[Senate Hearing 111-]
[From the U.S. Government Publishing Office]



 
  DEPARTMENTS OF LABOR, HEALTH AND HUMAN SERVICES, AND EDUCATION, AND 
          RELATED AGENCIES APPROPRIATIONS FOR FISCAL YEAR 2011

                              ----------                              


                         WEDNESDAY, MAY 5, 2010

                                       U.S. Senate,
           Subcommittee of the Committee on Appropriations,
                                                    Washington, DC.
    The subcommittee met at 9:35 a.m., in room SD-124, Dirksen 
Senate Office Building, Hon. Tom Harkin (chairman) presiding.
    Present: Senators Harkin, Pryor, Specter, and Cochran.

                DEPARTMENT OF HEALTH AND HUMAN SERVICES

                     National Institutes of Health

STATEMENT OF FRANCIS S. COLLINS, M.D., Ph.D., DIRECTOR, 
            NATIONAL INSTITUTES OF HEALTH

                OPENING STATEMENT OF SENATOR TOM HARKIN

    Senator Harkin. The Senate Subcommittee on Labor, Health, 
Human Services, and Education, and Related Agencies 
appropriations will come to order.
    I want to start, first, by welcoming Dr. Francis S. 
Collins, who, of course, has appeared before this subcommittee 
many times over the past 20 years. Until now, he always 
testified as the Director of the National Human Genome Research 
Institute (NHGRI), today, wearing a much different and bigger 
hat, as Director of the entire National Institutes of Health 
(NIH).
    The fiscal year 2010 budget for the NHGRI is $516 million. 
The budget for NIH as a whole is $31 billion. Well, at least 
that's where it is right now, anyway; we're looking at that. 
And, of course, the portfolio as NIH Director is much larger 
than the one that Dr. Collins had at the NHGRI.
    But, having known Dr. Collins for all these years, I can't 
tell you how proud I am, and honored, that he is, now, the 
Director of the NIH.
    I can remember when you first took over at the Genome 
Project--I think it was called a ``project'' at that time--
1992? 1993? I knew I was close, Dr. Collins. I was close. And 
to take the project to the complete mapping and sequencing of 
the human genome was a singular accomplishment. And as I said, 
watching you during that whole time, and watching you shepherd 
that thing through, I'm telling you, you're in the right place 
at the right time, right now, as Director of NIH.
    One of the things that--when you think about the issues 
that confront NIH today--what role does biomedical research 
play in healthcare reform? How can we capitalize on the Human 
Genome Project that we completed? How can we do a better job of 
translating basic research in the field? How can we encourage 
some of our brightest young minds to enter this field when 
we've got tight budgets? So, we need someone who thinks big to 
head up NIH, and that's why we have Dr. Collins here, because 
he does think big, and he accomplishes big things.
    So, the President's budget for the NIH for 2011 calls for a 
$1 billion increase more than the 2010 level, a total of $32 
billion; it's about a 3.2 percent increase, which I am told is 
the same as the biomedical inflation rate.
    But, fiscal year 2011 will bring with it a very special set 
of challenges; namely, how to achieve the softest possible 
landing for NIH after the $10.4 billion that was appropriated 
in the American Recovery and Reinvestment Act (ARRA). That is 
one area that I hope to explore with Dr. Collins in our 
question-and-answer period.
    I also want to spend some time discussing one of the 
questions I raised earlier, how we can more effectively 
translate basic science into treatments and practices that 
actually improve people's health.
    I know you've heard me say this many times before, Dr. 
Collins, that there's a reason it's called the National 
Institutes of Health, not the National Institutes of Basic 
Research.
    But, before we hear from Dr. Collins, I would yield to 
Senator Cochran for his opening statement.

                   STATEMENT OF SENATOR THAD COCHRAN

    Senator Cochran. Mr. Chairman, thank you very much for 
conducting this hearing, looking at the budget requests for the 
next fiscal year for the Department; that is, the NIH; 
specifically, under the generalship of Dr. Collins.
    We appreciate very much your fine leadership and good work 
not only as a researcher, but also to manage and help identify 
priorities that help this subcommittee decide how much funding 
we need to place in the different accounts in this bill. It's a 
very large bill. We wish it could be larger, but the budget 
constrains us. But, within that budget framework, we have to 
identify the highest priorities, and your testimony will help 
us do a better job of that. And so, we appreciate your 
assistance to the subcommittee and your leadership in your 
role.
    Thank you.
    Senator Harkin. Thank you, Senator Cochran.
    I didn't read that before I sat down, I just thought 
``turning discovery into health.'' That's one of the things I 
wanted to talk about.
    [The information follows:]

    www.nih.gov/about/discovery

    Senator Harkin. Well, Francis S. Collins, M.D., Ph.D., was 
sworn in as the 16th Director of the NIH in August 2009, after 
being unanimously confirmed by the Senate. A physician-
geneticist noted for his discoveries of diseased genes, his 
leadership of the Human Genome Project. Prior to becoming NIH 
Director, he served as the Director of the NHGRI at NIH. He 
received his B.S. from the University of Virginia, Ph.D. from 
Yale, and an M.D. from the University of North Carolina at 
Chapel Hill.
    Well, Dr. Collins, welcome. You're no stranger to this 
subcommittee. Your statement will be made a part of this record 
in its entirety, and you can please proceed as you so desire.

                SUMMARY STATEMENT OF FRANCIS S. COLLINS

    Dr. Collins. Well, thank you, Senator. And it is a great 
pleasure to be here. Good morning to all of you. It's an honor 
to appear to present the NIH's budget request for fiscal 2010 
and to discuss my vision for the future of biomedical research.
    I'd like for my written testimony to be included in the 
record, and I'm going to deviate from it quite a bit this 
morning in this opening set of remarks.
    First of all, I'd certainly like to thank all of you for 
your steadfast support of NIH's mission: to discover 
fundamental knowledge about the nature and behavior of living 
systems, but then to apply that knowledge to fight illness and 
to reduce the burdens of disability. And this is--of course, we 
are the National Institutes of Health--I think I've quoted you 
on that, actually, Senator Harkin--not the National Institute 
of Basic Science. We are passionate about taking the 
discoveries that are pouring out of research laboratories, and 
moving them quickly toward clinical benefits.
    Over the course of 15 years as Director of the NHGRI, I 
must say I was grateful for this subcommittee's strong support. 
Even at a time, early on, when the scientific community was 
somewhat divided about whether the Genome Project was worth 
investing in, this subcommittee was a strong supporter. And 
you, particularly, Mr. Chairman, were a vocal and articulate 
visionary for what this project might do. And your vision has 
been coming true ever since. And I--I'm personally grateful to 
you for that leadership.
    So, I want to introduce you today, instead of going through 
some specific scientific advances, to some people.
    Let's begin with Kate Robbins. Eight years ago, at the age 
of 44, this nonsmoking mother of two, was diagnosed with lung 
cancer; specifically, non-small-cell lung cancer. It had 
already metastasized to her brain. Normally this would be a 
death sentence. Despite surgery, radiation, chemotherapy, the 
cancer continued its deadly march, moving into her liver, into 
her pancreas. Still, she kept on fighting. And in early 2003, 
she enrolled in a trial of a drug called gefitinib, which is 
trade name Iressa, which is a new genome-based drug for cancer, 
based on a molecular understanding of what has gone wrong in 
certain cases of lung cancer.
    Now, after she started the drug, most of her metastases 
vanished. Look at these CT-scans. This was her original one. In 
2002, all of those dark areas are cancer in her liver. Just 6 
months later, all but one is gone. And today there is no 
evidence of cancer in her liver, at all.
    Now, why doesn't this work in all cases? In her case, a 
miraculous recovery. She's 7\1/2\ years out, with no sign of 
cancer in her liver or her lungs or her pancreas.
    The disappointing news is that this drug only works in 
about one-fifth of lung cancer patients. But, we now know why. 
If your tumor has a specific mutation in a gene called EGFR, 
this drug is for you. If your tumor does not have that 
mutation, this drug probably will not work. So, this 
demonstrates the potential of personalized medicine, which is a 
major frontier right now for cancer, for heart disease, for 
virtually all conditions; that we can individualize treatment 
instead of doing the one-size-fits-all approach.
    Well, next I'd like you to meet 9-year-old Corey Haas. This 
is Corey and his mom and dad. Corey was affected by a disease 
that was robbing him of his vision, a disease called Leber's 
congenital amaurosis, which is quite a mouthful, but it leads 
to progressive vision loss. And by age 7, Corey was legally 
blind. But, he underwent, in an experimental procedure 
supported by NIH at the University of Pennsylvania, a gene-
therapy approach. Basically, the idea here was to take a normal 
copy of RPE65 and inject it, in a viral vector, into the back 
of his eye. And let me show you what happened, in the videos 
that you can see.
    One eye was treated, and then, by patching one eye and 
looking to see how he would do in being able to follow some 
arrows on the floor, you can see what the effects were.
    So, let's start here. Now, at this point, his treated eye 
has been blocked, so you're seeing what he's able to see 
without treatment, trying to follow these little arrows on the 
floor. And he's basically being asked to follow them, he's 
saying, ``I can't see them.'' He's frustrated; he's standing 
there, he really can't see where anything is. They're asking, 
``Do you want a clue?'' He finally says, ``I can't see 
anything.''
    Now, same day, they now patch the untreated eye so he can 
see with the eye that's received the gene therapy. And watch 
what happens. ``Okay, follow those arrows, Corey.'' No 
mistakes. He even had to climb over an obstacle, there, and go 
all the way around. And he decided he was doing so well, he 
wouldn't even stop, he'd just walk outside the door.
    And if we had the audio, you would have heard wild applause 
from the researchers, at that point.
    So, isn't that dramatic? And this has been, in Corey, 
sustained for more than a year, and now the consideration is to 
treat the other eye.
    A third story. This is one that features prevention-
oriented research. Now this is about Leslie Cook. She smoked 
for 25 years, half of her life, a habit that put her at 
increased risk for heart attack, cancer, and many other 
diseases. She's a high-powered real estate lawyer; she tried to 
kick the habit many times. She tried the gum, the patch, you 
name it; nothing worked for her.
    And then she enrolled in a phase II trial of a vaccine 
against nicotine, called NicVAX. The vaccine spurs the immune 
system to generate antibodies against nicotine. Those bind to 
it, preventing it from entering the brain, and therefore no 
pleasure response occurs after smoking. NicVAX did the trick 
for Leslie; she has not smoked in 3\1/2\ years.
    And there is now a phase III trial underway here, supported 
by the ARRA, to test this in 1,000 smokers at 20 centers. It's 
the first-ever phase III trial of a smoking cessation vaccine.
    So, thanks to the discoveries you have funded----
    Senator Harkin. Working on a broad basis? Now, this is not 
personalized, it doesn't depend on a certain gene, or----
    Dr. Collins. No. In this case, the vaccine is actually 
raised against the nicotine itself, so the antibodies are 
against the material in the cigarette smoke that gives people a 
high, and it blocks that effect, and so there's no point in 
smoking and they have an easier time quitting. It's pretty 
dramatic. That has not, I think, previously been tried for this 
purpose.
    So, we're mixing immunology and drug addiction in 
interesting ways. There are efforts underway to do this, also, 
for other drugs of addiction.
    Well, let me quickly conclude, here, by just quickly 
pointing out to you that these represent just a few of the 
exciting areas of opportunity. When I first came to this job--
and it is an incredible responsibility, of leading the NIH--I 
scanned the landscape a bit, of biomedical research, to 
identify areas that seemed ripe for major advances and, in the 
process of doing so, identified five themes that I thought were 
particularly ripe for investment. And you have in front of you 
this publication from Science, published in January, that goes 
through a description of those five themes, and I think that's 
been reasonably well received by the scientific community.
    One of them is to use the high-throughput technologies that 
have been invented in the last few years--genomics, 
nanotechnology, imaging, computational biology--to really 
tackle questions in a comprehensive way; questions like the 
causes of cancer or autism or what role microbes play in 
disease when we can't actually culture them in the laboratory 
but we can detect their presence by DNA analysis.
    A second opportunity, and one that you've mentioned 
already, Mr. Chairman, the importance of translating the basic 
science discoveries into new and better treatments, of building 
a bridge, as you see done here for San Francisco, but a bridge 
between basic research and drugs and empowering academic 
investigators to play a larger role in that. And the Cures 
Acceleration Network (CAN), which is part of the healthcare 
reform bill, is an important aspect of this that we're very 
excited about.
    I should also say, stem cells fit into here, and I'm happy 
to tell you there are now 64 human embryonic stem cell lines 
that are on the NIH registry and approved for Federal funding, 
followed up on Obama's Executive order from a year ago.
    A third area, represented by these banners here, is to 
reach out with NIH research results and actually have an effect 
on our healthcare system. And that means personalized medicine 
research, health disparities research, comparative 
effectiveness research, behavioral research, and even 
healthcare economics. We're having a major meeting on that next 
week.
    A fourth area is to recognize that we have both 
opportunities and perhaps responsibilities to apply our medical 
research efforts to those in less fortunate parts of the world, 
and that means a focus on AIDS, tuberculosis, and malaria, but, 
going beyond that, to neglected tropical diseases and 
noncommunicable disorders, which are the most rapidly growing 
cause of morbidity and mortality in the developing world.
    And finally, the reinvigoration and empowerment of the 
research community, which is a challenge, especially at times 
of stressed budgets, to be sure that we're encouraging young 
investigators, that we're encouraging innovation, that we're 
training the next generation, using the Ruth Kirschstein 
awards. And I should, for a moment here, just say how much we 
miss Dr. Kirschstein, such a remarkable leader of NIH. We're 
having a special symposium in her honor, later this month, 
bringing back many of the people who were supported by those 
Kirschstein awards, in recognition of the role she's played in 
so much of what we've done in training.
    Also in front of you is this pamphlet. And let me just 
conclude by saying, if our Nation can be bold enough to act 
upon these many unprecedented opportunities, we'll be amazed at 
what tomorrow will bring, and how swiftly we can turn discovery 
into health, as this title says. The one-size-fits-all approach 
to medicine will be a thing of the past; we will be using 
genetic information to personalize our healthcare.
    But, if you'll allow me, I see a future in which we will 
use stem cells to repair spinal cord injuries. We'll 
bioengineer bones and cartilage to replace wornout joints. 
We'll use nanotechnology to deliver therapies with exquisite 
precision. We'll pre-empt heart disease with minimally invasive 
image-guided procedures, and use an artificial pancreas or 
other new technologies to manage diabetes better.
    I look forward to a universal vaccine for influenza, so 
that you don't have to get a shot every year for the new 
strain. I look forward to the possibility, more possible now 
than ever, of an AIDS vaccine and a malaria vaccine. And I 
dream of a day when we'll be able to prevent Alzheimer's 
disease, Parkinson's disease, and many others that rob us, too 
soon, of family and friends.

                           PREPARED STATEMENT

    As you've heard, the fiscal year 2011 request from this 
subcommittee is $32.157 billion, an increase of $1 billion. 
These funds will enable the biomedical research community to 
pursue a number of substantial opportunities in these major 
scientific and health opportunity areas.
    So, I'm really grateful for the chance to be here this 
morning. I'm pleased to respond to any questions that you might 
have.
    Thank you very much.
    [The statement follows:]

                Prepared Statement of Francis S. Collins

    Good morning, Mr. Chairman and distinguished members of the 
subcommittee: It is a great honor to appear before you today to present 
the fiscal year 2011 budget request for the National Institutes of 
Health (NIH), and to discuss my vision for the future of biomedical 
research.
    First, I'd like to thank each of you for your steadfast support of 
NIH's mission: discovering fundamental knowledge about living systems 
and then applying that knowledge to fight illness, reduce disability, 
and extend healthy life. In particular, I want to thank the 
subcommittee for the fiscal year 2010 budget level of $31 billion, and 
the $10.4 billion provided to NIH through the American Recovery and 
Reinvestment Act. I was very grateful for the subcommittee's interest 
and support over the course of my 15 years as Director of the National 
Human Genome Research Institute, most notably during our successful 
effort to sequence the human genome. Now, as steward of NIH's entire 
research portfolio, I truly believe that the opportunities for us to 
work together to improve America's health have never been greater.
    One of my first actions upon being named NIH Director was to scan 
the vast landscape of biomedical research for areas ripe for major 
advances that could yield substantial benefits downstream. I found many 
of the most exciting opportunities could be grouped under five main 
themes: taking greater advantage of high-throughput technologies; 
accelerating translational science, that is, turning discovery into 
health; helping to reinvent healthcare; focusing more on global health; 
and reinvigorating the biomedical research community.
    The administration's request of $32.1 billion for NIH's biomedical 
research efforts in fiscal year 2011 would help more researchers take 
greater advantage of these unprecedented opportunities, all with the 
aim of helping people live longer, healthier, more rewarding lives. We 
at NIH are fortunate to have a very solid foundation upon
    which to build, established by such extraordinary leaders as James 
Shannon, Nobel laureate Harold Varmus, Elias Zerhouni, and the late and 
much missed Ruth Kirschstein.

                         THE RESEARCH MARATHON

    In his fiscal year 2009 budget remarks, Dr. Zerhouni warned that 
our Nation's biomedical research effort is in a race that we cannot 
afford to lose. I wholeheartedly agree, and want to provide a few more 
insights about what that race involves.
    Science is not a 100-yard dash. It is a marathon--a marathon run by 
a relay team that includes researchers, patients, industry experts, 
lawmakers, and the public.
    Thanks to discoveries funded through NIH appropriations, we have 
covered a lot of ground in this marathon. Let us take a moment to look 
back at a few of the advances made possible by NIH-supported research, 
and then look ahead to some of our Nation's biggest health challenges 
and how NIH intends to meet them.

                           HOW FAR WE'VE COME

    U.S. life expectancy has increased dramatically over the past 
century and still continues to improve, gaining about 1 year of 
longevity every 6 years since 1990. A baby born today can look forward 
to an average life span of 77.7 years, almost three decades longer than 
a baby born in 1900.
    Not only are people living longer, they are staying active longer. 
From 1982 through 2005, the proportion of older people with chronic 
disabilities dropped by almost one-third, from 27 percent to 19 
percent.
    Some of the most impressive gains have been made in the area of 
cardiovascular disease. In the mid-20th century, cardiovascular disease 
caused half of U.S. deaths, claiming the lives of many people still in 
their 50s or 60s. Today, the death rate for coronary heart disease is 
more than 60 percent lower--and the death rate for stroke, 70 percent 
lower--than in the World War II era.
    What fueled these improvements? One major contributor has been the 
insights from the NIH-funded Framingham Heart Study, which began in the 
late 1940s and is still going strong. This population-based study, 
which changed the course of public health by defining the concept of 
disease risk factors, continues to break new ground with its recent 
move to add a genetic component to its analyses.
    Other factors include NIH-supported research that led to minimally 
invasive techniques to prevent heart attacks and to highly effective 
drugs to lower cholesterol, control high blood pressure, and break up 
artery-clogging blood clots. Science also played a crucial role in 
formulating approaches to help people make lifestyle changes that 
promote cardiovascular health, such as eating less fat, exercising 
more, and quitting smoking.
    Many chronic conditions have their roots in the aging process. One 
such disease, osteoporosis, can lead to life-threatening bone fractures 
among older people. NIH-funded research has led to new medications and 
management strategies for osteoporosis that have reduced the 
hospitalization rate for hip fractures by 16 percent since 1993. 
Science has also transformed the outlook for people with age-related 
macular degeneration, a major cause of vision loss among the elderly. 
Twenty years ago, little could be done to prevent or treat this 
disorder. Today, because of new treatments and procedures based on NIH 
research, 750,000 people who would have gone blind over the next 5 
years will continue to have useful vision.
    Biomedical research also has benefited those at the other end of 
the age spectrum. NIH-funded research has given hearing to thousands of 
children who were born profoundly deaf. This hearing is made possible 
through a cochlear implant, an electronic device that mimics the 
function of cells in the inner ear. Since the Food and Drug 
Administration (FDA) approved cochlear implants for pediatric use in 
2000, more than 25,000 children have received the devices, enabling 
many to develop normal language skills and succeed in mainstream 
classrooms.
    Then, there are the infectious diseases--diseases that often know 
no boundaries when it comes to age, sex, or physical fitness. One of 
NIH's greatest achievements over the past 30 years has been to lead the 
global research effort against the human immunodeficiency virus (HIV)/
acquired immunodeficiency syndrome (AIDS) pandemic. With discovery 
building upon discovery, researchers first gained fundamental insights 
about how HIV works, and then went on to develop rapid HIV tests, 
identify a new class of HIV-fighting drugs, and, finally, figure out 
how to combine those drugs in life-saving ways in the clinic. As a 
result, HIV infection has changed from a virtual death sentence into a 
manageable, chronic disease. Today, HIV-infected people in their 20s 
who receive combination therapy may expect to live to age 70 or beyond.

