1 00:00:00,240 --> 00:00:02,480 Speaker 1: Hi, this is newt twenty twenty is going to be 2 00:00:02,520 --> 00:00:05,160 Speaker 1: one of the most extraordinary election years of our lifetime. 3 00:00:05,360 --> 00:00:07,560 Speaker 1: I want to invite you to join my Inner Circle 4 00:00:07,920 --> 00:00:10,280 Speaker 1: as we discuss each twist and turn in the race 5 00:00:10,320 --> 00:00:13,640 Speaker 1: and my members only Inner Circle Club. You will receive 6 00:00:13,760 --> 00:00:18,960 Speaker 1: special flash briefings, online events, and members only audio reports 7 00:00:18,960 --> 00:00:21,599 Speaker 1: from me and my team. Here's a special offer to 8 00:00:21,640 --> 00:00:24,880 Speaker 1: my podcast listeners. 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On this episode of news World, when the 17 00:01:02,240 --> 00:01:05,640 Speaker 1: idea of gene therapy really surfaced, I wanted to develop 18 00:01:05,800 --> 00:01:09,200 Speaker 1: treatments for these diseases, not just understand them. The child 19 00:01:09,440 --> 00:01:13,200 Speaker 1: in Miami has thrown away his cane, which was his 20 00:01:13,360 --> 00:01:17,360 Speaker 1: lifelong dream, his blind cane, and is doing things that 21 00:01:17,400 --> 00:01:20,200 Speaker 1: he never could do before. There are many times when 22 00:01:20,520 --> 00:01:23,920 Speaker 1: I began to feel like a pioneer who was trying 23 00:01:23,959 --> 00:01:33,240 Speaker 1: to move west on a poorly defined trail. On this 24 00:01:33,280 --> 00:01:36,520 Speaker 1: episode of news World, we're going to meet three engaging 25 00:01:36,560 --> 00:01:39,280 Speaker 1: and passionate doctors who are on the cutting edge of 26 00:01:39,319 --> 00:01:45,560 Speaker 1: medical breakthroughs using gene therapy. Doctor Jim Wilson, doctor Catherine High, 27 00:01:45,600 --> 00:01:48,960 Speaker 1: and doctor Jean Bennett. All three were honored last year 28 00:01:49,000 --> 00:01:53,320 Speaker 1: by Sanford Health as nominees for their million dollars Lorraine 29 00:01:53,320 --> 00:01:56,840 Speaker 1: Cross Award, an award that's given for innovative breakthroughs in 30 00:01:56,880 --> 00:02:00,960 Speaker 1: medicine or science that has the ability to transform global 31 00:02:01,000 --> 00:02:05,280 Speaker 1: health and change lives. I work with Sandford as an adviser. 32 00:02:05,720 --> 00:02:09,600 Speaker 1: I was there at the award ceremony. Frankly, I left 33 00:02:09,639 --> 00:02:13,120 Speaker 1: the evening amazed, amazed by the potential of gene therapy 34 00:02:13,160 --> 00:02:17,560 Speaker 1: to cure disease, even more amazed by the determination of 35 00:02:17,600 --> 00:02:21,720 Speaker 1: these doctors and amazed by the courage of their patients. 36 00:02:22,440 --> 00:02:25,000 Speaker 1: That's why I want to focus this episode of Newts 37 00:02:25,080 --> 00:02:29,600 Speaker 1: World letting them tell their stories. You're going to hear 38 00:02:29,639 --> 00:02:32,800 Speaker 1: the doctor's early struggles in the field, you know, starting 39 00:02:32,800 --> 00:02:36,000 Speaker 1: out in medical school or a lab environment, only to 40 00:02:36,080 --> 00:02:40,240 Speaker 1: suddenly have funding for gene therapy dry up. There they were. 41 00:02:40,280 --> 00:02:42,520 Speaker 1: They thought they knew the entire future of their lives, 42 00:02:42,960 --> 00:02:48,800 Speaker 1: and suddenly the roadmap was destroyed as funding changed. But 43 00:02:48,880 --> 00:02:51,200 Speaker 1: they believe so much what they were doing. They just 44 00:02:51,320 --> 00:02:56,520 Speaker 1: persevered and they've made an incredible difference in many people's lives. 45 00:02:57,440 --> 00:02:59,640 Speaker 1: We're also going to hear about the patients they've treated 46 00:03:00,080 --> 00:03:04,440 Speaker 1: and the miraculous outcomes they've experienced after years of clinical troughs. 47 00:03:05,280 --> 00:03:07,480 Speaker 1: I want you to close your eyes for a minute 48 00:03:07,520 --> 00:03:11,679 Speaker 1: playter listening so you understand what I mean by miraculous. 49 00:03:11,919 --> 00:03:15,840 Speaker 1: Imagine you're a child. You're born with a genetic defect 50 00:03:16,520 --> 00:03:19,440 Speaker 1: and you can't see, so keep close your eyes. Think 51 00:03:19,440 --> 00:03:21,960 Speaker 1: about that. You're going to spend the entire rest of 52 00:03:21,960 --> 00:03:24,000 Speaker 1: your life, at three or four or five years of age, 53 00:03:24,560 --> 00:03:29,040 Speaker 1: not being able to see, and suddenly somebody finds some 54 00:03:29,160 --> 00:03:34,040 Speaker 1: breakthroughs and you can see because due to these breakthroughs, 55 00:03:34,560 --> 00:03:39,680 Speaker 1: Christian can see. Before I introduce my first guest, Doctor Wilson, 56 00:03:40,440 --> 00:03:42,520 Speaker 1: I want you to imagine for the moment, being the 57 00:03:42,600 --> 00:03:46,960 Speaker 1: parent of a child who's missing a chromosome that contains 58 00:03:46,960 --> 00:03:51,920 Speaker 1: a specific gene U B E three A, but just 59 00:03:52,080 --> 00:03:56,200 Speaker 1: that one mutation affects the quality of your child's life. 60 00:03:57,280 --> 00:04:00,760 Speaker 1: That is Alison Baron's story, a mother of a beautiful 61 00:04:00,840 --> 00:04:08,400 Speaker 1: daughter named Quincy. My name is Alison Brent and I'm 62 00:04:08,440 --> 00:04:10,400 Speaker 1: the mother of a four year old little girl. Her 63 00:04:10,480 --> 00:04:13,960 Speaker 1: name is Quincy, who is diagnosed with a rare monogenetic 64 00:04:14,040 --> 00:04:18,200 Speaker 1: disorder called Angelman syndrome. It's neurogenetic, which means that she's 65 00:04:18,240 --> 00:04:21,760 Speaker 1: missing a single gene and that ultimately results in her 66 00:04:22,240 --> 00:04:25,160 Speaker 1: having a lot of challenges. Is what we call a 67 00:04:25,279 --> 00:04:29,320 Speaker 1: taxic or. She has real significant balance issues. Her body 68 00:04:29,720 --> 00:04:32,680 Speaker 1: is moving a lot, and when she's trying to walk, 69 00:04:32,720 --> 00:04:34,560 Speaker 1: she's trying to figure out where her body is in 70 00:04:34,640 --> 00:04:37,240 Speaker 1: space and how does she balance herself so she can 71 00:04:37,279 --> 00:04:40,840 Speaker 1: walk safely. Every time we take a step, we tell 72 00:04:41,040 --> 00:04:43,599 Speaker 1: ourselves to activate one leg and relax the other leg, 73 00:04:43,600 --> 00:04:46,640 Speaker 1: and she doesn't have that relaxation. So really learning that 74 00:04:46,720 --> 00:04:49,920 Speaker 1: motor plan and learning how to do very gross things 75 00:04:50,000 --> 00:04:53,200 Speaker 1: like walking or very fine things like pointing, it's very 76 00:04:53,200 --> 00:04:57,000 Speaker 1: difficult for kids like Quincy. Those challenges are something that 77 00:04:57,480 --> 00:05:01,200 Speaker 1: she is incredibly inspiring to face the because she fights 78 00:05:01,240 --> 00:05:04,520 Speaker 1: them every day with so much determination and effort. She 79 00:05:04,600 --> 00:05:06,920 Speaker 1: knows she's different. She knows things are harder for her, 80 00:05:07,320 --> 00:05:09,960 Speaker 1: and she'll do thirty five hours a week of physical 81 00:05:10,000 --> 00:05:12,480 Speaker 1: therapy in order to do her best to keep up 82 00:05:12,520 --> 00:05:16,160 Speaker 1: with her peers. Finally, doctor Wilson was the biggest blessing 83 00:05:16,480 --> 00:05:19,200 Speaker 1: that has probably ever happened to our family, and I 84 00:05:19,200 --> 00:05:22,040 Speaker 1: would venture to say our community. What happened was my 85 00:05:22,120 --> 00:05:25,080 Speaker 1: daughter was diagnosed with the Angelman syndrome in twenty fourteen, 86 00:05:25,600 --> 00:05:28,599 Speaker 1: and so I said, who cured cystic fibrosis? And I 87 00:05:28,680 --> 00:05:31,600 Speaker 1: need to find this person? And a press release came 88 00:05:31,600 --> 00:05:33,960 Speaker 1: out as I was looking for who this was saying 89 00:05:34,000 --> 00:05:36,479 Speaker 1: that somebody was giving a lecture and it was the 90 00:05:36,520 --> 00:05:40,320 Speaker 1: person who pioneered gene therapy and cured cystic fibrosis. And 91 00:05:40,360 --> 00:05:42,040 Speaker 1: I was like, I need to meet this person. And 92 00:05:42,080 --> 00:05:44,000 Speaker 1: I looked in the press release and I found his name, 93 00:05:44,120 --> 00:05:46,920 Speaker 1: doctor James Wilson, and so I sent him an email, 94 00:05:47,200 --> 00:05:50,880 Speaker 1: a very brief email describing myself and describing Quincy and 95 00:05:50,960 --> 00:05:52,960 Speaker 1: asked him if he would be willing to have a conversation. 