[House Hearing, 108 Congress]
[From the U.S. Government Publishing Office]



   ASSESSING DIGESTIVE DISEASES RESEARCH AND TREATMENT OPPORTUNITIES

=======================================================================

                                HEARING

                               before the

                         SUBCOMMITTEE ON HEALTH

                                 of the

                    COMMITTEE ON ENERGY AND COMMERCE
                        HOUSE OF REPRESENTATIVES

                      ONE HUNDRED EIGHTH CONGRESS

                             SECOND SESSION

                               __________

                              JULY 8, 2004

                               __________

                           Serial No. 108-94

                               __________

       Printed for the use of the Committee on Energy and Commerce


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

                    COMMITTEE ON ENERGY AND COMMERCE

                      JOE BARTON, Texas, Chairman

W.J. ``BILLY'' TAUZIN, Louisiana     JOHN D. DINGELL, Michigan
RALPH M. HALL, Texas                   Ranking Member
MICHAEL BILIRAKIS, Florida           HENRY A. WAXMAN, California
FRED UPTON, Michigan                 EDWARD J. MARKEY, Massachusetts
CLIFF STEARNS, Florida               RICK BOUCHER, Virginia
PAUL E. GILLMOR, Ohio                EDOLPHUS TOWNS, New York
JAMES C. GREENWOOD, Pennsylvania     FRANK PALLONE, Jr., New Jersey
CHRISTOPHER COX, California          SHERROD BROWN, Ohio
NATHAN DEAL, Georgia                 BART GORDON, Tennessee
RICHARD BURR, North Carolina         PETER DEUTSCH, Florida
ED WHITFIELD, Kentucky               BOBBY L. RUSH, Illinois
CHARLIE NORWOOD, Georgia             ANNA G. ESHOO, California
BARBARA CUBIN, Wyoming               BART STUPAK, Michigan
JOHN SHIMKUS, Illinois               ELIOT L. ENGEL, New York
HEATHER WILSON, New Mexico           ALBERT R. WYNN, Maryland
JOHN B. SHADEGG, Arizona             GENE GREEN, Texas
CHARLES W. ``CHIP'' PICKERING,       KAREN McCARTHY, Missouri
Mississippi, Vice Chairman           TED STRICKLAND, Ohio
VITO FOSSELLA, New York              DIANA DeGETTE, Colorado
STEVE BUYER, Indiana                 LOIS CAPPS, California
GEORGE RADANOVICH, California        MICHAEL F. DOYLE, Pennsylvania
CHARLES F. BASS, New Hampshire       CHRISTOPHER JOHN, Louisiana
JOSEPH R. PITTS, Pennsylvania        TOM ALLEN, Maine
MARY BONO, California                JIM DAVIS, Florida
GREG WALDEN, Oregon                  JANICE D. SCHAKOWSKY, Illinois
LEE TERRY, Nebraska                  HILDA L. SOLIS, California
MIKE FERGUSON, New Jersey            CHARLES A. GONZALEZ, Texas
MIKE ROGERS, Michigan
DARRELL E. ISSA, California
C.L. ``BUTCH'' OTTER, Idaho
JOHN SULLIVAN, Oklahoma

                      Bud Albright, Staff Director

                   James D. Barnette, General Counsel

      Reid P.F. Stuntz, Minority Staff Director and Chief Counsel

                                 ______

                         Subcommittee on Health

                  MICHAEL BILIRAKIS, Florida, Chairman

RALPH M. HALL, Texas                 SHERROD BROWN, Ohio
FRED UPTON, Michigan                   Ranking Member
JAMES C. GREENWOOD, Pennsylvania     HENRY A. WAXMAN, California
NATHAN DEAL, Georgia                 EDOLPHUS TOWNS, New York
RICHARD BURR, North Carolina         FRANK PALLONE, Jr., New Jersey
ED WHITFIELD, Kentucky               BART GORDON, Tennessee
CHARLIE NORWOOD, Georgia             ANNA G. ESHOO, California
  Vice Chairman                      BART STUPAK, Michigan
BARBARA CUBIN, Wyoming               ELIOT L. ENGEL, New York
JOHN SHIMKUS, Illinois               GENE GREEN, Texas
HEATHER WILSON, New Mexico           TED STRICKLAND, Ohio
JOHN B. SHADEGG, Arizona             DIANA DeGETTE, Colorado
CHARLES W. ``CHIP'' PICKERING,       LOIS CAPPS, California
Mississippi                          CHRIS JOHN, Louisiana
STEVE BUYER, Indiana                 BOBBY L. RUSH, Illinois
JOSEPH R. PITTS, Pennsylvania        JOHN D. DINGELL, Michigan,
MIKE FERGUSON, New Jersey              (Ex Officio)
MIKE ROGERS, Michigan
JOE BARTON, Texas,
  (Ex Officio)

                                  (ii)




                            C O N T E N T S

                               __________
                                                                   Page

Testimony of:
    Carron, Adam.................................................    21
    DeRose, Rodger, President and CEO, Crohn's and Colitis 
      Foundation of America......................................    17
    Peura, David, Digestive Disease National Coalition...........    24
    Spiegel, Allen M., Director, National Institute of Diabetes 
      and Digestive and Kidney Diseases..........................    11
Additional material submitted for the record:
    Peura, David, Digestive Disease National Coalition, response 
      for the record.............................................    38

                                 (iii)

  

