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NCI CANCER RESEARCH: TODAY’S PROGRESS; 

TOMORROW’S CHALLENGES 

HEARING 

BEFORE THE

SUBCOMMITTEE ON HEALTH 

OF THE

COMMITTEE ON ENERGY AND 

COMMERCE 

HOUSE OF REPRESENTATIVES 

ONE HUNDRED ELEVENTH CONGRESS 

SECOND SESSION 

MARCH 23, 2010 

Serial No. 111–108 

( 

Printed for the use of the Committee on Energy and Commerce 

energycommerce.house.gov 


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COMMITTEE ON ENERGY AND COMMERCE 

HENRY A. WAXMAN, California, Chairman 

JOHN D. DINGELL, Michigan 

Chairman Emeritus 

EDWARD J. MARKEY, Massachusetts 

RICK BOUCHER, Virginia 

FRANK PALLONE, JR., New Jersey 

BART GORDON, Tennessee 

BOBBY L. RUSH, Illinois 

ANNA G. ESHOO, California 

BART STUPAK, Michigan 

ELIOT L. ENGEL, New York 

GENE GREEN, Texas 

DIANA DEGETTE, Colorado 

Vice Chairman 

LOIS CAPPS, California 

MICHAEL F. DOYLE, Pennsylvania 

JANE HARMAN, California 

TOM ALLEN, Maine 

JANICE D. SCHAKOWSKY, Illinois 

CHARLES A. GONZALEZ, Texas 

JAY INSLEE, Washington 

TAMMY BALDWIN, Wisconsin 

MIKE ROSS, Arkansas 

ANTHONY D. WEINER, New York 

JIM MATHESON, Utah 

G.K. BUTTERFIELD, North Carolina 

CHARLIE MELANCON, Louisiana 

JOHN BARROW, Georgia 

BARON P. HILL, Indiana 

DORIS O. MATSUI, California 

DONNA M. CHRISTENSEN, Virgin Islands 

KATHY CASTOR, Florida 

JOHN P. SARBANES, Maryland 

CHRISTOPHER S. MURPHY, Connecticut 

ZACHARY T. SPACE, Ohio 

JERRY MCNERNEY, California 

BETTY SUTTON, Ohio 

BRUCE BRALEY, Iowa 

PETER WELCH, Vermont 

JOE BARTON, Texas 

Ranking Member 

RALPH M. HALL, Texas 

FRED UPTON, Michigan 

CLIFF STEARNS, Florida 

NATHAN DEAL, Georgia 

ED WHITFIELD, Kentucky 

JOHN SHIMKUS, Illinois 

JOHN B. SHADEGG, Arizona 

ROY BLUNT, Missouri 

STEVE BUYER, Indiana 

GEORGE RADANOVICH, California 

JOSEPH R. PITTS, Pennsylvania 

MARY BONO MACK, California 

GREG WALDEN, Oregon 

LEE TERRY, Nebraska 

MIKE ROGERS, Michigan 

SUE WILKINS MYRICK, North Carolina 

JOHN SULLIVAN, Oklahoma 

TIM MURPHY, Pennsylvania 

MICHAEL C. BURGESS, Texas 

MARSHA BLACKBURN, Tennessee 

PHIL GINGREY, Georgia 

STEVE SCALISE, Louisiana 


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SUBCOMMITTEE ON HEALTH

FRANK PALLONE, JR., New Jersey, Chairman 

JOHN D. DINGELL, Michigan 

BART GORDON, Tennessee 

ANNA G. ESHOO, California 

ELIOT L. ENGEL, New York 

GENE GREEN, Texas 

DIANA DEGETTE, Colorado 

LOIS CAPPS, California 

JANICE D. SCHAKOWSKY, Illinois 

TAMMY BALDWIN, Wisconsin 

MIKE ROSS, Arkansas 

ANTHONY D. WEINER, New York 

JIM MATHESON, Utah 

JANE HARMAN, California 

CHARLES A. GONZALEZ, Texas 

JOHN BARROW, Georgia 

DONNA M. CHRISTENSEN, Virgin Islands 

KATHY CASTOR, Florida 

JOHN P. SARBANES, Maryland 

CHRISTOPHER S. MURPHY, Connecticut 

ZACHARY T. SPACE, Ohio 

BETTY SUTTON, Ohio 

BRUCE L. BRALEY, Iowa 

NATHAN DEAL, Georgia, 

Ranking Member 

RALPH M. HALL, Texas 

BARBARA CUBIN, Wyoming 

JOHN B. SHADEGG, Arizona 

STEVE BUYER, Indiana 

JOSEPH R. PITTS, Pennsylvania 

MARY BONO MACK, California 

MIKE FERGUSON, New Jersey 

MIKE ROGERS, Michigan 

SUE WILKINS MYRICK, North Carolina 

JOHN SULLIVAN, Oklahoma 

TIM MURPHY, Pennsylvania 

MICHAEL C. BURGESS, Texas 


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C O N T E N T S 

Page 

Hon. Frank Pallone, Jr., a Representative in Congress from the State of 

New Jersey, opening statement .......................................................................... 1 

Hon. Donna M. Christensen, a Representative in Congress from the Virgin 

Islands, opening statement ................................................................................. 9 

Hon. Joe Barton, a Representative in Congress from the State of Texas, 

prepared statement .............................................................................................. 11 

Hon. Zachary T. Space, a Representative in Congress from the State of 

Ohio, opening statement ...................................................................................... 15 

Hon. Henry A. Waxman, a Representative in Congress from the State of 

California, prepared statement ........................................................................... 19 

Hon. Gene Green, a Representative in Congress from the State of Texas, 

prepared statement .............................................................................................. 24 

Hon. Marsha Blackburn, a Representative in Congress from the State of 

Tennessee, prepared statement .......................................................................... 116 

WITNESSES

Anna D. Barker, Ph.D., Deputy Director, National Cancer Institute ................. 27 

Prepared statement .......................................................................................... 31 

Answers to submitted questions ...................................................................... 129 

Kristin Fitzgerald, Naperville, Illinois ................................................................... 63 

Prepared statement .......................................................................................... 66 

Megan Gordon Don, M.H.S., Chair, Deadly Cancer Coalition, Director of 

Government Affairs, Pancreatic Cancer Action Network ................................. 74 

Prepared statement .......................................................................................... 77 

Answers to submitted questions ...................................................................... 137 

Robert S. DiPaola, M.D., Member, American Association of Cancer Research, 

and Director, Cancer Institute of New Jersey ................................................... 86 

Prepared statement .......................................................................................... 88 

Answers to submitted questions ...................................................................... 139 

Jeff Allen, Ph.D., Executive Director, Friends of Cancer Research ..................... 94 

Prepared statement .......................................................................................... 96 

Answers to submitted questions ...................................................................... 143 

SUBMITTED MATERIAL

Statement of the International Myeloma Foundation, submitted by Ms. 

Capps ..................................................................................................................... 4 

Statement of the Lung Cancer Alliance ................................................................. 118 

Statement of the Association of American Cancer Institutes .............................. 124 


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NCI CANCER RESEARCH: TODAY’S PROGRESS; 

TOMORROW’S CHALLENGES 

TUESDAY, MARCH 23, 2010 

HOUSE OF REPRESENTATIVES, SUBCOMMITTEE ON HEALTH, COMMITTEE ON ENERGY AND COMMERCE, Washington, DC. 

The Subcommittee met, pursuant to call, at 2:05 p.m., in Room 

2322 of the Rayburn House Office Building, Hon. Frank Pallone, 

Jr. [Chairman of the Subcommittee] presiding. 

Members present: Representatives Pallone, Eshoo, Green, Capps, 

Schakowsky, Baldwin, Christensen, Castor, Space, Waxman (ex 

officio), Shimkus, Buyer, Burgess, Gingrey and Barton (ex officio). 

Also present: Representative Biggert. 

Staff present: Sarah Despres, Public Health Counsel; Anne Morris, Professional Staff; Stephen Cha, Professional Staff; Alvin 

Banks, Special Assistant; Aarti Shah, Minority Professional Staff; 

Clay Alspach, Minority Counsel; and Ryan Long, Minority Professional Staff. 

OPENING STATEMENT OF HON. FRANK PALLONE, JR., A REPRESENTATIVE IN CONGRESS FROM THE STATE OF NEW JERSEY 

Mr. PALLONE. I call the hearing to order. 

Let me mention that votes were just called, so we will try to do— 

maybe we will see if we can get in the three people that are here, 

or at least myself and Mr. Shimkus, and then we will have to take 

a break and come back. 

But let me say today, as you know, we are having a hearing on 

NCI Research: Today’s Progress; Tomorrow’s Challenge, and basically examining the cancer research efforts at the National Cancer 

Institute. It is a very important topic that is of great interest to 

many of my colleagues. Many of my colleagues have been asking 

us to have this hearing for some time. 

Cancer is an ugly word. It is an even uglier disease. Unfortunately, cancer touches us all and everyone in this room has had either direct or indirect contact with cancer. Either a mother, a father, a brother, a sister, in almost all cases a friend has been diagnosed with some form of cancer. And some have fought the odds 

and survived. Others tragically have lost the battle. The bottom 

line is, far too many of us have lost people we care deeply about 

to this horrible illness. Cancer is the second leading cause of death 

in the United States. In fact, it accounts for almost every one in 

four deaths. Half of all men and one-third of all women will deVerDate Mar 15 2010 13:13 Dec 14, 2012 Jkt 076020 PO 00000 Frm 00008 Fmt 6633 Sfmt 6633 E:\HR\OC\A020.XXX A020

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velop cancer during their lifetimes, and today, millions of people 

are living with cancer or have had cancer. 

The issue is a very emotional and personal one for me. In the 

spring of 2008, my mother was diagnosed with pancreatic cancer, 

and she passed away late that same year. Pancreatic cancer is obviously one of the diseases that we are looking at today in particular. 

Fortunately, we have made great progress in cancer research 

over the last decades. Just 40 years ago, there were only 3 million 

cancer survivors. Today, 3 percent of the U.S. population has survived cancer. We have new therapies that specifically target the 

malignant tumors in an attempt to lessen the impact of the therapy on the patients. We have better screening and early detection 

methods which help identify cancer in the stages when it is more 

successfully treatable. 

With the support of Congressional efforts over the next 2 years, NCI will grant $1.3 billion to cancer researchers across the country, and this money will go to fund additional grants for first-time 

investigators, thereby providing additional opportunities for the 

next generation of investigators and ensuring that the pipeline for 

new researchers remains stable. This additional funding will also 

go to initiatives that are expected to propel us forward in our understanding of cancer in the near future including efforts on the 

cancer genome atlas. I want to find out more about that today obviously. Also, research on personalized cancer care and new therapy 

development, collaborative cancer care work, just to name a few. 

Nevertheless, we face serious challenges regarding rare and 

deadly cancers. Cancers that have been termed the deadliest cancers have a 5-year survival rate of less than 50 percent. These cancers include ovary, brain, myeloma, stomach, esophagus, lung, liver 

and, of course, pancreas. Pancreatic cancer is the deadliest cancer 

with a 5-year survival rate of only 5 percent. Combined, the deadly 

cancers make up half of all cancer deaths yet they receive a fraction of the research funding as compared to other cancers. It is 

clear that we still know far too little about these cancers. We have 

no or limited early detection and screening. By the time one of 

these cancers is diagnosed, it has often progressed too far for treatment to be successful. And I know some of our witnesses here 

today will speak to these issues and we look forward to their comments and recommendations. 

As I think you know, we are a legislative subcommittee and so 

when we have oversight hearings like this, they are designed to try 

to see whether we should be legislating, so I want everyone to keep 

that in mind in terms of their recommendations as we move forward today. 

Mr. PALLONE. And now I would like to recognize our ranking 

member, Mr. Shimkus. 

Mr. SHIMKUS. Thank you, Chairman Pallone, for holding this important hearing about the progress and the challenges we face in 

tracking rare forms of cancer. 

I want to thank all the witnesses for being here today to help 

educate members on this issue. 

I have long been a supporter of cancer research but my former 

legislative director, Ray Fitzgerald’s, battle with gastric cancer 


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brought this issue much closer to my heart. As Chairman Pallone 

said, everybody has personal stories and experiences. 

I want to thank Ray’s wife, Kristin, and their three young girls, 

Nora, Maggie and Lucy, who I think are hiding somewhere not disrupting—oh, they are back there—for testifying before the committee today. They were an inspiration to me and many others as 

they publicly shared the highs and lows of Ray’s cancer. Kristin 

has continued to work tirelessly to expand efforts in the field of 

gastric cancer. I know her knowledge on the subject here today will 

help the committee advance efforts in that area. Also just for the 

record, she is a former congressional staffer, having worked for 

Harris Faywall, who is well known in the health legislative area, 

Judy Biggert and now our Republican leader, John Boehner. I also 

would like to thank Dr. Barker for being here today from the National Cancer Institute. I commend you and the NCI on the many 

things you do to make cancer curable, and I look forward to your 

institutional knowledge on what we can do from the federal side to 

progress our efforts in research on rare forms of cancer. 

With that, Mr. Chairman, I yield back my time. 

Mr. PALLONE. Thank you, Mr. Shimkus. 

I think we have time for Mr. Gingrey, the gentleman from Georgia. 

Mr. GINGREY. Mr. Chairman, I will waive my opening. 

Mr. PALLONE. You want to waive? OK. 

Ms. Capps, our vice chair. 

Mrs. CAPPS. Thank you, Mr. Chairman. Before I make a brief 

statement, I wish to insert for the record written testimony of the 

International Myeloma Foundation regarding NCI Cancer Research: Today’s Progress, Tomorrow’s Challenges. 

[The information follows:] 


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IMF 

Written Testimony of the 

INTERNATIONAL MYELOMA FOUNDATION 

regarding 

NCI CANCER RESEARCH: TODAY'S PROGRESS; TOMORROW'S CHALLENGES 

Subcommittee on Health 

Energy and Commerce Committee 

US House of Representatives 

March 23, 2010 

The International Myeloma Foundation (IMF) appreciates this opportunity to submit 

testimony related to research on the most deadly forms of cancer at the National Cancer 

Institute (NCI). IMF is the oldest and largest myeloma foundation dedicated to improving 

the quality of life of myeloma patients while working toward prevention and a cure. 

Background on Myeloma 

Myeloma is a cancer of plasma cells that develops in the bone marrow and affects the 

production of red cells, white cells, and stem cells. It is also called "multiple myeloma" 

because multiple areas of bone marrow and bone may be involved. Myeloma is the 

second most common blood cancer after lymphomas, but it is also a relatively rare or 

"orphan" disease as defined by the Orphan Drug Act. 

Myeloma is an incurable form of cancer. In 2009, 20,580 Americans were diagnosed 

with myeloma and 10,580 lost their battle with this disease. At anyone time there are 

over 100,000 myeloma patients undergoing treatment for their disease in the U.S. The 

5-year survival for multiple myeloma patients diagnosed in 2009 was estimated at only 

about 35% by the American Cancer Society. 

Over the past 40 years the incidence of many cancers has decreased, but myeloma 

cases are increasing in incidence. Once a disease of the elderly, it is now found in 

increasing numbers in people under 65 and patients in their 30s are not uncommon. 

IMF-funded research suggests that this increase is associated with environmental toxins; 

as just one example, a disproportionate number of myeloma cases have been 

diagnosed in clean-up and rescue workers at the 9/11 World Trade Center site. 

Although important advances have been and continue to be made in the treatment of 

myeloma since the IMF was founded in 1990, these treatments all come with significant 

and problematiC side effects that significantly impact quality of life. Even while they live 

with the disease, myeloma patients can suffer debilitating fractures and other bone 

12650 Riverside Drive Suite 206, North Hollywood, CA 91607 

800-452-CURE (2873) 

818-487-7455 telephone 818-487-7454 fax 

www.myeloma.org 
5 


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IMF 

Written Testimony of the Intematlonal Myeloma Foundation 

regarding 

NCl Cancer Research: Today's Progress; Tomorrows Challenges 

Subcommittee on Hea!th 

Energy and Commerce Committee 

US House of Representatives 

March 23, 2010 

disorders, severe side effects of their treatment, and other problems that profoundly 

affect their quality of life, and have major impact on the cost of their health care. 

Deadly Cancer Research at the National Cancer Institute 

The IMF joins with its partner members of the Deadly Cancers Group in bringing to the 

attention of this Committee that, while a number of cancers have achieved 5-year 

survival rates of over 80% since passage ofthe National Cancer Act of 1971, significant 

challenges still remain for other types of cancers, particularly the most deadly forms of 

cancer. More than half of the 562,340 cancer deaths in 2009 were caused by eight 

forms of cancer with 5-year survival rates of 45% or less; pancreatic, liver, lung, 

esophageal, stomach, brain. multiple myeloma, and ovarian. Yet, these eight 

cancers have historically also received the least amount of federal funding. As indicated 

earlier, the 5-year mortality rate for myeloma is still only about 35%. 

The IMF is highly supportive of long-term initiatives of the NCI such as the Cancer 

Genome Atlas and associated projects; however, these initiatives are highly unlikely to 

have any impact whatsoever on the care or survival of people already diagnosed with 

myeloma, or even those diagnosed with myeloma in the next 5 years, many of whom will 

die long before such projects bear fruit. In addition to such long-term initiatives that will 

benefit all those at risk for cancer in the long term, the NCI needs to emphaSize the 

critical importance of focused research monies that support "out-of-the-box" innovative 

research into the causes and progression of myeloma and the other deadly cancers. It 

is widely understood that research on one form of cancer may yield basic or applied 

knowledge related to other cancers, and this has been particularly true for the 

application of blood cancer research to other cancers. Treatments that were initially 

developed for blood cancers are now used in treatment of solid tumors, and the basic 

research on blood cancers fuels basic research advances in other cancers, as well. 

The IMF is fully supportive of the proposals laid out by the Deadly Cancers Group for the 

establishment of a "Targeted Cancers" program within NCI for cancers with 5-year 

survival rates of less 50%, This initiative would require a comprehensive plan of the 

research activities necessary to increase survival well above the 50% survival threshold; 

be authorized at a level commensurate to these cancers' mortality and public health 

impact; conduct research activities through its own grant making authority and through 

leveraging the grant making authority of other NCl divisions and programs. 

Environmental Risks Associated with Myeloma 

Once considered a "rare disease of the elderly," myeloma is increasingly being 

diagnosed in patients under 45 years old. Data from the IMF's Bank On A Cure® 

initiative has already identified several changes in DNA sequences called SNPs (single 

12650 Riverside Drive Suite 206, North Hollywood, CA 91607 

800-452-CURE (2873) 

818-487-7455 telephone 818-487-7454 fax 

vvww.myeloma.org 
6 


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IMF 

Written Testimony oftlle Internationa! Myeloma Foundation 

regarding 

NCI Cancer Research; Today's Progress; Tomorrow's Challenges 

Subcommittee on Health 

Energy and Comm erce Committee 

US House of Representatives 

March 23, 2010 

nucleotide polyrnorphisms) that are associated with the risk of bone disease in myeloma. 

Further analysis has shown that many of these DNA changes may be involved with the 

way the human body responds to certain environmental toxins, providing a possible link 

bel\veen myeloma and the environment. 

The findings may help explain a widely reported study published last year in the Journal 

of Occupational and Environmental Medicine, that found more cases of myeloma among 

younger responders to the 9/11 World Trade Center site than would normally be 

expected. The findings are also supportive of a study published in 2009 that suggests a 

link between exposure to certain pesticides among agricultural workers and a precursor 

to multiple myeloma. Previous studies have also shown an increased risk for myeloma 

among firefighters. 

The federal government has already recognized its responsibility to military personnel 

whose health may be endangered simply through their service to the country; the 

Institute of Medicine conducts periodic evaluations of the health consequences of 

environmental exposures and the Department of Veterans Affairs provides benefits to 

those diagnosed with blood cancers caused by or associated with environmental toxins. 

These toxins include Agent Orange for Vietnam veterans, ionizing radiation for atomic 

veterans, and benzene for Gulf War veterans. 

Grant Funding for Young Investigators 

For the past 15 years, the IMF's research initiatives have been highly focused on funding 

promising young researchers and clinical investigators from around the world in an effort 

to improve outcomes for myeloma patients, Giving young researchers the belief that 

there will be long-term opportunities for them to conduct scientific and clinical research 

into myeloma is fundamental to the improvement of opportunities for myeloma 

prevention, better treatments, and longer survival. But more needs to be done to provide 

grant funding for young investigators and to stimulate careers in research into myeloma 

and other forms of deadly cancer. 

The IMF supports the development by the NCI of specialized training programs and 

education programs for early career PhD and clinician scientists that protects their time 

for research, to attract and retain a broader pool of investigators for myeloma and the 

other deadly cancers. 

Conclusion 

The IMF stands ready to work with policymakers to advance pOlicies and support 

programs that work toward prevention and a cure for myeloma. Thank you for this 

opportunity to discuss research needs at the NCI to ensure thai our nation continues to 

12650 Riverside Drive Suite 206, North Hollywood, CA 91607 

800-452-CURE (2873) 

818-487-7455 telephone 818-487-7454 fax 

wwv>i.myeloma,org 
7 


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IMF 

Written Testimony of the International Myeloma Foundation 

regarding 

NCI GancerRosearch: Today's Progress; Tomorrow's Challenges 

Subcommittee on Health 

Energy and Commerce Committee 

US House of Representatives 

March 23. 2010 

make gains in the fight against myeloma and other deadly cancers. Please do not 

hesitate to contact us should you have any questions or need more information. 

12650 Riverside Drive Suite 206, North Hollywood, CA 91607 

800-452-CURE (2873) 

818-487-7455 telephone 818-487-7454 fax 

ww·vv.myeloma.org 
8 

Mr. PALLONE. Without objection, so ordered. 

Let me—it has been very busy and crazy around here the last 

few days so I overlooked, and I do want to mention that not only 

is Lois Capps our vice chair and one of the hardest working members of this subcommittee but also she has taken a particular interest in this subject, asked that we have this hearing today, and I 

think is also developing legislation—— 

Mrs. CAPPS. Yes. 

Mr. PALLONE [continuing]. On some of this, so thank you. 

Mrs. CAPPS. Thank you, Mr. Chairman, and thank you to our 

witnesses and to all of you for being here. 

It is so appropriate in my opinion that we are holding this particular hearing just 2 days after our historic passage of comprehensive health reform legislation. After all, while our witnesses today 

will be able to highlight just how advanced and cutting-edge cancer 

research is in the United States, now passage of legislation will finally mean that more American patients can take advantage of the 

treatment and the therapies that you all have developed. 

I am eager to learn from our witnesses today about the direction 

of cancer research and how we can develop better policies in Congress that mesh with the work that you are doing. I think we know 

now that collaboration is key and it is important that legislation 

and funding are geared toward facilitating collaboration. 

We also know that research is incomplete unless we also include 

research on best practices for providing cancer care in the clinical 

setting. I am particularly interested in that phase of it, but I see 

it as a collaboration with what you are doing. While it isn’t the 

focus of today’s hearing, I urge the chairman to consider holding 

a hearing in the near future to discuss this issue of cancer care as 

well. 

With the passage of health reform, a lot of us are getting back 

to our other health care priorities that we have held long, and this 

is one of mine. I am proud to be working with Chairman Pallone 

and several members of this committee to prepare a House companion to one of Senator Ted Kennedy’s priorities, the 21st Century 

Cancer ALERT Act. This bill will put emphasis on enabling federal 

research dollars to model the trends of modern cancer research. Additionally, it will focus on patients as survivors and the concept of 

living with cancer. With the right types of investment, we can truly 

put an end to the days of cancer as a death sentence as it has been 

for so many of our loved ones. 

I look forward to continuing to work with the committee to advance this legislation, and I hope we can use what we learn today 

to perfect the language that we are developing, and with that, I 

yield back, Mr. Chairman. 

Mr. PALLONE. I think, Mr. Buyer, there is maybe 3 or 4 minutes 

left. Do you want to go now or would you rather—— 

Mr. BUYER. I will reserve. 

Mr. PALLONE. OK. Then we will go in recess until we have—oh, 

Donna, did you want to do your statement? The gentlewoman from 

the Virgin Islands, Mrs. Christensen. 


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OPENING STATEMENT OF HON. DONNA M. CHRISTENSEN, A 

REPRESENTATIVE IN CONGRESS FROM THE VIRGIN ISLANDS 

Mrs. CHRISTENSEN. Thank you, Chairman Pallone and Ranking 

Member Shimkus. Thank you for holding this hearing. 

You know, there is not one of us here whose life has not been 

touched by cancer, and in my 21 years of practice, my patients with 

cancer have given me some of the highest highs, like an elderly 

lady with early-found colon cancer who is still surviving many 

years later, and some of the lowest lows such as women with breast 

cancer who came so very late that nothing could be done, and African Americans, while we may not have the highest incidence of 

many cancers, have the shortest survival rates and the highest 

deaths of any other population group in this country and so this 

hearing is very important to me personally and as chair of the Congressional Black Caucus’s Health Brain Trust. If we look at some 

other population groups, cancer has already surpassed heart disease as a leading cause of death for Asian Americans and Pacific 

Islanders. 

There has been a lot of progress that we are very proud of in cancer research at the National Cancer Institute, and all of NIH is to 

be commended for the transdisciplinary programs, the linking of 

genomics and cancer and advances in molecular biology and technologies. I don’t know where the balance should be but I do hope 

that the role of environmental and behaviors is given adequate attention as well. 

Of great concern given the disparities that exist in African American and the projections for even greater incidence of cancer in the 

future is how are we going to be able to ensure that the new diagnostic and treatment modalities reach everyone and reduce rather 

than exacerbate the disparities that now exist. So I would like to 

know what progress is being made and including racial ethnic minorities and women adequately in clinical trials, and just as important, how many principal investigators are at minority-serving institutions, which is what would really help to increase our participation, which is very critical to reducing the longstanding disparities. 

I would like to welcome you, Dr. Barker, and the other panelists 

on the second panel. We look forward to your testimonies. Thank 

you. 

Mr. PALLONE. Thank you, Dr. Christensen. 

So we will now recess, and I think there are three votes so I 

guess maybe 45 minutes, maybe less. The subcommittee hearing is 

in recess. 

[Recess.] 

Mr. PALLONE. The subcommittee will reconvene. Now, I know 

that there were some that did not have a chance to do openings so 

we will have that now for those of you who didn’t. I think next was 

the gentlewoman from Florida, Ms. Castor. 

Ms. CASTOR. In the interest of time, I would really like to hear 

the witnesses so I will submit my opening statement for the record. 

Thank you, Mr. Chairman. 

[The information was unavailable at the time of printing.] 

Mr. PALLONE. Thank you. 

The gentleman from Texas, our ranking member. 


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Mr. BARTON. Mr. Chairman, I will put my statement in the 

record. I agree, we need to get to the witness, but thank you for 

giving me the opportunity. 

[The prepared statement of Mr. Barton follows:] 


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March 23, 2010 

STATEMENT OF THE HONORABLE JOE BARTON 

RANKING MEMBER COMMITTEE ON ENERGY AND 

COMMERCE 

SUB-COMMITTEE HEARING: 

"NCI-CANCER RESEARCH" 

Thank you, Mr. Chainnan, for having this hearing. 

Cancer has impacted most of the 100 million households in America, 

and my family has been typical. I imagine that everyone of us in this room 

today can tell about a loved one lost to cancer. That's because cancer is an 

equal-opportunity destroyer of lives. It does not discriminate according to 

ethnicity; it afflicts men, women and children, and it lays low the rich, the 

middle class, and the poor with great vigor. Cancer is the second leading 

cause of death in our country. 1.48 million Americans are diagnosed with 

cancer each year, and last year, 560,000 died of it. 

The disease even extracts a toll from the U.S. economy. The National 

Institutes of Health (NIH) estimates that, quote, "the economic cost of 

cancer in 2005 was estimated at over $200 billion." That includes $74 
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billion in direct health care costs and another $135 billion in indirect costs 

caused by lost productivity. 

I am pleased to have Kristen Fitzgerald, the wife of a longtime staffer 

for Congressman Shimkus, Ray Fitzgerald. Ray passed away from gastrointestinal cancer. GI cancer is rare and like other rare cancers, the mortality 

rate is high. It is important that we are having this hearing to spotlight those 

types of cancers that often are overlooked. Rarer forms of cancer are often 

diagnosed after the tumors have grown, and because the patient population is 

smaller, research dollars are scarcer and progress on treatments, slower. 

The NIH's National Cancer Institute (NCI) is the federal agency that 

we have charged with researching cancer. The NCI' s work has produced 

many significant advances in our knowledge and treatment of cancer. I'm a 

strong supporter of NIH because I know that good science saves lives, and 

today I hope to hear from the Cancer Institute how they are approaching 

rarer fonns of cancer. 

We're here today to learn about the research efforts and progress 

made towards curing cancer. I want to thank our witnesses today for their 
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commitment to finding a cure and to educating the public. I look forward to 

hearing their testimony. 

I yield back the balance of my time. 
14 

Mr. PALLONE. Thank you. 

Ms. Eshoo. 

Ms. ESHOO. Thank you, Mr. Chairman. I will put my full statement in the record, but let me thank you for having this hearing 

today. 

I am pleased that our subcommittee has the stamina to push forward, especially on this important hearing because cancer is the 

second leading cause of death in our country, and it is really 

amongst the most dreaded words that anyone can ever hear. Everyone in this room and beyond this room has been affected by this 

disease in some way, shape or form, either themselves, someone in 

their family, a colleague, a child, a grandparent, an aunt, an uncle, 

a neighbor. So really, so many of us, I believe, we are all like one 

diagnosis away from something. 

There are several bills, Mr. Chairman, that members have authored in the committee, and what I would urge you to do is to put 

together a compendium of these bills from both sides of the aisle 

and really see what we can move in this Congress. Sometimes bills 

don’t have to be gigantic to really have an impact, especially in a 

specific area. So—— 

Mr. PALLONE. Would the gentlewoman yield? 

Ms. ESHOO. Certainly. 

Mr. PALLONE. Let me just mention that—I mean, not that we are 

making excuses but as you know, there was so much of the legislation that many of you have introduced on both sides of the aisle 

was either in the health care reform or impacted by the health care 

reform, and I think what we are going to do during the break, you 

know, the 2 weeks, is to try to sift through all that and see what 

is still relevant and not included in the bill, you know, once we go 

through reconciliation and then get back to all of you and say, OK, 

these are the things that we need to consider now between now 

and the end of the session. 

Ms. ESHOO. That would really be wonderful, Mr. Chairman. 

Thank you. 

And I just want to add how proud I am that in my Congressional 

district that we have a National Cancer Institute-designated cancer 

center at Stanford University and the work is really extraordinary, 

so the investments in this are amongst the best we can make as 

a society. 

I look forward to hearing from the witnesses and thank all the 

people that have been advocates for years and years and years. 

Hang in there. We all need each other and we have got to get more 

done on this. Thank you. 

[The information was unavailable at the time of printing] 

Mr. PALLONE. I thank the gentlewoman. 

The gentleman from Ohio, Mr. Space. 

Mr. SPACE. Thank you, Mr. Chairman. I too waive my oral statement orally and submit it for the record. 

[The prepared statement of Mr. Space follows:] 


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ZACKSPACE 

OPENING STATEMENT - CANCER RESEARCH 

March 23,2010 

Thank you, Mr. Chairman, and thank you for holding a hearing on an issue that 

affects all of us so much. 

In fact - that's the first point I'd like to bring up. What disease other than cancer 

has had such an impact on the lives of American people? 

Who hasn't lost a loved one to the disease? 

Who hasn't felt the pain and the agony of watching someone they know suffer as 

their body rebels against them? 

Simply put, we all have stories relating to cancer, and we all know the critical 

importance of addressing the disease. 

We all have also, at some point, wished that we could cure this disease, and do 

away with it altogether. 

Fortunately, while too many stories about people with cancer end in tragedy, there 

are many good stories to tell from those who have bested the disease and gone on 

to live long and fruitful lives. 

These stories are in thanks to the researchers around the country who are blazing a 

trail toward improving our understanding of cancer, and well as finding new and 

exciting ways to treat it. As we will hear from our witnesses today, there is 

groundbreaking research going on all over the country that provides much reason 

for optimism. 

My home state of Ohio is home to two of the NCI's designated cancer centers 

Case Western University and The Ohio State University. Ohio State is in the midst 

of exciting work to expand its hospital facilities, which will allow for critical care 

to cancer patients all over the state and in my home state of Ohio. 
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Even with all of this important work, I think that we as a Congress have a 

challenge before us today. A challenge, but also an oppOliunity. 

As the federal government of the most powerful country in the world, we have the 

resources and capability to sponsor research that will make new and definite strides 

toward ridding our society of this plague. 

I support proposals to significantly increase the budget of the NCI this year. It is 

past time that we make these kinds of investments into cancer research - research 

that will directly improve the lives of aIJ those suffering from cancer, as well as 

their friends and loved ones. 

In closing, Mr. Chairman, I want to thank all the witnesses for appearing today. 

am hopeful that we can use their testimony to develop positive, bipartisan ideas to 

what is a very real problem before us. 

I thank you, and 1 yield back. 
17 

Mr. PALLONE. Thank you, and I think that concludes our opening 

statements. Did you want to say something? 

Mr. SHIMKUS. Mr. Chairman, I ask unanimous consent that Congresswoman Judy Biggert be allowed to make an introduction of a 

former staffer on the second panel, and Judy. 

Mr. PALLONE. Without objection, so ordered. 

Ms. BIGGERT. Thank you, Chairman Pallone, and Ranking Member Shimkus for giving me this opportunity to address the subcommittee. I am honored to introduce to the subcommittee today 

Kristin Fitzgerald, who not only is a constituent of mine from 

Naperville, Illinois, but she is also a valued former member of my 

staff and the staff of the Education and Workforce, now the Labor 

Committee. But it is not Kristin’s expertise as an outstanding Congressional staff member that brings her to this subcommittee 

today. Rather, it is her experience in seeking treatment and a cure 

for a rare cancer that afflicted her late husband and dear friend of 

mine and Ranking Member Shimkus, Ray Fitzgerald. Kristin kept 

me, Mr. Shimkus and the rest of us on Capitol Hill informed via 

daily e-mails as to what was happening with her husband’s treatment, and we felt like we were able to be with her every day during that difficult time. Unfortunately, just over a year ago, Ray 

passed away from gastric cancer a mere six months after he was 

diagnosed. 

