>> charlie: welcome to our program. tonight, a "charlie rose" special on the brain and blindness. >> as you indicated, there are close to 300 million people worldwide that have various degrees of visual impairment and in the past the only thing you could do for people like that is to give them non-visual guides -- teach them how to read braille, a seeing-eye dog. but recently this has changed. we're sitting here in the midst of revolution in the treatment of macular degeneration and opens the treatment of many kinds of blindness. that is because three major developments have occurred. gene therapy, stem cell therapy and retinal chips. >> charlie: >> charlie: funding for "charlie rose" is provided by the following:

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from our studios in new york city, this is charlie rose. >> charlie: to be blind is not miserable. not to be able to bear blindness, that is miserable. john milton wrote that. more than 285 million people currently live with visual impairment. for many of these cases, there is no cure. yet in recent years there have been breakthroughs in our understanding and treatment of blindness. sanford greenberg lost his vision to glaucoma at age 18 and chairman of the board of governors of johns hopkins wilmer eye institute. jean bennett of the university of pennsylvania, steven schwartz of the jules stein eye institute, herb hart zrenner of the university of tubingen, carla shatz shatz of stafford university school of medicine. i begin with eric to give me afteroverview of our subject tonight.

eric? >> the last program is treatment of deafness. tonight we consider approaches to the treatment of blindness. as in the case with deafness. blindness is not a life-threatening situation, but it's tremendously disabling, and in some ways it's more disabling than deafness because, as you pointed out, there are a number of very important blindness conditions for which there is no treatment. now, why is that so? unlike deafness, the sensory organ for vision, the retina, which is evident in the lower image which shows the eye. the retina lines the inside surface of the eye. that is the most complex sensory organ that we have. in fact, it's not a peripheral organ. it's actually a part of the central nervous system. it's an extension of the central nervous system and as a result has the complexity of central nervous system structure. it's not uniform.

it has a small area in the center that is clear on that image which is called the macular. the macular is the area of greatest visual acuity. if i focus on you, i turn my head, my eyes focus on you and the macular analyzes your features. unfortunately, the cells of the macular are extremely sensitive to damage that leads to blindness and, so, this is a really serious problem, and we, at the moment, have no treatment for that, even though this is a point of great visual acuity. blindness, as you indicated, is a range of conditions. it ranges from complete to partial blindness. complete blindness means that you don't see images, you don't see figures, you don't see people. you can't tell the difference between day and night. this is sandy's condition.

partial blindness varies from having tunnel vision to cloudy vision to having night blindness. now, you can have difficulty with vision from two sources. one is if you have a damage to the visual pathway that goes from the retina into the brain, and carla will discuss that more, but we are not going to focus that as a source of blindness. we'll limit ourselves to disorders of the retina. as you indicated, there are close to 300 million people worldwide that have various degrees of visual impairment, and these categories, treble diseases -- treatable diseases and at the moment things that are essentially untreatable. cataracts, glaucoma and diabetic retinopathy are treatable diseases. cataracts you have cloudy vision. with glaucoma, tunnel vision. with diabetic retinopathy, you have trouble with night vision. what i find tragic is still

there are a number of people who suffer from these conditions and this is a graph only of the developed world -- the united states, canada and europe. because these are inmatly treatable diseases in case of diabetic retinopathy, these are preventible. in the underdeveloped world these are major forces of blindness because people don't have access to medical care and that's an absolute tragedy. in the developed world, the major problem is macular degeneration, and as we indicated, there's a form that counts for most of it called dry macular degeneration, which is an age-related disorder, in which people really lose a lot of their vision, their visual acuity. in the past, the only thing you could do for people like that is to give them non-visual guides. teach them how to read braille, a seeing eye dog, handrails in their apartment, and speech compression devices the kind

sandy developed which makes it easy for them to handle auditory information. but we're sitting here in the middle of a revolution in the treatment of macular degeneration and it opens the treatment of many kinds of blindness. that is because three major developments have occurred. gene therapy, stem cell therapy and retinal chips. gene therapy is an attempt to replace a defective gene with a normally functioning one. genes encode for proteins, either a mutation or the reasons they're not functioning, one can insert a normally functioning gene into the cell and in many cases arrest the situation. in stem cell therapy, you're not only rescuing a gene, you're rescuing a whole cell time. for the last 15, 18 years, it's been impossible to take on the

properties of any cell in your body and with the appropriate chemical mixture, get them to be retinal cells of various kinds and replace a defective stem cell line. with retinal chips, you implant that deep in the retina, and you stimulate directly electrically pathways that lead to the brain, stimulating neural elements that leadso the brain. and we have with us, by coincidence, the three pioneers in this area. jean, appropriately named jean, jean bennett, has pioneered the study of gene therapy. steve pioneered the study of stem cell therapy. eberhart is one of the outstanding leaders in developing retinal chips, these microchips that are really amazing for people who can't respond to these other treatments. we also have the privilege of having two other people here.

