New approach to target the stem cells that drive acute myeloid leukemia
Targeting leukemic stem cells in acute myeloid leukemia
This project aims to make the leukemia stem cells that cause relapses in people with acute myeloid leukemia more vulnerable by targeting a mitochondrial enzyme.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Emory University NIH-funded |
| Lab location | 1 site (Atlanta, United States) |
| Project ID | NIH-11251191 on NIH RePORTER |
What this research studies
This work focuses on the small population of leukemia stem cells (LSCs) that can survive treatment and cause relapses. Researchers will manipulate a mitochondrial enzyme called PTPMT1 in laboratory cells and animal models to create energy stress that may force LSCs to stop self-renewing and begin to differentiate. They will test this approach in models of AML driven by mutations such as FLT3-ITD, MLL-AF9, and PTEN loss to see if it blocks disease growth. The goal is to identify a vulnerability that could be turned into a therapy to reduce relapse risk.
Who could benefit from this research
Good fit: People with acute myeloid leukemia—especially those with subtypes driven by FLT3-ITD, MLL rearrangements, or PTEN loss—would be the type of patients most likely to benefit from future therapies derived from this work.
Not a fit: Because this is preclinical lab and animal research, patients needing immediate treatment or those with non-AML cancers are unlikely to receive direct benefit from the grant alone.
Why it matters
Potential benefit: If successful, this work could lead to therapies that better eliminate the leukemia stem cells responsible for relapse and improve long-term remission in AML patients.
How similar studies have performed: Related preclinical studies targeting leukemia stem cell metabolism have shown promise in lab and animal models, but clinical translation to effective patient treatments remains limited so far.
Where this research is happening
Atlanta, United States
- Emory University — Atlanta, United States (Active)
Researchers
- Principal investigator: Qu, Cheng-Kui — Emory University
- Study coordinator: Qu, Cheng-Kui
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.