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?
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.
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, 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.
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?
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.