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First Effective Medical Treatment For Rare Stomach Disorder Introduced
Ménétrier's disease causes thickening of the stomach lining, severe abdominal pain, nausea and vomiting, as well as anemia and swelling in the feet and ankles due to protein loss. Patients are at increased risk for gastric cancer. Previously, the only effective treatment was gastrectomy – surgical removal of the stomach.
The targeted cancer drug cetuximab, brand name Erbitux, relieved symptoms of severe Ménétrier's disease in seven patients who completed a one-month course of treatment. Four of them showed near-complete remission, the Vanderbilt researchers report in the Nov. 25 issue of the journal Science Translational Medicine.
"We have identified the first effective medical therapy for this disorder," said Robert Coffey, M.D., Ingram Professor of Cancer Research and the paper's senior author. Erbitux is a monoclonal antibody that blocks the binding of transforming growth factor-alpha, or TGF-alpha, a signaling protein, to the epidermal growth factor (EGF) receptor. Patients with Ménétrier's disease have abnormally high levels of TGF-alpha.
In studies dating back 20 years, Coffey and his colleagues found that TGF-alpha causes proliferation of the stomach lining and stimulates mucous production while suppressing acid secretion. Transgenic mice that over-express TGF-alpha in the stomach exhibit all of the hallmarks of Ménétrier's disease.
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