Stopping treatment-resistant pediatric leukemia caused by the NUP98‑NSD1 gene fusion
Overcoming Therapy Resistance in Fusion Oncoprotein Driven Pediatric Leukemia
Scientists are developing ways to understand and block chemotherapy resistance in children with AML driven by the NUP98‑NSD1 gene fusion.
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 ID | NIH-11162369 on NIH RePORTER |
What this research studies
The team is using human-derived blood stem cells and engineered lab models that carry the NUP98‑NSD1 fusion, with and without WT1 mutations, to mimic the leukemia seen in children. They compare how the fusion acts in prenatal fetal liver stem cells, neonatal cord blood cells, and adult bone marrow cells to uncover why the leukemia develops and resists treatment in young patients. By mapping the molecular pathways that allow the fusion protein to drive disease and resist therapy, researchers aim to identify specific weak points that drugs or other therapies could target. This work combines lab experiments with analysis of patient-derived samples to connect findings back to children who have this high-risk form of AML.
Who could benefit from this research
Good fit: Children (and young adults) diagnosed with acute myeloid leukemia that tests positive for the NUP98‑NSD1 fusion—particularly those with concurrent WT1 mutations—are the patient group most directly relevant to this work.
Not a fit: Patients with other AML subtypes that lack the NUP98‑NSD1 fusion or unrelated cancers are unlikely to benefit directly from findings focused on this specific fusion.
Why it matters
Potential benefit: If successful, this work could point to new treatments that overcome chemotherapy resistance and improve survival for children with NUP98‑NSD1–positive AML.
How similar studies have performed: Similar lab-based approaches have helped find targets in other fusion-driven leukemias, but targeting NUP98‑NSD1—especially in the context of WT1-linked resistance—is relatively novel and less tested clinically.
Where this research is happening
New York, United States
- 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
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 below.