Targeting Rac1 to treat medulloblastoma in children
Official title: Rac1 Inhibition for the treatment of medulloblastoma
Funded at Georgetown University · NIH-10995293 · Data updated 2026-11-12
This study is testing a new drug called GYS32661 to see if it can help treat medulloblastoma, a type of brain cancer in kids, by blocking a protein that helps cancer cells grow.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Georgetown University NIH-funded |
| Lab location | 1 site (Washington, United States) |
| Project start | December 6, 2023 |
| Project end | November 30, 2025 |
| Funding year | 2025 |
| Award (this year) | $195,000 |
| NIH project number | 5R21NS135506-02 |
| Project ID | NIH-10995293 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are children diagnosed with medulloblastoma, particularly those who have not responded well to traditional therapies.
✕Less likely to help
Patients with other types of brain tumors or those who are not diagnosed with medulloblastoma may not benefit from this research.
What this research studies
This research investigates a new treatment approach for medulloblastoma, a type of brain cancer that primarily affects children. The study focuses on inhibiting a protein called Rac1, which is involved in cancer cell growth and signaling. Researchers will use a specially developed drug, GYS32661, to see if it can reduce the activity of key signaling pathways that contribute to tumor growth. The research includes both laboratory tests and animal models to evaluate the effectiveness of this treatment.
Why it matters
Potential benefit: If successful, this research could lead to a novel therapy that improves outcomes for children with medulloblastoma and reduces the side effects associated with current treatments.
How similar studies have gone: While targeting Rac1 in cancer treatment is a novel approach, there have been successful studies targeting similar pathways in other cancers, suggesting potential for success in this area.
Where this research is happening
-
Georgetown University
Washington, United States Active
Researchers
- Principal investigator: Ayad, Nagi G — Georgetown University
- Study coordinator: Ayad, Nagi 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.