[House Hearing, 107 Congress]
[From the U.S. Government Printing Office]



 
THE NATIONAL INSTITUTES OF HEALTH: INVESTING IN RESEARCH TO PREVENT AND 
                              CURE DISEASE
=======================================================================

                                HEARING

                               before the

                         SUBCOMMITTEE ON HEALTH

                                 of the

                    COMMITTEE ON ENERGY AND COMMERCE
                        HOUSE OF REPRESENTATIVES

                      ONE HUNDRED SEVENTH CONGRESS

                             SECOND SESSION

                               __________

                              JUNE 6, 2002

                               __________

                           Serial No. 107-122

                               __________

       Printed for the use of the Committee on Energy and Commerce


 Available via the World Wide Web: http://www.access.gpo.gov/congress/
                                 house

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                    COMMITTEE ON ENERGY AND COMMERCE

               W.J. ``BILLY'' TAUZIN, Louisiana, Chairman

MICHAEL BILIRAKIS, Florida           JOHN D. DINGELL, Michigan
JOE BARTON, Texas                    HENRY A. WAXMAN, California
FRED UPTON, Michigan                 EDWARD J. MARKEY, Massachusetts
CLIFF STEARNS, Florida               RALPH M. HALL, Texas
PAUL E. GILLMOR, Ohio                RICK BOUCHER, Virginia
JAMES C. GREENWOOD, Pennsylvania     EDOLPHUS TOWNS, New York
CHRISTOPHER COX, California          FRANK PALLONE, Jr., New Jersey
NATHAN DEAL, Georgia                 SHERROD BROWN, Ohio
RICHARD BURR, North Carolina         BART GORDON, Tennessee
ED WHITFIELD, Kentucky               PETER DEUTSCH, Florida
GREG GANSKE, Iowa                    BOBBY L. RUSH, Illinois
CHARLIE NORWOOD, Georgia             ANNA G. ESHOO, California
BARBARA CUBIN, Wyoming               BART STUPAK, Michigan
JOHN SHIMKUS, Illinois               ELIOT L. ENGEL, New York
HEATHER WILSON, New Mexico           TOM SAWYER, Ohio
JOHN B. SHADEGG, Arizona             ALBERT R. WYNN, Maryland
CHARLES ``CHIP'' PICKERING,          GENE GREEN, Texas
Mississippi                          KAREN McCARTHY, Missouri
VITO FOSSELLA, New York              TED STRICKLAND, Ohio
ROY BLUNT, Missouri                  DIANA DeGETTE, Colorado
TOM DAVIS, Virginia                  THOMAS M. BARRETT, Wisconsin
ED BRYANT, Tennessee                 BILL LUTHER, Minnesota
ROBERT L. EHRLICH, Jr., Maryland     LOIS CAPPS, California
STEVE BUYER, Indiana                 MICHAEL F. DOYLE, Pennsylvania
GEORGE RADANOVICH, California        CHRISTOPHER JOHN, Louisiana
CHARLES F. BASS, New Hampshire       JANE HARMAN, California
JOSEPH R. PITTS, Pennsylvania
MARY BONO, California
GREG WALDEN, Oregon
LEE TERRY, Nebraska
ERNIE FLETCHER, Kentucky

                  David V. Marventano, Staff Director
                   James D. Barnette, General Counsel
      Reid P.F. Stuntz, Minority Staff Director and Chief Counsel

                                 ______

                         Subcommittee on Health

                  MICHAEL BILIRAKIS, Florida, Chairman

JOE BARTON, Texas                    SHERROD BROWN, Ohio
FRED UPTON, Michigan                 HENRY A. WAXMAN, California
JAMES C. GREENWOOD, Pennsylvania     TED STRICKLAND, Ohio
NATHAN DEAL, Georgia                 THOMAS M. BARRETT, Wisconsin
RICHARD BURR, North Carolina         LOIS CAPPS, California
ED WHITFIELD, Kentucky               RALPH M. HALL, Texas
GREG GANSKE, Iowa                    EDOLPHUS TOWNS, New York
CHARLIE NORWOOD, Georgia             FRANK PALLONE, Jr., New Jersey
  Vice Chairman                      PETER DEUTSCH, Florida
BARBARA CUBIN, Wyoming               ANNA G. ESHOO, California
HEATHER WILSON, New Mexico           BART STUPAK, Michigan
JOHN B. SHADEGG, Arizona             ELIOT L. ENGEL, New York
CHARLES ``CHIP'' PICKERING,          ALBERT R. WYNN, Maryland
Mississippi                          GENE GREEN, Texas
ED BRYANT, Tennessee                 JOHN D. DINGELL, Michigan,
ROBERT L. EHRLICH, Jr., Maryland       (Ex Officio)
STEVE BUYER, Indiana
JOSEPH R. PITTS, Pennsylvania
W.J. ``BILLY'' TAUZIN, Louisiana
  (Ex Officio)

                                  (ii)











                            C O N T E N T S

                               __________
                                                                   Page

Testimony of:
    Bonow, Robert O., President-Elect, American Heart Association    40
    Hargis, Eric R., President and CEO, The Epilepsy Foundation..    45
    Jones, Daniel, Vice Chancellor, University Medical Center, 
      University of Mississippi..................................    53
    Lenfant, Claude, Director, National Heart, Lung, and Blood 
      Institute, National Institutes of Health, U.S. Department 
      of Health and Human Services...............................     6
    Penn, Audrey S., Acting Director, National Institute of 
      Neurological Disorders and Stroke, National Institutes of 
      Health, U.S. Department of Health and Human Services.......    11
    Sanchez, Eduardo J., Commissioner, Texas Department of Health    49

                                 (iii)














THE NATIONAL INSTITUTES OF HEALTH: INVESTING IN RESEARCH TO PREVENT AND 
                              CURE DISEASE

                              ----------                              


                         THURSDAY, JUNE 6, 2002

                  House of Representatives,
                  Committee on Energy and Commerce,
                                    Subcommittee on Health,
                                                    Washington, DC.
    The subcommittee met, pursuant to notice, at 10:05 a.m., in 
room 2123, Rayburn House Office Building, Hon. Michael 
Bilirakis (chairman) presiding.
    Members present: Representatives Bilirakis, Deal, Norwood, 
Shadegg, Pickering, Bryant, Buyer, Brown, Strickland, Barrett, 
Capps, Stupak, and Green.
    Staff present: Cheryl Jaeger, majority professional staff; 
Steven Tilton, health policy coordinator; Eugenia Edwards, 
legislative clerk; John Ford, minority counsel; and Jessica 
McNiece, staff assistant.
    Mr. Bilirakis. Good morning. I don't like to start a 
hearing without at least one member of the minority being 
present. But, as you may have just heard, we have a journal 
vote on the floor. So it is a start of maybe a tough day; I 
don't know. I think probably the best bet is for us to break 
before we even start, run over and make that vote. By then, I 
am sure Mr. Brown and others will be here and then we could 
start. So if you will forgive us, we will go ahead and do that. 
Thank you.
    [Brief recess.]
    Mr. Bilirakis. The hearing will come to order. I again 
apologize on behalf of the committee. There may be another 
similar type of vote called in a few minutes, unfortunately. We 
shall see.
    All right, I call this hearing to order. I would like to 
thank our witnesses for appearing before the subcommittee 
today, particularly those of you that altered your schedules to 
be here. The subcommittee certainly values your expertise, and 
we are grateful for your cooperation and attendance.
    Over the past 5 years Congress has shown its commitment to 
scientific research by setting a path that would double the 
budget of the National Institutes of Health. I am proud to have 
been an active participant in that effort.
    These increased resources have ensured that our best 
scientists and researchers have access to the funds they need 
to develop treatments and cures for diseases. This funding has 
enabled the NIH to maintain its exalted status as the premiere 
research institution in the world.
    President Bush's fiscal year 2003 budget includes the final 
installment in the 5-year plan to double the NIH budget. I know 
we all hope and expect that Congress will follow suit and 
appropriate the necessary funds to complete this important 
effort.
    As is always appropriate with large investments of taxpayer 
dollars, I believe that it is our responsibility to review, 
with the assistance of the scientific experts at the NIH, how 
these new resources are being used. More specifically, how are 
the various institutes managing these large annual increases? 
Have we found new cures? If so, are they helping Americans live 
healthier lives?
    Given the vastness and the complexity of the NIH, the 
subcommittee is focusing on two institutes today: the National 
Heart, Lung, and Blood Institute and the National Institute of 
Neurological Disorders and Stroke. These institutes are 
critical in the discovery of basic causes of a number of 
diseases. These discoveries will help put researchers on the 
correct paths to cure illnesses like Parkinson's, alpha-1, 
heart disease, and stroke, which devastate millions of 
Americans every year.
    I would like to thank Dr. Lenfant and Dr. Penn for 
appearing before this subcommittee today to outline how they 
are progressing in the war against disease.
    I am particularly pleased that part of today's hearing will 
focus on the Stroke Treatment and Ongoing Prevention Act of 
2001, which was introduced by two members of our subcommittee, 
Mr. Pickering and Ms. Capps. I am very supportive of the 
provisions contained in this bill and look forward to working 
with my colleagues on this issue.
    Now I am pleased to yield to the ranking member, Mr. Brown, 
for an opening statement.
    Mr. Brown. I thank the chairman for holding this morning's 
hearing. I want extend a special welcome to Dr. Bonow with the 
American Heart Association. The Heart Association, as the 
chairman said, has been working with Ms. Capps and others on 
the stop strokes bill.
    Congress will double the NIH budget by 2003. It is rare for 
virtually all Members to endorse any kind of large increase in 
Federal spending for one purpose like this, but when I think 
about constituents I have met over the past 10 years who rely 
on research funded by NIH, doubling the budget is an easy sell 
to Congress and to the American public.
    We have all met children who give themselves daily shots of 
insulin, families who have lost a loved one to lupus or heart 
disease or Duchenne's muscular dystrophy. I have a constituent 
in my district who lost her husband to CJD 3 months after he 
woke up with a headache. Our increased investments at NIH 
afford even more opportunities to confront these diseases.
    I want to briefly touch on other aspects of NIH's role that 
I hope we will devote more attention to during future hearings 
on NIH. I am interested in how the institutes respond to a 
medical need that is not being addressed by the private sector. 
I have been told repeatedly by infectious disease specialists, 
especially talking to people involved in tuberculosis and 
malaria and AIDS, that one area where such a gap currently 
exists, especially in the area again of tuberculosis, is in the 
development of new antibiotics.
    In April 2000 the FDA approved Zyvox, the first in a new 
class of antibiotics to be approved in more than 3 decades. We 
desperately need new antibiotics, especially as antibiotic 
resistance becomes more and more of a problem to fight 
infectious diseases like drug-resistant tuberculosis, like 
pneumococcus, and other bacterial infections.
    According to WHO, too few new drugs are being developed to 
replace those antibiotics that have lost their effectiveness. 
Take tuberculosis, for instance, where four drugs are 
administered to people that have drug-sensitive tuberculosis, 
and then if their tuberculosis is drug-resistant, two other 
antibiotics are given to those patients. They are old 
antibiotics. They are weaker antibiotics that are drug-
sensitive because they have not been on the market for so long, 
and they have much worst side effects than other kinds of 
antibiotics.
    Fourteen thousand Americans die of resistant infections 
each year. Tens of millions die worldwide of treatable 
infections. Eleven hundred people a day in India die of 
tuberculosis. Instead of reverting to older drugs with greater 
and worst side effects, we should be encouraging drug companies 
to devote their considerable resources to antibiotic R&D, but 
if the private sector, as has been the case apparently, is 
unwilling to develop these needed antibiotic drugs, this 
responsibility should fall, and must fall, on NIH with many of 
its new resources available from taxpayers.
    Another area I am interested in is the role NIH invariably 
plays in the economics and allocation of health care in this 
country. Ideally, NIH could steer clear of thorny issues like 
health care costs and access, and focus exclusively on 
producing and supporting medical research.
    Unfortunately, the agency's technology transfer policies 
have an obvious direct bearing on the return consumers receive 
on our investment as taxpayers in NIH. When NIH licenses a 
patent on an NIH-developed medical breakthrough like Taxol, the 
agency's private sector partner is awarded a period of market 
exclusivity. During that exclusivity periods, consumers pay 
monopoly prices for a drug that their tax dollars produced. How 
long should that exclusivity period be? Affordability and 
access hinge on NIH driving a hard bargain. I haven't seen them 
do much of that. This subcommittee has a responsibility to 
ensure that NIH does that.
    NIH also has the power to break the patent on any product 
that was developed with U.S. tax dollars. That is a pretty big 
stick to use to convince drug companies to stop overcharging 
the American consumers.
    If drug inflation weren't a major issue, if American 
consumers weren't paying two, three, and four times what 
consumers in Canada and France and Israel and Japan and England 
and Germany were paying, I am sure no one would look at that 
option given to NIH seriously. But prescription drug inflation 
is a major problem. We all know that Americans are paying more 
than consumers in any other country for the same drugs.
    NIH has information on drug costs that this country needs 
that this committee should see. You know how much it costs to 
develop a drug, including the cost of failures. You have the 
information necessary to clear the air to reality-check the 
drug industry's claim that R&D costs average $800 million per 
drug, which the media obediently picks up and repeats over and 
over and over. We have never seen the facts from your agency, 
from the FDA, or from the drug companies themselves.
    We want to respond appropriately to the public's outrage at 
prescription drug prices. We need to understand how these 
prices relate to costs. There is no way around it.
    Because this hearing is not, however, focused on the issues 
I have just raised, I obviously don't expect answers today. I 
will ask for written responses, share those responses with the 
subcommittee, and my other colleagues, and hope that we can 
pursue, Mr. Chairman, these thoughts and questions and ideas in 
subsequent hearings.
    Our investment in NIH, again, is compromised when Americans 
are priced out of access. Research and access and costs are 
linked. We can't ignore that.
    Thank you, Mr. Chairman.
    Mr. Bilirakis. I thank the gentleman. Mr. Deal, for an 
opening statement?
    Mr. Deal. I have none.
    Mr. Bilirakis. Mr. Stupak?
    Mr. Stupak. I will waive my opening statement, Mr. 
Chairman.
    [Additional statements submitted for the record follow:]
    Prepared Statement of Hon. Charlie Norwood, a Representative in 
                   Congress from the State of Georgia
    Mr. Chairman, I would like to thank you for holding this hearing 
this morning. In the interests of our witness's time, I will be brief.
    The National Institutes of Health are one of our most important 
national resources. The research done at the NIH makes a real 
difference in the health of Americans. From basic research to 
collaborative efforts to cutting edge science, the NIH leads the way in 
health research.
    Work done at the National Institute of Neurological Disorders and 
Stroke has taught us the importance of early intervention when people 
suffer a stroke. We have taken that research and translated it into 
legislation to educate the public on the importance of recognizing the 
signs of stroke. My colleagues Ms. Capps and Mr. Pickering have 
introduced the Stroke Treatment and Ongoing Prevention Act to improve 
stroke care and increase public awareness. I am pleased to be a 
cosponsor of their effort. This is what makes the NIH so valuable--the 
application of NIH research into valuable public policy.
    Mr. Chairman, I would like to thank our witness for appearing 
before us today. I look forward to their testimony and yield back the 
balance of my time.
                                 ______
                                 
 Prepared Statement of Hon. W.J. ``Billy'' Tauzin, Chairman, Committee 
                         on Energy and Commerce
    Thank you, Mr. Chairman, for holding this hearing today. I commend 
the Chairman for taking a closer look at the National Institutes of 
Health, one of the most promising investments we have made to advance 
public health.
    Taxpayer dollars invested in medical research will yield untold 
benefits to all Americans. It is absolutely essential that we ensure 
that the investments we have put in place at the National Institutes of 
Health are maximized.
    Today's hearing will be the first in a series of hearings the 
Committee plans to hold to learn more about the amazing research being 
conducted at the Institutes and Centers of the NIH, and to explore 
options to strengthen the research programs. For the last five years, 
Congress has committed to doubling the budget of the National 
Institutes of Health. If we move to adopt the President's request to 
fund the NIH for fiscal year 2003 at $26.5 billion, we will have 
completed the fifth and final year of this investment initiative. Given 
that we have expanded the budget for NIH rather rapidly, I believe this 
hearing is particularly timely.
    I am pleased that we have the opportunity to hear from not only 
one, but two directors of the institutes at NIH today: Dr. Claude 
Lenfant and Dr. Audrey Penn, of the National Heart, Lung and Blood 
Institute and the National Institute for Neurological Disorders and 
Stroke, respectively. Thank you, for taking the time to address our 
Committee this morning. I look forward to becoming more familiar with 
the advancements being made at these institutes with the additional 
resources Congress has allocated.
    Cardiovascular disease is currently the leading cause of death in 
America, and stroke the third leading cause of death. Although great 
strides have been made to reduce the burden of cardiovascular disease 
and stroke through improvements in detection and treatment, the death 
rate for both are still too high. Furthermore, when we talk about 
doubling the overall budget of the NIH, this increase in funding did 
not necessarily translate into a unilateral doubling of all budgets in 
all scientific areas. Funding for stroke research, for example, has 
remained relatively flat over the past five years. This obviously begs 
the question: are we investing taxpayer dollars at NIH wisely? Are we 
capitalizing on real opportunities for scientific innovation that will 
have a major impact on public health?
    The National Institutes of Health truly is a shining example of a 
public-private partnership. Over 80 percent of NIH dollars are 
distributed through extramural grants. The grant structure we have 
built through the National Institutes of Health has become a 
significant resource for both public and private institutions across 
the United States. Scientists are competing for the opportunity to be 
the next Jonas Salk, to be the one who discovers a vaccine that is so 
widespread that a deadly disease like polio is no longer an immediate 
threat. Scientists are competing for the opportunity to discover new 
scientific theories and laws that will help guide and advance research 
on all disease fronts. It is easy to forget that two decades ago, 
mapping the humane genome seemed an unattainable goal. But yet, now, we 
are there, and I would like to think that the investments we have made 
at the federal level have helped to speed the development.
    Funding medical research and innovation is a worthwhile investment 
of limited taxpayer dollars. Research takes time and patience, and not 
all of our investments in research can be clearly tracked to a tangible 
end product like a new diagnostic or vaccine. But this research, 
nonetheless, helps us as a nation move forward in our efforts to 
improve public health.
    We all need to better understand how medical research is conducted. 
We also need to better understand what impediments are currently in 
place that unnecessarily delay new research developments and 
shortchange the potential impact of research findings. Only when we 
learn about the barriers to high-quality research, can we begin to 
remove them.
    I would like to thank all of the witnesses for coming before the 
Committee today to demonstrate the impact critical research plays in 
saving and improving American lives. I hope and pray that we will 
ultimately reach a point in time that all Americans will be free from 
disease. Congress needs to be a proud partner with the NIH and the 
public in this important goal.
    Thank you, Mr. Chairman. I look forward to hearing from the 
witnesses.
                                 ______
                                 
    Prepared Statement of Hon. John D. Dingell, a Representative in 
                  Congress from the State of Michigan
    Mr. Chairman, thank you for holding this hearing to examine how the 
National Institutes of Health (NIH) is investing taxpayer dollars to 
improve and expand their research activities. I would also like to 
thank the directors from the National Heart, Lung, and Blood Institute 
(NHLBI) and the National Institute of Neurological Disorders and 
Strokes (NINDS), as well as all of our other witnesses, for their 
testimony before us today.
    NIH is a vital and significant institution which conducts basic 
research, observational- and population-based research, clinical 
research, and health services research. The contribution that NIH, and 
its 27 individual institutes, makes to our medical community and the 
general public is unparalleled and invaluable.
    Our stewardship of this multibillion dollar investment of public 
money is crucial to maintaining public support for these programs. 
Taxpayers expect their money to be spent in an efficient and effective 
manner. I hope that we will ask tough questions.
    Continued funding for NHLBI is important, as this institution seeks 
to conduct research on diseases of the heart, blood vessels, lungs, and 
blood; sleep disorders; and blood resources management. Cardiovascular 
disease is the leading cause of death in America. NHLBI conducts 
research related to the causes, prevention, diagnosis, and treatment of 
some of today's most pressing and dangerous health problems.
    NINDS, another valued research institution, currently leads the 
neuroscience community in research on brain disease. This institution 
works to address problems in minority health disparities, Parkinson's 
disease, brain tumors, epilepsy, and stroke. Since stroke is the third 
leading cause of death in the United States, claiming the life of one 
American every three and a half minutes, the work of NINDS is vitally 
important to future of America's health.
    My friend and colleague, Representative Capps, has worked very hard 
to increase funding and focus attention on stroke research, and I 
encourage all of my colleagues to join me in sponsoring Representative 
Capps's STOP Stroke Act.
    Thank you again for holding this hearing, and I look forward to the 
testimony of our distinguished guests.

    Mr. Bilirakis. All right, we will go right into the panel 
then. First, Drs. Lenfant and Penn, you know that you submitted 
your written statement; it is a part of the record. We would 
hope you would complement, if you will, or supplement it 
orally.
    The first panel consists of Dr. Claude Lenfant--am I 
pronouncing that correctly?
    Dr. Lenfant. Yes.
    Mr. Bilirakis. [continuing] Director of the National Heart, 
Lung, and Blood Institute here in Bethesda, Maryland, and Dr. 
Audrey S. Penn, Acting Director for the National Institute of 
Neurological Disorders and Strokes, also out of Bethesda.
    Dr. Lenfant, why don't we start off with you, please, sir?

 STATEMENTS OF CLAUDE LENFANT, DIRECTOR, NATIONAL HEART, LUNG, 
   AND BLOOD INSTITUTE, NATIONAL INSTITUTES OF HEALTH, U.S. 
 DEPARTMENT OF HEALTH AND HUMAN SERVICES; AND AUDREY S. PENN, 
 ACTING DIRECTOR, NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS 
 AND STROKE, NATIONAL INSTITUTES OF HEALTH, U.S. DEPARTMENT OF 
                   HEALTH AND HUMAN SERVICES

