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Promising results for expensive cystic fibrosis treatment
Published Friday, December 28, 2012 4:29PM MST
Last Updated Friday, December 28, 2012 6:32PM MST
13-year-old Ryan Richards has cystic fibrosis which requires him to clear his airways daily.
People like Ryan manage their symptoms in an effort to avoid permanent damage to their lungs and digestive system.
Without proper maintenance, cystic fibrosis can be deadly, with many patients requiring lung transplants.
“There's always a worry about the future and what that looks like,” says Rhona Shore, Ryan’s mother. “What I try to remind myself of, and what we remind Ryan of, we are at a time in science when every day is changing.”
The latest development in the battle against cystic fibrosis is a new drug called kalydeco. The drug treats the cause rather than the symptoms.
Kalydeco fixes a defective protein and only works for patients with a one type of genetic mutation.
The drug works so well, most patients in the clinical trials stopped their other methods of treatment.
“The idea is it corrects the basic problem, so it replaces all of that,” says Dr. Mark Montgomery of the Alberta Children’s Hospital. “When you’re looking at how well that protein has to work it only has to have 15 percent of the residual function to be able to have no problems at all.”
The drug has been approved for sale in Canada but it carries a hefty price tag.
Kalydeco treatments cost approximately $300,000 a year.
Cystic Fibrosis Canada is working on finding a way to get the cost covered.