good morning or is it afternoon i should say we're having a hearing today and we're hearing from just one witness dr francis collins the director of the national institutes of health and i think the members of the energy and commerce committee of course well acquainted with your great leadership that you've shown today and i aged dating back to one thousand nine hundred three when you started your fifteen year successful tenure as a director of the national human genome research institute your efforts as director of an h g r r resulted in discovering a number of important genes including those responsible for cystic fibrosis neuro neurofibromatosis huntington's disease a familial and doctrine cancer syndrome and most recently genes for type two diabetes and this work contributed to your being awarded the presidential medal of freedom in two thousand and seven as well as the national medal of science in two thousand and nine the highest honor bestowed on scientists by the united states government so welcome we welcome your your well informed perspective today i just wanted to say that the outstanding biomedical research and i support has had a transformative effort on our national health u s life expectancy has dramatically improved over the past century as diseases once fatal have enjoyed scientific discoveries resulting in targeted effective and personalized treatment strategies patients once crushed by no treatment or toxic treatments of nominal value benefit from the collaborative innovative work done by some of the world's best researchers here bethesda in all fifty states and in ninety countries around the world and i'm personally proud of the great partnership the n h has with rutgers university and my district as well as with the university of medicine and dentistry of new jersey medical school which is the nation's largest freestanding public health science university now we commend the progress and it has made and cheer you on for the discoveries we know you'll realize in the future from alzheimer's to autism to cancer to heart disease we know that our federal dollars a hard at work well i could go on in length about all the research you and your team are undertaking i just want to mention a couple of examples as you certainly are well aware the war against cancer continues and for good reason nearly five hundred thousand people die annually of the disease but research conducted by any age has resulted in the overall cancer rate decreasing and treatment is no longer reactive but proactively targeted to the genetic profile of each patient's cancer further and i collaboration with public and private entities strengthens the global effort to fight infectious diseases into microbial drug resistance which has been a subject of examination by the health subcommittee is a top priority of an i as national institute of allergy and infectious diseases and i a id has shepherded basic research on resistance as well as silicate of partnerships with industry and nonprofit organizations to develop programs aimed at better controlling into microbial drug resistance and just this week and i each researchers announce the identification of two previously unknown steps in the spread of the malaria in the bloodstream progress like this could have a private prevent effect on our global health and today malaria kills over million million people annually the majority of whom are young children in sub saharan africa i want to say the energy and commerce health subcommittee is proud of its long and productive relationship with n h we support the cross cutting collaboration of researches across disciplines and empower the n h to continue to promote innovation in the bipartisan and i h reform act of two thousand and six we look forward to hearing an update today on the fully implemented act including take a minute if i could from my colleagues time or you know it to our time to thank a long time member of my staff and the energy and commerce health subcommittee bob clark or bobby clarke i always call him bob and everybody else calls him bobby would you stand up a minute mr clark all right most of you know him as being a staff member of the health subcommittee but actually he's kind of  i don't know i guess the best example i've ever had of someone who actually walked into my district office as an intern at rutgers university worked as an intern then was hired to work on the campaign then worked as a staff person in the district office for a couple years and then said he wanted to go to washington which i thought was a good idea came down to washington worked as a as a legislative person did you work as a reception ist before you even did that i think it was even the receptionist for a while and then he became a legislative assistant and then he went to georgetown and got an m a in public health or health then he came back and worked as the legislative person on health care and then finally went to the health subcommittee now he's moving on again but i think he's probably he may have been the only person who did absolutely everything  you know from being the rector's in turn all the way now to being on the health subcommittee staff so i'm very and you can imagine the things i had him do not as health care but native american and everything else on earth and he's also obviously a proud alumni rutgers university so i just want to take the time to to mention that bob is leaving at the end of this week and we wish him godspeed and i will now yield to mr shimkus ranking member thank you thank mr chairman let me let me join you in wishing bob godspeed and his new profession profession he now gets to go into the private sector yes he may become more republican because of that and i look forward to visiting with him real soon so the but so thanks for the hearing it is an important hearing and something that we all have said for numerous years is an important thing even when i first came here just doubling energy funding which you know happened under republican stewardship is a big deal we never have enough money for basic research and we always ask for more so you know we're we're all very supportive of what what can be done and we're particularly excited about as as a ranking member on the health subcommittee and even before  that the personal experiences of individuals with diseases or cancers we had one and we're excited that in this cancer genome atlas gastric cancer is now going to be included and everybody wants more money for every research facility but i do think that in the discussion of cancers focus on those that aren't cured right now or can't be delayed and doing the research like gastric survival rate getting people to survive then we can move in a direction so i know that the committee was gracious in testimony we had earlier this year that's been followed up by  some appropriation hearings and you know for americans age twenty five thirty nine one is likelihood of being diagnosed with gastric cancer has increased almost seventy percent since one nine hundred seventy seven so it's something that everyone has causes and that's one that we've been working on so we will focus on that i'm making my comments short we we are concerned about the influx of cash in the stimulus bill and the two year focus because most your stuff goes longer years and that and we're concerned about well what went well and what went poorly and and because of the limited time frame i can't leave a health care hearing without you know talking about the new health care law you know we were told it would save the average premiums twenty five hundred dollars a year c b l says going to increase premiums two thousand dollars a year but next week the new temporary high risk p pre existing condition exclusions those are things we could fix if we would just talk about this new law and try to address some of the reforms so not as long as i normally go mr chairman clarke while there is part of your subcommittee staff he was part of the team working tirelessly on health reform he was famous among the staff for his amazing command of detail and his calm and storm demeanor and we will miss him and we achievements led the government's effort to map the human genome and i preeminent health research institution in the world it's recognize across the country and around the globe for its outstanding work and with good the research and i ate supports and conducts not only has resulted in cutting its scientific breakthrough breakthroughs is also led to really meaningful improvements in the public health but the work of an i truly is never done learn more about disease in the human condition the list of research challenges grows some forty years ago for example we thought of cancer as a single disease now we know it's much more complicated because of its outstanding work we continue to look to and i age to help solve the trickiest of medical rid of riddles such as alzheimer's disease diabetes autism spinal cord injury parkinson's disease and many others and we also look to and i h to figure out how to prevent disease and disability wherever we can our job in the congress is to ensure that and i as a finding a need for researchers to continue their world class work money is in short supply i know but federal support for and i age is not where we can afford to cut back in this time of both endless research possibilities basic enshrines relational and difficult economic challenges dr collins has come forward to lead and i h and the brief brilliant researchers it supports to hear from him about his plans and how best in the years ahead to meet and i his mandate and continue the institute's longstanding legacy for making a measurable difference i thank the chairman and welcome dr collins to our humble little committee i'm