May 11, 2025 • 14 mins
NFL quarterback Boomer Esiason’s career high came just days before his son was diagnosed with cystic fibrosis (CF) – a genetic disease characterized by thick and sticky mucus that interferes with breathing, digestion and the body’s ability to fight infections. Esiason now dedicates his free time to raising awareness to fund the search for a cure. We explore how Vertex’s Chief Scientific Officer, Dr. David Altshuler, and the research team at Vertex have spent the last 20+ years researching the underlying cause of the disease – and their work is not yet finished.
This episode of Targeting the Toughest Diseases is produced by Bloomberg Media Studios and Vertex Pharmaceuticals and is sponsored by Vertex Pharmaceuticals.
See omnystudio.com/listener for privacy information.
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Episode Transcript
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Speaker 1 (00:00):
Since you're a subscriber to this Bloomberg podcast, we thought
you'd be interested in a six episode sponsored podcast called
Targeting the Toughest Diseases, produced by Vertex Pharmaceuticals and Bloomberg
Media Studios. It explores the innovative tools, methods, and unique
philosophy Vertex Pharmaceuticals is using to search for treatments for
(00:22):
some of humanity's most challenging diseases. Here's a recent episode.
Speaker 2 (00:29):
Yeah, Well, I was getting ready to step on the
field for the New York Jets for the first time
in nineteen ninety three.
Speaker 3 (00:38):
NFL quarterback Boomer Asiasin had just been traded to the
Jets after ten incredibly successful seasons with the Cincinnati Bengals,
a team where he'd wrapped up a Super Bowl appearance
and an MVP Award. He was born and raised in
New York, so it was exciting to play for one
of his hometown teams. But then, just as he was
(01:01):
about to walk into the field, he was called back.
Speaker 2 (01:05):
The head coach's secretary came out onto the field and said,
your wife is on the phone. You need to take
the phone call. So I took the phone call.
Speaker 3 (01:12):
It was Boomer's wife, Cheryl, who was back in Cincinnati
with their son, Gunner.
Speaker 2 (01:17):
Cheryl told me I needed to get back to Cincinnati,
that Gunner was in Cincinnati Children's Hospital and he was
having trouble breathing.
Speaker 3 (01:25):
Boomer grabbed the next flight to Cincinnati, then went straight
from the airport to the hospital.
Speaker 2 (01:31):
Seeing a two year old Gunner laying in a crib
hooked up to all sorts of different machines receiving antibiotics,
was on oxygen, and really wasn't sure what to think.
Speaker 3 (01:43):
The doctor came into the room and told Boomer and
Cheryl that their little boy had cystic fibrosis.
Speaker 2 (01:48):
That day will live with me forever as one of
the saddest days of my life.
Speaker 3 (02:00):
Hi. I'm Jordan Gospore. I'm a member of the University
of Southern California's Center for Health Journalism. This is Targeting
the Toughest Diseases a podcast produced by Bloomberg Media Studios
and Vertex Pharmaceuticals. In this series, we look at some
of humanity's most challenging diseases and how Vertex, a Boston
based biotech company, is using innovative tools, methods, and a
(02:24):
unique philosophy to search for treatments and cures. Today we're
looking at cystic fibrosis, also known as CF, a rare
chronic genetic disease affecting the lungs and other organs. It
can lead to all sorts of complications, including lung disease,
liver disease, diabetes, and even premature death. It affects about
(02:46):
thirty thousand Americans and more than eighty thousand people globally,
making it a disease not a lot of people know about,
but it was not new to Boomeris Siasin.
Speaker 2 (03:03):
Here's the really weird part of this, and maybe the
destiny and irony part of all of this. Back in
nineteen eighty nine, I was receiving award as the best
quarterback in the NFL, and at that awards banquet, there
was a speaker that night because there was a fundraising
attachment to that banquet, and his name was Frank DeFord.
Speaker 3 (03:22):
DeFord was a legendary writer at Sports Illustrated, but he
was also a CF dad.
Speaker 2 (03:27):
Frank spoke so eloquently about how he lost his daughter
Alex to the disease of cystic fibrosis, which I had
never heard of. She was eight years old, and everybody
was captured by his story. That night. I was very captured.
