Testing new drugs using patient-derived pediatric brain tumor models
Official title: In vivo Drug Testing of Pediatric CNS Tumors Using Patient Derived Orthotopic Xenograft Models
Funded at Lurie Children's Hospital of Chicago · NIH-11331847 · Data updated 2026-11-12
Researchers are using tumor tissue from children with brain tumors to test which drugs may work best.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Lurie Children's Hospital of Chicago NIH-funded |
| Lab location | 1 site (Chicago, United States) |
| Project start | July 14, 2015 |
| Project end | June 30, 2026 |
| Funding year | 2025 |
| Award (this year) | $237,825 |
| NIH project number | 3U01CA199288-10S1 |
| Project ID | NIH-11331847 on NIH RePORTER |
Who could benefit from this research
✓Could help
Children with pediatric brain tumors who can donate tumor tissue during surgery, at relapse, or at autopsy would be ideal candidates to contribute to this program.
✕Less likely to help
Children who cannot provide tumor tissue or people with non-pediatric or non-brain cancers would not be eligible and are unlikely to benefit directly.
What this research studies
Tumor samples from children are implanted into mouse brains so the tumors keep the same appearance and genetic changes as the original patient tumors. The team maintains a panel of about 150 patient-derived orthotopic xenograft (PDOX) models that represent many pediatric brain tumor types and clinical stages. Candidate cancer drugs are given to those mouse models to see which drugs slow or shrink the tumors, and tumor cells are cryopreserved for future testing. The work aims to prioritize the most promising drug candidates to guide later clinical trials.
Why it matters
Potential benefit: If successful, this could speed up finding treatments that are more likely to help children with brain tumors.
How similar studies have gone: Patient-derived xenograft models have been widely used to mirror human tumor biology and prioritize drugs in preclinical testing, though translating those leads into confirmed clinical cures has been inconsistent.
Where this research is happening
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Lurie Children's Hospital of Chicago
Chicago, United States Active
Researchers
- Principal investigator: Li, Xiaonan — Lurie Children's Hospital of Chicago
- Study coordinator: Li, Xiaonan
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.