After seeing firsthand the strengths and weaknesses of the cancer treatment system here in America, Kristin has used her knowledge of government to advocate for improvements in the dissemination of best practices at the NCI and other research facilities across 

the country. I believe that her ideas have the power to speed cancer research by better leveraging and coordinating our current efforts. Given the excellent work that she did in my staff, I have no 

doubt that she is up to the task. And with that, I would yield back. 

Mr. PALLONE. Thank you. Kristin is actually going to be on our 

second panel. 

Before we go to our witnesses, though, let me just mention that 

our chairman, Chairman Waxman here is here, and if he would 

like to give an opening statement, we haven’t begun with the witnesses yet. 

Mr. WAXMAN. Thank you very much, Mr. Chairman. I thank you 

for holding this hearing and giving us the opportunity to hear from 

the National Cancer Institute and other witnesses about the Institution’s cancer research efforts. 

Cancer is a complex disease. We know that genetic, environmental and other factors all contribute to an individual’s risk for 

developing cancer, so discovering cures and developing effective 

treatments are complex, difficult and expensive endeavors as well. 

We have made tremendous progress in reducing cancer deaths and 

new cancer cases due in large part to scientific advances over the 

last decade. However, cancer remains the second leading cause of 

death in this country and may soon overtake heart disease as the 

Nation’s number one killer. One and a half million people are diagnosed with cancer each year. Eleven million people are cancer survivors. Cancer is particularly devastating for members of certain 

communities. Racial and ethnic minorities experience disproportionately high rates of cancer cases and death. All these individVerDate Mar 15 2010 13:13 Dec 14, 2012 Jkt 076020 PO 00000 Frm 00024 Fmt 6633 Sfmt 6633 E:\HR\OC\A020.XXX A020

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18 

uals, their families and friends know all too well the tremendous 

physical, emotional and financial toll of this disease. 

In the past 5 years, we have made strides in combating certain 

forms of cancer such as breast and cervical cancer. Other cancers 

pose new challenges. For example, while colorectal cancer rates 

have decreased overall, the number of people under 50 with this 

type of cancer is on the rise, and eight types of cancer, those that 

we often don’t hear much about, account for half of all cancer 

deaths. I know these so-called deadly cancers are of particular interest to the chairman, Mr. Shimkus and other members of the 

subcommittee. 

Today we have an opportunity to learn more about cancer research conducted and supported by the NCI to better understand 

where we are and where we hope to go in making advances to beat 

this terrible disease. This research spans the continuum of discoveries starting at the laboratory bench, then translating scientific 

breakthroughs into clinical application and finally testing for safety 

and efficacy to determine if new innovations really work at the patient’s bedside. 

In every regard and throughout the world, NCI is considered the 

preeminent institution for biomedical research on cancer. The research funded by NCI will enable us to discover and ultimately deliver the best possible prevention, detection, diagnosis and treatment tools to all Americans. As we will hear, there is much to be 

excited about and, of course, there remains much work to do. I 

want to thank Dr. Barker and all of our witnesses for being here 

today, and I look forward to their testimony. Thank you. 

[The prepared statement of Mr. Waxman follows:] 


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Opening Statement of Rep. Henry A. Waxman 

Chairman, Committee on Energy and Commerce 

Hearing on "NCI Cancer Research: 

Today's Progress; Tomorrow's Challenges" 

March 23, 2010 

Thank you, Chairman Pallone, for holding this hearing 

today and giving us the opportunity to hear from the 

National Cancer Institute and our other witnesses about the 

Institute's cancer research efforts. 

Cancer is a complex disease. We know that genetic, 

environmental, and other factors all contribute to an 

individual's risk for developing cancer. So discovering 

cures and developing effective treatments are complex, 

difficuh, and expensive endeavors as well. 

1 
20 


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We have made tremendous progress in reducing 

cancer deaths and new cancer cases, due in large part to 

scientific advances over the last decade. However, cancer 

remains the second leading cause of death in this country 

and may soon overtake heart disease as the nation's number 

one killer. One and a half million people are diagnosed 

with cancer each year. Eleven million people are cancer 

survIvors. 

Cancer is particularly devastating for members of 

certain communities. Racial and ethnic minorities 

experience disproportionately high rates of cancer cases 

and death. 

All these individuals, their families, and friends know 

all too well the tremendous physical, emotional, and 

financial toll of this disease. 

2 
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In the past five years, we've made great strides in 

combating certain forms of cancer, such as breast and 

cervical cancer. Other cancers pose new challenges. For 

example, while colorectal cancer rates have decreased 

overall, the number of people under 50 with this type of 

cancer is on the rise. And eight types of cancer -- those 

that we often don't hear much about -- account for half of 

all cancer deaths. I know these so-called deadly cancers 

are of particular interest to Chairman Pallone, Mr. 

Shimkus, and other Members of the Subcommittee. 

3 
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Today, we have an opportunity to learn more about 

cancer research conducted and supported by NCI - to better 

understand where we are, and where we hope to go in 

making advances to beat this terrible disease. This research 

spans the continuum of discoveries -- starting at the 

laboratory bench, then translating scientific breakthroughs 

into clinical application, and finally testing for safety and 

efficacy to determine if new innovations really work at the 

patients' bedside. In every regard and throughout the 

world, NCI is considered the preeminent institution for 

biomedical research on cancer. 

The research funded by NCI will enable us to discover 

and ultimately deliver the best possible prevention, 

detection, diagnosis, and treatment tools to all Americans. 

As we will hear, there is much to be excited about. And of 

course, there remains much work to do. 

I want to thank Dr. Barker and all of our witnesses for 

being here today. I look forward to their testimony. 

4 
23 

Mr. PALLONE. Thank you, Chairman Waxman. 

Mr. Green, would you like to make an opening? 

Mr. GREEN. Thank you, Mr. Chairman, and I would like to have 

my full statement in the record, but I can’t get away without—I 

represent a district in Houston and we have there great NCI facilities at the University of Texas, Health Science Center, Baylor College of Medicine and of course the University of Texas M.D. Anderson Cancer Center. I thank NCI for what you are doing because I 

see it every day when I am home, so thank you. 

I ask unanimous consent to place my full statement in the 

record. 

[The prepared statement of Mr. Green follows:] 


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Statement of Congressman Gene Green 

Committee on Energy and Commerce 

Subcommittee on Health 

National Cancer Institute Cancer Research: Today's Progress; Tomorrow's Challenges 

March 23,2010 

Thank you for holding this hearing today on research at the National 

Cancer Institute and their designated research centers. 

Today we will be talking about ongoing research at the NCl and its 

research centers. We will also be discussing rare cancers and the 

challenges associated with diagnosing and treating these deadly cancers. 

Texas has three NCI designated cancer research centers- UT Health 

Science Center, Baylor College of Medicine, and the University of 

Texas' MD Anderson Cancer Center. 

MD Anderson Cancer Center is frequently recognized as the top cancer 

center in the country. It is one of the original three Comprehensive 

Cancer Centers designated by the National Cancer Act of 1971, and it 

remains the only one in Texas. 

Baylor College of Medicine in Houston coordinates more than $100 

million in total cancer-related research support, including nearly $40 

million from the National Cancer Institute. 

UT Health Science Center in San Antonio is known for its Cancer 

Therapy & Research Center Institute for Drug Development as largest 

integrated drug development programs in the world. Patients from 

around the world participate in Phase I, II and III clinical studies. 

Over the years our office has helped many individuals access cancer 

treatment at these wonderful institutions. 
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Most recently, one of our constituents was diagnosed with Stage 4 

melanoma. Like many individuals who are diagnose with late stage 

cancer-- treatment is particularly difficult. 

After a long battle with his insurance company, he was able to access an 

experimental treatment program at MD Anderson. After long months of 

difficult treatment, the therapy seemed to be successful. 

Unfortunately, his condition worsened and our office worked with MD 

Anderson to get him into an additional clinical trial. I am sad to say that 

my constituent, who was a community figure with a wife and small 

children, passed away before we could get him into the new clinical 

trial. 

When facing any type of cancer, especially the rare and potentially fatal 

type, knowing that you can access all types of treatment- even 

experimental is especially important. Patients want to explore every 

option available to them and that is why the research conducted at the 

NCI and its research centers is so important. 

We continue to diagnose many individuals with deadly cancers like 

pancreatic, lung, brain, liver, and ovarian in the very late stages, which 

makes them even more difficult to treat. It is important we begin to 

explore how to make these diagnoses earlier so individuals have 

increased survival rates. 

With continued funding and research the diagnostic tools and treatments 

we consider experimental could one day be routine. 

NCI research performed in Texas - and other impressive research 

facilities across the nation will yield continued contributions to our 

understanding of various forms of cancer and the development of 

effective treatments to improve the health and well-being of all 

Americans. 
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I want to thank out witnesses for appearing before the committee today 

and thank you Mr. Chairman for holding this hearing. 

I yield back my time. 
27 

Mr. PALLONE. Without objection, so ordered. And all statements 

by members in full will be put in the record if they so desire. 

Let me move to our first panel and only witness, who is Dr. Anna 

D. Barker, deputy director of the National Cancer Institute. Welcome, and thank you for being here today. I am not going to go 

through the drill. You know how we operate, so I recognize you for 

an opening statement. 

STATEMENT OF ANNA D. BARKER, PH.D., DEPUTY DIRECTOR, 

NATIONAL CANCER INSTITUTE 

Ms. BARKER. Thank you very much, Mr. Chairman and members 

of the subcommittee. It is my great pleasure to be here, and I want 

to thank you for the opportunity to testify today and for holding 

this hearing. The timing couldn’t be more appropriate, I think. 

I am the deputy director of the National Cancer Institute. I also 

have the singular pleasure actually of directing the strategic 

science initiatives for the Cancer Institute, and I will be talking 

about a couple of those today that I think you will find of some interest. It is really an exciting time in cancer research, as you heard 

earlier today. I would like to highlight a few of the advances in my 

testimony today and we can discuss them later, but I think we 

have unprecedented opportunities now to really increase progress 

against this disease that still tragically affects almost all of us in 

one way or another. 

So an unprecedented convergence of advances in molecular biology and advanced technologies is beginning to transform our understanding of the mechanisms of cancer, and that is incredibly important. Cancer is a disease of changes in our genes, so if I don’t 

deliver any other message today, maybe we can take that away. 

Some of these genes are inherited. You can’t choose your parents. 

But some of them, actually most of them are actually acquired as 

a consequence of the way we lives our lives. As you will hear, we 

are systematically identifying these genomic changes in cancer, 

which is allowing us to finally identify the molecular basis of subclasses of cancer and develop targeted interventions. That is quite 

a step. 

These discoveries really represent a paradigm shift. It will take 

time but knowing the molecular makeup of a cancer will allow a 

patient to be treated according to their tumor’s signature. As the 

cost of sequencing the human genome continues to fall, and it is 

falling almost daily, and bioinformatics facilitates the availability 

of an electronic medical record that captures all of a patient’s data 

in this next decade, and beyond, we are going to transform the way 

cancer is diagnosed, treated and prevented. 

NCI is leading this revolution through programs that range from 

studies that define specific changes in the genomes of cancer patients to nanotechnology-based diagnostics that can detect miniscule amounts of cancer in a patient. I would like to tell you a little bit about a few of these programs in this limited time that I 

have, but I think the programs I am going to tell you about will 

change our definition of cancer, allow us to better understand why 

certain cancers have poor outcomes and provide new approaches to 

control all cancers. 


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28 

Led by Dr. Francis Collins and myself in 2005, the NCI and the 

NHGRI, which is the National Human Genome Research Institute, 

launched a groundbreaking collaboration called The Cancer Genome Atlas—I will refer to it from now as TCGA, which you should 

remember—to ultimately identify and catalog all of the relevant 

genomic changes in most types of cancer. This is an enormous undertaking. The Cancer Genome Atlas is one of those paradigmshifting programs that I mentioned earlier. It employs state-of-theart genomic characterization and sequencing technologies, engages 

a network of multidisciplinary centers, which is actually composed 

of tens of institutions and hundreds of experts in genomics in cancer biology and deposits all of the data in a public database. TCGA 

is the largest and most comprehensive analysis of the molecular 

basis of cancer ever undertaken and the project has already faced 

and overcome a large number of technical and scientific challenges 

in the first three years. 

We launched TCGA with a study of glioblastoma, which is the 

most prevalent human brain tumor in adults, and ovarian cancer 

and squamous cell cancer of the lung had followed soon thereafter. 

In 2008, the first major results of the TCGA pilot program produced a map of these three key pathways that are disrupted in 

GBM and defined the four specific molecular subtypes of this cancer, paving the way for identifying the right patient for the right 

drug. The availability of a highest-quality, multidimensional data 

set on a statistically valid set of high-quality samples is bringing 

new investigators and teams forward to study GBM in large numbers. This is one of the goals of TCGA, so we are bringing tons of 

people to the table to study this cancer. 

We are about to do the same thing for high-grade serous ovarian 

cancer, which is responsible for most ovarian cancer and a major 

contributor to the overall 5-year survival rate of only 31 percent. 

These data are being finalized for publication. I haven’t talked to 

anybody about the data but I am going to tell you a few things that 

no one else will know. First, at one level, ovarian cancer looks quite 

different from GBM at the genomic level but in another way it 

looks quite similar. Ovarian cancer is a study of genome instability 

with a highly disrupted genome. It is a disease of copy number 

change, which actually means a disrupted genome. This instability 

is likely driven by nearly wholesale changes in only three genes. 

There are three distinct molecular subtypes of ovarian cancer confirmed at multiple levels of the genome. The distinct pathways disrupted in ovarian cancer where there are signatures can predict 

survival duration. That is quite a finding. Overall, the tumor is 

driven by defects in genes that are responsible for repairing damaged DNA, and there are a number of other rare genes that may 

represent new targets for drug development. The data is going to 

open a whole range of new windows of exploration for diagnosis 

and treatment of ovarian cancer that I predict will change the future for ovarian cancer patients hopefully on an accelerated schedule. 

The value of TCGA will ultimately be measured in many advances but perhaps one of the most striking is the value of the integration and analysis of multidimensional data about the many cancers it will study. Using ARRA funds, we are currently expanding 


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29 

the scope of TCGA to explore 20 additional cancers over the next 

5 years, 10 in the next 2 years. Many of these studies will include 

the rare and deadly cancers that you will all be asking me about 

later. 

One additional comment I would like to make is that one of the 

rate-limiting steps in TCGA has been the availability of rare cancer 

samples and very high-quality cancer samples, and we are hoping 

to work with the advocacy community to fix that problem. Achieving high-quality biospecimens for the country is a major challenge. 

This is the foundation for personalized cancer medicine. It is also 

a problem in terms of the way standard of care is practiced in this 

country and it is also a problem with most of the tumor samples 

in the country where only about 30 percent of those samples are 

available and can be used in a high-quality study like TCGA. NCI 

has launched something called the cancer Human Biobank to deal 

with that issue, so we are going to have a national biobank. 

The other program that I wanted to highlight just for the last 

minute is basically what we are doing in nanotechnology. Nanotechnology, as you know, is allowing us to measure things at levels 

we only dreamed about a few years at 1 to 100 nanometers, roughly the size of a water molecule to the size of a bacterium. Recently, 

we have been able to show with nanotechnology that you can measure in this particular case cancer changes roughly six times more 

sensitively than some of the diagnostic tests that we are using 

today. Northwestern University reported just 2 weeks ago using a 

barcode assay in DNA, they can actually detect prostate cancer at 

a level that is unheard of, and we believe going forward we will be 

able to use that kind of information to detect these cancers much, 

much earlier. 

Another recent breakthrough that was only published yesterday 

from the Nanotechnology Alliance for Cancer, which was started by 

NCI 5 years ago, we are now able to know that there is a piece 

of the genome actually called RNAI that actually blocks the expression of certain genes. We haven’t been able to deliver that. It gets 

actually degraded in the body. We had a nanotechnology investigator from California, actually from Cal Tech, report just yesterday 

that they have been able to deliver this to patients and it is going 

into phase II trials. Again, this is another breakthrough from the 

nanotechnology program that NCI started about 5 years ago. 

At NCI, we are really proud of the progress we are making in 

these advanced technology programs. We are excited by the opportunities that lie ahead and challenged by the daunting amount of 

work that we have yet to do. We are dedicated to achieving a future where the shadow of cancer is removed from our lives and 

those of our children and our grandchildren. This increasingly 

seems to me like an achievable mission and a vision for every 

American that we can achieve hopefully in this next decade. NCI 

and NIH and are committed to moving us all forward toward this 

future, and I have never been more excited about believing that we 

will achieve this future for every patient, for every family and everyone touched by cancer, including my own. I have lost all of my 

family to cancer. I have been in this field for my entire career. I 

lost my grandmother to pancreatic cancer, two additional relatives 


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to pancreatic cancer, my mother to breast cancer, my sister to 

breast cancer and my father to lung cancer. 

Thank you. 

[The prepared statement of Dr. Barker follows:] 


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Testimony 

Before the 

Subcommittee on Health 

Committee on Energy and Commerce 

United States House of Representatives 

National Cancer Institute Research: Today's Progress; 

Tomorrow's Challenges 

Statement of 

Anna D. Barker, Ph.D. 

Deputy Director 

National Cancer Institute 

National Institutes of Health 

U.S. Department of Health and Human Services 

For Release on Delivery 

Expected at 2:00 p.m. 

Tuesday, March 23, 2010 
32 


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Good afternoon, Chairman Pallone and members of the Subcommittee. Thank you for the 

opportunity to testifY this afternoon. I am Dr. Anna Barker, Deputy Director of the National 

Cancer Institute within the National Institutes of Health (NIH), an agency of the Department of 

Health and Human Services. I also serve as the NCI Deputy Director for Strategic Scientific 

Initiatives, a program focusing on trans-disciplinary programs in strategic areas of cancer 

research and advanced technologies including programs such as: the Nanotechnology Alliance 

for Cancer; The Cancer Genome Atlas (TCGA); the Clinical Proteomics Technologies Initiative 

for Cancer and Physical Sciences-Oncology Centers. It is my privilege to appear before you 

today to share some exciting new advances in our understanding of a disease that is tragicalIy 

familiar to each of us. 

Unfortunately nearly everyone has a personal story to telI of the toll taken by cancer. One out of 

every three women and one out of every two men in America will develop cancer over their 

lifetime. In addition to the enormous physical and emotional toll it takes, cancer represents a 

huge economic burden to the U.S., amounting to over $200 billion in total healthcare costs in 

2004. 1 NCI's key challenge is to understand the changes in the genome and associated biology 

that ultimately cause cancer in order to enable the development of more effective diagnostics, 

therapies, and prevention strategies that can be delivered to cancer patients. 

Cancer is an extraordinarily complex disease of uncontrolled cellular growth, proliferation, and 

spread beyond the original tumor. Cancer is also an integrated network of signaling pathways 

1 Smith SO, Smith, E, Hurria, A, et al; Future of Cancer Incidence in the United States; Burdens Upon an Aging, 

Changing Nation. J Clin Oncol published ahead of print on April 29, 2009 at 

http:Uico.ascopubs.org!cgilcontent/abstract/JCO.2008.20.8983vi 

National Cancer Institute Research: Today's Progress; Tomorrow's Challenges 

House Energy and Commerce Subcommittee on Health 

March 23, 2010 

Pagel 
33 


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and chemical interactions between the cancer and its human host. In fact, cancer is a large 

number of different diseases - over 200 - and many of these cancers may be comprised of a 

number of different subtypes, depending on which pathways are altered by changes in the cancer 

genome. These changes result in uncontrolled growth, and once cancer spreads (metastasizes), it 

is extremely difficult to control. 

In the 1980s, new tools in molecular biology led to the discovery that cancer is a disease of 

genetic alterations - some are inherited and others after birth due primarily to environmental 

exposures. Clearly the complex of environmental exposures that impact all of us plays a major 

role in who gets cancer and the mechanism by which it occurs. For example, environmental 

factors such as radiation can cause changes in genes (mutations) that increase cancer risk - and 

these changes, called somatic mutations, are responsible for most cancers. Some people inherit 

genes that may predispose them to cancer - but many people who inherit these genes never 

progress to cancer. Therefore, it is critical to systematically explore how environmental factors 

combine with genetic variants to produce cancer. For example, in one such study, researchers 

are assessing breast cancer risk during puberty following specific environmental exposures. 

The current convergence of advances in molecular biology and advanced technologies is already 

beginning to transform our understanding of the mechanisms by which cancer arises in humans. 

Increasingly, knowledge of the genomic changes in selected cancers is beginning to allow 

oncologists to categorize cancers based on technologies that defme these alterations These 

molecularly-based subclasses of cancers are beginning to support the development of more 

National Cancer Institute Research: Today's Progress; Tomorrow's Challenges 

House Energy and Commerce Subcommittee on Health 

March 23, 2010 

PageZ 
34 


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specific diagnostics through specific disease biomarkers - and drive the discovery of new cancer 

drug targets. 

Ultimately, knowledge that is deriving from 21 st century biomedical and cancer research will 

allow us to move from a "one size fits all" approach to disease - from a chemotherapeutic 

approach to cancer, for example - to safer and more effective interventions tailored to each 

individual's genetic makeup. NCI has developed a number of programs that are aimed at 

harnessing the power of molecularly-based interventions for cancer - ranging from studies that 

define specific changes in the genomes of cancer patients to nanotechnology-based diagnostics. 

As the cost of sequencing the human genome continues to fall and bioinformatics facilitates the 

availability of an electronic medical record that captures all of a patient's data, the next 10 to 20 

years promises to transform the way cancer is diagnosed, treated, and prevented. The following 

is a brief sampling of recent advances in cancer research that highlights and supports the promise 

of personalized cancer medicine. 

Recent Advances in Understanding the Genetics of Cancer 

Well before the $1000 genome becomes a reality, advances from genome research are already 

leading to important new understanding of the role of genetic changes in a number of common 

cancers. For example, use of a technology called Genome-Wide Association Studies (GWAS) 

scans the genomes of many individuals to identify markers that may predict whether or not an 

individual may be susceptible to the development of a specific cancer. These studies have 

already shown that, generally, multiple genetic changes are required for an individual to be 

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predisposed to developing cancer. GWAS also is proving invaluable in identifying genetic 

changes that are predictive for how an individual may metabolize a specific drug. This is 

proving especially valuable in reducing side effects in patients. 

The Cancer Genome Atlas 

In 2005, the National Cancer Institute and the National Human Genome Research Institute 

launched a groundbrealcing collaboration called The Cancer Genome Atlas (TCGA) to ultimately 

identify and catalogue all of the relevant genomic alterations in most types of cancer. TCGA 

employs state of the art genomic characterization and sequencing technologies, engages a 

network of multidisciplinary centers that involve over 200 experts in genomics and cancer 

biology and all of the data is placed in a public database. TCGA is the largest and most 

comprehensive analysis of the molecular basis of cancer ever undertaken which has faced and 

overcome a large number of technical and scientific challenges in its three year pilot period. In 

addition to achieving the goal of establishing the network and supportive structure for this firstever large-scale, high-throughput cancer genomic program - TCGA has already produced 

scientific advances in the most common fonn of adult brain cancer (glioblastoma multifonne, or 

GBM), ovarian and lung cancers. 

Because of TCGA's enonnous potential, NIH chose it as one of seven "signature projects" to 

receive special emphasis using American Recovery and Reinvestment Act of 2009 (ARRA) 

funds. Utilizing the latest technologies and molecular insights, TCGA is expanding its scope, to 

explore approximately 20 additional tumors over the next 5 years - lOin the two years of the 

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ARRA funding period. Results from TCGA will for the first time map the complex pathways 

involved in specific cancers which will re-define cancer targets and provide a rational basis for 

the development of new targeted diagnostics and therapeutics. 

In 2008, the first major results of the TCGA pilot program produced a map of the three key 

pathways that are disrupted in glioblastoma - and defined the four specific molecular subtypes -

paving the way for identifying the right patient for the right drug. Another exciting and 

unexpected fmding points to a potential mechanism of resistance to a common chemotherapy 

drug used for brain cancer - which will influence clinical practice almost immediately. 

Although quite different from GBM, the TCGA team has discovered a large number of large 

scale genomic alterations called copy number changes in the DNA of patients with ovarian 

cancer. The value of TCGA will ultimately be measured in many advances - but perhaps one of 

the most striking is the value of the integration and analysis of multidimensional data to fully 

understand all of the genomic changes that impact the biology of specific cancers. We have seen 

unprecedented new investigations and interdisciplinary collaboration as these data become 

accessible to so many scientists. NCI has made a major commitment of ARRA funds to collect 

the tissues for TCGA sequence the genes and integrate and analyze the vast amount of data 

coming from the project. The rate-limiting step in TCGA is high quality samples; and NCI is 

working with survivors and advocates to ensure that cancers such as pancreas, melanoma, gastric 

cancer and other rare tumors are able to enter TCGA as soon as possible. 

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The Next Generation of Cancer Treatments 

Prior to the emergence of the molecular diagnostic field, cancers were categorized solely by 

pathology, i.e., according to their appearance under a microscope. However, programs such as 

TCGA are changing this picture by producing molecular "maps" that allow scientists to 

determine how genes and proteins are interacting in a cell. This approach focuses upon patterns 

- gene and protein activity patterns - in different types of cancerous or precancerous cells. 

Molecular diagnostics uncovers these sets of changes and captures this information as expression 

patterns of the proteins that are coded for by the changes in DNA. Also called "molecular 

signatures," these expression patterns are improving clinicians' ability to diagnose cancer more 

specifically. This new approach extends to treatment as well as diagnosis, with the recent 

emergence of therapies specifically designed to hit a specific target defied by a molecular 

signature. 

Targeted cancer therapies are drugs or other substances that block the growth and spread of 

cancer by interfering with specific molecules involved in tumor growth and progression. By 

focusing on molecular and cellular changes that are specific to cancer, targeted cancer therapies 

promise to be more effective than other types of treatment, including chemotherapy and 

radiation, and less harmful to normal cells. Molecularly-targeted cancer treatments are expected 

to yield the next generation of therapies that complement, or even replace, today,s standard 

therapies for most cancers. The classic example is Gleevec, a small molecule drug that targets a 

class of overactive proteins formed due to a genetic change related to uncontrolled tumor growth. 

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NCr-funded research on Gleevec has shown it to be highly effective for treatment of chronic 

myelogenous leukemia and gastrointestinal stromal tumors. 

A Coordinated Cancer Platform 

NCI is initiating a number of key programs that will link the genomics of cancer with new ways 

to diagnose and treat the disease. The first step will be to ensure the availability of high-quality 

human biospecimens for cancer research, accomplished through the cancer human biobauk 

(caHUB). TCGA and other high-tech initiatives demand the highest quality tissue, blood, and 

tumor samples, all rigorously and ethically collected, properly stored, and extensively annotated. 

caHUB, begun in 2009 after several years of work developing techniques and best practices, will 

be a national biobauk: a repository for difficult-to-obtain biological materials and associated data 

that can be used for medical research and the sophisticated molecular assays necessary for 

identifying biomarkers that are key to understanding the biology of specific cancers. 

Biomarkers aDd Proteomics 

One of the key challenges in oncology is determining when and why cancer may recur in treated 

patients. Today, biomarkers, which are parameters that can be measured that reflect the biology 

of the disease, or in some cases the therapeutic effects of treatments, represent one of our best 

strategies to address these hurdles. These "biomarkers" of cancer can be measured through a 

variety of means, including dynamic imaging tests and laboratory tests on blood, tissue, and 

other biologic samples. The measurement of these parameters reflects changes in genes, 

proteins, or some combination that tell a story of the state of the cancer in a patient. The NCI is 

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actively investigating biomarkers through TCGA, an initiative in clinical proteomics, and 

working with other sectors through several partnerships. One such unique collaboration is the 

Biomarkers Consortium, a public-private partnership organized and managed by the Foundation 

for the NIH (FNIH) with funding provided by philanthropic groups and industry. The 

Consortium is currently investigating fluorodexyglucose-positron emission tomography (FDGPET) as a potential biomarker in clinical trials for lung cancer and lymphoma. These studies 

could have significant impact on patient management by validating a tool that can identify 

response to treatment and facilitate drug development. 

A related area of examination involves determining what proteins are expressed in patients as a 

result of the various genetic changes identified by large-scale genornics programs such as TCGA. 

NCI has undertaken a program called the Clinical Proteomics Technology Initiative to develop 

needed standards for the discovery of proteins, so they can serve as biomarkers. There are 

clearly large numbers of proteins that are important in the development and maintenance of 

cancer, but the technologies, reagents and assays to measure them must be standardized and 

transferable across laboratories to maximize benefit to patients. Proteins are likely our best hope 

to identify cancer early from small amounts of blood - meaning they offer great hope to improve 

screening assays for a number of cancers. NCI is assembling new transdisciplinary teams to 

harvest the promise of biomarkers for both cancer diagnosis and new targeted drug development 

through a number of strategies - one of the most promising and exciting at the intersection of 

molecular biology and advanced technology is the area of nanotechnology for cancer. Cancer's 

complexity and the numbers of biomarkers that will be needed to diagnose and treat cancer will 

require new experimental platforms that can capture and simultaneously analyze large numbers 

of different parameters that define the state of the cancer. 

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Nanotechnology 

Because of their small size, nanoscale devices can readily interact with biomolecules on both the 

surface and inside cells, giving them the capability to detect disease and deliver treatment in 

ways unimagined before now. Nanotechnology has the potential to enable all areas of cancer 

research including improving molecular imaging, early detection, cancer prevention, and 

treatment. In 2004, the National Cancer Institute established the NCI Alliance for 

Nanotechnology in Cancer program. The network is built on a strong foundation of superb 

science and technology deVelopment and is designed to accelerate the application of 

nanotechnology to remove some the m,yor barriers in clinical oncology and basic cancer 

research. 

One example of an exciting advance in nanotechnology involves the use of gold nanoparticles 

coded with "barcode" DNA to allow ultrasensitive detection of cancer biomarkers - six times 

greater than conventional assays. The technology has already been translated to bedside and has 

demonstrated preliminary success in clinical evaluations of the prostate cancer samples of 

approximately 400 patients, with a larger study currently being planned. Other advances that 

have derived from this multi-disciplinary program include a nanotechnology platfonn to detect 

multiple biomarkers of brain cancer and delivery of nanoparticles that contain a promising new 

cancer therapy called small interfering RNA (siRNA) directly to cancer cells. This type of drug 

delivery has already shown promising results in a small Phase I study of pancreatic cancer, a 

highly lethal type of cancer for which there are few treatment successes. The Alliance has 

placed significant emphasis on translating nanotechnology to patients, and as a result over 50 

companies have been formed from these efforts over the past 5 years. 

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A New Framework of Physical Sciences 

Understanding cancer at a fundamental level depends on more than just defining the genomic 

changes that characterize these diseases, as cancer occurs in space over time. To build a new 

framework for the advances and data that are deriving at a dizzying pace from present day cancer 

research, the NCI has created an unprecedented scientific network called the Physical SciencesOncology Centers (PS-OCs) program. These centers bring together teams of experts in the 

physical sciences with cancer biologists and oncologists to work together in innovative ways to 

understand how the physical forces in cells and organs impact on the expression of genomic 

changes that drive cancer biology. The fields of physics, mathematics, chemistry, engineering 

and biology are all working together to ask questions such as: How does evolution affect the 

way we look at cancer? How is information transferred and translated in cancer cells? What 

impact do the physical laws that govern the universe have across the scales ranging from 

molecules and atoms to whole organisms? It is anticipated that the PS-OCs will generate new 

bodies of knowledge that define the physical, chemical, and engineering processes that operate in 

cancer. Some examples of work already in progress at the POCs include examination of the 

physical dynamics of cancer progression, selective metastasis and specific mechanical properties 

of cancer cells. Studying cancer cells and tumors in terms of their physical properties will open 

new areas of research to drive the development of better diagnostic and treatment strategies for 

cancer (e.g., physical properties as potential cancer signatures). 

We at NCI are proud of the progress we have made, excited by the opportunities that lie ahead, 

and challenged by the daunting amount of work that must yet be done. We are dedicated to 

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achieving a future where the shadow of cancer is removed from onr lives and those of onr 

children and grandchildren. TIris increasingly seems like an achievable futnre - as thousands of 

dedicated intramural and extramnral scientists work tirelessly to make personalized cancer 

medicine a reality for every American. 

Thank you, Mr. Chairman. I would be pleased to answer any questions that the Committee 

might have. 

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Mr. PALLONE. Thank you, Dr. Barker. We are going to take questions from the panel beginning with me, and I have to say that as 

much as I am tired and I know many of us are from the long weekend, I am also very excited by your testimony. You mentioned 

nanotechnology, which I think is so important for the future. We 

just had a—well, not just but within the year or so I went to an 

all-day conference at Rutgers on nanotechnology and some of the 

things that were really interesting that they were doing, and when 

you were talking about your relatives, I feel the same way, you 

know, my mother with pancreatic cancer, father-in-law with brain 

cancer. These deadly cancers are just around us, you know. 

When I went to Johns Hopkins with my mom, I was surprised 

to find out how much of a hereditary factor there was. Now, I don’t 

think it was that significant. I think they said 10 or 11 percent 

that they could say were hereditary, which isn’t really that much, 

but the first day we arrived and they were looking at my mom, 

they took myself and my brother into a separate room and said by 

the way, there is a certain hereditary factor here, and they put us 

in some kind of a genetic bank or something to get back to us if 

there are breakthroughs because of the genetic factor, and I was 

of course—the first question they asked, Do you have a history of 

this in your family, and my mother actually was adopted so we had 

absolutely no information to relate to them other than her. But I 

know that all these things you mentioned are so important, really. 

I can’t stress that enough. 

I mentioned in the opening about this genome atlas that I guess 

examines genetic material of various tumors and looks into gene 

mutations and different things genetically, and I know that you 

have some new findings in that respect with regard to ovarian cancer, but this atlas I guess from what I understand gets updated 

and may be expanded, I suppose, to include some of these other 

deadly cancers, so I wanted to ask you, you said it is going to be 

expanded to include 20 additional forms of cancer over the next 5 

years and NCI will look at half of these within the next 2 years. 

So tell me, what is the basis for selecting the 20 additional cancers 

that you research and what is the timeline for that selection? How 

is that process gone about? 

