Scientists have identified an enzyme inhibitor that slows down tumour growth and increases survival in an animal model of the most deadly brain tumour in children, opening the door to a promising new treatment strategy.
Currently, there are no approved drugs for treating diffuse intrinsic pontine glioma (DIPG), according to the study published in the journal Nature Communications.
The inhibitor of the enzyme called ACVR1 slows tumour growth and increases survival in an animal model of DIPG.
"Our results are encouraging and suggest that it might be reasonable to test an inhibitor of this enzyme in a clinical trial," said Oren Becher, Associate Professor at Northwestern University Feinberg School of Medicine 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 said in a statement.
In 2014, Becher's lab co-discovered that ACVR1 mutations are found in about 25 per cent of DIPGs, leading the enzyme to be overactive.
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In the current study, Becher and colleagues demonstrate for the first time in an animal model that this enzyme mutation cooperates with a histone mutation 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-foot long DNA strand into the tiny nucleus of every cell.
Histones also help regulate which genes turn on and off, a process that goes awry when there is a histone mutation.
"Our future work will examine why and how the ACVR1 and histone mutations work together to trigger DIPG development," said Becher.
"Greater insight into this process will bring us closer to identifying a successful therapy for children with DIPG," Becher said.