I was crying.
Speaker 3 (03:42):
Bumeris Iyasin was twenty seven at the time, with no
kids of his own. He was living a great life
as an NFL quarterback, but something in that speech touched
him deeply. He asked to Ford how he could help.
Speaker 2 (03:53):
And he said, well, if you go back to Cincinnati
and you can get involved in some of the fundraising
aspects of cystic fibrosis a face on it, if you will,
and then maybe visit some of the kids at Children's Hospital,
that would really be helpful.
Speaker 3 (04:06):
And that's exactly what he did. He used his celebrity
status to help raise awareness and funds for cystic fibrosis,
and he visited kids in the hospital who were sick. Now,
his son, Gunner had this same terrible disease. We all
have mucus that lines her lungs and our airways. This
mucus has an important job. It captures dust and germs
(04:27):
in the air we breathe. When mucus traps those germs
in dust, tiny little hairs on the outside of our
cells called cilia push all that mucus out of our lungs. Normally,
this mucus is slippery and watery, but for people with
cystic fibrosis, this mucus is sticky and thick. As a result,
the cilia can't push the mucus out and it ends
(04:48):
up blocking lung passages, making it hard to breathe. Also,
germs and bacteria get trapped in a person's airways, making
them more prone to infections. Clearing the mucus could be
a daily task for people with CF. There are medications
that help fin it. Some patients use a vibrating vest
to literally shake the mucus out of their airways. Others
(05:10):
may have to visit the hospital periodically for a cleanout.
And because the mucus can also block the ducks to
the pancreas, many people with CF may struggle to absorb
nutrients efficiently. Solving that can require periodic IV treatments or
taking enzymes to help break down food for absorption. IV
treatments are also used to fight frequent infections and lungs
(05:31):
that come about in many people with CF. It's a
demanding regiment, something Gunner Asiasin had to confront when he
was still very young.
Speaker 4 (05:41):
My dad, who was driving the car just looked at
me while we were driving and said, this isn't gonna hurt.
And for the entire rest of the car ride, he
continued to say, this isn't gonna hurt, This isn't gonna hurt,
this isn't gonna hurt, as if he was reassuring me
about something they weren't telling me about.
Speaker 3 (05:55):
Everything was fine until the moment Gunner realized the antibiotic
treatment he was getting for a CF related infection involved needles.
Speaker 4 (06:05):
I looked at my dad's grinding at I said, you
lied to me, and I ran out the door, down
the hallway and into the lobby of Cincinnati Children's Hospital. So,
if you can picture the scenario, there's my dad, the
starting quarterback of the Cincinni Bengals at the time, chasing
his belligerent child, and he finally comes up to me
and he's like, quiet, please be quiet, you're making a scene.
I will do whatever you want if you go back
into that room with me.
Speaker 3 (06:25):
Eventually, after a lot of persuasion, Boomer agreed to buy
Gunner a video game console if he would go and
get the treatment.
Speaker 4 (06:33):
So we walked back into interventual raiology and they scrapped
me down to the table, and I cried and screamed
to the entire thing. But you better believe that I
got my Nintendo at the end of that possible visit.
Speaker 3 (06:46):
In college, Gunner reached a turning point with how he
coexisted with the disease.
Speaker 4 (06:51):
My senior year was a real slog like I went through,
like several bounds of pneumonia. I knew that I was
at a point in my life where I had no
other options.
Speaker 3 (07:01):
That uncertainty, having big questions about the future is one
of the hardest parts of living with CF. Cystic fibrosis
is genetic. Our genes give our cells instructions on how
to produce certain proteins. They tell the cells in our lungs, pancreas,
or sweat glands to make a special protein called cystic
(07:22):
fibrosis transmembrane conductance regulator or CFTR for short. This CFTR
protein is sent to the surface of a cell. Here,
it acts like little channels between the inside and the
outside of a cell. These channels open and close, and
this allows particles like chloride ions to move in or
out of a cell, and those chloride ions help a
(07:44):
cell maintain a healthy balance of salt and water. But
for people with CF, a mutation causes the gene to
give faulty instructions to the body. Those proteins, those little
channels don't work right. This means water and salt can't
flow freely in and out of a cell like they should.
