1 00:00:00,320 --> 00:00:03,840 Speaker 1: Since you're a subscriber to this Bloomberg podcast, we thought 2 00:00:03,840 --> 00:00:07,320 Speaker 1: you'd be interested in a six episode sponsored podcast called 3 00:00:07,760 --> 00:00:12,760 Speaker 1: Targeting the Toughest Diseases, produced by Vertex Pharmaceuticals and Bloomberg 4 00:00:12,800 --> 00:00:17,720 Speaker 1: Media Studios. It explores the innovative tools, methods, and unique 5 00:00:17,720 --> 00:00:22,479 Speaker 1: philosophy Vertex Pharmaceuticals is using to search for treatments for 6 00:00:22,520 --> 00:00:35,120 Speaker 1: some of humanity's most challenging diseases. Here's a recent episode. 7 00:00:35,600 --> 00:00:40,000 Speaker 2: I'm so goal oriented. I'm always just charging ahead, like 8 00:00:40,040 --> 00:00:42,160 Speaker 2: we're going to get it done by any means possible. 9 00:00:42,640 --> 00:00:46,200 Speaker 3: Focused, and fearless. That's the best way to describe Madison Carter. 10 00:00:46,520 --> 00:00:49,239 Speaker 3: Those qualities have helped Madison break a lot of important 11 00:00:49,280 --> 00:00:52,800 Speaker 3: stories as an award winning investigative reporter in Atlanta, Georgia. 12 00:00:53,040 --> 00:00:57,320 Speaker 3: She's investigated white nationalists, political corruption, and the parole system. 13 00:00:57,760 --> 00:00:59,880 Speaker 3: Those are stories that take a lot of guts to 14 00:01:00,480 --> 00:01:02,680 Speaker 3: and Madison has done it all without hesitation. 15 00:01:02,960 --> 00:01:05,919 Speaker 2: People will tell you I go for everything, I say 16 00:01:05,959 --> 00:01:08,240 Speaker 2: everything that needs to be said, like I have no fear, 17 00:01:08,319 --> 00:01:09,559 Speaker 2: like I don't have a lot to lose. 18 00:01:11,000 --> 00:01:13,199 Speaker 3: That fearlessness comes from a dark place. 19 00:01:14,080 --> 00:01:16,280 Speaker 2: It's because of my diabetes. I'm like I could die 20 00:01:16,280 --> 00:01:23,720 Speaker 2: at any second. Hi. 21 00:01:23,840 --> 00:01:27,120 Speaker 3: I'm Jordan Gospore, a member of the University of Southern 22 00:01:27,120 --> 00:01:30,720 Speaker 3: California's Center for Health Journalism. This is Targeting the Toughest 23 00:01:30,720 --> 00:01:35,080 Speaker 3: Diseases produced by Bloomberg Media Studios and Vertex Pharmaceuticals. In 24 00:01:35,160 --> 00:01:38,120 Speaker 3: this series, we look at some of humanity's most challenging 25 00:01:38,200 --> 00:01:42,040 Speaker 3: diseases and how Vertex, a Boston based biotech company, is 26 00:01:42,160 --> 00:01:45,800 Speaker 3: using innovative tools, methods, and a unique philosophy to search 27 00:01:45,840 --> 00:01:50,960 Speaker 3: for treatments and cures. Today, we're targeting type one diabetes, 28 00:01:51,280 --> 00:01:54,040 Speaker 3: a chronic disease that affects more than eight million people 29 00:01:54,080 --> 00:01:57,160 Speaker 3: around the world, a number that's expected to increase rapidly. 30 00:01:57,400 --> 00:02:00,960 Speaker 3: In the US alone, approximately one point four million people, 31 00:02:01,120 --> 00:02:03,640 Speaker 3: including nearly one hundred and seventy thousand under the age 32 00:02:03,640 --> 00:02:05,559 Speaker 3: of twenty, have type one diabetes. 33 00:02:12,040 --> 00:02:14,720 Speaker 2: I don't think I ever had the opportunity to be 34 00:02:14,760 --> 00:02:15,200 Speaker 2: a kid. 35 00:02:16,000 --> 00:02:19,079 Speaker 3: Since she was three years old, Madison's life has included 36 00:02:19,120 --> 00:02:23,120 Speaker 3: a highly controlled diet, constantly measuring her blood sugar levels, 37 00:02:23,120 --> 00:02:24,359 Speaker 3: and insolent shots. 