Drug Combo Safe for Children of 2-5 Years Having Cystic Fibrosis

by Mohamed Fathima S on January 25, 2019 at 10:09 AM

Lumacaftor and ivacaftor, a combination of cystic fibrosis drugs were found to be safe and effective in children of 2-5 years, two copies of F508del-CFTR gene mutation cause the most common and severe form of the disease. Earlier, the drug combination was approved for treatment in 6 years and older patients. However, the treatment with the drugs for two years and older administered as per the recently extended approval of Food and Drug Administration.

"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

Cystic fibrosis is a genetic disease involving the mucus and sweat glands and the medical world has still to find its cure

‘Children suffering from cystic fibrosis responded well to the treatment of the drugs showing an improvement in growth by a significant increase in height, weight, and body mass index.’

Tweet it Now

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."

DNA Finger Printing

DNA fingerprinting is a technique which helps forensic scientists and legal experts solve crimes, identity thefts, legal suits and terrorism cases.

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.

Source: Eurekalert

Respiratory Diseases

Respiratory diseases affect any part of the respiratory system. Some of the lung diseases like chronic obstructive pulmonary disease can lead to respiratory failure.

<< New Research Could Improve Blood Pressure Treatments

Reduce, Reuse and Recycle Should be Our Mantra on Plastic Us... >>

Post-Nasal Drip

Post-nasal drip occurs as dripping of the excessive mucus from the nasal mucosa caused by allergy or infection. Post-nasal drip can cause bad breath and snoring.