WEBVTT - Targeting Type 1 Diabetes (Sponsored Content)

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<v Speaker 1>Since you're a subscriber to this Bloomberg podcast, we thought

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<v Speaker 1>you'd be interested in a six episode sponsored podcast called

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<v Speaker 1>Targeting the Toughest Diseases, produced by Vertex Pharmaceuticals and Bloomberg

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<v Speaker 1>Media Studios. It explores the innovative tools, methods, and unique

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<v Speaker 1>philosophy Vertex Pharmaceuticals is using to search for treatments for

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<v Speaker 1>some of humanity's most challenging diseases. Here's a recent episode.

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<v Speaker 2>I'm so goal oriented. I'm always just charging ahead, like

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<v Speaker 2>we're going to get it done by any means possible.

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<v Speaker 3>Focused, and fearless. That's the best way to describe Madison Carter.

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<v Speaker 3>Those qualities have helped Madison break a lot of important

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<v Speaker 3>stories as an award winning investigative reporter in Atlanta, Georgia.

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<v Speaker 3>She's investigated white nationalists, political corruption, and the parole system.

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<v Speaker 3>Those are stories that take a lot of guts to

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<v Speaker 3>and Madison has done it all without hesitation.

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<v Speaker 2>People will tell you I go for everything, I say

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<v Speaker 2>everything that needs to be said, like I have no fear,

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<v Speaker 2>like I don't have a lot to lose.

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<v Speaker 3>That fearlessness comes from a dark place.

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<v Speaker 2>It's because of my diabetes. I'm like I could die

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<v Speaker 2>at any second. Hi.

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<v Speaker 3>I'm Jordan Gospore, a member of the University of Southern

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<v Speaker 3>California's Center for Health Journalism. This is Targeting the Toughest

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<v Speaker 3>Diseases produced by Bloomberg Media Studios and Vertex Pharmaceuticals. In

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<v Speaker 3>this series, we look at some of humanity's most challenging

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<v Speaker 3>diseases and how Vertex, a Boston based biotech company, is

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<v Speaker 3>using innovative tools, methods, and a unique philosophy to search

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<v Speaker 3>for treatments and cures. Today, we're targeting type one diabetes,

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<v Speaker 3>a chronic disease that affects more than eight million people

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<v Speaker 3>around the world, a number that's expected to increase rapidly.

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<v Speaker 3>In the US alone, approximately one point four million people,

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<v Speaker 3>including nearly one hundred and seventy thousand under the age

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<v Speaker 3>of twenty, have type one diabetes.

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<v Speaker 2>I don't think I ever had the opportunity to be

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<v Speaker 2>a kid.

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<v Speaker 3>Since she was three years old, Madison's life has included

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<v Speaker 3>a highly controlled diet, constantly measuring her blood sugar levels,

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<v Speaker 3>and insolent shots.

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<v Speaker 2>And it's something that I have spent the past couple

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<v Speaker 2>years in therapy really working through because it's not fair

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<v Speaker 2>it's not just that I feel like it's not fair.

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<v Speaker 4>It is not.

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<v Speaker 3>When she was just a toddler, Madison started showing signs

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<v Speaker 3>that something was off.

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<v Speaker 2>I was getting really, really skinny, which is weird because

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<v Speaker 2>the people in my family are not very skinny.

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<v Speaker 3>She was thin, feeling thirsty all the time, and constantly tired.

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<v Speaker 2>I would be falling asleep in random places. My mom

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<v Speaker 2>found me asleep next to my oatmeal one morning, found

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<v Speaker 2>me asleep on the toilet one day. You know, I'm

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<v Speaker 2>a toddler. I'm three years old, so that was really strained,

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<v Speaker 2>especially when I was supposed to be running around. And

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<v Speaker 2>one night my mom was putting me to bed and

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<v Speaker 2>she said that she could count every single one of

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<v Speaker 2>my ribs, and in that moment, she picked me up,

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<v Speaker 2>put me in the back of the car, gave me

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<v Speaker 2>a sprite, which later on we would find out was

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<v Speaker 2>a very bad idea, and took me to Children's Hospital.

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<v Speaker 3>When they got there, the medical team found Madison's blood

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<v Speaker 3>sugar level was dangerously elevated, nearly two hundred percent higher

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<v Speaker 3>than it should have been.

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<v Speaker 2>They told my mom I should be dead. Had she

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<v Speaker 2>had put me to bed that night, I would have

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<v Speaker 2>been dead, and that's how I was diagnosed. I think

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<v Speaker 2>my parents had about a year of giving me injections

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<v Speaker 2>until I told them to never touch me with the

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<v Speaker 2>needle again.