going to submit my statement for the record and reserve time for questions because we rarely get a witness with this much firepower and the time is better spent spent for question next is the gentleman from georgia mr gingrey as chairman thank you united as a global leader in medical research and innovation and we in large part have the national institute of health to think that these efforts today roughly thirty percent of the funding for disease research equaling almost three percent of our gross domestic product comes from the end i h and our federal government these investments have advanced our understanding and treatment of many life threatening diseases recently and  a funded organization of the universal or chicago made national news when they reported finding key genomic markers common in autistic children these findings may one day support our effort to understand what factors contribute to this consultation with an eye will spark new ideas for treatment of that dreaded disease i'd like to commend you dr collins for helping to foster this groundbreaking breaking collaborative approach in addition  grants that resulted in this cover and development of many life saving medical treatments since coming on the market the combination of the drug herceptin with chemotherapy has been shown to increase both survival and response rates for patien with breast cancer including reducing the risk or relapse back fifty percent when given to patients of mediately following surgery however i do want to strike one note of caution the drug herceptin is widely available in the united states but this was not always the case in britain england initially denied coverage of the drug and only relented after many public demonstrations including a protest march thousands of women through the streets of london  points because as many of you know dr donald berwick was recently nominated director of the centers for medicare medicaid services by president obama this is the same donald berwick who just last year stated that quote that the decision is not whether we will ration care the decision is whether we will ration with our as open to be frank i fear that the groundbreaking pritam is funded by and that we are celebrating here today will not be made available to our seniors if the political philosophies of dr berwick are in charge of our medicare program mr chairman our seniors cannot afford that and that kind of support of health care you mr chairman i too will submit my statement to the record in order to have more time to ask the wide range of questions i have for dr collins required to have him here thank you and as i said all member statements will be submitted  for the record next is the gentlewoman from tennessee miss blackburn thank you mr chairman thank you for the hearing and i want to thank our witnesses for taking the time to be with us i will have to tell you that i am one of those who really enjoys watching the research that is taking place in my state of tennessee we have over one thousand and i research awards totaling over four hundred forty million that were granted in two thousand and nine tennessee organizations such as harry medical vanderbilt university st jude's children's hospital a well as several small privately held companies are making advances in the medical field that will improve the lives everyone through the use of an eye funding the vanderbilt institute for clinical  is the only in c i designated comprehensive cancer center in tennessee that conducts basic translational and clinical research like why st jude's children's hospital in memphis is using grant money from  the way in cancer research and i think we've all noted that st jude's was recently named the best children's cancer hospital in the nation by u s news and world report in addition to funding large research institutions i grant two small privately owned businesses throughout to in the se such as max mobility which is a small business of less than ten employees located in ne iraq tennessee are producing great results max mobility has used and i funds to create innovative wheelchair designs and products to greatly assist regular wheelchair users and it's clear that research grants from and i h provide opportunities for organizations to make and continue these discoveries in zero six this committee passed the and i preform act to cut bureaucracy increase transparency and streamline interagency communication in order to increase efficiency and to hopefully decrease waste and the funding process my hope is that we're going to continue on that path my concern is that with the thank you the gentleman from connecticut mr murphy thank you very much chairman welcome dr collins discussions before and we realize that dr collins spent some time in my hometown of connecticut most kids actually went to the elementary school in our local neighborhood so it's good to see that it's a small small world dr collins in connecticut we have been wildly successful in the last four to five years investing in stem cell research lines to the federal restrictions we passed the nation's first law putting a state funds into our two main research institutions of yale and yukon been able to leverage about twice as some stem cell labs that we have at u conn today to move into private sector commercialized developments it's very exciting but it presents our state sort of new to the area of scientific investment with some very interesting questions about how to match public and private partnerships and how to make sure that our state investment in some of the basic stem cell research ends up with us sharing in some of the monetary gain to be happen d through the commercialization of some of those partnerships and so whether it's in your testimony or in some of the questioning one of the things that i'm eager to hear about is how and continues to evolve evolve in its thinking about how the scientific research that you fund when it becomes commercialized accrues to the benefit of the taxpayers that have made those investment i know you're not a biotech venture capital firm the you are in this for the the science of it in the lifesaving research but i hope that we are constantly thinking as we are in connecticut of new ways to protect taxpayer investments when the it leads to a private sector solution that can large rewards to a private company even though it's based in part on federally funded research we're thrilled that you're here very eager to hear your testimony about the new and exciting things happening in so we can bring chairman for calling the hearing and welcome dr collins i think medical research in america today is very exciting and the potential for continued breakthroughs and advances the potential is unlimited in my hometown of tampa where i represent a t the public health but they're important for our workforce as well as we try to find our way out of economic recovery what it means to do our workforce needs in the potential for young people to have good paying jobs in a in a profession that has a great future just cannot be understated sawed on i'm very interested in your in your testimony today about the future of and i shall we can improve all the grant making that you do and i appreciate the fact that you're holding this hearing today and dr collins i'm very grateful that you're here today to share information about the vital role of the national institutes for health in our health care system and in our economy the and i each is especially important to my hometown of madison wisconsin it is the largest and most critical source of research funds for the university of wisconsin madison which in my own biased or unbiased opinion is one of the world's premier research institutions basic applied better medical research has and will continue to have a significant impact on the health of wisconsin's residents and the state's economic growth biomedical research has helped raise the average life expectancy of wisconsin residents from fifty two years in one nine hundred twenty eight to seventy nine years in two thousand and five and every year wisconsin citizens have an estimated one point six billion dollars in health care save an estimated one point six billion dollars in health care costs thanks to public investments in biomedical research and development and finally wisconsin researchers bring in a greater percentage of federal bio science funding per capita than any other federal program or expenditure in the state generating thousands of high quality high paying jobs for our residents and i want to add just a little personal note as have trained students and created jobs they have fostered significant scientific progress and saved lives yet certainly challenges lie ahead budget constraints and limited funding for infrastructure improvements restrict the capacity of research institutions to take biomedical research to the next level and we must nurture young researchers and ensure that the next generation of scientists stay in the field so i look forward to your testimony dr collins and thank you for being here today and  been waiting for this occasion and i will take less than a minute just to say this is really about us listening to you about the fascinating work being done at the institutes now the direction you'd like to take the institute into the future and how congress can best legislate the authorities for you to meet your goals which are our goals as well most importantly how can we keep up with the pace and the direction of scientific discovery how can we move to more quickly translate those discoveries into practice both in treatment and in prevention i bring up prevention because so often we think about basic research that leads to the development of chemical drugs biologics medical