When that chloride gets trapped in a cell, it doesn't
have the fluids it needs to hydrate its surface properly.
(08:06):
Without those fluids, mucus becomes dehydrated, and that's what causes
it to be thick and sticky. Doctor Chinadou Nikuoro is
a pediatrician in London. He has spent decades researching the
condition and says we've known about CF since ancient times.
Speaker 5 (08:23):
If you go back to European proverbs, you may have
heard there was a saying, woe to that child which,
when kissed on the forehead, tastes salty. He is bewitched
and soon must die.
Speaker 3 (08:33):
Salty skin is a symptom because people with cystic fibrosis
have two to five times the normal amount of salt
in their sweat.
Speaker 5 (08:40):
And these old grandmothers in northern Germany were describing children
with cystic fibrosis.
Speaker 3 (08:46):
The genetic mutation that causes CF survived because it provides
its carriers with some extra immunity.
Speaker 5 (08:52):
Having one copy of the cistic vibrosius mutation seems to
protect against diarrehal diseases like cholera and typho. That would
have meant that people who were carriers of that gene
would be more likely to survive plagues of cholera and typhoid,
where people who didn't have any copies would have been
more likely to succumb.
Speaker 3 (09:12):
It was nineteen thirty eight that the disease was named
and made it into medical literature. It was discovered in
children who had died of malnutrition.
Speaker 5 (09:19):
The US pathologist Dorothy Anderson found that their pangresses were
damaged through being bummed up by sticky secretions, and they
were cystic and fibrotic, and that was the name given
to the disease.
Speaker 3 (09:31):
Doctor Niicuro says life expectancy for children living with CF
has been extended, thanks in large part to newborn screening
and improve treatments, but he is hopeful that treatments will
not just extend life for patients living with the disease,
it will improve their quality of life as well.
Speaker 5 (09:47):
What I hope that will lead to is a reduction
in the need for the kind of intense therapy, both
in terms of antibiotics, hospital admissions and in the even
physiotherapy that has been characteris consistent fibrousus care over the years.
Speaker 3 (10:04):
Coming up with therapies that can both prolong and improve
life is a pretty tall order, but it's a passion
for doctor David Altschuler, chief Scientific Officer at Vertex Pharmaceuticals.
As a physician and a researcher, he spent the last
thirty five years of his career scouring our DNA code
for clues about some of the world's toughest diseases.
Speaker 6 (10:26):
So the first thing we do is we look for
diseases with great unmet need, where the disease is very
serious and people's lives are impacted and there's nothing that
really is is transformational, that can really change their lives,
and cystic fibrosis certainly was that. The second was we
look for a case where the underlying human biology, the
(10:47):
underlying cause of the disease is known, and again in CF,
the cystic fibrosis gene mutation was known. What was missing
was therapeutics that could do what needed to be done,
which is, in this case, restore CF function. And that's
kind of thing that really gets our scientists and doctors
fired up, and so they worked on it for twenty
years to get where we are today.
Speaker 3 (11:08):
Can you speak a little bit more about how you
and Vertex are tackling cystic fibrosis.
Speaker 6 (11:14):
So the work that Vertex has done in CF really
goes back decades ran in families, but until nineteen eighty
nine when that gene was cloned, no one actually knew
what caused the disease.
Speaker 3 (11:25):
That changed with the discovery of CFTR.
Speaker 6 (11:28):
But then the question was could you repair it? And
for a period of decades that seemed impossible. People tried
lots of different things and couldn't figure it out. But
twenty years or more ago, scientists at Vertex had an idea.
They had an idea of how to use chemicals to
coax the CFTR protein that's not working to function again.
Speaker 3 (11:49):
That's what Vertex has been working on for the last
twenty years and now has approved medicines with the potential
to treat up to ninety percent of all people with CF,
and they're not done yet.
Speaker 6 (11:59):
The things that keeps us working hard on this problem
is that less than ten percent don't make any of
the protein.
