from our studios in new york city, this is "charlie rose." >> to be blind is not miserable, bind messable to bear

is miserable. john milton wrote that. 285 million people live with visual impairment. in recent years, there have been breakthroughs in our understanding and treatment of blindness. sanford greenberg lost his vision at the age of 19. he is chairman of the board of governors of john hopkins eye institute and joins me today to talk about his experience and mission to and blindness. also joining me, jean bennett, steven schwartz, eberhart ofnner, and carla shatz stanford university and once again, eric kandel a nobel laureate and a howard hughes medical investigator. toegan with my friend eric give me an overview of our subject tonight. >> the last program we did was approaches to deafness. tonight we are going to consider

lines miss. as is the case with deafness, blindness is not a life threatening situation but it is disabling. in some ways it is more disabling than deafness because there are a number of important conditions for which there is no treatment. now, why is that so? deafness, the sensory organ for vision, the retina, which is evident in the lower image, it lines the inside surface of the eye. oft is the most complex set organs we have. in fact, it is not a proof real organ. it is actually a part of the central nervous system. as a result, it has the complexity of the structure. it is not uniform. it has a small area that is clear which is called the macula.

area of greatest visual acuity. so if i turn my head towards analyzes youra features. unfortunately, the sales -- cells are sensitive to damage. so this is a really serious problem. we have no treatment for that, even though this is a point of great visual acuity. a range ofs conditions. it ranges from complete blindness to partial blindness. complete blindness means you do not see images, you do not see figures were people. you can't tell the difference -- you can tell the difference between day and night. partial blindness varies from television, cloudy vision, night blindness.

have difficulty with division 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, we are not going to focus on that. we are going to limit ourselves to disorders of the retina. you indicated, there are close to 300 million people worldwide that have various degrees of impairment and they fall into two categories, treatable diseases and things that are untreatable. glaucoma, you have cloudy vision, what, you have time no vision. diabetic retinopathy, you have difficulty with night vision. what i find tragic, a number of people suffer from these conditions and this is a graph

of the developed world. the united states, canada, and europe. because these are treatable in the case of diabetic retinopathy. they are preventable. in the underdeveloped world, people don't have access to medical care. in the developed world, the major problem is macular degeneration. as we indicated, there is a form that accounts for this, which is really an age-related disorder in which people really lose a lot of their vision, their visual acuity. in the past, the only thing we could do for people like that is to give them nonvisual guy, teach them how to read braille, hand rails in their apartment, and speech compression devices. it makes it easier for them to handle auditory information.

recently this has changed. we are sitting here in the midst of a revolution in the treatment of macular degeneration and it opens up treatment of many kinds of blindness. that is because tree major developments have occurred. gene therapy, stem cell therapy, tinalretched chips -- re chips. gene therapy is an attempt to replace a defective gene with a normal one. they are noton functioning, one can insert a normal gene into the cell. with stem cell therapy, you are not rescuing i jean, you are rescuing a cell type -- a gene, you are rescuing a cell type. -- it can take on any cell in your body and get them to be retinal cells and replace

them. chips, you implant it in the retina and you stimulate pathways that lead to the brain. stimulating those elements that lead to the brain. and we have with us, by coincidence, the three pioneers of this area, jean, j jean bennett has pioneered the study of gene therapy. steve has pioneered the study of stem still therapy. t is one of the outstanding leaders in developing our retinal chips, these microchips that are really amazing for people who can't respond to other treatments. we also have two other people ine, carla shatz, a leader visual physiology and she is going to explain the visual

system to us and my friend sandy is amazing. had a severe case of clock, from which he became blind. he will tell us what it is like to be blind. but the amazing thing about hasy is despite the fact he this tremendous handicap, he is a remarkable human being. he has had a rich and productive life. he has invented things and now he is thinking of how he can help other people who are blind. >> let me welcome you again. great to have you. blindo us about becoming and the sense of the journey for you. years, ieven after 50 can still feel what it was when i went blind. for many months, i had declining

