Targeting BRG1/BRM proteins to treat acute myeloid leukemia
Role of targeting ATPases BRG1/BRM in therapy of AML
['FUNDING_R01'] · UNIVERSITY OF TX MD ANDERSON CAN CTR · NIH-11320842
This project uses drugs that block or destroy BRG1/BRM proteins to kill or make leukemia cells mature in adults with acute myeloid leukemia, including types that stop responding to Menin inhibitors.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF TX MD ANDERSON CAN CTR (nih funded) |
| Locations | 1 site (HOUSTON, UNITED STATES) |
| Trial ID | NIH-11320842 on ClinicalTrials.gov |
What this research studies
The team is testing two approaches against BRG1/BRM, key proteins that help control gene activity in blood cells: a catalytic inhibitor (FHD-286) and a PROTAC degrader (AU15330). They will use lab-grown AML cells, patient-derived samples, and preclinical models to see whether these agents stop AML cells from growing and push them to differentiate, including AML cases with MLL rearrangements or NPM1 mutations. The project will compare effects on normal blood progenitor cells to check for selective toxicity and will measure gene-expression changes linked to treatment response and resistance to Menin inhibitors. Strong preclinical results could support moving these agents into early clinical testing for people with relapsed or treatment-resistant AML.
Who could benefit from this research
Good fit: Adults with acute myeloid leukemia—particularly those whose cancer has MLL rearrangements or NPM1 mutations or who have relapsed after Menin inhibitors—would be the most relevant candidates for future trials stemming from this work.
Not a fit: People with other types of leukemia that do not depend on BRG1/BRM, or those unable to participate in early translational work, are unlikely to receive direct benefit from this preclinical grant.
Why it matters
Potential benefit: If successful, this work could lead to new targeted treatments for AML patients, especially those who relapse after Menin-inhibitor therapy.
How similar studies have performed: Menin inhibitors have produced remissions in some AML patients but commonly see relapse, while BRG1/BRM inhibitor and PROTAC strategies are newer and show promising laboratory results but are not yet proven in patients.
Where this research is happening
HOUSTON, UNITED STATES
- UNIVERSITY OF TX MD ANDERSON CAN CTR — HOUSTON, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: FISKUS, WARREN CAMPBELL — UNIVERSITY OF TX MD ANDERSON CAN CTR
- Study coordinator: FISKUS, WARREN CAMPBELL
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.