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POLICIES TO SPUR INNOVATIVE MEDICAL 

BREAKTHROUGHS FROM LABORATORIES 

TO PATIENTS 

HEARING 

BEFORE THE

SUBCOMMITTEE ON RESEARCH AND TECHNOLOGY 

COMMITTEE ON SCIENCE, SPACE, AND 

TECHNOLOGY 

HOUSE OF REPRESENTATIVES 

ONE HUNDRED THIRTEENTH CONGRESS 

SECOND SESSION 

JULY 17, 2014 

Serial No. 113–87 

Printed for the use of the Committee on Science, Space, and Technology 

( 

Available via the World Wide Web: http://science.house.gov 
(II) 

COMMITTEE ON SCIENCE, SPACE, AND TECHNOLOGY 

HON. LAMAR S. SMITH, Texas, Chair 

DANA ROHRABACHER, California 

RALPH M. HALL, Texas 

F. JAMES SENSENBRENNER, JR., 

Wisconsin 

FRANK D. LUCAS, Oklahoma 

RANDY NEUGEBAUER, Texas 

MICHAEL T. MCCAUL, Texas 

PAUL C. BROUN, Georgia 

STEVEN M. PALAZZO, Mississippi 

MO BROOKS, Alabama 

RANDY HULTGREN, Illinois 

LARRY BUCSHON, Indiana 

STEVE STOCKMAN, Texas 

BILL POSEY, Florida 

CYNTHIA LUMMIS, Wyoming 

DAVID SCHWEIKERT, Arizona 

THOMAS MASSIE, Kentucky 

KEVIN CRAMER, North Dakota 

JIM BRIDENSTINE, Oklahoma 

RANDY WEBER, Texas 

CHRIS COLLINS, New York 

BILL JOHNSON, Ohio 

EDDIE BERNICE JOHNSON, Texas 

ZOE LOFGREN, California 

DANIEL LIPINSKI, Illinois 

DONNA F. EDWARDS, Maryland 

FREDERICA S. WILSON, Florida 

SUZANNE BONAMICI, Oregon 

ERIC SWALWELL, California 

DAN MAFFEI, New York 

ALAN GRAYSON, Florida 

JOSEPH KENNEDY III, Massachusetts 

SCOTT PETERS, California 

DEREK KILMER, Washington 

AMI BERA, California 

ELIZABETH ESTY, Connecticut 

MARC VEASEY, Texas 

JULIA BROWNLEY, California 

ROBIN KELLY, Illinois 

KATHERINE CLARK, Massachusetts 

SUBCOMMITTEE ON RESEARCH AND TECHNOLOGY

HON. LARRY BUCSHON, Indiana, Chair 

STEVEN M. PALAZZO, Mississippi 

MO BROOKS, Alabama 

RANDY HULTGREN, Illinois 

STEVE STOCKMAN, Texas 

CYNTHIA LUMMIS, Wyoming 

DAVID SCHWEIKERT, Arizona 

THOMAS MASSIE, Kentucky 

JIM BRIDENSTINE, Oklahoma 

CHRIS COLLINS, New York 

BILL JOHNSON, Ohio 

LAMAR S. SMITH, Texas 

DANIEL LIPINSKI, Illinois 

FEDERICA WILSON, Florida 

ZOE LOFGREN, California 

SCOTT PETERS, California 

AMI BERA, California 

DEREK KILMER, Washington 

ELIZABETH ESTY, Connecticut 

ROBIN KELLY, Illinois 

EDDIE BERNICE JOHNSON, Texas 
(III) 

C O N T E N T S 

July 17, 2014 

Page 

Witness List ............................................................................................................. 2 

Hearing Charter ...................................................................................................... 3 

Opening Statements 

Statement by Representative Larry Bucshon, Chairman, Subcommittee on 

Research and Technology, Committee on Science, Space, and Technology, 

U.S. House of Representatives ............................................................................ 7 

Written Statement ............................................................................................ 9 

Statement by Representative Daniel Lipinski, Ranking Minority Member, 

Subcommittee on Research and Technology, Committee on Science, Space, 

and Technology, U.S. House of Representatives ............................................... 11 

Written Statement ............................................................................................ 13 

Statement by Representative Lamar S. Smith, Chairman, Committee on 

Science, Space, and Technology, U.S. House of Representatives ..................... 15 

Written Statement ............................................................................................ 16 

Witnesses: 

Dr. Harold Varmus, Director, National Cancer Institute (NCI) at the National 

Institutes of Health (NIH) 

Oral Statement ................................................................................................. 18 

Submitted Biography ....................................................................................... 21 

Dr. Marc Tessier-Lavigne, President and Carson Family Professor, Laboratory 

of Brain Development and Repair, The Rockefeller University 

Oral Statement ................................................................................................. 30 

Written Statement ............................................................................................ 32 

Dr. Jay Keasling, Hubbard Howe Jr. Distinguished Professor of Biochemical 

Engineering, University of California, Berkeley; Professor, Department of 

Chemical & Biomolecular Engineering, University of California, Berkeley; 

Professor Department of Bioengineering, University of California, Berkeley; 

Director, Synthetic Biology Engineering Research Center 

Oral Statement ................................................................................................. 39 

Written Statement ............................................................................................ 41 

Dr. Craig Venter, Founder, Chairman, and Chief Executive Officer, J. Craig 

Venter Institute, Synthetic Genomics, Inc., and Human Longevity, Inc. 

Oral Statement ................................................................................................. 46 

Written Statement ............................................................................................ 48 

Discussion ................................................................................................................. 57 

Appendix I: Answers to Post-Hearing Questions 

Dr. Harold Varmus, Director, National Cancer Institute (NCI) at the National 

Institutes of Health (NIH) ................................................................................... 70 

Dr. Marc Tessier-Lavigne, President and Carson Family Professor, Laboratory 

of Brain Development and Repair, The Rockefeller University ....................... 78 
Page

IV 

Dr. Jay Keasling, Hubbard Howe Jr. Distinguished Professor of Biochemical 

Engineering, University of California, Berkeley; Professor, Department of 

Chemical & Biomolecular Engineering, University of California, Berkeley; 

Professor Department of Bioengineering, University of California, Berkeley; 

Director, Synthetic Biology Engineering Research Center ............................... 81 

Dr. Craig Venter, Founder, Chairman, and Chief Executive Officer, J. Craig 

Venter Institute, Synthetic Genomics, Inc., and Human Longevity, Inc. ....... 83 

Appendix II: Additional Material for the Record 

Statement submitted by Representative Eddie Bernice Johnson, Ranking 

Member, Committee on Science, Space, and Technology, U.S. House of 

Representatives .................................................................................................... 88 

Letter submitted by Representative Larry Bucshon, Chairman, Subcommittee 

on Research and Technology, Committee on Science, Space, and Technology, 

U.S. House of Representatives ............................................................................ 90 

Article submitted by Representative Dana Rohrabacher, Committee on 

Science, Space, and Technology, U.S. House of Representatives ..................... 91 
(1) 

POLICIES TO SPUR INNOVATIVE MEDICAL 

BREAKTHROUGHS FROM LABORATORIES 

TO PATIENTS 

THURSDAY, JULY 17, 2014 

HOUSE OF REPRESENTATIVES, SUBCOMMITTEE ON RESEARCH AND TECHNOLOGY, COMMITTEE ON SCIENCE, SPACE, AND TECHNOLOGY, Washington, D.C. 

The Subcommittee met, pursuant to call, at 9:05 a.m., in Room 

2318 of the Rayburn House Office Building, Hon. Larry Bucshon 

[Chairman of the Subcommittee] presiding. 
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Chairman BUCSHON. Subcommittee on Research and Technology 

will come to order. Good morning. Welcome to today’s hearing, entitled ‘‘Policies to Spur Innovative Medical Breakthroughs from Laboratories to Patients.’’ I recognize myself for five minutes for an 

opening statement. 

As a cardiothoracic surgeon and medical professional, I know 

firsthand there are many complexities surrounding the human 

body, and understanding human disease is one of the most challenging problems facing the scientific and medical communities. 

Complex human diseases will likely require an interdisciplinary 

and multifaceted approach, with the right scientific questions being 

asked and debated, with clear goals and endpoints being articulated. 

The creative drive of American science is the individual investigator, and I have faith they will continue to tackle, understand, 

and contribute original approaches to these problems. Medical diseases such as cancer, Alzheimer’s, Parkinson’s, autism, epilepsy, 

dementia, stroke, and traumatic brain injury have an enormous impact, and enormous economic impact, and personal impact for affected Americans. For example, Alzheimer’s disease is a severe 

form of dementia, and the sixth leading cause of death in the U.S. 

