A young cousin of mine died years ago from complications stemming from cystic fibrosis – he didn’t make it to legal age, if I recall. I’m glad there’s a new treatment, sad that it’s too late for so many.
Drug could stop mucus production in cystic fibrosis sufferers – Salt Lake Tribune
What is cystic fibrosis? » Cystic fibrosis is a genetic condition that causes cells to improperly manufacture a protein. The flawed protein results in the production of a thick, sticky mucus.
The mucus clogs and damages lungs and breaks down ducts in the pancreas, causing digestion problems.
It also coats the inside of the lungs, collecting bacteria that a healthy lung would expel. That leads to infection and triggers the immune system.
“Unfortunately, the immune cells’ weapons [against the bacteria] are just as lethal against human cells,” Liou said. “Most of the harm done to the lungs is actually collateral damage.”
That means many people with cystic fibrosis progress from difficulty walking up stairs to using oxygen and a wheelchair.
There are different genetic mutations that cause cystic fibrosis. The trial for VX-770 focuses on a mutation that affects only about 2 percent of people. Researchers hope they can adapt the drug to work for a much wider population.
If the drug stops cells from producing mucus, it won’t reverse any existing lung damage. But for those with minimal damage — younger patients, or those with milder cases — the drug could provide the closest thing to a cure.
Our cousin lived to 23 which is very unusual for this type patient.