News / Health

    New Therapy Gives Hope to Cystic Fibrosis Patients

    FILE - A photo provided by Laura Mentch, shows Laura Mentch of Bozeman, Montana, with her daily treatments for cystic fibrosis.
    FILE - A photo provided by Laura Mentch, shows Laura Mentch of Bozeman, Montana, with her daily treatments for cystic fibrosis.
    Jessica Berman

    Cystic fibrosis is one of the most common genetic disorders. It compromises a patient’s ability to breathe, digest food and ultimately leads to early death among the disease’s young patients. A Massachusetts biotech company this week announced a new combination therapy that could help people with the most common form of cystic fibrosis breathe easier.

    Cystic fibrosis is an inherited disease that causes sticky mucus to settle in the lungs and block the flow of air, causing life-threatening infections. According to the American Lung Association, approximately one in 2,500 white Americans of European descent and one out of every 15,000 Americans of African descent are born with CF. The Cystic Fibrosis Foundation estimates it affects some 70,000 people around the world.  

    One of them is Aaron Stocks, who participated in the clinical trial of the new therapy. Within a couple of weeks of starting the two-drug treatment, Stocks noticed a big change.

    "I was able to feel a difference in just the way I was breathing; being able to absorb my food better.  That has always been an issue for me," he said. "So, it has really been eye-opening and making the future even more exciting for us.”

    The CF foundation says about half of people born with the disorder die before their 30th birthday. But for the first time ever, one 28-year-old CF patient does not worry about his approaching mortality. In fact, he and his wife are thinking about starting a family.  

    “Now, we having different conversations planning for the future instead of, 'If' there is going to be a future,” he said.

    With a combination of the CF drug Kalydeco and the experimental compound lumacaftor, those with the most common form of the disease could be breathing almost normally. There are many forms of CF, and the trial only included patients with the most common mutation.

    The drug combination is manufactured by the biotech company Vertex. It treats the underlying cause of the disease, (which is abnormal proteins that cause thick mucus to accumulate in the lungs and impair digestion,) making it difficult for patients to get adequate oxygen and nourishment.  

    Aaron Stocks still spends several hours a day battling the disease, pounding his chest to loosen the mucus, taking vitamins and digestive enzymes as well as exercising to slow down progression of the disorder.

    But the drugmaker says the combination therapy has given Stocks, and some 22,000 people around the world, the prospect of a new lease on life.
     
    “It is going to help in the fact that there should be less of a decline in our health, which is the most exciting part,” he said.

    Vertex will reportedly seek approval for the drug combination from the U.S. Food and Drug Administration by year's end.  The company hopes the therapy becomes available to cystic fibrosis patients by the end of 2015. 

    You May Like

    Ethiopia's Anti-terrorism Law: Security or Silencing Dissent?

    Yonatan Tesfaye was detained in December 2015 on charges under Ethiopia's Anti-Terrorism Proclamation; eleven statements from his Facebook page were used as evidence

    Egypt Orders Trial for Journalists Charged With Harboring Reporters

    Order targets journalists' union chief Yehia Qalash, Khaled al-Balshy and Gamal Abdel Rahim for allegedly spreading false news, harboring fugitive colleagues

    Nigerian Oil Production Falls as Militant Attacks Take Toll

    Country no longer Africa's petroleum king due to renewed militancy in its oil-producing region

    This forum has been closed.
    Comment Sorting
    Comments
         
    by: Stephanie from: Texas
    July 02, 2014 2:14 PM
    This is great new for those u suffer from cf. i lost my best friend to the diesease at age 16. i know she's smiling from heaven over this news
    In Response

    by: Kinga from: Port kennedy
    July 06, 2014 10:09 PM
    We a full of hope ! We have a 6 yo with Cf. Sorry for your loss.

    Featured Videos

    Your JavaScript is turned off or you have an old version of Adobe's Flash Player. Get the latest Flash player.
    New Chapter for Tunisia's Ennahdai
    X
    Lisa Schlein
    May 31, 2016 1:56 PM
    Tunisia’s moderate Islamist Ennahda party says it is separating its religious and political activities in a broader bid to mark its so-called Muslim Democratic identity. The move appears to open a new chapter for a party that bounced back from the political wilderness of Tunisia’s pre-revolution days to become a key player in the North African country, and a member of the current coalition government. From Tunis, Lisa Bryant takes a look at how Tunisians are viewing its latest step.
    Video

