In a partnership between Michigan State University in East Lansing and Spectrum Health in Grand Rapids called the Cystic Fibrosis Translational Research program, a team of researchers is studying the effectiveness of a promising FDA approved treatment called Trikafta that is a combination of the drugs Elexacaftor, Tezacaftor, and Ivacaftor.
What makes Trikafta unique is that it has the potential to eliminate the mucus that causes breathing difficulty in cystic fibrosis patients.
Cystic fibrosis is caused by a genetic mutation that results in the body producing extraordinarily high volumes of thick mucus in the lungs that leads to chronic lung infections.
“It’s amazing,” says Robert Quinn, an assistant professor in the Department of Biochemistry and Molecular Biology in the College of Natural Sciences at MSU. “Patients were reporting a decrease in symptoms within weeks.”
By comparing the mucus from 24 patients before and after they were treated with Trikafta, Quinn and his team identified changes in the patients’ microbiome (the consortium of bacteria infecting the lungs) and metabolome (its collective biomolecules). The research was published Nov. 22 in the Journal of Cystic Fibrosis.
What the researchers found was encouraging. Usually, patients with cystic fibrosis have a unique microbiome dominated by a pathogenic bacterium that is known to cause damage to their lungs.
Trikafta changes their microbiome by making it more diverse with many different bacteria, so it looks more like that of a healthy person without the disease. Furthermore, the number of amino acids in the mucus is shown to decrease, which is believed to be the primary food source for some pathogens in the lungs. If the pathogen’s food source goes away, it may no longer be able to survive in the airways.
While the researchers may not fully understand yet how Trikafta works, the treatment is proving to be successful in reducing the amount of mucus patients’ lungs are producing. But this presents a new challenge for researchers.
“The thing that makes life better for patients is making the research more difficult,” Quinn says. “Trikafta reduces the amount of sputum (mucus) in the lungs, which is life-changing for the patients, but if we don’t have any mucus to sample, it’s difficult to learn about what lung infections will look like in the future.”
The use of Trikafta could mean an increase in the life expectancy of cystic fibrosis patients, and for young children who begin the treatment early. “They (children) might never have to experience the symptoms of cystic fibrosis in the same way prior to Trikafta,” Quinn says.
The research was funded by a National Institute of Allergy and Infectious Disease Grant R01AI145925.