Ms. BARKER. It is an excellent question. It turns out that as I 

said, the biggest issue we have with doing these very sophisticated 

studies is the availability of the samples. So as you can imagine, 

we are looking everywhere for samples, and to give you some feel 

for how difficult this was for GBM, glioblastoma, which is this 

adult brain tumor we did first, we had to look everywhere in the 

United States and outside the United States to get enough samples 

to actually do that study. It turns out that only about 30 percent 

of those samples qualify, and that is not a bad number for most 

of the samples in the country right now. Why is that? Well, people 

collected these samples for many years. They weren’t thinking a lot 

about when they collected the samples the advanced technologies 

we would be using today. So in terms of selecting cancers for the 

atlas, the first priority is, are the samples there, are they available, 

and so we are doing very well. We are moving ahead on lung cancer. We are just introducing kidney cancer into The Cancer Genome Atlas, already have actually. Colon cancer is already being 


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introduced into the atlas. We have cervical cancer and potentially 

gastric cancer to come into the atlas. Now, the latter one obviously 

is a rare tumor and so we are having to look in many, many areas 

to find these samples. By and large, or at least as we move forward, we think that we will also be able to prospectively collect 

samples and that will be driven mostly by which tumors are prevalent and which ones we can actually lay our hands on in the most 

rapid fashion, but we can collect tumors prospectively now and actually make sure that they meet our very stringent criteria. So 

going forward, we are going to use a combination of retrospective 

samples and prospective samples. 

But it turns out to be the biggest barrier we have. I mean, who 

would have guessed going into this that this—what most scientists 

would have considered a trivial issue is non-trivial and it is going 

to be actually the basis for personalized medicine overall so as a 

country we have to face up to the fact that we have to start treating our patient samples as well as we treat our patients, if not better, because they are going to actually define what personalized 

cancer medicine can be. So at NCI we started a lot of initiatives 

including best practices for how you collect samples and store them 

and actually what the stewardship is going to be of those samples. 

We think it is critically important. 

Mr. PALLONE. I would just ask you, I know that I and several 

members, maybe all the members of the committee, are interested 

in tracking any progress that you make on this cancer genome 

atlas, and keep us abreast of your efforts as you go along because 

this is of interest to me and to all of us. We appreciate it. 

The gentleman from Illinois, Mr. Shimkus. 

Mr. SHIMKUS. Thank you, Mr. Chairman. 

Dr. Barker, welcome and we are glad to have you here. The 

budget request this year is $5.26 billion. It is a $163 million increase over fiscal year 2010. Add to that $1.26 billion provided by 

the stimulus bill. How do you go about apportioning out—we as 

members have disease groups all the time that descend upon us, 

and it is a sad kind of a food fight. So how do you apportion research dollars to cancer, the various cancers? 

Ms. BARKER. With great difficulty. 

Mr. SHIMKUS. Like we do, I think. 

Ms. BARKER. I think at the last count there were a little over 400 

survivors’ groups, I think, relative to cancer of one sort or another, 

and so what we try to do is to look at what is really promising in 

terms of what is going to enable cancer research that will actually 

be translatable to patients and backing into that what is really 

going to inform the biological space that will allow that to happen. 

I mean, if you think about what is really required for cancer, it is 

to understand the mechanisms that actually drive this disease and 

that is understanding the biology, and that has taken some time 

and we have built a really strong base for that. So what we try to 

do at NCI is obviously about half of our portfolio is driven by what 

we call individual investigator-initiated research, which is the 

strongest part of what we still do in science today. It is innovative. 

It is driven by innovation and individuals’ ideas. The rest of our 

portfolio is dedicated to things like our cancer centers, our specialized programs of research excellence, our clinical trials infrastrucVerDate Mar 15 2010 13:13 Dec 14, 2012 Jkt 076020 PO 00000 Frm 00051 Fmt 6633 Sfmt 6633 E:\HR\OC\A020.XXX A020

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ture, et cetera. So cancer is a very, very big problem in the country 

so there is a fair amount of infrastructure that we have established 

in the early 1970s, actually the early 1960s. 

The other thing we try to do is the area that I lead up, the NCI, 

through the Center of Scientific Initiatives, we try to focus on areas 

that we think are enormously promising like cancer genomics or 

nanotechnology or proteomics, the biospecimen issue, cancer 

bioinformatics. These are all things that we have tried to focus on 

over the last several years. 

Mr. SHIMKUS. If I can, because I have only got a couple more 

questions that I want to ask, but I appreciate that. But I want to 

follow up on—— 

Ms. BARKER. So balance, I guess, is my answer. 

Mr. SHIMKUS. I know, I expected not an easy answer of how you 

do it. 

In the appropriations bills of last year, we addressed both sides 

of the Hill. We encouraged NCI to put a high priority on GI cancers 

in people age 40 and under and for NCI to consider developing an 

interconnected gastrointestinal cancer biorepository. Where are we 

at on that, and can you give us any response as far as what 

progress has been made, what factors are you considering to determine if a GI repository should be developed? What else can we do? 

Obviously that is a particular focus of this hearing and my interest. 

Ms. BARKER. I think the GI spores are really well under way and 

they are collecting their samples and so we will have samples from 

this specific cancer, so I think that is going alone fine. The other 

thing that we have done just recently is, we held a meeting just 

last week on infectious agents in cancer, and as you know, gastric 

cancer especially is related to H. pylori, an infestation of a bacterium in the gut, but not all stomach cancer is caused by H. 

pylori, and you heard about one of those today, I am guessing. So 

we are trying to also now investigate other areas that are related 

to intestinal cancers other than just the kinds of normal things we 

have been looking at over the years, specifically, infectious agents. 

Mr. SHIMKUS. What about the cancer Human Biobank, which 

seems very promising? Can you talk about that? 

Ms. BARKER. Yes. We had hoped to—we were planning on 

launching that probably next year, but with the aid of the ARRA 

funding, we were able to launch it in 2009 with about $60 million. 

So it is under way. We expect it to be well under way in the next 

3 years. Certainly by the end of the ARRA period we will have it 

well under way and samples are already beginning to flow in. The 

infrastructure is being created. So thanks to the ARRA funds, we 

were able to get it under way at least a year earlier than we would 

have been able to do it before. 

Mr. SHIMKUS. And I appreciate your quick answers. I still have 

20 seconds left. 

There are some difficult biological materials to obtain in this 

process. Can you identify some of those? 

Ms. BARKER. Well, any of the rare tumors basically. I mean, the 

rare tumors are really hard to get. Sometimes it is because of 

standard of care. Sometimes it is because people hang on to their 

samples; they are very precious and they don’t want to share them. 

So I would say any time that we can encourage and I think really 


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engage the advocacy groups to help us to collect these samples, 

that is where we should be focusing. 

Mr. SHIMKUS. Thank you, Mr. Chairman. 

Mr. PALLONE. Our vice chair, Ms. Capps. 

Mrs. CAPPS. Thank you. 

I found myself, Dr. Barker, getting goose bumps while you were 

giving your testimony. I think it is very stunning what you were 

telling us, and I would like to have you use my time to talk more. 

The whole notion of personalized medicine is kind of floating 

around there. You are kind of pinpointing it so we can know, but 

you mentioned Cancer Genome Atlas, and is that what Mr. Shimkus asked you about? 

Ms. BARKER. That is what the chairman is talking about. 

Mrs. CAPPS. Yes, and the Human Biobank would be part of that 

atlas? 

Ms. BARKER. Separate ideas and separate concepts and separate 

initiatives but very much related to each other because the cancer 

Human Biobank will collect these samples and make them available to The Cancer Genome Atlas. 

Mrs. CAPPS. And then I guess the part that is so intriguing to 

me, you could talk on and on, hours, I suppose, just about that—— 

Ms. BARKER. And I am Irish, so you don’t want to do that. 

Mrs. CAPPS. But maybe another time. I have a feeling we are just 

getting into this, and I feel myself wanting very much to be educated. 

I want to try something on you because there is a—we found that 

there is a piece of our health reform language that is about research on cancer, and the part that intrigues me is the Cures Action Network, another new initiative. I am just quoting from the 

language of the legislation. ‘‘New initiative created in the legislation, an effort to help translate promising discoveries into cures 

with an emphasis on priority health issues where incentives for development are inadequate guided by’’—this is all to be—‘‘guided by 

a board of scientific experts and venture capitalists, individuals 

who have experience identifying promising projects. These experts 

are teamed with advocates who can represent the needs of patients. 

How would you see what you do there? I sort of call that pure research, if you will, and then also with the atlas and those samples, 

how can these results, your results, be translated more quickly to 

treatment and cures? 

Ms. BARKER. So I think it is an end-to-end solution. I mean, basically you are starting with the samples. You are going through The 

Cancer Genome Atlas identifying all of these pathways that are 

disrupted. It allows you now to sort of attack that valley of death 

where things get lost and we don’t have a real opportunity to take 

things to the next step. I think it is a very exciting idea. 

Mrs. CAPPS. Will you be doing that or do you see yourself collaborating? I don’t mean you personally but—— 

Ms. BARKER. Probably both. We do a lot in translational research 

already but this allows actually—if I understand it correctly, it allows for more direct relations and public-private partnerships to 

occur, which is something that is missing now in terms of really 

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overdue and I think we can make good use of both the mechanisms 

and obviously the resources. 

Mrs. CAPPS. So we need to stay in touch with you. I am suggesting this to my chairman at the same time. This is a work in 

progress, both where you are and also this is a brand-new piece of 

legislation, to find out those ways that those connections should be 

made and using the advocacy groups because they are so useful to 

all of us. 

Ms. BARKER. Right. 

Mrs. CAPPS. And then connecting to the private sector. 

Ms. BARKER. Once you know which of these pathways is disrupted and which of these pathways are driving a specific cancer, 

then you are going to have to be able to make a new intervention 

or a drug, and The Cancer Genome Atlas is already sending the 

private sector in a new direction to discover new drugs. We are already seeing changes in the way that they are actually doing business. So the government is actually having a lot to do with sort of 

redirecting the way discovery is going to occur in the future. 

Mrs. CAPPS. Again, it is very exciting. I have 48 seconds left that 

is yours. What else would you like to tell us in that very short 

time? 

Ms. BARKER. Well, you can’t say much in these prepared statements so the couple things I would say is that The Cancer Genome 

Atlas is an example of something that is really changing science. 

I mean, it really is bringing hundreds of people together to look at 

a disease as complex as cancer and integrate all the data and make 

it available. 

Mrs. CAPPS. Let me just interrupt. Say there is someone who 

walks into a doctor’s office and they take a biopsy and it comes 

back, and that is the point that I think we all can relate to. How 

does what you are talking about connect there in any way? 

Ms. BARKER. So in a few years—when we started The Cancer Genome Atlas it was a couple hundred million dollars to sequence a 

cancer genome. It is now down to tens of thousands of dollars, and 

I would predict in as little as 5 years it is going to be down to less 

than $5,000. You get your genome sequenced, and we are going to 

know what the disruptions are and we are going to know what subclass of cancer that you have and you will get the right drug. You 

will not be treated with a generic sort of treatment. You will get 

the right drug for your subclass. 

Mrs. CAPPS. And you will have it in the bank, or someplace it 

will be? 

Ms. BARKER. Well, if we can move ahead quickly on the things 

that you just talked about in terms of developing these new treatments, absolutely. So I think it is going to change the way medicine is practiced completely. 

Mrs. CAPPS. Stunning. That is all I can say. We have to do this 

more, Mr. Chairman. Thank you. 

Mr. PALLONE. I realize that this is a subject of great interest and 

we will look into possibly doing additional hearings. We only have 

6 months left, though, before the end of the session. 

The ranking member, the gentleman from Texas, Mr. Barton. 

Mr. BARTON. Thank you, Mr. Chairman. 


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Thank you, Doctor, for appearing. Dr. Andy von Eschenbach is 

not only a professional acquaintance but a personal friend and has 

helped me in treatment decisions for members of my family, so he 

is somebody that I have a lot of respect for. He has told me that 

with the proper approach and proper funding, we could actually 

find a cure for cancer in the next 15 years. Do you share that view? 

Ms. BARKER. I think it depends on how we define ‘‘cure.’’ I think 

what will happen in this field, and Andy and I have discussed this 

on many occasions, is we are going to learn enough about these 

cancers to actually be able to treat them and have people live normal lives, and if you call that a cure, then that is a cure, but I 

think if you can stop the growth of a cancer, and let us just say 

even the cancer comes back, much as you see with AIDS patients, 

if you now can go in with another treatment that is equally effective and someone gets another 10 years, let us say, which is what 

is happening with breast cancer patients today, for example, then 

you are going to live out your life normally and die from something 

else. So that is the road we are on with cancer. Cancer is an extraordinarily complex disease, probably the most complex disease 

we have ever undertaken, but we are going to understand it well 

enough to be able to control the pieces that need to be controlled 

on a time frame that will allow you, I think, to live a fairly normal 

life. So I think that is the future. 

Mr. BARTON. Now, I don’t have the article in front of me but several months ago I believe Newsweek had a cover story about the 

war against cancer and was the wrong battle being fought. The article was not necessarily negative but it did raise some questions. 

Are you familiar with that article? 

Ms. BARKER. Yes. 

Mr. BARTON. What is your general view of that? It implied to me 

that we are spending a lot of money and kind of doing things the 

conventional way, which is somewhat contrary to what you just discussed with Congresswoman Capps. 

Ms. BARKER. Well, those that know me know that I do very little 

in a conventional way. I mean, most of the programs that we started at NCI are pretty unconventional, whether it is nanotechnology 

or The Cancer Genome Atlas or some of the other things we have 

done. You know, this happens three or four times a year that we 

have a national story that says, you know, why aren’t we winning 

the war on cancer, and, you know, I think it varies from time to 

time as to what the issues are, but I think we do have to look at 

these diseases differently. If we can actually balance the amount 

of individual investigator-initiated research with some of these 

larger programs, I think we will proceed faster. So I think we have 

to come up with sort of piece coexistence of programs that enable 

everybody in individual investigator-initiated grants. 

Mr. BARTON. But you do have a mechanism with NCI to review 

that type of—— 

Ms. BARKER. Yes. 

Mr. BARTON. So there are people thinking about different ways 

to do things? 

Ms. BARKER. Yes. I think it is the biggest change that we are 

going to have in cancer research and ultimately in the way products are developed for cancer patients. The one thing I didn’t say 


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but maybe it is obvious to everybody on the subcommittee is that 

cancer is digital, it is knowable, it is information. Think about it. 

I mean, you know, you pick up this device or another now and it 

is turning over every 5 years or it seems like every 2 months you 

are getting a new device. Well, once we digitize cancer, which is 

what we are doing with The Cancer Genome Atlas, going back to 

the Human Genome Project, that is what the Human Genome 

Project taught us, that DNA is digital, so as you begin to learn the 

information about cancer, you are going to be able to move much 

more rapidly, I think, and I think if cancer is knowable and we can 

decipher all the genes that cause these cancers and make them 

available to everybody, that is going to move the field exponentially. 

Mr. BARTON. Now, one of the things that we did in the NIH Reauthorization Act several years ago was create a common fund that 

requires a certain amount of money each year to go into this fund 

and requires those that wish to take advantage of grants from that 

fund to work across different areas of NIH. That fund has been in 

existence now I believe for 2 years, maybe 3. Is that type of approach something that would tend to help the approaches that you 

are focusing on at NCI? 

Ms. BARKER. They are very similar, and some of them have actually come from the common fund or enabled the common fund as 

many of the NIH institutes actually work in this way. I think the 

common fund is a great idea. I know Dr. Collins is now looking for 

new ideas for the common fund, so yes, they are very complementary and I really commend you for the common fund and the idea. 

It has been very, very helpful. 

Mr. BARTON. And my last question, we are bombarded every year 

on this committee with requests from advocacy groups for special 

bills for specific diseases and specific cancers, and each group, 

whether it is the breast cancer group or the cervical cancer group 

or the prostate cancer group or the autism group or the Alzheimer’s 

group or you name it, the heart group, theirs is always in their 

minds the priority that we should fund. What is your advice to the 

Congress on how we should handle those kinds of bills? 

Ms. BARKER. So we see the same groups, and I think increasingly 

I believe that understanding the biological space or understanding 

the biology that drives cancer and other diseases is going to be critical to everybody. So I think that what we do—and we are learning 

so much now from The Cancer Genome Atlas that says that, for example, GBM, the pathways that are disrupted in GBM are going 

to look like the same pathways only a bit different in terms of the 

quantity of the genes and the exact numbers of genes that are actually disrupted in ovarian cancer so there are going to be some interesting overlaps here that are going to tie these cancers together. 

So I would encourage us all to begin to unravel that mystery that 

is cancer as opposed to, you know, what is prostate cancer, what 

is breast cancer, what is this cancer, what is that cancer. I think 

that is all going to converge and it is starting to converge now. So 

we need to work together to fill in the biological space and find out 

what drives cancer, and I believe it is going to help all these diseases. 

Mr. SHIMKUS. Will the gentleman yield? 


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Mr. BARTON. Yes, I would be happy to yield. 

Mr. SHIMKUS. Just to follow up. Thank you, Dr. Barker. In talking about again the cancer Human Biobank, how do we envision 

researchers across the board having access and using that? It is 

kind of tied to the same question based upon research. Because a 

lot of the advocacy groups are also doing research that I am not 

sure how—if we are getting access to that information or how that 

all works out. Can you in the last minute of—— 

Ms. BARKER. So in the last minute—it is a complex issue. The 

cancer biobank is just getting underway. The Human Biobank is 

just getting underway. But what we want to do is, we will have to 

prioritize access because some of the samples will be quite precious. 

So what we envision is having a board for the biobank that will actually prioritize requests and make samples available and information available on a priority basis but we generally will also offer 

services to investigators who are just looking for or need samples 

and so I think both of those will be possible in the biobank. 

Mr. SHIMKUS. And how do we incentivize people? We will have 

testimony in the next panel of someone with a rare cancer who 

when they had the availability there was no place to go. How do 

we encourage a place to go? 

Ms. BARKER. For? 

Mr. SHIMKUS. I am not a doctor, but the tissues from this stuff. 

Some of these rare diseases, they are rare. It happens, it is gone 

and then you have no place to get the tissue to go to. 

Ms. BARKER. Well, I have proactively—for The Cancer Genome 

Atlas, the first three choices were rare tumors basically: squamous 

cell carcinoma of the lung, ovarian cancer and GBM, GBM being 

the toughest. We spend a lot of time thinking about and working 

on these highly lethal tumors, and we will do more. The Cancer 

Genome Atlas will allow us now to look at these tumors. We are 

going to need help with getting the samples, and this is where the 

advocacy groups can really play a role, and I spoke just recently 

with the Deadly Cancer Coalition and we are working with several 

of those groups now to get those samples into The Cancer Genome 

Atlas. So I think it is working. 

Mr. BARTON. Thank you, Mr. Chairman. 

Mr. PALLONE. Thank you. 

The gentlewoman from the Virgin Islands, Mrs. Christensen. 

Mrs. CHRISTENSEN. Thank you, Mr. Chairman. Thank you, Dr. 

Barker. 

It is very exciting. It was very exciting to read and to listen to 

your testimony today. You focus a lot on of course the genomics 

and the molecular biology, but when you talk about bringing all of 

the experts together and all the data together, does that also include experts on environmental and psychosocial? Does that come 

into the discussion as well? 

Ms. BARKER. Yes, because we are collecting data. I mean, I didn’t 

have time to mention but all the samples we are using are actually 

from patients that are extraordinarily well characterized. They 

have to be to get into The Cancer Genome Atlas. So we have survival data on these patients, exposure data, et cetera. So as we go 

through these processes and learn more and more about these cancers, we will be able to begin to dissect out some of the questions 


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relative to environmental exposures. I know Dr. Collins is planning 

a larger initiative in that regard to look at longitudinal data in 

terms of looking specifically at lifetime exposures in patients. We 

don’t have that capability right now. 

Mrs. CHRISTENSEN. Thank you. A Dr. Foege, I guess is how that 

name is pronounced at CDC, is quoted as saying, ‘‘The challenge 

to genomics is to overcome the inequitable allocation of benefits, 

the tragedy that would befall us if we made the promise of genetics 

only for those who could afford it and not for all of society.’’ So how 

do we incorporate population-level considerations of personalized 

cancer medicine and ensure that the emphasis on molecularly targeted therapies won’t exacerbate the health disparities? 

Ms. BARKER. I have lots of friends who always ask me this question—Harold Friedman from New York, who was at the NCI and 

actually founded our disparities program. I honestly think that this 

is going to do a lot to remove many of those barriers because one 

thing we are finding actually is that early on I think people assumed that maybe if you take the African American population, for 

example, that their cancers look the same as other cancers, and in 

fact, it is likely not to be the case. So we are studying these cancers 

now and we have cohorts of these cancers where we will be able 

to determine if there are differences, if there are inherited differences and if there are population differences. We are also working across borders with several countries. Almost all the countries 

now have actually—The Cancer Genome Atlas has become very 

popular. It is being cloned in lots of countries now. There is a meeting going on in Spain today that I was supposed to be at, but the 

United States, we are actually providing all these countries with 

the construct for how we are doing this. So there are going to be 

lots and lots of data on many countries including Africa where we 

will be able to sort out a lot of these differences. So I think actually 

this could reduce the digital divide and I think it will because I 

think we are going to know enough to do what is right for all these 

populations, not just for some. 

Mrs. CHRISTENSEN. And are you doing anything, any specific research on the more aggressive forms of breast cancer that black 

women are disproportionately likely to get? I noticed that in your 

plan you say you are using ARRA money to look at health disparities and really target these kinds of issues. 

Ms. BARKER. Right. So you are talking primarily about triplenegative breast cancer, which is a devastating cancer and it is 

quite high in African American women and quite devastating, and 

we have a number of programs in the NCI looking at that. We just 

launched a new program which I don’t have time to talk about last 

week actually called the I–SPY 2 trial which is actually a program 

to look at breast cancer in the neoadjuvant setting. Patients are 

going to have surgery. Many of those patients will be triple-negative breast cancer patients but the idea is to use an adaptive trial 

and test a large number of agents which we have not been able to 

do before and move drugs through very quickly. So I think we have 

got a lot of attention on that disease right now. 

Mrs. CHRISTENSEN. Great. We could probably look that up on 

your Web site? 

Ms. BARKER. Yes. 


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Mrs. CHRISTENSEN. One last question. I notice that you have a 

center to reduce cancer health disparities at the NCI. I would like 

to just know how do you relate and interact with the National Center for Minority Health and Health Disparities research. 

Ms. BARKER. Very close interactions, and I think our center for 

reducing health disparities is a real flagship for all of the initiatives that are ongoing around cancer and disparities, and everything from our centers, you know, where we are actually training 

and bringing in new investigators, minority investigators especially, to some of the special research programs. It is really quite 

a success. 

Mrs. CHRISTENSEN. Thank you. 

Thank you, Mr. Chairman. 

Mr. PALLONE. The gentleman from Indiana is recognized for 8 

minutes. Mr. Buyer. 

Mr. BUYER. I thank the gentleman. 

Ma’am, when you were holding up your cell phone, I couldn’t 

help but sense that as you talked about the genome and how fast 

our discoveries are moving, I am going to ask a question. I don’t 

know the answer to this but I am going to do a systems analytical, 

and this would be our delivery systems. Sometimes our delivery 

systems can be caught in time. It is logistics, it is who is doing 

what in the preparation of what you described as that proper drug. 

So if you take yourself back actually maybe 15 years ago, some of 

the larger pharmaceutical companies did some discoveries and they 

held the drugs and they realized that they couldn’t keep up and 

some would then spin them off either into the compound pharmacies or some nuclear pharmacies and they would be responsive 

to some cancer hospital or clinic or particular doctor, and so we 

have a unique delivery system on how that proper mix is getting 

there. Is that delivery system keeping pace in order for us to do 

that specialized proper care as you envision? 

Ms. BARKER. Well, that is a tough question. 

Mr. BUYER. I don’t know the answer to it either. 

Ms. BARKER. Well, I think I do know the answer. I mean, I think 

the answer is, as of now I think we are keeping pace pretty well 

because the targeted drugs we are using in cancer research today, 

drugs like Gleevec and Avastin, are getting to patients. I think the 

bigger question is, when we have hundreds of these kinds of drugs 

and you are going to be hard-pressed to know everything there is 

about every one of these drugs, will people keep pace then. I think 

that information is going to change and the amount of information 

available is going to change at a pace that will allow people to keep 

up. But I think delivery is going to be the major challenge. I don’t 

disagree with that. I think it is a huge systems challenge. 

Mr. BUYER. I asked that question from a systems analytical approach. We have a regulatory—all these regulations that are in 

place. Take, for example, a doctor that—you said the personalized 

sampling increasing personalized care. So when the doctor now 

knows what my patient’s needs and requirements are, sends off to 

someone for a particular proper drug mix and it is then placed into 

the delivery system whether it is FedEx or UPS. Now it comes 

under some other regulatory schematic and maybe it is held in 

FedEx, it is brought in overnight and it is now held in a different 


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place because of the regs and it has a time—sensitive in time. And 

now it may sit there for 2 days, and by the time doc gets it, how 

stale is the mix with regard to toxicity? You know what I am saying? I look at our system here and you say OK, the more we personalize, the better care we can get, but are we doing what we need 

to do because by the time that proper mix gets, is it going to do 

what the doc wants. This is my question. 

Ms. BARKER. Well, I am not funded for FedEx but I can tell you 

right now that the delivery of drugs in this country is a very high 

priority for the pharmaceutical companies that actually send them 

to physicians, and the delivery thereof is highly monitored by physicians because everybody knows that many of these drugs are actually not terribly stable. They have to be kept at certain temperatures, et cetera. I don’t think we see that problem very often but 

to your point, as you have more and more of these, it could become 

more of an issue. So it is a systems problem. 

Mr. BUYER. That is why I am asking the question. If this is 

where we are going, when you say what is our over-the-horizon vision, will our delivery system match your vision. Maybe I 

should—— 

Ms. BARKER. No, and I think—in oncology we have been working 

on that for many years and I think, you know, because we do have 

an oncology community, especially a community of physicians that 

are very, very good at keeping track of the information. I think it 

will morph according but it is going to be a huge challenge, I mean, 

for all diseases, not just cancer actually. 

Mr. BUYER. I yield to Mr. Shimkus. Are you good? I yield back 

my time. Thank you. 

Ms. BARKER. You are welcome. 

Mr. PALLONE. Thank you. Recognize the gentlewoman from Florida, Ms. Castor, for 8 minutes. 

Ms. CASTOR. Thank you, Chairman Pallone, very much. 

Dr. Barker and everyone at the National Cancer Institute, thank 

you very much for all that you are doing on cancer research and 

that vital—it is fundamental to American families and the struggles they have every day with cancer, and as my colleague said, 

there is just no one that has been unaffected. 

I am particularly enthused about your focus on genomics. I represent the Tampa Bay area that is home to the Moffitt Cancer Center, and they are really at the forefront of the national genomics 

project and have been educating me over the past few years, and 

let me tell you, even the Recovery Act money now, the NCI money, 

the Recovery Act that has gone to even greater research dollars 

and jobs, kind of high-wage jobs we want in our community and 

this new emphasis on genomics, and we also get support through 

the defense bill to even bolster the NCI funding. Moffitt launched 

their database in 1999 and they have been gathering that genomic 

data, the genetic profiles and the clinical history data for each enrolled patient, and I guess the next big step was to reach out to 

other researchers and other hospitals and begin that important 

partnership on the biorepository. So we are spreading out across 

Florida through the Moffitt, and I would like you to expand a little 

bit more on that data collection and the sample collection. I know 

the Moffitt, we have got 50,000 cancer patients so far have conVerDate Mar 15 2010 13:13 Dec 14, 2012 Jkt 076020 PO 00000 Frm 00060 Fmt 6633 Sfmt 6633 E:\HR\OC\A020.XXX A020

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sented to have their clinical data and molecular profiles added to 

the database and that is going to be accessible to physicians, researchers and the patient themselves. They call it the total cancer 

care database that then flows into the personalized treatment that 

you are talking about. 

But could you drill down farther on the details of collection and 

the challenges that we are going to face? You did mention in passing the protocols, ensuring that the sample meets your standards. 

Talk a little bit about that in the data collection. 

Ms. BARKER. So first of all, I just have to say that Dr. Bill Dalton 

at the Moffitt Center is one of the prime examples of a state that 

has actually put their whole state ahead. I think of the curve here. 

It is an amazing amount of work that they have done and they 

have actually taken a lot from The Cancer Genome Atlas directly 

and implemented it in Florida, a very commendable effort. 

In terms of some of the specifics, I mean, we have established at 

the NCI a set of best practices guidelines which I think have enormous merit for all investigators to be held accountable to, and so 

we are working on that, but I think in terms of the specimens 

themselves, they are very prescribed SOPs, or standard operating 

procedures, for how long in the operating room, you know, when it 

goes into storage, how it’s stored, how you access it, the kind of 

bioinformatics platforms that you need to keep track of the samples, the consent that goes with the samples, and in this era of 

genomics they are quite different consents for patients than they 

were before. So there is an enormous amount of information. 

Moffitt has all of that, and they have been using our standard operating procedures now for a couple of years. 

Ms. CASTOR. So you have got how many research institutes that 

are part of genomics data gathering right now? 

Ms. BARKER. Well, there are literally hundreds of those, but for 

The Cancer Genome Atlas, there are probably 20 different institutions that are contributing. 

Ms. CASTOR. And then is it their responsibility to use your framework and the SOPS—— 

Ms. BARKER. Yes. 

Ms. CASTOR [continuing]. To continue the outreach to gather 

those samples? 

Ms. BARKER. That is correct, and what you want is a standardized approach so we are all talking about the same things when we 

get to experimentation. Now, I thought you were going to ask a different question, and that is—— 

Ms. CASTOR. What did you think I was going to ask? 

Ms. BARKER. That is that there is something about these samples—we are actually doing some research on what causes changes 

in samples. We don’t know. I mean, patients undergo anesthesia, 

all kinds of things in the OR. We don’t know what impact that has. 

We have undertaken some research at NCI to try to figure that 

out. It is an interesting problem. 

Ms. CASTOR. How often does that occur? Is that standard, happening throughout all the samples? 

Ms. BARKER. Well, it is actually—we have just undertaken that 

research in the last 2 years. No one has ever asked the question 

before. No one has ever said, what is the impact of anesthesia on 


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a cancer sample, I mean, until I hired a pathologist to come to NCI, 

who I have known for a very long time, and the first question she 

asked me was, how do you know the anesthesia patients get is not 

actually responsible for the biomarkers you are measuring versus 

the biology itself. Nobody ever asked that question before. So we 

are doing research now to answer that question but it is a very important question, along with several other questions that we are 

trying to answer, so it is an interesting set of questions driven now 

by personalized cancer medicine because we want to know what 

those samples are actually telling us about the biology. 

Ms. CASTOR. And as you build this digitized database, I guess it 

is the molecular footprint for each of these cancers? 

Ms. BARKER. Right. 

Ms. CASTOR. And then you have control factors based on all sorts 

of considerations. Can you state again in plain language what that 

means? Maybe go back to the latest ovarian cancer findings, what 

that means to a patient now and in the future. 

Ms. BARKER. So what that will mean is that take ovarian cancer. 

We have identified three subclasses of ovarian cancer, very clear 

molecular subclasses. So we are going to know which drugs work 

on which subclasses. We already have survival data that says these 

drugs predict survival and these subclasses, at least two of the subclasses, so you are going to know as a patient what you should get, 

and you are also going to know as a physician whether or not anyone should get any drug basically. You know, right now we generically treat everybody with the same drug. If you have ovarian cancer, you either get a platinum-based drug or you get Taxol, and you 

may benefit from one, you might benefit from both, you may benefit 

from neither. So now we are going to be able to tell you which of 

these you are going to benefit from and the physician will have 

some idea about the expected longevity. As we go forward, I think 

you are going to see more and better cancer therapeutics for those 

specific subclasses, and that is the whole goal of The Cancer Genome Atlas. So patients can benefit very quickly by actually being 

put on the right drugs, I mean, going into the right clinical trials, 

putting patients in the right trials and then ultimately treating patients with the right drugs. 

Ms. CASTOR. And they benefit by submitting their samples? 

Ms. BARKER. Yes, and increasingly every patient coming in for 

any diagnosis of cancer should have their sample collected, stored 

and kept, period. 

Ms. CASTOR. Well, again, thank you very much for what you are 

doing. I will pass along to Dr. Dalton that you have kudos of the 

Moffitt. 

Ms. BARKER. He is a great catalyst. 

Ms. CASTOR. Thank you. 

Mr. PALLONE. Thank you. Now, we have our last series of votes, 

a 15-minute and a 2-minute—I am sorry, a 15-minute and two 5- 

minutes. 

Mr. Gingrey, you are next and you have 8 minutes. Do you want 

to try to use it? 

Mr. GINGREY. Why don’t I take a shot at it, Mr. Chairman? 

Mr. PALLONE. All right. You are recognized. The other members, 

if you want to go vote and then we will—— 


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Mr. GINGREY. I think if you don’t mind, Mr. Chairman, and I appreciate, I will go ahead. This is so interesting, I hate to lose my 

train of thought. 

Dr. Barker, thank you so much for being with us and sharing 

this information. I have a medical background, but I can tell you, 

on this issue I don’t know any more, maybe less than my colleagues 

on the subcommittee. You talked a good bit about the state of the 

art in regard to the TCGA and being able to figure out subsets. You 

just mentioned, I think, in ovarian cancer and being able to tailor 

specific drug therapy depending on the subset, which I think is fantastic, and this is a great thing and I am glad that we are continuing to fund it, and I will certainly continue to support that. 

I want to take it maybe a step further into the future and maybe 

you are already there, you haven’t talked about it yet, but, you 

know, there was a study in the New England Journal of Medicine 

last year, I think it was called the REVEAL study. Are you familiar 

with that, Dr. Barker? 

Ms. BARKER. Yes. 

Mr. GINGREY. Well, let me continue down that line then. In that 

study, it was on the impact of educating about genomic predispositions had on a patient’s emotional state. Now, let me make it a little more personal and anecdotal. I had a first cousin deceased several years ago of Lou Gehrig’s disease, amyotrophic lateral sclerosis, and he had a wife and three sons and a great life until he 

got this disease and it took him 3 or 4 years to die from Lou 

Gehrig’s disease. Now, there will come a day, maybe it is already 

here and I want you to tell us about it, where every individual will 

go in for an annual routine screening physical examination, and instead of just having several tubes of blood drawn and checking for 

blood type and different things and maybe an X-ray and an EKG, 

that they will have an opportunity to have their own genomes studied and maybe a printout that would suggest well, you are 20 years 

old, Mr. Jones, and your likelihood of developing Lou Gehrig’s disease or Huntington’s chorea or prostate cancer at some time in 

your life is a certain statistic or to a woman, same thing with 

breast cancer and ovarian cancer. But it is no assurance that that 

will happen to them, it is just a statistical likelihood or chance. 