devices but there are also very many opportunities to nip disease in the bugs to diagnose more efficiently and and more quickly and to prevent diseases before they ever develop so i look forward to hearing from you thank you very much thank you i think we've had every member of the  subcommittee to do an opening statement so now move to you dr collins and let me say to the members that dr collins had asked to speak longer than usual i don't know if it was twelve or team minutes something like that and i said fine because he's our only witness today and i think he needs some extra time to basically develop his testimony so with that we welcome you and thank you thank you mr chairman and thank all of you for those wonderful opening statements it's clear i'm speaking to a very well informed group of members and i'm been looking forward to this chance for a conversation for some time and will very much enjoy the questions and hopefully some answers that occur after a little bit but i did want  chance to say something in the way of an introductory set of remarks about it and i h it is a great honor to have a chance to share my vision for the future of biomedical research as we move into this second decade of the twenty first century i have submitted my full written testimony for the record and i'm not going to read from that i'd like to share with you some major themes that were in that statement and i have a few visuals that will appear up there on the screen so i would like to think this committee for your steadfast support mission which you see here on the slide is twofold to discover fundamental knowledge about the nature and behavior of living systems yes but also to apply that in the extension of healthy life and the reduction of the burdens of  disability and illness and as has already been pointed out by a couple of you in your opening statements we are making real progress there as you can see us life expectancy has been climbing over the course of the past few decades and if you look at now less than twenty percent but let me and emphasise that human biology is not an end to itself we wake up every day trying to think about how to further improve those outcomes right now course one of the most urgent goals that everyone is concerned about relates to the b p deepwater horizon oil spill this is an environmental tragedy of unprecedented proportions for the past several weeks and i has been partnering with our sister agencies in the department of health and human services to protect the health and meet the medical needs of gulf residents and cleanup workers for example the national institute of environmental health sciences has provided training and safety information to protect the health of responders its educational course on hazard awareness and safety is now required for all oil spill workers hired by b p more than thirty thousand to date have been trained by an i h s including the distribution of a safety handbook i have a copy here that's been distributed to first line responders and beach workers but we have to do more here and so today i am announcing that and i will devote another ten million dollars in existing funds to support research on the potential human health effects of the oil spill and i each through and i h s will recruit a cohort of fifteen to twenty thousand exposed clean up workers and gulf residents we will collect their health histories and tissue samples as well as information about cleanup work that they performed and the nature of their exposures in the near term and i will establish a baseline of such information and then we will monitor cohort members for respiratory immunological and neuro behavioral effects so that's an example of some urgent responses and we do feel that is part of our job to respond to those as they arise but we also have to look ahead to tackle our nation's many ongoing health problems obesity cancer heart disease diabetes autism to name just a few and you all in opening statements have mentioned quite a number of these one of my first actions upon being named and i each directory was to scan this landscape of biomedical research opportunity to try to identify areas that are really ripe for major advances that could yield substantial benefits in the coming years and while that list of specific projects could go on forever i've identified five areas of exceptional opportunity at your place there is a reprint of an article in science magazine and i would like to very briefly tell you what these five themes are that seem particularly ripe now for investments by and researchers and i'll go through them one of the time first of all we have seen invented in just the last four or five years a number of dramatically powerful high throughput technologies that give us the chance to ask comprehensive questions about how life works what are all the proteins in the cell what are all the components of the immune system what are all the steps in development we couldn't really ask those questions before and now with technologies like genomics and imaging computational biology nanotechnology we can do those things and the chance to apply them is unprecedented in its scope a second area that i think is a natural partner to this is to take all those basic science discoveries that are beginning to pour out of our laboratories and accelerate the pace by which those are brought into clinical benefits and i traditionally has partnered with the private sector in doing this and we intend to continue that but we have a chance now to play an ever larger role in the front end of drug development pipelines that previously academic investigators get involved in and we aim to try to push that agenda as quickly as we can in order to take basic science discoveries to the bedside at an unprecedented pace and i by the way i might say the common fund which by the act putting it into legislation is now a major opportunity for me as the director will be investing substantially in both of these areas in the coming years because this is an example of the kind of research that touches on all diseases it's not limited to any single in a third area is for us to try to provide the kind of evidence that is going to be needed to make wise decisions about healthcare reform and that includes things like personalized medicine and  effectiveness research that includes health economics to try to understand how we can better figure out incentives that will both improve outcomes and result in better care and at the same time we also need to think about in the fourth team extending outside of our own boundaries to the rest of the world we have opportunities in the global arena now to both create and deploy new kinds of diagnostics in therapeutics for aids tuberculosis and malaria and for the neglected tropical diseases that have had relatively work little work done upon them but they affect hundreds of millions of people and finally the fifth of the five themes is our own research community probably our most critical resource we certainly will depend upon these wonderfully bright minds in order to make these discoveries in the coming years and we need to be sure that we're encouraging the best and brightest of the next generation to come and join us that we're inspiring innovation and not just crank turning that we're improving the diversity of our workforce and i might also say that we are encouraging and stimulating and insisting upon the integrity of the process to be at the highest level let me just say a word about that in the process of overseeing and i  research one of my priorities is to be sure that we are vigilant about managing any potential conflicts of interest or even the appearance of such conflicts i'm determined and i should lead in this area and that's why we are in the midst of proposing a new set of regulations that will require more complete disclosure from me and i funded investigators about their dealings with industry in order to maintain the public trust the need for enhanced oversight of financial conflicts of interest has been made particularly salient by the news last week that described really egregious case of failure of an investigator to disclose in this case the scientist was sanctioned by his university but then went on to move to a new university and thus evade the sanctions we're going to fix this problem the pending notice of proposed rulemaking about conflicts of interest gives us an opportunity to address cases where there are such violations of an eye and university financial disclosure rules we can clarify our options for enforcement actions against both individuals and institutions so i am committed to making sure that we earn the public trust and ensure the highest standards of research integrity and transparency but now i want to go on to share several exciting examples of how and i funded researchers are using some of these revolutionary tools and technologies to expand our understanding of human biology the more we learn about how the body works the greater our ability to transform that knowledge into cures and treatments for the many diseases that plague humankind alzheimer's disease has already been mentioned currently more than five million americans suffer from this degenerative brain disorder and with the aging of the american population that number could more than double by two thousand and fifty and would greatly increase the diseases already steeped in anshul told if current trends continue the annual health care costs associated with alzheimer's disease would rise from one hundred seventy two billions to more than one trillion dollars over the next forty years now because all timers disease has proved to be such a tough opponent in researchers are now attempting to fight it on many fronts and the