It affects both the 5.1 million Americans that have the disease and 

their friends and family, who must watch their loved ones suffer 

from its symptoms. The average annual cost of care for people with 

dementia over 70 in the U.S. is roughly between 157 and $210 billion in 2010. 

I want to stress my support for medical science research, in particular understanding diseases from an interdisciplinary perspective. As our witnesses will testify today, medical science has benefitted enormously from fields as diverse as applied mathematics, 

computer science, physics, engineering, molecular biology, and 

chemistry. 

More important basic science research results from NSF funded 

research will be the future experimental tools for a hypothesisbased, data-driven research for brain science researchers. I also see 

this as an important opportunity for continuing interdisciplinary 

work between the various federal science agencies, including NSF, 

NIST, and NIH, and I hope to see more collaboration and productive research opportunities. 

At the same time, I am interested in how private sector research 

can complement ongoing federal R&D investment, and what public 

policies may spur more innovation and investment from medical 

breakthroughs. Companies must carefully balance short term and 

long term interests of the company and their shareholders. Private 

sector research efforts use the results of basic science research in 

the physical, mathematical, and engineering sciences. For example, 

advances in computing have led to the development of software, 

with the goal of helping medical sciences make sense of cancer 

genomes. 

Watson, an advanced computer that was developed by IBM is 

being enlisted not only to identify mutations from a patient’s tumor 

biopsy in order to help understand how these mutations cause cancer, but also to produce a list of drugs that could potentially treat 
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the cancer. All of this can potentially be done in minutes, and I had 

a demonstration of Watson in my office. It was fascinating. 

Our witnesses today reflect the wide spectrum of research in the 

biomedical sciences, and each have been recognized in their respective fields. I would like to thank the witnesses for their being here 

today, and taking time to offer their perspectives on this important 

topic. I hope you will continue to work with us to maximize federal 

funding of biomedical research. I would also like to thank the 

Ranking Member, Mr. Lipinski, and everyone else participating in 

today’s hearing. 

[The prepared statement of Mr. Bucshon follows:] 
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Chairman BUCSHON. At this point now I recognize the Ranking 

Member, the gentleman from Illinois, Mr. Lipinski, for his opening 

statement. 

Mr. LIPINSKI. Thank you, Chairman Bucshon, for holding this 

hearing on policies to spur medical breakthroughs, something we 

all certainly want to do what we can here to make it as likely as 

possible to get those medical breakthroughs, and get them out to 

market and helping people. And I want to thank all of our witnesses for being here today. I look forward to your testimony. 

Innovation, whether in biomedical research or elsewhere, is an 

ecosystem that is more than the sum of its parts. Federal agencies, 

universities, and research institutions, entrepreneurs, and the private sector all have important roles to play. That is why I am glad 

we have witnesses from across these sectors here to testify today. 

In April we held a hearing in this committee on innovation prize 

competitions. We heard testimony about the need for a kidney prize 

to facilitate the development of more effective treatments for kidney disease and end stage renal disease. Innovation prizes, as well 

as other forms of pre-commercial support, such as proof of concept 

funding, and programs like NSF’s Innovation Corps, which recently 

announced a collaboration with NIH, could hold great promise for 

future biomedical breakthroughs. 

I hope that our panel could comment on these and other potential mechanisms for supporting technology transfer from the lab to 

the marketplace. And, of course, it bears repeating that our ability 

to innovate will be greatly limited without growing investments in 

the basic research that generates these technologies. 

The emerging field of engineering biology has grown out of the 

decades old field of genetic engineering. In the 1800s, Gregor Mendel established many of the rules of heredity that became the foundation of modern genetics by studying pea plants. But even before 

Mendel, farmers knew that by cross-breeding animals and plants 

you could favor certain traits. 

Since the 1970s, scientists have been using more advanced tools 

to directly insert new genes or delete genes from plant and 

microbio genomes. Engineering biology is the next step in this field, 

and is being accelerated by the development of technologies such 

as DNA sequencing, which has gone from taking years, and costing 

billions of dollars, to taking just days, and costing a few thousand 

dollars, which is truly amazing. 

We have already started seeing commercial applications from engineering biology. I look forward to hearing more about how Dr. 

Keasling and his research group were able to engineer a microorganism to produce a life-saving anti-malarial drug that is now being 

produced on a large scale by a pharmaceutical company. I also look 

forward to learning about other potential applications from engineering biology research, including energy, agriculture, chemicals, 

and manufacturing. 

Since this is an emerging field, and it could have significant economic benefit for the United States, it is important that we make 

the necessary federal investments in both the foundational research, and across the potential application areas. Several of the 

agencies under the Committee’s jurisdiction have significant programs in engineering biology. The Department of Energy has one 
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of the largest programs focused on bioenergy. The National Science 

Foundation is investing more in this area, both through individual 

research awards, and through their support of an engineering research center at Berkeley. NASA and NIST also have programs in 

this area. And, of course, NIH and the Department of Agriculture 

are significant players this research. 

The nation would benefit not just from increased investment at 

individual agencies, but also from coordination of federal efforts 

under some kind of plan or strategy. Other countries have identified this area specifically as an important area to make investments in. The European Union’s Europe 2020 strategy calls out 

this field as a key element as it develops a strategy and an action 

plan for investment. 

I have never seen that one happen before. I didn’t even run over 

time yet. 

Chairman BUCSHON. That was your warning. 

Mr. LIPINSKI. Don’t worry, I am almost done. I am concerned if 

the United States does not take the necessary steps, we will lose 

our leadership position in this field. That was symbolic of losing 

our leadership position, the lights going out. We should also ensure 

that we are facilitating public/private partnerships. Given the potential of commercial applications across nearly all sectors of our 

economy, there is a need to engage and encourage private sector 

collaboration at a pre-competitive level. And, finally, we must pay 

careful attention to issues of human environmental safety and ethics when it comes to engineering biology research, including by supporting research on those topics. 

I look forward to all witnesses’ testimony, and the Q&A. Thank 

you all for being here, and I yield back the balance of my time. 

[The prepared statement of Mr. Lipinski follows:] 
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Chairman BUCSHON. Thank you, Mr. Lipinski. I now recognize 

the Chairman of the full Committee, the gentleman from Texas, 

Mr. Smith, for his opening statement. 

Chairman SMITH. Thank you, Mr. Chairman. 

Basic biomedical research is increasingly interdisciplinary in nature. Advances in applied mathematics, physics chemistry, computer science, and engineering provide a better understanding of 

medical conditions, and the tools to help find cures. The National 

Science Foundation can play an important and vital role in understanding the basic science behind many debilitating conditions. 

For example, developments in basic scientific research have provided deep insight into how the brain and other neurological structures are organized. NSF research could help us better understand 

conditions such as cancer, Alzheimer’s, Parkinson’s, autism, stroke, 

dementia, traumatic brain injury, epilepsy, and many other disorders. Countless lives have unfortunately been lost to these diseases, and the economic impact, physical and emotional toll they 

can put on families can make them even more devastating. 

The National Science Foundation should support interdisciplinary research in conjunction with the National Institutes of Health 

to help us better understand medical illnesses. The Frontiers in Innovation, Research, Science and Technology Act, or FIRST Act, 

supports basic research that has the potential to improve the daily 

lives of millions of Americans. The FIRST Act increases funding for 

subjects such as math, physical sciences, biological sciences, computer sciences, and engineering for Fiscal Year 2015. 

The FIRST Act, which was successfully reported out of Com- mittee this past May, includes a $270 million increase for Fiscal 

Year 2015 over current NSF spending for these important subject 

areas. Federally funded basic research has supported the creation 

of technologies that have changed and improved our daily lives, including the MRI and laser technology. Efficient and effective use of 

NSF funding geared toward basic research will help us better understand medical conditions, and lead to medical breakthroughs 

that benefit both doctors and patients alike. 

Thank you, Mr. Chairman, for holding this hearing. And I want 

to say, at the risk of offending any other panel, we have an unusually distinguished panel of witnesses today, and we look forward to 

hearing from their testimony. 

[The prepared statement of Mr. Smith follows:] 
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Chairman BUCSHON. Thank you, Chairman. At this point, if 

there are other Members who wish to submit additional opening 

statements, your statements will be added to the record. 

[The prepared statement of Ms. Johnson appears in Appendix II] 

Chairman BUCSHON. At this time I would like to introduce our 

witnesses, and it is a distinguished panel. Thanks for being here. 

