New hope for cystic fibrosis patients

Faculty of Arts and Science researcher Lucia Lee has received funding from Cystic Fibrosis Canada to support a new project focused on developing new therapeutic strategies for individuals living with cystic fibrosis.

Cystic Fibrosis Canada is investing $325,000 in seven innovative early-stage research projects with a focus on advancing the greatest health-related needs of Canada’s cystic fibrosis community. Dr. Lee’s project, Development of Synthetic Chloride Channels and Transporters, is one of those seven projects receiving funding from the Cystic Fibrosis Canada Grant for Innovative Research.

People with cystic fibrosis, an inherited progressive condition that affects multiple organs, lack the healthy proteins needed to transport salt (chloride ions) in and out of their cells, leading the mucus in their lungs and gut to become thick and sticky and affecting breathing and digestion. Some therapies can help to address this, but they often have side effects – and do not work for everyone with CF.  

Dr. Lee (Chemistry) and her research team will develop and test new ways that salt transfer in and out of cells could be fully restored. This work could provide a foundation for developing treatments that could help anyone with CF – regardless of underlying genetic mutation – to better digest food and breathe more easily.    

“In patients with cystic fibrosis, the protein responsible for transporting the chloride ions is shut down. So, when that happens, the chloride and water movement across airway cells is disrupted, dehydrating the airway surface liquid. This causes thick mucus to build up in the airways, and people with CF sometimes describe this as feeling like they are drowning from the inside. This problem stems from the protein not doing its transportation job.”

Dr. Lee adds patients also become susceptible to infections because a proper amount of water in the airways will wash away bad bacteria, but excessive mucus build up allows the bacteria to become trapped which causes infections.

There are drugs available, but they only currently work for those with specific mutation, leaving some patients without targeted treatment options. “Our idea is to build a molecule that can mimic this transportation protein which could provide a universal therapy for assisting people with cystic fibrosis, no matter what mutation of the disease they have.”

This innovative approach could provide a foundation for future treatments that work independently of specific genetic mutations.

Learn more about the projects on the Cystic Fibrosis Canada website.