first prong of attack is an interesting one that might not have seemed obvious a couple of years ago and that's immunotherapy this involves enlisting the body's own immune system to prevent the accumulation of beta amyloid which is the protein thought to be a major culprit in all timers disease about a decade ago there was much exciting about a clinical trial of the vaccine but enthusiasm flagged when some participants developed brain inflammation obviously a serious side effect since then much work has been devoted to figuring out ways to improve the safety profile of such vaccines one new approach which combines immunotherapy with anti inflammatory agents has produced encouraging results so far in animal test other recent work by and i funded researchers has uncovered a possible new drug target for all timers disease and one which interesting really brings this disease into closer molecular harmony with parkinson's disease and with huntington's disease and a few others and a study just published last week researchers described how a particular gene that's associated with all timers disease precent milind one acts to cause this disorder and interesting light what they learned was that this protein normally helps clear debris from the brain but mutations blocked the normal plan for the trash pickup system in the brain leading to a potentially toxic accumulation of discarded proteins we always already know of a few drugs that would stimulate the trash pickup and this is raising new ideas about an approach to alzheimer's that people had not thought about before a third effort and this is a public private partnership is the all timers disease neuroimaging initiative which is combining various clinical neuro imaging genetic and spinal fluid measures to understand the events that lead from normal cognition to alzheimer's disease defining biomarkers such data have been rapidly made available to the worldwide research community and now thanks in this case to an infusion of recovery act dollars for me and i each this initiative a d n i is stepping up its efforts even further i also want to talk about cancer several of you have mentioned it already a major project that has been accelerated by cover react funds is this cancer genome atlas or t c g already t c g a has produced comprehensive molecular classification systems for ovarian cancer and for which is the most common form of brain cancer and a very serious one team survey of glioblastoma recently revealed five new molecular subtypes for the disease that we didn't know existed before and in addition researchers found that responses to therapy for glioblastoma varies by subtype and that may help doctors do a better job of matching individual patients with the therapies that are most likely to work for them what's more the findings may lead to new therapies directed at the molecular changes underlying each type of glioblastoma providing targets that we very much need to have in order to develop the next generation of therapy so now in the next phase of the project again encouraged by the recovery act p c g i will build comprehensive catalogues of the key genomic changes in twenty major types and some types of cancer including gastric cancer they are being shared rapidly with the worldwide scientific community and will provide powerful new clues for all who strive to develop better ways to diagnose treat and prevent cancer those are general stories but to give you a real life example i'd like to share the story of beverly so to hear when she was diagnosed with stage fo non small cell lung cancer several years ago this nonsmoker told she probably had six months to live beverly received standard chemotherapy but her tumors kept growing devastating news for her and her children and grandchildren but then last july based on a genetic analysis of her tumor at the dana farber cancer institute in boston she signed up for a clinical trial of a new genome based a drug called crizotinib now look at this game it's maybe a little hard to see where you are the maybe the red circles will help you but if you look on the left in both of those images her lungs the black areas have big white spots in them those are large tumors growing in her lungs on the right is after treatment some five months later and those tumors are essentially gone so she has had a dramatic result and in the first six months of treatment most of her tumors have disappeared but this doesn't work for everyone this particular drug actually seems only to be successful for about five percent of people with lung cancer but we know now which five percent because you see this drug works on specific genomic changes called a fusion of a gene called a all k with another gene if your lung cancer has that fusion this is the drug for you because most of those patients about three four quarters of them respond dramatically to this new therapy this was just announced a few days ago at the american society for clinical oncology people in the room were blown away by this kind of response and by the ability to make such a precise prediction about who was going to respond so this demonstrates the potential of personalized medicine of the value of matching the right treatment with the right patient at the right time interestingly beverly's story also points out how rapidly this can happen when she was first diagnosed nobody knew about this genomic change in lung cancer that was discovered just three years ago and now here she is showing this dramatic response to a new drug so it need not be the case that the new therapeutics take many many years where clearly we need a lot more stories like beverly's not only for cancer but for as not diabetes depression heart disease and many other conditions the brochure you have at your place outlined some of those exciting advances so it is my hope and my expectation mr chairman that and i funded research will bring us much closer to turning discovery into health for all americans in to do so quickly if our nation is bold enough to act today upon the many research opportunities that lie before us i believe we will all be amazed at what tomorrow brings thank you very much and i'll be glad to answer your collins i'm just regularly visited as you can imagine by families and advocates with very compelling stories about diseases they have grappled with and of course there the message almost always is please increase  research for you know that particular disease and particularly of course during the appropriation and and you know of course every one of us has had one disease that may have had a profound effect on our lives either personally or our families the reason i bring that up is it because many of us believe that we should allow science in the public health not politics obviously to determine the research priorities of and i h and i think that generally speaking that's true and that has been the case but to be sure there are times when the relationship between national spending science and public policy have to be considered some diseases like alzheimer's for example are responsible for a massive strain on our medicare medicaid programs other developmental disabilities like autism have a significant effect on our families from a very young age and on our school budgets and social services programs obesity for example is skyrocketing and has a major economic impact act and is responsible for loss of productivity restricted activity absenteeism point i'm trying to make is that it's difficult because many times the effects of some diseases or disorders is has a huge impact on the economy and the question of how much we spend and how much how many dollars a loss so it's hard to completely take out funding and politics from the scenario but what i want to ask you is what factors you consider when formulating and i age budget and how can we best work with you to ensure that and i age is maximizing his research dollars do you think that you know perhaps we're being too you know that we're not giving you enough input or that you know perhaps that the way we're going about prioritizing things needs to be changed well thank you mr chairman this is certainly an issue that it and i we discuss probably just about daily are we getting our priorities right the factors that go into that are numerous and they do change over time certainly the burden of a disease has to be a major consideration i mention all timers in my opening statement particularly because of the concern about the burden this disease places on people today and that it may place on people tomorrow and if we certainly need breakthroughs in a disease alzheimer's is near the top of that list sorry i seem to have a disease myself today to struggle with the aftermath of some sort of respiratory virus so certainly the burden is one factor but if we only studied common diseases that affect hundreds of millions of people then what about the rare diseases of a rare disease strikes your family it probably doesn't matter to you too much that it's rare you hope that somebody will be doing something for that gastric cancer was mentioned which is growing in its frequency we don't know why but it's still rare cancer or should we ignore it and just study the common cancers that doesn't sound right either and then of course there's the matter of scientific opportunity in a circumstance where there has been a new realization of opportunity to make a real advance in a disease even if that's not as common as a different disease you would want to pass that up so that folds in there fortunately peer review which an eye does probably better than any organization in the world is one of our ways of trying to make sure we're using our investments wisely and we do have a two level peer review system the first