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Study shows: Experimental drug helps cancer patients gain weight

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Pfizer Inc.'s experimental weight-loss drug for cancer patients has been shown to help them regain weight in a mid-stage study, offering new prospects for treating this dangerous muscle-wasting disease.

In cancer patients, a syndrome called cachexia causes changes in metabolism and appetite. It can lead to the loss of important skeletal muscle and fat tissue, which weakens the body and in some cases can make cancer treatments less effective. Studies suggest that up to 30% of all cancer deaths are due to cachexia and about 80% of patients with advanced cancer are affected by the condition. There are currently no approved drugs to treat the condition.

Pfizer's drug, ponsegromab, is a monoclonal antibody that blocks GDF-15, a protein produced when the body is exposed to extreme stress. High levels of GDF-15 in cancer patients are thought to be linked to loss of appetite and nausea, leading Pfizer scientists to hypothesize that blocking protein formation could prevent the onset of cachexia.

The drug was discovered by Pfizer itself, which is looking to cancer drugs as a growth engine for the company. Last year, the company bought cancer drug maker Seagen Inc. for $43 billion, and investors are eagerly awaiting positive news as sales of the COVID-19 vaccine continue to decline.

Pfizer enrolled 187 patients with cancer and high GDF-15 levels in a Phase II trial testing the effect of ponsegromab on body weight compared with a placebo. The patients in the trial had non-small cell lung cancer, pancreatic cancer or colon cancer, and about 75% of them had already progressed to stage 4 of the disease.

At the highest dose of 400 milligrams, patients regained about 5.6 percent of their body weight within 12 weeks, according to the New England Journal of MedicineAccording to the results, which will be presented simultaneously at the conference of the European Society for Medical Oncology in Barcelona, ​​these patients also experienced improvements in appetite, physical activity and skeletal muscle.

Charlotte Allerton, head of discovery and early development at Pfizer, said in an interview that the company hopes ponsegromab will enable weight gain, allowing patients to tolerate treatment longer and “be more active, which will of course improve their overall health.”

Pfizer is discussing development plans with regulators and aims to begin pivotal trials in 2025. The company is also testing ponsegromab in patients with heart failure and high GDF-15 levels. Allerton said the company is also researching other conditions associated with elevated GDF-15 levels, such as chronic kidney disease.

Further information:
John D. Groarke et al, Ponsegromab for the treatment of cancer cachexia, New England Journal of Medicine (2024). DOI: 10.1056/NEJMoa2409515

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