                         HOW FAR WE HAVE TO GO

    Although we have accomplished much, and as tempting as it may be 
for NIH to rest upon its laurels, we all know that biomedical research 
still has an enormous amount of ground to cover before discovery is 
turned into health for all Americans.
    Consider the challenge posed by cancer. This disease still claims 
the lives of more than 500,000 Americans annually--about one every 
minute. But in 2007, for the first time in our Nation's history, the 
absolute number of cancer deaths in the United States went down. And, 
over the past 15 years, cancer death rates have dropped 11.4 percent 
among women and 19.2 percent among men, which translates into some 
650,000 lives saved--more than the population of Washington, DC. These 
are very encouraging milestones, but they are not nearly enough.
    NIH-funded research has revolutionized how we think about cancer. A 
decade or two ago, cancer treatment was mostly reactive, diagnosis was 
based on the organ involved and treatment depended on broadly aimed 
therapies that often greatly diminished a patient's quality of life. 
Today, basic research in cancer biology is moving treatment toward more 
effective and less toxic therapies tailored to the genetic profile of 
each patient's cancer.
    Among the early success stories in this area is the drug 
trastuzumab (Herceptin) for breast cancer. An NIH-sponsored clinical 
trial found that when breast cancer patients whose tumors were 
genetically matched to trastuzumab received the drug, along with 
standard chemotherapy, their risk of cancer recurrence fell 40 percent. 
That improvement is the best ever reported in postsurgical treatment of 
breast cancer. Studies also have found that the chemotherapy drugs 
gefitinib (Iressa) and erlotinib (Tarceva) work much better in the 
subset of lung cancer patients whose tumors have a certain genetic 
change.
    To accelerate the development of more individualized strategies for 
more types of cancer, NIH has tapped into the promise of high-
throughput technologies to launch The Cancer Genome Atlas (TCGA). Over 
the next few years, TCGA's research team will build comprehensive maps 
of the key genomic changes in 20 major types and subtypes of cancer. 
This information, which is being made rapidly available to the 
worldwide scientific community, will provide a powerful new tool for 
all those striving to develop better ways to diagnose, treat, and 
prevent cancer.
    Already, TCGA has produced a comprehensive molecular classification 
system for ovarian cancer and glioblastoma, the most common form of 
brain cancer. The survey of glioblastoma recently revealed five new 
molecular subtypes of the disease. In addition, researchers found that 
responses to aggressive therapies for glioblastoma varied by subtype. 
The findings hold promise for matching the most appropriate therapies 
with brain cancer patients and may also lead to therapies directed at 
the molecular changes underlying each subtype, as has already happened 
for some types of breast cancer.
    Diabetes is another disease that is inflicting much damage on U.S. 
health. More than 23 million Americans currently have diabetes--nearly 
8 percent of the population. Another 57 million have blood sugar levels 
that indicate they are at serious risk of developing the disease, which 
is a major cause of kidney failure, stroke, heart disease, lower-limb 
amputations, and blindness.
    For type 2 diabetes, prevention appears to be the name of the game. 
This form of the disease, which accounts for more than 90 percent of 
diabetes among adults, often can be averted or delayed by lifestyle 
factors. The NIH-funded Diabetes Prevention Program (DPP) trial showed 
that one the most effective ways to lower the risk of type 2 diabetes 
is through regular exercise and modest weight loss. There is good 
reason to believe that such efforts may lead to a lifetime of health 
benefits. A recent follow-up study of DPP participants found the 
protective effects of weight loss and exercise persist for at least a 
decade. The United Health Group has recently announced a partnership 
with Walgreen's and the YMCA to implement the results of this 
groundbreaking NIH-funded research on a broad scale.
    More than one-third of adults in the United States are obese, 
according to the latest data from the National Health and Nutrition 
Examination Survey which is conducted by the Centers for Disease 
Control and Prevention (CDC). And there are signs that the next 
generation may face an even greater struggle. Over the past 30 years, 
obesity has more than doubled among U.S. children ages 2 through 5 and 
nearly tripled among young people over the age of 6. Those statistics 
translate into tens of millions of Americans who face an increased risk 
of type 2 diabetes, as well as cardiovascular disease, high blood 
pressure, certain cancers, osteoarthritis, and other serious health 
problems associated with excess body fat.
    To address America's growing problem with obesity, NIH has launched 
a variety of initiatives aimed at developing innovative approaches for 
weight control. One such effort, called the National Collaborative on 
Childhood Obesity Research, has pulled together experts from four NIH 
Institutes, the CDC, and the Robert Wood Johnson Foundation. One 
example of their work is the Trial of Activity for Adolescent Girls, a 
national study to develop and test school- and community-based 
interventions to get girls more involved in gym class, organized 
sports, or recreational activities. Another NIH program, called We 
Can!, provides families with practical tools for weight control at more 
than 1,000 community sites nationwide. How to get more people to lose 
weight is also among the questions being explored by OppNet, a new 
trans-NIH initiative for basic behavioral and social sciences research.
    Meanwhile, other NIH-funded researchers are busy uncovering 
information about genes and environment that may pave the way for more 
personalized, targeted strategies for controlling weight and preventing 
diabetes. For example, in just the past few years, we have identified 
more than 30 genetic risk factors for type 2 diabetes.
    A better understanding of genetic and environmental factors may 
also help solve a longstanding medical puzzle: the causes of autism. 
Children with autism spectrum disorders experience a range of problems 
with language and social interactions, sometimes accompanied by 
repetitive behaviors or narrow, obsessive interests. Recent studies 
funded by NIH have associated autism risk with several genes involved 
in the formation and maintenance of brain cells, but much more work is 
needed to follow up on these clues.
    In fiscal year 2011, NIH will support comprehensive and innovative 
approaches to piece together the complex factors that contribute to 
autism spectrum disorders. One ambitious effort will involve sequencing 
the complete genomes of 300 people with autism and their parents. Other 
researchers will examine a mother's exposure during pregnancy to 
identify possible environmental contributions. NIH hopes to use these 
insights to develop new molecular and behavioral therapies for such 
disorders, as well as to identify possible strategies for prevention.
    Another brain disorder, depression, presents a different set of 
challenges. Although researchers have made significant progress in 
understanding the biology of depression, improving treatment, and 
lessening the social stigma associated with mental illnesses, suicide 
still claims the lives of twice as many Americans as homicide. And it 
does not end there--untreated depression also increases the risk of 
heart disease and substance abuse.
    How can medical research reduce depression's tragic toll? One way 
may be getting people into treatment more quickly. Researchers today 
are using functional magnetic resonance imaging and other innovative 
technologies to see how the brains of people with depression differ 
from those without the disorder. Rapid diagnosis is just part of the 
equation. Finding the right antidepressant drug for any particular 
patient currently is a lengthy, trial-and-error process that can take 
weeks before symptoms are relieved. NIH supports laboratory research 
aimed at developing quicker-acting antidepressants, as well as genetic 
studies that will help to match individuals with the drugs most likely 
to work for them.
    In 2008, 143 soldiers died by suicide--the highest rate since the 
Army began keeping records three decades ago. To address this problem, 
NIH and the U.S. Army recently partnered to launch the largest study 
ever of suicide and mental health among military personnel. The Army 
Study to Assess Risk and Resilience in Service Members will identify 
risk factors that may inform efforts to develop more effective 
approaches to suicide prevention.

                   TRANSFORMING DISCOVERY INTO HEALTH

    Whatever the disease, be it depression, diabetes, or something much 
rarer, NIH's emphasis in fiscal year 2011 and beyond will be on 
translating basic discoveries into new diagnostic and treatment 
advances in the clinic.
    In the past, some have complained that NIH has been too slow to 
convert fundamental observations into better ways to diagnose, treat, 
and prevent disease. Although some of that criticism may have been 
deserved, most of the delay has stemmed from the lack of good ideas 
about how to traverse the long and winding road from molecular insight 
to therapeutic benefit.
    That is now changing. For many disorders, there are new 
opportunities for NIH to shorten and straighten the pathway from 
discovery to health. This expectation is grounded in several recent 
developments: the dramatic acceleration of our basic understanding of 
hundreds of diseases; the establishment of NIH-supported centers that 
enable academic researchers to use such understanding to screen 
thousands of chemicals for potential drug candidates; and the emergence 
of public-private partnerships to aid the movement of drug candidates 
identified by academic researchers into the commercial development 
pipeline.
    Let me give you one example of how NIH plans to implement this 
strategy: the Therapeutics for Rare and Neglected Diseases (TRND) 
program. This effort will bridge the wide gap in time and resources 
that often exists between basic research discoveries and the human 
testing of new drugs.
    A rare disease is one that affects fewer than 200,000 Americans. 
However, if all 6,800 rare diseases are considered together, they 
afflict more than 25 million Americans. Private companies seldom pursue 
new therapies for these types of diseases because of the high cost of 
research and low likelihood of recovering their investments. Effective 
drugs exist for only about 200, or less than 3 percent, of rare 
diseases. Unlike rare diseases, neglected diseases may be quite common 
in some parts of the world, especially in developing countries. 
However, there also is a dire shortage of effective, affordable 
treatments for many of these major causes of death and disability.
    Working in an open environment in which all of the world's top 
experts on a disease can be involved, TRND will enable certain 
promising compounds to be taken through the preclinical development 
phase--a time-consuming, high-risk phase often referred to as ``the 
valley of death'' by pharmaceutical firms focused on the bottom line. 
Besides speeding development of drugs for rare and neglected diseases, 
TRND will serve as a model for therapeutic development for common 
diseases, many of which are being resolved into smaller, molecularly 
distinct subtypes.
    NIH will also take other steps to build a more integrated pipeline 
that connects all of the steps between identification of a potential 
therapeutic target by a basic researcher and the point when the FDA 
approves a therapeutic for clinical use. Among the tools at our 
disposal is the NIH Clinical and Translational Sciences Award program, 
which currently funds 46 centers and has awardees in 26 States and 
plans to add even more in fiscal year 2011. This national network is 
pulling together interdisciplinary clinical research teams to work in 
unprecedented ways to develop and deliver tangible health benefits. We 
also need to take advantage of the Nation's largest research hospital, 
the Mark O. Hatfield Clinical Research Center, located on the NIH 
campus in Bethesda, Maryland. Just as they blazed a trail for safe and 
effective human gene therapy, NIH clinical researchers may be well-
positioned to move the ball forward for other pioneering approaches, 
such as those using human embryonic stem cells or induced pluripotent 
stem cells derived from skin cells.
    To make the most of these new opportunities, the NIH and FDA 
recently forged a landmark partnership with the formation of a Joint 
Leadership Council. Members of this Leadership Council will work 
together to ensure that regulatory considerations form an integral 
component of biomedical research planning, and that the latest science 
is integrated into the regulatory review process. Such collaboration 
will advance the development of products to treat, diagnose and prevent 
disease, as well as enhance the safety, quality, and efficiency of 
clinical research and medical product approval.

                BIOMEDICAL RESEARCH PROPELS U.S. ECONOMY

    It is crucial to keep in mind that investing in NIH not only 
improves America's health and strengthens our Nation's biomedical 
research potential, it empowers the entire U.S. economy. Consider the 
following statistics:
  --A report issued by Families USA calculated that in 2007, every $1 
        in NIH funding resulted in an additional $2.11 in economic 
        output in the United States.
  --In fiscal year 2007, a typical NIH grant supported the salaries of 
        about 7 high-tech jobs in full or in part.
  --The 351,000 jobs resulting from NIH awards paid an average annual 
        wage of more than $52,000 per annum and account for more than 
        $18 billion in wages for fiscal year 2007.
  --Long-term, NIH-funded R&D sparks U.S. economic innovation in the 
        high-technology and high value-added pharmaceutical and 
        biotechnology industries. For example, between 1982 and 2006, 
        one-third of all drugs and nearly 60 percent of promising new 
        molecular entities approved by the FDA cited either an NIH-
        funded publication or an NIH patent.
  --Gains in average U.S. life expectancy from 1970-2000 were worth an 
        estimated $95 trillion.

                           IMAGINE THE FUTURE

    If our Nation is bold enough to act today upon the many 
unprecedented opportunities now offered by biomedical research, we may 
be amazed at what tomorrow will bring.
    In the world I envision just a few decades from now, we will use 
stem cells to repair spinal cord injuries; bioengineered tissues to 
replace worn-out joints; genetic information to tailor health outcomes 
with individualized prescriptions; and nanotechnology to deliver 
therapies with exquisite precision. I also dream of a day when, in ways 
yet to be discovered, we will be able to prevent Alzheimer's, 
Parkinson's, and other diseases that rob us much too soon of family and 
friends.
    Just imagine what such a future would mean for our Nation and all 
humankind. This is what keeps NIH in the research marathon, and why we 
ask you to go the distance with us.
    Thank you Mr. Chairman.

    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
    
                         NICVAX SMOKING VACCINE

    Senator Harkin. Well, Dr. Collins, thank you very much.
    I asked my staff to get me some more information on that 
smoking vaccine. It's just something I had not heard about. 
That could be phenomenal.
    [The information follows:]
                            Smoking Vaccine
    Tobacco remains the leading cause of preventable death in the 
United States, linked to more than 400,000 deaths each year. That is 
why the National Institutes of Health is accelerating research to 
eradicate tobacco addiction, including working with a private partner, 
Nabi Biopharmaceuticals, via a $10 million grant from the National 
Institute on Drug Abuse, to achieve that goal.
    American Recovery and Reinvestment Act (ARRA) funding released in 
September will help pay for the first phase III trial of NicVAX, a 
smoking cessation vaccine designed to help people quit and remain 
abstinent. It was given fast track designation by the Food and Drug 
Administration and has already successfully completed a proof-of-
concept trial; successful completion of the phase III study will bring 
the vaccine closer to final approval.
    As a result of ARRA funding, Nabi entered an agreement with 
GlaxoSmithKline to receive an additional $40 million to exclusively in-
license NicVAX on a worldwide basis and develop follow-on, next-
generation nicotine vaccines, with the possibility of additional $500 
million depending on the outcome of the trial. This work is an 
excellent example of leveraging Government resources to further develop 
and market a medication for tobacco addiction.
    Similar to vaccines for infectious diseases, NicVAX works by 
stimulating the immune system to produce antibodies; in this case, 
however, to the drug nicotine. Nicotine (a small molecule) normally 
travels quickly through the lungs into the bloodstream and then to the 
brain. However, when nicotine molecules are bound to antibodies, they 
become too large to enter the brain, thus subverting the behavioral 
effects of the drug. Results to date show that smokers who achieved 
high antibody levels had higher rates of quitting and longer stretches 
of abstinence than those given placebo (18 percent vs. 6 percent 
complete abstinence after 52 weeks). The vaccine was also well 
tolerated, with few side effects.
    NicVAX's unique immunological mechanism of action elicits anti-
nicotine antibodies lasting for several months--a potential benefit 
over current therapies. Early results showed that it reduced craving 
and withdrawal symptoms, which often prompt relapse. This should 
improve smokers chances to end the addiction/relapse cycle that plagues 
the great majority of those trying to quit.
    A successful phase II proof-of-concept trial was completed in late 
2007, in which NicVAX showed significant improvement in smoking 
cessation rates and continuous long-term smoking abstinence compared to 
placebo, in those who achieved high antibody levels. For the phase III 
trial, modifications were made to the original protocol to improve the 
likelihood of success. An additional vaccination was added and the 
timing of the quit attempt was modified to coincide with the optimal 
level of antibody response. Twenty-two investigative sites have been 
selected, and include highly experienced academic-based smoking 
cessation centers and experienced nonacademic sites. The study will 
enroll 1,000 subjects who want to quit smoking. They will be randomized 
to 1 of 2 treatment groups: (1) placebo control or (2) active vaccine 
treatment.
    Participants will be followed for 1 year from the start of 
immunization. The study's main goal is to determine the percentage of 
those who are abstinent during the final 16 weeks of the study (weeks 
37-52). Other endpoints include safety, withdrawal symptoms, craving, 
cigarette consumption, evaluation of the smoking experience, short-term 
cessation rates after each injection, and assessment of abstinence.
    Recruitment for the phase III trial is on target and the study is 
going well. Final data are expected within 2 years of study start, 
which was in November 2009.

    Dr. Collins. Yes, indeed.
    Senator Harkin. I mean, from prevention we know what 
smoking leads to, and all the diseases it leads to, and the 
cost to society. And most people I meet that have been on 
smoking want to stop, but they just have a tough time.
    Dr. Collins. They do, indeed.
    Senator Harkin. So, this could be remarkable. Do you know 
when--how--that trial is ongoing right now?
    Dr. Collins. It's ongoing, reasonably recently started. I 
can find out for you the expected end date of the trial, but 
they're certainly pushing this forward with all due speed.
    [The information follows:]

    To find the recent clinical trials go to: http://www.cancer.gov/
clinicaltrials/lung-cancer-updates.

    Senator Harkin. Now, let me ask you this, Doctor----
    Well, let's start a 6-minute round? Is that what we have, 
here? Who's operating my clock? There we go. Okay, fine.
    Dr. Collins, I noticed, on the funding, here, for next 
year, how some Institutes go up by 3.2 percent, some by 2.5 
percent, some by 2.8 percent, some by--and they're all over the 
place. I assume they are some of these differences accounted 
for by focusing on those thematic areas that you just 
mentioned, those five theme areas? Is that what is driving that 
now?
    Dr. Collins. That's exactly right.
    Senator Harkin. What----
    Dr. Collins. Those five themes seem to be areas of 
exceptional opportunity. When we looked at the investments of 
the various Institutes in those areas a couple of years ago--
which is not a perfect, but a somewhat good predictor of what 
might be possible in fiscal year 2011--it was clear that those 
opportunities are not entirely evenly distributed. And so, 
recognizing that that $1 billion, although it's only going to 
keep up with inflation, still ought to be invested in 
innovative ways, we attempted to do some arranging of the 
budget to reflect that, and that's what you see in those 
differences between Institutes. They're modest, but they are 
important, I think, to point out, that we're not just doing 
everything in lockstep.
    Senator Harkin. Well, one has to always be careful when 
you're dealing in percentages.
    Dr. Collins. Yes.
    Senator Harkin. As I've often pointed out, zero-to-one is 
an infinite increase. So, sometimes those that get very little 
funding, to get them up a little bit, looks like it's a huge 
percentage increase. So, I always want to be careful and look 
at the percentage increases there.
    Dr. Collins. Point taken.
    Senator Harkin. Well, for instance, the Library of Medicine 
has 4 percent. Well, but it's so small, line of increase 
amounts for that. So, I always like to look at that very 
carefully.
    Dr. Collins. You're quite right, Senator.

                     FISCAL YEAR 2010 AND POST-ARRA

    Senator Harkin. The other one I wanted to get into, here, 
with you is on the funding cliff. So, we put the money in the 
ARRA. At the time, it was decided that we'd put that in, it was 
a 2-year slug of money for at least the following reasons: one, 
because we didn't want researchers being laid off; we wanted to 
keep people employed. A lot of researchers were in the middle 
of projects and studies that we did not want to interrupt. But, 
we knew that we were probably going to face this, 2 years from 
now. So, I guess my question is, What kind of challenges are 
you facing? How do you provide for this soft landing? Are we 
facing any interruptions at all--in terms of some science 
that's being done right now because of this cliff?
    Dr. Collins. So, Senator, this is the question that keeps 
me up at night. On the screen there, you'll see what the total 
funding for NIH has been over the last 10 years, and those red 
bars there are the dollars that came from the ARRA, which we 
are deeply grateful for, and which provided a real shot in the 
arm for some exciting, innovative research that, otherwise, 
would have had to wait a long time to get started; things like 
the Cancer Genome Atlas, for instance, which really was able to 
move forward at an unprecedented pace because of the 
availability of those funds.
    But, as you can see, the difference between fiscal year 
2010, total, when you include the $5.2 billion of ARRA dollars, 
compared to the President's budget for fiscal year 2011 is 
certainly a drop, and that's the cliff that everybody talks 
about, right there, about $4 billion.
    Senator Harkin. Right.
    Dr. Collins. We have done what we can, in anticipation that 
this might be a really challenging year, to try to be sure that 
the ARRA dollars were invested, as much as possible, in short-
term needs. So, for example, $1 billion of this has gone to 
construction in the extramural community. Additional dollars 
have gone to equipment needs, things that were one-time 
requirements. And some dollars have gone to projects that we 
thought we could get done in 2 years, although that's a very 
short cycle time for a scientific project.
    But, we also felt that this was an opportunity to stimulate 
some real innovations and to get people to put forward some 
out-of-the-box ideas; and they did, in huge numbers. The 
Challenge Grants, for example, we thought we might get 4,000 
applications; we got 20,000. There was a great pent-up need 
here for support for new ideas. And many of those are, in fact, 
funded and will have, now, the question in their minds, ``What 
do we do after the 2 years is expended?''
    One thing we are doing is to encourage those who believe 
that they can't quite finish their project and they haven't 
quite spent all the money in 2 years, to ask for a no-cost 
extension, and we will consider those quite seriously. And if 
it seems reasonable, and they're making reasonable progress, we 
will grant that, so at least to stretch out this cliff a little 
bit.
    But, there's no question that the consequences of this 
situation are going to be significant. We currently estimate 
success rates for NIH grantees--which have been in the 25 to 35 
percent level for most of the last 30 years, and are now at 21 
percent, are going to drop further in fiscal year 2011, at this 
budget level, probably to about 15 percent. That's one chance 
out of seven that a given grant would get supported. And 
there's no question that is going to be stressful for all of 
us.
    Senator Harkin. That's not good.
    Well, we've been wrestling with this, ourselves. I am of 
the opinion that we need to do more at NIH. The question is, 
Where do we get the funding and--with all of the other things 
that the Appropriations Committee has to do, and with budget 
constraints? But, we'll see what we can do.
    I want to get one question--well, I'm down to zero. I'll 
ask the question after Senator Cochran gets through with his.
    Senator Cochran.

