Researchers say a widely-used antifungal drug may hold promise for treating people with cystic fibrosis, a life-threatening genetic disorder that causes serious damage to the lungs. In studies using human cells and animals models, the researchers found that the medication, called amphotericin, helps lung cells function in a way that could make it easier for patients to fight chronic bacterial lung infections that are a hallmark of the disease. The findings from the study will appear in the journal Nature.
If human studies validate the findings, the use of the drug could be good news to the more than 30,000 people in the United States and 70,000 worldwide who live with cystic fibrosis, a disease with no cure and few treatment options. It holds special promise for a subset of patients, about 10 percent of the people with cystic fibrosis, who do not respond to any treatment.
"The really exciting news is that amphotericin is a medicine that's already approved and available on the market," said the senior author. "We think it's a good candidate."
Cystic fibrosis is caused by a defect in a gene called CFTR (cystic fibrosis transmembrane conductance regulator). This gene normally makes a protein that controls or channels the movement in and out of cells of such materials as salt, bicarbonate, and water--all of which are important to normal lung function. In people with cystic fibrosis, however, the defective gene makes a protein that is itself defective, causing the accumulation of acidic and sticky mucus that not only clogs the lungs and makes it hard to breathe, but also makes the lungs vulnerable to bacterial infection.
While some treatments are currently available, they are limited because different people have different types of mutated proteins, and because 10 percent of people with cystic fibrosis make no protein at all. But amphotericin, the author said, has the potential to work regardless of the kind of mutation, and even when the protein is missing.
"Instead of trying to correct the protein or do gene therapy - the latter of which is not yet effective in the lung - we use a small molecule surrogate that can perform the channel function of the missing or defective protein," the author said. The researchers call this surrogate--the amphotericin-- a "molecular prosthetic," because it restores function much like a prosthetic device does when it replaces a limb.
In their studies, the researchers used lung tissue from patients with cystic fibrosis, as well as pig models of cystic fibrosis, and found that amphotericin spurred a host of changes associated with improved lung function--restoration of pH levels, improved viscosity, and increased antibacterial activity, among others.
The researchers noted that amphotericin can be delivered directly to the lungs to avoid common side effects. They cautioned that more experimental studies are needed before the drug is safe to treat cystic fibrosis in people. But experts are hopeful.
https://www.nhlbi.nih.gov/news/2019/scientists-find-new-approach-shows-promise-treating-cystic-fibrosis
https://www.nature.com/articles/s41586-019-1018-5
Latest News
TB blood test which could d…
By newseditor
Posted 27 Mar
Propionate supplementation…
By newseditor
Posted 27 Mar
Role of human Kallistatin i…
By newseditor
Posted 26 Mar
Addressing both flu and COV…
By newseditor
Posted 26 Mar
How the brain senses body p…
By newseditor
Posted 26 Mar
Other Top Stories
Detection of human brain tumor infiltration with quantitative stimu…
Read more
Sex neurons identified in the worms
Read more
A small peptide blocks abnormal blood vessel growth in retinal disease
Read more
Blood clotting protein triggers immune attack on the brain
Read more
Iron and brain cancer
Read more
Protocols
All-optical presynaptic pla…
By newseditor
Posted 23 Mar
Epigenomic tomography for p…
By newseditor
Posted 20 Mar
A mouse DRG genetic toolkit…
By newseditor
Posted 17 Mar
An optogenetic method for t…
By newseditor
Posted 13 Mar
Profiling native pulmonary…
By newseditor
Posted 08 Mar
Publications
Integrated plasma proteomic…
By newseditor
Posted 27 Mar
APP antisense oligonucleoti…
By newseditor
Posted 27 Mar
Targeting Erbin-mitochondri…
By newseditor
Posted 27 Mar
Regulation of Zbp1 by miR-9…
By newseditor
Posted 27 Mar
Pain-free oral delivery of…
By newseditor
Posted 27 Mar
Presentations
Hydrogels in Drug Delivery
By newseditor
Posted 12 Apr
Lipids
By newseditor
Posted 31 Dec
Cell biology of carbohydrat…
By newseditor
Posted 29 Nov
RNA interference (RNAi)
By newseditor
Posted 23 Oct
RNA structure and functions
By newseditor
Posted 19 Oct
Posters
A chemical biology/modular…
By newseditor
Posted 22 Aug
Single-molecule covalent ma…
By newseditor
Posted 04 Jul
ASCO-2020-HEALTH SERVICES R…
By newseditor
Posted 23 Mar
ASCO-2020-HEAD AND NECK CANCER
By newseditor
Posted 23 Mar
ASCO-2020-GENITOURINARY CAN…
By newseditor
Posted 23 Mar