Study: New Chemical Kills Glioblastoma Tumor Cells, Boosts Survival in Mice


A synthetic chemical caused the self-destruction of glioblastoma tumor cells transferred from human patients into mice.

A newly-discovered chemical may lead to new treatment options for glioblastoma, according to a recent study published in Science Translational Medicine.

The researchers found that a synthetic chemical, called KHS101, successfully destroyed glioblastoma tumor cells taken from human patients. The results represent a promising step toward finding new therapy options for combatting the aggressive brain cancer, according to the study authors.

Glioblastoma is associated with poor prognosis, showing a 5-year survival rate of less than 5%, which emphasizes the need for urgent improvements in therapy options.

“Treatment for glioblastoma has remained essential unchanged for decades, so there is a pressing need for preclinical research like this to identify and characterize potential new drugs,” professor Richard Gilbertson, Cancer Research UK’s brain tumor expert, said in a statement.

In the study, researchers from the University of Leeds tested the chemical’s effect on the growth of glioblastoma, initially hypothesizing that it may be able to slow down the cancer’s progression. The study showed that KHS101 disrupted the mitochondria and metabolism within tumor cells, shutting off the energy supply and leading to their self-destruction.

“When we started this research we thought KHS101 might slow down the growth of glioblastoma, but we were surprised to find that the tumor cells basically self-destructed when exposed to it,” lead author Heiko Wurdak, PhD, from the University of Leeds, said in a press release.

The researchers analyzed KHS101 by transferring tumor cells from human patients into mice. According to the study, the chemical successfully crossed the blood-brain barrier and significantly decreased tumor growth in treated mice, compared with mice given a placebo. Although the chemical was able to destroy the tumor cells, it had no effect on normal brain cells, the researchers noted.

Additionally, the researchers found that KHS101 maintained efficacy even when faced with different genetic profiles of cells within a tumor and between tumors in different patients. Importantly, all tested variation of glioblastoma subtype cells responded to the treatment.

“This is the first step in a long process, but our findings pave the way for drug developers to start investigating the uses of this chemical, and we hope that one day it will be helping to extend people’s lives in the clinic,” Dr Wurdak said.

Although the results are promising, further studies need to be explored before trials can begin for human patients. Moving forward, the researchers indicated that investigating similar drugs that target cell metabolism could offer hope in treating the deadly disease.


Polson ES, Kuchler VB, Abbosh C, et al. KHS101 disrupts energy metabolism in human glioblastoma cells and reduces tumor growth in mice. Science Translational Medicine. 2018. Doi: 10.1126/scitranslmed.aar2718

A new weapon against one of the deadliest cancers? [news release]. University of Leeds’ website. Accessed August 15, 2018.

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