Screening method reveals glioblastoma drug target

Northwestern Now: Summaries

Craig Horbinski, MD, PhD, associate professor of Pathology and Neurological Surgery, was a co-author of the Nature paper.
A team of scientists has developed a new method for identifying drug targets in the aggressive brain tumor glioblastoma and discovered that a key gene — previously overlooked by traditional approaches — may represent a promising new target for therapy.
The study, published in the journal Nature, was co-authored by Craig Horbinski, MD, PhD, associate professor of Pathology and Neurological Surgery.
Glioblastoma, the most common form of adult brain tumor, is a lethal cancer with a median survival of just 15 months. There is no cure, and current treatment options tend to extend patients’ lifespan only modestly.
Previously, most laboratory research in the area has focused on in vitro cellular models. But drug therapies based on targets discovered in vitro have had limited clinical success for glioblastoma patients, in part because such cell models poorly match how the cancer behaves in humans.
“What works against cancer cells in a dish doesn’t necessarily work when the same cells are grown in mice. Something that works very well in mice is more likely to work in patients, but it is far too expensive and laborious to test every new drug in mice without first testing it in a dish,” Horbinski said. “It’s very frustrating for everyone, including scientists, doctors and especially patients.”
In the current study, Horbinski and collaborators demonstrated a new, more efficient method to screen for potential drug targets in glioblastoma.
The team developed an in vivo functional screening strategy — using models of mice implanted with human glioblastoma cells, which closely mirrored the natural tumor environment —  to identify changes in cancer gene regulation. They then compared those results with screening results from cells in a dish. The scientists discovered that many of the genes necessary for cancer cell growth in the brain were ...

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