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<v Speaker 3>By the time she was five, Madison was doing all

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<v Speaker 3>her injections herself.

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<v Speaker 2>It was harder for me to see my mom inflict

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<v Speaker 2>pain me than it was for me to just do

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<v Speaker 2>it myself. Like seeing her face that upset me. It

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<v Speaker 2>wasn't even that it hurt, because it hurts sometimes when

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<v Speaker 2>I would do it myself. But I just didn't want

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<v Speaker 2>her to have that responsibility of feeling like she was

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<v Speaker 2>hurting me.

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<v Speaker 3>Our pancreas produces insulin, a special hormone that the body

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<v Speaker 3>needs to process glucose, a key source of energy. For

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<v Speaker 3>people who have type one diabetes, those insulin producing cells

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<v Speaker 3>in the pancreas are destroyed. That means the glucose stays

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<v Speaker 3>in the blood, resulting in high blood sugar levels, also

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<v Speaker 3>known as hyperglycemia. The symptoms include increased thirst, hunger, frequent urination,

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<v Speaker 3>and weight loss. If left untreated, this can lead to

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<v Speaker 3>excessively high glucose levels and eventually could even be fatal.

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<v Speaker 3>People with type one diabetes rely on giving themselves insulin

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<v Speaker 3>every single day to survive and have to be super vigilant.

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<v Speaker 3>Not enough insulin can lead to high blood glucose levels

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<v Speaker 3>and over time complications like kidney disease, vision loss, nerve damage,

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<v Speaker 3>heart attacks, and strokes. Too much insulin can result in

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<v Speaker 3>low blood sugar. If left unaddressed, blood glucose levels can

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<v Speaker 3>fall dangerously low and can lead to severe hypoglycemia, resulting

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<v Speaker 3>in seizures, loss of consciousness, or even death.

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<v Speaker 2>I remember it was President's Day weekend. She was playing volleyball,

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<v Speaker 2>so she had this tournament.

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<v Speaker 3>Madison's younger sister, Cameron, also had type one diabetes.

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<v Speaker 2>She was fourteen and I got pulled out of class.

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<v Speaker 2>They said, hey, you know we're going to take you

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<v Speaker 2>to the hospital. Your sister had a seizure.

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<v Speaker 3>Madison rushed to the hospital.

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<v Speaker 2>My parents met us at the door and they said,

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<v Speaker 2>you know, she didn't make it. She was dead when

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<v Speaker 2>they took her in the ambulance.

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<v Speaker 3>Her sister's sudden death from diabetes devastated Madison.

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<v Speaker 2>I would call her the good diabetic, like she really

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<v Speaker 2>did everything she needed to do, and I was kind

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<v Speaker 2>of in that phase of doing just enough to stay alive.

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<v Speaker 2>And she died and that upset me, and I was like, well,

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<v Speaker 2>what is the point of doing everything you're supposed to

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<v Speaker 2>do like my sister Cameron did, if it's still not enough.

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<v Speaker 3>Madison lost all motivation to manage her own health for.

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<v Speaker 2>A year after she died. I told people that I

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<v Speaker 2>took a year off of being diabetic. The responsibility is

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<v Speaker 2>the checking your blood sugar and the making sure you're

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<v Speaker 2>taking your injections or changing your pump. I refused to

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<v Speaker 2>do and my mom stepped in and did it for me.

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<v Speaker 3>After a year, Madison resumed doing her own care. It's

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<v Speaker 3>a daily grind, but it keeps her alive.

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<v Speaker 5>It's a very, very difficult disease to manage.

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<v Speaker 3>The constant managing of type one diabetes is something doctor

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<v Speaker 3>Aaron Kowalsky knows intimately.

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<v Speaker 5>When my brother was diagnosed in the late seventies, you

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<v Speaker 5>were pretty much told that you had about twenty years

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<v Speaker 5>before complications would form. Your life would be shortened pretty significantly.

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<v Speaker 5>You had a very high likelihood of going blind. You

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<v Speaker 5>had the dosing of insulin issues that could cost of

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<v Speaker 5>your hypoglycemia and potentially death on a daily basis.

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<v Speaker 3>A few years after his brother was diagnosed, Kowalski discovered

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<v Speaker 3>he had type one diabetes as well. Today he's the

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<v Speaker 3>CEO of JDRF, the world's largest nonprofit funder of type

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<v Speaker 3>one diabetes research.