Our first witness is Dr. Harold Varmus, Director of the National 

Cancer Institute. He previously served for ten years as President 

of Memorial Sloan Kettering Cancer Center, and six years as Director of the National Institutes of Health. Dr. Varmus is a co-recipient of the Nobel Prize for studies on the genetic basis of cancer. 

Dr. Varmus was a co-chair of President Obama’s Council of Advisors on Science and Technology. And Dr. Varmus majored in 

English, which I found interesting, Literature at Amherst, and 

earned a Master’s Degree in English at Harvard, and is a graduate 

of Columbia University’s College of Physicians and Surgeons. Welcome. 

Our second witness is Dr. Marc Tessier-Lavigne. Did I say that 

right? President of the Rockefeller University, where he is also 

Carson Family Professor, and head of the laboratory of brain development and repair. Previously he served as Chief Scientific Officer 

of Genentech, a leading biotechnology company. He obtained undergraduate degrees from McGill and Oxford Universities, and a Ph.D. 

from the University College London, and was a post-doctoral fellow 

over there also, and at Columbia University. Prior to joining 

Genentech, he held faculty positions at the University of California 

San Francisco and at Stanford, where he was the Susan B. Ford 

Professor and Investigator with the Howard Hughes Medical Institute. Welcome. 

Our third witness is Dr. Jay Keasling, the Hubbard Howe Jr. 

Distinguished Professor of Chemical and Biomolecular engineering, 

and Professor of Bioengineering at the University of California at 

Berkeley. He is the Director of the Synthetic Biology Engineering 

Research Center, Associate Laboratory Director for Biosciences at 

the Lawrence Berkeley National Laboratory, and Chief Executive 

Officer of the Joint Bioenergy Institute. 

Dr. Keasling earned his Bachelor’s Degree from the University of 

Nebraska, and his graduate degrees in Chemical Engineering from 

the University of Michigan. In 2006 he was cited by Newsweek as 

one of the country’s 10 most esteemed biologists. Welcome. 

And our fourth witness is Dr. Craig Venter, Founder, Chairman, 

and Chief Executive of the J. Craig Venter Institute, Synthetic 

Genomics, Incorporated, and Human Longevity, Incorporated. Dr. 

Venter contributed to sequencing the first draft human genome in 

2001, the first complete diploid human genome in 2007, and the 

construction of the first synthetic bacterial cell in 2010. Dr. Venter 

is the recipient of the 2008 National Medal of Science. 

Dr. Venter earned both a Bachelor’s Degree in Biochemistry, and 

a Ph.D. in Physiology and Pharmacology from the University of 

California at San Diego. Thank you for being here. 

And, again, thanks to all our witnesses. It is a very impressive 

panel, and I think this is going to be a great hearing. As our witnesses should know, spoken testimony is limited to five minutes, 

after which the Members of the Committee will have five minutes 
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each to ask questions. I now recognize Dr. Varmus for five minutes 

to present his oral testimony. 

TESTIMONY OF DR. HAROLD VARMUS, DIRECTOR, 

NATIONAL CANCER INSTITUTE (NCI) 

AT THE NATIONAL INSTITUTES OF HEALTH (NIH) 

Dr. VARMUS. Chairman Bucshon, Chairman Smith, Mr. Lipinski, 

and other Committee Members, I thank you for your strong, supportive opening statements, and for holding this important hearing 

about the state of the American scientific enterprise. This is a pivotal moment. On the one hand, our investments in science and 

technology continue to lead the world. Our discoveries and applications of knowledge have enriched the country, improved the world, 

and expanded opportunities for yet further discover and application. 

But in recent years, we have been fiscally constrained. The place 

I work, the NIH, has lost 25 percent of its buying power over the 

last decade. We are able to support fewer than one in seven of our 

grant applications. In the meantime, other countries have quickened their pace of investment. Under these circumstances, the nation needs to determine how the parts of the enterprise can most 

effectively work together, and I take that to be the ultimate goal 

of this hearing. 

But that isn’t easy. The scientific landscape is complex, with at 

least four dimensions. First, many defined disciplines, which often 

intersect. Second, a spectrum of activities, from free ranging fundamental research, to more programmatic—or pragmatic efforts to 

use basic knowledge. Third, a variety of funding sources, including 

many government agencies, small and large companies, academic 

institutions, and private philanthropies. And fourth, several kinds 

of mechanisms to support research from each of our sources. Balancing these elements is of obvious interest to the Subommittee 

and to your witnesses. 

I would like to make four points about the landscape to help 

guide our discussion today. The first three are operating principles. 

The fourth illustrates some novel ways in which my agency, the 

NCI, has tried to increase our effectiveness. 

First, the importance of interdisciplinary work, which has already been alluded to. Historically, major advances in medicine 

have been especially dependent on physical sciences—on physics 

and chemistry. The body is an object that can be studied with those 

tools. Just consider microscopes, X-ray machines, radio isotopes, 

pharmacology, electrocardiograms, Mr. Bucshon, and the electroencephalogram. 

More recently, the studies of genomes that have been alluded to, 

proteins and cells, have revolutionized our understanding of normal 

and diseased human beings, thanks to inventions that required, 

again, physical, mathematics, engineering, and chemistry, as well 

as, importantly, computational science to handle the massive sets 

of data that we have accrued. Now newly launched initiatives, such 

as the President’s BRAIN project, or the NCI’s therapeutics efforts 

that are based on genetic signatures, so-called precision medicine, 

are going to require these in still other fields. In short, the future 
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of medicine will depend on maintaining the vibrancy and the interaction of allied fields of science and technology. 

Second point, sustained fundamental research is essential for 

further developments in medicine. Yet, when financial support is 

highly competitive, as is the case now, the choice of research 

projects veers toward applications of existing knowledge, and away 

from basic science, posing a serious risk to future productivity. 

I have mentioned that medicine is being transformed today by 

the unveiling of genetic blueprints, and by the identification of the 

specific damage that occurs in most human diseases, specifically 

like cancers. But discovery is not finished. Despite these enormous 

increases in knowledge, fundamental features of biological systems 

have yet to be discovered. We know this from some very recent examples, the discovery of unanticipated forms of RNA that perform 

functions other than its well-known roles in the synthesis of proteins, or the discoveries of enzymes from strange organisms that 

allow rapid and efficient re-engineering of genomes of many kinds 

of cells. Such unanticipated results and methods, and their subsequent applications, can come only from unfettered basic research. 

The third point is that funders of research had aimed for a balanced and synergistic portfolio. Each component of the scientific 

landscape has a limited range of action, and government science 

agencies, academic institutions, and some charities have a strong 

mandate to invest in fundamental science. 

Commercial entities are constrained from a deep commitment to 

unfettered basic research, but invest heavily in applied research, 

and these observations articulated over 70 years ago by Vannevar 

Bush have been the basis for the success of American science. But 

still, all these elements need to interact, and to learn where and 

how scarce resources are being committed, to engage in collaborative work, and to accelerate progress across the full spectrum of 

research and development. 

Finally, the fourth point, which I will ask for some indulgence 

just to describe briefly, leaders of funding agencies, especially in 

government, can help in the situation by using their various mechanisms to encourage interdisciplinary team science to protect investigators working in—on fundamental studies, and to work with our 

funding partners, especially in these fiscally challenging times. The 

NCI has exploited the flexibility of our funding mechanisms in a 

variety of ways that are listed in my written testimony, just to 

mention a few extremely briefly. 

The Cancer Genome Atlas Project has supported many hundreds 

of DNA sequences, geneticists, bioinformaticians, oncologists, and 

others to compile an extensive set of characteristics about over 20 

different time—kinds of human cancer in a way that is now transforming the way we approach cancer patients through precision 

medicine. 

Our Frederick National Laboratory for Cancer Research in Frederick, Maryland, a contract program modeled on—in part on the 

Department of Energy’s national programs, carries out both general service functions through nanotechnology, and clinical collaboration with 19 other agencies, and specific projects, like a project 

that addresses a collection of genes known as rash genes that drive 

about a third of human tumors. 
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And, finally, our provocative questions exercise is intended to 

bring scientists of many disciplines together to identify the great 

unsolved, and sometimes not closely attended to, problems in a way 

that now allows us to fund proposals to answer those questions. 

I will be pleased to answer any questions you might have. Thank 

you, Mr. Chairman. 

[The prepared statement of Dr. Varmus follows:] 
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Chairman BUCSHON. Thank you very much. 

Now I recognize Dr. Tessier-Lavigne for five minutes. 

