Targeting leukemia stem cells to improve treatment outcomes

Targeting Leukemia Stem Cells with Small Molecule Heat Shock Transcription Factor 1 Degrader

NIH-funded research Case Western Reserve University · NIH-11129207

This study is testing a new treatment called SISU-102 that aims to help people with acute myeloid leukemia (AML) by targeting and eliminating the stubborn leukemia stem cells that make the disease hard to treat, while still allowing healthy blood cells to grow.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionCase Western Reserve University NIH-funded
Lab location1 site (Cleveland, United States)
Project IDNIH-11129207 on NIH RePORTER

What this research studies

This research focuses on acute myeloid leukemia (AML), specifically targeting the leukemia stem cells (LSCs) that contribute to the disease's persistence and recurrence. The approach involves using a small molecule called SISU-102, which degrades a protein known as heat shock transcription factor 1 (HSF1) that helps LSCs survive and thrive. By selectively targeting and degrading HSF1, the research aims to eliminate these resistant cells while sparing normal blood cell production. This could lead to more effective treatments for AML patients who currently have limited options.

Who could benefit from this research

Good fit: Ideal candidates for this research are patients diagnosed with acute myeloid leukemia, particularly those who have not responded well to conventional therapies.

Not a fit: Patients with other types of leukemia or those who have not been diagnosed with acute myeloid leukemia may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to more effective therapies for AML, potentially reducing the risk of disease recurrence.

How similar studies have performed: While there have been studies targeting HSF1 in cancer, this specific approach using SISU-102 is novel and has not been extensively tested in clinical settings.

Where this research is happening

Cleveland, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.