The NIH Director
Director's Speech: 2003 National Press Club Luncheon
SUBJECT: BIOMEDICAL CHALLENGES
MODERATOR: TAMMY LYTLE, NPC PRESIDENT
LOCATION: NATIONAL PRESS CLUB, WASHINGTON, D.C.
TIME: 1:01 P.M. (EST) DATE: THURSDAY, MARCH 6, 2003
SOURCE: FEDERAL NEWS SERVICE, INC.
MS. LYTLE: Good afternoon, and welcome to the National Press Club. My name is Tammy Lytle, and I'm the Washington bureau chief of the Orlando Sentinel and the president of the National Press Club. I'd like to welcome Club members and their guests in the audience today, as well as those of you watching on C-SPAN or listening to this program on National Public Radio.
The video archive of today's luncheon is provided by Connect Live and is available through the National Press Club web site at Press.org. For more information about joining the Press Club, contact us at 202-662-7511.
Press Club members also can access transcripts of our luncheons at our web site. Non-members may purchase transcripts and audio and videotapes by calling 1-888-343-1940.
Before introducing our head table, I would like to remind our members of future speakers. Senator Chuck Hagel of Nebraska will be rescheduled soon. On March 12th, Glenn Close, Oscar-nominated actress and co-narrator of "A Closer Walk," will discuss "A Revolution in AIDS Awareness." On April 8th, Ted Turner, CEO of Turner Broadcasting, will be our guest at the National Press Club. On April 22nd, Senator Gaylord Nelson, counselor to the Wilderness Society and father of Earth Day, will be with us.
If you have any questions for our speaker, please write them on the cards provided at your table and pass them up to me. I will ask as many as time permits.
I'd now like to introduce our head table guests and ask them to stand briefly when their names are called. Please hold all applause until all the head table guests are introduced.
From your right, Albert Teich, director, Science and Policy Programs, the American Association for the Advancement of Science and a new Press Club member; Alberto Cardenas, Texas Tech University and Press Club member; Joyce Winslow, independent health writer and a Press Club member; her excellency Elena Lilly, ambassador-at-large, Grenada, and a Press Club member; John Burklow, associate director for communications at the National Institutes of Health and a guest of our speaker; Larry Lipman, reporter for Cox Newspapers and a former president of the Club; Dr. Raynard Kington, deputy director of the National Institutes of Health and a guest of our speaker; Bill McCarren of U.S. Newswire and vice chairman of the NPC speakers committee.
Skipping over our guest for a second: Ira Allen, Center for the Advancement of Health, and the speakers committee member who arranged today's luncheon; Dr. Alan Spiegel, director of the National Institutes of Diabetes and Digestive and Kidney Diseases and a guest of our speaker; Brenda Craine, director of Washington media for the American Medical Association and a Press Club member; Leslie Gianelli, director of public outreach for the Global Health Council and a Press Club member as well; Kristen Hallam, reporter for Bloomberg News and a Press Club member; and Frank Blanchard, director of communications for the Whitaker Foundation and a Press Club member. Thank you. (Applause.)
Dr. Elias Zerhouni is a rare federal administrator, not only for his talents but for the fact that he runs an agency whose budget has grown faster than the ratings for reality TV. (Laughter.) In the past five years, in fact, the budget of the National Institutes of Health has doubled.
The NIH, of course, is the focal point for American health research, spending about $28 billion a year pursuing basic research into cancer, heart disease, diabetes, AIDS and other life-threatening diseases. Most of the U.S. advances in health in the past 30 years have come from the agency that Dr. Zerhouni now heads.
Since taking office in May 2002, Dr. Zerhouni has faced controversial issues such as stem-cell research, cloning, hormone replacement therapy, the role of corporate funding in NIH projects, and the federal response to the threats of bioterrorism.
A native of Algeria, he got his medical degree there and then emigrated to the United States. Dr. Zerhouni rose quickly in academic medicine to become vice dean of John Hopkins University School of Medicine. A radiologist by training, he looks at familiar images in new ways. He was, in the words of the Hopkins president, "the smartest man I know."
Our guest is also a mathematician and engineer, someone who believes NIH should be moved by data, not opinions. President Bush called him a "quadruple threat" for his abilities as a researcher, clinician, educator and entrepreneur. He also is the father of three who met his wife, Nadia Azza, on the Algerian national swim team.
One of his many talents honed during a youth time of swimming and diving in the Mediterranean Sea is his ability to hold his breath for up to three minutes. (Laughter.) Now, that's a talent that could prove useful in Washington policy debates. (Laughter.)
In addition to believing in evidence and data, Dr. Zerhouni believes in the value of communicating federal science efforts clearly to the public. He has said scientists have an obligation to communicate to the public both the excitement and the accomplishments of science.
