A temporary metabolic shutdown to help kill AML cells
Creating a transient metabolic catastrophe for AML therapy
['FUNDING_R01'] · CASE WESTERN RESERVE UNIVERSITY · NIH-11300980
This project will try turning on a metabolism switch together with proteasome-blocking drugs to make acute myeloid leukemia (AML) cells more likely to die.
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-11300980 on ClinicalTrials.gov |
What this research studies
Researchers are combining a new small molecule that activates mTORC1 with proteasome inhibitors to stress and damage leukemia cell mitochondria and reverse cell dormancy. Experiments will be done in lab-grown AML cells and in animal models to track mitochondrial damage, loss of membrane potential, and how dormant cells re-enter the cell cycle. The team will study why this combination forces cancer cells into a short-lived metabolic collapse and whether that makes standard chemotherapy more effective. Findings will guide whether this approach could move toward human clinical testing.
Who could benefit from this research
Good fit: People with acute myeloid leukemia, especially those with relapsed or treatment-resistant disease or evidence of dormant AML cells, would be the likely candidates for future clinical testing.
Not a fit: Patients with cancers other than AML or those who cannot tolerate proteasome inhibitors or drugs that affect mTOR signaling are unlikely to benefit from this approach.
Why it matters
Potential benefit: If successful, this approach could make existing drugs kill more AML cells and help overcome treatment resistance.
How similar studies have performed: Proteasome inhibitors have proven very effective in multiple myeloma but not broadly in AML, and early lab studies suggest combining mTORC1 activation with proteasome inhibition can kill AML cells, though clinical testing is still novel.
Where this research is happening
CLEVELAND, UNITED STATES
- CASE WESTERN RESERVE UNIVERSITY — CLEVELAND, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: TIROSH, BOAZ — CASE WESTERN RESERVE UNIVERSITY
- Study coordinator: TIROSH, BOAZ
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.