WEBVTT - Targeting Sickle Cell Disease (Sponsored Content) 0:00:00.360 --> 0:00:03.880 Since you're a subscriber to this Bloomberg podcast, we thought 0:00:03.880 --> 0:00:07.360 you'd be interested in a six episode sponsored podcast called 0:00:07.800 --> 0:00:12.760 Targeting the Toughest Diseases, produced by Vertex Pharmaceuticals and Bloomberg 0:00:12.840 --> 0:00:17.760 Media Studios. It explores the innovative tools, methods, and unique 0:00:17.760 --> 0:00:22.520 philosophy Vertex Pharmaceuticals is using to search for treatments for 0:00:22.560 --> 0:00:27.400 some of humanity's most challenging diseases. Here's a recent episode, 0:00:29.800 --> 0:00:30.360 and it's. 0:00:30.240 --> 0:00:33.879 Like, if someone grabs your body and twists in the 0:00:33.880 --> 0:00:40.080 opposite direction with each hand, it's like you can feel 0:00:40.120 --> 0:00:43.640 the pressure in your bones and you can just feel 0:00:43.760 --> 0:00:46.159 every part of your body is hurting. 0:00:47.800 --> 0:00:51.320 Twenty years ago, during her sophomore year in college, Terry 0:00:51.360 --> 0:00:54.600 Booker was hit with the worst pain of her life. 0:00:56.640 --> 0:01:01.040 My legs were burning in then it just went up 0:01:01.080 --> 0:01:04.480 throughout my whole entire body to the point where I 0:01:04.600 --> 0:01:09.560 was unconscious and I was put on life support. I 0:01:09.600 --> 0:01:12.560 wasn't able to breathe on my own. They told my 0:01:12.680 --> 0:01:17.040 mom I had kidney failure, I had lung failure. 0:01:17.600 --> 0:01:20.959 There was no clear path to treatment for her, just uncertainty. 0:01:21.760 --> 0:01:24.600 They said, you just prayed because we don't know what's 0:01:24.600 --> 0:01:25.559 going to happen to her. 0:01:29.120 --> 0:01:33.320 What happened was she got better for a while, but 0:01:33.360 --> 0:01:35.319 then the pain returned and she was back in the 0:01:35.360 --> 0:01:39.399 emergency room. Her recurring visits to the er seeking relief 0:01:39.400 --> 0:01:43.080 from the pain revealed a second problem, not a medical one, 0:01:43.560 --> 0:01:48.040 a societal one. Terry is black, and as an African 0:01:48.040 --> 0:01:51.160 American woman seeking pay meds, she was presumed to be 0:01:51.200 --> 0:01:54.120 a drug addict and her medical issues were ignored. 0:01:55.120 --> 0:01:58.920 It is infuriating because when you go into the er, 0:01:59.000 --> 0:02:02.840 you're expecting to receive help. You're expecting to be treated 0:02:02.960 --> 0:02:06.720 as a patient the need of something, not treated as 0:02:06.760 --> 0:02:10.160 someone who is wanting to get their next high. 0:02:14.040 --> 0:02:18.360 Hi. I'm Jordan Gospore. I'm a member of the University 0:02:18.400 --> 0:02:23.079 of Southern California's Center for Health Journalism. This is Targeting 0:02:23.080 --> 0:02:27.360 the Toughest Diseases a podcast produced by Bloomberg Media Studios 0:02:27.520 --> 0:02:31.600 and Vertex Pharmaceuticals. In this series, we look at some 0:02:31.639 --> 0:02:36.040 of humanity's most challenging diseases and how Vertex, a Boston 0:02:36.080 --> 0:02:40.400 based biotech company, is using innovative tools, methods, and a 0:02:40.480 --> 0:02:46.160 unique philosophy to search for treatments and cures. Today, we're 0:02:46.200 --> 0:02:49.240 looking at. Sickle cell disease a blood disorder which can 0:02:49.280 --> 0:02:53.600 cause organ failure, stroke, and even death. It's a disease 0:02:53.600 --> 0:02:57.720 that affects roughly one hundred thousand Americans, including Terry Booker. 