Cystic fibrosis (CF) patients suffering from chronic pulmonary infections may soon be offered a new treatment option thanks to a recent “Orphan Drug Designation” granted to McGill researchers by the U.S. Food and Drug Administration. The FDA initiative funds research into treating diseases or medical conditions affecting fewer than 200,000 people in the U.S., too small a market to excite significant interest of pharmaceutical companies.
Chronic pulmonary infections are the leading cause of sickness and death among the 70,000 people diagnosed with CF worldwide. In late spring 2011, Dr. Danuta Radzioch and Dr. Elias Matouk hope to begin clinical trials to test the safety, tolerability and efficacy of using fenretinide to treat a common infection called Pseudomonas aeruginosa in CF patients. Fenretinide is a drug mostly used in cancer research. “Our research has demonstrated that fenretinide reduced lung inflammation as well as the severity of pulmonary infections,” says Dr. Radzioch, a Senior Fellow of the American Asthma Foundation, and Professor of Medicine and Human Genetics at McGill’s Faculty of Medicine and the MUHC. “Based on our research thus far, we believe that this drug can reduce the number and length of lung infections which would improve the quality of life of patients, protect their fragile lungs and hopefully prolong their lives.”
The seeds of this project were planted in 1992, when Dr. Diane Gosselin (now Vice President of Research and Development at Quebec Consortium for Drug Discovery) and Dr. Charles Matouk (then a McGill undergraduate student, now the Endovascular Neurosurgery Fellow at University of Toronto) characterized CF mice and their response to Pseudomonas aeruginosa pulmonary infections. The project resulted in a patent submission—by Dr. Radzioch, Dr. Juan De Sanctis (then a visiting professor at McGill) and PhD student Claudine Guilbault—demonstrating the protective effect of fenretinide against abnormal metabolism of omega-3 and omega-6 fatty acids and other oxidation sensitive lipids important in launching proper defense against lung infections and osteoporosis; the work was built on PhD student Claudine Guilbault’s thesis project and Sahar Saeed’s Masters’ thesis project, respectively. Recently, genetics PhD student Gabriella Wojewodka validated the findings using plasma collected from CF patients at the Montreal Chest Hospital and the University Hospital in Olomouc in the Czech Republic; her work was done in collaboration with Dr. Marian Hajduch, Dr. Frantisek Kopriva and Dr. Jan Potesil in Olomouc
After receiving the Orphan Drug Designation, the CF researchers received 700,000$ from the Ministère du Développement économique, de l’Innovation et de l’Exportation, as well as contributions from MSBi Valorisation and Pharmascience. This work is also funded by CIHR, Canadian Cystic Fibrosis Foundation, American Asthma Foundation and the U.S. Department of Defense.
Note: The print version of this article omitted important information about the history of this research. The online version has been corrected.