- A collaboration between the University of Illinois and the University of Iowa found a new treatment for cystic fibrosis.
- It uses an FDA approved anti-fungal drug to mitigate the frequency of lung infections common in cystic fibrosis patients.
- While the treatment is promising, its results must be verified in clinical trials before its efficacy is proved.
A new research collaboration between the University of Illinois and University of Iowa shows promise as a new treatment for patients with cystic fibrosis. While efficacy still must be proved in the clinic, Dr. Martin Burke, a professor of chemistry and researcher at the University of Illinois and associate dean for research at the Carle Illinois College of Medicine, said what began as a “crazy” idea might prove to be a powerful discovery.
“A lot of human diseases are caused by too much protein function,” Burke said. “In those cases, small molecules (drugs) are good at binding to proteins and turning them off.”
As an example, Burke turns to aspirin. The drug works by inhibiting the function of cyclooxygenase enzymes which can provide relief from symptoms of inflammation and pain. The strategy often, Burke said, was to find a drug that inhibits the right protein.
“The challenge is there are other diseases caused by deficiencies of proteins,” Burke said. “You are missing something, and that means there is no target to shut down.”
This is a problem for patients with cystic fibrosis. The genetic disorder refers to patients that lack cystic fibrosis transmembrane conductance regulators or CFTRs for short. This protein is responsible for releasing bicarbonate (the same chemical as baking soda) in the lungs. The release bicarbonate helps neutralize the pH of the airway surface liquid preventing it from becoming too acidic.
“Bacteria love acidity,” Burke said. It was the University of Iowa’s own Dr. Mike Welsh who explored this relationship between cystic fibrosis patients’ acidic airways and patients with high rates of lung disease.
According to this new research, an anti-fungal drug already approved by the FDA could help substitute for some of the bicarbonate releasing functionality cystic fibrosis patients need.
Burke compares it to a prosthetic hand. While it will not offer a 100 percent return of functionality, the drug was shown to allow patient’s lungs to release bicarbonate. While it does not do so at the level of CFTRs, Burke said even the amount it does release could be an effective form of treatment.
“One of the key pieces is maybe it doesn’t have to be perfect. Maybe partial functionality can be returned anyway,” Burke said. “It looks like imperfection is still enough to get back function.”
Burke emphasized that the collaboration between the two universities was key for the outcomes of their study.
“Mike Welsh’s lab (at the University of Iowa) is a world leader in cystic fibrosis research,” Burke said.
Burke and two graduate students, Katrina Muraglia and Rajeev Chorghade, made frequent trips to Welsh’s lab to work with samples not readily available elsewhere. Of course donated tissue was helpful, but Welsh’s lab also developed pigs genetically selected to not have CFTR proteins. Pigs are one of the few animals other than humans that can contract cystic fibrosis.
“Welsh opened up his lab to my students,” Burke said. “They drove out and spent a lot of time at the Carver School of Medicine. They couldn’t have been more hospitable.”
The research findings are pre-clinical study, meaning there is still a great deal of vetting necessary to confirm that results from the lab can be replicated in patients. Burke is careful not to hype the results for the reason.
But Burke, whose research is funded by the National Institute of Health, said he is happy to be coming forward with results. Part of the excitement is having something to show for the investment into his team’s work.
“Much of this work was funded by taxpayers,” Burke said. “I want people to know that that money is getting put to good use. That every dollar we get goes into helpful treatments.”
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Zachary Oren Smith writes about government, growth and development for the Press-Citizen. Reach him at firstname.lastname@example.org or 319-339-7354, and follow him on Twitter @zacharyos.
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