0:03:04.000 --> 0:03:07.040 Sickle cell disease is not something you catch, it's a 0:03:07.040 --> 0:03:10.919 genetic condition you're born with. Often babies are diagnosed before 0:03:10.919 --> 0:03:14.840 they're even born. Terry was an exception. She didn't know 0:03:14.919 --> 0:03:16.280 she had it until she was eleven. 0:03:18.360 --> 0:03:22.400 They pricked my finger and I remember them putting it 0:03:22.520 --> 0:03:25.760 under a microscope, and when they headed under a microscope, 0:03:26.280 --> 0:03:29.160 all these people were rushing over and I remember this 0:03:29.360 --> 0:03:33.960 so vividly, and they were like, oh, come here, come. Look. 0:03:34.560 --> 0:03:36.680 Terry was in fifth grade and her mom had taken 0:03:36.760 --> 0:03:39.280 her to the doctors because she had been complaining of pain. 0:03:39.920 --> 0:03:42.560 And I'm like okay, and they say you see all 0:03:42.600 --> 0:03:47.360 these little funny shaped cells and I'm like yeah. They're like, yeah, 0:03:47.560 --> 0:03:51.080 that's sickle cell and I said okay, and I'm like, 0:03:51.200 --> 0:03:52.160 well what does that mean. 0:03:56.840 --> 0:04:00.360 Sickle cell disease, or sickle cell anemia, as it's often called, 0:04:00.800 --> 0:04:04.600 affects red blood cells. Those are the cells that carry 0:04:04.600 --> 0:04:08.800 oxygen to all the tissues in our bodies. Normally, our 0:04:08.840 --> 0:04:11.400 red blood cells are soft and shaped like a doughnut, 0:04:11.800 --> 0:04:14.920 so they can squeeze through even the smallest of blood vessels. 0:04:15.960 --> 0:04:19.919 But sickle cell disease changes them. Specifically, it causes the 0:04:19.920 --> 0:04:24.160 hemoglobin proteins inside the red blood cells to change their structure. 0:04:24.920 --> 0:04:28.359 Instead of being donut shaped, they become curved like a 0:04:28.400 --> 0:04:31.960 cresset moon, or, as the name implies, like a sickle. 0:04:33.560 --> 0:04:37.360 These odd shaped cells are also hard and sticky, which 0:04:37.400 --> 0:04:40.880 means sometimes they can't flow smoothly through blood vessels, and 0:04:40.920 --> 0:04:43.919 when that happens, they start piling up like cars on 0:04:43.960 --> 0:04:47.640 a busy highway, making it really hard, if not impossible, 0:04:47.920 --> 0:04:51.040 for oxygen to reach where it needs to go. It's 0:04:51.040 --> 0:04:54.320 that lack of oxygen to tissues that causes the stabbing pain. 0:04:58.120 --> 0:05:00.720 That's the medical side of the disease, but as Terry 0:05:00.760 --> 0:05:03.760 mentioned earlier, there's also a societal component. 0:05:05.160 --> 0:05:08.400 To address circle cell disease, we also have to address 0:05:08.480 --> 0:05:13.680 the fundamental issues that relate to systemic racism and how 0:05:13.720 --> 0:05:15.480 it impacts on assets to care. 0:05:16.600 --> 0:05:19.960 That's doctor Isaac O. Dame. He studies the ways racism 0:05:20.040 --> 0:05:23.000 and medical care intersect when it comes to sickle cell disease. 0:05:23.960 --> 0:05:26.800 He's the medical director of the Global Sickle Cell Disease 0:05:26.839 --> 0:05:34.120 Network at the Center for Global Child Health. Doctor Odame 0:05:34.240 --> 0:05:35.320 grew up in West Africa. 