Speaker 3 (12:06):
The way current therapies work is to coax the protein
that has an inherited mutation to function again. But some
people don't make any protein, so a different approach is needed, and.
Speaker 6 (12:17):
So we have research ongoing in our laboratories and with
a number of partners, are working hard for the last
ten percent. We're still very much in the CF story.
We still have a long way to go till we
reach our goal.
Speaker 3 (12:31):
For people with CF, like Gunner as siasin things that
once felt unimaginable now seem possible, like returning to school
to see old friends.
Speaker 4 (12:40):
I had my five year college reunion, and most people
remember me from my college a's at my last year
or two, being very very sick, like always missing things,
missing social activities, missing class, missing things like that. Yeah,
here we are at the reunion and it's like the
chance to go back to college, right, you know, We're
staying out late, and next thing you know, I'm up
every morning at like six point thirty getting breakfast for everyone.
And my friends were like, what is going on with you?
Speaker 3 (13:00):
But as great as the weekend was The drive home
with his girlfriend was even more impactful.
Speaker 4 (13:04):
We were stuck into New Jersey Turnpike and she looked
at me and she was like, do you realize what
we did this weekend? We went away and we're able
to do something. What do you want to do next
with your life? And I paused for a moment. It
hadn't occurred to me that I could do something with
my life. I didn't have to live at home anymore.
Speaker 3 (13:21):
What they decided to do next was get married.
Speaker 2 (13:24):
I've never been at a wedding where everybody, I mean
all two hundred and fifty people just came together as
one group and we're crying and we're laughing and we're cheering,
and it was like the greatest moment of all of
our lives.
Speaker 3 (13:38):
Going back to school, getting married, those experiences are possible
because of one thing. Hope. Well.
Speaker 2 (13:45):
I don't know how much of a football fan you are,
but when you play for the Jets, Cardinals and Bengals,
it's all about hope. You need as much hope as
you possibly can get.
Speaker 4 (13:53):
You know. I think for me, I mean honestly, I
hope is going to define the rest of my career.
When I was going through the worst today, I had
a little quote on my desk and it was a
Wizard Churchill quote and it was something like, if you're
going through hell, keep going. And that meant a lot
to me back then.
Speaker 3 (14:17):
This is Targeting the Toughest Diseases a podcast from Bloomberg
Media Studios and Vertex Pharmaceuticals. If you like what you hear,
subscribe and leave us a review. I'm Jordan Gosfore. Thanks
for listening.
Since you're a subscriber to this Bloomberg podcast, we thought
you'd be interested in a six episode sponsored podcast called
Targeting the Toughest Diseases, produced by Vertex Pharmaceuticals and Bloomberg
Media Studios. It explores the innovative tools, methods, and unique
philosophy Vertex Pharmaceuticals is using to search for treatments for
(00:22):
some of humanity's most challenging diseases. Here's a recent episode.
Speaker 2 (00:29):
Yeah, Well, I was getting ready to step on the
field for the New York Jets for the first time
in nineteen ninety three.
Speaker 3 (00:38):
NFL quarterback Boomer Asiasin had just been traded to the
Jets after ten incredibly successful seasons with the Cincinnati Bengals,
a team where he'd wrapped up a Super Bowl appearance
and an MVP Award. He was born and raised in
New York, so it was exciting to play for one
of his hometown teams. But then, just as he was
(01:01):
about to walk into the field, he was called back.
Speaker 2 (01:05):
The head coach's secretary came out onto the field and said,
your wife is on the phone. You need to take
the phone call. So I took the phone call.
Speaker 3 (01:12):
It was Boomer's wife, Cheryl, who was back in Cincinnati
with their son, Gunner.
Speaker 2 (01:17):
Cheryl told me I needed to get back to Cincinnati,
that Gunner was in Cincinnati Children's Hospital and he was
having trouble breathing.
Speaker 3 (01:25):
Boomer grabbed the next flight to Cincinnati, then went straight
from the airport to the hospital.
Speaker 2 (01:31):
Seeing a two year old Gunner laying in a crib
hooked up to all sorts of different machines receiving antibiotics,
was on oxygen, and really wasn't sure what to think.
