As Executive Vice Dean at Johns Hopkins I was instrumental in creating an Institute for Cell Engineering. Primarily because I was concerned about the lack of federal funding to advance the fundamental research needed in this new and fledging field. This is why I believe that in the current state of science the August 9 policy set by the president was an important advance. For the first time it allowed NIH funding for stem cell research, something which had not been in the previous administration.
We still have to go on, but there is another important topic that all of you have raised. And, that is that after years of effort from you, Senators, and the rest of the members of Congress, the doubling of the budget is almost here to be completed, as proposed by President Bush. This occurred despite all the difficulties faced by the nation.
KENNEDY: One of the essential functions of NIH is, as you mentioned, provide the best scientific information to Congress and the public and you reiterated that in your comments. Nowhere is this important than on the complex issue of stem cells and cloning. We're all aware that you must carry out the administration's policy, but can you assure the committee that the Congress and the American that you'll provide the objective scientific information on cloning and other scientific issues regardless of whatever political winds may be blowing?
ZERHOUNI: I think my statement reflected that. I mean I've lived by that principle before. And, I will continue to live by that principle. I think science is to be open and transparent. It has to fit from a certain quality standards such as peer review and replication by others. But, all of that should be shared transparently and with everyone, as I told you in our visits.
KENNEDY: Just, if my colleague would yield, I just have a few other areas that I wanted to touch on. And, that is one on disease prevention. The NIH has made astonishing progress in diagnosing and treating disease. Millions of Americans still suffer diseases that are preventable. Do you think that the NIH role -- or the NIH has any role in researching disease prevention?
ZERHOUNI: It has to have a role, Senator. If you look at the statistics, for example, in diabetes, the rise -- the fast rise in incidence should be characterized as a pandemic. This isn't something that we can ignore. And, in many ways, many of the diseases we suffer from are often almost self-inflicted because of, you know, lifestyles and dietary intake and other characteristics.
How we do the research to address these aspects of behavioral modification, a better mix between -- or match between what we know about the genetics of disease and the environment in which we'll live, the better we'll be to prevent these preventable diseases.
I think there are diseases we can't prevent for sure, but those that are preventable we need to do a lot more than what we're doing. Particularly, in terms of the drivers of behavior that leads people to really hurt themselves.
MIKULSKI: It's OK.
ZERHOUNI: ... be careful of the time. Well, first of all, let me say this, I think that the issue of diversity is a very important issue. [...] So, I have a very specific sense about it. And, that is that we need to absolutely understand why is it that we can, to an extent, create more diversity up front and less at the backend?
Managing a complex organization between fire trucks and Nobel prizes is very difficult, as you know. It's combining, essentially corporate management with academic cultures and that is very hard to do. I don't think anyone has the answer, by the way, Senator, in terms of how to best manage an enterprise like this. It's a day-to- day fight by finding the right people to mange those enterprises.
MIKULSKI: Thank you, doctor. I'm going to now yield to my colleague, Senator Wellstone. But, you say you're exactly what I'm talking about. And, even going through our own beloved institution of Johns Hopkins, when I was a young social worker at Catholic Charities I knew that there were no African American physicians at Johns Hopkins. 50 years ago Hopkins had a quota in terms of Jewish physician staying on the faculty. Now, that's gone. We see Levi Watkins and Ben Carson, and Dr. Vogelstein (ph) and so on. You, as a man of the Muslim faith and an immigrant from a medical school in Algiers and you know our snobby our medical establishment can be.
ZERHOUNI: No
WELLSTONE: So, I can get some sense from you, we covered this, but.
ZERHOUNI: Actually, as I indicated to the committee before, I do believe that, in fact, the impact of mental health in itself is a significant burden on society. And, many times the burden is hidden, as you know.
But, in addition to that, I think that all the sciences of behavioral modifications, behavioral sciences, social evaluation of the (inaudible) to which we evolve as individuals, human beings. And, in relation to diseases that are, in many ways, driven by our own behavior, we need to really understand that better because, as I mentioned, epidemics like diabetes are profoundly related to, you know, mental processes and we to do more research in that. There's no question we need to do that.
WELLSTONE: I appreciate that. And, on a personal note, I have other quick question. I would say to Senator Mikulski and you actually, years ago my brother was, who had had a severe mental breakdown, wound up at Johns Hopkins and probably that first year of treatment is what saved him, although it took my parents 20 years to pay off the bill, which is part of the problem. You know, there isn't the coverage, which is we're hoping to change.
Let me just, the last question is on Parkinson's disease. I mean the science, you can -- I don't think you'll disagree, scientists tell us, I say us in this community that it's perhaps the most curable brain disorder. I mean there's a lot of potential here. And, that the -- you know, with adequate research, three to five years we could really have a cure. They also say that these kind of discoveries in Parkinson's also spill over to Alzheimer's, spinal cord injury, ALS, Huntington Disease, you name it.
So, I'm interested in the question of stem cell research because this key and researchers have argued that the 78 stem cell lines available through the president's policy are not sufficient to pursue the therapies for diseases, such as Parkinson's or diabetes and spinal cord injury. They say that a greater diversity on the number of lines will be needed for them to meet the promise of research.