 
   ASSESSING DIGESTIVE DISEASES RESEARCH AND TREATMENT OPPORTUNITIES

                              ----------                              


                         THURSDAY, JULY 8, 2004

                  House of Representatives,
                  Committee on Energy and Commerce,
                                    Subcommittee on Health,
                                                    Washington, DC.
    The subcommittee met, pursuant to notice, at 11:05 a.m., in 
room 2322, Rayburn House Office Building, Hon. Michael 
Bilirakis (chairman) presiding.
    Members present: Representatives Bilirakis, Upton, Buyer, 
Barton (ex officio), Brown, Pallone, Capps, and Rush.
    Also present: Representative Kelly.
    Staff present: Cheryl Jaeger, majority professional staff; 
Eugenia Edwards, legislative clerk; and John Ford, minority 
counsel.
    Mr. Bilirakis. Good morning.
    Today's hearing assessing digestive disease research and 
treatment opportunities addresses an important issue that is 
often, unfortunately, overlooked.
    Digestive diseases such as ulcers, heartburn, celiac 
disease and inflammatory bowel disease, IBD, effect 
approximately 70 million Americans. Some of these diseases are 
minor and easily manageable while others are debilitating and 
extremely painful. All are increasing, unfortunately, in their 
frequency.
    One example of IBD, Crohn's disease, was once a very rare 
disorder but today there are twice as many cases as there were 
30 years ago, and we are not sure why.
    Now, it is common to hear the personal stories about how 
IBD's affect an individual's family or a close friend. Members 
of my own family, for example, suffer from IBD.
    What also troubles me greatly is that these conditions are 
increasingly afflicting children and teenagers. The health of 
our children is already in crises, especially with the 
skyrocketing obesity rates. Children with digestive diseases 
often have to live with symptoms such as pain, intestinal 
bleeding and weight loss. And that is why I am so honored to 
welcome on of our witnesses here today, Adam Carron who will 
speak about his personal experience with Crohn's disease. Adam, 
really no one should have to endure what you have experienced 
over the years with this terrible illness. You have my 
gratitude and my admiration and that of my colleagues for 
speaking out and educating others on Crohn's disease. It is not 
easy to discuss the terrible ordeals you have gone through, and 
we certainly thank you so very much for being here today.
    We have an excellent panel of witnesses here this morning. 
In addition to Adam, I would like to welcome Dr. William 
Spiegel, Director of the National Institute of Diabetes and 
Digestive and Kidney Disease, NIDDK, within the National 
Institutes of Health.
    NIDDK is the lead institute responsible for the strategic 
planning and priority setting of federally funded research in 
digestive diseases. NIDDK already has highlighted digestive 
diseases and specifically IBD as a priority. And we are, of 
course, very happy to know that. In fact, NIDDK has a center 
for the study of inflammatory bowel disease and a center for 
gastrointestinal biology and diseases which focus on IBD 
research.
    In the 108th Congress, this subcommittee has held 5 
hearings to highlight research activities and budget and 
priority setting at the NIH. Both Chairman Barton and I have 
expressed our commitment to reauthorize the NIH and assisted 
Director of NIH, Dr. Elias Zerhouni to implement his NIH 
Roadmap for Medical Research. During this process I am working 
to ensure that NIDDK has the resources it needs to maintain its 
reputation of excellence in the digestive diseases field, but 
also increases the transparency in the way that research is 
disseminated to the public. After all, what good is research if 
it is not in some good way disseminated to the public.
    I am also excited to have representatives from two 
digestive disease patient advocacy groups. Rodger DeRose is the 
President and CEO of the Crohn's and Colitis Foundation of 
America, CCF. And Dr. David Peura is the Associate Chief in the 
Division of Gastroenterology and Hepatology and Professor of 
internal medicine at the University of Virginia Health Science 
Center. Mr. Peura is testifying on behalf of the Digestive 
Disease National Coalition, DDNC.
    I would like to commend both of your organizations, 
gentlemen, on your work to improve education, access to and 
quality of digestive disease health care for current and future 
patients.
    I cannot stress enough how dedicated I am to increasing 
research and education for digestive diseases. I was fortunate 
to able to speak to CCF last month and I was touched by the 
level of commitment there that there is in the effort to 
fighting these debilitating diseases. Many Members of Congress 
are concerned about digestive diseases, and leading that group 
is our colleague and friend, Congresswoman Sue Kelly and then 
additionally Congressman Roy Blunt and Bobby Rush have 
introduced bills to increase the focus on digestive diseases. 
Ms. Kelly is not a member of this committee, but at my 
invitation and I know without any objection from Mr. Brown or 
the other members, she is here today and is sitting up here as 
a member of the panel, and we certainly welcome her.
    I believe it is important that we work together to find a 
cure and ways to prevent these very serious diseases. At the 
end of the day I know most of our concern is that if we do not 
find a solution, the prevalence of digestive diseases will 
continue to grow.
    Again, thank you for being here today.
    And I gladly yield to the ranking member of the 
subcommittee, my good friend from Ohio Mr. Brown for an opening 
statement.
    Mr. Brown. Thank you, Mr. Chairman. Thank you all the 
witnesses for joining us today.
    The term digestive disorder covers a wide range of 
illnesses, as the chairman said, several of them deadly, all of 
them significant from a public health perspective. Liver 
disease and inflammatory bowel disease or IBD, acid reflux, 
cancer of the stomach and pancreas, of these illnesses and many 
more are considered digestive disorders. More than 62 million 
Americans are diagnosed with one of these illnesses every year.
    I was contacted by Paul Levin, who is the father of Sarah, 
a young woman with Crohn's disease. Crohn's is a form of IBD, 
it is incurable and it is insidious. It causes inflammation of 
the lining of the intestine. When inflamed, the lining becomes 
ulcerated and bleeds. It can cause severe diarrhea, abdominal 
pain, cramping, fever and rectal bleeding.
    Sarah Levin takes 11 medications everyday. She has major 
surgery. She has taken steroids that stunted her growth and 
compromised her physical health. She has been forced to miss 
work again and again because Crohn's can flare up at anytime.
    Regrettably there is no cure at this time. Medical 
treatment can only try to ease the pain and control the 
inflammation.
    Like my digestive disorders, IBD does not get the attention 
it deserves given its prevalence and its disabling effects. 
Approximately 1 million Americans suffer from inflammatory 
bowel disease, and numbers keep increasing as there are about 
twice as many cases today of IBD as there were 30 years ago. 
Sarah and I hope all of us are determined to do about this. 
Along with her father and other advocates she has been lobbying 
very successfully for legislation focusing on IBD. H.R. 290 
sponsored by Sue Kelly and be Jessie Jackson, Jr. would raise 
public awareness about IBD, jump start IBD related research and 
help IBD patients get the treatment that they need. I am 
pleased to support this bill.
    I am interested in learning more about H.R. 3756 which 
would establish a national commission on digestive disorders. A 
commission was formed many years ago in 1976 to get a better 
handle on the prevalence of these diseases and the nature and 
score of available treatments. Obviously medicine has evolved 
significantly over these past three decades. The demographics 
of our nation and relevant lifestyle factors such as diet and 
exercise have also changed over time. If congressional action 
is needed to update the original commission's findings in 
regard to digestive disorders, reinstating the commission is a 
step Congress should consider.
    I would add, Mr. Chairman, as you know the Bush 
Administration budget memo recently leaked to the Washington 
Post revealed the Administration's intent to cut overall 
funding for NIH after the election, I might add, by roughly 
$600 million for fiscal year 2006. If anything, this hearing 
has demonstrated that our national investment in medical 
research is more important than ever. We have no business 
cutting NIH. In fact, we frankly have no business in slowing 
down its growth and its increased spending, which this Congress 
has already done. And next year if President Bush gets his way, 
it will be much more.
    We have, as I said, witnessed a slowdown in funding 
increase and this proposed cut that the President has suggested 
is absolutely going in the wrong direction. I hope, Mr. 
Chairman, we can learn more about that during this hearing.
    Thank you.
    Mr. Bilirakis. The Chair yields to the gentleman from 
Indiana, Mr. Buyer.
    Mr. Buyer. I just want to note for the record, sometimes I 
find it breathless. The record is very clear that when 
Republicans took over Congress we set out as a goal, and it was 
to double the funding of NIH.
    So I almost find it breathless whenever I hear someone ``My 
God, Republicans may be cutting NIH.''
    Mr. Brown. Would the gentleman yield?
    Mr. Buyer. Mr. Brown, I just want to make clear, I have not 
seen this document but it makes it even better when you would 
testify to--you know, and I thank and appreciate the leadership 
the Republicans have done to double NIH funding as we push the 
bounds of science that improve the quality of life of people in 
our country. That is a good thing.
    Mr. Brown, I yield to you.
    Mr. Brown. Yes. Thank you, Mr. Buyer.
    I only mention that that initiative was originally 
President Clinton's, that the Congress then worked with in 
partnership bipartisanally, and now that President Bush has 
decided to make room----
    Mr. Buyer. What initiative was Bill Clinton's?
    Mr. Brown. Well, the initiative of doubling the NIH budget, 
which began in----
    Mr. Buyer. Boy, that is----
    Mr. Bilirakis. Regular order. These are opening statements. 
We are not debating.
    Mr. Buyer. I will just reclaim my time, because I am not--I 
do not revitalize history. I do not rewrite history.
    Mr. Bilirakis. Let us keep our eye on the ball.
    Mr. Buyer. No, no. I----
    Mr. Bilirakis. Now all these good people have come here 
because they feel that we can work together for a good purpose. 
And then we get into partisanship----
    Mr. Buyer. Mr. Chairman, yes, we can work together for good 
purpose.
    Mr. Bilirakis. Well, I would repeat that I was a part of 
that promise that was made that we would double NIH funding----
    Mr. Buyer. That is right.
    Mr. Bilirakis. [continuing] over the next 5 years.
    Mr. Buyer. That is right.
    Mr. Bilirakis. So I do not know whether President Clinton 
had it in his mind to do so, whatever the case may be. I know 
it was the Congress that made that particular version, and we 
fulfilled it. We do not fulfill too many of the promises we 
make up here, unfortunately, but that is certainly what we 
will--we did do and we should be proud of it.
    But I do not think we should be going into partisanship or 
President this or President that. Let us stick to what we are 
supposed to be doing here.
    Mr. Buyer, please continue, sir.
    Mr. Buyer. Well, part of what we are doing here is trying 
to improve society and take care of our people. And when 
private industry do not make certain investment, and if we can 
partner with government, NIH to do that, we all benefit. And 
that is what all that is about.
    I just want you to know that I just feel uncomfortable when 
we have got a very good hearing and people take shots; and that 
is what we just had here. So I just felt the need to correct 
the record.
    I appreciate the witnesses being here today. And this is a 
very important issue. And Chairman is right, this is an issue 
that does not seem to get a lot of attention; you do not read 
on the headlines but it affects a lot of people in our society. 
And we appreciate you being here today.
    Mr. Bilirakis. The Chair recognizes Mr. Pallone for an 
opening statement.
    Mr. Pallone. Thank you, Mr. Chairman, for holding this 
hearing. And as a member of the House Digestive Disease Caucus, 
I understand that these types of disorders are often 
overlooked. And my colleague, Representative Sue Kelly and all 
the members of the Caucus, have been working hard to raise 
awareness of these digestive diseases in Congress. And I 
appreciate that as members of the Health Subcommittee we are 
today affirming our commitment to recognize digestive diseases 
and confront their challenges.
    I wanted to take a moment to specifically mention Crohn's 
disease. Mr. Brown has discussed the disabilities related to 
Crohn's disease, and so I will not repeat them. And I do not 
necessarily want to get into this argument about what President 
Bush is going to do, but I do mention that there is in fact a 
memo that we have expressing the fact that if he is reelected, 
that we will see cuts in NIH. And I hope that my Republican 
colleagues with us in opposing those. But I am not surprised if 
some members on the other side have not seen the document 
because I am sure that the Administration does not want anybody 
to know about it.
    Crohn's disease affects an estimated 1 million Americans. 
Many victims of Crohn's are children and, unfortunately, for 
these children afflicted with the disease Crohn's becomes a 
constant feature of their lives. And I trust we will be 
learning much more about this and other disorders from our 
expert witnesses. But my point today is that Crohn's disease is 
about more than just numbers and biological dysfunction, as I 
learned a number of years ago when I met with a remarkable 
young man named Gideon Sofer. Gideon is a constituent of mine 
from Highland Park, New Jersey and a victim of Crohn's disease. 
When I met him he was 16 years old, and I listened to his story 
about the years it took him to get a proper diagnoses and how 
little is still known about treating Crohn's disease. Despite 
all of the medical and emotional obstacles Crohn's has thrown 
his way, Gideon has refused to sit ideally by. He founded the 
IBD Cure Foundation which works to raise awareness about 
digestive disorders.
    In addition, he was the leading inspiration behind HCON 
resolution 455, a resolution I recently introduced that 
advocates from a commemorative stamp to increase awareness of 
and research for Crohn's. And Gideon has created an online 
petition for the stamp, which has garnered over 4,000 
signatures in just a short time.
    Since I have been privileged to know Gideon Sofer, this 
battle for Crohn's awareness has struck a personal cord with 
me. But the reality is that this disorder and other diseases 
have always been personal because health care is about real 
people and real families who have been dealing with these 
diseases with no understanding of the cause or cure. We must 
work together to spread awareness, increase research and 
improve care. And it is for this reason that I have proposed 
HCON res. 455, and I hope that members of this subcommittee 
will also lend their support to legislation such as H.R. 290 
introduced by Sue Kelly which expands research for IBD. I 
believe these types of legislation represent a step in the 
right direction.
    And, again, I just want to thank the chairman and our 
ranking member for supporting this issue. I think this 
subcommittee and the Digestive Disease Caucus to work together 
in removing some of the harm and enormous burden placed on 
millions of Americans suffering from digestive diseases.
    Thank you. Mr. Chairman.
    Mr. Bilirakis. I thank the gentleman.
    Mr. Upton for an opening statement?
    Mr. Upton. Thank you, Mr. Chairman. I am going to put my 
full statement in for the record. I just want to say a couple 
of things.
    I helped spearhead the bipartisan effort to double the 
funds for the NIH over a 5 year span, a successful effort. And 
I can assure anyone listening out there that there is going to 
be no cut in the NIH. President Bush is absolutely committed to 
increased funding for the NIH, and we have to thank folks like 
Chairman Young on Appropriations and Chairman Bilirakis and 
Chairman Tauzin, and now Chairman Barton for their support for 
the NIH as well. And let me just dispel any myth out there that 
the NIH is going to get cut. It ain't going to happen. My mom 
always told me it ain't right to say ain't, but it ain't going 
to happen.
    I also want to welcome Sue Kelly. I am a co-sponsor of H.R. 
290. It is something that needs to happen.
    I have a dear friend that suffers with Crohn's disease. I 
am anxious to see us get a cure and to extend her lifespan and 
make sure that she and so many others that suffer from that 
disease have a great future ahead of them instead of one that 
is a dark shadow.
    And I appreciate the efforts of Chairman Bilirakis to have 
this hearing. I look forward to being engaged and see 
legislation perhaps come out of it. And, obviously, increase 
the incentives and the encourage the NIH in their great work.
    And I yield back my time.
    Mr. Bilirakis. I thank the gentleman.
    Ms. Capps for an opening statement?
    Ms. Capps. Thank you, Mr. Chairman, for holding this 
hearing, a very important hearing. And I want to salute our 
colleague and welcome Sue Kelly, and thank you for your 
leadership in the legislation.
    A particular thanks to our witnesses for appearing today, 
especially Mr. Carron. Yours is a very important testimony that 
is significant for us to have on the record here in Congress. 
So thank you for being here.
    I did not prepare an opening statement. I look forward to 
the testimony.
    Thank you.
    Mr. Bilirakis. I thank the gentlelady.
    By the way, the opening statement of all members of this 
committee have been a part of the record, obviously.
    Let us see, Mr. Rush. Mr. Rush, who is co-author of one of 
the pieces of legislation regarding these diseases. Bobby, you 
are recognized.
    Mr. Rush. Thank you, Mr. Chairman.
    And to members of the subcommittee, let me begin by 
commending the chairman and my good friend Mr. Bilirakis and 
the ranking member for their participation in this hearing and 
for the outstanding work. And I want to thank you for convening 
this hearing.
    I have been long concerned about the devastating toll that 
digestive diseases and disorders have taken on our country. 
Each year more than 62 million of our citizens are diagnosed 
with a wide range of gastrointestinal disorders ranging from 
the inflammatory bowel disease to acid reflux disease, to GI 
cancers; just to name a few of them.
    A study conducted by the Lewin Group in 2000 concluded that 
the direct and indirect costs of just 17 of these diseases 
exceeds $41 billion, however the human pain and suffering 
associated with these conditions are enormous and defy 
quantifying.
    In the State of Illinois the screening rates for the most 
serious forms of GI diseases is low, and as a result the 
mortality and morbidity rates from these diseases are much too 
high.
    I am encouraged by the progress that have been made in some 
areas of GI research and treatment. Yet, on the other hand, I 
find it disheartening that with respect to many of these 
afflictions we still lack even a basic understanding of the 
causes and the treatments of these dreaded diseases.
    I am convinced that what we need is an energized and 
coordinated focus to digestive diseases research. And to this 
end, Mr. Blunt and I have introduced H.R. 3756, the National 
Commission on Digestive Diseases Act of 2004, which we urge the 
committee to favorably consider. This bill will direct the 
Secretary of HHS to convene a national commission on digestive 
diseases to be composed of the leading scientists in the field, 
physician specialists who treat patients in hospitals, clinics 
and offices everyday, patients who suffer from these 
afflictions and the National Institute of Health officials who 
manage the digestive disease research portfolio. This 
commission will survey the state of GI research and develop a 
long range plan with detailed recommendations by areas of 
research policy and programmatic development. The commission 
would issue its report within 18 months of it enactment.
    There is precedent for what we are proposing. A little more 
than 25 years ago this committee approved the bill which 
created the original National Digestive Diseases Commission. 
The Commission's report issued in 1979 laid the foundation for 
countless important research developments and breakthroughs in 
the areas of digestive diseases.
    Mr. Bilirakis. Please summarize, Mr. Rush.
    Mr. Rush. I sure will, Mr. Chairman.
    But the work of this first Commission serves as a 
precedent, the successor national commission on digestive 
diseases will galvanize the government, the research community 
and the public to undertake a comprehensive and cost effective 
campaign to end the scourge of digestive diseases.
    Mr. Chairman, I look forward to the hearing today. I 
welcome the witnesses. And I look forward to affirmative action 
by this committee on the bill that Mr. Blunt and I introduced 
in the near future.
    Thank you, and I yield back.
    Mr. Bilirakis. Well, I assure you we will have affirmative 
action regarding this issue and trying to do something about 
it. I cannot assure that we will have affirmative action 
strictly on your piece of legislation.
    Mr. Rush. I am really disappointed----
    Mr. Bilirakis. But as you and I discussed yesterday, I 
would like to include you in all the discussions that we have.
    In any case, without objection I would yield 3 minutes to 
Ms. Sue Kelly who has the principal piece of legislation that 
has been introduced for some time for an opening statement.
    Ms. Kelly. Thank you so much, Mr. Chairman. I thank you for 
the opportunity to be with you today.
    As the sponsor of H.R. 290, the Inflammatory Bowel Disease 
Act, I greatly appreciate your leadership in convening today's 
hearing which will bring much needed attention and awareness to 
a disease that has long been in the shadows of society.
    Crohn's disease, an ulcerative colitis collectively known 
as inflammatory bowel disease are chronic disorders of the 
gastrointestinal track that cause severe pain and suffering in 
the more than 1 million Americans who are affected. As you 
pointed out, Mr. Chairman, the IBD can cause severe abdominal 
pain, fever, bleeding, ulcerations and chronic diarrhea. But 
there are complications as well that are related to this 
disease; arthritis, osteoporosis, malnutrition, liver disease, 
colon cancer. IBD represents one of the major causes of 
morbidity from digestive illness, and it can be absolutely 
devastating.
    I want to thank our witnesses for joining us today, and 
particularly Adam Carron, a very courageous young Crohn's 
disease patient from my home State who is going to share his 
story with us.
    And I also want to recognize Bill and Shelby Modell who are 
here today attending the hearing. The Modells are co-founders 
of the CCFA.
    I want to thank you, Mr. Chairman, for your interest in 
this issue and for your leadership in convening this hearing.
    As I mentioned previously, I have introduced the 
Inflammatory Bowel Disease Act which would authorize an 
expansion of Federal support for IBD research at the National 
Institutes of Health and the Centers for Disease Control and 
Prevention.
    We are at an exciting time with respect to research on 
these challenging diseases. A few years ago, the scientific 
community discovered the first gene associated with Crohn's 
disease. This is a landmark discovery, and other advancements 
in the field have opened up exciting new research pathways 
which have the potential to lead to better treatments and, 
hopefully, 1 day soon I hope a cure.
    My legislation seeks to further this momentum by 
capitalizing on these promising opportunities. H.R. 290 builds 
upon recommendations put forth in a concurrent resolution on 
IBD, which I sponsored in the 107th Congress.
    As you know, Mr. Chairman, that resolution was passed by 
the Energy and Commerce Committee in September of 2002.
    The Inflammatory Bowel Disease Act currently has 177 
bipartisan co-sponsors in the House, including 17 members of 
this distinguished subcommittee. I want to express my 
appreciation to each and everyone of these members who have co-
sponsored this legislation.
    Mr. Chairman, I look forward to working with you and 
Ranking Member Brown who is a co-sponsor of H.R. 290, to pass 
this important bill this year. H.R. 290 represents a landmark 
opportunity to help improve the quality of life for IBD 
patients and their families.
    Thank you so much for allowing me to be here today. And I 
very much look forward to hearing from our witnesses.
    Mr. Bilirakis. The Chair thanks the gentlelady.
    I see that the chairman of our full committee Mr. Barton 
has joined us. Joe, would you like to make an opening 
statement.
    Chairman Barton. Thank you, Mr. Chairman. I will submit my 
formal statement in the record.
    I want to thank you for holding this hearing. I want to 
thank Congresswoman Kelly for the effort that she has put into 
this issue for many years. And hopefully we can have a good 
hearing today and create a bipartisan sense of support to move 
some legislation.
    [The prepared statement of Hon. Joe Barton follows:]

 Prepared Statement of Hon. Joe Barton, Chairman, Committee on Energy 
                              and Commerce

    Thank you, Chairman Bilirakis, for holding this hearing today. 
Talking about digestive diseases is not easy. These diseases, however, 
are serious, painful and dramatically impact how many Americans carry 
out their daily lives. I am pleased that the Committee is starting a 
dialogue about improving our current efforts to address this problem.
    In preparation for this hearing, I was informed that relatively 
little data exists regarding the causes, prevalence and control of 
digestives disease. Although scientists have made considerable progress 
in understanding the many complex diseases that affect the digestive 
system, the lack of solid data limits researcher's abilities to 
pinpoint the causes of these diseases and identify new treatment 
options. In addition, this lack of data limits our understanding of how 
many individuals are afflicted and whether these numbers are 
significantly increasing.
    This lack of basic data is one more example of why I am making it a 
priority to reauthorize critically important public health agencies 
like the National Institutes of Health (``NIH'') and the Centers for 
Disease Control and Prevention (``CDC''). As part of this effort, the 
Committee will explore how the CDC conducts surveys and how their data 
collection activities may be improved. This review should help us 
identify how the CDC can make improvements that will bring real 
benefits to patients suffering from digestive diseases, in addition to 
many other infectious and chronic diseases.
    I am pleased that Dr. Spiegel is here to talk about how the 
research programs underway at the National Institutes of Health. These 
programs hold the potential to fundamentally improve our understanding 
of digestive diseases. As Director of the National Institute of 
Diabetes and Digestive and Kidney Diseases for the past five years, and 
its scientific director for the previous 9 years, Dr. Spiegel has been 
at the forefront of scientific discoveries that have already improved 
patient outcomes.
    By now, it is no secret to anyone that I am very committed to 
making improvements at our public health agencies, and particularly at 
the National Institutes of Health. Although NIH's research portfolio is 
largely dedicated to basic research that transcends disease specific 
research, applying this research so that it directly benefits patients 
suffering from specific diseases is critical. I look forward to 
learning more about the scientific opportunities NIDDK is exploring and 
how we can encourage these developments by improving the organization 
and structure of NIH to maximize our investments in public health.
    Once again, I appreciate all of the time the witnesses have taken 
to make this an informative hearing. Thank you for helping us to raise 
awareness about digestive diseases so that more Americans recognize the 
symptoms of these diseases and can begin treatments early.

    Mr. Bilirakis. I thank the gentleman.
    [Additional statement submitted for the record follows:]

 Prepared Statement of Hon. Eliot Engel, a Representative in Congress 
                       from the State of New York

    Mr. Chairman, thank you for having this hearing today. Digestive 
diseases affect millions of Americans and I believe it is highly 
appropriate that our Committee examine the issues and the potential for 
moving research forward.
    Mr. Chairman, I am pleased to welcome our witnesses. I appreciate 
all of their time and especially the testimony of Adam Carron from the 
Crohn's and Colitis Foundation of America. I appreciate your courage in 
coming before the Committee this morning to talk about your experienced 
with IBD. I also welcome Rodger DeRose from the Crohn's and Colitis 
Foundation. Both are from my home state of New York, and they have been 
instrumental in raising awareness about these diseases and helping 
patients in New York City and across the country. I am pleased to be a 
co-sponsor of H.R. 290, legislation focused on Crohn's disease and 
ulcerative colitis. This legislation has the support of 175 bipartisan 
members of the House. I look forward to working with the Chairman and 
Ranking Member to advance this important legislation in the Committee 
this year.
    I am also interested in hearing what work the NIH is pursuing with 
regards to Irritable Bowel Syndrome (IBS). Obviously IBS doesn't get 
nearly the attention of IBD because it is a less severe condition and 
not life threatening but it still terribly disrupts the lives of its 
victims and we need to focus on this condition as well. I look forward 
to hearing from Dr. Spiegel about NIH's research efforts and a 
strategic plan regarding IBS.
    Mr. Chairman, I thank you for your efforts to examine digestive 
disease and look forward to working with you on this important issue.

