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

Video Migrants Caught in No-Man's Land In French Port

The deaths of hundreds of migrants in the Mediterranean this week has only recast the spotlight on the perils of reaching 'Fortress Europe' More

Video Westgate Mall Attack Survivors Confront Painful Memories

On anniversary of terror attack, survivors discuss how they have coped with trauma they experienced that day More

New Hints That Dark Matter Exists

New evidence from International Space Station hints at existence of dark matter and dark energy More

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.
Migrants Caught in No-Man's Land Called Calaisi
X
Lisa Bryant
September 19, 2014 5:04 PM
The deaths of hundreds of migrants in the Mediterranean this week has only recast the spotlight on the perils of reaching Europe. And for those forunate enough to reach a place like Calais, France, only find that their problems aren't over. Lisa Bryant has the story.
Video

Video Migrants Caught in No-Man's Land Called Calais

The deaths of hundreds of migrants in the Mediterranean this week has only recast the spotlight on the perils of reaching Europe. And for those forunate enough to reach a place like Calais, France, only find that their problems aren't over. Lisa Bryant has the story.
Video

Video Westgate Siege Anniversary Brings Back Painful Memories

One year after it happened, the survivors of the terror attack on Nairobi's Westgate Shopping Mall still cannot shake the images of that tragic incident. For VOA, Mohammed Yusuf tells the story of victims still waiting for the answer to the question 'how could this happen?'
Video

Video Militant Assault in Syria Displaces Thousands of Kurds

A major assault by Islamic State militants on Kurds in Syria has sent a wave of new refugees to the Turkish border, where they were stopped by Turkish border security. Turkey is already hosting about 700,000 Syrian refugees who fled the civil war between the government and the opposition. But the government in Ankara has a history of strained relations with Turkey's Kurdish minority. Zlatica Hoke reports Turkey is asking for international help.
Video

Video CERN Accelerator Back in Business

The long upgrade of the Large Hadron Collider is over. The scientific instrument responsible for the discovery of the Higgs boson -- the so-called "God particle" -- is being brought up to speed in time for this month's 60th anniversary of the European Organization for Nuclear Research, known by its French acronym CERN. Physicists hope the accelerator will help them uncover more secrets about the origins of the universe. VOA’s George Putic reports.
Video

Video Whaling Summit Votes to Uphold Ban on Japan Whale Hunt

The International Whaling Commission, meeting in Slovenia, has voted to uphold a court ruling banning Japan from hunting whales in the Antarctic Ocean. Conservationists hailed the ruling as a victory, but Tokyo says it will submit revised plans for a whale hunt in 2015. Henry Ridgwell reports from London.
Video

Video Russian Economy Reeling After New Western Sanctions

A new wave of Western sanctions is hitting Russia’s economy hard. State-owned energy firms continue to bleed profits and Russia’s national currency plunged to a new low this week after the U.S. and the European Union announced new sanctions to punish Russia's aggressive stance in eastern Ukraine. But as Mil Arcega reports, the sanctions could also prove costly for European and American companies.
Video

Video Belgian Researchers Discover Way to Block Cancer Metastasis

Cancer remains one of the deadliest diseases, despite many new methods to combat it. Modern medicine has treatments to prevent the growth of primary tumor cells. But most cancer deaths are caused by metastasis, the stage when primary tumor cells change and move to other parts of the body. A team of Belgian scientists says it has found a way to prevent that process. Zlatica Hoke has more.
Video

Video Mogadishu's Flood of Foreign Workers Leaves Somalis Out of Work

Unemployment and conflict has forced many young Somalians out of the country in search of a better life. But a newfound stability in the once-lawless nation has created hope — and jobs — which, some say, are too often being filled by foreigners. Abdulaziz Billow reports from Mogadishu.
Video

Video A Dinosaur Fit for Land and Water

Residents and tourists in Washington D.C. can now examine a life-size replica of an unusual dinosaur that lived almost a hundred million years ago in northern Africa. Scientists say studying the behemoth named Spinosaurus helps them better understand how some prehistoric animals adapted to life on land and in water. The Spinosaurus replica is on display at the National Geographic museum. VOA’s George Putic has more.
Video

Video Iraqi Kurdistan Church Helps Christian Children Cope find shelter in churches in the Kurdish capital, Irbil

In the past six weeks, tens of thousands of Iraqi Christians have been forced to flee their homes by Islamic State militants and find shelter in churches in the Kurdish capital, Irbil. Despite U.S. airstrikes in the region, the prospect of people returning home is still very low and concerns are starting to grow over the impact this is having on the displaced youth. Sebastian Meyer reports from Irbil on how one church is coping.
Video

Video NASA Picks Boeing, SpaceX to Carry Astronauts Into Space

The U.S. space agency, NASA, has chosen Boeing and SpaceX companies to build the next generation of spacecraft that will carry U.S. astronauts to the International Space Station by the year 2017. The deal with private industry enables NASA to end its dependence on Russia to send space crews into low Earth orbit and back. Zlatica Hoke has more.
Video

Video Future of Ukrainian Former President's Estate Uncertain

More than six months after Ukraine's former President Viktor Yanukovych fled revolution to Russia, authorities have yet to gain control of his palatial estate. Protesters occupy the grounds and opened it to tourists but they are also refusing to turn it over to the state. VOA's Daniel Schearf reports from Mezhigirya, just north of Kyiv.


Carnage and mayhem are part of daily life in northern Nigeria, the result of a terror campaign by the Islamist group Boko Haram. Fears are growing that Nigeria’s government may not know how to counter it, and may be making things worse. More

AppleAndroid