TESTIMONY OF DR. MARC TESSIER–LAVIGNE, 

PRESIDENT AND CARSON FAMILY PROFESSOR, 

LABORATORY OF BRAIN DEVELOPMENT AND REPAIR, 

THE ROCKEFELLER UNIVERSITY 

Dr. TESSIER-LAVIGNE. Thank you, Chairman Bucshon, Chairman 

Smith, Mr. Lipinski, and other Members of the Subommittee for 

the invitation to speak today about how best to harness public and 

private sector activities to drive critical breakthroughs for poorly 

treated diseases. As president of the Rockefeller University, I bring 

the perspective of the academic sector. Rockefeller is a graduate 

biomedical research university with a distinguished record. Over 

our 113-year history, our faculty has been honored with 24 Nobel 

Prizes in medicine and chemistry, more than any other institution 

in the world. As former Chief Scientific Officer at Genentech, a 

leading biotechnology company, I also bring a perspective from industry on how best to enable tomorrow’s scientific and medical innovation. 

I will start by noting that, despite great advances in health and 

life expectancy in past decades, as Chairman Bucshon noted, there 

is an urgent need for new therapies. Death rates from cancer remain stubbornly high, and chronic diseases, like Alzheimer’s and 

diabetes, are on the rise. The suffering is immense, and the costs 

of care could bankrupt us. 

The good news is that we are in a golden age of disease research, 

thanks to technological advances like genome sequencing. If we 

make the necessary investments, we can understand why tumors 

spread, why nerve cells die in Alzheimer’s disease, and the secrets 

of our immune system. But gaining this knowledge is only half the 

battle. Translating discoveries into new therapies is a complex 

process with substantial attrition. 

For every 24 drug discovery projects initiated based on basic 

science discoveries, only nine candidate drugs eventually enter 

human clinical trials, only one of which will make it all the way 

to approval to help patients in the marketplace. Twenty-four down 

to one. This process takes, on average, 10 to 15 years, and more 

than a billion dollars for every approved drug, a huge and lengthy 

investment. 

Complex as it is, this process is remarkably successful thanks to 

four major groups of stakeholders working closely together. The 

first are biomedical scientists in academia and government, who 

create new knowledge with federal support. They explore the inner 

workings of cells and organs in health and in disease, relying in 

important ways on instruments, tools, and methodologies provided 

by the harder sciences, physics, chemistry, math, and computer 

science, as has already been noted. 

Second are the large biopharmaceutical companies who lead the 

complex drug development process based on that knowledge. Two 

additional stakeholders, disease foundations and small biotechnology companies, facilitate progression at the interface of the 

first two. This ecosystem plays to the strength of each participant. 

Academia provides an unfettered environment where researchers 
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can best explore scientific leads to break open new fields, whereas 

companies, with their tightly defined structure, are better suited to 

mounting the directed studies needed for drug discovery and development. And only the federal government has the resources and 

time horizon to invest in basic research that may not see a return 

for many decades. Companies already stretched thin by the duration and expense of drug development do not. 

Historically, this ecosystem has worked successfully, so much so 

that approximately half of all new drugs today are discovered in 

the United States. This investment has benefitted patients, saved 

trillions in overall healthcare costs, and boosted the economy enormously, generating high paying jobs and increased economic activity, and it has stimulated massive biotech and pharmaceutical investments in the U.S. 

How, then, should we maximize this vital drug discovery and development ecosystem, and what risks do we face? The logic of the 

biopharmaceutical sector is simple. Companies locate their R&D 

operations near the sites of scientific innovation in academia to tap 

into the best scientists and a highly skilled work force. And companies will make significant, even multibillion dollar, investments in 

breakthrough therapies on two conditions: if basic scientists provide sufficient understanding of disease processes to justify the 

bets, and if they see a path to getting an adequate return on their 

investment. 

The government’s role in supporting a vibrant basic research sector is, therefore, essential to understanding poorly treated diseases. 

If the academic sector generates the knowledge, the private sector 

will then rush in to apply it. Programs like the NIH sponsored 

BRAIN initiative, and its accelerating medicines partnership with 

industry can help focus on areas of high unmet medical need, like 

psychiatric disease, and facilitate translation of discoveries into 

drugs. 

Conversely, reductions in federal support for science over the 

past decade have weakened our ecosystem, with promising young 

investigators turning away from the field to pursue more stable careers, and scientists relocating to countries where research funding 

is less challenging. If this trend continues, we will see industry relocate to emerging sites of innovation abroad. Countries in Asia, 

like China and South Korea, as well as in Europe, are investing to 

become new epicenters of biomedicine, and they are succeeding. 

Beyond supporting the research sector, government must also 

continue to address important structural issues to ensure our country is attractive to private sector investment. Key requirements include sufficient protections of intellectual property, tax policies that 

favor R&D investments, and support of STEM education to provide 

a highly trained work force. 

In conclusion, we now find ourselves at a time of huge medical 

need, but also enormous scientific and economic opportunity. To retain its preeminence in this golden age of biomedicine, the United 

States must pursue the necessary investments and structural policies. Thank you for your attention. 

[The prepared statement of Dr. Tessier-Lavigne follows:] 
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Chairman BUCSHON. Thank you very much. 

I now recognize Dr. Keasling for five minutes to present his testimony. 

TESTIMONY OF DR. JAY KEASLING, 

HUBBARD HOWE JR. DISTINGUISHED PROFESSOR 

OF BIOCHEMICAL ENGINEERING, 

UNIVERSITY OF CALIFORNIA, BERKELEY; 

PROFESSOR, DEPARTMENT OF CHEMICAL & 

BIOMOLECULAR ENGINEERING, 

UNIVERSITY OF CALIFORNIA, BERKELEY; 

PROFESSOR DEPARTMENT OF BIOENGINEERING, 

UNIVERSITY OF CALIFORNIA, BERKELEY; 

DIRECTOR, SYNTHETIC BIOLOGY ENGINEERING RESEARCH 

CENTER 

Dr. KEASLING. Chairman Bucshon, and distinguished Members of 

the Committee, I thank you for the opportunity to testify at this 

important hearing, and for your strong and sustained support for 

science and technology. Today I would like to begin to tell a story 

of how we engineered a microbial production process for a much 

needed drug to combat a deadly disease that affects millions of children around the world, and how repurposing that same process allows us to meet needs not only for health, but also for energy, and 

the environment. 

There are approximately 250 million cases of malaria every year, 

causing nearly a million deaths annually. Most of the victims are 

children under the age of five. A child dies of malaria every 

minute. Conventional quinine-based drugs are no longer effective. 

While plant derived artemisinin combination therapies are highly 

successful, for many malaria victims, they are simply too expensive. 

To bring down the cost of the therapy and stabilize the supply, 

we engineered a microbe, a yeast, to produce a precursor chemical 

to the drug. To do this, we transferred genes responsible for making the drug from the plant to a microorganism. The process of producing artemisinin is akin to brewing beer. Rather than spitting 

out ethanol, the microbes spit out artemisinin. The microbe consumes that sugar, and produces the drug from that sugar. 

We licensed this microbial production process to Sanofi-Aventis, 

who scaled the process to industrial levels. This year, SanofiAventis produced 70 million doses of artemisinin, and is on track 

to produce 100 to 150 million every year for the next few years, 

roughly half the world’s needs. We predict that the drug produced 

by this process could save a large fraction of the annual one million 

children that die of malaria. 

Begun in 2004, the artemisinin project was supported by a $42 

million grant from the Bill and Melinda Gates Foundation, and 

took roughly 150 person years’ worth of work to complete the 

project. We were able to complete the project largely due to readily 

available, well characterized biological components, a significant 

point that I will return to shortly. 

The artemisinin story demonstrates the significant medical benefits of engineering biology, but also reveals how these benefits ex-
40 

tend to chemical manufacturing. Unfortunately, engineering biology is still time consuming, unpredictable, and expensive, and 

many urgent challenges in health, and energy, and the environment remain needlessly unresolved. Efforts to—aimed at making 

biology easier to engineer have come to be known as synthetic biology. 

As was the case with the development of synthetic artemisinin, 

synthetic biology represents a convergence in the advances in 

chemistry, biology, computer science, and engineering. Experts in 

the fields work together to create reusable methods for increasing 

the speed, scale, and precision with which we engineer biological 

systems. In essence, this work can be thought of as the development of biological based toolkits that enable improved products 

across many industries, including medicine and health. 