    Mr. Bilirakis. All right. Let us go right into the 
testimony part of the witnesses. I have already introduced 
them.
    Dr. Spiegel is the Director of National Institute of 
Diabetes and Digestive and Kidney Diseases here in Bethesda. 
Mr. DeRose is President and CEO of the Crohn's and Colitis 
Foundation of America. Adam Carron is a young man who 
unfortunately has been inflicted with this disease, but I think 
that is probably God's way of saying to Adam hey this is your 
opportunity to help an awful lot of people around the world by 
virtue of your personal experience. And Dr. Peura is here on 
behalf of the Digestive Disease National Coalition.
    Your written statements are already a part of the record. 
And I would hope, I am going to set this at 5 minutes. I will 
not cut you off, but I would hope you would stay as close to it 
as you can. But hopefully you would sort of supplement and 
compliment your written statement.
    Dr. Spiegel, let us start off with you, sir.

STATEMENTS OF ALLEN M. SPIEGEL, DIRECTOR, NATIONAL INSTITUTE OF 
  DIABETES AND DIGESTIVE AND KIDNEY DISEASES; RODGER DeROSE, 
 PRESIDENT AND CEO, CROHN'S AND COLITIS FOUNDATION OF AMERICA; 
   ADAM CARRON; AND DAVID PEURA, DIGESTIVE DISEASE NATIONAL 
                           COALITION

    Mr. Spiegel. Thank you very much, Mr. Chairman and members 
of the committee. I am please to testify today regarding NIH 
efforts to combat digestive diseases.
    I am accompanied today by Dr. Stephen James, who is the 
newly appointed Director of the Institute's Division of 
Digestive Diseases and Nutrition. He's an expert on digestive 
disease research, particularly immunologically mediated 
diseases including the inflammatory bowel diseases.
    I have provided the written statement with a detailed 
account of the public health burden of digestive diseases, the 
vigorous research efforts NIH has underway in this area and 
future research directions based on our planning. I would like 
to just give you a brief overview this morning.
    Digestive diseases research encompasses many serious, 
potentially life threatening illnesses. Examples, hepatitis, 
gastrointestinal cancer as well as highly prevalent diseases 
such as acute gastroenteritis, gastroesophageal reflux, the 
cause of heartburn, and irritable bowel syndrome. While these 
generally are not fatal, they cause significant morbidity.
    In fiscal year 2003 the NIH invested nearly $1.2 billion in 
research on digestive diseases. The National Institute of 
Diabetes and Digestive and Kidney Disease, which I head, the 
National Cancer Institute and the National Institute of Allergy 
and Infectious Diseases accounted for 34, 32 and 17 percent of 
support, respectively.
    During the period of the doubling of the NIH budget for 
which, by the way, we're extraordinarily appreciative of the 
bipartisan efforts of this Congress, NIDDK was able to fund a 
significant number of large scale digestive disease research 
initiatives. We were able to fund four new digestive disease 
development centers, a women's health center focusing on 
irritable bowel syndrome and to expand the number of digestive 
disease research centers from 12 to 16, including our newest 
one at the University of Virginia where another panel member, 
Dr. Peura, is the associate chief of the gastroenterology 
division.
    There are active collaborations in digestive disease 
research among many NIH components. Just one example, NIDDK and 
NCI have joint efforts on Barrett's esophagus, which can be a 
precursor to cancer of the esophagus. Also, we collaborate on 
the diagnosis and treatment and prevention of liver cancer, 
which may be a consequence of infection with the hepatitis 
virus.
    The NIDDK plays an important role in disseminating 
information on digestive disease through the National Digestive 
Diseases Information Clearinghouse. And this speaks to the 
chairman's point about the need not only to do the research, 
but to get the information out to the American public.
    The clearinghouse develops and distributes health 
information for patients, the public and healthcare providers 
to improve understanding of digestive diseases. Just last week 
the NIH sponsored a consensus development conference on celiac 
disease, an immune mediated disorder that primarily affects the 
digestive tract. This disease affects about 1 percent of the 
U.S. population, but is currently recognized in only about one-
tenth of patients with current medical practice. In addition to 
providing useful guidance to the NIH, we are hopeful this 
conference will raise awareness of the disease among medical 
practitioners and the public in order to promote early, 
accurate diagnosis and treatment.
    As a major example of NIH digestive diseases research, I 
would like to give the committee a brief description of 
research advances in the inflammatory bowel diseases, 
ulcerative colitis and Crohn's disease. Many patients are 
diagnosed in childhood or in their teens and must cope for the 
rest of their lives with problems that include intestinal 
inflammation, abdominal pain, fever, diarrhea and rectal 
bleeding. We will hear momentarily from Mr. Carron whose 
testimony, I am sure, will be more eloquent than anything I 
could say about the impact of these inflammatory bowel 
diseases.
    Now for decades there were no truly effective medications. 
Surgical removal of the affected parts of the intestine was 
often necessary, particularly in ulcerative colitis which can 
lead to colon cancer. Investigator initiated basic research on 
the immune system, however, began to illuminate the fundamental 
mechanisms responsible for intestinal inflammation. These 
discoveries set the stage for an ambitious long range plan for 
IBD research with the goals of improving lives with more 
effective treatment and ultimately, prevention. The plan, 
importantly, was formulated with external input from patient 
groups such as the Crohn's and Colitis Foundation of America 
and investigative groups such as the American 
Gastroenterological Association, both valued partners of NIDDK.
    Through NIH research efforts significant improvements in 
therapy for Crohn's disease have resulted, notably the 
development of infliximab, a drug that targets an inflammation 
causing protein. The FDA's approval of infliximab was an 
important step forward in treating Crohn's disease.
    Then as Congresswoman Kelly has alluded to, a major 
discovery occurred in 2001 when investigators announced the 
discovery of a gene that confers susceptibility to Crohn's 
disease. It is fine to find a new gene, but you need to 
actually then build on that to do something for patients, that 
is really the important part; and we have. The role of this 
particular gene illuminated that abnormalities in the innate 
immune system, the first line of defense against foreign 
invaders, was an important potential cause of Crohn's disease. 
This was translated into a clinical trial demonstrating the 
potential benefit of a new treatment which boosts the function 
of the innate immune system. Because other genes conferring 
susceptibility to inflammatory bowel disease remain to be 
discovered and could provide similar insights, the NIDDK 
established a new multicenter genetics consortium to speed this 
research.
    In another example, and I think a particularly important 
one in terms of the crosscutting nature of research, NIDDK 
investigators demonstrated that the drug Rosiglitazone, 
currently used to treat diabetes, has anti-inflammatory effects 
in an animal model of IBD. Subsequently, an NIDDK sponsored 
multicenter clinical trial of this drug for treatment of 
ulcerative colitis has been initiated and is currently in 
progress.
    There are several promising agents in the pipeline and I 
hope the committee sees, as these highlights show, that 
progress in IBD research reflects a convergence of public 
health need, stakeholder input, scientific opportunity and the 
critical peer review system which assures that only the most 
meritorious proposals submitted to the NIH are funded.
    On a broader level the NIH is now embarking on a new 
planning process for digestive diseases generally under the 
auspices of the statutory Digestive Diseases Interagency 
Coordinating Committee chaired by Dr. James. The first step in 
that process is the development of a liver disease research 
action plan, a commitment that I made to the chairman back in 
September of 2002. This is being done in consultation with 
external scientific and lay experts. We will be following a 
similar planning process for other specific digestive diseases. 
We believe this planning effort will produce useful guideposts 
for prioritization in NIH program development and importantly, 
will help synergize cross-cutting research efforts across the 
NIH.
    Finally, let me mention that on March 9th Dr. James and I 
were pleased to present an overview of the digestive disease 
research program, recent advances and future plans to the newly 
created Congressional Digestive Disease Caucus. The caucus was 
founded through the leadership of Congresswoman Sue Kelly, and 
we are really appreciative of her commitment as Chair of the 
caucus to increase awareness of the burden of digestive 
diseases and to encourage research in this important area.
    Mr. Chairman and members of the committee, I hope these few 
examples convey the firm commitment of the NIH to combatting 
the many digestive diseases within its research mission. 
Through research we seek to relieve the burden these chronic, 
debilitating, frustrating diseases place on individuals, 
families and the nation.
    I appreciate the opportunity to address the committee on 
behalf of the NIH and the NIDDK, and we would be pleased to 
respond to any questions you may have.
    [The prepared statement of Allen M. Spiegel follows:]

Prepared Statement of Allen M. Spiegel, Director, National Institute of 
  Diabetes and Digestive and Kidney Diseases, National Institutes of 
          Health, U.S. Department of Health and Human Services

    Mr. Chairman and Members of the Committee: I am Allen Spiegel, 
Director of the National Institute of Diabetes and Digestive and Kidney 
Diseases (NIDDK), the Institute that has lead responsibility for 
digestive diseases research at the National Institutes of Health (NIH 
of the Department of Health and Human Services. I am pleased to testify 
today regarding NIH efforts to combat digestive diseases. Through basic 
and clinical research studies, we can gain greater insights into the 
causes of these diseases, find more effective treatments, and develop 
prevention strategies. I am accompanied today by Dr. Stephen James, the 
newly appointed Director of the Institute's Division of Digestive 
Diseases and Nutrition. Dr. James is an expert on digestive disease 
research, particularly immunologically mediated diseases, including 
inflammatory bowel diseases.
    In my testimony today, I will give you a brief overview of the 
public health burden of digestive diseases, the vigorous research 
efforts NIH has under way in this area, highlighting Crohn's disease 
research as an illustrative example, and closing with future directions 
based on our planning for research in digestive diseases. BURDEN OF 
DIGESTIVE DISEASES
    The digestive system is critically important to human health and 
well being. This complex system includes the pharynx, esophagus, 
stomach, liver, biliary tract, pancreas, small and large intestines, 
and anorectum. Thus, digestive diseases research encompasses many 
serious and potentially life-threatening illnesses, such as cirrhosis 
of the liver, inflammatory bowel diseases, hepatitis, gastrointestinal 
cancer, ulcers, and gallstones. This constellation of diseases also 
includes highly prevalent diseases such as acute gastroenteritis, 
gastroesophageal reflux disease (the cause of heartburn), and irritable 
bowel syndrome that, while generally not fatal, cause significant 
morbidity.
    Digestive diseases and their associated long-term complications 
have significant social and economic consequences for the Nation. 
According to a report published in 2002, the estimated annual total 
cost for digestive diseases, in 1998 dollars, was $85.5 billion 
(Sandler RS, Everhart JE, Donowitz M, Adams E, Cronin K, Goodman C, 
Gemmen E, Shah S, Avdic A, Rubin R. The burden of selected digestive 
diseases in the United States. Gastroenterology. 2002 May; 122(5):1500-
11).
    Digestive diseases rank second among all causes of disability due 
to illness in the United States. They result in an estimated 200,000 
absences from work each day with a mean time lost of nine days. More 
than two million Americans are impaired to some degree by digestive 
diseases, limiting an estimated 1.2 million people in the type of 
occupation they can seek. Approximately 140,000 veterans receive 
payments for service-related disabilities due to digestive diseases.
    The chronic nature of digestive diseases results in approximately 
11 percent of all admissions to general hospitals in the United States 
and in 15 percent of all surgical procedures performed in this country. 
Approximately 200,000 deaths annually are caused by digestive diseases, 
including cirrhosis and other liver diseases, cancer of the digestive 
system, gallbladder disease, ulcers, and pancreatitis. Digestive 
diseases also complicate the treatment of other life-threatening 
conditions, such as cardiovascular disease.
    In fiscal year 2003, the NIH invested nearly $1.1 billion in 
research on digestive diseases. The NIDDK, the National Cancer 
Institute (NCI), and National Institute of Allergy and Infectious 
Diseases (NIAID) accounted for 34 percent, 32 percent, and 17 percent 
of this support, respectively. Research mechanisms include regular 
research grants, cooperative clinical trials, epidemiologic studies and 
data systems, and cooperative consortia. In addition, during the period 
of the doubling of the NIH budget, we were able to expand the number of 
NIDDK Digestive Disease Research Centers from 12 to 16, as well as to 
add four new digestive disease Development Centers, plus a Women's 
Health Center (with the NIH Office of Research on Women's Health) 
focusing on irritable bowel syndrome. We are also vigorously supporting 
physician-scientists in digestive diseases through our research 
training and career development awards and the loan repayment program.
    A statutory Digestive Diseases Interagency Coordinating Committee 
serves to coalesce and synergize the efforts of the many NIH Institutes 
and Centers that support research in this field, as well as the efforts 
of other Federal agencies. Intersecting research and active 
collaboration are found among many NIH components. For example, NIDDK's 
research on the development of islet cells of the pancreas complements 
the NCI's work on the cellular origins of pancreatic cancer. Similarly, 
NIDDK and NCI have joint efforts on both Barrett's esophagus, which can 
be a precursor to cancer of the esophagus, and on the diagnosis, 
treatment, and prevention of liver cancer, which may be a consequence 
of infection with hepatitis virus. Hepatitis is a shared research focus 
of the NIDDK, NIAID, the National Institute on Alcohol Abuse and 
Alcoholism, and the National Institute on Drug Abuse. The NIDDK and the 
National Center for Complementary and Alternative Medicine are working 
together to test silymarin, or milk thistle, in treatment of liver 
disease. These are just a few examples of the many ongoing 
collaborative endeavors at NIH in digestive diseases research.
    The NIDDK also plays an important role in disseminating information 
on digestive diseases through the National Digestive Diseases 
Information Clearinghouse (NDDIC). The Clearinghouse develops and 
distributes health information for patients, the public, and health 
care providers to improve understanding of digestive diseases, such as 
Crohn's disease (http://digestive.niddk.nih.gov/ddiseases/pubs/crohns/
index.htm). The Clearinghouse is available via the web 
(www.digestive.niddk.nih.gov), a toll-free phone line (1-800-891-5389), 
e-mail ([email protected]), mail (NDDIC, 1 Information Way, 
Bethesda, MD 20892-2570), and fax (301-907-8906). Each year, the 
Clearinghouse meets with representatives of professional and patient-
advocacy groups to share information and seek feedback. The NDDIC's 
most recent national meeting was held on June 10, 2004, during which 
Michael Dolan reported on activities at the Crohn's and Colitis 
Foundation of America.