    Mr. Lenfant. Thank you very much, Mr. Chairman. I truly 
welcome the opportunity to appear before you today and to 
present some of our programs, a panoramic view, I should say, 
of some of the programs that we are supporting. As requested, I 
will limit my comments to cardiovascular diseases, specifically 
coronary heart disease, stroke, and congestive heart failure, 
and also on blood diseases and resources.
    When the institute was created 50 years ago, Mr. Chairman, 
this country was in the middle of a true epidemic of heart 
disease. Now today, thanks to decades of research, heart 
disease death rates have receded quite markedly. Just to give 
you an example, since the peak of the epidemic, which was in 
1968, the death rate from coronary heart disease has declined 
by 68 percent, and likewise for stroke.
    In addition, all of us in this room today can expect to 
live 6 years longer than was the case 30 years ago. Four of the 
6 years are due to the decline of the death rate from heart 
disease and the progress that we have accomplished with regard 
to this condition.
    However, I should say that we are far from out of the 
problems of heart disease. Today the majority of Americans will 
die from heart disease. In addition, the societal burden of 
living with heart disease is absolutely tremendous. Patients 
spend up to $30 billion each year to take care of their 
condition in acute care, hospital, medication, whatever else. A 
recent study has revealed that 13 of the top 22 prescriptions 
taken in the United States are for cardiovascular diseases.
    Thus, it is very clear that the research that we are 
pursuing is an important step and vital to reduce the 
tremendous burden. Let me take the case of heart failure, which 
accounts for a new public health problem and a new epidemic in 
this country. Ironically, I should say that this problem which 
is emerging is actually the cost of our success. Indeed, having 
saved many people from dying from acute events such as heart 
attack, we have created a large population with damage of their 
hearts.
    What we now have at our disposal is a number of palliative 
measures ranging from medications to instrumental intervention 
such as left ventricular assist devices, but that is not a cure 
for these patients. However, I should say that we are seeing 
surprising new research directions which may eventually bring 
us to a cure. I am referring to cell transplantation treatments 
which may really contribute a great to the treatment of heart 
failure in the future.
    The mapping of the human genome, which has been very much 
discussed in the last months, few months, years, I should say, 
gives us another group of new opportunities on which we are 
capitalizing as much as we can. We can expect that in years 
from now, hopefully sooner than later, we would be able to help 
in the prevention of cardiovascular disease, to predict the 
evolution of a disease if it develops, and, finally, perhaps 
more important, to develop treatments which will be 
personalized for the patients.
    If you will allow me to be a bit futuristic, I can envision 
the time when a child is born, that child will be given at the 
same time a birth certificate and a small CD that will include 
each of her genetic profiles that could be used for the whole 
life of this patient, to be sure that this patient, that this 
subject is treated very adequately when diseased.
    As we pursue these and other avenues, we are working very 
hard to strengthen our clinical research programs. As you might 
expect, we are pursuing a number of clinical trials which 
include medical as well as surgical intervention. They also 
include trials to examine the value of lifestyle interventions.
    We have clinical trials today to evaluate dietary 
approaches which could be acceptable to the public. We have 
clinical trials to prevent excessive gain of weight, which, as 
you know, is a very significant problem in this country, as 
well as to prevent a decline in physical activities, which, 
unfortunately, occurs in our younger population, and that, in 
turn, will lead eventually to excess weight and obesity.
    Let me now turn briefly to work in blood disease, and 
especially in sickle cell disease, a condition which affects 
70,000 of our minority citizens. Here again, I am glad to 
report to you some progress. In 1960, the lifespan of a patient 
with sickle cell disease, the most severe cases, was 
approximately 10 years. Today I am glad to tell you that it is 
between 40 and 50 years.
    Studies are ongoing to address the problem of sickle cell 
anemia and other hemoglobin disorders, as well as the problem 
of the transplantation of hematopoietic cell, that is, a cell 
which supplies all the other cells in the blood.
    I am also pleased to know that as today we talk a lot about 
gene therapy, hemophilia, a very serious condition, inherited 
blood condition, may as it turns out not too far away be the 
first disease to be treated by gene therapy.
    I was asked to make some comments on blood safety. Here 
again, I have to report to you that over the last 20 years, 
when the problems of blood contamination and transmission of 
disease by way of blood transfusion became so apparent with the 
occurrence of AIDS, at that time the risk of contracting 
hepatitis C, for example, from a transfusion was about 1 in 
100--no, it was 1 in 25 units of blood transfused. Today that 
number has been reduced to one chance out of 1.7 million 
transfusions.
    The last thing that I want to mention, Mr. Chairman, is 
that all this research that we have supported will do no good 
to anyone unless it is translated and disseminated to the 
public and to the health professionals. To this end, the 
institute has undertaken a number of programs to assure 
benefits to the patients.
    One which was referred to by Mr. Brown is the Stroke Belt, 
an initiative that was begun by the institute years ago. The 
Stroke Belt is in 11 States from the southeastern United States 
where the prevalence of stroke and high blood pressure, which 
is one of the main causes of stroke, was very high. The 
mortality rate in these States from coronary heart disease and 
from stroke was the highest in the country.
    I am pleased to tell you that today, between the early 
eighties and the mid-nineties, the reduction in stroke in these 
States has been the highest that we have witnessed in the 
entire United States. This shows that, indeed, we can do 
something, and an organization like me, like my colleague from 
the NINDS and all the other institutes are working very hard to 
take to the patients what we can.
    That concludes my remarks, Mr. Chairman, and I would be 
pleased to answer questions.
    [The prepared statement of Claude Lenfant follows:]
 Prepared Statement of Claud Lenfant, Director, National Heart, Lung, 
           and Blood Institute, National Institutes of Health
    Mr. Chairman and Members of the Subcommittee: I am pleased to have 
this opportunity to discuss the programs and activities of the National 
Heart, Lung, and Blood Institute (NHLBI). As requested, my comments 
will focus specifically on cardiovascular diseases--which include, 
among others, coronary heart disease, stroke, and congestive heart 
failure--and on blood diseases and resources.
                        cardiovascular diseases
    To begin with a historical perspective, let me mention that when 
the NHLBI was founded more than 50 years ago, this country was in the 
throes of an epidemic of heart disease. Beginning at the turn of the 
20th century, and particularly after the end of World War I, heart 
disease death rates increased quite precipitously among men and 
ominously among women. One could envision no end to this trend, as 
medical science was largely ignorant about the causes of heart disease 
and extremely limited in its ability to treat or prevent it. Now, 
thanks to decades of research, heart disease death rates among men have 
receded to the level of 100 years ago, and among women they are about 
37 percent lower. Stroke death rates have plummeted, due in great 
measure to improvements in detection and treatment of high blood 
pressure. The average American can expect to live 5+ years longer today 
than was the case 30 years ago, and nearly 4 years of that gain in life 
expectancy can be attributed to our progress against cardiovascular 
diseases. I believe it is fair to say that medical science has made 
more advances in this area than in any other major disease.
    Nonetheless, many challenges remain. As the following chart 
illustrates, we in this country are far more likely to die of 
cardiovascular diseases than of any other cause.
    Moreover, the societal burden of living with these diseases is 
considerable. Cardiovascular disease patients spend more than 30 
million days each year in acute-care hospitals--far more than patients 
with other diagnoses. And, a recent study revealed that 13 of the top 
22 prescription drugs taken in the United States address cardiovascular 
problems. Thus, beyond the suffering caused by these diseases, the 
health-care costs demand our attention.
    Heart failure accounts for a large and growing public health burden 
that has, in effect, become our next epidemic. Ironically, it is a cost 
of our success: having saved many people from dying of acute events, 
such as heart attack, we have created a large and vulnerable population 
with heart muscle damage. We now have at our disposal a number of 
palliative measures, ranging from drugs to instrumental interventions 
such as the left ventricular assist device. While they improve 
patients' quality of life by alleviating symptoms and reducing 
hospitalizations, they are by no means a cure. However, current 
research provides grounds for cautious optimism that a cure may 
ultimately be found. For example, we are stimulating research on cell-
based therapy in the wake of astonishing discoveries that, contrary to 
everything we thought we knew before, cells of the heart and other 
organs are capable of regeneration. If we could find a way to harness 
and direct the body's ability to grow new cells, we would have an 
entirely new approach to therapy for diseases such as end-stage heart 
failure.
    The mapping of the human genome has provided an extraordinary 
opportunity to understand the genetic underpinnings of disease. We have 
initiated Programs of Genomic Applications, which seek to maximize the 
fruits of the new information in order to identify the causes of 
disease, determine who is susceptible to it, and tailor treatments and, 
possibly, cures to the individual. We have also launched a program to 
identify genetic modifiers of disease--genes that determine, for 
instance, why some people with high blood pressure suffer heart 
attacks, while others have strokes, still others experience kidney 
failure, and some escape with few ill effects. The ability to predict 
the course of disease in a given patient will open up a new era of 
therapeutic approaches. Accumulating evidence suggests that 
inflammation--the body's normal, protective response to injury or 
infection--may be at the core of many chronic degenerative diseases 
such as atherosclerosis. This notion is supported by recent findings 
that blood levels of a substance called C-reactive protein, a marker of 
inflammatory activity, are correlated with risk of heart attack and 
stroke. Understanding the delicate balancing act of the immune system 
could pave the way for new preventive and therapeutic strategies. 
Related work from a number of laboratories has found that exposure to a 
variety of infectious agents is associated with development of vascular 
disease. We are vigorously pursuing basic research to elucidate the 
mechanisms underlying these phenomena in the expectation that it may 
ultimately lead to new approaches, perhaps even vaccines, to prevent 
cardiovascular disease.
    As we pursue these and other basic research avenues, we are working 
to strengthen clinical research to ensure that findings from the 
laboratory have a swift and effective impact on patient care. Our 
research centers program has been reconfigured as Specialized Centers 
of Clinically Oriented Research to sharpen its focus on the patient. We 
also conduct numerous clinical trials of promising approaches to treat 
or prevent disease. As you might expect, they include trials of medical 
and surgical interventions, but they also include trials that examine 
the value of lifestyle interventions such as the Dietary Approaches to 
Stop Hypertension (DASH) diet--an eating pattern that is rich in 
fruits, vegetables, and low-fat dairy products and low in fat and 
cholesterol--which has been shown to lower blood pressure. The DASH 
diet is now being tested in the context of an intensive behavioral 
intervention to promote other lifestyle changes to lower blood pressure 
(e.g., decreased salt and alcohol consumption, increased physical 
activity, and weight control). Two other trials focus on preventing 
excessive weight gain among teenaged African American girls--a 
population that is highly susceptible to weight-related problems such 
as high blood pressure and diabetes in adulthood--and on preventing the 
decline of physical activity that typically occurs among girls during 
the middle-school years.
                      blood diseases and resources
    Turning to blood diseases and resources, we also have much progress 
to report. In sickle cell disease, which affects approximately 70,000 
Americans, we have found that hydroxyurea, a chemotherapeutic drug that 
is taken by mouth, decreases the frequency of acute pain crises in 
adults and may actually prolong the life span. We are funding a study 
to determine whether benefits of this drug can be extended to very 
young children, thereby preventing primary damage to organs such as the 
spleen and kidneys. Clinical studies funded by the NHLBI also have 
proven the efficacy of transfusions in preventing the recurrence of 
stroke in young children with sickle cell disease.
    Clinical trials are also in progress to establish whether a cure is 
possible for Cooley's anemia and other hemoglobin disorders such as 
sickle cell disease through transplantation of hematopoietic (blood-
forming) stem cells obtained from sibling donors. The cells can come 
from the circulating blood of the sibling or from umbilical cord blood, 
in cases where there is a newborn brother or sister. Also in this area, 
the NHLBI is funding studies on cord blood transplantation in children 
and adults to determine the most appropriate role for this source of 
stem cells in blood diseases such as acute leukemia. This approach may 
provide new hope for thousands of patients in need of a transplant, 
because cord blood is readily available, can be collected at no risk to 
the newborn donor, is less likely than bone marrow to transmit 
infection, and may work well despite less precise tissue matching,
    Gene therapy for the eventual cure of hemophilia is now under 
development by several companies. The original research leading to the 
actual commercial development of this approach came from funding 
provided by the NHLBI. Our own research in this area is gaining 
addition momentum with recent funding of Programs of Excellence in Gene 
Therapy, which are designed to move these studies rapidly into the 
clinical arena within the context of careful and appropriate safeguards 
for patient safety and welfare.
    In the early 1980s the Institute created a research program in 
transfusion medicine that has actively pursued methods to improve the 
safety of the U.S. blood supply. I am happy to report great success in 
this endeavor. For instance, the risk of contracting hepatitis C from a 
transfusion--a great public health concern--is now about 1 in 1.7 
million units, whereas it was an estimated 1 in 25 units 2 decades ago. 
Taken as a whole, our investment in transfusion medicine research has 
given the United States a blood supply that is the safest in the world.
                         education and outreach
    To maximize the impact of research findings on the people whom we 
serve, the NHLBI is strongly committed to educating patients, health 
professionals, and the public about disease risk, diagnosis, treatment, 
and prevention. Over the past 3 decades, we have conducted education 
programs in high blood pressure, cholesterol, blood resources, smoking, 
asthma, heart attack awareness, obesity, and sleep disorders. Two 
campaigns--one that has been under way for some time and one that is 
brand new--may be of particular interest to the Subcommittee.
    The NHLBI Stroke Belt Initiative had its origins in observations 
during the 1980s that a band of states located generally in the 
southeastern portion of the country (depicted in the graphic on the top 
of the page that follows) suffered an excessive death toll from stroke, 
and that extraordinary rates of high blood pressure were the culprit. 
In subsequent years, we worked with state health departments and other 
groups to address improvement of blood pressure control in these 
populations. The approaches taken are too numerous to mention, but they 
included church-based screenings (``High Blood Pressure Sunday,'' the 
first Sunday in May, is now established in many communities, and 
features sermons, gospel music, and cooking related to lowering blood 
pressure) and screening at baseball games (the ``Strike out Stroke'' 
campaign, which began with the Atlanta Braves). As we look back on 
these efforts, it is clear that stroke is still a major problem in the 
Southeast. However, it is also apparent (see second graphic) that some 
of the greatest gains in reducing the number of stroke deaths per 
100,000 population over the past 2 decades have occurred in the Stroke 
Belt states. Building on what has been learned about improving the 
health of high-risk communities, we are now working to extend our reach 
to other vulnerable subsets of the population. We have established what 
we call EDUCs (Enhanced Dissemination and Utilization Centers) in 
communities whose residents are at especially high risk of developing 
cardiovascular disease. These projects are mobilizing community 
resources--including health centers, churches, schools, businesses, and 
soup kitchens--to increase awareness and control of cardiovascular 
disease risk factors.
    Our very recent campaign, Act in Time to Heart Attack Signs, 
addresses a missed opportunity to save lives. More than 1 million 
Americans suffer heart attacks each year, and about 460,000 of these 
attacks are fatal. In many cases, the deaths occur because heart attack 
victims do not get to the hospital in time to benefit from the 
treatments we have to offer. Why? Often, patients fail to recognize the 
symptoms of heart attack, shrink from the notion of calling an 
ambulance, or worry that they will feel foolish if their distress turns 
out to be ``indigestion.'' The new educational initiative seeks to 
counteract misconceptions about heart attack symptoms, alleviate 
patient fears, and emphasize the importance of getting treatment 
promptly. Materials have been developed--for the public and for 
doctors--to teach people the key messages: (1) recognize the symptoms 
and (2) call 9-1-1. Although the program is only in its 9th month, the 
Act in Time message is already an official course of the American Red 
Cross, and the National Council on the Aging is offering Act in Time in 
senior centers throughout the country.
                               conclusion
    We are confident that our approach, which is driven both by 
compelling public health needs and by extraordinary scientific 
opportunities, will continue to yield progress in the future. I would 
be pleased to answer any questions that the Subcommittee may have about 
the programs and plans of the NHLBI.

    Mr. Bilirakis. Thank you very much, Doctor.
    Dr. Penn?

                   STATEMENT OF AUDREY S. PENN

    Ms. Penn. Mr. Chairman and members of the committee, I am, 
indeed, Dr. Audrey Penn, Acting Director of the National 
Institute of Neurological Disorders and Stroke. I am here today 
to discuss our efforts at addressing stroke, the third leading 
cause of death and a leading cause of disability in the United 
States, with a total cost to the Nation estimated to be in 
excess of $40 billion and immeasurable personal and emotional 
costs to the victims and their families.
    As the institute name implies, stroke is a priority for 
NINDS. We are committed to developing safe and effective 
treatments for all forms of stroke, including strategies to 
maximize knowledge of warning signals, to apply known 
preventative measures, to minimize damage, and protect 
compromised brain, to avert recurrences, and to restore full 
function.
    Historically, NINDS has committed more funding to stroke 
research than any other single disease or disorder within our 
mission. In fiscal year 2001 our funding for stroke research 
was more than $117 million, and across NIH the total was $239 
million.
    Now, as you all know, a stroke is a brain attack which 
occurs when a clot blocks blood flow supplying the brain. An 
ischemic stroke occurs then or, when a blood vessel ruptures, 
you have a brain hemorrhage. In contrast to a heart attack, a 
stroke doesn't usually hurt. Instead, specific regions of the 
brain supplied by the compromised blood vessels stop 
functioning, resulting in unilateral loss of strength or 
sensation, loss of speech or vision, and even loss of 
consciousness, if it is big.
    In some persons, there may be brief episodes, transient 
ischemic attacks, which, if recognized, provide warnings that 
can allow us to use preventative strategies. It is critically 
important that all be instructed in the warning signs of 
stroke, and I would encourage everyone here to take home a copy 
of these bookmarks which list the risk factors and the warning 
signs, and are supplied actually in English and Spanish.
    Over 3 decades, NINDS has supported a series of productive 
clinical studies of stroke. Atrial fibrillation and irregular 
heart rhythm significantly predisposes to embolic stroke, 
especially in those over 60. We have supported clinical trials 
in over 3,800 patients which confirm that aspirin and warfarin, 
which is a blood-thinning agent, were so beneficial that stroke 
incidence was cut by 50 to 80 percent. Optimal use of warfarin 
in appropriate patients could prevent 40,000 strokes per year 
and save $600 million per year in health care costs. In 60 
percent of patients with atrialfibrillation younger than the 
age of 75, a daily adult aspirin provides adequate protection 
against stroke, with minimal complications.
    Transient ischemic attacks, which serve as warnings of 
impending stroke, can suggest the presence of stenosis in the 
carotid arteries in the neck, which is related to 
arteriosclerosis. So we have studied different surgical 
strategies and examined them in a series of major clinical 
trials leading to changes in practice and standard use of 
carotid endarterectomy to clean out the plaque.
    In another trial, which required over 10 years to complete, 
we developed the first FDA-approved acute treatment for 
ischemic stroke, and this is tissue plasminogen activator, or 
t-PA, which dissolves blood clots and restores blood flow, if 
you give it intravenously within 3 hours of the stroke. The 
impressive results show that more patients were out of the 
hospital, free of major neurological impairments, not in 
rehabilitation centers or nursing homes, and back to their 
usual activities at the end of 3 months.
    So to develop units that can deliver rapid treatment for 
strokes and conduct high-quality translational research, the 
institute has issued a grant solicitation for Specialized 
Programs of Translational Research in Acute Stroke, which are 
known as SPOTRIAS. The SPOTRIAS programs will combine the 
latest methods used in neurology critical care units with 
research into neuro-protection, reversal of brain damage, and 
restoration of function after acute stroke.
    In the past several years, research--actually, it started 
in the early 1990's in our institute as well--has revealed 
remarkable capacity of alternate parts of the brain to take 
over functions which have been lost in response to injury. So 
new brain imaging techniques that measure the activity of the 
brain cells involved are providing insights into how they do 
this, and rehabilitation medicine and neurology are also 
beginning to apply what has been learned about this, this so-
called brain plasticity, to encourage stroke recovery through a 
method called constraint-induced therapy.
    Dr. Lenfant referred to the increased incidence of stroke 
in the Stroke Belt which involves, in particular, our African 
American population, but also the general population. We are 
working with the National Heart, Lung, and Blood Institute and 
the National Center for Research Resources in developing a 
Stroke and Cardiovascular Prevention-Intervention Research 
Program at the Morehouse School of Medicine in Atlanta, 
Georgia. We also have an Acute Brain Attack Research Program in 
the Baltimore-Washington area, the pilot of which is the 24-
Hour Stroke Research Program at Suburban Hospital in Bethesda, 
Maryland, part of our intramural program.
    It is critical to continue to pursue and encourage basic 
research into mechanisms of stroke, and continuous advances in 
our knowledge of the biology of brain cells and of brain blood 
vessels, both normal and abnormal, are critical, including 
mechanisms of cell survival and death, neural growth factors, 
stem cell therapy, neuronal plasticity, and glial cell biology. 
We have funded many new projects to study strategies to protect 
these brain cells from the loss of glucose and oxygen 
consequent to stroke. We even have evidence that inflammation 
is involved right at the brain blood vessels.
    We recognize that scientific opportunities and research 
needs, coupled with the increases in the NINDS budget, as a 
result of the recent doubling efforts, mandate the 
identification of clear scientific priorities, so that the 
institute can determine the best uses for its resources. So we 
convened a Stroke Progress Review Group, the PRG, of over 140 
prominent scientists, clinicians, consumer advocates, 
representatives of several concerned NIH institutes, and 
industry representatives, which developed a comprehensive 
document that identifies the scientific priorities to achieve 
breakthroughs in stroke. I believe all of you have copies of 
the Stroke PRG report.
    So, we also recognize that supporting research is only part 
of the battle, and it is critical to help people recognize that 
they are having a stroke, to think of stroke as an emergency 
and as a treatable disease, so that they call 911 to seek help 
immediately. So, we direct an extensive education and outreach 
effort for health care professionals and the general public, 
and these include ``Know Stroke: Know the Signs. Act in Time.'' 
campaigns, where we have a variety of extremely well-received 
public education materials, including television and radio 
spots really given to us by the industry.
    Some of our public education strategies are targeted to 
specific at-risk minority communities. We had a ``Stroke 
Sunday'' program at a local African American church in October 
2000, which included participation by the then Surgeon General.
    We have partnerships, including the Brain Attack Coalition, 
which is a group of professional voluntary and government 
groups. We have signed a Memorandum of Understanding with the 
National Heart, Lung, and Blood Institute, the Centers for 
Disease Control and Prevention, and the Health and Human 
Services Office of Disease Prevention and Health Promotion, and 
the American Heart Association, to foster cooperation in 
reaching the heart disease and stroke goals for the Nation, 
which were articulated in the ``Healthy People 2010'' 
Initiative.
    So we feel we have made, and continue to make, 
contributions, significant contributions to achievements in 
stroke research which have impacted, and will impact, 
prevention, treatment, and rehabilitation. Encouraged by the 
recent progress in understanding the vascular biology of the 
brain, and enabled by the support we are getting from Congress, 
I can assure you that NINDS is committed to pursuing all of 
these opportunities to alleviate the devastating effects of 
stroke on our society.
    Thank you very much for the opportunity to talk to you, and 
I will be glad to answer any questions.
    [The prepared statement of Audrey S. Penn follows:]
    Prepared Statement of Audrey S. Penn, Acting Director, National 
Institute of Neurological Disorders and Stroke, National Institutes of 
                                 Health
    Mr. Chairman and Members of the Committee, I am Dr. Audrey Penn, 
Acting Director of the National Institute of Neurological Disorders and 
Stroke (NINDS). I am pleased to be here before you today to discuss our 
efforts in addressing stroke--the third leading cause of death in the 
United States after heart disease and cancer, and a leading cause of 
long-term disability. The National Institute of Neurological Disorders 
and Stroke at the National Institutes of Health (NIH) is the leading 
federal organization committed to research on improving stroke 
prevention, treatment, and recovery, through increased understanding of 
how to protect and restore the brain. Historically, NINDS has committed 
more funding to stroke research than to any other single disease or 
disorder within our mission. In Fiscal Year (FY) 2001, NINDS funding 
for stroke research was more than $117 million, and the NIH total was 
nearly $239 million. More importantly, our stroke programs impact all 
areas of scientific opportunity and public health priority--from stroke 
awareness to rehabilitation--and are advancing the state of cutting-
edge knowledge about the ways to prevent, diagnose, treat, and educate 
the public and health professionals about stroke.
                               background
    As many of you know, a stroke is a ``brain attack'' caused by an 
interruption of blood flow to the brain. There are two different types 
of stroke--ischemic and hemorrhagic. Ischemic strokes occur when blood 
flowing to a region of the brain is reduced or blocked, either by a 
blood clot or by the narrowing of a vessel supplying blood to the 
brain. Approximately 80 percent of all strokes are ischemic. The 
remaining 20 percent of strokes are caused by the rupture of a blood 
vessel, and leakage of blood into the brain tissue. These hemorrhagic 
strokes can occur from the rupture of an aneurysm, which is a blood-
filled sac ballooning from a vessel wall, or leakage from a vessel wall 
itself weakened by an underlying condition like high blood pressure.
    At every conceivable level, stroke is a tremendous public health 
burden to our country. More than 600,000 people experience a stroke 
each year. Of the more than 4 million stroke survivors alive today, 
many experience permanent impairments of their ability to move, think, 
understand and use language, or speak--losses that compromise their 
independence and quality of life. Furthermore, stroke risk increases 
with age, and as the American population is growing older, the number 
of persons at risk for experiencing a stroke is increasing. Over the 
past several decades, NINDS has supported some of the most significant 
achievements in stroke research, which have contributed to reductions 
in the death rate from stroke. We continue to be committed to reducing 
this burden.
         historical progress in stroke prevention and treatment
    NINDS has a long and distinguished history of supporting productive 
clinical studies in the field of stroke prevention and acute treatment. 
Indeed, successes in prevention date back more than twenty years, and 
there has been remarkable progress in stroke prevention--which reflects 
sustained efforts of private organizations, NIH, and other government 
agencies. Stroke prevention is also highly cost-effective because it 
averts the direct costs of hospitalization and rehabilitation. As NINDS 
celebrated its 50th anniversary, the U.S. Centers for Disease Control 
and Prevention estimated that the age-standardized stroke death rate 
declined by 70 percent for the U.S. population from 1950 to 1996 [MMWR 
Weekly 48:649-56 1999], and the American Heart Association tallied a 15 
percent decline just from 1988 to 1998. I would like to briefly 
summarize a few of the major NINDS-supported efforts, which have 
included dozens of clinical trials, that have contributed significantly 
to our knowledge of stroke.
    Several early studies investigated medical management approaches to 
the prevention of recurrent strokes in people with atrial fibrillation 
(AF). This irregular heart rate and rhythm is a common disorder in 
older Americans, and a significant stroke risk factor. It has been 
estimated that two million Americans, primarily over the age of 60, 
have AF and are six times more likely to have a stroke as a result. The 
drugs aspirin and warfarin had been used to prevent recurrent stroke in 
these individuals, however their use was based on little hard 
scientific evidence. To address this issue, NINDS supported a series of 
three trials in Stroke Prevention in Atrial Fibrillation-- referred to 
as the SPAF trials. The SPAF I, II and III trials evaluated the use of 
aspirin and warfarin for stroke prevention in more than 3,800 human 
subjects. The SPAF I study reported in 1990 that both aspirin and 
warfarin were so beneficial in preventing stroke in patients with 
atrial fibrillation that the risk of stroke was cut by 50 to 80 
percent. The results suggested that 20,000 to 30,000 strokes could be 
prevented each year with proper treatment. The SPAF II study results in 
1994 identified the 60 percent of people with atrial fibrillation for 
whom a daily adult aspirin provides adequate protection against stroke 
with minimal complications. This group consists of those younger than 
75 and those older than 75 with no additional stroke risk factors such 
as high blood pressure or heart disease. SPAF III, which included 1,044 
patients at 20 medical centers in the U.S. and Canada, studied the 
remaining 40 percent of atrial fibrillation patients with additional 
risk factors for stroke and for whom warfarin had been shown to be 
effective. The study was stopped ahead of schedule in 1996 because 
early results clearly demonstrated the benefit of standard warfarin 
therapy over the combination therapy of aspirin and fixed-dose 
warfarin, in these high-risk patients. Other reports have estimated 
that the use of warfarin to prevent strokes in persons with AF costs as 
much as $1,000 annually, but a year of post-stroke treatment can cost 
$25,000. Based on these estimates, optimal use of standard warfarin 
therapy in the appropriate patients could prevent as many as 40,000 
strokes a year in the U.S., and save nearly $600 million a year in 
health care costs.
    Other studies supported by the Institute, such as the Warfarin 
Antiplatelet Recurrent Stroke Study, the Vitamin Intervention for 
Stroke Prevention study, the African-American Antiplatelet Stroke 
Prevention Study, and the Women's Estrogen for Stroke Trial, build on 
these earlier findings, and continue to add to our knowledge about 
medical interventions that can affect the incidence of stroke in 
different at-risk groups.
    The NINDS has also supported several major studies of surgical 
approaches to the secondary prevention of stroke. This work has 
particular significance for people with carotid artery stenosis, a 
narrowing of the major blood vessels that supply the brain. One 
definitive study in the late 1970s examined a procedure called 
extracranial/intracranial (EC/IC) bypass. EC/IC bypass had been used 
for several years as a means to restore blood flow to the brain. The 
NINDS-funded study of the procedure's effectiveness found that the data 
did not support its continued use in medical practice to prevent 
stroke. These findings were of significant benefit to patients, who 
could avoid the risks and costs of this surgery, and to researchers, 
who used this information to redirect their attention to other 
promising approaches. As a result, investigators explored an 
alternative surgical strategy, called carotid endarterectomy, which 
involves the removal of fatty deposits, or plaque, in the carotid 
arteries. In two NINDS-funded trials--the North American Symptomatic 
Carotid Endarterectomy Trial (NASCET), and the Asymptomatic Carotid 
Atherosclerosis Study (ACAS)--this approach was examined more 
extensively.
    The results of the 12-year NASCET trial were reported in two 
stages. The investigators' early data led to a radical change in the 
recommended treatment for severe (70-99 percent) carotid stenosis, or 
blockage, when it was determined that, together with appropriate 
medical care, carotid endarterectomy for patients with severe blockage 
prevented more strokes than did medical treatment alone. NINDS 
responded to this finding by halting the part of the study involving 
patients with severe blockage, and issuing a nationwide alert to 
physicians asking them to consider the study results in making 
recommendations to their patients. The rest of the study focused on 
determining the efficacy of this surgery for symptomatic patients with 
moderate carotid stenosis (30-69 percent blockage). Those results 
showed that patients with the higher grades of moderate stenosis (50-69 
percent) clearly benefit from surgery. There was no significant benefit 
for patients with less than 50 percent stenosis. As a result of the 
NASCET trial, patients with moderate stenosis are better able to decide 
whether to risk surgery in order to prevent possible future strokes.
    In the ACAS trial, carotid endarterectomy was found highly 
beneficial for persons who are symptom-free, but have a carotid 
stenosis of 60 to 99 percent. In this group, the surgery reduces the 
estimated 5-year risk of stroke by more than one-half, from about 1 in 
10 to less than 1 in 20.
    To the long list of studies contributing to improvements in 
secondary stroke prevention, we can add a more recent NINDS-funded 
trial, which resulted in the first FDA-approved acute treatment for 
ischemic stroke, in 1996. This therapy--tissue plasminogen activator or 
t-PA--dissolves blood clots and restores blood flow, if given 
intravenously within the first three hours after an ischemic stroke. 
Patients must be screened carefully before receiving t-PA, since it is 
not appropriate for use in treating hemorrhagic stroke, and should not 
be given beyond the three-hour window. However, in carefully selected 
patients, use of t-PA can achieve a complete recovery. Unfortunately 
many, indeed most, stroke patients do not receive t-PA because they do 
not arrive at the hospital in time to be evaluated and treated within 
the crucial three-hour window of effectiveness. Or, in many cases, 
hospitals are not prepared to rapidly identify and treat these 
patients. It is this dual challenge that NINDS is actively pursuing 
through the development of model systems and through education and 
outreach efforts that are discussed later in my testimony.
                            recent advances
    Within the framework of these historical successes, NINDS continues 
to build its basic science and clinical stroke programs, and to reap 
the rewards of past investments. A sampling of these recent advances 
includes:
The use of medical therapy to prevent recurrent stroke in people 
        without cardiac risk factors
    As described above, past clinical studies provided important 
information about preventing recurrent stroke in people with cardiac 
arrhythmias. However, it has been difficult for physicians to choose 
between aspirin and warfarin for patients who do not present with 
cardiac risk factors. To help address these questions, another large 
clinical trial--the Warfarin versus Aspirin Recurrent Stroke Study 
(WARSS) was initiated with NINDS support. More than 2000 individuals 
with a history of stroke unrelated to cardiac problems participated in 
this study, with equal groups receiving aspirin and warfarin. After two 
years of treatment, there was no significant difference in the 
prevention of recurrent stroke or death, or in the rate of brain 
hemorrhage, in the aspirin and warfarin groups. This finding will 
likely have a major impact on the standard of care for this group of 
stroke survivors, since aspirin is considerably less expensive, safer, 
and easier to administer than warfarin.
The use of the ``warning signs'' of stroke to aid in prevention
    Recently, NINDS-funded researchers evaluated the risk of stroke 
after a transient ischemic attack (TIA), or ``mini-stroke.'' The 
symptoms of TIAs pass quickly, within a day or even hours, and are 
often ignored. After following 1700 people with a TIA, the study found 
that these episodes warn of a dramatically increased likelihood of 
experiencing a stroke within the subsequent 90-day period. Other risk 
factors, such as advanced age, other health conditions, and severity of 
the TIA, also helped to predict stroke risk, and may be useful in 
determining whether patients should be hospitalized immediately and/or 
receive preventive interventions following a TIA.
The development of clinical tools that can be used to predict stroke 
        recovery
    In order to offer clinicians the best possible methods for 
evaluating patients after a stroke, intramural investigators at NINDS 
have explored the types of clinical measurements and diagnostic tools 
that might be used to predict how well a person will recover from a 
stroke. They found that the combined use of a unique type of magnetic 
resonance imaging, the score on the NIH Stroke Scale--a diagnostic tool 
developed at NINDS for evaluating stroke patients, and the time from 
the onset of symptoms to the brain scan, can effectively predict the 
extent of stroke recovery. Future studies will focus on the potential 
of computerized tomography (CT) scanning to predict recovery as this is 
a technology more commonly available in most hospitals. We expect that 
all of these tools will help physicians manage patients more 
efficiently and reduce distress and anxiety among patients and their 
families.
Brain plasticity
    Over the last several years research has revealed the remarkable 
extent of brain plasticity--that is, the capacity of the brain to 
change in response to experience or injury. Scientists are now using 
brain imaging techniques that reveal the activity of brain cells, as 
well as structure, to understand why some patients recover lost 
abilities following stroke and others do not. In other efforts, 
researchers are trying to apply what has been learned about brain 
plasticity to encourage stroke recovery through a method called 
``constraint-induced therapy.'' This therapy involves constraining an 
unaffected extremity while actively exercising the affected one, 
thereby inducing use-dependent brain reorganization.
The use of stem cells to treat stroke in animal models
    Stem cells are immature cells that can multiply and form more 
specialized cell types. Recent animal studies have provided evidence 
that transplanted stem cells can help restore brain function after 
stroke. Other animal research suggests that the adult brain may itself 
have a latent capacity to regenerate new cells following stroke, which 
might be encouraged in efforts to repair the brain. The continuing 
efforts to develop these approaches to restoration of function in 
survivors of stroke build on active NINDS support to understand the 
basic biology of animal embryonic stem cells and adult human stem 
cells. Within the President's policy guidelines, the Institute is 
encouraging research to evaluate the capabilities of human embryonic 
stem cells.
                       current stroke initiatives
    The generous appropriations provided by Congress have made it 
possible for us to expand our programs in stroke, and we are grateful 
for the opportunity. Since the doubling of the NIH budget began in FY 
1999, the Institute has initiated many new clinical and basic science 
projects. Currently, the Institute is supporting 14 Phase III clinical 
trials in stroke, eight of which have been initiated since the start of 
the doubling effort. Even more importantly, the doubling effort has 
enabled NINDS to fund 17 Phase I and II clinical trials in stroke. 
These numbers are impressive and indicate that many novel prevention 
strategies, therapeutic interventions, and rehabilitation techniques 
for stroke are closer to the clinic as a result of the significant 
investments in NIH over the past several years. Areas of clinical 
research that are under exploration include the use of hypothermia to 
improve outcome following aneurysm surgery, the use of magnesium to 
treat stroke, and improvements in stroke imaging techniques. Several 
studies, including research in the NINDS intramural program at the NIH 
Clinical Center, are examining various strategies for rehabilitation 
after stroke including the use of constraint therapy, exercise, 
anesthesia, and electrical stimulation to improve functional recovery.
    NINDS also continues to be committed to exploring stroke at the 
basic science level, and has provided funding for many new projects 
since the doubling effort began. These include studies of procedures 
and drugs that may protect the brain against further injury, a possible 
vaccine for stroke, the role of inflammation, the expression of genes 
and proteins in response to stroke, and pre-clinical testing of 
therapies--just to name a few. Cellular ``communications'' between 
blood vessels, neurons, and glia, and the role of the blood-brain 
barrier, are also subjects of intense interest. In addition to studies 
specifically targeted to stroke, NINDS also provides support for many 
areas of basic neuroscience research that have broad applicability to 
stroke and other brain injuries. These include mechanisms of cell 
survival and death, neural growth factors, stem cell therapy, neuronal 
plasticity, and glial cell biology.
    In addition to the investigator-initiated projects that make up the 
core of our grant programs, NINDS is constantly looking for 
understudied areas in stroke research that the Institute could address 
through the use of targeted initiatives. Several years ago, NINDS 
identified a need for acute stroke centers, and in May 2001, we issued 
a grant solicitation for Specialized Programs of Translational Research 
in Acute Stroke (SPOTRIAS). The goal of the SPOTRIAS program is to 
reduce disability and mortality in stroke patients, by promoting rapid 
diagnosis and effective interventions. It will support a collaboration 
of clinical researchers from different specialties whose collective 
efforts will lead to new approaches to early diagnosis and treatment of 
acute stroke patients. In its report language for the Institute's 
FY2001 appropriation, the Senate also encouraged the creation of acute 
stroke research or treatment research centers to provide rapid, early, 
continuous 24-hour treatment to stroke victims, and noted that a 
dedicated area in a medical facility with resources, personnel and 
equipment dedicated to treat stroke, would also provide an opportunity 
for early evaluation of stroke treatments. The SPOTRIAS program is 
responsive to the recommendation highlighted by the Senate. 
Institutions supported under this program must be able to deliver rapid 
treatment for acute stroke and to conduct the highest quality 
translational research on the diagnosis and treatment of acute ischemic 
and hemorrhagic stroke. They will also help to recruit and train the 
next generation of stroke researchers. The SPOTRIAS initiative will 
facilitate the translation of basic research findings into clinical 
research, and ultimately, the incorporation of clinical research 
findings into clinical practice. The first two centers have recently 
been approved for funding under this program, and as more centers are 
added, it is expected that they will form a national network that will 
lead to significant changes in the care of stroke patients.
    On a more local level, NINDS is also developing the ``Acute Brain 
Attack Research Program'' in the Baltimore-Washington Area. This effort 
has already established a 24-hour stroke research program in diagnosis 
and treatment at Suburban Hospital in Bethesda, Maryland, and our plan 
is to replicate this program in other medical facilities in the 
Baltimore-Washington metropolitan area, next targeting those serving 
predominantly inner city minority populations.
                        stroke research planning
    While a significant knowledge base about stroke has been amassed 
through research supported by the NINDS, continually emerging 
discoveries and new technologies create constantly increasing research 
needs and scientific opportunities. Coupled with the increases in the 
NINDS budget as a result of the recent NIH doubling effort, it is 
necessary to identify clear scientific priorities, so that the 
Institute can determine the best uses for its resources. Such 
priorities will also serve as benchmarks for the broader scientific 
community against which progress can be measured. NINDS convened a 
Stroke Progress Review Group (Stroke PRG) to identify priorities in 
stroke research. The Stroke PRG had its origins in Fiscal Year 2001 
report language from the House and Senate Appropriations Committees to 
the NINDS urging us to develop a national research plan for stroke. 
Following on the success of the Brain Tumor Progress Review Group, a 
joint collaboration between NINDS and the National Cancer Institute to 
identify priorities for research on brain tumors, NINDS decided to use 
a Progress Review Group to develop a plan for stroke research. Members 
of the Stroke PRG include approximately 140 prominent scientists, 
clinicians, consumer advocates--including leaders from the American 
Stroke Association and the National Stroke Association, industry 
representatives, and participants from other NIH Institutes. Together, 
these individuals represent the full spectrum of expertise required to 
identify and prioritize scientific needs and opportunities that are 
critical to advancing the field of stroke research.
    At the Stroke PRG Roundtable meeting in July 2001, and in many 
subsequent discussions, the Stroke PRG report was developed--a 
comprehensive document that identifies the national needs and 
opportunities in the field of stroke research. The final draft of this 
report was submitted for deliberation and acceptance by the National 
Advisory Neurological Disorders and Stroke Council in February, and the 
final report was published in April 2002. The PRG report will be widely 
disseminated to the stroke community, and is available online at 
www.ninds.nih.gov (Search: Stroke PRG); copies were provided to the 
Committee earlier this week.
    Several areas of scientific need are identified in the Stroke PRG 
report, but five consensus priorities emerged from the PRG:

