WEBVTT - Targeting the Toughest Diseases (Sponsored Content) 0:00:00.120 --> 0:00:03.640 I'm Edward Adams of Bloomberg Media Studios. Since you're a 0:00:03.640 --> 0:00:07.040 subscriber to Bloomberg BusinessWeek, we thought you'd be interested in 0:00:07.080 --> 0:00:12.080 a six episode sponsored content podcast called Targeting the Toughest Diseases. 0:00:13.000 --> 0:00:15.720 It shows how the battle against some of humanity's most 0:00:15.800 --> 0:00:20.800 challenging diseases is happening at the intersection of business and medicine. 0:00:21.320 --> 0:00:25.720 Here's a full episode featuring NBA great Alonzo Mourning recounting 0:00:25.760 --> 0:00:27.560 his fight against kidney disease. 0:00:33.120 --> 0:00:37.440 Miami Arena, March twenty ninth, nineteen ninety six. The Miami 0:00:37.479 --> 0:00:43.159 Heat are facing the Washington Bullets all night long. Alonzo 0:00:43.240 --> 0:00:45.839 Morning of the Heat has been guarded by a giant 0:00:45.960 --> 0:00:49.600 of a man named George Morrissan. At seven foot seven, 0:00:49.960 --> 0:00:52.560 Morrisson is one of the fiercest players in the league, 0:00:52.800 --> 0:00:56.840 the tallest player in NBA history, an impenetrable wall with 0:00:56.920 --> 0:01:03.080 a NonStop motor, but Alonzo Morning is one of the 0:01:03.080 --> 0:01:08.600 greatest ever to step on the court. When the final 0:01:08.600 --> 0:01:11.400 buzzer goes, the Heat have won one hundred and twelve 0:01:11.480 --> 0:01:15.800 to ninety three, and Morning has scored an incredible fifty points. 0:01:16.240 --> 0:01:18.840 That game was a career high for Morning. He would 0:01:18.880 --> 0:01:21.080 go on to be a seven time All Star and 0:01:21.120 --> 0:01:24.479 a gold medalist in the two thousand Olympics, But little 0:01:24.480 --> 0:01:27.720 did he know his toughest opponent was yet to come. 0:01:29.240 --> 0:01:32.679 After returning from the Olympics in Sydney, morning started into 0:01:32.720 --> 0:01:36.560 his usual off season training, but he noticed something was wrong. 0:01:37.000 --> 0:01:42.640 I was experiencing lethargy, edema in my legs, swelling and 0:01:42.680 --> 0:01:48.000 my lower extremities. Extremely tired, worn out. 0:01:48.880 --> 0:01:52.320 He thought it was jetlag, maybe the flu. He figured 0:01:52.360 --> 0:01:55.280 he'd just take a couple days off then resume his training. 0:01:55.840 --> 0:01:59.120 His doctor had a different idea. He suggested they run 0:01:59.160 --> 0:01:59.720 some tests. 0:02:00.360 --> 0:02:04.000 I answered the phone next to the bed and he said, 0:02:04.080 --> 0:02:07.000 you know, I got your results back. He said that 0:02:07.080 --> 0:02:14.400 you've got this rare genetic disorder called focal segmental glamary 0:02:14.480 --> 0:02:17.640 los carosis. And I said, Doc, what is that? He said, 0:02:17.639 --> 0:02:20.040 It's a disease that scars the filters in the kidney. 0:02:20.480 --> 0:02:25.000 Focal segmental glamory you losclerosis or FSGS causes scar tissue 0:02:25.040 --> 0:02:27.080 to develop on the small parts of the kidneys that 0:02:27.120 --> 0:02:28.360 filter waste from the blood. 0:02:28.639 --> 0:02:32.480 I asked them three questions. I said, is there a 0:02:32.520 --> 0:02:37.720 cure for this? He said, no, I said, can I 0:02:37.720 --> 0:02:43.400 play basketball again? He said, I don't know, And then 0:02:43.560 --> 0:02:45.480 I said, well, am I going to die? 0:02:54.080 --> 0:02:54.240 Hi? 0:02:54.560 --> 0:02:57.560 I'm Jordan Gospore. I'm a member of the University of 0:02:57.600 --> 0:03:01.200 Southern California's Center for Health Journalism. This is Targeting the 0:03:01.240 --> 0:03:05.360 Toughest Diseases a