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<v Speaker 5>We are just celebrating the one hundred year anniversary of

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<v Speaker 5>the discovery of insulin, which won multiple Nobel Prizes and

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<v Speaker 5>saved millions of lives. Before the discovery of insulin in

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<v Speaker 5>nineteen twenty one, every single Type one person died.

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<v Speaker 3>That's every single person for centuries.

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<v Speaker 5>The disease actually was described in ancient Egyptian and Greek writings,

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<v Speaker 5>and this observation that people made was the fact that

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<v Speaker 5>people with this syndrome wasted away.

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<v Speaker 3>The term diabetes melitis was coined because it roughly translates

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<v Speaker 3>to sweet siphon.

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<v Speaker 5>The sugar which just passed through people, the food which

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<v Speaker 5>just passed through them, and the sweetness was observed when

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<v Speaker 5>ants would be attracted to the urine of these people.

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<v Speaker 3>Modern times have brought us modern discoveries. Apart from the

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<v Speaker 3>breakthrough of insulin, there was a landmark study in nineteen

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<v Speaker 3>eighty three called the DCCT trial, which stands for the

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<v Speaker 3>Diabetes Control and Complications Trial it showed the connection between

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<v Speaker 3>high blood sugar and complications. It also showed that with

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<v Speaker 3>more intensive therapy, people with type one diabetes could prevent

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<v Speaker 3>long term eye, kidney, and nerve complications. And more recently,

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<v Speaker 3>there's been huge improvements in glucose monitoring. This includes smartphone

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<v Speaker 3>apps with sensors which can monitor blood sugar levels continually.

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<v Speaker 5>Not surprisingly, if you have more information, you do better.

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<v Speaker 5>I certainly think that's been one of the biggest changes

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<v Speaker 5>in my life, in my brother's life.

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<v Speaker 3>Despite all those improvements in managing type one diabetes, there's

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<v Speaker 3>still no cure and people with the disease are still

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<v Speaker 3>at risk of developing really severe, potentially fatal complications.

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<v Speaker 6>Type one diabetes has such a significant burden on individuals,

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<v Speaker 6>on families, on communities, and it is a twenty four

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<v Speaker 6>to seven relentless job.

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<v Speaker 3>That's doctor Felicia Pelayuka. She's the vice president and Disease

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<v Speaker 3>Area Executive for type one diabetes at Vertex. They are

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<v Speaker 3>researching type one diabetes at the cellular level, something she

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<v Speaker 3>first got interested in when she was a student.

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<v Speaker 6>It was really a lecture that I saw when I

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<v Speaker 6>was a PhD student by Professor Doug Maltain that turn

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<v Speaker 6>that paradigm on its head, that not only could we

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<v Speaker 6>think about cells as being a cause of disease, but

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<v Speaker 6>think about cells as being a solution to diseases.

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<v Speaker 3>That's what Vertex is investigating cell therapy as a potential

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<v Speaker 3>treatment for type one diabetes.

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<v Speaker 6>Vertex has a really unique research and development strategy that

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<v Speaker 6>focuses first and foremost on diseases where we understand the

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<v Speaker 6>causal biology really well, so that we can rationally design

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<v Speaker 6>therapies that could have a major transformative impact on patients

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<v Speaker 6>if we're successful in developing them.

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<v Speaker 3>Type one diabetes definitely fits that description clearly.

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<v Speaker 6>It's a disease with enormous unmet medical need. It's a

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<v Speaker 6>disease where we understand that these missing insulin producing islet

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<v Speaker 6>cells are at the heart of the disease.

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<v Speaker 3>And this knowledge is what drives vertexs forward.

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<v Speaker 6>Science has advanced to a place where even a decade

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<v Speaker 6>ago go, it was unthinkable, it was science fiction to

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<v Speaker 6>think that you could make replacement isletself in the laboratory,

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<v Speaker 6>and so to be at the moment now it really

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<v Speaker 6>feels like an inflection point, and we hope it really

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<v Speaker 6>will be in the history of the treatment of type

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<v Speaker 6>one diabetes.

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<v Speaker 3>The professor who first inspired doctor Palayuka to think of

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<v Speaker 3>cells as living problem solvers was doctor Doug Melton, one

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<v Speaker 3>of the world's leading stem cell researchers. A former Harvard professor,

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<v Speaker 3>He's been in this area of research for more than

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<v Speaker 3>twenty years, and this year he joined Vertex as a

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<v Speaker 3>Distinguished Fellow. But working on an investigational treatment for type

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<v Speaker 3>one diabetes wasn't always on his radar.