 bIdentification of the genes and proteins that contribute to 
        stroke;
 An improved understanding of the relationship of blood, blood 
        vessels, and brain tissue;
 A better appreciation of how blood flow is regulated and how 
        it can be improved after stroke;
 The development of combination therapies based on molecular 
        and cellular pathways of injury; and
 A better understanding of the neural mechanisms that regulate 
        recovery after stroke.
    Participants also identified a number of scientific resource needs 
including:

 Access to new technologies that allow for large numbers of 
        genes or proteins to be analyzed simultaneously;
 Improved animal models of stroke that better simulate the 
        human disease;
 Improved methods of imaging the brain;
 Improvements in clinical trial design and methods;
 Development of a network of stroke centers;
 A national database that would capture information on the 
        burden of stroke; and
 Better education and training for clinicians in the care of 
        stroke patients.
    The full PRG report expands on all of these issues, and provides 
in-depth analysis of the status of 15 different fields of stroke 
research. As we move forward from the planning process into the 
implementation phase, the Stroke PRG members will work with NINDS staff 
to ``map'' the Institute's current stroke research efforts to the 
recommendations of the report. Using this approach, we will be able to 
identify existing research gaps and resource needs, and to incorporate 
these into a formal implementation plan.
                      health disparities in stroke
    NINDS recognizes that stroke is one of several neurological 
disorders that has a disproportionate effect on minority and 
underserved populations. For example, African Americans are twice as 
likely to die of stroke or complications from stroke as people in any 
other racial or ethnic group in the country, and Hispanics have a 
stroke rate two times higher than that of Caucasians. For this reason, 
we have identified stroke as a critical health disparities issue in 
several Institute planning efforts: health disparities in stroke was 
considered as an over-arching issue by the Stroke PRG panel; stroke is 
one of the top research priorities in the NINDS Five-Year Strategic 
Plan on Minority Health Disparities; and the Institute is also in the 
process of establishing a planning panel that will specifically address 
health disparities in stroke.
    The NINDS is also working to establish prevention/intervention 
research networks throughout the extramural community, particularly in 
regions of the ``Stroke Belt,'' an area in the Southeastern U.S. with 
stroke mortality rates approximately 25 percent above the rest of the 
nation. The goal is to foster stronger linkages between investigators 
at minority and majority institutions and community-based organizations 
in order to improve minority recruitment and retention in clinical 
studies--as one way of addressing health disparities. As part of this 
program, NINDS, working with the National Heart, Lung and Blood 
Institute (NHLBI) and the National Center for Research Resources, is 
developing the ``Stroke and Cardiovascular Prevention-Intervention 
Research Program.'' The pilot phase of this program is at the Morehouse 
School of Medicine in Atlanta, Georgia.
    In addition to these programs, NINDS supports a number of ongoing 
clinical projects that specifically address stroke in minority 
populations, including a new study that will examine the phenomenon of 
the ``Stroke Belt.'' In this study, the role of geographic and racial 
differences as contributors to differential mortality rates will be 
examined and risk factors estimated. We are also engaged in targeting 
special public education efforts to minority populations, as I will 
describe later in my testimony.
                            stroke in women
    In addition, we recognize that stroke is a major health problem for 
women. To address this critical research area, NINDS is supporting 
studies that will help us to better understand gender differences in 
stroke. Specific projects include a clinical study to determine if 
hormone replacement therapy affects stroke severity, and a study 
examining blood flow in the brain and the role of female hormones in 
protecting brain tissue during ischemia. In all clinical trials, we 
ensure that appropriate numbers of women are enrolled, and many of 
these trials involve specific analyses to examine the effects of the 
intervention tested in the female participants. For example, we are 
currently supporting a clinical study that is comparing the efficacy of 
two procedures--carotid endarterectomy and carotid stenting--that 
unblock a clogged carotid artery in the neck, a significant risk factor 
for stroke. Previous research has shown that women may not benefit from 
carotid endarterectomy as much as men do, so one facet of the trial 
will examine gender differences in these procedures.
                    education and outreach programs
    NINDS recognizes that supporting research into new prevention 
strategies and treatment options is only part of the battle in reducing 
the health burden of stroke. Helping people to recognize that they are 
having a stroke, so that they can seek help immediately, is a critical 
first step. To address this problem, the NINDS directs an extensive 
health promotion effort to raise awareness of the signs and symptoms of 
stroke, the need for urgent action if experiencing a stroke, and the 
possibility of a positive outcome with timely hospital treatment.
    In May 2001, the NINDS launched the ``Know Stroke. Know the Signs. 
Act in Time'' campaign, a multi-faceted public education campaign to 
educate people about how to recognize stroke symptoms, and then to call 
911 to get to a hospital quickly for treatment. The campaign's target 
audiences are those most at-risk for stroke--primarily people over the 
age of 50--and their family members, caregivers and health care 
providers. Because stroke attacks the brain, a stroke patient often 
cannot act alone to call 911 and seek medical treatment, so bystanders 
are integral to acting quickly and getting stroke patients to the 
hospital. For this activity, the NINDS developed a wide variety of 
public education materials including airport dioramas jointly sponsored 
with the National Stroke Association, billboard displays, an award-
winning eight minute film, consumer education brochures, exhibits, and 
new radio and television public service announcements (PSAs). All 
indications are that the ``Know Stroke'' campaign has been extremely 
well-received and effective. The television PSA garnered more than 87 
million viewer impressions and hundreds of thousands of dollars worth 
of free broadcast time; the radio PSAs received more than 46,000 
broadcasts on 272 stations; the airport dioramas received more than 800 
million annual impressions; and thousands of nursing homes, hospitals, 
senior centers and other organizations have received consumer education 
materials.
    All of our public education strategies are designed to increase 
awareness of stroke. However, since the problem of stroke is even more 
acute in the African American and Hispanic communities, some are 
targeted to specific at-risk minority communities. These campaigns 
started with outreach to the media in May 2002 for Stroke Awareness 
Month and, in the coming months and years, will include public service 
advertising and grassroots community education components. NINDS also 
co-sponsored a ``Stroke Sunday'' program in October 2000, with the 
American Stroke Association and the Black Commissioned Officers' 
Advisory Group of the U.S. Public Health Service. This program was led 
by the former U.S. Surgeon General, Dr. David Satcher, and I 
participated on behalf of the NINDS. Held at a Rockville, Maryland 
church, the event was designed to bring attention to the major impact 
of stroke in the African American community and to help inform 
participants about reducing their stroke risk.
    NINDS also participates in ``Operation Stroke,'' a coalition of 
health care professionals, allied health providers, civic leaders and 
representatives of community organizations for stroke education. This 
effort is being coordinated by the American Stroke Association, and is 
aimed at the public as well as medical professionals. An intramural 
investigator at NINDS, who is a stroke clinician, is chairing this 
coalition in the greater D.C. and Maryland suburban areas.
    Finally, NINDS has held several meetings and workshops to help 
educate health care professionals about advancements in stroke 
research, like t-PA. For example, our Institute held a major national 
scientific meeting after the publication of the t-PA study that 
involved more than 400 medical professionals. We plan to convene 
another conference later this year to revisit stroke treatment, and to 
explore how more people can be encouraged to recognize stroke as an 
emergency medical situation. The Institute hopes to use this symposium 
to educate healthcare professionals about the benefits of early 
treatment for all stroke patients. In addition, NINDS scientists speak 
at medical meetings all over the country in order to educate physicians 
about effective stroke care, and our grantees produce educational 
videos and offer continuing medical education courses on proper 
administration of t-PA. To complement these efforts, NINDS also 
distributes free copies of the NIH Stroke Scale.
                              partnerships
    As part of our ongoing prevention efforts, we have formed 
collaborative relationships with other NIH Institutes and federal 
agencies, and numerous voluntary organizations. NINDS coordinates the 
Brain Attack Coalition--a group of professional, voluntary, and 
government groups dedicated to reducing the occurrence, disabilities, 
and death associated with stroke--to increase awareness of stroke 
symptoms. To encourage improvements in stroke care, the Brain Attack 
Coalition published an article in June 2000 designed to help physicians 
and hospitals set up stroke centers.
    In February 2001, the NINDS signed a memorandum of understanding 
(MOU) with NHLBI, the Centers for Disease Control and Prevention (CDC), 
the HHS Office of Disease Prevention and Health Promotion, and the 
American Heart Association to foster cooperation in reaching the heart 
disease and stroke goals for the nation articulated in the Healthy 
People 2010 initiative. These goals include: the prevention of risk 
factors for cardiovascular disease (CVD) and stroke; the detection and 
treatment of risk factors; the early identification and treatment of 
CVD and stroke, especially in their acute phases; and the prevention of 
recurrent CVD and stroke, and their complications.
    In order to achieve these goals, we will work with the 
participating partners on focused initiatives such as population- and 
community-based public education and health promotion programs; 
activities to bring about improvements in the nation's cardiovascular 
health care delivery systems; media-based public awareness campaigns 
about the warning signs and symptoms of heart attack and stroke; 
promoting professional education and training, and other activities. 
CDC has already used our public education materials in cooperation with 
their networks, and we are enthusiastic about this partnership, and 
anticipate that it will continue for the next several years.
    NINDS is also participating in the development of a comprehensive 
National Action Plan for Cardiovascular Health--A Comprehensive Public 
Health Strategy to Combat Heart Disease and Stroke. This planning 
process was initiated last year by the CDC. It will chart a course for 
the CDC with the states, territories and other partners--including 
public health agencies, health care providers, and the public--for 
achieving national goals for heart disease and stroke prevention over 
the next two decades. The pillars of this public health strategy 
incorporate the three core functions of public health: assessment, 
policy development, and assurance.
                               conclusion
    NINDS has made, and continues to make, significant contributions to 
the achievements in stroke prevention, treatment, and rehabilitation, 
and we are extremely proud of our accomplishments. However, the 
incremental nature of progress in stroke prevention has confirmed that 
there is no easy route to success. There are still difficult challenges 
to be addressed, and we have invested more than a year in gathering 
recommendations from the best clinicians and researchers in the field, 
as well as our committed partners in the advocacy community, in order 
to help us make the best use of our resources.
    Our planning efforts tell us we must continue to pursue, in 
parallel, several areas of basic, translational, and clinical research 
that may have an impact on stroke. We must find better ways to prevent 
strokes before they occur. We must improve upon and encourage 
acceptance of pioneering diagnostic tools and acute treatments for when 
stroke happens. We must capitalize on the prospect, for the first time, 
of actually repairing the brain damaged by stroke and recovering 
function. The broad portfolio of NINDS research on stroke offers a 
glimpse of what the future might bring--the possibility of vaccines, 
genetic tests to tailor preventive measures for each individual, 
studies that may link infections or inflammation within blood vessels 
to stroke, biological markers that could aid in the identification of 
stroke risk, and new information about how chronic stress and hormones 
may affect susceptibility to stroke damage. Encouraged by the recent 
progress in neuroscience, guided by extensive and inclusive planning, 
and enabled by the support from Congress, I assure you that NINDS is 
committed to pursuing all of these opportunities to alleviate the 
devastating effects of stroke on our society.
    Thank you again for the opportunity to speak with you today. I 
would be happy to answer any questions you may have.