podcast produced by Bloomberg Media Studios and 0:03:05.480 --> 0:03:09.079 Vertex Pharmaceuticals. In this series, we look at some of 0:03:09.160 --> 0:03:13.480 humanity's most challenging diseases and how Vertex, a Boston based 0:03:13.480 --> 0:03:17.560 biotech company, is using innovative tools, methods, and a unique 0:03:17.560 --> 0:03:22.639 philosophy to search for treatments and cures. Today, we're looking 0:03:22.720 --> 0:03:26.240 at apol one mediated kidney disease, a disease caused by 0:03:26.280 --> 0:03:30.280 a genetic abnormality, one that thirteen percent of African Americans carry. 0:03:30.880 --> 0:03:34.200 This abnormality can cause a number of types of kidney diseases, 0:03:34.560 --> 0:03:37.880 one of which is FSGS, the condition alonzo morning was 0:03:37.920 --> 0:03:38.520 diagnosed with. 0:03:41.760 --> 0:03:48.600 I thought to myself, not why me, but why right now? God? 0:03:48.760 --> 0:03:51.920 Why am I dealing with dish right now? Of all times. 0:03:52.560 --> 0:03:55.440 I had just come back from the Sydney, Australia Olympics 0:03:55.440 --> 0:03:57.520 win in the gold medal. I had just come off 0:03:57.520 --> 0:04:01.360 from an amazing basketball season where I had first team 0:04:01.400 --> 0:04:04.960 All NBA All Star Team. The list goes on, you know, 0:04:05.120 --> 0:04:09.320 all the accolades, and then all of a sudden, bam. 0:04:09.480 --> 0:04:10.080 This happens. 0:04:10.600 --> 0:04:14.880 Lots of young professional athletes view themselves as invincible. Alonso 0:04:15.000 --> 0:04:17.800 suddenly found out he wasn't hung off the. 0:04:17.760 --> 0:04:22.680 Phone and my face fell in my hands. I felt 0:04:22.760 --> 0:04:24.240 like I was gonna play till I was in my 0:04:24.360 --> 0:04:27.680 forties because I was in such great shape. Just airing 0:04:27.720 --> 0:04:30.240 it from somebody saying hey, you got to stop playing. 0:04:31.240 --> 0:04:33.839 It was humbling and it was deflating. Yeah, and I 0:04:34.040 --> 0:04:37.000 just was just sitting there just trying to figure out, Okay, 0:04:37.480 --> 0:04:39.640 how is this all going to materialize? 0:04:44.200 --> 0:04:47.680 We can't survive without our kidneys. They played a vital role. 0:04:48.279 --> 0:04:51.080 They help our bodies maintain just the right balance of 0:04:51.080 --> 0:04:55.800 electrolytes like potassium, They control blood pressure, they cleaner blood, 0:04:56.320 --> 0:05:00.000 and they even help maintain our hormone levels. Each kidney 0:05:00.200 --> 0:05:02.760 is made up of a million or so tiny filters 0:05:02.880 --> 0:05:07.520 called gloomerulie. They're like little coffee filters. The filtered liquid 0:05:07.560 --> 0:05:10.799 becomes urine and the protein left behind stays in our blood. 0:05:11.000 --> 0:05:14.719 But when the glomerulaie become damaged, those proteins start leaking 0:05:14.760 --> 0:05:18.159 into the urine. The scary thing is it's estimated thirty 0:05:18.160 --> 0:05:20.960 seven million adults in the United States have kidney disease, 0:05:21.279 --> 0:05:23.960 and ninety percent of them don't even know they have it. 0:05:24.480 --> 0:05:27.320 And in the case of apol one mediated kidney disease, 0:05:27.640 --> 0:05:32.279 the prime causal factor is invisible. It's genetic caused by 0:05:32.400 --> 0:05:40.400 mutations in the apoel one gene. Back in the early 0:05:40.480 --> 0:05:44.320 nineteen nineties, doctor David Friedman, a doctor researcher and an 0:05:44.360 --> 0:05:47.560 associate professor with Harvard Medical School who currently works with 0:05:47.680 --> 0:05:51.240 Vertex on its clinical trials, first started to notice something unusual. 