carla shatz is the leader in study of visual physiology and development and made wonderful works and will explain the visual system to us. my friend sandy here is amazing. he, as he pointed out, is a college undergraduate, had a severe case of glaucoma from which he became blind, and he will tell us what it's like to be blind. but the amazing thing to me about sandy is, despite the fact he has this tremendous handicap, he's a remarkable human being. he's had a rich, happy and productive life. he's invented things and now he's recently begun to think of how we can help other people who are blind to make their life er? sandy, welcome you again. great to have you at this table. >> thank you. >> charlie: talk to us about becoming blind and the whole sense of the journey for you. >> well, even after 50 years, i can still feel what it was when

i went blind. for many months, i had declining vision, and my mother and i went to see a glaucoma specialist, and he examined me and then turns to me with my mo"owjrg the room and says, well, son, you're going to be blind tomorrow. i guess that was my moment. i think there is a moment that occurs in just about everyone's life that, the instant before bad news is given, after which nothing else in your life will ever be the same and after which you look back on your life and say, my god, i didn't realize how good i've had it until now. before that moment, all, for me, anyway, was possible and all was

rather self-evidently actual. after that moment, zero possible, zero actual. one moment you're at the top of your game. the next moment, there is no game. after surgery, i felt empty, eviscerated, my vital parts cut out, and there was a fair amount of pain in my eyes, but nothing in comparison with the pain in my heart, knowing that my mother had just witnessed her 20-year-old son go blind, you know, his eyes cut open. so there was really no reason to live. so i prayed, you know, in my own way. my girlfriend, sue, i was

convinced was going to leave me. after all, i'm a dropout, had no eyes, no money, no future. but she didn't. and it is really because of her that i'm here tonight with you. and she and my college roommate, art garfunkel, set me, really, on the way out of my horrific wilderness. so i returned. something happened back then not too far from where we're sitting tonight that turned me away from blindness. art and i were walking toward grand central station at rush hour when he abandoned me. so i got down the steps in that

hole in the ground on my own. grand central station, and you're behind, rush hour. so i stumbled to the train that got me back to columbia. as i walked through the large iron gates of that great university, a guy bumps into me and says, oops, excuse me, sir. it was, of course, my roommate, this guy called garfunkel. and he had not abandoned me. he followed me the entire way. and as i bumped into his chest, that instant, i knew that if i could get through the new york city subway system blind, there were no limitations to what i

could accomplish. >> charlie: that's great. all was possible for me. >> charlie: so what do you hope for today? >> what do i hope for today is that eric and the people who are sitting at this table who, in my view, embody the achievements of human genius for them to end this plague, and it's been 6 million years that we humans and our bipedal ancestors have been inflicted by this thing. it's got to end. a group of us started end blindness by 2020. and sue and my family and college roommates art and jerry spire, who is actually a witness to my subway odyssey, have been

with me and for me since we both entered columbia college together, we launched an effort a few years ago to end blindness by 2020. >> charlie: you know, sandy, what i find very interesting about you -- first of all, i must say i'm awed. jerry spires is a very good beginning to life. but how did you accomplish what you accomplished with this handicap? you went to harvard. you made a major invention. you've led a rich life. you're happily married. >> well, i told you, the centerpiece is sue, and then my family, my children, paul and jimmy and katherine have stuck by me, and these two guys, who as you say, i was really fortunate enough to meet when we all started columbia together.