vision. my mother and i went to see a specialist. he examined me and then turned to me with my mother in the room you're going, son, to be blind tomorrow. i guess that was my moment. i think there is a moment that lifes in about everyone's that the instant before bad news is given, after which nothing else in your life will be the same and after which you look back and say, my god, i did not realize it until now. meore that moment, all for was possible and all was self-evidently actual. after that moment, zero

possible, zero actual. at one moment you're at the top of your game, and then there is no gain. felt empty.y, i even serrated. -- eviscerated. there was a fair amount of pain in my eyes. wtih thein comparison pain in my heart knowing my mother had witnessed her 20 road sons go blind -- her 20-year-old son go blind. so there was really no reason to live. so i prayed in my own way. , i was convinced, was going to leave me. after all, i had no eyes, no

money, no future. but she did not. her thatlly because of i am here tonight with you. and she and my college roommate, me on thekel, helps way out of my wilderness. so i returned to columbia and then something happened back then not too far from where we are sitting tonight that turned me away from blindness. i were walking toward grand central station during rush hour when he abandoned me. so i got to down the steps to the hole in the ground, on my own. grand central station. and you are blind. so i stumbled to the train that

caught me back to columbia and as i walked through the large orange gates of that great university, a guy bumps into me and says, excuse me, sir. course, my roommate. this guy called garfunkel. me.he had not abandoned he followed me the entire way. and as i bumped into his chest, i knew if i could get through the new york city subway system blind, there were no limitations to what i could accomplish. >> all was possible. so what do you hope for today?

today iso i hope for that eric and people at this table who, in my view, embody the achievements of human genius for- human them to end this plague. it has been 6 million years since we humans and our ancestors have been afflicted by this thing. it's got to end. a group of us have started something called and blindness by 2020. sue and my family and my college wasmates, art, jerry, he ,ctually a witness to my subway who has been with me and for me since we entered columbia college together. we have launched an effort a few

years ago to end blindness b 2020. >> what i find interesting about you, first of all, having art and jerry has roommates is a very good beginning for life. from any point of view. how did you accomplish what you accomplished with this handicap? harvard, you made a major invention. you have led a rich life. you are happily married. sue.e centerpiece is my family, my children, have stuck by me and these guys, who i was really fortunate enough when we all started columbia together. all black or dark.

i don't want to leave that impression with you. distracted byot visual images, you develop a life within your mind. so i can see you now, the way i saw art back before it was 19 in the city and you look pretty damn good. [laughter] >> your mind is playing tricks on you. [laughter] >> charlie, you look like the david. [laughter] >> sandy, that is remarkable. do thiswhy we television program. let me turn to carve up. give me an overview, so we understand. exactly what it is we are talking about in terms of the human eye. >> vision is a miraculous

process. alsoar about losing it is very heartrending. thank you for what you have said. and i want to tell you about the visual system and talk about the basic layout of the system before we get into talking about the eye. andon starts in the eye there is a special layer at the back which is called the retina. you can think of the retina is a fancy digital camera. and the light sensitive part of the eye is like the pixels in your camera, although the pixels are made out of silicon. in your eye, they are the nerve cells in very specialized nerve ands which absorb light converted to a nero signal. and then the information is sent structures ina to

the central part of the brain. this is called the central visual pathway. the first place is the lateral geniculate nucleus and then the primary agile cortex. you can kind of think, we talk about the digital camera idea. this is kind of like the central processing unit where a lot of image analysis happens. butt starts in the eye, most of the information processing and data crunching is happening in the central visual pathway. you can ask, what is the nature of the information that comes out of the eye? i was thinking about how to convey this. i can't do it any better than this, there's this wonderful painting. and you will notice in this painting that he has broken up the visual world into thousands

