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Preclinical Development of Atr Inhibitor VE-822, Delivered Systemically in Nanoparticles, for Medulloblastoma Therapy

University of North Carolina
Timothy Gershon, MD, PhD
Grant Type: 
Innovation Grants
Year Awarded: 
Type of Childhood Cancer: 
Project Description: 


Cancer is the second leading cause of death in children aged 5-14, and medulloblastoma is the most common malignant brain tumor of childhood. Current treatments for medulloblastoma damage both cancer cells and normal cells, and this can have lasting consequences on a child's brain development. We have developed a promising new method to treat medulloblastoma. Our approach utilizes a drug called VE-822, which targets a protein (called ATR) that medulloblastoma cells need to survive. Through a multi-disciplinary collaboration with the UNC Eshelman School of Pharmacy, we have packaged VE-822 into tiny structures called nanoparticles that can pass into brain tumors. Our early mouse studies show that this nanoparticle VE-822, called pVE-822, gets into medulloblastomas in mice and slows tumor growth. VE-822 caused DNA damage and cell death in tumors, while sparing normal brain. Our novel agent, pVE-822 may produce fewer side effects than typical chemotherapy because it more specifically targets cancer cells.

Project Goal:

In this project, we will study:

1) the best way to dose pVE-822 for medulloblastoma using a mouse model,

2) how pVE-822 destroys tumor cells, and

3) how pVE-822 can be combined with existing medulloblastoma therapies.

Results from these studies will provide a novel way to more effectively and safely treat children with medulloblastoma.

Co-funded by: 
Cure4Cam Childhood Cancer Foundation
I Care I Cure