but, you know, it's not all black or dark. i don't want to leave that impression with you, because when you're not distracted by visual images, you develop a life within your mind. so i can see you now, eric, the way i saw art back before i was 19 here in this city, and you look pretty damn good, you know. (laughter) >> charlie: you are why we do this television program, so thank you. let me turn to carla. let me give you an overview so we understand exactly what it is

we're talking about in terms of the human eye. >> well, vision is really miraculous process, and to hear about losing it is also very heart-rending. thank you so much, sandy, for what you just said. and i want to tell you a little bit about the visual system and talk about the basic layout of the system before we get into really talking about the eye. of course, vision starts in the eye, and light enters the eye, and there's a special layer at the back of the eye which is called the retina. you can think of the retina as kind of a fancy digital camera, and the light-sensitive part of the eye, you can kind of think of it is like the pixels in your camera, though the pixels in the camera are made out of silicon, but in your eye they're very specialized nerve cells which

absorb light and convert it to a neural signal. then the information is sent from the retina -- as you can see theae arrows -- to structures in the central part of the brain. this is called the central visual pathways. the first place is the lateral jeniculate nucleus, and the second place the information from the eye goes is to the primary visual cortex. again, we talk about this visual camera idea that this is kind of like the central processing unit where a lot of image analysis happens. so it starts in the eye, but most of the information processing and data crunching for the visual system is happening in these central, visual pathways. now, you can ask, what is the nature of the information that comes out of the eye? what's it like? and i was thinking about how to convey this and i thought, i can't do it any better than this wonderful painting.

you will notice in this painting that the artist has broken up the visual world into thousands of dots of color, and, you know, if you stand up really close, you put your nose to the painting, basically you're just going to see a lot of dots. but the remarkable thing is if you stand back away from the painting, what you actually see is a seated woman. and if we talk about, again, this idea of the retina versus the central visual pathways, the concept here is that the retina is sending thousands of dots to the central visual pathways, and, in fact, each dot is carried by a single nerve fiber, and there are about a million of these nerve fibers going to the central visual pathways. so it's the job of the retina actually to deconstruct the visual world into a pixelated

view of the world, so thousands of pixels. it's the job of the central visual pathways to reconstruct the world by making kind of a seamless view of the world again, kind of connecting the dots back. so we really need our central visual pathways to appreciate that this is a woman seated, but we only need our retina to appreciate the dots. so this is kind of seeing starts in the eye, but really it's the computing power of the rest of the visual system that's needed for what you can call visual perception. you can see what you need in the central pathways for visual perception. damage to any part of this pathway will produce blindness. so damage to the eye will produce blindness and also damage to any of these other connections will produce blindness. but what we really want to talk about today is blinding eye diseases. so i would like to kind of get a

little more nitty gritty and talk about what's in the eye. if you look on the left, you see that light comes through the lens and, just like a camera, the lens is focusing light on the back of the eye, on the structure that's called the retina. you will see that the back of the eye, the retina, is not uniform. you will see there's kind of a dip or a pit that's present in the eye. this is called the macular region of the retina. it's in this region that we have our highest resolution vision. by the way, it's also the part of the retina that lets you see in color. the other part of the retina outside of the macula called the peripheral retina. this is very important, too, because it's the part of the retina that lets us see in the dark. so this is low-light vision.

actually, you can appreciate this part of the retina, too, because if you've ever looked at a starry night, if you've noticed if you don't point your eye directly at the star but you look a little off kilter, the star gets brighter, and that's because you're using the edges of your retina, the peripheral retina, which has very high sensitivity for light but isn't as good for high resolution images. so we have these two aspects of the retina. now, the last thing i want to talk about is what are the types of neurons in the retina. so we can look at a blowup of this macular region. there are nerve cells -- specialized nerve cells in the retina that are capable of converting light energy to a neural signal, and those are called the photoreceptors. there are two kinds of photoreceptors. there are the rods and there are the cones. the cones are the photoreceptors that are crucial for high

resolution vision, and they're in the macula, in the center. the rods are the photoreceptors in the peripheral retina, and those are really essential for high-sensitivity vision. the next step of visual information processing is that the information from the photoreceptors is passed along to the bipolar cells, and from the bipolar cells the information is passed along to the ganglion cells, and the ganglion cells are the output neuron of the eye. so they are the nerve cells that send the million long fibers out into the optic nerve and into the optic track into the central visual pathways. one last very important point i want to make and that is there is another critical cell type in the retina. it's called the pigment epithelium, and the pigment epithelial cells essentially provide crucial support and