38 00:02:24,720 --> 00:02:27,240 Speaker 2: And it's something that I have spent the past couple 39 00:02:27,320 --> 00:02:31,360 Speaker 2: years in therapy really working through because it's not fair 40 00:02:31,400 --> 00:02:33,560 Speaker 2: it's not just that I feel like it's not fair. 41 00:02:33,639 --> 00:02:34,040 Speaker 4: It is not. 42 00:02:39,120 --> 00:02:42,560 Speaker 3: When she was just a toddler, Madison started showing signs 43 00:02:42,600 --> 00:02:43,840 Speaker 3: that something was off. 44 00:02:44,200 --> 00:02:47,360 Speaker 2: I was getting really, really skinny, which is weird because 45 00:02:47,360 --> 00:02:49,519 Speaker 2: the people in my family are not very skinny. 46 00:02:49,919 --> 00:02:54,240 Speaker 3: She was thin, feeling thirsty all the time, and constantly tired. 47 00:02:54,639 --> 00:02:57,280 Speaker 2: I would be falling asleep in random places. My mom 48 00:02:57,639 --> 00:03:00,520 Speaker 2: found me asleep next to my oatmeal one morning, found 49 00:03:00,560 --> 00:03:03,400 Speaker 2: me asleep on the toilet one day. You know, I'm 50 00:03:03,440 --> 00:03:06,160 Speaker 2: a toddler. I'm three years old, so that was really strained, 51 00:03:06,240 --> 00:03:09,200 Speaker 2: especially when I was supposed to be running around. And 52 00:03:09,280 --> 00:03:11,320 Speaker 2: one night my mom was putting me to bed and 53 00:03:11,360 --> 00:03:13,840 Speaker 2: she said that she could count every single one of 54 00:03:13,880 --> 00:03:17,120 Speaker 2: my ribs, and in that moment, she picked me up, 55 00:03:17,160 --> 00:03:19,120 Speaker 2: put me in the back of the car, gave me 56 00:03:19,160 --> 00:03:21,560 Speaker 2: a sprite, which later on we would find out was 57 00:03:21,600 --> 00:03:24,600 Speaker 2: a very bad idea, and took me to Children's Hospital. 58 00:03:26,000 --> 00:03:28,800 Speaker 3: When they got there, the medical team found Madison's blood 59 00:03:28,800 --> 00:03:33,120 Speaker 3: sugar level was dangerously elevated, nearly two hundred percent higher 60 00:03:33,120 --> 00:03:33,919 Speaker 3: than it should have been. 61 00:03:35,040 --> 00:03:38,240 Speaker 2: They told my mom I should be dead. Had she 62 00:03:38,280 --> 00:03:40,120 Speaker 2: had put me to bed that night, I would have 63 00:03:40,240 --> 00:03:45,960 Speaker 2: been dead, and that's how I was diagnosed. I think 64 00:03:46,000 --> 00:03:48,400 Speaker 2: my parents had about a year of giving me injections 65 00:03:48,480 --> 00:03:50,400 Speaker 2: until I told them to never touch me with the 66 00:03:50,440 --> 00:03:51,120 Speaker 2: needle again. 67 00:03:51,440 --> 00:03:54,320 Speaker 3: By the time she was five, Madison was doing all 68 00:03:54,320 --> 00:03:55,600 Speaker 3: her injections herself. 69 00:03:56,280 --> 00:03:59,360 Speaker 2: It was harder for me to see my mom inflict 70 00:03:59,440 --> 00:04:01,600 Speaker 2: pain me than it was for me to just do 71 00:04:01,760 --> 00:04:05,080 Speaker 2: it myself. Like seeing her face that upset me. It 72 00:04:05,120 --> 00:04:07,480 Speaker 2: wasn't even that it hurt, because it hurts sometimes when 73 00:04:07,520 --> 00:04:09,600 Speaker 2: I would do it myself. But I just didn't want 74 00:04:09,600 --> 00:04:12,040 Speaker 2: her to have that responsibility of feeling like