                       DISCOVERIES ON THE HORIZON

    Senator Cochran. Mr. Chairman, thank you very much.
    Dr. Collins, thank you again for being here and helping us 
review the budget request and pointing out your views of how we 
should identify the priorities and the most important ways we 
can use the funds available to this subcommittee.
    We know that you're a research scientist, and you've been 
rewarded with a lot of recognition, medals, and honors, because 
of the outstanding research you have done, and it reminds me of 
Dr. Arthur Guyton's success as a researcher at the University 
of Mississippi Medical Center. The University continues to 
perform research there. And although he's no longer with us, he 
had a fascinating and very influential impact on heart disease 
and its understanding and therapies to help people live longer 
and have better lives.
    Is there anything going on in the research field right now 
that rivals the work you, personally, did and were praised so 
highly for, and Dr. Arthur Guyton, as well? Do we have any, 
really, blockbuster researchers out there that you've 
identified in helping us provide funding for?
    Dr. Collins. Well, yes, I'm happy to tell you, there is an 
amazing cadre of creative, innovative, productive scientists 
now involved in biomedical research. I certainly agree that Dr. 
Guyton was a legendary character. I studied his book when I was 
in medical school; that's how I learned a lot about physiology 
and about the heart.
    And when you look around today--well, you could count Nobel 
Prizes, I suppose. NIH has been the source of support for no 
less than 131 Nobel Prizes over the last few decades. And, in 
fact, this past fall, when the Nobel Prizes were given out, 
both for medicine and for chemistry, of the six awardees, five 
of them were our grantees. Remarkable people, people like Liz 
Blackburn and Carol Greider, who were awarded the prize for 
discovering telomeres and the enzyme that maintains those ends 
of the chromosomes, so they don't get ratty, like your 
shoelaces, if you didn't have some way to protect those ends. 
Remarkable stories, all of those.
    Many of them coming from a direction you couldn't have 
predicted, but one of the wonders of the way NIH has been able 
to support research is that we base our decisions, many of 
them, on what comes across to us by investigators with ideas 
that go through the most rigorous peer-review system in the 
world, and then are given the funds to chase after those ideas.
    A new program that we're investing in, called the Pioneer 
Awards, is particularly trying to identify those very creative 
individuals who we could unleash to follow their ideas, and not 
have them quite so constrained by the systems that sometimes 
are in place, that--we need to track research, but there are 
times where you want to let somebody just go for it. And we're 
determined to use those kinds of mechanisms and things like New 
Innovators to make that happen.
    In that--particular areas that NIH is supporting, I will 
mention cancer, because I think we are, actually, at a 
remarkable moment, in terms of being able to understand, at 
that most detailed DNA level, what goes wrong in a cancer cell; 
not just some of the things, but all of the things that go 
wrong in a cancer cell. Why does a good cell go bad? And what 
could we use as--with that information, to develop therapies 
that are targeted--like Kate Robbins, the case I told you 
about--specifically toward their tumor? That was a pipedream 5 
or 6 years ago. Now it is absolutely transforming people's 
ideas of how to go forward. And the researchers working on 
that--many of them 20-somethings, many of them with 
computational backgrounds, because a lot of the challenge now 
is to figure out how to analyze the mountains of data that can 
be produced. They are remarkable to hang out with.
    So, I'm actually quite inspired by our cohort of 
researchers. My concern is, we need to be sure we're giving 
them the confidence that that support is going to be there, so 
that they stick it out and are willing to take risks and not 
just do the obvious next steps.

                          JACKSON HEART STUDY

    Senator Cochran. One of the undertakings in our State is 
the Jackson Heart Study, which has been a comprehensive review 
of the individual medical histories of people who have heart 
problems, and seeing if we can identify factors that can be 
changed or corrected to help us do a better job of providing 
opportunities for healthy lives, rather than a destiny that is 
more likely to involve heart problems. What is the status of 
that study? And are you requesting funding, in this budget 
request, to continue or go forward from that study to something 
else?
    Dr. Collins. We are very enthusiastic about that study, 
Senator, and delighted by your strong support of this from the 
beginning. So, this is carried out in Mississippi, in Jackson, 
with the University of Mississippi and Tougaloo College 
participating. NIH has a big role in this, supported by the 
National Heart, Lung, and Blood Institute (NHLBI). And already, 
a lot of very important observations have come forward 
studying, particularly, cardiovascular disease in African 
Americans, about which we didn't know enough, and now we're 
starting to learn.
    So, for instance, we're learning that hypertension and 
obesity and diabetes, the three of those together, the so-
called ``metabolic syndrome,'' occurs at phenomenally high 
rates in this group. We're also learning that even individuals 
of normal body weight have a higher incidence of hypertension 
and diabetes in this group, and that's a puzzle, and a question 
is trying to be answered now: Is that diet? Is that 
environment? Is that genetics? We have to figure out what are 
those causes, because obviously these are diseases that have a 
great deal of consequence, in terms of heart disease and 
strokes.
    We are learning that this kind of gathering together is 
also a great way to get community involvement. And the ways in 
which the Jackson Heart Study has embraced the community, and 
been embraced by the community, is a wonderful model for doing 
research on health disparities.
    The funding for 2011 for the Heart Study is very much a 
part of this budget, and the NHLBI intends to continue that at 
least through 2013. At that point, they will be evaluating what 
progress has been obtained. But, everything I have heard from 
the leadership is, they're--they expect to continue this for a 
long time.
    Senator Cochran. Well, thank you very much.
    Thank you, Mr. Chairman.

         INSTITUTE OF MEDICINE (IOM) REPORT ON CLINICAL TRIALS

    Senator Harkin. Thank you, Senator Cochran.
    I've got two or three things I'd like to follow up on, 
here.
    Dr. Collins, last year President Obama vowed to find, 
quote, ``a cure for cancer in our time.'' But, I remember when 
President Nixon declared a war on cancer. They've been fighting 
that thing ever since. So, while I appreciate the President's 
vow, I just wonder if we're going in the right direction.
    Now, you've come up with some things here that give us a 
lot of hope, but, just recently, the IOM issued a report that 
was very critical of the National Cancer Institute's (NCI) 
Clinical Trial Network (CTN). According to the IOM, the CTN is 
underfunded, and is approaching, ``a state of crisis.'' Most 
disturbing of all, about 40 percent of its cancer trials are 
never completed, which might suggest that we're wasting 
valuable time and money.
    So, again, I want to give you the opportunity to respond to 
that. The IOM report found that the CTN is too bureaucratic, 
its research is poorly coordinated. Due to cumbersome review 
procedures, the average time between developing an idea for a 
trial and getting it started is about 2 years. Another problem 
they pointed out was the distressingly low participation rate 
of adults in clinical trials. So, I wanted to kind of go over 
that with you and how are you responding to this IOM study.
    Dr. Collins. Senator, I think all of us are quite concerned 
about this situation. Certainly, I've studied that IOM report 
carefully and talked to the leadership at the NCI about this. 
The cooperative groups, 10 of them, that have been conducting 
clinical trials on cancer for as long as 50 years, have 
certainly produced wonderful data over the course of time. But, 
there's no question that the current system is not functioning 
as well as it should. And that's what this report pointed out.
    I should mention that it was Dr. Niederhuber and the 
leadership of the NCI that asked for the IOM to look at this, 
so they were fully aware of the need for some changes, and 
asking IOM to help out with this, and are now, I think, 
embracing that report and already moving forward to try to make 
such changes.
    Clearly, there are a number of serious issues here. One is 
the very long time, as you've mentioned, between the time when 
a protocol is conceived and when the first patient is enrolled. 
And that had stretched out to 2\1/2\ years. Well, here we have 
a field that's moving so quickly, by the time you get to the 
point of enrolling a patient, sometimes the protocol didn't 
seem like one that you would really want to support at that 
point. So, that timetable has to be shortened. NCI has moved 
forward, now, to make changes that will limit that to 1 year, 
and no more.
    And obviously, part of this is our own system of trying to 
run multicenter trials, which has gotten really quite 
convoluted and complicated, in the sense that, particularly, 
for human-subjects approval, every center has its own IRB, and 
the IRB has to review the consent form. And if you're trying to 
run a trial that involves dozens of centers, and every IRB 
wants to tweak things a little bit, you can see how time passes 
and you don't end up with things getting underway very quickly.
    Senator Harkin. Why can't----
    Dr. Collins. Furthermore, there may be----
    Senator Harkin. Why don't we consolidate that?
    Dr. Collins. Well, exactly. We need central IRBs, and there 
is a major move underway to implement that. It has been, I 
think, delayed by the fact that many legal minds have been 
involved, saying that institutions shouldn't really deem anyone 
other than their own IRB as capable of reviewing----
    Senator Harkin. Do we have to do anything legislatively, 
Dr. Collins?
    Dr. Collins. I think this actually can be handled without 
legislation. I will tell you, there's a great groundswell now, 
not just from cancer, but from many other areas of clinical 
research, to do something to streamline our human-subjects 
effort, that we are not really, in every instance, using this 
in a way to protect participants in research, but we've gotten 
all tangled up in the bureaucracy. And sometimes we are mixing 
up the things that are really high risk with things that are 
very low risk. And we need a revamping there. And I think this 
is something that's going to get attention quite soon.
    Other areas--there's a problem, in some instances, where 
protocols may be run in too many centers, and each center is 
only enrolling a very small number of patients. And so, it's 
not an efficient way to do things.
    There may not be a sufficient evaluation of whether a 
protocol is actually the best use of the money for that disease 
at that point. There needs to be more of a scientific rigor in 
the process.
    All of those are accepted, now, I think, by the NCI.
    There will be new leadership of the NCI; an announcement of 
that sort is imminent. And I am sure the new NCI Director will 
take this on as a very high priority, to try to understand how 
best to re-engineer this CTN, because this is critical for our 
future. We're going to have a much higher throughput of new 
molecular entities coming forward from this molecular 
understanding of cancer, and we have to have an engine in place 
to test them and see what works and what doesn't. So, this 
could not be more important, and I appreciate your raising the 
issue.

                          ALZHEIMER'S DISEASE

    Senator Harkin. Well, thank you. I have a couple more. I 
had a question that has to do with Alzheimer's, but maybe a 
little bit broader than that.
    A panel, convened by NIH, issued a finding, last month, 
that left a lot of people confused, I think, about Alzheimer's. 
According to this panel, there is no evidence that any of the 
strategies that people have been told to use to prevent 
Alzheimer's actually makes any difference. That includes 
getting exercise, taking supplements, keeping your mind active, 
doing crossword puzzles, and so forth. According to this panel, 
there's no evidence that any of these measures prevent you from 
getting this disease.
    So, one question on that would be how we interpret a 
finding like that. The other question about Alzheimer's has to 
do with a broader level of funding, and how we think about 
funding for different diseases.
    But, let's focus on this one, first, about the finding. 
What do we tell people? How do we interpret this finding?
    Dr. Collins. Well, I think there have been a lot of 
messages out there that people were confused by--what works, 
what doesn't work. The whole point of the NIH panel was to 
actually look at the evidence and try to see, What do we 
objectively know about measures that could be used to delay or 
prevent this disease? Because this is a disease that affects, 
obviously, very large numbers of people, and we're all 
concerned about it. I just turned 60; I'm thinking about this 
more than I used to.
    And, basically, all of the things that were put forward as 
potentially being beneficial in reducing the risk haven't held 
up very well to rigorous scientific evaluation. It looks as if 
doing crossword puzzles or doing Sudoku, it makes you better at 
doing crossword puzzles and doing Sudoku.
    It isn't clear that there's evidence it has a more global 
effect, in terms of protecting your mental capacities as you're 
getting older.
    The one exception that they thought perhaps there was some 
evidence for is diet, and particularly Omega-3 fatty acids, 
which are something that you find in fish. And there is some 
data supporting that as a possible preventive measure, and that 
one deserves more study. But, it was one bright light.
    And then, of course, there are well-documented 
environmental influences that we know about. Smoking, for 
instance, is clearly a risk factor for Alzheimer's, as well as 
a long list of other things. And certainly, obesity seems to 
have a connection, as well.
    But, in terms of the specific mental exercises, which I 
think was one of the disappointments for a lot of people who 
hoped that that would be a way that you could take control of 
the situation and help yourself, there didn't seem to be 
evidence to support that.
    Senator Harkin. Thank you.
    Senator Cochran.

                       INSTITUTIONAL DEVELOPMENT

    Senator Cochran. Mr. Chairman, thank you.
    We were talking, in my first round of questions, about the 
University of Mississippi and the legacy of Dr. Arthur Guyton. 
One thing that this subcommittee decided to do a few years ago 
was to earmark--oh, heaven forbid--some money, in this 
particular bill, and target the funding for grants and research 
to institutions in States that were getting less money and less 
attention to their work and applications than many other States 
had--which had long records of success and notoriety in certain 
areas.
    Now, the University of Mississippi Medical Center, it was 
benefited greatly from one person's influence--Dr. Arthur 
Guyton. We talked about that. But, there are other 
institutions--within small States, in particular--who just come 
out on the short end of the stick when they apply for grants 
and try to get Federal support for work they're doing. Some of 
the ideas may be good, but the money is just never--never finds 
its way to those institutions.
    So, we set aside, in fiscal year 2009, $224 million in a 
program designated for Institutional Development Awards. The 
purpose of that is to spread the money out in areas that would 
not, probably, be seriously considered for grants, finding and 
looking for the activities and the research that's being done, 
and having national impact and importance.
    I guess my question is--Mississippi received $5 million--a 
little over--of the amount appropriated. That's only 2.4 
percent of the total, so it's not like we out-maneuvered 
everybody; we didn't. But--and I guess that's the reason for my 
question. Some States do better than others in this, and I was 
just wondering, Is there any way for--a more careful review can 
be made to be sure that the intent of the set-aside is carried 
forward and that some States are not treated too much better 
than everybody else, so--the consequences of being left out?
    Mississippi shares 2.4 percent, for example. That doesn't 
sound like much to me. What are your thoughts about how we 
could better define what this money is for to make sure it 
carries out the intent of the Congress?
    Dr. Collins. Well, thank you, Senator.
    So, yeah, the Institutional Development Awards (IDEA), have 
been strongly supported by NIH. They're administered by the 
National Center for Research Resources. And, yes, the budget 
for fiscal year 2010 was--went up $229 million. These are 
competitive, they are available to the States who are 
identified as IDEA States, one of which is Mississippi, but 
there are a number of others that are traditionally underfunded 
by NIH, oftentimes because they have a lower proportion of 
institutions that are heavy in research efforts. But, we felt 
that we needed to be sure--we were finding opportunities in 
those States, and that those States had opportunities for NIH 
funding.
    There are a couple of specific programs: The Centers of 
Biomedical Research Excellence, COBRE, or ``Cobra,'' is one. 
There's an IDEA Network of Biomedical Research Excellence, 
INBRE. And, in fact, most of the States in the IDEA Network 
have been applying for those, and many of them with 
considerable success. But, it is a competitive program, where 
the peer-review system kicks in. And so, because of our 
interest in making sure that, with the funds available, we 
support what seems to the experts, who are not biased toward 
any particular State, but are trying to identify the best use 
of the money--we have to see where those outcomes fall.
    Another program, though, that is, I think, relevant, here, 
is actually the ability, through the ARRA, to support 
construction efforts that have been asked for in the IDEA 
States. And Mississippi recently received such a construction 
grant; Arkansas did. In fact, a number of the IDEA States, for 
this $1 billion of construction money, that were part of the 
ARRA, have been quite successful. And we're delighted to see 
that, because that may be a way, then, to build that capacity, 
so that, in the coming years, they'll be in an even better 
position to be highly competitive for these funds.
    Senator Cochran. Thank you very much.
    Senator Harkin. Senator Specter.

                   STATEMENT OF SENATOR ARLEN SPECTER

    Senator Specter. Thank you, Mr. Chairman.
    Dr. Collins, I join my colleagues in welcoming you here. 
Thank you for taking on this important job.
    My view, as expressed repeatedly, is that the National 
Institutes of Health are the crown jewels of the Federal 
Government--perhaps the only jewels. And in an era where we are 
searching for ways to prolong lives, save lives, and save 
money, it seems to me that we ought to be funding NIH a lot 
more aggressively than we are.
    Senator Harkin and I led the way, with Senator Cochran's 
concurrence, and others, to raise NIH funding from $12 to $30 
billion, $10 billion more than the stimulus. And the stimulus, 
I have heard, has created a whole wave across America of a--may 
the record show the witness is nodding in the affirmative----
    Dr. Collins. Yes, he is.

                                  CAN

    Senator Specter [continuing]. Great surge of enthusiasm and 
rekindled a lot of interest in young people, who had been very 
much concerned because the funding had tapered off. There had 
been a loss of real dollars--in excess of $5 million--when we 
had to--accommodated for cost of living adjustments and also 
some across-the-board cuts.
    And last year's funding was disgraceful, at $772 million. 
And this year's funding is also disgraceful, in my opinion, at 
$1 billion, with the comment made, ``Well, you got $10 billion 
before,'' but it wasn't meant to lessen the annual funding. So, 
I'm going to repeat a message to you, which I have made 
frequently; that is that the scientific is going to have to 
become a lot more politically active blowing your horn. The 
statistics are very impressive as to what the increased funding 
did for NIH on mortality rates, on strokes, and much progress 
on many strains of cancer, and heart disease, and right down 
the line. And I think what you have to do, for the Congress and 
for the administration, is show how many dollars it saves.
    Senator Harkin has been a real leader here on what he has 
done on wellness, the new concept, the Harkin Wellness 
Doctrine, a little exercise and annual exams and catching off 
ailments before they become chronic and debilitating and 
expensive. A lot of money to be saved by research; tremendous 
amounts of money to be raised by research.
    And your medical communities have gotten a lot of money. 
University of Pittsburgh has gotten $4 billion in the last 
decade. And it's so across the country. You got a lot of 
prominent people on those boards, politically influential 
people. And appropriations run on politics, on the pressure. 
You've got a great case, but it hasn't been expressed very 
well. And I don't fault Dr. Zerhouni or the prior--he was a 
great director----
    Dr. Collins. I agree.
    Senator Specter [continuing]. And staffed by great people.
    Now, I understand that you convened a meeting of your 27 
Institutes to talk about CAN, which is new. And it has been put 
forward to bridge the gap, so-called valley of death, as I've 
heard it expressed in the scientific community, between the 
bench and bedside, between research and practical application. 
It has an authorization of $500 million, not a whole lot of 
money for that kind of a project, but what is--first of all, 
can you confirm the meeting that the 27 Institutes got together 
on CAN and what was the thrust of the conversation?
    Dr. Collins. Well, thank you, Senator. And let me, first, 
say how appreciative your leadership has been over these years 
in supporting the cause of biomedical research, and 
particularly the critical role you've played for NIH support, 
including the ARRA funding, which, as you've alluded to, 
provided a remarkable shot in the arm for the research 
community and is being spent, I think, in truly exciting ways.
    With regard to the CAN, this part of the healthcare reform 
legislation, as you know, puts forward a proposal of having the 
NIH take on, in new and flexible ways, the acceleration of the 
process of going from a basic science discovery to a clinical 
advance; a drug therapy, most likely, but this would also apply 
to other kinds of clinical advances. We did discuss this last 
Thursday, all of the Institutes' directors together for a full-
day retreat.
    Senator Specter. I heard there was a lot of enthusiasm for 
it.
    Dr. Collins. There was a lot of enthusiasm. People were 
delighted about the potential, here, because the science has 
reached the point of making this a real possibility. Not that 
NIH would become a drug development company, but the 
partnerships that we could now establish between NIH and the 
private sector through this kind of legislation are really 
exciting and unprecedented and are being very well received, 
both by the academics and people in companies.
    Senator Specter. What is your professional judgment as to 
the kind of priority attention that the CAN ought to receive?
    Dr. Collins. From my perspective, this is one of the five 
themes that I published in Science magazine as being most 
worthy of high-priority attention. The CAN fits very nicely 
into that, but provides some additional flexibility. So, this 
is a very high priority for us, and obviously we are mindful of 
the fact that, at the moment, this is authorized, but not 
appropriated. And we are also mindful of the fact that this may 
be a difficult year, in fiscal year 2011, with the ending of 
the ARRA dollars. But, certainly, from my perspective, as the 
NIH Director, and speaking for all those other Institute 
directors, this is something people are very anxious to get 
started on, and they have great hopes for, recognizing this is 
high-risk research, that many drug development programs fail, 
that if we're going to undertake this, we have to be prepared 
for that. But, I think we could learn a lot by doing this in a 
new way.
    Senator Specter. Many programs fail and many programs 
succeed.
    Dr. Collins. Indeed.
    Senator Specter. And the successes have been monumental in 
what you have done for prolonging and saving lives. What could 
you do with the $500 million, Dr. Collins? Tell this 
subcommittee how much you could accomplish with it.
    Dr. Collins. So, to undertake a project where you go from a 
basic science discovery to a Food and Drug Administration (FDA) 
approval of a drug is several years and expensive effort. With 
$500 million, we could probably proceed with about 20 projects, 
simultaneously, that went all the way from soup to nuts in that 
pipeline, and probably another 20 where we identify compounds, 
that are already in freezers of companies, that have been 
abandoned for various reasons, because they didn't work out for 
one application, but they might work out for a different one, 
so-called ``repurposing,'' which would allow you to skip over 
many expensive steps. That would be quite a bold effort, 
indeed, to take on roughly, then, 40 projects on 40 different 
targets.
    Senator Specter. One final comment, with the red light on. 
I would like you to go back to your office and review what 
could be accomplished with the $500 million, in as specific 
terms as you could, what you project you could do with that. 
And I know it is very hard to talk about saving lives, but you 
have some experience in what has gone on in other lines, 
statistically; and to the extent you could quantify it on 
saving lives, prolonging lives, or saving money, I think it 
would be very helpful, when the Chairman and the rest of us sit 
down to allocate the funds, here.
    This is a very difficult subcommittee, having the Labor and 
Health and Human Services, and Education Departments. The 
competition for the money is absolutely fierce. So, the more 
specific you can be, the stronger the case can carry.
    Thank you, Dr. Collins.
    Thank you, Mr. Chairman.
    Senator Harkin. I just want to, first of all, say that this 
whole CAN that we put into the healthcare reform bill was a 
singular effort by Senator Specter.
    [The information follows:]

                    Cures Acceleration Network (CAN)

