WEBVTT - Targeting Cystic Fibrosis (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 --> 0:00:12.799 Targeting the Toughest Diseases, produced by Vertex Pharmaceuticals and Bloomberg 0:00:12.840 --> 0:00:17.680 Media Studios. It explores the innovative tools, methods, and unique 0:00:17.760 --> 0:00:22.479 philosophy Vertex Pharmaceuticals is using to search for treatments for 0:00:22.520 --> 0:00:31.120 some of humanity's most challenging diseases. Here's a recent episode. 0:00:29.160 --> 0:00:34.760 Yeah, Well, I was getting ready to step on the 0:00:34.760 --> 0:00:37.120 field for the New York Jets for the first time 0:00:37.159 --> 0:00:38.279 in nineteen ninety three. 0:00:38.840 --> 0:00:42.400 NFL quarterback Boomer Asiasin had just been traded to the 0:00:42.479 --> 0:00:47.120 Jets after ten incredibly successful seasons with the Cincinnati Bengals, 0:00:47.720 --> 0:00:50.640 a team where he'd wrapped up a Super Bowl appearance 0:00:50.800 --> 0:00:54.240 and an MVP Award. He was born and raised in 0:00:54.280 --> 0:00:56.960 New York, so it was exciting to play for one 0:00:57.000 --> 0:01:01.400 of his hometown teams. But then, just as he was 0:01:01.440 --> 0:01:04.240 about to walk into the field, he was called back. 0:01:05.840 --> 0:01:08.680 The head coach's secretary came out onto the field and said, 0:01:08.720 --> 0:01:10.400 your wife is on the phone. You need to take 0:01:10.400 --> 0:01:12.000 the phone call. So I took the phone call. 0:01:12.600 --> 0:01:15.440 It was Boomer's wife, Cheryl, who was back in Cincinnati 0:01:15.480 --> 0:01:16.480 with their son, Gunner. 0:01:17.040 --> 0:01:19.600 Cheryl told me I needed to get back to Cincinnati, 0:01:19.880 --> 0:01:23.480 that Gunner was in Cincinnati Children's Hospital and he was 0:01:23.520 --> 0:01:24.720 having trouble breathing. 0:01:25.959 --> 0:01:29.119 Boomer grabbed the next flight to Cincinnati, then went straight 0:01:29.160 --> 0:01:31.240 from the airport to the hospital. 0:01:31.160 --> 0:01:33.559 Seeing a two year old Gunner laying in a crib 0:01:33.800 --> 0:01:37.880 hooked up to all sorts of different machines receiving antibiotics, 0:01:38.319 --> 0:01:42.520 was on oxygen, and really wasn't sure what to think. 0:01:43.160 --> 0:01:45.240 The doctor came into the room and told Boomer and 0:01:45.319 --> 0:01:48.040 Cheryl that their little boy had cystic fibrosis. 0:01:48.440 --> 0:01:50.800 That day will live with me forever as one of 0:01:50.840 --> 0:01:52.080 the saddest days of my life. 0:02:00.680 --> 0:02:03.960 Hi. I'm Jordan Gospore. I'm a member of the University 0:02:04.000 --> 0:02:07.440 of Southern California's Center for Health Journalism. This is Targeting 0:02:07.440 --> 0:02:11.399 the Toughest Diseases a podcast produced by Bloomberg Media Studios 0:02:11.600 --> 0:02:15.520 and Vertex Pharmaceuticals. In this series, we look at some 0:02:15.560 --> 0:02:19.800 of humanity's most challenging diseases and how Vertex, a Boston 0:02:19.840 --> 0:02:23.920 based biotech company, is using innovative tools, methods, and a 0:02:24.000 --> 0:02:28.720 unique philosophy to search for treatments and cures. Today we're 0:02:28.720 --> 0:02:32.520 looking at cystic fibrosis, also known as CF, a rare 0:02:32.680 --> 0:02:36.680 chronic genetic disease affecting the lungs and other organs. It 0:02:36.680 --> 0:02:40.520 can lead to all sorts of complications, including lung disease, 0:02:40.919 --> 0:02:46.000 liver disease, diabetes, and even premature death. It affects about 0:02:46.000 --> 0:02:49.280 thirty thousand Americans and more than eighty thousand people globally, 0:02:49.639 --> 0:02:52.160 making it a disease not a lot of people know about, 0:02:52.720 --> 0:02:54.880 but it was not new to Boomeris Siasin. 