Testing new drugs using patient-derived pediatric brain tumor models

In vivo Drug Testing of Pediatric CNS Tumors Using Patient Derived Orthotopic Xenograft Models

['FUNDING_U01'] · LURIE CHILDREN'S HOSPITAL OF CHICAGO · NIH-11331847

Researchers are using tumor tissue from children with brain tumors to test which drugs may work best.

Quick facts

Phase['FUNDING_U01']
Study typeNih_funding
SexAll
SponsorLURIE CHILDREN'S HOSPITAL OF CHICAGO (nih funded)
Locations1 site (CHICAGO, UNITED STATES)
Trial IDNIH-11331847 on ClinicalTrials.gov

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.

Who could benefit from this research

Good fit: 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.

Not a fit: 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.

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 performed: 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

CHICAGO, UNITED STATES

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.

View on NIH RePORTER →

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.