Speaker 3 (01:43):
The doctor came into the room and told Boomer and
Cheryl that their little boy had cystic fibrosis.
Speaker 2 (01:48):
That day will live with me forever as one of
the saddest days of my life.
Speaker 3 (02:00):
Hi. I'm Jordan Gospore. I'm a member of the University
of Southern California's Center for Health Journalism. This is Targeting
the Toughest Diseases a podcast produced by Bloomberg Media Studios
and Vertex Pharmaceuticals. In this series, we look at some
of humanity's most challenging diseases and how Vertex, a Boston
based biotech company, is using innovative tools, methods, and a
(02:24):
unique philosophy to search for treatments and cures. Today we're
looking at cystic fibrosis, also known as CF, a rare
chronic genetic disease affecting the lungs and other organs. It
can lead to all sorts of complications, including lung disease,
liver disease, diabetes, and even premature death. It affects about
(02:46):
thirty thousand Americans and more than eighty thousand people globally,
making it a disease not a lot of people know about,
but it was not new to Boomeris Siasin.
Speaker 2 (03:03):
Here's the really weird part of this, and maybe the
destiny and irony part of all of this. Back in
nineteen eighty nine, I was receiving award as the best
quarterback in the NFL, and at that awards banquet, there
was a speaker that night because there was a fundraising
attachment to that banquet, and his name was Frank DeFord.
Speaker 3 (03:22):
DeFord was a legendary writer at Sports Illustrated, but he
was also a CF dad.
Speaker 2 (03:27):
Frank spoke so eloquently about how he lost his daughter
Alex to the disease of cystic fibrosis, which I had
never heard of. She was eight years old, and everybody
was captured by his story. That night. I was very captured.
I was crying.
Speaker 3 (03:42):
Bumeris Iyasin was twenty seven at the time, with no
kids of his own. He was living a great life
as an NFL quarterback, but something in that speech touched
him deeply. He asked to Ford how he could help.
Speaker 2 (03:53):
And he said, well, if you go back to Cincinnati
and you can get involved in some of the fundraising
aspects of cystic fibrosis a face on it, if you will,
and then maybe visit some of the kids at Children's Hospital,
that would really be helpful.
Speaker 3 (04:06):
And that's exactly what he did. He used his celebrity
status to help raise awareness and funds for cystic fibrosis,
and he visited kids in the hospital who were sick. Now,
his son, Gunner had this same terrible disease. We all
have mucus that lines her lungs and our airways. This
mucus has an important job. It captures dust and germs
(04:27):
in the air we breathe. When mucus traps those germs
in dust, tiny little hairs on the outside of our
cells called cilia push all that mucus out of our lungs. Normally,
this mucus is slippery and watery, but for people with
cystic fibrosis, this mucus is sticky and thick. As a result,
the cilia can't push the mucus out and it ends
(04:48):
up blocking lung passages, making it hard to breathe. Also,
germs and bacteria get trapped in a person's airways, making
them more prone to infections. Clearing the mucus could be
a daily task for people with CF. There are medications
that help fin it. Some patients use a vibrating vest
to literally shake the mucus out of their airways. Others
(05:10):
may have to visit the hospital periodically for a cleanout.
And because the mucus can also block the ducks to
the pancreas, many people with CF may struggle to absorb
nutrients efficiently. Solving that can require periodic IV treatments or
taking enzymes to help break down food for absorption. IV
treatments are also used to fight frequent infections and lungs
(05:31):
that come about in many people with CF. It's a
demanding regiment, something Gunner Asiasin had to confront when he
was still very young.
Speaker 4 (05:41):
My dad, who was driving the car just looked at
me while we were driving and said, this isn't gonna hurt.
And for the entire rest of the car ride, he
continued to say, this isn't gonna hurt, This isn't gonna hurt,
this isn't gonna hurt, as if he was reassuring me
about something they weren't telling me about.
Speaker 3 (05:55):
Everything was fine until the moment Gunner realized the antibiotic
treatment he was getting for a CF related infection involved needles.
