Improving treatments for acute myeloid leukemia (AML)
Official title: Acute myeloid leukemia (AML) Research Project
Funded at University of Pennsylvania · NIH-11135489 · Data updated 2026-11-12
This program will use patient-derived leukemia samples and lab models to develop targeted treatments for people with genetically defined AML.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pennsylvania NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | July 5, 2023 |
| Project end | June 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $230,344 |
| NIH project number | 5U54CA283759-03 |
| Project ID | NIH-11135489 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with a diagnosis of AML—especially those whose leukemia carries known genetic mutations—who can provide tumor samples or be considered for future trials would be most relevant to this work.
✕Less likely to help
People without AML, or patients whose leukemia lacks the targeted genetic alterations or who cannot provide samples, are unlikely to gain direct benefit from this program.
What this research studies
Researchers are collecting and standardizing large numbers of patient-derived AML samples and building mouse and lab models that reflect specific genetic subtypes. They will test targeted drugs and drug combinations in these patient-derived xenograft (PDX) models to see which genetic changes respond best. The team plans to link model results to genetic features so promising therapies can be moved toward clinical trials for patients with matching AML genetics.
Why it matters
Potential benefit: Could identify targeted drugs or combinations that produce better responses for people with specific AML genetic subtypes.
How similar studies have gone: Targeted drugs such as FLT3 and IDH inhibitors have produced meaningful responses in AML but have not cured most patients, and using patient-derived models to guide therapy is a known but still improving approach.
Where this research is happening
-
University of Pennsylvania
Philadelphia, United States Active
Researchers
- Principal investigator: Carroll, Martin — University of Pennsylvania
- Study coordinator: Carroll, Martin
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
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.
-
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.
-
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.
-
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.