Today we will get to see his own communication skills tested. It is my honor to introduce Dr. Elias Zerhouni. (Applause.)
DR. ZERHOUNI: Well, good afternoon, and thank you, Tammy, for this introduction. My standard line now is that I would like to record all the introductions I've received since I became NIH director, because they are all wonderful. And I'd like to record them so I can play them back to my mother-in-law. (Applause.)
And, frankly, I'm really pleased to be here. And as I looked at the notes about the National Press Club, I found out that you were created in 1980 with a budget of $300. And, in fact, in 1887, the NIH was founded with a fund of $300. Now, obviously I'm very pleased that the growth rate of the two budgets has not been the same – (laughter) – and that this year we've been able to complete the doubling of the NIH budget, which I'm very pleased to see happen.
This has been a dream for the biomedical research community. But what I want to talk to you about today is, really, what is the landscape of both health and disease and biomedical sciences? And what has happened over the past 10 years that has radically changed our expectations and our hope for biomedical research in the way we diagnose, prevent and treat disease?
Now, what I'd like to do is really take you back a little bit in time and show you how the challenges that we have today have increased in many ways. The opportunities that we have today have never been better, but also, the fact that things have changed in a revolutionary way, both in the way we understand disease and in the way we treat and do research on diseases.
There's no doubt that NIH has been a terrific federal investment. It has been at the basis, if you will, of most of the discoveries made in the past 50 years that have advanced our health. Of over 100 Nobel prizes, half of all the American Nobel prizes have been trained or funded, developed with NIH's help.
The accomplishments in terms of real health benefits are obvious. The life expectancy of our population has grown tremendously. Let me give you some specifics. If we had not invested into NIH in the research that was done 30, 40 years ago, the question I always ask, what would the picture be today?
In cardiovascular disease, for example, what would be the picture of our health if we had not invested? Well, the number that I found is this: Today we would experience 1.3 million deaths a year from cardiac disease if we had not invested in all of the things we've discovered so far in improving that figure. The real figure today is half a million a year. While a change of 60 percent in mortality is enormous, 500 thousand is still enormous and we still need to make progress there.
If you looked at our investment in AIDS and if we had not invested in AIDS, the natural growth rate of that epidemic would have led us today to experience almost 80,000 deaths a year of young people. We're experiencing five times less than that because of all the discoveries in terms of multiple therapies that are now transforming HIV-AIDS from an acute lethal disease to a more chronic long-term disease.
And this concept that disease has changed over the past 50 years from acute lethal to chronic long-term is a fundamental aspect of the strategy for research that we have to follow.
And let me be more specific. Forty years ago, if you went to the hospital with an acute myocardial infarction, your chances of surviving that event were very low. Today your chances of surviving that event, if you make it to the hospital, is over 90 percent. But that also implies that that patient is going to survive that event but eventually have a more chronic disease.
In cancer, your chances of surviving cancer for five years have doubled. But because of that, surviving with the disease has become a pattern that is increasing. Aging of the population because of the progress that we've made in acute diseases is now leading to the rise in more chronic disease of aging. So the changes in the landscape of disease are important for us to consider as a federal agency.
In addition to that, you also realize that environmental gene interactions affect our population all the time. And I wanted to show you this, which is my salt shaker here. In this shaker here, there is salt that a few hundred years ago would have cost more than its weight in gold. It was extremely scarce. You had to go across deserts to get it. There was a huge industry based on salt trade.
Salt is plentiful. Salt is available for all of us. Well, salt is the basis of many of the cases of high blood pressure that we're experiencing, because this in our environment has changed. And that has changed also the disease landscape that we face.
Another example is food. Food was scarce. Human populations were really designed in times immemorial for scarce food supply. Now we have a plentiful food supply and, lo and behold, we are seeing a huge increase in obesity rates in our society, and it's really a phenomenon that occurs worldwide.
It is something that reflects the constant interplay between our environment and our own ability to adapt to that environment as a population of humans. And the same is true in terms of infection. If you heard the predictions 30 years ago about our ability to control infection, you would have thought the problem was over. We had antibiotics and we were doing terrifically well.
Well, re-emerging infections have now occurred because of the rise of resistance to antibiotics, a new challenge. But we also have the science to address that, hopefully.
In the context of globalization, you see diseases that are arriving on our shores which we never experienced before – West Nile virus, HIV-AIDS. So the landscape of challenges has also changed. And we need to grapple with that, in addition to the fact that we are a diverse society where, in fact, our interactions with the environment, our socioeconomic conditions, is not all the same for all groups in our society.
And one of the concerns I have is this persistent difference in disparities of health between populations of different socioeconomic and economic backgrounds. We need to grapple with that. That's a priority also that remains an important priority, because we're seeing progress across all diseases. If you looked at the mortality rates in cardiac disease, it's going down for all populations, but there's still a persistent difference between several groups in our society, and we need to understand that.