96 00:05:53,520 --> 00:05:55,719 Speaker 1: I got a response back from him one hour later, 97 00:05:55,760 --> 00:05:59,159 Speaker 1: on a Sunday night, at ten thirty pm, that said, 98 00:05:59,200 --> 00:06:02,039 Speaker 1: when are you available to talk this week? The next 99 00:06:02,120 --> 00:06:05,160 Speaker 1: day we got on the phone and we talked about 100 00:06:05,240 --> 00:06:07,240 Speaker 1: what Angelman syndrome was. He had never heard of it. 101 00:06:07,920 --> 00:06:11,400 Speaker 1: We explained what it was and how it could be treated, 102 00:06:11,560 --> 00:06:14,839 Speaker 1: and what the literature was that was available for Angelman syndrome, 103 00:06:15,080 --> 00:06:18,080 Speaker 1: and he said, this is incredibly promising. I have never 104 00:06:18,120 --> 00:06:19,520 Speaker 1: heard of it, but I'm going to do my due 105 00:06:19,520 --> 00:06:21,680 Speaker 1: diligence and I'm going to look into it. Can you 106 00:06:21,680 --> 00:06:24,760 Speaker 1: come to Philadelphia and meet with us in person? And 107 00:06:24,800 --> 00:06:27,400 Speaker 1: I said, I can be there in ninety minutes. And 108 00:06:27,480 --> 00:06:29,560 Speaker 1: so we set up a meeting for him and about 109 00:06:29,560 --> 00:06:32,719 Speaker 1: fifteen other scientists around the world to meet and talk 110 00:06:32,720 --> 00:06:35,440 Speaker 1: about Angelman syndrome and all of the science behind it. 111 00:06:35,880 --> 00:06:37,719 Speaker 1: And at the end of the meeting there was a 112 00:06:37,760 --> 00:06:40,400 Speaker 1: consensus that he felt that he could treat Angelman syndrome. 113 00:06:40,960 --> 00:06:43,920 Speaker 1: He's such a personable man and he has been very 114 00:06:43,920 --> 00:06:46,719 Speaker 1: close not only to our community, but to my family. 115 00:06:47,279 --> 00:06:50,440 Speaker 1: He has reached out to us on numerous occasions just 116 00:06:50,560 --> 00:06:54,800 Speaker 1: because he is enamored and he's touched by Quincy, and 117 00:06:54,880 --> 00:06:59,479 Speaker 1: so because of that, the personality that he has is 118 00:06:59,560 --> 00:07:02,679 Speaker 1: all this, Let's get to the point and let's fix 119 00:07:02,720 --> 00:07:07,000 Speaker 1: this disorder because we can because of her challenges, and 120 00:07:07,040 --> 00:07:10,680 Speaker 1: because of the coordination issues that she has, Quincy can't 121 00:07:10,680 --> 00:07:13,440 Speaker 1: talk because her tongue and her face and all the 122 00:07:13,480 --> 00:07:16,440 Speaker 1: muscles required in the mouth, her lips, all of that 123 00:07:16,520 --> 00:07:19,280 Speaker 1: have to be coordinated, and coordinations really hard for kids 124 00:07:19,280 --> 00:07:21,800 Speaker 1: with Angelman syndrome. And because of that, she has a 125 00:07:21,800 --> 00:07:23,960 Speaker 1: lot she wants to say, and when you ask her 126 00:07:23,960 --> 00:07:25,800 Speaker 1: to talk, she opens her mouth and tries to talk, 127 00:07:26,200 --> 00:07:28,760 Speaker 1: but she can't. So what we can provide her with 128 00:07:28,840 --> 00:07:31,200 Speaker 1: is she wears an iPad and that iPad has a 129 00:07:31,240 --> 00:07:34,560 Speaker 1: symbol system, and she can point to symbols of words 130 00:07:34,600 --> 00:07:37,280 Speaker 1: that she wants to say. And then her voice she 131 00:07:37,320 --> 00:07:39,640 Speaker 1: wears all day and she carries that on her body, 132 00:07:39,960 --> 00:07:41,760 Speaker 1: so if she has something she'd like to say, she 133 00:07:41,800 --> 00:07:44,680 Speaker 1: has to point to a symbol. And so now she's 134 00:07:44,680 --> 00:07:46,760 Speaker 1: been able to tell us all the things she's thinking about, 135 00:07:47,080 --> 00:07:51,240 Speaker 1: like she wants a hug, or she's hungry, or she's thirsty, 136 00:07:51,800 --> 00:07:54,440 Speaker 1: or she's tired, or her tummy hurts, and those are 137 00:07:54,440 --> 00:07:57,920 Speaker 1: all things she couldn't do. When you have a kid 138 00:07:58,000 --> 00:08:00,400 Speaker 1: who has so much that they want to say and 139 00:08:00,480 --> 00:08:02,800 Speaker 1: it's so hard for them to let that out, all 140 00:08:02,840 --> 00:08:04,920 Speaker 1: you want to do is provide for them to make 141 00:08:04,920 --> 00:08:07,400 Speaker 1: that easier so that they can let out the words 142 00:08:07,440 --> 00:08:10,800 Speaker 1: that are in there. But things are either possible or impossible, 143 00:08:10,800 --> 00:08:14,520 Speaker 1: and there's no one between. And doctor Wilson realizes it's possible, 144 00:08:14,800 --> 00:08:17,280 Speaker 1: and he's not going to stop until he is successful, 145 00:08:17,640 --> 00:08:20,480 Speaker 1: and he believes that he can treat children with rare 146 00:08:20,600 --> 00:08:25,200 Speaker 1: neurogenetic disorders. And I feel fullheartedly that the success of 147 00:08:25,280 --> 00:08:29,560 Speaker 1: gene therapy is really because of him. Now I want 148 00:08:29,560 --> 00:08:33,199 Speaker 1: to introduce you to doctor Jim Wilson, who treated Quincy 149 00:08:33,240 --> 00:08:37,760 Speaker 1: in our opening story. He's considered the godfather of gene 150 00:08:37,760 --> 00:08:42,640 Speaker 1: therapy due to his pioneering innovations and his lifelong commitment 151 00:08:43,080 --> 00:08:46,960 Speaker 1: to new discoveries in this field. Oh I was interested 152 00:08:47,000 --> 00:08:52,360 Speaker 1: in science from the very beginning, but when I was 153 00:08:52,440 --> 00:08:56,320 Speaker 1: at a point where I was considering graduate school or 154 00:08:56,320 --> 00:08:59,880 Speaker 1: what to do after college, I felt that it was 155 00:09:00,160 --> 00:09:02,600 Speaker 1: important to me that the work that I did would 156 00:09:02,640 --> 00:09:06,880 Speaker 1: be more directly related to helping people, and particularly in 157 00:09:06,920 --> 00:09:10,439 Speaker 1: the area of medicine. So I decided to enter into 158 00:09:10,480 --> 00:09:13,600 Speaker 1: a combined MD and a PhD program at the University 159 00:09:13,640 --> 00:09:18,160 Speaker 1: of Michigan. So my initial project was to define the 160 00:09:18,360 --> 00:09:23,280 Speaker 1: molecular basis of a rare neurologic disease called the lesh 161 00:09:23,400 --> 00:09:26,960 Speaker 1: Nyon syndrome. It was quite exciting, incredibly productive time in 162 00:09:27,000 --> 00:09:30,120 Speaker 1: my life where I was able to participate in cutting 163 00:09:30,200 --> 00:09:34,559 Speaker 1: edge molecular science but also gets to know the patients, 164 00:09:34,600 --> 00:09:37,439 Speaker 1: these young boys with this disease and their families. One 165 00:09:37,480 --> 00:09:39,120 Speaker 1: of these young boys that I got to know very 166 00:09:39,160 --> 00:09:42,880 Speaker 1: well because we would transport him back and forth from 167 00:09:42,920 --> 00:09:46,319 Speaker 1: his home in North Carolina to the Clinical Research Center 168 00:09:46,360 --> 00:09:48,880 Speaker 1: at the University of Michigan. During one of his trips 169 00:09:48,920 --> 00:09:52,599 Speaker 1: with us, I was able to identify the mutation that 170 00:09:52,720 --> 00:09:55,720 Speaker 1: caused his disease. And it was this kind of insight 171 00:09:55,840 --> 00:09:59,319 Speaker 1: that one rarely gets in science, these so called Eureka moments. 