Scientists from the Karolinska Institute in Stockholm, Sweden, have discovered a new molecule which tackled a particular type of brain cancer cell in an entirely new manner; by causing them to "explode". The encouraging results have been published in the journal Cell.
Scientists first exposed cancer cells derived from a particular type of brain cancer, a glioblastoma, to over 200 different potential molecules, looking for any that may have had adverse effects on the cells. Glioblastoma, or more specifically glioblastoma multiforme, is the most aggressive form of brain cancer known, and the life expectancy even with treatment is exceedingly poor. Essentially, it is incurable. Of the 200 tested on these cancer cells, only one molecule turned out to be of any interest to the researchers.
This molecule, named Vacquinol-1, acted in an intriguing and novel way on the cancer cells. It induced a process called vacuolization to occur in a rather dramatic manner. Vacuolization is where substances are taken up from the outside of the cell by deformation of the cell membrane into pockets inside the cell called vacuoles. The cells went into over drive, and the huge accumulation of vacuoles meant that the cell membrane eventually collapsed, causing the cell to essentially explode all of its contents and die.
Of course, it's all well and good putting a substance onto cells in a cell culture dish and saying "Eureka! It's killed them!". Killing cells in a dish is easy, bleach will do the trick quite nicely. So the researchers turned to mice with glioblastomas to see how they would fare with or without this substance. The mice that did not receive the treatment had a median survival rate of roughly 30 days, whereas 6 out of the 8 mice on treatment lived longer than 80 days. The drug therefore conveyed a significant survival advantage in this animal model.