Now, why is it, then, that I'm optimistic and I think that NIH is a good investment in that the investment will be well-used? And the reason for that is I think the landscape of science has changed at the same time. The field of knowledge that we have sought and that we are seeing the results of today is just incredible. I don't have to tell you about molecular biology and sequencing of the genome. You all know about this. But there are things under this, under these events that you are observing, that are fundamentally changing the way we do research.
First, computational power. The ability for us to compute extremely large experimental data sets is leading us to understand biology in a way we never understood it before. I'm an imaging scientist. I can tell you that for the past 15 years my research in imaging sciences has been entirely dependent on the growth of computing power, not on the ground of our understanding of the physics of imaging.
This year there was a publication in Nature from Germany that described computer tomography of a single cell. I started with computer tomography of the entire body. When I started, it took five minutes to acquire one scan and five minutes to process that scan. Today we can obtain the entire body in 30 seconds and we can process that online in three dimension.
The same thing is affecting our basic research, our ability to localize molecules and understand how these molecules are interacting with each other. The research technologies have also exploded to the point where today we can ask questions we couldn't even ask five years ago.
A good example is it took you 10 years to discover the gene for one disease in the '80s. It takes you a week today. Why? Because we have robotics technology; we've advanced in the way to do high through-put experimentation. And clearly there is tremendous hope there.
So where do we go from here? The question that I always get asked is, does that mean that we need to adopt new strategies? You're talking about a changing landscape of disease, a changing landscape of science. Are there new strategies we should consider? Absolutely.
It seems to me that if you look at our problems as a society, where you see a marked increase in health-care expenditures, when our GDP percentage for health care is growing, the aging of our population is going to put even more pressure on that.
The conclusion that you have to draw, which I draw, is this: There is no way that we can sustain the rate of increase as a society without some (auxiliary?) discoveries for us to understand the biology that is affecting us at a rate and at an impact on costs that should be orders of magnitude greater than what we do today.
And I'm talking to you from the basis of my experience as a physician. I was at Hopkins before, and we studied very carefully what is it that we could do to improve the delivery of what we know today, all the methods that we have in hand?
And we found, through multiple computations, that if we applied what we knew today extremely well, we would gain a 10-15 percent advantage. Well, 10-15 percent is the growth rate per year of the total health-care bill. It's not sustainable. So what can we do different? What should we do different? Obviously you heard about Medicare reform and the need to modernize Medicare. Those are good efforts. We need to have them.
One of the aspects of the proposal that has come out to me as a scientist is this: For the first time we're going to focus on prevention, because prevention is, in my mind, and prevention research is, in my mind, one of the most effective ways that we can make a difference.
Now, prevention has been talked about for a long time. It's not a new topic. But why am I optimistic about it? Because for the first time I see the possibility of influencing prevention at a fundamental level. If we understand the genetic events and the molecular events that lead, for example, to the creation of a cancer, the formation of a cancer, you could think of intervening before the cancer occurs.
And the paradigm for the 21st century is going to have to be intervention before the crash of disease – intervention before, not after. Don't pick up the pieces but intervene so that you don't have the accident of disease. So the whole concept of being able to trace back the pre-disease state, the development of the disease in its pathway molecularly and maybe find a way to intervene, is going to be key.
Dr. Spiegel has done studies in diabetes, for example, where he shows that if you could detect the disease earlier, you could in many ways prevent it, using exercise or drugs. But more importantly, it also allows you to delay the onset of the disease and reduce the rate of progression of the disease.
So what you're going to see are different strategies for research and implementation of that research. Molecular prevention is very important; behavioral research to find out how we make the right decisions as human beings. How do we decide that the decision you make today, which may be gratifying but deleterious over the long term, how do we make sure that those decisions are the right one? How do we intervene in molecular prevention?
Today in the news, there was a large study showing that aspirin, taking aspirin, reduces the rate of emergence of colon cancer, early forms of colon cancer, by almost 50 percent. So we have proof of concepts, experiments, that show that molecular intervention early in the development of the disease can make a huge difference.
If you look at our disabled population, patients with disability, we have been able to reduce, over the past 20 years, the rate of disability from about 29 percent to 21 percent. So we have a population where we need to prevent disease, we need to delay the onset of disease if we cannot prevent it, and we need to reduce the rate of progression of disability. Those are the three strategies that we need to implement.
Now, you will see results of this kind over and over again – slow progression. Cystic fibrosis was a disease that essentially was lethal in 15 years. Now you have patients who survive 35, 40 years. Deafness – there is a new implant that I think offers tremendous promise, because we are able to diagnose the disease very early after birth and intervene very early so that the consequences of the disease of congenital deafness are not what they used to be.