172 00:10:00,080 --> 00:10:02,360 Speaker 1: Was quite proud of the accomplishment and it was widely 173 00:10:02,400 --> 00:10:05,240 Speaker 1: acclaimed because it was cutting edge at the time. And 174 00:10:05,280 --> 00:10:08,120 Speaker 1: I remember when I brought him back home to meet 175 00:10:08,200 --> 00:10:10,480 Speaker 1: with his mother, who would pick him up at the 176 00:10:10,640 --> 00:10:14,640 Speaker 1: Raleigh Durham airplane terminal, and I was so excited to 177 00:10:14,679 --> 00:10:16,800 Speaker 1: meet with her and to share with her that we 178 00:10:16,880 --> 00:10:21,000 Speaker 1: identify the mutation that caused the son's disease. And she 179 00:10:21,120 --> 00:10:24,440 Speaker 1: looked at me and asked a very probing question, but 180 00:10:24,480 --> 00:10:27,000 Speaker 1: a very simple question, how is this going to help 181 00:10:27,000 --> 00:10:30,400 Speaker 1: my son? There was just silence, and I felt like 182 00:10:30,440 --> 00:10:32,480 Speaker 1: I was punched in the stomach, not by her, but 183 00:10:32,640 --> 00:10:37,200 Speaker 1: by my own kind of view and how excited I 184 00:10:37,320 --> 00:10:40,560 Speaker 1: was about the work, but realizing that its impact was 185 00:10:40,679 --> 00:10:43,640 Speaker 1: quite marginal. And it was really at that moment I 186 00:10:43,720 --> 00:10:46,079 Speaker 1: decided that whatever I was going to do going forward, 187 00:10:46,400 --> 00:10:49,800 Speaker 1: that I wanted to develop treatments for these diseases, not 188 00:10:49,840 --> 00:10:52,880 Speaker 1: just understand them. When the idea of gene therapy really 189 00:10:52,960 --> 00:10:55,439 Speaker 1: surfaced in a way that I could not only treat 190 00:10:55,679 --> 00:10:58,440 Speaker 1: this young man who are clearly too late to do that, 191 00:10:58,520 --> 00:11:02,559 Speaker 1: but others with diseases like his and other genetic diseases, 192 00:11:02,600 --> 00:11:05,679 Speaker 1: And therein started my journey. How difficult was it to 193 00:11:05,679 --> 00:11:08,520 Speaker 1: find support? As I understand it at the time you're 194 00:11:08,559 --> 00:11:11,600 Speaker 1: doing it, this is really a brand new approach and 195 00:11:12,400 --> 00:11:17,640 Speaker 1: sort of exploring an unknown territory. When it started back 196 00:11:17,920 --> 00:11:22,120 Speaker 1: in the late eighties, there was a lot of enthusiasm, 197 00:11:22,240 --> 00:11:25,840 Speaker 1: almost unbridled enthusiasm for what we were doing, in that 198 00:11:26,280 --> 00:11:32,199 Speaker 1: the expectations were quite high and ended up being unrealistic. 199 00:11:33,280 --> 00:11:35,320 Speaker 1: And what we did at the time they always to 200 00:11:35,440 --> 00:11:40,440 Speaker 1: use whatever technology viruses so called viruses that were available 201 00:11:40,720 --> 00:11:47,119 Speaker 1: and spent years to try to attenuate them and incorporate 202 00:11:47,200 --> 00:11:50,920 Speaker 1: normal genes and then into the clinic to see if 203 00:11:50,960 --> 00:11:53,440 Speaker 1: they would work. And during that period of the nineties 204 00:11:53,440 --> 00:11:56,760 Speaker 1: there was a tremendous amount of activity in moving gene 205 00:11:56,760 --> 00:11:59,120 Speaker 1: therapy into the clinic. But what we learned at the 206 00:12:00,000 --> 00:12:04,160 Speaker 1: really the turn of the century was that the technology 207 00:12:04,160 --> 00:12:07,320 Speaker 1: we had available to us at the time was not 208 00:12:07,600 --> 00:12:12,000 Speaker 1: up to the task, and there were clinical trials that 209 00:12:12,160 --> 00:12:14,800 Speaker 1: essentially didn't succeed. In fact, none of them were succeeding, 210 00:12:15,720 --> 00:12:21,640 Speaker 1: and unfortunately, there were several examples where there were tragic 211 00:12:21,679 --> 00:12:27,040 Speaker 1: consequences where our attempts to try to benefit someone led 212 00:12:27,120 --> 00:12:31,280 Speaker 1: to toxicity. In particular, a young man by the name 213 00:12:31,320 --> 00:12:36,679 Speaker 1: of Jesse Gelsinger, who courageously volunteered to participate in one 214 00:12:36,679 --> 00:12:40,920 Speaker 1: of the trials conducted here at Penn, had an unexpected 215 00:12:40,960 --> 00:12:45,520 Speaker 1: reaction to this experiment and eventually died from it. And 216 00:12:45,559 --> 00:12:48,520 Speaker 1: it was about that time that there were sort of 217 00:12:48,559 --> 00:12:52,839 Speaker 1: other events that occurred that led to a dramatic erosion 218 00:12:53,760 --> 00:12:56,160 Speaker 1: of support for the field. In fact, it was very 219 00:12:56,160 --> 00:12:59,000 Speaker 1: hard to get any support right around the turn of 220 00:12:59,000 --> 00:13:01,679 Speaker 1: the century, so new the challenging time and the time 221 00:13:02,240 --> 00:13:03,880 Speaker 1: that was sort of critical to get where we are 222 00:13:03,920 --> 00:13:08,200 Speaker 1: today was that first decade, kind of two thousand and ten, 223 00:13:08,559 --> 00:13:11,800 Speaker 1: where we really had no support, but a few of 224 00:13:11,880 --> 00:13:15,160 Speaker 1: us still believed in the concept and just continued to 225 00:13:15,160 --> 00:13:19,679 Speaker 1: sort of plug away and quietly made some important discoveries 226 00:13:19,760 --> 00:13:21,440 Speaker 1: during that period of time that sort of set the 227 00:13:21,480 --> 00:13:24,920 Speaker 1: stage for where we are right now. Jim Wilson's breakthrough 228 00:13:25,160 --> 00:13:30,520 Speaker 1: in developing a benign virus delivery system suddenly created enormous 229 00:13:30,600 --> 00:13:34,319 Speaker 1: new opportunities in gene therapy, and in that sense, Wilson, 230 00:13:34,360 --> 00:13:41,200 Speaker 1: in many ways, really was the godfather. When we come back, 231 00:13:41,400 --> 00:13:44,400 Speaker 1: we'll meet doctor Jean Bennett, one of the doctors who 232 00:13:44,520 --> 00:13:48,559 Speaker 1: built upon doctor Wilson's breakthroughs and find out why her 233 00:13:48,640 --> 00:14:12,720 Speaker 1: trows with dogs led to a cure for blindness. Doctor 234 00:14:12,800 --> 00:14:16,480 Speaker 1: Catherine High and doctor Jean Bennett began working together in 235 00:14:16,559 --> 00:14:21,080 Speaker 1: nineteen ninety. Twenty seven years later, they succeeded in getting 236 00:14:21,120 --> 00:14:24,320 Speaker 1: the first gene therapy medication approved by the Food and 237 00:14:24,400 --> 00:14:29,000 Speaker 1: Drug Administration. Their story is one of determination and commitment 238 00:14:29,400 --> 00:14:31,840 Speaker 1: to bring a treatment that cured a form of blindness 239 00:14:31,840 --> 00:14:35,320 Speaker 1: to market, but it wasn't without struggle along the way. 240 00:14:36,080 --> 00:14:39,640 Speaker 1: Here doctor Bennett explains the early years that she spent 241 00:14:40,040 --> 00:14:44,600 Speaker 1: working with dogs in clinical trials. My collaborators at University 242 00:14:44,600 --> 00:14:47,800 Speaker 1: of Pennsylvania in the Vet School had the wisdom early 243 00:14:47,920 --> 00:14:53,840 Speaker 1: on of studying dogs that were born blind in order 244 00:14:53,920 --> 00:14:57,840 Speaker 1: to be able to better understand human blindness, to use 245 00:14:57,840 --> 00:15:02,160 Speaker 1: them as models of human blindness. That was a unique 246 00:15:02,320 --> 00:15:08,760 Speaker 1: view and plan that they had because usually kennel clubs 247 00:15:09,000 --> 00:15:14,440 Speaker 1: and other organizations which breed dogs breed out genetic problems 248 00:15:14,480 --> 00:15:17,880 Speaker 1: such as