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New treatment could help with cystic fibrosis

August 28, 2008 12:00:00 AM PDT
Researchers are working on a new experimental drug that targets the cause of cystic fibrosis. The preliminary research is giving hope to patients who fight cystic fibrosis every day. Facing a pile of pills is part of 19-year-old Elyse Campbell's daily routine. Breathing isn't easy either, twice a day Elyse needs a lung treatment.

"The vibration breaks up the mucus and helps me cough it out so that I can breathe better," said Campbell.

It's all part of living with cystic fibrosis, a disease linked to a faulty protein called CFTR.

People with cystic fibrosis suffer from an imbalance of water and salt. It causes thick mucus and inflammation in the lungs and other organs, sometimes leading to infection. There is no cure, but there is new hope.

Pulmonologist Dr. Steven Rowe is leading a research team that's testing a new drug called v-x-seven-seventy.

"Oh, it's one of the more exciting things that's happened in CF research," said Dr. Rowe.

CF patients who took the drug for two weeks showed significant improvement in lung function and sweat chloride levels, indicating the drug may fix the faulty protein.

"It really allows us to be very optimistic that our strategies to try and attack the underlying defect," said Dr. Rowe.

Now, as the v-x-seven-seventy trial begins the next phase, a very excited Elyse is among the participants.

"If I didn't have to deal with this for at least a day, that would be amazing," said Campbell.

The Cystic Fibrosis Foundation has invested $79 million in the v-x-seven-seventy research project.

Even if it doesn't prove to be a cure, patients like Elyse hope it will help make life easier for her and the thousands of others with CF.

Web Extra Information:

BACKGROUND: Cystic fibrosis is an inherited chronic disease that affects the lungs and digestive system. About 30,000 adults and children in the United States suffer from cystic fibrosis. Worldwide there are about 70,000 sufferers.

According to the Cystic Fibrosis Foundation, the disease causes a defective gene to produce a protein that makes the body produce a thick, sticky mucous that can clog the lungs and lead to lung infections. It can also obstruct the pancreas, stopping natural enzymes that help the body break down food. About 1,000 new cases of cystic fibrosis are diagnosed each year and 70 percent of patients are diagnosed by age two.

Cystic fibrosis can produce an array of symptoms including, but not limited to:

  • Very salty tasting skin
  • Persistent coughing, sometime accompanied by phlegm
  • Frequent lung infections
  • Wheezing or shortness of breath
  • Poor growth/weight gain, despite normal appetite
  • Frequent greasy, bulky stools or difficulty in bowl movements
TREATMENTS: People suffering from cystic fibrosis must perform airway-clearing techniques (ACT) to loosen or get rid of the mucous from their lungs. ACTs may be done on one's own or with the help of another person. One technique commonly used is called postural drainage percussion. It involves the patient standing or sitting in a position that will help clear mucous while another person pounds of claps on his or her chest. Other options include blowing into a devise that shakes mucous out and using a mechanical vest to loosen mucous. Certain drugs are also helpful in treating the symptoms of cystic fibrosis. Mucolytics are used to thin mucous so it can be coughed out easier. Antibiotics to treat any infection in the lungs are crucial. Inhaled antibiotics are common for this type of treatment. Hypertonic saline is also used to draw water into their airways making it easier to cough.

NEW HOPE: Currently, a new drug called VX-770 is being tested for the treatment of cystic fibrosis, with promising results. The drug specifically targets the protein that's responsible for cystic fibrosis. The protein, called a chloride channel protein, in effect causes a door in the body to shut too tightly. Research shows that VX-770 is able to repair the protein, thus making the door in the body open and shut regularly. After 14 days on the drug, study participants showed significant improvements in lung function, nasal potential difference measurements and sweat chloride levels. This is the first time any potential therapy has improved the abnormal sweat chloride levels in a patient with cystic fibrosis.

 

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