It’s a rare disease that damages the lungs and the digestive system. An estimated 70,000 people around the world are living with Cystic Fibrosis. Although there’s still no cure – people with the genetic disorder are living longer. One drug made in the U.S. is benefiting a number of patients. But it remains off-limits to many in Europe.
CCTV America’s Hendrik Sybrandy reports.
Newly developed drug used to help treat cystic fibrosisTreating Cystic Fibrosis has bringing Fielder boys and their family a new hope. CCTV America's Hendrik Sybrandy reports.
In Henderson, Colorado, you’ll often find Josh and Matthew Fielder at their home away from home, the lacrosse field. Lacrosse is their favorite sport, a great form of exercise.
Josh said they’re practicing lacrosse to get better and build bonds with teammates.
Lacrosse also helps these teenagers’ lungs. Both boys have cystic fibrosis, a disorder marked by lung infections and digestive problems that requires them to spend hours each day clearing mucus from their airways and doing other treatments.
“It’s definitely a different lifestyle. You have to get used to all the treatments and medication,” Matthew said.
Not long ago, the Fielders added this drug to their mix of therapies. Orkambi is an oral tablet that treats patients with a genetic mutation that’s a leading cause of cystic fibrosis.
Dr. Scott Sagel from Children’s Hospital Colorado Pediatric Pulmonologist stated that this is one that is targeting the basic defect, the protein abnormality in the body so we do think of it as a disease modifying treatment, one that will be life extending for individuals with cystic fibrosis.
The Fielders’ doctor said the results have been encouraging.
Dr. Scott Sagel said he did sense some clinical improvement.
Josh and Matthew’s lung function and energy are up.
Orkambi does not come cheaply. The drug costs close to $300,000 per patient per year. U.S. health insurance generally covers patients like the Fielders. Access to the drug is much tougher in places like Europe.
Katie Murphy of Cystic Fibrosis Ireland said Orkambi is so expensive, her country’s public health service won’t prescribe the drug, not yet.
“I have friends with CF who could benefit from this therapy. I’m speaking with parents of children who are really very anxious of course understandably to get access to another therapy for their children,” said Murphy.
Josh and Matthew’s mother said Orkambi has given her family new hope.
Heather Fielder said each new drug could help prolong her sons’ lives and make the challenge of living with cystic fibrosis a little easier. As long as they do what they’re supposed to do, she said, the world is theirs.