she was 75 00:04:12,120 --> 00:04:12,640 Speaker 2: hurting me. 76 00:04:17,640 --> 00:04:21,119 Speaker 3: Our pancreas produces insulin, a special hormone that the body 77 00:04:21,160 --> 00:04:24,440 Speaker 3: needs to process glucose, a key source of energy. For 78 00:04:24,520 --> 00:04:28,080 Speaker 3: people who have type one diabetes, those insulin producing cells 79 00:04:28,080 --> 00:04:31,160 Speaker 3: in the pancreas are destroyed. That means the glucose stays 80 00:04:31,240 --> 00:04:34,640 Speaker 3: in the blood, resulting in high blood sugar levels, also 81 00:04:34,720 --> 00:04:40,279 Speaker 3: known as hyperglycemia. The symptoms include increased thirst, hunger, frequent urination, 82 00:04:40,640 --> 00:04:43,640 Speaker 3: and weight loss. If left untreated, this can lead to 83 00:04:43,720 --> 00:04:47,680 Speaker 3: excessively high glucose levels and eventually could even be fatal. 84 00:04:48,279 --> 00:04:51,600 Speaker 3: People with type one diabetes rely on giving themselves insulin 85 00:04:51,720 --> 00:04:55,240 Speaker 3: every single day to survive and have to be super vigilant. 86 00:04:55,560 --> 00:04:58,680 Speaker 3: Not enough insulin can lead to high blood glucose levels 87 00:04:58,920 --> 00:05:03,600 Speaker 3: and over time complications like kidney disease, vision loss, nerve damage, 88 00:05:03,760 --> 00:05:07,440 Speaker 3: heart attacks, and strokes. Too much insulin can result in 89 00:05:07,600 --> 00:05:11,280 Speaker 3: low blood sugar. If left unaddressed, blood glucose levels can 90 00:05:11,320 --> 00:05:16,000 Speaker 3: fall dangerously low and can lead to severe hypoglycemia, resulting 91 00:05:16,000 --> 00:05:18,799 Speaker 3: in seizures, loss of consciousness, or even death. 92 00:05:26,800 --> 00:05:30,920 Speaker 2: I remember it was President's Day weekend. She was playing volleyball, 93 00:05:30,960 --> 00:05:31,880 Speaker 2: so she had this tournament. 94 00:05:32,320 --> 00:05:35,480 Speaker 3: Madison's younger sister, Cameron, also had type one diabetes. 95 00:05:35,839 --> 00:05:39,520 Speaker 2: She was fourteen and I got pulled out of class. 96 00:05:39,720 --> 00:05:41,920 Speaker 2: They said, hey, you know we're going to take you 97 00:05:41,960 --> 00:05:43,640 Speaker 2: to the hospital. Your sister had a seizure. 98 00:05:44,360 --> 00:05:45,840 Speaker 3: Madison rushed to the hospital. 99 00:05:46,520 --> 00:05:48,760 Speaker 2: My parents met us at the door and they said, 100 00:05:48,880 --> 00:05:51,880 Speaker 2: you know, she didn't make it. She was dead when 101 00:05:51,920 --> 00:05:53,160 Speaker 2: they took her in the ambulance. 102 00:05:56,520 --> 00:05:59,839 Speaker 3: Her sister's sudden death from diabetes devastated Madison. 103 00:06:00,240 --> 00:06:02,840 Speaker 2: I would call her the good diabetic, like she really 104 00:06:02,880 --> 00:06:05,320 Speaker 2: did everything she needed to do, and I was kind 105 00:06:05,360 --> 00:06:07,840 Speaker 2: of in that phase of doing just enough to stay alive. 106 00:06:08,560 --> 00:06:12,000 Speaker 2: And she died and that upset me, and I was like, well, 107 00:06:12,080 --> 00:06:15,000 Speaker 2: what is the point of doing everything you're supposed to 108 00:06:15,000 --> 00:06:19,279 Speaker 2: do like my sister Cameron did, if it's still not enough. 