Cachexia Research Targeting the Brain Holds Significant Promise

By Daniel L. Marks, MD PhD

As scientists research treatments for cachexia, a wasting syndrome characterized by significant involuntary weight loss and muscle atrophy, there is a growing recognition that medications for this condition need to target the brain.

There is currently no drug on the market in the U.S. that effectively treats cachexia, which is typically associated with cancer and other severe chronic diseases. But multiple companies are working on candidates, including Endevica Bio and Pfizer.

A recent study published in the New England Journal of Medicine related to the Pfizer drug candidate highlights how the brain has become the focus of treating cachexia. Pfizer’s approach targets a specific pathway in the brain involved in regulating appetite. In preclinical models, this drug showed promising results in reducing cachexia symptoms. A drug on the market in Japan to treat cachexia targets the ghrelin receptor, a key part of the gut-brain axis.

The research behind these drugs represents significant progress. But there is a lot more work to be done as these drugs may be beneficial for a subset of patients – not the majority of individuals who suffer from cachexia.

To truly revolutionize the treatment of cachexia, we would ideally identify therapeutic avenues that target the brain in a more comprehensive manner. This could involve targeting a wide range of neural pathways involved in appetite, and energy balance.

Regardless of te approach different researchers are taking, we find all of these advances exciting. For too many years, too many people have suffered from cachexia -- a largely unknown disease -- without any effective treatments. Daniel Marks is Endevica Bio’s Chief Medical and Scientific Officer