of dots of color. if you stand up close, if you put your nose to the painting, you're going to see a lot of pot -- a lot of dots. if you stand away from the painting, what you actually see is a seated woman. if we talk about this idea of the retina versus the central pathway, the retina is sending thousands of dots to the central visual pathway. is carried bydot a single nerve fiber and there are about a million of them going to the central visual pathway. so it is the job of the retina to deconstruct the visual world into a pixelated view of the world. thousands of pixels. it is the job of the pathways to reconstruct the world by making

a kind of seamless view of the world, kind of connecting the dots. so we've really need our central visual pathways to appreciate this is a woman seated. we only need the retina to appreciate the dots. the -- it starts in theeye, but it is computing power that is needed for reception. damage to any part of the pathway will produce blindness. damage to the eye will produce blindness and any of these other connections will produce blindness. but what we want to really talk about today is blinding eye diseases. i like to get more nitty-gritty and talk about what is in the eye. if you look on the left, you see

that right comes through the lens and just like the camera, the lenses focusing the light on the back of the eye on the structure that is called the retina. you will see that the back of the eye is not uniform. dip thatkind ofa a is present. this is called the macular region of the retina. that we this region have our highest resolution vision. it is also the part of the retina that lets you see in color. the other part of the retina outside of the macular is called the peripheral retina. this is very important because it is the part of the retina that lets us see in the dark. this is low light vision. actually you can appreciate this part of the retina because if you have ever looked at a starry night, if you don't point your

eye at the start, you look a little off, the stars brighter and that is because you are using the edges of your retina which has very high sensitivity for light but is not as good for high resolution images. so we have these two aspects of the retina. the last thing i want to talk about is what are the types of neurons in the retina? we can look at a blowup of this region. that are nerve cells capable of converting light energy. and those are called the photoreceptors. there are two kinds of photoreceptors. the rods and tones. the cones are crucial for high resolution vision and they are in the center. photoreceptorse

in the peripheral retina and those are really essential for high since it -- high sensitivity vision. is that thep 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. they are the nerve cells that spend their long fibers into the optic nerve and the optic track. one important point i want to make, there is another critical type in the right now. it is called the pigment. cells provide crucial support and nutrients to the photoreceptors. they are really essential for the health and survival.

involveeye diseases these cells in the red number. we will hear about them. i think they will feature quite large in our conversation. >> that is helpful. understanding how we see. and how important the brain is and the pathways are. ♪

>> i want to come to jean, we mentioned stem cell therapy and other things, how can gene therapy be helpful? >> carla has set the stage for this because she explained how the pigment epithelium provides nutrients to the cells ticking away waste products. -- taking away waste products. if there is a problem in function of one particular gene, it affects the other cells. so when 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 epithelium cells next to the photoreceptors, which are stunted because they are sickly. is the pigment epithelium sickly. not because they have a mutation makingrevents them from a vitamin a derivative, which they normally supplied to photoreceptors and that is the central for vision. without the photoreceptors receiving that vitamin a, they can't respond to light and there is no vision. this makes them sick and they die off as the person ages. can go on ifg there is a mutation in the photoreceptor. possibleng this, it is , and knowing the genes that

cause these diseases, it is possible to intervene. i would like to give you an which isf a disease one of the most severe forms of retinal degeneration. manifestcause it is first in babies. it is present at birth and usually parents first notice these children aren't not responding to -- aren't responding to visual cues, smiles, and instead they have abnormal eye movements because they're retina is not giving a signal to the brain and brain is not getting a signal back to the eye muscles to tell them to hold still. this is a disease that has been studied very carefully over the past two decades and we now know there are about 19 different genes which can give rise to this. we have studied one of them, the rpe 65 gene.

