January 28, 2026
Scientists have identified a small molecule called compound A that shrinks glioblastoma brain tumors in mice by targeting advillin (AVIL), a protein found at abnormally high levels in this aggressive cancer. Because AVIL regulates the cell’s actin cytoskeleton and lacks obvious drug-binding sites, it was long considered an extremely difficult target, but screening efforts led to a compound that can bind AVIL, cross the blood–brain barrier, reduce tumor size even in temozolomide-resistant models, and show little toxicity. The researchers are now optimizing the molecule into an oral pill candidate and hope to begin human trials within the next couple of years, with potential relevance to other cancers as well.
Dr. Khasraw highlighted how notable it is to successfully target a cytoskeletal regulator and emphasized that compound A meets several rare translational benchmarks at the preclinical stage. He also cautions that resistance and long-term effectiveness still need further study.
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