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

Obama: Alaskans Feel Signs of Climate Change

They're seeing bigger storm surges as sea ice melts, more wildfires, erosion of glaciers, shorelines More

Katrina Brought Enduring Changes to New Orleans

The city’s recovery is the result of the people and culture the city is famous for, as well as newcomers and start-up industries More

Magical Photo Slides Show Native Americans in Late 1800s

Walter McClintock spent 20 years photographing the Blackfoot Indians and their vanishing culture at the dawn of the modern age 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.
Colombians Flee Venezuela as Border Crisis Escalatesi
X
August 27, 2015 2:08 AM
Hundreds of Colombians have fled Venezuela since last week, amid an escalating border crisis between the two countries. Last week, Venezuelan President Nicolas Maduro ordered the closure of a key border crossing after smugglers injured three Venezuelan soldiers and a civilian. The president also ordered the deportation of Colombians who are in Venezuela illegally. Zlatica Hoke reports.
Video

Video Colombians Flee Venezuela as Border Crisis Escalates

Hundreds of Colombians have fled Venezuela since last week, amid an escalating border crisis between the two countries. Last week, Venezuelan President Nicolas Maduro ordered the closure of a key border crossing after smugglers injured three Venezuelan soldiers and a civilian. The president also ordered the deportation of Colombians who are in Venezuela illegally. Zlatica Hoke reports.
Video

Video Is China's Economic Data Accurate?

Some investors say China's wild stock market gyrations have been made worse by worries about the reliability of that nation's economic data. And some critics say the reports can mislead investors by painting an unrealistically-strong picture of the economy. A key China scholar says Beijing is not fudging ((manipulating)) the numbers, but that the economy is evolving quickly from smoke-stack industries to services, and the ways of tracking new economic activity are falling behind the change. V
Video

Video Next to Iran, Climate at Forefront of Obama Agenda

President Barack Obama this week announced new initiatives aimed at making it easier for Americans to access renewable energy sources such as solar and wind. Obama is not slowing down when it comes to pushing through climate change measures, an issue he says is the greatest threat to the country’s national security. VOA correspondent Aru Pande has more from the White House.
Video

Video Shipping Containers Provide Experimental Housing

Housing prices around the San Francisco Bay area are out of reach for many people, so some young entrepreneurs, artists and tech industry workers are creating their own houses using converted shipping containers. But as VOA's Mike O’Sullivan reports from Oakland, the effort requires ingenuity and dealing with restrictive local laws.
Video

Video Arctic Draws International Competition for Oil

A new geopolitical “Great Game” is underway in earth’s northernmost region, the Arctic, where Russia has claimed a large area for resource development and President Barack Obama recently approved Shell Oil Company’s test-drilling project in an area under U.S. control. Greg Flakus reports.
Video

Video Philippine Maritime Police: Chinese Fishermen a Threat to Country’s Security

China and the Philippines both claim maritime rights in the South China Sea.  That includes the right to fish in those waters. Jason Strother reports on how the Philippines is catching Chinese nationals it says are illegal poachers. He has the story from Palawan province.
Video

Video Technique May Eliminate Drill-and-Fill Dental Care

Many people dread visiting dentists because they're afraid of drills. Now, however, a technology developed by a British firm promises to eliminate the need for mechanical cleaning of dental cavities by speeding a natural process of tooth repair. VOA’s George Putic reports.
Video

Video China's Spratly Island Building Said to Light Up the Night 'Like A City'

Southeast Asian countries claim China has illegally seized territory in the Spratly islands. It is especially a concern for a Philippine mayor who says Beijing is occupying parts of his municipality. Jason Strother reports from the capital of Palawan province, Puerto Princesa.
Video

Video Ages-old Ice Reveals Secrets of Climate Change

Ice caps don't just exist at the world's poles. There are also tropical ice caps, and the largest sits atop the Peruvian Andes - but it is melting, quickly, and may be gone within the next 20 years. George Putic reports scientists are now rushing to take samples to get at the valuable information about climate change locked in the ice.
Video

Video French Experiment in Integrating Roma Under Threat

Plans to destroy France’s oldest slum have sparked an outcry on the part of its Roma residents. As Lisa Bryant reports from the Paris suburb of La Courneuve, rights groups argue the community is a fledgling experiment on integrating Roma who are often outcasts in many parts of Europe.
Video

Video Kenyans Turn to Agriculture for Business

Each year Kenyan universities continue to churn out graduates for the job market despite the already existing high rate of unemployment among youth in the country. Some of these young men and women have realized that agriculture can be as rewarding as any other business or job, and they are resorting to agribusiness in large numbers as a way of tackling unemployment. Rael Ombuor reports for VOA.
Video

Video First Women Graduate Elite Army Ranger School

Two women are making history for the U.S. Army by proving they are among the toughest of the tough. VOA's Carla Babb reports from Fort Benning, Georgia as 94 men and those two women rise as graduates of the difficult Ranger school.

VOA Blogs