How much of this information should be given to patients and what 

effect would it have on their lives in regard to something like, let 

us say, Lou Gehrig’s disease which has no cure or Huntingdon’s 

chorea, which has no cure? Should they know this? Is this something that people want to know or would it drive them nuts to have 

that information, say, 40 years before the occurrence of the disease? 

Ms. BARKER. So you raise an excellent point. I think we have 

reached a point in our society where advanced technologies are 

ahead of the extent to which we have actually incorporated this 

into our thinking and our planning. Genetic counseling is something that we are investing in but probably not enough, and I think 

we are going to have to do a lot more and I think genetic counseling will become very much a practice of medicine in the future. 

I am a bit of futurist so I believe that what is going to happen is 

that you will get your genome. I think you will know that your 

probabilities of getting certain diseases are, but as you point out, 


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the chances are really going to be dependent on the environmental 

exposure you receive and dozens of other factors and so many people inherit predisposition for breast cancer, don’t get breast cancer, 

and frankly, all the inherited genes that we know about in cancer 

today are responsible for only probably less than 5 percent of all 

cancers. 

So I do think that people will want to know, and I think as we 

go forward, and I think the whole idea here is, we will develop prospective strategies for dealing with that both in terms of genetic 

counseling and ultimately interventions. So we haven’t talked 

much about prevention today but I think as you understand the 

genomics of these diseases, you will be able to go in and figure out 

ways to prevent them. Now, it is going to take some time but I 

think it is going to catalyze a whole new field here of, you know, 

sort of genomics-based prevention, and that is what I am in favor 

of. But as you know, there are companies already today that are 

giving people their genomes, I mean, at least your germline DNA, 

the changes that you inherited, and they do have genetic counselors. There are two or three of these companies now. And we are 

kind of watching that to see what impact that is having on patients. 

Mr. GINGREY. Well, in the couple of minutes that I have left, let 

me ask your opinion on this. Do you think ultimately that this will 

reduce the cost of medicine or that it ultimately will drive it up 

drastically? 

Ms. BARKER. Well, I don’t know the answer to the question but 

my guess is that there will be a blip as in all things and people 

will take a very high interest in this, and we just discussed, they 

will then realize that there are certain strategies that you have to 

undertake to mitigate you risk but that is pretty much what you 

can do until we can develop interventions to actually either prevent 

the disease or treat the disease effectively. So I think there may 

be some blip once everybody—you know, I think it is not going to 

be that long until everybody can get their genomes sequenced. So 

I think there will be a period when people have a high level of interest but I am guessing that will become integrated into our society and we will ease off. So I think in the long run it will reduce 

the cost of health care. 

Mr. GINGREY. Well, I think in the long run it will too, and I think 

I agree with you on that. 

The final point that I wanted to ask you about of course is the 

issue of discrimination, and of course as people get this information 

at a very young age and go out into the job market, how do we protect them from the possibility of being discriminated against because they have a high risk of developing breast cancer or colon 

cancer or whatever and as employers look at that or have access 

to that information? It would be so important to make sure that 

we protect that information. 

Ms. BARKER. And we do. I mean, I didn’t mention that all of the 

data in The Cancer Genome Atlas is protected at a second level. 

If you want any access to patient data, you have to actually apply 

for it, and any good, you know, sort of validated investigator can 

access that data. We are also protecting the patients’ data through 

informed consent, and of course, ultimately the Nation is protecting 


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that data though GINA and many people have worked tirelessly to 

pass that bill a few years ago. So I think we have anticipated this. 

That doesn’t mean that there might not be somebody who falls 

through the cracks here but we have done everything we can but 

we are going to have to stay ahead of this so that as more and 

more of this information becomes available, that opportunity 

doesn’t become too much of a temptation for people to actually 

abuse their privileges in that regard. 

Mr. GINGREY. Dr. Barker, thank you. My time is about up, and 

I thank you so much. 

Mr. Chairman, I yield back. 

Mr. PALLONE. Thank you. Now, I don’t think there is any time 

left on the floor so I suggest everyone go over there. But Ms. Eshoo 

wanted to be recognized. 

Ms. ESHOO. Thank you, Mr. Chairman. Unfortunately, I can’t 

come back after votes because I have to attend an Intelligence 

Committee meeting, but I want to thank you, Dr. Barker. I am 

going to submit my questions to you. 

I just want to make an observation, and that is that I think that 

there needs to be a healthy balance all the way around. You used 

that word in the beginning of your testimony. But also about advocates and advocacy. I remember a time where the NIH was reluctant for whatever reasons, maybe it was whatever was going on at 

the time with HIV/AIDS. It was the Congress that pushed it and 

they pushed the money into it in order to make things happen, especially to help care for the people that were afflicted, which we 

thought was going to be a pandemic at the time. So I have a lot 

of regard for the advocacy groups, and there is one that is going 

to be testifying today on behalf of pancreatic cancer. I think what 

I would like to know more about at some point is where the balance is between the atlas, the samples, what we are doing with it, 

The Cancer Genome Atlas, but also the funding of the rest as we 

do that, because I think that there is a correlation between low investment in research and poor survival rates, and I don’t think 

anyone can really get around that. We have never made a low and 

gotten a big bang for it. 

So I think we need to explore these things and I hope, Mr. Chairman, we will invite Dr. Barker back because there is a great deal 

of hope riding on you and I think in your words we found a great 

deal of hope. Thank you, Mr. Chairman. 

Ms. BARKER. Thank you. 

Mr. PALLONE. Thank you. We stand in recess until these votes. 

[Recess.] 

Mr. PALLONE. The subcommittee will reconvene. I think we have 

at least one more member who would like to ask Dr. Barker some 

questions. I yield to the gentleman from Texas, Dr. Burgess. 

Mr. BURGESS. Thank you, Mr. Chairman. I apologize for missing 

the earlier part of the hearing. As you know, we have several 

things happening simultaneously. That almost never happens up 

here on Capitol Hill, but today it did. 

You know, one of the things that has come up from time to 

time—we had the FDA in last week or the week before. There was 

a significant amount of funding coming to the National Institute of 

Health out of the stimulus bill last year. As I recall, that was a $10 


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billion amount that came to the NIH. What portion of that came 

to the National Cancer Institute? 

Ms. BARKER. About $1.28 billion. 

Mr. BURGESS. And can you give us an idea of where that funding 

is now? Has it all been allocated? Is there research now underway 

as a result of that? Where are we in the process with that? 

Ms. BARKER. Most of the funds have been allocated, probably the 

lion’s share of the funds have been allocated, and we will spend 

about $849 million obligated in 2009 and the remainder will be obligated in 2010. The funds went for special initiatives like The Cancer Genome Atlas, for example, where we invested in acquiring the 

samples and actually building the database and then Dr. Collins 

actually also invested money in sequencing these tumors. So some 

of the money went into the cancer Human Biobank. Much of the 

money went into, as you might imagine, individual investigator-initiated grants, and some of the money went into supplements to existing grants for investigators who had special projects. We funded 

quite a number of the innovation grants that actually came in, and 

overall, the lion’s share of the funds as they usually do go to individual investigators but we were able to fund several infrastructure, you know, kinds of programs like The Cancer Genome Atlas 

and also a lot of translational science, new clinical trials. This is 

probably the boost for cancer research that I have ever seen, I 

think the biggest and most catalytic action I have ever seen in cancer research. It has stimulated more thinking and more activities 

that I have seen in 35 years, and I think it is going to have a huge 

impact downstream. 

Mr. BURGESS. When you fund these research projects, was this 

money that was all allocated to be spent over 1 year’s time or have 

you funded a research project that may go on over multiple years? 

Ms. BARKER. Well, as you know, people are encouraged to spend 

their money in 2 years and to actually maximize the numbers of 

people that are hired newly and minimize the number of people 

who had to be let go in some of these institutions, but some of the 

grants will be extended over a couple of additional years, especially 

those that require longer-term objectives be met. So generally 

speaking, we are encouraging people to spend the money in the 2- 

year period allotted but there are certain kinds of studies that 

could proceed another couple of years if they have permission to do 

so. 

Mr. BURGESS. What about the sustainability of research? It is an 

odd way to get money to get a big chunk of dollars like that. Obviously you have to make a commitment to scientists who are going 

to stay with you. What do you expect to see in the funding stream 

and the appropriations process over the next several years? How 

are we going to make that pitch to the appropriators? As I recall, 

we did the NIH reauthorization in 2006. It was a base of $30 billion with an increase of 5 percent, so roughly $1.5 billion a year 

over the 6 years of the authorization. We are coming to the end of 

that time. Has that been adequate in the way of funding, and now 

with this additional money that is coming in? Are we going to have 

other projects that need to be sustained? What do we look for in 

the future? 


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Ms. BARKER. Well, I think the additional of $10 billion to NIH 

was an extraordinarily wise strategic move on the part of Congress 

and the President. I think it will be extraordinarily difficult, as you 

would expect, to reduce the numbers of investigators, to reduce the 

number of programs coming out of the ARRA period. So I think the 

decisions will be tough ones but I am hopeful that the pace of research and the pace at which things are moving right now in terms 

of moving us towards a health care system that I think will be 

much more responsive in terms of reducing costs because we know 

what the disease is and how to treat it than continuing on the 

paths that we are on. I think it is one of the best investments we 

have ever made actually. 

Mr. BURGESS. And I don’t disagree, but putting a bunch of money 

in, you are going to come up—you probably have some things that 

are in clinical trials. How do you see the interaction with the FDA 

going forward? You are going to have a lot of stuff that has to go 

through the FDA. And I can actually remember having this discussion with Andy von Eschenbach when he was at NCI before he was 

at FDA, and he was concerned about the FDA’s ability to keep up 

with the pace of research, and this was back in 2004 and 2005 that 

he was doing when he was at NCI. So how do you see this playing 

out? Is the FDA going to be able to keep up with the pressure that 

you are going to putting on with the demand for approval of new 

drugs and new therapies and new techniques? 

Ms. BARKER. So Andy von Eschenbach, the same year you talked 

to him talked to me and said we need to put together an interagency oncology task force with FDA. We did that. And that task 

force has been working now for almost 7 years now, and I would 

say that—and we have been working on the science that is required to enable FDA to actually, you know, review these drugs 

and devices accordingly. So many of our things fail because there 

hasn’t been enough science done to actually inform the process. So 

what we have tried to do over the last 6 or 7 years is to sort of 

identify the regulatory science that has to be done, to work with 

them to determine what is a clinical trial going to have to look like, 

what do the biomarkers have to do to perform in a regulatory 

sense. We made a lot of progress in that regard, and so I think 

FDA—and there is a new announcement even a few weeks ago between the Secretary and Dr. Collins of a new relationship between 

FDA and NIH to do exactly what we have been doing, and that is, 

to really enhance this regulatory pathway by actually informing 

the science up front. So I think it is a relationship that has been 

built and will continue to be built, and I think FDA is anticipating 

this influx of new agents. For example, we work with them on 

nanotechnology products and so they are actually coming along and 

I think are on the same page with where we are. 

Mr. BURGESS. I am going to run out of time. 

Mr. PALLONE. You are out of time. 

Mr. BURGESS. How do you—— 

Mr. PALLONE. You can’t ask any more questions. Your time is 

over. Let us move on. 

Ms. Schakowsky. 


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Ms. SCHAKOWSKY. Thank you, Mr. Chairman. I thank you, Dr. 

Barker, and actually all the witnesses who have been patient and 

waited with us. 

This is really just thrilling, the genomics and the nanotechnology 

and the next generation. I am just wondering if we have all the 

personnel we need. I have had concerns that once an upcoming scientist leaves NIH for the private sector they don’t often come back, 

and I wondered if this is a concern at NCI. What impact does it 

have on NCI’s ability to advance promising research? Is this a 

problem where there is a brain drain at all? 

Ms. BARKER. So I think it is always going to be a problem because I don’t think in our country we are placing enough emphasis 

in high schools and even earlier on science and mathematics, and 

that is a problem. We need to do more about that. I just came from 

China last November, and looking at the number of computational 

scientists they are training, for example, we are those computational scientists, and fortunately we can partner with these countries. But at NCI what we have attempted to do over the last 7 

years is actually really look at the first-time investigator coming in 

for a grant. We pull out those applications. We call them star R1s. 

And we preferentially fund those young investigators. So I think 

we are doing fine in terms of funding young investigators. I think 

where we are going to fall short is having individuals who know 

how to work in teams and actually areas like mathematics, as I 

said before, because the amount of data that we are creating, 

terabytes every week, has to be analyzed and we have very few individuals that we have trained to analyze this quantity of data. We 

don’t have many Google experts in the biomedical research enterprise. So I think we are doing fine but I think we really need to 

be very proactive about this and our universities and our high 

schools and even in our grade schools have to start thinking about 

putting science and mathematics back on the agenda, the top of the 

agenda, so they can bring the best and brightest into this field. I 

can’t imagine if I were entering college today, I mean, I cannot 

imagine what an exciting future they are looking at. 

Ms. SCHAKOWSKY. Exactly. 

Ms. BARKER. It is absolutely stunning. You know, the choices are 

amazing but these kids all grew up with their computer games and 

game theory and those are the kids that will be able to analyze the 

data. 

Ms. SCHAKOWSKY. Right, and they are used to changing technologies and being early adopters and being flexible. I think that 

we need to do a better sales job about just what is possible and 

soon in the fields of science. I was told by the Northwestern University in my hometown that were it not for Chinese students that 

postgraduate programs in science and math would probably have 

to shut down, and now the foreign students that we certainly want 

to come in go home. They are taking the skills that they learn here 

and going back home. 

Ms. BARKER. In large numbers. 

Ms. SCHAKOWSKY. Yes, and so I appreciate your emphasizing the 

STEM programs. I think we all have to get behind that. But I 

think if we could just paint a picture of the kinds of achievements 

that are possible for them as individuals, the kinds of teams that 


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they could be working on that are going to deliver cures, it is just 

incredible. So I thank you for all your work, and Mr. Chairman, I 

yield back. 

Ms. BARKER. My pleasure. 

Mr. PALLONE. I think that completes our questions. Thank you 

for bearing with us and for your input. I know members talk about 

doing further hearings on this subject. We will certainly look at it. 

I don’t know what exactly we can do but I do appreciate you being 

here today. You may get some additional written questions within 

the next 10 days or so from the clerk, and if you do, I would ask 

that you respond to them. Thanks a lot. 

Ms. BARKER. I really appreciate the opportunity. Thanks to all. 

Mr. BURGESS. Mr. Chairman, may I just make an inquiry? Do we 

get at the committee level—it seems like in years past the NCI 

used to produce an annual report? Is that made available to the 

committee? 

Ms. BARKER. Yes. 

Mr. BURGESS. Mr. Chairman, could I just ask that we all get a 

copy of the most recent report? I think it would be—— 

Mr. PALLONE. Is it something that is very voluminous? 

Ms. BARKER. No. 

Mr. PALLONE. You can get hard copies? 

Ms. BARKER. It is quite short. 

Mr. PALLONE. All right. Let us get some hard copies. Thank you. 

Ms. BARKER. All right. We will follow up with that. Thank you. 

Mr. PALLONE. I ask the second panel to come forward, if you 

would. Thank you for bearing with us. Let me introduce each of the 

members of this panel. Beginning on my left, she was introduced 

before, is Ms. Kristin Fitzgerald from Naperville, Illinois. Thanks 

for being with us today. And then we have Megan Gordon Don, 

who is the chair of the Deadly Cancer Coalition and director of government affairs for the Pancreatic Cancer Action Network. And 

then from New Jersey is Dr. Robert DiPaola, who is a member of 

the American Association of Cancer Research and director of the 

Cancer Institute of New Jersey. And last is Jeff Allen, who is the 

executive director of the Friends of Cancer Research. We ask you 

each to give us a 5-minute statement, if you will. That becomes 

part of the hearing record and after that our own questions for 5 

minutes each. As I mentioned, we may give you some written questions in the next 10 days or so. 

So let us start with Ms. Fitzgerald. Thank you so much. 


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63 

STATEMENTS OF KRISTIN FITZGERALD, NAPERVILLE, ILLINOIS; MEGAN GORDON DON, M.H.S., CHAIR, DEADLY CANCER 

COALITION, DIRECTOR OF GOVERNMENT AFFAIRS, PANCREATIC CANCER ACTION NETWORK; ROBERT S. DIPAOLA, 

M.D., MEMBER, AMERICAN ASSOCIATION OF CANCER RESEARCH, AND DIRECTOR, CANCER INSTITUTE OF NEW JERSEY; AND JEFF ALLEN, PH.D., EXECUTIVE DIRECTOR, 

FRIENDS OF CANCER RESEARCH 

STATEMENT OF KRISTIN FITZGERALD 

Ms. FITZGERALD. Mr. Chairman, members of the subcommittee, 

I want to thank you for the opportunity to testify at today’s hearing. 

My name is Kristin Fitzgerald. As a former health staffer, I have 

participated in many Congressional hearings. This is, however, my 

first time on this side of the dais, and I greatly appreciate the opportunity to speak to the challenges facing cancer research. I am 

here today not only on my own behalf, but that of my husband, Ray 

Fitzgerald, who as you have heard was a staffer for Congressman 

Shimkus. 

Ray died last January of gastric, or stomach, cancer. Until his diagnosis in May of 2008, Ray was a healthy 36-year-old man. He 

had no risk factors for cancer. He had never smoked, drank infrequently, and lived a healthy lifestyle. With a large Irish family before him, there were only four unrelated incidences of cancer before 

him. Nothing would ever have put him at high risk of a cancer diagnosis. 

Ray’s cancer symptom was burping, which appeared for a period 

of 2 months before his cancer was diagnosed. When Ray was diagnosed, his cancer was an advanced stage IV. His gastric tumor had 

spread throughout the lining of his stomach and progressed to his 

esophagus and throughout his liver. We were told that there was 

no hope of a cure but that chemotherapy could reduce the cancer 

for a time. That time was 8 months. 

This is not an abnormal scenario for gastric cancer. It is the second deadliest cancer worldwide. It very often presents in Stage IV, 

and is always incurable at that point. Ray however, was 40 years 

younger than the average gastric cancer patient, and thus the grim 

prognosis impacted not just Ray, but myself and our three young 

daughters, Nora, Maggie and Lucy. Nora and Maggie are here. 

It is my belief that Ray’s diagnosis and prognosis is our worst 

cancer nightmare: diagnosis of a deadly cancer with very few warning signs at an advanced stage where a cure is impossible. It is a 

death sentence. And if we think it can’t or won’t happen to us, we 

are wrong. Ray was one of us, or at least in your case, your staff. 

And as I have learned, it could even be happening to one of us 

right now, and we would never know it. 

After Ray died, I spent some time talking with Ray’s doctors to 

see how this kind of scenario can be prevented so that more young 

dads and moms aren’t violently stolen from their families by cancer. As a former health staffer, I assumed that gastric cancer research was ongoing and would utilize Ray’s tumor specimen and 

facts about his age and health status to find a cure for this deadly 

cancer. However, far too little is being done to research gastric canVerDate Mar 15 2010 13:13 Dec 14, 2012 Jkt 076020 PO 00000 Frm 00070 Fmt 6633 Sfmt 6633 E:\HR\OC\A020.XXX A020

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64 

cer and other gastrointestinal, or GI, cancers that have a similar 

deadly prognosis. CBS News analyzed the disparity in research dollars in May of 2009. For each cancer death, the most federal research dollars are spent on cancer of the cervix at $18,000 per fatality and breast at $14,000 per fatality, contrasted by the least 

funded, which is gastric cancer at $1,100 per cancer fatality. 

GI cancers are some of the deadliest cancers in the United States 

with deaths attributed to the digestive system second only to those 

in the respiratory system. Four out of the five lowest 5-year cancer 

survival rates for metastatic cancer are GI, pancreas, liver, esophagus and stomach. And GI cancers are rising, particularly in young 

people. A recent NCI article documented the rise in 

gastroesophageal cancers of the stomach and esophagus. This article compared the incidence rates in two 4-year periods, 1975 

through 1979 and 2000 through 2004. Overall, there was a 44 percent increase in these cancers. Within gastroesophageal cancers 

there was an explosion of a particular type, adenocarcinoma, which 

is what Ray had. The increase in adenocarcinoma was 465 percent, 

with a 190 percent increase in young white males. And the situation for young people with GI cancers is particularly grim. Because 

GI cancers are considered to be diseases of middle or advanced age, 

the diagnosis in people under 40 is often delayed. As a result, the 

disease is usually in an advanced stage with a poor prognosis by 

the time the diagnosis is established. And their very age works 

against them. The strength and relative health of their bodies is 

passed on to their cancers, making them even more aggressive 

than in older patients. As a result, GI cancers in young people tend 

to be fatal. 

Yet, unlike other deadly cancers, gastric cancer and many other 

GI cancers do not have a national clinical registry and tissue bank 

to utilize tumor tissue and clinical records for research purposes. 

In my view, this is intolerable. Congress and NCI can and should 

do more to ensure that researchers can have access to the tools 

they need to prevent and diagnose these cancers before it is too 

late. Though these cancers are growing, they are poor candidates 

for wide-scale screening programs due to the smaller population of 

people impacted and the invasive nature of screening available. 

More research is essential in order to understand the unique 

characteristics of the disease in younger people and develop a 

screening test based on molecular markers to allow for earlier detection. In order to accomplish this research, NCI must develop a 

coordinated national GI cancer tissue biorepository and accompanying research project to focus research in this area and make 

tumor tissue available for research purposes. 

Last year the Labor, HHS, and Education Appropriations Report 

included language asking NCI to do just that. To date, this has not 

yet been accomplished. Congress must act to ensure that these cancers can be detected and cured so that more lives are not lost. 

Ray was a wonderful man and his spirit will live on always. 

However, it is my belief that Congress should fund a research 

project, tissue bank and registry so that the physical legacy of a patient like Ray can live on forever, giving eternal gifts to researchers 

and scientists throughout the world. 


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65 

Members of the subcommittee, thank you for your time and consideration. I am happy to answer any questions. 

[The prepared statement of Ms. Fitzgerald follows:] 


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Kristin W. Fitzgerald 

Testimony before the Health Subcommittee of the House Energy and Commerce Committee 

uNCI Cancer Research: Today's Progress; Tomorrow's Challenges" 

March 23, 2010 
67 


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Kristin W. Fitzgerald Testimony Page 1 of 3 

Members of the Subcommittee, I want to thank you for the opportunity to testify at today's 

hearing, "NCI Cancer Research: Today's Progress; Tomorrow's Challenges." 

My name is Kristin Fitzgerald. As a former health staffer for Representatives John Boehner, 

Judy Biggert, and Harris Fawell, I have participated in many Congressional Hearings. This is 

however, my first time on this side of the dais, and I greatly appreCiate the opportunity to speak 

to the challenges facing cancer research. 

I am here today not only on my own behalf, but that of my husband, Ray Fitzgerald. 

Ray was also a Congressional staffer. In fact, he worked for six years as legislative director for 

the Ranking Member of this Commerce Health Subcommittee, Congressman John Shimkus. 

Ray died last January of gastric or stomach cancer. 

Until his diagnosis in May of 2008, Ray was a healthy 36 year old man. He had no risk factors 

for cancer. He had never smoked, drank infrequently and lived a healthy lifestyle. With 12 

aunts and uncles and 72 first cousins in his large Irish family, there were only four unrelated 

incidences of cancer before him. Nothing would ever have put him at high risk of a cancer 

diagnosis. 

Ray's cancer symptom was burping which appeared for a period of two months before his 

cancer was diagnosed. 

When Ray was diagnosed, his cancer was an advanced stage IV. His gastric tumor had spread 

throughout the lining of his stomach and progressed to his esophagus and liver. 

We were told that there was no hope of a cure but that chemotherapy could reduce the cancer 

for a time. 

That time was eight months. 

This is not an abnormal scenario for gastric cancer, it is the second deadliest cancer worldwide. 

It very often presents in Stage IV, and is always incurable at that point. 

Ray however, was forty years younger than the average gastric cancer patient, and thus the 

grim prognosis impacted not just Ray, but myself and our three young daughters, Nora (5), 

Maggie (3) and Lucy (1). 

Members of the Subcommittee, it is my belief that Ray's diagnosis and prognosis is our worst 

cancer nightmare: diagnosis of a deadly cancer at an advanced stage where a cure is impossible. 

It is a death sentence. 
68 


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Kristin W. Fitzgerald Testimony Page 2 of 3 

And if we think it can't or won't happen to us, we are wrong. Ray was you -- or at least, your 

staff. And as I have learned, it could even be happening to one of us right now, and we would 

never know it. 

After Ray died I spent time talking with Ray's doctors to see how this kind of scenario can be 

prevented so that more young dads and moms aren't violently stolen from their families by 

cancer. 

As a former health staffer, I assumed that gastric cancer research was ongoing and would utilize 

Ray's tumor specimen and facts about his age and health status to find a cure for this deadly 

cancer. 

However, far too little is being done to research gastric cancer and other gastrointestinal 

cancers that have a similar deadly prognosis. CBS news analyzed the disparity in research 

dollars in May of 2009. For every cancer death, the most federal research dollars were spent 

on cancer of the cervix ($18,870) and breast ($14,095) and on Hodgkin lymphoma ($12,791). 

The least funded was gastric cancer ($1,168), with esophageal cancer a close third at ($1,542).; 

Gastrointestinal (GI) cancers are some of the deadliest cancers in the U.S. with deaths 

attributed to the digestive system second only to those in the respiratory system. Four out of 

the five lowest five year cancer survival rates for metastatic cancer are GI: Pancreas 1.7%; Liver 

2.8%; Esophagus 2.9% and Stomach 3.4%.'; 

And, gastrointestinal cancers are rising, particularly in young people. Though it is very difficult 

to track current trends in cancers in a timely way because of the slow reporting of Surveillance 

Epidemiology and End Results (SEER) data, a recent NCI article documented the rise in 

gastroesophageal cancers of the stomach and esophagus. The article compared the incidence 

rates in two four year periods, 1975-1979 and 2000-2004. Overall there was a 44 percent 

increase in these cancers. Within gastroesophageal cancers there was an explosion of a 

particular type, adenocarcinoma. The increase in adenocarcinoma was 465 percent, with an 

190 percent increase in young white males.iii Attachment 1 

And the situation for young people with GI cancers is particularly grim. Because GI cancers are 

considered to be diseases of middle or advanced age, the diagnosis of GI cancers in people 

under 40 is often delayed. As a result, the disease is usually in an advanced stage with a poor 

prognosis by the time the diagnosis is established. And their very age works against them as 

the strength and relative health of their bodies is passed on to their cancers making them even 

more aggressive than in older patients. As a result of the delay in diagnosis and the more 

aggressive phenotype of cancers in young people, GI cancers in young people tend to be fatal. 
69 


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Kristin W. Fitzgerald Testimony Page 3 of 3 

Yet, unlike other deadly cancers, gastric cancer and many other gastrointestinal cancers do not 

have a national clinical registry and tissue bank, to utilize tumor tissue and clinical records for 

research purposes. 

In my view, this is intolerable. Congress and the National Cancer Institute can and should do 

more to ensure that researchers can have access to the tools they need to prevent and 

diagnose these cancers before it is too late. 

Though these cancers are growing, they are poor candidates for wide-scale screening programs 

due to the smaller population of people impacted and the invasive nature of screening 

available. 

More research is essential in order to understand the unique characteristics of the disease in 

younger people and develop a screening test based an molecular markers to allaw for earlier 

detection. In order to accomplish this research, the National Cancer Institute must also develop 

a coordinated national GI cancer tissue biorepository, and accompanying research project to 

focus research in this area and make tumor tissue available for research purposes. 

last year the labor, HHS, and Education Appropriations Report included language asking the 

National Cancer Institute to develop a research project and accompanying tissue repository to 

study GI cancers in young people. To date, this has not been accomplished. 

Congress must act to ensure that these cancers can be detected and cured so that more lives 

are not lost. 

Ray was a wonderful man and the legacy of his spirit will live on always. However, it is my 

belief that Congress should fund a research project, tissue bank and registry so that the physical 

legacy of patients like Ray can live an farever, giving eternal gifts to researchers and scientists 

throughout the world. 

Members of the Subcommittee thank you for your time and consideration. I am happy to 

answer any questions. 

, Data compiled and reported by CBS Evening News. May 27, 2009. 

" American Cancer Society. Cancer Facts & Figures 2008. Atlanta: American Cancer Society; 2008. 

", "Incidence of Adenocarcinoma olthe Esophagus Among White Americans by Sex, Stage, and Age," Linda Morris 

Brown, Susan S. Devesa, Wong-Ho Chow, Journal olthe National Cancer Institute 2008;100: 1184-1187. 
70 


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Incidence of Adenocarcinoma of the 

Esophagus Among White Americans by 

Sex, Stage, and Age 

Linda Morris Brown, Susan S. Devesa, Wong~Ho Chow 

Rapid increases in the incidence of adenocarcinoma of the esophagus have been 

reported among white men. We further explored the temporal patterns of this disease among white individuals by sex, stage, and age by use of data from the with adenocarcinoma (R128 men 1398-

Surveillance, Epidemiology, and End Results program. We identified 22759 patients women). Among white men, total agefrom January 1, 1975. through December 31, 2004, with esophageal cancer, of adjusted cancer rates increased 

whom 9526 were diagnosed with adenocarcinoma of the esophagus. Among white 

men, increases in the incidence of esophageal cancer were largely attributed to a 

463% increase in the incidence of adenocarcinoma over this time period, from 1.01 

per 100000 person-years (95°/(1 confidence interval [CI};:; 0.90 to 1.13) in 1975-1979 

to 5.69 per 100000 person-years (95% CI = 5,47 to 5.91) in 2000-2004. A similar rapid 

increase was also apparent among white women, among whom the adenocarcinoma rate increased 335%, from 0.17 (95% CI = 0,13 to 0,21) to 0,74 per 100000 person-years (95% CI == 0.67 to 0.81), over the same time period. Adenocarcinoma rates 

rose among white men and women in all stage and age groups, indicating that 

these increases are real and not an artifact of surveillance, 

J Natl Cancer Inst 2008;100:1184-1187 

Tot,l1 cancer incidence and 

mortality been increasing among 

stable among \\·hite women, 

R,lpid increJ~e<; in the incidence of adenahan' been 

patlerns are in contrast to those for 

mollS cell carcinoma of the 

which rates 11<1\'e been 

blacks than whites ,mel hwe 

underSLmd the 

!loral trends among white lndividwlls by sex, 

tries in the 

Sun'cillance, Epidemiology. and End Results 

(SEER) program (I) were used to calculate 

incidence rates of primary inqlSiyc CSOph'lcancer (fnternatiOJli11 ClrJrsijic"thm of 

Oncology, 3rd coition. 

150-159) for 22759 

1184 BriefCommunicatiQns i JNCI 

the period from 

Decemher 31, 

are located in 

the metropolitan areas (If Atlanta, Detroit, 

San Francisco-Oakhlnd, <lnd Seattle-Puget 

Sound and the states of Connecticut, 

FIawaii. Iowa, :-;e\v .\'1e,ico, and l'tah. All 

SEER Registries 

Standard Registry from the 

North American ;\<;50ciation of Central 

Cancer Regit>tries, Inc, for cOinpleteDess 

(at least 95%), accuracy (<3% of cases 

rates 

lldenocarcinoma hy sex, An rntes were 

expressed pcr 100000 person-years, and 

only data points \\-ith at Jellst 10 observation" were presented. Temporai trends 

ste::ldily from per 100 000 

reus (95% CI '" 5.+9 to 6.03) 

J975~t979 to 8.H per 100000 personyears (95% C1 '" R,08 to 8.60) during 

2000,2004, because of a +63 % 

increase in ,"cnoem,nm'"" rates from 

1.01 (95';{' Cf "" 0.90 to l.lJ) to 5.69 (95% 

CI =: 5..+7 to 5.91) (Table 1 and Fi~..Jre 1). 

The incidence of squamous cel! c2rcinoma 

decreased 50'Yo across the stlldy period, 

from :;.81 per 100000 

(:1;= 3.59 toof.O.'!) in 

100000 

2.03) in 

squamous cell carcinoma and the increases 

in adenocarcinoma, the r::lte for adenocarcinoma amon~ white men surpassed that 

for squamous cell carcinoma around 1490, 

,"\mong white women, total esophageal 

cancer rates remained comtant at around 

the 

time 

29% in squamous cell carcinoma 

from 1.38 per 100000 person-years (95°1" 

Cl '" 1.28 to !. '1"0) during 1975-1979 to 

0.98 per 100000 in 2000-

2004 (9'i% CI '" 

i1lcrease in aaenncarcinnma from O.! 7 

100000 person-years (9'1"% C1 

0.21) in ]075-1979 to 0.74 per 100000 

person~years in 2000--2004 (95';'S Cl '" O.fi7 

to 0.8i). The gap hetween "qmllllous cell 

Correspondence to: Linda Morris Brown, MPH, 

DrPH, RTI International, 6110 Executive Blvd, 

Suite 902, Rockville, MD 20852-3907 (email 

lindabrowni~rti.org). 

See '"Funding" and "Notes" follOWIng "References." 

001: 10.1093Ijnciidjn211 

Published by Oxford University Press 2008 

Vot. 100, Issue 16 I August 20, 2008 

m 

N 

o 

<5 
71 


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CONTEXT AND CAVEATS 

Prior knowledge 

Rapid increases in the incidence of adenocarcinoma of the esophagus have been 

reported among white men. 

Study design 

Registry study in which data from the 

Surveillance, Epidemiology, and End 

Results program from January 1, 1975, 

through December 31, 2004, was used to 

explore the temporal patterns of this djs~ 

ease among white individuals by sex, stage, 

and age. 

Contribution 

Adenocarcinoma incidence rates rose from 

1975 through 2004 among white men .and 

women in all stage and age groups, The 

incidence of adenocarcinoma among white 

men increased 463%, from 1.01 per 100000 

person-years in 1975-1979 to 5.69 per 

100000 person~years in 2000-2004. A similar rapid increase was also apparent among 

white women, with an increased incidence 

of 335'"10 from 0,17 per 100000 person-years 

to 0.74 per 100000 person-years, 

Implications 

Because the increases in incidence are 

independent of stage and age group, they 

appear to be real and not an artifact of 

surveillance. 

Limitations 

Cases of adenocarcinoma may have been 

missed or misclassified. Data for the most 

recent years may have been underreported, 

Pathological diagnoses were not reviewed 

centrally. 

