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: I'm so goal oriented. I'm always just charging ahead, like
Speaker 2: we're going to get it done by any means possible.
Speaker 3: Focused, and fearless. That's the best way to describe Madison Carter.
Speaker 3: Those qualities have helped Madison break a lot of important
Speaker 3: stories as an award winning investigative reporter in Atlanta, Georgia.
Speaker 3: She's investigated white nationalists, political corruption, and the parole system.
Speaker 3: Those are stories that take a lot of guts to
Speaker 3: and Madison has done it all without hesitation.
Speaker 2: People will tell you I go for everything, I say
Speaker 2: everything that needs to be said, like I have no fear,
Speaker 2: like I don't have a lot to lose.
Speaker 3: That fearlessness comes from a dark place.
Speaker 2: It's because of my diabetes. I'm like I could die
Speaker 2: at any second. Hi.
Speaker 3: I'm Jordan Gospore, a member of the University of Southern
Speaker 3: California's Center for Health Journalism. This is Targeting the Toughest
Speaker 3: Diseases produced by Bloomberg Media Studios and Vertex Pharmaceuticals. In
Speaker 3: this series, we look at some of humanity's most challenging
Speaker 3: diseases and how Vertex, a Boston based biotech company, is
Speaker 3: using innovative tools, methods, and a unique philosophy to search
Speaker 3: for treatments and cures. Today, we're targeting type one diabetes,
Speaker 3: a chronic disease that affects more than eight million people
Speaker 3: around the world, a number that's expected to increase rapidly.
Speaker 3: In the US alone, approximately one point four million people,
Speaker 3: including nearly one hundred and seventy thousand under the age
Speaker 3: of twenty, have type one diabetes.
Speaker 2: I don't think I ever had the opportunity to be
Speaker 2: a kid.
Speaker 3: Since she was three years old, Madison's life has included
Speaker 3: a highly controlled diet, constantly measuring her blood sugar levels,
Speaker 3: and insolent shots.
Speaker 2: And it's something that I have spent the past couple
Speaker 2: years in therapy really working through because it's not fair
Speaker 2: it's not just that I feel like it's not fair.
Speaker 4: It is not.
Speaker 3: When she was just a toddler, Madison started showing signs
Speaker 3: that something was off.
Speaker 2: I was getting really, really skinny, which is weird because
Speaker 2: the people in my family are not very skinny.
Speaker 3: She was thin, feeling thirsty all the time, and constantly tired.
Speaker 2: I would be falling asleep in random places. My mom
Speaker 2: found me asleep next to my oatmeal one morning, found
Speaker 2: me asleep on the toilet one day. You know, I'm
Speaker 2: a toddler. I'm three years old, so that was really strained,
Speaker 2: especially when I was supposed to be running around. And
Speaker 2: one night my mom was putting me to bed and
Speaker 2: she said that she could count every single one of
Speaker 2: my ribs, and in that moment, she picked me up,
Speaker 2: put me in the back of the car, gave me
Speaker 2: a sprite, which later on we would find out was
Speaker 2: a very bad idea, and took me to Children's Hospital.
Speaker 3: When they got there, the medical team found Madison's blood
Speaker 3: sugar level was dangerously elevated, nearly two hundred percent higher
Speaker 3: than it should have been.
Speaker 2: They told my mom I should be dead. Had she
Speaker 2: had put me to bed that night, I would have
Speaker 2: been dead, and that's how I was diagnosed. I think
Speaker 2: my parents had about a year of giving me injections
Speaker 2: until I told them to never touch me with the
Speaker 2: needle again.
Speaker 3: By the time she was five, Madison was doing all
Speaker 3: her injections herself.
Speaker 2: It was harder for me to see my mom inflict
Speaker 2: pain me than it was for me to just do
Speaker 2: it myself. Like seeing her face that upset me. It
Speaker 2: wasn't even that it hurt, because it hurts sometimes when
Speaker 2: I would do it myself. But I just didn't want
Speaker 2: her to have that responsibility of feeling like she was
Speaker 2: hurting me.
