Hope for a Fatal Brain Cancer

Bringing a sick child to the doctor can be challenging enough, but a fatal diagnosis is absolutely heartbreaking. Each year 300 children under 10 in America will learn they have DIPG, diffuse intrinsic pontine glioma. Survival is just one year.

This type of cancer arises from brain cells called glia. Their duties include providing the myelin covering surrounding nerves to insulate it and ensure speedy transmission of signals. The glial cells that become cancerous in DIPG are in the deepest area of the brain called the brainstem which regulates breathing, blood pressure and heart rate.

Now scientists have identified a molecule that stops the growth of this aggressive brain tumor. The molecule is called PRC-2, vital in regulating the expression of other genes. Without it, certain cells can grow uncontrollably. The mutation in DIPG may be keeping PRC-two from suppressing genes that keep cells in an immature state. These cells should be maturing into specific cells and when they don't, they can become the most aggressive tumors.

To test whether PRC-2 activity is important in these tumors, the scientist developed a molecule called JQ-1 that binds to and inhibits the action of PRC-2. They then injected human DIPG cells into the brainstems of mice. Once the mice developed tumors, they were treated with JQ-1 for 10 days. There was a dramatic decrease in the tumor sizes. JQ-1 even reversed the dysregulation of gene expression and shut down the genes that cause the cells to grow out of control. Though still in lab testing, this offers the first hope to DIPG patients that their cancer is survivable.