0:05:51.600 --> 0:05:55.120 He was seeing African American families where multiple members all 0:05:55.160 --> 0:05:56.040 had kidney disease. 0:05:56.880 --> 0:06:00.320 When there's an important inherited component of a disay disease, 0:06:00.560 --> 0:06:02.400 it tends to cluster in families. 0:06:03.000 --> 0:06:06.600 Understanding there was a genetic cause was just the beginning. Next, 0:06:06.640 --> 0:06:08.480 they had to find the exact gene. 0:06:10.800 --> 0:06:14.040 I think a real breakthrough in our understanding came in 0:06:14.040 --> 0:06:17.320 two thousand and eight when some teams at Johns Hopkins 0:06:17.320 --> 0:06:21.280 and the NIH were the first to find a location 0:06:21.440 --> 0:06:25.040 in the genome. My chromosome twenty two, where it became 0:06:25.080 --> 0:06:28.800 apparent that there was something strong that was impacting kidney 0:06:28.800 --> 0:06:30.880 disease in people of African ancestry. 0:06:31.200 --> 0:06:34.800 Then in twenty ten, doctor Friedman and his colleagues identified 0:06:34.960 --> 0:06:38.320 the specific mutations that led to this type of kidney disease. 0:06:38.800 --> 0:06:44.120 There were two important advances in technology which really helped 0:06:44.200 --> 0:06:48.400 us to pinpoint these two genetic variants in April one. 0:06:48.880 --> 0:06:54.200 The first was related to tools for identifying positive selection 0:06:54.560 --> 0:06:59.800 in the genome, and these mathematical tools helped us in 0:07:00.040 --> 0:07:02.720 vision the genome in a slightly different way. And the 0:07:02.760 --> 0:07:09.760 second major technological advance was a database of genetic variants 0:07:09.800 --> 0:07:14.560 in people of widely diverse ancestries called the Thousand Genomes Project. 0:07:14.800 --> 0:07:17.640 Up until that time, most of what we knew about 0:07:17.720 --> 0:07:23.760 genetic variation came from people of European ancestry, and this new, 0:07:24.200 --> 0:07:28.679 very powerful tool was a really equivalent of an encyclopedia 0:07:29.160 --> 0:07:31.560 of genetic variation around the world. 0:07:32.160 --> 0:07:36.320 From there, our understanding of apol one has continued to increase, 0:07:36.680 --> 0:07:40.600 including why the risk variants in this gene only affect 0:07:40.640 --> 0:07:45.360 people of recent African descent, including African American, LATINX and 0:07:45.440 --> 0:07:51.400 Afro Caribbean communities. Thousands of years ago, a genetic mutation 0:07:51.560 --> 0:07:54.760 in the apol one gene developed in Sub Saharan Africa 0:07:54.800 --> 0:07:56.280 as a protective mechanism. 0:07:57.120 --> 0:08:00.600 We all have a gene that encodes for or the 0:08:00.640 --> 0:08:04.800 april one protein, but the version which causes kidney disease 0:08:05.120 --> 0:08:10.120 contains some very slight changes in the instructions for building 0:08:10.160 --> 0:08:14.440 that protein that turn out to make it very effective 0:08:14.840 --> 0:08:20.240 for killing the trapanosomes that cause African sleeping sickness in humans. 0:08:20.880 --> 0:08:23.640 Sleeping sickness is a disease spread by the bite of 0:08:23.640 --> 0:08:26.640 an infected fly, and it can cause death within a 0:08:26.680 --> 0:08:29.920 matter of weeks. But this genetic mutation stops it in 0:08:29.960 --> 0:08:30.560 its tracks. 0:08:31.720 --> 0:08:34.640 Because it was so effective, it spread very quickly in 0:08:34.679 --> 0:08:39.360 the population. But it's these proteins with a slightly different 0:08:39.440 --> 0:08:42.280 version of april one. It's very very effective for killing 0:08:42.320 --> 0:08:46.559 trypanosomes and preventing African sleeping sickness, which is the same 0:08:46.720 --> 0:08:51.480 version of the protein which makes the kidneys of people 0:08:51.640 --> 0:08:53.720 sick who have this genetic variant. 