WEBVTT - Targeting the Toughest Diseases (Sponsored Content) 0:00:00.640 --> 0:00:04.320 I'm Edward Adams of Bloomberg Media Studios. Since you're a 0:00:04.400 --> 0:00:08.039 subscriber to Bloomberg Prognosis, we thought you'd be interested in 0:00:08.080 --> 0:00:12.640 a new six episodes sponsored content podcast called Targeting the 0:00:12.680 --> 0:00:16.200 Toughest Diseases. It shows how the battle against some of 0:00:16.320 --> 0:00:20.640 humanity's most challenging diseases is happening at the intersection of 0:00:20.680 --> 0:00:26.200 business and medicine. The podcast explores how Vertex Pharmaceuticals, a 0:00:26.280 --> 0:00:31.159 Boston based biotech company, is using innovative tools, methods, and 0:00:31.240 --> 0:00:36.199 a unique philosophy to search for treatments and cures. Produced 0:00:36.200 --> 0:00:40.440 by Bloomberg Media Studios and sponsored by Vertex, the podcast's 0:00:40.520 --> 0:00:44.960 latest episode features nb a great Alonzo Mourning, recounting his 0:00:45.040 --> 0:00:48.720 fight against kidney disease and how scientists are working hard 0:00:49.120 --> 0:00:53.639 to provide hope for future generations of patients. You can 0:00:53.640 --> 0:00:58.000 subscribe today on Apple Podcasts, Spotify, or wherever you listen 0:00:58.040 --> 0:01:06.320 to your favorite podcasts. Here is the full episode. Miami 0:01:06.400 --> 0:01:12.280 Arena March. The Miami Heat are facing the Washington Bullets 0:01:14.600 --> 0:01:17.319 all night long. Alonso Morning of the Heat has been 0:01:17.360 --> 0:01:20.560 guarded by a giant of a man named George Morrisson 0:01:21.319 --> 0:01:24.360 at seven ft seven. Morrisson is one of the fiercest 0:01:24.360 --> 0:01:27.479 players in the league, the tallest player in NBA history, 0:01:27.920 --> 0:01:35.199 an impenetrable wall with a NonStop motor, but Alonso Morning 0:01:35.440 --> 0:01:37.480 is one of the greatest ever to step on the court. 0:01:40.800 --> 0:01:43.360 When the final buzzer goes, the Heat have won a 0:01:43.480 --> 0:01:46.440 hundred and twelve to ninety three, and Morning has scored 0:01:46.480 --> 0:01:50.360 an incredible fifty points. That game was a career high 0:01:50.400 --> 0:01:52.760 from Morning. He would go on to be a seven 0:01:52.800 --> 0:01:55.160 time All Star and a gold medalist in the two 0:01:55.200 --> 0:01:59.520 thousand Olympics. But little did he know his toughest opponent 0:01:59.680 --> 0:02:04.240 was to come. After returning from the Olympics in Sydney, 0:02:04.480 --> 0:02:08.400 Morning started into his usual offseason training, but he noticed 0:02:08.480 --> 0:02:14.040 something was wrong. I was experiencing lethargy, a demon in 0:02:14.120 --> 0:02:20.760 my legs, swelling in my lower extremities. Extremely tired, worn out. 0:02:21.680 --> 0:02:24.600 He thought it was jet lag, maybe the flu. He 0:02:24.720 --> 0:02:27.079 figured he'd just take a couple of days off then 0:02:27.160 --> 0:02:30.840 resume his training. His doctor had a different idea. He 0:02:30.960 --> 0:02:34.360 suggested they run some tests. I answered the phone next 0:02:34.360 --> 0:02:37.560 to the bit and he said, you know, I got 0:02:37.560 --> 0:02:42.640 your results back. He said that you've got this rare 0:02:43.919 --> 0:02:49.399 genetic disorder called focal segmental glamorio los carrosis, and I said, Doc, 0:02:49.440 --> 0:02:51.640 what is that? He said, It's a disease that cards 0:02:51.680 --> 0:02:55.520 the filters in the kidney. Vocal Segmental glamoryo los clerosis 0:02:55.600 --> 0:02:58.320 or f s GS caused a scar tissue to develop 0:02:58.400 --> 0:03:00.600 on the small parts of the kidneys that alter waste 