Targeting KAT6A in MLL-rearranged acute myeloid leukemia
Investigating the role of KAT6A in MLL-rearranged acute myeloid leukemia
['FUNDING_R01'] · UNIVERSITY OF PENNSYLVANIA · NIH-11192259
This project tests whether blocking a protein called KAT6A can make leukemia cells with MLL rearrangements mature and stop growing.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF PENNSYLVANIA (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-11192259 on ClinicalTrials.gov |
What this research studies
Researchers are studying KAT6A, a protein that helps keep AML cells immature, with a focus on leukemias that have MLL rearrangements. Using chromatin-focused CRISPR screens, cell lines, and small molecule inhibitors they showed that stopping KAT6A encourages leukemia cells to differentiate and reduces their growth. The team will map how KAT6A works with other epigenetic factors and test KAT6A inhibitors alone and in combinations in lab and animal models. The aim is to identify drug approaches that could be advanced into clinical trials for patients with this subtype.
Who could benefit from this research
Good fit: People with MLL-rearranged acute myeloid leukemia, especially those with relapsed or treatment-resistant disease, would be the most relevant patient group for this work.
Not a fit: Patients with AML subtypes that do not have MLL rearrangements or whose disease does not depend on KAT6A activity are less likely to benefit.
Why it matters
Potential benefit: If successful, this could lead to new therapies that force MLL-rearranged AML cells to mature and stop proliferating, potentially improving remission rates.
How similar studies have performed: Other epigenetic drugs (like LSD1, BRD4, DOT1L inhibitors) have partially reactivated differentiation in AML models, and early lab data suggest KAT6A inhibition may produce stronger differentiation in MLL-rearranged models, but clinical benefit has not yet been demonstrated.
Where this research is happening
PHILADELPHIA, UNITED STATES
- UNIVERSITY OF PENNSYLVANIA — PHILADELPHIA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BLANCO, MARIO ANDRES — UNIVERSITY OF PENNSYLVANIA
- Study coordinator: BLANCO, MARIO ANDRES
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.