Transcript for Fighting Cystic Fibrosis
A new study in The New England Journal of Medicine uncovers a breakthrough new therapy in the fight against cystic fibrosis. Joining us now to explain his doctor Pamela Davis volcanologist and dean at case western reserve school of medicine. Doctor Davis welcome thanks so much for being here. Thank you for having me. So let's start with this study because it it looks like this really could be a breakthrough this is the first time -- rug. Has been shown to fight and they -- needs rather than just. The symptoms -- that right. Yes this drug is directed at the defective protein in cystic fibrosis or the kind of protein that's defective. In about 5% of patients with cystic fibrosis it's a very exciting development. That's a little bit let's back up a little bit and talk about cystic fibrosis itself -- more familiar what exactly. Is -- cystic fibrosis -- know the genetic diseases that it's the largest disease. That of affects caucasians cracked. It's a very common genetic disease film among caucasians it occurs in about one in 3500. Live births in the United States and it's a disease of salt transport. -- and several of the manifestations. Are very high content of salt in the sweat. And lung disease from failure of transport of salt in the Airways. And you get infected and the infection progresses and it can't be cured. And it's the lung disease and ultimately takes the life -- lives of most of the patients nausea. And it drastically shortens the lives of most patients -- backtracked. Well the average of the media and survival agent the United States now is about 37 years and that's a vast improvement from when I started in the field when it was only ten years of age. But still 37 is a very short life for. Vital and interest in -- absolutely and is it diagnosed at birth. It's very often diagnosed at birth and now -- days all fifty states have screening programs. So that most if not all patients with cystic fibrosis are identified in the first month -- life. And so once it is diagnosed what. Is the current treatment -- -- this drug could change all of -- what has been the current treatment. -- the current treatment. Is two every place. The digested and signs that the patient doesn't make and give that extra vitamins and because it's hard for the -- to absorb them. And for the lung disease. We give drugs to help break up the mucus and allow the patient to cough it out. And periodic treatment with antibiotics. To kill off the bacteria. That take up residence in the in the long. Some patients have other complications as well. Some of our patients take as many as sixty pills a day and do two to three -- even for treatments to clear their -- every day. So it really intrudes on people's lives. Yes it sounds like a very onerous treatment tell us about this new drug I believe it's called either -- -- -- And what is the potential that this drug -- Well this drug will strike directly -- They the protein itself that defective in cystic fibrosis. And it allows. -- to be transported across the Airways across the -- that's across the got. That wouldn't happen if the patient weren't treated. This drug is given twice today by mouth. -- had very few side effects and this study that's being reported now shows that over a year's period of time. -- -- be effective the drug did not -- And that's very important for our for our patient population while that's certainly exciting news what is the next step with this drug are more tax needed or is it available now occupation. Well -- the drug will be going to the FDA for approval and I think. CF. Interested parties are hoping that it will be approved in 2012. This drug was tested in patients with a particular mutation. That only occurs in 5% of the population. But in the test tube that works in other mutations as well so we're hoping that as many as 20% of the patients can benefit directly from this drug. The very very exciting news so if you are a loved one is suffering from cystic fibrosis. You recommend talking to that your doctor or their doctor about this right away. Absolutely. Absolutely and it's it shows the power of the discovery of the -- for cystic fibrosis. And the studies that moved systematically. Along. To find a drug to change the function of the protein. One very exciting scientific story and exciting indeed doctor -- thank you so much for joining us. Well thank you for having me.
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