0:03:00.600 --> 0:03:04.200 from the blood. I asked them three questions. I said, 0:03:04.960 --> 0:03:10.360 is there a cupidis He said no. I said, can 0:03:10.400 --> 0:03:15.919 I play basketball again? He said, I don't know? And 0:03:15.960 --> 0:03:27.480 then I said, well, am I gonna die? Hi? I'm 0:03:27.560 --> 0:03:30.360 Jordan's Gospel A. I'm a member of the University of 0:03:30.360 --> 0:03:34.000 Southern California's Center for Health Journalism. This is Targeting the 0:03:34.040 --> 0:03:38.160 Toughest Diseases a podcast produced by Bloomberg Media Studios and 0:03:38.280 --> 0:03:41.920 Vertex Pharmaceuticals. In this series, we look at some of 0:03:42.000 --> 0:03:46.280 humanity's most challenging diseases and how Vertex, a Boston based 0:03:46.280 --> 0:03:50.360 biotech company, is using innovative tools, methods, and a unique 0:03:50.360 --> 0:03:55.440 philosophy to search for treatments and cures. Today, we're looking 0:03:55.520 --> 0:03:58.480 at a po L one mediated kidney disease, a disease 0:03:58.560 --> 0:04:03.480 caused by a genetic abnormal one of African Americans carry 0:04:03.680 --> 0:04:06.960 this aftermality can cause a number of types of kidney diseases, 0:04:07.360 --> 0:04:10.560 one of which is f SGS, the condition Alonso Morning 0:04:10.600 --> 0:04:18.200 was diagnosed with. I thought to myself, not why me, 0:04:19.120 --> 0:04:22.880 but why right now? God? Why am I dealing with 0:04:22.920 --> 0:04:26.000 this right now of all times. I had just come 0:04:26.040 --> 0:04:29.200 back from the Sydney, Australia Olympics winning the gold medal, 0:04:29.600 --> 0:04:32.359 had just come off an amazing basketball season where I 0:04:32.360 --> 0:04:36.640 had First Team All NBA All Star Team. The list 0:04:36.680 --> 0:04:41.240 goes on, you know, all the accolades, and then all 0:04:41.279 --> 0:04:44.520 of a sudden, bam, this happens. Lots of young professional 0:04:44.560 --> 0:04:49.599 athletes view themselves as invincible. Alonso suddenly found out he wasn't. 0:04:50.080 --> 0:04:52.960 All of the phone and my face fell in my hands. 0:04:55.120 --> 0:04:56.800 I felt like I was gonna play until I was 0:04:56.839 --> 0:04:59.159 in my forties because I was in such great shape. 0:04:59.560 --> 0:05:02.719 Just airing it from somebody saying hey, you gotta stop 0:05:02.720 --> 0:05:06.479 playing it was humbling. He was deflating, you know, and 0:05:06.560 --> 0:05:09.880 I just was just sitting there just trying to figure out, Okay, 0:05:10.240 --> 0:05:17.920 how is this all going to materialize. We can't survive 0:05:17.960 --> 0:05:21.440 without our kidneys. They play a vital role. They help 0:05:21.480 --> 0:05:26.000 our bodies maintain just the right balance of electrolytes like potassium, 0:05:26.040 --> 0:05:29.839 They control blood pressure, the cleaner blood, and they even 0:05:29.880 --> 0:05:33.480 help maintain our hormone levels. Each kidney is made up 0:05:33.520 --> 0:05:36.760 of a million or so tiny filters called gloomy uli. 0:05:37.440 --> 0:05:41.080 They're like little coffee filters. The filtered liquid becomes urine 0:05:41.440 --> 0:05:43.880 and the protein left behind stays in our blood. But 0:05:43.920 --> 0:05:47.800 when the glomerulie become damaged, those proteins start leaking into 0:05:47.800 --> 0:05:51.239 the urine. The scary thing is it's estimated thirty seven 0:05:51.240 --> 0:05:54.200 million adults in the United States have kidney disease, and 0:05:54.400 --> 0:05:57.359 nine of them don't even know they have it. And 0:05:57.400 --> 0:05:59.719 in the case of a poe l one mediated kidney 0:05:59.720 --> 0:06:04.120 disease is the prime causal factor is