0:08:54.880 --> 0:08:57.680 We now understand that having one apo l one risk 0:08:57.800 --> 0:09:00.640 variant could protect you from many forms of sleeping sickness, 0:09:00.679 --> 0:09:04.000 but if you were unlucky enough to inherit too, one 0:09:04.000 --> 0:09:06.760 from your mother and one from your father. Your chances 0:09:06.760 --> 0:09:21.880 of getting kidney disease goes up tenfold, Doctor Fire, Doctor j. 0:09:23.120 --> 0:09:25.800 Once your kidneys start to fail, there really are only 0:09:25.840 --> 0:09:29.440 a few ways to stay alive. One is dialysis. It 0:09:29.480 --> 0:09:33.360 replaces the function of the kidney. Doctor Janice Lee studies 0:09:33.440 --> 0:09:37.520 kidney disorders at Emory University School of Medicine in Atlanta, Georgia. 0:09:38.040 --> 0:09:43.000 Kidneys excrete waste products, and they get rid of excess 0:09:43.120 --> 0:09:47.920 fluid from our bodies. So that's what the dialysis machine does. 0:09:48.679 --> 0:09:51.920 Two needles are inserted into a patient's arm. One draws 0:09:51.960 --> 0:09:54.240 blood out and sends it through the machine to be cleaned. 0:09:54.840 --> 0:09:57.760 The other needle returns the clean blood back into the patient, 0:09:58.320 --> 0:10:01.760 which is pretty much exactly the way kidney's work, except 0:10:02.000 --> 0:10:04.480 while your kidney's work slowly. Twenty four hours a day, 0:10:04.559 --> 0:10:07.839 seven days a week. Dialysis means sitting in a chair 0:10:08.200 --> 0:10:10.440 plugged into a machine for two to three hours at 0:10:10.440 --> 0:10:14.200 a time, three times a week. It works, but it's 0:10:14.240 --> 0:10:15.600 not ideal if. 0:10:15.480 --> 0:10:19.360 You really think about it. We go to the bathroom 0:10:19.440 --> 0:10:23.520 excrete our urine two or three four times or more 0:10:23.600 --> 0:10:27.360 a day. So when patients are in dialysis, they're really 0:10:27.400 --> 0:10:31.800 getting their blood cleansed just three days a week for 0:10:31.920 --> 0:10:36.720 a few hours, and so patients can feel washed out 0:10:36.880 --> 0:10:39.920 when they get off of dialysis because they've had all 0:10:40.000 --> 0:10:43.959 this fluid from two days worth of not excreting any 0:10:44.520 --> 0:10:46.360 waste products are fluid. 0:10:47.000 --> 0:10:50.560 There's no cure available for chronic kidney disease. The medications 0:10:50.600 --> 0:10:53.840 currently on the market focus on making sure a person's 0:10:53.880 --> 0:10:58.720 kidneys don't deteriorate further. The current options include medications that 0:10:58.800 --> 0:11:04.040 suppress the immune system, diuretics, ace inhibitors or ARB medications 0:11:04.080 --> 0:11:08.160 to control blood pressure, or lower urine protein anticoagulants to 0:11:08.240 --> 0:11:11.480 prevent blood clots. And then there's the option Alonzo Morning 0:11:11.520 --> 0:11:15.720 had done for his FSGS, a kidney transplant. That approach 0:11:15.800 --> 0:11:18.880 requires you to be fortunate enough to find a suitable donor. 0:11:19.280 --> 0:11:22.400 On average, it also only lasts ten to twelve years. 0:11:25.120 --> 0:11:28.760 Vertex Pharmaceuticals is a company that is researching apol one 0:11:28.800 --> 0:11:32.200 mediated kidney disease and other tough diseases where there's a 0:11:32.280 --> 0:11:35.680 huge unmet need. They are targeting conditions where the human 0:11:35.720 --> 0:11:39.680 biology is understood, the technology already exists, or Vertex thinks 0:11:39.679 --> 0:11:42.120 it can develop it and where Vertex has an approach 0:11:42.240 --> 0:11:45.800 they think maybe transformative. They have several programs in their 0:11:45.840 --> 0:11:53.320 investigational research, including apol one mediated kidney disease. Doctor ogo 0:11:53.360 --> 0:11:56.959 Egbunna leads clinical development for the team researching kidney disease 0:11:57.000 --> 0:11:57.640 at Vertex. 