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

Analyst: Joint-Arab Military Force Poses Perilous Challenge

Although international forces are desperately needed to counter the threat of the Islamic State group, analysts say conflicting alliances could escalate fighting More

Asia’s Middle Class Changes Demand for Wheat Grain Exporters

Changes in tastes and diets are boon for wheat exporters such as Australia and the United States More

S. African Comedian Taking Over Popular TV Show

Mixed-race comedian Trevor Noah, who is loved for his edgy jibes about race and language, is taking the helm from Jon Stewart at The Daily Show in US 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.
Film Tells Story of Musicians in Mali Threatened by Jihadistsi
X
Greg Flakus
March 30, 2015 6:48 PM
At this year's annual South by Southwest film and music festival in Austin, Texas, some musicians from Mali were on hand to promote a film about how their lives were upturned by jihadists who destroyed ancient treasures in the city of Timbuktu and prohibited anyone from playing music under threat of death. As VOA’s Greg Flakus reports from Austin, some are afraid to return to their hometowns even though the jihadists are no longer in control there.
Video

Video Film Tells Story of Musicians in Mali Threatened by Jihadists

At this year's annual South by Southwest film and music festival in Austin, Texas, some musicians from Mali were on hand to promote a film about how their lives were upturned by jihadists who destroyed ancient treasures in the city of Timbuktu and prohibited anyone from playing music under threat of death. As VOA’s Greg Flakus reports from Austin, some are afraid to return to their hometowns even though the jihadists are no longer in control there.
Video

Video With Coalition Airstrikes, Iraq Entering 'Last Page' of IS Battle

American warplanes joined Iraq's battle against the so-called 'Islamic State' in northern Iraq late Wednesday, as Iraqi ground troops launched a massive assault on Tikrit. Analysts say the offensive could take the coalition a step further towards Mosul, the largest city held by Islamic State forces. Others say it could also deepen already-dangerous sectarian tensions in the region. VOA's Heather Murdock has more from Cairo.
Video

Video Philippines Wants Tourists Spending Money at New Casinos

Tourism is a multi-billion dollar industry in the Philippines. Close to five million foreign visitors traveled there last year, perhaps lured by the country’s tropical beaches. But Jason Strother reports from Manila that the country hopes to entice more travelers to stay indoors and spend money inside new casinos.
Video

Video Civilian Casualties Push Men to Join Rebels in Ukraine

The continued fighting in eastern Ukraine and the shelling of civilian neighborhoods seem to be pushing more men to join the separatist fighters. Many of the new recruits are residents of Ukraine made bitter by new grievances, as well as old. VOA's Patrick Wells reports.
Video

Video Islamic State Prisoners Talk of Curiosity, God, Regret

Islamic State fighter, a prisoner of Kurdish YPG forces, asked his family asking for forgiveness: "I destroyed myself and I destroyed them along with me." The Syrian youth was one of two detainees who spoke to VOA’s Kurdish Service about the path they chose; their names have been changed and identifying details obscured. VOA's Zana Omer reports.
Video

Video Germanwings Findings Raise Issue of Psychological Testing for Pilots

More is being discovered about the co-pilot in the crash of Germanwings Flight 9525 in the French Alps. Investigators say he was hiding a medical condition, raising questions about the mental qualifications of pilots. VOA's Carolyn Presutti reports.
Video

Video Hi-tech Motorbike Helmet's Goal: Improve Road Safety

In cities with heavily congested traffic, people can get around much faster on a motorcycle than in a car. But a rider who is not sure of his route may have to stop to look at the map or consult a GPS. A Russian start-up company is working to make navigation easier for motorcyclists. Designers at Moscow-based LiveMap are developing a smart helmet with a built-in navigation system, head-mounted display and voice recognition. Zlatica Hoke has more.
Video

Video DOJ: Illinois National Guard Soldier Tried to Join ISIS

U.S. federal law enforcement agents arrested two suburban Chicago men accused of trying to join ISIS overseas, while also plotting attacks in the United States. As VOA’s Kane Farabaugh reports from the Midwest state of Illinois, one of those arrested is a soldier of the Illinois National Guard.
Video

Video New Wheelchair Is Easier to Use, Increases Mobility

Traditional push-rim wheelchairs create a lot of stress for arm, shoulder and neck muscles and joints. A redesigned chair, based on readily available bicycle technology, radically increases mobility while reducing the physical effort. VOA’s George Putic reports.
Video

Video Liberia's Almost Last Ebola Patient Grateful but Still Grieving

Beatrice Yardolo was to make history as Liberia’s last Ebola patient. Liberians recently started counting down 42 days, the period that has to go by without a single new infection until the World Health Organization can declare a country Ebola-free. That countdown stopped on March 20 when there was another new case of Ebola, making Yardolo’s story a reminder that Ebola is far from over. Benno Muchler reports from Monrovia.
Video

Video Cambodian Land Grabs Threaten Traditional Communities

Indigenous communities in Cambodia's Ratanakiri province say the government’s economic land concession policy is taking away their land and traditional way of life, making many fear that their identity will soon be lost. Local authorities, though, have denied this is the case. VOA's Say Mony went to investigate and filed this report, narrated by Colin Lovett.
Video

Video Space Program Status Disappoints 'Last Man on the Moon'

One of the films that drew big crowds last week at the annual South by Southwest festival in Austin, Texas, tells the story of the last human being to stand on the moon, U.S. astronaut Eugene Cernan. It has been 42 years since Cernan returned from the moon and he laments that no one else has gone there since. VOA’s Greg Flakus reports.

VOA Blogs

Circumventing Censorship

An Internet Primer for Healthy Web Habits

As surveillance and censoring technologies advance, so, too, do new tools for your computer or mobile device that help protect your privacy and break through Internet censorship.
More