I don't want to go too far in giving you too many details, but there is one thing I'd like to share with you, and that is that if you ask the scientists today, "What do you think the 21st century is going to be like, what's your philosophy of research?" and I would like to share mine with you.
Basically I have this notion that we are part of a long history. And I always like to read astronomy research, and I'm amazed that there's a discovery that the universe came from a big bang about 14 billion years ago. When you find out the discoveries, the background irradiation and the discovery of black holes and how this led to the theoretical understanding of how the universe came about, you realize that mankind is trying to traverse backwards astronomically in terms of astronomy.
And I always ask myself, is the same process not the one we see in life, in our life sciences? Well, think about it. About 14 billion years ago, the universe started with a big bang. It took about 9 billion years for the earth to appear, so planet earth has been around for about 5 billion years.
And then when you look at life, well, about 4 billion years ago something happened. Some molecules got together. We do not know how. They self-assembled. We do not know how. We suspect that RNA was the primordial molecule that started the whole process.
But something happened which I call the big bang of life about 4 billion years ago. When that happened with the ability to have these molecules reproduce, have variations, natural selection – and the thing that went on, really, to me the mystery is that all these processes led to more and more complex life forms, that led to us about 3-4 million years ago.
So my dream as a scientist would be to take a time machine equipped with the scientific tools of today and try to be able to study, from that very first big bang, what happened, how did life together, see in an accelerated mode how DNA appeared and how DNA controlled the membranes and proteins.
Well, you know what I'm describing to you is exactly what science is all about in the 21st century. We're trying to reverse, reconstruct, travel in time our entire history, the history of our species. When you think about it, and if I told you, "Well, think in terms of time scale, trying to rebuild life's complexity, the immune system, the reproduction" – if you think in terms of time schedule about our own conversation today, one hour together, and if I told you, "Well, let's think that this hour represents the entire time since the big bang of life, 4 billion years ago, until today," well, the total history of modern humans would cover about one second of our conversation during that hour. And the total duration of our scientific exploration of biological systems to date would be much less than a millisecond. The next century will be a millisecond, or thereabouts.
So we have traversed a tremendous amount of territory in time over the past 50 years, from the discovery of the DNA structure to the sequencing of the human genome. And in the 21st century, I think we're going to realize something that I believe will be also a scientific priority, besides this reverse-engineering process which we are undergoing right now.
We're now, after sequencing the genome, we're going to try to understand all of the proteins that are coded by the genome, how they interact. And then we're going to try to understand the real miracle, and that is that one species a hundred thousand years ago developed a brain which is the only organ that tries to know itself in nature. There's no organ like this. Your liver doesn't try to know its itself, but your brain does. And in the 21st century I think you are going to see a major focus on cognitive neurosciences, on brain-mind interactions behavior. So those would be the two major priorities. And you might ask, How do we do this? Well, clearly the scope and complexity of the problems that I'm describing are daunting, and clearly they will require novel forms of scientific teams that are multidisciplinary, that know how to work together, know how to ask large questions. For example, if you wanted to know how cells signal each other, well that problem is intractable by any one laboratory. There are 2,400 proteins that are playing with each other all the time, and we don't understand how they are talking to each other. We don't understand it within the single cell, we don't understand it across cells fully. In a single cell there's a billion molecules, each one of them with the ability to interact very specifically with its neighboring molecules. That's more than what you would have in a galaxy. And we need to do that. So it will require different mind-sets, it will require the introduction of quantitative sciences, mathematics, biology, physics and modeling of the systems. It will also require new ways of making discovering.
For example, today, as we sit here today, there's 10,000 or more what we call punitive targets – enzymes, proteins, which we think have a major role in certain diseases that we would like to be able to modify with molecules. You know how much – how many drugs are addressing how many targets in the entire pharmaceutical industry? In other words, after a hundred years of pharmaceutical research, there are only 400 biological targets that are targeted by the drugs that we know today. So we have a lot of work to do. But we can do it, because we have the technology today that we never had before. We have the science and the people to be able to do it. We just need to make sure that they are organized appropriately.
And last but not least, we need to translate that into a reality, and that is I think the greatest challenge that I face as the director of NIH is to combine those three poles, the basic science pole that looks at the scope and complexity of the challenge I described to you, and then we have to have the people pole – how do we organize scientific teams in a way that we haven't seen in the past? And the third pole is how do we translate that effectively and quickly to our people, because we are in a race. There's no doubt that if you look at the growth of the problems in health, the potential of our research, I really look at it as a race, and we cannot be complacent or rest on our laurels. And I can assure you that the NIH is doing that. I have great colleagues there. I am amazed at the commitment actually of the NIHers to that mission.
So it's a pleasure to have been here, and I am really looking forward to your questions. Thank you.
