WEBVTT - Targeting Sickle Cell Disease (Sponsored Content) 0:00:00.320 --> 0:00:03.840 Since you're a subscriber to this Bloomberg podcast, we thought 0:00:03.840 --> 0:00:07.320 you'd be interested in a six episode sponsored podcast called 0:00:07.760 --> 0:00:12.760 Targeting the Toughest Diseases, produced by Vertex Pharmaceuticals and Bloomberg 0:00:12.800 --> 0:00:17.640 Media Studios. It explores the innovative tools, methods, and unique 0:00:17.720 --> 0:00:22.479 philosophy Vertex Pharmaceuticals is using to search for treatments for 0:00:22.520 --> 0:00:27.320 some of humanity's most challenging diseases. Here's a recent episode, 0:00:29.760 --> 0:00:30.360 and it's like. 0:00:30.400 --> 0:00:34.440 If someone grabs your body and twists in the opposite 0:00:34.479 --> 0:00:40.159 direction with each hand, it's like you can feel the 0:00:40.360 --> 0:00:44.080 pressure in your bones and you can just feel every 0:00:44.320 --> 0:00:46.120 part of your body is hurting. 0:00:47.760 --> 0:00:51.320 Twenty years ago, during her sophomore year in college, Terry 0:00:51.320 --> 0:00:54.560 Booker was hit with the worst pain of her life. 0:00:56.560 --> 0:01:01.000 My legs were burning in then it just went up 0:01:01.040 --> 0:01:04.440 throughout my whole entire body to the point where I 0:01:04.560 --> 0:01:09.480 was unconscious and I was put on life support. I 0:01:09.560 --> 0:01:12.480 wasn't able to breathe on my own. They told my 0:01:12.600 --> 0:01:17.000 mom I had kidney failure, I had lung failure. 0:01:17.520 --> 0:01:20.919 There was no clear path to treatment for her, just uncertainty. 0:01:21.720 --> 0:01:24.520 They said, you just prayed because we don't know what's 0:01:24.560 --> 0:01:25.520 going to happen to her. 0:01:29.080 --> 0:01:33.280 What happened was she got better for a while, but 0:01:33.280 --> 0:01:35.280 then the pain returned and she was back in the 0:01:35.280 --> 0:01:39.319 emergency room. Her recurring visits to the er seeking relief 0:01:39.360 --> 0:01:43.039 from the pain revealed a second problem, not a medical one, 0:01:43.520 --> 0:01:48.000 a societal one. Terry is black, and as an African 0:01:48.000 --> 0:01:51.080 American woman seeking pay meds, she was presumed to be 0:01:51.120 --> 0:01:54.080 a drug addict and her medical issues were ignored. 0:01:55.080 --> 0:01:58.880 It is infuriating because when you go into the er, 0:01:58.960 --> 0:02:02.800 you're expecting to receive help. You're expecting to be treated 0:02:02.920 --> 0:02:06.680 as a patient the need of something, not treated as 0:02:06.720 --> 0:02:13.600 someone who is wanting to get their next high. 0:02:14.000 --> 0:02:18.320 Hi. I'm Jordan Gospore. I'm a member of the University 0:02:18.320 --> 0:02:23.000 of Southern California's Center for Health Journalism. This is Targeting 0:02:23.040 --> 0:02:27.320 the Toughest Diseases a podcast produced by Bloomberg Media Studios 0:02:27.480 --> 0:02:31.520 and Vertex Pharmaceuticals. In this series, we look at some 0:02:31.600 --> 0:02:35.960 of humanity's most challenging diseases and how Vertex, a Boston 0:02:36.000 --> 0:02:40.360 based biotech company, is using innovative tools, methods, and a 0:02:40.440 --> 0:02:46.120 unique philosophy to search for treatments and cures. Today, we're 0:02:46.160 --> 0:02:49.200 looking at. Sickle cell disease a blood disorder which can 0:02:49.200 --> 0:02:53.519 cause organ failure, stroke, and even death. It's a disease 0:02:53.560 --> 0:02:57.680 that affects roughly one hundred thousand Americans, including Terry Booker. 