WEBVTT - Targeting the Toughest Diseases (Sponsored Content) 0:00:00.680 --> 0:00:04.240 I'm Edward Adams of Bloomberg Media Studios. Since you're a 0:00:04.280 --> 0:00:07.440 subscriber to Bloomberg Business Week, we thought you'd be interested 0:00:07.440 --> 0:00:11.840 in a new six episode sponsored content podcast called Targeting 0:00:11.840 --> 0:00:15.680 the Toughest Diseases. It shows how the battle against some 0:00:15.720 --> 0:00:19.680 of humanity's most challenging diseases is happening at the intersection 0:00:19.880 --> 0:00:25.360 of business and medicine. The podcast explores how Vertex Pharmaceuticals, 0:00:25.680 --> 0:00:30.240 a Boston based biotech company, is using innovative tools, methods, 0:00:30.480 --> 0:00:34.880 and a unique philosophy to search for treatments and cures. 0:00:35.280 --> 0:00:39.159 Produced by Bloomberg Media Studios and sponsored by Vertex, the 0:00:39.200 --> 0:00:44.400 podcast's latest episode features NBA great Alonzo Mourning, recounting his 0:00:44.440 --> 0:00:48.040 fight against kidney disease and how scientists are working hard 0:00:48.440 --> 0:00:52.440 to provide hope for future generations of patients. You can 0:00:52.479 --> 0:00:57.240 subscribe today on Apple Podcasts, Spotify, or wherever you listen 0:00:57.280 --> 0:01:05.720 to your favorite podcasts. Here's the full EPISO showed Miami 0:01:05.760 --> 0:01:11.640 Arena March, the Miami Heat are facing the Washington Bullets 0:01:13.959 --> 0:01:16.720 all night long. Alonso Morning of the Heat has been 0:01:16.720 --> 0:01:19.920 guarded by a giant of a man named George Morrisson. 0:01:20.720 --> 0:01:23.720 At seven ft seven. Morrisson is one of the fiercest 0:01:23.760 --> 0:01:26.880 players in the league, the tallest player in NBA history, 0:01:27.280 --> 0:01:34.559 an impenetrable wall with a NonStop motor, but Alonso Morning 0:01:34.840 --> 0:01:36.839 is one of the greatest ever to step on the court. 0:01:40.200 --> 0:01:42.760 When the final buzzer goes, the Heat have won a 0:01:42.840 --> 0:01:45.840 hundred and twelve to ninety three, and Morning has scored 0:01:45.840 --> 0:01:49.680 an incredible fifty points. That game was a career high 0:01:49.760 --> 0:01:52.120 from Morning. He would go on to be a seven 0:01:52.160 --> 0:01:54.560 time All Star and a gold medalist in the two 0:01:54.600 --> 0:01:58.880 thousand Olympics. But little did he know his toughest opponent 0:01:59.120 --> 0:02:03.600 was yet to come. After returning from the Olympics in Sydney, 0:02:03.840 --> 0:02:07.760 Morning started into his usual offseason training, but he noticed 0:02:07.880 --> 0:02:13.400 something was wrong. I was experiencing lethargy, a dima and 0:02:13.520 --> 0:02:20.119 my legs swelling in my lower extremities. Extremely tired, worn out. 0:02:21.040 --> 0:02:24.000 He thought it was jet lag, maybe the flu. He 0:02:24.120 --> 0:02:26.440 figured he'd just take a couple of days off then 0:02:26.520 --> 0:02:30.240 resume his training. His doctor had a different idea. He 0:02:30.360 --> 0:02:33.720 suggested they run some tests. I answered the phone next 0:02:33.720 --> 0:02:36.919 to the bit and he said, you know, I got 0:02:36.960 --> 0:02:42.000 your results back. He said that you've got this rare 0:02:43.280 --> 0:02:48.760 genetic disorder called focal segmental glamorio los carrosis, and I said, Doc, 0:02:48.840 --> 0:02:51.000 what is that? He said, It's a disease that scars 0:02:51.040 --> 0:02:55.040 the filters in the kidney. Focal Segmental glamoryo losclerosis, or 0:02:55.200 --> 0:02:57.840 f s GS caused a scar tissue to develop on 0:02:57.880 --> 0:03:00.040 the small parts of the kidneys that filter waste in 