0:05:35.800 --> 0:05:39.040 Psyco cell disease was something that I was very familiar with. 0:05:39.760 --> 0:05:42.720 Not only did I have this some family members who 0:05:43.120 --> 0:05:47.640 had children with psycosol disease, but some classmates and schoolmates 0:05:47.680 --> 0:05:51.600 who also lived with the condition. So I was exposed 0:05:51.720 --> 0:05:54.320 very early to the ravages of this disease. 0:05:55.080 --> 0:05:58.000 The first documented case of psycho cell anemia in America 0:05:58.200 --> 0:06:01.800 was in nineteen ten, just over a century ago, but 0:06:01.920 --> 0:06:04.520 its history dates back thousands of years. 0:06:04.920 --> 0:06:08.599 Sucer cell disease has been known for centuries in parts 0:06:08.600 --> 0:06:13.480 of Africa, and if you go into the oral tradition, 0:06:14.040 --> 0:06:18.960 they did have descriptions of the disease which typified the 0:06:19.279 --> 0:06:23.799 chronic and intercurrent as creating pain associative of the disease. 0:06:24.560 --> 0:06:28.200 Oddly enough, the mutation that changes the cell shapes was 0:06:28.240 --> 0:06:31.680 once an evolutionary advantage protecting against malaria. 0:06:32.400 --> 0:06:37.919 It predominantly affects people of African descent because malaria is 0:06:38.120 --> 0:06:42.760 very endemic in Africa. It occurs in people of Indian descent, 0:06:43.320 --> 0:06:48.040 Mediterranean descent, and parts of the Middle East. Arabian Peninsula, 0:06:48.800 --> 0:06:51.520 and when you look at the common link between all 0:06:51.560 --> 0:06:57.200 these areas is malaria anddemicity. So it was a survival 0:06:57.240 --> 0:07:01.560 advantage it provided against malaria, and the percentages of people 0:07:01.560 --> 0:07:04.800 with traits in the population will rise over time. 0:07:05.720 --> 0:07:09.800 Having sickle cell trait is actually quite common in African populations. 0:07:10.360 --> 0:07:13.040 It evolved over time because it prevents the development and 0:07:13.080 --> 0:07:17.920 spreading of malaria. That historical context explains why the majority 0:07:17.920 --> 0:07:21.560 of sickle cell patients in the United States are African Americans. 0:07:22.200 --> 0:07:24.960 That means sickle cell care provides a clear example of 0:07:25.000 --> 0:07:28.680 the racial inequities and disparities in the medical system. That 0:07:28.720 --> 0:07:32.640 includes access to care and quality of care, both of 0:07:32.640 --> 0:07:36.800 which are lower for African Americans. Doctor Odami is hopeful 0:07:36.800 --> 0:07:39.600 that sickle cell treatments in development will not just treat 0:07:39.600 --> 0:07:42.880 the disease, but will address those other complex issues as well. 0:07:44.120 --> 0:07:47.800 I think more and more the patients are becoming more empowered, 0:07:48.760 --> 0:07:52.560 and I think they call themselves warriors, which is the 0:07:52.760 --> 0:07:55.760 appropriate term. They're not only dealing with the ravages of 0:07:55.800 --> 0:07:59.400 the disease, but they're also having to fight a system 0:07:59.680 --> 0:08:05.520 that doesn't recognize their needs appropriately, and so the warriors 0:08:05.600 --> 0:08:09.360 are beginning to speak out aloud. The system is beginning 0:08:09.400 --> 0:08:10.040 to respond. 0:08:11.640 --> 0:08:14.320 Vertex Pharmaceuticals is a leader in that response. 0:08:15.000 --> 0:08:17.720 For it to actually help people's lives, it has to 0:08:17.760 --> 0:08:19.920 work for them and it has to work for society. 0:08:20.440 --> 0:08:24.240 That's doctor David Altschuler, the chief scientific officer at Vertex. 