0:03:03.960 --> 0:03:07.000 Sickle cell disease is not something you catch. It's a 0:03:07.000 --> 0:03:10.840 genetic condition you're born with. Often babies are diagnosed before 0:03:10.880 --> 0:03:14.760 they're even born. Terry was an exception. She didn't know 0:03:14.840 --> 0:03:16.240 she had it until she was eleven. 0:03:18.320 --> 0:03:22.360 They pricked my finger and I remember them putting it 0:03:22.440 --> 0:03:25.639 under a microscope, and when they had it under a microscope, 0:03:26.200 --> 0:03:29.200 all these people were rushing over and I remember this 0:03:29.280 --> 0:03:33.840 so vividly, and they were like, oh, come here, come, Look. 0:03:34.520 --> 0:03:36.640 Terry was in fifth grade and her mam had taken 0:03:36.680 --> 0:03:39.200 her to the doctors because she had been complaining of pain. 0:03:39.880 --> 0:03:42.520 And I'm like okay, and they say you see all 0:03:42.520 --> 0:03:47.320 these little funny shaped cells and I'm like yeah. They're like, yeah, 0:03:47.520 --> 0:03:51.040 that's sickle cell and I said okay, and I'm like, 0:03:51.160 --> 0:03:52.119 well what does that mean. 0:03:56.800 --> 0:04:00.280 Sickle cell disease, or sickle cell anemia, as it's often called, 0:04:00.720 --> 0:04:04.520 affects red blood cells. Those are the cells that carry 0:04:04.560 --> 0:04:08.760 oxygen to all the tissues in our bodies. Normally, our 0:04:08.800 --> 0:04:11.360 red blood cells are soft and shaped like a doughnut, 0:04:11.720 --> 0:04:14.840 so they can squeeze through even the smallest of blood vessels. 0:04:15.920 --> 0:04:19.839 But sickle cell disease changes them. Specifically, it causes the 0:04:19.880 --> 0:04:24.120 hemoglobin proteins inside the red blood cells to change their structure. 0:04:24.880 --> 0:04:28.320 Instead of being donut shaped, they become curved like a 0:04:28.320 --> 0:04:31.920 cresset moon, or, as the name implies, like a sickle. 0:04:33.480 --> 0:04:37.280 These odd shaped cells are also hard and sticky, which 0:04:37.320 --> 0:04:40.840 means sometimes they can't flow smoothly through blood vessels, and 0:04:40.880 --> 0:04:43.880 when that happens, they start piling up like cars on 0:04:43.920 --> 0:04:47.560 a busy highway, making it really hard, if not impossible, 0:04:47.880 --> 0:04:50.960 for oxygen to reach where it needs to go. It's 0:04:51.000 --> 0:04:54.280 that lack of oxygen to tissues that causes the stabbing pain. 0:04:58.080 --> 0:05:00.640 That's the medical side of the disease, but as Terry 0:05:00.680 --> 0:05:03.720 mentioned earlier, there's also a societal component. 0:05:05.120 --> 0:05:08.320 To address circle cell disease, we also have to address 0:05:08.440 --> 0:05:13.640 the fundamental issues that relate to systemic racism and how 0:05:13.680 --> 0:05:15.440 it impacts on assets to care. 0:05:16.560 --> 0:05:20.040 That's doctor Isaac Odame. He studies the ways racism and 0:05:20.120 --> 0:05:22.960 medical care intersect when it comes to sickle cell disease. 0:05:23.920 --> 0:05:26.760 He's the medical director of the Global Sickle Cell Disease 0:05:26.800 --> 0:05:34.040 Network at the Center for Global Child Health. Doctor Odame 0:05:34.200 --> 0:05:35.279 grew up in West Africa. 