109 00:06:19,760 --> 00:06:23,480 Speaker 3: Madison lost all motivation to manage her own health for. 110 00:06:23,520 --> 00:06:26,200 Speaker 2: A year after she died. I told people that I 111 00:06:26,240 --> 00:06:29,560 Speaker 2: took a year off of being diabetic. The responsibility is 112 00:06:29,600 --> 00:06:31,400 Speaker 2: the checking your blood sugar and the making sure you're 113 00:06:31,440 --> 00:06:34,560 Speaker 2: taking your injections or changing your pump. I refused to 114 00:06:34,640 --> 00:06:38,080 Speaker 2: do and my mom stepped in and did it for me. 115 00:06:40,640 --> 00:06:44,279 Speaker 3: After a year, Madison resumed doing her own care. It's 116 00:06:44,279 --> 00:06:46,440 Speaker 3: a daily grind, but it keeps her alive. 117 00:06:49,720 --> 00:06:52,480 Speaker 5: It's a very, very difficult disease to manage. 118 00:06:52,760 --> 00:06:56,320 Speaker 3: The constant managing of type one diabetes is something doctor 119 00:06:56,320 --> 00:06:58,320 Speaker 3: Aaron Kowalsky knows intimately. 120 00:06:58,960 --> 00:07:02,400 Speaker 5: When my brother was diagnosed in the late seventies, you 121 00:07:02,440 --> 00:07:05,559 Speaker 5: were pretty much told that you had about twenty years 122 00:07:05,600 --> 00:07:09,720 Speaker 5: before complications would form. Your life would be shortened pretty significantly. 123 00:07:10,160 --> 00:07:13,240 Speaker 5: You had a very high likelihood of going blind. You 124 00:07:13,480 --> 00:07:16,000 Speaker 5: had the dosing of insulin issues that could cost of 125 00:07:16,000 --> 00:07:18,960 Speaker 5: your hypoglycemia and potentially death on a daily basis. 126 00:07:19,200 --> 00:07:22,520 Speaker 3: A few years after his brother was diagnosed, Kowalski discovered 127 00:07:22,560 --> 00:07:25,720 Speaker 3: he had type one diabetes as well. Today he's the 128 00:07:25,760 --> 00:07:29,640 Speaker 3: CEO of JDRF, the world's largest nonprofit funder of type 129 00:07:29,640 --> 00:07:30,880 Speaker 3: one diabetes research. 130 00:07:34,280 --> 00:07:37,640 Speaker 5: We are just celebrating the one hundred year anniversary of 131 00:07:37,680 --> 00:07:42,120 Speaker 5: the discovery of insulin, which won multiple Nobel Prizes and 132 00:07:42,680 --> 00:07:46,440 Speaker 5: saved millions of lives. Before the discovery of insulin in 133 00:07:46,520 --> 00:07:49,680 Speaker 5: nineteen twenty one, every single Type one person died. 134 00:07:50,280 --> 00:07:54,120 Speaker 3: That's every single person for centuries. 135 00:07:56,720 --> 00:08:02,600 Speaker 5: The disease actually was described in ancient Egyptian and Greek writings, 136 00:08:03,480 --> 00:08:08,400 Speaker 5: and this observation that people made was the fact that 137 00:08:08,640 --> 00:08:11,600 Speaker 5: people with this syndrome wasted away. 138 00:08:12,160 --> 00:08:16,160 Speaker 3: The term diabetes melitis was coined because it roughly translates 139 00:08:16,200 --> 00:08:17,920 Speaker 3: to sweet siphon. 140 00:08:18,120 --> 00:08:21,160 Speaker 5: The sugar which just passed through people, the food which 141 00:08:21,160 --> 00:08:24,760 Speaker 5: just passed through them, and the sweetness was observed when 142 00:08:24,800 --> 00:08:27,400 Speaker 5: ants would be attracted to the urine of these people. 