Lipid-based therapy targeting multiple growth pathways in pediatric brain cancer
Official title: Lipid nanomedicine targeting multiple signaling pathways of medulloblastoma
Funded at University of Nebraska Medical Center · NIH-11333316 · Data updated 2026-11-12
Aimed at exploring a lipid-based medicine to slow or stop growth of medulloblastoma, a common childhood brain tumor.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Nebraska Medical Center NIH-funded |
| Lab location | 1 site (Omaha, United States) |
| Project start | July 15, 2022 |
| Project end | May 31, 2027 |
| Funding year | 2026 |
| Award (this year) | $475,579 |
| NIH project number | 5R01NS128336-05 |
| Project ID | NIH-11333316 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with medulloblastoma or related pediatric brain tumors could participate in future translational steps once a safe clinical pathway is established.
✕Less likely to help
Early-stage preclinical research may not yet translate into immediate patient benefits.
What this research studies
This project focuses on medulloblastoma, a brain tumor of children, and looks at how a specially designed lipid nanomedicine can hit several growth pathways at once. Researchers will study how the therapy interacts with key signals like PI3K/AKT and BRD4, and how it affects tumor cells and cancer stem cells. The team will address challenges such as drug delivery across the blood-brain barrier and potential neurotoxicity, with structural studies to understand how the compound binds to target proteins. Preclinical experiments will guide future steps toward making a treatment more effective and less harmful for patients.
Why it matters
Potential benefit: If successful, the approach could lead to a new treatment option that more effectively slows tumor growth while reducing side effects.
How similar studies have gone: Similar dual inhibitors have shown activity in MB models, but this work advances toward a multi-pathway targeting strategy with brain delivery.
Where this research is happening
-
University of Nebraska Medical Center
Omaha, United States Active
Researchers
- Principal investigator: Mahato, Ram I. — University of Nebraska Medical Center
- Study coordinator: Mahato, Ram I.
Related research
Other NIH-funded projects on the same topics.
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Glial origins and signaling in childhood brain cancer (medulloblastoma)
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Targeting medulloblastoma with multispecific T-cell engagers
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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.
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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.