0:05:35.760 --> 0:05:38.960 Psyco cell disease was something that I was very familiar with. 0:05:39.680 --> 0:05:42.640 Not only did I have this some family members who 0:05:43.040 --> 0:05:47.600 had children with psycosol disease, but some classmates and schoolmates 0:05:47.600 --> 0:05:51.560 who also lived with the condition. So I was exposed 0:05:51.640 --> 0:05:54.239 very early to the ravages of this disease. 0:05:55.040 --> 0:05:57.960 The first documented case of psycle cell anemia in America 0:05:58.160 --> 0:06:01.760 was in nineteen ten, just over a century ago, but 0:06:01.880 --> 0:06:04.479 its history dates back thousands of years. 0:06:04.880 --> 0:06:08.480 Sucer cell disease has been known for centuries in parts 0:06:08.560 --> 0:06:13.440 of Africa, and if you go into the oral tradition, 0:06:14.000 --> 0:06:18.920 they did have descriptions of the disease which typified the 0:06:19.240 --> 0:06:23.760 chronic and intercurrent as creating pain associative of the disease. 0:06:24.520 --> 0:06:28.159 Oddly enough, the mutation that changes the cell shapes was 0:06:28.200 --> 0:06:31.640 once an evolutionary advantage protecting against malaria. 0:06:32.320 --> 0:06:37.839 It predominantly affects people of African descent because malaria is 0:06:38.040 --> 0:06:42.680 very endemic in Africa. It occurs in people of Indian descent, 0:06:43.240 --> 0:06:47.960 Mediterranean descent, and parts of the Middle East Arabian Peninsula, 0:06:48.760 --> 0:06:51.480 and when you look at the common link between all 0:06:51.480 --> 0:06:57.159 these areas is malaria anddemicity. So it was a survival 0:06:57.200 --> 0:07:01.479 advantage it provided against malaria, and the percentages of people 0:07:01.520 --> 0:07:04.760 with traits in the population will rise over time. 0:07:05.680 --> 0:07:09.760 Having sickle cell trait is actually quite common in African populations. 0:07:10.280 --> 0:07:13.000 It evolved over time because it prevents the development and 0:07:13.040 --> 0:07:17.840 spreading of malaria. That historical context explains why the majority 0:07:17.880 --> 0:07:21.520 of sickle cell patients in the United States are African Americans. 0:07:22.120 --> 0:07:24.880 That means sickle cell care provides a clear example of 0:07:24.960 --> 0:07:28.600 the racial inequities and disparities in the medical system. That 0:07:28.640 --> 0:07:32.560 includes access to care and quality of care, both of 0:07:32.600 --> 0:07:36.720 which are lower for African Americans. Doctor Odami is hopeful 0:07:36.720 --> 0:07:39.559 that sickle cell treatments in development will not just treat 0:07:39.560 --> 0:07:42.800 the disease, but will address those other complex issues as well. 0:07:44.040 --> 0:07:47.760 I think more and more the patients are becoming more empowered, 0:07:48.720 --> 0:07:52.520 and I think they call themselves warriors, which is the 0:07:52.680 --> 0:07:55.680 appropriate term. They're not only dealing with the ravages of 0:07:55.720 --> 0:07:59.320 the disease, but they're also having to fight a system 0:07:59.640 --> 0:08:05.440 that doesn't recognize their needs appropriately, and so the warriors 0:08:05.520 --> 0:08:09.320 are beginning to speak out aloud. The system is beginning 0:08:09.360 --> 0:08:10.000 to respond. 0:08:11.600 --> 0:08:14.240 Vertex Pharmaceuticals is a leader in that response. 0:08:14.960 --> 0:08:17.680 For it to actually help people's lives, it has to 0:08:17.720 --> 0:08:19.840 work for them and it has to work for society. 0:08:20.400 --> 0:08:24.160 That's doctor David Altcheler, the chief scientific officer at Vertex. 