The results of the randomized phase 2 clinical trial, which included187 individuals who experienced cachexia with lung, pancreatic, or colorectal cancer, were reported in the New England Journal of Medicine on Sept. 14, 2024. Richard Dunne, MD, MS, a Wilmot Cancer Institute oncologist and cachexia expert was part of the large group of investigators who ran the nationwide clinical trial.

Cachexia involves loss of appetite and weight, muscle-wasting, fatigue, and weakness. It affects more than 50 percent of people who have cancer, and currently there are no FDA-approved treatments.

Scientists discovered that a drug, ponsegromab, blocks a hormone known as GDF-15 that regulates appetite and body weight. The patients in the trial had elevated levels of GDF-15, a primary driver of cachexia. Ponsegromab is a type of drug known as a monoclonal antibody, and in this trial, it improved many aspects of cachexia and its symptoms. Side effects were minimal, Dunne said, and in fact ponsegromab appeared to be safer than common appetite stimulants used by cachexia patients.

Drugmaker Pfizer supported the study.

“This is super exciting,” said Dunne, an associate professor of Medicine at the University of Rochester Medical Center. “This study is an important step in providing treatment for the hundreds of thousands of patients who suffer from poor quality of life due to cachexia.”

Several academic medical centers participated in the clinical research, which was led by John D. Groarke, MB, BCh, MPH, at Pfizer. Investigators are continuing to study GDF-15 and the importance of the biomarker in several types of cancer. Other clinical trials are also testing additional cachexia treatments that do not target the GDF-15 pathway.