About ten years ago, around the start of the artemisinin effort, 

several colleagues and I set out to develop these more generalized 

approaches to making biology easier to engineer. We believed that 

we could engineer microbes to produce virtually any important 

chemical from sugar, yet there was a severe lack of publicly accessible tools for building biological processes and products, so we 

went out to the National Science Foundation, proposed a center 

dedicated to building these tools for the research community. In response, NSF granted us the Synthetic Biology Engineering Research Center, a ten year multi-institutional research project designed to lay the foundations for engineering biology. 

Now, eight years later, SynBERC has produced a broad range of 

toolkits that are being developed in the fields of energy, agriculture, health, and security, and offer an array of economic benefits. When SynBERC was established in 2006, it was the nation’s 

single largest research investment in synthetic biology. 

Eight years later, this, and other federal funding, have catalyzed 

the growth of academic research centers around the country, the 

production of many synthetic biology enabled chemicals in the private sector, five startup companies from SynBERC itself, and a robust private/public consortium that helps guide the research from 

lab bench to bedside. 

The U.S. model has been so successful that other countries, particularly China and the U.K. are developing aggressive, nationally 

coordinated research programs in an effort to surpass the U.S. to 

become the global leaders in biological engineering. These investments in synthetic biology are already making their mark on national economies. By some estimates, domestic revenues from biologically engineered systems was thought to account for more than 

2.5 percent of U.S. GDP in 2012, with a growth rate of 10 percent. 

The U.S. has been a leader in this field because of early and focused federal investment, but we now face stiff competition from 

overseas, and uncertainty in our pre-competitive investments here 

at home. I believe that now is the time for federal government to 

work with academic and industrial researchers to launch a national 

initiative in engineering biology, to establish new research directions, technology goals, and improve inter-agency coordination. I 

thank you for your time. 

[The prepared statement of Dr. Keasling follows:] 
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Chairman BUCSHON. Thank you very much. 

I now recognize Dr. Venter for five minutes to present his testimony. 

TESTIMONY OF DR. CRAIG VENTER, 

FOUNDER, CHAIRMAN, AND CHIEF EXECUTIVE OFFICER, 

J. CRAIG VENTER INSTITUTE, SYNTHETIC GENOMICS, INC., 

AND HUMAN LONGEVITY, INC. 

Dr. VENTER. Chairman Bucshon, distinguished Committee Members, thank you for the invitation to be here today. I represent a 

not-for-profit independent research institute, the J. Craig Venter 

Institute, and two biotech companies, Synthetic Genomics, and 

Human Longevity, Inc. We have a combination of funding from the 

private sector, from donations, from DOE, from DARPA, from 

NASA, from NSF, NIH, BARDA, and a range of interactions that 

range from 100 percent privately funded to 100 percent publicly 

funded. 

This is a very exciting time in science, as you have heard from 

my colleagues. We now have the ability to interchange the genetic 

code and the digital code in the computer. We can read the genetic 

code, put the data in the computer, and now we have shown, as my 

colleague Jay Keasling has discussed, we can go the other way, and 

actually write the genetic code. And, four years ago, we announced 

the creation of the first synthetic organism, completely writing the 

chemical genetic code. 

This is having implications in lots of areas. We have had a great 

collaboration with BARDA and Novartis for making the first synthetic vaccine against flu. When H7N9 flu broke out in China, a 

team in China sequenced the genome from a patient, posted it on 

the Internet. We downloaded it, and within a few hours synthesized the H7N9 virus. That was immediately started in development for a vaccine. BARDA has now stockpiled a substantial 

amount of the H7N9 vaccine before the first case has appeared in 

the U.S. It is the first time in history where the U.S. is ready for 

a deadly pandemic before the first case has reached this country. 

We can send vaccines through the Internet. Biological information now moves around the world digitally. It is not a matter of 

sending DNA in clones. We are using this in lots of different ways. 

We had recently announced, at Synthetic Genomics, a collaboration 

with United Therapeutics to re-engineer the pig genome, 

humanizing the pig genome to allow organ transplantation of 

hearts, kidneys, and lungs into humans to meet a huge medical 

need of lack of organ transplants. This comes from all these new 

tools for writing and editing the genome. You have heard from Jay 

Keasling how this can be done to create chemicals. We have engineered a synthetic genomic algae to produce large amounts of 

Omega-3 fatty acids that ADM is taking into extremely large scale 

production. 

The ultimate application of all this is in medicine. We have recently announced that Human Longevity formed the largest human 

DNA sequencing facility in the world. We are scaling up from 15 

years ago, when we sequenced one genome over nine months for 

roughly $100 million to doing 100,000 genomes a year, hopefully 

within 18 months, with the goal to have one million human 
47 

genomes by 2020 in a database to allow this data driven practice 

of medicine. 

This is a very exciting era, but it is a challenge, as you have 

heard from my colleagues, with the changes in government funding, and the competition from overseas, as Dr. Keasling talked 

about. In this same field, the Chinese government supports their 

industry to the tune of billions of dollars, versus competition with 

industry. These challenges are important, exciting. Also we deal 

with the public policy issue. Bob Friedman, my colleague, is head 

of policy at the Venter Institute. We have been asking ethical questions before anybody else. We have driven them, and the latest 

iteration of this was when we announced the first synthetic cell. 

The Obama Administration asked their new bioethics commission 

to take this on as their number one challenge. 

These are exciting times, they are challenging times, but this 

science has a chance to revolutionize medicine, and perhaps be a 

new industrial revolution. I am pleased to take any questions. 

Thank you very much. 

[The prepared statement of Dr. Venter follows:] 
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Chairman BUCSHON. Thank you, and I agree. This is an exciting 

time in health care. I miss health care. I have been out of it now 

for four years. From an overall federal budget standpoint, usually 

when I am at a hearing, and we are talking about discretionary 

funding programs, I like to say that right now in Washington, D.C., 

unfortunately, we are not addressing the entire piece of the federal 

spending pie. And—I will. I recognize myself. Because he pointed— 

he told me I had to, so I do, for whatever time I have left. 

And that is a challenge, because many people know that 60 or 

65 percent of all federal spending right now is mandatory spending, 

and the remaining part is discretionary spending, including Department of Defense, and that is where we start to see discretionary programs, like research funding, being pinched in an effort 

to balance the overall federal budget. 

So I am hopeful that in the next number of years, or short timeframe, that we will begin to address the entire piece of the pie, and 

take some of the pressure off the discretionary spending, particularly research funding, which I think many—most of us on this 

panel would agree needs—is extremely important, and needs to be 

probably increased to keep up. 

I will ask Dr. Venter this question. The return on investment on 

R&D, like in the pharmaceutical industry, has been a subject of recent debate because there are companies that are adept at R&D, 

and these returns can be significant both—from a both clinical and 

economic perspective. However, out there there are some forces 

that are, specifically in the health care industry, that have maybe 

the opposite perspective, people that are controlling companies, and 

believe that R&D is no longer productive in the private sector, for 

example. And you—seeing this, as some companies are bought and 

sold, that some people don’t value the R&D that was being done 

by the company. 

Do you disagree with this? Can we talk about the benefits of robust R&D, at the same time the potential consequences of cuts to 

R&D budget in the private sector, based on the shareholder investment in the companies? 

Dr. VENTER. Well, I think the experience that I have, and if you 

look at the biotech industry as a whole, it is largely based on basic 

research. It is only when you get way past that, into the manufacture and development of drugs, that I think you get into some of 

those conflicts. 

What I see is many people turn to biotechnology, and the robust 

funding that we have with capital investment, as an alternative 

way to fund basic research, because every breakthrough that we 

rely upon in the field of synthetic genomics, we have been doing 

basic research there for eight years. With these new efforts to sequence large numbers of human genomes, and have them impact 

medicine, these are large research projects that, in their places, are 

taken on by government funding, not by private capital. 

So I see it from the opposite point of view. I see much more private money, private investment, going into supporting basic research, because it is—I think we all agree, it is the basic research 

that drives these breakthroughs in every field. 

Chairman BUCSHON. Yeah, I would agree. R&D research in the 

private sector, you know, is important, and hopefully we can con-
58 

tinue to encourage all of our companies to continue to value this 

as a very valuable thing. 

Dr. Varmus, in your opinion, do we have the right balance between basic and applied science research, particularly in the biomedical science? Do we spend too many resources, or over-emphasize applied science—sciences at the expense of basic science research? Do we—where is that balance? Where do you see that? 

Dr. VARMUS. Well, thank you for the question, Chairman. It 

is—— 

Chairman BUCSHON. Turn on your mike. 