invisible. It's genetic 0:06:04.600 --> 0:06:12.719 caused by mutations in the APO l one gene. Back 0:06:12.720 --> 0:06:16.160 in the early nineteen nineties, Dr David Friedman, a doctor, 0:06:16.240 --> 0:06:19.560 researcher and an associate professor with Harvard Medical School who 0:06:19.640 --> 0:06:22.760 currently works with Vertex on its clinical trials, first started 0:06:22.800 --> 0:06:26.320 to notice something unusual. He was seeing African American families 0:06:26.400 --> 0:06:30.640 where multiple members all had kidney disease. When there's an 0:06:30.640 --> 0:06:34.320 important inherited component of a disease, it tends to cluster 0:06:34.480 --> 0:06:38.080 in families. Understanding there was a genetic cause was just 0:06:38.240 --> 0:06:41.159 the beginning. Next, they had to find the exact gene. 0:06:43.600 --> 0:06:46.800 I think a real breakthrough in our understanding came in 0:06:46.839 --> 0:06:50.120 two thousand and eight when some teams at Johns Hopkins 0:06:50.120 --> 0:06:53.120 in the n i H were the first to find 0:06:53.400 --> 0:06:56.839 a location in the geno my chromosome twenty two where 0:06:57.120 --> 0:07:00.480 it became apparent that there was something strang on that 0:07:00.600 --> 0:07:04.160 was impacting kidney disease in people of African ancestry. Then 0:07:05.279 --> 0:07:09.440 Dr Friedman and his colleagues identified the specific mutations that 0:07:09.560 --> 0:07:12.160 led to this type of kidney disease. There were two 0:07:12.200 --> 0:07:18.320 important advances in technology which really helped us to pinpoint 0:07:18.360 --> 0:07:22.480 these two genetic variants in April one. The first was 0:07:22.560 --> 0:07:28.080 related to tools for identifying positive selection in the genome, 0:07:28.320 --> 0:07:33.760 and these these mathematical tools helped us envisioned the genome 0:07:33.800 --> 0:07:37.520 in a slightly different way. In the second major technological 0:07:37.560 --> 0:07:43.760 advance was a database of genetic variants in people of 0:07:43.960 --> 0:07:47.680 widely diverse ancestry, is called the Thousand Genomes Project. Up 0:07:47.760 --> 0:07:50.960 until that time, most of what we knew about genetic 0:07:51.040 --> 0:07:56.600 variation came from people of European ancestry, and this new, 0:07:57.000 --> 0:08:01.840 very powerful tool was a really equivalent of an encyclopedia 0:08:02.000 --> 0:08:06.560 of genetic variation around the world. From there, our understanding 0:08:06.560 --> 0:08:10.960 of apoel one has continued to increase, including why the 0:08:11.040 --> 0:08:14.400 risk variants in this gene only affect people of recent 0:08:14.440 --> 0:08:19.800 African descent, including African American, Latin X and Afro Caribbean communities. 0:08:21.800 --> 0:08:25.040 Thousands of years ago, a genetic mutation in the apoel 0:08:25.120 --> 0:08:29.840 one gene developed in sub Saharan Africa as a protective mechanism. 0:08:29.920 --> 0:08:33.840 We all have a gene that encodes for the april 0:08:33.880 --> 0:08:38.400 one protein, but the version which causes kidney disease contains 0:08:38.400 --> 0:08:43.080 some very slight changes in the instructions for building that 0:08:43.160 --> 0:08:47.960 protein that turn out to make it very effective for 0:08:48.280 --> 0:08:53.040 killing the trepanisomes that cause African sleeping sickness and humans. 