is one of the more common forms of this condition. there also happens to be an animal model of this disease, puppies that are born with a spontaneous mutation are born blind. we actually began our work with these puppies restoring vision thought, wouldn't it be great to do this with children? so how would you carry out to gene therapy? is highly charged. that is a problem for getting across thees of dna cell membrane. you have to be able to encapsulate the normal copy of of thee inside recombinant virus. we have taken the shell of a virus and packaged it with the normal copy of the dna. is injected into

the space between the retina and the nerve cells, exposing those cells to the experimental reagent. the next image you can see a close-up of the virus entering the sub retinal space and delivering the pigment epithelium cells, then it goes into the nucleus of the cells, where it starts to encode the protein that is missing, which happens to be rp 65. effect invery stable the first animals we studied. injection, the protein was produced and the dogs could see after 11 years. so this was -- the next question is, how would this work and children?

so the bottom line is that we have run a clinical trial using gene therapy for this condition all of theund children involved can lead normal lives. and they walked into the hospital using a cane or holding their parents hands because they could not see well enough to navigate, they can walk independently, they can sit in the classroom and read books and teacher is- what the writing on the board. i would like to show you an example of one of the children. my team was the first to do this. you are going to see a video image in a minute of a little boy, an eight-year-old boy, one of the first children in the world and rolled in a gene therapy trial for a nonlethal disease.

he is shown three months after he received a single injection in one of his eyes, and in this iseo is injected eye patched. what you will see when the video plays is the boy is put through an obstacle course, which is in the hospital exam room. it is full of arrows and obstacles he is supposed to wayd and navigate his around the course. what you can see in this video is the boy takes a step and does not know what to do. he can't see anything. video -- you can hear him. "this is hard." he has to be coaxed. he takes a step and bumps into the object, the sign, because he cannot see it.

he goes off course immediately. 17takes him a total of minutes to make it through the course. hand, when his other eye is patched and he is using his injected eye, this is the same child. he is walking through the course, stepping over an object, avoiding the obstacles, confident, and makes it without any problem. so how does this translate into his daily life? this is what is fantastic. could not walk around and is now riding his bicycle to his friends houses, playing baseball, he was on a championship little league team. hammering objects. laying video games. ,itting targets with rocks doing things his parents would rather him not do, leaving the knife -- the life of a normal child.

>> to whom it is that treatment going to help? what kind of blindness? >> it will be effective for individuals who have mutations in the rep 65 gene. intois the dna delivered the cells. if they had a mutation in a different gene, it would not be effect if. -- effective. the same approach can intervene are other diseases and they now, at least a dozen targets, several of which are now in human clinical trials, with exciting results. >> and they are learning from each other. >> right. >> but this is not restricted to the eye. therapy is used in other areas as well. talk to us about it.

>> gene therapy is in late stage lyrical child and showing clinicalkabul -- trials and showing great improvements. it is poised for approval. stem cell is different. the studies are currently and hold great hope and promise, but are in the earliest stages of development. i would characterize them as highly experimental. the first trial is what i'm going to talk to you about today. our hypothesis centers on the idea we could replace the cell. we could take a stem cell capable of becoming any cell type in the body, we could take a stem cell and coax it into become a retinal pigment epithelium cell and then take those differentiated cells and

injected them into patients who are missing the pigment epithelium with the hope of restoring vision. our aim is to transplant these cells and replace the diseased cells. >> what is wonderful, she is treating an individual gene. he is replacing the whole population. >> may be replacing. we are trying. so in macular degeneration, the arenal epithelium cells lost and we are trying to replace them. we are using a strategy where we take human stem cells and we induce the differentiation. once they become the pigment epithelium cells, we harvest them where and are most likely to succeed and then the next slide you can see what we're doing. through surgery, we're transplanting them into the same

space jean is using in her trials to replace these cells. they either rescue or restore vision by taking care of the photoreceptors. >> i don't understand how you coax them. >> it is a surprisingly straightforward method by which stem cells can be induced. it turns out pigment epithelium are low hanging fruit in terms of, easy to induce the transformation. it is also important to realize the retinal pigment epithelium are attractive targets for stem cell therapy. integrated theow days. there are no synaptic connections. it is protected inside the barrier of the blood and brain. and it is surgically accessible.