    As you know Senator Specter, the Cures Acceleration Network (CAN), 
authorized in the Patient Protection and Affordable Care Act of 2010, 
would provide the National Institutes of Health (NIH) with new 
authorities to advance the development of ``high need cures'' by 
smoothing the pathway for developing new drugs, biologics, and devices, 
particularly through the so-called ``valley of death'' phase of the 
therapeutic pipeline. CAN would provide NIH with new authorities and 
flexible funding mechanisms, including the ability to leverage the 
Government's investment through matching funds. In addition to 
supporting the development of novel compounds and the repurposing 
abandoned products, it would provide NIH with an opportunity to carry 
out systematic process engineering that would result in a more 
efficient and effective therapeutic development pipeline. The program 
would operate in close coordination with the Food and Drug 
Administration and private sector stakeholders. CAN's authorities would 
allow us to use three novel funding mechanisms--Cures Acceleration 
Grant Awards, which could allow up to $15 million per award and 
additional funds in subsequent years; Cures Acceleration Partnership 
Awards, which could allow us to leverage additional funds so that a 
total of $20 million could be put toward every $15 million award; and, 
Cures Acceleration Flexible Research Awards, which could allow 
discretionary use of other funding mechanisms for up to 20 percent of 
the appropriation.
    Methicillin-resistant Staphylococcus aureus (MRSA) provides an 
example of how CAN could contribute to improving health, saving lives, 
and lowering healthcare costs. MRSA is a major and growing clinical and 
public health challenge, and there is a need to develop antibiotics 
that are effective in treating this potentially life-threatening 
infection. MRSA occurs in hospitals and other settings where people are 
in close contact with one another, including nursing homes, 
dormitories, military barracks, athletic centers, and prisons. All 
sectors of the population are vulnerable, and certain groups are at 
higher risk, including children, the elderly, and people with 
concurrent health conditions. In 2005, MRSA caused approximately 94,000 
invasive infections and 19,000 deaths. Total hospital costs for 
patients with MRSA infections were more than twice as high as those for 
patients with methicillin-treatable Staph infections ($34,657 compared 
to $15,923).
    Industry interest in developing new antibiotics for drug-resistant 
infectious diseases like MRSA has declined considerably in recent 
years. Since 1999, 10 of the 15 largest companies have fully abandoned, 
or cut down significantly, discovery efforts in this field.\1\ CAN 
could help address the deficits in the antibiotic drug development 
pipeline for treatments for MRSA and other drug resistant pathogens by 
leveraging established research resources, bringing together the 
pharmaceutical industry, regulatory and the financial communities, and 
applying necessary incentives to identify compounds for later phase 
development of new antibiotics. CAN's approach could make important 
contributions to this area.
---------------------------------------------------------------------------
    \1\ Kresse, H et al. The antibacterial drugs market. Nature Reviews 
Drug Discovery, January 2007.
---------------------------------------------------------------------------
    The de novo development and characterization of each new drug ready 
for clinical testing would require approximately $20 million. The 
repurposing of a drug, which has already undergone considerable 
chemical and biologic characterization, would require approximately $5 
million. An appropriation of $500 million would therefore allow us to 
support approximately 20 novel drug development projects and another 20 
projects using compounds that have been abandoned for lack of capital, 
market demand, or regulatory and developmental hurdles. We anticipate 
that the program would eventually make major contributions to improving 
health, saving lives, and lowering healthcare costs associated with 
many serious human disorders and conditions that currently lack 
effective therapies and pose major burdens for individuals, their 
families, and society.

    Senator Specter. Thank you, Mr. Chairman.
    Senator Harkin. He really dogged that one. And since I wear 
the other hat, as chairman of that other committee, too--this 
is one that Senator Specter championed and got in there and was 
on us all the time to make sure that it was not dropped. And 
so, it was held in there, and I thank him for that.
    I agree that this is something that really needs to be 
done, and we've talked about it personally many times in the 
past. And, Senator Specter, I think, has really been the great 
leader on this one.
    Again, of course, Arlen also put his finger on it--we have 
a lot of competition for a lot of money here, and we have 
constrained budgets. So, I'm going to play a little bit of the 
devil's advocate here.
    What would funding the CAN up to that $500 million, or 
however close--what would that allow NIH to do, that it can't 
do now?
    Dr. Collins. No, it's appropriate to----

          THERAPEUTICS FOR RARE AND NEGLECTED DISEASES PROGRAM

    Senator Harkin. Why can't you do it now?
    Dr. Collins. It's appropriate to ask those questions. So we 
are, in fact, pushing this translational agenda in innovative 
ways. There's a program that this Congress has funded, the 
Therapeutics for Rare and Neglected Diseases, the TRND program, 
which aims to try to fill in some of the missing pieces in the 
``valley of death'' that's necessary to cross if you're going 
to go from a promising compound to an FDA application for a 
clinical trial. And we're pursuing that quite vigorously.
    And, Senator, I do understand the pressures on the budget 
system are severe. And I should have said earlier that, in that 
condition, the fact that the President's budget was able to 
come up with a $1 billion increase for NIH is something that--
we should all, sort of, credit the administration with their 
vision for science. And I, personally, am delighted to see that 
this is an administration that has put science at such a high 
priority, even with frozen discretionary budgets.
    What we could do that the CAN legislation provides is not 
just about money, though, it's also about flexibilities. So, 
what that legislation allows is that some proportion of that 
money can be used in a Defense Advanced Research Projects 
Agency (DARPA) like model, where you have flexible research 
authority to go beyond traditional grants, contracts, and 
cooperative agreements, to manage projects in very forward-
looking ways. And that, for this kind of science, where you 
need to make decisions quickly, where you need to bring in 
other partners in a quick turnaround when you see you need to 
fill a void in what the science is showing you needs to be 
done, can be quite valuable. And we do not, at the present 
time, have that kind of flexibility for this sort of project. 
And we could benefit from that.

                      FLEXIBLE RESEARCH AUTHORITY

    Senator Harkin. But, Dr. Collins, you have the flexibility, 
now that it's authorized. I know, you have that--what you're 
saying is, you don't have the money.
    Dr. Collins. Well actually, the way the bill was written, 
it says that the flexibilities of this bill may not be utilized 
unless the appropriation is put forward. Some appropriation is 
required before this is activated. So, unless, in the 
appropriations process that you all are thoughtfully leading, 
there is a green light offered to this project by providing 
some kind of funding, I am not permitted to take advantage of 
the authorized flexibilities. That's the way the legislation 
was put together.
    Senator Harkin. Even if we just appropriate a dollar?
    Dr. Collins. A dollar would, I suppose, do it, although it. 
It might be a little hard to do a DARPA program with $1. I 
don't know.
    Senator Harkin. I mean, I'm just talking about the trigger 
mechanism that allows this--you just told me something I didn't 
know. I didn't know that. So, this is very interesting.
    Dr. Collins. And, of course, Senator, the other question 
is, in trying to figure out all of the priorities that I now 
struggle with, How does this fit? And obviously, you might say, 
``Well, why don't you just do this with the budget you've 
got?'' Well, that would mean I would have to do less of 
something else. And already, with our 15 percent success rates 
looming, you can imagine how much of a stress and strain that 
is.
    Senator Harkin. Dr. Collins, I feel your pain.
    Dr. Collins. I'm sure you do.
    Senator Harkin. That same thing is hitting us here--not 
just here, but in health, education--we're going to have some 
real problems in education, meeting our needs in higher 
education. So, we've just got a lot of things that are pulling 
at us, and we just are not going to have the funds to do it. 
So, we've got to make some pretty tough decisions, too. And 
some of our friends are not going to be very happy with some of 
the decisions that we make, but we're all going to have to 
sharpen our pencils and just try to prioritize things. And what 
I'm hearing about the CAN is--it's a very high priority.
    Dr. Collins. That's correct.
    Senator Harkin. The translational research. And so, I'm 
going to take a look at what you just told me about--that 
there's a trigger mechanism in the legislation.
    I think, Senator Specter, that's something we're going to 
have to take a look at here.
    And I accept your word on that. We'll just have to see how 
much we need to put in there that would trigger that.
    Now, I know Senator Specter would like the full $500 
million. Yes.
    Senator Harkin. Actually, so would I.
    Senator Specter. We could----
    Senator Harkin. I don't have any problem with the $500 
million, but I----
    Senator Specter. We could do more than that. That was the 
appropriation for fiscal year 2010.
    Senator Harkin. Oh----
    Senator Specter. And now it's a set sum, so we could do $1 
billion.
    Senator Harkin. It was $500 million for 2010, such sums 
after that.
    Senator Specter. So, we're now at a set sum, so it could be 
$1 billion or $2 billion.
    Senator Harkin. You tell me where to get the money, and----
    Senator Specter. I will.
    Senator Harkin. Okay. And we'll just put it out there, who 
we're going to take it away from to get that money. Like I 
said, we just have a lot of different demands on our money.
    I had one follow up----
    Senator Specter. Mr. Chairman, you and I have found as much 
as $3.77 billion, in the past. And it was just exactly what you 
mentioned, it was the sharp pencil.
    Senator Harkin. Well, in the past----
    Senator Specter. And there are other accounts which do not 
rate with curing cancer or Parkinson's or Alzheimer's. And you 
and I did it before, and we can do it again.
    Senator Harkin. Yeah, we did it before, when we had some 
budget flexibility. I don't see much of that there right now. I 
just don't. Unless you've got some way of getting it.
    Anyway, I ran up my time. I'm yielding to Senator Specter 
for another round. Do we have another round?
    Senator Specter. No, that's it, Mr. Chairman. That really 
is.
    Well, I have one other item that I would like to take up, 
and that is the funding on minority health.

   NATIONAL CENTER ON MINORITY HEALTH AND HEALTH DISPARITIES (NCMHD)

    Senator Specter. I note that it is in the budget for $219 
million. The health reform bill elevated the NCMHD at NIH to an 
Institute. And the administration requested a budget of $219 
million, which, by comparison, seems low. What do you recommend 
on that, Dr. Collins?
    Dr. Collins. Well, actually, the NCMHD, is a major 
coordinator of minority health and health disparity research at 
NIH, but certainly all of the Institutes are invested in this 
area. If you look at the graph, here on the screen, the total 
investments estimated for 2011, with this budget, would be more 
like $2.7 billion, so more than 10 times what the funding is, 
specifically for that Institute.
    Because we actually think that minority health and health 
disparities ought to be a priority for all of the Institutes. 
Whether it's the NCI or the NHLBI, or the Diabetes Institute, 
these are all areas where health disparities are a critical 
matter.
    Senator Specter. Well, then why was a new Institute 
established for minority health, if it's accommodated at other 
places?
    Dr. Collins. I think there was a desire to have it more 
visible, to have a coordinating function, which that----
    Senator Specter. $219 million doesn't give you a whole lot 
of visibility.
    Dr. Collins. It has provided an opportunity to give 
endowments, for instance, to some of the traditionally 
minority-serving institutions. That's a major part of what that 
Center, and now Institute, has done, when that flexibility 
didn't exist before. And certainly this Institute, every 4 
years, puts forward a strategic plan, which they coordinate, on 
health disparities. And that didn't really have a home before, 
in terms of doing that kind of strategic plan coordination; and 
now it does.
    Senator Specter. Thank you.
    Thank you, Mr. Chairman.

                           BURDEN OF DISEASE

    Senator Harkin. Thanks, Senator Specter.
    Let me follow up on the Alzheimer's thing that I started 
off with on. The first part just had to do with that finding of 
that panel. But, here's the whole issue of how NIH decides how 
much to spend on individual diseases. It's something that keeps 
coming up; year after year, I hear about it.
    First of all, Congress does not earmark funding levels by 
disease. And I hope we never do. As long as I'm chairman, we 
never will.
    I'm often asked, by patients and advocates, for example, 
how to explain the NIH funding level for a disease like 
Alzheimer's.
    As we know, Alzheimer's is an enormous burden on our 
society, not just in human terms, but in terms of our overall 
economy. There's an estimate out there that, from 2010 to 2050, 
the Medicare and Medicaid costs of Alzheimer's will total--
ready for this one?--about $20 trillion. That's just for the 
care of Alzheimer's. Now, I don't know if that's high or low; 
I'm just tossing this estimate out there. Even if it was half 
that, it would be staggering.
    And yet, if you look at the NIH budget, funding for 
Alzheimer's makes up a much smaller share than one might 
expect; about 1.5 percent.
    Another example: pancreatic cancer is the fourth leading 
cause of cancer-related death, but less than 2 percent of the 
NCI's budget is devoted to this disease.
    So, my question, basically, is this, Dr. Collins. What role 
does the burden of a disease--the burden on society--play in 
where NIH allocates its money?
    Dr. Collins. Senator, it's a great question, and it's a 
question that all of the people who have sat in this chair in 
prior years have also wrestled with. From the very beginning of 
NIH and its system of trying to define how to set priorities, 
there have always been debates about what are the right 
weighting factors to apply to particular diseases. And I would 
say that it's a complicated enough calculus that it'll take a 
minute to explain.
    So, first of all, some of what NIH does needs not to be 
focused on a specific disease; otherwise, we will not have the 
foundational discoveries that result in Nobel Prizes and 
transformative understandings about neuroscience and immunology 
and cell biology and all of those things that are the really 
important foundation upon which everything rests. So, we would 
not want to have our entire budget specifically focused on 
disease research, or we would probably be mortgaging our 
future.
    When it comes to those things that are clearly in need of 
attention, how do we decide? So, this--certainly, the burden of 
disease has to be a big factor, and the cost of that disease 
has to be a big factor. And you've quoted numbers for 
Alzheimer's that are staggering in that regard. And diabetes 
could also be cited in that way--and cancer and heart disease.
    But, if we based our decisions solely on those issues, then 
rare diseases would tend to get ignored, or funded in only the 
very smallest amounts. If a rare disease happens to strike your 
own family, it's hard to say it doesn't matter. For that 
person, the burden of disease is very high. So, we clearly have 
a responsibility there, as well.
    And oftentimes, studying rare diseases gives us insights 
into common diseases. We study progeria, that affects maybe 30 
kids in this country, and we learned something about aging that 
we never knew before, which affects all of us. Those kinds of 
connections keep popping up over and over again. We wouldn't 
have statins if we hadn't started out by studying a rare cause 
of very high cholesterol levels. All of those, I think, are 
reasons not to focus solely on burden of disease.
    And then, there's scientific opportunity, which has got to 
be a big part of this. To say, ``We have a disease problem, and 
we're going to throw money at it,'' if nobody has an idea about 
what to do, is unlikely to be productive.
    And to take another area, which maybe is not quite as much 
of a burden, or quite as much of an expense, but where you can 
see the scientific field is just poised for a breakthrough, you 
don't want to miss that opportunity.
    So, the job of those 27 Institute Directors, and my job, is 
to try to survey the landscape, sort of, weekly, and figure out 
how to do that steering of the ship to try to be sure we are 
investing most wisely. Do we always get it completely right? I 
wouldn't say we could claim that, but I think we do pretty 
well. And we are supported, of course, by this remarkable peer-
review system. There's two levels which both looks at the 
scientific rigor of a grant proposal and then, at the second 
level, tries to figure out where are the highest program 
priorities, factoring in things like burden of disease. And 
when you look at the landscape of what we do across diseases, 
it doesn't match up precisely with what you might have guessed, 
just based on epidemiology, but I think it's fair to say 
there's a pretty strong connection.
    Alzheimer's--you know, we are working hard on that. There 
are 30 new drugs that are in various stages of being developed 
for this approach, using things that we've learned about the 
amyloid deposits in the brain, and the enzymes that are 
involved in breaking that down, and how to encourage them to do 
a better job.
    Vaccination--we talked about vaccination against nicotine; 
maybe a vaccination against amyloid, for Alzheimer's, which, 
unfortunately, in the early trials, a few years ago, ran into 
some unfortunate side effects. But, people are developing new 
ideas about how to get around that.
    I couldn't agree more that, if there's an area that 
desperately needs a breakthrough, it's Alzheimer's disease. A 
lot of people trying.

                           PANCREATIC CANCER

    Senator Harkin. Again, that gets me to another question 
about causes and the rapid growth of certain diseases. It just 
seems like Alzheimer's is exploding.
    Pancreatic cancer--the huge increase in pancreatic cancer 
in just the last few years. And different medical personnel 
I've talked to about this says that there's something going on 
out there; something is causing this huge increase in 
pancreatic cancer, but no one can quite figure out what it is.
    And so, that's why I say, you need to look at this--I mean, 
it--I'd like to have some sort of satisfaction, or some 
feeling, positive feeling, that NIH is pivoting a little bit on 
this and saying, ``What is causing this? Why?'' and guiding 
some more research into pancreatic cancer and what's happening 
there.
    We always knew that it was one of those secret kinds of 
cancers; in other words, you didn't know about it until it was 
too late----
    Dr. Collins. Yeah.
    Senator Harkin [continuing]. Because there was no markers 
for it or anything. But, it's not only that now, but it's just 
the huge increase. I forget the figure, but it's just up 
tremendously, the number of people being diagnosed with 
pancreatic cancer.
    Do you think NCI is pivoting and looking at this and 
putting more emphasis on it?
    Dr. Collins. I think pancreatic cancer is a cause of major 
concern at NCI, and is for me, personally, when you see the 
number of individuals being diagnosed with this disease, which, 
as you say, often comes to light after it's already too late, 
because it doesn't reveal itself until it's already, 
oftentimes, spread. It is, all too often, a disease that we 
don't do much for, at the present time, except chemotherapy, 
which may gain a few months. And, of course, some notable 
figures--Patrick Swayze, diagnosed with this disease, and the 
way in which that created a new personal face, has brought even 
more attention to this, as well it should.
    So, pancreatic cancer is one of the cancers being pursued 
by the Cancer Genome Atlas. This comprehensive effort to try to 
identify what exactly goes wrong in a pancreatic cell to cause 
it to grow out of control this way, and not just look under the 
lampposts, where we've been looking all along for clues, but 
actually using the tools of genomics to get all the answers 
that--all of the ways that a cell in the pancreas can start to 
go bad. And that will, I am confident, Senator, give us a 
comprehensive ability, both to do a better job of early 
diagnosis, but, most importantly, to identify new therapeutic 
magic bullets that will go to the heart of that cancer, like 
Gleevec does for leukemia; except we need a Gleevec for 
pancreatic cancer, don't we? And the problem right now is, we 
don't know what the target is that we're shooting at. The 
Cancer Genome Atlas will reveal the complete list of targets.
    Of course, that doesn't happen overnight. That's a process. 
And again, the CAN, we talked about a minute ago, may assist, 
once the target's identified, in speeding up the process of 
getting something ready for a clinical trial. All of those 
steps have to be integrated together.
    Again, I think having new leadership, imminently, for the 
NCI, is going to be quite timely in this regard. I am 
impatient, just as you are--frustrated, as you are--about this 
terrible disease of pancreatic cancer, and how many people we 
lose to it, and how impotent we seem to be, so often, in being 
able to stop the course of the disease.
    Senator Harkin. Yes.
    Dr. Collins. And I would not want to have a day go by where 
we were passing up on the opportunity of new ideas to do 
something about this.
    Senator Harkin. Yes, because like B-cell lymphoma and 
things like that, and what NCI has done has been miraculous.
    Dr. Collins. Yes.
    Senator Harkin. The cure rate there is just phenomenal.
    Dr. Collins. Yes.
    Senator Harkin. It's very, very good.
    Dr. Collins. Well, that's a good point, because there you 
have targets, and----
    Senator Harkin. Yes.
    Dr. Collins [continuing]. There, the drugs have developed 
against those targets. And, boy, they work.

                            FDA AND THE NIH

    Senator Harkin. Yes, they sure do. Okay, we'll follow up on 
that.
    You recently joined Secretary Sebelius and FDA Commissioner 
Hamburg in announcing a new partnership between NIH and FDA 
that, again, is intended to speed up the process of turning 
basic scientific discoveries into treatments. Well, what is 
this effort? How does this correlate with CAN? What are the 
goals? Is this different than what we've been talking about?
    Dr. Collins. It's a part of the whole system that needs to 
be coordinated, integrated, optimized. I think it's clear that 
relationships between NIH and FDA have to be really well 
orchestrated in order for all of those complicated steps, in 
going from an idea to having a successful clinical trial, to go 
forward without missteps that cost time and cost money.
    The FDA has enormous challenges in front of them, in terms 
of the way in which the development of therapeutics is 
evolving. The idea that you might, for instance, for cancer, 
need to get to a place where most patients are not being given 
one compound, but maybe two or three, that's targeted 
specifically to their tumor. Because you're going to know, in 
their tumor, exactly what's gone wrong. So, you look at your 
list of drugs, and you pick the combination that you know is 
zeroed in on their problem. Well, how does FDA evaluate a 
clinical trial of thousands of patients, where they aren't all 
taking the same thing? So, they need scientific research 
efforts to prepare them for that.
    The regulatory science that Peggy Hamburg has been talking 
about is exactly what's needed. We, at NIH, agree. Fact, we 
have funded, with FDA, for the first time, a research program 
on regulatory science. We just announced that. We got 59 
letters of intent. There are really interesting things being 
put forward, that the scientific community thinks they could 
offer to help FDA with the things that are coming down the 
pike, as far as regulatory challenges.
    And many academic investigators, if they're getting more 
involved in the development of therapeutics--and the CAN will 
make that happen--they're not familiar with exactly how to do 
this, and there's a risk that they might sort of get very close 
to an FDA application, and then find out they've left out 
something really important, and have to backtrack, and waste 
time and money. So, we have to tighten up those relationships.
    So, Peggy Hamburg and I have been meeting--and since last 
summer--to talk about how to do that. This new leadership 
council, which she and I will cochair, will involve senior 
leadership of both agencies, and will involve many people at 
middle level, so that we could prepare for the opportunities 
that are coming, and not end up in some sort of bureaucratic 
mixup, which would be really heartbreaking to see.
    I think the atmosphere is just right for this.