blindness or hip displays and so forth, and 249 00:15:18,120 --> 00:15:22,000 Speaker 1: people get rid of those animals. But here this group 250 00:15:22,080 --> 00:15:26,040 Speaker 1: was searching for them as models. Because dogs, just like humans, 251 00:15:26,040 --> 00:15:30,160 Speaker 1: there are dogs that are born spontaneously blind or with 252 00:15:30,280 --> 00:15:35,680 Speaker 1: other deficits. And there was a pet owner who owned 253 00:15:36,240 --> 00:15:40,560 Speaker 1: a very large dog, a Swedish Briard dog. His name 254 00:15:40,600 --> 00:15:44,600 Speaker 1: was Merlin. And they brought him over to the clinic 255 00:15:44,640 --> 00:15:47,720 Speaker 1: and said, our you inter snurt dog, and they said, 256 00:15:47,760 --> 00:15:52,400 Speaker 1: of course yes. They borrowed the dog and bred him 257 00:15:52,480 --> 00:15:57,520 Speaker 1: with one of their small, normal sighted laboratory dogs and 258 00:15:57,600 --> 00:16:03,680 Speaker 1: then crossbred and back to that dog. Realizing that this 259 00:16:03,760 --> 00:16:08,800 Speaker 1: original owner's dog was probably had an autosomal recessive disease 260 00:16:08,880 --> 00:16:14,080 Speaker 1: that means it's a disease where two different alleles are 261 00:16:14,120 --> 00:16:18,320 Speaker 1: required to be affected. You need two badgings in order 262 00:16:18,360 --> 00:16:23,520 Speaker 1: to get a disease. And so they did what people 263 00:16:23,600 --> 00:16:25,920 Speaker 1: commonly do in the laboratory with mice. It's a lot 264 00:16:25,920 --> 00:16:29,400 Speaker 1: easier with mice. But they did a back cross with 265 00:16:29,480 --> 00:16:34,000 Speaker 1: the dogs in order to see if they would then 266 00:16:34,160 --> 00:16:38,120 Speaker 1: generate puppies that had this same sort of blindness as 267 00:16:38,160 --> 00:16:43,560 Speaker 1: the original founder dog, and lo and behold, they had 268 00:16:43,800 --> 00:16:46,880 Speaker 1: three puppies that were born. All three of them were blind, 269 00:16:47,960 --> 00:16:53,200 Speaker 1: and in fact we named them Lancelot, Guenevere and King 270 00:16:53,440 --> 00:16:58,800 Speaker 1: Arthur because their grandfather had been named Merlin. And these 271 00:16:58,840 --> 00:17:02,720 Speaker 1: were the puppies that we then used to test whether 272 00:17:02,840 --> 00:17:08,040 Speaker 1: gene therapy could reverse their blindness, and that was what 273 00:17:08,240 --> 00:17:12,840 Speaker 1: led to the Eureka moment. These dogs were maintained by 274 00:17:12,960 --> 00:17:17,720 Speaker 1: the Veterinary School at University of Pennsylvania by my collaborators 275 00:17:17,760 --> 00:17:23,600 Speaker 1: Greg Ackland and Gussaghery, and we joined forces together with 276 00:17:23,680 --> 00:17:28,880 Speaker 1: some other investigators to test the possibility that gene therapy 277 00:17:29,119 --> 00:17:33,960 Speaker 1: could rescue the vision in these little puppies. And we 278 00:17:34,040 --> 00:17:39,080 Speaker 1: had three animals available, we went ahead and injected them 279 00:17:39,480 --> 00:17:43,280 Speaker 1: and the results were nothing short of miraculous Within a 280 00:17:43,320 --> 00:17:48,199 Speaker 1: few weeks, these puppies who had to be led to 281 00:17:48,280 --> 00:17:51,040 Speaker 1: a water bowl because they couldn't see where it was, 282 00:17:51,119 --> 00:17:53,199 Speaker 1: who would just crunch in a corner because they were 283 00:17:53,280 --> 00:17:57,200 Speaker 1: scared of bumping themselves if they walked into a door 284 00:17:57,320 --> 00:18:00,880 Speaker 1: a wall. Their behavior changed from radically and they were 285 00:18:00,960 --> 00:18:05,000 Speaker 1: playing with each other, catching toys, playing tug of war, 286 00:18:05,200 --> 00:18:09,440 Speaker 1: running around, wagging their tails. It was just stunning result, 287 00:18:10,040 --> 00:18:14,320 Speaker 1: and it was the sort of thing which scientists it 288 00:18:14,400 --> 00:18:16,639 Speaker 1: hardly ever gets to see in a career. It was 289 00:18:16,720 --> 00:18:22,440 Speaker 1: really a dream result, a Eureka moment, And unfortunately, at 290 00:18:22,440 --> 00:18:25,560 Speaker 1: that point in time, it was very difficult to get 291 00:18:25,640 --> 00:18:29,159 Speaker 1: funding to carry out the next set of steps that 292 00:18:29,240 --> 00:18:32,280 Speaker 1: we wanted to carry out, which would be to go 293 00:18:32,440 --> 00:18:37,920 Speaker 1: to a human clinical trial. Match Doctor Bennett's clinical trials 294 00:18:37,960 --> 00:18:41,440 Speaker 1: are threatened by a lack of funding across all gene 295 00:18:41,480 --> 00:18:50,200 Speaker 1: therapy research. 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Doctor Bennet had developed some promising data 304 00:19:27,320 --> 00:19:31,280 Speaker 1: with the Swedish Briard Dogs, but funding for gene therapy 305 00:19:31,359 --> 00:19:35,959 Speaker 1: dried up. Then along came her longtime colleague and friend, 306 00:19:36,480 --> 00:19:40,359 Speaker 1: doctor Catherine High, with a knock at her door. Here's 307 00:19:40,440 --> 00:19:44,959 Speaker 1: doctor High. I went to the CEO of Children's Hospital 308 00:19:44,960 --> 00:19:48,160 Speaker 1: of Philadelphia, where I was working then, and asked if 309 00:19:48,160 --> 00:19:51,199 Speaker 1: he thought that he could put resources into this, and, 310 00:19:51,359 --> 00:19:57,280 Speaker 1: to my continuing surprise, even many years later, after thinking 311 00:19:57,320 --> 00:19:59,119 Speaker 1: about it for a week, he agreed to do that. 312 00:20:00,080 --> 00:20:04,080 Speaker 1: He told me, he said, he said, I'm going to 313 00:20:04,160 --> 00:20:09,040 Speaker 1: invest in gene therapy because if it works, it will 314 00:20:09,119 --> 00:20:13,639 Speaker 1: be very important for genetic disease, and that means it 315 00:20:13,680 --> 00:20:17,240 Speaker 1: will be important for children's hospitals. But I have one 316 00:20:17,280 --> 00:20:20,400 Speaker 1: condition for you, and that is that you cannot use 317 00:20:20,480 --> 00:20:22,240 Speaker 1: all the resources I'm going to provide to you to 318 00:20:22,280 --> 00:20:24,280 Speaker 1: work on hemophilia. You have to work on some other 319 00:20:24,280 --> 00:20:28,480 Speaker 1: genetic diseases that affect children, and I said, Okay, well, 320 00:20:28,480 --> 00:20:31,760 Speaker 1: then I know exactly what I'm going to do because 321 00:20:31,800 --> 00:20:35,520 Speaker 1: I had been a long time colleague of gene Bennett's 322 00:20:36,240 --> 00:20:40,560 Speaker 1: in terms of our interest in working on gene therapy approaches, 323 00:20:41,000 --> 00:20:45,919 Speaker 1: and I knew how great her dog model data was. 324 00:20:46,000 --> 00:20:50,560 Speaker 1: So she had essentially cured this rare form of inherited 325 00:20:50,600 --> 00:20:56,120 Speaker 1: blindness in dogs, and I really felt that that would 326 00:20:56,160 --> 00:21:01,520 Speaker 1: have an excellent chance of translating into success in people. 