From the Editors 

carcinoma and lldenocarcinoma continued 

to narrow bet'ause rate<; in 2004 were 1.13 

per 100000 (95% CI 0.95 

to LH) and 100000 pcrson-yeJrs 

(95'X, C1 0.68 to 1.02). rcspectlvely. 

disea<;e. There wa~. bowe\(~r, some suggesTable 1. cancer incidence among whites in nine SEER registries, 

1975-1979 2000-2004 

No. of No, of % change 

Characteristic patients Ratet (95% el) patients Ratet (95<>;" cn in ratet ._------------------

76 j5 49 ~o 6.03) 3943 8.34 {8 08 to 8 60) 44.8 

1303 381 (3.59 ~o 4 03) 889 1.90 (L77 to 2.03) -501 

!'l..denocarClnoma 344 101 (090tol.13) 2706 569 {547 to 5.91) 

O:her 3'"id NOS 281 094(083 to 1 06) 348 0.76 (0 68:0084) 

AdenocarCinorna 

65 020 to.1S to 0.25) 692 1.4811 37 to 1.60) 6400 

Regional 101 0.28 1022 "to 0.34) 892 1.86 (1 74 to 1 99) 5643 

Djs~ant 111 031 (075 -:0 0 38) 88'- 1 81 (1 69 to 1 94) 4839 

Unstaged 67 0.22 (0.17 to 0 29) 238 053 i0.47 to 0 60} 1409 

i'l..denocarclnO'T1a 

16 0.17 (0.10 to 0.28) 78 0.50 (0 39 to 0 62) 194.1 

67 1.40 \1 08 1./8) 386 5.05 14 55 to 5.57) 260."1 

55-64 118 2 85 (2.35 ~o 3.42) 684 14.11 (13,0710 1521) 395.1 

65-74 88 383 (3.07 to 4.73) 774 26.27 (2445 to 28.19) 5859 

>75 58 4.43 (3.36 to 5 75\ 784 31 33 (29.17 ~o 33 SOl 607.2 

White females 

Tot,,; esophag:;s 855 1.93 (1.80 to 2 07) 1227 200 (189 to 2.12) 36 

622 1.38 (1.28 to 1.50) 589 0.98 (O 90 to 1 06) -290 

73 017 (0 13 ~o 0.21) 454 0.74 (0 67 to 0 31) 3353 

O(>:ef and \lOS 160 0.38 10.32 to C.M) 184 029 (O 25 to 0 33) 

,L\denOCarGInOMa 

18 0.04 (O 02 to 0.07) 135 021 (0.18 toO 25) 425,0 

13 0.03 (002 to 0 05) 131 022 iO.18 to 0 26) 6333 

25 0.06 (0 C4 to 0.09) 129 0221018 to 0.26) 2667 

Unstageo 17 0.04 W 02 to 0 06) 59 009\o07to011) 1250 

/\denocarClnoMa 

by age, Y 

0.10(006,00,16) 

45--54 0.47 (0 33 ~o 0 65) 

55-64 028iO.14to048) 1 57 (1 24 ~o 1 95) 460.7 

65-74 

,vas ob:.en'ed I~)r \\ omen, although have been missed or mischssifled, hut this is 

rat.::s were much lower for each stage 

(ranging from 0.06 or less per 100000 perin 1975-1979 to 0.21",0.22 per 

2000 ·20CH). 

decades studied, rhe 

increa~es in :ldenocarcinoma occurred 

the upward trends in adenocarcinonu Illay 

be cyen more than wh,lt we 

have rep()rted. diaplO:;es 

the lIlicro~ 

tion that the rate of increase may be slow- This study h.ls sc\·crallimitatjons. Cases 

distinction het\\een ,idenocarcinOlI1<l 

squamous cell carcinoma has been 

for decades. Furthermore, 

ing, especially for lOGllized disease ;\ simibr of adenocarcinoma of the esophagus may in dle histological t)Te category 

jnci.oxfordjournals.org JNCI i Brief Communications 1185 
72 


erowe on DSK2VPTVN1PROD with Insert offset folio 78 here 76020A.035

White Ma!es White Females 

"" 10 Esophaseal Cancer 

t by Histology 

~ ....... Total 

---0- AdenocarCinoma 

f ~. 

~ ~ Unknown 

I , 

~ 

11. 

0,1 0,,1 

~ 1980 1990 2000 198019902000 

10

1 

10

1 Adanocatcinoma 

by Stage 

~ ....... locahzed 

I I -D- Regional _____ Distan\ .J; ~UnknoWIl 

~ 

11. 

g 

§ 

~ 

"L~ ! 0,1 

198019902000 198019902000 

4°1 40 Adenocarcinoma 

"/; ~ 

"'75+ 

-0-65-74 

........ 55-64 

~ 10 -v-- 45-54 

~ --e-- 25-44 

~ l / Q 

g 

~ 

":~ 

. 

~ 

,; 

0: 

01 

196019902000 1960 19902000 

--Year of dlagnasis-------------

1186 Brief Communications 1 JNCI 

noses were modest ;1nd CDuld not exphlin the 

observed increases in adenocarcinoma. 

and women. This increase '\-vas not dear in 

earlier reports (3- 6) because of the rarity of 

ease, indicating that the overall increase in 

adenocarcinomll incidence " unlikely to 

reflect heightened surveilbnce and earlier 

diagnosis. The increases appeared across all 

0 

0 

~ 

I 

3 

3 

~ 

~ 0 

z, 

~ ~ 

'" " C3 

~ 

;: 

0 

~ ~ 

Barrett g 

be related to the higher I 

0 

pressure {'l 

pre\<11ence of ~ 

ease in ohese g 

presented (6) (bta [ 

'" for adenocarCInoma. ,\1so, increl1ses in ooe- '" by and the lJ1iJ~1970s :: 

0 

appear to parallel increase.;; in adenoc1r,jnoma (15). and recent data (16) indicllte 

abdominal 

that the increase in adenocarcinoma is reill 

Vol. 100, Issue 16 : August 20, 2008 
73 


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~OO-, b,\~td on the November 2006 17. Kubn 

B. SFER'StM: SUITcillance Rese,lrth 

References 

235-:56, 

7, Survcil1'l:1('C, Epidemiology, and Fnrl R('~u![S 15. 

16. 

jnci.oxfordjourna1s.org 

~ational Ca;Kcr Institute SEFR'Stat 50!rof t~\e A3tional 

Cancer In'ititllt~'. 

and 

JNCI I Brief Communications 1187 
74 

Mr. PALLONE. Thank you so much. 

Ms. Don. 

STATEMENT OF MEGAN GORDON DON 

Ms. DON. Mr. Chairman and members of the subcommittee, my 

name is Megan Gordon Don and I am here on behalf of the Pancreatic Cancer Action Network as well as the Deadly Cancer Coalition. I appreciate the opportunity to testify today regarding deadly 

cancers and the state of cancer research. 

I am going to give you some background on the research problems associated with deadly cancers and why the Deadly Cancer 

Coalition was formed as well as provide you with four key recommendations for the committee’s consideration. While a number 

of cancers have achieved 5-year survival rates of over 80 percent 

since passage of the National Cancer Act in 1971, and the average 

5-year survival rate for all cancers has increased during that time 

from 50 percent to 66 percent, significant challenges still remain 

for other types of cancer. In fact, there are eight major cancers that 

have yet to reach the 1971 benchmark. Nearly half of the over 

562,000 cancer deaths in 2009 were caused by these same eight 

forms of cancer, which are ovarian, brain, myeloma, stomach, 

esophageal, lung, liver and pancreatic. I have provided for the 

record a fact sheet on these cancers, which we refer to as the deadly cancers. 

As grim as the statistics are now for deadly cancers, the future 

looks even worse. According to an article in the June 2009 edition 

of the Journal of Clinical Oncology, cancer incidence is not only 

projected to dramatically increase in the next 20 years, but some 

of the deadliest cancers will be among those of the greatest relative 

increase in incidence. 

In contrast to many of the other cancers with significantly better 

survival rates, research into the deadly cancers has been relatively 

underfunded, and as a result, these cancers have no early detection 

or treatment tools or the available treatments are still considered 

controversial. While there has been some work through the TCGA, 

as Dr. Barker highlighted, for three of the deadly cancers, and 

while NCI has expressed interest in expanding TCGA to more 

deadly cancers, biomarkers have yet to be identified or validated 

for the majority. Also, survival for these cancers is measured in 

weeks and months, not years. 

Research into deadly cancers has also faced many hurdles including low priority status, little accountability, low average funding, 

little understanding of the research complexities by grant reviewers, deadly research grants are rarely reviewed by experts in the 

field, and a shortage of available tissue. 

Our coalition started with the premise that all cancer patients 

deserve at least a 50–50 chance of survival. And, at a minimum, 

survival from all types of cancers should be above the starting line 

that was established nearly 40 years ago. The fact that there are 

a set of cancers that have not reached that starting line, in most 

cases are not even close, indicates that the time has come to establish a targeted effort to focus on the greatest challenges with the 

greatest need: deadly cancers. These challenges are not insurmountable but it will take leadership, vision and a change in the 


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75 

current research paradigm at NCI. Specifically, we are calling for 

an increase in funding, the creation of a targeted deadly cancer 

program to provide structure and accountability, a dedicated grant 

program, and expert review of grants. 

I want to note that our recommendations are not about telling 

NCI how to do the science. We are calling for an increase in funding because the data clearly shows that deadly cancers are currently not research priorities at NCI. 

I would like to call the subcommittee’s attention to two charts 

that I have provided for the record. The first shows NCI funding 

for the top five cancer killers. Lung and pancreatic are two of the 

most deadly, and they also have the lowest funding levels. Looking 

at the survival rates, you also see that survival is the lowest. This 

chart demonstrates in very dramatic fashion that there is a clear 

correlation between low investment in research and poor survival 

rates, and vice versa. The second chart shows that less than 18 

percent of NCI’s funding has gone to the deadly cancers, and yet 

over half of the cancer deaths are caused by the eight deadly cancers. Across all types of cancer combined, the NCI spent just over 

$7,000 per cancer death in 2008. For the deadly cancers, NCI devoted only about $2,500. Taken together, these charts clearly demonstrate that the status quo is not working for the deadly cancers 

and some sort of targeted funding is needed. 

Mr. Chairman, creating structure and accountability is absolutely essential to making progress. Therefore, a second recommendation is to create a targeted cancers program that includes 

strong accountability measures such as requiring the NCI director 

to develop a strategic plan to increase survival rates for the deadly 

cancers. Furthermore, we call for annual reports to Congress showing progress against the strategic plan to further ensure accountability. 

Our third recommendation is to establish a new targeted grants 

program to create a protected pool of research funds for the deadliest cancers. This additional grant opportunity will help to compensate for the limited existing data on deadly cancers which put 

these grant applications at a competitive disadvantage with the 

better researched cancers and allow these grants to be evaluated 

in a way that would foster more funding opportunities for both experienced and young investigators in order to attract more scientists to this field of study. Grant review committees would include scientific experts in the specific disease areas of interest, another critical point for deadly cancers in our fourth and last recommendation. We have presented the idea for a targeted cancers 

program to the NCI and have also been working with the House 

and Senate sponsors of the ALERT Act. 

Chairman Pallone, on behalf of the Deadly Cancer Coalition, I 

would like to commend you and Representative Capps for your 

work on the ALERT Act. It captures many of the recommendations 

outlined above. Additionally for the pancreatic cancer community, 

passage of H.R. 745, the Pancreatic Cancer Research and Education Act, introduced by Representatives Anna Eshoo and Ginny 

Brown-Waite, is another important step to tackling the changes I 

have discussed. 


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Mr. Chairman, in conclusion, I want to thank you and members 

of the subcommittee again for allowing me the time to testify. The 

Deadly Cancer Coalition believes that the time has come to create 

a new research paradigm at NCI that will lead to the institute 

tackling the hardest and most complex problems. It is by solving 

the hardest problems that we will likely see the greatest rewards 

for the entire field of cancer research. I am happy to answer any 

questions. 

[The prepared statement of Ms. Don follows:] 


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Mr. Chairman and members of the Subcommittee: 

My name is Megan Gordon Don; I am here on behalf of the Pancreatic Cancer Action Network 

as well as the Deadly Cancer Coalition, a coalition of organizations that represent high mortality 

cancers, defined as those with five-year survival rates below 50 percent. I appreciate the 

opportunity to testify today regarding deadly cancers and the state of cancer research. 

I'm going to give you some background on the research problems associated with deadly cancers 

and why the Deadly Cancer Coalition was formed as well as provide you with four key 

recommendations for the Committee's consideration. 

I would like to first provide some background on what are best described as the deadliest 

cancers. While a number of cancers have achieved five-year survival rates of over SO 

percent since passage of the National Cancer Act in 1971, and the average five-year 

survival rate for all cancers has increased during that time from 50 percent to 66 percent, 

significant challenges still remain for other types of cancers, particularly the most deadly 

forms of cancer. In fact, nearly half of the 562,340 cancer deaths in 2009 were caused by 

eight forms of cancer with five-year relative survival rates of less than 50 percent: ovary 

(45.5%), brain (35.0%), myeloma (34.9%), stomach (24.7%), esophagus (l5.S%),lung 

(IS.2%),liver (11.7%), and pancreas (5.1 %). I have provided forthe record a facl sheet on 

the deadliest cancers. 

It is no coincidence that cancers with significantly better five year survival rates, such as breast, 

prostate, colon, testicular, and chronic myelogenous leukemia. also have effective treatment 

options in some cases, several and/or early detection tools thanks to research programs 

championed and supported by Congress. By contrast, research into the cancers with the lowest 

five-year survival rates has been relatively under-funded, and as a result, these cancers have no 

early detection or treatment tools. Available treatment protocols for many of the deadly cancers 

are still considered controversial. In further contrast, while there has been some work through 

The Cancer Genome Atlas (TCGA) for lung, brain, and ovarian cancer, which arc three deadly 

cancers, biomarkers have yet to be identified or validated for the majority of deadly cancers. 

Also, survival for these cancers is measured in weeks and months, rather than years. 

As grim as the statistics are now for the deadly cancers, the future looks even bleaker. 

According to an article in the June 2009 edition of the Journal of Clinical Oncology, cancer 

incidence is not only projected to dramatically increase in the next 20 years, but "certain cancer 

sites with particularly high mortality rates. such as liver, stomach, pancreas, and lung, will be 

among those with the greatest relative increase in incidence." In fact, the article projected that 

lung cancer incidence would increase by 52 percent, pancreatic cancer would increase by 55 

percent, liver cancer would increase by 59 percent and stomach cancer would increase by 67 

percent. 

Our coalition started with the premise that all cancer patients deserve at least a 50-50 chance of 

survival. And, at a minimum, survival from all types of cancers should be above the starting line 

that was established 30 years ago when the overall cancer survival rate was 50 percent. The fact 

remains that there are a number of cancers, which make up nearly half of all cancer deaths 

Pancreatic Cancer Action Network 

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annually, that have not yet reached that starting line and in most cases are not even close. This 

shortcoming indicates that thc time has come to establish a targeted effort to focus on the greatest 

challenges with the greatest need: [he high mortality cancers. 

Research into high mortality cancers has faced many hurdles including: low priority status, little 

accountability, below average funding. little understanding of the research complexities by grant 

reviewers (high m0l1ality research grants are rarely reviewed by experts in that field), and a 

shortage of available tissue for research caused by the complexities of the diseases. To help you 

better understand what these hurdles have actually meant in the fight (0 increase survival for 

patients diagnosed with one of the deadliest cancers. I would like to use pancreatic cancer as an 

example. With a five year survival rate of jus I 5 percent, it is the deadliest of the deadly cancers. 

It is estimated that over 42.000 Americans were diagnosed with pancreatic cancer in 2009 -- a 12 

percent increase over the year before. In the past few years there has been increased pUblicity of 

this deadly disease with the deaths of Patrick Swayze, the actor, Dr. Randy Pausch. a computer 

science professor at Carnegie Mellon University and author of the widely acclaimed "Last 

Lecture", as well as the diagnosis of U.S. Supreme Coun Justice Ruth Bader Ginsberg. 

But while these prominent individuals' diagnoses have increased national awareness, the fact 

remains that a pancreatic cancer patient diagnosed today has roughly the same chance of survival 

as someone diagnosed 30 years ago. Today, 95 percent of pancreatic cancer patients die within 

five years of diagnosis. Seventy-six (76) percent die within the first year after diagnosis. There 

are still no early detection tools or effective treatments. Just as it has been for decades, the 

majority of patients diagnosed with pancreatic cancer hear that they should get their final affairs 

in order. instead of hearing about treatment options to help them see another birthday, wedding 

anniversary. or child's graduation. 

Admittedly, pancreatic cancer is a particularly challenging disease to research: 

• Pancreas tissue is very difficult to obtain for research. The pancreas is located deep 

within the body. Therefore, most tissue samples are obtained only if a patient has surgery 

to remove the tumor. However. because the majority of pancreatic cancers are caught 

very late, only 15 percent of all pancreatic cancer patients are eligible for this surgery. 

Further, even if tissue is obtained at the time of surgery. the tissue sample is usually 

small, making this resource extremely valuable and scarce. 

• Pancreatic tumors arc unique in the types of cells that makc up the tumor. Tumors are 

often comprised of a variety of cell types, including dense fibrotic cells that may 

contribute to the remarkable resistance of the tumor to chemotherapies. 

• Participation in clinical trials is often limited because patients are extremely sick and die 

quickly of the disease. 

• Currently. there are no biomarkers sensitive and specific enough to be useful in the 

diagnosis of pancreatic cancer. 

It should be noted that at this point studies have demonstrated that individual pancreatic cancer 

patients respond differently to various treatments. Therefore. personalized medicine holds great 

promise for people facing pancreatic cancer. However, as is the case for many of the deadly 

Pancreatic Cancer Action Network 

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cancers, much more research is needed to understand these differences, as well as a system to 

share data and analyze similarities and differences amongst individual patients. 

These challenges in pancreatic cancer are not insurmountable. But, as with research into all of 

the deadly cancers, it will take leadership, vision and a change in the current research paradigm 

at NCr. Specifically, we are calling for an increase in fnnding, the creation of a targeted cancers 

program focused on deadly cancers to provide structure and accountability on making progress 

on these diseases, a dedicated grant program, and expert review of grants. 

I want to note that our recommendations are not about telling NCr how to do the science. We 

simply believe that the slatus quo has not worked for deadly cancers and the time has come to 

take specific steps to ensure that there is sufficient focus on the deadliest cancers to ensure true 

progress. 

Deadly cancers, like pancreatic cancer, are currently not research priorities at NCr and as a 

consequence they are severely underfunded. For example, pancrcatic cancer currently receives 

less than 2 percent of NCT's nearly $5 billion budget - a figure much too low to foster any 

significant progress against this leading cancer killer. I have included for the record a chart of 

NCr funding for the top five cancer killers which includes both lung and pancreatic cancer, 

also two of the most deadly cancers and their respective survival rates. This chart 

demonstrates in very dramatic fashion that there is a clear correlation between low investment in 

research and poor survival rates. When an investment has been made, the five year survival rates 

reflect those efforts. 

Funding is obviously an important part of the problem. NCI's budget has declined by nearly 

$700 million, or IS percent since fiscal year 2003, after adjusting for inflation. More 

importantly, NCI has not made the deadliest cancers a funding priority. As indicated in the fact 

sheet on deadly cancers r have included for the record, less than 18 percent of the NCr s 2008 

research funding budget was dedicated to the eight deadly cancers even though these cancers 

cause half of all cancer deaths. Across all types of cancer combined, the NCr spent just over 

$7,000 per cancer death in 2008. For the eight highest-mortality cancers, NCI devoted only 

about $2,500. 

r do want to note that Dr. Barker has reached out to the Deadly Cancer Coalition about 

expanding TCGA to more of the deadly cancers. We are vcry interested in having TCGA 

address more of these cancers and, specifically, the most complex problems such as improving 

tissue collection methods when tissue is particularly scarce. However, while efforts like TCGA 

and nanotechnology are important parts of the solution for deadly cancers, these efforts on their 

own are insufficient. Targeted funding for research into deadly cancers also will be critical, but 

again, on its own, is not enough. Mr. Chairman, we believe that creating structure and 

accountability also is absolutely essential to making progress in these diseases. Specifically, the 

deadly cancer community recommends establishing a targeted cancers program within the NCr 

for the high mortality cancers. It should include a strategic plan for progress, an annual repot1 

from Ncr to Congress, and a new grant program specifically focused on the deadly cancers. 

Pancreatic Cancer Action Network 

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The targeted cancers program would require the NCI Director to work with staff, top scientists, 

patient advocates and other stakeholders to develop a plan of research activities necessary to 

increase survival rates for the high mortality cancers. The strategic plan would identify the steps 

required to reduce mortality rates for each cancer over a five year period. The plan would 

include specific areas of research needed, as well as an estimated budget that can be factored into 

the NCr's Professional Judgment Budget. It would also include NCI-wide initiatives, such as 

nanotechnology and TCGA, to help ensure that all deadly cancers are included in these types of 

programs. The strategic plan will also have the added benefit of helping the deadly cancer 

research community ensure that privately funded research is not duplicating federally funded 

research. Furthermore, we believe that annual reports on the new program are necessary to 

ensure accountability. Reports should indicate progress that has been made against the plan in 

the previous year, changes in survival rates, and newly available early detection tools or 

treatments. 

We also recommend the establishment of a new targeted grants program to create a protected 

pool of research funds for the deadliest cancers. Researchers studying deadly cancers often have 

relatively limited initial data, due in pan to the historical lack of research into these cancers. In 

the NCr competitive application process, grant applications with limited initial data tend to be 

less competitive versus applications regarding more researched cancers, such as breast and 

prostate. Limited data reflects high-risk/high-reward research and the NCr tends to fund "safe 

bets" While researchers studying the deadly cancers would be encouraged to continue to submit 

proposals through standard grant mechanisms, they would also have the opportunity to submit 

grant applications under the targeted cancers program. This additional opportunity would help to 

compensate for the limited existing data in deadly cancers. For example, basic research 

investigating the biology and progression of some of these historically under-studied cancers 

may be considered high risk/high reward due to lack of research data available and would not 

receive funding. However, it is clear that the basic biology and progression of the disease are 

essential building blocks of knowledge, critical to moving these fields forward. With a targeted 

cancer program, these grants could be evaluated in a different way and would have greater 

opportunities to get funded. 

Additionally, grants under this program could include a directed portion, similar to the challenge 

grant process the NIH used to administer ARRA funding, in order to meet the goals of the 

strategic plan discussed above. Funding would be open both to experienced investigators and to 

early-career investigators to attract more scientists to this field of study. Grant review 

committees would include scientific experts in the specific disease areas of interest, another 

critical point for deadly cancers. 

We have presented the idea for a targeted cancers program to the NCr and have also been 

working with the House and Senate sponsors of the 21 51 Century Cancer Access to Life-Saving 

Early detection, Research and Treatment (ALERT) Act. Chairman Pallone, on behalf of the 

deadly cancer community, I would like to commend you and Representative Capps for your 

leadership in drafting a House version of this bill that would establish a targeted cancer research 

program and many of the ideas outlined above. 

Pancreatic Cancer Action Network 

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Additionally. for the pancreatic cancer community. passage of HR 745. the Pancreatic Cancer 

Research and Education Act. introduced by Representatives Anna Eshoo and Ginny BrownWaite. is another important step to tackling the challenges I have discussed. Specifically. this 

bill would put in place a strategic plan for pancreatic cancer research and establish a cancer 

research incubator pilot project for the highest mortality cancers. The bill would also strengthen 

and expand Centers of Excellence for pancreatic cancer and promote awareness of the disease 

amongst health professionals and the public. 

The time has come to tackle the hardest and most complex problems. We must fund new 

progress and give researchers the opportunity to do more with more, not less. It is by solving the 

hardest problems that we will likely see the greatest rewards for the entire field of cancer 

research. We have seen greatly reduced mortality rates of diseases like breast cancer, prostate 

cancer, AIDS and childhood leukemia as a result of targeted. comprehensive and well-funded 

programs. These research programs have produced early detection tools and effective treatments 

for these cancers. We must also shine a bright light on the deadliest cancers to achieve these 

same results. 

Mr. Chainnan, in conclusion. I want to thank you and members of the subcommittee again for 

allowing me the time to testify. Creating a targeted research program for the deadly cancers that 

includes a strategic plan and a dedicated grants program reviewed by scientific experts in the 

respective fields. is a critical first step toward reducing the mortality rates and developing early 

detection tools and treatments for the deadliest cancers. 

The Pancreatic Cancer Action Network, along with the Deadly Cancer Coalition, hopes that in 

the near future a diagnosis of ovarian. brain. myeloma, stomach. esophageal, lung. liver, or 

pancreas cancer does not carry an automatic death sentence, but rather the first step in effectively 

treating and ultimately curing the disease. With your help. we know this hope can be a reality 

for all cancer patients. 

In addition to the Pancreatic Cancer Action Network. this testimony is endorsed by the following 

organizations: 

American Association for the Study of Liver Diseases 

American College of Gastroenterology 

American Gastroenterological Association 

American Liver foundation 

American Pancreatic Association 

Digestive Disease National Coalition 

Esophageal Cancer Action Network (ECAN) 

Hepatitis B Foundation 

Hepatitis Foundation International 

International Myeloma foundation 

Leukemia & Lymphoma Society 

Lung Cancer Alliance 

National Brain Tumor Society 

National Ovarian Cancer Coalitioll 

National Pancreas Foundation 

Pancreatic Cancer Action Network 

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Ovarian Cancer National Alliance 

Sarcoma Foundation of America 

Society of Gynecologic Oncologists 

Attachments: 

The Deadly Cancer Fact Shect 

Top 5 Canccr Killers Chart 

Pancreatic Cancer Action Net work 

p. 60f6 
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The Deadliest Cancers 

Nearly half of the 562,340 cancer deaths expected in 2009 will be caused by eight forms 

of cancer with five-year relative survival rates of less than 50%: ovary (45.5%), brain 

(35.0%), myeloma (34.9%), stomach (24.7%)' esophagus (15.8%). lung (15.2%). liver 

(11. 7%), and pancreas (5.1%). 

2009 Total Cancer Deaths 

562,340 

l'erc"ntageof2009carn;erDea\II, 

eau.edbV£ightH~l>e~l·M(lr!;ahtycan<er. 

2008 Total Cancer Funding 

$4,000,422,516 

Percentag""fNOfundedPortfol;';' 

Ded1<:~ted T"w~rd$ 8ght Highest-Mortality Qoneers 

<omb,~ed, 

$3,2S8,178,QS5 

Most Common Causes of Cancer Incidence, Deaths, and Five-year Relative Survival Rates 2009 

Deadliest 

Cancers: 

cancers for which 

the jive year 

survival rate is 

less than 50 

percent 

r-- Pancreas 

Liver 

Lung 

Esophagus 

Stomach 

Myeloma 

Brain 

Ovary 

Leukemia 

Colon 

Non-Hodgkin 

Kidney 

~' . Urinary Bladder 

Uterine Corpus 

Breast 

Melanoma of the Skin 

Prostate 

2009 Incidence 2009 Deaths five-year. 

Relative 

Survival R'ate 

42,470 35,240 5.1% 

22,620 18,160 11.7% 

219,440 159,390 15.2% 

16,470 14,530 15.8% 

21,130 10,620 24.7% 

20,580 10,580 I 34.9% 

22,070 12,920 35.0% 

21,550 14,600 45.5% 

44,790 21,870 50.0% 

106,100 49,920 64.4% 

65,980 19,500 64.5% 

57,760 12,980 66.5% 

70,980 14,330 79.8% 

42,160 7,780 I 82.9% 

194,280 40,610 I 88.7% 

68,720 8,650 91.2% 

192,280 27,360 98.9% 

Insert offset folio 90 here 76020A.044erowe on DSK2VPTVN1PROD with 

Pancreatic ca

is the 4th lead

cancer killer 

continues to 

least funded 

among the to

cancer killers
85 


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86 

Mr. PALLONE. Thank you. 

Dr. DiPaola. 

STATEMENT OF ROBERT S. DIPAOLA 

Dr. DIPAOLA. Good afternoon, Mr. Chairman and members of the 

subcommittee. My name is Dr. Robert DiPaola. I am director of the 

Cancer Institute in New Jersey, our State’s only NCI-designated 

comprehensive cancer center. The mission of CINJ, similar to 65 

other NCI-designated centers nationwide, is to reduce cancer incidence, morbidity and mortality through multidisciplinary cancer research including researchers in the laboratory collaborating with 

physician researchers in the clinic. I am also a member of the 

American Association of Cancer Research, AACR, which is dedicated to advancing cancer research, to prevent and cure cancer 

through research, education, communication and collaboration. 

Thank you for convening this hearing and recognizing that cancer 

research is critical to making and translating the discoveries needed to reduce the toll that cancer takes on the people and the economy of our Nation. Through its oversight and legislative activities, 

this committee has played an important role in advancing cancer 

research, and I commend Chairman Pallone and all the members 

of the committee for their achievements and ongoing commitment 

to this national priority. 

Today we estimate, as you have heard, one in two men and one 

in three women will develop cancer in their lifetimes. This year 

alone, almost 1.5 million Americans will be diagnosed with cancer 

and more than half a million Americans are expected to die of the 

disease. That is approximately 1,500 people a day, one per minute. 

The toll on the economy is staggering and predicted to increase 

with the increased risk of cancer to our aging Baby Boomer population if there is not dramatic intervention supporting the need to 

increase the investment in cancer research. 

The Nation’s prior investment in cancer research is reaping benefits to millions of Americans. According to the ACS, as you just 

heard, the 5-year survival rate for cancer has improved. The overall 5-year survival rate improved to approximately 66 percent compared to 50 percent in earlier years but we now need to go further, 

especially for rare and aggressive cancers. An example of a major 

advance that is providing for accelerated progress is the sequencing 

of the human genome and The Cancer Genome Atlas, as you just 

heard from Dr. Barker, which is now allowing us to answer difficult questions more rapidly. Research to improve diagnosis, treatment and prevention of cancer can improve patient outcome. 

Currently, only approximately 5 to 10 percent of drugs that are 

first tested in cancer clinical trials are ultimately approved. We 

now have the models to improve this rate including a better understanding of molecular pathways that allow a more targeted and individualized approach. Much of the progress made in this country 

against cancer has been the result of organizations such as the 

AACR and of research in cancer care done at NCI-designated cancer centers. A culture of collaboration is also a hallmark of NCIdesignated cancer centers in which collaboration between laboratory and clinical researchers and collaboration with other research 

institutions, industry and other cancer centers is encouraged. 


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At CINJ, as an example, with NCI’s support, we have been able 

to foster a consortium model with researchers at multiple institutions in the State including the best researchers at CINJ, Rutgers 

and Princeton universities. These efforts recently have led to the 

discovery of critical metabolic pathways involved in tumor cell starvation and survival and translated these laboratory findings into 

several clinical trials that are now ongoing and available for patients with both common and more rare cancers that are attempting to better starve tumor cells. 

Another area of funded research important to improve outcomes 

for patients is comparative effectiveness research which seeks to 

optimize emerging and existing therapies. For example, a team of 

researchers in epidemiology recently published a landmark study 

that better defines the use of hormone therapy for men with early 

stages of prostate cancer. Other novel therapeutic trials with NCI 

support include a recently opened clinical trial to look at harnessing our immune system to tackle pancreatic cancer and other 

cancers, in this case administering in pancreatic cancer a new vaccine combination directly into the tumor, now offering many patients in need a new clinical trial option. 

Other efforts to foster collaboration include the work of the 

AACR that underpins many efforts and groups such as Stand Up 

To Cancer. This new initiative provides large grants awarded to 

multidisciplinary research dream teams. 

Cancer’s economic burden is staggering. The NIH estimates that in 2008 the overall cost to society was more than $200 billion. Fortunately, we are at a most promising time in cancer research, as 

you have heard, but much more remains to be done. I think we 

have the potential to accelerate through this tipping point in time 

by supporting a new era of cancer treatment and prevention and 

gain on our investment to reduce the toll of cancer on the people 

and the economy of our Nation. Thank you. 

[The prepared statement of Dr. DiPaola follows:] 


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ROllfRT WOOD JOHNSON 

MEDICAL SCHOOL 

The Cancer 

Institute of 

New JerseT Brillgin,~ 1\'SL'dr~h 10 liJ~: 

Testimony before the House Committee ou Energy and 

Commerce Subcommittee on Health - March 23, 2010 

Robert S. DiPaola, MD 

Director, The Cancer Institute of New Jersey and 

Member, American Association for Cancer Research 

NCI CCC 

-

Good morning Mr. Chairn1an and Members of the Subcommittee. My name is Dr. Robert 

DiPaola. and [am the Director of The Cancer Institute of New Jersey (CINJ), our State's only 

National Cancer Institute (NCI)-designated Comprehensive Cancer Center at UMDNJ-Robert 

Wood .!ohnson Medical School. The mission of CIN.!, similar to the 65 other NCI-designated 

Centers Nationwide, is to reduce cancer incidence, morbidity, and mortality through multidisciplinary cancer research. I am also a member of the American Assoeiation for Cancer 

Research (AACR). The mission of AACR, as the world's oldest professional organization 

dedicated to advancing cancer research, is to prevent and cure cancer through research, 

education, communication, and collaboration. 

Thank you for convening this hearing and recognizing that cancer research is critical to making 

and translating the discoveries needed to reduce the toll that canccr takes directly on the people 

and the economy of our Nation. Through its oversight and legislative activities, this Committee 

has played 8n important role in advancing cancer research at the national and local levels and I 

commend Chairman Pallone and all of the Members of the Committee for their achievements 

and ongoing commitment to this national priority. 

Today, we estimate that one in two men and one in three women will develop cancer in their 

lifetimes. This year alone almost 1.5 million Americans will be diagnosed with cancer and more 

than half a million Americans are expected to die of the disease that's approximately 1,500 

people a day, and one per minute, In fact, in the United States. cancer accounts for nearly one of 

every four deaths (American Cancer Society, Cancer Facts and Figures 2009). The toll on the 

195 Little Albany Street· Ne\v Bruns\vick. Ne\v Jersey 08903-2681 -Phone 732-235-2465· www.cinj.org 
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2 

economy is also staggering and predicted to increase if there is not dramatic intervention, 

supporting the need to increase investment in cancer research. Compounding these concerns is 

the increased risk of cancer to our aging "baby boomer" population. confinning our need to 

increase the investment in preventive and therapeutic research, which has the promise to 

decrease health care spending by effectively treating and preventing cancer before the disease 

becomes advanced and more costly. 