MS. LYTLE: Thank you. Secretary Thompson today is announcing a new plan to encourage health care workers, very few of whom so far have been willing to get inoculated, to go ahead and get the smallpox vaccine. Will there be enough inoculated health care workers? And if it's this difficult to encourage them to get it, how will you convince the larger population to get vaccinated down the road?
DR. ZERHOUNI: I should have quit when I was ahead. Can I have the question? I think the issue here is an issue that from the scientific standpoint when we look at it is really an issue of assessment of risks and benefits. There's no doubt that the vaccine as we know it today has a certain level of risk. You need to absolutely build the ability for those who receive the vaccine to be able to withstand a risk or be covered for that risk if that risk exists.
Will there be enough health care workers? We know that if there was an event in smallpox, as soon as – you can still vaccinate people within three, four, five days of their exposure. So if there was an event, we have enough doses in the country, we have expanded our stocks. We are very confident that if there was an event – which means that the risk level is much higher than currently – then the risk benefit computation is so easy to make, the essentially we can vaccinate over half of the population in that five days, and the entire population in 10 days. That's the one response that I would say is important to appreciate.
The second is NIH is working right now on developing what we call a kinder, gentler vaccine, so that we can reduce the background risk level, so that the decision will not be as contentious as it is today.
MS. LYTLE: Editors of 30 leading science journals have agreed to self-censorship when research might provide a benefit to bioterrorists. What role will NIH have in that?
DR. ZERHOUNI: Well, it's very important to understand why we have the need to have some control over the publications and self- control in many ways – because scientists are the best ones to find out and to know what should be transmitted. And we – our bias is to try to be open, because we believe in fact that exchanging information allows you to develop science at a faster rate than anyone else would for malintent. So there is no question in my mind that the bias should be in that direction. But we do believe that since we use – we have made a strategic decision. The president has made that decision to make sure that research on bioagents is made by the civilian biomedical scientific enterprise. This is a very important distinction, because you could have had two choices. You could have used the nuclear research model, where you had closed labs, where everybody is checked out, and you basically censor everything, which was one part, which was used in the nuclear weapons research area; or you believe that an open approach, where all scientists participating in the biodefense efforts bring their own talents and exchange those ideas. This is the model that the administration has chosen, because we believe that if you are going to do research on anthrax or securitization, the basic knowledge you have in molecular biology and other infectious diseases is central for you to make rapid progress, while the counterpart of that is obviously some restrictions on materials and the ability to access that material, and some restrictions on the publications that come out.
So you can't separate the two issues. One is inherent to the decision which I think is a good one to make biodefense something that all of our researchers are committed to.
MS. LYTLE: To what extent will behavioral research supported by NIH be incorporated in the homeland security agenda?
DR. ZERHOUNI: I am assuming that the question relates to psychological biodefense and preparedness for that and research in that regard. I can only tell you from what I know so far that this is a very important topical area, both for NIH research and for the Department of Homeland Security. But I don't know the details at this point.
MS. LYTLE: Of course we discussed the fact that your budget has doubled. But the president's 2004 budget request is much below the 15 percent increases that NIH has gotten in recent years. Can you comment on how you feel about the level of funding as we go forward?
DR. ZERHOUNI: This is actually an area that we have worked a lot on over the past nine months, since I became director of NIH. There's no doubt that you can make sure that whatever happens we preserve what I consider the most precious asset, and that is people, and the people's commitment to do research and continue in their research careers. So when we engineered the budget between 2003, 2004, we on purpose limited the spending that we had on buildings and one-time items, to build for example biodefense laboratories and so on, and reconverted all of these dollars into research dollars. So when you look at the net impact, we have a 15 percent increase in budget in 2003. When you look at the net impact on the research portfolio, it's less than 15 percent, because we have buildings and facilities. But when you look at next year, the administration, what we are proposing is about a two percent total increase. But when you look at the impact on research, it's seven and a half percent. And the reason is because you have a research portfolio and a non-research portfolio, and we try to make sure that there would be a soft landing, if you will, for the research enterprise. And seven and a half percent, including biodefense, obviously the question then becomes, Well, doesn't that distort the picture, because biodefense is growing at a very fast rate? And the answer is if you look at non-biodefense, it's still in the four and a half percent range, which is above scientific inflation. So as a director I work very hard. I think it is something that at least in the 2003-2004 timeframe hopefully we can manage well.
MS. LYTLE: Some members of Congress have talked about holding hearings on NIH funding. Are you concerned at all about cutbacks coming from the Hill?
DR. ZERHOUNI: Is this a nightmare or – (laughs; laughter). I'm concerned about any cutbacks coming from anywhere. I'd be happy to really participate in those hearings, because at the end of the hearings, I'm absolutely convinced that the American representatives, you know, the legislators, and the American people, will realize this one of the best investments. I cannot think of another investment that has been as good as the NIH investment.