nutrients to the photoreceptors. they're really essential for photoreceptor health and for photoreceptor survival. and several blinding eye diseases involve -- and their treatments -- involve these pigment epithelial cells in the retina. we'll hear about these cells. they'll feature really quite large in our conversation. >> charlie: that's enormously helpful. thank you very much for the huge understanding of how we see and how important the brain is and how important the pathways are. i want to come now to jean and talk about -- we mentioned gene therapy and stem cell therapy and other things. how can gene therapy be helpful? >> well, carla has set the stage for this perfectly because she's explained how the pigment epithelium provides nerve function for the photoreceptors, providing nutrients to the cells, taking away waste products, and the two cell types are interdependent so that, if

there's a problem in function of one particular gene in one of those cells, it affects the other cell secondarily. so in the next image is an illustration of one particular example where a mutation can cause disease in both of these cells. you see some pigment epithelial cells next to the photoreceptor cells which are stunted because they're sickly, and the pigment epithelial cells memeselves are sick -- themselves are sickly. they're accumulating liquid droplets because they have a mutation which prevents them from forming a particular form of vitamin a derivative which they normally supply to photoreceptors that's essential for vision. without the photoreceptors receiving that vitamin a derivative, they can't respond to light and there is no vision. this makes them sick and they die off as the person ages.

the same thing can go on if there's a mutation in a photoreceptor, it can cause diseases in retinal pigment epithelium. knowing this, it's possible to, in knowing the genes which cause these diseases, it's possible to intervene. so i would like to give you an example of labors congenital amarosis, one of the most severe forms of retinal degeneration, one of the most severe forms of retinal pig me pigmentosa. it's usually in babies. parents notice the children aren't responding to things and instead have roving eye movements because the retina is not giving signal to the brain and the brain isn't giving signals to the eye muscles to tell them to hold still. this disease has been studied

carefully over the past two decades or so and we now know there are ant 19 different genes which can give rise to this same phenotype. we've studied the rpe65 gene, stands for retinal epithelium 65kilidalton, and this is one of the more common forms of this condition, and there also happens to be an animal model of this disease. puppies born with a spontaneous mew triggs and born -- mutation and born blind. we begin working with the puppies and restoring vision with them and thought it would be great to do it with children. so how would you carry out gene therapy? the dna is very highly charged and trait that's a problem for g a normal copy of dna, a corrective copy across the cell membranes. so you have to be able to encapsulate the normal copy of

the gene inside of what's call a recominant virus. we've taken essentially the shell of a virus and packaged it with the normal copy of the dna, and that dna inside the virus has been injected into the subretinal space, which is the space between the neural retina and the nerve cells, exposing those cells to the experimental reagent. then the next image you can see a closeup of the virus entering the subretinal space and delivering -- entering the retinal pigment epithelial cells. the dna inside that virus then goes into the nucleus of these cells where it sets up shop and starts encoding the protein that is missing, which happens to be rp65, in this instance. this is a very stable effect in the first animals that we've

studied, after one single injection the protein was produced and the dogs could see after eleven years. and so this is really quite stable. so the next question is how would this work in children? so the bottom line is that we've run a clinical trial using gene therapy for this condition and have found that all of the children involved in the study can now lead essentially normal lives, whereas when they walk into the hospital using a cane or holding their parents' hands because they couldn't see well enough to navigate, they can walk independently, they can sit in the classroom and read books, sit in front of the classroom and see what the teacher is writing on the board, play sports, et cetera. so i'd like to show you an example -- >> who was the first to do this? ell, my team was the first to do this. so you will see a video image in a minute of an 8-year-old boy

who was one of the first children in the world enrolled in a gene therapy trial for a non-lethal disease. he is shown three months after he received a single injection in one of his eyes, happened to be his left eye. in this video, his injected eye is patched. what you will see when this video plays is that the boy is put through an obstacle course which is in the hospital exam room. it's full of arrows and obstacles that he's supposed to avoid and navigate his way around the course and find the door. what you can see in this video is the boy takes a step and he doesn't know what to do because he can't see anything. he talks to this video and you

can hear what he says. this is hard. he has to be coaxed. he takes a step and bumps into the object, the sign in front of his eyes because he can't see it. he goes off course immediately, and it takes him a total of 17 minutes to make it through this course. >> charlie: he has a great heart, though. >> oh, absolutely. on the other hand, when his uninjected hand is patched and he's using his newly-injected eye, this is the same child, he's walking through the course, stepping over an object jutting in his path, avoiding the observe strackles, can have -- obstacles, confident and makes it to the end of the path without any problem whatsoever. how does this translate to daily life? this is what is so fantastic. this child who came in walking with a gain is riding bicycles,

on a championship little league team, hammering objects, playing video games, hitting targets with rocks, maybe doing things his parents would rather him not do but leading the life of a normal child. >> charlie: what we want wie all want to know is to whom is that treatment going to help? what kind of blindness? >> this treatment will be effective for individuals who have mutations in the rpe65 gene because that is the type of dna that is delivered into the cells. if they have a mutation in a different gene, that wouldn't be effective. however, the exciting thing is that the same sort of approach can be used to intervene with other diseases that are due to other mutations and there are now at least a dozen other targets out there, several of which are now in human clinical trials with very exciting results. >> charlie: and they're all learning from each other.