327 00:21:02,080 --> 00:21:06,639 Speaker 1: Doctor Bennett. I was totally shocked when she knocked on 328 00:21:06,680 --> 00:21:10,440 Speaker 1: my office door one day July two thousand and five 329 00:21:11,200 --> 00:21:13,800 Speaker 1: and asked, Jean, how would you like to run a 330 00:21:13,800 --> 00:21:18,679 Speaker 1: clinical trial at shop. It was just shocking. I was 331 00:21:18,760 --> 00:21:21,080 Speaker 1: not prepared for that at all, but it was just 332 00:21:21,119 --> 00:21:27,720 Speaker 1: such a wonderfully surprising shock. Is that I responded within milliseconds, 333 00:21:27,760 --> 00:21:30,440 Speaker 1: and from then on we've been tied at the hip 334 00:21:30,600 --> 00:21:35,360 Speaker 1: with this common goal of bringing together our complimentary expertise 335 00:21:35,400 --> 00:21:40,199 Speaker 1: and making this happen safely and fast. So doctor Catherine 336 00:21:40,240 --> 00:21:43,440 Speaker 1: High was successful in finding the funding to the Children's 337 00:21:43,520 --> 00:21:46,399 Speaker 1: Hospital of Philadelphia. Doctor Bennett was ready to move from 338 00:21:46,400 --> 00:21:50,320 Speaker 1: her trous with dogs to clinical trials with people. The 339 00:21:50,480 --> 00:21:54,159 Speaker 1: year is two thousand and seven. Doctor High explains what 340 00:21:54,280 --> 00:21:58,080 Speaker 1: happened next. The first three subjects were all adults. That 341 00:21:58,200 --> 00:22:00,480 Speaker 1: was the agreement that we had reached with both the 342 00:22:00,520 --> 00:22:05,560 Speaker 1: local and the federal regulatory agencies, and because that looked safe, 343 00:22:05,720 --> 00:22:08,959 Speaker 1: then after that we were able to begin enrolling children. 344 00:22:09,720 --> 00:22:13,159 Speaker 1: So we enrolled twelve people in the phase one to 345 00:22:13,359 --> 00:22:19,520 Speaker 1: study a mix of pediatric and adult participants, and the 346 00:22:19,680 --> 00:22:24,840 Speaker 1: data looked really strong, and so then the problem became, 347 00:22:26,280 --> 00:22:29,280 Speaker 1: how do we take the information that we're getting in 348 00:22:29,280 --> 00:22:33,639 Speaker 1: this clinical trial and set up a way of measuring 349 00:22:33,680 --> 00:22:40,120 Speaker 1: what we're seeing in a way that's quantitative, reproducible, and 350 00:22:40,280 --> 00:22:43,919 Speaker 1: convincing to other people so that we can actually get 351 00:22:43,920 --> 00:22:50,480 Speaker 1: a drug approved. So we worked closely actually with the 352 00:22:50,600 --> 00:22:55,080 Speaker 1: regulators to set up a new endpoint. So this is 353 00:22:55,760 --> 00:22:59,520 Speaker 1: I think a very important point because if gene therapy 354 00:22:59,760 --> 00:23:04,520 Speaker 1: is really to realize all the potential represented in the 355 00:23:04,600 --> 00:23:09,520 Speaker 1: Human Genome Project, we are going to be tackling diseases 356 00:23:10,800 --> 00:23:13,920 Speaker 1: that currently have no treatment. And if a disease has 357 00:23:13,960 --> 00:23:19,119 Speaker 1: no treatment, it means that nobody has figured out what 358 00:23:19,359 --> 00:23:23,240 Speaker 1: are the clinical endpoints that you have to develop evidence 359 00:23:23,280 --> 00:23:28,280 Speaker 1: for that will tell other people and the regulators and 360 00:23:28,320 --> 00:23:33,920 Speaker 1: clinicians and everybody, Yes, this drug is working. So that's 361 00:23:33,920 --> 00:23:36,160 Speaker 1: what we had to tackle. We had to tackle, how 362 00:23:36,160 --> 00:23:39,320 Speaker 1: do we make a novel end point for people with 363 00:23:39,560 --> 00:23:47,440 Speaker 1: this specific clinical condition where they have very diminished sensitivity 364 00:23:47,600 --> 00:23:51,800 Speaker 1: to light and if you can't pick up light that's 365 00:23:51,840 --> 00:23:56,720 Speaker 1: coming in. You may be able to operate okay in 366 00:23:56,760 --> 00:24:00,760 Speaker 1: a location on the beach on a right sunny day, 367 00:24:01,560 --> 00:24:05,840 Speaker 1: but if you're in the setting of dimmer light, then 368 00:24:06,119 --> 00:24:09,200 Speaker 1: you can't see Doctor High. Doctor Bennett worked through these 369 00:24:09,240 --> 00:24:13,320 Speaker 1: clinical challenges and moved to clinical trials with patients who 370 00:24:13,320 --> 00:24:18,639 Speaker 1: are diagnosed with this rare disease, libre congenital amurosis. The 371 00:24:18,760 --> 00:24:23,160 Speaker 1: four original patients came from Italy, and in fact the 372 00:24:23,200 --> 00:24:26,840 Speaker 1: first two were twins, fraternal twins. The first was a girl, 373 00:24:26,960 --> 00:24:30,560 Speaker 1: the second was a boy, and they had all been 374 00:24:30,600 --> 00:24:36,040 Speaker 1: diagnosed with LCIA or Labor's congenital amaurosis. That's a disease 375 00:24:36,200 --> 00:24:39,840 Speaker 1: which a form of impaired vision, which it fits within 376 00:24:39,880 --> 00:24:43,800 Speaker 1: the classification of retinitis pigmentosa. But it's the most severe 377 00:24:43,960 --> 00:24:49,399 Speaker 1: form of retinitis pigmentosa because it's found at birth, so 378 00:24:49,800 --> 00:24:55,320 Speaker 1: babies with LA don't see the same detail that normal 379 00:24:55,359 --> 00:25:00,560 Speaker 1: sighted babies would and they are very very light impaired. 380 00:25:00,960 --> 00:25:03,920 Speaker 1: In fact, if they are in a room where they 381 00:25:04,080 --> 00:25:06,440 Speaker 1: can look at the sun or look at some very 382 00:25:06,520 --> 00:25:10,040 Speaker 1: bright object, their eyes tend to look in that direction 383 00:25:10,119 --> 00:25:13,880 Speaker 1: because they don't see regular room lights, or they don't 384 00:25:13,880 --> 00:25:17,480 Speaker 1: see well in lighting that people use generally in their homes. 385 00:25:18,280 --> 00:25:21,639 Speaker 1: They are attracted to very very bright objects. They have 386 00:25:21,880 --> 00:25:28,000 Speaker 1: very poor resolution, and that's reflected by testing visual acuity. 387 00:25:28,160 --> 00:25:31,959 Speaker 1: They don't see letters on an eye chart or for children, 388 00:25:31,960 --> 00:25:35,800 Speaker 1: they don't see pictures that are drawn very largely. They 389 00:25:36,119 --> 00:25:41,120 Speaker 1: have very poor side vision and whatever they have, however, 390 00:25:42,280 --> 00:25:46,200 Speaker 1: gets progressively worse because this is also a degenerative condition. 391 00:25:46,800 --> 00:25:50,600 Speaker 1: As the cells in the retina die off, they lose 392 00:25:50,880 --> 00:25:53,880 Speaker 1: what they have, even though that's poor to begin with. 393 00:25:54,760 --> 00:25:58,960 Speaker 1: So these first four Italian the first three Italians who 394 00:25:59,480 --> 00:26:03,280 Speaker 1: enrolled in our study were adults, as mandated by our 395 00:26:03,400 --> 00:26:07,600 Speaker 1: local institutional review board, who wanted to make sure that 396 00:26:07,960 --> 00:26:13,040 Speaker 1: this was tested first in individuals who could fully understand 397 00:26:13,080 --> 00:26:18,080 Speaker 1: what they were agreeing to participate in, give proper informed consent, 398 00:26:19,000 --> 00:26:22,360 Speaker 1: and to make sure that the intervention was safe enough 399 00:26:22,400 --> 00:26:26,480 Speaker 1: to then move forward to what are called quote vulnerable 400 00:26:26,720 --> 00:26:31,679 Speaker 1: subjects I eat children who may not understand all of 401 00:26:31,720 --> 00:26:36,760 Speaker 1: the details of the study. So these individuals, the first 402 00:26:36,800 --> 00:26:39,960 Speaker 1: two individuals were in their twenties when they came here 403 00:26:40,000 --> 00:26:43,040 Speaker 1: and the third was nineteen years old, and they each 404 00:26:43,119 --> 00:26:48,520 Speaker 1: stayed for several weeks. They had testing before their injection 405 00:26:48,680 --> 00:26:52,119 Speaker 1: and after their injection, and then they came for follow 406 00:26:52,240 --> 00:26:57,160 Speaker 1: up visits at regular intervals three months, six months, nine months, 407 00:26:57,240 --> 00:27:01,480 Speaker 1: a year, and so forth. So it was just a 408 00:27:01,760 --> 00:27:05,560 Speaker 1: huge contribution that they gave in terms of their time 409 00:27:05,960 --> 00:27:10,680 Speaker 1: investment to participate in these studies which were aimed at 410 00:27:11,000 --> 00:27:13,840 Speaker 1: both looking at the safety of this intervention and also 411 00:27:13,920 --> 00:27:19,200 Speaker 1: whether it worked. And of course these individuals were born 412 00:27:19,400 --> 00:27:24,959 Speaker 1: severely vision impaired, so they traveled with a family member 413 00:27:25,040 --> 