    Mr. Bilirakis. Thank you very much, Dr. Penn.
    I will start off the questioning. First of all, by the way, 
the bookmarks that you referred to are back at that rear table.
    Ms. Penn. Yes.
    Mr. Bilirakis. They are in English also? The one that is 
available up here is in Spanish.
    Ms. Penn. Yes, there should be two sets.
    Mr. Bilirakis. Good enough.
    I guess this question may be for both of you: The 
Department of Defense, as we know, has also invested, Dr. 
Lenfant, significant dollars in blood research. So they are 
conducting blood research also. We have represented here today 
by the two of you two institutes, the Institute of National 
Heart, Lung, and Blood Institute, your institute as well as Dr. 
Penn's institute.
    So I guess my question goes to coordination. It is 
something that has always concerned me. Maybe I am placing too 
much emphasis in my own mind on that, but there's Veterans' 
Administration research, Department of Defense research, 
similar type of research by your two institutes. My question 
goes to coordination.
    Is much duplication taking place? Is that duplication, if 
it is taking place, necessary? In order to reach the ultimate 
result, is some duplication a necessary evil, if you will, if I 
can call it that? What coordination takes place among the 
institutes with other departments of the government, et cetera, 
university research, all that? Yes, sir?
    Mr. Lenfant. I think the varieties of cooperation and 
collaboration are many, and I would say ideal. First of all, 
Mr. Chairman, we are all on the same campus. We bump into each 
other all day. So we have this free-wheeling discussion about 
topics and whatever.
    But there are, as Dr. Penn mentioned to you, some formal 
ways to do it. For example, a few months ago, our two 
institutes signed a Memorandum of Understanding to assure our 
corroboration and cooperation in areas of mutual interest. But, 
furthermore, not only do we do it with the two of us, but we 
worked with the American Heart Association and other agencies 
of the government such as CDC. There was also the Office of 
Prevention from the Department, and we discuss things.
    If you are looking at the initiatives which are issued by 
NIH, that is, new programs which are initiated, you probably 
would be amazed to see that there may be as many as 10 
institutes sponsoring one program. We all contribute the 
technical contributions, technical support, but also monetary 
support.
    Mr. Bilirakis. How do you tie into other departments, the 
VA, the Defense, the universities?
    Mr. Lenfant. I will go into that. With the VA, we have many 
cooperations. In fact, we have a number of joint studies in our 
institute with the VA. You mentioned blood safety earlier. We 
do have exchange of information. In fact, I believe--and I 
would have to verify that--but I believe that we have a project 
on blood safety and substitutes that is, quote/unquote, 
artificial blood. We have programs which are jointly supported 
between our institutes and the Department of Defense. So there 
is a lot of cooperation.
    The idea is that you want enough in the open so that people 
know about it. We cannot, with the rules of NIH, we cannot 
issue a new program, initiate a new program, without posting it 
for, I believe it is, 2 weeks for anyone to see it, and to 
indicate its intents, that is, another institute or somebody 
else. That happens all the time.
    We could probably do more.
    Mr. Bilirakis. Does that information get to the other 
departments in some way? Does it become known to the 
universities?
    Mr. Lenfant. The NIH publishes widely on the Internet and I 
believe also on paper all the things which are being developed.
    You also asked about the academic community. Most, not to 
say all, of the things which are initiated, new programs, by 
the National Institutes of Health, and certainly by our two 
institutes, are actually the result of deliberations and 
discussions and debates, I should say sometimes, with the 
scientific community. In fact, we do have an obligation, either 
by rule or legislation--I must admit I don't know which one it 
is--but to basically seek the support of our national advisory 
council, which is made up of representatives of the public as 
well as the scientific community, before we can start the new 
programs.
    Mr. Bilirakis. Well, my time has expired.
    Mr. Lenfant. I'm sorry.
    Mr. Bilirakis. Dr. Penn, if you wanted to take 30 seconds 
or a minute to maybe expand upon what Dr. Lenfant said?
    Ms. Penn. I would say it depends somewhat on what kind of 
science or medicine you are trying to drive. In the case of 
issues actually of a particular type of therapy for Parkinson's 
disease, we are working with the Veterans' Administration, also 
through a Memorandum of Understanding between the two agencies, 
because they have patients that the general academic health 
centers don't have. They started this, and they asked us to 
work with them. We helped design this large clinical trial of 
deep brain stimulation. So that is one example.
    Another example depends somewhat, as Dr. Lenfant just said, 
on who the investigators are, and the investigators really do 
work both through the Department of Defense, some of them are 
in the VA system; some of them are coming to us for grants. We 
just have to--you know, we know who is getting support from 
whom. In some cases, such as the prion diseases that Mr. Brown 
referred to, we have an action plan in the Department, which 
involved all the institutes as well as several other units of 
HHS. That really did require collaboration, and that was not 
overlap. The prion diseases are a major problem or potentially 
a major problem.
    Mr. Bilirakis. So I am unduly concerned then? I am unduly 
concerned about that problem? There isn't that much 
duplication?
    Ms. Penn. Well, we do talk to each other first, is what I 
am getting at.
    Mr. Bilirakis. Thank you.
    Mr. Brown?
    Mr. Brown. Thank you, Mr. Chairman.
    I think that probably Members of Congress, when they look 
at NIH and look at CDC, they think of, first of all, they fund 
NIH to the tune of about $4, $5 for every dollar that Congress 
funds CDC, and there are a lot of reasons for that. One of 
them, I think, is that all of us know people that have awful 
diseases in our families, our friends, our constituents. NIH is 
a terrific agency responding to those challenges.
    Most of us don't know, frankly, because of the way we live 
our lives and run our offices, don't know a lot of people that 
are poor, where CDC, which is not an agency only for the poor, 
but an agency that seems to be that to many people in this 
body. I think of NIH, I think of both CDC and NIH as public 
health agencies. I am not sure you would characterize 
yourselves that way, but you are a public health agency in the 
sense that you respond to health demands, to public health 
demands, in terms of basic research, sometimes in the case of 
Taxol more than simply basic research.
    But your charge in many ways, in my mind, is that when 
neither the private sector in terms of antibiotics, as an 
example perhaps, and a host of other awful developing world 
illnesses where there is not much money for private 
pharmaceutical--not much monetary incentive for private 
pharmaceutical companies to respond, or in the sense of 
sometimes the public where we didn't respond publicly quickly 
enough, to the public sector, on something like coming up with 
responses to the awful AIDS epidemic.
    So my questions is, how does NIH respond when it is clear 
that a medical need is not being sufficiently addressed? Do you 
see your model in part the way that the Department of Defense's 
Walter Reed took it upon themselves, with little private 
involvement and private money, simply to develop malarial drugs 
and not exactly go to market with them, but really develop them 
almost in toto--do you see your role fulfilling a public need 
in that way?
    How poorly we have done developing antibiotics, how we 
haven't done well enough in malarial vaccines, how we haven't 
done well enough for--I know less about this--but river 
blindness and various awful Third World diseases that none of 
our constituents will have, and certainly nobody that dresses 
the way I do will probably have.
    Not that I dress that well. I have heard from several 
people how ugly this tie is, but because it is from Children's 
Hospital I get a break on it.
    Go ahead.
    Ms. Penn. The mission of the National Institute of 
Neurological Disorders and Stroke is to apply research in basic 
neuroscience to solving our major disorders. We, obviously, 
focus on disorders of the central and peripheral nervous 
system, and to some degree muscles. So muscular dystrophy is in 
there.
    So we look for opportunities by looking, on the one hand, 
at the scientific opportunities that are there that our basic 
scientists and our clinical scientists are providing us, and 
then saying, okay, how can we move this into the clinic? We 
don't go so far, necessarily, as to start by saying--well, we 
do say we want to treat this. This is underserved or not 
treated well or it has got way too many side effects. We could 
do that.
    But we concentrate really--and it is becoming increasingly 
clear to all of us--that we have to do the translational 
research. So that we are right in the middle of taking the 
mechanisms and saying, okay, if you tweak this or you do that, 
you are going to get a therapy. Then we have to least start to 
work out the therapy and say, it's safe; it isn't safe; how 
much do you need, and all of this.
    At some point in there we do have to talk, and we often do, 
to small or large pharmaceutical companies because they are 
going to finally make this drug and market it. I am really not 
into what they do. We leave it a lot to the NIH Office of 
Technology Transfer, as you mentioned.
    But we do look, and sometimes get a little concerned, as 
all citizens do, about how things are being done in terms of 
delivery of care and all of that, but we are really into taking 
the science, making sure the science is done, making sure the 
science--some of our science is really purely at the bench and 
purely doesn't seem to be related to anything, but sooner or 
later it is. It is amazing how much it is. We think that is the 
key, to figure out where, all of a sudden, okay, that 
breakthrough is going to work on that.
    Mr. Brown. But the other key is when there is not private 
sector incentives. There apparently hasn't been in antibiotics, 
and with multi-drug resistance. Was NIH taking--not your 
institute, but NIH as a whole--taking on that public burden?
    Ms. Penn. I would just say this is not something we think 
of first; we really don't. But, I mean, sooner or later, we do 
have to consider that.
    Mr. Brown. But your charge as a public health agency needs 
to be broad enough, as we have just piled on money for you--to 
think of doubling the budget over 5 years, when of course there 
are untold number of scientific opportunities and thoughts and 
proposals and ideas and ideals, but we need not you and 
necessarily your institute, but the NIH as a whole needs to 
think what ultimate, not just basic scientific research, but 
what ultimate public goals do you have.
    When you see antibiotic resistance, you see what happens 
with TB, you see we are going back to the 1940's and 1950's 
antibiotics, which have terrible side effects, that people have 
to take for 2 years to treat multi-drug-resistant TB, as it is 
getting worse and worse, there is a public need there that the 
drug industry probably won't address, and who else will if not 
you with a doubled budget? Just comment.
    Mr. Bilirakis. Yes, a brief comment to that.
    Mr. Brown. Dr. Lenfant, if you----
    Mr. Lenfant. Yes, I would like to add to what Dr. Penn has 
said. I cannot speak about antibiotics because that is not 
something that we are doing and are involved with. But what I 
can say to you, Mr. Brown, is that there are medications which 
have the potential to have an effect on disease. Well, let me 
put it this way: on diseases which basically are not on the 
label of the medication. You can be sure that the 
pharmaceutical industry will not engage in exploring the whole 
possibilities that one medication may offer.
    In our institute we initiate many clinical trials, and the 
reason we do it, it is basically because we know that industry 
will not do it, either because, if it is done, it may not have 
enough impact and the business aspect is small to get into 
that, but we take it. In effect, we know that there are some 
significant benefits which are given to the public and the 
patients by this kind of approach to explore what it is and 
investigate what it is that is not being done by the industry, 
for whatever reason they may have, which may be business, which 
may be--I don't know what it is, sometimes liability issues. We 
have the capability to do it, and we do it.
    Mr. Bilirakis. Would you say that you speak for the NIH as 
a whole?
    Mr. Lenfant. I believe--well, you know, I can tell you at 
least one institute that I know a little bit better than most, 
it is the Cancer Institute. I know that the Cancer Institute 
has a drug development program which is extraordinarily active. 
Basically, I suppose if the industry would do it, they would 
not have that program. I am sure that there are many other 
examples with regard to NIH.
    Mr. Bilirakis. Well, thank you. I am not sure that Mr. 
Brown is completely satisfied, but we've really got to go on.
    Mr. Brown. Thank you.
    Mr. Bilirakis. But it is a good start.
    Mr. Deal, to inquire?
    Mr. Deal. Thank you, Mr. Chairman.
    As I understand it, each of you represent 27 institutes or 
centers under the umbrella of NIH. I would like to find out how 
the operational activities take place within each institute. 
Could you give me an idea, first of all, as to what percentage 
of the research is done through extramural grants as opposed to 
in-house research? And what process do you use to determine to 
whom those extramural grants will be given? Do you have a 
review panel within each institute, I assume? Is that review 
panel made up of people from the outside who are recognized 
experts? Would you elaborate on those two areas? First of all, 
how much is done in-house versus grants, and how do you decide 
how the grant process is going to work?
    Mr. Lenfant. Well, okay, let me take it. These are very 
important questions.
    Let me say that, as a rule, the amount, the fraction of the 
total NIH budget which is for intramural research is pretty 
much fixed. I think the average for the NIH as a whole is 11 
percent. Eleven percent of the total is intramural. It varies 
greatly. My institute is the one with the smallest intramural 
budget. It is, I believe, 6 percent, but of course it is 6 
percent of $2.7 billion. So it is a significant amount of 
money.
    It varies. It may go up and down a little a bit, but not 
significantly. But years ago there was a review of the 
intramural programs, and the decision was made by all of us 
that we would pretty much limit our intramural research to that 
amount for the corporation.
    Now the extramural research is, indeed, all decided by a 
peer review process. Applications for grants are all coming 
into a central place at NIH. That place has two 
responsibilities. The first one is to assign them to one of the 
institutes. Say, for example, something comes on strokes, 
depending on what it is going to be specifically, it may go 
here or it may go here.
    Now once the application is assigned, it does not come to 
my institute until it has been reviewed, evaluated 
independently of me and my staff. The people who are going to 
do that are people from the institutions which are in all of 
the States that you are from. So it is a independent peer-
review process.
    What they do, they give a numerical score, 100 being the 
best, 500 being the worst. By and large, these things are 
funded on the ranking of the score that you have received.
    Ms. Penn. Right. We have to follow the NIH guidelines for 
the intramural program. It has its own review committee made of 
external experts that come in three times a year to look at--
the same cycle as extramural--how 4 years of work has 
proceeded. It is at least as tough as any academic tenure 
committee or worse. So people really have to show that they 
have done good work, really good work, and that they are 
fulfilling the mission of the institute in what they are doing.
    Our intramural program has a set of units, laboratories, 
that have to do with clinical research and another set that 
have to do with basic laboratory research, all in related 
neuroscience aspects. The extramural is exactly as Dr. Lenfant 
described. We receive, following assignment by the Center for 
Scientific Review, grants that will then--we just know they are 
there, and we monitor until they have gone through the review 
process.
    Study sections are the review process. That is the other 
name for these committees. The set up and the organization of 
the Center for Scientific Review has recently been looked at by 
a star committee of outside experts, looking at whether they 
have the proper sets of science covered. I would say we all, 
all the institutes, through central NIH, contribute to the 
funding of the Center for Scientific Review. But it is critical 
that they are peers, but they are not in any way run by our 
institute or Dr. Lenfant's institute.
    Mr. Deal. Just a real quick question: During the course of 
the 4-year grant period--as I understand, it is usually 4-year 
grant periods--what oversight is exercised by the respective 
institutes over what is being done in these research projects? 
Could you give us some idea of what percentage may be renewed 
after an initial 4-year period?
    Mr. Bilirakis. Please try to answer the question, but try 
to be as brief as you can.
    Ms. Penn. Be as brief as possible.
    Mr. Bilirakis. I will tell you why. We are going to have 
another, I will call it, nonsense vote in a few minutes. So we 
are trying to get through----
    Ms. Penn. Quickly, both institutes have a group of 
extramural persons who are all scientists, or who are 
physicians themselves, who work on different parts of this 
program and are experts, pretty expert in the topics. They keep 
an eye on their particular set of people with grants. They are 
charged to review the progress every year to sort of let them 
know when they are not doing anything, and then to decide 
whether they possibly would get some kind of monetary 
supplement or not. But it is really monitored very carefully.
    Mr. Lenfant. Yes, I can only echo what has been said. There 
are some instances where actually your progress is not deemed 
to be satisfactory, and there are some consequences which may 
be from reducing the budget to in some instances, very rare, I 
have to say, but sometimes to basically closeing the grant.
    Ms. Penn. It varies, but it is somewhere between, I guess, 
25 and 40 percent are successful on the first pass on their 
renewal. This is, I think, your question.
    Mr. Deal. Thank you.
    Mr. Bilirakis. I thank the gentleman. Mr. Stupak?
    Mr. Stupak. Thank you, Mr. Chairman.
    In the NIH, you are required to transfer new biomedical 
technologies to the private sector for further research and 
commercialization and development. Also, Congress intends that 
NIH research will lead to new products such as diagnostics and 
vaccines.
    My question is, how far along do you go in developing new 
vaccines? Do you work with the pharmaceutical companies? Do you 
say, ``Look, we have an idea that this is where we think it 
should go. Here's the idea. Take it from here.''? How do you do 
it?
    Ms. Penn. The vaccine program, all of it pretty much, is 
under--except for very unusual new cases in Alzheimer's and 
potentially even in stroke--but most of the vaccines for 
infectious agents are in the hands of the National Institute 
for Allergy and Infectious Disease and the Vaccine Program, 
including the one for HIV/AIDS.
    When something is developed, we do talk to industry, or 
industry may be developing something also. There comes the NIH 
Office of Technology Transfer. I mean, we do monitor, they do 
monitor, they do come in and talk at various of our committees. 
They are always involved in all these groups that I talked 
about because they are very interested, obviously. I mean they 
are really interested in fixing diseases, too.
    Mr. Stupak. Okay, you say, well, here comes your Office of 
Technology, and you work with them. You transfer that to, let's 
say, the pharmaceutical company----
    Ms. Penn. Yes.
    Mr. Stupak. [continuing] to develop whatever the vaccine 
may be, correct?
    Ms. Penn. Yes. I mean, they are the group that handles 
things like intellectual property, things like, you know, we 
don't always take brand-new ideas because the brand-new ideas 
are really--our investigators have developed the brand-new 
ideas most of the time.
    Mr. Stupak. Okay.
    Ms. Penn. But it is a very active component of our 
intramural program, where our investigators really are us, and 
they are developing new technologies, diagnostics, and drugs.
    Mr. Stupak. Okay. Then in response to Representative 
Brown's question, you mentioned clinical trials. Are you 
involved in the clinical trials as these new vaccines are 
developed?
    Mr. Lenfant. I would assume that the answer to that is yes, 
but again----
    Mr. Stupak. Well, I don't want you to assume.
    Mr. Lenfant. Neither our institute nor Dr. Penn's institute 
are involved with vaccine and drug developments, especially 
antibiotics. So it is a bit difficult for us, at least for me, 
to tell you what another institute is doing.
    Mr. Stupak. Okay. If we assume for the sake of discussion 
here this morning that you do do the clinical trials, do you do 
post-marketing surveillance on drugs then after they have been 
on the market?
    Mr. Lenfant. I mean, if I understand the question, if 
clinical trials were said to assess the possibility of a new 
application of a medication of some sort, we work very closely 
with the FDA. Basically, any event, good or bad, that may occur 
during that process is reported to the FDA.
    Mr. Stupak. Sure. Well, we all are painfully aware up here 
that FDA doesn't do post-marketing surveillance on drugs, or 
very little, if any. So my question, I was wondering if you did 
then.
    Mr. Lenfant. Post-marketing?
    Mr. Stupak. Yes.
    Mr. Lenfant. No, not post-marketing. We don't have the 
authority to do it. We are not a regulatory agency.
    Mr. Stupak. Pardon?
    Mr. Lenfant. We are not a regulatory agency. We do not have 
the authority to do it.
    Mr. Stupak. Oh, I agree, but you are also a public health 
agency, as we established earlier. It would seem to me, if you 
helped to produce a vaccine or a drug an do clinical trials, 
you would really want to know what happens once it is out in 
the real world, and would probably do some post-marketing 
surveillance or work with the drug company, whoever it may be, 
who is making this vaccine, to make sure that the good 
intentions that you had in producing or going down this route 
is actually being fulfilled in the real world.
    Mr. Lenfant. Well, these events are not reported to us.
    Ms. Penn. Not in that way.
    Mr. Lenfant. If you ask about adverse events that are in 
the post-marketing phase, I think they probably appear in the 
newspaper long before they come to us actually. I am not being 
flippant about it.
    Mr. Stupak. I just think of the recent publicity around the 
drug Lariam which they indicated that NIH helped to develop. 
That is to fight malaria. There's been some side effects on it, 
mental health, especially some suicide. In the articles I read, 
NIH was more or less protecting the quality of this drug.
    But if you are telling me you don't do the post-marketing 
surveillance and it is not really your job, and it is not 
important, I certainly would think it would be important to all 
those people who may have been harmed by a drug. While it might 
help you out with malaria, but Lariam also has some side 
effects that, if the FDA isn't doing the post-marketing and you 
are not doing the post-marketing, who in the heck is?
    Ms. Penn. Now I would add that, of course, we work with our 
investigators in academic medical centers who are then using 
this drug. Not only will it come out in the newspapers, it will 
come out in medical journals. We certainly would hear.
    Now the question then is still, who's got the 
responsibility? We really don't. We don't like what is 
happening. Actually, our best drugs have a lot of side effects.
    Mr. Stupak. Yes, they have a lot of side effects, and they 
are out in the general public. They are not in the academic 
world or the research world anymore, and the people who are 
suffering are the people who don't have the scientific 
background, medical expertise, and we look to NIH when we 
double your budget and FDA to do it. If you don't do it and FDA 
doesn't do it, the pharmaceutical companies aren't doing it, 
who has to do it then?
    Ms. Penn. I hear you.
    Mr. Bilirakis. The gentleman's time has expired.
    Dr. Penn, following up, you say it is not part of your 
responsibility, but if you wanted to do it, could you do it? I 
realize you don't have the regulatory authority.
    Ms. Penn. Yes, we simply don't have the authority.
    Mr. Bilirakis. You don't have the authority to do it even 
if you wanted to?
    Ms. Penn. No. Our Program Director certainly and our staff, 
we are all aware when something that we have invested a lot of 
time and effort, and we think it is going to work, and then it 
goes out--this is true, for instance, of some anti-convulsants, 
anti-epilepsy agents, and we have a big program for that. If 
something goes out, the best possible studies we thought were 
done, and then a side effect occurs, then we would get the 
investigators to look at it again. Actually, we found out why 
Felbamate gave a hepatic effect, that kind of thing.
    Mr. Bilirakis. Dr. Norwood, to inquire.
    Mr. Norwood. Thank you very much, Mr. Chairman. I would 
like to state at the outset that probably NIH is one of our 
most important national resources, and I am happy to be part of 
the group that doubled your funding. I know ever since I have 
been here we have increased NIH funding almost every year, but 
that does, then, lead us into the realm of being responsible to 
some degree for that funding.
    I just want to follow up on some of the questions that have 
been asked. For example, Congressman Deal asked you the 
question of what percent of your research is done in-house, and 
I would like to know the answer to that.
    Mr. Lenfant. Well, Mr. Norwood, as I indicated, for the NIH 
as a whole, it is 11 percent of the budget, 11.
    Mr. Norwood. Percent of the budget goes for in-house 
research?
    Mr. Lenfant. Correct. Correct. And that varies from 
institute to institute.
    Mr. Norwood. But, in general, the average is 11 percent?
    Mr. Lenfant. Correct.
    Mr. Norwood. I understand. Would you describe for me, 
briefly, because I think I have lost my way, not you, what is 
the simple mission statement of NIH?
    Mr. Lenfant. It is to do research to improve the health of 
the American people.
    Mr. Norwood. I have always labored under the thought that a 
lot of your research was basic science.
    Mr. Lenfant. Well, the research process begins with basic 
science.
    Mr. Norwood. I understand.
    Mr. Lenfant. From basic science, it moves to applications, 
and applications means clinical research.
    Mr. Norwood. I know it does move to that, but does it move 
to that at NIH? Is that part of your mission----
    Mr. Lenfant. I would say yes. I can speak for our 
institute. We spend a lot of our budget to basically carry into 
clinical practice the basic research that is conducted either 
from us or from Dr. Penn, or from any of the institutes.
    The beauty and the problems with clinical research is that 
you address an idea, but you don't know for sure where it is 
going to take you. Our job, my job, is to identify things which 
appear to be promising and important and move them into the 
next step. The end of all these steps is basically the practice 
of medicine, what is going to happen between the physician and 
the patient.
    Mr. Norwood. So you do, then, a lot of clinical research--
--
    Mr. Lenfant. Oh, yes.
    Mr. Norwood. [continuing] as well as basic science?
    Mr. Lenfant. Yes.
    Mr. Norwood. Do you think Congress emphasizes enough the 
need to conduct basic scientific research? Do we imply that to 
your agency in different ways, how important we think basic 
science research is?
    Mr. Lenfant. Yes. Mr. Norwood, you know, I have been at the 
NIH for 32 years and the Director of this Institute for 20. I 
am not aware that the Congress ever said to us and to our 
institute, ``You shall do this much clinical research or this 
much basic research,'' or whatever. What the Congress has done, 
and I would say in its wisdom, is to say, ``You have to address 
them all.'' Sometimes it is going to be more of this; the next 
time it is going to be more of that.
    Mr. Norwood. Do you define yourself as a public health 
agency? Is that how you think of yourself?
    Mr. Lenfant. Absolutely. I can tell you that, I mean 
speaking for myself, I am entirely committed to the public 
health mission of our institute.
    Mr. Norwood. Let me just conclude with a last questioning 
sort of thought about it. Dr. Penn, you said that many, if not 
most, of our best drugs have side effects.
    Ms. Penn. Yes, sir.
    Mr. Norwood. Actually, most drugs have side effects, don't 
they?
    Ms. Penn. Yes, they do. Well, I'm thinking----
    Mr. Norwood. Isn't it also true that the side effects don't 
occur the same in each and every patient? Some may have them; 
some may not. Some may have drastic side effects; some may not.
