TOPICS > Science

Dr. Yury Verlinsky

August 9, 2004 at 12:00 AM EST

TRANSCRIPT

SUSAN DENTZER: Let’s start by talking just a little bit about RGI [Reproductive Genetics Institute]. You do pre-implantation diagnosis on about a thousand couples a year now, or a thousand cases a year, I should say, from all over the country.

DR. VERLINSKY: Right.

SUSAN DENTZER: What are those, what are the conditions that people generally want to have diagnosed pre-implantation?

DR. VERLINSKY: Right now, we do pre-implantation genetic diagnosis for more than hundred different genetic conditions, single gene disorders.

SUSAN DENTZER: Give me some examples.

DR. VERLINSKY: Cystic fibrosis, thalassemia, Fanconi’s anemia, Tay-Sachs.. diseases that are more common in different ethnic groups and also rare disorders carried by the family.

SUSAN DENTZER: And let’s walk through just what some of those disorders are. For example, if we take Fanconi’s anemia, explain that particular disease to me.

DR. VERLINSKY: This is an inherited disorder that involves deformity of the children born, it’s somewhat recessive, two parents have to be carriers to produce abnormal baby. In these patients, the immune system is also jeopardized. They usually are affected by other leukemia and they do not live very long.

SUSAN DENTZER: So these children die in a relatively–

DR. VERLINSKY: In a relatively younger age, except if we are [able to help] them with treatment by selecting the embryo for the sibling, for the brother or sister, who is not only going to be free of disease but also whose stem cells from the umbilical cord [can be used to] make this sibling live.

For example, the Nash case, that occurred here, which is very well-publicized, when the brother saved his sister’s life and the sister is still alive and goes to school. Otherwise she would be dead.

SUSAN DENTZER: And that was done here?

DR. VERLINSKY: Yes. It’s [an] example of collaboration, by the way, of different institutions. Pre-implantation genetics was done here, the in vitro fertilization was done in Colorado, the transplant of the stem cells was done in Dr. Wagner’s clinic, and that’s how we are usually working.

We are specialized in preimplantation genetic diagnosis, and actually any genetic diagnosis, prenatal, and for the rest of the things we cooperate with different institutions.

SUSAN DENTZER: Let’s take another disease that you also conduct pre-implantation diagnosis for, Fragile X syndrome. Tell me how that manifests itself.

DR. VERLINSKY: Fragile X syndrome is a disease where there is expansion of different repeats on X chromosomes. It’s linked to the X chromosome and that’s how it diagnosed. So we can diagnose the disease by analyzing polar bodies, through this special technology that we developed. We also do pre-implantation diagnosis of thalassemia, a disease with defective hemoglobin production.

These patients need transfusions all the time because they have the defective hemoglobin gene, and they usually die at early ages. Now with, with treatment and everything else, it’s very easy to control in the beginning, but then it leads to more and more complications. 
So all diagnoses that can be made after initiating pregnancy can be made before initiating the pregnancy, before transferring the embryo to the mother’s uterus. We don’t do screening. We do testing or diagnosis. That’s major misunderstanding. We’re saying, “This embryo has Down syndrome.” That’s it. It’s not screening.

SUSAN DENTZER: Okay. So let’s say I’m a woman and I and my husband, we’re both carriers of Fanconi’s anemia. We come here. We produce four or five embryos. You test them. What do you say to me once you determine that–

DR. VERLINSKY: I tell you that this embryo, No. 1 or 2, is free of mutation. Or it could be carrier, in which case it has one abnormal chromosome from the father or mother, and a normal one from other partner, and that’s going to be normal baby. And then we transfer a normal one. That’s what we’re doing.

Then, with abnormal embryos, we ask the patient, and this was approved by our internal review board, we ask to have this embryo for research and development.

In one [area] of the research, what we’re doing is examining stem cells to look at how they are growing and how they differentiate into different tissue, and maybe in the future, organs.

SUSAN DENTZER: And how many stem cell lines have you now created from these genetically defective embryos?

DR. VERLINSKY: Right now we have 12 stem cell line with different diseases. For example, muscular dystrophy, Fragile X, thalassemia, Fanconi’s anemia [a carrier], Marfan syndrome. Any embryo, any patient, we ask now [if we can have the abnormal embryos]. If they agree, that’s what we’re doing.

SUSAN DENTZER: And what is, what is the response you typically get from couples or from individuals when they’re asked about this?

DR. VERLINSKY: You can figure yourself. We have 12 lines. This means they are very positive. The alternative [is to] to throw away the embryos, because they come to us to diagnose, and they do not transfer abnormal embryos. Or else they can [contribute to] some benefit for future treatment, or analysis of what disease is all about. It’s very positive, I would say.