Dr. VARMUS. —quite difficult—sorry. This is a very difficult thing 

to measure, because the definitions of basic versus applied science, 

especially in this day and age in which the approach of basic 

science to clinical application is very, very close. I would argue, 

based on my observations, it is hard to document numerically that 

there is, in this moment of difficulty in obtaining funds for research, a tendency to think more about how the research that is 

being done, even in government supported labs, can be applied to 

the very real problems of human disease, and that this creates a 

situation in which scientists think their chances of being funded 

are augmented, and it may well be, by making specific claims for 

how the work they do will be applied in the short run. 

We have tried to defuse that somewhat recently at the National 

Cancer Institute by announcing a new award, a seven-year outstanding investigator award, that provides stable funding for at 

least 50 percent of an investigator’s work, so they are more willing 

to take risky approaches to science, to say, this is an important 

question. I don’t know where it is going to come out, it may or may 

not be useful. That is an element that we need to protect, and I— 

and we are making an effort to do that. 

I would say one more thing about the previous question you 

asked, about research in companies, and I agree with Craig that 

the major companies do recognize the importance of research. In 

my observation over the last few years, large companies and small 

are more willing to come to the NIH to work with us, we doing 

more of the more basic work, they bringing in the more applied approach. 

And we see this in the design of our clinical trials, where—which 

are increasingly becoming dependent on genetic analysis of tumors, 

targeted therapies being provided for tests by the companies, companies eager to collaborate with us, either through the NIH Foundation, or through work that we do at the NCI. 

Chairman BUCSHON. Thank you very much. I now recognize Mr. 

Lipinski for his questions. 

Mr. LIPINSKI. Thank you, Mr. Chairman. I want to start with Dr. 

Keasling. And I just want to say, Dr. Keasling, it was—trying to 

remember how many years ago, five or six years ago, that I came 

out to JBEI specifically at that time mostly to look at the bioenergy 

work that was going on there. But I wanted to ask you about technology transfer. 

You successfully co-founded a company, Amyris, to bring your 

discovery to the marketplace, so I would like you to talk about the 

challenges that you have faced trying to launch your company, or 

otherwise transfer your discoveries into commercial applications, 
59 

and then talk about what role do you see federal government can 

play at helping transfer academic research into the marketplace, 

and touch on what—at what stages should the federal government 

be involved, and what is the best way for the federal government 

to be involved? 

Dr. KEASLING. All right. So I will start answering that kind of— 

the last part first, and that is that the work that went into the 

anti-malarial drug was based on basic science that we did that was 

funded through the National Science Foundation to try to understand how microbes produce cholesterol-like molecules, and how 

plants produce molecules that are flavors and fragrances. And we 

then took that science, and engineered a microbe, and happened to 

learn about this anti-malarial drug. 

And that attracted funding from the Bill and Melinda Gates 

Foundation that allowed us to both develop this microbe, but also 

build Amyris, a company that makes no profit, and neither does 

Sinofi-Aventis, on this anti-malarial drug. In fact, they gave the 

technology away. It is being used free. And so did the University 

of California, which has title to the patents. 

What Amyris was able to do was take that same microbe that 

produces the anti-malarial drug and swap out a few genes, put in 

a few others, and it produces a diesel fuel that is now running in 

buses in Sao Paulo and Rio. In fact, they have clocked about five 

million miles on that diesel, and is now a molecule that is in flavors, and fragrances, and cosmetics. In fact, you can buy cosmetics 

from these yeast produced molecules. 

Our ability to get that technology out to companies is critical. 

Amyris came into the University of California, licensed that technology, and that allowed them to build the company. And that— 

federally funded research, and research funded by the Bill and 

Melinda Gates Foundation made all of that possible. 

I think it is critical that the federal government continue to fund 

basic science and basic research because, as we heard in this hearing today, that leads to the development of companies, and those 

companies tend to be located near the science that is being done 

so they can have access to those scientists, and build the companies 

further. Amyris now has about 400 employees, about 500, actually, 

in the U.S., and in Brazil, that are working on producing more molecules like this that will make the U.S. competitive. 

Mr. LIPINSKI. Thank you. And you had talked about doing a—the 

time may be right for some kind of national initiative. What 

would—you think that would—should look like? 

Dr. KEASLING. I think that the U.S. could, and should, make investments in biomanufacturing. And generally, in this area of engineering biology, we have been the leader since the discoveries of 

genetic engineering in the early ’70s. But that leadership is being 

challenged by China and many other countries, and they are building on a lot of the discoveries here, and the fact that we don’t have 

federally coordinated effort. An effort that would coordinate all the 

federal agencies, so that they are moving in the same direction toward engineering biology, I think, could have a huge impact on the 

field, and also on our national economy. 

As I mentioned earlier in my talk, this area is an area that is 

growing rapidly, and will continue to grow. We want to make sure 
60 

that it grows in the United States, and an effort by the federal government around engineering biology could ensure that. 

Mr. LIPINSKI. Do any of the other witnesses have any comments 

or suggestions along those lines? 

Dr. TESSIER-LAVIGNE. I just want to reinforce the last point, that 

the basic science discoveries and their commercialization leads to— 

not just to great outcomes, like the generation of these molecules 

or new medicines, it also creates real economic activity locally, as 

the industry will locate next to the sites of innovation. 

Mr. LIPINSKI. Thank you. And Dr. Varmus or Dr. Venter, 

any—— 

Dr. VARMUS. Well, I just would emphasize that, at the Cancer Institute, for example, the fundamental tools of genetic engineering 

are in use almost every day to change the behavior of cells, experimental animals that allow us to probe the secrets of cancer more 

profoundly, and new developments in this area are much to be welcomed by us in our experimental approaches to cancer. 

Mr. LIPINSKI. Thank you. I yield back. 

Chairman BUCSHON. Thank you. Recognize Mr. Johnson from 

Ohio, five minutes. 

Mr. JOHNSON. Thank you, Mr. Chairman, and really appreciate 

our witnesses being here today for this hearing. I am an information technology professional for most of my life before I came here 

to Congress, so I am always looking at how advances in technology 

affect different industries, particularly yours, so I would like to go 

in that direction just a little bit, if I could. 

So, for Dr. Varmus and Dr. Venter, if you would, you know, we 

are increasingly seeing the need for big data to help us decipher 

scientific problems, including understanding the genome, and complex diseases, like cancer. What is the future of cloud computing 

and big data in biomedical science research, and what role will 

they play, do you think? 

Dr. VARMUS. Thank you. This is a very timely question, because 

the NIH, and NCI in particular, are now housing the largest data 

sets in the world as a result of the accumulation of genetic information about cancer. As you may understand, cancer—— 

Mr. JOHNSON. But can’t find Lois Lerner’s e-mails, go figure. 

Dr. VARMUS. No comment. 

Mr. JOHNSON. Go ahead, go ahead. 

Dr. VARMUS. As you know, cancer is a disease largely driven by 

changes that occur during life and genomes, and being able to understand the patterns which differ from every tumor to another is 

critical. We had built, through the exercise I mentioned, The Cancer Genome Atlas, a huge database that needs interpretation. 

The question about cloud computing is particularly apt for us at 

the moment. We now have a—we are about to launch a cloud pilot 

exercise in which we will fund three—two or three competitors to 

do experiments with cloud computing, to allow investigators around 

the world to work with our lab’s large data sets. The NIH more 

generally has an initiative called Big Data to Knowledge, BD2K, 

that was attempting to learn both the computational rules that will 

make best use of that data, but also to do so in the context of privacy, which is important in medical research, and in a way that al-
61 

lows fair access of our investigators throughout the world to those 

data sets. 

In addition, there is a movement underway internationally to 

create something called a Global Alliance for Genomics and Health 

that will—has attracted the attention of literally hundreds of institutions around the world to be sure that data sets, initially in the 

area of oncology, and various genetic diseases to have access to 

those data sets, both to understand the underlying nature of the 

disease, and to make informed decisions about prognosis and treatment of those diseases. 

Mr. JOHNSON. Okay. Thank you. Dr. Venter? 

Dr. VENTER. Thank you for your question. It is—it is, as Harold 

said, very timely. There are two thresholds we just passed that actually allowed us to form Human Longevity. One was a sequencing 

technology that just barely passed the threshold of cost and accuracy. 

But the most important changes are in the computer world, and 

we are going to rely very heavily on cloud computing, not only to 

house this massive database, but to be able to use it internationally. We will have operations in different parts of the U.S., and 

even in Singapore, to allow us to do computation 24 hours a day. 

The cloud sort of makes that seamless, instead of trying to transport this massive amount of data. 