level is to look at scientific excellence of a proposal but the second level looks at program needs and there again that's an opportunity for our experts to come to our advisory councils from universities and companies all over the country and sometimes the world to give us advice about should you steer your ship a little bit further in this direction or the other the other thing i should say is that basic science not attached to any particular disease is also a critical part of our portfolio and needs to be so because that's the foundation upon which we build the future and if we soley made decisions on the basis of diseases we might miss out on those important investments i guess what i'm saying is it's a very complex calculus and it should be but i do think it's best made by people who have a sense of the entire landscape i'm sympathetic with what must happen to you every day as someone with a very compelling story where more resources clearly would help in terms of the advances that are needed in research is asking for your help because i have those same conversations all the time and obviously more resources in many instances would help but we have a limited opportunity here in terms of the budget the resources that the taxpayers are willing to give us i do think the best place is for those decisions to be made on the basis of science and i think it and i not just speaking for myself but for those twenty seven institute and center directors and all the advisers we have with all of their expertise i think we do a pretty reasonable job all right thank you very much mr sh first i'd like to ask you now as consent to that we can submit this letter into the record it's a very historic letter because it's a letter signed by senator durbin and myself and you will not see these letters signed on a similar issue  so the questions about the basic part of cancer research i think we're at a remarkably exciting time in terms of our understanding of what makes cells grow and what makes them stop growing and then really that is the nature of cancer is the loss of control that a cell normally has when you see cells that have lost that control even in their proper location or worse yet if they've spread to other parts of the body that is a malignancy we have learned a lot in the last ten or twenty years about the signals that control be on and off switches for cells but we have a lot more to learn there particularly delighted to welcome on july twelfth the new director of the national cancer institute dr harold varmus who will be coming back to and i after ten years as the president of the sloan kettering cancer center in new york and nobel laureate as he is and somebody who won his nobel prize in this very area and i think it could hardly be a more exciting time for exploring and finding the additional answers we don't know about yet in terms of the basic understanding of cancer and that's going to shine a bright light on prevention and treatment strategies great thank you my time's woman let me go to in my opening statement i mention this in the stimulus bill but also just regular funding the two year program versus a four year program and how does that help or how does that  hurt and then the real question is would you would you have would have been better in the stimulus bill had we went to longer term strategies and if in the remaining stimulus stars that haven't been spent would you like the opportunity to be able to shift that to longer term research programs that you have i appreciate that question certainly the availability of support from the recovery act was a wonderful opportunity for an age we had gone through a five year period where the budget had been relatively flat and that meant inflation had been eroding our buying power to the point where we had lost about sixteen seventeen percent of our purchasing power and there was a great pent up interest and need on the part of the scientific community for new resources to spur innovation so the availability of those dollars from the recovery act resulted in a incredible outpouring of grant applications one particular kind of application that we designed very quickly and put out there for people to respond to called challenge grants we thought we might get four or five thousand applications we got more than twenty thousand and it took quite a lot of work to get the reviews done and decide what we could find so clearly there was a need and i would argue that the dollars that came to and i from the recovery act are extremely well spent they are going to produce very interesting scientific vance's but you're quite right that two years is a very short cycle time for most scientific projects i understood and i think all of us did why that was the case we were after all and still are in an economic downturn of considerable magnitude and the point here is not just to do great science but also to  encourage job creation and to stimulate the production of goods and services and i think money has done that really quite remarkably well fifty thousand job times right now but can you address the remaining stimulus dollars and putting it to longer term prospects so we have essentially allocated all of our ten billion dollars that we were given a chance for a few of those dollars to be put into multi year projects but most of them are into your effort versus what what you have decided and it's not a significant amount of money it's for two thousand and nine it's seven thousand but and two hundred sixty thousand for two thousand and eight so it's a pretty big chunk of change the deep concerns of people who have this as part of the disease how do you how do you prioritize stopping of high cost diseases institute and center directors need to look at those priorities and to try to balance those considerations between the burden of the disease which means how serious is it how many people does it affect what is its cost to society what is its personal cost of the individual and to the family also factor into that the scientific opportunities which may in some instances be more ripe than in others as i said earlier i think that we also have to be sure we're not neglecting those rare diseases which are in many instances opportunities not only to help people who are suffering from a condition that might otherwise be a bit neglected but often shed light on common diseases we would not for  instance right now have the staten drugs had it not been for the study of a rare disease called familial hypercholesterolemia that browning goldstein who later won the nobel for they figured out this whole pathway that led to the discovery  do things like were done with regard to viruses are not accrue search dress the question that would lead is for example a cure for polio but let's come back two thousand and nine five hundred thirty four million dollars of it money was put into this and that includes seventy seven million dollars from the recovery act and that included a wide range of research investigations  from such things as the ability to understand the genetic contributions to alzheimer's going yawn the well known a potty for two other variants that have now been demonstrated as playing a role to new imaging approaches that allow us to assess whether a treatment  working without having to wait years to see if that's the case biomarkers of response to therapy to immune therapies which are actually looking quite promising as a way to potentially reduce the formation of these beta amyloid plaques to new ideas that suggest maybe a new drug therapy approach there are thirty drug trials that are either in in place or getting ready to go into action in terms of all timers approaches so there is a lot of activity here but i  the sense that this is a time bomb i mean it's already upon us but it's going to get worse if we don't come up with better strategies for prevention and treatment in the future let's talk about cooperation and collaboration with biomedical research planning and regulatory review of goods covered therapies how do you feel this will provide predictability for industry make investment decisions very important question mr dingell and certainly from my perspective the need for close connections between and i each and f d a has never been greater accordingly peggy hamburg the commissioner of the f d a and myself have established and then i f d a leadership council and we had a public input meeting just ten days ago to see what issues that council might take on a we've begun a scientific research effort in regulatory science to try to give the f d a some new tools to be able to do review of new and creative research clinical trials in order to speed up the process of assessing whether truly new approaches are safe and effective i think it is timely indeed to have those kinds of opportunities brought forward and i think industry is welcoming the way in which dr hamburg has brought the idea of regulatory science into the present time in order to try to handle some of these more creative approaches to design and testing of new therap credits produce the new therapies and addressing unmet medical medical needs so this is the program called the qualifying therapeutic development program which is supposed to make it possible a biotechnology companies with less than two hundred fifty employees to be able to get a tax credit if they are involved in therapeutic or other clinical advances in medicine i actually think this could be a very useful program at a time where many of these companies are really starved for capital with the economy and it's downturn venture capital has been hard to come by and so this is a chance for companies that have projects that have stalled out for lack of support to be able to obtain tax credits o grants to get