Speaker 4 (06:05):
I looked at my dad's grinding at I said, you
lied to me, and I ran out the door, down
the hallway and into the lobby of Cincinnati Children's Hospital. So,
if you can picture the scenario, there's my dad, the
starting quarterback of the Cincinni Bengals at the time, chasing
his belligerent child, and he finally comes up to me
and he's like, quiet, please be quiet, you're making a scene.
I will do whatever you want if you go back
into that room with me.
Speaker 3 (06:25):
Eventually, after a lot of persuasion, Boomer agreed to buy
Gunner a video game console if he would go and
get the treatment.
Speaker 4 (06:33):
So we walked back into interventual raiology and they scrapped
me down to the table, and I cried and screamed
to the entire thing. But you better believe that I
got my Nintendo at the end of that possible visit.
Speaker 3 (06:46):
In college, Gunner reached a turning point with how he
coexisted with the disease.
Speaker 4 (06:51):
My senior year was a real slog like I went through,
like several bounds of pneumonia. I knew that I was
at a point in my life where I had no
other options.
Speaker 3 (07:01):
That uncertainty, having big questions about the future is one
of the hardest parts of living with CF. Cystic fibrosis
is genetic. Our genes give our cells instructions on how
to produce certain proteins. They tell the cells in our lungs, pancreas,
or sweat glands to make a special protein called cystic
(07:22):
fibrosis transmembrane conductance regulator or CFTR for short. This CFTR
protein is sent to the surface of a cell. Here,
it acts like little channels between the inside and the
outside of a cell. These channels open and close, and
this allows particles like chloride ions to move in or
out of a cell, and those chloride ions help a
(07:44):
cell maintain a healthy balance of salt and water. But
for people with CF, a mutation causes the gene to
give faulty instructions to the body. Those proteins, those little
channels don't work right. This means water and salt can't
flow freely in and out of a cell like they should.
When that chloride gets trapped in a cell, it doesn't
have the fluids it needs to hydrate its surface properly.
(08:06):
Without those fluids, mucus becomes dehydrated, and that's what causes
it to be thick and sticky. Doctor Chinadou Nikuoro is
a pediatrician in London. He has spent decades researching the
condition and says we've known about CF since ancient times.
Speaker 5 (08:23):
If you go back to European proverbs, you may have
heard there was a saying, woe to that child which,
when kissed on the forehead, tastes salty. He is bewitched
and soon must die.
Speaker 3 (08:33):
Salty skin is a symptom because people with cystic fibrosis
have two to five times the normal amount of salt
in their sweat.
Speaker 5 (08:40):
And these old grandmothers in northern Germany were describing children
with cystic fibrosis.
Speaker 3 (08:46):
The genetic mutation that causes CF survived because it provides
its carriers with some extra immunity.
Speaker 5 (08:52):
Having one copy of the cistic vibrosius mutation seems to
protect against diarrehal diseases like cholera and typho. That would
have meant that people who were carriers of that gene
would be more likely to survive plagues of cholera and typhoid,
where people who didn't have any copies would have been
more likely to succumb.
Speaker 3 (09:12):
It was nineteen thirty eight that the disease was named
and made it into medical literature. It was discovered in
children who had died of malnutrition.
Speaker 5 (09:19):
The US pathologist Dorothy Anderson found that their pangresses were
damaged through being bummed up by sticky secretions, and they
were cystic and fibrotic, and that was the name given
to the disease.
Speaker 3 (09:31):
Doctor Niicuro says life expectancy for children living with CF
has been extended, thanks in large part to newborn screening
and improve treatments, but he is hopeful that treatments will
not just extend life for patients living with the disease,
it will improve their quality of life as well.
Speaker 5 (09:47):
What I hope that will lead to is a reduction
in the need for the kind of intense therapy, both
in terms of antibiotics, hospital admissions and in the even
physiotherapy that has been characteris consistent fibrousus care over the years.
Speaker 3 (10:04):
Coming up with therapies that can both prolong and improve
life is a pretty tall order, but it's a passion
for doctor David Altschuler, chief Scientific Officer at Vertex Pharmaceuticals.
As a physician and a researcher, he spent the last
thirty five years of his career scouring our DNA code
for clues about some of the world's toughest diseases.
