Creating new treatments for acute leukemia by targeting specific genes
Official title: Development of ASH1L inhibitors for acute leukemia
Funded at University of Michigan at Ann Arbor · NIH-10740903 · Data updated 2026-11-12
This study is looking at a new way to help people with acute myeloid leukemia (AML) by testing a treatment that targets a specific protein to lower the activity of certain genes linked to the disease, and patients may have the chance to participate in trials to see how well this treatment works.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Michigan at Ann Arbor NIH-funded |
| Lab location | 1 site (Ann Arbor, United States) |
| Project start | December 1, 2019 |
| Project end | November 30, 2025 |
| Funding year | 2024 |
| Award (this year) | $544,236 |
| NIH project number | 5R01CA244254-05 |
| Project ID | NIH-10740903 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are patients diagnosed with acute myeloid leukemia (AML) who have high levels of HOX gene expression.
✕Less likely to help
Patients with other types of leukemia or those who do not have high HOX gene expression may not benefit from this research.
What this research studies
This research focuses on developing inhibitors that target the ASH1L protein, which plays a crucial role in the expression of HOX genes linked to acute leukemia. By reducing the expression of these genes, the research aims to create a novel treatment strategy for patients suffering from acute myeloid leukemia (AML), a condition with a low survival rate. The approach involves using small molecules to inhibit ASH1L, which has shown promise in preclinical studies. Patients may be involved in clinical trials to test the effectiveness and safety of these new treatments.
Why it matters
Potential benefit: If successful, this research could lead to more effective therapies for acute leukemia, potentially improving survival rates and patient outcomes.
How similar studies have gone: Previous research has shown promise in targeting HOX gene dysregulation in leukemia, suggesting that this approach could be a viable treatment strategy.
Where this research is happening
-
University of Michigan at Ann Arbor
Ann Arbor, United States Active
Researchers
- Principal investigator: Grembecka, Jolanta — University of Michigan at Ann Arbor
- Study coordinator: Grembecka, Jolanta
Related research
Other NIH-funded projects on the same topics.
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Investigating a specific type of leukemia in children caused by genetic changes.
This study is looking at how certain proteins linked to leukemia affect kids and teens with high-risk blood cancers, and the researchers hope to find new ways to treat these conditions by learning more about how the disease works.
-
Reducing leukemia disparities in Latino children and teens
A large, multi-project effort aims to understand why Latino children with acute lymphoblastic leukemia have worse outcomes and how to reduce those gaps.
-
Genomic advances to improve outcomes in high-risk acute leukemia
A physician-scientist team is studying the genetic drivers of pediatric acute leukemia to find new ways to diagnose and treat it, with the goal of improving cure rates for kids.
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Understanding and Targeting Extramedullary Acute Leukemia
A clinician-research team is studying myeloid sarcoma, a form of AML that forms solid tumors outside the bone marrow, to develop new treatments for patients.
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Diversifying acute leukemia trial enrollment through multilevel approach
Aiming to broaden participation in acute leukemia trials by testing a multi-layer plan that addresses barriers for adults with AML.
-
Blocking menin to treat acute leukemias with high HOX gene activity
This work develops new drugs that block a protein called menin to help people with certain aggressive acute leukemias driven by HOX gene activation.
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.