SUSAN DENTZER: They feel they’re giving something to the future?

DR. VERLINSKY: Absolutely; absolutely. They feel, not only a feeling, they give something to the future. They’re feeling they participate in something to help the couples as they are, and that’s a very positive and very encouraging moment.

SUSAN DENTZER: So how long have you been growing these 12 cell lines?

DR. VERLINSKY: Oh, they are all frozen already. We’ve tested them, they have the characteristic of stem cells, and then we freeze them. So they’re frozen and in multiple tubes, and, and are available for different researches who are interested to work in this area.

SUSAN DENTZER: And what is the status of that, those efforts on the part of researchers to get those cells?

DR. VERLINSKY: We have a few responses, people who are interested to work in it. A problem needs to be resolved because [people working with federal funding cannot] work on these lines.

SUSAN DENTZER: Right. You’re doing this entirely with private money.

DR. VERLINSKY: Right, it’s the nature of our institution. From the beginning, 50 percent of our revenues are spent on research. When I founded this Reproductive Genetic Institute, that’s what the idea was, not just to have a business.

SUSAN DENTZER: So you’ve been contacted by, by several organizations that are interested in–

DR. VERLINSKY: Individuals and organization, yes.

SUSAN DENTZER: And some disease research foundations also.

DR. VERLINSKY: Right.

SUSAN DENTZER: Who, for example?

DR. VERLINSKY: I don’t know if I am privy to disclose this, but in addition, the Muscular Dystrophy Foundation, the Juvenile Diabetes Foundation. But it’s all just contacts so far.

SUSAN DENTZER: Because there are some issues you need to work out about–

DR. VERLINSKY: Yeah, distributing, [making sure we are not] crossing the line with patents. I can tell you we developed a different way to create stem cell line. We are using different technology for it. But it’s still stem cells. That’s why we need to work it out and not step on the mine field.

SUSAN DENTZER: And then there are also liability considerations you need to resolve–

DR. VERLINSKY: Yeah, liability, but it’s very, very simple because we are giving these lines for research and they [disclose] what they are going to do with this line, and give us a waiver of liability. Then there should be no problem.

But you cannot step any way without lawyer, so that’s–that’s what we’re doing now.

SUSAN DENTZER: You’ve also created a number of normal, if you will, human stem cell lines from–have you not?

DR. VERLINSKY: Yes.

SUSAN DENTZER: What have you done?

DR. VERLINSKY: We–you know, some of the embryos [created here] are normal, [and are] donated by patients after they get pregnant or they don’t want to have another pregnancy. They donate for research and for stem cell research.

So we have, right now, 80 stem cell lines, eight zero, that are not affected by any disease. So altogether about 100 stem cell lines, or approaching that. It’s a dynamic process; we do it all the time. Probably 70 percent of them are fully characterized already, and 30 percent, we still are looking at different markers and different characteristics.

SUSAN DENTZER: So just here, in this lab, you have more than four times as many stem cell lines as are currently authorized for federal funding?

DR. VERLINSKY: Right. I know it’s a surprise, well, for public, but I think they shouldn’t be surprised, considering our leading position in pre-implantation genetic diagnosis. We are in a unique situation. We are testing embryos for disease, and then, with surplus embryos, we know we can [use them for] research, if patients [give their] consent.

So other centers have only one part of all the operations that we have. Some have a very good IVF program but no stem cell research laboratory. Some have the stem cell research laboratory but no pre-implantation genetic diagnosis lab.

SUSAN DENTZER: Have you given away your normal stem cell lines to other researchers?

DR. VERLINSKY: We will.

SUSAN DENTZER: You will?

DR. VERLINSKY: Yeah. We’re willing to share this, all of these lines, because now we’ve actually exhausted our funding opportunities. For us, we need an additional million dollars of funding to be able to do anything.

SUSAN DENTZER: And just give me a concrete example of what some of these other researchers might want these genetically mutated lines for.

DR. VERLINSKY: I think it’s very obvious things. I mean, people create animal models for different diseases, just to study disease. And then they have to move to human model. This is a human model – a very good human model.

SUSAN DENTZER: It’s human.

DR. VERLINSKY: It’s human, and it’s human cells [that will grow into] any kind of tissue. Heart, liver, blood, bone marrow, brain. Anything; you name it. All this differentiation process, it’s a very, very dark room as yet. It only can be studied from molecular point of view. [But one day] you can move to different medicine. You can get from patients their stem cell and then treat disease. I mean, I’m talking about very hypothetical issues, but that’s what it’s all about.