0:08:24.880 --> 0:08:27.800 They have a unique approach to choosing the diseases they target. 0:08:28.800 --> 0:08:33.520 Vertex has decided to focus on a set of diseases 0:08:33.600 --> 0:08:36.360 where we see great unmet need, where we see the 0:08:36.559 --> 0:08:39.920 human biology is clear, and we feel that we have 0:08:40.040 --> 0:08:43.240 the technology, the insights, the wherewithal to make a difference. 0:08:43.600 --> 0:08:46.880 When it comes to Vertex's sickle cell program, Doctor William 0:08:46.920 --> 0:08:48.880 Hobbs is in charge of clinical development. 0:08:49.840 --> 0:08:53.800 You know, I started in medicine being interested in hematology 0:08:53.840 --> 0:08:58.120 and sickle cell disease in particular, mainly from a research standpoint, 0:08:58.520 --> 0:09:01.559 and that was because sickle cell disease has long been 0:09:01.640 --> 0:09:06.280 considered a disease where the underlying cause of disease could 0:09:06.360 --> 0:09:11.960 be targeted, if innovative approaches could be identified, and so 0:09:12.040 --> 0:09:14.720 I really came at it from this scientific point of view, 0:09:14.920 --> 0:09:18.079 and then along the way I started to meet sickle 0:09:18.120 --> 0:09:22.400 cell disease patients and their families coordinate their care, and 0:09:22.480 --> 0:09:24.920 realized there was really a lot more to it than 0:09:25.120 --> 0:09:28.720 a scient just a scientific question. And early on in 0:09:28.720 --> 0:09:31.920 my academic career as a physician scientist, I started an 0:09:31.920 --> 0:09:36.160 adult sickle cell disease treatment center, and I still remember this. 0:09:36.200 --> 0:09:39.200 One of the first patients that I started seeing was 0:09:39.640 --> 0:09:42.840 a woman who had long standing and severe disease and 0:09:42.920 --> 0:09:46.760 at her first clinic visit with me, as she walked in, 0:09:46.960 --> 0:09:50.600 sat down and said, Hi, doctor Hobbs, I've just got 0:09:50.600 --> 0:09:53.920 to know one thing, and that is if I ever 0:09:54.040 --> 0:09:57.760 need you, if I'm in an emergency room or a hospital, 0:09:57.800 --> 0:10:01.120 it's two in the morning, it's a night weekend, I 0:10:01.280 --> 0:10:03.000 just need to know that you're there on the other 0:10:03.080 --> 0:10:05.400 end of the phone twenty four hours a day, seven 0:10:05.480 --> 0:10:07.720 days a week, whenever I need you, because if you're not, 0:10:08.720 --> 0:10:11.400 then there's no reason for me to be here. And 0:10:11.480 --> 0:10:15.240 it was that awareness that I realized at that point 0:10:15.280 --> 0:10:17.559 that if you're going to take care of patients living 0:10:17.559 --> 0:10:19.600 with sickle cell disease, that you really need to be 0:10:19.720 --> 0:10:20.200 all in. 0:10:21.000 --> 0:10:21.800 That's a big ask. 0:10:22.679 --> 0:10:25.520 It was a big ass, but you know, it's actually 0:10:25.559 --> 0:10:27.840 a fair ask. And I think if you look at 0:10:27.840 --> 0:10:30.880 the history of how sickle cell disease patients have generally 0:10:30.920 --> 0:10:34.120 been treated by the medical system. I think sickle cell 0:10:34.200 --> 0:10:37.600 patients aren't asking for anything unique or above and beyond 0:10:37.679 --> 0:10:42.120 or special. They're really asking for equitable care and what 0:10:42.120 --> 0:10:44.840 we would offer to anyone else with the chronic severe disease. 0:10:45.840 --> 0:10:48.880 So what treatment options are available for patients with sickle 0:10:48.920 --> 0:10:49.480 cell disease. 