0:03:03.600 --> 0:03:05.920 Here's the really weird part of this, and maybe the 0:03:06.040 --> 0:03:08.960 destiny and irony part of all of this. Back in 0:03:09.000 --> 0:03:11.880 nineteen eighty nine, I was receiving award as the best 0:03:11.960 --> 0:03:15.120 quarterback in the NFL, and at that awards banquet, there 0:03:15.160 --> 0:03:17.760 was a speaker that night because there was a fundraising 0:03:17.919 --> 0:03:21.560 attachment to that banquet, and his name was Frank DeFord. 0:03:22.080 --> 0:03:25.760 DeFord was a legendary writer at Sports Illustrated, but he 0:03:25.840 --> 0:03:27.040 was also a CF dad. 0:03:27.440 --> 0:03:31.320 Frank spoke so eloquently about how he lost his daughter 0:03:31.360 --> 0:03:35.080 Alex to the disease of cystic fibrosis, which I had 0:03:35.080 --> 0:03:38.560 never heard of. She was eight years old, and everybody 0:03:38.640 --> 0:03:41.600 was captured by his story. That night. I was very captured. 0:03:41.640 --> 0:03:42.240 I was crying. 0:03:42.480 --> 0:03:45.240 Bumeris Iyasin was twenty seven at the time, with no 0:03:45.440 --> 0:03:47.760 kids of his own. He was living a great life 0:03:47.800 --> 0:03:51.200 as an NFL quarterback, but something in that speech touched 0:03:51.240 --> 0:03:53.960 him deeply. He asked to Ford how he could help. 0:03:53.920 --> 0:03:55.520 And he said, well, if you go back to Cincinnati 0:03:55.600 --> 0:03:57.600 and you can get involved in some of the fundraising 0:03:57.800 --> 0:04:01.280 aspects of cystic fibrosis a face on it, if you will, 0:04:01.520 --> 0:04:04.080 and then maybe visit some of the kids at Children's Hospital, 0:04:04.440 --> 0:04:05.760 that would really be helpful. 0:04:06.240 --> 0:04:09.000 And that's exactly what he did. He used his celebrity 0:04:09.000 --> 0:04:12.080 status to help raise awareness and funds for cystic fibrosis, 0:04:12.240 --> 0:04:15.160 and he visited kids in the hospital who were sick. Now, 0:04:15.200 --> 0:04:20.840 his son, Gunner had this same terrible disease. We all 0:04:20.880 --> 0:04:23.640 have mucus that lines her lungs and our airways. This 0:04:23.800 --> 0:04:27.359 mucus has an important job. It captures dust and germs 0:04:27.360 --> 0:04:30.280 in the air we breathe. When mucus traps those germs 0:04:30.279 --> 0:04:33.200 in dust, tiny little hairs on the outside of our 0:04:33.240 --> 0:04:37.560 cells called cilia push all that mucus out of our lungs. Normally, 0:04:37.760 --> 0:04:40.560 this mucus is slippery and watery, but for people with 0:04:40.640 --> 0:04:45.080 cystic fibrosis, this mucus is sticky and thick. As a result, 0:04:45.400 --> 0:04:48.000 the cilia can't push the mucus out and it ends 0:04:48.040 --> 0:04:51.640 up blocking lung passages, making it hard to breathe. Also, 0:04:51.920 --> 0:04:55.240 germs and bacteria get trapped in a person's airways, making 0:04:55.279 --> 0:05:00.320 them more prone to infections. Clearing the mucus could be 0:05:00.400 --> 0:05:03.720 a daily task for people with CF. There are medications 0:05:03.760 --> 0:05:06.800 that help fin it. Some patients use a vibrating vest 0:05:06.880 --> 0:05:10.279 to literally shake the mucus out of their airways. Others 0:05:10.320 --> 0:05:13.160 may have to visit the hospital periodically for a cleanout. 