>> we're all learning from jean. both are true. but this is not restricted to the eye. gene therapy and stem cell therapy is being used in other areas of medicine as well, really major advances. >> charlie: talk to us about stem cell. >> gene therapy is in late-stage clinical trial development and showing remarkable safety, efficacy and durability, meaning it's working well within the clinical trials guidelines and poised, in my opinion, for approval. stem cell or regenerative medicine is different. these studies are early, hold great hope and promise but are in the early stages of development and i would characterize them as highly experimental. appropriately the first stage of any new trial is a safety trial or a phase one trial and that's what i'll talk to you about today. our hypothesis centers on the idea that we could replace a cell, so we could take a stem cell which means that cell is capable of becoming any cell type in the body. so we could take a stem cell,

coax it, to use eric's term, into becoming the retinal pigment epithelial cell, differentiate, so to speak, and then take those differentiated cells and inject them into patients who are missing the retinal pigment epithelium with the hope of rescuing or restoring vision. our aim is to safely transplant stem cell pigment epithelial cells and replace the diseased cells. >> what's wonderful about these two people is she's treating an individual gene, he's replacing the whole population. >> we're trying hard to replace it. in macular degeneration, the retinal pigment epithelial cells are lost and we're trying to replace them using a strategy similar to jean and her team. we've taken human stem cells and reduce the differentiation and cokes them into becoming the retinal pigment epithelial

cells. buns they become them, we optimize them and harvest them at a time when they're most likely to succeed in transplantation. the next slide, through surgery, we're transplanting them into the same space jean is using in her gene therapy trials to replace these cells, and once they're replaced, they either rescue or restore vision by taking care of the photoreceptors that are adjacent to them. >> charlie: i don't understand how you coaxed them. >> it's a very surprisingly astonishingly, straight-forward method by which stem cells can be induced to certain cell times. turns out retinal pigment epithelium are relatively low-hanging fruit in terms of easy to induce the transformation to a terminally differentiated cell type. it's important to realize the retinal pigment epithelium are attractive targets for stem cell therapy. the retina pigment epithelium

has no synaptic connections. it's inside the blood-brain barrier, as eric would say, and surgically accessible so we can get to it surgically, relatively safely, it doesn't require synaptic connections. in the laboratory we can take the stem cells and turn them into essentially brand-new, young, juvenile retinal pigment epithelium and give somebody a fresh layer of cells -- if they have macular degeneration, say 40, 50, 60 years old, to the duration. we have, to date, transplanted a number of patients. these early stem cell studies seem safe and astonishingly give us a signal there might be some restoration of vision. i want to share with you the first patient we tranc transplaa young lady who was a set designer and lost her vocation because of her blindness. >> when i was younger i played a

lot of competitive tennis. in my later teens, i started playing a little more poorly. it was seeing the lines or the ball not quite as, you know, precisely and calling the lines and stuff was a little more difficult. i woke up one morning and i looked across my room and i have a piece of furniture there that's kind of a larg armwoir ad a lot of detail on it. i opened the operated eye and could see a lot of the detail but not from the distance where i was lying down. after that, i got up and looked at everything around the house, looked at the grass, everything with one eye, then the other eye because they only operated on the one eye. i could see it a lot better than i had before and i thought, wow, maybe there's something that would really be working.

