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Cancer cachexia is a common and severe cancer complication characterized by significant weight and muscle loss, often associated with increased morbidity and mortality. Recent research highlights the central role of growth differentiation factor 15 (GDF-15) in its pathophysiology. Ponsegromab, a humanized monoclonal antibody targeting and inhibiting GDF-15, has shown promising effects, including increased body weight, improved appetite and physical activity, and reduced serum GDF-15 levels. This study evaluates the efficacy and safety of ponsegromab in treating cancer cachexia.

Can ponsegromab transform cancer cachexia management?

A total of 187 patients with cancer cachexia and elevated GDF-15 levels were included in the study. Participants were divided into four groups:
  • Ponsegromab treatment at three doses (100 mg, 200 mg, 400 mg)
  • Placebo group

The primary endpoint was body weight change. Secondary endpoints included appetite, cachexia symptoms, physical activity, and treatment safety.

The results show that patients treated with ponsegromab experienced significant weight gain compared to the placebo group. Significant improvements in appetite and reductions in cachexia-related symptoms were also observed, particularly in patients receiving the 400 mg dose. Additionally, non-sedentary physical activity increased in this group. The safety profile was favorable, with comparable rates of adverse events between the ponsegromab and placebo groups.


Ponsegromab: a promising new therapeutic pathway for cancer cachexia

This study demonstrates that ponsegromab significantly improves weight, cachexia symptoms, and physical activity, confirming the central role of GDF-15 as a therapeutic target. This treatment offers a promising option for patients with cancer cachexia. However, further research is necessary to validate these results and assess their long-term sustainability.  

Source(s) :
Groarke, J. D., et al. (2024). Ponsegromab for the treatment of cancer cachexia. New England Journal of Medicine ;

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