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Synthetic chemical discovered that may lead to glioblastoma treatment

A synthetic chemical (KHS101) has been discovered by researchers that has the ability to kill aggressive brain tumour cells by removing their source of energy.

This research, led by the University of Leeds and published in Science Translational Medicine, demonstrates promising results and may lead to the development of a therapy to fight glioblastoma — a deadly cancer that has a five-year survival rate of less than five percent.

“When we started this research, we thought KHS101 might slow down the growth of glioblastoma, but we were surprised to find that the tumour cells basically self-destructed when exposed to it,” explained Dr Heiko Wurdak, from the University of Leeds who led the international research team. “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.”

In the study, the researchers used mice implanted with human tumour cells in order to assess whether or not the chemical could cross the blood-brain barrier — which is a limiting factor to treatment options and as such important to assess for efficacy.

From their research, the team found that KHS101 successfully crossed the blood-brain barrier and caused a significant reduction in tumour growth in the mice treated when compared with those administered with placebo. Additionally, the team found that normal brain cells were unaffected by the chemical.

Further to these tests, the team looked at whether or not the chemical would be effective against genetic variations in tumours. They found that all the tested variations of glioblastoma subtype cells responded to the treatment.

“Treatment for glioblastoma has remained essentially unchanged for decades, so there is a pressing need for preclinical research like this to identify and characterise potential new drugs,” stated Professor Richard Gilbertson, Cancer Research UK’s brain tumour expert who wasn’t involved in the research. “While the findings are encouraging, as an experimental chemical, further rigorous testing and refinement of KHS101 is required before trials in people can begin.”

It is hoped that further research into the properties of KHS101 will lead to the discovery of similar chemicals/drugs that can disturb the energy sources of tumour cells and thus cause the cells to self-destruct. This should in turn widen the range of treatment options available in the fight against brain tumours.