143 00:08:32,800 --> 00:08:36,440 Speaker 3: Modern times have brought us modern discoveries. Apart from the 144 00:08:36,480 --> 00:08:39,600 Speaker 3: breakthrough of insulin, there was a landmark study in nineteen 145 00:08:39,640 --> 00:08:43,440 Speaker 3: eighty three called the DCCT trial, which stands for the 146 00:08:43,480 --> 00:08:47,679 Speaker 3: Diabetes Control and Complications Trial it showed the connection between 147 00:08:47,800 --> 00:08:51,400 Speaker 3: high blood sugar and complications. It also showed that with 148 00:08:51,440 --> 00:08:55,000 Speaker 3: more intensive therapy, people with type one diabetes could prevent 149 00:08:55,160 --> 00:08:59,200 Speaker 3: long term eye, kidney, and nerve complications. And more recently, 150 00:08:59,280 --> 00:09:03,079 Speaker 3: there's been huge improvements in glucose monitoring. This includes smartphone 151 00:09:03,120 --> 00:09:07,000 Speaker 3: apps with sensors which can monitor blood sugar levels continually. 152 00:09:07,440 --> 00:09:10,200 Speaker 5: Not surprisingly, if you have more information, you do better. 153 00:09:10,440 --> 00:09:12,880 Speaker 5: I certainly think that's been one of the biggest changes 154 00:09:12,920 --> 00:09:14,560 Speaker 5: in my life, in my brother's life. 155 00:09:19,000 --> 00:09:22,360 Speaker 3: Despite all those improvements in managing type one diabetes, there's 156 00:09:22,360 --> 00:09:25,240 Speaker 3: still no cure and people with the disease are still 157 00:09:25,280 --> 00:09:29,720 Speaker 3: at risk of developing really severe, potentially fatal complications. 158 00:09:30,160 --> 00:09:35,320 Speaker 6: Type one diabetes has such a significant burden on individuals, 159 00:09:35,320 --> 00:09:38,280 Speaker 6: on families, on communities, and it is a twenty four 160 00:09:38,320 --> 00:09:40,000 Speaker 6: to seven relentless job. 161 00:09:40,720 --> 00:09:44,400 Speaker 3: That's doctor Felicia Pelayuka. She's the vice president and Disease 162 00:09:44,480 --> 00:09:47,800 Speaker 3: Area Executive for type one diabetes at Vertex. They are 163 00:09:47,840 --> 00:09:51,520 Speaker 3: researching type one diabetes at the cellular level, something she 164 00:09:51,600 --> 00:09:53,600 Speaker 3: first got interested in when she was a student. 165 00:09:54,600 --> 00:09:57,040 Speaker 6: It was really a lecture that I saw when I 166 00:09:57,120 --> 00:10:01,199 Speaker 6: was a PhD student by Professor Doug Maltain that turn 167 00:10:01,320 --> 00:10:04,360 Speaker 6: that paradigm on its head, that not only could we 168 00:10:04,400 --> 00:10:07,320 Speaker 6: think about cells as being a cause of disease, but 169 00:10:07,400 --> 00:10:11,040 Speaker 6: think about cells as being a solution to diseases. 170 00:10:11,440 --> 00:10:15,240 Speaker 3: That's what Vertex is investigating cell therapy as a potential 171 00:10:15,280 --> 00:10:16,880 Speaker 3: treatment for type one diabetes. 172 00:10:17,440 --> 00:10:22,319 Speaker 6: Vertex has a really unique research and development strategy that 173 00:10:22,880 --> 00:10:27,400 Speaker 6: focuses first and foremost on diseases where we understand the 174 00:10:27,440 --> 00:10:31,280 Speaker 6: causal biology really well, so that we can rationally design 175 00:10:31,640 --> 00:10:35,840 Speaker 6: therapies that could have a major transformative impact on patients 176 00:10:35,840 --> 00:10:38,000 Speaker 6: if we're successful in developing them. 177 00:10:38,400 --> 00:10:42,040 Speaker 3: Type one diabetes definitely fits that description clearly. 178 00:10:42,080 --> 00:10:45,600 Speaker 6: It's a disease with enormous unmet medical need. It's a 179 00:10:45,640 --> 00:10:49,800 Speaker 6: disease where we understand that these missing insulin producing islet 180 00:10:49,880 --> 00:10:52,600 Speaker 6: cells are at the heart of the disease. 