"This is really a breakthrough," said Polineni. It is currently approved for people age 12 and over. The new drug, the first "triple-combination therapy" to treat the disease, was fast-tracked by the FDA through four of its programs that advance treatments for serious illnesses with unmet patient needs. Approximately 90 percent of all cystic fibrosis patients have this mutation. Trikafta, a pill meant to be taken twice a day, is actually a combination of three drugs (elexacaftor, ivacaftor, and tezacaftor) designed for people who have the most common cystic fibrosis genetic mutation. Approximately 1,000 new cases are diagnosed every year. In the United States, 30,000 people have cystic fibrosis, and there are more than 70,000 people with the disease worldwide. The results of that clinical trial were published today in the New England Journal of Medicine. Food and Drug Administration on October 21. The clinical trial that Carollo participated in at the KU Medical Center was one of two phase 3 trials that led to the approval of the drug, known by its brand name, Trikafta, by the U.S. "At the next visit, seven days later, my mother asked me to slow down because I was walking too fast."Īfter a few weeks, as her health continued to improve, Carollo became able to do more and more, including keeping up with the 3- to 5-year-old kids she teaches. "The first day of the study, walking in, I had to take several breaks and sit down just to get to the room," she said. After just a few days, she began to suspect she was on the drug. Because it was a double-blind study, Carollo had no idea whether she was receiving the new drug or was in the control group receiving a placebo. Polineni was the site principal investigator from KU Medical Center on a multi-center clinical trial for a new drug, produced by Vertex Pharmaceuticals, to treat the disease.Ĭarollo had already participated in two clinical trials, neither of which led to improvement in her disease, but she said yes. Then while on vacation in Colorado, she received a phone call from Deepika Polineni, M.D., MPH, assistant professor of internal medicine in the Division of Pulmonary, Critical Care and Sleep Medicine at the University of Kansas Medical Center, and a pulmonologist at the cystic fibrosis clinic for The University of Kansas Health System, where Carollo received treatment. "Once, I had to hook up to an IV at school, in the teacher's lounge," she said. But by last summer, Carollo's health had declined to the point where she was spending hours every day coughing, doing various therapies to clear her lungs, and administering antibiotics via a nebulizer and intravenously at home to fight chronic lung infections. The 28-year-old, who had been expected not to live past the age of 10, had spent her lifetime battling cystic fibrosis, a lethal genetic disease that causes thick mucus to build up in the lungs, digestive tract and other parts of the body. In the summer of 2018, Sarah Carollo, a special education teacher in Grain Valley, Missouri, was on the brink of quitting her job.
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