                           PATIENT ADVOCATES

    Senator Harkin. Tell me about the role of what I would call 
``patient advocacy groups.'' When you're going out to conduct 
human trials and, as you say, there's always risks when you 
conduct human trials--I think it's important to inform 
patients, from the beginning, help them understand what you're 
going through, in terms of the regulatory end of it. So, I'm 
just wondering when you're setting up this regime of involving 
these patient advocacy groups so that they can be supportive 
because they want to get the human trials out there. I think it 
might be wise to have them involved so that they understand 
what you're doing and that they can be a proponent of it, that 
they can be out in the public, advocating for this and sort of 
acting as a shield for you out there, perhaps, because a lot of 
people don't understand what you might be doing, and these 
groups could help you. So, I hope you'll look at involving them 
in this process.
    Dr. Collins. Senator, I completely agree with you. I think 
there are many heroes, and ``sheroes,'' out there in the 
advocacy organizations----
    Senator Harkin. Yes.
    Dr. Collins [continuing]. Who have remarkable insight into 
what we could do to improve the success of our whole 
enterprise. And we listen to them, with great attentiveness. 
And certainly, with regard to this relationship, we have 
already had some of those informal consultations. And on June 
2, we're holding a public, sort of, town meeting about this new 
NIH-FDA Leadership Council, and asking advocates and other 
members of the public to come forward and tell us what they 
think are the highest-priority matters for this council to 
address.
    Senator Harkin. So, it's an online town meeting?
    Dr. Collins. I think we're web casting it, and it's also, 
certainly, encouraging people to come live and come to the 
microphone.
    Senator Harkin. Ah. Is that going to be out at the campus?
    Dr. Collins. It is.

                               STEM CELLS

    Senator Harkin. Very good. That's on June 2. Well, I 
appreciate that. I think that would be important.
    Is there anything--oh, yeah, of course. How could I leave 
you without asking about stem cells?
    I wouldn't let this go.
    You recently announced that--as you did, also, in your 
opening statement--that some additional human embryonic stem 
cell lines have been approved for NIH funding, and including 
the line that's been studied more than any other. Again, what's 
the significance of this? How many lines are we up to now? And 
give me some crystal-ball-gazing. Where are we headed?
    Dr. Collins. Thanks for the question, because this is a 
very exciting area of biomedical research.
    There are now 64 human----
    Senator Harkin. Sixty-four?
    Dr. Collins [continuing]. Embryonic stem cell lines that 
have been approved by this NIH process that was stimulated by 
Obama's Executive order and that are up on the NIH registry and 
may now be used by researchers using Federal funds. And that is 
a number that is going to continue to grow. We have more than 
100 additional lines that are in the process of being reviewed.
    The goal, of course, of the review is to be sure that the 
consent process that was utilized for the embryo donors was 
above reproach. We want to be sure that these lines were 
obtained in a way that is entirely open to ethical scrutiny. 
And that is why the NIH has been conducting the reviews of 
those documents before certifying such a line.
    We were very happy to be able to get the materials, just 
about a month ago, on a few of the lines that had been 
particularly heavily used since 2001, when, as you recall, 
President Bush's decision was that lines could not be used that 
were derived after that. But, there were 21 lines that were 
allowed, at that point.
    Senator Harkin. Right.
    Dr. Collins. And there were a couple of them that were used 
particularly heavily. One, called H1, we were able to approve 
right away, because we had the documentation. The one that was 
causing a lot of anxiety in the community is a line called H9, 
and it just took a while for the deriver of that line--
derivers, because it involved both Israel and the United 
States--to locate all the documents and to get them to us. Once 
we had them, we did a rigorous review, in a very short 
turnaround. We're happy to see that everything was totally in 
order and approved that line. And I think that settled down 
some of the concerns that people had about whether that line 
was still going to be available to them, or not. We had allowed 
researchers to continue to work with it, with an existing 
grant; but, if somebody came back for a competing renewal, we 
wanted them to start working with approved lines. They can now 
use H9 as long as they want; it's fine. And there will be 
hundreds more coming through.
    On top of that, of course, there's great excitement about 
this additional way of making a pluripotent stem cell by taking 
a skin cell and, with just four genes, carefully chosen--and 
this is the remarkable work of Shinya Yamanaka, who I'm sure 
someday ought to win the Nobel Prize--you can take that skin 
cell and turn that into a pluripotent cell that basically can 
make any cell type that you would want it to, if you stimulate 
it with the right cocktail of cytokines and so on. Just 
phenomenal, Senator, that there's this much plasticity in the 
system, and that a cell that's been sitting in your skin all 
those years that--since you were originally born--is capable of 
having that ability. But, I guess it sort of makes sense, from 
a genome perspective; after all, that skin cell has the whole 
genome.
    Senator Harkin. Yes, right.
    Dr. Collins. It just needs to be woken up again and 
encouraged to think that it's young and has all those 
potentials to do everything you could imagine.
    That is an area that is just bursting with potential. We 
are actually starting, on the NIH campus, a special center for 
the so-called induced pluripotent stem cells (iPS)----
    Senator Harkin. Oh.
    Dr. Collins [continuing]. And the specific goal there is to 
push the agenda toward actual clinical applications.
    Senator Harkin. Great.
    Dr. Collins. The beauty of these, if it turns out to be as 
successful as we all hope, is that these are your cells; and 
so, if you were to need them for Parkinson's disease, because 
you develop that, or for a liver problem, you should be able to 
receive that kind of autotransplant, without the rejection 
problems that would otherwise apply if the cells came from 
somebody else. So, that is a big positive about this.
    The questions are safety, particularly, because a 
pluripotent cell sometimes grows when it isn't supposed to. And 
one of the ways we actually characterize pluripotent stem 
cells, like iPS cells or embryonic stem cells, is by whether 
they can make tumors if you put them into----
    Senator Harkin. Oh.
    Dr. Collins [continuing]. A particular mouse model. And 
obviously, we have to be very sure, before we try this in human 
applications, that we're not creating more trouble.
    There is, as you may know, a single FDA-approved trial for 
clinical use of human embryonic stem cells. It's for spinal 
cord injury. It's by a company called Geron. They have not yet 
enrolled their first patient, but expect to later this year. 
Obviously, everyone is watching that, although I think, 
realistically, one should not assume that the very first trial 
of any brand new therapy is going to tell the whole tale about 
its promise.
    But, of all the areas that are going forward right now in 
biomedical research, that I think have been breathtaking in 
their potential, this is right near the top of the list. And I 
think NIH, as you can maybe tell from my remarks, is pretty 
excited about pushing this forward with as much energy and as 
many resources as we're able to.
    Senator Harkin. I'd just ask my staff to get me all the 
information on this spinal cord. I had read about it, know a 
little bit, but I don't have--but, if you can get me some 
information on that, I'd appreciate it.
    Dr. Collins. Happy to do that.
    [Information follows:]

                  Stem Cells for Spinal Cord Injuries

    Geron Corporation is a biotechnology company based in California. 
Its lead human embryonic stem cell (hESC)-based therapeutic candidate, 
GRNOPC1, contains human embryonic stem cell hESC-derived neural support 
cells developed for the treatment of acute spinal cord injury. In pre-
clinical studies, GRNOPC1 has been demonstrated to repair myelin, a 
protective nerve coating, and to stimulate nerve growth leading to the 
restoration of function in animal models of acute spinal cord injury. 
The initial proof-of-principle animal studies were conducted by Dr. 
Hans Keirstead, an investigator at the University of California, Irvine 
with funding from the National Institute of Neurological Disorders and 
Stroke.
    In January 2009, Geron's Investigational New Drug application for 
GRNOPC1, which application the company had submitted to the U.S. Food 
and Drug Administration (FDA), went into effect. In May 2009, FDA 
placed a hold on the start of the phase 1 clinical trial and requested 
that Geron conduct additional pre-clinical studies to provide further 
assurance of GRNOPC1's safety. Geron has recently reported that 
additional data have been submitted to FDA, and its Web site now 
indicates that phase 1 clinical trials are expected to proceed in the 
third quarter of 2010.
    If Geron's clinical trial is allowed to proceed and GRNOPC1, as the 
subject of a biologics license application, is shown to be safe and 
effective, the therapy may provide a treatment option for thousands of 
patients who suffer severe spinal cord injuries each year.
    http://www.gemcris.od.nih.gov

    Senator Harkin. And the last issue--the last issue of 
Scientific American, which I always call the ``layman's 
magazine of an NIH report''--something I can understand; it's 
my must-reading every month, the Scientific American--but, the 
last cover--get a copy of--it was all on the iPS, on the adult 
stem cells, as they say. And it was a fascinating article about 
turning the clock back. And Dr.--I forget his name.
    Dr. Collins. Yamanaka.

                          SICKLE CELL DISEASE

    Senator Harkin.--Yamanaka, yes--is featured in that, and 
the way it was written is--just makes you think that this could 
be the--the way to go. I don't know. That's why I've always 
been in favor of all stem cell research, whether--whatever it 
is, whatever pathway it leads us down, within the ethical 
guidelines that we've established.
    Dr. Collins. Well, think about sickle cell disease as a 
possible application for iPS. This has already been done in a 
mouse model, which is one of the reasons I think I'm----
    Senator Harkin. Yes.
    Dr. Collins [continuing]. Particularly excited about its 
potential for humans. If you could take somebody with sickle 
cell disease, this terrible disorder, where a hemoglobin 
mutation causes the red cells to clog up in the vessels and 
cause all manner of organ damage and much pain. Take a skin 
cell, make it into an iPS cell, grow up a bunch of those, and 
then, using well-established experimental protocols, convert 
those iPS cells into bone marrow stem cells, and infuse them 
back in, after you've fixed the sickle mutation, which you can 
do while the--you're still working with a iPS cell in a culture 
dish. So, you can kind of do the whole cycle.
    That has been done by Rudy Jaenisch, at MIT, in a mouse 
model, and cured sickle cell disease in the mouse. Now, 
everybody will say, ``We've cured a lot of diseases in mice,'' 
and we have. But, by this protocol, it's pretty radical and 
pretty exciting, and certainly--one of the diseases that I hope 
will be high on the list for first human applications will be 
sickle cell. It's a 100 years since that disease was first 
described. This year, 100 years.

                         AUTOLOGOUS STEM CELLS

    Senator Harkin. Amazing. Yes.
    Let me ask you about autologous stem cells. I've been 
meeting somewhat with FDA on this, in terms of a change in 
their approval process that took place in the--in about 2005, 
if I'm not mistaken. And--but, that's another--that's the 
regulatory end. I'm just more interested in the scientific end, 
because I've had people in my office who have had autologous 
stem cell treatment. And--interesting group of people. One was 
a pilot who had been in an airplane crash and was, basically, 
paralyzed from his waist down. And through a process of 
autologous stem cells--I mean, he's not walking like you and I, 
but with canes and crutches. I mean, he's actually walking. 
But, you know, not fully recovered.
    Another person that had some heart problems brought in his 
different PET scans and different things like that, and, 
through autologous stem cells, has never had to have heart 
surgery.
    And there were a few others that I met. But, this is all 
through autologous stem--and some of that's being done in our 
country right now. Some of that's being done.
    Can you enlighten me as to what this involves? And what is 
NIH doing in autologous stem cells?
    Dr. Collins. So, this is an interesting area, and a rather 
controversial one----
    Senator Harkin. Yes, I know.
    Dr. Collins [continuing]. In terms of, what capability 
these autologous stem cells have to home in on the site where 
they're needed and how they actually turn into the kind of 
cells that are needed there in order to compensate for what's 
happened, whether it's a spinal cord injury, whether it's a 
heart attack and you're trying to provide an opportunity to 
repair itself?
    Frankly, the NIH-supported studies on this have not been as 
encouraging as many people had hoped. Take the approach to 
heart attack. Ten years ago, there was a lot of suggestion--
enthusiasm, here--that bone marrow stem cells might, if given 
directly into the heart muscle after a heart attack, allow 
repair of that area that had suffered damage. And there were 
experiments done in animals that looked encouraging; and human 
trials that were done, in many centers, that had somewhat mixed 
results.
    And I think, now, looking back on that, the evidence that 
that has actually been beneficial is not nearly as convincing 
as one would like.
    That has not stopped, of course, the research from going 
forward. And it shouldn't. And I can't tell you, but I could 
for the record, exactly what the total is--now is, of NIH-
supported autologous stem cell trials.
    I will say that I've heard some heartbreaking stories of 
people who have gone outside of the United States to undergo 
these kinds of trials, in the hands of people who really are 
not scientifically very rigorous, and bad things have happened, 
in terms of the consequences--infections, stem cells that got 
in the wrong place, people basically spending large sums of 
money for the kinds of therapies that really had no scientific 
basis, in hopes that it would help them.
    So, anybody contemplating that ought to be sort of eyes 
wide open, as far as what the evidence is.
    And we will continue to push this approach. We spend more 
money on adult stem cells than we do on embryonic stem cells, 
because of the potential opportunities there. And obviously, 
there are great successes, particularly bone marrow transplant, 
that we can all point to, that has saved many, many lives. But, 
the broader applications for curing problems that involve solid 
organs, I think, are much more challenging.
    There's a protocol just getting started, not with 
autologous cells, but with fetal cells, to try to treat Lou 
Gehrig's disease, ALS, which is obviously a disease of great 
frustration and great tragedy when it strikes.
    So, these kinds of approaches deserve every bit of 
attention, as long as they're done rigorously and as long as we 
find out, at the end of the study, ``Did it work, or did it 
not?'' so that we can guide people who are interested in that 
outcome.
    Senator Harkin. I'd like to know more about autologous stem 
cells. Get me some information. I'd just like to know, you 
know, what's being done at NIH in research on autologous stem 
cells.
    Dr. Collins. We're happy to provide a summary of that----
    Senator Harkin. Oh, good.
    Dr. Collins [continuing]. For you, Senator.
    [The information follows:]

                         Autologous Stem Cells

    Autologous stem cell transplantation (ASCT) is the use of an 
individual's own stem cells for the treatment of disease. The best 
known application of this technique is commonly referred to as ``bone 
marrow transplantation,'' where an individual's hematopoietic (blood) 
stem cells are harvested and then reintroduced to reconstitute the 
blood and immune system. This form of ASCT has been in use for many 
years, and has demonstrated clinical effectiveness for the treatment of 
several diseases.
    However, the concept of ASCT can be expanded to include stem cells 
harvested from one organ system to treat another organ system. Proof of 
principle animal studies revealed that stem cells harvested from organs 
such as bone marrow, skin, gut or endometrium, may be able to treat 
diseases in or ameliorate damage to solid organs such as the heart, 
brain, or spinal cord. These findings have raised hopes that these 
treatments could be transferred to the clinic and have led to the 
development of a growing cellular therapy industry within the United 
States and abroad. The application of ASCT across organ systems in 
humans is still in early experimental phases, and, unfortunately, the 
controlled studies conducted thus far have demonstrated mixed results, 
with some even having severe negative consequences.
    The National Institutes of Health (NIH) continues to support 
research into the development of safe and effective treatments for 
diseases and disorders using ASCT. I am providing you with a summary of 
NIH-supported clinical trials using autologous stem cells. This summary 
is a broad overview of the many research projects being conducted.
National Cancer Institute (NCI)
    ASCT is an important treatment option for several hematologic 
cancers as well as other types of cancer and other diseases. In this 
case, a patient's own bone marrow is used as a source of stem cells to 
reconstitute his/her blood cell producing capability following high-
dose curativeintent chemotherapy. However, ASCT is not curative for all 
patients and NCI continues to support research to refine and improve 
outcomes using ASCT in both intramural and extramural research 
settings. Strategies under investigation include adding novel agents 
and agent combinations following transplant and adding 
immunotherapeutic drugs in conjunction with transplant. These 
strategies are a therapeutic tool in treatment of the following disease 
states (among others): multiple myeloma and other plasma cell disorders 
such as amyloidosis and Waldenstrom's macroglobulinemia; Hodgkin's 
disease and non-Hodgkin's lymphoma; acute myelogenous leukemia and 
acute lymphoblastic leukemia; neuroblastoma; inflammatory breast 
cancer; systemic lupus erythematosus; and leukocyte adhesion 
deficiency.
National Heart, Lung, and Blood Institute (NHLBI)
    ASCT holds great potential for treating cardiovascular, lung, and 
blood diseases and the development of clinically feasible applications 
is an important part of NHLBI's strategic plan.
    In the cardiovascular area, ASCT is being investigated in phase I/
II trials for the treatment of damaged or malfunctioning heart muscle, 
and in an upcoming phase I trial for treatment of peripheral artery 
disease. Bone marrow mononuclear cells and mesenchymal cells are being 
tested for treatment of acute myocardial infarction (heart attack) and 
heart failure by injecting stem cells directly into the heart. In 
another study, cardiac-derived progenitor cells, obtained via cardiac 
biopsy, are being tested for treatment of individuals with ischemic 
left ventricular dysfunction. Finally, parent-banked umbilical cord 
blood-derived stem cells will be tested for treatment of limb muscle 
damage by injection into the affected muscle.
    In the hematology area, ASCT has been performed for more than five 
decades. In 2001, NHLBI initiated a network specifically to conduct 
multi-center trials to improve outcomes in blood and marrow 
transplantation, including eight clinical trials involving ASCT. 
Examples include a comparison of cell sources (autologous vs. 
allogeneic), a comparison of conditioning regimens used prior to ASCT, 
and the possible benefit of combining intensive chemotherapy with an 
autologous stem cell transplant. Investigator-initiated studies have 
also been implemented including a long-running program project grant on 
stem cell transplantation.
National Institute of Allergy and Infectious Diseases (NIAID)
    NIAID researchers are investigating potential opportunities for 
improving immune function in patients with certain rare genetic 
disorders, including X-linked Chronic Granulomatous Disease, X-linked 
severe combined immune deficiency, and WHIMS (warts, 
hypogammaglobulinemia, infection, and myelokathexis syndrome) through 
gene therapy and other treatments targeting human hematopoietic stem 
cells. NIAID also is supporting two trials to assess autologous 
hematopoietic stem cell transplantation ``to reset'' the human immune 
system in patients who suffer from the autoimmune diseases multiple 
sclerosis and systemic sclerosis.
National Human Genome Research Institute (NHGRI)
    NHGRI is supporting a gene therapy trial for a rare form of 
inherited immunodeficiency called adenosine deaminase (ADA) deficient 
severe combined immunodeficiency (SCID). Eligible children with ADA-
SCID are admitted to the Clinical Center where their autologous bone 
marrow stem cells are collected and subjected to retroviral-mediated 
gene transfer to correct the genetic defect before being reinfused. 
Results from treated ADA-SCID patients indicate that this approach can 
regenerate immune responses in these severely immune-compromised 
subjects.
National Center for Research Resources (NCRR)
    NCRR supports ASCT through its General Clinical Research Centers. 
Researchers are investigating the use of ASCT in patients with relapsed 
Hodgkin's or non-Hodgkin's lymphoma. Other scientists are transfusing 
autologous umbilical cord blood to regenerate pancreatic islet insulin-
producing beta cells and improve blood glucose control is being tested. 
Finally, other researchers are comparing disease-free survival between 
two different clinical protocols for ASCT.
National Institute of Dental and Craniofacial Research (NIDCR)
    Bone marrow contains a population of stromal stem cells capable of 
regenerating bone and supporting the formation of marrow. NIDCR-
supported scientists are planning a study that would involve harvesting 
bone marrow from the hip of patients with cranial (skull) defects that 
have failed standard treatments (metal plates, plastic overlays). The 
stromal cells in the marrow will be expanded and then attached to 
ceramic particles and placed into the cranial defects. Patients will be 
monitored to determine if new bone is formed.
National Institute on Neurological Disorders and Stroke (NINDS)
    NINDS is supporting a clinical protocol that receives biospecimens 
from patients with multiple sclerosis who have received autologous 
hematopoietic stem cells. The NINDS intramural researchers perform 
immunological analysis on the specimens to elucidate mechanisms of 
treatment action.

    Senator Harkin. That'd be good. I'd appreciate that.
    Well, that's good. I enjoyed this session very much.
    As you know, Dr. Collins, I have always, in the past, tried 
to have sessions with each of the Directors of the Institutes. 
However, because of some added responsibilities I have this 
year, now, I--my time is being crunched a lot, and I can't do 
that right now. I am hopeful, though--and I say this for the 
record--that sometime during this year, when I find some space 
opened up a little bit, that I might ask Mr. Fatemi and Ms. 
Taylor to also see if we can pull this together again, where I 
can set up a few days and have three or four down at a time, 
and sit down, because it's very enlightening. It's better than 
reading Scientific American, so, I just want you to know that 
I'm contemplating that. I hope I can do that, at some point yet 
during this calendar year.
    Dr. Collins. All of us at NIH would love that opportunity, 
Senator, and we do appreciate the many heavy loads that you're 
carrying this year, and your strong support of medical 
research.