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<v Speaker 4>Well. I didn't think much about diabetes until my six

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<v Speaker 4>month old son contracted the disease. From a parental point

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<v Speaker 4>of view, you have to manage that little baby's blood

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<v Speaker 4>sugars by picking their fingers or their toes and squeezing

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<v Speaker 4>blood out and measuring how much sugar there is and

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<v Speaker 4>then inject them with insulin. You can imagine how hard

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<v Speaker 4>it is. And a six month old that doesn't talk.

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<v Speaker 3>His son, Sam, was the youngest person at Children's Hospital

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<v Speaker 3>in Boston to be diagnosed with type one diabetes.

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<v Speaker 4>My wife had to deal with the fact that she

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<v Speaker 4>was also breastfeeding Sam at the time. When you're eating

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<v Speaker 4>food like a candy bar or an apple or something,

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<v Speaker 4>you know how much sugar you're taking in, but you

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<v Speaker 4>can't tell how much sugar you take in or energy

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<v Speaker 4>from breast milk because you don't know what the volume is.

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<v Speaker 4>So she's up all night testing his blood sugars and

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<v Speaker 4>injecting insulin. And she looked at me more or less

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<v Speaker 4>and said, look, you're a scientist, go do something about this.

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<v Speaker 3>And so he set out to do just that. It

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<v Speaker 3>was understood that people with type one were missing insulin

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<v Speaker 3>producing cells, also known as beta cells. Doctor Melton figured

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<v Speaker 3>if the beta cells were missing, they needed to make

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<v Speaker 3>new ones.

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<v Speaker 4>There was this nice commercial about a man he would

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<v Speaker 4>say time to make the donuts, and so I used

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<v Speaker 4>to go to lab and say time to make beta cells.

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<v Speaker 4>That was our job.

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<v Speaker 3>I love that. What are the differences between type one

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<v Speaker 3>and type two diabetes.

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<v Speaker 4>Well, type one diabetes is caused by the problem that

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<v Speaker 4>one's immune system makes a mistake. Instead of just attacking

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<v Speaker 4>foreign entities like a virus, it decides, for reasons we

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<v Speaker 4>don't understand, to attack the patient's own beta cells and

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<v Speaker 4>destroys those cells. That is not the cause of type

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<v Speaker 4>two diabetes. And type two diabetes the patient still makes insulin,

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<v Speaker 4>but their body has a greater and greater demand for insulin.

0:13:36.760 --> 0:13:41.559
<v Speaker 4>So it's called insulin resistance, meaning that the patients muscle

0:13:41.720 --> 0:13:45.120
<v Speaker 4>and fat cells require more and more insulin all the time.

0:13:45.280 --> 0:13:48.720
<v Speaker 4>Type two diabetes can be treated in many cases by

0:13:48.880 --> 0:13:52.280
<v Speaker 4>changes in diet and exercise. That is not true for

0:13:52.400 --> 0:13:56.360
<v Speaker 4>type one, so no behavioral change will prevent or treat

0:13:56.520 --> 0:13:57.640
<v Speaker 4>type one diabetes.

0:13:58.040 --> 0:13:59.120
<v Speaker 3>What was that aha moment?

0:13:59.480 --> 0:14:03.520
<v Speaker 4>The real turning point was about in twenty thirteen twenty

0:14:03.600 --> 0:14:07.640
<v Speaker 4>fourteen where we figured out the right factors or molecules

0:14:07.679 --> 0:14:10.440
<v Speaker 4>to give to the cells that made them respond to glucose.

0:14:10.679 --> 0:14:13.760
<v Speaker 4>Because the beta cells job is to read or measure

0:14:13.800 --> 0:14:16.440
<v Speaker 4>the amount of sugar in the blood and then squirt

0:14:16.480 --> 0:14:19.480
<v Speaker 4>out just the right amount of insulin. So the moment

0:14:19.680 --> 0:14:23.120
<v Speaker 4>where we had a test and when we added glucose,

0:14:23.160 --> 0:14:25.680
<v Speaker 4>the cells squirted out insulin and then we knew we

0:14:25.680 --> 0:14:26.600
<v Speaker 4>were on the right track.

