Ft. Collins boy participating in cystic fibrosis clinical study

Noah Colbert started going to school this past year. Besides being especially vigilant about washing his hands and taking pills to help him digest fats, he is just like any other four-year-old student. For 15 minutes in the morning and at night, however, he inhales a solution through a nebulizer as part of a clinical trial through The Children’s Hospital in Denver.

Noah lives in Ft. Collins with his parents, Jim and Libby. He was diagnosed with cystic fibrosis when he was two and half weeks old. Cystic fibrosis is an inherited disease that affects one’s lungs and digestive system. The disease’s defective gene and its resulting protein cause the body to produce excessively thick, sticky mucous that clogs the patient’s lungs. Those with cystic fibrosis are much more prone to lung infections.

Noah goes to The Children’s Hospital every six weeks for checkups on the study which, if successful, could lead to new information about how to help cystic fibrosis patients live for several decades past the current mid-30s life expectancy.

Scott Sagel, Noah’s pediatric pulmonologist, said that neither he nor Libby Colbert knows if the solution Noah is breathing in every day is the drug – a hypertonic saline solution – or a placebo. The drug is a seven percent saline solution, which is about twice as salty as the Atlantic Ocean, Sagel said. Currently, the solution is used as a treatment for older children and adults with cystic fibrosis. The Children’s Hospital and other clinics around the country are participating in the study to test the safety and efficacy of the solution in younger children.

“We believe that a hypertonic saline solution improves mucous clearance both by promoting coughing and by hydrating one’s airways,” Sagel said. “We’re evaluating this therapy in toddlers and young children to see if it’s both safe and tolerated, but also to see if it improves lung function in children like Noah.”
Libby Colbert said she and her husband didn’t hesitate before deciding to participate in the trial, which he began in September 2009.

“It was a fairly easy decision because we were already researching,” she said. “Information for us is comfort”
In addition to participating in the study, Noah wears a special vest twice a day that shakes him enough to loosen mucous in his lungs. Other treatments for cystic fibrosis, like a drug called Pulmozyme, are very expensive, Libby said. The hypertonic saline solution would be a much more cost effective treatment option.

Noah’s family and doctors won’t know if he was breathing the solution or a placebo until August, when the study ends. Libby Colbert said she suspects he is receiving the drug, as he has had fewer infections this year than in previous years – before he started the trial.
“He was on antibiotics [for infections] 8-10 times his first few years,” Libby Colbert said. “He’s only been on them five times since the study started.”

Sagel said he is optimistic about the study and its possibilities.

“If this works by enhancing and improving mucous clearance, could help to prevent lung disease, injury and progression [of the illness] and improve quality of life and prolong life,” he said.

For now, Noah will continue to live his life to the fullest as he approaches his fifth birthday in July.

“Looking at Noah you would never know that he has cystic fibrosis,” Libby Colbert said. “He does a little extra to stay healthy but he’s a happy-go-lucky kid.”