0:11:58.200 --> 0:12:00.559 As we speak here in the US, there probably more 0:12:00.559 --> 0:12:04.560 than one hundred thousand people waiting for a kidney, and 0:12:04.679 --> 0:12:07.440 not everyone is fortunate to be able to get one. 0:12:07.640 --> 0:12:09.840 Many people will die waiting for a kidney. 0:12:10.240 --> 0:12:12.719 The staggering number of people living with and dying from 0:12:12.800 --> 0:12:15.160 kidney disease is one of the main reasons why doctor 0:12:15.240 --> 0:12:17.160 ed Buna joined Vertex's efforts. 0:12:17.360 --> 0:12:20.080 It was really heartbreaking, you know, at the beginning of 0:12:20.080 --> 0:12:23.320 the year, I'd have a whole host of patients on 0:12:23.440 --> 0:12:26.800 dialysis and at the end of the year, one or 0:12:26.800 --> 0:12:30.240 two out of every three would have, you know, passed on. 0:12:30.760 --> 0:12:33.840 That was just too depressing for me. 0:12:34.400 --> 0:12:38.000 Why did Vertex choose to focus on a pall one 0:12:38.400 --> 0:12:40.400 versus other types of kidney disease. 0:12:41.000 --> 0:12:46.079 This is actually one of the most difficult kidney diseases 0:12:46.160 --> 0:12:51.600 that have lagued a minority population and underserved population for 0:12:52.280 --> 0:12:56.800 so long and for the longest time we have attributed 0:12:56.840 --> 0:12:59.800 this to either a bad diet or lack of ex 0:13:00.120 --> 0:13:04.000 size or nutrition, but we do know now that there 0:13:04.160 --> 0:13:07.480 is a genetic basis for a lot of this disparity. 0:13:07.720 --> 0:13:11.840 And I think in typical Vertex fashion, we go after 0:13:11.960 --> 0:13:16.000 diseases that have a serious unmet need, and in addition 0:13:16.080 --> 0:13:20.280 to that, we go after diseases where the underlying cause 0:13:20.640 --> 0:13:25.400 is well understood and for which we apply the best 0:13:25.440 --> 0:13:27.080 science available to try and address it. 0:13:27.280 --> 0:13:28.320 What can we look forward to. 0:13:28.880 --> 0:13:31.200 Part of the reason why I'm actually so excited about 0:13:31.320 --> 0:13:33.880 what we're doing here at Vertex is because we have 0:13:34.080 --> 0:13:40.079 found small molecule therapies that are investigational to the underlying 0:13:40.160 --> 0:13:43.800 cause of what I've described as equal one mediated kidney disease. 0:13:44.160 --> 0:13:49.319 We have evaluated these potential therapies in experimental settings and 0:13:49.600 --> 0:13:53.800 first in human studies as well as a preliminary proof 0:13:53.800 --> 0:13:56.400 of concept study in patients. 0:13:57.760 --> 0:14:01.120 Developing a potential small molecule therapy is no easy feat, 0:14:01.520 --> 0:14:04.480 doctor ed Buna, says scientists a Vertex have gone through 0:14:04.679 --> 0:14:08.840 hundreds of thousands of candidate molecules in their kidney disease research. 0:14:09.760 --> 0:14:14.160 Nothing good or great comes easy. Therein lies the promise 0:14:14.200 --> 0:14:16.480 and the excitement While. 0:14:16.320 --> 0:14:19.360 Their research continues. Doctor Egbunna says he and his team 0:14:19.400 --> 0:14:22.360 at Vertex will do everything they can to raise awareness 0:14:22.360 --> 0:14:26.840 for the disease. That includes educating on preventative measures like genotyping, 0:14:27.040 --> 0:14:29.920 which can help determine whether a patient carries the apol 0:14:29.960 --> 0:14:31.040 one genetic variant. 