Transcript of Charlie rose show.. Dr Schwartz and his first hesc rpe patient Cathy...
Courtesy of ICELL Cocopuff
Transcript from the Charlie Rose Show...
Dr. Stephen Schwartz on Charlie Rose show, 4/22/14
Dr. Schwartz: Well, as we have just discussed, gene therapy is now, you know, in late stage clinical trial development and showing remarkable safety, efficacy and durability, meaning it's working well within clinical trials guidelines, and it's poised in my opinion for approval. Stem cell, or regenerative medicine, is different. These studies are early. They hold great hope and promise, but are in the earliest stages of development. I would categorize them as highly experimental. Appropriately, the first stage of any new trial is a safety trial, or Phase I trial and that's what I'm going to talk to you about today. Our hypothesis centers on the idea that we could replace cells, so we can take a stem cell, which means that 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 a 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 a hope of rescuing or even restoring vision. In this, our aim is to safely transplant these stem cell-derived retinal pigment epithelial cells and thereby replace the diseased or lost cells.
Eric Kandel: What's wonderful about these two people is, she's treating an individual gene in the pigment epithelium. He's replacing the whole population of pigment epithelium. It's really amazing.
Dr. Schwartz: MAYBE replacing…. We're trying to replace it. So for example, in macular degeneration, the retinal pigment epithelial cells are lost and we are trying to replace them, and we're using a strategy very similar to Jean and her team. We've taken human stem cells and we induce the differentiation, or we coax them into becoming the retinal pigment epithelial cells. Once they become the retinal pigment epithelial cells, we optimize them and harvest them at a time where they are most likely to succeed in transplantation, and then in the next line you can see what we're doing here is through surgery, we're transplanting them into the same space that Jean is using in her gene therapy trials to replace these cells. And once they are replaced, they rescue or restore vision by taking care of the photoreceptors that are adjacent to them.
Charlie Rose: Yeah, I don't understand how you coax them?
Dr. Schwartz: The truth is, it's a very surprisingly, astonishingly, straightforward method by which stem cells can be induced into certain cell types and it just turns out that retinal pigment epithelium are relatively low-hanging fruit in terms of being easy to induce the transformation to a terminally differentiated cell type. It's also important to realize that the retinal pigment epithelium are really attractive targets for stem cell therapy. Carla told us how integrated the retina is. The retinal pigment epithelium has no synaptic connections. It's protected by an immune privilege. It's inside the blood retinal barrier or blood brain barrier as Eric would say, and it's surgically accessible. So we can get to it surgically, relatively safely. It doesn't require synaptic connections. We can, in the laboratory, take the stem cells and turn them into essentially brand new, young, juvenile retinal pigment epithelium and give somebody a fresh layer of these cells that could take them, if they have macular degeneration and say they're 40, 50, 70 years old, for the duration.
We've today transplanted a number of patients. These early stem cell studies seem safe. They seem astonishingly to be giving us a signal that there might be some restoration of vision. I wanted to share with you the first patient that we transplanted, a young lady who was a set designer and lost her vocation because of her blindness.
Cathy: When I was younger I played a lot of competitive tennis, and I think when I was like in my later teens, I started playing a little more poorly. It was seeing the lines or the ball not quite as 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 this large armoire and it has a lot of carved detail on it, and I actually had my head to the side and I opened the operated eye and I looked at it, and for the first time I could see the detail in it, that I hadn't been able to see from the distance I was lying down. After that I just got up and I started looking at everything around the house, and looked at the grass, I mean looking at everything with one eye and then the other eye, because you know they only operated on the one eye, and I could see it a lot better than I had before, and I thought, wow, maybe there is something here that will really be working you know. It was pretty exciting.
Charlie Rose: How long will it be before you operate on the other eye?
Dr. Schwartz: Oh, a long time. We're very, very careful and step-wise and guided by you know the FDA appropriately, and by our own ethics committees at UCLA and at other universities, so only the worst eye for when we're not certain about safety, as opposed to say gene therapy, where now they're operating on the second eye of these kids. We have not. We're far from that, but we certainly have given this opportunity to a number of patients.
Eric Kandel: How many patients have you done so far?
Dr. Schwartz: Between 20 and 30, and they're actually the heroes. They're the people who willingly go into this with huge risks and they do it for….
Charlie Rose: What are the risks?
Dr. Schwartz: Well the risks in stem cell therapy and regenerative medicine are enormous. We're very careful about making certain that we've differentiated these cells into retinal pigment epithelium, but if we don't and we give them a straight stem cell, it could turn into anything, plus the body could reject. So we've been very lucky to have a heroic group of patients, essentially volunteer and put themselves in harm's way. It's really sort of miraculous what these people do for those who follow.
Eric: The encouraging thing is that so far things seem to be moving safely.
