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