Speaker 3: Our pancreas produces insulin, a special hormone that the body
Speaker 3: needs to process glucose, a key source of energy. For
Speaker 3: people who have type one diabetes, those insulin producing cells
Speaker 3: in the pancreas are destroyed. That means the glucose stays
Speaker 3: in the blood, resulting in high blood sugar levels, also
Speaker 3: known as hyperglycemia. The symptoms include increased thirst, hunger, frequent urination,
Speaker 3: and weight loss. If left untreated, this can lead to
Speaker 3: excessively high glucose levels and eventually could even be fatal.
Speaker 3: People with type one diabetes rely on giving themselves insulin
Speaker 3: every single day to survive and have to be super vigilant.
Speaker 3: Not enough insulin can lead to high blood glucose levels
Speaker 3: and over time complications like kidney disease, vision loss, nerve damage,
Speaker 3: heart attacks, and strokes. Too much insulin can result in
Speaker 3: low blood sugar. If left unaddressed, blood glucose levels can
Speaker 3: fall dangerously low and can lead to severe hypoglycemia, resulting
Speaker 3: in seizures, loss of consciousness, or even death.
Speaker 2: I remember it was President's Day weekend. She was playing volleyball,
Speaker 2: so she had this tournament.
Speaker 3: Madison's younger sister, Cameron, also had type one diabetes.
Speaker 2: She was fourteen and I got pulled out of class.
Speaker 2: They said, hey, you know we're going to take you
Speaker 2: to the hospital. Your sister had a seizure.
Speaker 3: Madison rushed to the hospital.
Speaker 2: My parents met us at the door and they said,
Speaker 2: you know, she didn't make it. She was dead when
Speaker 2: they took her in the ambulance.
Speaker 3: Her sister's sudden death from diabetes devastated Madison.
Speaker 2: I would call her the good diabetic, like she really
Speaker 2: did everything she needed to do, and I was kind
Speaker 2: of in that phase of doing just enough to stay alive.
Speaker 2: And she died and that upset me, and I was like, well,
Speaker 2: what is the point of doing everything you're supposed to
Speaker 2: do like my sister Cameron did, if it's still not enough.
Speaker 3: Madison lost all motivation to manage her own health for.
Speaker 2: A year after she died. I told people that I
Speaker 2: took a year off of being diabetic. The responsibility is
Speaker 2: the checking your blood sugar and the making sure you're
Speaker 2: taking your injections or changing your pump. I refused to
Speaker 2: do and my mom stepped in and did it for me.
Speaker 3: After a year, Madison resumed doing her own care. It's
Speaker 3: a daily grind, but it keeps her alive.
Speaker 5: It's a very, very difficult disease to manage.
Speaker 3: The constant managing of type one diabetes is something doctor
Speaker 3: Aaron Kowalsky knows intimately.
Speaker 5: When my brother was diagnosed in the late seventies, you
Speaker 5: were pretty much told that you had about twenty years
Speaker 5: before complications would form. Your life would be shortened pretty significantly.
Speaker 5: You had a very high likelihood of going blind. You
Speaker 5: had the dosing of insulin issues that could cost of
Speaker 5: your hypoglycemia and potentially death on a daily basis.
Speaker 3: A few years after his brother was diagnosed, Kowalski discovered
Speaker 3: he had type one diabetes as well. Today he's the
Speaker 3: CEO of JDRF, the world's largest nonprofit funder of type
Speaker 3: one diabetes research.
Speaker 5: We are just celebrating the one hundred year anniversary of
Speaker 5: the discovery of insulin, which won multiple Nobel Prizes and
Speaker 5: saved millions of lives. Before the discovery of insulin in
Speaker 5: nineteen twenty one, every single Type one person died.
Speaker 3: That's every single person for centuries.
Speaker 5: The disease actually was described in ancient Egyptian and Greek writings,
Speaker 5: and this observation that people made was the fact that
Speaker 5: people with this syndrome wasted away.
Speaker 3: The term diabetes melitis was coined because it roughly translates
Speaker 3: to sweet siphon.
Speaker 5: The sugar which just passed through people, the food which
Speaker 5: just passed through them, and the sweetness was observed when
Speaker 5: ants would be attracted to the urine of these people.