In your capacity as Director, how will you ensure that researchers have the necessary supply of stem cell lines to develop treatments and cures? And, if necessary, will you recommend that the president's policy be broadened to include additional lines?
ZERHOUNI: Well, in terms of where the science is today, and I know a little bit about stem cell research. I organized the institute at Hopkins. There's no question that there is a lot of fundamental research that needs to be done before even considering what pathways we're going to take for cures.
Fundamentally, the process by which DNA's program reprograms is not well-known. And, that needs to be done now. When you talk about the 78 cell lines, let me point out to you, for example, that a lot of research can be done when -- on the limited number of cell lines when they're well characterized. In fact, what I'm saying is the same in other fields of research, for example, if you look at the on the human genome, if you look at the human genome, how many DNA's do we have in the human genome that we're using to do our research? It's a -- well, closely held secret, but we know the number is two to six individuals. And, actually in the last New York Times, one of the individuals said on one of them.
So, you can do a lot when you have these lines. In embryonic stem cell research in mice for 20 years there's been a number of cell lines, about 20, that's been used for fundamental research for understanding the mechanism to know where you go next.
Now, to the second part of your question, it becomes evident to this research that there are pathways to develop cures and so on, I'm going to be the first one to assemble that information, to get the experts to give that information to provide that in the sense of well- established scientific facts and share that with everyone.
WELLSTONE: I appreciate it. I'm not sure whether it was a yes or no answer. And, I'm not the scientist and I appreciate that. But, I'm...
ZERHOUNI: Right.
WELLSTONE: ... in politics and public affairs. I don't think it was quite the commitment that I was hoping to hear. But, I'll follow- up with you on it. OK?
ZERHOUNI: That'll be fine.
KENNEDY: Dr. Zerhouni, we talked about -- or you did about the establishing a national molecular library at NIH. Why is that important? What will that mean for researchers?
ZERHOUNI: Again, this is a concept that we discussed in our interviews. And, it's an idea that has evolved from my experience at Hopkins. I don't know all the ins and outs of how to do it. So, with that caveat, let me describe to you what I believe is needed. When we look at our research and our researchers we have tremendous abilities to technologies like this one to identify which proteins, which genes, are actually affected by disease. But, we do not have the molecules to affect those pathways. And, scientists have a difficult time accessing molecules for research, now not for therapy, not for drugs, for research out of the molecular libraries that are available in the world.
One of us said that I know our scientists at Hopkins, our basic scientists have mentioned to me as an important, and I agree, an important step forward would be to have a national resource where any scientists with a catalog of the appropriate molecules that would be known to effect the genetic pathway X or biological pathway Y. In such a way that over time we will build a library that will allow us to go from fundamental discovery to clinical testing much faster.
That's the idea, but, again, this is my own and not tested and vetted by other colleagues.
KENNEDY: No, it's enormously interesting. I could listen to your answer two or three more times to try and get a better handle on it. But, I think it's very exciting and something that deserves a good deal of thought.
Senator Mikulski, is there anything further?
MIKULSKI: Just one other, as you can see, the Liberal Arts graduates are trying to hang in there. Just a question about, on Page 3 of your testimony, Dr. Zerhouni, about the Institute for Stem Cell Engineering at Hopkins. If we go to the third paragraph from the bottom on page three, you say I was instrumental in creating an institute for stem cell engineering. Primarily because I was concerned about the lack of federal funding to advance the fundamental research still needed in this promising but fledging field.
Question, one, what is the stem cell -- what was and is the Institute for Stem Cell Engineering? You cite its creation, the lack of federal funds to advance fundamental research. In your perspective are federal funds still lacking and should federal funds be available?
ZERHOUNI: At the time, I was aware of the progresses by Dr. John Gerhardt (ph), in particular at Hopkins. I also realized by talking to many scientists at the time that they were shying away from entering this exciting field because...
MIKULSKI: Yes, and tell us what year you established the institute.
ZERHOUNI: We starting working on establishing in 1999. And, we succeed by 2001 because I had to raise funds for it through philanthropy. And, at the time, basically the scientists will tell me; I don't want to go into this field because there's no federal funding. If I have to depend on commercial funding from a company then there are lots of strings attached to that.
So, I said well, we need to get into this field. We need to understand the fundamental mechanism of this differentiation of cells into different tissues and I will work to try to get an institute up and going because I needed two things. One, was the resource, but also the multi-disciplinary teams working together.
Without federal funding it is hard for me to see how you develop a field of science in our country. So, I do believe federal funding is needed.
MIKULSKI: Yes, and then I know that you go on and say that the president's policy was an important breakthrough and that you'll work with whatever rules that Congress passes. Hopefully, Congress will be forward -- hopefully Congress will be as forward thinking as you have been in establishing this institute. |
President George W. Bush embraces his nominee for Surgeon General, Dr. Richard Carmona, as Dr. Elias Zerhouni, left, applauds in the East Room Tuesday, March 26. Dr. Zerhouni is nominated to be the Director of the National Institute of Health. White House photo by Tina Hager. 
