Researchers Advance Treatments for Cancer Cachexia Muscle Wasting Syndrome

Between 50% and 80% of cancer patients experience cachexia, a wasting syndrome causing severe muscle loss, metabolic disruption, and fatigue. While long viewed as an inevitable part of disease progression, researchers are advancing new trials and treatments backed by a multi-million-dollar global research initiative.

Mealtimes were once a central family affair for Dave Kochanczyk’s household in Indiana. As an electrical contractor, volunteer firefighter, and property manager, he led an active life that included barbecuing, cooking Polish fare, and tending to fresh garden vegetables. That routine shifted after a pancreatic cancer diagnosis in 2010. Over seven years of intensive treatment, his appetite dwindled, his muscle mass dropped, and he grew gaunt and fatigued.

His struggles stemmed not only from the tumor and treatments, but also from cachexia, a syndrome that links cancer with muscle wasting and reduced appetite. Martin Kochanczyk, Dave’s son, who became an advocate for awareness through the Cancer Cachexia Society following his father’s death in 2017, recalls that it is very hard to watch. As Martin notes, his father was far from alone, with the condition affecting between 50% and 80% of cancer patients depending on the tumor type and disease stage.

Understanding the Physical Toll of Cancer Cachexia

The syndrome reaches far beyond simple physical wasting or lack of appetite. Patients often describe the exhaustion in striking terms. Abigail Newell, senior director of research at the Cancer Support Community in Washington D.C., says that she has heard people describe it as lying down under a thousand-pound blanket, adding that sitting up feels like running a marathon.

Beyond quality of life, the condition significantly impacts survival rates. Severe fatigue and psychological symptoms can leave individuals unwilling or unable to maintain their treatments. In some cases, extreme cachexia makes therapies completely intolerable or leads to exclusion from clinical trials.

Global Funding Drives the CANCAN Research Initiative

For years, cachexia was treated by many physicians as an unavoidable consequence of cancer progression, with medical focus placed almost entirely on eliminating the tumor. Treatments remain scarce globally. While the appetite stimulant anamorelin is approved and available in Japan, the U.S. Food and Drug Administration and the European Medicines Agency previously found evidence for its benefits insufficient for approval. Furthermore, forcing patients to eat more or providing tube feeding fails to reverse the metabolic wasting.

Researchers Advance Treatments for Cancer Cachexia Muscle Wasting Syndrome
Photo: fredhutch.org

That clinical blind spot began shifting in 2020 when the global research initiative Cancer Grand Challenges launched a funding call dedicated to the syndrome. The initiative ultimately awarded an international team spanning 16 research groups across 14 institutions to the tune of £20 million, equivalent to $25 million, to investigate cachexia’s drivers, subtypes, and treatments.

Now in its fifth year, the project—known as CANCAN—has produced roughly 30 published papers alongside independent studies. Researchers now view cachexia not as basic malnutrition, but as a complex whole-body syndrome involving metabolism gone haywire, impacting behavior and motivation alongside physical health.

Pharmaceutical Testing and Future Outlook

As more patients survive longer with cancer, medical demand for effective anti-cachexia interventions continues to rise. A handful of pharmaceutical companies, including industry giant Pfizer, have begun testing medicines designed specifically to target the syndrome.

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Ryan Schoenfeld, chief executive of the Mark Foundation for Cancer Research in New York City, notes that it is still an area of mystery, but the burst of activity over the past few years has made him optimistic, feeling confident in the future of the research there, that they will uncover ways to treat cachexia that will make it more reversible than it is today, and ideally, that they will prevent it.

Tobias Janowitz, a physician-scientist at Cold Spring Harbor Laboratory in New York and co-principal investigator of CANCAN, notes that a dedicated ecosystem of cachexia researchers has finally formed. With clinical trials advancing, scientists aim to transform a once-overlooked condition into a manageable and ultimately preventable target in cancer care.

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