Patient-based mouse models to find better treatments for chronic myelomonocytic leukemia
Official title: Developing and credentialing patient-derived xenograft models to advance therapeutic approaches for chronic myelomonocytic leukemia
Funded at H. Lee Moffitt Cancer Ctr & Res Inst · NIH-11179159 · Data updated 2026-11-12
Researchers are using mice implanted with patients' CMML cells to try out new treatment approaches for people with chronic myelomonocytic leukemia.
Quick facts
| Grant type | R37 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | H. Lee Moffitt Cancer Ctr & Res Inst NIH-funded |
| Lab location | 1 site (Tampa, United States) |
| Project start | August 12, 2019 |
| Project end | July 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $477,698 |
| NIH project number | 5R37CA234021-07 |
| Project ID | NIH-11179159 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are people diagnosed with CMML, especially those willing to donate blood or bone marrow samples or whose disease includes a small mast cell component.
✕Less likely to help
People without CMML or those seeking immediate treatment effects should not expect direct clinical benefit from this laboratory-based modeling work.
What this research studies
I have CMML, and the team makes mouse models using actual patient leukemia cells so the disease behaves more like it does in people. They have built models from samples of 92 patients using immune-deficient mice engineered to support human blood cells. The next step is to add small mast cell clones found in about 20% of patients to create hybrid models and study how the different cell clones interact. These hybrid models will be used to try drugs that target abnormal cytokine signaling and mRNA splicing seen in specific genetic subgroups.
Why it matters
Potential benefit: If successful, this work could help identify more effective, personalized treatments for CMML patients.
How similar studies have gone: Researchers have already created CMML patient-derived xenografts and used them to find promising targets, while adding mast cell clones to make hybrid models is a new and untested advance.
Where this research is happening
-
H. Lee Moffitt Cancer Ctr & Res Inst
Tampa, United States Active
Researchers
- Principal investigator: Padron, Eric — H. Lee Moffitt Cancer Ctr & Res Inst
- Study coordinator: Padron, Eric
Related research
Other NIH-funded projects on the same topics.
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Targeted therapies for a blood cancer called CMML (CBL-mutant CMML)
A physician-scientist is testing targeted drugs to treat a hard-to-kill blood cancer called CMML in patients whose cancer has CBL mutations.
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Understanding how CMML develops and becomes aggressive
Looks at blocking overactive MEK/ERK signals and BET protein-driven gene activity to slow or reverse aggressive CMML and lower its chance of turning into AML for people whose leukemia has NRAS and ASXL1 mutations.
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Blocking PLK1 in RAS‑mutated chronic myelomonocytic leukemia
People with relapsed or refractory proliferative CMML that has RAS pathway mutations will receive an oral drug called onvansertib that blocks the cell‑division protein PLK1 to look for safety and early signs of benefit.
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How inflammation changes blood stem cells and drives progression in chronic myelomonocytic leukemia
Looking at whether inflammation changes blood stem cells and speeds disease in adults with CMML.
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Understanding how chronic myelomonocytic leukemia resists targeted therapy
This study is looking at how some people with chronic myelomonocytic leukemia (CMML) might not respond to a treatment called cobimetinib, and it aims to help those patients by figuring out why that happens and finding better ways to treat them.
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Targeting a specific protein to treat a type of blood cancer
This study is looking at a new pill called Onvansertib to see if it can help people with a tough type of blood cancer called chronic myelomonocytic leukemia (CMML) that hasn't responded to other treatments, especially for those with specific gene changes.
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.