Childhood cancer may have a new rival.

According to research, a new drug that “targets genetic weakness” in an untreatable childhood brain could help treat the disease. This treatment could help assist patients who have ‘stone man syndrome’, in which “muscles and ligaments turn to bone” (“New treatment”, 2019).

It was found that this new drug can kill brain cancer cells and shrink tumors specifically within the ACVR1 gene – this could be promising, considering there has been no new drug developed to treat brain cancer for 20 years (“New treatment”, 2019).

This particular drug “targets the protein molecule produced by mutated versions of the ACVR1 gene called diffuse intrinsic pontine glioma (DIPG)”. These mutations are the cause of a variety of devastating conditions, such as ‘stone man syndrome’. This drug could offer an exceptional amount of ground, since brain tumors that develop in children often cannot be removed with surgery or treated by chemotherapy (“New treatment”, 2019).

After experimenting on mice, the introduction of the drug to mice with DIPG tumors had their lives extended by 25 percent and shrunk the tumors.

“DIPG is a relatively rare childhood brain cancer, but it is always deadly. Learning more about the biology of DIPG, and trying to find ways to translate that knowledge into new treatments, has been a passion of mine for years. My lab discovered that mutations in the ACVR1 gene occur in a quarter of DIPG cancers and it’s extremely exciting to see this now lead to potential new drugs for the disease,” stated Professor Chris Jones, Professor of Paediatric Brain Tumour Biology (“New treatment”, 2019).

Reference: Institute of Cancer Research. (2019, May 9). New treatment could become first targeted therapy designed for ‘untreatable’ childhood brain cancer. ScienceDaily. Retrieved May 12, 2019 from www.sciencedaily.com/releases/2019/05/190509101946.htm