0:14:30.120 --> 0:14:32.680
<v Speaker 3>As part of his work at Vertex, doctor Melton is

0:14:32.720 --> 0:14:36.520
<v Speaker 3>helping develop a potential treatment that addresses the root cause

0:14:36.640 --> 0:14:40.600
<v Speaker 3>of type one diabetes, the absence of those insulin producing

0:14:40.640 --> 0:14:43.760
<v Speaker 3>cells in the pancreas. The company is investigating how to

0:14:43.800 --> 0:14:47.920
<v Speaker 3>replace those destroyed cells with insulin producing cells and making

0:14:47.960 --> 0:14:51.120
<v Speaker 3>sure they can get into patients in a way where

0:14:51.160 --> 0:14:54.760
<v Speaker 3>they can survive and function for a long time, and

0:14:54.880 --> 0:14:58.360
<v Speaker 3>progress so far gives doctor Melton competence and their path forward.

0:14:58.840 --> 0:15:01.120
<v Speaker 4>Let's put it this way. If thirty years ago you

0:15:01.160 --> 0:15:04.040
<v Speaker 4>had told someone you would take a human stem cell

0:15:04.160 --> 0:15:07.520
<v Speaker 4>and turn it into functional insulin producing cells, people would

0:15:07.560 --> 0:15:08.880
<v Speaker 4>have said, yeah, good luck with that.

0:15:11.640 --> 0:15:14.440
<v Speaker 3>One last thing that doctor Milton told me something that

0:15:14.480 --> 0:15:16.720
<v Speaker 3>made me smile the rest of the day. He says

0:15:16.760 --> 0:15:20.160
<v Speaker 3>that everywhere he goes he always carries a little vial

0:15:20.240 --> 0:15:21.000
<v Speaker 3>in his pocket.

0:15:21.240 --> 0:15:24.480
<v Speaker 4>It has in it cells that have been turned into

0:15:24.560 --> 0:15:27.440
<v Speaker 4>insulin producing cells. Sometimes I take it out and like

0:15:27.480 --> 0:15:29.160
<v Speaker 4>a snow globe and just turn it up and down

0:15:29.160 --> 0:15:31.240
<v Speaker 4>and look at it, because it reminds me that this

0:15:31.440 --> 0:15:33.840
<v Speaker 4>is possible. We now know the problem. We just have

0:15:33.920 --> 0:15:34.360
<v Speaker 4>to do it.

0:15:40.240 --> 0:15:43.160
<v Speaker 3>Madison Carter is hoping for a better treatment for type

0:15:43.200 --> 0:15:47.200
<v Speaker 3>one diabetes because she doesn't want another generation of young

0:15:47.240 --> 0:15:49.040
<v Speaker 3>people to feel how she did.

0:15:49.200 --> 0:15:50.680
<v Speaker 2>I was like, I don't want to take the shot.

0:15:50.960 --> 0:15:53.960
<v Speaker 2>I don't want to do this again tomorrow. I don't

0:15:54.000 --> 0:15:56.640
<v Speaker 2>want to not that I wanted to die, but I

0:15:56.720 --> 0:16:01.400
<v Speaker 2>just did not want to work this hard to be alive, to.

0:16:01.320 --> 0:16:04.000
<v Speaker 3>Ease that burden. She's now looking to technology.

0:16:04.360 --> 0:16:07.440
<v Speaker 2>Things have improved so much. Having a continuous glucose monitor

0:16:07.760 --> 0:16:11.480
<v Speaker 2>is probably one of the best inventions. So now I

0:16:11.520 --> 0:16:14.840
<v Speaker 2>can glance at my blood sugars and I can then

0:16:15.040 --> 0:16:16.800
<v Speaker 2>pick up my insulin pump, which is right next to

0:16:16.840 --> 0:16:19.160
<v Speaker 2>that device, and just input the numbers.

0:16:19.840 --> 0:16:23.200
<v Speaker 3>And maybe more important than tech, Madison still has her

0:16:23.280 --> 0:16:24.440
<v Speaker 3>fearless determination.

0:16:24.920 --> 0:16:27.080
<v Speaker 2>I had a friend point out to me. She goes, Oh,

0:16:27.120 --> 0:16:28.880
<v Speaker 2>that's why you are the way you are, you know,

0:16:29.760 --> 0:16:32.200
<v Speaker 2>And I didn't even realize that. I didn't even associate

0:16:32.240 --> 0:16:33.520
<v Speaker 2>it back to my diabetes.

0:16:43.400 --> 0:16:46.480
<v Speaker 3>This is Targeting the Toughest diseases. A podcast from Bloomberg

0:16:46.520 --> 0:16:50.280
<v Speaker 3>Media Studios and Vertex Pharmaceuticals. If you like what you hear,

0:16:50.600 --> 0:16:54.240
<v Speaker 3>subscribe and leave us a review. I'm Jordan Gosbory. Thanks

0:16:54.240 --> 0:16:54.800
<v Speaker 3>for listening.