synapticot require connections. in the laboratory we can take the stem cells turn them into brand-new young pigment epithelium and give somebody a fresh layer of the cells that for theke them duration. we have transplanted a number of patients. these early studies it seems to be giving a signal there might be some restoration of vision. >> how many patients have you had? and 30.en 20 they are the heroes. they are the people who go into this with huge risks and they do it -- >> what are the risks? >> you normans. we make certain we have differentiated the cells into pigment epithelium, but if we don't, it can turn into anything. and the body could reject.

we have been lucky to have a heroic roup of patients volunteer and put themselves in harm's way. >> i am glad you pointed that out. seemed to be moving safely. >> there are people who do not qualify. you stem forward through the use of technology. >> right, if we look at that slide again. there is nothing to treat anymore. therapy does not work. replace them. is what we did is to build a chip littles photodiodes, boxes on the graph. imagehotodiode takes an like the seurat, and it amplifies it and sends an electric signal back.

so it is really a kind of replacement of the natural photo receptor. so how does that work? we can see this three times 3 million millimeter chip. it is like a camera chip and an iphone, but has a different life. it has 1500 photodiodes. the image goes onto the chip and point by point the image is mirrord and turn to a image, the cells are stimulated point by point and sends the image through the natural pathways of the optic nerve back to the central visual system. but now we have amplifiers.

they need a current. how to do that? what you see is the retina, the eye, we put the chip to train the macular region. -- right beneath the macular region. foil is a tiny yellow cable going under the retina to the top of the eyeball, crossing the eye to a power supply cable which goes back to a place behind the year. let's look through the people pupil onto his retina. you will see this round shaped like the moon and left of it there is this square. it is the new window to the

world of this patient, approximately the size of an ipad at arms length. cableis this yellowish that is the power supply to control the chip. you see the head of the patient on the left in the x-ray. chip, it isa camera like a pretzel like cable which goes to a receiver behind the a magnet in the center. so on the right side, you see the patient if he wants to switch on the chip, he clips the year, itoil behind the is the size of the one dollar coin. in his hand he has a power box where he can control brightness just like an old tv.

in this tiny cable powers the usenna and now this reminds about a car killer implant. implant.ar this is more complex because here you have to have 1500 atallel points to address the same time. it is much more difficult. when you speak, there is a sequence to the sound. that is easier to analyze than paintingok at a seurat where you have to analyze all of the components of the image. >> let's look at the seurat printing -- painting. how many dots are there? i don't know. thousands. our chip does not have the power

to resolve each of those dots. probably 20 or so which fallen one pixel. so the image is not colorful anymore because it all mixes to gray and the borders are not so sharp, they are a little bit blurred. that is what the patients tell us. there is a movie clip next. >> is going across there. lighter light up there, there. there is a bridge going across. that is the building there. and up there. the building goes up there. very, it is not a wide bridge. >> here she is, completely blind. completely blind.

she got the chip into the eye. >> she is seeing white as she describes the bridge? >> she is describing it. in a moment you will see exactly what it looks like. shows what shege is describing. blackreally consisting of and white and grayish levels and because we see so well. but somebody who had been blind, it makes a lot of difference to see again at least your surroundings in a blurry way and to be able to be self-confident and walking and we have 40 patients and many of them are glass and left a glass or a cup, knife, a spoon

or something like that. >> who is eligible for this? >> patients who have lost the there and the retina is still intact. -- lost the photo receptor and the retina is still intact. 4000 here in new york. >> sandy, tell me what you are thinking. well, it is inspiring. -- you know,y there are millions of these have zerohere they actual, and there are millions that will be born that are blind. short, welong story toe established a prize end blindness for the work that

best achieves our objective of developing a treatment for eye disease. we just reached $3 million in gold bullion as the prize. we are doing whatever we can to encourage the scientists around the world. these are without a doubt the leaders. >> what is wonderful about this approach, the three of them are taking, is that it shows there is no single approach to cure blindness, because it takes many forms. and it gets more complicated. gene andrts a single boom. a stem cell population. he takes cases where you do not even have the epithelium cells.

