A new medication may allow some people with cystic fibrosis to live near full lifespans, says Ran Anbar, MD, director of Pediatric Pulmonary Medicine at Upstate University Hospital.
People with CF have thick mucus that plugs the lungs and digestive tract, because their bodies either do not make a particular protein or make a defective version of the protein. In the 1950s, babies born with CF lived only a couple of years. Statistics today project a lifespan of about 40 years. Anbar says this is continuing to improve.
Ivacaftor (Kalydeco) is the first drug to treat the underlying cause of CF, a disease that is inherited when both parents carry defective genes. Since it was approved by the Food and Drug Administraiton in 2012, the drug has been used to help thin the mucus in patients with a small number of CF protein mutation. More than 150 different mutations cause the disease.
“For the 4 percent of patients who received it, it’s been marvelous. Their lung function improved. Their weight has improved. They feel better,” Anbar says. “This drug is powerful, and we’re still learning how it works.”
He says ivacaftor is being researched in combination with another drug, lumacaftor, for people with other types of CF. Recently, this combination was shown to be effective for patients with CF who carry the most common mutation combination, which affects about half of patients with CF. Ivacaftor/lumacaftor may be available for prescription to CF patients in early 2015. In combination with a third drug that is under development, this pair may be even more effective.
Of the estimated 30,000 Americans with CF, 225 live in Central New York and receive their care at Upstate.
Hear an interview with Dr. Anbar about Ivacaftor