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Cystic Fibrosis: Drug Treatment Offers New Hope for Patients

May 18, 2015 12:14 PM EDT
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Cystic fibrosis is life threatening to anyone born with the disease, but now a new combination of drugs is offering new hope for afflicted patients.

That's at least according to a recent study published in the New England Journal of Medicine, which finds that a unique drug cocktail can successfully treat the underlying cause of cystic fibrosis for patients age 12 and older with two copies of the F508del gene mutation - the most common form of the genetic disease found in over half of the cystic fibrosis (CF) population. Approximately 8,500 people in the United States, and 22,000 people in North America, Europe and Australia, age 12 and older with cystic fibrosis carry this gene mutation.

Results from two phase 3 clinical trials, which involved more than 1,000 cystic fibrosis patients age 12 and older, revealed that a combination of the drugs Kalydeco (ivacaftor) and lumacaftor - an experimental drug that has yet to be approved by the Food and Drug Administration (FDA) - successfully treated the defective CF protein and improved lung function.

The drugs also led to a 40 percent decrease in pulmonary exacerbations, the leading cause of death in cystic fibrosis patients.

"Being able to treat not just the symptoms, but the underlying cause of cystic fibrosis by targeting this specific gene mutation, is a major game changer because it is by far the most common in patients with this disease," Dr. Bonnie Ramsey from Seattle Children's Hospital, who led the research, said in a press release. "Previously, we found that Kalydeco treated just four percent of cystic fibrosis patients, but this new combination treats the defective CF protein for a much greater number of patients."

So what is it about this unique combination of drugs that makes it so effective? When they work together, the drugs act as a salt channel and allow proteins within the cell to fold properly, thereby fixing the gene mutation that causes CF. Today, a person diagnosed with CF dies on average in their mid-20s, but this new research may give patients hope for living a longer life.

"What gives me tremendous confidence and excites me the most are the comprehensive health improvements seen in patients across all age groups, in all geographies and with all levels of disease severity," said Ramsey. "Patients can now have longer periods of good health, greatly improving their quality of life."

If approved by the FDA, a decision slated for July, the new combination of medications will be prescribed to patients age 12 and older with F508del/F508del mutation.

For more great nature science stories and general news, please visit our sister site, Headlines and Global News (HNGN).

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