he stimulates the central connections that lead to the central never system. these are more severe diseases each of these approaches address. >> as we were watching, i was a society that worships celebrities and sports stars and sports figures and here we are with scientists and people experimenting with their own lives, giving a new definition to what it means to be heroic and make contributions to society. we are all in your debt. as we run down the clock, let me go to each of you and ask about what you hope is possible from the research you are doing. immediate level, we are running a phase 3 clinical trial, aimed at getting this material approved as a drug so people who have this condition

can benefit from the intervention. this is being run and children's hospital philadelphia and is the gene therapy going on in the world. there is no approved gene therapy in the united states and only one other gene therapy approved in the world. we think this could the unapproved gene therapy, the first one, 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 is just the beginning. is right. jean it is like anti-bodies were 20 years ago. she has taped the way and many will follow for prevalent diseases. as far as stem cells, as hopeful , i want to emphasize how early it is. i am mindful of not increasing

suffering because there is so much stigma around stem cell, people think, everybody is cured. it is also worth recognizing the social and political complexities of stem cells. it is an interesting story, we have had two political leaders that have contributed to this from different parties. arnold schwarzenegger started the california institute for regenerative medicine, which paved the way for a lot of this work in california and has allowed us to stand on the shoulders of great scientists and universities like ucla. the other is president obama. he was very courageous in leading us, following the science, and supporting the national institutes of health through complex times. they have both been really important and i think, it is really the team.

i could not do this myself. my hope for the future is that efforts like this allows people .o 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 michael. macular degeneration, other gene therapy or stem cell therapy or a chip would be a great thing to knock off the podium as the leader of blindness. >> we have operated so far on 40 patients and it does not work in all of them. some of them have a very degenerated retina. only half of them have useful vision in daily life. there are more than 30 groups working worldwide on this approach. some of them are in the united states.

on the brain.king especially for those patients like sandy, who have lost the optic nerve were other people who have lost the eye. tois possible in monkeys connect to the implants to the brain. that may be a way to help those other patients which are not, the reason is not the retina. and of course there are technical improvements. we can't put only a single chip, but many to increase the visual computer we can use software to translate it into simple graphs like sketches or can be grasped. there is a lot of room for improvement. arla, you talked about how

the brain and the retina are connected. own would you say is your future? >> what we have heard today is from three pioneers who have used three approaches trying to ure blinding eye diseases. what is amazing is that the eye is an extension of the brain. it is part of the brain. and so what they have done gives us a great deal of hope for dealing with other brain cells arewhere nerve lost and where they could be replaced or circuits could even be rebuilt. my hope is from a combination of their work and the work of many others who are taking and

working on these problems, that soon we will have a lot of options for dealing with the loss of neurons in the brain are ever it happens. heree groundbreaking work can affect a whole range of diseases and acted to the brain. >> right. to the brain. >> right. we discussed deafness and now blindness. if you go back 20 years, there was no treatment for almost anything. there was hearing aids, they were primitive. the progress in these areas has been spectacular. what we are talking about here is for five years old. this has become very recent. if you look at my field, psychiatry, you can't think of the 20 years as being comparable. so we can take hope from these areas in which we have

progressed. very and you, sandy. thank you, jean. thank you for joining us. see you next time. ♪

>> this is "taking stock" for tuesday, april 22, 2014, earth day. i'm pimm fox. today's theme is redefining boundaries. aereo will have its say in front of the supreme court. justices will look at the legality of the service that is shaking up the television industry. plus, i will speak with a marijuana executive is capitalizing on the legalization of pot. and i will sit down with the cofounder of paperless post. this company wants to redefine