Targeted treatment for adults with t(8;21) AML (AML1-ETO)
Official title: Targeted therapy for t(8;21)+ AML
Funded at University of Miami School of Medicine · NIH-11303330 · Data updated 2026-11-12
This project tests drugs that block proteins AML1-ETO needs to grow, aimed at adults with t(8;21) acute myeloid leukemia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Miami School of Medicine NIH-funded |
| Lab location | 1 site (Coral Gables, United States) |
| Project start | September 17, 2012 |
| Project end | February 28, 2029 |
| Funding year | 2026 |
| Award (this year) | $456,796 |
| NIH project number | 5R01CA166835-13 |
| Project ID | NIH-11303330 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be adults diagnosed with t(8;21) (AML1-ETO) acute myeloid leukemia, especially those with relapsed or refractory disease or who can provide diagnostic/leukemia samples.
✕Less likely to help
People with other AML subtypes that do not have the t(8;21) translocation or pediatric patients are unlikely to benefit from these targeted approaches.
What this research studies
You would be helping researchers develop drugs that stop the AML1-ETO fusion protein from turning on genes that keep leukemia cells growing. The team targets partner proteins and chemical tags (like p300/CBP and the Taf1 bromodomain) that AML1-ETO uses, and they are also studying other factors such as ETV6 and Rac1 that help the cancer repress normal cell maturation. Work combines lab models of t(8;21) leukemia with testing on human-derived leukemia samples to see which inhibitors block disease features. The goal is to find drug approaches that could move toward clinical testing for people with this specific AML subtype.
Why it matters
Potential benefit: If successful, this could lead to targeted treatments that are more effective and less toxic for people with t(8;21) AML.
How similar studies have gone: Related drugs that target bromodomains and p300/CBP have shown promise in lab studies and early clinical work for some cancers, but directly targeting AML1-ETO biology is a newer and less-tested approach.
Where this research is happening
-
University of Miami School of Medicine
Coral Gables, United States Active
Researchers
- Principal investigator: Nimer, Stephen D. — University of Miami School of Medicine
- Study coordinator: Nimer, Stephen D.
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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.