0:08:53.679 --> 0:08:56.400 Sleeping sickness is a disease spread by the bite of 0:08:56.440 --> 0:08:59.400 an infected fly, and it can cause death within a 0:08:59.440 --> 0:09:02.680 matter of weeks. But this genetic mutation stops it in 0:09:02.720 --> 0:09:06.920 its tracks. Because it was so effective. It's spread very 0:09:07.000 --> 0:09:11.120 quickly in the population, but it's these proteins with a 0:09:11.320 --> 0:09:14.000 slightly different version of eight pl one. It's very very 0:09:14.040 --> 0:09:18.720 effective for killing japanizomes and preventing African sleeping sickness, which 0:09:18.760 --> 0:09:23.280 is the same version of the protein which makes the 0:09:23.360 --> 0:09:27.760 kidneys of people sick who have this genetic variant. We 0:09:27.800 --> 0:09:31.200 now understand that having one apoel one risk variant could 0:09:31.200 --> 0:09:33.800 protect you from many forms of sleeping sickness. But if 0:09:33.840 --> 0:09:37.160 you were unlucky enough to inherit too, one from your 0:09:37.160 --> 0:09:39.920 mother and one from your father, your chances of getting 0:09:40.000 --> 0:09:57.000 kidney disease goes up tenfold fire Once your kidneys start 0:09:57.040 --> 0:09:59.480 to fail. There really are only a few ways to 0:09:59.520 --> 0:10:02.960 stay a Lot five. One is dialysis. It replaces the 0:10:02.960 --> 0:10:07.040 function of the kidney. Dr Janice Lee studies kidney disorders 0:10:07.080 --> 0:10:11.400 at Emory University School of Medicine in Atlanta, Georgia. Kidneys 0:10:11.760 --> 0:10:16.360 excrete waste products, and they get rid of excess fluid 0:10:16.920 --> 0:10:20.760 from our bodies, so that's what the dialysis machine does. 0:10:21.480 --> 0:10:24.720 Two needles are inserted into a patient's arm. One draws 0:10:24.720 --> 0:10:27.040 blood out and sends it through the machine to be cleaned. 0:10:27.640 --> 0:10:30.520 The other needle returns the clean blood back into the patient, 0:10:31.120 --> 0:10:33.480 which is pretty much exactly the way our kidneys work. 0:10:34.040 --> 0:10:37.320 Except while your kidneys work slowly. Twenty four hours a day, 0:10:37.360 --> 0:10:40.640 seven days a week. Dialysis means sitting in a chair 0:10:40.960 --> 0:10:43.240 plugged into a machine for two to three hours at 0:10:43.240 --> 0:10:47.000 a time, three times a week. It works, but it's 0:10:47.040 --> 0:10:51.079 not ideal if you really think about it. We go 0:10:51.200 --> 0:10:54.880 to the bathroom excrete our urine two or three four 0:10:54.960 --> 0:10:59.600 times or more a day, so when patients are on dialysis, 0:10:59.640 --> 0:11:04.079 there getting their blood clans just three days a week 0:11:04.520 --> 0:11:09.240 for a few hours, and so patients can feel washed 0:11:09.280 --> 0:11:12.480 out when they get off of dialysis because they've had 0:11:12.559 --> 0:11:16.319 all this fluid from two days worth of not excreting 0:11:16.440 --> 0:11:21.040 any waste products or fluid. There's no cure available for 0:11:21.120 --> 0:11:25.040 chronic kidney disease. The medications currently on the market focus 0:11:25.120 --> 0:11:29.320 on making sure a person's kidneys don't deteriorate further. The 0:11:29.360 --> 0:11:34.280 current options include medications that suppress the immune system, diuretics, 0:11:34.320 --> 0:11:38.000 ace inhibitors or a r B medications to control blood pressure, 0:11:38.080 --> 0:11:42.440 or lower urine protein anticoagulants to prevent blood clots. And 0:11:42.480 --> 0:11:45.080 then there's the option Alonso Morning had done for his 0:11:45.240 --> 0:11:49.280 f s GS a kidney transplant. That approach requires you 0:11:49.360 --> 0:11:52.760 to be fortunate enough to find a suitable donor. On average, 0:11:52.880 --> 0:11:59.160 it also only last ten to twelve years. Vertex Pharmaceuticals 0:11:59.280 --> 0:12:02.000 is a company that is researching a poll one mediated 0:12:02.040 --> 0:12:05.360 kidney disease and other tough diseases