Nav: Home

Cystic fibrosis drug combo found safe and effective for 2- to 5-year-olds

January 24, 2019

An open label Phase 3 study conducted at Ann & Robert H. Lurie Children's Hospital of Chicago and other centers established that a combination of cystic fibrosis drugs lumacaftor and ivacaftor is safe and effective in children aged 2-5 years, whose disease is caused by two copies of F508del-CFTR gene mutation - the most common and severe form of cystic fibrosis. These data, published in Lancet Respiratory Medicine, were the basis for the Food and Drug Administration's extended approval for this treatment to include children 2 years and older. Previously, the drug combination was approved for patients 6 years and older.

"With earlier treatment that targets the genetic cause of cystic fibrosis we hope to arrest disease progression and substantially improve long-term outcomes," says study author Susanna A. McColley, MD, from Stanley Manne Children's Research Institute at Lurie Children's and Professor of Pediatrics at Northwestern University Feinberg School of Medicine. "In this study, we also saw evidence of improved pancreatic function, which suggests that we might be able to reverse damage to the pancreas from cystic fibrosis."

Cystic fibrosis is a progressive genetic disease that damages multiple organs, including the lungs and pancreas, with average predicted survival of 47 years. It is caused by mutations in the CFTR gene that lead to insufficient flow of salt and water in and out of cells. In the lungs, this creates buildup of thick, sticky mucus that can result in chronic lung infections and severe lung disease. Damage to the pancreas occurs even before birth, which interferes with nutrition absorption and growth.

"Our study also showed that treatment with lumacaftor/ivacaftor can improve growth for children with cystic fibrosis," says Dr. McColley. "We saw significant increases in weight, height and body mass index over six months of treatment."

In addition to efficacy, the study also confirmed that lumacaftor/ivacaftor is well tolerated in the younger population. No new safety concerns were identified.

Dr. McColley is now leading an open label Phase 3 trial of this drug combination for children 1-2 years of age.
-end-
The study was funded by Vertex Pharmaceuticals Inc. Dr. McColley has received personal fees as a speaker for Vertex Pharmaceuticals Inc., but not for the conduct of this research.

Research at Ann & Robert H. Lurie Children's Hospital of Chicago is conducted through the Stanley Manne Children's Research Institute. The Manne Research Institute is focused on improving child health, transforming pediatric medicine and ensuring healthier futures through the relentless pursuit of knowledge. Lurie Children's is ranked as one of the nation's top children's hospitals in the U.S.News & World Report. It is the pediatric training ground for Northwestern University Feinberg School of Medicine. Last year, the hospital served more than 212,000 children from 49 states and 51 countries.

Ann & Robert H. Lurie Children's Hospital of Chicago

Related Cystic Fibrosis Articles:

In cystic fibrosis, lungs feed deadly bacteria
A steady supply of its favorite food helps a deadly bacterium thrive in the lungs of people with cystic fibrosis, according to a new study by Columbia researchers.
Cibio knocks out cystic fibrosis
The fight against cystic fibrosis continues, targeting in particular some of the mutations that cause it.
Hypertonic saline may help babies with cystic fibrosis breathe better
Babies with cystic fibrosis may breathe better by inhaling hypertonic saline, according to a randomized controlled trial conducted in Germany and published in the American Thoracic Society's American Journal of Respiratory and Critical Care Medicine.
Understanding antibiotic resistance in patients with cystic fibrosis
Patients with cystic fibrosis who carried antibiotic-resistant bacteria in their lungs had significantly lower microbial diversity and more aggressive disease, according to a small study published in Heliyon.
Research shows that cystic fibrosis impacts growth in the womb
New research, published in Thorax, funded by the Cystic Fibrosis Trust has shown that babies with cystic fibrosis (CF) are born weighing less than babies without the condition, even allowing that they are more likely to be born prematurely.
More Cystic Fibrosis News and Cystic Fibrosis Current Events

Best Science Podcasts 2019

We have hand picked the best science podcasts for 2019. Sit back and enjoy new science podcasts updated daily from your favorite science news services and scientists.
Now Playing: TED Radio Hour

Erasing The Stigma
Many of us either cope with mental illness or know someone who does. But we still have a hard time talking about it. This hour, TED speakers explore ways to push past — and even erase — the stigma. Guests include musician and comedian Jordan Raskopoulos, neuroscientist and psychiatrist Thomas Insel, psychiatrist Dixon Chibanda, anxiety and depression researcher Olivia Remes, and entrepreneur Sangu Delle.
Now Playing: Science for the People

#537 Science Journalism, Hold the Hype
Everyone's seen a piece of science getting over-exaggerated in the media. Most people would be quick to blame journalists and big media for getting in wrong. In many cases, you'd be right. But there's other sources of hype in science journalism. and one of them can be found in the humble, and little-known press release. We're talking with Chris Chambers about doing science about science journalism, and where the hype creeps in. Related links: The association between exaggeration in health related science news and academic press releases: retrospective observational study Claims of causality in health news: a randomised trial This...