0:05:13.480 --> 0:05:16.359 And because the mucus can also block the ducks to 0:05:16.400 --> 0:05:19.960 the pancreas, many people with CF may struggle to absorb 0:05:20.080 --> 0:05:24.680 nutrients efficiently. Solving that can require periodic IV treatments or 0:05:24.720 --> 0:05:28.200 taking enzymes to help break down food for absorption. IV 0:05:28.360 --> 0:05:31.719 treatments are also used to fight frequent infections and lungs 0:05:31.800 --> 0:05:34.440 that come about in many people with CF. It's a 0:05:34.480 --> 0:05:38.440 demanding regiment, something Gunner Asiasin had to confront when he 0:05:38.600 --> 0:05:39.679 was still very young. 0:05:41.160 --> 0:05:43.400 My dad, who was driving the car just looked at 0:05:43.440 --> 0:05:46.119 me while we were driving and said, this isn't gonna hurt. 0:05:46.240 --> 0:05:48.240 And for the entire rest of the car ride, he 0:05:48.400 --> 0:05:51.120 continued to say, this isn't gonna hurt, This isn't gonna hurt, 0:05:51.120 --> 0:05:53.039 this isn't gonna hurt, as if he was reassuring me 0:05:53.360 --> 0:05:55.200 about something they weren't telling me about. 0:05:55.480 --> 0:05:59.360 Everything was fine until the moment Gunner realized the antibiotic 0:05:59.360 --> 0:06:04.160 treatment he was getting for a CF related infection involved needles. 0:06:05.880 --> 0:06:07.359 I looked at my dad's grinding at I said, you 0:06:07.440 --> 0:06:09.719 lied to me, and I ran out the door, down 0:06:09.720 --> 0:06:12.440 the hallway and into the lobby of Cincinnati Children's Hospital. So, 0:06:12.520 --> 0:06:15.080 if you can picture the scenario, there's my dad, the 0:06:15.120 --> 0:06:17.640 starting quarterback of the Cincinni Bengals at the time, chasing 0:06:17.640 --> 0:06:20.320 his belligerent child, and he finally comes up to me 0:06:20.360 --> 0:06:22.600 and he's like, quiet, please be quiet, you're making a scene. 0:06:22.640 --> 0:06:24.480 I will do whatever you want if you go back 0:06:24.480 --> 0:06:25.240 into that room with me. 0:06:25.560 --> 0:06:29.400 Eventually, after a lot of persuasion, Boomer agreed to buy 0:06:29.440 --> 0:06:32.320 Gunner a video game console if he would go and 0:06:32.360 --> 0:06:33.080 get the treatment. 0:06:33.640 --> 0:06:36.840 So we walked back into interventual raiology and they scrapped 0:06:36.839 --> 0:06:38.599 me down to the table, and I cried and screamed 0:06:38.600 --> 0:06:40.400 to the entire thing. But you better believe that I 0:06:40.400 --> 0:06:42.320 got my Nintendo at the end of that possible visit. 0:06:46.360 --> 0:06:49.080 In college, Gunner reached a turning point with how he 0:06:49.200 --> 0:06:50.719 coexisted with the disease. 0:06:51.160 --> 0:06:54.159 My senior year was a real slog like I went through, 0:06:54.240 --> 0:06:56.839 like several bounds of pneumonia. I knew that I was 0:06:56.960 --> 0:06:59.640 at a point in my life where I had no 0:06:59.680 --> 0:07:00.479 other options. 0:07:01.960 --> 0:07:05.840 That uncertainty, having big questions about the future is one 0:07:05.920 --> 0:07:11.680 of the hardest parts of living with CF. Cystic fibrosis 0:07:11.720 --> 0:07:15.120 is genetic. Our genes give our cells instructions on how 0:07:15.160 --> 0:07:19.240 to produce certain proteins. They tell the cells in our lungs, pancreas, 0:07:19.320 --> 0:07:22.720 or sweat glands to make a special protein called cystic 0:07:22.800 --> 0:07:28.840 fibrosis transmembrane conductance regulator or CFTR for short. This CFTR 0:07:28.920 --> 0:07:32.040 protein is sent to the surface of a cell. Here, 0:07:32.440 --> 0:07:35.560 it acts like little channels between the inside and the 0:07:35.600 --> 0:07:39.040 outside of a cell. These channels