0:08:24.800 --> 0:08:27.760 They have a unique approach to choosing the diseases they target. 0:08:28.760 --> 0:08:33.480 Vertex has decided to focus on a set of diseases 0:08:33.559 --> 0:08:36.280 where we see great unmet need, where we see the 0:08:36.520 --> 0:08:39.880 human biology is clear, and we feel that we have 0:08:40.000 --> 0:08:43.160 the technology, the insights, the wherewithal to make a difference. 0:08:43.559 --> 0:08:46.840 When it comes to Vertex's sickle cell program, Doctor William 0:08:46.880 --> 0:08:48.840 Hobbs is in charge of clinical development. 0:08:49.800 --> 0:08:53.760 You know, I started in medicine being interested in hematology 0:08:53.800 --> 0:08:58.080 and sickle cell disease in particular, mainly from a research standpoint, 0:08:58.480 --> 0:09:02.880 and that was because disease has long been considered a 0:09:03.000 --> 0:09:06.960 disease where the underlying cause of disease could be targeted, 0:09:07.520 --> 0:09:12.280 if innovative approaches could be identified, and so I really 0:09:12.360 --> 0:09:15.000 came at it from this scientific point of view, and 0:09:15.040 --> 0:09:18.280 then along the way I started to meet sickle cell 0:09:18.320 --> 0:09:22.800 disease patients and their families coordinate their care and realized 0:09:22.840 --> 0:09:25.160 there was really a lot more to it than a 0:09:25.640 --> 0:09:29.280 just a scientific question. And early on in my academic 0:09:29.320 --> 0:09:32.640 career as a physician scientist, I started an adult sickle 0:09:32.679 --> 0:09:36.280 cell disease treatment center, and I still remember this. One 0:09:36.320 --> 0:09:39.680 of the first patients that I started seeing was a 0:09:39.720 --> 0:09:43.000 woman who had long standing and severe disease, and at 0:09:43.040 --> 0:09:46.720 her first clinic visit with me, as she walked in, 0:09:46.880 --> 0:09:50.560 sat down and said, Hi, doctor Hobbs, I've just got 0:09:50.559 --> 0:09:53.880 to know one thing, and that is if I ever 0:09:53.960 --> 0:09:57.679 need you, if I'm in an emergency room or a hospital, 0:09:57.720 --> 0:10:00.439 it's two in the morning, it's a night, it's a weekend, 0:10:00.960 --> 0:10:02.760 I just need to know that you're there on the 0:10:02.760 --> 0:10:04.880 other end of the phone twenty four hours a day, 0:10:05.080 --> 0:10:07.320 seven days a week, whenever I need you, because if 0:10:07.360 --> 0:10:10.520 you're not, then there's no reason for me to be here. 0:10:11.240 --> 0:10:14.840 And it was that awareness that I realized at that 0:10:14.880 --> 0:10:17.200 point that if you're going to take care of patients 0:10:17.240 --> 0:10:19.440 living with sickle cell disease, that you really need to 0:10:19.480 --> 0:10:20.160 be all in. 0:10:20.960 --> 0:10:21.760 That's a big ask. 0:10:22.640 --> 0:10:25.480 It was a big ass but you know, it's actually 0:10:25.520 --> 0:10:27.800 a fair ask. And I think if you look at 0:10:27.800 --> 0:10:30.760 the history of how sickle cell disease patients have generally 0:10:30.840 --> 0:10:34.080 been treated by the medical system. I think sickle cell 0:10:34.160 --> 0:10:37.520 patients aren't asking for anything unique or above and beyond 0:10:37.600 --> 0:10:42.040 or special. They're really asking for equitable care and what 0:10:42.080 --> 0:10:44.760 we would offer to anyone else with the chronic severe disease. 0:10:45.800 --> 0:10:48.840 So what treatment options are available for patients with sickle 0:10:48.840 --> 0:10:49.400 cell disease? 