Experimental Drug Shows Promise Against Cushing’s Disease

WEDNESDAY, March 7 (HealthDay News) — An experimental drug
called pasireotide reduced levels of the “stress hormone” cortisol and
improved symptoms in patients with Cushing‘s disease, a new study
found.

Cushing’s disease is a rare (three to five cases per million people)
hormonal disorder that causes a wide range of health problems and, if
untreated, significantly increases a patient’s risk of dying at a much
younger age than normal, researchers said in a news release.

Weight gain, high blood pressure, mood swings, irregular or absent
menstrual periods, insulin resistance, glucose intolerance and type 2
diabetes are among the symptoms of Cushing’s disease. It is a form of
Cushing’s syndrome, which is caused by prolonged exposure of the body’s
tissues to high levels of the hormone cortisol.

This phase 3 study of 162 patients in 18 countries found that treatment
with pasireotide reduced cortisol secretion by an average of 50 percent
and returned some patient’s cortisol levels to normal.

A phase 3 study means that a drug is in the final stages of testing
that drugs undergo before they can be approved for treatment of a specific
disease.

The study, funded by Novartis Pharma, appears in the March 8 issue of
the New England Journal of Medicine.

Dr. Spyros Mezitis, an endocrinologist at Lenox Hill Hospital in New
York City, is not associated with the study but is familiar with its
findings.

Mezitis said the study showed that the experimental treatment “improved
metabolic abnormalities and emotional difficulties. Therefore, pasireotide
injections become an alternative to surgical resection of the pituitary
ACTH-secreting tumor, and may be shown to work with the FDA-approved
mifepristone, which blocks the action of cortisol at receptors in the
body.”

Elevated blood sugar (glucose) levels occurred in 73 percent of the
patients who took the drug, a side effect that requires close attention,
according to senior study author Dr. Beverly Biller, of Massachusetts
General Hospital.

Cushing’s patients already have difficulty processing glucose, she
noted.

“Those patients who already were diabetic had the greatest increases in
blood sugar, and those who were prediabetic were more likely to become
diabetic than those who began with normal blood sugar,” Biller said in the
hospital news release. “So this is real and needs to be monitored
carefully.”

Mezitis agreed that careful patient monitoring is important.
“Blood-sugar elevations are dose-dependent with pasireotide and will need
to be managed as indicated for diabetes,” he said.

More information

The U.S. National Institute of Diabetes and Digestive and Kidney
Diseases has more about Cushing’s syndrome.

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