NEW ADVANCEMENTS IN CYSTIC FIBROSIS TREATMENT
Modulator treatments are a highly beneficial and widespread therapy for people with cystic fibrosis. However, until recently, no modulator treatments were available for children aged two to five who have the most common form of cystic fibrosis caused by a mutation in the F508 gene. A new study published on May 6, 2021 by Children's Hospital Colorado shows that based on new understandings of cystic fibrosis in young children, the (CFTR) modulator treatment is safe and well-accepted for children in the two to five age range.
Cystic fibrosis is a chronic, genetic disorder that inflicts serious hardships on the digestive system, respiratory system, and many major organs. People with cystic fibrosis are born with a mutation in the F508 gene which causes their transmembrane conductance regulator (CFTR) protein to be defective, or in extreme cases, completely absent from the cell. The malfunction of the CFTR protein directly affects the cells that create mucus, digestive juices, and sweat. Cells with a perfectly functioning CFTR protein produce thin and slippery fluids, while cells in people with cystic fibrosis produce thick and sticky fluids. Fluids inside people with this condition clog the ducts and passageways in the lungs and pancreas instead of acting as a smooth coating, causing many health complications for people with cystic fibrosis (CF).
Cystic fibrosis affects more than 70,000 people worldwide; their symptoms include persistent coughing that produces thick mucus, wheezing, exercise intolerance, repeated lung infections, inflamed nasal passages, poor weight gain and growth, intestinal blockage, and chronic constipation. Despite the daily care needed to treat cystic fibrosis, most people can attend school and work. Unfortunately, due to all the health complications, people with cystic fibrosis have shortened life spans ranging from thirty to fifty years. The lifespan of people with cystic fibrosis used to be much shorter, but now with new developments in treatment for cystic fibrosis, people are getting the help they need and are living longer.
In children from ages two to five, the cystic fibrosis modulator treatment uses doses of a combination of lumacaftor and ivacaftor every twelve hours for more than 96 weeks. This treatment is used to slow and hopefully prevent the difficult side effects of modulator treatments on children. Modulator treatments can cause lung function decline, lung damage, and frequent illnesses or hospitalizations. The modulator treatment for children is revolutionary, as most previous treatments were reactive as opposed to preventative. For example, manual chest percussion to clear mucus from the airways was a reactive treatment that attempted to fix what was already damaged, while the new lumacaftor and ivacaftor treatment aims to prevent the damage before it even happens.
The medical community and cystic fibrosis patients all around the world are elated and grateful for the green light on preventative treatments for young children with cystic fibrosis and hope for the discovery of even more treatments to improve quality of life and increase the lifespan of people with cystic fibrosis.
What is Cystic Fibrosis by Mayo Clinic
New advancements in cystic fibrosis treatment