    Ms. Penn. Yes. I am thinking of the big drugs. I am 
thinking of penicillin. I am thinking of aspirin and all of the 
ones that we do use and sometimes we absolutely have to use 
them, side effects or no side effects. But they do have side 
effects, and you are absolutely correct, it has to do with the 
genetic makeup of the individual to some degree. It has to do 
with how it is given in some cases. But everybody tries. What 
you need to do, again, is the doctor-patient communication, as 
soon as something happens. You hope it doesn't happen 
seriously.
    Mr. Norwood. Because anaphylactic shock may occur with 
penicillin, it is probably not a wide decision to conclude from 
that that we shouldn't use penicillin?
    Ms. Penn. Yes, sir, because some bacterial infections are 
best treated with penicillin. So you do your best.
    Mr. Norwood. And that is what I am trying to get you to say 
here.
    Ms. Penn. Yes.
    Mr. Norwood. I think we all do our best that we possibly 
can, and there are sometimes some very drastic outcomes, but 
these drugs are very important to many other people.
    Ms. Penn. But I would say that part of our mission, as Dr. 
Lenfant just said, is to say, okay, if we change one chemical 
part of penicillin, can we get a better penicillin?
    Mr. Bilirakis. The gentleman's time has expired. Ms. Capps, 
and then we are going to break right after her inquiry.
    Ms. Capps. Thank you, Mr. Chairman. Thank you for holding 
this very important hearing. I was frustrated by being detained 
on the floor and didn't get here for opening statements. I 
understand that you spoke to the importance of a particular 
bill that my colleague, Mr. Chip Pickering, and I are working 
on on Stroke Treatment and Ongoing Prevention Act of 2001. 
Thank you for your support of that legislation.
    I am sorry I didn't get your opening testimony either, but 
I am so appreciative that you are both here. I have three 
questions I am going to wrap into one. They have to do with the 
report that came recently from your group, Dr. Penn, I think. 
This is about stroke.
    You have written a report that will serve as a blueprint 
for a long-range strategic plan on stroke research. I want to 
hear from you about it. I know you have talked about it some, 
but I want to focus on the cost, the breakdown of cost, the 
aspects of it that will address women's health in perhaps a 
reinforced way, and also some concerns that I have that the 
doubling of the NIH budget has meant the Heart and Stroke 
Coalition is concerned in the House that the stroke and heart 
disease budget has not kept pace as it should have, and that 
there is a letter that I have signed, written and signed on 
with 80 of my colleagues, to see if we can fix this 
discrepancy.
    Ms. Penn. I said in my opening statement----
    Ms. Capps. Yes, I'm sure.
    Ms. Penn. [continuing] that we really do spend more on 
stroke than any of our other major disorders at NINDS. Part of 
what we did at the Stroke Progress Review Group meeting was to 
prioritize the kinds of science and medicine that they felt, as 
over 140 of our investigators thought should be done as we move 
ahead, as we implement this plan.
    Ms. Capps. I am actually not----
    Ms. Penn. I have a summary here for you, but I don't know 
if it is back there. You have the book.
    Ms. Capps. But I want to clarify that.
    Ms. Penn. Yes.
    Ms. Capps. I am not talking about within your agency. I am 
talking about within NIH as a whole.
    Ms. Penn. NIH. Well, we do work, as we said this morning 
with Dr. Lenfant, with all the other institutes----
    Ms. Capps. Yes.
    Ms. Penn. [continuing] at NIH that could impact on, not 
only figuring out what stroke really is, but treating it. I 
haven't even mentioned the hemorrhagic strokes this morning.
    Ms. Capps. Yes, exactly.
    Ms. Penn. So I believe that we have an excellent plan. We 
know roughly where we have to go, and we will fund it. We are 
very grateful for the help that we have gotten in funding it.
    Ms. Capps. So you see that the ability that you have to 
work among other institutes, that----
    Ms. Penn. We are definitely talking to other institutes, 
and we are talking, again, to the FDA. We can talk to them when 
the time comes about some of the things because we have to be 
concerned about safety. We talked, as we said before, to the 
Veterans' Administration about some of the things.
    Ms. Capps. Right.
    Ms. Penn. I mean, we do interact and we know the folks.
    Ms. Capps. Then your blueprint, we can expect that it 
really is going to----
    Ms. Penn. It will go forth. It won't all go forth at one 
time.
    Ms. Capps. Right.
    Ms. Penn. We would like to get grants in on some of the 
issues here, right, and set up new clinical trials, the whole 
thing.
    Ms. Capps. Then how much do you think it is going to cost 
and how much do we need to be prepared to address?
    Ms. Penn. We are going to do this in the context of the 
budgets that we think, and we think that----
    Ms. Capps. What would you like?
    Ms. Penn. I am not even going to try. Just to say, again, 
that stroke is extraordinarily important.
    Ms. Capps. Yes.
    Ms. Penn. We, of course, are hearing from a lot of other 
disorders because we have some really major and disabling 
disorders, but we will fund this.
    Ms. Capps. Thank you.
    Ms. Penn. You're welcome.
    Mr. Bilirakis. I thank the gentlelady.
    We are going to break to make this vote. I plan, Mr. Brown 
and I, I think, plan to get back immediately, and then we 
will--I am just not going to discharge the panel. I apologize 
again. You are going to have to just wait a few more minutes. 
Thank you.
    [Brief recess.]
    Mr. Bilirakis. I think Mr. Brown is on his way, is he not? 
There must be some Greek blood in Mr. Brown because he is 
always late.
    Until he comes and others, Mr. Pickering is here. I think I 
will yield to Mr. Pickering at this point.
    Mr. Pickering. Mr. Chairman, I want to thank you for 
holding this hearing today, and I want to thank you for calling 
attention to legislation that I have introduced with my 
colleague on the committee and from the other side of the 
aisle, Mrs. Capps, the Stop Stroke Act.
    I want to commend both Dr. Lenfant and Dr. Penn for their 
institutions and the great contributions that they are making 
and efforts across the board. I wanted to ask them for their 
comment on how important this legislation is, if they have had 
a chance to review the legislation, any recommendations they 
may make. What difference do you think this legislation may 
make and add to your ongoing efforts, as we try to take it one 
step up in trying to get the grants, the resources, and the 
information out to both our physician and medical community, 
but also to individuals who need to know what the signs are, 
how they can get treatment, what are the medical and 
technological and pharmaceutical breakthroughs that have really 
made a tremendous difference as we look at how to treat and 
effectively prevent strokes.
    With that, Dr. Lenfant.
    Mr. Lenfant. Mr. Pickering, with your permission, I would 
defer to Dr. Penn whose institute is overseeing the stroke 
program at NIH.
    Ms. Penn. We feel that we have made a start with this, and 
we have several campaigns that will fit right into the Stop 
Stroke Campaign, which are the public service announcements. We 
are trying to get a network out, so that in some communities we 
work through the churches, in some communities through 
professional societies.
    The Stroke Progress Review Group report will certainly 
address some of the issues that are in the Stop Stroke Act. It 
is extraordinarily important still to make sure that everybody 
knows the signs of stroke, and we finally think we have a 
therapy, which can intervene immediately. So people really have 
to get to the hospital.
    So we are foursquare behind this campaign. As I said, we 
have started, but this can only help. The bookmarks in the back 
are to address our populations in a bilingual way.
    So in terms of the idea, it is just terrific.
    Mr. Pickering. Let me follow up. What is your current 
spending at NIH as it relates to strokes? As you know, it is 
the third leading cause of death, approximately $50 billion in 
economic impact to our country on an annual basis. What I would 
like to know is, what is the research commitment that we are 
making as a country, given that tremendous loss of life or the 
quality of life of victims of stroke and to their families, 
their community, and to our economy because of strokes? What is 
our research commitment in NIH?
    Ms. Penn. NIH-wide, it is for fiscal year 2001, it is $239 
million. We, as an institute, we are spending more on stroke 
than any of our other disorders at the moment, and that is over 
$117 million.
    Everything, of course, is relative, but we are getting done 
what needs to be done. I just answered your colleague in terms 
of mobilizing the resources and using them to fulfill what is 
in the plan.
    Mr. Pickering. Again, just putting it in context, if it is 
the third leading cause of death, how would you say that $239 
million is in the priority at NIH, as we look at other diseases 
and research? Would you be able to put it into, what are the 
leading five areas of overall spending at NIH on research?
    Ms. Penn. On research, I would have to get you those 
answers for the written record.
    In terms of our own priorities, as I said, we are spending 
more on stroke. The rest of NIH has, obviously, institute-per-
institute--I mean, we've got cancer; we've got AIDS; we've got 
diabetes. So they have their own priorities. The diabetes 
impacts strokes. So there you go.
    Mr. Pickering. If you could, I would just like, as a 
policymaker, to understand where we are putting our resources 
and our priorities, and to put it in context to both the 
medical and the economic impact of our communities, of our 
families, of individuals, and to make sure that our priorities 
are right.
    Ms. Penn. Yes. Well, I think everybody, including all the 
people at NIH--it is just that you don't know exactly what is 
going to strike, but I did this on ``Stroke Sunday.'' I asked 
the audience, you know, ``How many of you have hypertension or 
high blood pressure?'' It was well over half the congregation. 
I mean, you could do that with stroke. Most of them had had 
relatives with stroke. That was the second question. So we all 
know the impact and we all know that it is a major, major 
problem.
    I think we are doing something for it, though, and I 
think----
    Mr. Pickering. I want to commend you and congratulate you 
for what you are doing. I would just like to know as to our 
research budget on AIDS, cancer, diabetes, hypertension, 
stroke, where we are in the allocations.
    Ms. Penn. Right. I can give you stroke, and I did, numbers, 
but I will have to get the relative amounts for you.
    Mr. Bilirakis. There will be a series of questions that 
will be presented to you after the hearing in writing which you 
would be asked to respond to in writing. So that certainly 
would be one, Chip.
    Mr. Lenfant. If I may add something, Mr. Pickering, our 
institute, the National Heart, Lung, and Blood Institute has 
spent approximately over $60 million in research on stroke, 
either basic but mostly clinical research. But, as you know, 
one of the main risk factors of stroke is elevated high blood 
pressure. Our institute supports $120 million in high blood 
pressure, and we have an extensive program of public and 
professional education and dissemination of what we know. It is 
called the National High Blood Pressure Education Program, 
which has been in existence for 30 years.
    I believe that we can fairly say that it has been a great 
contributor in the decreasing prevalence and, more importantly, 
in the control of high blood pressure. See, the issue is, 
again, to be sure that what we know is used, in this case that 
people who have high blood pressure are treated and controlled; 
that is, that we keep their blood pressure low.
    I think these programs do that, and I think that jointly 
with our colleagues we endeavor to do that with much increased 
intensity now, because we do realize the importance of that 
significant problem, especially in the Stroke Belt that was 
mentioned before you came this morning. But we are beginning to 
see some significant successes.
    Mr. Bilirakis. Thank you. Mr. Green, to inquire.
    Mr. Green. Thank you, Mr. Chairman. Again, I apologize to 
the panel for lots of us coming and going. We actually had 
votes scheduled, and some of us didn't know when we would vote 
on the House floor.
    I have one question of both witnesses. It is great 
following up my colleague from Mississippi. One of the most 
common concerns, I guess, with the NIH is that funding 
allocation is not proportionate with the burden of the certain 
diseases. For example, diabetes, which affects 17 million 
Americans and costs our Nation more than $100 billion in 
medical costs each year, actually receives only about $769 
million in research funding at the NIH. This amounts to about 
.7 of 1 percent of the cost of the treating the disease.
    The NIH is probably the only agency I know that Congress is 
reluctant to micromanage, simply because none of us have the 
expertise. But it seems to me the NIH would be emphasizing 
those diseases that have the largest burden both physically and 
economically on our society. If each of you could comment on 
that?
    Mr. Lenfant. Yes. First of all, I would like say, sir, that 
the diabetes research is not precisely in the purview of our 
two institutes. There is an institute where they do that.
    Having said that, it is a sad state of affairs that most 
diabetic patients suffer development of vascular disease which 
often leads to arteriosclerosis and then to death eventually, 
coronary heart disease and what have you.
    Our institute has a very significant program. In fact, 
yesterday I spent the day in New York discussing the 
development of a program which is precisely for the prevention 
of cardiovascular disease in diabetes.
    So if you take this commitment in addition to the basic 
research and the work that has been done by our colleague who 
is not here today whose responsibility is diabetes, we almost 
doubled, not quite, but we certainly had a very solid amount of 
money to the research, not only of the disease, but of the 
consequences of the disease.
    Mr. Green. Okay.
    Ms. Penn. Yes, sir, I was thinking somewhat of consequences 
of some of our disorders. You can talk about numbers. You can 
also talk about consequences because some of our disorders kill 
people sort of slowly. We have things like amyotrophic lateral 
sclerosis in our portfolio. We have the dystrophies where the 
kids are going to die by the age of 25. Now Parkinson's 
certainly and MS, and, therefore, what we look for are ways to 
make breakthroughs. We really want to go after the mechanisms 
of these, so we can figure out how to fix them.
    So sometimes small diseases can give you information on big 
diseases. We have a small disorder, very rare, genetic, which 
is probably giving us ideas on how to treat some major diseases 
that have to do with loss of balance.
    So it isn't just always dollars. It is how the whole 
package is coming together. If I really thought that, if I just 
added more and more dollars to some of these, something would 
happen tomorrow, I would probably do it. But I'm not always 
sure of that.
    We need to get the scientists involved. We need to train 
more to look at these disorders. We need to look at all of our 
disorders. We've got like 300 genetic diseases with some 5 or 
10, or 20 patients, but they are children. Again, these are 
very important also.
    So it is a balance. We have to balance the disorders in our 
mission, and we certainly consider burden. As I said, we all 
have--the more common the disorder, the more likely all of us 
will have somebody around who's got the disorder.
    Mr. Green. I guess I understand, hopefully, there is better 
correlation in NIH between your institutes than we have between 
our intelligence agencies.
    I hope that is the case. When we look at the loss, for 
example, in my home State of Texas with diabetes and juvenile 
diabetes--in fact, the House passed a resolution 2 days ago 
encouraging increased investment in diabetes research in 
juvenile diabetes.
    But I appreciate the correlation, and, hopefully, that will 
happen, because you are right, one success in one research area 
or one illness may also turn into something else.
    Ms. Penn. And I don't want to say that we aren't--you know, 
and Dr. Lenfant, too, we are a working--we have, again, a 
Memorandum of Understanding with the Juvenile Diabetes Research 
Foundation to deal with the complications in the nervous system 
for diabetes. So we do talk to people.
    Mr. Green. Thank you, Mr. Chairman.
    Mr. Bilirakis. Well, and just if I may, and Mr. Green has 
basically said it, really sometimes the toughest part of our 
job is having witnesses come here and pleading for additional 
funding for research for their particular diseases. Muhammad 
Ali has been here, just so many others.
    We try to stay on the path of not interfering with NIH 
because we are an ivory tower and don't really know, and we 
like to think that the funding is going where maybe they are 
closer to a particular cure and emphasize that sort of thing, 
but it is frustrating sometimes when we feel strongly, as Mr. 
Green does about diabetes, and don't see maybe a little more 
funding going that way.
    That is something that I don't want to take the time here 
now because I would really like to be able to excuse you in a 
few minutes, after Mr. Buyer has asked you questions, but that 
is something you might want to give a little thought to, giving 
us a little more rationale to sort of stay on the path that we 
are on, rather than to press NIH to allocate so much funding to 
a particular disease versus any others. You might want to think 
about doing that in writing to us.
    Mr. Buyer, to inquire.
    Mr. Buyer. Thank you.
    Dr. Lenfant, I am new to this Health Subcommittee. This is 
my first term. I am more challenged than I was before I walked 
into this hearing 2 hours ago. I am more challenged because 
maybe it is my logical reasoning; I can follow methodologies; I 
don't follow things very well if it is said, well, it is not 
always about dollars. I must interpret, then, that you are 
asking for great deference.
    I then hear your testimony in response to a very good 
question by Mr. Deal, and I know that you didn't mean to be 
flippant, but you said, well, we've spent a little more here 
and we've spent a little more there, and makes it sound like 
this or that, which then I must interpret that you like being 
capricious. It is a strong word, ``capricious.'' So if 
Congress--let me pause for a second. Mr. Pickering asked a very 
simple question: What are the five leading areas for which you 
do funding? And, Dr. Penn, you wouldn't even answer that. You 
said, ``Well, I want to answer that on the record, give you a 
written answer.'' Well, excuse me, I am almost challenged here 
at the moment.
    If this Congress is going to double the investments into 
NIH, please, we are not being intrusive into your territory. We 
would like you to be responsive because this Member here is 
challenged at the moment.
    So let me ask Dr. Lenfant, please respond to Mr. 
Pickering's question, what are the five leading areas in which 
you invest in your research? You ought to be able to do this 
off the top of your head. Thirty years, what are they?
    Mr. Lenfant. Well, I am going to let Mr.--would you allow 
me to----
    Mr. Buyer. I will allow you to say what are the five 
leading areas. What are they?
    Mr. Lenfant. Okay. In heart disease, congestive heart 
failure, coronary heart disease, and congenital heart disease. 
In----
    Mr. Buyer. Those are your five leading areas?
    Mr. Lenfant. That is three in our cardiovascular area. Our 
institute is the National Heart, Lung, Blood, blood resources, 
that is, blood safety, sleep, these are all areas, sir. So I 
could give you examples for each of these areas.
    Mr. Buyer. All right. No, that's fine.
    Now let me ask this question: I am trying to understand, 
quote, ``methodology.'' If you know this answer, what are the 
percentage of applications that receive funding compared to the 
total applications received?
    Mr. Lenfant. That's very easy, Mr. Buyer. In our institute 
during the last2 or 3 years, it has been between 30 and 33 
percent.
    Mr. Buyer. All right. Is it fair for us--I am looking at 
this chart by the American Stroke Association that uses some of 
your statistics here on funding. It shows the investments. It 
lists cancer, AIDS, heart disease, and stroke. I can understand 
why we doubled your budget, we've got this huge increase in 
cancer, but I don't understand this large increase in AIDS 
funding relative to a flat line in stroke and a minimal 
increase with heart disease. So could you explain it to me?
    Let me ask, before you jump to this, what can you tell me 
about the death rates of cancer versus AIDS versus heart 
disease and versus stroke? Is that a fair question for me to 
say?
    Mr. Lenfant. It is a fair question. I do not know, Mr. 
Buyer, whether you have in front of you a copy of my written 
statement. If you would turn on page 2----
    Mr. Buyer. No, I don't want to go to your written 
statement.
    Mr. Lenfant. Okay.
    Mr. Buyer. I want you to answer this question without----
    Mr. Lenfant. The answer is that half of the people who are 
here in this room will die from heart disease.
    Mr. Buyer. All right.
    Mr. Lenfant. That is a fact. That is a fact. Now why the 
budget is higher than for heart disease? If I was in a position 
to make that decision, I would probably take half of the budget 
of the institute, of the NIH, for heart programs, but I am not 
the one to make that decision. So I just cannot answer your 
question.
    Mr. Buyer. So must I interpret, then, by your answer that 
we have a disproportionate in funding for AIDS as compared to 
the illnesses out there?
    Mr. Lenfant. No, no. I would say that the----
    Mr. Buyer. Wait a second. You just said that if you were 
the decisionmaker, that you would prefer this investment to be 
in heart disease. If you think half of us in this room are 
going to die from heart disease and not from AIDS, why do we 
have the funding increases for that as compared to AIDS?
    Nothing on AIDS--it is just that there are four things on 
this chart.
    Mr. Lenfant. Well, if you look at cancer, that is a 
condition where you see a steady increase today of the death 
rate, whereas in cardiovascular disease you see a steady 
decline. I suppose that people who are making these decisions 
say, here we have an emergency, a situation which is becoming 
more serious year after year, and that probably influences the 
decision.
    With regard to AIDS, that is an infectious disease. An 
infectious disease always leads to a sense of emergency. I 
think that is why you see that situation. It may be that fewer 
people die from AIDS than from heart disease, but AIDS is a 
global emergency which can affect any one of us almost at any 
time.
    Mr. Buyer. Mr. Chairman, I am going to yield back to you, 
but I think Mr. Green asked a great question, along with Mr. 
Deal and Mr. Pickering. I think what we have here is sort of an 
invitation for greater scrutiny. I yield back.
    Mr. Bilirakis. All right, the gentleman's time has expired. 
Mr. Brown?
    Mr. Brown. I have two things. Thanks for the extra time.
    Mr. Bilirakis. I will ask consent for an extra 30 seconds.
    Mr. Brown. That is a unanimous consent request. I would 
like to submit questions. I specifically want to raise one, and 
we will submit it in writing and ask for your response in 
writing. But I just wanted to mention a brief moment the status 
of the graduate training and clinical investigation award, 
which has not yet been implemented and the eligibility 
requirements for the Clinical Research Loan Repayment Program. 
Researchers at Case Western University near my district near 
Cleveland and Ohio State tell me these criteria are effectively 
excluding many qualified students. I will submit that as a 
question for your response.
    I want to make one more point. This is not the time to 
debate this in length, but I had a physician that was in my 
office, who works in international health, the day before 
yesterday. He said unequivocally that the AIDS epidemic is the 
worst epidemic in human health for 600 years.
    For us to question funding because, for whatever political 
agenda people have, to question the funding when we are 
doubling the NIH budget, to question any commitment, any 
expenditure we have on AIDS, not that we shouldn't do 
oversight, and I wish this agency would take more leadership, 
as I mentioned two or three times earlier, on things like 
vaccines and antibiotics.
    But the politics of this issue disgusts me, frankly, the 
politics of the AIDS issue, when it is an epidemic, not nearly 
as big in this country, but the epidemic in Africa, the 
epidemic when AIDS and TB intersect in India, in China, in 
Russian prisons, in Estonian prisons, in Latvian prisons, and 
millions, 1100 people, as I said, a day in India die of TB. 
That number is going to skyrocket when AIDS hits India and 
China in the way that it almost inevitably will. We simply 
can't do enough.
    We spend less than .1 percent of our GDP on foreign aid and 
we should be ashamed of ourselves.
    Mr. Bilirakis. Well, you can see the reasons why we would 
rather not interfere in terms of the use of----
    Mr. Brown. Thank you, Mr. Chairman.
    Mr. Buyer. Mr. Chairman, I would like a chance to respond 
to that.
    Mr. Bilirakis. Well, this----
    Mr. Buyer. Mr. Chairman----
    Mr. Bilirakis. I don't think we ought to get into debate. 
This is a hearing.
    Mr. Buyer. No, he's--Mr. Chairman, he's pulling me into an 
area in which I was not going. For him to interpret my words as 
though he's personally disgusted, as though I was attacking 
AIDS insults me.
    I am referring to a chart right here, Mr. Brown. And Mr. 
Green asked some very good questions. Mr. Deal asked good 
questions. Mr. Pickering asked good questions. This is not a 
debate about AIDS. Diabetes was a very good thing to go over. I 
asked a very pertinent question here. So please don't pull me 
into your political disgust into some other form of agenda----
    Mr. Brown. My disgust----
    Mr. Buyer. [continuing] which I find personally insulting.
    Mr. Bilirakis. Without objection, the opening statements of 
all members of the subcommittee will be made a part of the 
record, and, as I have indicated earlier, there will be written 
questions submitted to you. We would appreciate a relatively 
prompt response to those.
    I thank you both very, very much. Again, I apologize for--I 
thank you for your patience, but that is the kind of a day we 
are going through here, is running back and forth. Thank you so 
very much, Doctors.
    Mr. Lenfant. Thank you, sir.
    Mr. Bilirakis. The second panel consists of Dr. Robert O. 
Bonow, President-Elect, American Heart Association, Goldberg 
Distinguished Professor of Medicine, and Chief, Division of 
Cardiology, Northwestern University, Feinberg School of 
Medicine; Mr. Eric Hargis, President and CEO of the Epilepsy 
Foundation; Dr. Edward Sanchez, Commissioner of Texas 
Department of Health, and Dr. Daniel Jones, whom Mr. Pickering 
would like to introduce.
    Would you like to do that at this point?
    Mr. Pickering. Yes, Mr. Chairman. Thank you for giving me a 
point of personal privilege and honor to introduce Dr. Daniel 
Jones from Jackson, Mississippi, who is the Vice Chancellor of 
the University of Mississippi Medical Center.
    But he is also a family friend. He had a private practice 
in my home town of Laurel, Mississippi, where he treated my 
family as well as me as I was growing up. Not only did he 
practice in a small town in Mississippi and contribute to the 
community, I grew up attending the same church as he, but he 
also went overseas to Korea as a medical missionary for 7 
years. For his humanitarian contributions and leadership and 
example, we are always very proud to point to his role in 
giving back not only to the community, but going overseas to 
help those in Korea.
    We also are very proud as he now is leading the effort in 
Mississippi at the University of Mississippi Medical Center in 
a number of different areas, especially in research and in 
hypertension and in stroke, as he has been published and is a 
leading figure and voice in that area, not only for our State, 
but around the country.
    So it is my great privilege today to introduce him, and I 
look forward to his testimony and the testimony of the rest of 
the panel.
    Mr. Bilirakis. Thank you. Thank you, Mr. Pickering.
    Mr. Green would like to add to my very brief introduction 
of Dr. Sanchez.
    Mr. Green. Thank you, Mr. Chairman. I would like to welcome 
Dr. Sanchez, our Texas Commissioner of Health. Having served 10 
years in Congress, I have worked with four different Texas 
Health Commissioners, and 20 years in the legislature before 
that, many dedicated people. Dr. Sanchez, he has been there 7 
months or a little more than 7 months, but he brings an 
aggressiveness and innovation, I think, to the department.
    I had a chance to meet him, and our staffs have worked 
together. So I am glad he is here today.
    Thank you, Mr. Chairman.
    Mr. Bilirakis. Thank you, Mr. Green.
    Gentlemen, your written statements are a part of the 
record. We will set the clock at 5 minutes. I would appreciate 
it if you would stick as close to it as you possibly can.
    Dr. Bonow, is that correct, sir?
    Dr. Bonow. Bonow.
    Mr. Bilirakis. Bonow. All right, Dr. Bonow, please proceed.