open and close, and 0:07:39.080 --> 0:07:41.960 this allows particles like chloride ions to move in or 0:07:41.960 --> 0:07:44.560 out of a cell, and those chloride ions help a 0:07:44.640 --> 0:07:47.600 cell maintain a healthy balance of salt and water. But 0:07:47.640 --> 0:07:50.720 for people with CF, a mutation causes the gene to 0:07:50.720 --> 0:07:54.480 give faulty instructions to the body. Those proteins, those little 0:07:54.560 --> 0:07:58.080 channels don't work right. This means water and salt can't 0:07:58.080 --> 0:07:59.960 flow freely in and out of a cell like they should. 0:08:00.840 --> 0:08:03.360 When that chloride gets trapped in a cell, it doesn't 0:08:03.400 --> 0:08:05.920 have the fluids it needs to hydrate its surface properly. 0:08:06.320 --> 0:08:10.760 Without those fluids, mucus becomes dehydrated, and that's what causes 0:08:10.800 --> 0:08:15.800 it to be thick and sticky. Doctor Chinadou Nikuoro is 0:08:15.800 --> 0:08:19.400 a pediatrician in London. He has spent decades researching the 0:08:19.440 --> 0:08:23.000 condition and says we've known about CF since ancient times. 0:08:23.640 --> 0:08:25.880 If you go back to European proverbs, you may have 0:08:25.920 --> 0:08:29.320 heard there was a saying, woe to that child which, 0:08:29.360 --> 0:08:32.040 when kissed on the forehead, tastes salty. He is bewitched 0:08:32.080 --> 0:08:33.280 and soon must die. 0:08:33.800 --> 0:08:36.960 Salty skin is a symptom because people with cystic fibrosis 0:08:37.120 --> 0:08:39.880 have two to five times the normal amount of salt 0:08:39.880 --> 0:08:40.600 in their sweat. 0:08:40.960 --> 0:08:45.040 And these old grandmothers in northern Germany were describing children 0:08:45.040 --> 0:08:46.040 with cystic fibrosis. 0:08:46.520 --> 0:08:50.160 The genetic mutation that causes CF survived because it provides 0:08:50.200 --> 0:08:52.160 its carriers with some extra immunity. 0:08:52.960 --> 0:08:56.480 Having one copy of the cistic vibrosius mutation seems to 0:08:56.480 --> 0:09:00.960 protect against diarrehal diseases like cholera and typho. That would 0:09:00.960 --> 0:09:03.880 have meant that people who were carriers of that gene 0:09:03.960 --> 0:09:07.479 would be more likely to survive plagues of cholera and typhoid, 0:09:07.880 --> 0:09:10.760 where people who didn't have any copies would have been 0:09:10.760 --> 0:09:11.800 more likely to succumb. 0:09:12.120 --> 0:09:14.600 It was nineteen thirty eight that the disease was named 0:09:14.600 --> 0:09:17.319 and made it into medical literature. It was discovered in 0:09:17.440 --> 0:09:19.240 children who had died of malnutrition. 0:09:19.760 --> 0:09:23.200 The US pathologist Dorothy Anderson found that their pangresses were 0:09:23.360 --> 0:09:27.720 damaged through being bummed up by sticky secretions, and they 0:09:27.720 --> 0:09:30.320 were cystic and fibrotic, and that was the name given 0:09:30.400 --> 0:09:31.000 to the disease. 0:09:31.440 --> 0:09:35.079 Doctor Niicuro says life expectancy for children living with CF 0:09:35.120 --> 0:09:38.320 has been extended, thanks in large part to newborn screening 0:09:38.440 --> 0:09:41.559 and improve treatments, but he is hopeful that treatments will 0:09:41.600 --> 0:09:44.440 not just extend life for patients living with the disease, 0:09:44.720 --> 0:09:47.560 it will improve their quality of life as well. 0:09:47.720 --> 0:09:50.920 What I hope that will lead to is a reduction 0:09:51.160 --> 0:09:54.920 in the need for the kind of intense therapy, both 0:09:54.960 --> 0:09:57.840 in terms of antibiotics, hospital admissions and in the even 0:09:57.920 --> 0:10:02.600 physiotherapy that has been characteris consistent fibrousus care over the years. 