181 00:10:52,800 --> 00:10:55,160 Speaker 3: And this knowledge is what drives vertexs forward. 182 00:10:55,880 --> 00:10:59,800 Speaker 6: Science has advanced to a place where even a decade 183 00:10:59,800 --> 00:11:03,000 Speaker 6: ago go, it was unthinkable, it was science fiction to 184 00:11:03,120 --> 00:11:07,679 Speaker 6: think that you could make replacement isletself in the laboratory, 185 00:11:07,720 --> 00:11:10,720 Speaker 6: and so to be at the moment now it really 186 00:11:10,800 --> 00:11:14,160 Speaker 6: feels like an inflection point, and we hope it really 187 00:11:14,200 --> 00:11:16,920 Speaker 6: will be in the history of the treatment of type 188 00:11:16,960 --> 00:11:17,679 Speaker 6: one diabetes. 189 00:11:21,280 --> 00:11:24,080 Speaker 3: The professor who first inspired doctor Palayuka to think of 190 00:11:24,120 --> 00:11:27,559 Speaker 3: cells as living problem solvers was doctor Doug Melton, one 191 00:11:27,600 --> 00:11:31,160 Speaker 3: of the world's leading stem cell researchers. A former Harvard professor, 192 00:11:31,440 --> 00:11:33,600 Speaker 3: He's been in this area of research for more than 193 00:11:33,640 --> 00:11:36,559 Speaker 3: twenty years, and this year he joined Vertex as a 194 00:11:36,600 --> 00:11:40,080 Speaker 3: Distinguished Fellow. But working on an investigational treatment for type 195 00:11:40,080 --> 00:11:42,559 Speaker 3: one diabetes wasn't always on his radar. 196 00:11:43,440 --> 00:11:47,120 Speaker 4: Well. I didn't think much about diabetes until my six 197 00:11:47,160 --> 00:11:51,240 Speaker 4: month old son contracted the disease. From a parental point 198 00:11:51,320 --> 00:11:54,559 Speaker 4: of view, you have to manage that little baby's blood 199 00:11:54,559 --> 00:11:58,439 Speaker 4: sugars by picking their fingers or their toes and squeezing 200 00:11:58,480 --> 00:12:01,000 Speaker 4: blood out and measuring how much sugar there is and 201 00:12:01,080 --> 00:12:04,360 Speaker 4: then inject them with insulin. You can imagine how hard 202 00:12:04,400 --> 00:12:06,480 Speaker 4: it is. And a six month old that doesn't talk. 203 00:12:06,920 --> 00:12:10,400 Speaker 3: His son, Sam, was the youngest person at Children's Hospital 204 00:12:10,400 --> 00:12:13,200 Speaker 3: in Boston to be diagnosed with type one diabetes. 205 00:12:13,800 --> 00:12:16,520 Speaker 4: My wife had to deal with the fact that she 206 00:12:16,679 --> 00:12:20,040 Speaker 4: was also breastfeeding Sam at the time. When you're eating 207 00:12:20,240 --> 00:12:22,760 Speaker 4: food like a candy bar or an apple or something, 208 00:12:22,800 --> 00:12:25,200 Speaker 4: you know how much sugar you're taking in, but you 209 00:12:25,240 --> 00:12:28,160 Speaker 4: can't tell how much sugar you take in or energy 210 00:12:28,240 --> 00:12:30,840 Speaker 4: from breast milk because you don't know what the volume is. 211 00:12:31,480 --> 00:12:34,760 Speaker 4: So she's up all night testing his blood sugars and 212 00:12:34,800 --> 00:12:38,840 Speaker 4: injecting insulin. And she looked at me more or less 213 00:12:38,840 --> 00:12:42,240 Speaker 4: and said, look, you're a scientist, go do something about this. 214 00:12:42,600 --> 00:12:47,360 Speaker 3: And so he set out to do just that. It 215 00:12:47,400 --> 00:12:50,240 Speaker 3: was understood that people with type one were missing insulin 216 00:12:50,280 --> 00:12:54,000 Speaker 3: producing cells, also known as beta cells. Doctor Melton figured 217 00:12:54,120 --> 00:12:56,760 Speaker 3: if the beta cells were missing, they needed to make 218 00:12:56,800 --> 00:12:57,320 Speaker 3: new ones. 219 00:12:57,960 --> 00:13:00,440 Speaker 4: There was this nice commercial about a man he would 220 00:13:00,440 --> 00:13:02,520 Speaker 4: say time to make the donuts, and so I used 221 00:13:02,520 --> 00:13:04,960 Speaker 4: to go to lab and say time to make beta cells. 