How Menin-blocking drugs work in certain acute myeloid leukemias
Defining mechanisms of action of Menin inhibition in AML
This project looks at Menin-blocking drugs as a treatment approach for people with KMT2A-rearranged or NPM1-mutant acute myeloid leukemia across children and adults.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Dana-Farber Cancer Inst NIH-funded |
| Lab location | 1 site (Boston, United States) |
| Project ID | NIH-11196542 on NIH RePORTER |
What this research studies
If I have KMT2A-rearranged or NPM1-mutant AML, this project aims to understand how Menin-blocking drugs change the cancer cells' DNA packaging and gene activity. Researchers will map chromatin structures at key oncogenes and test whether combining Menin inhibitors with other drugs (like DOT1L or KAT6A inhibitors) or using Menin-degrading approaches more effectively shuts down the cancer program. Lab and preclinical models will be used to find the most promising combinations and mechanisms that could guide future patient trials. The work builds on early clinical responses to Menin inhibitors and seeks to make those responses more durable and broadly applicable.
Who could benefit from this research
Good fit: Ideal candidates are children and adults with KMT2A-rearranged or NPM1-mutant acute myeloid leukemia, particularly those with high-risk or relapsed disease.
Not a fit: Patients whose leukemia does not have KMT2A rearrangements or NPM1 mutations, or who have other unrelated blood cancers, are unlikely to benefit directly from Menin-targeted approaches.
Why it matters
Potential benefit: If successful, this work could lead to better targeted or combination therapies that improve outcomes for patients with KMT2A-rearranged or NPM1-mutant AML.
How similar studies have performed: Early clinical trials of Menin inhibitors have produced promising responses in KMT2A-rearranged and NPM1-mutant AML, but optimal combinations and mechanisms are still being defined.
Where this research is happening
Boston, United States
- Dana-Farber Cancer Inst — Boston, United States (Active)
Researchers
- Principal investigator: Armstrong, Scott a — Dana-Farber Cancer Inst
- Study coordinator: Armstrong, Scott a
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.