New Hope for Cystic Fibrosis Could Save Over 90%, Doctors Say

[TO BREATHE EASIER Patient with approved treatment for Cystic Fibrosis]

For several years now, scientists have continuously struggled to find a therapeutic treatment suitable to aiding most victims of cystic fibrosis. Now, with a newfounded triple-drug approach, although still being tested, scientists are one step closer to a revolutionary uncovery.

Cystic fibrosis, a mutation in a gene also known as CFTR, forces the body to either produce defective versions of a protein, or none at all. With the hereditary disorder affecting the exocrine glands, an abnormally thick mucus is generated, and thus the pancreatic ducts as well as intestines are blocked.

So how can this be treated?

Well, as of recent laboratory studies conducted, two triple-drug approaches, both of which underwent processes of four week periods, showed a breakthrough with the improvement in lung function of people with the most common cystic fibrosis-causing mutation. About 90% of people with the disease have this mutation, and so with continued testing, this particular non-invasive triple-drug combination could potentially provide an effective treatment for the vast majority of cystic fibrosis patients.

This new triple-drug treatment builds upon the success of an earlier drug, that of which had been approved in 2012 for patients who had a much less common mutation. This particular treatment achieved high rates of success, as proven with detailed data analysis. Steven Rowe, a pulmonologist at the University of Alabama states, “They are in the hospital much less,” he says, and “the way they feel on a day-to-day basis is substantially improved.”

Thus, with the oncoming development of the triple-drug treatment, scientists are predicting a breakthrough in cystic fibrosis treatments, with some believing that, once approved, it will be life-changing for over 90% of patients living with cystic fibrosis.

 

Credit:

Cunningham, Aimee. “New Therapies Pack a Triple-Drug Punch to Treat Cystic Fibrosis.” Science News, Science News Organization, 19 Oct. 2018, www.sciencenews.org/article/new-therapies-pack-triple-drug-punch-treat-cystic-fibrosis?tgt=nr.

Image Credit:

News, CKOM. “Mom Reacts as Coverage Denied for $240K Cystic Fibrosis Drug.” 650 CKOM, www.ckom.com/2018/10/18/422789/