0:10:49.840 --> 0:10:52.600 Yeah, you know, the causal biology of sickle cell disease 0:10:52.679 --> 0:10:56.400 has been known for many years, but for a long time, 0:10:56.840 --> 0:11:01.400 the only therapies available were able to deuce, but not 0:11:01.480 --> 0:11:05.800 really eliminate complications of disease in some but not all patients. 0:11:06.360 --> 0:11:08.400 And we've come a long way in the last few years, 0:11:08.400 --> 0:11:10.600 but there's still a lot of work yet to be done. 0:11:11.080 --> 0:11:15.000 And I often think about the sickle cell disease patients 0:11:15.000 --> 0:11:17.840 that I've cared for over the years, knowing that they 0:11:17.840 --> 0:11:21.840 were in desperate need of new treatment options, and so, 0:11:22.440 --> 0:11:25.000 you know, we continue to look at multiple angles and 0:11:25.240 --> 0:11:29.200 innovative options with the goal of treating the underlying cause 0:11:29.240 --> 0:11:29.880 of the disease. 0:11:30.520 --> 0:11:34.240 I'm curious, from Vertex's point of view, why has Vertex 0:11:34.280 --> 0:11:37.360 decided to invest so heavily in R and D specifically 0:11:37.360 --> 0:11:38.040 with sickle cell. 0:11:38.400 --> 0:11:42.720 For the most part, sickle cell disease has been largely 0:11:43.040 --> 0:11:46.800 ignored by the pharmaceutical industry for a long time, and 0:11:46.840 --> 0:11:49.880 I think there's always historically been a perception that it's 0:11:49.920 --> 0:11:53.280 a very difficult disease to go into from a commercial 0:11:53.280 --> 0:11:57.600 and marketing standpoint. However, if one has an approach that's 0:11:57.640 --> 0:12:01.640 based on solid biology and rectizes the unmet need that 0:12:01.720 --> 0:12:04.680 exists in sickle cell disease where there's millions of patients 0:12:04.720 --> 0:12:08.520 waiting worldwide, it becomes very compelling to do it. And 0:12:08.600 --> 0:12:12.120 so this is really about patients. It's understanding the medical need, 0:12:12.160 --> 0:12:17.080 it's understanding the causal biology and then innovating on treatment approaches, 0:12:17.240 --> 0:12:20.160 which is what we Advertex do And you could say 0:12:20.200 --> 0:12:22.040 that it's this is in our DNA. 0:12:22.720 --> 0:12:25.320 Sickle cell isn't a new disease. There's been a lot 0:12:25.360 --> 0:12:27.760 of work done and a lot of innovation. What is 0:12:27.920 --> 0:12:30.480 Vertex bringing to this that's new. 0:12:32.360 --> 0:12:36.679 Our aim is to investigate multiple different types of approaches 0:12:37.280 --> 0:12:41.120 that can give us the best opportunities to hopefully treat 0:12:41.120 --> 0:12:43.559 the causal biology of sickle cell disease for as many 0:12:43.640 --> 0:12:47.360 patients as possible. Whenever you do something like that, there 0:12:47.400 --> 0:12:49.920 are a number of unknown questions that have to be answered, 0:12:49.960 --> 0:12:52.959 both scientific and medical, and we're learning to understand what 0:12:53.040 --> 0:12:55.280 all of those are. And I think what we're also 0:12:55.440 --> 0:12:58.960 understanding is that for diseases like sickle cell disease, where 0:12:58.960 --> 0:13:02.120 there's such a huge need for treatment options, that serial 0:13:02.120 --> 0:13:05.120 innovation is required. And I think this is a focus 0:13:05.120 --> 0:13:08.520 for us to continue to innovate, because, as we all know, 0:13:08.640 --> 0:13:11.640 technology doesn't stand still, and so neither should we. 0:13:13.480 --> 0:13:16.880 That's the approach philosophically, but what does it actually look 0:13:16.960 --> 0:13:18.480 like when you put it into practice. 