Swedish researchers have discovered a substance that makes brain tumors explode. They said the molecule —called Vacquinol-1 — could be developed into a medicine that might provide an entirely new way to treat glioblastoma, the most aggressive type of brain cancer.
In findings published in the journal Cell, researchers from the Karolinska Institutet said they treated laboratory mice engineered to develop brain cancer with the substance, which can be administered in tablet form, and found Vacquinol-1 halted their tumor growth and the animals lived longer than expected.
Current treatments for the brain cancer include surgery, radiation, and chemotherapy, but the average glioblastoma patient lives just 15 months after diagnosis — even with such aggressive therapy.
The Karolinska researchers discovered Vacquinol-1 by testing about 200 molecules for their potential impact on tumor cells. The found it caused the tumors to form blister-like structures called "vacuoles" that eventually simply collapsed, causing the tumors to explode and die.
"This is an entirely new mechanism for cancer treatment," said Patrik Ernfors, professor of biology at the Department of Medical Biochemistry and Biophysics at Karolinska Institutet.
"A possible medicine based on this principle would therefore attack the glioblastoma in an entirely new way. This principle may also work for other cancer diseases; we have not really explored this yet.
"We now want to try to take this discovery in basic research through preclinical development and all the way to the clinic. The goal is to get into a phase 1 trial."
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