0:03:00.120 --> 0:03:04.400 the blood. I asked them three questions. I said, is 0:03:04.440 --> 0:03:09.760 there a cure forness? He said no. I said, can 0:03:09.800 --> 0:03:15.280 I play basketball again? He said, I don't know? And 0:03:15.320 --> 0:03:26.880 then I said, WHOA am I gonna die? Hi? I'm 0:03:26.960 --> 0:03:29.720 Jordan's gospel A. I'm a member of the University of 0:03:29.760 --> 0:03:33.400 Southern California's Center for Health Journalism. This is Targeting the 0:03:33.400 --> 0:03:37.560 Toughest Diseases a podcast produced by Bloomberg Media Studios and 0:03:37.640 --> 0:03:41.280 Vertex Pharmaceuticals. In this series, we look at some of 0:03:41.360 --> 0:03:45.640 humanity's most challenging diseases and how Vertex, a Boston based 0:03:45.640 --> 0:03:49.760 biotech company, is using innovative tools, methods, and a unique 0:03:49.760 --> 0:03:54.840 philosophy to search for treatments and cures. Today, we're looking 0:03:54.880 --> 0:03:58.280 at APO l one mediated kidney disease, a disease caused 0:03:58.280 --> 0:04:03.080 by a genetic abnormality. One of African Americans carry this 0:04:03.200 --> 0:04:06.320 oftermality can cause a number of types of kidney diseases, 0:04:06.760 --> 0:04:09.880 one of which is f SGS, the condition Alonso Morning 0:04:10.000 --> 0:04:17.560 was diagnosed with. I thought to myself, not why me, 0:04:18.480 --> 0:04:22.279 but why right now? God? Why am I dealing with 0:04:22.320 --> 0:04:25.360 this right now of all times. I had just come 0:04:25.400 --> 0:04:28.599 back from the Sydney, Australia Olympics winning the gold medal, 0:04:29.000 --> 0:04:31.719 had just come off an amazing basketball season where I 0:04:31.760 --> 0:04:36.000 had First Team All NBA All Star Team. The list 0:04:36.080 --> 0:04:40.640 goes on, you know, all the accolades, and then all 0:04:40.640 --> 0:04:43.880 of a sudden, bam. This happens. Lots of young professional 0:04:43.920 --> 0:04:49.000 athletes view themselves as invincible. Alonso suddenly found out he wasn't. 0:04:49.440 --> 0:04:52.320 All of the phone and my face fell in my hands. 0:04:54.480 --> 0:04:56.160 I felt like I was gonna play until I was 0:04:56.200 --> 0:04:58.520 in my forties because I was in such great shape. 0:04:58.960 --> 0:05:02.400 Just airing from somebody saying hey, you gotta stop playing. 0:05:02.400 --> 0:05:05.839 It was humbling and he was deflating, you know, and 0:05:05.920 --> 0:05:09.280 I just was just sitting there just trying to figure out, Okay, 0:05:09.600 --> 0:05:17.279 how is this all going to materialize? We can't survive 0:05:17.320 --> 0:05:20.839 without our kidneys. They play a vital role. They help 0:05:20.880 --> 0:05:25.400 our bodies maintain just the right balance of electrolytes like potassium, 0:05:25.440 --> 0:05:29.240 They control blood pressure, the cleaner blood, and they even 0:05:29.240 --> 0:05:32.800 help maintain our hormone levels. Each kidney is made up 0:05:32.880 --> 0:05:36.120 of a million or so tiny filters called gloomy uli 0:05:36.839 --> 0:05:40.520 They're like little coffee filters. The filtered liquid becomes urine 0:05:40.800 --> 0:05:43.279 and the protein left behind stays in our blood. But 0:05:43.320 --> 0:05:47.120 when the gloomeryulie become damaged, those proteins start leaking into 0:05:47.200 --> 0:05:50.600 the urine. The scary thing is it's estimated thirty seven 0:05:50.640 --> 0:05:53.560 million adults in the United States have kidney disease, and 0:05:53.800 --> 0:05:56.040 ninety percent of them don't even know they have it. 0:05:56.680 --> 0:05:58.760 And in the case of a po l one mediated 0:05:58.800 --> 0:06:03.520 kidney disease, the prime causal