those things going again her doctor but there's progress years i want to get as a record why is it important that this could be targeted to the smaller biotech firms and second how will the applications be prioritized among diseases among the hope of a particular firm i think the goal particularly was to try to provide relief for small companies who have been dependent on venture capital that's no longer easy to come by hence the reason for small companies as far as the prioritizing and i each has been asked to do the reviews of the applications and we have welcomed the chance to do that we will prioritize on the basis of the likelihood of success of particular projects benefit our health system certainly thank you for that question high throughput is essentially a referred to new approaches to understanding how life works in the cell usually a human cell where you don't just look at one protein at a time or one gene at a time or one metabolite at a time you're trying to look at a very rapid fashion at a very large amount of data genomic is one of those high throughput technologies where we used to satisfy ourselves with focusing in on a single gene that cause for instance sickle cell disease but now we can sequence the entire genome all three billion last heard of the code in the space of about a week that's high throughput now that is a challenge then to be able to not just to generate the data but to make sense of it so another part of high throughput technologies is a lot of computational needs so that marriage of biology and computers which has been coming along for a while think the gentleman from connecticut and the gentlewoman from la tennessee both mentioned about job creation and that sort of thing where an application is going through the peer review process how much weight is given to the number of jobs that the grant will support in contrast to say what chairman emeritus dingell was talking about also hammers and and the significance of that disease on the cost to our overall  system how do you balance that so our primary reason for doing that first level of review is to look at the excellence of the science we want to be sure we're supporting the very best scientific ideas the second level of review then really looks at program relevance and balance and may very well take some account then into what kind of project is this and how many people would be supported but frankly the difference between the types of grants that we would support in this process in terms of how many jobs they would create is not going to be very huge most of this will be defined by the dollars that are being invested in research because in general about thirty to forty percent of our research dollars are going to support the salaries the jobs of the individuals doing the work and so whether we pick this grant or that whatever i'm sorry what percentage that thirty to forty percent somewhere in the neighborhood of what we're spending in terms of our direct costs are generally going on the personnel involved in the rectory this hour the average grant that we give out well the reason i asked that question i spent six years on the house armed services committee and was a strong strong advocate for the f twenty two raptor now it happened to be finally assembled in my congressional district and it involved at least two thousand jobs and whenever i advocated on behalf of that program i think i always rightly challenge to justify my support based on air superiority and the defense of the country and not just jobs it would be created in mocking were actual district and then again i mean i think it's the same thing with the university of georgia as well and the medical college of georgia in my hometown of augusta georgia all of these institutions are absolutely fantastic and have great research programs and i hope to see them continue to utiliz final guidelines for human embryonic stem cell research what is the status of the embryonic stem cell research being funded by the age well i'm happy to say that has resulted in  significant set of advances in terms of both stem cell lines that are available now for federally funded researchers and for new projects funded with that new capability that the president's executive order has made possible and in fact as of now there are seventy three human embryonic stem cell lines that have gone through our very careful process to make sure that they have been collected with the most stringent kind of ethics involved in the informed consent and are now available for federally funded researchers to use and they're all listed on the registry that and i has put up for investigators to look at are there additional cell lines that are waiting for approval there are more than one hundred other lines that are either already have their applications complete they're in the process of being reviewed or have started the application process and the investigator who is going to submit has not quite finish which we are course still right in the middle of so i can't give you exact numbers but i believe that is going to be higher because we've had a lot of new applications as a result of the availability of these new lines ok do you think that the scientific community would benefit from knowing that the ability to conduct this type of human embryonic stem cell research would be codified versus just contained in an executive order so i appreciate that question i think the executive order has provided the scientific community with a great deal of excitement in terms of the ability to begin to do experiments that were previously not allowable whether that would be further increased in terms of confidence about the future by having this c legislation is not something i'm probably in a good position to be able to comment upon but i understand that let me ask you this question then because what i hear from a lot of the researchers is oftentimes these research projects because their basic scientific research take a number of years to complete would that be correct to your knowledge and researchers would certainly like to be confident that their particular experimental approach can go forward without and i also want to ask you in november two thousand and seven scientists were able to successfully reverse human adult skins skin cells into an embryonic like state because they  research and is this something that the and i just also funding absolutely i have to tell you congresswoman when i read your yamanaka paper in two thousand and seven describing how he had been able to take a skin cell and by adding just fo genes to it cause it to essentially go back in time and become pluri potent the hair stood up on the back of my neck it was one of those moments where you realize this is a publication that changes everything who knew it was going to be that possible so yeah the potential here because you could then produce pluripotent cells from any of us that with therefore be possible to use in the future for therapeutic purposes without rejection because they would be a genetic match b m a now today let me ask you this to your knowledge dr collins have the i p s cells substituted for the embryonic stem cells because that's the press accounts that we've seen now we don't need embryonic stem cell research anymore they have not i think we are still trying to understand what is the same and what is different between a human embryonic stem cell which is the gold standard for a period potency and the i p s cell which has a lot of the same properties but if you look very closely by some of the measures that can be done about things like epi genetics d n a methylation and so on it's clear that an i p s cell is not identical to an embryonic and in fact a lot of the researchers have told me i'm a lay person so that's why i ask experts is is that in fact all these types of some stem cell research when done ethically are important to support because they the researchers can use all of them to sort of help validate the other research and to help in the lab i believe that those who are studying i p s cells are pretty much all committed to doing side by side comparisons to human embryonic stem cells to see what the similarities and the differences are and that comparison continues to be really important that's what the researchers tell me too i want to talk to you for just a moment about this the recent regulations that the and i h has developed on the conflicts of interest because many of us were on this committee in two thousand and four when we conducted our last investigation of the conflicts of interest at the at the n a s with your predecessor and i know that you've got draft guidelines out for comment my question  we sort of had thought we had solved this problem several years ago and i know i know doctors are when he was quite committed to to solving it what do these new draft regulations contain that are not in the regulations that were promulgated a few years and do we think this is going to help solve some of the conflict very real conflicts of interest that we're seeing with researchers so the discussion that you had with doctors  few years ago was largely focused on investigators who are and i employ these who work in the intramural program of the national institutes of health and there were some agree just examples of conflict of interest that came to light the consequence of that was to tighten those rules up very significantly and they remain i would say above reproach in terms of the way in which those have limited any kind of bad behavior what has not been taken on at that time was to consider all of those out in universities across the country who are not and i jim ploys their employees of universities and i therefore has a different relationship but recognizing that