An international team of researchers has used a synthetic chemical to induce glioblastoma, a particularly aggressive type of brain cancer, to self-destruct in laboratory tests. This is a potential first step towards finding an effective treatment for this type of tumor.
The study, published in Science Translational Medicine, discusses the effects of the chemical KHS101 on glioblastoma cells. This chemical is capable of cutting the energy source of these cells by disrupting its mitochondria. This led to the eventual shut down of the cells' metabolism and eventually the death of the cancer. Given that glioblastoma has only a five-year survival rate of less than 5 percent, this research is welcome news.
"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,” team leader Dr Heiko Wurdak, from the University of Leeds, said in a statement. "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."
The research was conducted in the lab, but the team also investigated two important factors that suggest the chemical could potentially be used as a treatment: its ability to cross the blood-brain barrier and its safety on regular brain cells. The blood-brain barrier stops most molecules from entering the brain and while it keeps us safe most of the time, it also reduces the number of viable treatments for brain cancer.
The researchers used KHS101 in mice that had human glioblastoma growing in them. The chemical was seen to successfully cross the blood-brain barrier, leading to a 50 percent reduction in tumor growth in the mice compared to those given a placebo. The team also showed that normal brain cells were unaffected by the chemical.
Since previous treatments have found genetics to play a role in the effectiveness, researchers successfully tested KHS101 in several different subtypes of glioblastoma.
"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,” Professor Richard Gilbertson, Cancer Research UK's brain tumor expert who wasn't involved in the research, explained. "While the findings are encouraging, as an experimental chemical, further rigorous testing and refinement of KHS101 is required before trials in people can begin."
KHS101 might not be the only chemical out there that can disrupt tumors cells to the point of non-return. Understanding this chemical's properties might lead to a wide range of treatments for brain cancers.