If you look at the entire country – world, having an NIH grant now has become a label of quality where promotions are decided on the basis of whether or not you've gone through the peer review competitive process of NIH, well, what agency in the federal government can issue certificates that essentially promote people to the highest reaches of science? I can assure you – I don't want to talk about my sister agencies, but I don't think the IRS would qualify! (Laughter.)
MS. LYTLE: Do you expect you'll have to increase the percentage of corporate funding, and does that cause you any concern over a public perception of a loss of NIH independence?
DR. ZERHOUNI: I'm absolutely concerned always about the independence of the agency in terms of making scientific decisions untainted by any conflict. In fact, I believe the NIH has a mission right now because as we see more interactions between the private sector and the academic sector, I think it's very important for NIH to remain a source of unconflicted information.
Now, partnerships, on the other hand, you can't just imagine that there is no synergy possible, there are lots of areas where synergy is, in fact, welcomed, because I think it will make us advance faster. So I think my view is that you can't live in isolation. Partnerships are important because each party brings added value to the equation, if we have a very specific project with very specific endpoints and no conflict, obviously, that would damage that. But clearly, leveraging is important. We leverage the private sector, the private sector leverages the federal investment. I think that's the way to go. But I have to agree that above all is the preservation of the independence of the science.
MS. LYTLE: NIH had been working on a plan to increase doctoral stipends by 10 percent each year. This questioner says the increase is only 4 percent this year, and what kind of problems does that present, and what's the long-term prognosis there?
DR. ZERHOUNI: This sounds like a hearing! (Laughs; laughter.) I read your Press Club motto and it says that we'd have a friendly and open – (laughter).
Actually, we have – we do want to see better stipends. And I'm not sure about the exact detail of this particular one. We even talked about also increasing indirect costs on career awards and so on. So I'm not totally familiar. But I know that we have made a significant investment in raising the stipends, especially at the post-doctoral levels. And to me, clearly, we want to competitive and we want to be able to attract the best and brightest.
MS. LYTLE: What medical discoveries do you foresee in the next few years, especially on diseases that kill people?
DR. ZERHOUNI: (Pause.) Are there diseases that don't kill people, I was thinking. (Laughter.)
I can name three. One is, I think you're going to see now the emergence of what I call multi-targeted therapy. In a sense, what is becoming obvious to us is that a disease does not just happen because of one cause. There are a series of events, series of genes interacting, proteins that are missing or poorly titrated, some things that happen during the aging process, we don't know. But it's clear that a single-cause-for-a-single-disease idea, even in diseases where we know there's only a single gene that has been mutated, is not a good concept. So the concept of magic bullets that will cure a disease, that is not a good concept. We need to understand the disease.
And what is going to happen – it's happening in cancer, it happened in AIDS – is that we're going to rationally understand the systems of target that need to be hit by drugs or other interventions. So what you're going to see is – in cancer, I think you've already seen the emergence of very targeted therapies, the change in leukemia rates and in lymphoma. You're going to see the same in immune diseases. We now understand better the immune system, and people are starting to think about intervening at multiple levels so that you can reduce the disease. That really, I think, has great potential. When I started medical school, we knew about one or two interleukins, which are messenger proteins between cells. There are now 27. So I think you're going to see that.
The other, I think, is definitely my biggest hope, is in infectious diseases and AIDS, a vaccine for AIDS. I think I'll be very pleased if we could do that during my tenure.
MS. LYTLE: The war on cancer is 30 years old now. When might we win?
DR. ZERHOUNI: We've won a lot of battles. And in fact, I think we're winning in many ways. I don't want to sound like a politician here, but really, when you look at the data and you look at the fact that cancer in many cases has gone from an acute, lethal, short-term disease to a more chronic state. There are 9 million people in this country that live with – have survived cancer.
I think if you define success as complete eradication of the disease overnight, we're not winning to that extent. If you define winning in the sense of delaying the onset, detecting it earlier and preventing its ill effects and allowing longer – good-quality survival, I think we're doing very well in that regard.
MS. LYTLE: What's your position on stem cell research?
DR. ZERHOUNI: (Pause.) (Laughter.) My position is this. I think that the stem cell phenomenon is a very important phenomenon to study. Ten years ago, no one thought that you could reprogram a cell to do something different than what it was programmed for. To me, that's a fundamental observation that needs to be pursued scientifically.
At this point, I think we need a major investment in the basic science of what happens to a stem cell. What are the molecular events that make a DNA be reprogrammed? That answer is not known. No one knows. What are the molecular events that drive a stem cell to do what it needs to do in a good way, replace dopamine neurons or islet cells, and prevents it from turning malignant? Because remember, stem cells can multiply without control. So all of those questions, to me, need to be addressed. And the fact that we have federal funding to look at the fundamentals of stem cell biology, that we can look at the questions that will inform our advances in that regard are key.
