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Science Translational Medicine Reports First Successful Treatment for Ménétrier's Disease

A new study points to the first effective medical treatment for Ménétrier's disease, over a hundred years after the rare stomach disorder was first described by French pathologist Pierre Ménétrier. Most commonly affecting adults, Ménétrier's disease causes ridges along the inside of the stomach wall—called rugae—to enlarge, forming giant folds in the lining of the stomach. The condition puts affected individuals at an increased risk for gastric cancer.


A portion of Ménétrier's disease patient's stomach.
[Image courtesy of Robert Coffey]

“There are probably about 500 reported cases of this disease in the literature since it was first identified,” said Robert Coffey, co-author of the study and professor of Medicine, Cell and Developmental Biology at Vanderbilt University.

What causes Ménétrier's disease is unclear, but it is thought to be an acquired disorder with no known genetic component. Recent studies suggest people with Ménétrier's disease have stomachs that make abnormally high amounts of a protein called TGF-alpha. With no effective drugs available for this disease, the last line of defense for patients has been removal of all or part of the stomach.

Now, Coffey and colleagues report a study of nine patients with severe Ménétrier's disease treated with the anti-cancer drug Cetuximab, which blocks the binding of TGF-alpha to a certain receptor in the stomach.

Because the disease is so rare, it took eight years to gather these patients. Of the seven patients who completed the one month treatment, all showed significant improvement in symptoms and biochemical features of the disease; including decreased production of TGF-alpha in the stomach, fewer stomach folds, and reduced abdominal pain, vomiting, and swelling in the feet and ankles. Remarkably, four of the participants' excess stomach folds disappeared almost completely.

Cetuximab is already approved by the FDA for treatment of patients with colorectal cancer, and there are ongoing efforts to seek approval of the drug for Ménétrier's disease.