Experimental Brain Cancer Drug Shows Potential

An experimental brain cancer drug has shown potential for treating patients with an aggressive form of the disease. Researchers screened over 1,000 different molecules in order to find the Vacquinol-1 compound. The compound has been used to treat patients with glioblasoma multiforme, or GBM. GBM is the most aggressive form of brain cancer, with an average patient life expectancy of only 15 months. GBM is extremely lethal, as only five percent of patients dignosed survive more than three years.

Researchers tested the Vacquinol-1 compound on mice to determine how it would affect GBM brain cancer tumors. The compound works by stopping the cell’s ability to control what goes in and out of cells. While normal brain cells are unaffected, GBM tumor cells eventually fill with water and other materials. Once at full capacity, the cells explode. Researchers did not observe negative side effects in mice. The researchd demonstrated that mice receiving treatment lived significantly longer than others without treatment.

Ravi Bellamkonda, a neuroscientist at the Georgia Institute of Technology, called the results “very exciting.” He was cautious, as the drug had not yet been tested on humans. He was also concenred about the reported high levels of the drug that were needed in order to make treatment effective. Treatment at that level may cause negative side effects in humans. Still, Bellamkonda’s view on the project is a promising one. “This said, enhancing survival by several fold in aggressive tumor models is encouraging. I'd love to see more studies."