Approximately 300 children born in Indiana this year will be diagnosed with cystic fibrosis (CF) or identified as a carrier for the disease, according to health officials. May is National Cystic Fibrosis Awareness Month. During this time, volunteers and people with CF and their families unite to help shine a spotlight on what CF is and the progress being made toward finding a cure.
Cystic fibrosis is an inherited chronic disease that affects the lungs and digestive system of about 30,000 children and adults in the United States. People with CF have two genetic changes that cause the body to produce unusually thick, sticky mucus that clogs the lungs, obstructs the pancreas and stops natural enzymes from helping the body break down and absorb food. Those with CF commonly experience frequent respiratory infections and digestive problems, such as failure to gain weight.
“Fifty years ago, most kids diagnosed with cystic fibrosis weren’t expected to live to attend school,” said State Health Commissioner Gregory Larkin, M.D. “Thanks to newborn screening and medical advances, most individuals with CF can now expect to live into their 30s and 40s. While this is a significant improvement, much more needs to be done to find improved treatments and a cure to this devastating disease.”
In Indiana, most children with CF are diagnosed within the first few months of life, meaning they receive treatment earlier and have an overall improved quality of life. Indiana has chosen to follow the national precedent by referring patients to Cystic Fibrosis Foundation-accredited centers for sweat chloride testing (to confirm CF in any person with a positive CF newborn screen) and/or management of the disease. Prior to CF being added to the Indiana’s newborn screen in 2007, most children with CF were not diagnosed until about age 4.
Individuals with CF undergo a strict daily treatment regimen, which involves taking dozens of pills and using a high-frequency chest wall oscillation (HFCWO) vest to break up the mucus in their lungs daily.
“Due to a limited CF patient population worldwide, the Cystic Fibrosis Foundation must fund its own research and drug development,” said Joanna Whitaker, Development Director of the Indiana Chapter of the Cystic Fibrosis Foundation. “In recent years, the groundbreaking drug, Kalydeco, has made a huge improvement in the quality of life for about 4 percent of patients battling CF. The Foundation, along with Vertex Pharmaceuticals, has been working feverishly toward the next step to help the remaining 96 percent of those who suffer from CF. We’re not there yet but are so very, very close.”
Last week, Vertex announced unexpectedly positive interim results from a clinical trial of Kalydeco in combination with VX-809, a CF drug in development. The results showed a significant improvement in lung function in people with two copies of the most common CF genetic mutation.
Hoosiers can help support cystic fibrosis awareness this month by participating in one of the many Great Strides Walks taking place around the state. On May 19 and 20, walkers will put their best foot forward at 13 locations across the state and more than 600 sites nationwide—all to help find a cure for CF.
To find a Great Strides walk in your area or to register, visit www.cff.org/Chapters/indiana/greatstrides/.
To learn more about Cystic Fibrosis, visit the Indiana State Department of Health’s website at www.StateHealth.in.gov or the Indiana Chapter of the Cystic Fibrosis Foundation at www.cff.org/Chapters/indiana/.