Drug that targets leukemia stem cells by removing HSF1
Targeting Leukemia Stem Cells with Small Molecule Heat Shock Transcription Factor 1 Degrader
['FUNDING_R01'] · CASE WESTERN RESERVE UNIVERSITY · NIH-11291313
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
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | CASE WESTERN RESERVE UNIVERSITY (nih funded) |
| Locations | 1 site (CLEVELAND, UNITED STATES) |
| Trial ID | NIH-11291313 on ClinicalTrials.gov |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: People without AML or whose leukemia does not depend on HSF1 activity are unlikely to benefit from this approach.
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 performed: 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
CLEVELAND, UNITED STATES
- CASE WESTERN RESERVE UNIVERSITY — CLEVELAND, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: ZHAO, CHEN — CASE WESTERN RESERVE UNIVERSITY
- Study coordinator: ZHAO, CHEN
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.
Conditions: Cancer Model