to improve treatments for people with acute myeloid leukemia who have certain gene mutations, by figuring out why some don’t respond to current medications and testing new combination therapies that could work better for them.
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 | 2024 |
| Award (this year) | $374,760 |
| NIH project number | 5P50CA254838-04 |
| Project ID | NIH-10862892 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients diagnosed with acute myeloid leukemia who have mutations in the IDH1 or IDH2 genes.
✕Less likely to help
Patients without IDH1 or IDH2 mutations or those with other types of leukemia may not benefit from this research.
What this research studies
This research focuses on enhancing the effectiveness of treatments for patients with acute myeloid leukemia (AML) who have specific mutations in the IDH1 and IDH2 genes. By analyzing patient samples and conducting preclinical studies, the team aims to identify why some patients do not respond to existing IDH inhibitors and to develop new combination therapies that could improve outcomes. The research includes clinical trials that will test these new approaches in genetically defined patient groups, providing a tailored treatment strategy based on individual genetic profiles.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatment options for patients with IDH-mutant acute myeloid leukemia, potentially improving survival rates and quality of life.
How similar studies have gone: Previous research has shown promising results with IDH inhibitors in treating relapsed/refractory IDH-mutant AML, indicating that this approach has potential for further success.
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.
-
A broader genomic risk picture to guide AML treatment
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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.