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For one in 10 cystic fibrosis patients, current therapies don’t work, leaving them ravaged by shortness of breath and bouts of pneumonia and other infections. Now, there hope for them in the form of a medicine that’s already approved to fight fungal infections, researchers report today. The treatment appears to restore healthy function in human cells affected by cystic fibrosis as well as in pigs with the disease.
“This is a wonderful development,” says Garry Cutting, a cystic fibrosis expert at the Johns Hopkins University School of Medicine in Baltimore, Maryland, who was not involved with the work. “This type of drug could provide a viable option for cystic fibrosis patients who are not eligible for any of the revolutionary treatments.”
Fibrosis - Results - Mutations - Gene - Protein
Cystic fibrosis results from mutations in a gene that codes for a protein called cystic fibrosis transmembrane conductance regulator (CFTR). The protein forms pores in cells that line the airways of the lungs, known as epithelial cells. These cells build up a compound called bicarbonate. The pores help the cells release bicarbonate into the lungs’ mucous lining, where it helps fight bacteria that infect lung tissue. In cystic fibrosis patients, these pores don’t work properly, so patients suffer from repeated and sometimes life-threatening lung infections.
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A series of revolutionary new drugs have helped restore at least partial function to CFTR channels. One helps ensure CFTR proteins form properly, for example, whereas others increase the transport of bicarbonate through the cellular pores. But some cystic fibrosis patients have CFTR mutations that don’t respond to the drugs, whereas others lack the CFTR gene altogether. “We don’t have many options for the patients that don’t produce CFTR,” Cutting says.
Years - Researchers - Martin - Burke
That may soon change. In recent years, researchers led by Martin Burke, a...
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