Speaker 6 (10:26):
So the first thing we do is we look for
diseases with great unmet need, where the disease is very
serious and people's lives are impacted and there's nothing that
really is is transformational, that can really change their lives,
and cystic fibrosis certainly was that. The second was we
look for a case where the underlying human biology, the
(10:47):
underlying cause of the disease is known, and again in CF,
the cystic fibrosis gene mutation was known. What was missing
was therapeutics that could do what needed to be done,
which is, in this case, restore CF function. And that's
kind of thing that really gets our scientists and doctors
fired up, and so they worked on it for twenty
years to get where we are today.
Speaker 3 (11:08):
Can you speak a little bit more about how you
and Vertex are tackling cystic fibrosis.
Speaker 6 (11:14):
So the work that Vertex has done in CF really
goes back decades ran in families, but until nineteen eighty
nine when that gene was cloned, no one actually knew
what caused the disease.
Speaker 3 (11:25):
That changed with the discovery of CFTR.
Speaker 6 (11:28):
But then the question was could you repair it? And
for a period of decades that seemed impossible. People tried
lots of different things and couldn't figure it out. But
twenty years or more ago, scientists at Vertex had an idea.
They had an idea of how to use chemicals to
coax the CFTR protein that's not working to function again.
Speaker 3 (11:49):
That's what Vertex has been working on for the last
twenty years and now has approved medicines with the potential
to treat up to ninety percent of all people with CF,
and they're not done yet.
Speaker 6 (11:59):
The things that keeps us working hard on this problem
is that less than ten percent don't make any of
the protein.
Speaker 3 (12:06):
The way current therapies work is to coax the protein
that has an inherited mutation to function again. But some
people don't make any protein, so a different approach is needed, and.
Speaker 6 (12:17):
So we have research ongoing in our laboratories and with
a number of partners, are working hard for the last
ten percent. We're still very much in the CF story.
We still have a long way to go till we
reach our goal.
Speaker 3 (12:31):
For people with CF, like Gunner as siasin things that
once felt unimaginable now seem possible, like returning to school
to see old friends.
Speaker 4 (12:40):
I had my five year college reunion, and most people
remember me from my college a's at my last year
or two, being very very sick, like always missing things,
missing social activities, missing class, missing things like that. Yeah,
here we are at the reunion and it's like the
chance to go back to college, right, you know, We're
staying out late, and next thing you know, I'm up
every morning at like six point thirty getting breakfast for everyone.
And my friends were like, what is going on with you?
Speaker 3 (13:00):
But as great as the weekend was The drive home
with his girlfriend was even more impactful.
Speaker 4 (13:04):
We were stuck into New Jersey Turnpike and she looked
at me and she was like, do you realize what
we did this weekend? We went away and we're able
to do something. What do you want to do next
with your life? And I paused for a moment. It
hadn't occurred to me that I could do something with
my life. I didn't have to live at home anymore.
Speaker 3 (13:21):
What they decided to do next was get married.
Speaker 2 (13:24):
I've never been at a wedding where everybody, I mean
all two hundred and fifty people just came together as
one group and we're crying and we're laughing and we're cheering,
and it was like the greatest moment of all of
our lives.
Speaker 3 (13:38):
Going back to school, getting married, those experiences are possible
because of one thing. Hope. Well.
Speaker 2 (13:45):
I don't know how much of a football fan you are,
but when you play for the Jets, Cardinals and Bengals,
it's all about hope. You need as much hope as
you possibly can get.
Speaker 4 (13:53):
You know. I think for me, I mean honestly, I
hope is going to define the rest of my career.
When I was going through the worst today, I had
a little quote on my desk and it was a
Wizard Churchill quote and it was something like, if you're
going through hell, keep going. And that meant a lot
to me back then.
Speaker 3 (14:17):
This is Targeting the Toughest Diseases a podcast from Bloomberg
Media Studios and Vertex Pharmaceuticals. If you like what you hear,
subscribe and leave us a review. I'm Jordan Gosfore. Thanks
for listening.
Hosts And Creators
Tim Stenovec
Carol Massar
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