it was pretty exciting. >> charlie: how long will it be before you operate on the other eye? >> a long time. we're very, very careful and guided by the fda appropriately and by our own ethics commutes at ucla and other universities. so only the worse eye when we're not certain about safety as opposed to gene therapy. we've certainly given this opportunity to a number of patients. >> how many tairpts so far? between 20 and 30 and they're actually the heros, the people who willingly go into this with huge risks and they do it -- >> charlie: what are the risks? >> in stem cell therapy and regenerative medicine are enormous. we're careful to make sure we differentiate cells into retinal pigment epithelium but if we give them a straight stem cell, it could turn into anything the

body can reject. we've been lucky to have heroic patients to volunteer themselves and put themselves in harm's way. >> so far things seem to be moving smoothly. >> charlie: there are people who continue qualify for either gene therapy or stem cell therapy and you step forward with the use of technology. >> right. if you look at one of the slides, what you do when nothing works anymore, we can replace them technically. we built a chip hat has little photo diodes, little boxes in the graph and each photodiode takes a spot of the image like the painting we've seen, amplifies it and sends an

electrical signal back. so it's kind of a replacement of the natural photoreceptor with a technical photoreceptor. how does it work? we can see this kind of like a chip in your iphone but has a totally different inner life. it has 1,500 photodiodes. the image falls through the lens on to the little chip and, point by point, the image is analyzed and turned into an electronic mirror image. the cells are stimulated, processes the image and sends the image through the natural pathway to the optic nerve to the output of the retina back to the central visual system. now we have amplifiers there.

they need currents. how do we do that? what we see on the left is again the retina and the eye, and we put the dhip right beneath the macular region where we see the best and the brain has to learn to see -- the brain has learned to see the things. and there is a tiny yellowish wire going under the retina to the top of the eyeball, crossing the eye, to empower to the part placed behind the ear. so first look to the patient's retina. the very right is like a moon optic nerve and left of it there is this grey area. a new window to the world of this patient, has approximately the size of an ipad at arm's

length. window size, again the patients looks into it. and the yellowish cables are the power supply and the lines to control the chip. you see, the head of the patient on the left, there's an x-ray, so there is a chip on the eye and almost a pretzel-like cable, and then we see behind the ear and the x-ray and it has a magnet. on the right side you see the patient, if he wants to switch on the chip, he simply clips the antenna coil behind the ear, the size of a $1 coin, and in his hand he has a power box so he can control brightness and contrast like an old black and white tv, and this tiny table

powers the antenna and, now, this reminds us a little bit about the cochlear implant. his is much more complex because, in here, we have a single signal in time to analyze, and here we have to have 1,500 parallel points to address at the same time so it's much more complex and more difficult. but what -- >> when you speak, there's a sequence to the sounds. that's much easier to analyze than if you look at a sarat painting where you simultaneously have to analyze all the components of the image. it's much more difficult. >> so let's look at the painting. how many dots? i don't know, several thousands. our chip has, of course, not the power to resource each of those

dots, so it's probably 20 dots or so which fall on one pixel of our implant. so the image is not colorful because it all mixes to grey, and the borders are not so sharp as you can see, they are a little blurred. that's what the patients tell us. there is a movie clip next. >> it's going across there. it's all light up there, and it's light there. it's going across. there and there. it goes up there. but it's not white. >> charlie: here she is, completely behind. >> completely blind and she got the chip into the eye and --

>> charlie: she's seeing what as she described the bridge at oxford? >> that's what she's seeing through the chip. >> in the moment, you will see on the next image exactly what that looks like. >> the next image shows what she is describing. it's really an image consisting of black and white and greyish layers and it's probably not much to us who are used to seeing so well, but to somebody who has been blind, it makes a lot of difference to see again at least the surroundings in the blurry greyish black and white way and to be able to be confident in marking. we have 40 patients and many are able to see a glass to lift a glass, to see a cup, a knife, a spoon, or to go out and see. >> charlie: who is eligible

for this? >> all the patients who have lost their photoreceptors and the retina is still in tact. so patients who have lost the rods and cones. there are thousands in new york who has this condition. >> charlie: tell me what you're thinking, sandy, as you're listening to this. >> well, it's inspiring, and i'm not a guy who likes the i word very much. but there are many children and millions that will be born blind. well, to make a very long story short, we established a prize to end blindness for the work that

best achieves our objective of developing treatment for eye disease, $3 million in gold bullion as the prize. so we're doing whatever we can to encourage the scientists around the world, and these are, without a doubt, the leaders. >> what's wonderful about this approach the three of them are taking is, first of all, it cheese ther, thereis no single r blindness. jean inserts a single gene into a particular cell type and, boom. he uses the stem cell to replace the whole cell population. he takes case in which you don't even have the epithelial cells, you've lost a good part of the retina and he stimulates the

