Blocking microRNA signals that keep leukemia stem cells alive
Targeting MicroRNAs to Eradicate Leukemia Stem Cells
This project tests a new molecule called miRisten that blocks miR-126 to try to kill the chemotherapy-resistant stem cells that drive acute myeloid leukemia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Beckman Research Institute/city of Hope NIH-funded |
| Lab location | 1 site (Duarte, United States) |
| Project ID | NIH-11139529 on NIH RePORTER |
What this research studies
From a patient's view, researchers are developing a targeted inhibitor called miRisten that lowers levels of a small RNA (miR-126) which helps leukemia stem cells hide and survive. The team has shown in the lab that miR-126 comes from both the leukemia cells themselves and from nearby blood vessel cells, and miRisten reduces both sources. Experiments in animal models, including serial transplants, show that lowering miR-126 damages stem-cell function and reduces leukemia growth. The work also looks at how blocking miR-126 changes leukemia cell metabolism and mitochondrial behavior to make the stem cells less fit.
Who could benefit from this research
Good fit: People with acute myeloid leukemia, especially those with relapsed or treatment-resistant disease, would be the most likely candidates for therapies developed from this work.
Not a fit: Patients with other blood cancers that do not depend on miR-126, or those whose disease is already cured without detectable leukemia stem cells, may not benefit from this approach.
Why it matters
Potential benefit: If successful, this approach could reduce relapses and improve long-term remissions by removing the treatment-resistant leukemia stem cells that drive AML.
How similar studies have performed: Preclinical laboratory and animal work by the investigators has shown promising results with miR-126 inhibition, but the approach has not yet been proven in human patients.
Where this research is happening
Duarte, United States
- Beckman Research Institute/city of Hope — Duarte, United States (Active)
Researchers
- Principal investigator: Kuo, Ya-Huei — Beckman Research Institute/city of Hope
- Study coordinator: Kuo, Ya-Huei
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 below.