An experimental drug called ponsegromab may be an effective treatment for a wasting syndrome called cachexia that often affects people with cancer, according to results from a clinical trial.
The hallmark sign that
cachexia has taken hold is the unintentional loss of significant amounts of weight, both from fat and skeletal muscle.
In addition to harming quality of life, cachexia is also estimated to be responsible for as many as 30% of deaths in some cancer types.
In the clinical trial, nearly 200 people with advanced cancer and cachexia were randomly assigned to receive one of three different doses of ponsegromab or a
placebo. Participants treated with
ponsegromab gained anywhere from 2 to 6 pounds on average over 12 weeks, depending on the dose they received. By comparison, people treated with the placebo lost an average of 1 pound.
The group of participants treated with the highest dose, 400 mg, in fact, gained back more than 5% of their body weight, reported one of the study’s investigators, Jeffrey Crawford, M.D., of the Duke Cancer Institute.
That amount of weight gain is “clinically significant for our patients,” said Dr. Crawford, who presented the trial’s findings on September 14 at the European Society for Medical Oncology (ESMO) annual congress in Barcelona. The study’s results were published the same day in the
New England Journal of Medicine.
Trial participants treated with the highest dose also reported having better appetites, fewer cachexia-related symptoms, and were physically more active than those who received a placebo. Ponsegromab caused few side effects, and few people stopped treatment because of them, he explained.
Ponsegromab, a type of drug known as a monoclonal antibody, targets a protein called GDF-15. Several drugs targeting GDF-15 or another that it interacts with in the brain, called GFRAL, are in development as possible treatments for cachexia, but ponsegromab is the first to advance this far in clinical trials.
No drugs are approved in the United States or Europe to treat cachexia, so the results with ponsegromab represent “a big breakthrough in the field of cancer cachexia research,” said Richard Dunne, M.D., of the Wilmot Cancer Center in New York and an investigator on the trial.
In a press release, Pfizer—which makes ponsegromab and funded the trial—said it is planning to launch a larger trial of the drug next year in people with cancer and cachexia. If the trial produces similarly positive results, it could lead to the drug’s approval by the Food and Drug Administration.