00:27:28,680 Speaker 1: or two. Sometimes we got to know their families really, 414 00:27:28,720 --> 00:27:31,280 Speaker 1: really well because of all the time that they spent 415 00:27:31,920 --> 00:27:37,320 Speaker 1: traveling back and forth here. So the first individual who 416 00:27:37,320 --> 00:27:41,640 Speaker 1: received the injection from Italy was one of those volunteers 417 00:27:41,680 --> 00:27:46,280 Speaker 1: to have her second eye injected first, and she did 418 00:27:46,320 --> 00:27:49,440 Speaker 1: not have a family member who could stay here for 419 00:27:49,640 --> 00:27:52,960 Speaker 1: three months because they all had jobs that required that 420 00:27:53,000 --> 00:27:56,640 Speaker 1: they be back in Italy for that time period. So 421 00:27:56,680 --> 00:28:00,359 Speaker 1: we said, why don't you stay with us, and actually 422 00:28:00,400 --> 00:28:04,840 Speaker 1: stayed in her house and she had the injection, and 423 00:28:05,200 --> 00:28:08,119 Speaker 1: a couple of days later she came downstairs and looked 424 00:28:08,160 --> 00:28:12,639 Speaker 1: in the mirror and said, Momma, she saw her face 425 00:28:12,800 --> 00:28:18,400 Speaker 1: for the first time. It was just amazing watching her 426 00:28:18,680 --> 00:28:22,880 Speaker 1: learn how to use her new vision. From day to day, 427 00:28:23,080 --> 00:28:26,800 Speaker 1: she'd look out the window and say, what is that? 428 00:28:27,000 --> 00:28:30,600 Speaker 1: And it would be branches on a tree, or we'd 429 00:28:30,600 --> 00:28:34,160 Speaker 1: be driving and she'd see a reflection from a pond. 430 00:28:34,640 --> 00:28:37,600 Speaker 1: Each thing she had to learn what it was, and 431 00:28:37,720 --> 00:28:41,040 Speaker 1: it was like a child seeing things for the first time, 432 00:28:41,360 --> 00:28:44,440 Speaker 1: but in fact she was twenty eight years old and 433 00:28:44,880 --> 00:28:48,680 Speaker 1: telling the brain what that was that the person was seeing. 434 00:28:49,440 --> 00:28:53,440 Speaker 1: So that was really quite remarkable and I'll never forget it. 435 00:28:53,920 --> 00:28:56,320 Speaker 1: I think one of the remarkable things about this story, 436 00:28:56,880 --> 00:28:59,840 Speaker 1: really this journey, is to understand that people with the 437 00:29:00,200 --> 00:29:04,480 Speaker 1: rare genetic mutation could receive one injection and have their 438 00:29:04,480 --> 00:29:10,480 Speaker 1: site restored almost overnight. It's truly a remarkable scientific breakthrough, 439 00:29:10,920 --> 00:29:13,360 Speaker 1: Doctor Barnett, can you tell us a few stories of 440 00:29:13,400 --> 00:29:18,080 Speaker 1: patients who were treated and what their experience was. Interestingly enough, 441 00:29:18,200 --> 00:29:23,680 Speaker 1: there were three patients who had near simultaneous injections around 442 00:29:23,680 --> 00:29:28,440 Speaker 1: the United States, one in Boston, one in Miami, and 443 00:29:28,560 --> 00:29:32,280 Speaker 1: one in LA and I believe it was the Boston 444 00:29:32,320 --> 00:29:37,160 Speaker 1: patient who was first. The Boston patient was injected at 445 00:29:37,200 --> 00:29:42,960 Speaker 1: Massachusetts Iron Ear Infirmary, which is a Harvard institution by 446 00:29:43,440 --> 00:29:47,160 Speaker 1: doctor Jason Commander, who's a retinal surgeon who would actually 447 00:29:47,400 --> 00:29:50,600 Speaker 1: come here to learn how to do these subretinal injections. 448 00:29:50,680 --> 00:29:54,160 Speaker 1: From my husband, I never tire of hearing these stories. 449 00:29:54,560 --> 00:29:59,760 Speaker 1: For example, Duboya treated at Massioneer now rides his bike 450 00:30:00,200 --> 00:30:04,120 Speaker 1: and can see things in school and goes to his 451 00:30:04,240 --> 00:30:10,680 Speaker 1: friend's houses by himself. The child in Miami has thrown 452 00:30:10,680 --> 00:30:14,400 Speaker 1: away his cane, which was his lifelong dream, his blind cane, 453 00:30:14,920 --> 00:30:18,000 Speaker 1: and is doing things that he never could do before. 454 00:30:18,640 --> 00:30:21,960 Speaker 1: The little girl in Los Angeles and had her injection 455 00:30:22,080 --> 00:30:25,640 Speaker 1: just before Halloween, and she was able to go trick 456 00:30:25,680 --> 00:30:28,920 Speaker 1: her treating and could not get over the fact that 457 00:30:28,960 --> 00:30:31,640 Speaker 1: she could see in the dark. She was not as 458 00:30:31,680 --> 00:30:34,240 Speaker 1: interested in the Halloween treats as the fact that she 459 00:30:34,320 --> 00:30:36,640 Speaker 1: could go down the street and see where she was going. 460 00:30:37,640 --> 00:30:41,240 Speaker 1: The dramatic importance of a scientific breakthrough is that it 461 00:30:41,320 --> 00:30:44,960 Speaker 1: suddenly sends a signal that maybe there's something really worth 462 00:30:45,040 --> 00:30:47,800 Speaker 1: investing in and all of a sudden, more and more 463 00:30:47,880 --> 00:30:50,680 Speaker 1: people come to an area because they now have hope 464 00:30:51,200 --> 00:30:55,440 Speaker 1: and they have a practical, realistic scientific basis for that hope. 465 00:30:56,240 --> 00:30:58,400 Speaker 1: Well we've read about this story is that it has 466 00:30:58,440 --> 00:31:01,280 Speaker 1: led to support of other research who are focused on 467 00:31:01,320 --> 00:31:05,120 Speaker 1: finding cures for blindness. Talk about it. Can you tell 468 00:31:05,200 --> 00:31:09,280 Speaker 1: us how the field has expanded? The availability of lextern 469 00:31:09,320 --> 00:31:14,720 Speaker 1: has already made a difference because people are now educating 470 00:31:14,800 --> 00:31:19,000 Speaker 1: their ophthalmologists saying, you know, there is this new drug, 471 00:31:19,360 --> 00:31:23,160 Speaker 1: do I qualify? And doctors are realizing that these individuals 472 00:31:23,160 --> 00:31:27,440 Speaker 1: with these inherited forms of blindness need to be studied 473 00:31:27,480 --> 00:31:31,920 Speaker 1: and genotyped to see what the genetic basis is of 474 00:31:31,960 --> 00:31:35,240 Speaker 1: their disease, to see if they qualify, and also whether 475 00:31:35,280 --> 00:31:38,440 Speaker 1: they qualify for other studies that we think are going 476 00:31:38,520 --> 00:31:42,560 Speaker 1: to take place in the near future. So it's also 477 00:31:43,160 --> 00:31:48,160 Speaker 1: fueled research in developing treatments for the other forms of 478 00:31:48,160 --> 00:31:51,840 Speaker 1: inherited blindness. There are more than two hundred and sixty 479 00:31:51,960 --> 00:31:57,520 Speaker 1: different genetic forms of blindness, and many of these are 480 00:31:57,680 --> 00:32:02,720 Speaker 1: now targets for developing gene based therapies, and many of 481 00:32:02,760 --> 00:32:05,400 Speaker 1: them are now actually in clinical trials. So I think 482 00:32:05,400 --> 00:32:08,800 Speaker 1: it paved a way for other groups to be able 483 00:32:08,840 --> 00:32:14,160 Speaker 1: to try to develop interventions based on gene delivery. Finally, 484 00:32:14,200 --> 00:32:17,320 Speaker 1: I think that this is going to be a stepping 485 00:32:17,320 --> 00:32:21,680 Speaker 1: stone for developing gene based treatments for blinding diseases that 486 00:32:21,760 --> 00:32:25,520 Speaker 1: affect a large number of people. I think there are 487 00:32:25,640 --> 00:32:30,600 Speaker 1: going to be ways to develop an intervention which could 488 00:32:30,800 --> 00:32:35,160 Speaker 1: address multiple different genetic defects, for example, to target a 489 00:32:35,200 --> 00:32:41,880 Speaker 1: specific biochemical pathway or a support pathway where a somewhat 490 00:32:41,960 --> 00:32:45,720 Speaker 1: generic therapy could be delivered which would prevent the disease 491 00:32:45,800 --> 00:32:50,560 Speaker 1: from progressing. Based on what we know of the biology 492 00:32:50,560 --> 00:32:53,640 Speaker 1: of the retina, and so I'm hopeful that this will 493 00:32:53,760 --> 00:32:57,200 Speaker 1: end up helping a lot of different people who have 494 00:32:57,240 --> 00:33:02,760 Speaker 1: different forms of blindness. Coming up, we'll hear from a 495 00:33:02,760 --> 00:33:06,760 Speaker 1: patient whose life was fundamentally changed for the better because 496 00:33:06,800 --> 00:33:15,280 Speaker 1: of doctor Bennett and doctor Hy's breakthrough discovery. The Westwood 497 00:33:15,280 --> 00:33:18,800 Speaker 1: One podcast Network, A Daily Wire's Ben Shapiro show. What 498 00:33:18,880 --> 00:33:21,680 Speaker 1: I do see is a system that is wildly broken. 