Researchers from the Northwestern University (NU) have discovered that a combination of drugs that has the potential to significantly improve the health of patients diagnosed with cystic fibrosis (CF).
According to reports, patients treated with a mixture of ivacaftor and lumacaftor medications have shown a noticeable decrease in lung infections and an improvement in their ability to breathe freely.
The treatment was given to 1,108 patients, from 12 years old and older, in randomized controlled tests during a six-month period.
Susanna McColley, a pediatrics professor at NU's Feinberg School of Medicine and one of the authors of the study, said the findings of their research are groundbreaking, and that it will help 15,000 patients with cystic fibrosis in the United States alone.
Cystic fibrosis is a medical condition that causes the body to overproduce thick mucus that often causes infections in the lungs and even problems with the pancreas.
The lung disease is known to manifest in different mutations. Most cystic fibrosis patients are diagnosed with two copies of the mutation known as F508del.
Health experts estimate that there are around 75,000 people who suffer from cystic fibrosis in various parts of the world including Europe, Australia and North America.
People in the United States with the F508del mutation are given a predicted midpoint of survival of 37 years.
In 2012, the U.S. Food and Drug Administration (FDA) approved the drug Kalydeco (ivacaftor) after it had shown the ability to improve the health of patients diagnosed with a rare cystic fibrosis mutation. This genetic mutation is seen in about four percent of all CF patients.
Lumacaftor, the second drug used in the combination, is yet to be approved by regulators.
Both ivacaftor and lumacaftor are manufactured by drug maker Vertex Pharmaceuticals.
Earlier this week, Vertex announced that the advisory panel of the FDA has approved the combination of ivacaftor and lumacaftor in a vote of 12-1. The medication will be known as Orkambi.
Officials of the Food and Drug Administration will release their final decision on July 5.
While the researchers note that the effects of the two drugs were not as dramatic as they were expecting, they believe that the drug combo can offer better treatment for patients with rare forms of cystic fibrosis.
Further tests will also be needed to confirm the long-term effectiveness of the ivacaftor and lumacaftor.
The Northwestern University study is published in the New England Journal of Medicine.
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