STATEMENTS OF ROBERT O. BONOW, PRESIDENT-ELECT, AMERICAN HEART 
 ASSOCIATION; ERIC R. HARGIS, PRESIDENT AND CEO, THE EPILEPSY 
FOUNDATION; EDUARDO J. SANCHEZ, COMMISSIONER, TEXAS DEPARTMENT 
   OF HEALTH; AND DANIEL JONES, VICE CHANCELLOR, UNIVERSITY 
           MEDICAL CENTER, UNIVERSITY OF MISSISSIPPI

    Mr. Bonow. Thank you, Mr. Chairman. Members of the 
committee, it is my great pleasure to participate in this 
discussion this morning. My name is Robert Bonow. I am 
Professor of Medicine and Chief of Cardiology at Northwestern 
University. I did serve 16 years as a commissioned officer in 
the National Heart, Lung, and Blood Institute at the NIH in 
Bethesda. As a volunteer member of the American Heart 
Association, I now serve as its President-Elect.
    I would like to first thank the committee for its 
leadership in including the Community Access to Emergency 
Defibrillation Act in the bioterrorism legislation. Your action 
will reduce cardiac arrest deaths by providing grants to 
purchase AEDs and to train first-responders in their use.
    Our association is devoted to fighting heart disease, 
stroke, and other cardiovascular diseases which are America's 
leading cause of death. Nearly 62 million Americans suffer from 
cardiovascular diseases, at an estimated cost this year of $330 
billion in medical expenses and lost productivity, more costly 
than any other diseases.
    Since Dr. Jones will focus on stroke in his testimony, I 
will address heart disease. Heart disease remains the number 1 
killer of Americans and is the leading cause of premature, 
permanent disability among American workers. Our association 
works to increase the number of Americans who receive 
immediate, high-quality care for sudden cardiac arrest, heart 
attack, and stroke by raising awareness of their warning signs 
and risk factors, and the need to seek immediate medical help. 
These efforts touch Americans throughout the country.
    We are particularly concerned about the elderly who suffer 
disproportionately from these diseases, yet benefit from 
preventative strategies. So we are leading the charge to add 
preventative cholesterol screening to Medicare benefits, which 
currently are not covered.
    Our association also invests in medical research. We are 
unique in that our local and national research programs for 
investigator-initiated research are supported wholly by 
publicly donated money, and our programs emphasize the support 
of investigators in the early stages of their careers, as they 
strive to become successful and become competitive for NIH 
grants.
    We do not accept Federal funds, but we do enjoy a 
productive relationship with the government in advancing our 
mission. For example, as mentioned by both Drs. Penn and 
Lenfant, our association has signed a Memorandum of 
Understanding with the Centers for Medicare and Medicaid 
Services, NHLBI, NINDS, and the Centers for Disease Control and 
Prevention. Through this partnership with the Department of 
Health and Human Services, we strengthen and enhance the 
information and services provided to the public to reduce the 
impact of heart disease and stroke.
    Also, we work with the NIH to coordinate and enhance vital 
research activities by participating mutually in each other's 
conferences, research committees, and advisory councils. For 
example, I now serve on the NHLBI's Board of Extramural 
Advisors, and our chairman of the board serves on the NIH 
Directors' Council of Public Representatives.
    Our association, including our extensive grassroots network 
and affiliates, actively advocates for the completion of the 5-
year, bipartisan congressional initiative to double the NIH 
budget. We applaud this committee's visionary leadership in 
this historic effort, and we urge you to complete this 
initiative in fiscal year 2003. Your action will benefit the 
health of all Americans for decades to come.
    Thanks to your investment in NIH, exciting medical advances 
have benefited countless Americans suffering from heart disease 
and those at risk. Major advances include: cutting-edge, life-
extending drugs that help prevent heart disease, including 
drugs to control blood pressure and cholesterol. Your 
investment has also produced revolutionary diagnostic tools, 
including exciting new imaging technologies to diagnose heart 
disease in its early and advanced stages, and simple blood 
tests that can rapidly diagnose even the smallest heart attack.
    Your investment in NIH has resulted in major changes in the 
heart patient care. Revolutionary clot-buster drugs can reduce 
disability from heart attack by dissolving the blood clots that 
cause the attack. Small, wire-mesh stents, now used in nearly 
80 percent of the 1 million angioplasty procedures performed 
each year to widen narrow arteries of the heart, greatly 
increase the success rate of these procedures.
    Other breakthrough technologies include pacemakers, 
implantable cardiac defibrillators, AEDs, and minimally 
invasive surgical techniques. Advances have clearly been made 
in the control and treatment of heart disease and its risk 
factors.
    However, as has already been mentioned, heart disease 
remains America's number 1 killer, and there still is no cure. 
An American dies from cardiovascular disease every 33 seconds. 
Much more needs to be done to address these challenges and 
their opportunities.
    Now is the time to capitalize on our potential to 
understand the fundamental causes of heart disease and to 
develop exciting new treatments. For instance, NHLBI research 
has shown the strongest evidence yet that human heart muscle 
cells may regenerate after a heart attack. This finding opens 
entirely new avenues in future investigation and clinical 
trials for treatment of failing hearts weakened by heart 
attacks.
    Also, implantable left ventricular assist devices and even 
artificial hearts show promise as replacement therapy for end-
stage heart failure. Promising breakthroughs for this and other 
heart conditions are on the horizon with the potential to 
improve the quality of life for all Americans and to reduce 
health care costs.
    Unfortunately, the NIH budget for heart disease and stroke 
has not kept pace with the doubling initiative, and NIH heart 
and stroke research remains disproportionately underfunded 
compared to the burden and to the many promising scientific 
opportunities. I will take some risk here by referring you 
again to the chart on page 5 of the brochure attached to my 
testimony. The point of my testimony is not whether or not this 
is disproportionate funding, but only as we double the NIH 
budget, we are currently not on track to double the amount of 
funding for heart-related research. The point is we would like 
the NHLBI to also share in the doubling effort for this very 
important disease.
    Importantly, these opportunities include research into 
improved health care delivery systems, not just basic science 
or clinical trials, but developing research to deliver the 
health care, to allow all Americans access to our current and 
future research advances. We urge Congress in the last year of 
this effort to provide funds necessary to ensure the NIH budget 
for heart disease and stroke also doubles over this 5-year 
period.
    Thank you very much for the opportunity to be with you 
today.
    [The prepared statement of Robert O. Bonow follows:]
Prepared Statement of Robert O. Bonow, President-Elect, American Heart 
                              Association
    Good morning, I am Robert Bonow, Goldberg Distinguished Professor 
of Medicine and Chief of the Division of Cardiology at Northwestern 
University Feinberg School of Medicine. Before joining Northwestern, I 
served 16 years as a commissioned officer in the U.S. Public Health 
Service at the National Institutes of Health's National Heart, Lung, 
and Blood Institute as Chief of the Nuclear Cardiology Section and 
Deputy Chief of the Cardiology Branch. I currently serve on the NHLBI's 
Board of Extramural Advisors. As a volunteer, I am President-Elect of 
the national American Heart Association and President of its Chicago 
Metro Board. The Association is the largest voluntary health 
organization fighting heart disease, stroke and other cardiovascular 
diseases.
    Before I begin my discussion on NIH, I would like to thank the 
Committee, on behalf of the American Heart Association, for its 
leadership in including the Community Access to Emergency 
Defibrillation Act in the bioterrorism legislation awaiting the 
President's signature. Your action will help reduce deaths from cardiac 
arrest by providing grants for the purchase and placement of AEDs where 
cardiac arrests are likely to occur and train first responders in their 
use.
     american heart association: fighting heart disease and stroke
    The Association, with 22 million volunteers and supporters, is 
devoted to reducing disability and death from heart disease, stroke and 
other cardiovascular diseases, which kill nearly 960,000 Americans each 
year. Cardiovascular diseases account for more than 40 percent of all 
American deaths. Nearly 62 million Americans suffer from cardiovascular 
diseases, many of whom are permanently disabled. Cardiovascular 
diseases cost Americans more than any other disease--an estimated $330 
billion in medical expenses and lost productivity this year.
    Since Dr. Jones will focus on stroke in his testimony, I will 
address heart disease--still the No. 1 killer of Americans across 
racial and ethnic groups, killing more than 725,000 people of all ages 
each year. Nearly 23 million Americans live with the often disabling 
effects of heart disease. Heart disease is the leading cause of 
premature, permanent disability among American workers, accounting for 
nearly 20 percent of Social Security disability payments.
    Our Association works to increase the number of Americans who 
receive immediate, high-quality care for sudden cardiac arrest, heart 
attack and stroke by raising awareness of the warning signs, risk 
factors and the need to seek immediate medical attention. Our awareness 
and educational efforts touch Americans in every area of their lives--
at home, work, school, church and in the hospital. For example, our 
national, community-based initiative, Operation Heartbeat, seeks to 
improve the sudden cardiac arrest survival rate. Search Your Heart is a 
faith-based program, involving approximately 3,000 places of worship 
and community-based organizations. It is a prevention program that 
teaches at-risk Hispanics, African-Americans, and Asians to recognize 
and control heart disease and stroke risk factors, such as high blood 
pressure, high cholesterol, obesity and diabetes. Another example is 
Get With The Guidelines, an acute-care, hospital-based program that 
helps manage risk factors in heart disease patients. It strives for 
long-term behavioral change to help prevent subsequent heart attacks.
    Also, we invest in medical research. In fiscal year 2000-2001, we 
expended nearly $135 million on research to increase knowledge of heart 
disease and stroke. Even this amount places us a distant second to the 
National Institutes of Health in the amount of research funding in 
these critical areas. However, we are unique in our research by 
providing both local and national resources for investigator-initiated 
research projects wholly supported by publicly donated money. In 
addition to sponsoring the highest meritorious research, we place 
emphasis on supporting beginning investigators as they progress to 
become competitive for national funding sources, such as the NIH.
                   partnering to advance our mission
    Our Association cannot accomplish our life-saving mission alone, so 
we join forces with the federal government. We do not accept federal 
funds, but enjoy a productive relationship with the government in 
advancing the battle against heart disease and stroke. For example, in 
February 2001, the Association and four federal health agencies signed 
a Memorandum of Understanding. An important example in public and 
private sector cooperation, this agreement creates a working 
partnership with the Department of Health and Human Services, including 
the Centers for Medicare and Medicaid Services, a Centers for Disease 
Control and Prevention component and two NIH institutes--the National 
Heart, Lung, and Blood Institute and the National Institute of 
Neurological Disorders and Stroke. Through this partnership of shared 
community-based education, health promotion programs, public awareness 
campaigns, media information and data collection, we strengthen and 
enhance the information and services provided to the public to 
significantly reduce the exorbitant impact of heart disease, stroke and 
other cardiovascular diseases on our nation.
    Also, we work with the NIH to coordinate and enhance vital research 
activities by participating in each other's national conferences, 
research committees and advisory councils. For example, I serve on the 
NHLBI's Board of Extramural Advisors and other Association volunteers 
have participated on the National Heart, Lung, Blood Advisory Council. 
Our Chairman of the Board serves on the NIH Director's Council of 
Public Representatives.
    NIH-supported research plays an essential role in advancing the 
fight against heart disease. So, our Association, including our 
extensive grassroots network, our affiliates nationwide and more than 
32,000 scientific council members, actively advocates for the 
completion of the five-year bipartisan congressional initiative to 
double the NIH budget. We laud this Committee's visionary leadership in 
this historic effort and urge you to complete this initiative by FY 
2003. Your action will benefit the health of all Americans for decades 
to come.
                    heart disease research advances
    Thanks to your investment in NIH, exciting medical advances benefit 
Americans suffering from heart disease and those at risk. Several major 
advances follow. Cutting-edge, life-extending drugs help prevent and 
treat heart disease, including drugs to control blood pressure and 
cholesterol. Now, a simple blood test can diagnose even the smallest 
heart attack within six hours of symptoms. When prevention fails, 
revolutionary ``clotbuster'' drugs, such as tPA, can reduce disability 
from heart attack by dissolving blood clots causing the attack.
    Your investment in NIH has resulted in major changes in heart 
patient care. For instance, stents--wire mesh tubes used to prop open 
an artery--are now used in nearly 80 percent of the more than 1 million 
angioplasty procedures performed each year to widen narrowed arteries 
to the heart. The use of stents as part of the angioplasty procedure 
significantly reduces the incidence of artery renarrowing within six 
months.
    Also, your investment in NIH has revolutionized imaging technology 
to diagnose heart disease and surgical techniques to treat heart 
disease. You probably know someone who has benefited from the research-
breakthrough of heart bypass surgery--355,000 Americans under-went this 
procedure in 1999. Patients who experience conventional bypass surgery 
to improve blood flow to the heart require several weeks to recover, 
but those who experience the new ``minimally invasive heart bypass 
surgery'' need a much shorter recovery period. Other amazing 
technologies include pacemakers, implantable cardiac defibrillators, 
and automatic external defibrillators.
    During our Association's recent lobby day, I teamed up with 
numerous heart disease and stroke survivors who traveled to Washington, 
D.C. asking you to make America's No. 1 killer, your No. 1 health 
priority. Many of them are alive today due to your investment in the 
NIH. They are living with stents, pacemakers, implantable cardiac 
defibrillators, or heart transplants or have benefited from other 
state-of-the-art procedures.
              heart disease: still america's no. 1 killer
    Thanks to research made possible by your Committee, great strides 
have been made in the control of heart disease risk factors and in the 
treatment of heart disease. However, heart disease remains America's 
No. 1 killer and there is still no cure for this devastating disease. 
Much more needs to be done to address the mounting challenges and 
numerous unanswered questions about heart disease.
    Now is the time to capitalize on a century of progress in 
understanding the causes of heart disease and in developing new 
treatments. According to a national expert panel supported by Congress, 
America's progress in reducing the death rate from cardiovascular 
diseases has slowed, suggesting the need for new strategies against 
these killers. Also, the panel reported striking differences in 
cardiovascular disease death rates by race/ethnicity, socio-economic 
status and geography.
                  heart disease research opportunities
    Promising, cost-effective breakthroughs are on the horizon, with 
the potential to reduce health care costs and to improve the quality of 
life for all Americans, including the 1.1 million who will suffer a 
heart attack this year and the nearly 5 million who live with the 
effects of heart failure. For instance, NHLBI-supported research has 
shown the strongest evidence to date that human heart muscle cells may 
regenerate after a heart attack, challenging previous beliefs that 
heart muscle damage from a heart attack remains permanent. Implantable 
left ventricular assist devices show promise as replacement therapy for 
end-state heart failure. This year, several patients received the first 
completely implantable artificial heart. Imagine what could be 
accomplished with more resources.
    Unfortunately, despite the tremendous advances and burgeoning 
opportunities, the NIH budget for heart disease has not kept pace with 
the doubling initiative. Heart research receives 8 percent of the NIH 
budget. We urge Congress in the last year of this historic effort to 
provide funds necessary to ensure that the NIH budget for heart disease 
also doubles over the five-year period. NIH heart research remains 
disproportionately underfunded compared to the enormous burden heart 
disease places on our nation and the abundant promising scientific 
opportunities that could advance the fight against heart disease.
                        preventing heart disease
    Research findings must be translated into effective prevention 
programs. More resources should be made available to bring research 
advances to Americans. We support the conviction of Dr. Elias Zerhouni, 
Director of the National Institutes of Health, who noted during his 
United States Senate confirmation hearing on April 20, 2002 that ``we 
still have to make discoveries to perhaps facilitate the way we deliver 
healthcare.'' For example, two separate independent panels, one 
convened by NHLBI, agreed that it is never too late to substantially 
lower heart attack risk by aggressively reducing cholesterol levels. 
So, we lead the charge to add preventive cholesterol screening to 
Medicare benefits. Now, Medicare covers cholesterol screenings only if 
beneficiaries already suffer from diseases associated with elevated 
cholesterol, such as heart disease, but does not cover screening of 
apparently healthy individuals to prevent heart disease.
    Thank you for this opportunity to speak with you today. I will be 
happy to answer questions.

    Mr. Bilirakis. Thank you so much, sir.
    Mr. Hargis?

                   STATEMENT OF ERIC R. HARGIS

    Mr. Hargis. Thank you, Mr. Chairman and distinguished 
members of the subcommittee, for the opportunity to be here 
this morning, or actually this afternoon, and talk about NIH 
research and the efforts to prevent and cure disease. My name 
is Eric Hargis. I am the President and Chief Executive Officer 
of the Epilepsy Foundation. This is a topic of critical 
importance to the 2.3 million Americans who live with epilepsy.
    Advances in medical treatment have enabled many people to 
live normal lives, free of seizures, and to achieve personal 
and professional success. These advances would not be possible 
without an aggressive public and private research effort. Yet, 
one in four of all newly diagnosed people will have persistent 
seizures despite treatment. More than 1 million Americans 
currently live with uncontrolled epilepsy.
    The Epilepsy Foundation is an aggressive advocate for the 
doubling of the NIH budget, for an expanded epilepsy public 
health program, and for quality and affordable health care for 
all Americans. Families are desperate for a cure for epilepsy 
and hopeful that in the short term research will provide new 
and more effective treatments.
    While much about the research program at NINDS could be 
praised, I want to single out a very successful joint effort of 
the NINDS and the epilepsy community. Our work together on the 
Curing Epilepsy Focus on the Future Conference illustrates 
several important principles that we believe have widespread 
application.
    First is the value of having the institutes collaborating 
with professional organizations, lay organizations, 
individuals, and family members affected by the condition.
    Second, the importance of Federal interagency coordination 
and cooperation.
    And, third, the value of creating measures to hold the 
various agencies accountable for their activities and their 
progress.
    This conference drew together experts from all parts of the 
scientific world, representatives of different agencies within 
NIH, and people with epilepsy to review the status of our 
current understanding of the epilepsies and to develop a 
framework for future directions in research. As a result of 
that conference, the research community has reached a dramatic 
turning point, and we are now able to talk about a potential 
cure for epilepsy.
    The next step was the community's creation, under the 
auspices of NINDS, of the Scientific Benchmarks Implementation 
Plan for the next 5 years. This will guide research activities 
and should serve as the springboard for the development of 
measurable goals both for the agency itself and for other 
affected agencies within the NIH. However, a shortcoming of 
these benchmarks was the failure to do funding projections for 
what would be needed to accomplish this research.
    One of the important aspects of the Conference on the Cure 
was NINDS's concerted effort to bring together multiple 
agencies within NIH to participate. NIH should ideally develop 
an interagency coordinating body to address epilepsy and its 
impact on all aspects of life. While we know that NINDS staff 
members do work with members of other agencies within NIH, this 
is on an informal basis, and this activity should become more 
formalized and systemized among the agencies.
    It is also important that the Federal Government be able to 
account for how it has spent the large increases in funding for 
research that has occurred over the last 5 years. While NINDS 
has been helpful in answering our questions on epilepsy 
funding, we do not know how much the NIH has spent overall on 
epilepsy research. Apparently, there are no cross-agency 
accounting systems for making that determination.
    Within NINDs, further detail and information describing and 
explaining the research that is being funded is needed to 
better educate the public about the work of NIH. This morning 
when we visited the NIH website to gather updated information 
about the level of funding, we found that, unfortunately, while 
there were 60 conditions listed with dollar amounts, epilepsy 
was not included.
    NINDS should also be recognized for another innovative 
program that fosters partnership between the public and the 
private sector. The Anti-Convulsant Screening Program was begun 
in recognition that private industry would not pursue research 
for products that addressed the needs of a limited number of 
people. This Federal program has now had a successful history 
of identifying promising compounds that develop into 
medications for seizures. In fact, most of the medications that 
have been introduced in the last decade have been developed as 
a result of this research.
    Thus, NINDS must overall be congratulated for its work and 
achievements in understanding of the brain and its support of 
research toward an ultimate cure. At the same time, NINDS needs 
to better address the continuing medical treatment issues of 
this population whose needs will not go away while cures are 
being sought.
    NINDS very recently funded a multi-center clinical trial on 
early surgical interventions for temporal lobe epilepsy. We 
urge NINDS to do more of these types of studies, including 
research on bioequivalence and bioavailability among various 
anti-epileptic drugs, or doing research to compare the outcomes 
of the various new treatments to help define best practices.
    These are but a few examples of clinical research projects 
that NIH could and should fund to a far greater extent than it 
does today. Again, our recent work with NINDS is really a model 
for bringing stakeholders together to address the future of 
research. That is a practice that must continue to ensure a 
close relationship between the needs of American citizens and 
the work of Federal agencies.
    We congratulate NINDS for the progress they have made and 
look forward to being a key partner in the efforts to prevent 
and cure epilepsy. Again, thank you for the opportunity to 
testify.
    [The prepared statement of Eric R. Hargis follows:]
   Prepared Statement of Eric R. Hargis, President & Chief Executive 
                      Officer, Epilepsy Foundation
    Good Morning. Thank you Mr. Chairman and Distinguished Members of 
the Subcommittee for the opportunity to be here this morning to talk 
about research at the National Institutes of Health and the efforts to 
prevent and cure disease. I am Eric Hargis, President and Chief 
Executive Officer of the Epilepsy Foundation. This is a topic of 
critical importance to the 2.3 million Americans who live with 
epilepsy.
    The Epilepsy Foundation is the national organization, formed in 
1968, that works for children and adults affected by seizures through 
research, education, advocacy and service. Approximately 181,000 new 
cases of seizures and epilepsy occur each year; 10% of the American 
population will experience a seizure in their lifetimes; 3% will 
develop epilepsy by age 75.
    Advances in medical treatment have enabled many people to live 
normal lives free of seizures and to achieve personal and professional 
success. These advances would not be possible without an aggressive 
public and private research effort. Yet one in four of all newly 
diagnosed people will have persistent seizures despite treatment and 
more than one million Americans currently live with uncontrolled 
epilepsy. For them, epilepsy remains a formidable barrier to normal 
life, affecting educational attainment, employment and personal 
fulfillment. People with epilepsy are at risk of brain damage and 
increased mortality when seizures resist control. Despite a decade of 
economic boom and record employment, 25% of people with epilepsy are 
unemployed, the majority a result of their epilepsy. Stigma remains a 
fact of life for many with epilepsy fueling discrimination and 
isolating them from the mainstream of American life. Epilepsy can 
strike at any age but tends to impact the very young and old, often the 
most vulnerable segments of our population. Epilepsy can produce 
developmental delays and brain damage in children that can lead to a 
lifetime of dependence on others and continually accruing costs to the 
health care system and society at large.
    The Epilepsy Foundation is an aggressive advocate for the doubling 
of the NIH budget, for an expanded epilepsy public health program, and 
for quality and affordable health care for all Americans. Spending time 
with families who live with frequent and persistent seizures 
underscores the need to address each of these areas. Families are 
desperate for a cure for epilepsy and hopeful that in the short-term, 
research will provide new and more effective treatments.
    While much about the research program at NINDS could be praised, I 
want to single out a very successful joint effort of NINDS and the 
epilepsy community. Our work together on the 2000 Curing Epilepsy: 
Focus on the Future conference illustrates several important principles 
that have widespread applications:

 The value of working with people with epilepsy, their family 
        members and the organizations that represent their interests
 The importance of federal interagency coordination and 
        cooperation
 The value of creating measures to hold the various agencies 
        accountable for their activities and progress.
    This conference drew together experts from the all parts of the 
scientific world, behavioral experts, representatives of different 
federal agencies within NIH, and people with epilepsy and family 
members to review the status of current understanding of the epilepsies 
and to develop a framework for future directions in research.
    As a result of that conference we are now able to talk about a 
potential cure for epilepsy--a turning point in the general approach 
that had been taken to epilepsy since the first medications were 
introduced sixty years ago. The conference identified important areas 
where continuing research may indeed lead to a cure in the course of 
the next twenty years. These include advances in genetics, 
understanding the plasticity of the brain and epileptogenesis, insights 
from new imaging and electrophysiology, the potential for cell therapy, 
and surgical and other mechanical interventions in the brain.
    After that conference, the NINDS again pulled together all 
stakeholders and created an epilepsy research benchmarks implementation 
plan for the next five years. This document will be used to guide the 
NINDS' activities, and should serve as the springboard for the 
development of measurable goals, both for the agency itself and for 
other affected agencies within the NIH. It is also important that 
progress towards these goals be assessed periodically, but certainly at 
least every five years, through future reviews with the participation 
of expert community, government and consumers. One shortcoming was the 
failure to do funding projections for what would be needed to 
accomplish the research.
    One of the important aspects of the Conference on the Cure was 
NINDS' concerted effort to bring together multiple agencies within NIH 
to participate. This is an effort that needs to continue in future work 
of the NINDS and NIH. Coordination of activities among the various 
agencies must be fostered and institutionalized so that each hand knows 
what the other is doing and a systematic cross agency approach to 
epilepsy research can be implemented. NIH should ideally develop an 
interagency coordinating body to address conditions like epilepsy and 
the many neurological and other diseases that impact one in all aspects 
of life, so as to prevent needless overlap, inconsistency, and promote 
optimal research among the agencies. While we know that NINDS staff 
members do work with members of other agencies within NIH, this is on 
an informal basis, and this activity should be more formalized and 
systemized among the agencies.
    It is also important that the federal government be able to account 
for how it has spent the large increases in funding for research that 
have occurred over the last five years. While NINDS has been helpful in 
answering our questions on epilepsy funding, we do not know how much 
NIH has spent overall through all the agencies on epilepsy research, 
because apparently there are no cross agency accounting systems within 
NIH for making that determination. Within NINDS, further detail and 
information describing and explaining the research that is being funded 
would be helpful. The Epilepsy Foundation is a logical partner that can 
assist NINDS in getting the word out about their good work, but we need 
more information from the agency.
    NINDS should also be recognized for another innovative program that 
fosters partnerships between the public and private sector. Over the 
last twenty years, NINDS has made considerable progress in the 
identification of compounds that affect seizure generation by 
interfering with various channels and receptors within the brain. This 
has occurred through the NINDS Anti-Epileptic Drug Development program, 
a program begun in response to federal recognition that the private 
marketplace would not invest in research for products which affect 
limited numbers of people. The Anti-Epileptic Drug Development program 
has led to the introduction of multiple new drugs for the treatment of 
epilepsy and has made a real difference in the lives of people with 
epilepsy by adding more treatment options.
    The program, now called the Anticonvulsant Screening Program, has 
identified many compounds that should continue to be explored for their 
potential to treat seizures and other conditions, and we urge Congress 
to continue its support for this activity. An interesting and difficult 
side issue is whether the private marketplace will be willing and 
equipped to explore these risky new potential treatments. Congress 
should consider a broader public role in the exploration and 
development of new treatment, particularly for conditions that affect 
only a limited segment of the public. That is a discussion for another 
time, but we raise it here because the federal government is sitting on 
a potential goldmine of treatment options for a variety of conditions 
that should not be lost.
    Thus NINDS must overall be congratulated for its work in 
understanding of the brain and its support of research towards a long 
term cure. At the same time, NINDS needs to address the continuing 
medical treatment issues of this population, whose needs will not go 
away while cures are sought. For example, NINDS recently funded a 
multi-center clinical trial on early surgical intervention for temporal 
lobe epilepsy. We urge NINDS to do more of these types of studies. 
Other types of outcomes research are also needed, such as a study of 
the efficacy of the various treatment modalities, including comparisons 
among them in terms of best outcomes; studies on bioequivalence and 
bioavailability issues among various products which are based on the 
same chemical compounds but which clinicians and patients will swear 
are different in their effectiveness in treating seizures. These are 
but a few examples of clinical research projects that NIH could and 
should fund. The private sector is generally not capable of funding 
this type of research, and in our view NIH could do much more to 
sponsor research that informs best medical practice.
    I would like to conclude this morning by again stating how 
critically important it is for the general public to understand what 
NINDS is doing. Too often, the work of the NIH is a mystery to the 
public. NIH and its agencies could do a better job of presenting itself 
simply and clearly to the public, and of reaching out to stakeholders 
for input and guidance on its activities. As discussed earlier our 
recent work with NINDS is really a model for bring stakeholders 
together to address the future of research. That is a practice that 
must continue to ensure a close relationship between the needs of 
American citizens, and the work of the federal agencies. We 
congratulate NINDS for the progress they have made and look forward to 
being a key partner in the efforts to prevent and cure epilepsy.
    Again, thank you for the opportunity to testify this morning, I am 
happy to answer any questions.

    Mr. Bilirakis. Thank you very much, Mr. Hargis.
    Dr. Sanchez, please proceed.

