Targeting MBD2 for medulloblastoma therapy
Funded at University of Alabama at Birmingham · NIH-11412727 · Data updated 2026-11-12
Aimed at developing brain-penetrant small molecules to treat medulloblastoma in children by reactivating a tumor-suppressing gene.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Alabama at Birmingham NIH-funded |
| Lab location | 1 site (Birmingham, United States) |
| Project start | September 9, 2025 |
| Project end | August 31, 2030 |
| Funding year | 2026 |
| Award (this year) | $564,200 |
| NIH project number | 5R01NS144449-02 |
| Project ID | NIH-11412727 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children diagnosed with medulloblastoma, across molecular subgroups, who could benefit from targeted epigenetic therapy.
✕Less likely to help
Patients whose tumors do not rely on the ADGRB1/MBD2 axis or who cannot tolerate small-molecule brain-penetrant therapies may receive little or no benefit.
What this research studies
Medulloblastoma is the most common malignant brain tumor in children and current treatments can cause lasting damage. This project focuses on designing and optimizing small molecules that inhibit MBD2 to reactivate the ADGRB1 (BAI1) tumor-suppressor pathway in MB cells, with the goal of moving a drug candidate toward clinical use. The work includes chemistry optimization, testing in brain-relevant models, and evaluating synergy with radiotherapy to improve outcomes. The team has already shown that a lead compound can reach the brain, extend survival in mouse MB models, and tolerate the animals well. If successful, this approach could offer a targeted, less toxic option for pediatric MB patients.
Why it matters
Potential benefit: It could provide a less toxic, brain-targeted therapy that improves survival and reduces long-term disabilities for children with medulloblastoma.
How similar studies have gone: Previous preclinical work showed that MBD2 inhibitors can reactivate tumor-suppressor genes and extend survival in MB models, supporting further development.
Where this research is happening
-
University of Alabama at Birmingham
Birmingham, United States Active
Researchers
- Principal investigator: Van Meir, Erwin G. — University of Alabama at Birmingham
- Study coordinator: Van Meir, Erwin G.
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.
-
Efflux-aware brain barrier model to guide pediatric medulloblastoma drug discovery
A study using a pediatric brain cancer model to test how radiation-activated digoxin-like compounds could safely reach brain tumors in children.
-
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.
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.