INFLAMMATORY BOWEL DISEASES: NEW DISCOVERIES AND PROMISE FOR THE FUTURE

    As a major example of NIH digestive diseases research, I would like 
to give the committee a brief description of the paths that have been 
taken to realize research advances in the inflammatory bowel diseases 
(IBD), ulcerative colitis and Crohn's disease. Ulcerative colitis is 
characterized by inflammation and ulceration of the inner surface of 
the large intestine. Crohn's disease may involve any portion of the 
gastrointestinal tract, but most commonly affects the lower portion of 
the small intestine. The lesions of Crohn's disease may penetrate 
through the bowel wall and lead to the formation of fistulas, which are 
ulcerating lesions that tunnel through the intestines of patients.
    The inflammatory bowel diseases are incurable, chronic, and 
debilitating. They affect an estimated 1 million Americans. Many 
patients are diagnosed in their teens and twenties and must cope for 
the rest of their lives with problems that include intestinal 
inflammation, abdominal pain, fever, diarrhea, and rectal bleeding. In 
children, symptoms can progress to malnutrition and growth retardation. 
These problems dramatically reduce quality-of-life and require 
lifelong, expensive medical care. For decades, there were no truly 
effective medications, so that surgical removal of the affected parts 
of the intestine was often necessary, particularly in ulcerative 
colitis, which can lead to colon cancer.
    Investigator-initiated basic research on the immune system, 
however, began to illuminate the fundamental mechanisms responsible for 
intestinal inflammation, leading to important therapeutic advances, 
particularly in Crohn's disease. Mice in which certain key genes of the 
immune system had been knocked out unexpectedly developed inflammatory 
bowel disease mimicking Crohn's disease. Importantly, bowel disease did 
not develop in mice raised under ``germ-free'' conditions in which the 
bacteria normally residing in the bowel are absent. Thus, the immune 
gene knockout alone is insufficient to cause disease, but combines with 
the normal gut bacteria in provoking a self-destructive immune 
response.
    These discoveries set the stage for an ambitious long-range plan 
for inflammatory bowel disease research with the goals of providing 
more effective treatment and, ultimately, prevention. The NIDDK has 
pursued the plan's aims to: (1) emphasize basic research on 
interactions between intestinal cells and bacteria; (2) augment the 
pool of researchers and foster interdisciplinary research; (3) 
establish and enhance appropriate biological resources and data 
collections; and (4) develop therapeutic applications and preventive 
approaches as basic research progresses.
    This plan was formulated with external input from patient groups, 
such as the Crohn's and Colitis Foundation of America, and investigator 
groups, such as the American Gastroenterological Association. Dr. James 
has worked closely with these advocacy groups to maximize efforts 
toward accomplishing common research goals. The plan was updated at a 
meeting of the NIDDK-led Digestive Diseases Interagency Coordinating 
Committee in April 2003. In implementing the plan, the NIDDK has 
deployed the full range of available mechanisms, including a robust 
portfolio of investigator-initiated grants and pilot-and-feasibility 
studies, research training grants, large program project grants, and 
four Digestive Diseases Research Centers that focus on inflammatory 
bowel disease.
    Through NIH research efforts, significant improvements in therapy 
for Crohn's disease have resulted, most notably the development of 
infliximab, a drug that targets an inflammation-causing protein whose 
role was illuminated by the mouse gene knockout experiments. The Food 
and Drug Administration's (FDA) approval of infliximab was an important 
step forward in treating Crohn's disease. A report in the New England 
Journal of Medicine earlier this year showed that it is particularly 
effective in healing the fistulas. An enormous new scientific 
opportunity emerged in 2001 when investigators announced the 
unprecedented discovery of a gene that confers susceptibility to 
Crohn's disease. This discovery represents an important payoff of the 
Human Genome Project. It is also a credit to the strong foundation laid 
by previous NIDDK research efforts--including an emphasis on targeted, 
interdisciplinary collaborations among researchers from different 
fields. Finding this gene illuminated the potential role of 
abnormalities of the innate immune system--the first line of defense 
against ``foreign invaders''--as a cause of Crohn's disease. This 
insight was then translated into a clinical trial demonstrating the 
potential benefit of a new treatment, GM-CSF, a natural protein in the 
body which boosts the function of the innate immune system. Because 
other genes conferring susceptibility to inflammatory bowel disease 
remain to be discovered, the NIDDK established a new multi-center 
Genetics Consortium to speed this search. The pace of discovery in 
genetics of IBD is accelerating, as evidenced by the publication in 
April of the identification of two new candidate genes involved in 
Crohn's disease.
    Most recently, results from a multicenter clinical trial were 
presented at the May 2004 meeting of the digestive diseases 
professional organizations. Researchers have shown that there is a 
benefit for Crohn's patients in the use of a monoclonal antibody 
targeting the cytokine IL-12. This work emanates from the intramural 
research program of the NIAID.
    Other clinical insights are expected to emerge from an ongoing 
NIDDK-funded multicenter clinical trial to investigate whether the 
dosing of the standard, now generic, immunosuppressive drug 
azathioprine can be improved. Researchers are studying new testing 
methods for the metabolizing enzyme and toxic metabolites of the drug. 
This trial is an example of the vital role NIH can play in conducting 
clinical trials of a treatment when there is no longer any incentive 
for commercial interest in such research.
    Other NIDDK initiatives include the detailed study of adult stem 
cells of the intestine that may ultimately lead to therapies 
stimulating gut regeneration. We are vigorously pursuing these research 
areas as we evaluate and monitor progress in attaining our goals. While 
this example illustrates how investments in investigator-initiated 
basic research lead to discoveries that improve the outcomes of 
patients with Crohn's disease, we recognize that we must take steps to 
accelerate the translation of basic science discoveries into patient 
benefits. For this reason, NIH and NIDDK are taking steps to bolster 
translational research.

   INFLAMMATORY BOWEL DISEASES: TRANSLATIONAL RESEARCH OPPORTUNITIES

    The NIH is strongly committed to spurring translational research in 
both Crohn's disease and ulcerative colitis. By translational research, 
we mean research to speed the movement of laboratory discoveries into 
research that holds promise of direct clinical benefits for patients--
also called ``bench-to-bedside'' research. NIH Director Elias Zerhouni, 
M.D., has stressed the importance of this type of research in his 
initiative to develop a Roadmap for medical research, and the many 
Institutes and Centers of the NIH are also emphasizing translation 
research in their respective programs. At the NIDDK, for example, we 
have recently completed an assessment of drugs in the pipeline for 
several diseases within our mission, including the inflammatory bowel 
diseases--Crohn's disease and ulcerative colitis. One good example of 
translational research involves the drug rosiglitazone, which is used 
to treat diabetes. NIDDK-funded investigators demonstrated that this 
drug has anti-inflammatory effects in an animal model of IBD, and 
subsequently, a NIDDK-sponsored multi-center clinical trial of this 
drug for treatment of ulcerative colitis has been initiated and is 
currently in progress. There are approximately 10 new drugs under 
development for one or both of these diseases, as well as many new 
studies of existing agents. The novel therapies have emerged from a 
foundation of basic research supported by the public sector, with drug 
development steps pursued by the private sector. We are encouraged when 
industry builds upon NIH-funded basic research discoveries because such 
activity offers promise of new and more effective treatments for IBD.
    We have identified roadblocks to translational research in IBD and 
steps that can be taken to address them, such as the development of 
surrogate markers of disease activity and better diagnostic tests. It 
is also essential to maximize the research investment in animal models 
of IBD, which can continue to provide insights into the underpinnings 
of the disease, and also serve as a source of potential genetic 
discoveries and a means of testing emerging new therapies. Research 
progress is often hampered by the difficulty of obtaining access to 
human samples. To overcome this barrier, NIDDK has recently initiated a 
repository that will collect and make available to investigators 
various types of human samples, including blood, biopsies, genetic 
material, and datasets. Another barrier to clinical research concerns 
the great complexity of modern clinical trial designs. To facilitate 
testing of additional new treatments under development, the NIDDK 
convened a meeting in January 2003 that included representatives from 
the FDA, industry, and the investigative community, to seek 
improvements in the design of clinical trials, with emphasis on 
improving trial endpoints. We will continue to foster such proactive 
partnerships with the FDA and industry, and also to pursue clinical 
studies in needed areas that industry does not have a commercial 
incentive to explore.
    As these highlights show, progress in IBD research reflects a 
convergence of public health need, scientific opportunity, stakeholder 
input, and the merit of research proposals submitted to the NIH for 
funding. While many strides have been made, we still recognize that our 
currently available therapies have many drawbacks and may not provide 
the adequate symptom relief that patients need. At the same time, 
however, we are encouraged by the advances being made through research 
and are committed to accelerating the pace of discovery and 
translation.
    To this end, NIDDK is fostering more cross-cutting initiatives, 
including emphasis on harnessing powerful new technologies in genomics, 
proteomics, and molecular imaging to address long-standing problems. 
Availability of a non-invasive imaging method to assess liver scarring 
or fibrosis, for example, would transform the clinical management of 
many liver diseases which now must rely on invasive biopsies. Such 
cross-cutting initiatives have broad application not only to digestive 
diseases, but also to a wide array of other diseases within the NIH 
research mission. By pursuing such endeavors, we can help to maximize 
NIH research investments by promoting their greatest yield and 
application.

                 ENHANCING DIGESTIVE DISEASES RESEARCH

    In building the digestive diseases research portfolio, we recognize 
the importance of input from the scientific and lay community external 
to the NIH. I would like to provide just a few examples.
    Stakeholder input is an important dimension of our planning and 
program development processes. As noted previously, our joint planning 
efforts with the Crohn's and Colitis Foundation of America have been 
very productive. Another example of input that guides NIH program 
development can be found in the insights and recommendations we obtain 
from a wide range of conferences and workshops. For example, in 
digestive diseases, the NIH has sponsored critically important 
Consensus Development Conferences on hepatitis C, and we recently 
submitted to the Congress a report on our implementation of the 
recommendations we received. Just last week, June 28-30, 2004, the NIH 
sponsored a Consensus Development Conference on celiac disease, an 
immune-mediated disorder that primarily affects the digestive tract. 
This disease affects about 1 percent of the U.S. population but is 
recognized in only about one-tenth of patients using current medical 
practice. In addition to providing useful guidance to the NIH, we are 
hopeful that this conference will raise awareness of the disease among 
medical practitioners and the public in order to promote early, 
accurate diagnosis and treatment.
    On a broader level, the NIH is now embarking on a new planning 
process for digestive diseases generally, under the auspices of the 
Digestive Diseases Interagency Coordinating Committee chaired by Dr. 
James. The first step in that process is the development of a Liver 
Disease Research Action Plan, in consultation with external scientific 
and lay experts. An open meeting provided significant input from 
representatives of professional organizations, patients, and the 
public. Six draft chapters of the Plan have already been posted on the 
NIDDK website for additional public comment, and remaining chapters 
will be posted as they are completed by the Committee. We will be 
following a similar planning process for other specific digestive 
diseases, and we believe that this planning effort will produce useful 
guideposts for prioritization in NIH program development, and will help 
synergize cross-cutting research efforts across the NIH.
    Finally, I also want to mention that on March 9, Dr. James and I 
were pleased to present an overview of the NIDDK digestive disease 
research program, recent advances, and future plans to the newly-
created Congressional Digestive Disease Caucus. The Caucus was founded 
through the leadership of Congresswoman Sue Kelly. We are most 
appreciative of her commitment, as Chair of the Caucus, to increase 
awareness of the burden of digestive diseases and to encourage research 
in this important area.
    Mr. Chairman and Members of the Committee, I hope that these few 
examples convey the firm commitment of the NIH to combating the many 
digestive diseases within its research mission. Through research, we 
seek to relieve the burden these chronic, debilitating, frustrating 
diseases place on individuals, families, and the Nation. I appreciate 
the opportunity to address the Committee on behalf of the NIH and the 
NIDDK, and would be pleased to respond to any questions you may have.

    Mr. Bilirakis. Thank you very much, Dr. Spiegel.
    Mr. DeRose, you are on, sir.

                   STATEMENT OF RODGER DeROSE

    Mr. DeRose. Well, thank you, Mr. Chairman. I appreciate the 
opportunity to testify on behalf of the patients across the 
country.
    I think you all have background now in terms of the 
complications of this disease, so I am not going to be 
repetitive in that. I think when you hear Adam's story, you 
will understand the dilemma that so many of these patients 
face; that you look at a young man like this that looks healthy 
and at the same time you know that on the inside they are not 
healthy. And that is the deceiving part about this disease. And 
we find this as we go across the country talking to patients 
that we do not get our due respect because individuals see our 
patients and do not recognize how debilitating this disease can 
be.
    I just wanted to give you some background in terms of our 
foundation. We were founded in 1967. And, actually, two of our 
co-founders are here today, Mr. Chairman. I want to recognize 
them, Bill and Shelby Modell from New York. And, you know, they 
started this organization with the Rosenthals in 1967 because 
they had a teenager that had the disease. And a few years ago 
they lost their son, Michael, to not complications to Crohn's 
but the fact that his immune system was compromised so 
dramatically that other diseases set in. And they lost him. 
They have continued to be 100 percent committed to this 
organization, day in and day out.
    From the time that we have started we have expanded to 42 
chapters around the country. We have a mission of research, 
education and support. To date, we have raised nearly $300 
million, $100 million of that has gone directly into research. 
Because we have such a high efficiency rate as an organization, 
the majority of the balance of that $200 million has gone into 
education and support to the patient community.
    And I think it is fair to say, Mr. Chairman, that the 
organization in its 30 plus years history has an impact on 
every major aspect of this disease in terms of improving the 
quality of life of the patient community in terms of: New 
therapies that have been introduced because of the basic 
science that CCFA has supported; safer alternatives to steroids 
that were available, only available 25 years ago; advanced 
surgical techniques that have given patients their life back; 
improved diagnostic tools that have allowed us to detect the 
disease much earlier; in the area of early warning signs in 
terms of dysplasia and cancer, and; also in the area of 
nutritional therapies because many of our patients are 
malnourished. They cannot get the calories into their system 
because they're going to the bathroom so often and flushing 
this through their system. And importantly, education programs.
    In the area of education while we run these education 
programs throughout the country and over 300 support groups 
around the country each year, we also reach out to the children 
with this disease in terms of running camps. We have 14 camps 
across the country that help children cope with the physical as 
well as emotional battles that they deal with with this 
disease. It is staffed by volunteer doctors, volunteer nurses 
and camp counselors 7 by 24.
    So it is a community that is really based on volunteerism 
and has a dramatic impact in the advances that are taking place 
in this disease state.
    I want to say one thing about the number of patients that I 
have talked about. We conservatively estimate that it is a 
million Americans, but we happen to believe that it is probably 
much higher than that. Anecdotally as we travel the country, as 
we get phone calls from patients and we talk to the 
professional community, it is our understanding that we are 
probably underestimating the size and the impact of this 
disease across our country. And as a result of that, the 
foundation 2 years actually went to the Center for Disease and 
Control and Prevention and we actually went to them and ask 
them to submit a grant request to CCFA so that we could start 
an epidemiology program with them, an epidemiology study to 
better understand the prevalence of this disease. So we funded 
it to the tune of three-quarters of a million dollars for those 
2 years.
    And our scientific advisory committee has been working very 
closely with the CDC, but despite repeated encouragement from 
your colleagues, Mr. Chairman, on the Appropriations Committee 
the CDC has yet to provide any financial support for this 
project. And now that the study has been initiated by funding 
from a patient group to a government agency, we are hoping that 
the CDC will contribute to the completion of this major study. 
And, of course, that is one of the aspects of the IBD bill.
    With respect to research, I have to tell you that it is a 
real privilege for us to work in partnership with the NIH 
within NIDDK and in particular with Dr. James as well as Dr. 
Allen Spiegel. Dr. James actually sits as an ad hoc member, a 
nonvoting member of our national scientific advisory committee. 
And so we have a very close working relationship with them, and 
we exchange intellectual knowledge as we try to advance the 
state of this disease on behalf of the patient community.
    I think one good example of that is the benefits that this 
approach has led to in terms of the discovery of the very first 
gene that was talked about were CCFA funded that and then the 
NIH came in and continued to invest the furtherance of 
understanding, the impact of that gene as well as other genes 
that may lead to getting us closer to the cure.
    We are very proud of the support that we have had and that 
we have played with the NIH. If you talk to some of the 
investigators, they would say that there is probably a 20 times 
multiplier of fact in terms of from the time that they first 
get their CCFA grant from us to the time that they meet the 
midpoint of their career in terms of the additional funding 
that they get from the NIH. So you can see that there is an 
infusion of knowledge that is taking place here from the 
preliminary data that CCFA is providing with the research that 
the NIH is picking up and continuing.
    So I am confident that working together that we can make 
dramatic strides here.
    Mr. Chairman, I do want to thank Congresswoman Sue Kelly 
for introducing the bill as well as Congressman Jessie Jackson, 
Jr. And I thank you for taking the time out to hear our 
testimonies today and everything that you are doing on behalf 
of the patient community to advance the quality of life for our 
patients.
    Thank you very much. And I would be happy to answer 
questions whenever you are ready.
    [The prepared statement of Rodger DeRose follows:]