0:10:49.760 --> 0:10:52.560 Yeah, you know, the causal biology of sickle cell disease 0:10:52.600 --> 0:10:56.360 has been known for many years, but for a long time, 0:10:56.760 --> 0:11:01.360 the only therapies available were able to reduce, but not 0:11:01.400 --> 0:11:05.720 really eliminate complications of disease in some but not all patients. 0:11:06.320 --> 0:11:08.319 And we've come a long way in the last few years, 0:11:08.360 --> 0:11:10.520 but there's still a lot of work yet to be done. 0:11:11.000 --> 0:11:14.920 And I often think about the sickle cell disease patients 0:11:14.960 --> 0:11:17.800 that I've cared for over the years, knowing that they 0:11:17.800 --> 0:11:21.760 were in desperate need of new treatment options, and so, 0:11:22.360 --> 0:11:24.960 you know, we continue to look at multiple angles and 0:11:25.200 --> 0:11:29.160 innovative options with the goal of treating the underlying cause 0:11:29.200 --> 0:11:29.800 of the disease. 0:11:30.480 --> 0:11:34.160 I'm curious, from Vertex's point of view, why has Vertex 0:11:34.240 --> 0:11:37.239 decided to invest so heavily in R and D specifically 0:11:37.320 --> 0:11:38.000 with sickle cell. 0:11:38.320 --> 0:11:42.679 For the most part, sickle cell disease has been largely 0:11:43.000 --> 0:11:46.760 ignored by the pharmaceutical industry for a long time, and 0:11:46.800 --> 0:11:49.839 I think there's always historically been a perception that it's 0:11:49.880 --> 0:11:53.240 a very difficult disease to go into from a commercial 0:11:53.240 --> 0:11:57.560 and marketing standpoint. However, if one has an approach that's 0:11:57.600 --> 0:12:01.280 based on solid biology and reckon is the unmet need 0:12:01.440 --> 0:12:04.120 that exists in sickle cell disease, where there's millions of 0:12:04.160 --> 0:12:07.760 patients waiting worldwide, it becomes very compelling to do it. 0:12:08.320 --> 0:12:11.440 And so this is really about patients. It's understanding the 0:12:11.440 --> 0:12:15.440 medical need, it's understanding the causal biology and then innovating 0:12:15.840 --> 0:12:18.760 on treatment approaches, which is what we ad Vertex do. 0:12:19.360 --> 0:12:21.960 And you could say that it's this is in our DNA. 0:12:22.640 --> 0:12:25.319 Sickle cell isn't a new disease. There's been a lot 0:12:25.320 --> 0:12:27.680 of work done and a lot of innovation. What is 0:12:27.880 --> 0:12:30.439 VerTech bringing to this that's new. 0:12:32.320 --> 0:12:36.640 Our aim is to investigate multiple different types of approaches 0:12:37.200 --> 0:12:41.040 that can give us the best opportunities to hopefully treat 0:12:41.080 --> 0:12:43.560 the causal biology of sickle cell disease for as many 0:12:43.600 --> 0:12:47.280 patients as possible. Whenever you do something like that, there 0:12:47.320 --> 0:12:49.840 are a number of unknown questions that have to be answered, 0:12:49.920 --> 0:12:52.920 both scientific and medical, and we're learning to understand what 0:12:53.000 --> 0:12:55.280 all of those are. And I think what we're also 0:12:55.400 --> 0:12:58.920 understanding is that for diseases like sickle cell disease, where 0:12:58.920 --> 0:13:02.040 there's such a huge need for treatment options, that serial 0:13:02.080 --> 0:13:05.040 innovation is required. And I think this is a focus 0:13:05.080 --> 0:13:08.480 for us to continue to innovate, because, as we all know, 0:13:08.600 --> 0:13:11.560 technology doesn't stand still, and so neither should we. 0:13:13.400 --> 0:13:16.840 That's the approach philosophically, but what does it actually look 0:13:16.920 --> 0:13:18.440 like when you put it into practice. 