New chemical causes deadly brain cancer to self-destruct

Scientists have discovered a chemical compound that cuts off the "energy supply" to glioblastoma cells, making them self-destruct. Glioblastoma is a highly aggressive brain tumor.

Glioblastoma is a deadly form of brain cancer. Glioblastoma tumors emerge from the sticky, supportive tissue of the brain, which gets an ample supply of blood.

This makes the cancer particularly difficult to treat; the malignant cells multiply very fast.

The median survival rate for this aggressive cancer is 10–12 months. According to some studies, the 5-year survival rate is below 10 percent.

However, new research carried out by an international team of scientists may have found a way to stop the cancer cells from spreading so fast. A new synthetic chemical compound called KHS101 cuts off the "energy supply" to the cancer cells.

Heiko Wurdak, from the University of Leeds in the United Kingdom, led the study, which was published in the journal Science Translational Medicine.

KHS101 cuts tumor growth in half

Laboratory experiments discovered that KHS101 disrupts the mitochondria of the cancer cells. Also known as the "powerhouses of the cell," mitochondria are tiny organelles responsible for turning nutrients into energy.

By disrupting the good functioning of mitochondria, KHS101 interfered with this fuel-producing metabolism and caused the cells to self-destruct.

"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," says Wurdak.

Next, the researchers wanted to see whether or not the compound could penetrate the blood-brain barrier, which is the "barrier between the brain's blood vessels (capillaries) and the cells and other components that make up brain tissue."

This barrier is essential because it helps protect our bodies from pathogens such as bacteria and viruses. However, the barrier may be an obstacle when specialists try to administer drugs.

So, Wurdak and colleagues transplanted human cancer cells into mice and administered the compound to examine its effects.

Rodents treated with the compound had a 50 percent decrease in their tumors, compared with the rodents that received placebo. KHS101 could indeed cross the blood-brain barrier.

The mice that were treated with KHS101 survived the disease, and the healthy tissue around the tumors remained unaffected.

Importantly, the researchers also found that the compound was successful in treating all the different genetic variations of cells within the tumors.

"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."

Heiko Wurdak

Prof. Richard Gilbertson — a brain tumor expert with nonprofit organization Cancer Research UK, who was not involved in the study — also comments on the new findings, saying, "Treatment for glioblastoma has remained essentially unchanged for decades, so there is a pressing need for preclinical research like this to identify and characterize potential new drugs."

"While the findings are encouraging," adds Prof. Gilbertson, "as an experimental chemical, further rigorous testing and refinement of KHS101 is required before trials in people can begin."

In the United States, almost 23,000 adults were diagnosed with brain tumors in 2015. Of all primary brain tumors, over 50 percent are glioblastomas.