Acute Myeloid Leukemia: Improving Targeted Therapies through Genetically Defined PDX Models
Official title: Acute myeloid leukemia (AML) Research Project
Funded at University of Pennsylvania · NIH-11401381 · Data updated 2026-11-12
Researchers are using patient-like leukemia models to develop therapies that target specific genetic features of AML to improve survival.
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 | 2026 |
| Award (this year) | $230,344 |
| NIH project number | 5U54CA283759-04 |
| Project ID | NIH-11401381 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with acute myeloid leukemia whose disease features align with the genetic subtypes studied in the AML PDX models.
✕Less likely to help
Patients whose AML does not harbor the targeted genetic alterations or who do not respond to the tested therapies may not benefit.
What this research studies
This project aims to advance AML treatment by using genetically defined patient-derived xenograft (PDX) models to test new therapies tailored to particular AML mutations. Researchers at the University of Pennsylvania will expand and characterize AML PDX collections and apply targeted therapies to these models to identify potentially effective combinations. The work focuses on translating genetic findings into more precise treatment options for people living with AML. The effort builds on existing knowledge of AML genetics and aims to move promising strategies toward clinical testing.
Why it matters
Potential benefit: If successful, patients with AML could gain access to more effective, genetics-guided therapies in the future.
How similar studies have gone: Previous work has shown that AML genetics-driven therapies can yield meaningful responses in preclinical models, with some targeted approaches progressing to clinical trials.
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.
-
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.
-
Making genetic risk predictions more accurate for adults with acute myeloid leukemia
This project is developing better genetic-based risk categories to help guide treatment decisions for adults with acute myeloid leukemia.
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.