Saturday, March 24, 2012

New drug shows promise in patients with rare illness

Two in Montreal with Cushing's disease were among 16 in international trial

By AARON DERFEL, The Gazette

Quebecers suffering from Cushing's disease - a rare hormonal disorder - have some reason to be hopeful after a clinical trial conducted partly in Montreal has shown promising results of a new drug.

About 400 to 500 Quebecers have Cushing's disease or syndrome, a disorder that produces tumours on the pituitary gland, leading to a spike in cortisol levels.

The excess cortisol can cause a wide range of problems, including obesity, hypertension and diabetes as well as sleep and mood disorders.

In some people, deposits of fat accumulate on the back of the neck and shoulders, an effect known as a "buffalo hump."

Until now, surgery and radiotherapy have been the only options for many patients. However, a drug developed by Novartis Pharmaceuticals has been found to act on the tumours, cutting cortisol levels an average of 50 per cent.

In some patients - two of them from Quebec - cortisol levels returned to normal.

The drug, pasireotide, involves twice-daily injections. The treatment has yet to be approved by Health Canada.

"This study is very promising, especially for patients for whom complete surgical removal of tumours - the standard treatment for this disease - was not possible," said Dr. André Lacroix, an endocrinologist at the Centre hospitalier de l'université de Montréal.

Lacroix and his colleagues tested the drug on four patients. In addition to the two who "experienced a complete regression of all symptoms" of Cushing's disease, the two others reported drops in their cortisol levels and an improvement in their health, Lacroix noted.

The findings of the international study of 16 patients were published in the latest edition of the New England Journal of Medicine.

Pasireotide acts by binding to certain receptors on the tumours. Lacroix's team has also carried out a separate study of another drug that targets a different receptor.

He suggested that using the two drugs together might prove even more beneficial, but this must be borne out by further research.

The ideal patients for the drug therapy would be those whose tumours are too small for surgery, Lacroix said.

Each year in Quebec, there are about 15 new cases of Cushing's disease, and doctors at the CHUM treat about 150 patients.

aderfel@montrealgazette.com

© Copyright (c) The Montreal Gazette

 

 

Read more: http://www.montrealgazette.com/health/
drug+shows+promise+patients+with+rare+illness/6340316/story.html