                 STATEMENT OF EDUARDO J. SANCHEZ

    Mr. Sanchez. Good afternoon, Mr. Chairman and members of 
the Subcommittee on Health. It is an honor to appear before you 
today to testify about the Texas Department of Health's efforts 
to disseminate public health interventions that reduce the 
health effects and related costs of cardiovascular disease, the 
leading cause of death in our country.
    My name is Eduardo Sanchez. I am the Texas Commissioner of 
Health with the Texas Department of Health.
    I want to thank you, Mr. Chairman and members of this 
subcommittee, for your support to help improve our Nation's 
health and for holding this hearing. As Texas State Health 
Officer and family physician experienced in community-based 
care, I know that are many who have yet to benefit from 
available research on effective prevention of cardiovascular 
diseases and other chronic illnesses. Getting research and 
proven public health interventions off the shelf and into 
communities is vital for all Americans.
    First, I want to explain why cardiovascular disease 
prevention is one of the Texas Department of Health's top 
priorities. Death, long-term illness, disability, 
hospitalization, and rising costs related to treatment and lost 
productivity are quite literally breaking the heart and 
breaking the bank of our State and of this country.
    Heart disease and stroke kill nearly 1 million men and 
women each year in the United States. This number represents 
more than 40 percent of all annual deaths. Sixty-one million 
Americans, almost 25 percent of our population, are living with 
some form of cardiovascular disease. Almost 6 million 
hospitalizations each year are due to cardiovascular disease. 
As you heard, the estimated annual cost of cardiovascular 
disease is $330 billion.
    The good news is that we know how to reduce this burden, 
but we must put this knowledge into action. I want to share 
with you one example of translating research into positive 
public health outcomes.
    We know the primary risk factors for cardiovascular disease 
are tobacco use, poor nutrition and obesity, and physical 
inactivity. These risk factors begin to have negative effects 
at a young age. Texas is 1 of 4 States that participated in a 
National Heart, Lung, and Blood Institute trial specifically 
designed to reduce these risk factors.
    The Child and Adolescent Trial for Cardiovascular Health, 
or ``CATCH,'' is the largest school-based health promotion 
study ever conducted in the United States that has 
scientifically documented positive changes in children's 
physical activity and dietary habits. Now it is a research-
based model, and TDH is providing curricula and training for 
teachers, cafeteria workers, and other school personnel.
    Our program evaluation shows that behavioral changes have 
continued 3, 5, and 7 years after the first Texas schools 
implemented the CATCH program. Former CATCH students have 
continued to eat diets low in fat and to exercise vigorously. 
CATCH-trained schools are still serving healthier meals than 
those not in the program.
    CATCH is a demonstrated success, and we have renamed the 
program a Coordinated Approach to Child Health, still 
``CATCH.'' It has been adopted by over 1,000 Texas elementary 
schools with support from public and private partnerships. 
Although schools are already challenged with developing optimal 
curricula for the basics, the CATCH curriculum can integrate 
important life-long health lessons with those already in 
teachers' lesson plans.
    At present, few States have comprehensive cardiovascular 
disease programs. If all States of the United States had 
comprehensive cardiovascular disease programs, then successful 
models such as CATCH might be introduced in schools across the 
country.
    Preventing cardiovascular and other chronic diseases 
increases our quality of life and life expectancy and might 
help lower health care and related costs. CATCH is an example 
of a solidly researched public health intervention strategy 
that needs nationwide funding and implementation to make a real 
difference in Americans' lives.
    Thank you for the opportunity to testify before you today.
    [The prepared statement of Eduardo J. Sanchez follows:]
Prepared Statement of Eduardo J. Sanchez, Commissioner of Health, Texas 
                          Department of Health
    Good morning. Mr. Chairman and members of the Subcommittee on 
Health, it is an honor to appear before you today to testify about the 
Texas Department of Health's ongoing efforts to disseminate public 
health interventions in communities to reduce the health impact and 
related costs of cardiovascular disease--the leading cause of death in 
our country. With your permission, I would like to submit my written 
testimony for the record.
    My name is Eduardo Sanchez, and I am the Texas Commissioner of 
Health. I want to thank you, Mr. Chairman, and the members of this 
Subcommittee for your support for improving the cardiovascular health 
of our nation and for holding this hearing. As the State Health Officer 
and a family physician experienced in community-based care, I've 
treated many who have yet to benefit from the available research on 
effective prevention of cardiovascular diseases and other chronic 
illnesses. Getting research and proven public health interventions 
``off the shelf'' and into communities is vital--for all Americans.
    First, I want to explain why cardiovascular disease prevention is 
one of the Texas Department of Health's top priorities: death, long-
term illness, disability, hospitalization and rising costs related to 
treatment and lost productivity are, quite literally, ``breaking the 
heart'' and breaking ``the bank'' of this country. Heart disease and 
stroke are the leading causes of death in the U.S.: together, these 
cardiovascular diseases kill nearly one million men and women each 
year--this number represents more than 40% of all annual deaths in this 
country. Almost 25% of our population is living with some form of this 
disease--61 million Americans. More than 1 million Americans are 
disabled every year by stroke; and almost 11 million persons over age 
65 report such disabilities as loss of speech or mobility--caused by 
heart disease and stroke. Almost 6 million hospitalizations each year 
are due to cardiovascular disease. For 2001, the costs--in both health 
care expenditures and lost productivity--are estimated at $298 billion 
(CDC, 2002). Both the human and economic costs are enormous. The good 
news is: we know how to reduce this burden; but we must put this 
knowledge into action. I want to share with you one example of 
translating research into positive public health outcomes.
    We know that the primary risk factors for cardiovascular disease 
are: tobacco use, poor nutrition and obesity, and physical inactivity--
these same risk factors also underlie the development of diabetes, 
cancer and other chronic diseases. Texas is one of four states that 
participated in a trial program specifically designed to reduce these 
risk factors. (The others are California, Louisiana and Minnesota.) The 
National Heart, Lung, and Blood Institute (NHLBI) conducted this 4-year 
trial of the most effective concepts and strategies distilled from 
previous studies that intervene on the three risk factors I mentioned 
earlier: sedentary lifestyle, poor dietary choices, and tobacco use. 
Appropriately named ``CATCH'', this method ``catches'' both the 
opportunity to develop healthy habits and prevents cardiovascular 
disease--at the earliest possible time, early childhood.
    During the trial period, this acronym stood for the ``Child & 
Adolescent Trial for Cardiovascular Health''. We not only helped 
demonstrate the effectiveness of the research-based interventions, the 
Texas Department of Health is helping to disseminate this program into 
communities across the state. The program has garnered international 
attention. When results showed clear success, Texas renamed it to 
reflect what really happens: a ``Coordinated Approach To Child 
Health.''
    Briefly, CATCH is the largest school-based health promotion study 
ever conducted in the United States that scientifically documented 
positive changes in children's physical activity and dietary habits. It 
is a research-based model that has proven results. The Texas Department 
of Health is helping diffuse this model statewide by providing 
curricula and training for teachers, cafeteria workers and other school 
personnel.
    CATCH is a comprehensive, coordinated school health program for 3rd 
through 5th graders that introduces healthy behavior through four 
components: classroom curriculum, physical education, school food 
service, and family involvement. Though focused on those three grade 
levels, CATCH teaches our children what healthy food choices are, what 
healthy activity levels are and models other healthy behaviors for 
life. Three years after CATCH was implemented in Texas, students in the 
program reported lower fat intake and more vigorous physical activity 
than those in the control group. Five to seven years down the road, 
schools that received CATCH training were still serving meals 
significantly lower in total fat and saturated fat than schools not 
introduced to this program. Former CATCH students were also spending 
the same amount of time in moderate-to-vigorous physical activity as 
when they were in the program.
    This program's effectiveness is linked not only to its 
comprehensive design, but with how seamlessly it fits into existing 
school curricula, addresses the need for physical activity, raises the 
health status of all school children, involves parents, cafeteria 
workers and entire communities, and has demonstrated long-term 
effectiveness. CATCH has been adopted by over 1,000 Texas elementary 
schools with support from local public health departments and 
professional and community organizations. Other states as well as 350 
Department of Defense schools worldwide are piloting their own CATCH 
programs. All components of this program have been cited 
internationally as examples of ``best practices.''
    We know that schools are already challenged with developing optimal 
curricula for ``the basics'' and ensuring that students are prepared to 
demonstrate their knowledge on standardized tests. The CATCH curriculum 
fits seamlessly into that knowledge base, and it includes a number of 
methods for teachers to integrate these important life-long health 
lessons with those already planned. One teacher commented that the 
curricula ``. . . are very user-friendly (and) easy for any teacher to 
modify or--enrich (what he or she already has planned). It allows for 
individuality in each classroom.'' So while the program is specific in 
its design, it doesn't tell teachers how to teach--CATCH provides the 
tools that teachers need and want to help our kids grow up healthy and 
strong.
    One of the greatest success stories in Texas comes from El Paso, an 
example of community investment. Collaborative efforts among school 
districts, the Texas Education Association, the Texas Department of 
Health, the American Cancer Society, the American Health Association, 
universities and other private and public partners have resulted in 
such important accomplishments as translating materials into Spanish 
and otherwise adapting them to be more culturally appropriate for that 
community. The original, 4-year initiative has grown into a 7-year 
program that reaches an estimated 52,000 students and their families 
through 83 schools in 13 school districts. Grant funds from the Paso 
del Norte Health Foundation (PNHF) are supporting this expansion, with 
an evaluation component funded by PNHF and the American Heart 
Association. Parents, teachers, and school principles have volunteered 
testimonials on the changes in children's health.
    One Texas parent, ``Will's mother,'' wrote that ``(w)hen--Will was 
first enrolled in the program, he was a chubby guy who regularly 
overate. Today, his eating habits are the best of our whole family. Now 
Will is slender and very active. At the age of 13, he plays football, 
baseball, and basketball and works out with weights twice a week. 
Thanks for your part in this healthy metamorphosis.'' This statement 
reflects the kind of changes needed in one of ``the nation's fattest 
cities''; Texas has 5 of the country's 50 fattest cities, according to 
Men's Fitness magazine. The alarming increase in childhood obesity 
demonstrates the urgent need for programs like CATCH--to intervene 
early, preventing disease before it strikes down our citizens, often in 
the prime of their lives.
    Much of this devastation on our people, on our economy, on our 
quality of life--can be prevented. We know what causes cardiovascular 
disease; and we have solid, tested methods for helping people make wise 
decisions to reduce their risk for these chronic diseases, as well as 
improve their overall health status. Right now, few states have 
comprehensive cardiovascular disease programs, yet all Americans 
experience these health problems. Research reveals that young people, 
members of racial and ethnic minorities, women, and people of low 
socioeconomic status are most at risk for developing these problems and 
suffering the greatest effects (CDC, 2002). The Texas Department of 
Health validated that the CATCH curriculum, as implemented in Texas 
communities, is flexible enough to ensure cultural appropriateness and 
is a good, comprehensive strategy for child health.
    If all states had comprehensive cardiovascular programs, then 
successful models--such as CATCH--can be taken directly to communities. 
We've known the risk factors for cardiovascular disease for over 50 
years--the result of the NHLBI's long-term research on cardiovascular 
health, the Framingham Heart Study. We've known how to prevent the 
development of these risk factors for over 10 years. Preventing 
cardiovascular and other chronic diseases increases the quality-of-life 
and life expectancy. It also lowers health care and related costs. 
CATCH is an excellent example of solidly researched public health 
intervention strategies that need adequate funding to make a real 
difference in people's lives.
    Thank you for the opportunity to testify before you today. I will 
be happy to answer any questions you may have.

    Mr. Bilirakis. Thank you very much, Doctor.
    Dr. Jones?

                    STATEMENT OF DANIEL JONES

    Mr. Jones. Mr. Chairman, I would like to express my 
appreciation to you and the members of the subcommittee for 
holding this hearing on this very important issue, and thank 
you, Congressman Pickering, for your kind introduction and for 
your continued leadership.
    I am here today to tell you about the enormous impact of 
stroke, our Nation's number 3 killer, and the opportunity to 
advance the fight against stroke through research. The American 
Heart Association and our American Stroke Association Division 
applauds Congress for its commitment to double the budget of 
the National Institutes of Health over 5 years by fiscal year 
2003. Fulfillment of this commitment will help us to develop 
new knowledge and tools to more effectively prevent and treat 
stroke and other cardiovascular diseases.
    Together, NIH and the stroke community have advanced our 
knowledge of stroke. Stroke was once viewed as an untreatable 
disease, but important new information shows promise for 
improved stroke diagnosis, treatment, rehabilitation, and 
prevention. Research during the last decade provided physicians 
new resources to prevent, diagnose, and treat stroke.
    For example, NINDS-sponsored clinical trials showed the 
effectiveness of a clot-busting drug when administered to 
appropriate patients within 3 hours of the onset of symptoms of 
a clot-based stroke. Another series of trials found that 
aspirin or warfarin reduced stroke risk to 80 percent in 
victims of atrial fibrillation and irregular heart beat 
associated with 70,000 strokes each year. NHLBI-sponsored 
clinical trials confirmed the benefit of treatment of high 
blood pressure and high cholesterol as ways to prevent stroke.
    The bad news is, despite this progress, stroke remains the 
Nation's third leading cause of death and, importantly, a major 
cause of disability. We must make a commitment to do more. With 
the aging of the population, the number of stroke patients in 
the United States will substantially grow in the coming 
decades.
    Despite the overall effort to double the NIH budget, stroke 
research, as has been pointed out, remains disproportionately 
underfunded in view of the enormous burden this disease places 
on our Nation and the numerous scientific opportunities in the 
future. Presently, only 1 percent of the NIH budget is invested 
in stroke research and related programs.
    We urge Congress to ensure that the NIH budget for stroke 
also doubles over the same 5-year period. An appropriation of 
$316 million for fiscal year 2003 is needed to accomplish this 
goal.
    Each year over 600,000 Americans suffer a stroke, and 
167,000 of them die. This devastating disease touches the lives 
of nearly all Americans. There are currently 4.6 million stroke 
survivors living in the United States, and as many as 30 
percent of survivors are permanently disabled, requiring 
extensive and costly care. My home State of Mississippi, which 
is in the Stroke Belt Region, has the seventh highest stroke 
death rate in the Nation.
    Stroke is a costly disease. Nationally, stroke is expected 
to cost our Nation $49.4 billion in 2002, including $30.8 
billion in direct medical costs. Since a large share of these 
costs are paid for by public payors like Medicare, these 
programs should be modernized to better address stroke.
    For example, Medicare should cover the cost of preventative 
cholesterol screening in order to better detect stroke risk. 
The American Heart Association and its American Stroke 
Association Division has set a bold goal to reduce stroke and 
risk of stroke by 25 percent by the year 2010. The association, 
which does not accept government funding, plans to reach this 
goal through its continued efforts to fund stroke research, 
educate the public about stroke, and implement successful 
community-based programs.
    We can reach this goal, but it will take commitment, hard 
work, a continued close relationship with Federal agencies like 
the National Institutes of Health and public and private 
resources. The American Stroke Association applauds the 
National Institute of Neurological Disorders and Stroke at the 
NIH for recognizing the need to do more in this area and for 
developing a road map for the next decade of stroke research.
    We are pleased that several of the critical priorities 
identified by NINDS include the development of stroke center 
networks with sufficient infrastructure to deliver quality 
stroke care, improvement of data bases for collection and 
analysis of stroke data, and expansion of efforts to raise 
public awareness and train medical professionals about stroke. 
Accomplishing these goals will require a shared responsibility 
on the part of Congress and the public health community.
    We encourage the members of this subcommittee to actively 
join our fight. We are pleased that the members of this 
subcommittee are committed to funding stroke research and 
ensuring that this research is translated into effective care.
    For example, we particularly want to thank Congresswoman 
Lois Capps and Congressman Chip Pickering for introducing the 
Stroke Treatment and Ongoing Prevention Act, or Stop Stroke 
Act, and the chairman for his support stated today. This vital 
legislation will ensure that stroke is more widely recognized 
by the public and treated more effectively by health care 
providers. The Stop Stroke Act unanimously passed the Senate 
and currently has strong bipartisan support from 175 co-
sponsors in the House.
    Last, I want to take a moment to congratulate Dr. Lenfant 
and the National Heart, Lung, and Blood Institute on the 30th 
anniversary of their National High Blood Pressure Education 
Program that was mentioned earlier. This is one of the most 
successful public awareness programs in existence, and it has 
helped dramatically increase the interest and awareness of 
hypertension or high blood pressure, one of the leading risk 
factors for stroke.
    Thank you, and I would be happy to answer any questions, 
and look forward to ongoing dialog with the subcommittee. 
Thanks.
    [The prepared statement of Daniel Jones follows:]
  Prepared Statement of Daniel Jones, American Stroke Association, a 
               Division of the American Heart Association
    Thank you Congressman Pickering. I appreciate your leadership on 
this issue. I would also like to thank Chairman Bilirakis and the 
members of the Subcommittee for holding this hearing.
    I am Dr. Daniel Jones, the Associate Vice Chancellor for Health 
Affairs and Associate Dean, School of Medicine at the University of 
Mississippi Medical Center. I am also the Herbert G. Langford Professor 
of Medicine, the Co-director of the Division of Hypertension and 
Associate Director of the Center for Excellence in Cardiovascular-Renal 
Research. In addition, I am also an active volunteer for the American 
Heart Association and its American Stroke Association Division. In this 
capacity, I serve on the NIH National High Blood Pressure Education 
Program Coordinating Committee and am the chairman of the AHA 
International Committee.
    I am here today to tell you about the enormous impact of stroke--
our nation's number three killer--and the opportunity to advance the 
fight against stroke through research.
                significant advances in the last decade
    The American Heart Association and our American Stroke Association 
division applaud Congress for its commitment to double the budget of 
the National Institutes of Health (NIH) over five years--by Fiscal Year 
2003. Fulfillment of this commitment will help us develop new knowledge 
and tools to more effectively prevent and treat stroke and other 
cardiovascular diseases.
    Together, NIH and the stroke community have advanced the knowledge 
of stroke. Stroke was once viewed as an untreatable disease. 
Importantly, new information shows promise for improved stroke 
diagnosis, treatment, rehabilitation and prevention.
    Research during the last decade provided physicians new resources 
to prevent, diagnose and treat stroke. Highlights of selected National 
Institute of Neurological Disorders and Stroke (NINDS) supported stroke 
studies follow.

 A clinical trial showed that when administered to appropriate 
        patients within three hours of the onset of symptoms of a clot-
        based stroke, tissue plasminogen activator (tPA), the only FDA-
        approved emergency treatment for stroke, can restore blood flow 
        and reduce the chances of permanent disability by 33 percent. 
        It is estimated to save $4.5 million to $5 million for every 
        1000 patients treated.
 A series of clinical trials found that aspirin or warfarin 
        reduced stroke risk by up to 80 percent in victims of atrial 
        fibrillation, an irregular heart beat that is associated with 
        70,000 strokes each year. Either aspirin or warfarin treatment 
        could prevent up to 30,000 strokes each year with an annual 
        savings of $200 million. For many atrial fibrillation victims, 
        the less expensive, less complicated aspirin can provide 
        sufficient protection from stroke.
 A clinical trial is evaluating medications, ticlopidine and 
        aspirin, to prevent recurrent stroke in African-Americans. 
        Importance of prevention and early risk factor treatment are 
        stressed.
 Clinical trials sponsored by the National Heart, Lung and 
        Blood Institute (NHLBI) confirmed the benefit of treatment of 
        high blood pressure and high cholesterol as ways to prevent 
        stroke.
            stroke is still the nation's number three killer
    The bad news is, despite this progress, stroke remains the nation's 
third leading cause of death, and importantly, a major cause of 
disability. We must make a commitment to do more. With an aging 
population, the number of stroke patients in the United States will 
substantially grow in the coming decades.
    Despite the overall effort to double the NIH budget, stroke 
research remains disproportionately underfunded in view of the enormous 
burden this disease places on our nation and the numerous scientific 
opportunities. Presently, only 1 percent of the NIH budget is invested 
in stroke research and related programs.
    We urge Congress to ensure that the NIH budget for stroke also 
doubles over the same five-year period. An appropriation of $316 
million for Fiscal Year 2003 is needed to accomplish this goal.
    Each year, more than 600,000 Americans suffer a stroke and 167,000 
of them die. This devastating disease touches the lives of nearly all 
Americans. There are currently 4.6 million stroke survivors living in 
the United States, and as many as 30 percent of the survivors are 
permanently disabled, requiring extensive and costly care.
    My home state of Mississippi, which is in the stroke belt region, 
has the seventh highest stroke death rate in the nation. Mr. Chairman, 
I have included as part of my testimony a chart that illustrates the 
impact of stroke on each state.
    Stroke is a costly disease. Nationally, stroke is expected to cost 
our nation $49.4 billion in 2002, including $30.8 billion in direct 
medical costs. Since a large share of these costs are paid for by 
public payors like Medicare, these programs must be modernized to 
better address stroke. For example, Medicare should cover the cost of 
preventive cholesterol screening in order to better detect stroke risk.
the american heart association--we're putting our heart behind fighting 
                                 stroke
    The American Heart Association and its American Stroke Association 
division have set a bold goal of reducing stroke and risk of stroke by 
25 percent by the year 2010.
    The Association, which does not accept government funding, plans to 
reach this goal through its continued efforts to fund stroke research, 
educate the public about stroke and implement its successful community-
based programs. The Association leverages credible science, a strong 
reputation and a nationwide infrastructure of Affiliates to advance its 
mission.
    We can reach this goal, but it will take commitment, hard work, a 
continued close partnership with federal agencies like the National 
Institutes of Health and public and private resources.
           nih sets roadmap for the future of stroke research
    The American Heart Association and its American Stroke Association 
division applauds the National Institute of Neurological Disorders and 
Stroke (NINDS) at the NIH for recognizing the need to do more in this 
area and for developing a roadmap for the next decade of stroke 
research.
    NINDS assembled leaders in the stroke field to form the Stroke 
Progress Review Group to develop the strategic plan, which was released 
in April, 2002. Our Association plays an active role in this group. The 
report identifies five research priorities for the next five to ten 
years as well as seven priorities needed to implement this important 
research so that patients benefit in a meaningful and timely manner.
    Several of the critical priorities identified in the NINDS report 
include the development of stroke center networks with sufficient 
infrastructure to deliver quality stroke care, improvement of databases 
for collection and analysis of stroke data and expansion of education 
and training.
     translating research into improved stroke care and prevention
    Accomplishing these goals will help ensure that stroke research 
advances are translated into practice, but their fulfillment will 
require a shared responsibility on the part of Congress and the public 
health community.
    We are pleased that Members of this Subcommittee are committed to 
funding stroke research. We ask that the Subcommittee also help ensure 
that this research is translated into effective stroke care and 
prevention by advancing legislation like the Stroke Treatment and 
Ongoing Prevention Act (STOP Stroke Act).
    We thank Congresswoman Lois Capps and Congressman Chip Pickering 
for introducing this vital legislation (H.R. 3431/S.1274), which will 
help ensure that stroke is more widely recognized by the public and 
treated more effectively by healthcare providers.
    The STOP Stroke Act addresses a number of barriers that prevent 
stroke patients from accessing quality care. These barriers include low 
public awareness, lack of awareness among medical professionals, lack 
of infrastructure and lack of data collection. Many of these barriers 
were also identified as priorities in the Report of the Stroke Progress 
Review Group, and their removal is critical to the translation of NIH 
stroke research advances into practice.
    For example, the legislation addresses the critical need to better 
educate the public about stroke. Despite being the nation's number 
three killer, the public knows very little about stroke. Only 68 
percent of the general public can name the most common stroke warning 
sign--sudden numbness or weakness on one side of the body. Even more 
alarming, those at the greatest risk--seniors, minorities and women--
know the least about the stroke warning signs.
    Stroke is a medical emergency and must be treated rapidly. 
Unfortunately, since many Americans do not recognize the stroke warning 
signs, stroke victims frequently wait as long as 22 hours before 
seeking medical attention. The treatment window for most strokes is as 
short as three hours from the onset of symptoms.
    We look forward to continuing to work with Representatives Capps 
and Pickering to advance this legislation.
    The STOP Stroke Act unanimously passed the Senate and currently has 
strong bipartisan support from 175 co-sponsors in the House, including 
21 Members of this Subcommittee.
    Lastly, I want to take a moment to congratulate Dr. Lenfant and the 
National Heart, Lung and Blood Institute on the 30th anniversary of the 
National High Blood Pressure Education Program. This is one of the most 
successful public awareness programs in existence and has helped 
dramatically increase awareness of hypertension, or high blood 
pressure, one of the leading risk factors for stroke.
    Thank you. I have included as part of my testimony several 
documents that provide additional background information about stroke 
as well as information about the American Heart Association and its 
American Stroke Association division, including what we are doing to 
fight this devastating disease.
    I would be pleased to answer any of your questions and look forward 
to an ongoing dialogue with the Subcommittee as you address these 
important issues.
                          stroke in your state
    In 1999, stroke was the number 3 killer in every state but Colorado 
(number 4), Nevada (number 4), New Mexico (number 5) and Wyoming 
(number 4).
    The following chart shows the number of stroke deaths in your 
state, your state's stroke death rate (number of deaths per 100,000 
people), and your state's rank.

------------------------------------------------------------------------
                                    Number of
                                      Stroke       Rank**      Rate***
              State                  Deaths*    (Highest to  (Deaths per
                                      (1999)      Lowest)      100,000)
------------------------------------------------------------------------
Alabama..........................        3,148           11         68.1
Alaska...........................          171           20         63.7
Arizona..........................        2,600           44         56.2
Arkansas.........................        2,255            2         85.4
California.......................       17,962           26         61.5
Colorado.........................        1,834           43         56.5
Connecticut......................        1,933           46         52.1
Delaware.........................          365           47         51.8
District of Columbia.............          297           33         60.4
Florida..........................       10,560           48         51.3
Georgia..........................        4,416            6         73.8
Hawaii...........................          762           39         58.9
Idaho............................          771           17         65.1
Illinois.........................        7,714           23         62.3
Indiana..........................        4,057            9         68.8
Iowa.............................        2,317           28         61.1
Kansas...........................        1,841           24         62.0
Kentucky.........................        2,710           13         67.6
Louisiana........................        2,684           14         67.2
Maine............................          879           42         57.0
Maryland.........................        2,892           35         60.0
Massachusetts....................        3,548           51         49.3
Michigan.........................        6,041           22         62.7
Minnesota........................        2,997           27         61.4
Mississippi......................        1,854            7         69.9
Missouri.........................        3,950           18         64.5
Montana..........................          595           31         60.6
Nebraska.........................        1,176           37         59.6
Nevada...........................          882           21         63.0
New Hampshire....................          669           29         61.0
New Jersey.......................        4,122           50         50.1
New Mexico.......................          817           45         53.1
New York.........................        8,124           52         42.9
North Carolina...................        5,626            4         77.8
North Dakota.....................          513           25         61.5
Ohio.............................        7,235           34         60.2
Oklahoma.........................        2,481           12         68.0
Oregon...........................        2,799            5         77.2
Pennsylvania.....................        8,600           38         58.9
Puerto Rico......................        1,814           40         58.5
Rhode Island.....................          633           49         51.2
South Carolina...................        2,974            1         86.0
South Dakota.....................          547           32         60.5
Tennessee........................        4,103            3         78.3
Texas............................       10,414           16         66.3
Utah.............................          869           30         60.9
Vermont..........................          344           41         58.4
Virginia.........................        4,110            8         69.3
Washington.......................        3,718           10         68.1
West Virginia....................        1,323           36         59.6
Wisconsin........................        3,869           15         66.3
Wyoming..........................          265           19         63.9
------------------------------------------------------------------------
*Source: National Center for Health Statistics: National Vital
  Statistics Reports for 1999. Age adjustments are based on the 2000
  standards.
**The rank is based on the state death rate and is listed from highest
  (1) to lowest (52). For the purposes of the chart, the District of
  Columbia and Puerto Rico are listed and ranked as states.
***Source: National Center For Health Statistics compressed mortality
  file for the years 1996 to 1998.

