2019 was a promising year for cystic fibrosis (CF) patients as a new drug called Trikafta was approved by the FDA and can benefit up to 90% of the patient population. It works with those patients that share the most common mutation (delta-F508) to the CFTR gene. Local news covered some of the exciting local results thanks to this recent FDA approved CF treatment.
Brief Cystic Fibrosis Overview
An inherited, progressive, life-threatening disease that results in the formation of thick accumulations of mucus in the lungs, digestive tract, and other areas of the body. The disease leads to dangerous respiratory and digestive challenges and other complications such as infections and diabetes. It is a rare disease and afflicts about 30,000 in America and 70,000 worldwide.
Cornhusker State: Research & Care in the Heart of America
With a population just under 2 million and at the heart of America, Nebraska covers a lot of ground. It wields influence in the medical research world with prominent institutions such as the University of Nebraska Medical Center/Nebraska Medicine, a nexus of advanced research and care.
The buzz in the medical community with interests in cystic fibrosis is palpable, as the buzz generated at the national CF meeting in Nashville was brought back to the Midwest CF community reported Peter J. Murphy, MD, associate professor, internal medicine, and program director of the Adult CF Program in the Nebraska Regional Cystic Fibrosis Center at the University of Nebraska Medical Center/Nebraska Medicine. Moreover, patients are directly benefiting, such as Nick Bell, whose story is covered by local news outlet Boone News-Republican.
UNMC Nebraska CF Program
The UNMC Adult CF program serves about 200 adult CF patients, and another 165 pediatric patients are followed at Children’s Hospital & Medical Center. The median survival age for people with CF is 47—this has risen from age 32 back in 2000. Dr. Murphy reports that Trikafta is “an amazing life-changing solution” but noted that the drug is not a cure, but rather serves as a form of the repair mechanism. He noted, “This is really the cutting edge of science—it’s protein repair. It has the potential to significantly change the course of the disease.”
Made by Vertex Pharmaceutics, Trikafta was approved by the FDA for patients aged 12 or older. Clinical trials of the drug reveal significant improvements in key areas of disease elements from lung function, sweat chloride, and quality of life. The Boone News-Republican reported that one patient from the Trikafta clinical trial was from Children’s Hospital & Medical Center. A combination of three drugs that target the defective CFTR protein, it helps the protein made by the CFTR gene mutation function more effectively. Previous therapies that target this defective protein are options for some CF patients, however, many more patients have mutations rendering them ineligible for treatment. Hence Trikafta is the first treatment that is effective for CF patients 12 and up with at least one F508del mutation which affects 90% of the population with CF, or approximately 27,000 people in the United States.
Early results reveal that Trikafta is offering CF patients about a 14% improvement in lung function—the biggest gains are experienced within the first month.
10% Left out
With another 10% or 3,000 in America alone that don’t have options, Dr. Murphy noted that the Cystic Fibrosis Foundation is offering $500 million in grant funding to stimulate new research that will hopefully go toward benefiting all CF patients.
UNMC is very active in clinical research representing a major hub of research and testing of advanced experimental therapies in America’s heartland. The Department of Internal Medicine division actively participates in clinical studies focused on smoking cessation, COPD, Pulmonary Hypertension, and Cystic Fibrosis.
UNMC/Nebraska Medicine CF Team
For those residing in or near the State of Nebraska, the heartland of America represents a potent nexus of CF research and care. This nexus is the Adult Cystic Fibrosis Clinic in the Durham Outpatient Center and includes:
· Peter J. Murphy, MD associate professor (program director)
· John Dickinson, MD, PhD, assistant professor (associate program director)
· Meilinh Thi, DO, assistant professor
This program offers an array of services to CF patients from nursing care, respiratory therapy, dietetics, and care coordination. Full support staff is available as well, led by Jill Fliege, APRN, program coordinator.
Call to Action: Interested in learning more about UNMC’s CF program? Follow the link for more information.