there were examples there as well our investigators who are enraged grantees were not disclosing as they were supposed to their financial conflicts and i has now stepped forward to require institutions to take a much more heavy hand here in terms of checking out whether their investigators are engaged in kind licks of interest and there are several proposals here as part of the n p r m that we are now seeking comment on that would require a much more extensive disclosure of any kind of financial conflicts that investigators might have including posting those on a public web site so that it's possible for anyone to see what's going on and not have this being done behind a curtain this also requires the invest the institutions to have a much clearer plan about how they're going to review and notify about those financial conflicts again we're seeking  comments on those over the course of the next month there has been this recent observation that in one instance an investigator left one university and was able to escape sanctions on that basis and we are now considering what could be done to prevent that in the future as  well so there is a lot that we can do here although we are to some extent limited in that our relationship is primarily with the institution and not with the investigation but if you do if you require the institution to make disclosure than that then that burden would be on that and that's the goal of the n chair now will recognize mr whitfield thank you very much and dr collins we appreciate your being with us here today very much i know that and i was involved in a lot of projects like the molecules that are biological they have years and so forth leading to what i guess we refer to as a big biology or human matter bone nano project and i was wondering if you could just give us sort of an update on the money that's being spent in agencies on this and what the what you view the prospects are for this type of initiatives so i really this is a very exciting area this is bringing chemistry and biology together in a new and exciting way and making those resources available to academic investigators is another kind of high throughput science the idea here is that if you're interested in a developing some kind of a compound an organic molecules that might ultimately become a therapeutic drug you need a way to empower academic investigators to get engaged in that process and not just hope it's going to happen and that is giving us the impetus to put together a series of four of these high throughput screening centers and you mentioned one of them the n h chemical genomics center but there are three others collectively we spend about sixty million dollars each year on these centers but it makes it possible for an academic investigator who's just made a basic science discovery to move into this possible translational application in ways that just weren't happening before i would like to see as push that really hard right now i don't know because we're in a circumstance where there's been a lot of basic discoveries that  perhaps even more ripe for translational applications than ever before but many of them are too early for the private sector to necessarily see them as ripe enough for them to take on if we could come up with a strategy where academic investigators take it a certain way to risk a project and then handed off to a private company who would then carry it the rest of the way to approval that's a pretty good model companies like that we had and i like that this may be our path towards the future you know and it looks like there was be some really exciting breakthroughs and an opportunity here for partnerships with private companies as well i totally agree and i think that is a new model that maybe hasn't really been tried to this extent before companies would tell you they are also really looking for new models the development of new therapeutics has not been going all that well for them in the last few years here's a way to speed that up and i've also been told that some of these new techniques quote  in analyzing the dispersants that they're saying about using in the gulf oil spill is that correct that is correct so we have a marriage here between the e p a and the national institute of environmental health sciences the national toxicology would welcome that congressman just let me know will set you up with a quite a tour and i guarantee you you will be amazed at the way in which this operation has taken shape with a robot that is capable of screening hundreds of thousands of chemical compounds the benefits on the tax credit not there but the health care bill i've been crying and asking for hearings on the health care law since i became ranking member i'm glad he took the opportunity to address the health care law i do think questions like that could be done in a hearing on the overall law i just want to put in the record that twenty seven billion in tax increases to the pharmaceutical companies makes it more difficult for them to do the r and d for new drug development the ten ninety nine provision for small companies that require a ten ninety nine form for every six hundred dollar cost added will add additional burden to small companies to be able to do that so where there may be some tax credit help i think a lot of people would say that the burden on the small pharmaceutical companies are increased under the health care law and i'll just and on the i p s system as my good friend was talking it may there may be scientists who want them both to go on but the i p s does a data very important question that would help unify us versus divide us it does address some moral moral ethical problem that many americans have on embryonic stem cell research now you know back this is christian some thank you madam chair and welcome again to the collins as you know one of the great successes in the health care reform bill was elevating the national center on minority and how this might be researched and institute something that we are very excited about and all of us who have worked for years on health equity or the elimination of health disparities are really anxious to hear how the role that you envision for this new institute and i so maybe my first question would be what are your plans to ensure that the new institute will play a pivotal role in helping to ensure and coordinate all research efforts across all of the other institutes and centers so that they would include health disparity elimination variable and measures health disparities under the direction of dr john ruff and and this does bring that part of it into the same status as the other institutes that direct many other kinds of research it certainly underlines the this further strengthens the hand that dr roughen holds and i think we all welcome back this will of course further add to the importance of those rolling five year health disparities strategic plans  which i am h d is charged with putting together with input from all of the other institutes it will take a lot of collaboration between the institute but i can tell you having been it and i know for seventeen years the importance of health disparities and the investments that need to be made there is certainly a shared goal across all of the leadership but it's nice now to have an institute that stands up in a very visible way to take charge of that well thank you and in a recent interview i think i was in science you spoke of the pressures of year to year budgeting and the possible impact of our nation's current economic situation but if you had defined as you said and you alluded to the possibility of if you find yourself grant budgets or back on cutting back on some areas of science i have to decide which is to get the three point two percent increase and which don't i hope that you'll consider that the new institute is starting out brand new is grossly underfunded so is part of your plan is to implement the patient protection and affordable care act to bring its budget in the line with the other instances of course i would love to see that happen and of the challenge of course as you probably well know is all of the other stresses that we have on the overall and i resource total as we face what may be a particularly difficult year in fiscal year zero levin recognizing the economic downturn the ending of the recovery act dollars we're going to be stressed in all kinds of ways trying to maintain  the sources in the environment of the by medical researchers who are so critical to our future estimates are that our success rates while evan may fall to historic lows across the board and of course you c appreciate i hope that that makes any special kind of corrections even more difficult than they otherwise would be we all hope for times where those things will be easier but i don't think that next year is likely to be an easy time but we don't expect it to be an easy time but they're starting out at such a low budget and if they're really going to function as an institute their budget would really have to be greatly increased and given the extent of the health disparities in racial and ethnic minorities you it really needs to be a priority in your testimony both written and what you gave here today talked about diabetes alzheimer's cancer all of which disproportionately impact african americans and other racial  ethnic minorities but african americans in particular yet there was no mention of how the disparities of the disparities or how i would direct research to close those gaps in that same interview that i read you talked about a big thing that heard was and the outcome of that big think with several areas that may need more focus and attention where health disparities one of them and if  not is there going to be a big