Arsenic trioxide has potential to be a powerful therapy that could extend the lives of certain glioblastoma patients by as much as three to four times the median expectation, according to research published yesterday in Molecular Cancer Research.
For years arsenic trioxide has been used to treat acute promyelocytic leukemia. Now a study led by Northwestern University Feinberg School of Medicine and the Translational Genomics Research Institute (TGen) shows this anti-cancer agent could also be effective in treating glioblastoma multiforme (GBM), the most common and aggressive type of deadly brain tumors.
The team screened a panel of 650 small molecules against patient-derived GBM cells to discover compounds targeting specific GBM subtypes. They discovered that a specific subtype of GBM cells were more responsive to the arsenic trioxide treatment.
The next step will be to validate the findings by initiating a new clinical trial specifically designed to match arsenic trioxide with glioblastoma patients that have a specific genomic signature, according to Dr. Michael Berens, a TGen deputy director and professor in TGen's cancer and cell biology division, and one of the study's authors. These patients also would receive TMZ, which is the current standard-of-care drug given to GBM patients.
"Rather than treat all patients, we want to design a prospective clinical trial that we can enrich for those patients whose genomic signatures indicate they would be the best candidates for success," said Dr. Harshil Dhruv, an assistant professor at TGEN and one of the study's authors. "This would be a biomarker-driven, precision-medicine clinical trial for glioblastoma, a way to match the right drug to the right patient."
According to the researchers, there are additional advantages of using arsenic trioxide. First, it is a small molecule able to penetrate the blood-brain barrier. This barrier that prevents most other anti-cancer drugs from attacking brain tumors. Second, its costs are minimal, since its source material, arsenic, is abundant in nature.