SHORT ANSWER
A More Effective Channel
PULMONOLOGY
Since the first modulator treatments for cystic fibrosis were approved in 2012 , they ’ ve been used primarily to treat kids older than 12 . And they ’ re effective . Cystic fibrosis is caused by various mutations to the gene CFTR , which makes proteins that channel chloride in and out of cells ; modulators correct the function of those proteins .
The first class of drug to treat the underlying mechanisms of cystic fibrosis rather than just the symptoms , modulators have the potential to benefit younger children even more .
Abnormalities in CFTR protein function damage organ systems over time . Modulators might arrest that damage before it begins . But long-term safety outcomes for the most commonly used modulator have not been studied in young children — until now .
A study conducted at the Colorado Clinical and Translational Sciences Institute at Children ’ s Hospital Colorado ’ s Mike McMorris Cystic Fibrosis Research and Care Center , the largest center of its kind in the nation , showed that children ages 2 to 5 can safely tolerate lumacaftor-ivacaftor for up to 120 weeks . Lumacaftor-ivacaftor was the first modulator approved to treat the most common form of cystic fibrosis , caused by two copies of the F508del mutation .
“ The modulator therapy is preventive rather than reactive ,” says pediatric pulmonologist Jordana Hoppe , MD , the study ’ s lead author . “ With these findings , we believe kids can start treatments at a younger age and hopefully prevent the devastating consequences of cystic fibrosis , such as lung damage or disease , respiratory failure , the need for lung transplant or even premature death .”
The study also showed improvements in lung clearance , pancreatic function , weight gain and growth . It was published in the Lancet Respiratory Medicine ( 1 ) in May 2021 .
1 . Hoppe JE , Chilvers M , Ratjen PF , McNamara , JJ , et al . Long-term safety of lumacaftor – ivacaftor in children aged 2 – 5 years with cystic fibrosis homozygous for the F508del-CFTR mutation : a multicentre , phase 3 , open-label , extension study . 2021 May . Online .
JORDANA HOPPE , MD
Pediatric pulmonologist , Children ’ s Hospital Colorado
Assistant professor , Pediatrics-Pulmonary Medicine University of Colorado School of Medicine
Keep in Training
Q : When you ’ re the best at what you do , how do you leverage your expertise to help those you don ’ t directly care for ?
Nick Cost , MD , co-director of the Surgical Oncology Program at Children ’ s Colorado , is one of the nation ’ s few physicians who is fellowship trained as both a pediatric urologist and urologic oncologist . He operates on 10 to 12 pediatric renal tumors a year — an impressive amount , considering they ’ re rare . For context , most pediatric urologists , surgeons and oncologists care for one patient or less with a renal tumor each year .
Physicians who don ’ t commonly see these cases are exactly who Dr . Cost is hoping to reach as the newly appointed Surgical Discipline Lead of the Children ’ s Oncology Group ’ s ( COG ) Renal Tumor Committee .
Known widely as the world ’ s largest pediatric cancer research organization , COG works with more than 230 children ’ s hospitals across North America to lead pediatric cancer studies . It also provides protocol-dictated clinical guidelines . Dr . Cost isn ’ t the only Children ’ s Colorado specialist in a leadership position with COG . Pediatric oncologist Lia Gore , MD , serves as the COG vice chair , helping to oversee the entire operation .
For their part , Dr . Cost and the 13 other surgeons on the Renal Tumor Committee support all of the pediatric urologists , surgeons and oncologists in North America who care for kids with renal tumors . That work has primarily involved educating pediatric urologists and surgeons about COG ’ s trials : the format , the part a surgeon plays and how to ensure that surgical care remains within study guidelines .
More recently , the committee has worked to expand its role . “ We give advice and offer up our experiences on past cases to help surgeons across the continent prepare for and manage these tumors ,” says Dr . Cost .
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