Trying to move things from my institute in Rockville, Maryland 

to La Jolla, we had dedicated fiber, but it is now so slow with these 

massive data sets, we use Sneakernet or FedEx to send discs, because we can’t send it by what you think would be normal transmission. So the use of the cloud is the entire future of this field. 

Mr. JOHNSON. Okay. All right. Well, Dr. Varmus, in your written 

testimony you discussed how supercomputers have created a powerful tool to analyze massive, complex data sets for genomics, proteins, and other biological sciences. In my final 30 seconds here, do 

you think that if the Department of Energy and National Science 

Foundation developed the next generation of computing—supercomputing, moving from petascale to exascale level, that even more 

medical breakthroughs would be made possible, and is supercomputing capabilities a limiting factor for future medical breakthroughs? 

Dr. VARMUS. Yes, absolutely, and we—this, in some way, would— 

we would obviously capitalize on that for its—the DOE’s agencies. 

We have, in the past, used DOE beam lines for our structural biology work. As Craig mentioned earlier, the number of genomes 

being sequenced is accelerating very rapidly, and the ability to sift 

through all that information, to look for patterns, to look for common mutations, and different tumor types, to try to understand the 

biological events as revealed by genetic analysis to the clinical 

events of real life experiences that the patients had is going to be 

a tremendous task that is going to—we have not yet achieved in 

solving simply by sequencing these genomes. We need to understand what those patterns mean, and it is going to require a tremendously heavy lift in the computer world to do that. 

Mr. JOHNSON. Okay. Well, thank you very much. Mr. Chairman, 

thanks for giving me the additional time. 
62 

Chairman BUCSHON. You are welcome. They are—we are going 

to have votes probably in the next five minutes or so, but once they 

call the vote, we still have plenty of time. We will be able to finish 

our line of questioning, and—so I now recognize Mr. Peters. 

Mr. PETERS. Thank you, Mr. Chairman. I want to thank all of 

you for being here, particularly my constituent, Dr. Venter. And we 

are so proud, and awed, and excited by what you have accomplished in the genome. 

And what I was—what—as I was listening to the testimony, and 

looking over some of what you presented, it strikes me that you, 

in particular, are someone who has been on both the private and 

the public side of this. And we have been talking for the last year, 

the model that we followed here with the NIH is that we provide 

a lot of funding, and much of it is competed, so that you have scientists who file these applications for grants. It is very competitive, 

it is peer reviewed, and that has been the basis of a lot of our 

science. 

And what I am inferring from this discussion is that now there 

is more a private sector kind of involvement, a lot of the—it is not 

the same model. So how should I, as a policymaker, be thinking 

about this, and is the old model, the model that has kind of been 

our playbook and so successful, is that still the same, or is that 

changed? 

Dr. VENTER. Well, thank you for the question. It is a very important one to answer—I also spent ten years in government at the 

NIH—— 

Mr. PETERS. Right. 

Dr. VENTER. —so I think I have been institutionalized many 

times. So I think the challenge, and the risk I see with government 

funding, aside from, as Harold said, the decreased buying power of 

it is the increased risk aversion of that funding. And I am pleased 

to hear what he says about the seven year grants. I think that is 

a step in the right direction. 

Finding a way to set aside a certain percentage of NIH funding 

to mandate risk is a challenge, and I can tell a story about it. With 

a previous NIH director, they started this new award for high risk 

research, and I was on the committee with other successful researchers, and the top 10 people we listed for this award were rejected because they were too risky. 

Mr. PETERS. Too risky, right, yeah. 

Dr. VENTER. So the challenge is how do you legislate risk taking 

when it is sort of not built into the fabric of the people and the government? But somehow we have to take greater risk with this 

funding to get more value for that funding. 

Mr. PETERS. Did that used to happen on the natural because 

there was more funding? And one of the things I have heard is 

that, because of the reduced buying power, the reduced investment, 

effectively, only the safe stuff is getting done, that if there were 

more funding, it is alleged that risky stuff would happen as part 

of the mix. 

Dr. VENTER. Well, there was more funding per capita. There 

were almost an order of magnitude less scientists when I started 

in my—— 

Mr. PETERS. Right. 
63 

Dr. VENTER. —career. And the funding from the Cancer Institute, there was much more on reputation of the investigator versus 

the sort of negotiated contract of the next stage of the research. 

And it sort of had to go that way, I think, because of fewer dollars 

per the number of researchers. So, you know, there is no—I don’t 

have a magic solution for it, but—— 

Dr. VARMUS. No. I—— 

Dr. VENTER. —we need to change something. 

Dr. VARMUS. I don’t have a magic solution. I would like to comment briefly on the question, which, of course, is a very important 

one. I don’t think the model is essentially changed. I think—and 

it is important to remember that, while much of our research is 

conducted through grants that are given to competing extramural 

investigators, we also have other ways of doing research. For example, through an intramural program, where there is a lot more stability, and a chance to encourage risk taking. 

And we also, within the NCI, have the privilege of having a contract laboratory, the national—the Frederick National Lab for Cancer Research out in Frederick, Maryland, where we can undertake 

projects that are extremely risky, like the new RAS initiative that 

I mentioned briefly in my testimony. 

The question of how we get both investigators and reviewers to 

take risks is a tricky one, because everyone recognizes this is a limited pot of money, and when you have a good proposal that seems 

very likely to yield tangible results, everybody’s focus tends to be 

on funding those first. And we have had to create programs, like 

our Outstanding Investigator Award, like the so-called Pioneer, 

and other innovation awards that are now awarded—— 

Mr. PETERS. Right. 

Dr. VARMUS. —throughout the NIH to try to encourage risk. But 

it is not the NIH, it is the whole community that is seized with this 

anxiety about how to undertake funding that is most productive. 

Dr. TESSIER-LAVIGNE. If I may just comment briefly also on the 

question. 

Mr. PETERS. We together have 15 seconds. Yeah, go ahead. 

Dr. TESSIER-LAVIGNE. The private sector is increasingly trying to 

tap into the discoveries in the basic science community, but they 

are not generating the knowledge, nor will they. So there isn’t a 

change in that sense. There is still—nothing can substitute for the 

federal support of basic research. 

Mr. PETERS. Well, thank you. Mr. Chairman, I appreciate the 

hearing. I yield back. 

Chairman BUCSHON. And, again, we—they have called votes, but 

for the first vote, we probably have 20, 25 minutes to get there to 

vote, so we are going to continue on with our—with recognizing Mr. 

Hultgren for five minutes. 

Mr. HULTGREN. Thank you, Chairman. Thank you all for being 

here. This is a very important hearing. It has been one of my primary goals on the Committee, to make sure that our laboratory 

system is set up, really, to provide the best bang for the buck, and 

to better work in our national interest. I just want to thank you 

for your work, and for your testimony here today. 

With the great innovation ecosystem in Illinois, I have seen how 

labs provide a valuable resource to industry to do work in facilities 
64 

that no individual company could build. The federal government 

does have a role in this space. Use of facilities such as the advanced photon source at Argonne have provided companies such as 

Abbvie with the unique research capability to make 

groundbreaking discoveries. 

What would normally take the company weeks on their own can 

be done in days with samples spending more in overnight deliveries 

than on the lab bench. My scientists at FERMI have also done key 

research in the accelerator technology necessary to finish the Linac 

Coherent Light Source upgrade at SLAC. 

Yesterday I introduced a bill to help modernize the national labs 

with my good friend Mr. Kilmer from Washington, along with 

Chairman Smith, and other Members from the Committee. We are 

looking to make sure that these facilities are open to partner with 

industry when it makes sense, ensuring that discoveries are not 

stuck in the lab. 

Dr. Tessier-Lavigne, how are the goals of pharmaceutical R&D 

different from federally funded research projects, and I wonder if 

you could explain—but also how can the federal government help 

to better accelerate innovation in this field? 

Dr. TESSIER-LAVIGNE. Well, thank you. The goals in this sense 

are complementary, they are not different, but it is really a staged 

process, where the fundamental insights into what goes wrong in 

disease, whether it is asthma, or Alzheimer’s Disease, or various 

cancers, are generated, for the most part, in the academic sector. 

The companies really come in when the discoveries are breaking, 

when insights are starting to coalesce, and they sift through them 

to try to find the most promising ones, and then deploy their horsepower, which is really focused around taking those insights, taking 

molecular targets, which they believe will be good targets against 

which to make drugs, and then start to make the drugs. That long 

odyssey of drug making takes, on average, 13 years, and over a billion dollars. They do that part of the work. 

The—so the research is complementary. It is not identical. There 

is some basic research, some fundamental research being done in 

the private sector, but very little compared to the academic sector, 

and vice versa. Some academic institutions will actually make 

drugs, and take them through clinical trials. Those are the exceptions that prove the rule. 