    As a division of the American Heart Association, the American 
Stroke Association's mission is to reduce disability and death from 
stroke through research, education, fundraising, and advocacy. The 
American Stroke Association leverages credible science, a strong 
reputation, and a nationwide infrastructure of Affiliates to advance 
its mission. The American Stroke Association's goal is to reduce stroke 
and stroke risk by 25 percent by the year 2010.
                    programs, products and services
    The American Stroke Association's initiatives are delivered through 
three primary categories:
I. Primary Prevention of Stroke
 The American Stroke Association produces educational 
        materials, including brochures and videos, for both 
        professional and consumer audiences, with a high level of focus 
        on women, African Americans and seniors.
 Search Your Heart is an educational program designed to reach 
        African Americans in a church setting, which encourages church 
        members to change their lifestyles in order to build heart-
        healthy bodies. The program contains several activity kits, 
        including a module called Stomp Out Stroke, that are designed 
        to educate people about risk factors and warning signs.
II. Acute Care/The Acute Event
 Operation Stroke--Created in 1997, Operation Stroke is a 
        comprehensive community initiative that pulls together local 
        resources necessary to provide optimal care for those 
        experiencing a stroke.
 Acute Stroke Treatment Program (ASTP)--This implementation 
        resource was developed as the tool for the Brain Attack 
        Coalition's Guidelines for Primary Stroke Centers, published in 
        the Journal of the American Medical Association (JAMA), which 
        the American Stroke Association co-authored. Since its launch, 
        more than 3,000 kits have been distributed to hospitals across 
        the United States, and the ASTP is considered the premier 
        resource for implementing primary stroke centers.
 The need for rapid action is communicated to consumers through 
        national media and call-to-action campaigns, radio public 
        service announcements and national alliances with other 
        organizations that can impact care at the time of an acute 
        event.
 Stroke: When Minutes Matter, is a senior education program 
        designed to help seniors identify the stroke warning signs and 
        to respond promptly by calling 9-1-1. Pilot results showed 10-
        15 percent improvement (between pre- and post-tests) in senior 
        recognition of stroke warning signs.
III. Secondary Prevention and Post-Stroke Rehabilitation
 Get with the Guidelines is a Web-based initiative delivered at 
        the hospital level to develop hospital-based protocols to 
        implement primary and secondary prevention guidelines for 
        cardiovascular disease and stroke. The stroke module is 
        currently in pilot with the Patient Management Tool (a Web-
        based data collection tool also used to support some states in 
        the Paul Coverdell stroke registry pilot).
 The American Stroke Association provides valuable resources 
        for stroke survivors and caregivers, including:
     1. Stroke Connection magazine;
     2. A toll-free ``Warmline'' (888-4-STROKE) staffed by 
            stroke survivors and caregivers; and
     3. Support Group Registry of more than 2,000 support 
            groups nationwide.
                         professional resources
 Attended by more than 2,400 people, the American Stroke 
        Association's International Stroke Conference provides an 
        educational experience for neurologists, surgeons, physicians, 
        nurses and allied health professionals. The conference, which 
        highlights major advances in fundamental and clinical stroke-
        related research, is considered among the most successful and 
        prestigious stroke conferences in the world.
 Stroke: A Journal of the American Heart Association is the 
        premier scientific journal for those involved in the care of 
        stroke patients.
 The Stroke Trials Directory is a one-of-a-kind Web site that 
        contains descriptions of completed and ongoing stroke 
        therapeutic trials, positive and negative.
 The Satellite Broadcast on Acute Stroke, one of a series of 
        live, interactive satellite professional education broadcasts, 
        reached more than 4,500 healthcare professionals 
        simultaneously. The Emerging Science broadcast reached more 
        than 8,600 health care professionals simultaneously. Future 
        topics may include Secondary Prevention and Comprehensive 
        Stroke Centers.
 Healthcare professionals and others interested in stroke can 
        sign up to receive a quarterly email communication, the Stroke 
        Information Alliance, to keep up with the latest activities and 
        initiatives of the American Stroke Association (by sending 
        email information to [email protected])
 Stroke volunteers and alliances may also keep up-to-date 
        through our Extranet community located at 
        www.strokecommunities.org
                      national strategic alliances
    In order to align goals and strategies at a national level to fight 
against heart disease and stroke, the American Heart Association and 
American Stroke Association recently signed an historic Memorandum of 
Understanding, considered a milestone in public and private sector 
cooperation, with:

 Centers for Disease Control;
 National Heart, Lung and Blood Institute;
 National Institute of Neurological Diseases and Stroke;
 Office of Disease Prevention and Health Promotion; and
 Centers for Medicare and Medicaid Services
    These collaborative efforts, coupled with the organization's 
ability to work through more than 2,000 local offices, provides a 
strong basis for delivering stroke messages to consumers and 
professionals across the United States.
   american heart association/american stroke association identified 
                     stroke research opportunities
Improved Treatments for Stroke
    Blood clots or bleeding into the brain can cause strokes. tPA use 
has dramatically improved treatment of clot-caused strokes when 
administered within three hours of the onset of stroke symptoms. Using 
coils, balloons and stents in arteries in the brain has dramatically 
prevented bleeding and strokes in small numbers of patients. However, 
many challenges to improving treatment remain. Strokes often are not 
recognized quickly. They can start with mild or confusing symptoms, or 
in the middle of the night. Also, techniques for treating strokes, 
other than using tPA, require extensive training for safe and effective 
use and so are often not available. Therefore, a compelling need exists 
to develop medications, devices and techniques to accomplish three 
specific but broad goals: 1) more effective stroke treatment; 2) safer 
treatment than is now available that can be administered later after 
stroke onset than is possible now; and 3) treatment that can be used 
more widely, in more patients, by more doctors.
Limiting Damage Due to Interruption in Blood Flow and Restoration of 
        Blood Flow
    Strokes damage the brain by depriving affected areas of blood and 
oxygen. If blood flow is restored quickly after a stroke occurs--for 
example, by using tPA--this damage is prevented. Even if certain areas 
are not completely deprived of blood, or are only without blood for a 
few hours, they may not recover when blood flow is restored. Much 
research has focused on damage caused when parts of the brain do not 
receive blood. But so far there has been little practical progress. 
Further research is needed to develop ways to 1) prevent damage to 
cells around the area deprived of blood, 2) prolong the time that areas 
of the brain can be partially deprived of blood and still recover and 
3) help damaged brain cells recover. This will require a better 
understanding of how brain cells respond to injury and developing new 
drugs to help in prevention, preservation and healing. Treatments based 
on reopening brain blood vessels are often complicated by bleeding into 
the brain, when blood flow is re-established. Studies are needed to 
understand why such bleeding occurs and how best to prevent it.
Brain Imaging Techniques
    To be considered for tPA, a patient must undergo imaging studies 
within three hours of the onset of stroke symptoms to 1) confirm that 
the symptoms stem from a stroke and 2) determine that the stroke 
results from a blood clot, not bleeding. Thus, an imaging technique 
must quickly provide information about stroke. Efforts must be directed 
toward 1) advancing the technology of imaging techniques to reduce the 
time to obtain images and improve image quality, and 2) investigating 
and expanding imaging to other applications that directly impact stroke 
care, such as telemedicine. Telemedicine is implementing radiological 
and communications technology to facilitate treatment of stroke 
patients in communities without immediate access to adequate emergency 
care.
Stroke Risk Factors
    The devastating impact of stroke will continue until proven methods 
and techniques prevent its long-term debilitating effects. After 
researchers identify and understand how risk factors predispose someone 
to stroke, more people at high risk for stroke can be identified, 
evaluated and treated to prevent a future stroke.
Stroke and Dementia
    Research on dementia has focused on Alzheimer's disease, but some 
of dementia in the elderly stems from stroke. Dementia remains a 
contributing factor in the overall outcome and quality of life of 
stroke survivors and of patients with high blood pressure. Lesions in 
the brain and some of its blood vessels can cause dementia either by 
producing multiple strokes (multi-infarct dementia) or by producing 
lesions in the white matter of the brain (leukoaraiosis). More research 
must define risk factors for multiple strokes, its underlying causes 
and effective preventive measures. Furthermore, more clinical and basic 
research is needed to characterize the risk factors for leukoaraiosis 
and to define its causes and potential treatments.
Functional Recovery from Stroke
    More Americans are expected to suffer from stroke as the ``baby 
boomer'' population ages. Managing high-risk patients and evaluating a 
stroke in progress must continue to focus on predicting and improving 
functional recovery from stroke.
Stroke Education and Awareness
    Studies underscore the importance of comprehensive and effective 
educational efforts to increase public awareness of stroke. They 
emphasize three important steps in improving stroke outcome: educating 
the public about stroke risk factors, recognizing stroke warning signs 
and seeking emergency medical attention. Several audiences must be 
targeted: the public, especially high-risk populations (blacks, 
elderly, diabetics), stroke survivors, healthcare professionals, 
emergency medical personnel, hospital administrators, civic leaders and 
government officials. Primary stroke center recommendations published 
by the Brain Attack Coalition in the June 21, 2000 Journal of the 
American Medical Association clearly define recommendations for 
hospitals to implement stroke centers, teams and other programs to 
improve stroke treatment.
Genomics and Proteomics in Stroke
    The factors that predispose someone to stroke are complex. They are 
influenced by multiple genes interacting with each other and the 
environment. Recent advances in genomics and proteomics help identify 
genes and their protein products involved in developing brain blood 
vessel disease and stroke. Knowledge of these genes will help 
scientists and physicians use an individual's genetic makeup to 
identify subgroups of the population at risk for stroke, establish 
which groups are most likely to benefit from specific treatments, and 
provide the scientific basis for developing innovative approaches to 
treat and prevent stroke. Important opportunities for research include 
1) developing new technologies for studying the differing patterns of 
gene expression of normal and diseased cells and tissues and 2) 
measuring interactions between genetic variants and specific 
environmental changes to identify genes that modify the impact of the 
environment on brain blood vessel disease.
    Proteomics builds on and complements knowledge gained from genomics 
and genetic screening approaches. It helps provide a functional 
understanding of how genes regulate the blood vessels. It also allows 
investigators to identify alterations in protein structure and function 
that lead to brain blood vessel disease and stroke. Genomic and 
proteomic studies are often complex and require sophisticated 
analytical tools to store and analyze data. So studies involving 
multiple investigators and centers and ways to share data among 
investigators should be encouraged.
                       frequently asked questions
What is a stroke?
    Stroke is a cardiovascular disease that affects the blood vessels 
supplying blood to the brain. A stroke occurs when a blood vessel 
responsible for supplying the brain with oxygen and nutrients bursts or 
becomes excessively clogged by a blood clot or some other particle.
What are the types of stroke?
    There are two main types of stroke: ischemic strokes and 
hemmorrahagic strokes.

 Ischemic strokes are caused by blood clots that form and block 
        blood flow to the brain. Ischemic strokes are most common and 
        account for 80 percent of all strokes.
 Hemmorrahagic strokes are caused by a break in an artery in 
        the brain, causing blood to fill the area and damage the 
        surrounding tissue.
What is a TIA (transient ischemic attack) or ``mini stroke''?
    A TIA is a sudden but temporary interruption of the blood supply to 
the brain resulting in symptoms that last from several minutes to 
several hours, but not more than 24 hours.
What are the effects of stroke?
    Strokes affect people in different ways, depending on the type of 
the stroke, the area of the brain affected and the extent of the brain 
injury. Strokes can cause devastating disability, including:

 Paralysis or muscle weakness
 Difficulty in speaking or swallowing
 Blindness
 Cognitive impairment or memory loss
 Incontinence
How can stroke be prevented?
    The American Heart Association has identified several factors that 
increase the risk of stroke. The more risk factors a person has, the 
greater the chance that he or she will have a stroke. The best way to 
prevent a stroke is to reduce the controllable risk factors, which 
include:

 High blood pressure
 Tobacco use
 High cholesterol levels
 Obesity
 Physical inactivity
    There are also a number of uncontrollable risk factors for stroke 
including age, gender, race, family history of heart disease, stroke or 
diabetes.
What are the stroke warning signs?
    The most common warning signs of stroke include the following:

 Sudden numbness or weakness of the face, arm or leg, often on 
        one side of the body.
 Sudden confusion, trouble speaking or understanding.
 Sudden trouble seeing in one or both eyes.
 Sudden trouble walking, dizziness, loss of balance or 
        coordination.
 Sudden severe headache with no known cause.
    Not all of these warning signs occur in every stroke. If some signs 
begin to occur, don't wait. Get help immediately. Stroke is a medical 
emergency.
How is stroke currently treated?
    Depending on the type and severity, stroke can be treated through 
surgery, drugs, acute hospital care and rehabilitation. If the stroke 
is caused by a blood clot (ischemic stroke), clot-busting drugs can 
sometimes be used to dissolve the clot. This treatment, which was 
approved by the Food and Drug Administration in 1996, is a significant 
advance in the war against stroke.
    For clot-busting drugs to be effective, treatment must be started 
within three hours of the onset of the stroke. Therefore, it's critical 
that care1ivers, medical professionals and the public recognize stroke 
as a medical emergency and respond immediately. Unfortunately, for a 
number of reasons, only 3 to 5 percent of patients who could benefit 
from these drugs actually receive the treatment.
Can stroke survivors be rehabilitated?
    Besides being the third leading cause of death in the United 
States, stroke is a leading cause of serious, long-term disability. 
Many survivors are left with mental and physical disabilities of 
varying severity, and nearly all stroke survivors can benefit from an 
appropriately structured and comprehensive rehabilitation program. 
People with the least impairment are likely to benefit from 
rehabilitation the most. Sometimes even modest gains can mean the 
difference between staying in an institution and returning home. The 
goal of rehabilitation is to increase independence and improve physical 
abilities. Rehabilitation is most successful if initiated early.
In addition to current treatments and therapies, there are many 
        promising medical advances on the horizon. What is the goal of 
        these new treatments?
    A number of new stroke technologies are in development. The primary 
objectives of these medical advances are to more effectively remove 
blood clots that cause stroke and to extend the therapeutic window.

 New drug therapies are under development that would, like 
        current treatments, dissolve blood clots. Physicians hope to 
        improve the performance of these drugs by delivering them 
        directly to the blood clot through small tubes threaded 
        directly into arteries or the brain itself. Researchers are 
        also trying to develop very small mechanical devices that can 
        be delivered through these small tubes to break-up or remove 
        blood clots.
 Once a blood clot occurs and a portion of the brain is starved 
        of blood, a series of chemical reactions take place. Many of 
        these chemical reactions occur in the first few hours of a 
        stroke and actually cause most of the damage to the brain. This 
        short time frame means that many patients are not able to 
        receive treatment in time to prevent significant brain injury. 
        Researchers hope to develop drugs that stop or delay these 
        chemical reactions, prevent permanent damage to the brain and 
        expand the therapeutic window so that there is more time to 
        provide treatment.

    Mr. Bilirakis. Thank you. Thank you very much, Dr. Jones.
    Dr. Bonow, certainly because of your past experience on the 
research staff at the institute, you're more than anybody here, 
I guess, in a unique position to explain to us how NIH research 
activities translate to patients and their providers. In the 
process of formulating your answer, I wish you would maybe 
expand my question, so that you can expand your answer, to go 
into the public advocacy organizations such as your 
organization, such as Mr. Hargis' organization, in terms of 
their relationship with NIH, in terms of interacting with NIH, 
and their having difficulty interacting with NIH, et cetera. 
Please proceed, sir.
    Mr. Bonow. Thank you, Mr. Chairman. I do have a unique 
perspective, both on the inside and outside, as a researcher 
and currently as somewhat of an administrator over a large 
number of cardiologists who are seeking NIH grants.
    I do believe that I can reassure the committee that, from 
my perspective, the administrative components of the National 
Institutes of Health are doing a good job. There is a large 
attention to basic research, but basic research is vital, 
getting to some of the issues raised in the previous panel. New 
drug development only comes when there is an understanding of 
the fundamental mechanisms of disease. You can develop a whole 
new class of drugs when you know what is going on in the cell, 
on the cell membrane. If we have cell regeneration, perhaps 
there are drugs that can be developed to simulate this. So the 
basic science is critically important.
    Translating that to clinical research is equally important. 
Intramurally, getting back to one of the questions I believe 
from Mr. Deal this morning, much of the research done in 
Bethesda is patient-based research. My 16 years there was 
spent, at least 14 years, involved in clinical trials in 
patients with either new drugs, new diagnostic tests, new 
indications for surgical advances, and so forth. I believe that 
is vitally important to continue this focused research in a 
very heady research environment, unencumbered by the rest of 
the issues that someone in a medical school environment must 
deal with in trying to accomplish research.
    On the other hand, one needs to be able to translate 
research in a protected environment like the NIH into the real 
world, and this is where the clinical trials become very 
important. In the current year of cardiovascular disease 
research, much of the clinical trials are being performed by 
drug companies. This is very important. This is the way we 
advance our understanding of drugs.
    But I believe the federally funded clinical trials are 
preeminent in their objectivity, their research 
accomplishments. Negative results are published equally with 
positive results without bias. These trials are fundamentally 
important.
    I do believe that the process by which clinical trials get 
developed and approved and implemented is a good one with 
appropriate peer review by external reviewers. So, therefore, I 
think that component is fine.
    The other component you raise of how this now gets 
implemented into outcomes and improvement in patient care is 
something the NIH does focus on, perhaps could focus on more, 
and perhaps better. But it can only do so in partnership with 
other agencies, I think, like CDC, or perhaps at the state 
level with state departments, such as Dr. Sanchez mentioned, 
partnerships with NIH and public health groups.
    Mr. Bilirakis. Is that being done on an adequate basis?
    Mr. Bonow. Well, as has been discussed in the first panel, 
the Memorandum of Understanding I think is critical. This is an 
official liaison between these Federal agencies. We in the 
American Heart Association are part of this as well, and we are 
proud to be. It does help to translate things beyond the 
research arena into outcomes or outcomes-based research, and 
then perhaps it can actually improve care nationwide.
    The CDC I believe should be focused on. I know it is not 
the topic for today's discussion, but CDC's chronic disease 
program, which can address cardiovascular disease and stroke at 
the state level, would require more funding to implement this 
nationwide. Currently, only six States are receiving full 
support from CDC to implement chronic disease programs, 
including cardiovascular disease and stroke. I would like to 
see more funding going to the CDC chronic disease components to 
do this.
    My district in Chicago for African American men has the 
third worst mortality rates in the country for cardiovascular 
disease. We are not doing enough to translate what we already 
know works. It is not new research; it is old research. What 
already works, but to implement that effectively and at the 
community level. To answer your question, I don't believe we 
are focusing on this enough.
    This also leads, I guess, to your final question, which is 
how the private agencies or groups before you today could help 
because we are focusing on this, too. We can't do it alone. We 
need to have partnerships with other peer groups. We work with 
the American College of Cardiology, the American Diabetes 
Association, but in partnership with the Federal agencies, I 
believe we can get the job done.
    Mr. Bilirakis. My time has expired, and we have a series of 
votes coming up. So we should finish up, would like to finish 
up, because I don't want to keep you that long.
    Mr. Hargis, I intended to ask you basically the same 
question, and consider that question asked, but will you 
respond in writing to us----
    Mr. Hargis. Certainly.
    Mr. Bilirakis. [continuing] regarding it? I would like to 
know your experience with NIH in terms of your relationship 
with them. Can you interact with them? Do they listen to you? 
You know, things of that nature.
    Obviously, we think the world of them in general, but we 
also want to make sure that they are spending the taxpayers' 
dollars the right way, and that the organizations such as 
yours, since you spend so much time on these particular 
epilepsy and heart and all these other diseases, we want to 
make sure that they listen; if not necessarily go along with it 
all the time, at least are listening.
    All right, I will now yield to Mr. Brown.
    Mr. Brown. Thank you, Mr. Chairman. I will do better than I 
did the last time you yielded to me. Thank you.
    Dr. Sanchez, just one pretty simple question: We have 
talked at length about translating basic research into basic 
health through both panels, in large part through the 
development of prescription drugs and antibiotics. I was 
interested in your discussion about translating basic research 
into prevention.
    Tell us, if you would, in my State of Ohio we have, I 
believe Columbus has some of the highest diabetes mortality 
rates in the country. Diabetes is a serious problem, obviously, 
everywhere. Ohio, you are at the top of the list by most 
measurements.
    Tell us, if you would, a little more about what you have 
done through the Texas Department of Health in terms of obesity 
and exercise and diet, and all the issues that clearly make the 
diabetes probably the worst.
    Mr. Sanchez. Sure. As was stated, I have been Commissioner 
of Health for about 7 months, and one of the first things that 
we did at TDA when I came on board was to have a discussion 
internally and with external partners about what might be Texas 
Department of Health priorities. The top five priorities for 
the Texas Department of Health are fitness promotion, in other 
words, obesity prevention, improving our immunization rates in 
our State, eliminating health disparities in our State, not 
just racial and ethnic but geographic disparities--we have 
great geographic disparities in our State--enhancing our public 
health preparedness, and then, last, as a State agency whose 
charge is to dispense public funds, look at improving our 
business practices.
    So far as obesity goes, we've got a number of programs. 
CATCH, which you heard about, is one. We feel that focusing at 
least some of our attention on children is very important in 
terms of the continuum and the upstream benefits of addressing 
lifestyle issues early on in life, before they become so 
ingrained as in someone like me, and try to get people early in 
life.
    We have a Texas Diabetes Council in Texas that works in 
collaboration with the Texas Department of Health. It is a 
legislatively created entity. That is entity that is doing 
interventions at the community level and at the practice level 
to try to improve our ability to prevent diabetes and our 
ability to improve the management of diabetes by physicians.
    Those programs are tailored for the community. A program in 
the Lower Rio Grande Valley, where 90 percent of the population 
is Hispanic and a substantial percentage of the population 
speaks Spanish, is going to look very different from a diabetes 
community program based in Dallas, for example.
    With regards to physical activity, the Texas Department of 
Health is now a part of the Governor's Council on Physical 
Fitness, and we are in the very initial stages of defining an 
agenda with regard to physical fitness.
    We also have a Cardiovascular Council that has created a 
State plan that was only published May 2002. We can provide 
that for this committee. That sets out a short-term and long-
term agenda to address cardiovascular disease in our State.
    One thing that I will say is obesity is one of our 
priorities because we feel that obesity prevention, if we were 
to do it properly, we would be preventing cardiovascular 
disease, diabetes, some forms of cancer, arthritis, and the 
list literally goes on and on. Our sense is that we should try 
to promote fitness, and in so doing we would address some of 
these more disease-specific issues, not to say that they aren't 
important, but at the Texas Department of Health we are trying 
to take of an upstream primary prevention approach in 
conjunction with already-existing secondary prevention 
approaches.
    I hope that answered your question.
    Mr. Bilirakis. I thank the gentleman. Mr. Pickering?
    Mr. Pickering. Mr. Chairman, I know our time is running 
short, and I want to submit for the record to Dr. Jones some 
questions for the record, to make sure that we get the full 
benefit as a committee from the very important research that 
you are doing at the University of Mississippi Medical Center 
as it relates to these issues of heart, cardiovascular, 
hypertension, stroke, and what that means for the Stroke Belt, 
what it means for a State like Mississippi, and especially in 
the African American community. We know that those findings can 
be very important not only to Mississippi, but for the rest of 
the country.
    But I would like to ask you a couple of different things. 
You stated in your testimony a very aggressive objective of 
reducing stroke incidences by 25 percent by the year 2010. 
Without the legislation that has passed the Senate unanimously, 
has 175 co-sponsors in the House, introduced by Mrs. Capps and 
myself here in the House, can you meet that objective? If you 
do not have the resources that that legislation calls for, can 
you meet that goal?
    Mr. Jones. No, we can't. As I said in my statement, this 
will take a partnership, and all of us need to work together. 
You have unique resources that we need to raise awareness both 
at the public level and at the professional level about stroke. 
There has been a true revolution in the management of stroke in 
the last few years, but we really must move forward to 
implement that. We can stop the devastating effect of stroke in 
individuals if they can receive modern treatment in an 
appropriate time fashion, but we have a lot to do in public 
awareness, in professional awareness, and in changing access to 
health care to make that happen.
    Mr. Pickering. Let me follow up. One of the key components 
of meeting that objective as well is to have the physicians 
that can treat stroke, In Mississippi we are in a state of 
crisis as it relates to the availability and the number of 
neurosurgeons. Now that has several components, but one of the 
key causes to the flight of neurosurgeons from Mississippi and 
the inability to recruit neurosurgeons to Mississippi is the 
condition that we now face in the tort or medical malpractice 
arena.
    Another piece of legislation that is coming before this 
subcommittee and before this committee and Congress in the 
coming days will be an effort that I co-sponsored, that 
Congressmen Cox and Greenwood and others have sponsored, that 
would limit medical malpractice liability. We are seeing in 
Mississippi 400 doctors leave our State. We are not being able 
to recruit others. We have seen 14 medical malpractice insurers 
go down to one insurance. We are seeing a quadrupling of 
insurance premiums for our small rural hospitals.
    Would you also support that effort, and how critical is 
that to your overall effort to bring good health to Mississippi 
and to make progress on heart and stroke disease?
    Mr. Jones. Thank you for that question and for your 
interest. The word ``crisis'' is overused sometimes, but this 
is a true crisis. It is a crisis in several States now, 5, 6, 
10, depending on how you define it, but it is a crisis that is 
spreading across our country.
    I would like to couch this in terms of what we are talking 
about today for cardiovascular disease management and 
particularly stroke management. Access to care is a critical 
issue. You have heard testimony from several that we have new 
treatments that are effective if they are implemented within 3 
hours. In a rural state like Mississippi, like Texas, where 
people have to go to receive that wonderful new treatment is 
really important. As part of a comprehensive stroke team, 
neurosurgeons are a vital part.
    In Mississippi the Mississippi Neurosurgical Association 
has on the public record said that 30 percent of the 
neurosurgeons have left Mississippi. Now that is beginning with 
a total of 38 neurosurgeons. That is a scarce resource in a 
State that has the seventh highest stroke rate in the country. 
There are parts of Mississippi now where on some evenings and 
on some weekends, if you experience a problem where you need a 
neurosurgeon, you may have to travel 200 or 300 miles to have 
that. If you add up that 3 hours, it is a magic 3 hours for the 
initiation of treatment of stroke.
    Our patients are confused about what they should do, about 
where they should go when they get to hospitals and they don't 
have adequate coverage. It is a real crisis in health care.
    Access to care is an important part of this issue of 
improving the treatment of stroke, and I thank you for your 
interest in that. I plead with you to bring some solution to 
that. If you don't care enough about physicians, and physicians 
are not always easy to love, care about our patients and 
providing access to care for our patients.
    Mr. Pickering. Mr. Chairman, let me just close real quick 
with, in my earlier questions----
    Mr. Bilirakis. We don't want to miss these votes.
    Mr. Pickering. I know. In my earlier questions I did not 
intend to set up any type of competition on research among the 
various priorities that we have. I simply want to find 
consensus on a very important issue that is important to my 
State, and from a personal point of view, having both a 
grandmother die of heart disease and a grandfather who passed 
away after a series of strokes. This affects all of our 
families.
    We just want to get the resources and the priorities and 
objectives to be able to give you the resources you need. Thank 
you for coming here today.
    Mr. Bilirakis. Hopefully, we can do that, and are doing 
that, on a bipartisan basis. This increase of funding, doubling 
the funding for NIH over 5 years, we make an awful lot of 
promises from up here, and many of them we just can't fulfill, 
but that is one we did, and we are very proud of it.
    The hearing now is terminated. I very much appreciate your 
being here. We have learned a lot from you. I know if we had 
more time, we would probably learn even more from you, but 
there will be a series of questions. We would like to hear from 
you. We would like to hear from you on this, particularly the 
one that I had asked. Again, anything, even if it hasn't been a 
question that has been asked, if you feel that you have 
anything to offer that might be helpful in these regards, 
please feel free to submit that information to the committee.
    Thank you very much.
    [Whereupon, at 12:59 p.m., the subcommittee was adjourned.]