499 00:33:21,840 --> 00:33:24,800 Speaker 1: When a lot of people who were brought here as 500 00:33:24,920 --> 00:33:27,040 Speaker 1: children in our net benefits to the United States, it 501 00:33:27,040 --> 00:33:28,560 Speaker 1: takes four ever for them to get a citizenship and 502 00:33:28,600 --> 00:33:31,520 Speaker 1: We're fucking blanket amnesty for people who come across the 503 00:33:31,520 --> 00:33:34,920 Speaker 1: border now. The Ben Shapiro Show download and subscribe at 504 00:33:34,960 --> 00:33:38,400 Speaker 1: Apple Podcasts, Google Play, and the Westwood one Podcast App 505 00:33:38,600 --> 00:33:49,680 Speaker 1: free free from the Westwood one Podcast Network. We've talked 506 00:33:49,680 --> 00:33:51,840 Speaker 1: a lot in this episode about the process of going 507 00:33:51,920 --> 00:33:55,840 Speaker 1: through clinical trials to bring a drug to market, but 508 00:33:55,960 --> 00:33:59,320 Speaker 1: the reason these doctors do what they do is ultimately 509 00:33:59,360 --> 00:34:03,200 Speaker 1: to affect the lives of their patients. Here's doctor Bennett 510 00:34:03,240 --> 00:34:08,000 Speaker 1: and Doctor Hy's patient, a teenage musician named Christian. My 511 00:34:08,120 --> 00:34:11,360 Speaker 1: name is Christian. Guardina was born the very rare retinal 512 00:34:11,400 --> 00:34:14,880 Speaker 1: disease called Lieber's congenital amaurosis, and what that is is 513 00:34:14,920 --> 00:34:17,600 Speaker 1: a very rare disease that causes you to go blind 514 00:34:17,800 --> 00:34:20,279 Speaker 1: over time. But we didn't know that when I was 515 00:34:20,320 --> 00:34:22,799 Speaker 1: a baby. What my mom heard when she noticed there 516 00:34:22,880 --> 00:34:25,520 Speaker 1: was something wrong was that I would have the type 517 00:34:25,520 --> 00:34:30,240 Speaker 1: of LCA that either improves slightly over time or remain stable. 518 00:34:30,680 --> 00:34:33,520 Speaker 1: But about eleven years old, we started to notice a 519 00:34:33,640 --> 00:34:36,439 Speaker 1: decline in my vision and we were really concerned, so 520 00:34:36,960 --> 00:34:41,120 Speaker 1: we got tested at the children's hospital. We met doctor Bennett, 521 00:34:41,400 --> 00:34:43,600 Speaker 1: and that's where we found out that I would be 522 00:34:43,640 --> 00:34:46,440 Speaker 1: going completely blind. By the time I hit age fifteen 523 00:34:46,480 --> 00:34:49,360 Speaker 1: to thirty, I started to get older and I started 524 00:34:49,360 --> 00:34:52,279 Speaker 1: to see my friends playing outside at nighttime. I was 525 00:34:52,320 --> 00:34:54,799 Speaker 1: like always had to sit inside and said, Mom, why 526 00:34:54,840 --> 00:34:56,640 Speaker 1: why can't I, you know, why can't I play outside? 527 00:34:56,640 --> 00:34:58,520 Speaker 1: Why can't I go with them? And that's when I 528 00:34:58,520 --> 00:35:00,640 Speaker 1: really started to notice something was wrong with my vision. 529 00:35:01,800 --> 00:35:04,840 Speaker 1: I always use music as something to cope with that, 530 00:35:05,640 --> 00:35:08,080 Speaker 1: you know, my body used my ears over my eyes. 531 00:35:08,560 --> 00:35:10,719 Speaker 1: But grown up with LCA was hard, mostly because I 532 00:35:10,760 --> 00:35:13,120 Speaker 1: wanted to do everything else that all the other kids 533 00:35:13,160 --> 00:35:15,840 Speaker 1: were doing, but I couldn't. I think one of the 534 00:35:15,880 --> 00:35:18,600 Speaker 1: biggest things that made me want to move forward with 535 00:35:18,640 --> 00:35:21,600 Speaker 1: the clinical trials. And even though it was experimental and 536 00:35:21,640 --> 00:35:25,359 Speaker 1: I was very nervous, We've already found out that I'm 537 00:35:25,400 --> 00:35:27,000 Speaker 1: going to be going blind by the time I hit 538 00:35:27,040 --> 00:35:30,319 Speaker 1: age fifteen to thirty, nothing I could do about it. 539 00:35:30,840 --> 00:35:33,600 Speaker 1: So there was something I could do about it. I 540 00:35:33,680 --> 00:35:37,240 Speaker 1: was going to take that opportunity, and there was that hope, 541 00:35:37,760 --> 00:35:39,960 Speaker 1: and it was all we had. I didn't want to 542 00:35:39,960 --> 00:35:43,399 Speaker 1: go blind. During the gene therapy, we'd go every few 543 00:35:43,440 --> 00:35:45,279 Speaker 1: weeks for a little bit. It was a lot of 544 00:35:45,360 --> 00:35:48,760 Speaker 1: visits and it was a lot of testing. So these 545 00:35:48,800 --> 00:35:52,480 Speaker 1: people really really helped me feel a sense of security 546 00:35:52,520 --> 00:35:56,640 Speaker 1: with that, and I trusted them. Doctor High, doctor Bennett 547 00:35:56,680 --> 00:36:00,279 Speaker 1: are all such incredible people, incredible at what they do. 548 00:36:00,360 --> 00:36:02,799 Speaker 1: They're some of the best doctors I've ever met. So 549 00:36:02,840 --> 00:36:05,680 Speaker 1: as soon as we took that patch off, I remember 550 00:36:06,200 --> 00:36:08,359 Speaker 1: at the hotel, I saw these patterns on the floor. 551 00:36:08,400 --> 00:36:11,320 Speaker 1: I was like, what what what? What's on the floor, 552 00:36:11,360 --> 00:36:14,240 Speaker 1: What's going on over here? I've been to this hotel 553 00:36:14,400 --> 00:36:18,040 Speaker 1: for years before the gene therapy. I was like, I 554 00:36:18,200 --> 00:36:22,000 Speaker 1: never remembered any patterns on this floor, and my mom 555 00:36:22,080 --> 00:36:23,959 Speaker 1: was like, those patterns have been here the whole time. 556 00:36:24,120 --> 00:36:27,520 Speaker 1: I was like, okay. So that's when I really started 557 00:36:27,560 --> 00:36:30,879 Speaker 1: to notice the gene therapy working, and that was really cool. 558 00:36:31,719 --> 00:36:34,400 Speaker 1: Fast forward a few weeks, we go home and I 559 00:36:34,520 --> 00:36:36,600 Speaker 1: see the moon for the first time. Out of performance, 560 00:36:37,400 --> 00:36:41,600 Speaker 1: which was absolutely amazing. I was at this amusement park 561 00:36:41,960 --> 00:36:45,359 Speaker 1: called Adventure End and I performed there every Wednesday nights 562 00:36:45,560 --> 00:36:49,600 Speaker 1: during the summer, and this one night, I'm on the 563 00:36:49,600 --> 00:36:51,959 Speaker 1: side of the stage. It's an outdoor thing. I'm looking 564 00:36:52,040 --> 00:36:55,120 Speaker 1: up at the sky and they're calling my name on stage. 565 00:36:56,200 --> 00:36:59,680 Speaker 1: Christian Christian Christian, I'm not paying attention. I'm looking straight 566 00:36:59,719 --> 00:37:02,240 Speaker 1: up at the sky, and the MC of the show 567 00:37:02,280 --> 00:37:04,200 Speaker 1: comes down. It was like, Christian, your turn to perform. 