Research gives hope of potential treatment for most deadly paediatric brain tumour

A study conducted by researchers from Northwestern Medicine and Ann & Robert H. Lurie Children’s Hospital of Chicago has given clues as to how diffuse intrinsic pontine glioma (DIPG) develops, opening up potential treatment.

DIPG is one of the most deadly forms of cancer in children, with only 5% of children surviving beyond two years after being diagnosed. The current standard of treatment is radiation therapy, however, this only decreases symptoms and does not offer an effective cure to patients.

The study found that the earliest sign of the development of DIPG can be linked to the mutation in histone H3, a protein that regulates gene expression. The mutation, known as H3K27M, has been linked to the development of 80% of tumours and is known to flip the ‘on switch’ for cancer promoting genes.

Researchers identified a compound that is able to alter the ‘on switch’. The compound was able to shrink tumour size and also increase overall survival in early studies.

"In this study we successfully targeted histone acetylation with a bromodomain inhibitor, a compound that is currently in studies for different types of advanced cancers in adults, but has never been studied in DIPG," said Senior Author Ali Shilatifard, Chair of Department of Biochemistry and Molecular Genetics.

Robert Francis Furchgott, Professor at Northwestern University Feinberg School of Medicine, said "We were able to show that it kills tumour cells in culture and shrinks the tumour in a mouse model of DIPG. These are very exciting results that we hope will be valuable for children with DIPG. A clinical trial is our next step."

As mentioned, the next stage of research will be to see if the bromodomain inhibitor is able to reproduce the same effect in humans as it did in animal studies. With such low survival rates for children with DIPG, the research may in its early stages but shows some promise towards providing a more effective form of treatment.