These cells are so important. These cells can be differentiated into any cell type, into organs, and that’s what will give answers to people who study disease, how the different body systems are affected, from the beginning. I think it’s a huge opportunity – huge. I know in England, where they’re not prohibited from creating embryos for research, they will try to create embryos with disease by nuclear transfer and then stem cells from those embryos. Here we have all of them.

SUSAN DENTZER: What do you think of this policy that we’ve now had in place, here, in the United States for the last three years, that has restricted federal funding to research only on those authorized stem cell lines?

DR. VERLINSKY: I have maybe not very standard answer. I think it’s good and bad. And I’ll explain what I mean. Bush got advice. He’s no geneticist. He probably doesn’t have a medical degree, I suppose. But he has an expert who told him, stem cells, we already have enough to study everything. That’s why the restriction was made. What we’ve found out is that it’s not enough, it’s not reliable, we need much more.

But on the other hand, it’s stimulated our creativity. We’ve started to think differently, how to get around it, how to do something to avoid cloning, for example.

SUSAN DENTZER: But you are totally immune from this policy. You can operate on private funding. You can make any stem cells that you want–

DR. VERLINSKY: Right; right.

SUSAN DENTZER: You think this policy has stimulated private funding?

DR. VERLINSKY: Oh, yeah. I think, definitely.

SUSAN DENTZER: And in any case, this policy has not harmed your research efforts at all.

DR. VERLINSKY: It does.

SUSAN DENTZER: It does?

DR. VERLINSKY: Oh, sure, because I don’t have enough money. I have limited capacity. That’s all. That’s–this way, yes, but, again, that’s–one thing it’s na–it’s make–put limitation on it. But limitation by itself push creativity. That’s what I feel.

SUSAN DENTZER: So let me see if I can restate this to make sure I have your argument correct.

SUSAN DENTZER: There are legislative proposals now to allow more stem cell lines to be created and to have federal funding available for research. If they are, if the new lines are created from embryos such as you have here, discarded embryos that are not going to be implanted into uteruses, that are otherwise going to be thrown away.

Is that a good idea?

DR. VERLINSKY: Absolutely. We’d, we’d like to have part of this funding also. I feel we will need as many as possible stem cell lines from human embryos. Hundreds of thousands of embryo are discarded. I think it’s, it’s unique material, and shouldn’t be discarded.

SUSAN DENTZER: So how many embryos do you have here that are not going to be transplanted into uteruses, that probably are not going to become babies?

DR. VERLINSKY: Usually it’s 70 percent of the embryos are not going to be transferred to produce babies.

SUSAN DENTZER: So you have up to several thousand a year of embryos that are not going to be transferred–

DR. VERLINSKY: Right, and particularly with pre-implantation genetic diagnosis, even more.

SUSAN DENTZER: So an endless supply, potentially,of–

DR. VERLINSKY: I think it’s, it’s a big supply, yes, a huge supply of embryos and potential stem cell lines.

SUSAN DENTZER: And you would have no trouble obtaining informed consent from parents to–

DR. VERLINSKY: Yeah. If they read, they’ sign the consent form, which was formulated by special institutional review board, and lawyers.

SUSAN DENTZER: So finally, what do you think is the promise of all of this research, in the largest possible sense?

DR. VERLINSKY: The largest one? I think it’s new medicineyou will have injection of your stem cell once a year to make you as young as you used to be.

SUSAN DENTZER: You really think that?

DR. VERLINSKY: Absolutely.

SUSAN DENTZER: And what do you think will be the application that comes soonest?

DR. VERLINSKY: We already using [hematopoietic] stem cells for treatment of the Fanconi’s anemia and leukemia. I think soon we’ll be using embryonic stem cells to create hemopoietic stem cells for transfer.

SUSAN DENTZER: And why will that be a useful application?

DR. VERLINSKY: Because in a lot of patients don’t have time, or don’t want to create a new baby, to treat a sick child. But creating an embryo and obtaining stem cells will be a short cut to have this.

SUSAN DENTZER: So you actually could have a couple come in with a child with Fanconi’s anemia, they don’t have to have a new baby–

DR. VERLINSKY: Right.

SUSAN DENTZER: –but they want to treat that one child–

DR. VERLINSKY: Absolutely.

SUSAN DENTZER: –So you’d create an embryo, isolate the stem cell, turn it into a hemopoietic cell–And how soon do you think that will happen?

DR. VERLINSKY: It all depends how much funding we’re going to have for it. If I have funding now, in one year we’ll do it.

SUSAN DENTZER: In one year?

DR. VERLINSKY: Uh-huh. [If we get] a million dollars, one year.

SUSAN DENTZER: What are the prospects of getting that?

DR. VERLINSKY: If it’s not going to be, we’ll not get this money. It’s going to be two years. Three years max. We’ll do it with our money. That’s all.