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