where there's a huge 0:12:05.480 --> 0:12:09.040 unmet need. They are targeting conditions where the human biology 0:12:09.120 --> 0:12:12.520 is understood, the technology already exists, or Vertex thinks that 0:12:12.559 --> 0:12:15.200 can develop it, and where Vertex has an approach they 0:12:15.240 --> 0:12:19.800 think maybe transformative. They have several programs in their investigational research, 0:12:20.080 --> 0:12:26.320 including a poll one mediated kidney disease. Dr Ogo ed 0:12:26.360 --> 0:12:29.800 Buna leads clinical development for the team researching kidney disease 0:12:29.840 --> 0:12:32.520 at Vertex. As we speak here in the US, there 0:12:32.520 --> 0:12:36.120 are probably more than a hundred thousand people waiting for 0:12:36.160 --> 0:12:39.680 a kidney, and not everyone is fortunate to be able 0:12:39.720 --> 0:12:42.840 to get one. Many people will die waiting for a kidney. 0:12:43.040 --> 0:12:45.520 The staggering number of people living with and dying from 0:12:45.600 --> 0:12:47.920 kidney disease is one of the main reasons why Dr 0:12:48.040 --> 0:12:52.000 ed Buna joined Vertexas efforts. It was really heartbreaking, you know, 0:12:52.080 --> 0:12:55.040 at the beginning of the year, I'd have a whole 0:12:55.040 --> 0:12:58.280 host of patients on theiralysis and at the end of 0:12:58.320 --> 0:13:01.960 the year one or two out of every three would 0:13:01.960 --> 0:13:06.640 have you know, costa. That was just too depressing for me. 0:13:07.240 --> 0:13:10.840 Why did Vertex choose to focus on a pole one 0:13:11.240 --> 0:13:15.560 versus other types of kidney disease? This is actually one 0:13:15.720 --> 0:13:21.160 of the most difficult kidney diseases that have lagged a 0:13:21.200 --> 0:13:26.000 minority population or underserved population for so long, and for 0:13:26.040 --> 0:13:31.240 the longest time, we have attributed this to either bad 0:13:31.320 --> 0:13:34.960 diet or lack of exercise or nutrition. But we do 0:13:35.040 --> 0:13:39.240 know now that there is a genetic basis for a 0:13:39.240 --> 0:13:43.560 lot of this disparity. And I think in typical Vertex fashion, 0:13:43.960 --> 0:13:47.560 we go after diseases that have a serious and met need, 0:13:48.080 --> 0:13:51.400 and in addition to that, we go after diseases where 0:13:51.720 --> 0:13:57.199 the underlying cause is well understood and for which we apply, 0:13:57.679 --> 0:13:59.880 you know, the best science available to try and address it. 0:14:00.120 --> 0:14:02.320 What can we look forward to. Part of the reason 0:14:02.360 --> 0:14:04.800 why I'm actually so excited about what we're doing here 0:14:04.800 --> 0:14:09.959 had Vertex, is because we have found small molecule therapies 0:14:10.280 --> 0:14:14.319 that are investigational to the underlying cause of what I've 0:14:14.360 --> 0:14:17.559 described as april one media to kidney disease. We have 0:14:18.160 --> 0:14:22.800 evaluated these potential therapies in experimental settings and first in 0:14:22.880 --> 0:14:27.160 human studies, as well as a preliminary proof of concept 0:14:27.160 --> 0:14:33.120 study in patients. Developing a potential small molecule therapy is 0:14:33.120 --> 0:14:36.600 no easy feat. Dr ed Buna says scientists a Vertex 0:14:36.600 --> 0:14:40.280 have gone through hundreds of thousands of candidate molecules and 0:14:40.280 --> 0:14:44.960 their kidney disease research. Nothing good or great comes easy. 0:14:45.240 --> 0:14:49.120 There in lies the promise and the excitement well the 0:14:49.240 --> 0:14:52.200 research continues. Dr ed Bunah says he and his team 0:14:52.200 --> 0:14:55.160 at Vertex will do everything they can to raise awareness 0:14:55.200 --> 0:14:59.680 for the disease. That includes educating on preventative measures like genotyping. 