Researchers have identified an enzyme inhibitor that may help fight the deadliest brain tumour in children.
The study, published in the journal Nature Communications, suggests that an inhibitor of ACVR1 enzyme slows tumour growth and increases survival in an animal model of diffuse intrinsic pontine glioma (DIPG) the deadliest brain tumour in children.
According to the researchers, currently, there are no approved drugs for treating DIPG.
"Our results are encouraging and suggest that it might be reasonable to test an inhibitor of this enzyme in a clinical trial," said senior author Oren Becher, Associate Professor at Northwestern University in the US.
"Prior to that, we need to evaluate different ACVR1 inhibitors in animal models to make sure we bring the most safe and effective agent to trials with children," Becher added.
In 2014, Becher's lab co-discovered that ACVR1 mutations are found in approximately 25 per cent of DIPGs, leading the enzyme to be overactive.
In this study, the team demonstrated for the first time in an animal model that this enzyme mutation cooperates with a histone mutation (H3.1 K27M) found in 20 per cent of DIPGs. Together, these mutations are important in initiating tumour development.
Histone is a protein that acts like a spool for DNA, helping to package the six-feet long DNA strand into the tiny nucleus of every cell.
"Our future work will examine why and how the ACVR1 and histone mutations work together to trigger DIPG development," Becher noted.