factor is invisible. It's genetic 0:06:04.000 --> 0:06:10.920 caused re mutations in the a po l one gene. 0:06:11.880 --> 0:06:14.960 Back in the early nineteen nineties, Dr David Friedman, a 0:06:15.040 --> 0:06:18.719 doctor researcher and an associate professor with Harvard Medical School 0:06:18.800 --> 0:06:21.800 who currently works with Vertex on its clinical trials, first 0:06:21.800 --> 0:06:25.200 started to notice something unusual. He was seeing African American 0:06:25.240 --> 0:06:29.880 families where multiple members all had kidney disease. When there's 0:06:29.880 --> 0:06:33.279 an important inherited component of a disease, it tends to 0:06:33.320 --> 0:06:37.240 cluster in families. Understanding there was a genetic cause was 0:06:37.360 --> 0:06:40.560 just the beginning. Next, they had to find the exact gene. 0:06:42.960 --> 0:06:46.159 I think a real breakthrough in our understanding came in 0:06:46.240 --> 0:06:49.440 two thousand and eight when some teams at Johns Hopkins 0:06:49.480 --> 0:06:52.520 in the n i H were the first to find 0:06:52.760 --> 0:06:56.200 a location in the geno my chromosome twenty two where 0:06:56.520 --> 0:07:00.120 it became apparent that there was something strong that was 0:07:00.120 --> 0:07:04.920 impacting kidney disease in people of African ancestry. Then Dr 0:07:05.000 --> 0:07:09.120 Friedman and his colleagues identified the specific mutations that led 0:07:09.160 --> 0:07:12.160 to this type of kidney disease. There were two important 0:07:12.280 --> 0:07:18.000 advances in technology which really helped us to pinpoint these 0:07:18.040 --> 0:07:22.400 two genetic variants in April one. The first was related 0:07:22.520 --> 0:07:27.920 to tools for identifying positive selection in the genome, and 0:07:28.040 --> 0:07:33.240 these these mathematical tools helped us envisioned the genome in 0:07:33.280 --> 0:07:37.440 a slightly different way. In the second major technological advance 0:07:38.160 --> 0:07:43.800 was a database of genetic variants in people of widely 0:07:43.880 --> 0:07:47.440 diverse ancestry, is called the Thousand Genomes project. Up until 0:07:47.480 --> 0:07:50.960 that time, most of what we knew about genetic variation 0:07:51.440 --> 0:07:56.560 came from people of European ancestry, and this new, very 0:07:56.600 --> 0:08:01.440 powerful tool was a really equivalent of an cyclopedia of 0:08:01.720 --> 0:08:06.040 genetic variation around the world. From there, our understanding of 0:08:06.080 --> 0:08:10.640 apoel one has continued to increase, including why the risk 0:08:10.760 --> 0:08:14.640 variants in this gene only affect people of recent African descent, 0:08:14.880 --> 0:08:21.720 including African American, Latin X and Afro Caribbean communities. Thousands 0:08:21.720 --> 0:08:24.680 of years ago, a genetic mutation in the apoel one 0:08:24.720 --> 0:08:29.200 gene developed in sub Saharan Africa as a protective mechanism. 0:08:29.280 --> 0:08:33.200 We all have a gene that encodes for the april 0:08:33.240 --> 0:08:37.720 one protein, but the version which causes kidney disease contains 0:08:37.800 --> 0:08:42.440 some very slight changes in the instructions for building that 0:08:42.559 --> 0:08:47.360 protein that turn out to make it very effective for 0:08:47.640 --> 0:08:52.400 killing the trepanisomes that cause African sleeping sickness and humans. 