Targeting a vulnerable splicing process to help neuroblastoma patients
Official title: Targeting splicing vulnerability of neuroblastoma
Funded at St. Jude Children's Research Hospital · NIH-11382134 · Data updated 2026-11-12
This grant aims to test a therapy that disrupts a cancer-specific splicing process to improve outcomes for children with high-risk neuroblastoma.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | St. Jude Children's Research Hospital NIH-funded |
| Lab location | 1 site (Memphis, United States) |
| Project start | August 1, 2025 |
| Project end | July 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $767,328 |
| NIH project number | 5R01CA303799-02 |
| Project ID | NIH-11382134 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children diagnosed with high-risk neuroblastoma who could participate in future clinical trials based on the preclinical results.
✕Less likely to help
Patients without neuroblastoma or those not matching the study's translational path may not benefit.
What this research studies
Neuroblastoma causes a significant share of childhood cancer deaths, and current treatments, while effective for some, have harsh long-term side effects. The project focuses on a spliceosome-related vulnerability in neuroblastoma cells and tests the idea that removing a key splicing factor (RBM39) with a small molecule (indisulam) could make the cancer more treatable. Researchers will examine combinations of indisulam with chemoimmunotherapy in preclinical neuroblastoma models to identify promising treatment strategies. The goal is to pave the way for a near-term clinical trial that could offer safer, more effective options for patients.
Why it matters
Potential benefit: If successful, this work could lead to safer, more effective therapies for children with high-risk neuroblastoma and improved long-term outcomes.
How similar studies have gone: Preclinical studies have shown that targeting splicing vulnerabilities can be effective in some cancers, with this approach now being explored for neuroblastoma.
Where this research is happening
-
St. Jude Children's Research Hospital
Memphis, United States Active
Researchers
- Principal investigator: Yang, Jun — St. Jude Children's Research Hospital
- Study coordinator: Yang, Jun
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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.