Summary of UVA health System News Release: Potential New Glioblastoma Treatment Discovered
Key Takeaways:
* New Target Identified: Researchers at the University of Virginia (UVA) have identified the AVIL gene as a key driver of glioblastoma, an aggressive and deadly brain cancer. AVIL normally maintains cell shape, but becomes overactive in glioblastoma, promoting cancer cell growth and spread.
* Promising Compound Found: the team has discovered a molecule that effectively blocks AVIL activity, selectively targeting tumor cells while sparing healthy brain tissue. Crucially, this molecule can cross the blood-brain barrier, a major hurdle for neurological treatments.
* Potential for Oral Medication: The compound is designed to be taken orally, offering a convenient delivery method.
* Early Stage, But Significant Hope: While still in the early stages of development, this research offers a new mechanism of action for treating glioblastoma, a disease where standard therapies have remained largely unchanged for decades and survival rates are low.
* Company Formed for Development: lead researcher Dr.Li has founded AVIL Therapeutics to further develop AVIL inhibitors.
Details:
* Previous Research: Dr. li’s team previously identified AVIL as an oncogene in glioblastoma in 2020. Initial lab tests showed blocking the gene could destroy cancer cells, but the method used wasn’t suitable for human application.
* High-Throughput Screening: Researchers used high-throughput screening to identify compounds that could block AVIL activity.
* Validation of AVIL Role: The research confirmed that the protein produced by AVIL is rarely found in healthy brains but is abundant in glioblastoma patients.
* Future Steps: Extensive research, clinical trials, and FDA approval are required before the compound can become a treatment option for patients.
Funding & Affiliations:
* Supported by the National Institutes of Health and the Ben & Catherine Ivy Foundation.
* Research conducted at UVA Comprehensive Cancer Center and UVA’s Paul and Diane Manning Institute of Biotechnology.
Source: University of Virginia Health System (https://www.uvahealth.com/)
Journal Reference: DOI: 10.1126/scitranslmed.adt1211