So I am extremely optimistic that we need to invest and we need to really understand that phenomenon at the fundamental level as well as the applied level. But we can't progress without more people in the field, more understanding of the biology of stem cells. It is actually quite difficult, when you talk to scientists who are involved – and I was peripherally involved. The difficulty is enormous to just culture the cells and make sure that they remain stem cells. In other words, this is different from anything we've seen before, in the sense that a stem cell has to remain undifferentiated; otherwise, it's not a stem cell. So to keep that state over multiple divisions is not that easy to do, and very few groups have succeeded. So my view is, we should go ahead, bring evidence to the table, do the work, do the science, and my sense is that we should go from the weight of opinion to the weight of evidence on this one.
MS. LYTLE: You've talked about the importance of communicating scientific information. How do you square that with the Department of Health and Human Services taking information off websites regarding abortion and teen sexuality?
DR. ZERHOUNI: I mean, my view is we should communicate. You're referring to the National Cancer Institute website. I think that that was followed by a scientific meeting. The fact is, that website, from what I'm told, had never been the object of a scientific conference, where people would get together and have a consensus. NIH would like to have information that is built on scientific evidence and consensus-driven. So no, I don't think that taking anything down is a good idea, but putting anything up that is not consensus-driven by scientists, with scientists meeting openly about it and reviewing the data, that's what I would like to have.
MS. LYTLE: Which side of the debate do you agree on on the issue of annual mammograms for women?
DR. ZERHOUNI: I'm a radiologist! (Laughs; laughter.) So I'm not the most unbiased person to ask. My sense is that from my experience and my own research, there is little doubt in my mind that screening helps. Now, the issue of what age – younger, older – there's no doubt in my mind that surveillance is important.
And I remember from my personal experience, when I was a resident in radiology and I just started my career, if you may recall, in 1976- '77, there was a radiation scare about mammography. For several years, no mammography was performed in this country until about – between '76 and '81, people – I remember that very distinctly because in about '81, '82, the pendulum swung the other way. I was in Virginia at the time, and I opened two of the very first mammography screening centers, and in the space of three months, we found more cancer that had evolved beyond the stage of cure than I did ever since.
So to me, my personal experiences indicated that there was a need for surveillance, there was a need for early detection. And I don't think that the down side is as stated in terms of whether or not it's justified. Now, I'm not an epidemiologist, I'm not someone to really be the expert on that, but clearly, this is what my bias is.
MS. LYTLE: What do you see as the role of traditional and natural remedies in the spectrum of health care?
DR. ZERHOUNI: As complementary sources of medicines, I think we need to consider that as part and parcel of a total treatment regiment. As replacements or alternatives, I find that a little bit difficult to accept because the scientific evidence, the evidentiary base, is not as advanced or as established as it is for the more conventional forms of therapy.
So from my standpoint, I think we need to forge ahead in building the scientific and evidentiary base for the clinical use of alternative medicines, but clearly send a message that they cannot be considered either as replacement of proven therapies; and second, that their combination with proven therapies in itself needs to be studied very carefully. As you know from studies that have been done on St. John's Wort, the metabolism of that herb can interfere in many cases with the metabolism of effective drugs that we're giving patients, in particular in cancer.
So I think we have to be very careful. There's not one answer that will satisfy. What we need to do scientifically for this field, we need to basically look at it and then invest in appropriate studies to evaluate the value of those approaches.
MS. LYTLE: Animal rights activists have complained about the use of animals in testing and experiments. Can you talk about whether you feel that the use of animals is important?
DR. ZERHOUNI: Well, the – I understand very well the concern of animal activists. There's no question that, you know, you cannot perform experiments without a strong rationale, a strong reason to do that. On the other hand, to say there is no reason whatsoever to use animal models to inform potential approaches for treatment, that I think is a position that I think is too extreme.
But on the other hand, we, as scientists, have to be really responsive and ask ourselves, to the greatest extent possible, what scientific methods, what animal models do we want to use or not use, and are we using them in the most appropriate fashion, so that there is a profound sense of scientific integrity in doing that. So I think it's important to preserve the ability of scientists to do experiments on animal models. They are the only predictors of what could or could not happen to improve our health eventually, or to understand our biology.
On the other hand, it has to be done appropriately so. There's no question that scientifically, as you look at the genomes of many, many different organisms, it has become clear that the similarities at the genomic level, even between small – I mean very early life forms and humans, is very great. The difference genomically between the mouse and the human is 3 percent. And those animal models are important to predict what may or may not happen when we're dealing with a very complex set of questions scientifically, where, as I told you, we're going to have to model multiple targeted therapies, where multiple targets are acted upon at the same time. Well, to be able to do this, I just don't see how we can make progress without well-thought-out, you know, ethical experiments.