                     ADDITIONAL COMMITTEE QUESTIONS

    Senator Harkin. Thank you.
    And congratulations, again, on taking over the reins, and 
we're looking forward to working with you on this terrible 
budget crunch that we have.
    Thanks, Dr. Collins.
    Dr. Collins. Thank you, Senator.
    [The following questions were not asked at the hearing, but 
were submitted to the Department for response subsequent to the 
hearing:]

               Questions Submitted by Senator Tom Harkin

                              MEDLINE PLUS

    Question. Dr. Collins, I am pleased at the importance you have 
placed on communicating to the American public about the valuable work 
done at NIH. As you may know, it was this subcommittee that first 
called on the National Institutes of Health (NIH) several years ago to 
start a magazine that would go directly to consumers to help people 
take charge of their health and provide reliable up-to-date information 
directly from the experts at NIH. What can be done to make sure that 
this NIH MedlinePlus magazine and its bilingual counterpart, NIH 
MedlinePlus Salud, gets out to every doctor's office and federally 
funded health center? Do you have the resources to do this?
    Answer. The NIH MedlinePlus magazine is the gold standard of 
reliable, up-to-date health information in plain language and in a 
reader-friendly format. I share your enthusiasm for it and its 
bilingual edition, the NIH MedlinePlus Salud, which is in both Spanish 
and English. As you know, the magazine contains no advertising and is 
produced through a partnership between NIH, particularly National 
Library of Medicine (NLM), and the Friends of the National Library of 
Medicine. The magazine is distributed through community health centers, 
hospital emergency rooms, physicians' offices, libraries, and other 
locations where the public receives health services and health 
information. Specific issues or sections of issues are also used for 
targeted health education and disease prevention campaigns. At its 
current budget level, NLM is able to support printing and distribution 
of an average of 260,000 copies of each issue of the English version. 
To date, private sector support has allowed printing and distribution 
of about 100,000 copies of the Spanish version. Both versions are now 
available online at: http://www.nlm.nih.gov/medlineplus/magazine/.
    To increase distribution of the magazines, we are working to extend 
our partnership to include other Government agencies and private 
organizations that have an interest in supporting the distribution of 
health information from NIH to their respective constituencies and 
audiences. For example, the Peripheral Arterial Disease Coalition and 
the American Diabetes Association supported the distribution of 
additional copies of two 2009 issues. The National Alliance for 
Hispanic Health supported the production and distribution of the first 
two issues of NIH MedlinePlus Salud. The NIH and the NLM will continue 
to encourage partnerships with other public and private organizations 
in an effort to ensure that this publication reaches the widest 
possible audience, every doctor's office, and every federally funded 
health center in America.

             AMERICAN RECOVERY AND REINVESTMENT ACT (ARRA)

    Question. NIH received $10.4 billion in ARRA--roughly $5 billion a 
year in fiscal years 2009 and 2010. That money is about to run out. How 
do you achieve the softest possible landing in fiscal year 2011? What 
are some of the challenges you will face?
    Answer. The $10.4 billion in ARRA for NIH has resulted in more than 
15,000 grants and contracts to date, with more expected by September 
30, 2010. These funds have served as a catalyst for inspiring 
innovative biomedical research in many areas of science relevant to 
health and disease.
    With regard to ensuring the softest possible landing beyond fiscal 
year 2011, NIH has taken steps to limit reliance on ARRA funding. From 
the outset, we decided to use these funds primarily for one-time 
expenditures, special equipment, construction, innovative grants, and 
special projects, which could either be advanced or completed within 2 
years. NIH also anticipated that some of the ARRA grantees who were 
awarded 2-year grants in fiscal year 2009 would seek continued funding 
in fiscal year 2011. These applications will be among those considered 
in the regular NIH competitive grant review process.
    The nature and pace of science is often unique to each research 
question. We expect a staggered increase in applications over the next 
few years resulting from the completion of the ARRA awards. Success 
rates of applicants may potentially be affected by gradual increases in 
application submission rates. NIH will continue to support applications 
that are rated by peer-reviewers to be meritorious and which address 
the programmatic priorities of the NIH Institutes and Centers.

                           GRANT RESTRICTIONS

    Question. Dr. Collins, in a January 2010 interview in The Chronicle 
of Higher Education, you suggested that universities are ``becoming too 
reliant on NIH money, allowing faculty members to obtain all their 
income from Federal research grants.'' You said that when faculty 
members run multiple research projects at the same time, ``that turns 
that investigator into a grant-writing machine perhaps more than a 
doing-of-science machine.'' You added that any new restrictions on NIH 
grants ``would have to be phased in over a fairly long period of time 
because many universities and faculty members would find that quite 
disruptive.'' What sorts of changes to the NIH grant system are you 
envisioning for the future? Would you favor limits on the number of 
grants scientists could receive simultaneously from NIH? If faculty 
members should not expect to obtain all their income from Federal 
research grants, what other sources could supply the funds?
    Answer. Over the past several years, the NIH has supported an 
increasing number of extramural research projects; ARRA provided 
additional support to expand and accelerate these efforts. In the 
upcoming and future years, we expect to see a higher number of 
applications for extramural awards, which could increase competition 
for the limited resources available. Given this, it simply may not be 
sustainable to have a large number of investigators deriving all or 
most of their salary from NIH grants. But before making any changes to 
our grants policy, we need to carefully explore alternatives and seek 
input from the relevant stakeholder groups and from the subcommittee. 
Any recommended changes would then have to be phased in over a period 
of time, as universities and researchers would find rapid change 
disruptive to the health of the American biomedical research community.
                                 ______
                                 
            Questions Submitted by Senator Daniel K. Inouye

                   LOWELL P. WEICKER CONFERENCE ROOM

    Question. I understand that you are considering dedicating a 
conference room in the National Institutes of Health (NIH) Neuroscience 
Research Center to Lowell P. Weicker. I greatly appreciate your 
commitment to preserving the honorable recognition of Governor Weicker 
and respectfully request an update on the status of the dedication of 
the conference room?
    Answer. NIH intends to dedicate a conference room to honor Senator 
Weicker's legacy of contributions to the advancement of human health 
through research. We anticipate the dedication to take place soon after 
the Porter Neuroscience Research Center phase II project is completed. 
The Porter Center, which is being built on the western portion of NIH's 
Bethesda campus with funding from the American Recovery and 
Reinvestment Act (ARRA), is scheduled to be completed in 2013. We will 
keep the Senate apprised of the specific plans for the dedication as 
the building's completion date approaches.

                            NURSING RESEARCH

    Question. Senator Burdick and I were instrumental in the 
establishment of the National Institute for Nursing Research (NINR) and 
for 25 years NINR has been dedicated to improving the health and 
healthcare of Americans through the funding of nursing research and 
research training Since it was established, NINR has focused on 
promoting and improving the health of individuals, families, 
communities, and populations. How does the NIH plan to further expand 
this critical arm of research?
    Answer. NINR supports clinical and basic research that develops 
knowledge to: build the scientific foundation for clinical practice; 
prevent disease and disability; manage and eliminate the symptoms 
caused by illness; enhance end-of-life and palliative care; and train 
the next generation of nurse scientists. In order to expand these vital 
areas of research at NIH, the President's fiscal year 2011 budget 
requests $150,198,000 for NINR, a 3.2 percent increase more than fiscal 
year 2010.
    In fiscal year 2011, NINR will build upon the important scientific 
research advances the Institute has supported more than its 25-year 
history. For example, NINR research in health promotion and disease 
prevention will explore strategies to understand and promote behavioral 
changes in individuals; evaluate health risks within communities; and 
explore biological factors that underlie susceptibility and mediate 
disease risk. To improve quality of life for those with chronic 
illness, NINR will continue to support symptom management research to 
illuminate the biological and behavioral aspects of symptoms such as 
pain, insomnia, and fatigue, and to enhance the ability of patients to 
manage their own conditions. NINR's end-of-life and palliative care 
program supports science to improve the understanding of the needs of 
dying persons, their families, and caregivers by examining such topics 
as the alleviation of symptoms; psychological care; advance directives; 
spirituality; and family decisionmaking. NINR training programs will 
ensure ongoing advancements in science and improvements in health 
through the support and development of an innovative, 
multidisciplinary, and diverse scientific workforce. In addition, 
across all of its research programs, NINR will continue its commitment 
to promoting health equity and eliminating health disparities in at-
risk and underserved populations through the development of culturally 
appropriate, evidence-based interventions.
    Finally, NINR will continue to support basic and clinical research 
to develop the scientific basis for clinical practice. These efforts 
will promote the translation of research into practice; assess cost-
effectiveness of clinical interventions; improve the delivery, quality, 
and safety of clinical care; and establish the foundation of evidence-
based practice. Evidence-based practice is essential to ensuring that 
all Americans receive the highest-quality, most-efficient healthcare. 
It is NINR's emphasis on clinical research that places NINR in a 
position to make major contributions to the NIH Director's goals for 
translating basic research to clinical practice, supporting science to 
enable better healthcare, and reinvigorating the biomedical workforce.

              ALLIED HEALTH SCHOOLS IN REMOTE COMMUNITIES

    Question. At my request, the University of Hawaii at Hilo 
established the College of Pharmacy. The College of Pharmacy's 
inaugural class of 90 students began in August 2007, will graduate in 
2011, and will hopefully stay in Hawaii to meet the growing demand for 
pharmacists. Historically, Hawaii's youth interested in becoming 
Pharmacists would travel to the mainland for school, and not return. It 
is my vision that the people of Hawaii will have educational 
opportunities in the health professions that will in turn increase 
access to care to residents in rural and underserved communities. Has 
there been any consideration of focusing research efforts on the 
benefit of establishing schools of allied health in remote communities 
to meet the growing needs for healthcare and improve access to care in 
rural America?
    Answer. Allied health education is an important part of the U.S. 
rural healthcare infrastructure. Allied health professionals form a 
vital part of the healthcare infrastructure necessary to support 
ambulatory, pharmacy and institutional primary and preventive care, yet 
the complement of allied health training and subsequent rural practice 
choices are limited. Several studies have highlighted the gross 
deficiencies in the health status of those living in rural areas, as 
well as the disparities in the distribution of health resources. Allied 
health education is offered in approximately 2,000 widely dispersed 
rural locations. Of significance, from a health policy perspective is 
the realization that primary healthcare profession shortage designation 
areas significantly lack allied health training education and 
resources. These concerns have served as a catalyst for the National 
Center on Minority Health and Health Disparities (NCMHD) and other 
Federal partners such as Health Resources and Services Administration 
to develop new directions for rural health research and workforce 
studies.
    Research indicates that targeted expansion of allied health 
training resources in rural underserved areas might improve the 
healthcare infrastructure, enhance access to care, and provide career 
opportunities for residents of rural areas. NCMHD will continue to 
support a rural health research agenda as part of its activities. This 
includes collaborative efforts to address the distribution of allied 
health professions training and workforce distribution, providing 
research infrastructure and capacity for rural-based institutions to 
support allied health education training and meet NIH's goal of 
developing scientific resources for disease prevention. Future research 
will be able to identify the optimal mix of allied health professionals 
necessary to support healthier rural communities.

                         CHRONIC KIDNEY DISEASE

    Question. Hawaii experiences a higher than average rate of Chronic 
Kidney Disease (CKD) with 1 person in 7, compared to a national average 
of 1 person in 9, afflicted with this disease. Among the Asian/Pacific 
Islander (API) population groups, Filipinos have one of the highest 
rates of incidence per capita. National Kidney Foundation of Hawaii in 
2007 it is estimated that of the 156,000 residents with CKD, 
approximately 32 percent are Filipino. Has there been any consideration 
to focusing research efforts on preventing chronic kidney disease among 
the API population groups?
    Answer. The National Kidney Disease Education Program (NKDEP) is an 
initiative of the National Institutes of Health that is designed to 
reduce the morbidity and mortality caused by chronic kidney disease 
(CKD) and its complications. NKDEP works to reduce the burden of CKD 
and focuses its efforts on those communities most affected by the 
disease including African Americans, American Indians, and APIs.
    In 2008, the NKDEP initiated the Community Health Center (CHC)-CKD 
Pilot to identify effective strategies or improving detection and 
treatment of chronic kidney disease in community health centers--
critical primary care settings for many people at increased risk for 
CKD. The pilot involves a small group of centers in the Northeast that 
work together to design, implement, and monitor performance 
improvements related to CKD. NKDEP is currently developing plans to 
broaden the pilot project nationally and will use data from the pilot 
phase pilot and lessons learned to inform this expansion. CHCs in 
Hawaii would be appropriate participants in this effort. 
Representatives from NKDEP have been in contact with Hawaii State 
Representatives and the Hawaii National Kidney Foundation since March 
2008 and have provided technical assistance on how NIH resources could 
potentially be utilized to reduce the burden of chronic kidney disease 
among Hawaiians.

                              HEPATITIS B

    Question. Hepatitis B and liver cancer, as caused by the hepatitis 
B virus (HBV), are the single greatest health disparities affecting the 
API populations in the United States. While up to 14 percent of the API 
population is infected with HBV, only 0.4 percent of the Caucasian-
American population is infected. Asian Americans, native Hawaiians, and 
APIs comprise more than half of the 2 million estimated HBV carriers in 
the United States and consequently have the highest rate of liver 
cancer among all ethnic groups. Has there been any consideration of 
focusing research efforts on preventing HBV in APIs and other groups 
disproportionately affected by HBV?
    Answer. The NIH supports research and education activities focusing 
on groups that are disproportionately affected by HBV. For example, the 
multi-center Hepatitis B Research Network, established in 2008, aims to 
advance understanding of disease processes and natural history, as well 
as to develop effective approaches to treating and controlling HBV. The 
network includes 21 clinical sites across the United States, including 
Hawaii, and a central data coordinating center. The network's centers 
are in the final stages of planning several clinical trials in both 
adults and children. Recognizing the health disparities affecting the 
API populations, the network plans to conduct trials testing antiviral 
therapy in these particularly at-risk groups. In another at-risk 
population, the NIH is conducting studies on the use of antiviral 
therapy during pregnancy to prevent the spread of HBV from a 
chronically infected mother to her newborn. The network will enroll 
pregnant women with HBV into clinical studies to assess risk factors 
associated with reduction in maternal-infant transmission.
    Research to develop new classes of drugs that are safe and 
effective in treating HBV infections is essential to effectively 
addressing HBV disparities. It is also critical to study how HBV 
develops resistance to new classes of drugs. For example, in studies 
conducted in nonhuman primates, NIH scientists and their colleagues 
determined that the replication rate for HBV is higher than previously 
thought. A higher replication rate increases the frequency of HBV 
genetic mutations, including those mutations that cause the virus to 
become resistant to drugs. This finding may help enhance the ability to 
predict when HBV virus will develop drug resistance which, in turn, 
will inform the use of existing antiviral therapies, including the use 
of a single antiviral drug versus combination therapies. NIH-funded 
researchers also discovered that selective combinations of existing 
drugs (nucleotides and nucleosides) may work better together not only 
to inhibit the emergence of mutated strains, but also to do a better 
job of reducing circulating virus.
    A workshop, arranged by NIH together with the U.S.-Japan 
Cooperative Medical Sciences Program and the Asia Pacific Association 
for the Study of Liver, was held in Hong Kong in February 2009. Its 
purpose was to understand the issues related to antiviral drug 
resistance encountered in the treatment of HBV infected patients in the 
countries of the Asia-Pacific region. Issues discussed included 
determining the extent and burden of resistance in Southeast Asia, 
which has the highest prevalence and incidence of HBV infection 
worldwide. Other issues discussed were the need for databases to 
catalogue and track virus mutations associated with resistance; to 
track patient management; and to study correlations between treatment 
and clinical outcome.
    Other NIH-supported basic and clinical research holds promise for 
populations disproportionately affected by HBV. For example, currently 
licensed antiviral drugs for HBV target a single step in the viral 
replication cycle. As resistance with this class of drugs seems 
inevitable, NIH-supported investigators, through partnership 
initiatives and investigator-initiated proposals, are redirecting their 
research to novel targets in the replication cycle and are pursuing the 
development of different classes of drugs. Other studies are ongoing to 
explore host responses to HBV infection, how the virus spreads in the 
liver, the influence of viral inoculum on outcome, and the cascade of 
host responses leading to chronicity or resolution.
    There are ongoing efforts to promote coordination and planning of 
all HBV research within NIH and across the Department of Health and 
Human Services. Strategic plans, such as the trans-NIH Action Plan for 
Liver Disease Research (http://liverplan.niddk.nih.gov) and the plan 
produced by the National Commission on Digestive Diseases (http://
NCDD.niddk.nih.gov), were developed with trans-NIH and trans-DHHS 
input, and highlight important research goals relevant to controlling 
HBV. In 2008, NIH convened a Consensus Development Conference on the 
Management of Hepatitis B. The conclusions of this conference can be 
found at the following Web site: (http://consensus.nih.gov/2008/
hepbstatement.htm). The NIH is also providing expert input on the HHS 
Viral Hepatitis Interagency Working Group to coordinate the responses 
to the challenges described in the recent Institute of Medicine report 
on HBV and liver cancer.
    In addition to research activities, the National Digestive Diseases 
Information Clearinghouse provides educational materials for the public 
on HBV to improve knowledge and awareness (available at: http://
digestive.niddk.nih.gov/diseases/pubs/hepatitis/index.htm). Materials 
on HBV are available in several languages, which include Chinese, 
Korean, Vietnamese, and Tagalog. There is a new series of fact sheets 
focusing on hepatitis B-related issues affecting API.

                                DIABETES

    Question. One of the gravest threats to the healthcare system is 
the chronic disease of diabetes with its impact on both the economy and 
on the quality of life for nearly 24 million Americans. In Hawaii, 
Native Hawaiians have more than twice the rate of diabetes as Whites 
and are more than 5.7 times as likely as Whites living in Hawaii to die 
from diabetes. Education and prevention are essential to controlling 
this serious, costly, and deadly disease. What innovative research 
efforts have been considered to improve diabetes outcomes and prevent 
diabetes?
    Answer. NIH research has helped to significantly increase the life 
expectancy of people with diabetes and led to the development of a 
proven method to help prevent or delay the most common form of the 
disease, type 2 diabetes. For example, the landmark Diabetes Prevention 
Program (DPP) clinical trial demonstrated that a lifestyle intervention 
aimed at modest weight loss achieved a 58 percent reduction in diabetes 
rates among people at risk in a 3-year trial. The intervention was 
effective in both men and women and in all ethnic groups tested and was 
especially effective in older participants. Results published since the 
original findings have shown that the intervention remains effective 
for at least 10 years. In addition to reducing rates of diabetes, the 
intervention also led to improved blood pressure and lipid levels with 
less use of medications. The study included a site in Hawaii.
    To develop lower cost methods to deliver the DPP intervention to 
the 57 million Americans with pre-diabetes who could benefit, the NIH 
has vigorously pursued DPP translational research. One innovative NIH 
sponsored study tested a group lifestyle intervention, modeled after 
the DPP's, that is delivered at YMCAs. This approach yields a sharp 
reduction of cost per patient, and appears to be achieving excellent 
interim results. Importantly, YMCAs are located throughout the United 
States, including in many communities at high risk of type 2 diabetes. 
For example, the State of Hawaii is home to 17 YMCA branches. A fully 
national implementation of these methods would have the potential to 
affect diabetes treatment for Native Hawaiians in significant ways. 
Because of the excellent results achieved in this program to date, the 
Centers for Disease Control and Prevention (CDC) is planning to expand 
it to 10 more YMCA locations around the country. Similarly, the United 
Health Group, a private insurer, has announced plans to pay for its 
subscribers in six cities who are at risk of diabetes to receive at no 
charge a YMCA-based diabetes prevention intervention modeled on the 
program. These are outstanding examples of the adoption of evidence-
based prevention methods to alleviate a serious national healthcare 
problem.
    The National Institute of Diabetes and Digestive and Kidney 
Diseases (NIDDK) is also sponsoring a major multi-center trial to study 
the effects of lifestyle change and weight loss on the course of type 2 
diabetes. Exciting preliminary results at 4 years have shown improved 
diabetes control and reductions in cardiovascular disease risk factors 
despite less use of medication. As with the DPP, the study includes a 
substantial representation of minority groups disproportionately 
affected by type 2 diabetes. To build on the findings from major NIH-
supported trials that have transformed diabetes care by establishing 
therapies that reduce diabetes complications and premature mortality, 
ongoing studies are examining translation of these approaches into 
communities at risk. One such research effort is employing community 
health workers in American Samoa, where diabetes rates are 3-fold 
higher than in the U.S. mainland, to test methods for delivering care 
there, as informed by results from previous NIH studies.
    It is particularly important to understand how diabetes is 
affecting children in America. The SEARCH for Diabetes in Youth study, 
a joint program of the CDC and the NIH, is collecting data on the 
incidence and prevalence of type 1 and type 2 diabetes in young people 
of diverse ethnicity, and thus is providing information to better 
understand the diabetes disparity among young APIs as well as other 
groups. One SEARCH center, located at the Kuakini Medical Center in 
Honolulu, will help provide the most accurate statistics to date on 
childhood diabetes in Hawaii. The National Diabetes Education Program 
(www.ndep.nih.gov), another joint effort of NIH and CDC, distributes 
educational materials conveying the vital health messages that have 
come from the major NIH-sponsored diabetes studies. Many of these 
materials have been translated into a wide array of languages, 
including the Pacific Island languages of Chamorro, Tagalog, Tongan, 
Chuukese, and Samoan, as well as Japanese, Indonesian, and other 
languages of the Pacific Rim. These programs are helping to extend the 
benefit of NIH diabetes research to people of diverse ethnicity in the 
United States and throughout the world.

