Finding ways to overcome treatment resistance in a type of childhood leukemia
Official title: Overcoming Therapy Resistance in Fusion Oncoprotein Driven Pediatric Leukemia
Funded at Icahn School of Medicine at Mount Sinai · NIH-10942305 · Data updated 2026-11-12
This study is looking into why some kids with acute myeloid leukemia (AML) don’t respond to regular treatments, focusing on specific genetic changes that might be causing this resistance, so they can find better ways to help those kids fight the disease.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Icahn School of Medicine at Mount Sinai NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | August 6, 2024 |
| Project end | July 31, 2029 |
| Funding year | 2024 |
| Award (this year) | $441,178 |
| NIH project number | 1R01CA292503-01 |
| Project ID | NIH-10942305 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include pediatric patients diagnosed with NUP98-NSD1 fusion-positive acute myeloid leukemia.
✕Less likely to help
Patients with other types of leukemia or those without the specific genetic alterations being studied may not benefit from this research.
What this research studies
This research focuses on understanding why certain genetic changes in pediatric acute myeloid leukemia (AML) lead to resistance against standard therapies. By studying a specific fusion oncoprotein, NUP98-NSD1, and its interaction with mutations in the WT1 gene, the researchers aim to uncover the mechanisms behind treatment failure. They have developed a humanized model to investigate how these genetic alterations affect blood stem cells from different developmental stages. The goal is to identify new therapeutic strategies that can effectively target these resistant leukemia cells.
Why it matters
Potential benefit: If successful, this research could lead to more effective treatments for children with therapy-resistant leukemia, improving survival rates.
How similar studies have gone: While research on leukemia treatment resistance is ongoing, this specific approach using the NUP98-NSD1 fusion is novel and has not been extensively tested in prior studies.
Where this research is happening
-
Icahn School of Medicine at Mount Sinai
New York, United States Active
Researchers
- Principal investigator: Wagenblast, Elvin — Icahn School of Medicine at Mount Sinai
- Study coordinator: Wagenblast, Elvin
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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.