Scientists Discover Potential Glioblastoma Treatment

UVA researchers identify molecule that blocks cancer-causing gene, offering hope for deadly brain tumor.

Feb. 4, 2026 at 8:23pm

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Researchers at the UVA Comprehensive Cancer Center have identified a small molecule that blocks the gene responsible for glioblastoma, the deadliest form of brain cancer. In lab tests and animal studies, the molecule proved effective at stopping the invasive cancer without harmful side effects. While more research is needed, the findings offer promise for a new treatment approach for this devastating disease.

Why it matters

Glioblastoma is an extremely aggressive and difficult-to-treat brain cancer, with a typical survival time of only 15 months after diagnosis. Current treatment options like surgery, chemotherapy, and radiation therapy provide limited benefits, so new therapeutic approaches are desperately needed to improve outcomes for glioblastoma patients.

The details

The researchers, led by Hui Li, PhD, previously discovered the "oncogene" or cancer-causing gene responsible for glioblastoma. In this latest study, they used high-throughput screening to identify a small molecule that blocks the activity of this gene, known as AVIL. In lab tests and animal models, the molecule was effective at destroying glioblastoma cells without harming healthy brain tissue. Importantly, the molecule can also cross the blood-brain barrier, a key challenge for many potential brain cancer treatments.

  • The research was published in Science Translational Medicine in February 2026.
  • Li first discovered the AVIL oncogene responsible for glioblastoma in 2020.

The players

Hui Li

A researcher at the UVA Comprehensive Cancer Center who previously discovered the AVIL oncogene responsible for glioblastoma and led the current study identifying a molecule that blocks this gene.

UVA Comprehensive Cancer Center

A National Cancer Institute-designated comprehensive cancer center at the University of Virginia that conducts cutting-edge cancer research and provides exceptional patient care.

AVIL Therapeutics

A company founded by Hui Li and Zhongqiu Xie to develop AVIL inhibitors as potential glioblastoma treatments.

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What they’re saying

“Glioblastoma is a devastating disease. Essentially no effective therapy exists. What's novel here is that we're targeting a protein that GBM cells uniquely depend on, and we can do it with a small molecule that has clear in vivo activity. To our knowledge, this pathway hasn't been therapeutically exploited before.”

— Hui Li, Researcher, UVA Comprehensive Cancer Center

“GBM patients desperately need better options. Standard therapy hasn't fundamentally changed in decades, and survival remains dismal. Our goal is to bring an entirely new mechanism of action into the clinic — one that targets a core vulnerability in glioblastoma biology.”

— Hui Li, Researcher, UVA Comprehensive Cancer Center

What’s next

The researchers will need to conduct further optimization and testing of the molecule to prepare it for clinical trials in human glioblastoma patients. If the compound continues to show promise, it would then undergo extensive testing in human volunteers before potentially being approved by the FDA as a new treatment.

The takeaway

This research represents a significant breakthrough in the search for more effective therapies for glioblastoma, one of the deadliest and most challenging forms of brain cancer. The identification of a small molecule that can selectively target the cancer-causing gene responsible for this disease offers hope that a new treatment approach may one day become available to improve outcomes for patients with this devastating diagnosis.