0:08:53.040 --> 0:08:55.800 Sleeping sickness is a disease spread by the bite of 0:08:55.800 --> 0:08:58.800 an infected fly, and it can cause death within a 0:08:58.840 --> 0:09:02.040 matter of weeks. But this genetic mutation stops it in 0:09:02.120 --> 0:09:06.319 its tracks. Because it was so effective. It's spread very 0:09:06.360 --> 0:09:10.480 quickly in the population, but it's these proteins with a 0:09:10.679 --> 0:09:13.920 slightly different version of april one. It's very very effective 0:09:14.000 --> 0:09:18.240 for killing japanizomes and preventing African sleeping sickness, which is 0:09:18.280 --> 0:09:23.160 the same version of the protein which makes the kidneys 0:09:23.160 --> 0:09:27.360 of people sick who have this genetic variant. We now 0:09:27.440 --> 0:09:30.520 understand that having one a poel one risk variant could 0:09:30.559 --> 0:09:33.120 protect you from many forms of sleeping sickness. But if 0:09:33.200 --> 0:09:36.520 you were unlucky enough to inherit to one from your 0:09:36.559 --> 0:09:39.319 mother and one from your father, your chances of getting 0:09:39.360 --> 0:09:56.360 kidney disease goes up tenfold dore once your kidneys start 0:09:56.440 --> 0:09:58.880 to fail, there really are only a few ways to 0:09:58.920 --> 0:10:02.960 stay alive. One is dialysis. It replaces the function of 0:10:03.000 --> 0:10:07.000 the kidney. Dr Janice Lee studies kidney disorders at Emory 0:10:07.120 --> 0:10:12.679 University School of Medicine in Atlanta, Georgia. Kidneys excrete waste products, 0:10:13.080 --> 0:10:17.200 and they get rid of excess fluid from our bodies, 0:10:17.840 --> 0:10:21.640 so that's what the dialysis machine does. Two needles are 0:10:21.640 --> 0:10:24.720 inserted into a patient's arm. One draws blood out and 0:10:24.760 --> 0:10:27.360 sends it through the machine to be cleaned. The other 0:10:27.400 --> 0:10:30.720 needle returns the clean blood back into the patient, which 0:10:30.760 --> 0:10:33.920 is pretty much exactly the way our kidneys work, except 0:10:34.120 --> 0:10:36.680 while your kidneys work slowly, twenty four hours a day, 0:10:36.760 --> 0:10:40.000 seven days a week. Dialysis means sitting in a chair 0:10:40.320 --> 0:10:42.600 plugged into a machine for two to three hours at 0:10:42.640 --> 0:10:46.400 a time, three times a week. It works, but it's 0:10:46.400 --> 0:10:50.439 not ideal if you really think about it. We go 0:10:50.600 --> 0:10:54.280 to the bathroom excrete our urine two or three four 0:10:54.320 --> 0:10:59.000 times or more a day, so when patients are on dialysis, 0:10:59.000 --> 0:11:03.080 they're really getting their blood clans just three days a 0:11:03.160 --> 0:11:07.640 week for a few hours. And so patients can feel 0:11:08.160 --> 0:11:11.600 washed out when they get off of dialysis because they've 0:11:11.600 --> 0:11:14.679 had all this fluid from two days worth of not 0:11:15.040 --> 0:11:20.320 excreting any waste products or fluid. There's no cure available 0:11:20.360 --> 0:11:23.840 for chronic kidney disease. The medications currently on the market 0:11:24.000 --> 0:11:28.000 focus on making sure a person's kidneys don't deteriorate further. 0:11:28.600 --> 0:11:33.559 The current options include medications that suppress the immune system, diuretics, 0:11:33.720 --> 0:11:37.400 ace inhibitors or a r B medications to control blood pressure, 0:11:37.480 --> 0:11:41.800 or lower urine protein anticoagulants to prevent blood clots. And 0:11:41.840 --> 0:11:44.440 then there's the option. Alonso Morning had done for his 0:11:44.640 --> 0:11:48.640 f s GS a kidney transplant that approach requires you 0:11:48.720 --> 0:11:52.120 to be fortunate enough to find a suitable donor. On average, 0:11:52.280 --> 0:11:58.600 it also only last ten to twelve years. Vertex Pharmaceuticals 0:11:58.679 --> 0:12:00.839 is a company that has researched ing a poll one 0:12:00.960 --> 0:12:04.360 mediated kidney disease and other tough diseases where there's