Why There’s New Hope for Cystic Fibrosis
The outlook for people with cystic fibrosis (CF) is better than ever. Treatment advances include new drugs that act on CF’s genetic defects.
When cystic fibrosis (CF) was first described in 1939, patients lived only days or months. Today, CF patients live well into adulthood with a better quality of life. And the outlook is getting better as treatments advance.
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Here’s a look at two ways current treatments are bringing new hope for kids and adults with CF.
CF is caused by genetic defects. Researchers have identified more than 1,000 of them.
Defects hinder salt and water flow into and out of cells in the lungs and other organs. That makes the mucus extra thick and sticky, leading to long-term lung infections and other issues.
Historically, treating CF’s symptoms and complications was the best doctors could do. But in 2012, the U.S. Food and Drug Administration (FDA) approved a drug (ivacaftor) that gets to the root of the problem — the genetic defect in about 8 percent of CF patients. In 2015, the FDA approved a similar drug (ivacaftor combined with lumacaftor) that treats the defect in about 50 percent of CF patients.
“These drugs act on the defective protein,” says Elliott Dasenbrook, MD, who directs Cleveland Clinic’s adult cystic fibrosis program. “When the protein works better, mucus doesn’t build up as much and patients breathe easier.”
Even the amount of chloride in a patient’s sweat — a marker for CF — can become normal, he notes.
CF patients take this medication by pill twice daily.
Could you take it too? It depends on which mutation is causing your CF. A genetic (blood) test can identify it.
“Therapies that treat the basic defect are game-changers for CF,” says Dr. Dasenbrook. “We are working toward developing similar therapies for all our CF patients.”
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In addition to new medications, multidisciplinary care — involving physicians from various specialties, as well as nurses, nutritionists, respiratory therapists and social workers — also is improving the outlook for CF patients.
CF isn’t just a lung disease. The thick mucus that makes breathing difficult can block ducts in the pancreas. That causes digestive issues and sometimes malnutrition.
CF also can affect the liver, sinuses, intestines and reproductive organs.
“Comprehensive care has improved the prognosis for many people,” says Silvia Cardenas, MD, a pediatric pulmonologist at Cleveland Clinic. “Multidisciplinary teams can provide preventive care, to help keep lungs, gastrointestinal tract and other organs healthy. They provide nutritional support, address psychological challenges of chronic disease and more.”
As CF patients are living longer, medical teams also are addressing more adult issues, such as fertility. Medical centers like Cleveland Clinic now include adult cystic fibrosis programs.
“With more multidisciplinary care and more medications to treat genetic defects, the health and longevity of CF patients will continue to improve,” says Dr. Dasenbrook.