0:10:04.720 --> 0:10:07.880 Coming up with therapies that can both prolong and improve 0:10:08.000 --> 0:10:10.840 life is a pretty tall order, but it's a passion 0:10:10.880 --> 0:10:15.320 for doctor David Altschuler, chief Scientific Officer at Vertex Pharmaceuticals. 0:10:15.640 --> 0:10:18.480 As a physician and a researcher, he spent the last 0:10:18.520 --> 0:10:21.600 thirty five years of his career scouring our DNA code 0:10:21.800 --> 0:10:24.800 for clues about some of the world's toughest diseases. 0:10:26.520 --> 0:10:29.320 So the first thing we do is we look for 0:10:29.400 --> 0:10:33.680 diseases with great unmet need, where the disease is very 0:10:33.760 --> 0:10:37.520 serious and people's lives are impacted and there's nothing that 0:10:37.600 --> 0:10:40.400 really is is transformational, that can really change their lives, 0:10:40.520 --> 0:10:43.760 and cystic fibrosis certainly was that. The second was we 0:10:43.840 --> 0:10:47.400 look for a case where the underlying human biology, the 0:10:47.480 --> 0:10:50.720 underlying cause of the disease is known, and again in CF, 0:10:50.800 --> 0:10:54.120 the cystic fibrosis gene mutation was known. What was missing 0:10:54.360 --> 0:10:57.360 was therapeutics that could do what needed to be done, 0:10:57.360 --> 0:10:59.880 which is, in this case, restore CF function. And that's 0:11:00.120 --> 0:11:02.840 kind of thing that really gets our scientists and doctors 0:11:02.880 --> 0:11:04.800 fired up, and so they worked on it for twenty 0:11:04.920 --> 0:11:08.559 years to get where we are today. 0:11:08.640 --> 0:11:10.760 Can you speak a little bit more about how you 0:11:10.840 --> 0:11:13.720 and Vertex are tackling cystic fibrosis. 0:11:14.120 --> 0:11:16.720 So the work that Vertex has done in CF really 0:11:16.720 --> 0:11:20.920 goes back decades ran in families, but until nineteen eighty 0:11:21.000 --> 0:11:24.200 nine when that gene was cloned, no one actually knew 0:11:24.240 --> 0:11:25.319 what caused the disease. 0:11:25.600 --> 0:11:28.960 That changed with the discovery of CFTR. 0:11:28.640 --> 0:11:31.079 But then the question was could you repair it? And 0:11:31.120 --> 0:11:35.000 for a period of decades that seemed impossible. People tried 0:11:35.080 --> 0:11:37.080 lots of different things and couldn't figure it out. But 0:11:37.160 --> 0:11:41.000 twenty years or more ago, scientists at Vertex had an idea. 0:11:41.280 --> 0:11:44.160 They had an idea of how to use chemicals to 0:11:44.520 --> 0:11:48.600 coax the CFTR protein that's not working to function again. 0:11:49.080 --> 0:11:51.280 That's what Vertex has been working on for the last 0:11:51.360 --> 0:11:54.760 twenty years and now has approved medicines with the potential 0:11:55.000 --> 0:11:57.720 to treat up to ninety percent of all people with CF, 0:11:58.080 --> 0:11:59.200 and they're not done yet. 0:11:59.800 --> 0:12:02.840 The things that keeps us working hard on this problem 0:12:02.960 --> 0:12:05.560 is that less than ten percent don't make any of 0:12:05.600 --> 0:12:06.200 the protein. 0:12:06.520 --> 0:12:09.880 The way current therapies work is to coax the protein 0:12:10.040 --> 0:12:13.080 that has an inherited mutation to function again. But some 0:12:13.120 --> 0:12:17.400 people don't make any protein, so a different approach is needed, and. 0:12:17.400 --> 0:12:20.440 So we have research ongoing in our laboratories and with 0:12:20.520 --> 0:12:23.199 a number of partners, are working hard for the last 0:12:23.240 --> 0:12:26.640 ten percent. We're still very much in the CF story. 