a 0:12:04.480 --> 0:12:07.880 huge unmet need. They are targeting conditions where the human 0:12:07.880 --> 0:12:11.840 biology is understood, the technology already exists, or Vertex thinks 0:12:11.840 --> 0:12:14.280 that can develop it, and where Vertex has an approach 0:12:14.440 --> 0:12:18.000 they think maybe transformative. They have several programs in their 0:12:18.000 --> 0:12:25.120 investigational research, including a poll one mediated kidney disease. Dr 0:12:25.160 --> 0:12:28.440 Ogo ed Buna leads clinical development for the team researching 0:12:28.520 --> 0:12:31.680 kidney disease at Vertex. As we speak here in the US, 0:12:31.760 --> 0:12:35.320 there are probably more than a hundred thousand people waiting 0:12:35.360 --> 0:12:38.840 for a kidney, and not everyone is fortunate to be 0:12:38.880 --> 0:12:41.440 able to get one. Many people will die waiting for 0:12:41.520 --> 0:12:44.400 a kidney. The staggering number of people living with and 0:12:44.480 --> 0:12:46.840 dying from kidney disease is one of the main reasons 0:12:46.840 --> 0:12:50.800 why Dr ed Buna joined Vertexas efforts. It was really heartbreaking, 0:12:51.120 --> 0:12:53.480 you know, at the beginning of the year, I'd have 0:12:54.080 --> 0:12:57.319 a whole host of patients on theiralysis and at the 0:12:57.440 --> 0:13:00.720 end of the year, one or two of every three 0:13:01.080 --> 0:13:05.680 would have you know, costa. That was just too depressing 0:13:05.760 --> 0:13:09.520 for me. Why did Vertex choose to focus on a 0:13:09.679 --> 0:13:14.040 pole one versus other types of kidney disease? This is 0:13:14.080 --> 0:13:18.840 actually one of the most difficult kidney diseases that have 0:13:19.200 --> 0:13:24.959 lagged a minority population and underserved population for so long, 0:13:25.080 --> 0:13:29.360 and for the longest time we have attributed this to 0:13:29.920 --> 0:13:33.920 either bad diet or lack of exercise or nutrition. But 0:13:34.040 --> 0:13:38.320 we do know now that there is a genetic basis 0:13:38.400 --> 0:13:40.960 for a lot of this disparity. And I think in 0:13:41.320 --> 0:13:45.800 typical Vertex fashion, we go after diseases that have a 0:13:45.880 --> 0:13:49.200 serious and met need, and in addition to that, we 0:13:49.280 --> 0:13:54.080 go after diseases where the underlying cause is well understood 0:13:54.280 --> 0:13:57.920 and for which we apply, you know, the best science 0:13:57.920 --> 0:14:00.000 available to try and address it. What can we look 0:14:00.080 --> 0:14:02.560 forward to. Part of the reason why I'm actually so 0:14:02.600 --> 0:14:05.400 excited about what we're doing here at Vertex is because 0:14:05.640 --> 0:14:11.200 we have found small molecule therapies that are investigational to 0:14:11.360 --> 0:14:14.640 the underlying cause of what I've described as april and 0:14:14.679 --> 0:14:19.200 one media to kidney disease. We have evaluated these potential 0:14:19.240 --> 0:14:23.760 therapies in experimental settings and first in human studies as 0:14:23.800 --> 0:14:27.680 well as a preliminary proof of concept study in patients. 0:14:29.920 --> 0:14:33.320 Developing a potential small molecule therapy is no easy feat. 0:14:33.720 --> 0:14:36.640 Dr ed Buna says scientists a Vertex have gone through 0:14:36.800 --> 0:14:41.000 hundreds of thousands of candidate molecules and their kidney disease research. 