0:13:18.760 --> 0:13:21.680 We've learned a lot from natural history studies and a 0:13:21.720 --> 0:13:24.280 lot of other data about how we hope to be 0:13:24.320 --> 0:13:27.520 able to target causal biology with the aim of treating 0:13:27.600 --> 0:13:30.720 the effects of sickle cell disease. So for Vertex, it 0:13:30.760 --> 0:13:34.880 really begins with a deep scientific insight into causal biology 0:13:34.920 --> 0:13:39.520 and then serially innovating using all available tools and modalities 0:13:39.760 --> 0:13:44.679 to identify potential treatment options. And so it's essentially identifying 0:13:44.920 --> 0:13:47.320 the right tool for the job. And one of the 0:13:47.360 --> 0:13:50.120 things that's unique about Vertex and the approach is just 0:13:50.200 --> 0:13:53.960 that it's so completely focused on the patient and what 0:13:54.000 --> 0:13:57.280 the potential treatment outcomes could be. And so the focuses 0:13:57.320 --> 0:14:00.200 really have we identified and cracked the right science that 0:14:00.240 --> 0:14:03.560 can translate into something that could be meaningful for patients. 0:14:04.040 --> 0:14:06.400 So it's the goal is a high bar, and we 0:14:06.520 --> 0:14:09.720 do that relentlessly. We do it very aggressively, and we 0:14:09.800 --> 0:14:14.360 constantly strive to out innovate even ourselves within our own 0:14:14.400 --> 0:14:18.920 programs here, and that's how we do science and develop medicines. 0:14:26.280 --> 0:14:29.760 The research, the testing, and the potential approvals are moving 0:14:29.800 --> 0:14:33.320 forward as quickly as possible, and in the meantime, Terry 0:14:33.320 --> 0:14:35.680 Booker is just trying to live her best life. 0:14:36.120 --> 0:14:39.800 I exercise, I hang out with my kat Lewis, who 0:14:39.840 --> 0:14:42.720 I love and who he always knows when it's something 0:14:42.760 --> 0:14:45.920 wrong because he'll stay under me a little bit longer 0:14:46.040 --> 0:14:49.600 or a little bit more. And I cook often. I 0:14:49.640 --> 0:14:52.080 cook most of my meals. And when I say I 0:14:52.120 --> 0:14:57.080 cook every day, I cook every day. And I pray. 0:14:58.080 --> 0:15:02.280 What she's praying for ultimately is a cure. Terry says, 0:15:02.320 --> 0:15:04.760 until that happens, she'll continue to keep the faith. 0:15:05.200 --> 0:15:08.880 My faith has grown so much through this process because 0:15:09.720 --> 0:15:13.720 I feel like as a human, I feel like it's 0:15:13.760 --> 0:15:17.160 no way I could be going through these things, I 0:15:17.240 --> 0:15:23.160 should be in a whole crying somewhere. So our pain 0:15:23.280 --> 0:15:28.560 is real and we cope and we do what we 0:15:28.680 --> 0:15:34.600 have to do to try to live a quote unquote 0:15:34.640 --> 0:15:44.040 normal life. But our pain is real. And when people 0:15:44.160 --> 0:15:48.040 understand that, I feel like you can understand a sickle 0:15:48.080 --> 0:15:48.760 cell patient. 0:15:53.280 --> 0:15:57.200 This is targeting the toughest diseases. A podcast from Bloomberg 0:15:57.240 --> 0:16:01.000 Media Studios and Vertex Pharmaceuticals. If you like what you hear, 0:16:01.320 --> 0:16:05.320 subscribe and leave us a review. I'm Jordan Gospore. Thanks 0:16:05.320 --> 0:16:05.840 for listening.