Speaker 3: Modern times have brought us modern discoveries. Apart from the
Speaker 3: breakthrough of insulin, there was a landmark study in nineteen
Speaker 3: eighty three called the DCCT trial, which stands for the
Speaker 3: Diabetes Control and Complications Trial it showed the connection between
Speaker 3: high blood sugar and complications. It also showed that with
Speaker 3: more intensive therapy, people with type one diabetes could prevent
Speaker 3: long term eye, kidney, and nerve complications. And more recently,
Speaker 3: there's been huge improvements in glucose monitoring. This includes smartphone
Speaker 3: apps with sensors which can monitor blood sugar levels continually.
Speaker 5: Not surprisingly, if you have more information, you do better.
Speaker 5: I certainly think that's been one of the biggest changes
Speaker 5: in my life, in my brother's life.
Speaker 3: Despite all those improvements in managing type one diabetes, there's
Speaker 3: still no cure and people with the disease are still
Speaker 3: at risk of developing really severe, potentially fatal complications.
Speaker 6: Type one diabetes has such a significant burden on individuals,
Speaker 6: on families, on communities, and it is a twenty four
Speaker 6: to seven relentless job.
Speaker 3: That's doctor Felicia Pelayuka. She's the vice president and Disease
Speaker 3: Area Executive for type one diabetes at Vertex. They are
Speaker 3: researching type one diabetes at the cellular level, something she
Speaker 3: first got interested in when she was a student.
Speaker 6: It was really a lecture that I saw when I
Speaker 6: was a PhD student by Professor Doug Maltain that turn
Speaker 6: that paradigm on its head, that not only could we
Speaker 6: think about cells as being a cause of disease, but
Speaker 6: think about cells as being a solution to diseases.
Speaker 3: That's what Vertex is investigating cell therapy as a potential
Speaker 3: treatment for type one diabetes.
Speaker 6: Vertex has a really unique research and development strategy that
Speaker 6: focuses first and foremost on diseases where we understand the
Speaker 6: causal biology really well, so that we can rationally design
Speaker 6: therapies that could have a major transformative impact on patients
Speaker 6: if we're successful in developing them.
Speaker 3: Type one diabetes definitely fits that description clearly.
Speaker 6: It's a disease with enormous unmet medical need. It's a
Speaker 6: disease where we understand that these missing insulin producing islet
Speaker 6: cells are at the heart of the disease.
Speaker 3: And this knowledge is what drives vertexs forward.
Speaker 6: Science has advanced to a place where even a decade
Speaker 6: ago go, it was unthinkable, it was science fiction to
Speaker 6: think that you could make replacement isletself in the laboratory,
Speaker 6: and so to be at the moment now it really
Speaker 6: feels like an inflection point, and we hope it really
Speaker 6: will be in the history of the treatment of type
Speaker 6: one diabetes.
Speaker 3: The professor who first inspired doctor Palayuka to think of
Speaker 3: cells as living problem solvers was doctor Doug Melton, one
Speaker 3: of the world's leading stem cell researchers. A former Harvard professor,
Speaker 3: He's been in this area of research for more than
Speaker 3: twenty years, and this year he joined Vertex as a
Speaker 3: Distinguished Fellow. But working on an investigational treatment for type
Speaker 3: one diabetes wasn't always on his radar.
Speaker 4: Well. I didn't think much about diabetes until my six
Speaker 4: month old son contracted the disease. From a parental point
Speaker 4: of view, you have to manage that little baby's blood
Speaker 4: sugars by picking their fingers or their toes and squeezing
Speaker 4: blood out and measuring how much sugar there is and
Speaker 4: then inject them with insulin. You can imagine how hard
Speaker 4: it is. And a six month old that doesn't talk.
Speaker 3: His son, Sam, was the youngest person at Children's Hospital
Speaker 3: in Boston to be diagnosed with type one diabetes.
Speaker 4: My wife had to deal with the fact that she
Speaker 4: was also breastfeeding Sam at the time. When you're eating
Speaker 4: food like a candy bar or an apple or something,
Speaker 4: you know how much sugar you're taking in, but you
Speaker 4: can't tell how much sugar you take in or energy
Speaker 4: from breast milk because you don't know what the volume is.
Speaker 4: So she's up all night testing his blood sugars and
Speaker 4: injecting insulin. And she looked at me more or less
Speaker 4: and said, look, you're a scientist, go do something about this.
