Children's Hospital Colorado

Dylan: Kalydeco™ Offers Hope to Fight Cystic Fibrosis

Taking the FIGHT to Cystic Fibrosis

Thanks to a revolutionary medication, Dylan Stellar —a 6-year-old with cystic fibrosis — has made remarkable progress.

Two years ago, Dylan was diagnosed with cystic fibrosis (CF), a rare genetic condition that causes mucus to build up and impact the function of vital organs, such as the lungs or pancreas. When Dylan was born in 2006, CF testing for newborns was not mandatory, so his condition went undetected until he moved to Colorado with his family in 2008.

“Dylan had a history of chronic respiratory infections, slow growth and low weight gain,” said Dylan’s mother, Camille Stellar Fontana. “When he was admitted to the hospital with pneumonia, the doctors recognized the symptoms and decided to perform a CF test. Dylan’s diagnosis was absolutely devastating for my family.”

Dylan was transferred to Children’s Hospital Colorado, where he was treated by Edith Zemanick, MD, MSCS, Pediatric Pulmonologist. 

Dr. Zemanick placed Dylan on a regimen of medications, as well as physical therapy and pancreatic enzymes. Although these treatments significantly improved Dylan’s symptoms, he still suffered from the respiratory problems and low weight gain associated with CF.

Advancing toward a cure

In late 2009, Camille learned of a new medication for CF, known as Kalydeco™. This medication offered hope for patients with a rare form of the genetic mutation responsible for CF. About 4% of CF patients — Dylan included — have the G551D mutation Kalydeco treats. 

A research team at Children’s Colorado helped test Kalydeco during the second phase of drug trials. The team was led by Frank Accurso, MD, Medical Director of the Core Laboratory at Children’s Colorado Clinical Translational Research Center. Through sweat analysis from CF patients around the country, Dr. Accurso’s team worked tirelessly to test the safety and effectiveness of Kalydeco, and found that it restored the function of the faulty protein present in CF patients.

“This study showed Kalydeco improves lung function, lowers risk for respiratory infections, decreases hospitalizations and helps children with CF experience normal weight gain,” said Dr. Zemanick. “It isn’t a cure, but it provides an important road forward for everyone who has the condition.”

Dylan began taking the new medication when he turned 6 in March. Although he will still require long-term specialized care at Children’s Colorado, his condition has significantly improved. 

“As a parent, this is such a dream come true,” Camille said. “Dylan has started growing and gaining weight, and he’s missing fewer school days. Overall he’s feeling much better.”

The Mike McMorris Cystic Fibrosis Research and Care Center at Children’s Hospital Colorado is the largest CF clinical care center in the country and the only pediatric hospital in the state accredited by the National CF Foundation.