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UC Berkeley professor collaborates on study to improve cystic fibrosis treatment

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FEBRUARY 04, 2019

UC Berkeley professor Terry Machen is collaborating with researchers from Canada to discover new treatments for cystic fibrosis, an incurable genetic disease that cuts decades off the lives of patients.

The research, a collaboration between UC Berkeley and the University of Saskatchewan, or USask, in Canada that began in 2016, is based on a unique method developed by the Canadian researchers to measure fluid secretion in the lungs. Cystic fibrosis is characterized by thick, viscous mucus that clogs the airways and causes bacteria to accumulate, leading to frequent infections that damage the airways.

“This treatment predicates an idea that if you inhale a solution that is more concentrated at the blood, the patient will get this salty solution in the lungs,” said Juan Ianowski, a study co-director and USask assistant professor of physiology. “The salt will cause osmosis or water movement through a concentration gradient and water movement from the blood side into the airways.”

The effects of this treatment change the properties of the mucus to become “normal,” allowing the patient to cough it out or have it moved through cilia, tiny hairlike structures, in the lung. This hypersaline treatment originated in Australia and has been used for decades to alleviate the symptoms of cystic fibrosis.

The research team — co-directed by Ianowski and Julian Tam, a respirologist at USask — found a way to develop this treatment by observing the way in which the hypersaline solution stimulated neuron cells that are present in the lungs rather than solely crediting the solution for its established osmotic effects.

“Neurons that are present in the airway are constantly monitoring the conditions of the airway, (becoming) deactivated in cystic fibrosis,” Ianowski said. “These neuron cells would also contribute to the production of water in the lumen of the airway.”

The research group determined that 50 percent of the liquid produced in the airway when the hypertonic saline is administered comes from the nervous system stimulating the production of fluids.

The impacts of these potential treatments enable cystic fibrosis patients to modulate the amount of the hypertonic saline solution as well as potentially disengage or engage certain aspects of their nervous system to manage a more desirable response.

“The main goal is to come up with new formulations that will allow us to make this treatment last longer and reduce negative reactions in the patient,” Ianowski said. “We want to make the treatment stronger, last longer, or cause it to inhibit some components such as a coughing reaction.”

The research team is funded by Cystic Fibrosis Canada and will be continuing to study developed treatments for cystic fibrosis.

Machen, campus professor emeritus of molecular and cell biology and study contributor, said “amazingly effective” drugs for cystic fibrosis have been made available for patients by pharmaceutical companies including Vertex. The disadvantage, however, is that these drugs cost $300,000 a patient per year.

“It would be a big addition to the field if we could develop something that made things better and is also cheaper,” Machen said.

Stanley von Ehrenstein-Smith covers research and ideas. Contact him at [email protected] and follow him on Twitter at @von_ehrenstein.
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FEBRUARY 05, 2019


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