                     COLLABORATIVE CANCER RESEARCH

    Question. What is the status of the administrations' efforts to 
continue collaborative cancer research and program efforts focused on 
reducing cancer health disparities in native Hawaiians?
    Answer. The administration's efforts to continue collaborative 
cancer research and program efforts focused on reducing cancer health 
disparities in Native Hawaiians are exemplified in a number of 
community-based participatory research programs supported by the Center 
to Reduce Cancer Health Disparities of the National Cancer Institute 
(NCl/CRCHD). These include:
Community Networks Program (CNP)
    This program was recently renewed and the new CNP centers 
initiative (RFA-CA-09-032) extends the previous efforts of NCI to 
support community-based participatory research (CBPR) in racial and 
ethnic minorities and other underserved populations. The goals of the 
CNP Centers are (1) to develop and perform evidence-based intervention 
research to increase use of beneficial biomedical and behavioral 
procedures for cancer prevention, detection and treatment, which may 
include related co-morbid conditions; and (2) to train and promote the 
development of a critical mass of competitive new researchers using 
CBPR to reduce health disparities. This program and its predecessors 
have promoted and continue to promote CBPR-based cancer health 
disparities research. As part of the current NCl/CRCHD CNP, NCI 
supports two projects aimed at reducing cancer health disparities in 
native Hawaiian populations.
    The 'Imi Hale Native Hawaiian Cancer Network is aimed at reducing 
cancer incidence and mortality among native Hawaiians by maintaining 
and expanding an infrastructure that: (1) promotes cancer awareness 
within native Hawaiian communities; (2) provides education and training 
to develop native Hawaiian researchers; and (3) facilitates research 
that aims to reduce cancer health disparities experienced by native 
Hawaiians. 'Imi Hale is housed at Papa Ola Lkahi, a nonprofit native 
Hawaiian community-based agency in Honolulu, is dedicated to improving 
native Hawaiian health and well being. They collaborate with key 
partners at the community, State, and national levels. Examples of 
clinical partners are the five Native
    Hawaiian Health Care Systems (NHHCS, providing access and 
prevention services to Native Hawaiians on the State's seven inhabited 
islands), the Queen's Medical Center, and Breast and Cervical Cancer 
Control Program. Examples of program partners include CIS, ACS, and 
Hawaii Primary Care Association. Examples of educational and research 
partners include the University of Hawaii, Oregon Health and Sciences 
University, and the NHHCS IRB.
    Weaving an Islander Network for Cancer Awareness, Research, and 
Training (WINCART) is a community-academic consortium employing CBPR to 
reduce preventable cancer incidence and mortality among five API 
communities in southern California. The specific aims of WINCART are 
to: (1) identify individual, community, and health service barriers to 
cancer control among APIs; (2) improve access to and utilization of 
existing cancer prevention and control services; (3) facilitate the 
development, implementation, and evaluation of community-based 
participatory research studies; (4) create opportunities to increase 
the number of well-trained API researchers through training, 
mentorship, and participatory research projects; (5) sustain community-
based education, training, and research activities by increasing 
partnerships with governmental and community agencies, funders, and 
policy makers; and (6) disseminate research findings to aid in the 
reduction of cancer health disparities for APIs. Project methods 
include implementation and evaluation of community awareness activities 
in each API population; conducting cancer prevention and control 
research; and recruitment/training/mentorship of API researchers.
Basic Research in Cancer Health Disparities (R21/U01)
    Two new NCI-supported funding opportunities, PAR09-160 and PAR09-
161, have been developed to encourage basic research studies to 
determine whether there are biological causes and mechanisms of cancer 
health disparities and support the development of a nationwide cohort 
of scientists with a high level of basic research expertise in cancer 
health disparities research. PAR09-160 will focus on the development of 
resources and tools, such as racial/ethnic specific biospecimens, cell 
lines and methods that are necessary to conduct basic research in 
cancer health disparities. PAR09-161 will provide an avenue for entry 
into cancer disparities research through collaboration and association 
with researchers with specific expertise in emerging technologies in 
cancer research.
Minority Institution/Cancer Center Partnership (MI/CCP)
    The MI/CCP program supports a partnership program that promotes 
research in cancer health disparities. The University of Guam (UOG), 
and the Cancer Research Center of Hawaii (CRCH), an NCI-designated 
cancer center at the University of Hawaii at Manoa, have been engaged 
in a unique and successful partnership over the past 6 years to 
establish a Cancer Research Center of Guam on the campus of UOG, to 
increase number of faculty and students engaged in cancer research at 
UOG, and to increase the number of faculty from CRCH addressing issues 
of particular relevance for cancer health disparities in the Hawaii/
Pacific region.

                           CANCER PREVENTION

    Question. How will the NIH continue to support an infrastructure 
that has identified and mentored more native Hawaiian researchers in 
cancer prevention and control than any other institution has done in 
the past 20 years?
    Answer. NIH is committed to enhancing workforce diversity within 
the research enterprise, and as part of that effort, seeks to support 
infrastructures that recruit and retain a strong cadre of competitive 
researchers from diverse backgrounds working in cancer prevention and 
control. Within NCI, there are a number of current activities that will 
continue to support an infrastructure to train and mentor native 
Hawaiian and other Pacific island cancer researchers. Examples of 
programs within NCI's CRCHD that support training infrastructure for 
native Hawaiians include:
MI/CCP
    The NCl/CRCHD supports a partnership program between minority 
serving institution partners and a NCI-designated cancer center to 
foster training and research activities. For example, the newly awarded 
5-year U54 University of Guam and the University of Hawaii at Manoa MI/
CCP partnership has a well-established infrastructure for mentoring of 
Hawaiian and Guamanian researchers in cancer research as part of their 
diversity training program.
CNP
    The goal of the NCl/CNP program is to develop and increase capacity 
building in support of community-based participatory education, 
research and training to reduce cancer health disparities. The program 
has increased the development of a cadre of new investigators, 
including among native Hawaiian researchers, in the field of cancer 
health disparities research. To date, a total of 34 native Hawaiians 
have been trained, representing 7 percent of the total CNP trainees. 
The CNP native Hawaiian trainees have submitted 40 grant applications 
and a total of 12 were funded for a 30 percent success rate. Building 
on the success of the CNP program, the new 5-year CNP centers program 
has been established, and will continue to support infrastructure for 
diversity training.
Promote Workforce Diversity (PAR-09-162)
    The Exploratory Grant Award to Promote Workforce Diversity in Basic 
Cancer Research (PAR-09-162) supports underrepresented minorities, such 
as native Hawaiians, in basic cancer research. Through this funding 
opportunity, NCI encourages institutions to diversify their faculty 
populations, and increase the participation of individuals currently 
underrepresented in basic cancer research, such as individuals from 
underrepresented racial and ethnic groups, individuals with 
disabilities, and individuals from socially, culturally, economically, 
or educationally disadvantaged backgrounds that have inhibited their 
ability to pursue a career in health-related research.
Continuing Umbrella of Research Experiences (CURE)
    The ongoing CURE program offers unique training and career 
development opportunities to enhance diversity in cancer and cancer 
health disparities research. With a focus on broadening the cadre of 
underrepresented investigators engaging in cancer research, the ongoing 
CURE program identifies promising candidates from high school through 
junior investigator levels and provides them with a continuum of 
competitive funding opportunities. Today, there are 30 CURE supported 
trainees and 14 high school and undergraduate students who are native 
Hawaiians.
Diversity Supplements
    These diversity supplements are designed to foster diversity in the 
research workforce. These supplements support and recruit students, 
postdoctoral, and eligible investigators from groups shown to be 
underrepresented in biomedical research. Currently, two native Hawaiian 
junior investigators are supported by diversity supplements.
NCI Community Center Centers Program (NCCCP)
    The NCCCP is designed to create a community-based cancer center 
network to support basic, clinical, and population-based research 
initiatives, addressing the full cancer care continuum--from 
prevention, screening, diagnosis, treatment, and survivorship through 
end-of-life care. The NCCCP pilot has added the Queen's Medical Center, 
Honolulu, Hawaii (The Queen's Cancer Center) to its 30-hospital 
network.
Cancer Health Disparities Geographic Management Program (GMaP)
    GMaP, a new initiative, is developing transdisciplinary regional 
networks dedicated to the coordination and support of cancer health 
disparities research training and outreach using regional management 
approach. Creating sustainable partnerships among institutions and 
agencies involved in cancer health disparities research and cancer 
care, this initiative seeks to advance cancer health disparities, 
diversity training and ultimately, contribute to disparities reduction. 
A companion program, the Biospecimen/biobanking Management Program, 
will support research and training infrastructure specific to 
biospecimen collections among underrepresented populations across the 
country.

                            CANCER RESEARCH

    Question. How will NCI support entities like 'Imi Hale, who engage 
Hawaiian communities in identifying and addressing cancer health 
disparities and invest in building community capacity to mobilize local 
resources and train local staff? The mission of the NCI CRCHD is to 
reduce the unequal burden of cancer in our society and train the next 
generation of competitive researchers in cancer and cancer health 
disparities research.
    Answer. The NCI's CRCHD coordinates multiple programs that focus on 
community based participatory cancer disparities research and multi-
institution collaborations to reduce the unequal burden of cancer and 
train the next generation of competitive cancer researchers. These 
programs include CNP, Patient Navigation Research Program (PNRP), MI/
CCP, and CURE. All of the following programs are either in Hawaii or 
extend to native Hawaiians and address cancer health disparities and 
community building among Hawaiian communities.
CNP
    The NCl/CRCHD CNP builds capacity in community-based participatory 
research, educational outreach, and professional training through 
partnerships with community organizations and institutions working with 
multiple racial/ethnic and underserved populations, including Hawaiian 
populations. The goal of the program is to improve access to beneficial 
cancer interventions and treatment in communities experiencing 
significant cancer health disparities. Currently, the NCI is supporting 
25 CNP projects developing programs to increase the use of cancer 
interventions in underserved communities. Interventions include proven 
approaches including smoking cessation, increasing healthy eating and 
physical activity, and early detection and treatment of breast, 
cervical, and colorectal cancers.
    Each CNP has put together an advisory group that serves as the 
``voice of the community.'' These advisory groups work with local 
community members to gather information and then deliver back results. 
A steering committee of community-based leaders, researchers, 
clinicians and public health professionals provides additional support.
    To sustain successful efforts in their communities, CNP grantees 
work closely with policymakers and nongovernmental funding sources. 
Together, CNP grantees and NCI train investigators, identify potential 
research opportunities, and work to ensure that best practice models 
are widely disseminated.
MI/CCP
    MI/CCP is designed to: (1) increase Minority Serving Institutions 
participation in cancer research and research training and (2) increase 
the involvement and effectiveness of NCI-designated Cancer Centers in 
developing effective research, education, and outreach programs to 
encourage diversity among competitive researchers and reduce cancer 
health disparities. These partnerships foster and support intensive 
collaborations to develop stronger cancer programs aimed at 
understanding the reasons behind significant cancer health disparities 
among racial and ethnic minority and socioeconomically disadvantaged 
populations. NCI supports grants under this program that establish such 
a partnership program in Hawaii.
    The NCl/CRCHD supports a partnership program with UOG and CRCH, an 
NCI-designated cancer center at the University of Hawaii at Manoa. 
Engaged in a unique and successful partnership over the past 6 years, 
this program has established a Cancer Research Center of Guam on the 
campus of UOG to (1) increase the number of faculty and students 
engaged in cancer research at UOG; (2) increase the number of minority 
scientists of API ancestry engaged in cancer research, and providing 
pertinent undergraduate, graduate, and postgraduate education and 
training opportunities for API students; (3) further strengthen the 
research focus at CRCH on cancer health disparities with particular 
emphasis on aspects of particular relevance for the people of Hawaii 
and the Pacific; and (4) enhance the awareness of cancer and cancer 
prevention and, ultimately, to reduce the impact of cancer on the 
population in the Territory of Guam, the other U.S.-associated Pacific 
island territories, and Hawaii.
CURE
    The CURE program is a strategic approach for training a diverse 
generation of competitive cancer researchers. The CURE provides 
educational support to students and junior investigators from high 
school through postdoctoral studies and mentors them in the early 
phases of their careers in cancer research. This approach builds on the 
success of the research supplements to promote diversity and 
strategically addresses each level of the biomedical research and 
education pipeline to increase the pool of researchers from underserved 
populations. There are currently 14 high school and undergraduate 
students being supported by a CURE supplement in Hawaii.
Diversity Supplements
    These research supplements are designed to foster diversity in the 
research workforce. They support and recruit students, postdoctoral, 
and eligible investigators from groups shown to be underrepresented in 
biomedical research. There are currently two junior investigators being 
supported by diversity supplements in Hawaii.
NCCCP
    Another program within NCI addressing health disparities is the 
NCCCP program. The NCCCP is designed to create a community-based cancer 
center network to support basic, clinical and population-based research 
initiatives, addressing the full cancer care continuum--from 
prevention, screening, diagnosis, treatment, and survivorship through 
end-of-life care. The NCCCP has seven major focus areas to: (1) improve 
access to cancer screening, treatment, and research; (2) improve 
quality of care at community hospitals; (3) increase participation in 
clinical trials; (4) enhance cancer survivorship and palliative care 
services; (5) participate in biospecimen research initiatives to 
support personalized medicine; (6) expand use of electronic health 
records and connect to cancer research data network; and (7) enhance 
cancer advocacy.
    Reducing and eliminating cancer disparities continues to be a major 
commitment for NCI, the research community, healthcare providers and 
policymakers. In recent years, the cancer research community has also 
begun to focus on understanding why members of some population groups 
experience higher cancer incidence and mortality rates than others.

                            CANCER RESEARCH

    Question. Hawaiian researchers have been very effective in 
addressing the unequal burden of cancer among native Hawaiians; however 
Hawaiian researchers are not equally represented in the researcher 
pool. How will the administration demonstrate its long-term commitment 
to programs like 'Imi Hale that address disparities at all levels and 
identify, mentor, and provide research training, fellowships and grant 
opportunities to native Hawaiians interested in cancer research?
    Answer. The NIH continues to promote its diversity programs to 
underrepresented individuals at the college, graduate school, 
postdoctoral, and faculty stages of a scientist's career. Native 
Hawaiians are a key target group within these programs. Examining NIH's 
efforts in its formal research training programs at the pre- and 
postdoctoral levels, the most recent data from 2007 are encouraging 
regarding native Hawaiians and APIs. They show that 4 percent of NIH 
trainees self-identified as native Hawaiian and APIs, which is higher 
than the proportion of this group in the total U.S. population.
    The challenge is to retain and sustain these individuals as they 
transition into their independent research careers. NIH has several key 
programs in place that are aimed at addressing this challenge. 
Specifically, CNP (http://grants.nih.gov/grants/guide/rfa-files/rfa-ca-
09-032.html) is designed to support community-based participatory 
research in underserved populations and provide a training venue for 
preparing a new cadre of scientists to address health disparities 
research. Second, new initiatives in research in cancer health 
disparities (http://grants.nih.gov/grants/guide/pafiles/PAR-09-160.html 
and http://grants.nih.gov/grants/guide/pa-files/PAR-09-161.html) are 
also designed to provide a venue for young scientists to prepare for 
careers in health disparities research. MI/CCP between the University 
of Hawaii and UOG, and community-based programs, including the 'Imi 
Hale Native Hawaiian Cancer Network supported by the NCI, are dedicated 
to health disparities research in the Hawaii and Pacific region.
    Finally, native Hawaiians and APIs are encouraged to apply for the 
Diversity Supplement to Research Grants Program (http://grants.nih.gov/
grants/guide/pa-files/PA0908190.html) both on the Mainland and in 
Hawaii. This program has supported more than 500 APIs at stages of 
their careers ranging from college education to faculty research 
scientists. NIH intends to continue its support for all of these 
programs.

                              TUBERCULOSIS

    Question. Dr. Collins, thank you for your continuing leadership on 
biomedical research issues. I would like to turn for a moment to 
tuberculosis (TB), one of the oldest diseases known to mankind. As you 
know, TB continues to impact millions of people around the world, 
including in my home State of Hawaii, which has the highest rates of TB 
in the Nation: 128 cases in 2008 or a rate of 9.6 per 100,000 
Hawaiians. Further, complicating this already serious situation is the 
20 percent increase Hawaii has experienced in the more difficult and 
expensive to treat multidrug resistant forms of TB, in part because of 
the decades that have passed since new treatments have been developed. 
Could you give me an overview of the research initiatives NIH is 
currently undertaking to address the drug resistant forms of TB.
    Answer. TB research at NIH is primarily conducted and supported by 
the National Institute of Allergy and Infectious Diseases (NIAID). 
Through grants and other mechanisms and through its intramural research 
program, NIAID supports a globally relevant TB research agenda. NIAID 
TB research is focused on all aspects of TB, including drug-susceptible 
and drug-resistant TB, as well as TB in HIV co-infected persons. NIAID-
sponsored basic TB research includes studies to better understand the 
biology of TB and the host-pathogen interaction, including latent TB 
infection in human hosts and in animal models of infection and disease. 
NIAID-supported translational and clinical research is focused on the 
identification and development of new diagnostics, drugs, and vaccines. 
To better understand TB in special populations, NIAID's research agenda 
includes studies of TB in children and immune suppressed persons as 
well as studies to clarify the interaction of HIV and TB to improve TB 
prevention and treatment. To date, NIAID's investment in basic, 
translational, and clinical science has led to the development of 
several new candidate TB drugs, diagnostics, and vaccines. In addition, 
the NIAID developed a research agenda in fiscal year 2008, the NIAID 
Research Agenda for Multidrug-Resistant and Extensively Drug-Resistant 
Tuberculosis (MDR/XDR-TB), to complement and leverage ongoing efforts 
and focus on specific research gaps for MDR/XDRTB.
    Specific NIAID research activities include the following:
    Research on the pharmacological basis of drug resistance in 
infectious diseases.
  --Studies to characterize drug-resistant TB strains, their 
        epidemiology and their impact on disease progression, host 
        immune response, and response to therapy.
  --An initiative in fiscal year 2010 to support targeted clinical 
        trials to evaluate and improve the optimal use of currently 
        existing therapies for TB and support for phase I clinical 
        studies of new TB drug candidates.
  --Intramural and extramural studies of a multitude of international 
        basic science, translational, diagnostic, and clinical research 
        activities to better characterize drug-resistant TB and gain 
        insight into what specific healthcare interventions need to be 
        developed to combat and prevent drug-resistant TB.
  --Collaborations with the HIV/AIDS clinical trials networks to expand 
        studies of drug- sensitive and drug-resistant TB as a co-
        infection in patients with HIV/AIDS, enhance the capacity for 
        international clinical trials on TB, and increase efforts to 
        combat the co-epidemics of TB and HIV.
  --An intramural research program project at the South Korean Masan 
        National Tuberculosis Hospital, which cares for the largest 
        population of MDR-TB inpatients in the world, to study the 
        natural history of MDR-TB and the occurrence of extensively 
        drug-resistant TB (XDR-TB) in patients who have completely 
        failed chemotherapy.
  --Coordination of drug-sensitive and drug-resistant TB research 
        activities with other Federal agencies through the U.S. TB Task 
        Force, as well as with other Government and nongovernmental 
        organizations such as the WHO/Stop TB Partnership, programs 
        funded by the Bill & Melinda Gates Foundation, and not-for-
        profit product development partnerships.

                UNDERREPRESENTED BIOMEDICAL RESEARCHERS

    Question. For the past 19 years, the Distance Learning Center has 
been pioneering a new training paradigm, the STEMPREP Project, to 
create the next generation of researchers from native Hawaiian and 
other underrepresented minority students. The project provides an 
earlier start in the training pipeline (7th grade) to a national pool 
of minority child prodigies who desire a career in STEM and medicine. 
As we continue our efforts to reduce and ultimately eliminate the 
racial and ethnic health disparities that plague our healthcare system, 
we must support a generation of physician scientists and researchers 
who have the skills to develop sound public health solutions and 
advance public health through scientific discovery. How will the 
administration demonstrate its commitment to programs like the 
Physician Scientist Training Program that has called for an increase in 
the supply of biomedical researchers from underrepresented racial and 
ethnic minority populations?
    Answer. The NIH has a history of creating and supporting policies 
and programs with the goal of promoting and providing a diverse 
workforce in the biomedical, behavioral, clinical, and social sciences. 
NIH programs are designed to recruit, train, retain, and develop the 
careers of underrepresented individuals, and every NIH research 
training, fellowship, career development, and research education 
project award Funding Opportunity Announcement explicitly States this 
policy. A number of programs target talented science undergraduates by 
providing funds for their college tuition and a stipend for living 
expenses to promote their pursuit of a career in biomedicine. At the 
doctoral level of education, the NIH awards fellowships, traineeships, 
and research grant supplements to individuals in support of their 
studies toward the research doctorate degree. At the postdoctoral 
level, NIH offers fellowships, career development, and research grant 
supplements to promote the transition of young scientists to 
independent investigators.
    In terms of a commitment to providing a diverse workforce in the 
future, the NIH continues to evaluate and explore new and creative 
programs to promote a diverse workforce. Most recently, the NIH has 
committed ARRA funds to support the NIH Director's Pathfinder Award to 
Promote Diversity in the Scientific Workforce (DP4) which was announced 
on March 5, 2010. This new research grant program encourages 
exceptionally creative individual scientists to develop highly 
innovative approaches for promoting diversity within the biomedical 
research workforce. The proposed research must reflect ideas 
substantially different from those already being pursued or apply 
existing research designs in new and innovative ways to unambiguously 
identify factors that will improve the retention of students, postdocs 
and faculty from diverse backgrounds in the workforce (http://
grants.nih.gov/grants/guide/rfa-files/RFA-OD-10-013.html).
    New studies and grant programs are also underway to identify 
barriers to underrepresented individuals being incorporated into the 
biomedical workforce and to more effectively address those barriers. 
The National Institute of General Medical Sciences has launched two new 
research grant programs to explore the development of new interventions 
to improve diversity (http://grants.nih.gov/grants/guide/rfa-files/RFA-
GM-10-008.html and http://grants.nih.gov/grants/guide/rfa-files/RFA-GM-
09-011.html).
    In addition, the Office of the Director is undertaking studies to 
more explicitly identify attrition points along the pathway between 
high school and achieving independence as a biomedical scientist. 
Relating this information to variables such as race, ethnicity and 
gender should enable NIH to target interventions more selectively and 
improve our ability to recruit and retain a diverse population of 
researchers.
                                 ______
                                 
              Questions Submitted by Senator Arlen Specter

                       CURES ACCELERATION NETWORK

    Question. Moving the new authorized Cures Acceleration Network 
(CAN) forward is of critical importance. What would the timeline be for 
getting the program started if funding is provided?
    Answer. If funding is provided, the first step would be to appoint 
CAN's advisory board and identify priority areas. After this, the 
National Institutes of Health (NIH) would prepare grant and contract 
solicitation announcements within approximately 2 months of the first 
board meeting. Applicants would be given 60 days to prepare 
applications in response to the solicitation(s). The application 
reviews would occur within several weeks following receipt, and awards 
made rapidly thereafter. Under this timetable, we would expect to 
disburse awards within the first year.

