Targeting a specific protein to treat a type of blood cancer
Official title: Targeting PLK1 in RAS mutant chronic myelomonocytic leukemia
Funded at Mayo Clinic Rochester · NIH-11067766 · Data updated 2026-11-12
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.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mayo Clinic Rochester NIH-funded |
| Lab location | 1 site (Rochester, United States) |
| Project start | May 1, 2023 |
| Project end | April 30, 2028 |
| Funding year | 2025 |
| Award (this year) | $427,131 |
| NIH project number | 5R01CA272496-03 |
| Project ID | NIH-11067766 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients diagnosed with relapsed or refractory proliferative chronic myelomonocytic leukemia who have RAS pathway mutations.
✕Less likely to help
Patients with other types of leukemia or those who do not have RAS mutations may not benefit from this research.
What this research studies
This research focuses on chronic myelomonocytic leukemia (CMML), a serious blood cancer with poor outcomes. It aims to develop targeted therapies for patients with the proliferative subtype of CMML, which often has mutations in the RAS pathway. The study will test a new oral medication called Onvansertib, which inhibits a protein known as PLK1, in patients whose cancer has returned or is resistant to other treatments. By using innovative trial designs, the research seeks to determine the safety and effectiveness of this treatment approach.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatment options for patients with relapsed or refractory chronic myelomonocytic leukemia.
How similar studies have gone: Previous studies have shown promise in targeting PLK1 in similar blood cancers, indicating potential for success in this novel approach.
Where this research is happening
-
Mayo Clinic Rochester
Rochester, United States Active
Researchers
- Principal investigator: Patnaik, Mrinal Shiv — Mayo Clinic Rochester
- Study coordinator: Patnaik, Mrinal Shiv
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.
-
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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Patient-based mouse models to find better treatments for chronic myelomonocytic leukemia
Researchers are using mice implanted with patients' CMML cells to try out new treatment approaches for people with chronic myelomonocytic leukemia.
-
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.
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.