LOS ANGELES (Oct. 28, 2024) -- A syndrome called cachexia, which triggers unexplained loss of weight and muscle mass, causes severe illness and death among patients with cancer and other serious health conditions. Cachexia (pronounced kuh·kek’·see·uh) can also be one of the earliest symptoms of some cancers, and there is currently no medication available to treat the condition.
November is a month designated for raising awareness about lung and pancreatic cancers, and Andrew Hendifar, MD, medical director of Pancreatic Cancer at Cedars-Sinai Cancer, sat down to talk with the Cedars-Sinai Newsroom about cancer cachexia. Hendifar is co-author of recent clinical trial results, published in The New England Journal of Medicine, that could yield the first Food and Drug Administration-approved medication for the condition.
More than 25% of cancer-related deaths can be attributed to cachexia. It affects 80% of patients with pancreatic cancer, and half of patients with advanced lung cancer. We also see it among patients with colon, kidney and stomach cancer, as well as heart, lung and kidney disease.
As many as 75% of patients with cancer have significant unexplained weight loss six months before they are diagnosed, but this clue is often missed. People just think the latest weight loss strategy they have tried is finally working. Unintentional weight loss is a significant health problem, and anyone who is losing weight unexpectedly should immediately seek the care of a health provider to investigate it further. It’s a huge problem and a huge unmet need. At this point, all we are really able to offer patients with cachexia is a referral to a dietitian.
For decades we've been trying to figure out what causes cachexia and what we can do to treat it. After pursuing many different pathways, we recently made significant progress with a drug that targets the GDF-15/GFRAL pathway.
The therapy we tested, a monthly injection that caused few side effects, blocks the action of GDF-15. Our Phase 2 clinical trial included patients with non-small cell lung, pancreatic and colorectal cancers. Patients taking this new drug had improved weight gain and physical activity as compared with patients receiving a placebo. Not only is this the first promising cachexia drug, it is also the first drug showing promise in blocking the action of GDF-15.
Several new clinical trials should be opening soon at various institutions, targeting cachexia not only in patients with cancer, but in those with other diseases as well. Patients with cachexia should be referred to a dietitian and also work with a physical therapist to help recover muscle mass. And they can ask their health care provider about opportunities to participate in one of these clinical trials.
Cedars-Sinai was recently part of an international consortium that conducted a Phase 3 trial called the MENAC trial. This trial found that a combination of protein and omega 3 supplementation, dietary counseling, a home exercise program and ibuprofen improved weight by roughly 2 pounds. But this hasn’t yet become part of official guidelines for cachexia care.
References
1. https://www.cedars-sinai.org/provider/andrew-hendifar-546093.html
2. https://www.nejm.org/doi/10.1056/NEJMoa2409515
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