And then at the interface, the small startup companies are very 

important in helping sift through the discoveries made in academia, and move them towards the private sector, with the big 

companies then partnering with them as well, and disease foundations providing an assist. So it is really an ecosystem with those 

four components. 

How can we facilitate it? There is a lot of effort being placed 

right now on that interface. It is really about the interface. How 

can we ensure that discoveries in academia don’t lie fallow, that 

people recognize them and develop them? And there are a number 

of initiatives that are being made on those fronts. 

I mentioned in my testimony the Accelerating Medicines Partnership, which brings together the NIH, the Foundation for NIH, and 

10 companies to focus on very important areas, like Type II diabetes and Alzheimer’s disease, to try to identify the best molecular 
65 

targets. What are the best insights from academia? What are the 

biomarkers of the disease? What are the best targets for the biopharmaceutical industry on which to deploy its horsepower? 

So I think it is initiatives at that interface that I think will yield 

the biggest bang for the buck. What we are not going to see is a 

change where the pharmaceutical industry does a lot of the basic 

research, or academia makes a lot of the drugs. But what we can 

really help with is that interface. 

Mr. HULTGREN. Thank you. Dr. Venter, and also Dr. Keasling, 

what—are you concerned about any government regulations that 

might adversely affect both research and technology transfer of advances in synthetic biology? 

Dr. VENTER. Thank you for the question. I have not seen anything at all. I think, you know, that the whole case of intellectual 

property being important in this new field I think is overplayed. I 

think, in this new field of applying genomics to medicine, and the 

rapid change of events in synthetic biology, it is first mover effects, 

and making great advances I would say are an order of magnitude 

better than IP is now. It is like the software industry. The changes 

are happening so fast that you can’t really protect things with intellectual property as much as you can by just trying to stay ahead 

of the curve. My colleagues may disagree. 

Mr. HULTGREN. Dr. Keasling, yeah, I wonder if you have any 

thoughts on government relations—or, I am sorry, government regulations that might adversely affect research and technology. 

Dr. KEASLING. I don’t think there is right now. We have had a 

very effective system that started with the dawn of genetic engineering. That system has changed over the years as the technology 

has changed, but it has proven very effective, and I think we 

should continue that regulation that works so well. 

Dr. TESSIER-LAVIGNE. And if I may just—— 

Mr. HULTGREN. Quickly, I am out of time. 

Dr. TESSIER-LAVIGNE. —that is right, comment on Dr. Venter’s 

point, I think that his point really applies to tools and technologies, 

which evolve quickly. I think when it comes to the pharmaceuticals, there the patent system and IP protection is absolutely 

essential. Otherwise, the industry just won’t invest. 

Mr. HULTGREN. Thank you. I am out of time, and I know we 

have got votes, so I will yield back. Chairman, thank you so much. 

Chairman BUCSHON. You are welcome. I ask unanimous consent 

to allow Mr. Rohrabacher to participate in the questions. Without 

objection, the Chair recognizes Mr. Rohrabacher for five minutes. 

Mr. ROHRABACHER. Thank you very much, Mr. Chairman, and let 

me note, on the last point that was just made, that patent rights 

have been considered vitally important to American progress from 

day one. In fact, it is the only right that is written into the body 

of the Constitution as the word right. The Bill of Rights came later. 

And the fact that we have had a diminishing of patent protection 

in our country is of great concern to me, as is the fact that we have 

had a medical device tax as a vehicle to try to provide some kind 

of mechanism. Seems to me to be showing that perhaps there isn’t 

as much appreciation for technological advance in the higher circles 

that we should have. 
66 

Also let me just note that the FDA has recently approved Al 

Mann’s ten year question to have an inhaler being used as a substitute for needles for diabetics, and in the treatment of diabetes. 

And it took him ten years and a billion dollars. These are things 

of great concern. That can’t go on. Having something held off the 

market for that long, and that expensive—added to the process are 

reasons for concern. 

But today, Mr. Chairman, I would like to ask the panel about another flaw in the system. I would like to submit with a—for the 

record an article from the New York Times. 

Chairman BUCSHON. Without objection. 

[The information appears in Appendix II] 

Mr. ROHRABACHER. This article details a real challenge that has 

surfaced in California, with a particular company that is being 

taken over by a hostile takeover. And it appears to me, after looking at this, and looking at the details behind this, that we have a 

basic flaw in our tax system, and in our basic corporate structure 

that we have set up that will discourage R&D in the private sector 

by companies. 

And what we have here is Allergan, a company that has hundreds, if not thousands, of employees engaged in research is being 

taken over—a hostile takeover by a company who is actually raising the money for the hostile takeover by a plan that includes 

eliminating all the R&D. And thus you have a profit in eliminating 

R&D from a company by other companies wanting to take over. 

I mean, this—if this methodology is seen by others, we are going 

to have basically a huge reduction—we have made it profitable for 

companies, then, to come in and eliminate R&D. Have any of you 

gentlemen got any thoughts on that? Or is this just maybe a 

new—— 

Dr. VENTER. I will—— 

Mr. ROHRABACHER. —concept—— 

Dr. VENTER. Yes. 

Mr. ROHRABACHER. —here? 

Dr. VENTER. This is not the—— 

Mr. ROHRABACHER. I didn’t—— 

Dr. VENTER. —a—it is not a new concept to the pharmaceutical 

industry. 

Mr. ROHRABACHER. Okay. 

Dr. VENTER. CEOs will come in, and think they can greatly improve the bottom line by getting rid of R&D, and—— 

Mr. ROHRABACHER. Right. 

Dr. VENTER. —that is true for a very short period of time, but 

they basically bankrupt the company very quickly for doing that. 

So anybody who takes that philosophy is just—— 

Mr. ROHRABACHER. Well, this—— 

Dr. VENTER. —extremely shortsighted. 

Mr. ROHRABACHER. Well, then, this is really a—well—shortsighted. They are not shortsighted for themselves. That is the 

whole point. They give themselves a million dollar bonus and buy 

a new yacht because they have now given themselves a profit at 

the expense of perhaps things—discoveries that could be made that 

would improve the lives of all of us ten years down the line. This 

is a catastrophe. This is a catastrophe for people whose lives will 
67 

now not be helped by the R&D that Allergan, and other companies 

like it, are conducting. And we need to correct this flaw in the system. 

All these other things I have heard today are important, but I 

am really—Mr. Chairman, I commend you for calling this hearing. 

And the fact is that—but what we are—what—this whole issue 

that I just brought up, this undercuts so much of whatever the government’s basic research is doing, and all the other things that 

have been mentioned, if our own private companies that invest in 

it, now we found—we have made it profitable for other companies 

to take them over and eliminate it. We are going to—our people are 

going to suffer needlessly in the future because of this. 

Mr. Chairman, again, thanks for holding this hearing. All of the 

points that were made today are really significant. I have learned 

a lot, and I appreciate your leadership in this issue. 

Chairman BUCSHON. Thank you, Mr. Rohrabacher. At this point 

I would like to thank all the witnesses for your testimony. This is 

very valuable testimony, as our Subommittee, and the full Committee, look to reauthorize National Science Foundation, and are 

very important in funding, you know, research, obviously. And the 

Members, thank them for their questions. 

The record will remain open for two weeks. There may be some 

additional written questions sent to you that didn’t get covered 

today from the Members, and just please respond to them as timely 

as you can. We appreciate your testimony. The witnesses are excused. The hearing is adjourned. Thank you. 

[Whereupon, at 10:18 a.m., the Subcommittee was adjourned.] 

(69) 

Appendix I 

ANSWERS TO POST-HEARING QUESTIONS
70 

ANSWERS TO POST-HEARING QUESTIONS

Responses by Dr. Harold Varmus 
71 
72 
73 
74 
75 
76 
77 
78 

Responses by Dr. Marc Tessier-Lavigne 
79 
80 
81 

Responses by Dr. Jay Keasling 
82 
83 

Responses by Dr. Craig Venter 
84 
85 
86 
(87) 

Appendix II 

ADDITIONAL MATERIAL FOR THE RECORD
88 

STATEMENT SUBMITTED BY RANKING MEMBER EDDIE BERNICE JOHNSON
89 
90 

LETTER SUBMITTED BY SUBCOMMITTEE ON RESEARCH AND TECHNOLOGY

CHAIRMAN LARRY BUCSHON
91 

ARTICLE SUBMITTED BY REPRESENTATIVE DANA ROHRABACHER
92 
93 

Æ