KAT6A as a target in MLL‑rearranged acute myeloid leukemia
Investigating the role of KAT6A in MLL-rearranged acute myeloid leukemia
['FUNDING_R01'] · UNIVERSITY OF PENNSYLVANIA · NIH-11223350
This research looks at whether blocking a protein called KAT6A can make leukemia cells mature and stop growing in people with MLL‑rearranged acute myeloid leukemia.
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-11223350 on ClinicalTrials.gov |
What this research studies
Researchers found KAT6A in a lab screen as a protein that helps AML cells stay immature. They use CRISPR gene editing and small‑molecule inhibitors in cell and animal models to see if blocking KAT6A forces cancer cells to mature and stop dividing. The team studies how KAT6A works with another protein called ENL and tests KAT6A inhibitors alone or with other epigenetic drugs. The goal is to generate data that could support future clinical trials for patients with MLL‑rearranged AML.
Who could benefit from this research
Good fit: Ideal candidates for future trials would be people diagnosed with MLL‑rearranged acute myeloid leukemia, especially if their disease remains immature or resistant to standard treatments.
Not a fit: Patients whose AML does not have MLL rearrangements or who respond well to existing targeted therapies are less likely to benefit from KAT6A‑directed approaches.
Why it matters
Potential benefit: If successful, targeting KAT6A could push leukemia cells to mature and stop growing, offering a new treatment approach for people with MLL‑rearranged AML.
How similar studies have performed: Other epigenetic drugs (like LSD1, BRD4, DOT1L inhibitors) have shown the ability to reactivate differentiation in lab models and early studies, while KAT6A inhibition is a newer approach with promising preclinical results but not yet tested in patients.
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.