222 00:13:04,960 --> 00:13:05,760 Speaker 4: That was our job. 223 00:13:05,960 --> 00:13:08,959 Speaker 3: I love that. What are the differences between type one 224 00:13:09,000 --> 00:13:10,160 Speaker 3: and type two diabetes. 225 00:13:10,480 --> 00:13:14,439 Speaker 4: Well, type one diabetes is caused by the problem that 226 00:13:14,559 --> 00:13:18,479 Speaker 4: one's immune system makes a mistake. Instead of just attacking 227 00:13:18,800 --> 00:13:22,320 Speaker 4: foreign entities like a virus, it decides, for reasons we 228 00:13:22,360 --> 00:13:26,120 Speaker 4: don't understand, to attack the patient's own beta cells and 229 00:13:26,240 --> 00:13:29,400 Speaker 4: destroys those cells. That is not the cause of type 230 00:13:29,440 --> 00:13:33,240 Speaker 4: two diabetes. And type two diabetes the patient still makes insulin, 231 00:13:33,400 --> 00:13:36,520 Speaker 4: but their body has a greater and greater demand for insulin. 232 00:13:36,760 --> 00:13:41,559 Speaker 4: So it's called insulin resistance, meaning that the patients muscle 233 00:13:41,720 --> 00:13:45,120 Speaker 4: and fat cells require more and more insulin all the time. 234 00:13:45,280 --> 00:13:48,720 Speaker 4: Type two diabetes can be treated in many cases by 235 00:13:48,880 --> 00:13:52,280 Speaker 4: changes in diet and exercise. That is not true for 236 00:13:52,400 --> 00:13:56,360 Speaker 4: type one, so no behavioral change will prevent or treat 237 00:13:56,520 --> 00:13:57,640 Speaker 4: type one diabetes. 238 00:13:58,040 --> 00:13:59,120 Speaker 3: What was that aha moment? 239 00:13:59,480 --> 00:14:03,520 Speaker 4: The real turning point was about in twenty thirteen twenty 240 00:14:03,600 --> 00:14:07,640 Speaker 4: fourteen where we figured out the right factors or molecules 241 00:14:07,679 --> 00:14:10,440 Speaker 4: to give to the cells that made them respond to glucose. 242 00:14:10,679 --> 00:14:13,760 Speaker 4: Because the beta cells job is to read or measure 243 00:14:13,800 --> 00:14:16,440 Speaker 4: the amount of sugar in the blood and then squirt 244 00:14:16,480 --> 00:14:19,480 Speaker 4: out just the right amount of insulin. So the moment 245 00:14:19,680 --> 00:14:23,120 Speaker 4: where we had a test and when we added glucose, 246 00:14:23,160 --> 00:14:25,680 Speaker 4: the cells squirted out insulin and then we knew we 247 00:14:25,680 --> 00:14:26,600 Speaker 4: were on the right track. 248 00:14:30,120 --> 00:14:32,680 Speaker 3: As part of his work at Vertex, doctor Melton is 249 00:14:32,720 --> 00:14:36,520 Speaker 3: helping develop a potential treatment that addresses the root cause 250 00:14:36,640 --> 00:14:40,600 Speaker 3: of type one diabetes, the absence of those insulin producing 251 00:14:40,640 --> 00:14:43,760 Speaker 3: cells in the pancreas. The company is investigating how to 252 00:14:43,800 --> 00:14:47,920 Speaker 3: replace those destroyed cells with insulin producing cells and making 253 00:14:47,960 --> 00:14:51,120 Speaker 3: sure they can get into patients in a way where 254 00:14:51,160 --> 00:14:54,760 Speaker 3: they can survive and function for a long time, and 255 00:14:54,880 --> 00:14:58,360 Speaker 3: progress so far gives doctor Melton competence and their path forward. 