  Prepared Statement of Rodger DeRose, President and Chief Executive 
           Officer, Crohn's and Colitis Foundation of America

    Mr. Chairman and members of the subcommittee, thank you for the 
opportunity to testify today. I am Rodger DeRose, President and Chief 
Executive Officer of the Crohn's and Colitis Foundation of America.
    CCFA is the nation's oldest and largest organization dedicated to 
finding a cure for inflammatory bowel disease. IBD (which includes both 
Crohn's disease and ulcerative colitis), is a chronic disorder of the 
gastrointestinal tract which afflicts conservatively 1 million 
Americans, 100,000 or 10% of whom are children under the age of 18. IBD 
can cause severe diarrhea, abdominal pain, fever, and rectal bleeding. 
Complications related to the disease include; arthritis, osteoporosis, 
anemia, liver disease, and colon cancer. IBD represents a major cause 
of morbidity from digestive illness, and although it is not usually 
fatal due to the major advances that have occurred in the last 30 
years, IBD can be devastating. We do not know its cause, and there is 
no medical cure.
    Founded in 1967, CCFA is headquartered in New York City and has 42 
chapters that provide education and support services to patients in all 
50 states. Since its beginning, CCFA has generated $300 million in 
revenues--$100 million of which has been directly invested in basic and 
clinical research related to IBD. Because CCFA prides itself on being 
an efficient organization, with low administrative costs, a large 
percentage of the remaining $200 million has been reinvested in patient 
education and support programs.
    Through CCFA's leadership, our organization has impacted every 
major advancement in Crohn's and colitis over the past 30 years 
including: new therapies and safer alternatives to steroids, advanced 
surgical techniques, improved diagnostic methods to detect the disease 
earlier, early warning of dysplasia for the treatment of colorectal 
cancer in patients, nutritional therapies for struggling patients who 
can't get calories into their malnourished bodies, and education 
programs for both the patient and professional community.
    In the area of education, CCFA plays a leadership role in patient 
based training through our 42 chapters and the 300 support groups that 
we offer to patients each year. Additionally, we reach out to children 
with IBD by sponsoring 14 camps throughout the US to help kids deal 
with the physical and emotional elements of this disease. We staff the 
camps with volunteer doctors, nurses and camp counselors that are 
available 24 hours a day, 7 days a week to meet the needs of the kids.
    As I mentioned earlier, it is conservatively estimated that 1 
million Americans suffer from either Crohn's disease or ulcerative 
colitis. CCFA and other leaders within the digestive disease community 
believe, however, that the actual number of patients is significantly 
higher. As a result, the Foundation has provided the Centers for 
Disease Control and Prevention with $750,000 over the past two years to 
initiate a national IBD epidemiology study to determine the true 
prevalence of the disease.
    Gaining a better understanding of the number of patients afflicted 
with IBD, and their demographic characteristics, will give us 
invaluable clues as to the role that environmental factors play in the 
development and progression of the disease.
    CCFA's National Scientific Advisory Committee has developed a 
strong working relationship with the CDC on this important study. 
However, Mr. Chairman, despite repeated encouragement from your 
colleagues on the Appropriations Committee, CDC has yet to provide any 
financial support for the project. Now that the study has been 
initiated by an investment from the patient community, we are hopeful 
that CDC will contribute to the continuation and completion of this 
major study. Mr. Chairman, we would welcome your support of this 
important initiative as we move forward.
    With respect to research, the IBD community is encouraged by the 
significant progress that has been made in recent years. We are 
extremely grateful for the leadership of Dr. Spiegel, and his colleague 
Dr. Stephen James, at the NIDDK. They are strong partners in our 
continuing effort to combat this disease.
    However, we all agree that much more needs to be done. To that end, 
CCFA's scientific leaders have developed a forward-thinking research 
agenda entitled, ``Challenges in Inflammatory Bowel Disease'' that 
outlines the many exciting opportunities that currently exist in the 
field. Next to the NIH, CCFA is the leading source of funding for IBD 
research. CCFA plays a critical role in providing ``seed funding'' to 
researchers interested in IBD. This support helps investigators gather 
preliminary findings which, in turn, enables them to pursue advanced 
IBD research projects through the NIH. A good example of the benefits 
of this approach is the research that led to the discovery of the first 
gene associated with Crohn's disease in 2001. Preliminary support for 
this historic research was provided by CCFA and enhanced by the NIDDK 
and other funding sources in subsequent years.
    We are proud of the enormous impact that support from CCFA has had 
on Crohn's and colitis research. Some investigators estimate that there 
is a 20 factor multiplier effect from the time a CCFA funded 
investigator receives their first CCFA grant to the peak of their 
career. In fact, more than 80% of CCFA-sponsored researchers have 
obtained subsequent funding from the NIH for further IBD research.
    Mr. Chairman, I am confident that working together, with additional 
resources, we will develop better treatments and eventually a cure for 
these diseases. In 5 years, I'm hopeful that we will have approximately 
5 biologic treatments available to target the disease. Today we have 1 
FDA approved biologic with several pursuing or finishing stage 3 
trials. In 10 years, I am hopeful that through our genetic 
understanding of the disease that we will have genetic treatments to 
alter the course of the disease. We are at an unprecedented time of 
optimism with respect to research on IBD. Opportunities for advancement 
and real progress have never been greater. An additional investment in 
this area has the potential to yield significant and tangible results 
for patients.
    Mr. Chairman, I would like to take this opportunity to thank 
Congresswoman Sue Kelly and Congressman Jesse Jackson, Jr. for their 
leadership in introducing the ``Inflammatory Bowel Disease Act'' in the 
House. This landmark legislation, which addresses IBD research at the 
NIH and CDC, as well as other issues that are important to our 
community, has over 175 bipartisan co-sponsors, including 17 members of 
this distinguished subcommittee. On behalf of CCFA and the entire IBD 
community, I want to express our sincere appreciation for all of the 
support that this important legislation has received. Our patients and 
family members across the nation encourage the subcommittee to pass 
legislation focused on inflammatory bowel disease this year.
    The chances are high that most of the subcommittee members here 
know someone with this disease: a co-worker, a family member, friend, 
acquaintance. What you may not know is the symptoms that they live with 
daily because most look normal outwardly but they are anything but 
normal on the inside.
    We need your help in bringing Crohn's and colitis out of the closet 
so patients can get the support they need in seeking the cure through 
the NIH and the CCFA. You can be sure that the CCFA will do its part to 
end the disease.
    In closing Mr. Chairman, I want to thank you for your interest in 
this disease and for convening this important hearing today. Our Long 
Island chapter was honored that you took time out of your busy schedule 
to join us for our annual dinner in May. Our Long Island Chapter 
President, Jamie Pappas and his family, send you their best. We are 
very grateful for your leadership and look forward to working with you 
to help improve the quality of life for IBD patients and their 
families.
    I would be pleased to respond to any questions that you may have.

    Mr. Bilirakis. Unfortunate news that we have 3 votes on the 
floor.
    Let us go into Adam.

                    STATEMENT OF ADAM CARRON

    Mr. Carron. Mr. Chairman and members of this committee, 
thank you for the opportunity to testify today.
    I am Adam Carron, an 18 year old ulcerative colitis patient 
from Long Island, New York and I appreciate the opportunity to 
share my story about living with inflammatory bowel disease 
with you.
    My experience with IBD began at age 9. It started with 
bloody stools and intense abdominal pian, and my parents' 
nightmare came true when my doctor told us all that I was 
probably suffering from ulcerative colitis; the same disease 
that lead to the death of my uncle at age 42 and the illness my 
mother was struggling with.
    Before I could even pronounce ``inflammatory bowel 
disease,'' let alone know what it was, I knew I had something 
bad and was in serious trouble. I even feared that I was going 
to die.
    I remember the insecurities affecting my everyday life; 
backing out of sleeping at friends' houses and going to camp; 
even being at school needing permission to use the bathroom 
frequently during the day fearful of what might happen if my 
teachers said no.
    I was given the responsibility to remember to take my 
medicine every day, both orally and anally, just so that I 
would not have stomach cramps and blood in my stools. I 
resented it because in a time when all I wanted was to fit in, 
none of my friends had the same issues, and therefore I did not 
always follow through.
    But at age 11 I got very sick. The medications were no 
longer able to control my symptoms. I was bleeding profusely 
and had severe pain. I could not stop vomiting. I was forced to 
go into the hospital and for several weeks I lay there being 
given massive doses of steroids. I will never forget having to 
miss trick or treating with my friends and then not being able 
to eat the Halloween candy my sister had so graciously gathered 
for me because I could not eat any food.
    Finally, I was released from the hospital but had to stay 
on the steroids for several months in order to wean my body 
from the harsh treatment. I could not even look at myself in 
the mirror, for my face had swelled up beyond relief.
    A little over 1 year later, I found myself in the hospital 
again. This time the steroids that I was given did not help. I 
kept bleeding and bleeding and suffering, and constantly going 
to the bathroom. I was there for over one mo nth of my summer 
vacation and lost 25 percent of my body weight. My joints, my 
insides, and my psyche were all inflamed. I thought that I 
would never leave the hospital that month. It was at that point 
that my parents and doctors decided to remove my colon. After a 
little while, including 2 years with an ostomy, the physical 
pain was gone but the suffering I went through will never leave 
me. Several years and several surgeries later my body is, more 
or less, back in tact. I wake up everyday and see red lines 
running across my stomach, these red lines which represents the 
scars from the surgery provide me with a constant reminder of 
my struggle with IBD, yet they also serve to remind me of how 
lucky I am.
    I am lucky because I had access to great physicians and I 
was able to have these successful operations and sit before you 
today, I hope and pray, physically cured of the disease. But 
not everyone with IBD gets to tell a happy ending to their 
story. I now spend some of my time working for the Long Island 
Chapter of the Crohn's and Colitis Foundation of America, 
helping adolescents cope with the disease, on both the 
individual and social fronts.
    Earlier this year, I visited with a young lady, Ariana 
Pappas, the daughter of the CCFA Long Island Chapter President, 
on the weekend before her surgery. I could see in her eyes the 
fear that she had of the pain and the resulting scars from the 
surgery. I also saw the hope that she had that she would be 
relieved of this pain if the operation were to be successful.
    I often see promising young kids forced to miss months of 
school at a time, causing test scores and grades to plummet 
from As to Cs. I have seen a young boy ridiculed everyday at 
school because his growth has been stunted so significantly by 
IBD that he looks 6 years younger than his 13 year old age. I 
see children so distraught and so mentally defeated, simply 
because they think that talking about the disease is 
unacceptable in today's society. Our society has inadvertently 
put a cap on bowel discussion, which does not usually become 
lifted until those days in the retirement home.
    Mr. Chairman, hope for IBD patients and their families lies 
in the promise of better treatments and a cure through 
biomedical research. To that end, I want to thank Congresswoman 
Sue Kelly for her leadership in introducing the Inflammatory 
Bowel Disease Act in the House. This landmark legislation, 
which addresses IBD research at the NIH and CDC, as well as 
other issues that are important to our community, has over 175 
bipartisan co-sponsors, including 17 members of this 
distinguished subcommittee. On behalf of the CCFA and the 
entire IBD community, I want to express our appreciation for 
your support, and encourage you to pass this important bill 
this year.
    In closing, I want to thank you, Mr. Chairman, for your 
interest in this disease and for convening this important 
hearing today. We are very grateful for your leadership and 
look forward to working with you to help improve the quality of 
life for IBD patients and their families. I would be pleased 
and honored to respond to any questions that you may have.
    [The prepared statement of Adam Carron follows:]

    Prepared Statement of Adam Carron, Member, Crohn's and Colitis 
                         Foundation of America

    Mr. Chairman thank you for the opportunity to testify today. I am 
Adam Carron, an 18 year-old ulcerative colitis patient from Long 
Island, New York and I appreciate the opportunity to share my story 
about living with inflammatory bowel disease with you.
    My experience with IBD began at age nine. It started with bloody 
stools and intense abdominal pain, and my parents nightmare came true 
when my doctor told us all that I probably was suffering from 
ulcerative colitis; the same disease that lead to the death of my uncle 
at age 42 and the illness my mother was struggling with. Before I could 
even pronounce ``Inflammatory Bowel Disease,'' let alone know what it 
was, I knew I had something bad and was in trouble. I even feared that 
I was going to die. I remember the insecurities affecting my everyday 
life; backing out of sleeping at friends houses and going to camp, even 
being at school, needing permission to use the bathroom frequently 
during the day, fearful of what might happen if the teachers said 
``no''. I was given the responsibility to remember to take medicine 
everyday, both orally and anally, just so that I would not have stomach 
cramps and blood in my stools. I resented it because in a time when all 
I wanted was to fit in, none of my friends had the same issues, and 
therefore I did not always follow through.
    At age 11 I got very sick. The medications were not controlling my 
symptoms. I was bleeding profusely and had severe pain. I could not 
stop vomiting. I had to go into the hospital. For several weeks I lay 
in the hospital, being given massive does of steroids. I'll never 
forget having to miss ``Trick or Treating'' with my friends and then 
not being able to eat the Halloween candy because I could not have any 
food. Finally I was released from the hospital and then had to stay on 
the steroids for several months in order to wean my body from the 
treatment. I could not even look at myself in the mirror, for my face 
had swelled up beyond belief. A little over one year later, I found 
myself in the hospital again. This time the steroids that I was given 
did not help. I kept bleeding and bleeding and suffering, and 
constantly going to the bathroom. I was there for over one month of my 
summer vacation and lost over 25% of my body weight. My joints, my 
insides, and my psyche were all inflamed. I thought that I would never 
leave the hospital. It was at that point that my parents and doctors 
decided to remove my colon. After a little while, the physical pain was 
gone but the suffering I went through will never leave me. Several 
years and several surgeries later my body is more or less back intact. 
The physical pain was finally gone but the suffering I went through 
will never leave me. I wake up everyday and see red lines running 
across my stomach. These scars provide me with a constant reminder of 
my struggle with IBD, yet, also serve to remind me just how lucky I am.
    I am lucky because I had access to great physicians and I was able 
to have these successful operations and stand before you today, I hope 
and pray, physically cured of the disease. But not everyone with IBD 
gets to tell a happy ending to their story. I now spend some of my time 
working for the Long Island Chapter of the Crohn's and Colitis 
Foundation of America, helping adolescents cope with the disease, on 
both the individual and social fronts. Earlier this year, I visited 
with a young lady, Ariana Pappas, the daughter of the CCFA Long Island 
Chapter President, on the weekend before her surgery. I could see in 
her eyes the fear that she had of the pain and the resulting scars from 
the surgery. I also saw the hope that she had that she would be 
relieved of her pain, if the operation were successful. I often see 
promising young kids forced to miss months of school at a time, causing 
test scores and grades to plummet from ``A's'' to ``C's.'' I have seen 
a young boy ridiculed everyday at school because his growth has been 
stunted so significantly by IBD that he looks 6 years younger than his 
13-year-old age. I see children so distraught, and mentally defeated, 
simply because they think that talking about the disease is 
unacceptable. Our society has inadvertently put a cap on bowl 
discussion, which does not usually become lifted until those days in 
the retirement home.
    Mr. Chairman, hope for IBD patients and their families lies in the 
promise of better treatments and a cure through biomedical research. To 
that end, I want to thank Congresswoman Sue Kelly for her leadership in 
introducing the ``Inflammatory Bowel Disease Act'' in the House. This 
landmark legislation, which addresses IBD research at the NIH and CDC, 
as well as other issues that are important to our community, has over 
175 bipartisan co-sponsors, including 17 members of this distinguished 
subcommittee. On behalf of CCFA and the entire IBD community, I want to 
express our appreciation for your support, and encourage you to pass 
legislation on IBD this year.
    In closing, I want to thank you Mr. Chairman for your interest in 
this disease and for convening this important hearing today. We are 
very grateful for your leadership and look forward to working with you 
to help improve the quality of life for IBD patients and their 
families. I would be pleased to respond to any questions that you may 
have.

