Promising New Drug Therapy to Fight DIPG

Diffuse Intrinsic Pontine Glioma is a type of cancer that involves highly aggressive tumors located in the brainstem. It impacts many of the body’s functions such as breathing, eating, and movement. Around 300 children are diagnosed with DIPG in the United States every year. When diagnosed, most are given 9 to 12 months to live. No cure has been found for this horrible disease. Although, there has been significant progress in research and clinical trials in recent years.

A research paper was published this month that reveals a promising new drug therapy combination to fight DIPG. Associate Professor David Ziegler works at the Children’s Cancer Institute and Sydney Children’s Hospital where he runs the clinical trials program for kids. He has devoted his career to researching drug therapies for brain cancers, particularly DIPG. Ziegler established Australia’s first DIPG research team in 2013.

Ziegler and his team have discovered that a combination of the drugs difluoromethylornithine (DFMO) and AMXT 1501, an investigational agent being developed by Aminex Therapeutics, has proven to be effective in laboratory models of DIPG. DFMO has recently been getting attention as a treatment for difficult to control cancers such as DIPG. This is because it targets the polyamine pathway. Polyamines are created from several amino acids and the polyamine pathway is an area that allows tumor cells to grow.

In animal studies and 3D models of the tumor, it has shown that DIPG can bypass the activity of DFMO by pumping polyamines directly into the cancer. This allows the tumor to continue growing, despite treatment with DFMO. They have now made the breakthrough discovery that treatment with DFMO and AMXT 1501 potently blocks the transport of polyamines into the DIPG cancer cell.

This drug combination, which is currently in early trials in adult cancer, is one of the most effective treatments ever tested in laboratory models for DIPG. In mouse models, researchers have found that the drug therapy has led to survival in two thirds of mice and that it completely stunted growth of these aggressive tumours in these mice.

Associate Professor Ziegler said that clinical trials of this drug combination are going to begin for DIPG patients this year at Sydney Children’s Hospital. This treatment looks very promising and we are hopeful that it will be a success. We will keep you updated on this new combination as we move Full Steam Ahead towards a cure for DIPG.

By Clara Jude


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