Breakthrough Drug Brings New Hope for Cystic Fibrosis Patients

May 25, 2012

The director of UNMC’s Adult Cystic Fibrosis program says he’s excited about a new treatment called Kalydeco.

Dr. Peter J. Murphy says Kalydeco is a drug that specifically targets a certain class of genetic mutation that causes cystic fibrosis in some patients.  Cystic fibrosis is a disease that primarily affects the lungs, with thick secretions that may sometimes become infected and make it difficult for patients to breathe.  

Dr. Murphy says the drug was recently approved by the FDA, and the few CF patients he has with that particular genetic mutation are taking Kalydeco.  He says so far the drug has had an amazing effect on many of the patients.

"I think everyone has seen increases in their lung function and a decrease in the number of times they have to be treated for exacerbations, for worsening of their lung infections.”

Dr. Murphy says Kalydeco is only effective on patients with the G551D mutation of CF.  He says that’s only about 4% of the CF population.  

The most common mutation is Delta F508 which affects 90% of patients with CF.  Dr. Murphy says studies are already underway to find ways to combine Kalydeco with other medications to target that population.  

The goal of UNMC’s Adult Cystic Fibrosis program is to provide the best possible care for adults with CF.