Speaker 0: Welcome everyone to the Becker's Healthcare podcast series. I'm Mariah Muhammad, writer and moderator with Becker's Healthcare. I'm thrilled to have with me today doctor Michael j Welsh, professor of internal medicine at the University of Iowa and Grace Lidget, a patient at University of Iowa Health Care. Thank you both for speaking with with me today, and welcome to the podcast. We're very excited to have you join us.

Today, we'll be discussing a fascinating fascinating story about the treatment of cystic fibrosis, between doctor and patient. To get us started, doctor Welsh, I'll get started with you first. For those who are not familiar with this, can you explain what cystic fibrosis is and how it affects patients' lives?

Speaker 1: So cystic fibrosis is a genetic disease. It's inherited in autosomal recessive fashion. It's the most common, severe, life threatening disease in Caucasians, particularly of Northern European ancestry. It affects multiple different organs. It affects the pancreas.

Destruction of the pancreas and intestinal problems used to be the major early claim on babies born with cystic fibrosis. Then, physicians and scientists developed pig, pancreas enzymes, and that helped with nutrition. But but for a long time, the lung disease is the major cause of suffering for people with cystic fibrosis. And there, the problem was infections that were really recalcitrant to treatment. Infections with many different organs.

Pseudomonas aeruginosa is a common widely recognized organism as well as staph aureus. And that treatments had progressed for cystic fibrosis, and those consisted of antibiotics, consisted of attempts to dislodge the mucus with mists and saline administration and, pounding on people's backs. And that's where the situation was prior to, the recent developments, recent developments beginning a long time ago, but accelerating and it's a turning point with the discovery of the CF gene.

Speaker 0: Wonderful. Thank you so much for giving us that background. Grace, going to you, you were diagnosed with CF as a baby. What were some of the biggest challenges, you faced growing up with the disease before the therapies were available? When I was born,

Speaker 2: it was about it was 02/2004. And so I don't ever remember not having the therapies because as soon as I was actually old enough to know what was happening around me, I was doing two nebulizers a day, and I didn't have a vest yet because I wasn't old enough. It straps to you like a vest that you'd normally wear except for it inflates with air, and it shakes you very violently to try to break up that mucus so that way you can cough it up. So once I reached that age, I I had a vest readily available. I was born during a very fortunate time where I didn't have to live without these, specific therapies, like that.

However, these modulator therapies, before those were avail available, I got sick quite often. I I remember having Pseudomonas three times in my life right when I was first diagnosed at 14 old, or yeah. Right when I was diagnosed at 14 old, once in fifth grade, and then once again in eighth grade. I was lucky enough that these didn't severely damage my lungs, but it was a consistent enough issue that I was at risk to become chronic with it. But it was only a couple years after my last diagnosis with Pseudomonas that these modulators came out.

My lung function skyrocketed, which was such a blessing because I am a long distance runner, and so it really helped me out to pursue that passion, something that I was already actively pursuing, but now could comfortably continue to do so with a little bit of support.

Speaker 0: Yeah. Yeah. Absolutely. Well, that's that's very good to hear. And, doctor Walsh, you've been studying CF for more than forty years.

What drew you to the this disease early in your career, and, also, what kept you focused on it for so long?

Speaker 1: So my first exposure to cystic fibrosis was when I was a medical student, junior medical student here at the University of Iowa. And, at that time, I I was in the pediatric clinic, and I met a young girl, probably seven or eight years old. And, she was struggling to breathe. I could see her using her accessory muscles, speaking in short sentences, just obviously having difficulty breathing, frequently coughing, a really harsh cough. And I learned about all the things she couldn't do, and I learned how short her life would be.

That made a made an indelible impression.