This week, Bobbi Conner talks with Dr. Christina Mingora about the impact of recent advances in treating cystic fibrosis. Dr. Mingora is an Assistant Professor in the College of Medicine and a pulmonologist at MUSC.
TRANSCRIPT:
Conner: I'm Bobbi Conner for South Carolina Public Radio with Health Focus here at the radio studio for the Medical University of South Carolina in Charleston. In recent years, advances in medical research have produced a dramatic shift in the treatment of cystic fibrosis. Doctor Christina Mingora is here to talk about the details. Doctor Mingora is an Assistant Professor in the College of Medicine, and she's a pulmonologist at MUSC. Doctor Mingora, tell us about cystic fibrosis.
Dr. Mingora: Cystic fibrosis is a genetic condition that is affecting approximately 34,000 people here in the United States and more worldwide. Cystic fibrosis is caused by abnormalities in a specific protein, the cystic fibrosis transmembrane conductance regulator, or CFTR protein. There can be abnormalities with protein creation, processing or function that can then lead to different abnormalities throughout the body. The main manifestation is predominantly in the lungs, but it's very important to know that cystic fibrosis can also cause sinus disease, diabetes, abnormalities of bone health, issues with nutrition and fat malabsorption, as well as male and female infertility.
Conner: Explain briefly the big treatment breakthrough about six years ago related to CF.
Dr. Mingora: So historically in CF, we had focused our treatments and therapies on things that would target just the symptoms that came from the disease. But as technologies and science improved over the years, researchers looked to see how can we instead fix the root cause of the disease? And they looked to see how we can improve that protein function. A series of different drugs were developed beginning in 2012, but in 2019, we had our most impactful drug at the time, which was an oral combination of three drugs that worked to enhance the function of that CFTR protein, leading to dramatic improvements in patient symptoms and outcomes.
Conner: Well, how has this new treatment impacted people living with CF?
Dr. Mingora: This drug has truly been remarkable and so impactful on individuals with cystic fibrosis. The initial trial focused on changes that we saw in pulmonary function and reduction in disease exacerbations. But over the years, as individuals began to take this drug, we also noticed that there were benefits to all of the other systems we talked about. We noticed reduction in sinus symptoms, reduction in GI and bowel complications, as well as improvement in fertility in females, leading to increased pregnancies in individuals with cystic fibrosis. This has been such a wonderful drug. It's completely revolutionized the way that people with CF have lived their lives. When people started this drug, they felt that they were given a whole new future that they didn't previously think was available to them.
Conner: Doctor Mingora, is this treatment available to everybody living with CF?
Dr. Mingora: So unfortunately, this drug is not available for all individuals with CF. The eligibility for the medication is related to your particular genetic mutations. And with that, approximately 90% of the population with CF is able to use this drug. So, we need to look for new therapies for the 10% that is not eligible, as well as a proportion of the 90% who can use the drug, but is unable to do so related to side effects or other intolerances.
Conner: Doctor Mingora, thanks for talking with us about cystic fibrosis.
Dr. Mingora: You're welcome. It's been a pleasure.
Conner: From the radio studio for the Medical University of South Carolina in Charleston, I'm Bobbi Conner for South Carolina Public Radio.
Health Focus transcripts are intended to accurately represent the original audio version of the program; however, some discrepancies or inaccuracies may exist. The audio format serves as the official record of Health Focus programming.
