Targeting complex mutations in leukemia treatment
Official title: Strategies to target BCR-ABL1 compound mutants in https://apps.era.nih.gov/facts/fcdataGrants/viewFCData.era?applId=11005218#app-addressesCML and Ph+ ALL
Funded at Versiti Wisconsin, INC. · NIH-11005218 · Data updated 2026-11-12
This study is looking at a new drug called asciminib to see if it can help people with a tough type of leukemia that has not responded to standard treatments, especially those with multiple gene mutations.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Versiti Wisconsin, INC. NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project start | November 10, 2021 |
| Project end | December 31, 2025 |
| Funding year | 2025 |
| Award (this year) | $339,694 |
| NIH project number | 5R01CA257602-05 |
| Project ID | NIH-11005218 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include patients with Philadelphia chromosome-positive leukemia who have compound mutations in the BCR-ABL1 gene and have not responded to standard TKIs.
✕Less likely to help
Patients without Philadelphia chromosome-positive leukemia or those who have not developed resistance to current TKI therapies may not benefit from this research.
What this research studies
This research focuses on developing new strategies to treat Philadelphia chromosome-positive leukemia, particularly in patients with compound mutations in the BCR-ABL1 gene. Current treatments using tyrosine kinase inhibitors (TKIs) are effective for some patients but fail for those with multiple mutations. The study aims to explore the effectiveness of a novel drug, asciminib, which targets a different site on the BCR-ABL1 protein, potentially offering new hope for patients who have not responded to existing therapies. By understanding the mutational profiles and resistance mechanisms, the research seeks to improve treatment options for these challenging cases.
Why it matters
Potential benefit: If successful, this research could provide new treatment options for patients with resistant forms of leukemia, improving their chances of survival.
How similar studies have gone: While there has been significant progress in targeting single mutations in leukemia, the approach to targeting compound mutations is relatively novel and has not been extensively tested in clinical settings.
Where this research is happening
-
Versiti Wisconsin, INC.
Milwaukee, United States Active
Researchers
- Principal investigator: Deininger, Michael W. — Versiti Wisconsin, INC.
- Study coordinator: Deininger, Michael W.
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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.