Successes and Challenges 

The Nation's investment in cancer research is reaping substantial benefits for millions of 

Americans. According to the American Cancer Society (ACS) (ACS, Surveillance and Health 

Policy Research, 2009), the five-year survival rate for cancer has improved. In a comparison of 

the time period 1996 to 2004 with the period 1975 to 1977, the overall five-year survival rate has 

improved from 50% to 66%. Accordingly, the five-year estimated survival rate for many of the 

most common types of cancer have improved in the same time frame: breast 75% to 89%, 

prostate 69% to 99%, and colon 52% to 65%. Some less common cancers have also improved, 

but remain at low rates of five-year survival. For example, Pancreas cancer has a 5% five-year 

survival compared to 3% in the past and liver cancer 11 % versus 4% in tbe past, according to the 

ACS Cancer Facts and Figures 2009. Therefore, despite substantial improvements, we need to 

go further, and we now have additional technology to move research forward at a faster pace. A 

major advance that is providing for accelerated progress is the sequencing of the human genome, 

which now is allowing us to answer difficult questions more rapidly. According to the NCI 

FY2011 annual plan and budget report, "what once took tens of millions of dollars and years to 

accomplish can now be done in about a week for $10,000 or less, thanks to next-generation 

sequencing technology." To capitalize on the remarkable scientific and teclmical advances, 

however, we need to continue to invest. We need to reinforce our anny of researchers who have 

already accelerated our ability to translate discoveries into results for patients, through research 

in diagnosis, treatment and prevention of cancer. 

Research to improve diagnosis of cancer at an earlier stage of disease improves our opportunity 

to have an impact on healthcare. It has been long known that the chance for a patient to be cured 

when diagnosed in early stages of cancer is far greater than tbe rate of cure [or patients 
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3 

diagnosed with more advanced stages of disease. Building on recent research discoveries 

identifying biomarkers of early disease, the opportunity to translate to effective new means of 

diagnosis is better than ever before. A major challenge, however, is limited funding for the 

clinical trials necessary to validate these approaches. Another challenge is the integration of 

many disciplines, including basic laboratory research, clinical research, and efforts in 

information technology to align specific genetic and molecular findings with clinical outcomes to 

accelerate high impact discoveries. Therefore, it will be important to increase etTorts in 

fundamental basic research to continue to discover genetic and molecular abnonnalities, efforts 

in translational research to move those discoveries to patients, efforts in collaborative research to 

combine many institutions to complete large clinical studies, and the integration of infonllation 

technology to catalyze research efforts by providing needed data for researchers. 

[n regard to treatment, we are beginning to more effectively translate scientific discoveries 

directly to new drugs for patients. There are a panoply of new anti-cancer drugs being studied in 

clinical trials, but very few will be approved without etTorts to optimize our approach. In fact, 

approximately 5-10% of drugs that are first tested in cancer clinical trials are ultimately approved 

by the US Food and Drug Administration. We now have new models to improve this success 

rate, all of which can be enhanced with increased research funding. First. we have improved the 

success of drug development by bettcr understanding molecular pathways that allow drug 

developers to take a more targeted approach. This has led to the succcss of the drug imatinib, 

which has rcyolutionized the treatment of chronic myeloid leukemia by targeting and inhibiting a 

specific molecular pathway responsible for this cancer. Second, we have improved the 

assessment of drug concentrations in the blood and the effect on cancer cells in clinical trials 

through better measurements, imaging, and new clinical trial designs. Third, we are improving 

the ability to individualize therapies, thereby improving results for patients, through molecular 

characterization of cancer cells. Fourth, we now have an improved our understanding of not only 

what makes a cancer cell grow and spread, but how a cancer cell survives drugs, radiation, and 

other stresses, and how to use this infonnation to improve the effectiveness of our therapies. 

Efforts to reduce the toll of cancer on the people of our Nation could be maximized by a greater 

investment in prevention. Efforts to develop preventive agents have been successful, including 
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4 

the approval of agents for prevention of breast cancer and vaccination to prevent liver and 

cervical cancer, with high impact by reducing cancer and the associated economic challenges. 

With continued support. further impact is possible with therapeutic research possibilities, 

increased efforts in behavioral research to identify methods to change lifestyles. and the potential 

for beller identification of high-risk populations through advances in genomic technology for a 

more personalized approach. 

The Importance of Collaboration and NCI-Designated Cancer Centers 

Much of the progress made in this country against cancer has been the result of research and 

cancer care done at NCI-designated Cancer Centers. of which 65 today are scattered throughout 

the United States. The NCI-designated Cancer Centers like the Cancer Institute of New Jersey 

(CINJ) are a major force in discovering the cures for cancer, and of development of more 

effective approaches to prevention, diagnosis, and therapy. NCI-designated Cancer Centers 

deliver medical advances to patients, educate healthcare professionals and the public. reach out 

to underserved populations. and collaborate with colleagues in academia and industry to bring 

the latest medicines directly to patients. As noted in the NCI FY20J 1 annual plan and budget 

report, "Cancer Centers arc key participants-the linchpin, some might say-across virtually every 

NCr initiative." 

A culture of collaboration is also a hallmark of NCr-designated Cancer Centers, as well as many 

NIH-supported grants that require multidisciplinary, multi-investigator, and consortium efforts. 

Collaboration with pharmaceutical and biotechnology companies also helps expedite drug 

development. In an era when targeted cancer treatments are now a reality, the challenge we face 

is to continue to turn groundbrcaking discoveries into lifesaving care in the clinic at an even 

greater speed. By fostering collaborations, we are laying the groundwork for breakthrough 

discoveries in cancer research that will translate into cutting-edge treatments for cancer patients. 

At CIN1, we have emphasized and fostered a consortium model with local researchers at 

multiple institutions including UMDNJ. Rutgers and Princeton Universities. For example, effOlis 

have led to combined science from a collaboration of laboratory and clinical researchers that 

resulted in the discovery of critical metabolic pathways involved in tumor cell survival and 
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5 

launched several clinical trials for patients with lung, breast, colon, skin and prostate cancer that 

are attempting to better starve tumor cells of their nutrients. NCI-funded centers and efforts like 

these provide opportunities for patients never before available, and a route to improve the 

outcomes through effective cancer research. 

Another area of funded research important to improve the quality of care of patients and reduce 

health care costs is comparative effectiveness research. Comparative effectiveness research 

seeks to optimize the use of emerging and existent therapies. For example, a team of researchers 

in epidemiology at CINJ recently published a landmark study that defined the proper usc of 

horn10ne therapy for men with early stages of prostate cancer, which will improve patient 

outcomes while hopefully leading to reduced healthcare expenditures. 

Other novel therapeutic trials at CINJ include a recently opened clinical trial to look at 

harnessing our immune system to tackle pancreatic cancer. Researchers are testing the 

effectiveness of a new vaccine combination injected directly into the tumor. If the results prove 

successful this will improve patient outcomes for one of the deadliest types of cancer. This trial 

is supported by the NCI and would not be possible without the ongoing research at CINJ. 

Other efforts to foster collaboration include the work of AACR that underpins groups such as 

Stand Up To Cancer. This ncw initiative provides large grants awarded to research "Dream 

teams," headed by multiple principle investigators who bring expertise and resources from basic, 

translational, and clinical research arcas to bear on a single cancer problem. 

Investment 

It may seem to some that increasing federal support for cancer research is something the nation 

can't afford right now. But we can't afford not to maintain our investment - given demographic 

shifts now underway. Cancer's economic burden is staggering. The National Institutes of 

Health estimates that in 2008 the overall cost to society was more than $200 billion: 

approximately $93 billion for direct medical costs, $116 billion for loss of productivity due to 

premature death, and approximately $19 billion for loss of productivity due to the acute illness 

and time necessary to undergo cancer treatment. Our population is aging rapidly, and cancer is 
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6 

largely a disease of aging. As a result, the total economic burden of cancer in the United States 

will likely continue to increase and interventions are required now to bend this health 

expenditnre curve. 

We are at a most promising time in cancer research. Our nation's investment in cancer research 

has brought us to the forefront of a revolution. But much more remains to be done. We have the 

potential to accelerate through this tipping point in time to welcome a new era of cancer 

treatment and prevention and gain on our investment to reduce the toll of cancer on the people 

and economy of our Nation. 
94 

Mr. PALLONE. Thank you, Dr. DiPaola. 

Dr. Allen. 

STATEMENT OF JEFF ALLEN 

Mr. ALLEN. Good afternoon, Chairman Pallone, Mr. Shimkus and 

members of the subcommittee. It is an honor to testify before you 

today. I am Dr. Jeff Allen, executive director of Friends of Cancer 

Research, a cancer research advocacy organization and think tank 

based in the Washington, D.C., area. I would like to thank the staff 

of this committee, who have worked tirelessly in putting together 

this hearing. 

The foundation of Friends of Cancer Research, Dr. Ellen Sigal, 

could not be here today as she is with a loved one right now who 

is being treated for a rare cancer. Dr. Sigal started this organization 15 years ago after having lost a sister to breast cancer, her father to prostate cancer and mother to pancreatic cancer. This is as 

personal for her as it is for you, Mr. Chairman, and likely everyone 

in this room including myself, who have been deeply affected by 

this terrible disease. It is with this in mind that I am here today 

to express what we feel needs to be done to end the suffering that 

millions of cancer patients and their families experience every year. 

Exceptional progress has been made in the treatment of cancers, 

due in large part to the investments in biomedical research. However, significant hurdles stand in the way of ending the burden of 

cancer. 

Today it is estimated that it requires over $1 billion, 12 to 15 

years and 1,000 patient volunteers to get a single drug to market. 

Chairman Pallone, 15 years to translate a new discovery to a 

therapeutic today by today’s rates would mean a loss of almost 8.5 

million Americans, approximately the population of your home 

State of New Jersey. 

The funding allocation to biomedical research as a part of the 

American Recovery and Reinvestment Act presented a renewed opportunity for American investigators to carry out research projects 

that otherwise may not have been possible. However, in order to 

truly halt the devastating impact of cancer, a comprehensive approach is needed. First, increased collaboration across all sectors is 

needed to turn the next corner of scientific advancement. The NCI 

continues to be an engine-driving process but we must also acknowledge the need for collaboration with other agencies. For example, increased scientific capacity at the FDA is needed to ensure 

that the discoveries being made at the NCI get to patients as safely 

and efficiently as possible. 

Second, the historic health reform bill passed this week takes 

many important steps to ensure that breakthroughs from research 

are available, accessible and affordable to all Americans. This includes the expansion of comparative effectiveness research, which 

can help to provide improved information for patients and their 

health care providers. While it will take careful thinking to finalize 

many of the details moving forward, we look forward to working 

with members of this committee and others to ensure the success 

of these programs. 

Finally, we must also tear down the silos that exist in biomedical 

research and focus on the common goal of reducing the cancer burVerDate Mar 15 2010 13:13 Dec 14, 2012 Jkt 076020 PO 00000 Frm 00101 Fmt 6633 Sfmt 6633 E:\HR\OC\A020.XXX A020

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den. Classifying and studying cancers based on their molecular 

characteristics as opposed to just their tumor site is in many cases 

the direction that science is leading. It is through the success of research that common molecular targets for abnormal growth have 

been identified in multiple cancers. While this adds to complexity, 

it also creates opportunities for shared success. This is not to diminish the important work of targeted focus but we must let our 

work support and inform those fighting for the common goal on our 

alternative fronts. The time of scientific opportunity is upon us. In 

order to ensure that multiple integral components of the health 

care system are prepared for the future of cancer research, we 

must act now. 

We respectfully ask that members of this committee, the Congress and the Administration be steadfast in their commitment to 

ending cancer. I cannot emphasis enough the need for collaboration. The advocacy community and entire research community 

must embrace our common goal and support science and collaboration that will enhance the battle against cancer on all fronts. It is 

our responsibility to represent patients’ needs and what must be 

done to end the burden of all diseases. Thank you. 

[The prepared statement of Mr. Allen follows:] 


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NCI Cancer Research: Today's Progress; Tomorrow's Challenges 

Jeff Allen, Ph.D 

Executive Director 

Friends of Cancer Research 

Testimony Before 

Committee on Energy and Commerce 

Subcommittee on Health 

United States House of Representatives 

March 23, 2010 
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Introduction 

Good afternoon, Chairman Pallone, Ranking Member Deal, Mr. Shimkus, and Members 

of the Subcommittee. I am Dr. Jeff Allen, Executive Director of Friends of Cancer 

Research, a cancer research advocacy organization and think tank based in the 

Washington area. I would like to thank the staff of this committee who have worked 

tirelessly in putting together this hearing. It is an honor to testify before you today on the 

importance of cancer research at the National Cancer Institute (NCI) and across all 

sectors. My testimony is intended to give perspective on the vital need for NCI to be in 

direct collaboration with other federal health agencies, the important role that publicprivate partnerships can play in spurring innovation, and the need to tear down the silos 

that currently exist within the biomedical research community. 

The founder of Friends of Cancer Research, Dr. Ellen Sigal, could not be here today, as 

she is with a loved one right now that is being treated for a rare cancer. Dr. Sigal started 

this organization 15 years ago after having lost a sister to breast cancer, her father to 

prostate cancer, and mother to pancreatic cancer. This is as personal for her, as it is for 

you Mr. Chairman, and likely everyone in this room, including myself, who have been 

deeply affected by this terrible disease. It is with this in mind that I am here today to 

express what we feel needs to be done to end the suffering that millions of cancer 

patients and their families experience every year. 

Nearly forty years ago President Nixon declared war on cancer by signing The National 

Cancer Act (P.L. 92-218) into law, and said, "The time has come in America when the 

same kind of concentrated effort that split the atom and took man to the moon should be 

turned toward conquering this dreaded disease. Let us make a total national 

commitment to achieve this goal.,,1 Four decades later, we also have a President that 

has committed to ending cancer in our lifetime. 

Over the past forty years, exceptional progress has been made in the treatment of 

cancers, due in large part to the investments in biomedical research. This has allowed 

for an understanding of the underlying biology of the disease in much more scientific 

detail. For example, since the declaration of a war on cancer, blood cancers have gone 

from being classified as five different types of leukemia and lymphomas to nearly 90 

different disease subtypes based on biological characteristics. This has lead to the 

development of more tailored treatments that are more effective and in many cases less 

toxic. The current five-year survival rates for leukemia and lymphomas now average 

upwards of 70%.2 That is at least twice, and in some cases, four times what they were 

in 1970. 3 

While this type of progress is compelling and observed in a few other cancers as well, 

there is much more to be done to alleviate the burden of cancer. It is estimated that, in 

2009, nearly 1.5 million Americans will have been diagnosed with some form of cancer. 

As a result, our healthcare system will be strained an additional $228 billion.4 Most 

1 Remarks by the President in State of the Union Address: January 22, 1971 http://www.cspanorg/Content/HTMLlExecut,vefT ranscripts/nixon 1971. pdf Accessed 3/19/10 

2 Allison, M: Is Personalized Medicine Finally Arriving? Nature Biotechnology. Vol. 26; No.5, May 2008: 

509-17. 

3 Facts 2009-2010. The Leukemia & Lymphoma Society. httpl/www.leukemia-. 

~homaorg/attachments/Nalional!br 1247234696.pdf Accessed 3/19/10 

The American Cancer SOCiety: 

bI\QlLwww.cancer.org/docrootiMIT/contentiMIT 3 2X Costs of Cancer asp Accessed 3/19/10 

2 
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importantly, this disease will claim the lives of 562,340 mothers, fathers, grandparents, 

sisters, brothers, and friends, per year. 5 

Biomedical research is an enterprise that is built on the benefits of capitalism, and all 

sectors gain from and are required for its success. Today, it is estimated that it requires 

over $1 billion, 12 to 15 years, and thousands of patient volunteers to get a single drug 

to market. Fifteen years to translate a new discovery to a therapeutic treatment, by 

today's rates, results in the loss of almost 8.5 million Americans approximately the 

population of state of New Jersey.6 

Let us join together and commit that hearings like this, which are so important to refocus 

on these issues, will not end with a few comments for the record, but will actually bring 

about new action. There is an ever growing need for bipartisanship; to not let political 

posturing hinder the progress we have made as we strive to discover new life-saving 

treatments and therapies. 

The strides forward in cancer research have been large, but due to the complexity of the 

disease, new multi-institutional approaches must be fostered. The funding allocated to 

biomedical research as a part of the American Recovery and Reinvestment Act of 2009 

(ARRA) presented a renewed opportunity for American investigators to carry out 

research projects that otherwise may not have been possible. The programs supported 

by the National Caner Institute (NCI), from both the ARRA and the traditional budget 

process, continue to advance cancer research. However, a sustained funding 

commitment for biomedical research is needed to build on our prior investments and see 

that the returns on those investments reach the clinics and benefit the millions of 

patients, as they were designed to do. 

Sustained funding is just one component to reducing the cancer burden. 

In order to truly halt the devastating impact of cancer, a comprehensive approach 

is needed. The NCI and its programs are an engine that drive progress, but 

increased collaboration with other entities, both public and private, are needed to 

turn the next corner of scientific advancement. 

We must also acknowledge the need for a streamlined process in getting the discoveries 

being made at the NCI to patients, as safely and efficiently as possible. 

To accomplish this Congress needs to support increased scientific capacity of the Food 

and Drug Administration, which serves as the nexus between the progression of 

laboratory research and the clinical use of new therapies. 

We ask that you support President Obama's fiscal year 2011 budget request which 

contains a much needed 6% increase to the budget authority for the FDA, including $25 

million allocated to Regulatory Science, which aims to develop, assess and provide new, 

validated tools and approaches to better evaluate the utility of new medical products. 

This initiative has been spearheaded by the visionary leadership of FDA Commissioner 

Dr. Margaret Hamburg. 

, Nel Surveillance, Epidemiology and End Results: http://www.seer.cancer.gov/ Accessed 3/19/10 

6 United States Census Bureau: 

http.l!iactfindercensus.gov/servletiSAFFPopulation? submenuld=population 0& sse=on Accessed 3/19/10 

3 
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The success of the NCI cannot be measured solely as the research accomplishments 

facilitated by the institute, but rather by its contribution to the larger goal of reducing the 

national cancer burden, and this cannot be achieved alone. It is critical to also examine 

the impact of other federal agencies on the ability for NCI-based discoveries to ultimately 

improve the lives of patients. For example, in 2007 the Food & Drug Administration 

(FDA) Science Board declared the agency's "mission at risk" due to its eroded scientific 

foundation? Clearly, without a scientifically rigorous regulatory body, discoveries 

facilitated by NCI-based research could be inefficiently or inappropriately evaluated, and 

ultimately not achieve the envisioned improvement to patient's lives. 

Even prior to the report that highlighted the need to advance the science of regulation, 

the FDA acknowledged the need for assembling oncology expertise at the agency 

through the establishment of the Office of Oncology Drug Products. 8 While this has 

made several improvements to the regulation of oncology programs, a comprehensive 

FDA Oncology Program is still needed to facilitate and increase the transparency of 

intra-agency collaborations, standardize review guidelines, and establish jurisdiction and 

sufficient interactions between FDA Centers that are frequently involved in the review of 

increasing complex new product applications. A robust cancer program can also build 

upon existing collaborations in order to increase the scientific methodologies used by the 

agency. 

The NCI knows the power of such collaborations first hand through the development of 

the NCI-FDA Interagency Oncology Taskforce (IOTF).9 This has allowed for new 

training mechanisms and an exchange of ideas that capitalizes on the great expertise at 

both agencies in order to efficiently translate NCI-based discovery to patient benefit. 

However, with additional resources the IOTF could expand its current portfolio to include 

additional scientific programming. It is in that spirit that on February 24th this year, the 

leaders of the National Institutes of Health (NIH) and the FDA announced the formation 

of the Joint Leadership Council in order to take the necessary steps to work together to 

advance the missions of each respective agency. 10 

It is these types of collaborations that are needed across the federal health agencies to 

streamline cancer research, detection, treatment, prevention, sUNeiliance, product 

regulation, care delivery, reimbursement of seNices, and to learn from all of these 

cancer-related functions as a routine by-product of care. This will ultimately lead to 

increased sUNivorship, reduction of cancer incidence and mortality, and improvement of 

the lives of all who may face this disease. 

The collaboration cannot occur within federal government agencies alone. Publicprivate partnerships, like that of the Foundation for the NIH or the Reagan Udall 

Foundation for the FDA, are the direction that we must go in order to capitalize on 

all research being conducted around the world. 

7 Report of the FDA Subcommittee on Science and Technology: FDA Mission at Risk. November, 2007 

http://w-NW.fdagov/ohrmsidocketsIACI07/briefinqI2007-

432gb 02 01 FDA%20Report%200n%20Science%20and%20Technology.pdl Accessed 3120110 

"FDA News Release, July 16, 2004: 

http://w-NW. fdagov/NewsEventsiNewsroom/PressAnnouncementsl20041ucm108326. htm Accessed 3120110 

9 NCI-FDA Interagency Oncology Taskforce: http://otir cancergoviprogramslpartnerships iotl.asp Accessed 

3120/10 

10 United States Department of Health and Human Services: 

http://w-NWhhs.gov/secretarylspeeches/sp20100224.htmIAccessed 3119/10 

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Last week, the Biomarker Consortium, a public-private partnership through the 

Foundation for the NIH, announced the opening of the I-SPY2 TRIAL. This clinical trial 

will utilize an innovative new model that use biomarkers from individual patients' tumors 

to screen promising new treatments for breast cancer and identify which treatments are 

most effective in specific types of patients. This will allow researchers to use early data 

from one set of patients to guide decisions about which treatments might be more useful 

for patients later in the trial, and eliminate ineffective treatments more quickly. Included 

in this public-private collaboration are NCI, FDA, the Center for Medicare and Medicaid 

Services (CMS), the Pharmaceutical Research and Manufacturers of America (PhRMA), 

the Biotechnology Industry Organization (BIO), major pharmaceutical companies, and 

numerous non-profit medical research organizations." It is through collaboration that 

true progress will continue to be made. 

In addition to fostering multi-agency and multi-institution collaboration, we must 

address the barriers to advancing the work achieved through NCI programs by 

modifying current policies. This includes the need for the streamlining of 

intellectual property agreements between academia, government, and industry, 

modifications to the HIPPA privacy rules that ensure patient protection but don't 

stand in the way of research, and data collection. 

The historic health reform bill passed this week, developed by this committee and others 

takes many important steps to aid cancer research and ensure that breakthroughs from 

research are available and accessible to all Americans. We applaud the protections it 

provides for patients by prohibiting insurance discrimination due to a pre-existing 

condition. The provisions that safe guard the coverage of routine costs of care for 

patients that participate in a clinical trial will undoubtedly allow more cancer patients to 

participate in clinical research as an option of their care. This is a critical step forward in 

alleviating the challenges created by the currently low levels of participation in oncology 

trials. Numerous steps were also included to help provide much needed access to 

important tools for prevention, the ultimate defense against cancer. However, we must 

remain steadfast in our research support to develop even more preventive measures for 

the many cancers that we have no current means to prevent. 

Another component of the health reform bill that Congress should be commended for 

addressing is the expansion of comparative effectiveness research (CER) programs. 

Additional support for CER can help to provide improved information for patients and 

their healthcare providers as they decide the best course of treatment. A focus on 

strengthening the information technology (IT) infrastructure in this country will help 

generate additional evidence to inform health outcomes research and CER. ,2 In fact, the 

development of large-scale data networks will create an expansive collection of 

outcomes data for which comparisons of different treatment options can be performed. 

Furthermore, the use of harmonized data networks will help increase transparency to 

research priorities and generate further hypotheses for clinical studies based upon 

subpopulation characteristics, which in turn, will help to further advance "personalized" 

medicine. 

11 I spy 2 TRIAL (Investigation of Serial Studies to Predict Your Therapeutic Response with Imaging And 

molecular Analysis 2): http://ispy2.orgl Accessed 3119110 

12 Improving Medical Decision Making Through Comparative Effectiveness Research: 

hJlR/lfocr.orglfileslCER REPORT FINALpdf Accessed 3119110 

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In order for these activities to be successful we will need the previously described 

agency collaborations to develop research priorities, design appropriate study 

methodologies, identify of the most appropriate agency/organization to conduct 

the CER studies, as well as provide the resources necessary. 

The NCI should be at the center of this type of work, and already has significant 

expertise in the development of data networks for cancer research that can support the 

development of more personalized medicine as well as be utilized or serve as a model 

for other health areas. 

To begin to address the challenges associated with translating early phase research to 

clinically meaningful treatments, the health reform bill establishes the Cures Acceleration 

Network. This provision aims to address areas of high medical need and support future 

innovation in such areas by providing directed funding and streamlining processes 

required for the development of a potentially beneficial medical product. 

These provisions in the health reform bill hold great promise for the future health of 

America. While it will take careful thinking to finalize many of the details moving forward, 

we look forward to working with members of this committee and others through the 

implementation process to ensure the success of these programs. 

We must also tear down the silos that exist in biomedical research and focus on 

how all sectors can work toward the common goal of reducing the cancer burden. 

Advances in cancer research have redefined how cellular abnormalities associated with 

cancer development and progression are studied and treated. Basic research findings 

have revealed that unregulated cell growth is often the result of altered signaling 

mechanisms that frequently involve multiple, complex molecular signaling pathways. 

This phenomenon is observed in many different cancers no matter what their tissue of 

origin may be. While the specific abnormality or mutation may likely be different in 

different cancers an improved understanding of the biology of cancers has identified 

potential commonalities that, when identified, may allow effective treatments to be 

utilized in multiple cancers that have been historically categorized differently based on 

their tumor site. 

Classifying and studying cancers based on their molecular characteristics as opposed to 

just their tumor site is in many cases the direction that science is leading. This 

alternative approach in classification of different cancers has, and will continue to parse 

the number of people that actually have the same form of disease. Much like the 

subtypes observed in blood cancers, similar subpopulations are being defined in other 

cancers. For instance we now understand that "breast cancer" is a collection of 

hundreds of molecularly defined different diseases. This causes many more cancers to 

actually be "rare" diseases than originally thought. 13 Attention to this growth in the 

overall number of rare disease has been shown by NIH Director, Dr. Francis Collins, 

13 A rare disease is defined as a disease that affects fewer than 200.000 people in the United States. The 

Orphan Drug Act of 1983 (P.L. 97-414) 
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through his exemplary leadership of the NIH effort to develop new therapeutics for rare 

and neglected diseases, 14 

The identification of multiple signaling pathways and their molecular components 

involved in the complexity of cellular communication has created hundreds of targets for 

potential new treatments, In fact, several effective cancer treatments have been shown 

to provide great benefit in multiple cancer types that may have common characteristics, 

For example, both imatinib (Gleevec®) and bevacizumab (Avastin®) target specific 

cellular components and have been shown to be effective in different types of cancers 

that may have historically thought to be very different from one another. It is through the 

success of research that these common molecular targets responsible for abnormal 

growth have been identified in multiple cancers, Many experts speculate that the future 

of cancer research and treatment will involve the combined use of multiple targeted 

agents that are developed and utilized based on the analysis of tumor characteristics, 

While this presents numerous challenges to developing, regulating, and utilizing such a 

treatment approach, it provides a great hope for the future of cancer research, 

The beneficial effect of the combination is often greater than the sum of its individual 

parts, This will hopefully hold true for the future of cancer therapies themselves, but the 

principle will also need to be applied by all the components of the cancer research 

enterprise if we are to accelerate the pace to reduce the burden of cancer. 

In order to create a synergistic effect in cancer research, existing institutional 

barriers will need to come down with a renewed focus on the common goal. While 

currently it is true that the health burden due to cancer can be seen greater in different 

diseases, taking from one to give to another is not an effective strategy, This is not to 

diminish the important work and targeted focus, but we must let our work support and 

inform those fighting for the common goal on an alternative front 

The field of cancer research has built an unprecedented infrastructure that needs to be 

further capitalized on, The NCI currently has 65 designated cancer centers that should 

encourage both internal and inter-center coordination of research, 15 Philanthropic 

institutions have facilitated great breakthroughs through goal-oriented funding, Tension 

continues to exist between individual initiated research and large scale science, often 

leading to difficult decisions of supporting one over another. In reality, we need support 

for all types of research with increased accountability, clear targeted goals, enhanced 

collaboration, and support for one another's efforts, 

The time of scientific opportunity is upon us. In order to ensure that the multiple, integral 

components of the health care system are prepared for the future of cancer research 

and treatment we must act now. In order to ultimately and successfully reduce the 

burden due to cancer, a renewed and unified commitment is needed - including 

researchers and caregivers, government officials and congress, industry and payors, 

patients and advocates, It is time for a new, unified approach to combating cancer as a 

whole, 

14 NIH Press Release, May 20, 2009: http://www.nih.gov/news/health/may2009/nhgri-20.htm Accessed 

3/20110 

15 NCI Designated Cancer Centers: http://cancercenters.cancer.gov/cancercenterslindex,htmIAccessed 

3/20/10 

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Conclusion: 

I cannot emphasize enough the need for collaboration. The advocacy community, and 

entire research community, must embrace our common goal and support science and 

collaboration that will enhance the battle against cancer on all fronts. It is our 

responsibility to represent patient's needs, and what is needed to end the burden of all 

diseases. 

We respectfully ask that the members of this committee, the Congress and the 

Administration, be steadfast in their commitment to ending cancer, through not only 

sustained funding research but supporting and facilitating inter-agency collaboration, 

regulatory science, tearing down the silos, and encouraging the development of publicprivate partnerships. 

8 
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Mr. PALLONE. Thank you, Dr. Allen. I thank all of you, really. 

I want to particularly mention Ms. Fitzgerald. I remember when 

your husband passed away last year and how sad it was and a difficult time for Mr. Shimkus in particular, and I just wanted to 

thank you for sharing Ray’s and your family’s story with us today. 

Thank you. 

I am going to recognize myself first. You know, I wanted to ask, 

I guess, Ms. Fitzgerald and Ms. Don, you know, you both described 

progress in our fight against cancer but that it has been limited 

progress with certain types of deadly cancers like pancreatic, stomach and esophageal cancer, and of course, I mentioned my mother, 

and one of the things that—I will try to be brief, but what happened was, I started doing research and I actually went on your 

Pancreatic Cancer Action Network site almost daily during that period of maybe 7 or 8 months from when she was diagnosed to when 

she passed away, and of course, the one thing that I kept finding 

out was that the problem was no early diagnosis. In other words, 

by the time you find out about pancreatic cancer, it is usually too 

late. In her case, though, there was a little bit of an early diagnosis 

because she had jaundice, and I basically found out from your Web 

site that that is one of the few cases where they do find it a little 

early if the person gets jaundice and that is because the tumor 

presses on something, I guess the bile ducts or whatever, and the 

person turns yellow, which is what happened to her, and then she 

did have an opportunity. They call it the Whipple operation. And 

I have talked to a lot of people, even Members of Congress, who 

had the operation. 

But what I wanted to ask you is, is that one of the reasons, and 

if not, what are the reasons why pancreatic cancer or a lot of these 

other deadly cancers are so challenging to researchers? Is the lack 

of an early diagnosis the main problem or what is it from a research perspective? 

Ms. DON. It is actually twofold. One is the fact that we don’t 

have early detection tools and the second is that pancreatic cancer 

in particular is a particularly difficult disease to diagnose. The pancreas is located deep in your body so it is not something that a doctor can feel on examination. As you mentioned, there really aren’t 

that many early warning signs. It is usually lower back pain or 

jaundice, and typically with jaundice, the tumor is pushing on the 

bile duct and typically that is too late. So if your mother was a candidate for the Whipple surgery, then that was actually fantastic 

news because most patients are not. Only 15 percent of pancreatic 

cancer patients are currently eligible for the Whipple procedure, 

which is a very, very difficult surgical procedure where they take 

out almost all of your digestive tract to get to your pancreas. So 

if only 15 percent of patients are eligible for the surgery, then that 

means that another problem is that it is very difficult to get tissue 

because you only get issue when you do surgery. 

Mr. PALLONE. Oh, you are saying because only 15 percent actually have the surgery, you just don’t get much tissue to work with? 

Ms. DON. Right. 

Mr. PALLONE. Of course, most of those don’t last more than a 

year or two anyway, even when they have the Whipple, but I see 

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Ms. DON. Pancreatic cancer also has a very high recurrence rate. 

Of the people who have surgery, 80 percent will have a recurrence 

within 5 years. 

Mr. PALLONE. Right. That is what they tell us. So now, is this 

lack of early warning the biggest problem not only with pancreatic 

but also with a lot of these other deadly cancers or that is just pancreatic? 

Ms. DON. No, it is—— 

Mr. PALLONE. And Ms. Fitzgerald can chime in too if she wants. 

Ms. DON. It is a similar problem across many of the cancers, and 

most of the deadly cancers we find at very late stages, so getting 

the cancers at earlier stages would be helpful but we also don’t 

have treatments for them once we get them. As I mentioned, the 

best treatment for pancreatic cancer is a surgical procedure that 

even in the best case, 80 percent, still don’t make it more than 5 

years. 

Mr. PALLONE. Ms. Fitzgerald. 

Ms. FITZGERALD. I think it is a combination. There are very few 

symptoms in many of the GI-tract cancers, gastric cancer. You 

know, like I mentioned, my husband’s symptom was burping which 

is, you know, pretty often reflux or something of the kind—— 

Mr. SHIMKUS. Ray did that all the time. 

Ms. FITZGERALD. But there are few symptoms, and I think it is 

also in the case of gastric cancer, a rapid progression. So you have 

very few symptoms, you have a rapid progression throughout the 

GI tract and then straight—because the stomach pumps out all 

your—you know, it gets your food and it pumps out your nutrients. 

It goes straight to your blood so it goes straight to your liver, so 

it is a rapid progression and right to basically all of your blood and 

so it goes throughout your body, and I think that is really similar 

on a number of the GI cancers, that they progress right to the liver. 

Mr. PALLONE. So is the problem then—in other words, is the reason why National Cancer Institute or others don’t do a lot of research because they just figure that with all these problems and 

the difficulty of determining early diagnosis and cures, it is just not 

worth the investment? I don’t want to put it that way but is that 

what is going on? They would rather spend money on other things 

where they think they can make more progress? Is that what we 

are getting? 

Ms. DON. I think that from our perspective, and obviously I can’t 

speak for NCI but from our perspective, it has been a case of we 

have gotten to a point where we encourage researchers to go after 

the lowest hanging fruit, and NCI funds the safe bets. 

