Eltombopag: a new way to boost blood stem cells in aplastic anemia
Official title: Eltombopag: Novel Mode of Action on Normal and Aplastic Anemia Hematopoietic Stem Cells
Funded at Cleveland Clinic Lerner Com-Cwru · NIH-11364969 · Data updated 2026-11-12
A new drug approach aims to help people with idiopathic aplastic anemia by stimulating blood-forming stem cells through mechanisms that may not depend on traditional pathways.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Cleveland Clinic Lerner Com-Cwru NIH-funded |
| Lab location | 1 site (Cleveland, United States) |
| Project start | August 15, 2022 |
| Project end | May 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $658,812 |
| NIH project number | 5R01DK131923-05 |
| Project ID | NIH-11364969 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with idiopathic aplastic anemia or related marrow failure conditions who could benefit from therapies that stimulate hematopoietic stem cells.
✕Less likely to help
Patients whose anemia is not driven by stem cell deficiency or who cannot tolerate eltrombopag may not benefit.
What this research studies
This grant explores how eltrombopag can promote the production of blood cells in patients with aplastic anemia. Researchers will study both normal and diseased hematopoietic stem cells to understand how eltrombopag works beyond its known receptor interactions. The study includes laboratory experiments using human and mouse cells to uncover iron-related and epigenetic effects that could drive stem cell growth. The goal is to identify how this drug could restore multi-lineage blood formation in patients who do not fully respond to current therapies.
Why it matters
Potential benefit: If successful, the work could lead to more effective treatments that restore multiple blood cell types in patients with aplastic anemia.
How similar studies have gone: Previous work has shown eltrombopag can improve blood counts and tri-lineage hematopoiesis in some patients, supporting further investigation.
Where this research is happening
-
Cleveland Clinic Lerner Com-Cwru
Cleveland, United States Active
Researchers
- Principal investigator: Maciejewski, Jaroslaw P. — Cleveland Clinic Lerner Com-Cwru
- Study coordinator: Maciejewski, Jaroslaw P.
Related research
Other NIH-funded projects on the same topics.
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Emapalumab for children with aplastic anemia
Doctors will give emapalumab to children with acquired aplastic anemia to try to stop the immune attack on the bone marrow and help it make blood cells.
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Clinical and immunologic study of pediatric severe aplastic anemia to personalize care
A pediatric-focused research effort will collect and study immune and clinical data to tailor treatments for children with severe aplastic anemia.
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Aplastic Anemia Registry to Understand How Immune and Genetic Discoveries Affect Disease
A collaborative registry study to learn how genetic background and immune factors influence aplastic anemia and related bone marrow failure conditions in patients.
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Understanding how Eltrombopag helps with aplastic anemia
This research explores how the medication Eltrombopag helps patients with aplastic anemia by looking at its effects on blood-forming cells.
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Investigating a new treatment for pediatric aplastic anemia
This study is looking at how well a new treatment called emapalumab works for kids with acquired aplastic anemia, a rare blood disorder where the body stops making enough blood cells, to see if it can help them produce more blood cells and feel better.
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Comparing bone marrow transplantation with immune therapy for severe aplastic anemia in young patients
This study is looking at whether bone marrow transplants from unrelated donors or immune suppression therapy works better for kids and young adults with newly diagnosed severe aplastic anemia, especially since not everyone has a sibling who can donate bone marrow.
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.