Drug breakthrough gives cancer patients with cachexia new hope

Researchers have discovered a drug that safely and effectively helps cancer patients suffering from cachexia (ku-KEK-see-uh), a common cancer condition associated with weight loss and muscle wasting.

The results of the randomized Phase 2 clinical trial, which included 187 people suffering from cachexia due to lung, pancreatic or colon cancer, were published in New England Journal of Medicine on September 14, 2024. Richard Dunne, MD, MS, an oncologist and cachexia expert at Wilmot Cancer Institute, was part of the large group of researchers who led the nationwide clinical trial.

Cachexia is characterized by loss of appetite, weight, muscle wasting, fatigue and weakness. It affects more than 50 percent of cancer patients and there are currently no FDA-approved treatment options.

Scientists have found that the drug ponsegromab blocks a hormone called GDF-15, which regulates appetite and body weight. Patients in the study had elevated levels of GDF-15, a major factor in cachexia. Ponsegromab is a drug known as a monoclonal antibody, and in this study it improved many aspects of cachexia and its symptoms. Side effects were minimal, Dunne said, and in fact, ponsegromab appeared to be safer than the usual appetite stimulants taken by cachexia patients.

The pharmaceutical company Pfizer supported the study and published this news.

This is super exciting. This study is an important step towards providing treatment to hundreds of thousands of patients who suffer from a reduced quality of life due to cachexia.”

Richard Dunne, MD, MS, Associate Professor of Medicine, University of Rochester Medical Center

The clinical trial, led by John D. Groarke, MB, BCh, MPH, at Pfizer, involved several academic medical centers. Researchers continue to study GDF-15 and the biomarker's importance in various cancers. Other clinical trials are also testing additional cachexia treatments that do not target the GDF-15 pathway.

Learn how Wilmot is supporting global efforts to research and prevent cancer cachexia.