Mr. PALLONE. Because this way they can show they are spending 

money, they want to show results. 

Ms. DON. That, and there is a limited amount of money. 

Mr. PALLONE. But in a sense, I mean, at one level it makes sense 

because then they can show they have results. On the other level, 

they may be spending money on things that aren’t as deadly and 

don’t need—I mean, you see my point. In other words, why not 

spend money on the areas where we have such problems rather 

than the ones where we don’t. 

Ms. DON. And that is our central point, that we really believe the 

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est problems, and the hardest problems are really the deadly cancers where we have made the least amount of progress. That 

doesn’t mean there is not worthwhile research to be done on other 

cancers, on breast cancer and colon cancer and prostate and the 

others, but there is definitely a set of cancers that have not gotten 

the same amount of attention or really any sufficient attention, and 

it is time to focus efforts there. 

Mr. PALLONE. Thank you. 

Mr. Shimkus. 

Mr. SHIMKUS. Thank you. And I want to thank Dr. Barker for 

staying here and listening to the testimony. That doesn’t always 

happen here in Congressional hearings, and I think it is to your 

credit. And I also now can see why the funding issue that we struggle with is very similar to the funding struggles that you have. 

Ms. Don, kind of on the same line, but the question would be, 

NCI’s efforts towards establishing The Cancer Genome Atlas, 

which was discussed at length, I think, how is that going to help 

research towards the deadly cancers, the pancreatic and gastrointestinal and others? 

Ms. DON. Well, we certainly appreciate and want to acknowledge 

Dr. Barker in particular for all of her work to try and ensure that 

deadly cancers are included. I mean, the three places where they 

started were three of the deadly cancers, and she has absolutely 

reached out to the deadly cancer community to try and get more 

deadly cancers included. I think the issue is that The Cancer Genome Atlas is one piece of a very large puzzle of things that we 

need to be doing for these cancers. It is a very important piece, as 

is nanotechnology, but we also need to be doing additional things 

to be able to understand the bigger picture and so we fully support 

TCGA moving forward, and from the Pancreatic Cancer Action Network’s perspective, we absolutely hope pancreatic cancer is included and we can figure out a way to deal with the tissue issue 

with pancreatic cancer, but we need to focus on other efforts too. 

Mr. SHIMKUS. Great. Thank you. 

Kristin, again, welcome. We all miss Ray. And your daughters 

have been precious in the back. Let me ask you a question on the 

issue of federally coordinated national cooperation. Why is that so 

important in cases like stomach cancer that develop in young people? 

Ms. FITZGERALD. First, I just want to publicly thank you and 

your staff for your tireless advocacy on behalf of Ray, and I know 

that he would be incredibly happy that we were here at the Commerce Committee, his favorite committee, and this is a Congressional family. Ray’s family was Congressional, many of whom are 

here, and I just want to thank you for helping and I know that it 

would mean a lot to him that we are helping to prevent this from 

happening to other people. 

I think that I would say that NCI federally coordinating cooperation and collaboration on the case of GI cancers like gastric cancer 

and esophageal cancer is one of what I would think would be their 

truest missions, which in the case of someone like Ray where they 

are younger and they have a more rare cancer, that is a time 

where there really isn’t a sample size that you need at any one institution. There are many institutions that are doing their best to 


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really make a difference in curing people that have gastric cancer 

but they might see a handful of patients that are young like Ray, 

so in really attacking that question of what is happening in these 

cases with gastric and esophageal cancers that are happening in 

young people, collaboration is absolutely essential. Otherwise they 

just won’t be able to make the kind of gains that they need. And 

they need—in the case of gastric cancer, they need a place to store 

tissue. They don’t have a biorepository where they can store things. 

They need to be able to share that so they can make the research 

happening at any one institution benefit from the kinds of tissues 

that are coming into other institutions. They need a clinical registry which is coordinated on a federal level, all the kind of things 

that they can use to determine the kinds of influences that are contributing to these cancers. The coordination on a federal level helps 

to be able to solve that problem because of the smaller sample size 

at all the institutions that are working so hard. 

And probably most importantly, they need a coordinated federal 

research project because there are difficulties in obtaining gastric 

cancer tumors. When you are diagnosed with gastric cancer, you 

can’t have surgery if you are stage IV like Ray because that would 

delay your chemotherapy and you would probably die. So you have 

to have mechanisms in order to develop a better tissue sample before it is treated with chemotherapy, and those are the kinds of 

things that NCI can perfect and disseminate throughout the United 

States because you are not having an endoscopy at a cancer center 

most likely. You don’t think you are going to find cancer. And so 

even the community centers need to be able to get a sample size 

of the tissue that is untreated in order to have the kinds of things 

they need to make the truest gains in research. So it is probably 

in my view one of the areas where NCI can most be effective is coordinating all these fantastic cancer centers that are doing their 

very best in these areas and really making those gains. 

Mr. SHIMKUS. Thank you, and I have got limited time, but the 

5-year survival rate for GI cancer is? 

Ms. FITZGERALD. Well, for metastatic cancer, five of the worst 

survival rates, and I put them in my testimony, are GI. I think it 

is 1.7 for pancreatic. Liver is, I think, 2.2, 2.4 for esophageal, 3.4 

for GI cancer. So, you know, folks with metastatic cancer, which is 

where it is often found for those GI cancers, they are not living. 

Mr. SHIMKUS. Which is compared to some of the others where we 

have 95 percent early diagnosis, and I know they are different, but 

that is part of this debate. 

My time is up but I just want to, if I can, Mr. Chairman, just 

go to Dr. DiPaola and Dr. Allen. Because we have had a big debate 

on this terminology and hopefully this terminology is maybe not 

the same, or I am trying to get—comparative effectiveness research, which in the health care debate we talk about funding. 

Comparative effectiveness research, I hope, I gather from what you 

are doing, is finding the right response for the right disease, kind 

of like Dr. Barker talked about on the genome and the right medicine or whatever to affect that. What is your definition of comparative effectiveness research? Is it for directed research or is it not 

for a funding process, is it? 


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Dr. DIPAOLA. It is a great question, and this comes up a lot. In 

terms of comparative effectiveness research, what we are really trying to do is do research to try to understand with many of the 

therapies that we have how to have better outcomes for patients, 

so how to take the therapies that we do have and appropriately use 

them to maximize their effect and outcomes for patients. 

Mr. SHIMKUS. Not possibly minimize care based upon the cost? 

Dr. DIPAOLA. I think that it should be focused on outcomes. 

Mr. SHIMKUS. Dr. Allen. 

Mr. ALLEN. Thank you. At Friends of Cancer Research, we spend 

a lot of time looking at this issue for really about the last 2 years, 

and in conjunction with the cancer community put together a policy 

white paper that I am happy to leave copies for the members of the 

committee if you are interested. But what this outlined was really 

from a broad perspective what can be obtained through comparative effectiveness research, largely focusing on the need for creating 

additional information, and I think we are in a fortunate position 

as far as the national infrastructure that is available in this country from the cancer research centers funded by NCI to be able to 

collaborate better to create additional information that then can be 

a starting point for comparative effectiveness research but it is important as this funding is being allocated to fund comparative effectiveness research moving forward that we capitalize on the 

strengths of the different agencies to be able to really get to the 

answers to the questions that we are looking for and so comparative effectiveness and personalized medicine can actually go hand 

and hand and inform each other better. 

Mr. SHIMKUS. Thank you, Mr. Chairman. 

Mr. PALLONE. Thank you. 

Next is our vice chair, Ms. Capps. 

Mrs. CAPPS. Thank you. Thank you, each of you, for your testimony. I had to miss some of the oral testimony but I am glad I 

could read the written statements that you made and I appreciate 

it very much. I have two questions, one to ask you, Dr. DiPaola, 

quite specific to the Cancer Institute of New Jersey. Then I have 

a more generic question to ask each of you. 

So if I could ask you, Dr. DiPaola, your testimony described the 

work of the Cancer Institute and the work of the consortia. Can 

you just briefly highlight the issues involved in setting this up and 

the way this could be replicated in other areas of the country, how 

it could be streamlined perhaps as a model? 

Dr. DIPAOLA. Sure, absolutely, and I think that the consortia 

model is being done throughout the country, you know, and I gave 

an example in terms of what we are doing. CINJ as an NCI-designated center does have a consortia model, meaning there can be 

members, great researchers from additional institutions, especially 

local institutions, that would basically allow us to share their 

science, work together and translate that science into clinical trials 

for patients. Right now we work together with many of the researchers at Rutgers and now even Princeton, and what I had described in my testimony was an example of that that has actually 

led to a better understanding on how to better starve tumor cells 

and has led to additional grant funding because of this team approach. Additionally, it has led to clinical trials that are operating 


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right now that are taking that biology and that understanding from 

researchers from multiple institutions and putting it into designing 

new clinical trials. 

Mrs. CAPPS. Thank you, and because of Dr. Barker’s previous testimony, I can see the connection with NCI and then to you and 

then perhaps other consortia around the country too. I see a really 

good model. 

Finally, I know that it has been mentioned already that our recovery dollars have been a real boost to the NCI. The downside of 

that is that it is one-time or limited-time funding. I would like each 

of you to respond to this question. We all know that NIH has essentially been flat-funded during a good portion of the past decade. 

In your experiences, how has this affected cancer research, and 

particularly research on the deadly cancers, and briefly, how can 

we avoid that in the future? Just down the panel, if you will start, 

Ms. Fitzgerald. 

Ms. FITZGERALD. I think that NIH and NCI are doing their very 

best. 

Mrs. CAPPS. I know they are. 

Ms. FITZGERALD. It is a difficult balance. I think one of the 

things that I would like to see is factoring in incidence but then 

factoring in mortality. You know, maybe there is a large portion of 

the United States that gets a particular cancer but if they have 

treatments, they are a little bit better off than, for example, my 

husband, who there just wasn’t treatment available and so factoring in that, when you get this cancer, then you die, that to me 

should mean that there is a priority there in the federal research. 

So to the extent that new dollars come into the pot, that those 

are—and even with the existing dollars, they are apportioned in a 

way that includes looking at those kinds of statistics. 

Mrs. CAPPS. Thank you. 

Ms. Don. 

Ms. DON. So 2 percent of NCI’s $5 billion budget goes to pancreatic cancer research. When you talk about ARRA funds, there 

are approximately 208 projects for all of the eight deadly cancers 

for a total of about 5.7 percent of NCI’s ARRA budget. So we look 

at it, if these eight cancers are causing half of all cancer deaths, 

we are not trying to say that it should be X percentage or X number of dollars that should go to these cancers but it seems like 

there should be more than 18 percent of the overall budget, 2 percent in the case of pancreatic, less than 6 percent of ARRA funds 

going to the cancers where we have the worst survival rates, and 

as I stated earlier, I think that part of the problem is that given 

the flat funding, NCI has been looking for the safest bets instead 

of the most difficult research and the deadly cancers clearly are 

some of the most difficult and complex research. 

Mrs. CAPPS. Let me shift the tone for you, Dr. DiPaola, given 

your testimony. Is there a way that the consortia could come partway to meet that diagnosis that has been given by the two previous, you know, how can the deadly cancers that need so much, 

is there a way that what you do can help meet the needs that they 

propose? 

Dr. DIPAOLA. Yes, I do think so. You know, as we especially work 

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ering new pathways and the biology and align them with the clinical researchers that are able to take it in the clinic or for clinical 

trials that are therapeutic or for new biomarkers that might be 

useful in diagnosis. I think as we support those teams coming together on a regular basis, we speed up that process so that that 

biology, which is cancer biology, can apply towards really any cancers, and especially rarer cancers. I can tell you that I spent this 

morning being part of a session at NCI, a symposium where we 

looked at a new biology focused on that area of metabolism actually 

called autophagy, and what was commented in the symposium was 

that that biology actually applies even best to the most aggressive 

cancers, so now what would be important, which we did there, was 

we had laboratory researchers presenting with clinical researchers 

is translate that to many different cancers but it is that same important biology. 

Mrs. CAPPS. That is important. 

Dr. Allen. 

Mr. ALLEN. Thank you. Well, I think like any situation where 

there is a budget, there comes hard choices. But I think that with 

NCI leading the way, it must be encouraged to look at what other 

components of the health care system can provide data so that cancer research is an ongoing and learning process. We need to look 

at the data that is available through other federal health-related 

agencies, and even as Ms. Fitzgerald mentioned earlier, with so 

many cancer patients being treated in a community setting, how 

cancer treatment can actually be research so that we learn more 

about the products we are even using now, because when they 

come to market, we don’t really fully understand, particularly the 

impact that these products might have in different populations that 

weren’t involved in the original clinical trials. So there is a degree 

of creativity in trying to capitalize and generate as much data as 

possible so that we are in a learning process continuously. 

Mrs. CAPPS. Thank you, each of you. 

Mr. PALLONE. The gentleman from Texas, Mr. Burgess. 

Mr. BURGESS. Thank you, Mr. Chairman. You know, I think it 

is interesting on a historical note, this building that we are in, the 

Rayburn Building, Mr. Rayburn did die of pancreatic cancer and 

the diagnosis was just as Ms. Don described it. He came down with 

back pain, came back to Texas, was hospitalized probably back 

then under the care of an orthopedist for a while without much 

help to his problem, and ultimately succumbed to his disease. So 

I don’t think things were a whole lot different in 1961 than they 

are in 2010. It is useful to reflect. 

Dr. Allen, I just wanted to ask you on the issue that has come 

up of course with The Cancer Genome Atlas and the issue of personalized medicine as far as treatment but what about prospectively? What about looking ahead at someone’s risk? What if there 

were a way to screen people to understand where their risks were? 

Is that something that is on the horizon out there from a genetic 

standpoint, not just from the family history, not just from the 

things we typically associate but you have companies out there now 

sequencing human genomes for $987. Is that a useful part of what 

lies ahead for the next generation of researchers and doctors? 


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Mr. ALLEN. I think it is quite possible. The challenge is that 

right now at this point in time we may not fully understand what 

some of that information could lead to down the road or what it 

actually means at a point in time or quite frankly what to do in 

order to act and when a problem is identified. But I think this also 

goes back to the earlier question about how we try and gather longterm longitudinal data so that we understand the outcomes associated with some of those genetic tests that are detected. Ms. Castor 

described really a large project that is going on at the Moffitt Cancer Center that is trying to do just that. As patients come in and 

are diagnosed with cancer, they are enrolled in a long-term database that allows for a great deal of follow-up. So not everyone can 

be enrolled in a clinical trial throughout their lifetime but if there 

is a way that we can try and capture more data about each individual person, share this across multiple centers and agencies, then 

we may be able to get to some of these longer-term answers much 

quicker. 

Mr. BURGESS. Where are you in that process? My father trained 

at Mayo Clinic back in the late 1940s. Part of his master’s thesis 

was on esophageal cancer. Mayo Clinic has tissue from every patient they have ever seen since patient number one. Is there an effort to sort of consolidate and be collaborative of this mass of data 

that is out there? 

Mr. ALLEN. I think there are some very good efforts underway. 

The challenge is the problem is so complex. One program that the 

NCI would be able to tell you more about is their cancer 

bioinformatics grid which looks to align clinical trial data from 

multiple centers across the country. This helps to essentially increase your patient base as well so hopefully like the others at the 

table mentioned, when you have smaller cancer populations enrolling for those trials is an increased challenge so kind of connecting 

the data between others is underway. I think the challenge is that 

the data sources are do disparate right now that it is very difficult 

to align what you gain from a clinical trial versus perhaps administrative records like CMS that have more of a longitudinal look to 

things rather than a point in time kind of where clinical trial is a 

little shorter span. 

Mr. BURGESS. Well, with other areas in medicine, particularly 

with the push to electronic health records, we talk about particularly for hospital-acquired infections, for example, deidentifying 

and aggregating data so that trends can be spotted sooner than 

perhaps in the past. Is this type of work going on as far as surveillance of cancer? It seems like The Cancer Genome Atlas being developed, this would be something that people would want to do and 

in fact would be a priority. 

Mr. ALLEN. Absolutely, and I think there is a great deal of interest in doing that. It is underway. It is expensive. But right now I 

think the biggest challenge, it is frequently done in isolated different centers so you mentioned the Mayo Clinic, which has a fantastic model and example and achieved many successes from it. 

But in order to really harness the power of this, it would be nice 

to try and adopt that and allow it to link in to other systems that 

are doing very much the same thing so that we increase the pool, 

so to speak, the means to the end will be much faster. 


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Mr. BURGESS. Dr. DiPaola, is that something that you are doing 

in New Jersey? 

Dr. DIPAOLA. Absolutely. In fact, you know, one of the areas that 

we have increased dramatically over the last follow-up years was 

our bioinformatics area, and they are working with NCI in their efforts so that we do a better job in having clinical data associated 

with all of the issue and genetic data. 

Mr. BURGESS. What about the concept Ms. Fitzgerald raised? A 

lot of, particularly, well, in this case not early stage gastric cancer 

is diagnosed at an endoscopy center or an ambulatory surgery center, kind of different from the days when I trained, whoever was 

in the hospital, and it was not as big a challenge to collect tissue 

and get it down to the lab. Is there an effort being made to get collaboration from the endoscopy centers and ambulatory surgery centers for just this type of data collection? 

Dr. DIPAOLA. There is. You just pointed out some of the things 

and challenges we need to overcome, and especially when things 

are in these trials including multidisciplinary approach, not only 

multidisciplines in terms of lab and clinical researchers but different disciplines in terms of surgery and radiation oncology and 

medical oncology. 

Mr. BURGESS. But it does seem, Ms. Fitzgerald was telling her 

story and we have the other paper where it talks about the incidence of adenocarcinoma of the esophagus increasing. I mean, I 

have heard that story different names and different places but the 

same story, advanced-stage esophageal cancer being diagnosed at 

an ambulatory surgery center or endoscopy center if the incidence 

is indeed increasing, and this is not—I mean, this is the third time 

I have this story told. I did practice medicine but not GI. It just 

seems like that is something we should be perhaps a little bit more 

aggressive about, about getting the word out to our clinicians who 

are doing the endoscopies out in the field. 

Ms. FITZGERALD. I don’t think that the technology exists to get 

the kind of tumor sample that they want, right now, anyways. I 

mean, I think that it is important to get that little biopsy but what 

they really need is they need that whole tumor with those properties before it is treated and so they need, like, a little hole punch 

that goes all the way down and gets more of those tumor properties, and I think NCI is working on that but I am not sure that 

it is totally the kinds of tissue that we need for the kind of research 

that we need to do. I am not sure that that technology is totally 

available. 

Mr. BURGESS. But, I mean, 15 years ago we were doing that for 

breast disease for estrogen receptors in the hospital. They weren’t 

large tissue samples with ovarian malignancy. 

Ms. FITZGERALD. And they haven’t been disseminated, and I 

think the other part of it too is that in terms of clinical trials, many 

patients do not qualify for clinical trials because they are late-stage 

diagnosis. For example, my husband could not qualify for that so 

I think clinical trials are really important in terms of driving some 

of the funding but I think in the case of pancreatic cancer and 

some of these other cancers like gastric cancer, you simply are not 

going to be able to participate because you don’t qualify and so 


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there has to be a way of obtaining those kinds of tissue samples 

too. 

Mr. PALLONE. OK. We are 3 minutes now. 

Mr. BURGESS. Sorry. 

Mr. PALLONE. That is all right. 

The gentlewoman from Illinois, Ms. Schakowsky. 

Ms. SCHAKOWSKY. Well, first of all, let me just express my condolences to you, Ms. Fitzgerald. As has been said, there is really not 

a family that hasn’t been affected. My 38-year-old daughter-in-law 

died of cancer 5 years ago. I admire your incredible composure. I 

am still not as good at talking about it as you were today. You are 

great. 

Here is what I want to really understand from this testimony. 

One is the issue just of diagnosis. If there is not really major symptoms of some of these deadly diseases, then inevitably when they 

are diagnosed, they have moved along. So maybe this has all been 

said in the testimony but it is not just what we do about the cure. 

Yet your husband was burping. I mean, who doesn’t, right? So 

what would drive—let us say he went for an annual checkup. What 

do we do about diagnosis? 

Ms. FITZGERALD. You have to have a molecular screen on a cellular level. You have to have a blood test that could pick him up 

because there is no other way that you can prevent it because there 

is not a large enough population for these cancers that they will 

ever have the invasive screening procedures necessary that there 

would be for like colon or breast cancer. You just won’t have an endoscopy screening program in the United States like you have a 

mammography or a colonoscopy program. 

Ms. SCHAKOWSKY. Right. 

Ms. FITZGERALD. You have to have that science. You have to understand that molecular change that somebody on their regular 

visit to their general practitioner has a blood draw and catches 

that and either gets put into a screening program or, you know, 

catches the cancer and takes it out. You know, that is the only 

thing that would be able to get somebody in this kind of a situation. 

Ms. DON. If I may comment, that is absolutely true. The other 

thing is that we do need more awareness for the symptoms that we 

know about. For example, in pancreatic cancer, we are beginning 

to see that there may be some evidence that otherwise healthy 

mean who all of a sudden have diabetes may actually have pancreatic cancer, and when they see their general practitioner their 

general practitioner isn’t thinking about pancreatic cancer, they 

are thinking about treating their diabetes, and so we need to get 

more information out there about even back pain. We need to get 

more information out there so that more physicians are thinking 

about some of these other cancers from very seemingly benign 

symptoms and we absolutely need a good early detection test but 

we also need to get information out as it becomes available. 

Ms. SCHAKOWSKY. Thank you. 

Dr. DiPaola. 

Dr. DIPAOLA. I absolutely agree with the other speakers. I guess 

what I would say is that, you know, with the research that is going 

on looking at the biology of the cancer that leads to an underVerDate Mar 15 2010 13:13 Dec 14, 2012 Jkt 076020 PO 00000 Frm 00120 Fmt 6633 Sfmt 6633 E:\HR\OC\A020.XXX A020

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standing of what potential markers we could assess, which might 

be early diagnostic markers. 

Ms. SCHAKOWSKY. Meaning when we take a blood sample or 

something? 

Dr. DIPAOLA. I think that we need to continue to work toward 

discovery of even better markers as we learn the biology of cancer 

even more, and the problem is that once we understand that in the 

laboratory to really apply it in the clinic. We need to conduct very 

large clinical trials to validate that, and that becomes difficult, especially in rarer tumors, and I think having partnerships and collaborations to conduct those types of trials is going to be critically 

important. So even if we found and there are a number of potential 

markers to prove it and make sure that it is doing what it should 

be doing, we need these larger clinical trials. 

Ms. SCHAKOWSKY. So if cancer were present but it is not one of 

these common ones, is our goal to develop now some kind of a simple one-size-fits-all diagnostic tool that is a blood test of some sort 

or, I don’t know, urine, whatever, I don’t know, that would at least 

say there is some abnormality that is worth looking at? 

Dr. DIPAOLA. Well, I mean, the goal would always be to define 

the best and the simplest. I think—— 

Ms. SCHAKOWSKY. But are we looking for that? Are we anywhere 

close to that? 

Dr. DIPAOLA. Absolutely. I mean, I think there are a lot of leads 

and it stems from understanding the biology better. I do think that 

kind of the partnership between the labs that are looking at the 

biology and these potential markers with the clinic need to continue to work together, but it will ultimately require validation in 

these larger clinical trials where many people need to be enrolled 

to really understand this. 

Ms. SCHAKOWSKY. But is the thought, though, that some manifestation of the disease is more likely to show up? You seem to 

imply that even then in some of the rarer forms of cancer that it 

may not necessarily show up in some kind of a mass test. 

Dr. DIPAOLA. No, I think that there is a lot of potential based 

on the biology and understanding new markers. There are new imaging modalities that are coming up all the time, so our ability to 

do things with greater technology have a lot of hope and I think 

our ability to collaborate so that we can conduct larger trials to 

prove or disprove and develop these different technologies is going 

to be important but I do think we need to look further, some relying on, as you have heard, the current symptoms and current imaging modalities for many of these cancers is just not enough and so 

we are relying on new science and discoveries, new markers and 

developing them all the way through clinical trials to prove them 

and use them in the best possible way. 

Ms. SCHAKOWSKY. Thank you. Thank you all. 

Mr. PALLONE. Thank you. 

Mr. SHIMKUS. Before you could, could I—— 

Mr. PALLONE. I yield to the gentleman. 

Mr. SHIMKUS. Thank you, and I will be brief. Kind of following 

up on my colleague from Illinois’s comments, I think part of that 

genome discussion I think earlier, one of my takeaways is that if 

we identify in essence an individual genome, and I would have 


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thought that they would never change. Obviously there are changes 

that may occur, and then if people have that as part of their medical record, then you may get a better heads-up than before. Is that 

fair to say? 

Dr. DIPAOLA. Absolutely. If you can start identifying populations 

that are at risk for certain cancers, then as you develop even, you 

know, the existing and the newer modalities whether they be imaging or new biomarker potential diagnostic tests, you would apply 

them more individualized and appropriate, especially for the higher 

populations. 

Mr. SHIMKUS. And I guess the other takeaway is that we do— 

as we know, we always have the disease groups here. This funding 

issue causes people to struggle, and it is very compelling when you 

talk about mortality rates and where should dollars go. Now, we 

would hope that NCI would take that into consideration as they 

make these decisions versus intervention by us or other people. I 

was never one to want to direct funding because you want it to go 

to the scientists and you want them to apportion based upon due 

diligence, but there is a question about should mortality be given 

a higher priority, and that is kind of a takeaway and I don’t know 

what we do from that. 

And last, I got an e-mail. Kristin, folks who are watching, they 

are saying you are doing a great job and they appreciate your 

strength and fortitude. 

Thank you, Mr. Chairman. 

Mr. PALLONE. Thank you. 

Let me say before we conclude, it is very obvious to me and I am 

sure to everyone in the room that there is a great deal of interest 

on this panel in everything you have discussed, and I know it was 

mentioned that Ms. Capps and I are trying to put together some 

legislation and there is already, I think that you mentioned the 

pancreatic cancer bill that is already out there. I mean, there are 

different things out there. So first of all, I will say we are probably 

going to have a lot of follow-up written questions to all of you just 

because we have so many questions. I know, for example, you both 

described the need for improved collaboration to ensure that scientific advances at NCI and others actually translate to safe and 

effective treatments, and I want to follow up with my staff about 

the three types of collaboration you discussed within the biomedical 

research community and with industry partners, so that is one 

thing I know we are going to get back to you on. But I am sure 

there are going to be others. So thank you so much. We really appreciate it. Usually we get back to you within 10 days or so with 

any written questions that we have, and I really appreciate your 

testimony. 

And without objection, the Subcommittee hearing is adjourned. 

[Whereupon, at 5:50 p.m., the Subcommittee was adjourned.] 

[Material submitted for inclusion in the record follows:] 


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Opening Statement for Energy and Commerce 

Health Subcommittee Hearing 

"NCI Cancer Research: Today's Progress; 

Tomorrow's Challenges." 

Congressman Marsha Blackburn 

March 10,2010 

I would like to thank the Chairman for calling this 

hearing to inform this Committee of the progress the 

National Cancer Institute (NCI) is making in cancer 

research and discuss the issues that lay ahead. 

I am excited about the cancer research that is taking 

place in my home state of Tennessee. The VanderbiltIngram Cancer Center in Nashville is the only National 

Cancer Institute-designated Comprehensive Cancer Center 

in Tennessee that conducts basic, translational and clinical 

research and offers adult and pediatric oncology treatment. 

It is the only center in Tennessee consistently ranked 

among the best hospitals for cancer care by us. News & 

World Report. 

The Cancer Research Institute of West Tennessee 

(CRIWT) aims to address two areas of cancer care that are 

often overlooked: Individualized Cancer Management and 

Treatment Therapy. The Institute is currently raising about 

$15,000,000 for research in early detection and treatment. 

CRIWT's research is unique in that it develops diagnostic, 

prognostic, monitoring and treatment protocols for cancer 

patients on an individual basis. Customized protocols may 
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be developed using these procedures to more accurately 

diagnosis a patient's tumor and prescribe the proper 

treatment. 

St. Jude Children's Research Hospital is the only 

pediatric research hospital supported by a National Cancer 

Institute Cancer Center Grant. It is also the only pediatric 

research hospital to be designated a Comprehensive Cancer 

Center by NCI. It is the only pediatric cancer research 

hospital that produces highly specialized medicines for 

cancer treatment through a Good Manufacturing Practices 

facility. The University of Tennessee Cancer Institute is the 

adult cancer partner of St. Jude in Memphis. 

Additionally, the Tennessee Breast Cancer Coalition 

provides assistance to more men and women than any other 

breast cancer grassroots organization in the state, and the 

Minnie Pearl Cancer Foundation offers free diagnostic 

mammograms and ultrasounds for uninsured and 

underinsured individuals in need of testing for their breast 

cancer symptoms. 

While these organizations have provided significant 

advances in fighting many types of cancer, there is still 

much more work to be done. I look forward to hearing the 

testimony today and learning more about the innovative 

work being done by NCI. 

Thank you, Mr. Chairman, and I yield back my time. 
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Written Testimony to the 

Subcommittee on Health 

Committee on Energy and Commerce 

United States House of Representatives 

Supplement to March 23 hearingNCI Cancer Research: Today's Progress; Tomorrow's Challenges 

Submitted April 6, 2010 

Testimony submitted by: 

Michael A. Caligiuri, MD 

President, Association of American Cancer Institutes 

Director, Ohio State University Comprehensive Cancer Center 

CEO, James Cancer Hospital & Solove Research Institute 

The Association of American Cancer Institutes (AACI), representing 95 of the nation's premier 

aeademic and free-standing cancer centers, appreciates the opportunity to submit this statement 

for consideration by the United States House of Representatives' Committee on Energy and 

Commerce, Subcommittee on Health. 

AACI applauds recent budgetary commitments-notably, increased funding for NIH and support 

from the Obama Administration tlu'ough the American Recovery and Reinvestment Act of2009 

(ARRA)--that have created a more encouraging landscape for cancer research compared to 

recent years. We hope that this support will continue in the years ahead, to ensure that this 

recognition of the importance of biomedical research is sustained. 

AACI congratulates the administration and Congress on their commitment to ensuring quality 

care for cancer patients, as well as for providing researchers with the tools that they need to 

develop better cancer treatments and, nltimately, to cure this disease. 

President Obama has released his FY2011 bndget which includes a $1 billion increase to the 

National Institutes of Health (NIH) budget for an expansion of support for biomedical research. 

This funding boost would make the NIH budget $32.1 billion, representing a 3.2% increase. The 

National Cancer Institute (NCI) would receive an additional $161 million, or 3.16% more, for a 

total of$5.26 billion. 

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AACI has joined its colleagues in the biomedical research community in supporting the proposed 

increase for NIH and in calling on Congress to fmiher strengthen the impact of the President's 

request hy increasing funding to $35 hill ion. 

With the extra NIH and NCI funding, the cancer community will be hetter equipped to leverage 

ARRA fInancial support. ARRA dollars have helped to sustain the momentum achieved in 

reducing death rates from cancer, and they are proving to he an effective means of stimulating 

local economies and creating or maintaining jobs throughout the country. 

For example, my own institution, The Ohio State University Comprehensive Cancer CenterJames Cancer Hospital & So10ve Research Institute, and the Winthrop P. Rockefeller Cancer 

Institute at the University of Arkansas for Medical Scienccs are moving fClrward with major 

construction projects supported hy ARRA funding. Another AACI mcmber, the University of 

New Mexico Cancer Center is buying equipment and hiring more staff with ARRA money, 

while a researcher at the Vanderbilt-Ingram Cancer Center, in Tennessee, is studying imaging 

techniques in colorectal cancer with help from ARRA grants (Association of American Cancer 

Institutes, AA CI Update, Fehruary 20 I 0). 

Maintaining the flow of sufficient, dependable funding streams for NCI will help to continue the 

work that stillied under the stimulus plilll. It will also serve as recognition that $70 million worth 

of great ideas--the approximate amount of ARRA funding for NCI to date--might not have been 

explored if it were not for the administration's unprecedented infusion of funds for cancer 

research. And much untapped scientific potential remains. 

Cancer Research: Benefiting all Americans 

Cancer's financial and personal impact on America is suhstantial and growing-- one in two men 

and one in three women will face cancer in thcir lifetimes, and cancer cost our nation morc than 

$228 billion in 2008 (Centers for Disease Control and Preventions, Addressing The Cancer 

Burden: Ar A Glance 2010). This year, cancer will become the world's numher one killer. 

Investing in cancer research is a prudent step - both for the health of our nation and for our 

nation's economic well-being. 

Cancer research, conducted in academic laboratories across the country, saves money by 

reducing healthcare costs associated with the disease, enhances the United States' global 

competitiveness, and has a positive economic impact on localities that house a major research 

center. While these aspects of cancer research are important, what cannot be overstated is the 

impact cancer research has had on individuals' lives-lives that have been lengthened and even 

saved hy virtue of discoveries made in cancer research laboratories at cancer centers across the 

United States. 

Biomedical research has provided Americans with better cancer treatments, as well as enhill1ced 

cancer screening and prevention efforts. Some of the most exciting breakthroughs in cunent 

cancer research are those in the field of personalized medicine. Tn personalized medicine for 

cancer, not only is the disease itself considered when determining treatments, but so is the 

individual's unique genetic code. This combination allows physicians to better identify those at 

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risk for cancer, detect the disease, and treat the cancer in a targeted fashio.n that minimizes side 

effects and refines treatment in a way to. provide the maximum benefit to. the patient. 

In the labo.ratory setting, multi-disciplinary teams o.f scientists are wo.rking to.gether to. 

understand the significance o.fthe human geno.me in cancer. For instance, the Cancer Genetic 

Markers o.fSusceptibility initiative is co.mparing the DNA o.fmen and wo.men with breast o.r 

prostate cancer with that o.f men and wo.men witho.ut the diseases to. better understand the 

diseases. The Cancer Geno.me Atlas is in develo.pment as a co.mprehensive catalo.g o.f genetic 

changes that o.ccur in cancer. 

These projects-along with the work being performed by dedicated physicians and researchers at 

cancer centers across the United States every day-have the po.tential to. radically change the 

way cancer, as a collection o.f diseases, affects the people who. live with it every day. Every 

discovery contributes to a future witho.ut cancer as we know it to.day. 

Clinical Trials 

Clinical trials are the cornersto.ne o.f cancer research, and it is commo.nly held that "yesterday's 

clinical trials arc toda)' s standard therapies". Without clinical trials we canno.t disco.ver new 

cancer drugs and better treatments, and witho.ut volunteers we canno.t conduct trials. 

