New gene therapy drug for inhalation helps cystic fibrosis patients

New gene therapy drug for inhalation helps cystic fibrosis patients

For people with the genetically inherited disorder cystic fibrosis, breathing becomes a constant battle.

This rare disease causes the lining of the lungs to secrete heavy mucus that makes it very tough to take a breath and also leads to repeated lung infections. After years of this type of assault on their lungs, most people with cystic fibrosis die of respiratory failure.

But researchers from the United Kingdom may have developed an important therapy that could help to stabilize or improve lung function. After years of failed attempts by scientists around the world to create an inhalable gene therapy for cystic fibrosis, members of the U-K team say they have achieved notable success.

Their latest trial of the inhaled drug included 140 participants age 12 or older, split into two groups.

Patients who underwent the treatment every four weeks for a year showed almost a 4 percent improvement in lung function. Others who inhaled a placebo at the same frequency saw their lung function degrade.

Doctors consider the 4 percent enhancement to be a stabilization of lung function rather than an improvement. But that’s obviously much better than a decline.

Experts say even if the treatment does no more than prevent lungs from getting worse, the therapy would be worthwhile.

This therapy works by delivering correct copies of a specific gene … the one that is mutated in cystic fibrosis patients … into the lungs, along with a vector that takes the D-N-A molecules into lung cells. There, the new genes can take over for defunct ones, orchestrating better function of the lungs.

More trials are needed to know if this drug is suitable for patient care. But the researchers say more drugs with similar delivery methods should be tested in trials.

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