    Video New Chapter for Tunisia's Ennahda

    Tunisia’s moderate Islamist Ennahda party says it is separating its religious and political activities in a broader bid to mark its so-called Muslim Democratic identity. The move appears to open a new chapter for a party that bounced back from the political wilderness of Tunisia’s pre-revolution days to become a key player in the North African country, and a member of the current coalition government. From Tunis, Lisa Bryant takes a look at how Tunisians are viewing its latest step.
    Video

    Video New Mobile App Allows Dutch Muslims to Rate their Imams

    If a young Dutch-Moroccan app developer has his way, Muslims in the Netherlands will soon be able to rate their imams online. Mohamed Mouman says imams rarely get feedback from their followers. He believes his app can give prayer leaders a better picture of what's happening in their communities — and can also keep young people from being radicalized. Serginho Roosblad reports from Amsterdam.
    Video

    Video Moscow Condemns NATO Plans to Beef Up Defense in Eastern Europe, Baltics

    NATO Secretary General Jens Stoltenberg said Monday an upcoming "landmark summit" will enhance the alliance's defensive and deterrent presence in eastern Europe and the Baltics. He is visiting Poland ahead of the NATO Summit in Warsaw. Zlatica Hoke reports
    Video

    Video Tech Startups Showcase Wares at Amsterdam Conference

    More than 20,000 tech enthusiasts, entrepreneurs and lovers of digital technology came together in Amsterdam recently at the Next Web Conference to discuss the latest developments in digital technology, look to the future and, of course, to connect. In recent years, there has been an explosion of so-called startup businesses that have created devices and applications that have changed the way we live; but, as Serginho Roosblad reports for VOA, there are pitfalls for such startups as well.
    Video

    Video US Military's Fallen Honored With Flags

    Memorial Day is a long weekend for most Americans. For some, it is the unofficial start of summer -- local swimming pools open and outdoor grilling season begins. But Memorial Day remains true to its origins -- a day to remember the U.S. military's fallen.
    Video

    Video Rolling Thunder Rolls Into Washington

    The Rolling Thunder caravan of motorcycles rolled into Washington Sunday, to support the U.S. military on the country's Memorial Day weekend
    Video

    Video A New Reading Program Pairs Kids with Dogs

    Dogs, it is said, are man's best friend. What some researchers have discovered is that they can also be a friend to a struggling reader. A group called Intermountain Therapy Animals trains dogs to help all kinds of kids with reading problems — from those with special needs to those for whom English is a second language. Faiza Elmasry has more on the New York chapter of R.E.A.D., or Reading Education Assistance Dogs, in this piece narrated by Faith Lapidus.
    Video

    Video Fan Base Grows for Fictional Wyoming Sheriff Longmire

    Around the world, the most enduring symbol of the U.S. is that of the cowboy. A very small percentage of Americans live in Western rural areas, and fewer still are cowboys. But the fascination with the American West is kept alive by such cultural offerings as “Longmire,” a series of books and TV episodes about a fictional Wyoming sheriff. VOA’s Greg Flakus recently spoke with Longmire’s creator, Craig Johnson, and filed this report from Houston.
    Video

    Video Chinese-Americans Heart Trump, Bucking National Trend

    A new study conducted by three Asian-American organizations shows there are three times as many Democrats as there are Republicans among Asian-American voters, and they favor Hillary Clinton over Donald Trump. But one group, called Chinese-Americans For Trump, is going against the tide and strongly supports the business tycoon. VOA’s Elizabeth Lee caught up with them at a Trump rally and reports from Anaheim, California.
    Video

    Video Reactions to Trump's Success Polarized Abroad

    What seemed impossible less than a year ago is now almost a certainty. New York real estate mogul Donald Trump has won the number of delegates needed to secure the Republican presidential nomination. The prospect has sparked as much controversy abroad as it has in the United States. Zlatica Hoke has more.
    Video

    Video F-35 Fighter Jet Draws Criticisms as Costs Mount

    America’s latest fighter plane, the F-35, has been mired in controversy. Critics cite cost, faulty design, and the attempt to use it to fill multiple roles. Even the pilot’s helmet is controversial. VOA’s Bernard Shusman reports from New York.
    Video

    Video Concerns Over Civilian Suffering as Iraqi Forces Surround Fallujah

    Thousands of residents are trapped inside the IS-held city ahead of a full scale Iraqi offensive aimed at retaking it.

    Special Report

    Adrift The Invisible African Diaspora