Patients with Cushing’s syndrome experience significant weight loss while taking oral medication

Sinai Hospital of Baltimore endocrinologist, Henry G. Fein, M.D., today presented new research showing that patients with Cushing's syndrome, a rare disease that can lead to extreme weight gain, type 2 diabetes, high blood pressure and psychological issues, experienced significant, sustained weight loss while taking oral medication daily over a number of years to manage symptoms of the disease.

Cushing's syndrome is a result of excessive levels of the stress hormone, cortisol, which is usually caused by a non-cancerous tumor in the pituitary or adrenal gland. The disease can have a significant impact on the body's natural chemical processes. In addition to the other symptoms, some patients also develop a 'buffalo hump' at the back of the neck and grow hair in unusual places.

Surgeons first try to treat the disease by removing the tumor, but for many patients, surgery is not an option. In some cases, it's only possible to remove a portion of the tumor, and the Cushing's syndrome symptoms continue. For these patients, who also continue to have high blood sugar, a medicine called Korlym® (mifepristone) is an option.

Fein participated in the late-stage study of Korlym that led to U.S. Food and Drug Administration (FDA) approval in 2012. He and other physicians have continued to follow patients who took part in the study in the years since it officially ended. He presented the outcomes of this long-term extension phase on June 23 at the joint meeting of the International Society of Endocrinology and the Endocrine Society (ICE/ENDO 2014) in Chicago, Illinois.

"Rapid weight gain is the most common symptom associated with Cushing's syndrome. Diabetes, severe fatigue, weak muscles, high blood pressure, depression and other negative health problems are common in these patients as well," said Fein. "We are very pleased to report that a large majority of patients with Cushing's syndrome who were enrolled in this study maintained their weight loss over the years as they continued to take the medication. We believe that this weight loss can have a positive impact on the patients' overall health and allow them to have a better quality of life."

The results, which include data on up to three and a half years of treatment, showed that patients experienced significant weight loss while taking Korlym, and they were able to keep the weight off, which may translate into other positive health outcomes for these patients. During the official study duration, a majority of the patients saw significant improvement in their type 2 diabetes and were able to reduce or stop taking insulin medications to manage the disease.

According to the American Association of Neurological Surgeons, an estimated 10 to 15 of every million people are affected by Cushing's syndrome each year. It is most common among adults aged 20 to 50; up to 70 percent of Cushing's syndrome patients are female.