- I think it's best to talk about treating cystic fibrosis
based on its symptoms in different organ systems.
So, let's go one by one and let's start with GI.
And I want to start with this because
I just remembered this little boy I took care of
in a camp once who has CF, and, before he ate anything,
breakfast, lunch, dinner, snack,
we had to give him these enzyme pills.
They're not that much hard to swallow,
but it just struck me that he really shouldn't eat anything
without them, because the nutrients he's eating
would be a waste without these enzymes to help him digest.
So, remember that, our pancreas here,
with their little head there, helps us digest
by naturally secreting these enzymes to digest
the sugars, the fats and proteins in our diet.
And you might think, well, I thought
absorption happened in the gut, and you'd be right.
So, let's see if I can draw this correctly.
If we have our stomach here, stomach,
and then our gut follows, I'm just going to
draw it like this, it's a big, tangled mess,
and you're right, that nutrients get absorbed in this tract
and goes to our blood, but the pancreas is responsible
for secreting the enzymes to help it break down.
For example, protein is really hard to break down.
Without these enzymes, the gut wouldn't be able to
do anything with the food.
In cystic fibrosis, since these ducts are clogged up,
there's nothing we can really do to unclog them
at this point.
So, what we can do is make these synthetic enzymes
that mimic what the pancreas usually makes.
You just give it to the person to take by mouth.
So, it's our way of trying to do nature's job,
in which case it's not happening
in the GI system of CF patients.
Aside from that, this little boy that we took care of
also had to way up his calories, because even with
all these enzymes and even with eating a lot,
it's hard for him to grab onto the nutrients,
and also in the GI system, the pancreas also makes insulin.
Let's write that here.
Which is the thing that helps us regulate blood sugar.
Not everyone who has CF has a problem with
the insulin tract, but sometimes they do,
and it's called CF-related diabetes.
So, if they need it, we might have to do diabetic therapy
and think about giving them insulin, but, again,
it doesn't happen to every CF patient.
Next, let's talk about the lungs.
The lungs, not only are they very affected by CF,
but this is where a lot of the
mortality and morbidity come from.
In fact, CF used to be a pediatric disease,
because not a lot of people lived to adulthood,
and thankfully, that's changed with all this therapy
that we're talking about, but, still,
there's a lot of lung infections,
and they have all that thick mucus in there.
The bacteria's just trapped.
It's the perfect environment for them to grow.
So, it's not uncommon for CF people to
keep getting one pneumonia after another,
and that's very taxing on the body
and people can die from that.
So, a lot of patients with CF have to spend
maybe half an hour each day wearing this vest.
Let me try to draw it.
It looks like a big, bulletproof vest.
It's heavy and it's got hoses that are connected here
that lead to a machine that kind of looks like
a big vacuum cleaner, and when you turn it on,
the whole thing just shakes while this person's wearing it.
It looks like they can't even complete a full sentence
because it's shaking so hard, and that's because
usually in the lungs, we have cilia,
these little green hair-like things
that clear the mucus for us, but since it's so thick,
we need this mechanical way to shake things up
so they can get the mucus out.
So, I'm going to write here, shake.
I imagine it's very uncomfortable and hard to get used to
in the beginning but the kids I met who do this every day,
they're so used to it, it's just another thing they do.
They put it on, read a book, and then they're done.
So, this vest has really helped a lot
to clear some of that mucus
and try to temper down the infection rate.
Now, this is mechanically trying to clear the lungs.
We also sometimes give them anti-inflammatory drugs,
anti-inflammatory, because the lungs are so inflamed
by all the mucus and the things happening in there,
it's nice to give them anti-inflammatory drugs
to try to open up the airways a little bit,
make the person a little more comfortable,
and, of course, if they have any signs of infection,
having a pneumonia again, then we do not hesitate
to give them antibiotics, ABX stands for antibiotics,
try to stop the infection at the source,
try to stop the infection before it begins.
Now, even with all this therapy, it's very hard on the lungs
to go through this every day, so, as a last resort,
sometimes the lungs can be so damaged
that people need lung transplants,
and this, of course, brings with it
a whole host of possible complications,
so we really try to keep them away from transplant
by doing these daily therapies
and see how it progresses.
Now, skin...
I've said before that CF patients have very salty sweat
because the chloride doesn't get absorbed,
therefore neither does the sodium,
and all the sodium chloride or salt ends up on the skin.
So, if we have a person's arm here, and their hand,
usually our sweat is salty, because not all the
sodium chloride gets absorbed,
but for CF patients especially, there's just a lot of salt
sitting on their surface of their skin.
Now, remember, we need a very careful balance
of sodium and water and all the chlorides in our body.
So, a lot of patients with CF might have to
put a lot of salt in their diet just to try to
replace what they're losing all the time through their skin.
So, these are some of the mainstay therapies,
kind of the basic things that we do for people with CF.
Of course, whatever complication comes up,
we'll deal with as they come, but the basic principle here
is to maximize their nutrition,
make sure their lungs are working as well as possible,
and keep their ions and fluids and everything balanced,
and those are the basic, daily,
really life-extending therapies for cystic fibrosis.