    Mr. Bilirakis. Thank you so much, Adam, for that very 
moving statement.
    Let us see, we have three votes on the floor. The first 
vote takes a few minutes and then two 5 minute votes after 
that. I would like to finish up maybe Dr. Peura's statement and 
then we can take a break.
    Proceed, sir, and hopefully I will not have to cut you off. 
We certainly do not want to miss the first vote.

                    STATEMENT OF DAVID PEURA

    Mr. Peura. Thank you very much.
    Mr. Chairman, members of the subcommittee, thank you for 
initiating this hearing on assessing digestive disease research 
and treatment opportunities, and particularly for allowing the 
Digestive Disease National Coalition to present testimony.
    I am Dr. David Peura. I am Associate Chief of the Division 
of Gastroenterology and Hepatology at the University of 
Virginia. I am Professor of Medicine at that institution. I am 
also an active clinician, clinical researcher for over 30 
years.
    Established in 1978, the Digestive Disease National 
Coalition is a national non-profit advocacy organization 
comprised of the major gastrointestinal volunteer patient 
organization and professional societies. Currently there are 25 
member organizations that belong to the DDNC. One of the 
original members of the coalition is the American 
Gastroenterological Association of which I am a member and 
currently serve as the President-elect.
    The mission of the Digestive Disease National Coalition is 
to work cooperatively to improve access to and quality of 
digestive health care in order to promote the best possible 
medical outcome and quality of life for current and future 
patients diagnosed with digestive diseases. The founder of the 
DDNC was a Crohn's disease patient who saw the need for 
increased digestive disease research and education.
    As we have heard, inflammatory bowel disease is just one of 
the scores of debilitating gastrointestinal conditions that 
afflict more than 62 million Americans. Others include 
hepatitis and other liver disease, irritable bowel syndrome, 
disease of the pancreas, ulcers, pediatric and adult 
gastroesophageal reflux, metabolic disorders, colon cancer, 
celiac disease, motility disorders, hemochromatosis and a 
number of other serious ailments.
    A recent study, ``The Burden of Gastrointestinal Disease,'' 
conducted by the Lewin Group concluded that a group of just 17 
digestive disease disorders accounted for $41 billion each year 
in direct and indirect health costs. In some of these areas, 
medical research has brought us closer to developing lifesaving 
treatments and cures.
    For example: The application of immunologic advances which 
have made liver transplantation a common life saving approach; 
the development of effective screening techniques for 
colorectal cancer; the genetic contributions of IBD, which we 
have heard about, acute and chronic pancreatitis, pancreatic 
and colon cancer and chronic diarrheal illnesses, these are all 
just on the cusp of clinical application.
    Yet for every breakthrough, there is still a lack of even 
basic understanding of the causes, prevention, transmission and 
treatments of a variety of other disease.
    IBD, the name given to Crohn's disease and ulcerative 
colitis, is a painful disrupting disorder, which currently has 
no cure. In Crohn's disease, the large and small intestines 
become inflamed. This inflammation can result in excessive 
diarrhea, severe rectal bleeding, anemia, fever, as well as 
abdominal pain and cramping, as we have heard. But I think it 
is important to emphasize this for the people that are 
afflicted with this condition.
    Those battling this disorder face the trauma of multiple 
surgeries and the effects of toxic and potentially dangerous 
drugs. Ulcerative colitis attacks the large intestine, as we 
have heard, causing painful diarrhea, bleeding, and can 
ultimately lead to colon cancer, the third most common cancer 
among the population in the United States, difficult to 
diagnose and often misdiagnosed.
    The goal of medical treatment of IBD is to suppress the 
inflammation in the large and small intestine, thereby 
permitting the intestines to heal and some of the symptoms to 
be relieved. While surgery is an option for some people, 
removing the segment of the small intestine of the colon, and 
may allow people to be symptom free for years, it is not always 
a cure.
    Mr. Chairman, the Digestive Disease National Coalition 
supports the passage of H.R. 290, The Inflammatory Bowel 
Disease Act. In addition to being endorsed by the coalition, 
the bill is endorsed by 7 of the DDNC member organizations.
    The DDNC commends Congresswoman Sue Kelly for her 
leadership in introducing this legislation. This bipartisan 
bill has 176 co-sponsors, 17 of whom sit on this committee. The 
broad support of this bill reflects the tremendous potential in 
biomedical research related to IBD. The scientific community, 
led by the CCFA, has developed a long range strategic plan for 
the future of IBD research and is in agreement that an 
additional investment in IBD research has the potential to 
yield greater scientific progress in clinical and general 
research in other areas as well.
    The DDNC also supports the passage of H.R. 3756, The 
National Commission on Digestive Diseases Act, introduced by 
Congressman Roy Blunt and Congressman Bobby Rush. In 1976, 
Congress passed legislation that authorized the first national 
commission on digestive disease----
    Mr. Bilirakis. Dr. Peura, forgive me, but I am doing 
something that I did not want to do. But we have less than 3 
minutes left to get over and cast this vote. So you will have 
to forgive us. And when we get back, you will finish up. It 
will be probably about 20 minutes or so. As soon as we cast the 
third vote, we will hustle back here.
    Mr. Peura. Fine.
    Mr. Bilirakis. Thank you.
    [Brief recess]
    Mr. Bilirakis. We are back in session.
    Dr. Peura, please proceed, sir. And, again, I apologize for 
the interruption.
    Mr. Peura. Well, actually it was a very good place to 
pause, because the presentation is sort of in two halves. We 
have covered the issue of the IBD bill.
    The DDNC also supports the passage of H.R. 3756, The 
National Commission on Digestive Disease Act, introduced by 
Congressman Blunt and Congressman Bobby Rush. In 1976, Congress 
passed legislation that authorized the first national 
commission on digestive diseases. Actually, I am probably 
chronologically gifted enough to have practices in the BC era, 
and that was before the first commission. And so I have 
actually seen the results of that commission.
    A quarter of a century later, H.R. 3756 would authorize a 
contemporary commission to provide a blueprint for the 
digestive diseases research endeavor of the future. Like the 
original commission, the new commission would be charged with 
assessing the state of digestive diseases in the United States, 
identifying areas in which improvement in the management of 
digestive diseases could actually be achieved and creating a 
long range plan to recommend resources to effectively promote 
the GI research portfolio.
    We are particularly thankful for the leadership of 
Congressman Blunt and Rush on this bill.
    In a time of limited fiscal resources to pursue an almost 
boundless reservoir of scientific opportunity, it is even more 
imperative that the most promising avenues of research are 
traveled and that only the highest quality grants, trials, 
centers, and other programs are awarded funds. If the work of 
the first commission serves as a precedent, this initiative 
will once again galvanize the government, the research 
community and the expanding population of people suffering from 
digestive diseases in a comprehensive and cost effective 
national campaign to end the scourge of digestive disorders.
    I would again thank you, Mr. Chairman, and this members of 
this subcommittee for holding this important hearing, and ask 
that the committee pass H.R. 290, The Inflammatory Bowel 
Disease Act and H.R. 2756, The National Commission on Digestive 
Diseases Act as soon as possible.
    And I will be more than happy to answer any questions.
    [The prepared statement of David Peura follows:]

   Prepared Statement of David A. Peura, Digestive Disease National 
                               Coalition

    Mr. Chairman and members of the subcommittee, thank you for 
initiating this hearing on Assessing Digestive Disease Research and 
Treatment Opportunities and allowing the Digestive Disease National 
Coalition and to present testimony. I am Dr. David Peura, Associate 
Chief, Division of Gastroenterology and Hepatology and Professor of 
Internal Medicine at the University of Virginia Health Sciences Center.

                         BACKGROUND ON THE DDNC

    Established in 1978, the Digestive Disease National Coalition 
(DDNC) is a national non-profit advocacy organization comprised of the 
major gastrointestinal volunteer patient organizations and professional 
societies. Currently there are 25 member organizations that belong to 
the DDNC. One of the original members of the coalition is the American 
Gastroenterological Association (AGA) of which I am a member and 
currently President-elect.
    The mission of the Digestive Disease National Coalition is to work 
cooperatively to improve access to and the quality of digestive disease 
health care in order to promote the best possible medical outcome and 
quality of life for current and future patients diagnosed with 
digestive diseases. The founder of the DDNC was a Crohn's disease 
patient who saw the need for increased digestive disease research and 
education.

                    THE IMPACT OF DIGESTIVE DISEASES

    Inflammatory bowel disease is just one of the scores of 
debilitating gastrointestinal conditions that afflict more than 62 
million Americans; others include hepatitis and other liver diseases, 
irritable bowel syndrome, diseases of the pancreas, ulcers, pediatric 
and adult gastroesophageal reflux, metabolic disorders, colorectal 
cancer, celiac disease, motility disorders, hemochromatosis, and other 
serious ailments.
    A recent study, The Burden of Gastrointestinal Diseases, conducted 
by the Lewin Group, concluded that a group of just 17 digestive 
diseases accounts for more than $41 billion each year in direct and 
indirect health care costs. In some of these areas, medical research 
has brought us closer to developing lifesaving treatments and cures. 
Examples of this include:

 The application of immunologic advances which have made liver 
        transplantation into a common life saving approach.
 The development of effective screening techniques for colon cancer.
 The genetic contributions to IBD, acute and chronic pancreatitis, 
        pancreatic and colon cancer and chronic diarrheal diseases 
        which are just on the cusp of recognition.
    Yet for every breakthrough, we still lack even a basic 
understanding of the causes, prevention, transmission and treatments 
for other diseases.
    IBD, the name given to Crohn's disease and ulcerative colitis, is a 
painful and disrupting disorder, which currently has no cure. We see in 
Crohn's disease the large and small intestines have become inflamed. 
This inflammation can result in excessive diarrhea, severe rectal 
bleeding, anemia, fever, as well as abdominal pain and cramping. Those 
battling this disorder have the trauma of multiple surgeries and the 
effects of toxic and potentially dangerous drugs. Ulcerative colitis 
attacks the large intestine, causing painful diarrhea, bleeding, and 
can ultimately lead to colon cancer, the third highest cancer 
population in the United States.
    IBD is an unpredictable disorder, symptoms vary in nature, 
frequency, and intensity. I wish I could say IBD was an easy disease to 
diagnose, but it is not. Misdiagnosis is common. Because there is no 
cure for IBD, the goal of medical treatment is to suppress the 
inflammation of the large and small intestine and the colon. By 
suppressing this inflammation, intestinal and colon tissue is permitted 
to heal and relieve many symptoms. Surgery can be an option to remove 
the diseased segments of the bowel or the colon. While surgery might 
allow patients to be symptom-free for many years, it is not a cure.

             LEGISLATIVE INITIATIVES IN DIGESTIVE DISEASES

    Mr. Chairman, the Digestive Disease National Coalition supports the 
passage of H.R. 290, The Inflammatory Bowel Disease Act. In addition to 
being endorsed by the coalition, the bill has been endorsed by many of 
the DDNC member organizations.
    The DDNC commends Congresswoman Sue Kelly (R-NY) for her leadership 
in introducing this legislation. This bipartisan bill has 176 co-
sponsors, 17 of which sit on this committee. The broad support of the 
bill reflects the tremendous potential in biomedical research related 
to IBD. The scientific community, led by CCFA, has developed a long-
range strategic plan for the future of IBD research and is in agreement 
that an additional investment in IBD research has the potential to 
yield greater scientific progress in clinical and general research.
    The DDNC also supports the passage of H.R. 3756, The National 
Commission on Digestive Diseases Act, introduced by Congressman Roy 
Blunt (R-MO) and Congressman Bobby Rush (D-IL). In 1976, Congress 
passed legislation that authorized the first National Commission on 
Digestive Diseases. The Commission was charged with assessing the state 
of digestive diseases in the United States identifying areas in which 
improvement in the management of digestive diseases could be achieved, 
and creating a long-range plan to recommend resources to effectively 
deal with promoting the GI research endeavor. The Commission, because 
it provided a credible roadmap for research and generated enthusiasm 
within the biomedical community, precipitated a number of research 
breakthroughs.
    In a time of limited fiscal resources to pursue an almost boundless 
reservoir of scientific opportunity, it is all the more imperative that 
the most promising avenues of research are traveled and that only the 
highest quality grants, trials, centers, and other programs are awarded 
funds. If the work of the first Commission serves as precedent, this 
initiative will once again galvanize the government, the research 
community, and the expanding population of people suffering from 
digestive diseases in a comprehensive and cost-effective national 
campaign to end the scourge of digestive disorders. Like its 
predecessor, the Commission should be directed to develop and recommend 
a long-range plan for the use and organization of national resources to 
effectively deal with digestive diseases.
    I would again thank you Mr. Chairman and the members of this 
subcommittee for holding this important hearing and ask that the 
committee pass H.R. 290, The Inflammatory Bowel Disease Act and H.R. 
3756, The National Commission on Digestive Diseases Act as soon as 
possible.

