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McGill's 'Orphan Drug' research offers hope
Date:6/1/2010

This release is available in French.

In addition to their suffering, rare disease patients often have to face the harsh reality that few pharmaceutical companies will ever be able to offer new treatments for their condition because the costs of new treatments will never be recovered from such a small market. But there are ways they can be helped. The U.S. Food and Drug Administration's "Orphan Drug Designation" offers a wide range of benefits that help organizations developing treatments for diseases and conditions affecting fewer than 200,000 patients in the United States. It was recently granted to McGill University for research conducted at the MUHC into the use of the drug fenretinide for the treatment of pulmonary infections caused by Pseudomonas aeruginosa in patients with Cystic Fibrosis (CF).

CF is a chronic, hereditary disease affecting the respiratory and digestive systems of approximately 70,000 people worldwide. Pseudomonas aeruginosa infects more than 80 per cent of CF patients and contributes to the decline in pulmonary function the ability of the body to pump blood between the heart and the lungs. The infection is a major cause of sickness and death and is difficult to treat since it is resistant to many drugs.

"We strongly believe that fenretinide has the potential to improve morbidity and prognosis in CF patients at different levels," said Dr. Danuta Radzioch, a professor of Experimental Medicine at the MUHC and McGill's Faculty of Medicine. "Our research has demonstrated that fenretinide reduced lung inflammation as well as the frequency and the severity of pulmonary infections." Fenretinide is a drug that was originally investigated for potential use in the treatment of cancer but has not been commercially available.

Orphan Drug Designation provides opportunities for grant funding toward clinical-trial costs, tax ad
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Contact: William Raillant-Clark
william.raillant-clark@mcgill.ca
514-398-2189
McGill University
Source:Eurekalert

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