                            CLINICAL CENTER

    Question. What is the current number of patients being treated at 
the Mark O. Hatfield Clinical Research Center in Bethesda? As the 
largest clinical research hospital in the world, what capacity is it? 
If it is not at full capacity when do you anticipate that it will be?
    Answer. As of May 26, 2010, the Mark O. Hatfield Clinical Research 
Center has seen 17,450 patients in the inpatient and outpatient 
settings; approximately 38,000 inpatient days and 61,000 outpatient 
visits this fiscal year. The current inpatient capacity at the Mark O. 
Hatfield Clinical Research Center is 234 beds. A new 6-bed high 
containment unit that will allow us to study patients with infectious 
diseases is scheduled to open shortly and will increase the Center's 
total capacity to 240 beds.
    In fiscal year 2010, the Mark O. Hatfield Clinical Research Center 
has been operating at an average daily census of 166 inpatients per day 
which represents an occupancy level of approximately 70 percent. Based 
on plans that the Institutes are making fiscal year 2011, we anticipate 
an increase in inpatient activity of approximately 2 percent more than 
fiscal year 2010. In addition, NIH leaders are exploring the 
feasibility of opening the Mark O. Hatfield Clinical Research Center to 
the outside research community, and discussions are underway with the 
NIH Scientific Management Review Board. Such a change could lead to 
increased utilization.

                           PANCREATIC CANCER

    Question. Pancreatic cancer research accounts for only about 2 
percent of NIH's budget, even though it is the forth leading cancer 
killer and has one of the lowest survival rates. What can be done to 
increase funding?
    Answer. Since the publication of Pancreatic Cancer: An Agenda for 
Action in 2001, the National Cancer Institute (NCI) has expanded its 
portfolio of pancreatic cancer research from $21.8 million in fiscal 
year 2001 to $89.7 million in fiscal year 2009, an increase of more 
than 300 percent. During this period, the total NCI budget increased by 
about 30 percent; thus, the growth in the pancreatic cancer portfolio 
has been approximately tenfold larger than the growth in the total NCI 
budget. As documented in Pancreatic Cancer: Six Years of Progress in 
2007, the NCI pancreatic cancer research portfolio has grown within 
each of the six major research priority areas identified in 2001.
    In addition to an increase in funding, there have also been 
increases in the number of projects funded (up more than 275 percent 
since fiscal year 2000), unique RO1 Grant Principal Investigators 
funded (up more than 200 percent since fiscal year 2000), and training/
career development awards (up more than 65 percent since fiscal year 
2005). Part of the growth came about through planned actions and 
funding opportunities specific to pancreatic cancer, and part grew out 
of an increasingly larger pool of pancreatic cancer researchers 
successfully competing for general funding opportunities and 
unsolicited research grants.
    In addition, pancreatic cancer projects were also funded through 
the American Recovery and Reinvestment Act of 2009 (ARRA). In fiscal 
year 2009, 79 pancreatic cancer-related projects received ARRA funding 
totaling $10.7 million. These projects include some focused on 
training/career development that are relevant to growing the critical 
mass of pancreatic cancer investigators, a group of traditional RO1 
research grants, a Challenge Grant, and a Grand Opportunity or ``GO'' 
grant. The NCI Community Cancer Centers Program, a group already 
working on pancreatic cancer, has been further developed with ARRA 
funds. The ACTNOW initiative, which supports high-priority, early-phase 
clinical trials of new cancer treatments on an accelerated timeline 
includes a clinical trial addressing pancreatic cancer. Finally, The 
Cancer Genome Atlas project (TCGA) is using ARRA funds to rapidly 
increase the number of cancers covered by the project, including 
pancreatic cancer. ARRA has provided a unique opportunity to accelerate 
progress in pancreatic cancer research.
    NCI has focused considerable expertise on assessing the state of 
the science in pancreatic cancer and developing a targeted network of 
pancreatic cancer experts for consultation with NCI program staff In 
2006, NCI created a Gastrointestinal Cancer Steering Committee (GISC) 
with seven specific disease-site task forces, including one focused on 
pancreatic cancer. GISC members include all Cooperative Group 
gastrointestinal disease committee chairs, representatives from the 
Specialized Programs of Research Excellence (SPOREs), Cancer Center and 
R01/P01 investigators, along with community oncologists, 
biostatisticians, patient advocates and NCI staff. Through GISC, NCI 
convened a Pancreas State of-the-Clinical Science meeting in 2007 to 
discuss the integration of basic and clinical knowledge into the design 
of clinical trials for pancreatic cancer and to define the direction 
for clinical trials investigation for pancreatic cancer over the next 3 
to 5 years. A Consensus Report from the meeting, published in the 
Journal of Clinical Oncology in November 2009, emphasized the 
importance of enhanced molecular targets and targeted drugs for 
pancreatic cancer, better preclinical models, and improved phase II 
studies. The GISC is an active part of NCI's programmatic development 
for pancreatic and other gastrointestinal cancers. The GISC' s 
pancreatic cancer task force provides important leadership, meeting on 
a monthly basis to coordinate strategy between the cooperative groups, 
identifying new leads to explore, and monitoring ongoing trials. Within 
the pancreatic cancer task force, a working group has been created to 
focus on development of trials for locally advanced disease. In 
addition, as part of the operational efficiency working group 
guidelines for the development of clinical trials, the pancreatic 
cancer task force is now operating under an accelerated timeline for 
the development of phase II and III clinical trials.
    Finally, in response to earlier congressional language, NCI will be 
holding an internal meeting this summer to discuss research and 
training initiatives relevant to pancreatic cancer.
    Question. In 2001, NCI developed a set of 39 recommendations for 
increasing pancreatic cancer research, including attracting more 
scientists to this field of study. Nine years later, only five of its 
own recommendations have been implemented. Over the same time period 
the NCI's budget has grown by more than $1 billion, so it's not a 
question of funds being available. Given the fact that pancreatic 
cancer deaths are increasing, what concrete steps will you take to make 
this field of study a higher priority?
    Answer. Since the publication of Pancreatic Cancer: An Agenda for 
Action in 2001, the NCI has expanded its portfolio of pancreatic cancer 
research from $21.8 million in fiscal year 2001 to $89.7 million in 
fiscal year 2009, an increase of more than 300 percent. During this 
period, the total NCI budget increased by about 30 percent; thus, the 
growth in the pancreatic cancer portfolio has been approximately 
tenfold larger than the growth in the total NCI budget. As documented 
in Pancreatic Cancer: Six Years of Progress in 2007, the NCI pancreatic 
cancer research portfolio has grown within each of the six major 
research priority areas identified in 2001.
    A genome-wide association study to uncover the causes of pancreatic 
cancer, known as PanScan, has identified five important genetic regions 
that greatly influence the risk of developing pancreatic cancer. NCI is 
now focused in detail on each of these genetic risk regions. NCI is 
active in the Pancreatic Cancer Genetic Epidemiology Consortium, 
founded to examine susceptibility genes in familial pancreatic cancer.
    Other initiatives include the Pancreatic Cancer Cohort Consortium, 
and pancreatic and GI SPOREs. In November 2009, NCI launched one of the 
largest phase III trials ever undertaken in pancreatic cancer (RTOG 
0848), intended to enroll 900 patients to evaluate both Erlotinib and 
chemoradiation as adjuvant treatment.
    Pancreatic cancer studies have been funded within the Cancer 
Nanotechnology Platform Partnerships, the Early Detection Research 
Network, and the Tumor Glycome Laboratories of the NIH Alliance of 
Glycobiologists for Detection of Cancer and Cancer Risk. NCI is 
collaborating with the Pancreatic Cancer Action Network (PanCAN) and 
the Lustgarten Foundation for Pancreatic Cancer research on the 
Pancreatic Cancer Research Map. This project facilitates collaborations 
among pancreatic cancer researchers to speed the development of 
national strategies, and leverage resources for pancreatic cancer 
research. The map provides a unified collection of pancreatic cancer 
research projects, funding opportunities, and investigators.
                                 ______
                                 
              Questions Submitted by Senator Thad Cochran

                     SPINAL MUSCULAR ATROPHY (SMA)

    Question. What role can the National Institutes of Health (NIH) 
play in laying the groundwork for SMA and to develop new therapies and 
work with the Food and Drug Administration (FDA) to support new 
therapies? Please update the subcommittee on what are the next steps 
that NIH is planning to take to prepare for, support and sustain the 
efforts that will be necessary up to and through clinical trials for 
SMA?
    Answer. Due to NIH's continued investment in SMA research, 
including studies on disease mechanisms and preclinical/translational 
therapy development, the first treatments for SMA are now advancing 
through the therapeutic development pipeline. The NIH has taken a 
number of steps to continue to support development of potential 
treatments up to and through clinical trials.
    NIH supports a variety of projects for translating basic research 
findings into therapies that can be tested in a clinical setting. The 
SMA Project, funded by the NIH and guided by experts from industry, 
academia, NIH, and the FDA, is an innovative, contract-based, 
``virtual-pharma'' program to develop drugs and test them in the 
laboratory. The project holds two patents on two sets of compounds that 
show significant promise and, assuming successful preclinical testing, 
a phase I clinical trial to assess safety should begin in 2011. The 
project is also continuing to pursue other leads.
    To complement the SMA project, the NIH also funds investigator-
initiated therapy development projects. This year, National Institute 
of Neurological Disorders and Stroke (NINDS) began funding a major 
milestone-driven collaboration between an academic lab and a biotech 
company to develop a lead compound into a drug that is ready for 
clinical testing in SMA patients. An investigator-initiated grant 
funded by the National Institute of Child Health and Human Development 
is designed to assess the natural history of the disease and perform 
pilot studies to evaluate potential interventions in a broad cohort of 
SMA patients. Additionally, NIH has used American Recovery and 
Reinvestment Act (ARRA) funds to make investments in rapidly developing 
opportunities, including a Grand Opportunity grant on delivery of 
therapeutic genes for motor neuron diseases. Stem cell research 
relevant to SMA has also been funded, including studies of induced 
pluripotent stem cells derived from SMA patients.
    NIH has also made a commitment to support high-quality clinical 
trials for SMA and other pediatric disorders. In February, the NINDS 
Council approved NINDS-NET, a multi-site clinical research network to 
expedite early phase clinical trials of therapies from academic 
research, foundations, or biotech companies. Because all network 
participants are required to have expertise in clinical trials for 
pediatric neurological disorders as well as adult diseases, this 
clinical research network provides the framework for high-quality 
trials for SMA and other rare disorders.
    The NIH, working with SMA volunteer organizations, has organized a 
workshop for later this year that will focus on therapies that are 
approaching readiness for clinical testing, what hurdles remain, and 
what is needed for effective SMA clinical trials. A second workshop, 
organized by both the NIH and FDA, will address specifically the use of 
anti-sense oligonucleotides in treating neuromuscular disorders 
including SMA, and will provide FDA input into clinical and preclinical 
studies. Both of these workshops will not only facilitate communication 
among SMA researchers, NIH, and the FDA, but will also help the 
research community plan for moving therapies into clinical trials.

                            CROHN'S DISEASE

    Question. Dr. Collins, I want to thank you and the leadership of 
the National Institute of Diabetes and Digestive and Kidney Diseases 
for advancing research on Crohn's disease and ulcerative colitis. As 
you know, these are extremely painful and debilitating disorders that 
are increasing in prevalence. Can you tell us what needs to be done to 
translate the remarkable genetic discoveries of recent years into 
better treatments for patients?
    Answer. The NIH support for research on the genetics of Crohn's 
disease and ulcerative colitis--the two major forms of inflammatory 
bowel diseases (IBD)--is providing the foundation for the development 
of unique and effective therapies for patients who suffer from these 
diseases. Following the discovery of the first IBD-associated gene, the 
NIH established a major program in 2002--the IBD Genetics Consortium--
to accelerate the discovery of genetic variants that are associated 
with the disease. To date, this very successful program has uncovered 
nearly 50 genetic variants that are associated with both major forms of 
IBD. Progress in this area was bolstered by recent investments from 
ARRA, which provided additional support for the consortium to enhance 
its ability to expand and develop resources. In addition, ARRA 
supported innovative projects to identify genetic variations that are 
less common amongst people with Crohn's disease and extend the success 
of genome wide association studies to identify genetic variations that 
predispose individuals from different ethnic groups to developing IBD. 
As researchers continue to discover additional genetic variants 
associated with IBD, it will be important for these advances to be 
translated into better treatments for patients. Through ARRA and 
regular appropriations, the NIH is supporting research to define the 
biological processes that are perturbed by genetic variants associated 
with IBD. In some cases, genetic variants that have limited direct 
associations with IBD may have significant biological consequences, and 
it will be important to consider these factors when developing models 
of disease risk. By further understanding the genetic variants 
associated with disease and their molecular consequences, researchers 
will be able to develop and validate biomarkers as indicators of 
disease risk, disease prognosis, and patient responses to therapies. In 
addition, as the biological pathways underlying IBD are better defined, 
researchers will identify targets for developing new therapeutics to 
help treat these painful and debilitating disorders.

                            MINORITY HEALTH

    Question. How will the new data collection requirements on race and 
ethnicity, primary language, geographic location, and disability status 
affect research at NIH? How will this information be used? Are you 
collaborating with the existing Department of Health and Human 
Services, Office of Minority Health (OMH) in order to coordinate and 
establish an effective Government effort to address minority health 
issues?
    Answer. The new data collection requirements will advance NIH's 
research-based efforts for improving the health of the Nation. The 
limited specificity, uniformity and quality of data collection and 
reporting procedures has been a significant restraint in identifying 
and monitoring efforts to reduce health disparities. According to a 
recent report by the Institute of Medicine (IOM) ``Race, Ethnicity, and 
Language Data: Standardization for Health Care Quality Improvement, 
``from the Subcommittee on Standardized Collection of Race/Ethnicity 
Data for Healthcare Quality Improvement,'' consistent methods for 
collecting and reporting healthcare data on racial and ethnic 
minorities are essential to informing evidence-based disparity 
reduction initiatives.
    In addition, as the demographics of the United States continue to 
shift, it is essential to understand the diversity of the Nation based 
on race, ethnicity, primary language, and disability status. Collecting 
information on the geographic distributions of racial and ethnic 
populations will aid researchers in understanding how geographic 
location and environmental factors for example, contribute to the 
existence and persistence of health disparities. During the past 10 
years there has been a growing appreciation of the role these factors 
play in health disparities. Collecting this data will assist 
researchers in understanding how these factors, working independently 
and dependently, contribute to the excess burden of disease, morbidity, 
and mortality experienced by racial and ethnic minorities relative to 
majority populations.
    This enhanced data collection will be useful in clinical research, 
especially in Comparative Effectiveness Research, where there will be 
the need to collect information on these racial and ethnic subgroups to 
produce statistically reliable evidence-based results. Statistical 
oversampling of certain subpopulations in clinical comparative 
effectiveness research will be done as needed. In addition to improving 
data collection across Federal categories of race and ethnicity, 
information is needed on racial and ethnic subgroups. This new data 
collection will be critical to monitoring the health status and needs 
of immigrant and language minority populations. This calculates to 
approximately 100 different ethnic groups with populations more than 
100,000 living in the United States.
    Health disparities are persistent and eliminating them requires an 
in-depth understanding of how multiple factors--social and biological--
act independently and dependently. Collecting information on race, 
ethnicity, primary language, disability status, and geographic location 
will allow researchers to better understand these factors and their 
interactions. Scientists will use it to design interventions tailored 
to meet the needs of racial and ethnic populations as a function of 
primary language or geographic location, or other factors.
    The NIH, through the National Center on Minority Health and Health 
Disparities (NCMHD), has had a long-standing tradition of collaboration 
and coordination of minority health and health disparities activities 
with the HHS OMH. Over the years the NCMHD and OMH have worked 
collaboratively to address a number of minority health issues both 
domestically and internationally, as well as support several minority 
health initiatives with funding from some of the Institutes and 
Centers. Most recently, the NIH has participated in:
  --The development of the HHS National Partnership Action Plan led by 
        OMH;
  --NIH is represented on the HHS Health Disparities Council which 
        deals with minority health and health disparities issues across 
        the HHS and for some time has been led by the OMH;
  --NCMHD and OMH are collaborating on an ARRA initiative to develop 
        Centers of Excellence for Comparative Effectiveness Research 
        through the NCMHD Centers of Excellence; and
  --NCMHD and OMH serve as two of three Federal Government co-leads for 
        the Federal Collaboration on Health Disparities Research 
        (FCHDR) which is aimed at enhancing wide Federal Government 
        coordination around minority health and health disparities.

                 INSTITUTIONAL DEVELOPMENT AWARD (IDEA)

    Question. Does the list of eligible States ever change to reflect 
their greater or lesser success over time in attracting competitive NIH 
research funding?
    Answer. When Congress authorized the Institutional Development 
Award (IDeA) program in 1993, its intent was to promote geographic 
distribution of NIH funding across the United States. in order to 
increase the research capacity in eligible States. The eligibility to 
participate in the IDeA program has been evaluated on a yearly basis 
and the list of eligible States has not changed over the years with the 
exception of Alabama, which was once an IDeA eligible State that became 
ineligible based on its success in obtaining NIH funding. The current 
list of IDeA eligible States can be found on the National Center for 
Research Resources' (NCRR) Web site at http://www.ncrr.nih.gov/
research_infrastructure/institutional _development_award/.
    The current IDeA eligibility criteria are based on two components: 
(1) a success rate for competing research projects and centers of less 
than 20 percent for obtaining NIH grant awards during 2001-2005; or (2) 
less than $120 million average NIH funding during 2001-2005 (regardless 
of success rate), excluding IDeA awards and R&D contracts.
    NCRR is currently evaluating whether the IDeA eligibility criteria 
are still appropriate to accomplish the legislative intent. As it does 
so, the eligibility criteria and the IDeA-eligible States will remain 
the same.
                                 ______
                                 
            Questions Submitted by Senator Richard C. Shelby

                               BIODEFENSE

    Question. In National Institute of Allergy and Infectious Diseases 
(NIAID)'s Strategic Plan for Biodefense Research 2007 Update, NIAID 
outlined three ``broad spectrum'' strategies as a way to maximize 
biodefense capabilities. One of these strategies was the exploration of 
broad spectrum platforms, which NIAID describes as standardized methods 
that can be used to significantly reduce the time and cost required to 
bring medical countermeasures to market. Please explain how much 
funding has been spent in this area and what milestones have been 
reached.
    Answer. NIAID's product development strategy has broadened from a 
``one bug-one drug'' approach toward a more flexible, broad-spectrum 
approach. This process involves developing medical countermeasures that 
are effective against a variety of pathogens and toxins, developing 
technologies that can be widely applied to improve classes of products, 
and establishing platforms that can reduce the time and cost of 
creating new products. The broad-spectrum strategy recognizes both the 
expanding range of biological threats and the limited resources 
available to address each individual threat. NIAID provided $653 
million in fiscal year 2009 to a number of initiatives that have the 
potential to lead to the development of broad spectrum platforms. 
Examples of milestones in the development of broad-spectrum strategies 
that have been facilitated by NIAID funding include:
  --The preclinical development of AdvaxTM, a vaccine 
        adjuvant platform technology. AdvaxTM has been 
        approved for human use in Australia for at least five different 
        candidate vaccines and currently is being tested in seasonal 
        and pandemic influenza vaccines and hepatitis B vaccines that 
        are ready to enter phase III clinical trials.
  --The development of LJ001, a broad-spectrum antiviral that has shown 
        activity against multiple viruses, including influenza, Ebola, 
        Marburg, hepatitis C, and West Nile.
  --Syntiron's broad-spectrum vaccine technology that is currently used 
        for candidate vaccines for Staphylococcus, Salmonella, plague, 
        and anthrax.

                               BIODEFENSE

    Question. Specifically, equine source plasma has been successfully 
used in the development of passive antibody therapy for postexposure 
treatment of agents such as botulinum toxin. I understand this same 
technique can be used for treatment of a number of the Category A 
biological threat agents such as Bacillus anthracis, hemorrhagic fevers 
(i.e., Ebola and Marburg), and Yersinia pestis. Is NIAID familiar with 
this platform of therapeutics and its successes? Has NIAID applied 
funding either from within its directly appropriated funds or from 
BARDA transferred funds to the development of passive antibody 
therapeutics? If so how much and on what projects?
    Answer. NIAID is significantly involved in the development and use 
of passive antibody therapy for postexposure treatment of agents such 
as botulinum toxin and has provided more than $92 million in funding 
over the past 3 years for the development of passive antibody therapy 
for Category A agents. Among other efforts, NIAID supported the 
development of the botulinum toxoid antibody from horses for a product 
that is now included in the Strategic National Stockpile; coordinated 
with the Biomedical Advanced Research and Development Authority (BARDA) 
for development of animal models in support of licensure of botulinum 
anti-toxins; and supported initial work to develop ricin polyclonal 
antibodies from equine antisera.

                         CONCLUSION OF HEARINGS

    Senator Harkin. The subcommittee will stand recessed.
    [Whereupon, at 11:05 a.m., Wednesday, May 5, the hearings 
were concluded, and the subcommittee was recessed, to reconvene 
subject to the call of the Chair.]
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