0:13:18.679 --> 0:13:21.640 We've learned a lot from natural history studies and a 0:13:21.640 --> 0:13:24.240 lot of other data about how we hope to be 0:13:24.240 --> 0:13:27.520 able to target causal biology with the aim of treating 0:13:27.559 --> 0:13:30.679 the effects of sickle cell disease. So for Vertex, it 0:13:30.720 --> 0:13:34.840 really begins with a deep scientific insight into causal biology 0:13:34.880 --> 0:13:39.439 and then serially innovating using all available tools and modalities 0:13:39.720 --> 0:13:44.640 to identify potential treatment options. And so it's essentially identifying 0:13:44.880 --> 0:13:47.280 the right tool for the job. And one of the 0:13:47.320 --> 0:13:50.080 things that's unique about Vertex and the approach is just 0:13:50.160 --> 0:13:53.920 that it's so completely focused on the patient and what 0:13:53.960 --> 0:13:57.240 the potential treatment outcomes could be. And so the focuses 0:13:57.280 --> 0:14:00.160 really have we identified and cracked the right science that 0:14:00.200 --> 0:14:03.520 can translate into something that could be meaningful for patients. 0:14:04.000 --> 0:14:06.360 So it's the goal is a high bar, and we 0:14:06.480 --> 0:14:09.640 do that relentlessly. We do it very aggressively, and we 0:14:09.760 --> 0:14:14.320 constantly strive to out innovate even ourselves within our own 0:14:14.360 --> 0:14:18.880 programs here, and that's how we do science and develop medicines. 0:14:26.240 --> 0:14:29.720 The research, the testing, and the potential approvals are moving 0:14:29.760 --> 0:14:33.240 forward as quickly as possible, and in the meantime, Terry 0:14:33.280 --> 0:14:35.520 Booker is just trying to live her best life. 0:14:36.080 --> 0:14:39.760 I exercise, I hang out with my kat Lewis, who 0:14:39.800 --> 0:14:42.680 I love and who he always knows when it's something 0:14:42.720 --> 0:14:45.840 wrong because he'll stay under me a little bit longer 0:14:45.960 --> 0:14:49.560 or a little bit more. And I cook often. I 0:14:49.600 --> 0:14:52.040 cook most of my meals. And when I say I 0:14:52.040 --> 0:14:57.040 cook every day, I cook every day. And I pray. 0:14:58.000 --> 0:15:02.200 What she's praying for ultimately is a cure. Terry says, 0:15:02.280 --> 0:15:04.720 until that happens, she'll continue to keep the faith. 0:15:05.160 --> 0:15:08.800 My faith has grown so much through this process because 0:15:09.680 --> 0:15:13.680 I feel like as a human, I feel like it's 0:15:13.720 --> 0:15:17.120 no way I could be going through these things. I 0:15:17.160 --> 0:15:20.560 should be in a whole crying somewhere. 0:15:21.880 --> 0:15:28.000 So our pain is real and we cope and we 0:15:28.040 --> 0:15:29.440 do what we have to do. 0:15:32.040 --> 0:15:36.160 To try to live a quote unquote normal life. 0:15:36.920 --> 0:15:44.800 But our pain is real. And when people understand that, 0:15:45.480 --> 0:15:48.720 I feel like you can understand a sickle cell patient. 0:15:53.240 --> 0:15:57.160 This is targeting the toughest diseases. A podcast from Bloomberg 0:15:57.200 --> 0:16:00.920 Media Studios and Vertex Pharmaceuticals. If you like what you hear, 0:16:01.240 --> 0:16:05.240 subscribe and leave us a review. I'm Jordan Gospore. Thanks 0:16:05.280 --> 0:16:05.800 for listening.