Targeted treatment for inv(16) acute myeloid leukemia (CBFβ‑SMMHC)
Official title: Therapeutic targeting of an oncogenic translational program in AML
Funded at Versiti Blood Health, INC. · NIH-11300259 · Data updated 2026-11-12
Using a targeted drug combination to kill leukemia cells in adults with inv(16) acute myeloid leukemia.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Versiti Blood Health, INC. NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project start | April 1, 2025 |
| Project end | March 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $545,170 |
| NIH project number | 5R01CA293588-02 |
| Project ID | NIH-11300259 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults (21+) with inv(16) acute myeloid leukemia, especially those whose leukemia carries the CBFβ‑SMMHC fusion or who have relapsed after standard treatments, would be the ideal candidates.
✕Less likely to help
Patients without the inv(16) genetic change, pediatric patients, or those medically unable to receive experimental therapies are unlikely to benefit from this targeted approach.
What this research studies
This work focuses on a specific genetic form of adult AML driven by the CBFβ‑SMMHC fusion (inv(16)). Researchers are developing a targeted drug called AI‑10‑49 that disrupts the cancer driver and have found it causes leukemia cell death in patient samples and improves survival in mouse models. They discovered that a protein called eIF4G1 can restore cancer cell survival and that a second drug, SBI‑756, blocks eIF4G1 to overcome resistance. The team plans to test combining AI‑10‑49 with eIF4G1 inhibition in laboratory models using human AML cells to support future patient-directed trials.
Why it matters
Potential benefit: If successful, this could lead to a new targeted therapy combination that reduces relapse and improves outcomes for patients with inv(16) AML.
How similar studies have gone: Preclinical studies show AI‑10‑49 kills primary inv(16) human AML cells and improves survival in mouse models, and lab data indicate SBI‑756 can overcome resistance, making the combined approach promising but still experimental.
Where this research is happening
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Versiti Blood Health, INC.
Milwaukee, United States Active
Researchers
- Principal investigator: Pulikkan, John — Versiti Blood Health, INC.
- Study coordinator: Pulikkan, John
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.