Targeting the TAF1 protein in acute myeloid leukemia
Selective targeting of TAF1 function in acute myeloid leukemia
Researchers are creating molecules that tag the TAF1 protein for destruction to try to stop AML driven by the AML1-ETO fusion.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | H. Lee Moffitt Cancer Ctr & Res Inst NIH-funded |
| Lab location | 1 site (Tampa, United States) |
| Project ID | NIH-11259574 on NIH RePORTER |
What this research studies
The team is designing CRBN-directed PROTAC molecules that bind TAF1 and recruit the cell's disposal machinery to degrade it. They use structure-guided chemistry and laboratory tests in AML cell lines and models to measure whether removing TAF1 kills leukemia cells while sparing normal blood stem cells. This work builds on findings that AML1-ETO-driven leukemia depends on TAF1, whereas normal hematopoietic stem cells tolerate TAF1 perturbation. If preclinical results are strong, the approach could move toward early human testing at cancer centers.
Who could benefit from this research
Good fit: People with acute myeloid leukemia—particularly those whose leukemia carries the AML1-ETO fusion or those with relapsed/refractory disease—would be the most relevant candidates for later clinical trials.
Not a fit: Patients without AML or with leukemia subtypes not driven by TAF1 (and those needing immediate standard treatment) are unlikely to benefit from this early preclinical work.
Why it matters
Potential benefit: If successful, this could produce new targeted drugs that eliminate certain AML cells while causing fewer harms to normal blood cells.
How similar studies have performed: PROTAC degraders have shown promise in other cancers, but direct TAF1 targeting is largely untested in patients and prior bromodomain inhibitors showed limited leukemia cell killing.
Where this research is happening
Tampa, United States
- H. Lee Moffitt Cancer Ctr & Res Inst — Tampa, United States (Active)
Researchers
- Principal investigator: Schonbrunn, Ernst — H. Lee Moffitt Cancer Ctr & Res Inst
- Study coordinator: Schonbrunn, Ernst
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.