Potential new treatment for pediatric brain cancer

Therapeutic options for an aggressive and lethal form of childhood brain cancer are presented in a mouse study published online this week in Nature Medicine. The findings illustrate the potential of precision medicine to pair cancer patients with drugs specific to their genetic alterations and may represent a therapeutic advance for this type of childhood tumour.

There are currently few treatment options for this type of childhood brain cancer, called diffuse intrinsic pontine gliomas, and surgery is not an option as the tumours are dispersed throughout important regions of the brain. Recent genetic studies identified that these tumours frequently have genetic alterations in one histone. Histones are proteins that package the DNA inside the cell and regulate gene expression. This specific genetic alteration blocks the activity of a regulator complex that would normally modify these histones, causing widespread cancerous consequences.

Rintaro Hashizume and colleagues test an existing drug that acts on the same pathway as the blocked regulator, in mice bearing tumours with the histone mutation, in an attempt to reverse the effects of the mutation. They report that the treatment of established tumours with this drug leads to an increase in survival of the mice.