Dr. Evan Snyder Discusses Advances in A-T Research
By Sheila Dwyer, Body1 staff
Dr. Snyder: The best way to think of a stem cell is as the most immature, primordial cell of the nervous system. Think of it almost like the smart seeds of the brain. These seeds are the kind of seeds that if you throw them on the lawn, they know to become grass, but when they land in the flower bed they also know to become tulips or roses or daffodils. So it’s reseeding the lawn with really smart seeds.
If we can understand how these seeds work, how to obtain them, disburse them, and get them to become what we want them to become, then we can use this knowledge for a whole range of diseases such as Alzheimer’s disease, Parkinson’s disease, or even acquired diseases like head injury, stroke, and spinal-cord injury. If our goal ultimately is to try to translate neural stem cell biology (in particular human neural stem cell biology) to a model that will somehow be a dress rehearsal for what we want to do with the kids so we can try to understand the pitfalls and how best to approach the kids, we need a model that’s about as close experimentally as we’re going to be able to come to kids, without actually being kids. We don’t want to make our mistakes in the kids. That brings us to what is the traditional model of the human being, which is a monkey. What we think we will be able to do is, after we have perfected the technique in a limited number of monkeys, we have worked out the bugs and we have figured out what the limitations and the potential of neural stem cells might be, in a monkey model where these particular cells are degenerating just as they do in the kids, that then we may be able to—if the biology is on our side and if things are safe and if things are effective—to at least think about doing clinical trials in some of the kids.
My dream situation would be to generate a pretty representative model of Purkinje cell degeneration and cerebellar degeneration in a primate, that the human neural stem cells can be administered effectively to these animal models, that the delivery is not going to be onerous, that a fairly simple delivery system will allow these cells to find the proper place. Once they find the proper place they will live, become the appropriate missing cells, they will send out and find the appropriate target and start functioning, and that they’ll do this in a very safe and effective manner. That we’ll know that fairly soon—certainly within the next year or two. Based on that, maybe A-T is not as complex and perhaps some of the neurological problems really can be addressed by fixing the cerebellum. We’ll have to try some human trials on some kids with A-T using our human neural stem cells and [hope] that it will work. We’re starting to get to the point where we’re thinking that the obstacle is no longer the biology, the obstacle is only resources. With enough resources, I would take many people and throw them on this project and start working literally on shift work. We’d be working around the clock. The quicker we work around the clock, the quicker the data becomes available, the quicker we can troubleshoot, fix it, and then start putting together a story that would be believable to a regulatory commission to say, yes, you’ve made your case. We think this may be a good approach for kids.
The A-T Children’s Project has provided an enormous amount of foresight. They got into the stem cell field probably earlier than almost any other organization. These parents are so intelligent and so well informed that they know that the only way to cure their kids is by still using the scientific method. They don’t want bad science, they don’t want to just hear a fairy tale told by somebody talking about pseudo-science because they know, ultimately, that’s not what’s going to help their kids. What they want is good, solid, rigorous sciences by the best minds in the country doing them. What they want to do is just make sure that the science and the biology are the only obstacles.
One day I was in my office and there was a knock on the door. It was a face staring in the window of somebody I knew didn’t work in the laboratory building and somebody I’d never seen before. I went and answered the door, and it was a mother of an A-T child. And she was at Children’s Hospital. She had heard that the A-T Children’s Project was supporting some of the new techniques in trying to approach and understand A-T, which was the stem cell field. She said she wanted to come and just see me. And I thought this was just great. I was so thrilled that she would even think enough to come and actually meet me and talk with me. And she brought her two kids. One was little and one was big. She said to me, “Which one do you think has A-T?” And I looked at them playing and I actually couldn’t see anything abnormal with either one of them. And I said, “Well, I guess neither one of them.” And she said, “Actually it’s the little one. If you look really carefully, she’s a little bit clumsy.” And then she kind of looked at me and said “Do you think you can help her before she gets worse?” And that, for me, was really a very poignant moment. It happens whenever I go away from the word processor and away from my slides, or away from talking to a room full of scientists and I’m actually meeting the patients. I get very frightened. So there’s this combination of both exhilaration and absolute dire panic. The exhilaration is the thought that maybe, maybe I do have in my power the ability to help this kid so that this kid will never develop the symptoms that I know happens with kids with A-T. Then the panic sets in and says “but what if you can’t do it.” And then the exhilaration comes back and says “well, you’ve got to do it.”
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Courtesy of the A-T Children’s Project