This week Bobbi Conner talks with Dr. Patrick Flume about new breakthrough treatments for cystic fibrosis. Dr. Flume is a Distinguished Professor of Medicine and Pediatrics and Director of the Cystic Fibrosis Center 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 the fall of 2019, a new triple combination of drugs became available for treating cystic fibrosis. Doctor Patrick Flume is here to talk about the impact of this therapy for children and adults living with this condition. Doctor Flume is a Distinguished Professor of Medicine and Pediatrics, and he's Director of the Cystic Fibrosis Center at MUSC. Doctor Flume, first, tell us briefly about cystic fibrosis.
Dr. Flume: Cystic fibrosis is a genetic condition in which people who have two altered genes for this protein end up having complications that range from sinus disease to lung disease, with chronic infections, to digestive problems, and a multitude of other conditions that result in a shortened lifespan.
Conner: And tell us about this triple combination drug therapy, that's been a big breakthrough in treating this condition.
Dr. Flume: This is a very exciting drug combination. Historically, all of our therapies in CF were treating the downstream consequences of CF. So, it was treating the infections and trying to clear secretions from the lungs. But these drugs actually work at the basic defect. Now, although they are not a cure, what they do is result in more of that protein and better functioning protein, so things operate more normally. So, it actually relieves them of many of the complications of their condition.
Conner: And is this in widespread use now and also is everybody eligible for this therapy who has cystic fibrosis?
Dr. Flume: Unfortunately, not everybody is eligible because they have to have the right gene mutations that will be responsive to these drugs. So, fortunately that is the majority of people with CF. about 90% have the right mutation. But there are some people who cannot even take the medication because of some adverse effects. So, there's probably 20 or 25% of the population who are not yet able to enjoy the benefits of this therapy.
Conner: How has this therapy improved outcomes for patients with CF who can take this new medication?
Dr. Flume: These drugs have been amazing for patients. They lived with a daily cough and sputum production. And when they start taking this medication, they experience what they call the purge, where all this stuff comes up and then they just stop coughing. Many of these patients would have exacerbations where their symptoms became more severe, often leading to therapies like antibiotics and hospitalization. And as an example, here in Charleston, it used to be that I had 4 to 6 people in the hospital every day, and now that number is about 0 to 1. So, it has been a dramatic improvement in the health of these patients.
Conner: Are we getting any closer to a cure for cystic fibrosis?
Dr. Flume: There is progress with genetic therapies. So there are ongoing studies looking at gene editing and gene replacement. There are studies looking at providing the messenger RNA much like was done with some of the newer vaccines, to try and get the person to make their own protein, and some other very novel technologies that try to get to fixing the basic defect.
Conner: Doctor Flume, thanks for this update about the new treatment for cystic Fibrosis.
Dr. Flume: Thank you.
Conner: Bobbi 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.
