Blocking menin to treat acute leukemias with high HOX gene activity
Official title: Targeting Menin in Acute Leukemia with Upregulated HOX Genes
Funded at University of Michigan at Ann Arbor · NIH-11312667 · Data updated 2026-11-12
This work develops new drugs that block a protein called menin to help people with certain aggressive acute leukemias driven by HOX gene activation.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project start | May 1, 2023 |
| Project end | April 30, 2028 |
| Funding year | 2026 |
| Award (this year) | $633,462 |
| NIH project number | 5R01CA272561-04 |
| Project ID | NIH-11312667 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be children or adults whose leukemia shows MLL1 translocations, NPM1 mutations, or NUP98 rearrangements and who are eligible for early-phase clinical trials.
✕Less likely to help
Patients whose cancers do not have HOX gene upregulation or these specific genetic alterations are unlikely to benefit from this menin-targeted approach.
What this research studies
Researchers are designing and improving small-molecule drugs that bind menin and stop it from helping leukemia-driving proteins turn on HOX and MEIS1 genes. They test these compounds in laboratory models and use medicinal chemistry to make more potent candidates such as MI-3454. The team aims to identify lead compounds safe and strong enough to take into early clinical testing. The work focuses on leukemias with MLL1 translocations, NPM1 mutations, or NUP98 rearrangements and includes efforts relevant to both children and adults.
Why it matters
Potential benefit: If successful, this could produce a new targeted therapy that controls or shrinks aggressive leukemias with these specific genetic changes.
How similar studies have gone: Related menin-MLL1 inhibitors have shown strong anti-leukemia effects in preclinical models and potent compounds like MI-3454 exist, although patient benefit remains to be proven in clinical trials.
Where this research is happening
-
University of Michigan at Ann Arbor
Ann Arbor, United States Active
Researchers
- Principal investigator: Grembecka, Jolanta — University of Michigan at Ann Arbor
- Study coordinator: Grembecka, Jolanta
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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.