568 00:37:04,239 --> 00:37:06,720 Speaker 1: You gotta go up. I was like, is that the moon? 569 00:37:07,360 --> 00:37:10,160 Speaker 1: It was probably one of the biggest nights in my life. 570 00:37:10,160 --> 00:37:13,960 Speaker 1: I don't think I'll ever forget that night before geene therapy, 571 00:37:14,000 --> 00:37:16,640 Speaker 1: when I found out that I was gonna go completely 572 00:37:16,640 --> 00:37:18,920 Speaker 1: blind by the time I hit age fifteen to thirty, 573 00:37:18,960 --> 00:37:22,640 Speaker 1: I was terrified of losing everything that I had seen. 574 00:37:23,280 --> 00:37:25,320 Speaker 1: I didn't want to forget what my mother looked like. 575 00:37:25,360 --> 00:37:27,759 Speaker 1: I didn't want to forget what my family looked like. 576 00:37:28,600 --> 00:37:31,480 Speaker 1: And now that I got this gene therapy, I don't 577 00:37:31,560 --> 00:37:37,480 Speaker 1: have to Doctor Bennett and doctor hy their kind. They are, 578 00:37:38,520 --> 00:37:42,600 Speaker 1: like I said, incredible, incredible people. I really can't emphasize 579 00:37:42,600 --> 00:37:46,400 Speaker 1: that enough. Like they really changed my life just because 580 00:37:46,440 --> 00:37:48,960 Speaker 1: of the human beings that they are, let alone the 581 00:37:49,040 --> 00:37:51,800 Speaker 1: doctors that they are, but just because of the human 582 00:37:51,960 --> 00:37:55,960 Speaker 1: beings that they are. And also gene therapy could pay 583 00:37:56,040 --> 00:37:59,120 Speaker 1: the way for other sciences, other diseases. There's so much 584 00:37:59,120 --> 00:38:02,160 Speaker 1: that could come out of this, and it is absolutely 585 00:38:02,160 --> 00:38:05,520 Speaker 1: incredible what they did with this technology and the science, 586 00:38:05,520 --> 00:38:08,359 Speaker 1: and honestly, it blows my mind. You know, I'm just 587 00:38:08,360 --> 00:38:11,680 Speaker 1: this eighteen year old. I don't understand it blows my mind. 588 00:38:12,120 --> 00:38:16,160 Speaker 1: It is so cool. I hope they'll take two things 589 00:38:16,200 --> 00:38:21,960 Speaker 1: away from today's episode. First that these cures are about 590 00:38:22,040 --> 00:38:27,040 Speaker 1: human beings, people, folks whose lives are made profoundly different 591 00:38:27,680 --> 00:38:32,240 Speaker 1: because something happens in science that translates into their health. 592 00:38:33,200 --> 00:38:37,760 Speaker 1: And second that achieving that kind of a breakthrough takes 593 00:38:37,920 --> 00:38:44,480 Speaker 1: years dedication, smart people who work endless hours, sometimes with 594 00:38:44,680 --> 00:38:49,160 Speaker 1: enormous frustration. Frustration because what they thought would work didn't 595 00:38:49,160 --> 00:38:50,800 Speaker 1: and they got to go back to the drawing board. 596 00:38:51,360 --> 00:38:55,160 Speaker 1: Frustration because they've got to deal with government bureaucracies to 597 00:38:55,160 --> 00:38:59,920 Speaker 1: get approval, frustration because funding drives up just as they 598 00:39:00,040 --> 00:39:03,520 Speaker 1: think they're about to make a breakthrough. And the pioneers, 599 00:39:03,560 --> 00:39:08,080 Speaker 1: the folks who courageously go out there year after year 600 00:39:09,080 --> 00:39:14,480 Speaker 1: because of their dedication change lives. It's always amazing to 601 00:39:14,520 --> 00:39:17,560 Speaker 1: me to have a chance to share with you the 602 00:39:17,680 --> 00:39:22,319 Speaker 1: kind of personal stories were suddenly a young man who 603 00:39:22,320 --> 00:39:26,399 Speaker 1: couldn't see looks at the moon for the very first time, 604 00:39:27,480 --> 00:39:30,719 Speaker 1: to listen to a mother who had no hope for 605 00:39:30,800 --> 00:39:36,440 Speaker 1: her daughter, but absolute determination that she would find hope. 606 00:39:38,000 --> 00:39:43,240 Speaker 1: This is what health americash out, finding the cures, helping 607 00:39:43,280 --> 00:39:47,120 Speaker 1: people have complete lives, having people have healthy, long lives. 608 00:39:47,920 --> 00:39:51,520 Speaker 1: When I was Speaker, we doubled the funding for the 609 00:39:51,600 --> 00:39:56,240 Speaker 1: Nationalist to its health. The number of people alive today 610 00:39:57,120 --> 00:40:01,680 Speaker 1: because of your generosity as a taxpayer is astounding. And 611 00:40:01,800 --> 00:40:04,600 Speaker 1: over the next twenty or thirty years, the number of 612 00:40:04,640 --> 00:40:09,160 Speaker 1: breakthroughs are going to grow and grow and grow, whether 613 00:40:09,200 --> 00:40:16,040 Speaker 1: it's Alzheimer's or sickle cell anemia, hemophilia, cancer, again and again, 614 00:40:16,480 --> 00:40:19,840 Speaker 1: you're going to see story after story, and I hope 615 00:40:20,239 --> 00:40:23,960 Speaker 1: you'll hear that people can be saved. But it's important 616 00:40:23,960 --> 00:40:27,600 Speaker 1: to remember that at the end of the laboratory is 617 00:40:27,640 --> 00:40:31,120 Speaker 1: not a cure. At the end of the laboratory is 618 00:40:31,160 --> 00:40:36,359 Speaker 1: an idea, a breakthrough, a new scientific discovery. And then 619 00:40:36,440 --> 00:40:40,759 Speaker 1: from that point, the treatment in your doctor's office requires 620 00:40:40,760 --> 00:40:45,319 Speaker 1: additional money, additional focus, additional specialties. And that's why this 621 00:40:46,200 --> 00:40:51,000 Speaker 1: unusual American model of a public private partnership where publicly 622 00:40:51,000 --> 00:40:55,200 Speaker 1: we fund enormous amounts of basic research, and then privately 623 00:40:55,600 --> 00:40:59,080 Speaker 1: we raise the money, organize the entrepreneurs and turned that 624 00:40:59,160 --> 00:41:03,560 Speaker 1: basic research and to cure after cure aftercure, and it's 625 00:41:03,640 --> 00:41:07,239 Speaker 1: that combination which has made us the health powerhouse of 626 00:41:07,280 --> 00:41:10,360 Speaker 1: the planet and which offers us, as a team a 627 00:41:10,560 --> 00:41:13,400 Speaker 1: chancel of the next fifteen or twenty years to truly 628 00:41:13,440 --> 00:41:16,840 Speaker 1: have breakthroughs on a scale that will seem like miracles. 629 00:41:20,680 --> 00:41:24,360 Speaker 1: Thank you to my guests doctor Jim Wilson, Doctor Catherine 630 00:41:24,400 --> 00:41:29,360 Speaker 1: High and doctor Jean Bennett and Alison Barant and Christian Guardino. 631 00:41:30,400 --> 00:41:33,600 Speaker 1: You can see the books, articles, and documents that we 632 00:41:33,680 --> 00:41:36,920 Speaker 1: relied on in researching this episode on our show page 633 00:41:37,320 --> 00:41:41,399 Speaker 1: at newtsworld dot com. Newtsworld is produced by Westwood One. 634 00:41:42,000 --> 00:41:46,120 Speaker 1: The executive producer is Debbie Myers. Our producers are Garnsey 635 00:41:46,160 --> 00:41:50,360 Speaker 1: Sloan and Joe De Sanis. Our editor is Robert Barowski. 636 00:41:50,760 --> 00:41:54,560 Speaker 1: Our researcher is Rachel Peterson. The artwork for the show 637 00:41:54,680 --> 00:41:58,680 Speaker 1: was created by Steve Penman. The music was composed by 638 00:41:58,760 --> 00:42:02,440 Speaker 1: Joey Salvia. Special thanks to the team of Gingwishtree sixty, 639 00:42:02,920 --> 00:42:06,880 Speaker 1: to Sanford Health, and to Westwood Ones, Tim Saban and 640 00:42:07,040 --> 00:42:12,319 Speaker 1: Robert Mothers. Please subscribe to Newsworld on Apple Podcasts, Spotify, 641 00:42:12,800 --> 00:42:16,920 Speaker 1: Google Play, or wherever you get entertaining podcasts. On the 642 00:42:16,960 --> 00:42:21,879 Speaker 1: next episode, of Newtsworld. A remarkable, candid and open conversation 643 00:42:22,440 --> 00:42:26,520 Speaker 1: with Congressman Patrick Kennedy, someone who in his own life 644 00:42:26,520 --> 00:42:29,319 Speaker 1: has had the challenge of addiction, someone who's been a 645 00:42:29,320 --> 00:42:33,319 Speaker 1: great leader in terms of mental health parity, dealing with 646 00:42:33,360 --> 00:42:37,399 Speaker 1: addiction and trying to solve the problems of healthcare. I 647 00:42:37,440 --> 00:42:41,759 Speaker 1: think you'll find it a fascinating, an important conversation and 648 00:42:41,920 --> 00:42:45,960 Speaker 1: one that you'll never forget. I am Newt kingwish this 649 00:42:46,200 --> 00:42:46,920 Speaker 1: is Newtsworld.