0:14:31.840 --> 0:14:35.600 So, in addition to supporting the clinical community on patients 0:14:35.680 --> 0:14:38.640 in increasing the rates of diagnosis of kidney disease, we 0:14:38.680 --> 0:14:43.360 also want an increase in the awareness and of genotyping 0:14:43.720 --> 0:14:46.560 so that precise diagnosis can be made so that the 0:14:46.720 --> 0:14:49.920 right therapy can be brought to the patient. Dollons of 0:14:49.960 --> 0:14:52.760 Money is one of those great examples of people that 0:14:53.040 --> 0:14:57.440 went through the science, symptoms, the worries, the challenges of 0:14:57.600 --> 0:15:01.440 n stage advanced kidney disease. I stunding and he got 0:15:01.520 --> 0:15:02.440 back on his feet. 0:15:05.760 --> 0:15:09.320 Alonzo Morning underwent a kidney transplant three years after his 0:15:09.360 --> 0:15:10.479 initial diagnosis. 0:15:11.160 --> 0:15:16.400 So the recovery process was grueling and it was extremely 0:15:16.520 --> 0:15:19.720 painful at times, and it was difficult. But if you 0:15:19.760 --> 0:15:23.200 think about anything in life worth having, it's very difficult 0:15:23.240 --> 0:15:25.400 to get it. And I was trying to get my 0:15:25.480 --> 0:15:27.200 help back in. I was trying to get back on 0:15:27.240 --> 0:15:29.800 the court, So I was trying to get back to 0:15:29.840 --> 0:15:33.360 some sense of normalcy, but it challenged me tremendously, and 0:15:33.920 --> 0:15:35.440 it really truly challenged me. 0:15:36.200 --> 0:15:38.920 Morning talks about his struggle to get and then adapt 0:15:38.960 --> 0:15:41.800 to living with a new kidney, knowing full well that 0:15:41.880 --> 0:15:43.800 he had a lot of advantages. 0:15:43.800 --> 0:15:47.600 That was in optimal condition, excellent health, you know, but 0:15:48.720 --> 0:15:53.360 that was the benefit of my recovery so fast, because 0:15:53.400 --> 0:15:56.800 the doctors told me, like, hey, you know, if you 0:15:56.920 --> 0:16:01.560 wasn't this high performance athlete, then you probably wouldn't have 0:16:01.560 --> 0:16:04.640 bounced back as fast after the transplant. 0:16:04.920 --> 0:16:08.640 And bounce back he did. Just three years after his transplant. 0:16:08.720 --> 0:16:11.280 Alonso Morning would go on and win a championship with 0:16:11.320 --> 0:16:14.080 the Miami Heat in two thousand and six, and today 0:16:14.360 --> 0:16:17.960 he's partnering with Vertex as a spokesperson, using his profile 0:16:18.040 --> 0:16:20.600 and his experience to advocate and educate. 0:16:21.280 --> 0:16:23.240 You know. A big part of it is to try 0:16:23.240 --> 0:16:28.560 to inspire and provide hope and encouragement for those who 0:16:28.640 --> 0:16:30.840 are going through the same ordeal. 0:16:31.040 --> 0:16:33.640 And Morning says he's hoping Vertex will be able to 0:16:33.640 --> 0:16:37.240 help kidney patients by raising awareness of the disease, encouraging 0:16:37.280 --> 0:16:40.320 people to visit their doctor and continuing to search for 0:16:40.360 --> 0:16:41.360 a potential treatment. 0:16:41.960 --> 0:16:45.240 So if this can happen, then all of what I've 0:16:45.280 --> 0:16:50.600 gone through is so much more of a worthwhile because 0:16:51.240 --> 0:16:53.840 I'm able to help save other people's lives. 0:17:04.320 --> 0:17:07.440 This is Targeting the Toughest diseases. A podcast from Bloomberg 0:17:07.480 --> 0:17:11.200 Media Studios and Vertex Pharmaceuticals. If you like what you hear, 0:17:11.520 --> 0:17:15.200 subscribe and leave us a review. I'm Jordan Gospore. Thanks 0:17:15.200 --> 0:17:15.760 for listening.