Speaker 3: And so he set out to do just that. It
Speaker 3: was understood that people with type one were missing insulin
Speaker 3: producing cells, also known as beta cells. Doctor Melton figured
Speaker 3: if the beta cells were missing, they needed to make
Speaker 3: new ones.
Speaker 4: There was this nice commercial about a man he would
Speaker 4: say time to make the donuts, and so I used
Speaker 4: to go to lab and say time to make beta cells.
Speaker 4: That was our job.
Speaker 3: I love that. What are the differences between type one
Speaker 3: and type two diabetes.
Speaker 4: Well, type one diabetes is caused by the problem that
Speaker 4: one's immune system makes a mistake. Instead of just attacking
Speaker 4: foreign entities like a virus, it decides, for reasons we
Speaker 4: don't understand, to attack the patient's own beta cells and
Speaker 4: destroys those cells. That is not the cause of type
Speaker 4: two diabetes. And type two diabetes the patient still makes insulin,
Speaker 4: but their body has a greater and greater demand for insulin.
Speaker 4: So it's called insulin resistance, meaning that the patients muscle
Speaker 4: and fat cells require more and more insulin all the time.
Speaker 4: Type two diabetes can be treated in many cases by
Speaker 4: changes in diet and exercise. That is not true for
Speaker 4: type one, so no behavioral change will prevent or treat
Speaker 4: type one diabetes.
Speaker 3: What was that aha moment?
Speaker 4: The real turning point was about in twenty thirteen twenty
Speaker 4: fourteen where we figured out the right factors or molecules
Speaker 4: to give to the cells that made them respond to glucose.
Speaker 4: Because the beta cells job is to read or measure
Speaker 4: the amount of sugar in the blood and then squirt
Speaker 4: out just the right amount of insulin. So the moment
Speaker 4: where we had a test and when we added glucose,
Speaker 4: the cells squirted out insulin and then we knew we
Speaker 4: were on the right track.
Speaker 3: As part of his work at Vertex, doctor Melton is
Speaker 3: helping develop a potential treatment that addresses the root cause
Speaker 3: of type one diabetes, the absence of those insulin producing
Speaker 3: cells in the pancreas. The company is investigating how to
Speaker 3: replace those destroyed cells with insulin producing cells and making
Speaker 3: sure they can get into patients in a way where
Speaker 3: they can survive and function for a long time, and
Speaker 3: progress so far gives doctor Melton competence and their path forward.
Speaker 4: Let's put it this way. If thirty years ago you
Speaker 4: had told someone you would take a human stem cell
Speaker 4: and turn it into functional insulin producing cells, people would
Speaker 4: have said, yeah, good luck with that.
Speaker 3: One last thing that doctor Milton told me something that
Speaker 3: made me smile the rest of the day. He says
Speaker 3: that everywhere he goes he always carries a little vial
Speaker 3: in his pocket.
Speaker 4: It has in it cells that have been turned into
Speaker 4: insulin producing cells. Sometimes I take it out and like
Speaker 4: a snow globe and just turn it up and down
Speaker 4: and look at it, because it reminds me that this
Speaker 4: is possible. We now know the problem. We just have
Speaker 4: to do it.
Speaker 3: Madison Carter is hoping for a better treatment for type
Speaker 3: one diabetes because she doesn't want another generation of young
Speaker 3: people to feel how she did.
Speaker 2: I was like, I don't want to take the shot.
Speaker 2: I don't want to do this again tomorrow. I don't
Speaker 2: want to not that I wanted to die, but I
Speaker 2: just did not want to work this hard to be alive, to.
Speaker 3: Ease that burden. She's now looking to technology.
Speaker 2: Things have improved so much. Having a continuous glucose monitor
Speaker 2: is probably one of the best inventions. So now I
Speaker 2: can glance at my blood sugars and I can then
Speaker 2: pick up my insulin pump, which is right next to
Speaker 2: that device, and just input the numbers.
Speaker 3: And maybe more important than tech, Madison still has her
Speaker 3: fearless determination.
Speaker 2: I had a friend point out to me. She goes, Oh,
Speaker 2: that's why you are the way you are, you know,
Speaker 2: And I didn't even realize that. I didn't even associate
Speaker 2: it back to my diabetes.
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 Gosbory. Thanks
Speaker 3: for listening.