With no more than five percent of adult cancer patients participating in clinical trials, attracting 

volunteers to. trials has been a Io.ng-standing struggle for cancer researchers. And yet, thanks in 

large part to. advances realized through clinical trials, two-thirds of cancer patients no.w survive 

at least five years after diagnosis, co.mpared with only half a generation ago.. 

Unfortunately, running a clinical triallTom start to finish can be prohibitively co.mplicated and 

expensive. While the natio.n's cancer centers represented by AACI work to untangle red tape and 

other factors that can derail trials, a serio.us obstacle stands largely beyond their control--the Co.st 

to patients of participating in trials. 

Scctio.n 2709 o.fthe Patient Protection and Affordable Care Act 0.[2010 requires health insurance 

plans, including those offered through the Federal Employee Health Benefit Program, to pro.vide 

coverage fo.r routine costs associated with participatio.n in clinical trials. 

Co.mmercial health insurers often refuse to. pay for routine care Co.sts associated with a clinical 

trial, arguing that the trial is "investigational" and thus optional or unnecessary. Co.nsequcntly, 

patients experience financial difficulties that limit their participation in trials. That. in tum, has a 

negative impact o.n research and patients' ability to receive promising treatments that are 

available thro.ugh trials. It slows the development o.fnew cancer therapies. 

Routine costs associated with clinical trials include physician visits, blood wo.rk, hospital stays 

and x-rays. These Co.sts would usually be reimbursed by the insurer if the patient was no.t 

participating in a clinical trial. The investigational portion ofthc trial (usually a new drug o.r 

devicc) is not charged to. the patient or the insurer. 

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About 30 percent of the insured volunteers participating in trials at The Ohio State University 

Comprehensive Cancer Center experience such insurance claim denials of payment for routine 

care. Denials tend to occur with Medicare Advantage HMO and PPO plans and with insurance 

plans that are based outside the state where the trial is conducted. 

Since 1994. 27 states and the District of Columbia have passed laws requiring insurance 

coverage for routine patient care costs when patients participate in clinical trials, and another five 

states have established cooperative agreements with insurers to do so. However. beyond the 

patchwork nature of such coverage, somc of these laws do not necessarily require insurers to 

cover all cancer patients. such as those in Phase I or II clinical trials, or those with employer sclfinsured plans, in which a large company self-insures its employees. With the new federal policy. 

all cancer patients can now afford to enroll in a potentially life-saving clinical trial. 

The Nation's Cancer Centers 

The nexus of cancer research in the United States is the nation's network of cancer centers 

represented by AACI. These cancer centers conduct the highest-quality cancer research 

anywhere in the world and provide exceptional patient care. The nation's research institutions, 

which house AACI's member cancer centers. receive an estimated $3.15 billion from NCI to 

conduct cancer research: this represents 65 percent ofNCTs total budget (U.S. Department of 

Health and Human Services. U.S. National Institutes of Health, National Cancer Institute 2008 

Fact Book). In fact, 84 percent of NCr's budget supports research at nearly 650 universities, 

hospitals, cancer centers, and other institutions in all 50 states. Because these centers are 

networked nationally, opportw1ities for collaborations are many-assuring wise and 110l1-

duplicative investment of scarce federal dollars. 

Collaboration between the cancer centers' and NCr is also essential, and extramural input in 

shaping Ncrs programmatic priorities is vital for effecting cancer research breakthroughs. 

Furthem1ore, AACI endorses the call for greater collaboration expressed in recent testimony 

before this committee by Robert S. DiPaola. MD, director of the Cancer Institute of New Jersey. 

The association is in strol1g agreement with Dr. DiPaola that "culture of collaboration" needs to 

be nurtured among NCI-designated cancer centers, as well as between such centers and the 

pharmaceutical and biotechnology companies that develop drug treatment for cancer and related 

illnesses. 

In addition to conducting basic. clinical, and population research. the cancer centers are largely 

responsible for training the cancer workforce that will practice in the United States in the years to 

come. Much of this training depends on federal dollars. via training grants and other funding 

irom NCI. Sustained federal support will significantly enhance the centers' ability to continue to 

train the next generation of cancer specialists-·· both researchers and providers of cancer care. 

By providing access to a wide array of expertise and programs specializing in prevention, 

diagnosis, and treatment of cancer, cancer centers play an important role in reducing the burden 

of cancer in their communities. The majority of the clinical trials of new interventions for cancer 

arc carried out at the nation' s network of cancer centers. 

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Ensuring the Future of Cancer Care and Research 

Because of an aging population, an increasing number of cancer survivors require ongoing 

monitoring and care from oncologists, and new therapies that tend to be complex and often 

extend life. 

Demand for oncology services is projected to increase 48 percent by 2020. However. the supply 

of oncologists expected to increase by only 20 percent and 54 percent of currently practicing 

oncologists will be of retirement age within that timeframe. Also. alarmingly. there has been 

essentially no growth over the past decade in the number of medical residents electing to train on 

a path toward oncology as a specialty (American Society of Clinical Oncology, Forecasting the 

Supply oland Demandlor Oncologists: A Report to the American Society olClinical Oncology 

(ASCO) from the AAMC Centerfor TYorkjorce Studies, 2007). 

Without immediate action, these predicted shortages will prove disastrous for the slate of cancer 

care in the United States. The discrepancy between supply and demand for oncologists will 

amount to a shortage of9.4 to 15.1 million visits, or a shortage of2,550 to 4.080 oncologists. 

(American Cancer Society. Cancer Facts and Figures 2008). 

Cancer physicians-while essential-are only one part of the oncology workforce that is in 

danger of being stretched to the breaking point. For example, the Health Resources and Services 

Administration has predicted that by 2020, over 1 million nursing positions will go unfilled. 

The Department of Health and Human Services projects that today's 10-percent vacancy rate in 

registered nursing positions will grow to 36 percent, representing more than 1 million unfilled 

jobs by 2020. 

Greater federal support for training oncology physicians. nurses, and other professionals who 

treat cancer must be enacted to prevent a disaster where demand for oncology services far 

outstrips the system's ability to provide adequate care for all. 

Conclusion 

These are exciting times in science and, particularly. in cancer research. The AACI cancer center 

network is unrivaled in its pursuit of excellence, and places the highest priority on affording all 

Americans access to superior cancer care, including novel treatments and clinical trials. It is 

through the power of collaborative innovation that we will accelerate progress toward a future 

without cancer. and research funding through the NIH and NCI is essential to achieving our 

goals. 

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DEPARTMENT OF }lEALT}I & }lUMAN SERVICES 

APR 292010 

The Honorable Anna Eshoo 

House of Representatives 

Washington, D.C. 20515 

Dear Ms. Eshoo: 

Public Health Service 

NatIonal Institutes of Health 

Bethesda, Maryland 20892 

Thank you for the opportunity to testify at the March 23, 2010 hearing entitled "NCI 

Cancer Research: Today's Progress; Tomorrow's Challenges" before the Committee on 

Energy and Commerce Subcommittee on Health. 

I appreciate your interest in the research being conducted at the National Cancer Institute. 

The enclosed responses to your questions for the record provide additional information 

on our strategies for addressing the many challenges we face in cancer research. 

Thank you again for your intcrest and support of cancer research at the NCI. Please do 

not hesitate to contact me for additional information. 

Enclosure 

Sincerely, 

Anna D. Barker, Ph.D. 

Deputy Director 

National Cancer Institute 
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How does the National Cancer Institute strike a balance between the more global 

approach to cancer versus the immediate needs of deadly cancers, like pancreatic? 

The NCr strives to maintain an overall portfolio that includes: (l) the best of the basic 

research that will yield data and scientific insights relevant to all cancers, and (2) 

significant investments in translational and clinical research to ensure that laboratory 

advances reach patients, Both are important to ensure that we continue to build a 

fundamental understanding of cancer, while ensuring that patients with cancer receive the 

best of what evidence based c,ancer medicine can deliver. 

Cancer is an extraordinarily complex collection of over 200 diseases that share a number 

of common hallmarks. In the past decade, our understanding of the molecular basis of 

cancer has evolved rapidly to a point where highly lethal cancers such as pancreatic 

benefit directly or indirectly from most if not all of the research that comprise the NCI's 

overall portfolio. Specifically, the highly lethal cancers have beeome a major focus for, 

and beneficiary of, several NCI strategic initiatives such as The Cancer Genome Atlas 

(TCGA) and the Nanotechnology Alliance for Cancer. 

Advances in technology now make it possible to obtain comprehensive genomic 

infonnation from multiple tumor types to catalog most, if not all, ofthe genomic changes 

associated with a specific cancer. To capitalize on this unprecedented opportunity, the 

NCI and the National Human Genome Research Institute (NHGRI) undertook a 3 -year 

pilot program to detennine the feasibility of developing an "atlas" of all of the relevant 

genomic changes in most cancer. After successfully completing the TCGA Pilot 

Program in 2009, TCGA was expanded to a five-year large scale, high throughput 

program that is designed to comprehensively profile all of the significant genomic 

changes in 20-25 cancers and make the data publicly available, The integrated TCGA 

network of 17 centers operates as a "pipeline" that includes acquisition of tumor and 

normal samples, extraction of the DNA and RNA, genome characterization and 

sequencing and data deposition and analysis. TCGA is a paradigm shifting program that 

will ultimately benefit all cancers - especially the rare tumors, 

For example, TCGA is rapidly characterizing the genomic alterations in rare tumors such 

as glioblastoma multifonne (most common of the adult brain tumors), ovarian and lung 

cancer and acute myelogenous leukemia to name a few. The standard of care for a 

disease such as pancreatic cancer presents a major challenge in terms of acquiring the 

needed samples, but TCGA is proceeding on two fronts to ensure that the genomics of 

this lethal tumor are fully characterized. TCGA is collecting samples for evaluation by 

the U.S. TCGA team - and in parallel is working with members of the International 

Cancer Genome Consortium to ensure that the ranges of subtypes of this tumor are 

studied. 

Results from TCGA are already allowing scientists to define tumor subtypes - paving the 

way for significant improvements in therapy. In addition, biomarkers that will evolve 

from an understanding of the changes in the genomes from specific cancers will drive the 
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design of new clinical trials for targeted therapies, more specific diagnostics for earlier 

detection and ultimately usher in a new era of personalized cancer medicine for all 

cancers. 

TCGA is a powerful example of how publically available, multidimensional data from 

high quality samples can change the future for patients with deadly tumors such as 

glioblastoma and ovarian cancers. The data are attracting numerous basic and clinical 

scientists from across all of cancer research to investigate the mechanisms that drive 

these diseases - and more importantly to use this new knowledge to deliver better 

interventions to patients. TCGA investigators will be analyzing a number of tumor types 

in the coming year, including pancreatic cancer. It is anticipated that these data along 

with those from the international cfforts will drivc unprecedented insights into the disease 

and enable the development of more strategic and targeted approaches to diagnosis and 

treatment. 

Beyond the TCGA example, the Ncr is also aggressively pursuing a broad research effort 

for pancreatic and other rare cancers. In 2001, NCI convened the Pancreatic Cancer 

Progress Review Group (PRG) to identify priority areas for research. Since that time, 

NCT's support for pancreatic cancer research has grown from $21.8 million in fiscal year 

(FY) 2001 to $89.6 million in FY 2009. Part of this growth came about through plmmed 

actions and funding opportunities specific to pancreatic cancer, and part grew out of an 

increasingly larger pool of pancreatic cancer researchers successfully competing for 

general funding opportunities and unsolicited research grants. 

Finally, in the past 8 years, the number of investigators funded through the standard 

principal investigator-funding ROI awards has more than quadrupled, increasing from 34 

to 159. The total number of research awards with a pancreatic cancer focus has increased 

substantially since FY 2000, from 85 projects to 589 projects in FY 2009. 
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Given the likelihood of shrinking resources, how will you sustain NCl's focus and 

commitment on basic research? 

The NCI spends about half of its total budget on basic research. This type of research 

contributes to our knowledge of the underlying biology of cancer, and it is essential that 

we continue our investment in basic research to inform our translational research efforts. 

In developing its budget, the NCI plans for research that will address critical unanswered 

questions about many types of cancer. More detailed information about the NCI's 

research priorities and its requested budget for Fiscal Year 2011 can be found in "The 

Nation's Investment in Cancer Research: Connecting the Nation's Cancer Community," 

which is available at http://plan.cancer.gov/ on the internet. 

In determining research and funding priorities, we have found that there is no simple 

correct formula for allocating funds. The NCI carries out a number of priority-setting 

activities to ensure that they are responsive to new discoveries and opportunities and can 

make the best use of resources. NCI leaders and staff work closely with researchers and 

representatives from the scientific, medical, and advocacy communities to identify 

research opportunities and resource needs - and determine how to best move the science 

forward. Some specific priority-setting activities include assembling State-of-theScience Meetings to discuss priorities for clinical trials, as well as frequent calls to the 

community for suggestions in broad scientific, public health emphasis, or capacitybuilding areas. 

In addition, with the completion of the sequencing of the human genome, it became 

possible to identify changes in the genomes from cancer patients with the normal 

sequence. However, to drive a new era of discovery and ensure that we develop an 

understanding of the molecular basis of every cancer, new innovati ve programs are 

required to provide basic scientists with the data they need to determine the mechanisms 

that drive cancer. Programs such as TCGA are making these data available to all 

scientists and it is already making a difference in what individual scientists and teams are 

able to accomplish. The co-existence of basic individual initiated research and large team 

science programs that provide data and knowledge for all of the communities to employ 

to generate hypotheses and ask bigger questions about cancer is one ofthe best ways to 

sustain momentum in basic research - and ensure its support. Innovation and discovery 

will always be the foundation of the NCI's research portfolio - and this new era holds 

unprecedented promise for basic research to flourish in a partnership with team science 

programs that will finally lead to a fundamental understanding of cancer. 
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DEPARTMENT OF HEALTH & HUMAN SERVICES 

APR 29 Z010 

The Honorable Mike Rogers 

House of Representatives 

Washington, DL 20515 

Dear Mr. Rogers: 

Public Health Service 

National Institutes of Health 

Bethesda, Maryland 20892 

Thank you for the opportunity to testify at the March 23, 2010 hearing entitled "NCI 

Cancer Research: Today's Progress; Tomorrow's Challenges" before the Committee on 

Energy and Commerce Subcommittee on Health. 

I appreciate your interest in the research being conducted at the National Cancer Institute. 

The enclosed responses to your question for the record provide additional information on 

our strategies for addressing the many challenges we face 10 cancer research. 

Thank you again for your interest and support of cancer research at the NCl. Please do 

not hesitate to contact me for additional information. 

Enclosure 

Sincerely, 

~-Arum D. Barker, Ph.D. 

Dcputy Director 

National Cancer Institute 
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The Cancer Genome Atlas holds great promise, but it will take time to produce the 

answers necessary to conquer some of the deadliest cancers. What can NCI do now 

to address pancreatic and other deadly cancers where there has been little progress 

in survival rates? 

NCI is committed to aggressively pursuing a broad research effort for pancreatic and 

other rare cancers. In 2001, NCI convened a Pancreatic Cancer Progress Review Group 

(PRG) to identify priority areas for research. Since that time, NCl's support for 

pancreatic cancer research has grown significantly. Based on the recommendations in the 

PRG Report, NCI expanded its portfolio of pancreatic cancer research from $21.8 million 

in fiscal year (FY) 2001 to $89.6 million in FY 2009. Part oftbs growth came about 

through planned actions and funding opportunities specific to pancreatic cancer, and part 

grew oUl of an increasingly larger pool of pancreatic cancer researchers successfully 

competing for general funding opportunities and unsolicited research grants. 

In the past 8 years, the number of investigators funded through the standard principal 

investigator-funding ROl awards has more than quadrupled, increasing from 34 to 159. 

The total number of research awards with a pancreatic cancer focus has increased 

substantially since FY 2000, from 85 projects to 589 projects in FY 2009, 

Early detection is also a critical challenge for pancreatic cancer, but NCI is working on 

many fronts to advance early detection, Specifically, early detection is a major focus of 

the NCl's Nanotechnology Alliance for Cancer. This program leverages the properties of 

matter at the nano scale to build and multiplex new technologies with unprecedented 

capacity to detect, treat and prevent cancer. One such example is the work of Dr. Chad 

Mirkin of Northwestern University, who has developed a gold nanoparticlc based 

biobarcode assay to measure changes in biomarkers in the blood of cancer patients. This 

assay, already submitted for regulatory approval, has protein detection sensitivity up to 

six orders of magnitude higher than standard ELISA assays and the potential for broad 

application in the detection of a number of cancers such as pancreatic cancer. 

NCI is also addressing pancreatic cancer along with rare cancers such as other 

gastrointestinal cancers. Recently, as part of the restructuring ofthe NCI Clinical Trials 

Enterprise, NCI fonned the Gastrointestinal Intergroup. Pancreatic cancer is one ofthe 

gastrointestinal cancers that the group will be looking at as they hannonize an efficient, 

cost-effective, science-driven, and transparent process that will identifY and promote the 

"Best Science" in gastrointestinal cancer clinical research by addressing the design and 

prioritization oflarge phase II studies and phase III trials in these cancers. 

The Cancer Genome Atlas (TCGA) can also playa critical role in advancing pancreatic 

cancer research, and research on other rare cancers. TCGA has already begun to make 

significant advances on rare cancers, The first three cancer types mapped by TCGA are 

all considered rare cancers: brain (glioblastoma multiforme), lung (squamous carcinoma) 

and ovarian (serous cystadenocarcinoma). Pancreatic cancer tissue samples, along with 
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other types of gastrointestinal cancers, are currently being collected for potential study 

through TCGA. 

After successfully completing the TCGA Pilot Program in 2009, TCGA was expanded to 

a five-year large scale, high throughput program that is designed to comprehensively 

profile all of the significant genomic changes in 20-25 cancers and make tile data publicly 

available. The integrated TCGA network of 17 centers operates as a "pipeline" that 

includes acquisition of tumor and normal samples, extraction of the DNA and RNA, 

genome characterization and sequencing and data deposition and analysis. TCGA is a 

paradigm shifting program that will ultimately benefit all cancers - especially the rare 

tumors. 

For example, TCGA is rapidly characterizing the genomic alterations in rare tumors such 

as glioblastoma multiforme (most common ofthe adult brain tumors), ovarian and lung 

cancer and acute myelogenous leukemia to name a few. The standard of care for a 

disease such as pancreatic cancer presents a major challenge in terms of acquirffig the 

needed samples, but TCGA is proceeding on two fronts to ensure that the genomics of 

this lethal tumor are fully characterized. TCGA is collecting samples for evaluation by 

the U.S. TCGA team - and in parallel is working with members of the International 

Cancer Genome Consortium to ensure that the ranges of subtypes of this tumor are 

studied. 

Results from TCGA are already allowing scientists to define tumor subtypes - paving the 

way for significant improvements in therapy. TCGA is already making these types of 

discoveries for glioblastoma, a deadly cancer and the most common malignant brain 

tumor in adults. TCGA research revealed two new molecular subtypes of glioblastoma 

and con finned two previously known subtypes. The findings indicate at least four distinct 

forms that are recognizable by their genetic signatures, and that the responses to 

aggressive therapies for glioblastoma vary by subtype. Based on the subtypes, paticnts 

can be identified in a logical manner and eventually receive therapies tailored for more 

individualized care. TCGA's work on glioblastoma is a model for defining the molecular 

basis of pancreatic and other rare cancers. 

In addition, biomarkers that will evolve frOID the an understanding of the changes in the 

genomes from specific cancers will drive the design of new clinical trials for targeted 

therapies, more specific diagnostics for earlier detection and ultimately usher in a new era 

of personalized cancer medicine for all cancers. 

TCGA is a powerful example of how publicaJly available, multidimensional data from 

high quality samples can change the future for patients with deadly tumors such as such 

as glioblastoma and ovarian cancers. The data are attracting numerous basic and clinical 

scientists from across all of cancer research to investigate the mechanisms that drive 

these diseases ~~ and more importantly to use this new knowledge to deliver better 

interventions to patients. TCGA investigators will be analyzing a number of tumor types 

in the coming year, including pancreatic cancer. It is anticipated that these data along 

with those from the international efforts will drive unprecedented insights into the disease 
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and enable the development of more strategic and targeted approaches to diagnosis and 

treatment. 
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Dear Chairmm-:. \\'Hxman: 

Question 1: ~,you described hyO strategies to 

research community. Can you elaborate 01] 

Stall;] Cn To Cancer (SL2C) rvlodci: 

The Cancer 

Institute of 

New Jersey" 

Bringing research tife 

Office 

:Vlarch 23, 20) 0, 

To Cancer hU'gc grants 

multiple l]1~tl-tution~ representing pivotal areas t,f 

,'",u"'-"'''o,C" one year 3g('. S1:2C mvardcd the first round of three-year 

discir,11l1carc), multi-institutional :-escarch D:-cal1; Teams. ti\"e Dream Tcams arc 

resc<llThers irom over 20 leading instilutions. 

195 Litt1!t Albany StrL'Ct ., New Bntnsv.ick, Nev.-Jersey 08901,2681 ,. Phone: 732.235.8064 * Fax: 712.235.8094 ~ 
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Page 2 

On hehalf (if SL2C. responsible 

leaders' institutions, 

through progran1 

pwgress during the funding period. and the ScieJ1titlc 

Ad\'lsory Cr!l1unittee conduct periodic rcvle\,vs ensure that n1ilestoncs and objectives 

achicycd. 1nade pubhc at 'W\v\v.su2,,;.org and W\V\V,aaCf,Orv" 

cancer research leaders. and \\lith 

data sharing~ fosters collaboration 

beginning. unique Dream 

interests of both today's cancer the future. 

eulturc of c()llaboratiol1 is hallmark of "CI-designated Cancer Centers. Mechanism;; such 

collahl'ration within );CI-designated Cancer Centers Cancer Center n;scarch programs. 

shared \:CI p:"ogranls such Specialized Programs of Research 

Excellence (SPORE). multi-investigator grants. and multi-center consortium Programs 

"lCI-designated Centers usually include multiple memhers on 

population research. );CI-dcsignatcd Centers add 

collaboration each program 

progra111nlatic cnllabora11on, resuhing in coHahorativc subnl1ssJ.or\S Bnd publlc:ations, intraand coiluboration between faculty cor:oucting research 

{(lsters translational efforts, Centers support sha-:-cd resources of 

both individual research and 

that focus type. SPORE investigators 

"rno'""'~' that may cancer 

SPORE programs 

collabc-ra.tions, 
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111ct..'hanjsrn that fosters collaboration is establishment 

its academic partners 

industry?" 

:nc1ude and rt~gulatory nnv,-',c,,', 

negotiations .. and creating precolnpetitlvc pO(lls 

Advanl'ed T iOchnology Partl1iOfships Initiative (A TrI), 

research lJY 

sciences COlTI!D3l11C:S, 
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Page 
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EXECUTIVE COMMlTTEE 

EUen V. Sigal. Ph.D. 

"AOI!ene A. Malek, R.N. 

Researcll 

BOARD OF DIRECTORS 

WilliamP. Ero 

Kidney ConCRf Associolion 

f-oun:lution 

Ronald 8. Herberman, M.D. 

CMO.intrexon Corrorollon 

Mary Woolley 

Reseorch!America 

Robert C. Young. MD 

RCYMedrcine 

Alston & Bird 

April 28, 2010 

The Honorable Henry A. Waxman. 

Committee on Energy and Commerce 

2125 Rayburn House Office Building 

Washington, DC 20515 

Dear Chairman Waxman: 

Thank you for the questions you submitted as follow up to the March 23, 2010 hearing, 

"NCI Cancer Research: Today's Progress; Tomorrow's Challenges." 

Attached, please find my written responses for the record. If there are any further 

questions you may have, please do not hesitate to reach out, we would be happy to 

provide further information or clarification. 

We look forward to continuing to work with you and your staff to address these vital 

issues. 

Sincerely, 

Jeff Allen, Ph.D. 

Executive Director 

Friends of Cancer Research 

2231 Crystal Drive, Suite 200, Arlington. Virginia 22202 I Phone 703.302.1503 i Fax 703.302.1568 I www.focr.org 
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1. You called for a unified, holistic approach to combating cancer. You also stated cancer 

research should focus on the classification and study of molecular characteristics of various 

forms of cancer. And then discoveries from this research would inform the development of 

targeted treatment. Both you and Dr. Barker mentioned the drug Gleevec as a classic example 

of a safe and effective treatment that resulted from this approach. I understand part of what 

made this discovery so ground breaking is that Gleevec received FDA approval in a record two 

months. 

Can you elaborate on how the research leading to Gleevec embodies the approach you believe 

cancer research should take? 

Historically, many cancers were treated by general chemotherapy which functions by inducing cellular 

toxicity in attempt to cause highly proliferative cancer cells to die. Because chemotherapy drugs attack 

healthy cells as well as cancerous cells. side effects are often quite difficult for patients to endure. 

Through significant progress made in biomedical research, we now have improved understanding of 

the characteristics unique to cancerous cells. This information has provided numerous molecular 

targets that have given us the ability to develop treatments that only the cancerous cells are impacted. 

For example, two genes that are unrelated in normal cells, were discovered to be mutated and fused 

together in patients with chronic myelogenous leukemia (CML).' The product of this gene fusion, 

called BCR/Abl, results in CML because cells expressing this mutation grow in an uncontrolled 

manner.' The mutated protein (BCR/Abl) can be a target to distinguish cancerous cells from normal 

cells, and if the action of the mutant protein can be blocked, then cancerous growth is stopped WITh 

relatively few side effects. This is how Gleevec works to impede CML.3 The results have been 

astounding and has transformed CML from a disease that had a fatality rate of one hundred percent to 

a ninety-five percent five year survival rate4 

It should be noted that, while Gleevec received FDA approval in 2002 after only 10 weeks of review, it 

took decades of research and hundreds of millions of dollars, to identify, characterize, and develop a 

drug toward the BCR/Abl target. Research programs like The Cancer Genome Atlas (TCGA) aim to 

identify many new potential targets unique to cancer cells. Researchers and patients remain hopeful 

that the identification of new targets will result in new treatments that will positively impact multiple 

types of cancer. For instance, while the original approval of Gleevec was for the treatment of CML, it 

has since been shown to have positive results in the treatment of a rare solid tumor, known as 

gastrointestinal stromal tumor (GIST).5 This positive activity occurs because the protein inhibition 

achieved by the use of Gleevec can occur in other cell types with similar abnormalities. Efficacy in 

multiple tumor types has been demonstrated with other targeted anti-cancer treatments as well. 

This observation of common cellular malfunctions in different cancer types has led researchers to 

pursue new treatments based on molecular pathways, as opposed to solely examining treatment 

strategies according to the tumor site. While targeted drugs are generally more efficient, effective, and 

less toxic, the development and application of such products is not without challenge. 

1 Rowley JD. Letter: a new consistent chromosoma! abnormality in chronic myelogenous leukaemia identified by quinacrine 

fluorescence and Giemsa staining. Nature. 1973;243290-293. 

2 Hochhaus A, Weisser A, La Rosee P, Emig M, Muller MC, Saussele S, Reiter A, Kuhn C. Berger U, Hehlmann R. Cross NC. 

Detection and quantification of residua! disease in chronic myelogenous leukemia. Leukemia, 2000;14:998-1005 

:1 Kanta~ian H, Talpaz M, et al. High-dose !matinib mesylate therapy in newly diagnosed Philadelphia chromosome-positive 

chronic phase chronic mye!oid leukemia. Blood. 2004;103:2873-2878. 

4Druker BJ, Guithot F, et al Five-year follow-up of patlents receiving imatinib for chronic myelOid leukemIa. N Engl J Med. 2006 Dec 

7;355(23):2408-17. 

:; Blanke CO, Oemetri GO, von Mehren M, et aJ. Long-term results from a randomized phase II trial of standard-versus higher-dose 

imatinib mesylate for patients with unresectable or metastatic gastrointestinal stromal tumors expresSing KIT. J elm Oneol. 

2008;26(4) 620-625. 

2 2231 Crystal Drive, Suite 200, Arlington. Virginia 22202 I Phone 703.302.1503 I fax 703.302.1568 : www.focr.org 
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The use of a targeted treatment may require a diagnostic test to identify the absence or expression of a 

protein, like BCRlAbl. In many diseases this is becoming increasingly complex. For example, breast 

cancers can be sub-characterized based on their expression of different protein receptors. Roughly 

60% of patients are estrogen receptor positive (ER+), 25% of tumors over express a protein called 

Her2 (Her2+), and 15% are identified as triple negative breast cancer (ER-, Her2-, and progesterone 

negative). These three subtypes include tumors that have additional mutations that impact the efficacy 

of current treatment options. 

The use of stratified or "personalized" medicine holds great hope for bettering the lives of patients 

through by delivering the right treatment, to the right patients, at the right time. 

2. You and Dr. DiPaola described the need for improved collaboration to ensure that promising 

scientific advances made by NCI and others actually translate to safe and effective treatments. I 

want to ask you about the three types of collaboration you discussed in your testimony: 

collaboration within the biomedical research community, interagency collaboration, and 

collaboration with industry partners. 

a. I'd like to ask you about collaboration with FDA. What can NCI, and NIH as a whole, do to 

build upon its relationship with its sister agency? 

As the fields of medical product development and regulation become increasingly complex, a stronger, 

collaborative relationship between the NIH and FDA is vital to continued progress and will benefit the 

mission of both agencies. While in some areas there are strong working relationship between FDA 

and specific NIH Institutes and Centers (ICs), the collaboration must be strengthened at multiple levels. 

A step in the right direction was the February 23, 2010 announcement by Secretary of Health and 

Human Services Kathleen Sebelius of the formation of a joint FDA-NIH Leadership Council consisting 

of top officials from both agencies. The Leadership Council has the potential to serve as model for 

identifying shared scientific priorities and coordination of trans-agency initiatives. 

In order to implement the priorities of the Leadership Council, working task forces between specific ICs 

and FDA divisions must be strengthened. This would allow and encourage utilization of the sister 

agency as a scientific resource for interaction on specific product applications, clinical trial and protocol 

design, and jointly developed studies. Additionally, to foster on-going collaboration between the 

agencies and the development of a well-rounded, expert work force, programs should be established 

for the joint training and development of FDA and NIH staff. An example of this has been developed 

through the NCI-FDA Interagency Oncology Taskforce6 While this taskforce has successfully 

developed cross-agency fellowship programs, other activities and potential scientific initiatives have 

been limited by a lack of resources. 

In addition to enhancing the interactions between NIH/NCI and FDA, increased collaboration across all 

federal health related agencies is vital to realize the full potential of cancer research. Through 

collaboration we can streamline cancer research, detection, treatment, prevention, surveillance, 

product regulation, care delivery, reimbursement of services, and learn from all of these cancer-related 

functions as a routine by-product of care. This will ultimately lead to increased survivorship, reduction 

of cancer incidence and mortality, and improvement of the lives of all who may face this disease. 

The challenges of establishing a collaborative, enduring relationship between federal health agencies 

and NIH/NCI are not insignificant. The core mandate of each agency is rightfully quite different, the 

organizational structures are not common, and there is, at times, resistance to modifications to current 

practices displayed by each based on professional experience and long standing cultural differences. 

(, NCt-FDA Interagency Oncology Taskforce: http://otir cancer.gov/programs/partnerships ioti.£§g Accessed 4/27/10 

2231 Crystal Drive, Suite 200, Arlington, Virginlo 22202 I Phone 703.302.1503 I Fox 703.302.1568 1 www.!ocr.org 

3 
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However, the common goal of improving public health must trump these barriers and should be 

embraced and promoted through scientific collaboration, 

The NCI drives the advancement of cancer research, but in order to truly impact this disease a 

comprehensive approach is needed, In 1971, the National Cancer Act was passed and provided the 

NCI Director, with the advice of the National Cancer advisory Board, the authority to "plan and develop 

an expanded, intensified, and coordinated cancer research program encompassing the programs of 

the National Cancer Institute, related programs of the other research institutes, and other Federal and 

non-Federal programs,'" As a part of this authority, the NCI regularly outlines their research portfolio 

and the broad plans for the National Cancer Program as a part of their annual report and budget 

estimate. However, many of the oncology activities that occur as routine functions of other health 

related federal agencies are not done in direct collaboration with the NCL This is not to imply that all 

cancer-related activities of the federal government should occur at the NCI, but rather that the scientific 

data and expertise housed at multiple agencies should be available and capitalized on by sister 

agencies. Each agency needs to ultimately retain their independence and autonomy, but improved 

coordination at all levels of cancer discovery, product regulation, delivery of services and surveillance 

will synergistically advance the field of oncology, Multi-agency collaboration for cancer research should 

not be delayed, We must tear down the silos that exist between all government agencies that aim to 

conquer cancer. 

b. How can NCI and its academic partners improve collaboration with industry? 

Partnerships with organizations outside of the federal government have an important role in providing 

access to technologies, resources, and data that may not be possible for duplication within the 

government and offer valuable additional perspectives to many scientific discussions, Therefore, it is 

important that external partnerships and collaborations with academic centers, industry, patient and 

advocacy groups continue to be fostered, Engaging external stakeholders and improving transparency 

will help provide public confidence in the decisions that are ultimately made. Effective partnerships 

have been developed between federal agencies and non-government entities, often via public-private 

partnerships like those at the Foundation for the NIH (FNIH) and potentially through the Reagan-Udall 

Foundation for the FDA. 

As I mentioned in my testimony, just last rnonth, the Biomarker Consortium, a public-private 

partnership through the Foundation for the NIH, announced the opening of the I-SPY2 TRIAl. This 

clinical trial will utilize an innovative new model that use biomarkers from individual patients' tumors to 

screen promising new treatments for breast cancer and identify which treatments are most effective in 

specific types of patients· 

This type of coordination will require a significant commitment from the leadership of multiple agencies 

and organizations to coordinate on-going activities, and demonstrate the importance of collaboration at 

various levels. Additionally, considerable resources will be needed to support meaningful projects 

designed to advance the common goal of improving the health of the American people, 

7 National Cancer Act of 1971. PL 92-218 

9 I SPY 2 TRIAL (Investigation of Serial Studies to Predict Your Therapeutic Response with Imaging And molecular Analysis 2)' 

http://ispy2 org! Accessed 4/27/10 

2231 Crystal Drive, Suite 200, Arlington. Virginia 22202 ! Phone 703.302,1503 i Fax 703.302.1568 I www.focr.org 

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