    Mr. Bilirakis. Thank you very much, Mr. Peura.
    I do not know whether any other members will return. Please 
do not think that there is a lack of interest in the subject; 
there is not. But it is a very hectic, frantic place, 
obviously, and a lot of other obligations and that sort of 
thing. Mr. Brown particularly extends his regrets because I 
know there is a group from Cleveland who--I guess they purchase 
from a charity, they purchase the right to have lunch with him 
or something. I think the Rotary Club or something of that 
nature. It is a critical that he has got to go. And so I know 
he apologizes.
    I am just going to ahead.
    For some time, Drs. Spiegel and Peura particularly, going 
back to the prior Administration in the sense that when the 
Democrats controlled the Congress and whatnot, we on this 
committee felt that we should not be determining what specific 
dollars should be earmarked for what specific disease. You 
know, the feeling was that the people at NIH know where the 
breakthroughs might be pretty darn close and, therefore, they 
are in a much better position if you will, to determine how 
many dollars should go to--Sue and I have talked about this. 
And so anyhow, it has always been our policy to not set out 
specific amount of dollars that will be going to research for a 
specific disease.
    We had doubled NIH funding. We had planned to continue to 
increase NIH funding. And we have been hoping that they would 
do the job adequately in terms of allocations of dollars.
    I mean, I have had Mohammad Ali come in here on behalf of 
Parkinson's and plead for more funding for Parkinson's. We can 
go on, ALS, Alzheimer's. You can just go on and on. And, of 
course, every group considers their disease, if you will, the 
highest priority and I do not blame me. So, you know, what can 
we really do here? So that has always been sort of our feeling.
    And I am going to ask for the two of you particularly, what 
your opinion is regarding that? Do you think it is right for 
Congress to decide what diseases deserve more attention than 
others or is this priority setting activity, as I have already 
said, best determined by the scientists and advisory counsels 
at the NIH.
    Dr. Spiegel?
    Mr. Spiegel. Thank you, Mr. Chairman.
    Let me just say that I again want to emphasize how 
enormously appreciative we are both of your leadership and the 
bipartisan support in Congress that permitted the doubling of 
the NIH budget and allowed us to initiate some of these 
important digestive disease initiatives that I alluded to.
    The reality is that science is extraordinarily complex. 
Because it is so complex, it is difficult often to predict 
where the next discovery is coming from. I gave the example of 
a diabetes drug which targets a receptor that turns out to be 
very highly expressed in the colon, and actually reduces 
inflammation in the colon first in animal models and then in 
the clinical trial, and which we are studying extensively and 
may become a therapy for ulcerative colitis. So for this reason 
we really do appreciate the flexibility in being able to set 
priorities.
    Having said that, and having met with your staff in April, 
it is vital for the NIH priority setting process to be as 
transparent as possible. I am committed to doing the best 
possible job we can in making that as transparent as possible. 
And we do that by inviting and sharing with stakeholders such 
as the CCFA, the AGA, that we are hearing from, from patient 
groups, traveling around the country meeting with patient 
groups. Again, as an example, the CCFA has crafted a tremendous 
strategic plan in IBD which was then extensively presented at 
an April 2003 meeting that Dr. James chaired at our Digestive 
Disease Interagency Coordinating Committee. This was an 
opportunity for all the NIH institutes to hear this plan and 
work toward its implementation.
    The bottom line ultimately is that, given the complexity of 
science, given the interrelation between things that you work 
on in one area that end up being very helpful to another area 
and the rapid pace with which these things change, we 
appreciate the flexibility to be able to set priorities, but in 
no way do we have any sense of arrogance or being isolated in 
saying that we are going to do this in a top down way. It has 
got to be transparent, it has got to have input from external 
groups and stakeholders, and that is what we are committed to.
    Mr. Bilirakis. Should it have in effect mandates from 
Congress in terms of the amount of dollars that should be 
allocated to that particular disease?
    Mr. Spiegel. Let me not be presumptuous. We are fortunate 
to live in an extraordinary democracy and you as the elected 
representatives of the people ultimately are responsible to 
your constituents. I view myself, as a physician, scientist and 
public servant, as accountable to you as the elected 
representative and want to work in partnership with you in each 
of these areas. To the extent that you offer us the flexibility 
to do this priority setting and do it in the most transparent 
way possible, we certainly appreciate that. And I know that Dr. 
Zerhouni as the NIH Director, who has interacted extensively 
with you, does. This gives us the opportunity.
    I will cite just one other example. There are many areas of 
both basic fundamental research as well as large projects. Take 
the human genome project. That was an enormous undertaking of 
the NIH. It was just the first step in a way, just getting all 
those 3 billion A, G, C and Ts, but it was the investment in 
that which was not specific to any disease that then allowed a 
tremendous acceleration in the discovery of the Crohn's 
susceptibility gene. That would not have happened without the 
investment in something that in this case was not disease 
specific. We have to keep that perspective in mind.
    I know you have heard from Dr. Zerhouni about the NIH 
Roadmap. In my many ways that is another example; the idea of 
increasing the harmonization of clinical research practices. 
That has got to be a benefit to people with IBD, with ALS, with 
every disease where we are desperate for better treatments and 
preventions, and where accelerating clinical trials and really 
being sure that we have the public's input is vital.
    Mr. Bilirakis. So you feel that you have adequate 
flexibility?
    Mr. Spiegel. We never take it for granted and are 
appreciative of having that flexibility.
    Mr. Bilirakis. Dr. Peura?
    Mr. Peura. Well, again, I want to take the opportunity 
again to thank you for having this hearing and allowing me to 
testify.
    Mr. Bilirakis. Is the mic on?
    Mr. Peura. It is a very difficult question you ask. I mean, 
as a clinician I see people with lots of different disease, but 
I know that inflammatory bowel disease, for example, what we 
are discussing here is a disease that causes significant 
morbidity, mortality. And we have heard from Adam about the 
morbidity that it caused him.
    Money spent for a particular disease is not just for that 
disease. And as Dr. Spiegel had mentioned, there are amazing 
outcomes that come from understanding immunology that cross 
many, many different disease states. Much of what we would 
learn by research in IBD would also be applicable to arthritis 
and neuro-degenerative disorders and other sorts of things. It 
is really not to say that anything is disease specific, because 
the fundamentals of research cross many disease entities.
    I do know that digestive diseases as a class of disease 
causes significant morbidity, mortality. And as I mentioned, I 
practiced in the BC era. Before really--and usually when I say 
that it is before cimetidine which was really probably one of 
the first advances in pharmacology in gastroenterology where we 
treated ulcer disease. Endoscopy was based on ulcer disease. 
Colostomy was based basically on ulcer disease, but now we 
recognize that ulcer disease is an infectious disease and 
research going in there, now we can cure a chronic disease. We 
spent a lot of money in the past on ulcer, but we recognize 
that the advances in endoscopy, colostomy, surgical techniques, 
all of that came from ulcer disease.
    So, I am obviously an advocate for money for digestive 
disease, but I think money is well spent on disease like IBD 
because of its implications for a variety of conditions.
    Mr. Bilirakis. You both said it well, but I think you said 
it more like a lawyer than you did--but you know, we have these 
good people, people like Adam coming and testifying here and 
wanting us, almost pleading with us--he has not done so, but 
really pleading with us that X amount of dollars, should be 
more funding for, as I said, Parkinson's or funding for ALS. 
And Sue has dollars in her particular piece of legislation that 
would be earmarked specifically for these diseases, and 
whatnot.
    So, you know, again the question is I mean what do we tell 
them? I cannot explain it the way you all have. What do we tell 
them? It is right for Congress to decide what diseases deserve 
more attention than others; and that is the question. And I 
think you both said in a sense that it is not right that 
Congress do that. At least I think Dr. Spiegel has said that in 
a round about way? It's important because we are met--you know, 
we do not have to meet with the patients just as you do. But 
they are pleading with us. Yes, they are pleading with you to 
get them well. They are pleading with us to allocate more 
money, which we can hope of course that more money would result 
in a cure. I mean, there is a hope thing there.
    Anything further?
    Mr. Peura. Well, Mr. Chairman, I mean I think that if I 
were answering that question and somebody came into my office 
and said that, I would probably say I want to be as committed 
to supporting research. Research in one particular area, as we 
have mentioned, is going to effect the disease that you have, 
the disease that other people have, too. The problem is when we 
do not fund research, when we shut the faucet off and, you 
know, research suffers and many generations of physicians 
suffer. I mean, I am faced with young physicians everyday that 
are making a decision whether they should have a research 
academic career or whether they should go into private 
practice. Private practice is vital. There have to be people 
out here taking care of the Adams in this world. But there also 
have to be people, the best and the brightest, that are 
available to apply for the NIH funding and for the other 
funding, CCFA funding that is out there.
    And to say, to sort of support that by being very positive 
in research and targeting those areas such as IBD that really 
will have an opportunity to make a major impact, not only on 
lives of sufferers of IBD, but patients with a variety of 
digestive conditions and nondigestive. I mean, I think that 
would be my answer, that I am committed to research. And I 
think it is important to have prioritization, but commitment to 
research and research that is going to bear fruit.
    As I said, following the best and most productive avenues I 
think is going to be important. And that is what a digestive 
disease commission would also help do.
    Mr. Bilirakis. Mr. DeRose, I believe it was you who 
accented maybe a lack of cooperation, lack of funding from CDC. 
Was it not you that made that comment? Yes. Why do you not 
expand on that?
    Mr. DeRose. For some time now the Appropriations Committee 
has had dollars allocated for an epidemiology study at the CDC, 
but those dollars have not been spent or focused in the area of 
IBD epidemiology study. And our frustration as an organization 
from a patient point of view has been that if the allocation is 
there and we can make a strong case as a patient group on the 
needs of this growing dilemma that we have in our country, why 
cannot those dollars be allocated appropriately by the CDC to 
move this research forward.
    And so our frustration stems from that. And to the point 
where we said we are going to put our money where our mouth is, 
and that is what we believe it is important. And we will go to 
the CDC and ask them to submit a grant, have it peer reviewed 
through the same process that we use for all our grants. And if 
it passes, and is approved, then we will fund dollars to it.
    But we felt it important enough that we take a lead on it 
on behalf of being able to come to you and say, Mr. Chairman, 
it's not a million Americans; it is 2-, it is 3-, it is 4 
million Americans.
    Mr. Bilirakis. We do not really know.
    Mr. DeRose. We do not know. The last study was done in 1991 
in Olmstead, Minnesota. And that, to me, is not the best 
representation of every aspect of the country, nor every ethnic 
group of the country. And there are some high incidents of this 
disease in ethnic groups.
    So I would say that that was our frustration with the CDC. 
And we still have a very positive relationship with them, and 
it will continue. But since they report to you as a 
stakeholder, I think it is incumbent on you and the 
Appropriations Committee to ask penetrating questions with the 
knowledge that you have to be able to say why are we not 
funding, why are we not proceeding with this study that has 
already been allocated.
    Mr. Bilirakis. Okay. And you will be available for any 
inquires we may have to help us?
    Mr. DeRose. Sure.
    Mr. Bilirakis. Good.
    Adam, you were at the CCFA event on Long Island, were you 
not? You were not there?
    Mr. Carron. I do not believe I was at that particular gala.
    Mr. Bilirakis. You were not at that particular one?
    Mr. Carron. I do not think--I am actually positive I was 
not there.
    Mr. Bilirakis. Well, I was there. And I started to get 
tears in my eyes during your testimony, and I guarantee I had 
them when she, of course, had something like a half hour to 
share her experiences with us. And, you know, the feeling was 
at the time that she was basically well. She had improved so 
tremendously, and then she went to camp, and you have told me. 
What has happened since she went to camp, tell us?
    Mr. Carron. She had her first surgery, which was at Mount 
Sinai, and it was supposed to be her final surgery.
    Mr. Bilirakis. Yes, she had had many surgeries. Well, she 
had surgeries over a period of years.
    Mr. Carron. Yes. I am sorry. This surgery at Mount Sinai 
was supposed to be her final surgery.
    Mr. Bilirakis. Right.
    Mr. Carron. And it was very similar to the operation that I 
had where they would take the small intestine and form a pouch 
basically and then remove the colon. And so kind of reconnect 
that there.
    But while she was at camp, she had an obstruction which 
formed by the scar tissue on the inside which had basically 
closed up and she was forced to come from camp and go back into 
the operating room to have that basically reopened up. And I 
believe she is currently recovering from that right now.
    Mr. Bilirakis. Yes. Well, I notice that you counsel a lot 
of these young people, adolescents and whatnot on this disease. 
Are these young people who have the disease?
    Mr. Carron. Yes. I am serving as kind of the co-director of 
youth activities for the Long Island Chapter. And through that 
I am--I get deferred to a lot of younger kids. And I will go to 
the hospital to visit them or call them, and basically kind of 
like relax them and let them know what they are about to go 
through and to share my experiences with them so that--to that 
basically so that they will be able to deal with the IBD as 
best as they can and feel most confident doing so.
    Mr. Bilirakis. Well, you are to be commended, Adam, for 
your courage, for your toughness, for your willingness to come 
here and share with us. And even though there are not other 
members other than Ms. Kelly here to ask our questions, a 
record is being taken. And I assure you it will all be very, 
very helpful.
    I have taken more than my time, because not only be the 
Chair but also because nobody is here to do it, but I will 
yield to Ms. Kelly for her inquiry.
    Ms. Kelly. I thank you, Mr. Chairman. I thank you so much 
for letting me here and be a part of this hearing today. I 
really appreciate your generosity on that.
    Mr. DeRose, I want to go back to the issue about the 
support that CCFA has given the Centers for Disease Control. As 
I understand it, the CDC has not provided any support for IBD 
epidemiology program, that program.
    The IBD Act includes the provision that will formally 
establish the epidemiology program, and it is really needed.
    Mr. Chairman, I think it is important that we recognize 
organizations that contribute their own resources to projects 
that benefit the general public good and to promote the types 
of public/private partnerships in the way that the CCFA has 
stepped forward to do. This is an unusual organization. The 
members of CCFA have demonstrated that they are willing to fund 
research, they have put a lot of money in research and in 
education. And I just want to go on record as saying I feel 
very strongly that we in Congress owe it to organizations like 
that to try to at least match the amount that they have 
collected and put into the research on their own because it is 
the type of research, as both the doctors on this panel have 
pointed out, will extend the life of many people from many 
different kinds of diseases. Autoimmune disease are a very 
particularly interesting group of diseases. And bits of 
research can be put together to help others all the way along 
the line.
    And the other thing I like to just go on record as saying 
is this Congress does fund specific diseases. We fund research 
in AIDS, we fund research in cancer and we just passed a bill 
to fund research on stroke. It seems to me if we do that for 
those three diseases where we have such a strongly committed 
group of people who have collected such large amounts of money 
on their own and offered it to the CDC, we in Congress really 
could help a lot of people if we would at least be able to 
match that by requesting of the CDC that amount of money.
    I do not know, Mr. DeRose, if you would like to--I mean, I 
welcome this opportunity to ask you question. I would like to 
hear if you have got any more thoughts on this, because I think 
this is extraordinary? I do not know of other groups that--you 
are not a large groups, you are a small group and you have 
collected a lot of money and you have put a lot of research 
grants out there on your own. You have done a lot just to try 
to build the information base. And that is really what, I 
think, in part you are asking for now. And I would just like to 
point out that that is the reception that I got from you, and I 
want to know if you would like to just respond to that?
    Mr. DeRose. Well, Congresswoman, I would be echoing some of 
the points that you have made and think I mentioned earlier. I 
really do think that it is important for organizations such as 
ours to take a leadership role. If a government agency is not 
going to take action on an objective that has been laid out for 
them, then we are. And we will make the investments so that we 
can show to them, as well as to the community, that we are in 
position to advance knowledge and advance information so that 
we can get the true picture of this disease into the right 
hands so that you can make those kinds of decisions that are 
needed.
    I personally feel that we as a disease state should be 
getting more dollars allocated to this disease. It is a complex 
disorder. It is not a one gene type of a disease, it is 
multiple genes. It is probably, depending on the literature, 10 
to 20 genes that affect this disease state and we have only 
discovered one. And there is great activity that is taking 
place right now in other chromosomes. Chromosome 5 and 10 that 
I think once verified, and it is being verified now in 
independent labs around the world, we may be on the 
breakthrough of other genes that it may behoove us for further 
understanding----
    Mr. Bilirakis. Well, but if the gentleman will yield. But 
do you not make that case to NIH? You made that case to CDC, 
and apparently they have not been cooperative. But you make 
that case.
    I mean, who are we to make that case to? Who are we to 
understand the breakthrough and the complexities, as you 
indicated, and the technicalities and whatnot, whereas those 
people are the experts? And as long as we give them the 
funding, it seems like you should be able to make the case to 
them and then, hopefully, the funding would be adequate.
    Mr. Spiegel. If you will permit me, I would like to 
interject something which is relevant to this point and 
actually harks back in a way to what Mr. Buyer I believe had 
said in some of his initial statements.
    We work with the CCFA in a unique kind of public/private 
partnership which I really value, and I think that Congressman 
Kelly has underscored.
    Mr. Bilirakis. I think it is terrific.
    Mr. Spiegel. In a sense they are providing seed money to 
investigators who would not be able to get NIH grants without 
preliminary data. This is the system that we have. And it is a 
conservative system, but it has to be because currently under 
the best of circumstances only one of three applications can be 
funded. The reality is that we have to be extremely circumspect 
and we want some preliminary evidence that they can accomplish 
what they are setting out to do.
    It is the CCFA and organizations like it, and there are 
some other organizations in our institute that we work with 
this way, who provide that seed money. As you heard from Mr. 
DeRose, it then leverages multifold because of the generous 
resources you have given us.
    That is one dimension that I think is very important, and I 
really do value that partnership.
    Let me just illuminate, and this comes to Mr. Buyer's point 
earlier, that we want to be extremely circumspect with every 
hard earned tax dollar that you give us in terms of how it is 
used. We do not want to reinvent the wheel. We do not want to 
do the work of private industry. We are not talking about 
nonprofit voluntary groups such as the CCFA, but industry in 
terms of the pharmaceutical and biotech industry. In that 
respect we are really now very much focused on what we call a 
translational emphasis for inflammatory bowel disease. We are 
spending at NIH as a total for fiscal year 2003 of $58.4 
million on IBD. That money, though, is really targeted to areas 
in which we believe only NIH, only government support could 
really make a difference. Let me just illuminate that.
    We led an almost groundbreaking kind of meeting with the 
FDA in January of 2003. Dr. James led that meeting, and 
organized that meeting. The drug industry and biotech industry 
were there trying to work out ways to get better indices of the 
disease. There's something called the Crohn's disease activity 
index, which is a very crude measure of disease. The fact is 
that you have to constantly do endoscopy, and Dr. Peura is an 
expert he knows how to do this, but it is invasive in a way. We 
need ways that are noninvasive, a simple blood test for example 
that could tell us how bad this disease is. These are the kinds 
of things, fundamental investments, which industry cannot do 
but which we can do; we have set these out as important goals.
    Finally, just as an example, there is an interesting 
situation vis-a-vis ulcerative colitis versus Crohn's disease. 
There are actually over 10 or more new agents in the pipeline 
at varying stages short of approval, some of which will come 
through, for Crohn's disease. There is lesser activity, 
actually, in ulcerative colitis. Why is that? The fact is that 
you have this drastic maneuver of colectomy, removing the 
entire large bowel as a ``cure.'' It is an alternative, but it 
is not the desirable outcome that we want. We want to be able 
to avoid that with effective kinds of treatments. For that 
reason we have focused extensive attention on ulcerative 
colitis. With this diabetes drug that I mentioned, there is no 
guarantee that is going to work, but we will not find out 
unless we are supporting that kind of effort.
    Another example is the a drug azathioprine. It is a generic 
drug which is an immunosuppressant. It is now generic so there 
is no patent exclusivity and there is less incentive for drug 
companies to really be working or testing that. Again, Dr. 
James' leadership is involved in funding studies based at the 
University of Chicago and other places that are looking at the 
metabolism of that drug. There are wide differences among 
children and others in terms of the metabolism of that drug so 
that we need to be able to know what is the most effective dose 
that will suppress the disease and yet not have dangerous side 
effects. This is the kind of work that only NIH can support, 
and this is why we are so grateful for the support you are 
giving us to focus our attention on these areas and the 
translational opportunities.
    Ms. Kelly. Reclaiming my time.
    Dr. Spiegel, I would like to follow up with you on that 
because I am not really clear right now. From what you said it 
seems to me that we need to do the epidemiologic study that the 
CCFA has asked for. We have not gotten the support for that 
study from the CDC. And what I do not understand is are you are 
talking about a lot of things. There is a lot of things other 
there. But what I do not understand is why if NIH and CDC are 
connected, there cannot be some pressure from NIH on CDC to get 
that epidemiologic study done.
    Mr. Spiegel. It is an excellent point. I can tell you, I am 
an ambitious guy, and I think an energetic guy, and I find that 
running my institute gives me a full time job as opposed to 
necessarily running other parts of the CDC. We are sensitive to 
what you are saying. We value colleagues at the CDC and we 
actually work with them in a variety of areas, often in very 
close partnership.
    The physician and epidemiologist, I think her name is 
Siobhan O'Connor, at the CDC has been involved in conducting 
this study. She is someone we have had up to the NIH to 
Bethesda. We have wanted to hear from her about her methods. We 
are in dialog about these kinds of things. Maybe you will 
accuse me of not being sort of forceful enough, but I have not 
seen it as within my purview to dictate what they should be 
doing in regard to this study.
    Ms. Kelly. Well, I am just a little puzzled about the 
money. We are giving NIH a lot of money, CDC has a lot of 
money. Crohn's and Colitis Foundation has given CDC a lot of 
money. And there is no movement on the part of CDC.
    Mr. Chairman, I am wondering if perhaps it would be helpful 
to the CCFA if you and I wrote a letter directly to the CDC and 
asked them why there is no movement on that and just weigh in 
on the fact that it might be----
    Mr. Bilirakis. We have been known to do that. And I have 
already talked to Cheryl about it. That is why I made the 
comment about maybe asking Mr. DeRose would be available when 
we might need some information toward that.
    Yes, we will follow up as far as that is concerned.
    Your time has basically expired.
    Ms. Kelly. Thank you.
    Mr. Bilirakis. Look, we want to do the right thing, okay. 
We sort of ask, you know, put yourselves in our shoes 
sometimes, too. You know everybody, I mean there is never 
enough money, No. 1. There certainly is not ever enough money 
for any of the diseases, and yet the squeaky wheel gets the 
grease and there have been some diseases that the wheels have 
been more squeaky than others and have gotten funding, mostly 
through the appropriation process. But we do want to do the 
right thing. And we also have to realize that money--money is 
significant as far as research is concerned, but there are 
other ways in addition to--in addition to, not in lieu thereof 
but obviously in addition to things that Adam have shared with 
us that he is doing with some of the younger people and things 
of that nature could be helpful.
    So, to adjourn the hearing, but I would like to invite you, 
and I mean it sincerely and I do it every hearing, is to feed 
into us--well, first of all we are going to have a series of 
questions to you that we will submit to you in writing and we 
are requesting that you respond to them in a timely fashion, 
number. Particularly as it might involve a piece of 
legislation.
    I am hopeful that before this year is up we are going to 
have some legislation on this issue. I cannot tell, I am not 
all powerful enough to tell Bobby Rush that his legislation 
will become law, and I am not powerful enough to tell Sue that 
her legislation will become law. But I am hoping that we will 
have a good piece of legislation that will combine some of the 
better parts of both pieces of legislation that, hopefully, 
will be set up in such a way that they will get the proper 
credit for it. Because that is important. They do not do it for 
credit, but I think it is obviously important that that be 
taken into consideration.
    So taking that into consideration, respond to our questions 
as soon as you can. Feed into us any additional points you want 
to make that you have not made here today that might come to 
mind, any suggestions you may have to crank into the 
legislation, things of that nature. Let us not fight the battle 
of what this President did or what the previous President did, 
or anything of that nature. I mean, that is the wrong way to 
go. That is what really gets us off the track when we get into 
this partisanship business, and it happens unfortunately much 
too much up here. And we throw stones at each other rather than 
get our heads together and do what is right for the public out 
there. And you saw a little bit of an idea of that earlier 
today. And that is unfortunate. But I do know that all of these 
people are concerned, and I do know that they ultimately want 
to do the right thing. It is how we go about it sometimes that 
is maybe not the right way to go.
    Adam, you come up with any ideas; first of all, if you see 
Ariana, give her my best, but also any ideas that you might 
have that would be helpful to us in order to be able to do a 
better job, please do not hesitate.
    Thank you so very much, gentlemen.
    The hearing is adjourned.
    [Whereupon, at 1:07 p.m. the hearing was adjourned.]
    [Additional material submitted for the record follows:]

