Despite being the most common fatal genetic illness in Canada for children and young adults, Cystic Fibrosis (CF) still has no cure. But new research at Queen’s hopes to improve that reality.
CF is an inherited condition that affects multiple organs, including the lungs, digestive system, and pancreas. It’s a progressive condition, which means it worsens over time.
New research at Queen’s led by Dr. Lucia Lee, assistant professor in the Department of Chemistry, has received the Cystic Fibrosis Canada Grant for Innovative Research, a seed grant designed to help researchers explore promising new directions and generate the evidence needed to pursue larger-scale studies and clinical impact.
The grant is valued at $325,000, divided among seven innovative early-stage research projects across Canada, which include Cystic Fibrosis Canada’s first-ever clinical trial planning grant, aiming to allow those with CF to be a part of the research.
In an interview with The Journal, Lee shared why the grant was awarded to her research team. “So, [Cystic Fibrosis Canada’s] specifically funding research ideas that are new and innovative and a little bit of a higher risk.”
At the molecular level, the disease stems from a broken protein called the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR). In healthy individuals, this protein sits in the cell membrane and regulates the flow of chloride ions and water in and out of cells. When it malfunctions, the results are severe.
According to Lee, the surfaces lining the airways and digestive organs become dangerously dehydrated on the outside, causing the body to produce excessive mucus.
“People with cystic fibrosis often complain of their symptoms as if they’re drowning inside because of the sticky surface of their airway,” Lee noted. “It was featured in the movie, Five Feet Apart, so maybe people will be able to relate because of it.”
Existing therapies attempt to repair the faulty CFTR protein rather than replace its function entirely. Some drugs known as CFTR modulators can help correct how the protein folds, partially restoring its ability to transport chloride.
However, these treatments don’t work for everyone. “The problem is that this is very mutation specific,” Lee said. “People who don’t have this specific mutation don’t benefit from these drugs.”
Among major concerns regarding CFTR modulating therapies are accessibility and cost.
“In Canada, thankfully, the government is able to cover the cost of the drugs,” Lee added. “It actually costs up to $300,000 per person per year, and people in different countries where this accessibility isn’t allowed are just left without even an option.”
Lee’s research takes a different approach—creating synthetic molecules that mimic the function of the CFTR protein rather than trying to repair it.
“The molecule, we can design it so that it sits in the cell membrane, just like the proteins. But it doesn’t matter what kind of mutation you have, we’ll just allow the chloride to be transported,” Lee stated.
What makes the research novel and potentially higher risk is its use of heavier elements such as selenium, tellurium, and bismuth. Since these elements aren’t common in conventional medical research, it raises concerns about toxicity. However, in small doses, they can be very effective.
The next stage of Lee’s research involves designing and testing the molecules in laboratory models that simulate cells. “We assess [the cells’] ability to transport chlorides in fake cells like liposomes,” Lee said.
If the molecules perform well, the research could move into more advanced testing using cystic fibrosis cell models in collaboration with other scientists. “The long-term goal is advancing successful candidates toward clinical testing,” Lee added.
While the work is still at an early stage, the new grant highlights growing interest in innovative and interdisciplinary approaches to tackling cystic fibrosis—and the role chemistry could play in developing the next generation of therapies.
For a disease that still has no cure, Lee’s chemistry-first perspective may open doors that biology alone can’t.
Tags
cystic fibrosis, Cystic Fibrosis Canada, research grant
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