0:12:26.800 --> 0:12:28.400 We still have a long way to go till we 0:12:28.440 --> 0:12:29.160 reach our goal. 0:12:31.800 --> 0:12:34.840 For people with CF, like Gunner as siasin things that 0:12:34.920 --> 0:12:39.320 once felt unimaginable now seem possible, like returning to school 0:12:39.360 --> 0:12:40.319 to see old friends. 0:12:40.640 --> 0:12:43.280 I had my five year college reunion, and most people 0:12:43.320 --> 0:12:45.320 remember me from my college a's at my last year 0:12:45.360 --> 0:12:48.040 or two, being very very sick, like always missing things, 0:12:48.080 --> 0:12:50.680 missing social activities, missing class, missing things like that. Yeah, 0:12:50.679 --> 0:12:52.360 here we are at the reunion and it's like the 0:12:52.440 --> 0:12:53.959 chance to go back to college, right, you know, We're 0:12:53.960 --> 0:12:55.360 staying out late, and next thing you know, I'm up 0:12:55.400 --> 0:12:57.520 every morning at like six point thirty getting breakfast for everyone. 0:12:57.720 --> 0:13:00.000 And my friends were like, what is going on with you? 0:13:00.240 --> 0:13:02.280 But as great as the weekend was The drive home 0:13:02.320 --> 0:13:04.360 with his girlfriend was even more impactful. 0:13:04.600 --> 0:13:07.360 We were stuck into New Jersey Turnpike and she looked 0:13:07.360 --> 0:13:09.199 at me and she was like, do you realize what 0:13:09.240 --> 0:13:12.040 we did this weekend? We went away and we're able 0:13:12.080 --> 0:13:14.720 to do something. What do you want to do next 0:13:14.720 --> 0:13:17.480 with your life? And I paused for a moment. It 0:13:17.559 --> 0:13:19.280 hadn't occurred to me that I could do something with 0:13:19.320 --> 0:13:21.120 my life. I didn't have to live at home anymore. 0:13:21.400 --> 0:13:23.840 What they decided to do next was get married. 0:13:24.320 --> 0:13:27.200 I've never been at a wedding where everybody, I mean 0:13:27.240 --> 0:13:29.880 all two hundred and fifty people just came together as 0:13:30.040 --> 0:13:35.240 one group and we're crying and we're laughing and we're cheering, 0:13:35.800 --> 0:13:37.520 and it was like the greatest moment of all of 0:13:37.559 --> 0:13:38.280 our lives. 0:13:38.679 --> 0:13:42.600 Going back to school, getting married, those experiences are possible 0:13:42.760 --> 0:13:45.640 because of one thing. Hope. Well. 0:13:45.679 --> 0:13:47.559 I don't know how much of a football fan you are, 0:13:47.559 --> 0:13:49.760 but when you play for the Jets, Cardinals and Bengals, 0:13:49.760 --> 0:13:52.120 it's all about hope. You need as much hope as 0:13:52.120 --> 0:13:53.400 you possibly can get. 0:13:53.400 --> 0:13:55.839 You know. I think for me, I mean honestly, I 0:13:55.880 --> 0:13:58.240 hope is going to define the rest of my career. 0:13:58.400 --> 0:14:00.440 When I was going through the worst today, I had 0:14:00.440 --> 0:14:02.719 a little quote on my desk and it was a 0:14:02.760 --> 0:14:04.920 Wizard Churchill quote and it was something like, if you're 0:14:04.920 --> 0:14:08.559 going through hell, keep going. And that meant a lot 0:14:08.600 --> 0:14:09.520 to me back then. 0:14:17.480 --> 0:14:20.560 This is Targeting the Toughest Diseases a podcast from Bloomberg 0:14:20.600 --> 0:14:24.320 Media Studios and Vertex Pharmaceuticals. If you like what you hear, 0:14:24.640 --> 0:14:28.320 subscribe and leave us a review. I'm Jordan Gosfore. Thanks 0:14:28.320 --> 0:14:28.880 for listening.