256 00:14:58,840 --> 00:15:01,120 Speaker 4: Let's put it this way. If thirty years ago you 257 00:15:01,160 --> 00:15:04,040 Speaker 4: had told someone you would take a human stem cell 258 00:15:04,160 --> 00:15:07,520 Speaker 4: and turn it into functional insulin producing cells, people would 259 00:15:07,560 --> 00:15:08,880 Speaker 4: have said, yeah, good luck with that. 260 00:15:11,640 --> 00:15:14,440 Speaker 3: One last thing that doctor Milton told me something that 261 00:15:14,480 --> 00:15:16,720 Speaker 3: made me smile the rest of the day. He says 262 00:15:16,760 --> 00:15:20,160 Speaker 3: that everywhere he goes he always carries a little vial 263 00:15:20,240 --> 00:15:21,000 Speaker 3: in his pocket. 264 00:15:21,240 --> 00:15:24,480 Speaker 4: It has in it cells that have been turned into 265 00:15:24,560 --> 00:15:27,440 Speaker 4: insulin producing cells. Sometimes I take it out and like 266 00:15:27,480 --> 00:15:29,160 Speaker 4: a snow globe and just turn it up and down 267 00:15:29,160 --> 00:15:31,240 Speaker 4: and look at it, because it reminds me that this 268 00:15:31,440 --> 00:15:33,840 Speaker 4: is possible. We now know the problem. We just have 269 00:15:33,920 --> 00:15:34,360 Speaker 4: to do it. 270 00:15:40,240 --> 00:15:43,160 Speaker 3: Madison Carter is hoping for a better treatment for type 271 00:15:43,200 --> 00:15:47,200 Speaker 3: one diabetes because she doesn't want another generation of young 272 00:15:47,240 --> 00:15:49,040 Speaker 3: people to feel how she did. 273 00:15:49,200 --> 00:15:50,680 Speaker 2: I was like, I don't want to take the shot. 274 00:15:50,960 --> 00:15:53,960 Speaker 2: I don't want to do this again tomorrow. I don't 275 00:15:54,000 --> 00:15:56,640 Speaker 2: want to not that I wanted to die, but I 276 00:15:56,720 --> 00:16:01,400 Speaker 2: just did not want to work this hard to be alive, to. 277 00:16:01,320 --> 00:16:04,000 Speaker 3: Ease that burden. She's now looking to technology. 278 00:16:04,360 --> 00:16:07,440 Speaker 2: Things have improved so much. Having a continuous glucose monitor 279 00:16:07,760 --> 00:16:11,480 Speaker 2: is probably one of the best inventions. So now I 280 00:16:11,520 --> 00:16:14,840 Speaker 2: can glance at my blood sugars and I can then 281 00:16:15,040 --> 00:16:16,800 Speaker 2: pick up my insulin pump, which is right next to 282 00:16:16,840 --> 00:16:19,160 Speaker 2: that device, and just input the numbers. 283 00:16:19,840 --> 00:16:23,200 Speaker 3: And maybe more important than tech, Madison still has her 284 00:16:23,280 --> 00:16:24,440 Speaker 3: fearless determination. 285 00:16:24,920 --> 00:16:27,080 Speaker 2: I had a friend point out to me. She goes, Oh, 286 00:16:27,120 --> 00:16:28,880 Speaker 2: that's why you are the way you are, you know, 287 00:16:29,760 --> 00:16:32,200 Speaker 2: And I didn't even realize that. I didn't even associate 288 00:16:32,240 --> 00:16:33,520 Speaker 2: it back to my diabetes. 289 00:16:43,400 --> 00:16:46,480 Speaker 3: This is Targeting the Toughest diseases. A podcast from Bloomberg 290 00:16:46,520 --> 00:16:50,280 Speaker 3: Media Studios and Vertex Pharmaceuticals. If you like what you hear, 291 00:16:50,600 --> 00:16:54,240 Speaker 3: subscribe and leave us a review. I'm Jordan Gosbory. Thanks 292 00:16:54,240 --> 00:16:54,800 Speaker 3: for listening.