 Response for the Record by David A. Peura, Digestive Disease National 
                               Coalition

    Q. As has been stated, Congressman Blunt and I have introduced a 
bill that would create a temporary commission to conduct research on 
digestive diseases. Historically, Congress has established similar 
research commissions on many disease areas. Can you please comment on 
why it makes sense for Congress to act and create a Digestive Diseases 
Research Commission?
    A. During the late 1990s, digestive diseases researchers and 
clinicians reported that there was a growing incidence in the number of 
patients with serious gastrointestinal diseases. In an effort to gain 
external validation for this growing problem, the AGA contracted with 
the Lewin Group in 2001 to conduct a study entitled ``The Burden of 
Gastrointestinal Diseases.'' The study focused on just 17 of the scores 
of digestive diseases and included a comprehensive analysis of at least 
5 nationally recognized health statistics data bases to determine the 
overall annual financial burden, in direct and indirect costs, 
associated with these 17 diseases. The study found that the combined 
direct and indirect costs to society for these diseases in 2000 were 
$42 billion. While the study only focused on a subset of all digestive 
diseases, the results have been recognized as substantive documentation 
that the U.S. faces a crisis in terms of the incidence of digestive 
diseases.
    A Digestive Diseases Research Commission would focus the research 
community on setting priorities and research goals and address these 
issues in a trans-NIH and trans-scientific community fashion. This 
mechanism has been recognized as a successful model by scientists for 
over 35 years, with the first Digestive Diseases Research Commission in 
the 1970s as a prime example. The commission would be required to 
report back to Congress to ensure accountability in its deliberations.
    Q. The first Digestive Diseases Research Commission was created 
back in the 1970s. Dr. Peura, can you comment on the benefits of 
creating a second Research Commission on digestive diseases?
    A. A second commission would provide an important opportunity to 
update the work of the very successful first Digestive Diseases 
Research Commission. Approximately 15-18 commissioners would be 
appointed by the Secretary of HHS, drawing from the ranks of 
distinguished scientists in digestive and other diseases, 
representatives from research in other federal agencies, patient 
advocates, the pharmaceutical industry, technology experts, and 
industry leaders impacted by the loss of productivity related to 
digestive diseases. Their efforts would concentrate on the following:

 Developing a comprehensive forward-looking strategic plan for 
        addressing the crisis in digestive diseases over the next 5-10 
        years;
 Addressing overarching approaches to new and effective clinical 
        trials, specific clinical studies, refining new technologies 
        and setting standards in these areas;
 Recommending more collaborative, inter-institute research and ways to 
        accelerate new initiatives to more quickly and efficiently find 
        effective treatments and cures;
 Assessing the need for more researchers in digestive diseases to meet 
        the challenges posed by the growing incidence of digestive 
        disorders;
 Updating the work of the first commission due to the advent of 
        genomics and genetics research, new technologies and the 
        discovery of new digestive diseases and how to apply this 
        information to new research initiatives.
    The efforts of the first commission led to the advent of powerful 
new therapies such as interferons and monoclonals and new technologies 
such as CT colonography and capsule endoscopy. The discovery of the H. 
pylori bacteria as the cause of ulcers and the IBD 5 gene's role in 
Inflammatory Bowel Disease are also major breakthroughs set in motion 
by the first commission. The first commission also recommended the 
establishment of the Digestive Disease Core Research Centers program 
which remains a model of collaborative research even today.
    We still face many new challenges in the area of obesity, hepatitis 
and hepatomas, and the potential use of foodborne pathogens as 
bioterrorism agents just to name a few of many existing diseases and 
threats.
    Q. Some critics of our bill contend that a Digestive Diseases 
Research Commission duplicates NIH functions already in place, such as 
the work done by the National Institute of Diabetes and Digestive and 
Kidney Diseases. Would you please tell us how a temporary research 
commission would supplement the work of the NIDDK and its advisory 
committee?
    A. Actually, the NIDDK Advisory Council, which meets 3 times per 
year for approximately 1\1/2\ days for each meeting, operates much 
differently than a commission. The Council is comprised of 18 members, 
only 2 of which are experts in gastrointestinal diseases. The primary 
function of the Council is to provide second level peer review for 
current research grants, grant issues and budget issues (i.e. the size 
of grants) which occupies approximately 50% of their meeting time. The 
other portion of the meeting is occupied with updates on ongoing NIH 
programs. As such, the NIDDK Advisory Council, as with other institute 
councils, does not address strategic issues or long-range research 
planning, but more of the ``here and now'' operational issues. It also 
does not operate as an inter-institute body. The Council serves an 
important operational role for the NIDDK but has a clearly distinct 
mission from that of a commission.
    It should also be noted that one of the major outcomes of the 
advisory council process, scientific ``consensus conferences'' on 
specific diseases, reflect a culmination of research and not new 
research and, as a result, are not intended to develop new knowledge. A 
Digestive Diseases Research Commission would complement the Advisory 
Council's work by providing a forward-looking mechanism for developing 
a long-range plan to help accelerate research into more effective 
treatments and cures for digestive diseases.
    Q. Can you tell us the differences between creating an internal 
research commission and an external commission as Mr. Blunt and I are 
proposing? What are the merits and/or disadvantages of each approach?
    A. An internal commission would impose upon NIH to create a 
potentially permanent structure which is currently not in place. The 
logistics of creating such a body would be cumbersome, potentially 
disruptive of the current work being undertaken by scientists and 
administrators and impractical as it would require individuals to 
change roles and methods of operating for a defined period of time. NIH 
might also be required to hire more full time staff to accommodate an 
internal commission and, thereby, create a more permanent and costly 
alternative to an external commission. Digestive diseases research is 
also such a broad area of investigation that it lends itself more to 
community participation than internal deliberation. Only 10% of all 
research monies are designated to intramural research so the depth of 
expertise does not exist within the structures of NIH. Finally, due to 
the restrictions placed upon federal agencies, an internal commission 
exercise would be confined to supporting the parameters of the 
President's budget request and would not be conducive to creative 
thinking and long-range planning, thereby being more reactive in 
nature. In addition, the effort would not be a trans-NIH initiative 
and, therefore, would not be a centerpiece of other institutes' 
programs.
    An external commission would provide an opportunity for academic 
gastroenterologists and other specialists to provide leadership and 
accept responsibility for the elements of a comprehensive long-range 
plan addressing digestive diseases. This exercise would also energize 
the digestive diseases community. Since 90% of NIH research is not 
conducted in Bethesda, but elsewhere in the country, it is essential to 
energize, and achieve buy-in, from the community that ultimately will 
implement the plan. This approach would also be the most effective in 
helping ensure any initiatives included in the plan are trans-
scientific community and trans-institute.

                                 
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