A Promising Lead Against Glioblastoma
Hui Li, PhD, has identified a molecule that can block the gene responsible for glioblastoma, the deadliest brain cancer.
Our Hui Li, PhD, previously discovered the oncogene responsible for glioblastoma, the deadliest brain cancer, and now has identified a small molecule that may be able to block the gene's harmful effects.
The molecule successfully blocked the gene’s activity in both cell samples and lab mice. Promisingly, the molecule proved effective in the mice without harmful side effects.
Additional research will be needed before we could begin testing it in people, but it's a much-needed lead toward a new treatment for an aggressive cancer that is extremely difficult to treat. The cancer threads its way through the healthy tissue in the brain, making it extremely hard to remove with surgery. Right now, surgeons take out as much as possible, and then patients typically receive radiation and chemotherapy to help them survive as long as possible. Unfortunately, most will still die within a couple of years of diagnosis.
Li discovered the oncogene responsible for glioblastoma back in 2020. That gene, AVIL, helps cells maintain their size and shape, but it can be shifted into harmful overdrive by a variety of factors, he found.
Li and his colleagues found that blocking the gene’s activity could completely destroy glioblastoma cells in mice, without any effect on healthy cells. But the lab technique they used is unsuitable for people. That put the researchers on the hunt for a molecule that could stop the gene’s harmful effects.
The molecule they have found appears to affect only tumor cells, sparing healthy brain tissue. Further, the molecule can cross the brain’s natural protective barrier that keeps out toxins but also blocks many potential treatments for neurological diseases.
Professor Li envisions that the molecule could be packaged as a medicine patients could take orally, assuming it is successful in the extensive testing needed before it could be approved by the federal Food and Drug Administration..
“Glioblastoma is a devastating disease. Essentially no effective therapy exists,” Professor Li told me. “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.”