WIKIMEDIA, RAMAResearchers have found that a drug called Vacquinol-1 causes cancer cells to burst open, an achievement that represents “an entirely new mechanism” of killing cancer, according to the authors of the study, published in Cell this week (March 20). The drug prompted cells to over-develop vacuoles, become perfectly round, break apart, and die. “They have shown very exciting results,” said Ravi Bellamkonda, a neuroscientist at the Georgia Institute of Technology, in an e-mail to The Verge.
Mice with glioblastoma—a tough-to-treat brain cancer in humans—that were given Vacquinol-1 orally for five days ended up with either smaller tumors than control mice or no tumors at all. Mice that were treated with the drug also lived longer than control mice; only two of eight mice given Vacquinol-1 died over the course of 80 days, whereas the median survival of the eight control mice was only 31 days.
The results are enticing, given that the average survival time for a human with glioblastoma is only 15 months. “We now want to try to take this discovery in basic research through preclinical development and all the way to the clinic,” Patrik Ernfors, a coauthor of the study and a researcher at the Karolinska Institute, said in a press release. “The goal is to get into a phase 1 trial.”
Bellamkonda pointed out that the levels of Vacquinol-1 used were “relatively high” and it's unclear whether side effects might emerge. “This said, enhancing survival by several fold in aggressive tumor models is encouraging,” he told The Verge. “I'd love to see more studies.”