Researchers identify EGFR protein as therapeutic target for Cushing disease

Cushing disease is a hormone disorder that causes a diverse array of symptoms, including fat accumulation, high blood pressure, osteoporosis, muscle wasting, and ultimately death. It is caused by a tumor in the anterior pituitary gland that results in the secretion of excess amounts of adrenocorticotropic hormone (ACTH). Treatment options are essentially limited to surgical resection. However, tumors commonly recur, meaning that new treatment options are needed.

A team of researchers, led by Shlomo Melmed, at Cedars-Sinai Medical Center, Los Angeles, has now identified a potential new therapeutic target -- the protein EGFR, which is the target of a drug used to treat some patients with non-small cell lung cancer (gefitinib). As discussed by Melmed and colleagues in their paper, as well as Frederic Wondisford, at Johns Hopkins University School of Medicine, Baltimore, in an accompanying commentary, the data generated in human, canine, and mouse models provide strong support to investigate the clinical effects of gefitinib in patients with Cushing disease.