Experimental Drug Shows Promise for Cancer-Related Weight Loss Condition #

A new experimental drug has shown positive results in a midstage trial for cancer cachexia, a life-threatening condition that causes cancer patients to lose their appetite and weight. The treatment demonstrated improvements in body weight, muscle mass, quality of life, and physical function for patients with the condition.

Cancer cachexia affects approximately 9 million people worldwide, with 80% of cancer patients suffering from it expected to die within one year of diagnosis. The condition is characterized by significant fat and muscle loss, leaving patients weak, fatigued, and sometimes unable to perform daily activities. It is currently defined as a loss of 5% or more body weight over six months in cancer patients, along with symptoms such as fatigue.

The symptoms of cancer cachexia can make cancer treatments less effective and contribute to lower survival rates. The experimental drug aims to address this unmet need in cachexia, potentially improving patients’ wellness, ability to care for themselves, and tolerance for cancer treatments.

The midstage trial involved 187 people with non-small cell lung cancer, pancreatic cancer, or colorectal cancer who had high levels of growth differentiation factor 15 (GDF-15), a key driver of cachexia. After 12 weeks, patients who received the highest dose of the drug saw a 5.6% increase in weight compared to those who received a placebo. Lower doses also showed weight increases of approximately 3.5% and 2%.

Experts consider a weight gain of greater than 5% as clinically meaningful for cancer patients with cachexia. The drug’s effect on other measures of wellness, such as increased appetite and physical activity, was also encouraging.

No significant side effects were observed with the drug. Treatment-related side effects occurred in 8.9% of people taking a placebo and 7.7% of those who took the experimental treatment.

The drug works by reducing the levels of GDF-15, a protein that binds to a certain receptor in the brain and impacts appetite. By lowering GDF-15 levels, the treatment aims to improve appetite and enable patients to maintain and gain weight.

Late-stage development plans for the drug are being discussed with regulators, with studies aimed at filing for approval expected to begin in 2025. The experimental treatment is also being studied in patients with heart failure, who can also suffer from cachexia.

If successful, this drug could become the first treatment approved specifically for cancer cachexia in the United States, offering hope for millions of cancer patients struggling with this debilitating condition.