Cystic Fibrosis (CF) is a terrible disease. According to Cystic Fibrosis Canada, it’s the “most common fatal genetic disease affecting Canadian children and young adults.”
CF can wreak absolute havoc upon the body, though it mainly impacts the digestive system and the lungs. The nature of CF can vary depending on each person who is diagnosed with it, “however, the persistence and ongoing infection in the lungs, with destruction of lungs and loss of lung function, will eventually lead to death in the majority of people with CF.”
Common complications that arise as a result of CF include having difficulty digesting fats and proteins, malnutrition, vitamin deficiencies, chronic infection and aberrant inflammation in the lungs causing damage, diabetes and sinus infections.
The CF Canada website explains that roughly “one in every 3,600 children born in Canada has CF.” It presents itself when “a child inherits two defective copies of the gene responsible for cystic fibrosis, one from each parent. Approximately, one in 25 Canadians carry one defective copy of the CF gene.”
Considering how debilitating this disease is, any news regarding treatment is arguably seen as progressive. And, as it turns out, the U.S. Food and Drug Administration has recently approved new treatment for the disease, and it’s the first treatment that has been created for CF in decades.
“The newly-approved Trikafta medication, which was developed by Vertex Pharmaceuticals, can be used to treat patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene,” says an article from The Good News Network. “While there are approximately 2,000 known mutations of the CFTR gene, the most common mutation is the F508del mutation—and Trikafta is the first approved treatment that is effective for these cystic fibrosis patients, which make up approximately 90% of the those with the disease.”
The new treatment “is a combination of three drugs that target the defective CFTR protein. It helps the protein made by the CFTR gene mutation function more effectively,” the article explains.
“The efficacy of Trikafta in patients over 12-years-old with cystic fibrosis was demonstrated in two trials. The first trial was a 24-week, randomized, double-blind, placebo-controlled trial in 403 patients while the second trial was a four-week, randomized, double-blind, active-controlled trial in 107 patients who had two identical F508del mutations.
“Trikafta increased the ppFEV1 in both trials. In the first trial, it increased mean ppFEV1 13.8% from baseline compared to placebo. In the second trial, it increased mean ppFEV1 10% from baseline compared to tezacaftor/ivacaftor. In the first trial, treatment with Trikafta also resulted in improvements in sweat chloride, number of pulmonary exacerbations (worsening respiratory symptoms and lung function), and body mass index (weight-to-height ratio) compared to placebo.
“The Cystic Fibrosis Foundation (CFF)—which is the world’s leader in the search for a cure for CF—hailed the FDA’s approval of the treatment as ‘the single greatest therapeutic advancement in the history of CF,'” the article says.
If this isn’t some much-needed good news, I’m not sure what is.
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