Targeted therapy for AML using DHODH inhibitors to boost differentiation and immune response
Official title: Pharmacodynamically directed targeted therapy for leukemia
Funded at University of Pittsburgh at Pittsburgh · NIH-11611081 · Data updated 2026-11-12
Aims to help adults with acute myeloid leukemia by using a special drug approach that makes cancer cells act more like normal blood cells and supports the immune system.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pittsburgh at Pittsburgh NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project start | May 1, 2025 |
| Project end | August 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $694,315 |
| NIH project number | 7R01CA292084-02 |
| Project ID | NIH-11611081 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with acute myeloid leukemia, including those with TP53 mutations who may benefit from DHODH inhibitor strategies.
✕Less likely to help
Patients whose leukemia does not respond to DHODH inhibition or who experience intolerable side effects may not benefit.
What this research studies
This project focuses on AML, a deadly adult leukemia, and investigates a drug strategy that both forces leukemia cells to mature and enhances immune activity. The team will study inhibitors of DHODH, a mitochondrial enzyme, to trigger cancer cell differentiation and stress that can lead to cancer cell death. They will test these inhibitors in combination with existing therapies in animal models to find safe, synergistic approaches. The goal is to translate promising results into more effective treatment options for patients with AML, especially those with difficult-to-treat disease.
Why it matters
Potential benefit: If successful, this approach could improve responses and survival for AML patients by combining differentiation and immune-boosting effects with current therapies.
How similar studies have gone: Prior studies in models suggest DHODH inhibitors can differentiate AML cells and activate immune responses, with synergy observed when combined with other therapies.
Where this research is happening
-
University of Pittsburgh at Pittsburgh
Pittsburgh, United States Active
Researchers
- Principal investigator: Byrd, John C. — University of Pittsburgh at Pittsburgh
- Study coordinator: Byrd, John C.
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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.