central connection that leads to the central nervous system, so these are progressively more severe diseases each of these therapeutic approaches address. >> charlie: you know, as we were watching this, i was thinking of a society that worships athletes and celebrities and sports figures and here we are with scientists and people who are experimenting with their own lives. >> yes. >> charlie: who give new definition to what it is to be heroic and make real contributions to society, so we're all in your debt. let me, as we run down the clock here, go to each of you and ask about what you hope is possible. from the research that you are doing. >> well, on an immediate level, we are running a phase 3 clinical trial -- that's a trial aimed at getting this material approved as a drug so that people who have this condition could benefit from the

intervention. this is being run at the children's hospital, philadelphia, the only phase 3 gene therapy clinical trial going on in the world, and there is no approved gene therapy in the united states, and only one other gene therapy approved in the world. we think this could be an approved gene therapy, the first approved gene therapy for a blinding condition and could pave the way for opening up the possibilities of developing similar approaches for many other blinding conditions. so we think it's just the beginning. >> i think jean's right, gene therapy is here to stay, a little like antibodies were 20 years ago. she's paved the way and many will follow for many different diseases. stem cells, as astonishing and hopeful, i want to emphasize how early it is. i'm mindful of not increasing

patients' suffering. there's so much stigma around stem cells, people think everyone's cured and it's a fountain of youth, but i think it's worth considering the social and political complexities of studying stem cells. it's an interesting study. we have two political leaders who have contributed to this and they're from different political parties. in california, arnold schwarzenegger created the california institute for degenerative medicine which paved a way for the work in california and allowed us to stand on the shoulders of great basic scientists and great universities like ucla. the other is president obama, barack obama is very courageous in leading us, following the science and supporting the national institute of health through very complect political and economic times so, to me, both those have been really important. and i think, as jean and

eberhart pointed out, it's really the team. i could not do this myself. for the future, efforts like what you do, this great work, allows people to understand the suffering, what sandy has gone through and the countless number of people who go blind unnecessarily, bringing blindness to the forefront and ending it is what my goal is as a treating physician, and macular degeneration, whether gene therapy or stem cell therapy or a chip, would be a great thing to knock off the podium as sort of the leader of blindness. >> charlie: well said. we have so far about 40 patients, and it doesn't work in all of them. some have very degenerated retina and we have to understand only half of the patients have useful vision in daily life. but there are more than 30 groups working worldwide on those approaches, some in the united states on the cell side,

the others the brain. for patients like sandy who have lost the optic nerve or other people who have lost the eye, it is possible to directly connect implants to the brain. that may be a way to help the other patients which are, for other reasons, not a retina situation. of course, there are technical improvements. we can't put only a single chip into the eye but many chips to increase the visual field and also we can transform these computer pictures by computer software into simple graphs like sketches or caricatures that can easily be grasped. i think there is still a lot of room for improvement. >> charlie: carla, i ge i give u

the last word. what would you say is your own hope that the future holds? >> well, i think what we've heard today is from three pioneers who have used three different approaches to trying to cure these blinding eye diseases. and what's amazing is that the eye is an extension of the brain. it's part of our brain. so what they've done, also, i think, gives us a great deal of hope for dealing with other neurodegenerative brain disorders where nerve cells are lost and where they could be replaced or circuits could even be rebuilt. so my hope is that both from a combination of their work and the work of many other scientists and physicians who are taking -- you know, also working on these problems that,

soon, we will have a lot of options for dealing with the loss of neurons and the brain, wherever it happens. >> charlie: that's hope blonde specifically blindness that the groundbreaking work here can affect a whole range of diseases that are connected to the brain. >> right. i was thinking, you know, we first discussed deafness, and now blindness, and both those areas, if you go back 20 years, there was no treatment for almost anything. there was help with deafness for hearing aids that were very primitive. the progress has been spectacular in each of the areas. what we're talking about here is four or five years old. this is very recent. if you look at my field, psychiatry, you cannot think of the 20 years that past as being anywhere comparable. so we can take hope from these areas in which we've progressed

brilliantly that will be applicable to other areas as well. >> charlie: thank you all very much. thank you for joining us. see you next time. captioning sponsored by rose communications captioned by media access group at wgbh access.wgbh.org

>> the following kqed production was produced in high definition. [ ♪music ] >> it's all about licking your plate. >> the food was just fabulous. >> i should be in psychoanalysis for the amount of money i spend in restaurants. >> i had a horrible experience. >> i don't even think we were in the same restauran