MS. LYTLE: Alzheimer's research has made great strides as the budget of NIH doubled, but still no cure. Now you've got 14 million baby boomers coming down the pike. What argument can you make to Congress to keep the momentum up there in the funding?
DR. ZERHOUNI: Well, this is an illustration of what I was describing as the changing landscape of disease. We are dealing with Alzheimer's, a phenomenon of late life. We are doing the research we need to do. But I think the strategies are going to be multi-pronged in the sense that, first of all, we need to understand, again, what are the molecular events that lead to the development of Alzheimer's. We're making great progress in that regard, understanding protein aggregation, which is actually a theme that is common to multiple neurodegenerative diseases. Parkinson's disease now is suspected to be also a protein aggregation disease. So we're making very important progress from the fundamental standpoint.
The second line of research is to try to delay the onset of Alzheimer's as much as we can, because if turns out that if you are able to diagnose it early and have any intervention that will delay its onset, you can really decrease its burden significantly.
And last but not least would be a cure. But I don't think that the standard should be the magic cure for every disease. We have to be realistic, and respectful of these 4 billion years of evolution that have created biological systems that are quite complex. And I think it would be pretentious to think that you can solve this issue in one fell swoop. It's going to require basic studies that will help to find the preventative strategy, studies that help us diagnose the disease before it occurs so that we can delay its onset, and then when the onset is there, delay its progression.
MS. LYTLE: Speaking of prevention, much of the focus in this country has been on disease management and cure. What sort of roadmap will you lay out, generally speaking, to promote prevention programs?
DR. ZERHOUNI: Well again, prevention is going to have two steps. One is what I will call molecular prevention, where you really understand at the molecular level what are the events that lead to that particular disease state and then intervene at that level, as early as you can, as rationally as you can. And we're seeing this. When you use cholesterol-lowering drugs, you are, in fact, performing molecular prevention in some ways, when you use aspirin to prevent heart attacks, or prevent colon cancer. The concept works. It needs to be informed, and we need to pose the question explicitly in our research programs and to our scientists, and drive programs towards that.
The other area of prevention research that needs to be done is this connection between mind, brain, behavior and lifestyles, and we need to understand that. So a lot of effort will be made in investing in neurobiology of behavior and decision-making that relates to differences in the way we respond to different stimuli in the environment. And a good example of that is obesity or other diseases. So, addiction, for example. We need to understand that. That's a frontier we need to go into. And that's what I meant by the 21st century is going to be the century where the brain finally will know itself.
MS. LYTLE: Name some areas that NIH should not be doing research on anymore.
DR. ZERHOUNI: That's a great question because I've asked, actually, the institute directors about that. And my concept of how you keep an organization vibrant is that you have to ask yourself the question, what are the new areas of investment you want to make, because there are opportunities, and oh, by the way, what is it you need to stop doing? And so I asked that question very, very explicitly, and I was actually pleased to see, for example, that the National Cancer Institute has terminated 25 percent of its programs over the past five years. If you look at NI – the Neurological Institute, almost half of the research programs in the intramural program have been terminated and started with new programs.
And I think the rate is increasing in terms of terminating programs and starting new ones, because of what I described in terms of the landscape of science. So, for example, the investments that we're making now in high throughput screening is increasing in multiple institutes. The Institute of Drug Abuse has made an investment and stopped the old approach, which was sort of empirical, and today they're doing systematic analysis of drugs that have an interaction with the receptors that we know have something to do with addiction. So in the past five years, you've seen two drugs appear – buprenorphine is one – that could be used for addiction control in the setting of general medical practice.
The Neurological Institute is doing something very unique and interesting. They've stopped doing the old approach, which is to wait for a drug to be specifically targeted to a particular disease. Well, the Neurological Institute has 600 diseases. Each one of them is a small group of patients, except for stroke and Parkinson's disease, that are very high numbers, and Alzheimer's disease. And what they've done is they've used, for example, all of the approved FDA drugs and tested them for effect in the neurological system. And I'm told that they've made six, eight discoveries of drugs that the effect of which weren't known on the nervous system. A good example of that is the drug Gleevac, which is, you know, the wonder drug for cancer. Gleevac was originally developed for cardiac disease but never worked in the heart, but is found to work in cancer. So those interactions are the new ones, and we've stopped (sizable ?) programs in many cases.
MS. LYTLE: I'm afraid we've run out of time for questions today, but let me just give you this Press Club mug and a certificate of our appreciation. And thank you for being here today.
DR. ZERHOUNI: Thank you very much. (Applause.)
MS. LYTLE: I'd like to thank you for coming today.
I'd also like to thank National Press Club staff members Melinda Cooke, Pat Nelson, Jo Anne Booze, Melanie Abdow Dermott, and Howard Rothman for organizing today's lunch. Also thanks to the NPC Library for their research.
And good afternoon.
END.
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