Efflux-aware brain barrier model to guide pediatric medulloblastoma drug discovery
Official title: Efflux-Aware Human BBB Assembloid Assay for Brain-Penetrant Therapeutic Discovery in Medulloblastoma
Funded at Mayo Clinic Rochester · NIH-11443917 · Data updated 2026-11-12
A study using a pediatric brain cancer model to test how radiation-activated digoxin-like compounds could safely reach brain tumors in children.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mayo Clinic Rochester NIH-funded |
| Lab location | 1 site (Rochester, United States) |
| Project start | September 15, 2026 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $500,986 |
| NIH project number | 1R61NS150150-01 |
| Project ID | NIH-11443917 on NIH RePORTER |
Who could benefit from this research
✓Could help
Pediatric patients and samples that model medulloblastoma biology could benefit from advances in brain drug delivery demonstrated by this work.
✕Less likely to help
Direct patient benefit may be limited until later-stage testing and clinical trials.
What this research studies
This project uses patient-derived iPSC brain and blood vessel organoids that mimic the human blood-brain barrier to study how radiotherapy can trigger the release of digoxin-like drugs inside brain tumors. The team will synthesize two pro-drug chemotypes and develop a method to measure intact pro-drug, released drug, and breakdown products in samples. By focusing on medulloblastoma in children, the work aims to improve drug delivery to tumors while limiting systemic exposure. The effort combines chemistry, radiotherapy timing, and organoid biology to model how a treatment could work in kids.
Why it matters
Potential benefit: If successful, it could lead to more effective and safer brain-targeted therapies for children with medulloblastoma.
How similar studies have gone: Similar organoid and pro-drug strategies have shown promise in preclinical models but require further validation in human-relevant systems.
Where this research is happening
-
Mayo Clinic Rochester
Rochester, United States Active
Researchers
- Principal investigator: Huang, Frank — Mayo Clinic Rochester
- Study coordinator: Huang, Frank
Related research
Other NIH-funded projects on the same topics.
-
Glial origins and signaling in childhood brain cancer (medulloblastoma)
A new study looks at how glial cells may drive relapse and growth in childhood medulloblastoma, with the goal of finding targeted treatments.
-
Next generation GPC2 CARs to treat pediatric medulloblastoma
A new CAR T-cell therapy approach targets a protein common on child brain tumor cells to improve outcomes for kids with medulloblastoma.
-
Targeting medulloblastoma with multispecific T-cell engagers
A pediatric brain cancer treatment study tests a new multi-targeted T-cell engager designed to reach brain tumors and help the immune system kill cancer cells.
-
Overcoming medulloblastoma resistance in kids using in vivo perturb-seq and preclinical therapies
A patient-focused study aiming to find new, less toxic ways to treat pediatric medulloblastoma by using advanced genetic screening to identify effective drug targets in preclinical models.
-
Dysregulation of a key gene complex and its role in Group 4 medulloblastoma
A study explores how disruption of a core gene complex in brain development may drive Group 4 medulloblastoma and seeks to understand new ways to help affected children.
-
Targeting MBD2 for medulloblastoma therapy
Aimed at developing brain-penetrant small molecules to treat medulloblastoma in children by reactivating a tumor-suppressing gene.
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.