0:15:00.000 --> 0:15:02.280 You can help determine whether a patient carries the A 0:15:02.640 --> 0:15:06.680 L one genetic variant. So, in addition to supporting the 0:15:06.680 --> 0:15:10.600 clinical community and patients in increasing the rates of diagnosis 0:15:10.600 --> 0:15:13.360 of kidney disease, we also want an increase in the 0:15:13.440 --> 0:15:18.680 awareness and of genotyping so that precise diagnosis can be 0:15:18.720 --> 0:15:21.680 made so that the right therapy come brought to the patient. 0:15:22.320 --> 0:15:24.840 Alons of Morning is one of those great examples of 0:15:25.120 --> 0:15:29.480 people that went through the sign symptoms, the worries, the 0:15:29.600 --> 0:15:33.440 challenges of stage advanced kidney disease, got a transplant and 0:15:33.520 --> 0:15:39.880 he got back on his feet. Alonso Morning underwent a 0:15:39.960 --> 0:15:44.240 kidney transplant three years after his initial diagnosis. So the 0:15:44.280 --> 0:15:50.400 recovery process was grueling and it was extremely painful at times, 0:15:50.440 --> 0:15:53.440 and it was difficult. But if you think about anything 0:15:53.480 --> 0:15:56.480 in life worth having, is very difficult to get it. 0:15:56.880 --> 0:15:58.800 And I was trying to get my help back, and 0:15:58.920 --> 0:16:00.680 I was trying to get back on the court. So 0:16:01.440 --> 0:16:04.120 I was trying to get back to some sense of normalcy. 0:16:04.160 --> 0:16:08.240 But it challenged me tremendously, and it really truly challenged me. 0:16:09.000 --> 0:16:11.720 Morning talks about his struggle to get and then adapt 0:16:11.760 --> 0:16:14.600 to living with a new kidney, knowing full well that 0:16:14.680 --> 0:16:18.680 he had a lot of advantages that was in optimal condition, 0:16:19.200 --> 0:16:23.240 excellent health, you know. But that was the benefit of 0:16:23.440 --> 0:16:28.320 my recovery so fast, because the doctors told me, like, hey, 0:16:28.800 --> 0:16:33.480 you know, if you wasn't this high performance athlete, then 0:16:33.480 --> 0:16:36.840 you probably wouldn't have bounced back as fast after the transplant, 0:16:37.720 --> 0:16:41.440 and bounced back he did. Just three years after his transplant. 0:16:41.480 --> 0:16:44.120 Alonso Morning would go on and win a championship with 0:16:44.120 --> 0:16:47.360 the Miami Heat in two thousand six, and today he's 0:16:47.400 --> 0:16:50.920 partnering with Vertex as a spokesperson using his profile and 0:16:51.000 --> 0:16:54.680 his experience to advocate and educate. You know, a big 0:16:54.720 --> 0:16:58.560 part of it is to try to inspire and provide 0:16:58.600 --> 0:17:02.280 hope and encouragement for the those who are going through 0:17:02.360 --> 0:17:05.960 the same ordeal. And Morning says he's hoping Vertex will 0:17:05.960 --> 0:17:08.639 be able to help kidney patients by raising awareness of 0:17:08.640 --> 0:17:12.359 the disease, encouraging people to visit their doctor, and continuing 0:17:12.440 --> 0:17:15.879 to search for a potential treatment. So if this can happen, 0:17:16.359 --> 0:17:20.440 then all of what I've gone through is so much 0:17:21.040 --> 0:17:25.840 more of a worthwhile because maybe to help save other 0:17:25.880 --> 0:17:39.560 people's lives. This is targeting the toughest diseases. A podcast 0:17:39.600 --> 0:17:43.480 from Bloomberg Media Studios and Vertex Pharmaceuticals. If you like 0:17:43.520 --> 0:17:46.359 what you hear, subscribe and leave us a review. I'm 0:17:46.440 --> 0:17:48.560 Jordan's Gospel. Thanks for listening.