A large-scale clinical trial has shown promising outcomes for cancer patients experiencing a common syndrome related to weight loss.
Researchers have found a medication that effectively and safely addresses cachexia (ku-KEK-see-uh), a prevalent condition in cancer that leads to muscle loss and weight decrease.
The findings from the randomized phase 2 clinical trial, which involved 187 participants suffering from cachexia due to lung, pancreatic, or colorectal cancer, were published in the New England Journal of Medicine on September 14, 2024. Richard Dunne, MD, MS, an oncologist at the Wilmot Cancer Institute and an expert in cachexia, was part of the extensive team conducting this nationwide trial.
Cachexia is characterized by reduced appetite and weight, muscle loss, fatigue, and weakness. It impacts over 50% of cancer patients, and at present, there are no FDA-approved therapies available for it.
Researchers found that ponsegromab, a drug that inhibits a hormone called GDF-15—which plays a role in regulating appetite and body weight—was effective in treating the condition. Participants in the study exhibited high levels of GDF-15, which is a key contributor to cachexia. Ponsegromab belongs to a category of drugs known as monoclonal antibodies, and in this study, it improved multiple symptoms associated with cachexia, with minimal side effects. According to Dunne, it seemed safer than common appetite stimulants used by patients with cachexia.
The study was funded by Pfizer.
Dunne, an associate professor of Medicine at the University of Rochester Medical Center, expressed his enthusiasm, stating, “This is incredibly exciting. This study marks a significant milestone in offering treatment options to hundreds of thousands of patients whose quality of life is severely affected by cachexia.”
Multiple academic medical institutions contributed to the research, led by John D. Groarke, MB, BCh, MPH, from Pfizer. Researchers are continuing to investigate GDF-15 and its significance as a biomarker across different cancer types. Additional clinical trials are also underway to explore alternative treatments for cachexia that do not involve targeting the GDF-15 pathway.