0:14:41.960 --> 0:14:46.320 Nothing good or great comes easy. Therein lies the promise 0:14:46.400 --> 0:14:50.560 and the excitement well the research continues. Dr ed Bunah 0:14:50.600 --> 0:14:53.200 says he and his team at Vertex will do everything 0:14:53.240 --> 0:14:56.240 they can to raise awareness for the disease. That includes 0:14:56.360 --> 0:14:59.880 educating on preventative measures like genotyping, which can help the 0:15:00.040 --> 0:15:04.120 ternaman whether a patient carries the A L one genetic variant. So, 0:15:04.160 --> 0:15:08.000 in addition to supporting the clinical community and patients in 0:15:08.000 --> 0:15:11.160 increasing the rates of diagnosis of kidney disease, we also 0:15:11.240 --> 0:15:16.040 want an increase in the awareness and of genotyping so 0:15:16.080 --> 0:15:19.040 that precise diagnosis can be made so that the right 0:15:19.120 --> 0:15:22.520 therapy can be brought to the patient. Alonso Morning is 0:15:22.560 --> 0:15:25.720 one of those great examples of people that went through 0:15:26.120 --> 0:15:30.640 the science symptoms, the worries, the challenges of stage advanced 0:15:30.720 --> 0:15:33.960 kidney disease, got a transplant, and he got back on 0:15:34.040 --> 0:15:41.040 his feet. Alonso Morning underwent a kidney transplant three years 0:15:41.080 --> 0:15:46.040 after his initial diagnosis. So the recovery process was grueling 0:15:46.720 --> 0:15:50.800 and it was extremely painful at times, and it was difficult. 0:15:51.320 --> 0:15:53.880 But if you think about anything in life worth having, 0:15:54.440 --> 0:15:57.160 is very difficult to get it. And I was trying 0:15:57.200 --> 0:15:58.840 to get my help back, and I was trying to 0:15:58.880 --> 0:16:01.440 get back on the court, So I was trying to 0:16:01.480 --> 0:16:04.400 get back to some sense of normalcy. But it challenged 0:16:04.440 --> 0:16:09.040 me tremendously, and it really truly challenged me. Morning talks 0:16:09.080 --> 0:16:11.520 about his struggle to get and then adapt to living 0:16:11.520 --> 0:16:14.320 with a new kidney, knowing full well that he had 0:16:14.320 --> 0:16:19.320 a lot of advantages that was in optimal condition, excellent health, 0:16:19.400 --> 0:16:23.720 you know. But that was the benefit of my recovery 0:16:24.440 --> 0:16:28.360 so fast, because the doctors told me, like, hey, you know, 0:16:28.680 --> 0:16:33.280 if you wasn't his high performance athlete, then you probably 0:16:33.320 --> 0:16:37.200 wouldn't have bounced back as fast after the transplant, and 0:16:37.240 --> 0:16:40.840 bounce back he did just three years after his transplant 0:16:40.880 --> 0:16:43.480 alongso Morning would go on and win a championship with 0:16:43.520 --> 0:16:46.720 the Miami Heat in two thousand six, and today he's 0:16:46.760 --> 0:16:50.320 partnering with Vertex as a spokesperson, using his profile and 0:16:50.360 --> 0:16:54.040 his experience to advocate and educate. You know, a big 0:16:54.080 --> 0:16:57.920 part of it is to try to inspire and provide 0:16:57.960 --> 0:17:01.800 hope and encouragement for those who are going through the 0:17:01.840 --> 0:17:05.399 same ordeal. And Morning says he's hoping Vertex will be 0:17:05.440 --> 0:17:08.560 able to help kidney patients by raising awareness of the disease, 0:17:08.880 --> 0:17:12.280 encouraging people to visit their doctor, and continuing to search 0:17:12.400 --> 0:17:15.960 for a potential treatment. So if this can happen, then 0:17:16.680 --> 0:17:20.600 all of what I've gone through is so much more 0:17:20.640 --> 0:17:26.000 of a worthwhile because maybe to help save other people's lives. 0:17:36.520 --> 0:17:39.640 This is targeting the toughest diseases. A podcast from Bloomberg 0:17:39.640 --> 0:17:43.400 Media Studios and Vertex Pharmaceuticals. If you like what you hear, 0:17:43.720 --> 0:17:47.320 subscribe and leave us a review. I'm Jordan's Gospel. Thanks 0:17:47.400 --> 0:17:47.919 for listening.