KU and The University of Kansas Health System are leading the way on research of a new drug, known by its brand name, Trikafta, which was approved by the U.S. Food and Drug Administration October 21. The results of that clinical trial were published today in the New England Journal of Medicine. 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, is a co-author on the study.
In the United States, 30,000 people have cystic fibrosis, and there are more 70,000 people with the disease worldwide. Approximately 1,000 new cases are diagnosed every year. One trial participant went from believing she would have to quit her teaching job because of exhaustion to running a 5K.
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, which includes about 90 percent of all cystic fibrosis patients. The new drug, the first “triple-combination therapy” to treat the disease, was fast-tracked by the FDA. It is currently approved for people age 12 and over.
In this video, Polineni explains more about the study and how the drug works, Steven Stites, M.D., director of the cystic fibrosis center at The University of Kansas Health System, talks about how the drug can change the lives of patients, and Sarah Carollo, a participant in the clinic trial, shares how the drug has changed her life. Also included is video of Sarah running the 5K.