Drug that targets leukemia stem cells by removing HSF1
Official title: Targeting Leukemia Stem Cells with Small Molecule Heat Shock Transcription Factor 1 Degrader
Funded at Case Western Reserve University · NIH-11291313 · Data updated 2026-11-12
This project aims to use a new small molecule that removes a protein called HSF1 to try to kill leukemia stem cells in people with acute myeloid leukemia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Case Western Reserve University NIH-funded |
| Lab location | 1 site (Cleveland, United States) |
| Project start | April 1, 2025 |
| Project end | March 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $502,516 |
| NIH project number | 5R01CA292487-02 |
| Project ID | NIH-11291313 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with acute myeloid leukemia, particularly those with relapsed or treatment-resistant disease or evidence of high HSF1 activity, would be the most relevant candidates for future trials or sample donation.
✕Less likely to help
People without AML or whose leukemia does not depend on HSF1 activity are unlikely to benefit from this approach.
What this research studies
Researchers are developing small molecules that bind and selectively degrade the active, nuclear form of HSF1, a protein that helps leukemia stem cells survive stress and resist treatment. Lab work includes experiments in mouse models and tests on human AML stem cells and patient-derived samples to see whether these degraders reduce stem-cell survival. The team has used an earlier compound called SISU-102 and is now working on newer, more potent HSF1 degraders with improved drug-like properties. This work is preclinical but is expressly focused on a drug mechanism that could move toward clinical testing if results remain promising.
Why it matters
Potential benefit: If successful, the approach could produce therapies that eliminate leukemia stem cells and lower the risk of AML relapse.
How similar studies have gone: Other HSF1 inhibitors have shown activity in cell and mouse models but were often indirect or mechanistically unclear, while direct HSF1 degraders like SISU-102 have shown promising preclinical activity against AML stem cells but remain experimental.
Where this research is happening
-
Case Western Reserve University
Cleveland, United States Active
Researchers
- Principal investigator: Zhao, Chen — Case Western Reserve University
- Study coordinator: Zhao, Chen
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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.