think about health disparities i appreciate your question yes it was one of the very significant conversations we had the big think was a meeting where i brought together about fifty five or sixty individuals that i think are able to really look at the entire landscape and give advice about where investments are most needed and most opportune and certainly one of the groups we had focused of the three groups focused very much on this general area of what can we do about the health of the nation and health disparities emerged as a part securely powerful part of that reed tuckson led that particular discussion good and i think i think we got some some strong messages and of course the summit that was held and a half ago  roughen organized very much focused on health disparities and itself produced a blueprint of additional needs only i would want to say although i would agree we're not putting enough resources into health disparities we're not putting enough resources into many other things too but i hope it is clear that the investments in research on health disparities are not limited to that one institute that every institute enrage has an important portfolio in this area and dr ruffins job is to be sure that we are coordinating that in a way that we get the maximum benefit from the dollars we're spending right i'm aware that the other institutes and centers are doing research but we want to make sure that he does that that court the ability to coordinate the also said in that interview that we have a lot to do and the thought of a quote at least part of it is a quote we have a lot to do to figure out how do people decide about altering behavior in the case of racial and ethnic disparities or any health issue  behaviors one of the many issues along with environment access and others that requires research does your response will you say we have a lot to do to figure out how people decide about all doing behavior those that respond mean that we will see a greater emphasis on behavioral research and i i think it's timely to focus a lot of attention on behavioral research if we're talking about prevention especially so much of what we're learning won't do any good if it's not transmitted to people in a way that they understand and if it doesn't motivate health behavior changes whether it's diet or exercise or other activities and i think you will see in fact we have one of our new initiatives in the common fund is called the science of behavior change how do we learn more about what actually is involved when somebody changes their health behavior and how do we inspire that in circumstances where we have the evidence but we haven't been very good at actually getting the result you're going to see a lot more that well you partly answered one of my other questions about what was what were you focusing on in the common fund but i'm going to yield back minutes i have to  pick thank you madam chairman dr collins aeration article in the new york times claims that the human genome project which you worked on has not yielded many medical benefits is that an accurate statement that came through loud and clear today's new york times and a different article by a different reporter has i think a much more upbeat and balanced view about the way in which general makes is inspiring new ideas about their beauty in sort of responding to mr wade's rant i sat down for ten minutes just to see if i could come up in ten minutes with twenty five significant advances in medicine that come out of genomics and i had no trouble doing so somehow they didn't appear in his article ok thank you to follow along the line of questioning of the chair chairwoman you said that regarding the regulations on conflict of interest that there had been agreed just examples of conflict of interest with federal employees can you explain the type of example you're talking about this well i'll tell you the one that got attention in the last week or so a psychiatrist who was doing research at emory university dr charles nemeroff was found to have been receiving a large sums of money from pharmaceutical companies that he had not disclosed those sums of money were in the same general area as his own research casting into some question whether his research results were in some way tainted by the receipt of those dollars when that finally came to light he was he admitted that he had been receiving those funds and claimed that he didn't know he had to disclose them emory university in consultation with the end i decided to strip him of his chairmanship and to also remove him as the head of his particular grant dr nemeroff then actually in the space  year or so after that sanction moved to a different university the university of miami where because of the current rules because and i choose arrangements or with institutions and not with individuals dr nemeroff was now able to apply for new grants he has not received any grant funding from and i am quick to tell you but he was able to apply and i think that raised a lot of questions let me say because i think it's important to put this into the record that the individual who was also involved in a certain way  innuendo in this dr tom insult who's the director of the national institutes of mental health was in some way implicated by a reporter as not having conducted himself properly i would really like to defend dr insel in that regard and to say that in his many years of service to the and i each dr insel has proven to be an exceptional scientific leader has led the n i m h with great skill has encouraged innovative advances in biomedical research he's been a person of great integrity a dedicated and visionary public  fortunate to have him on my team and i thank you let me go a little further on embryonic stem cell research about if you can ballpark it how many years have we been doing research cells that have produced improvement for human patients so there has only been one clinical trial for human embryonic stem cell that have been approved by the f d a and that is a trial by the company called hiran for spinal cord injury and they have not yet in rolled the first patient because of safety concerns but are expected to later this year so it's still early ok what about with adult stem cells have there been any clinical trials using adult stem cells that have produced improvement for patients so adult stem cells covers a lot of territory certainly a stem cells found in the bone marrow have been utilized for a long period of time for bone marrow transplantation and have saved many lives in circumstances where that was an important task but importantly those kinds of adult stem cells don't have the same pluri potency that embryonic stem cell would and so their applications would be limited to certain things it would not be possible for instance to use those for all the applications that embryonic stem cells are being contemplated but there have been human protocols developed for human treatment right for therapy for human patients with adult stem cells a year where again bone marrow would be the one where most of those experiments have happened so that's been very successful in that limited set of applications but would not have been possible to apply for other applications like part of what about with i b s shelves i p s cells are a new development as was also already raised very exciting not yet tried in any human applications we have just announced the formation of an i p s cell center on the n i j campus to try to speed up those applications for human therapeutic purposes but it will probably be a few more years before we are sure that they are safe because those cells are capable of forming new stem cell lines for research and i just wanted to ask a broad question on that subject as to how that process of a part of approving new lines is working and if you sort of feel that that process is moving swiftly enough and a sufficient manner so i think it was seven new cell eleven stem cell lines that were put forward by the advisory committee through advising me i have not formally made the decision about expect to do so in the very near future that brings to seventy three the number of human embryonic stem cell lines that have been approved for use with federal funds i think the process is actually working quite well basically we ask investigators who have derived the stem cell lines to supply documentation about the nature of the informed consent that was used to obtain those because we want to be absolutely sure that that was done at the highest ethical standards if they are able to do so in a fashion that is right down the line to every detail consistent with the  guidelines we approve those administratively and we can do that quite quickly once we have all the documents but some of the stem cell lines were derived years ago and the process of getting consent and the specifics of the details have changed a bit over that time so we have a working group of distinguished bioethicist and biologists who look at those circumstances to try to see whether those particular stem cell lines at least follow the general principles that we would like to see and if they agree that they have then they put those forward for approval as well they have approved quite a few of those interesting late last week though they recommended disapproving a whole set of lines where they thought the consent had a problematic clause in it which was basically what we call it sculpted tori language which according to the common rule really should not be informed consent process and they didn't feel if we were doing this carefully that we could allow that that was a bit of a heartache because that was a whole lot alliance that a lot of people were i think hopeful they would get access to but if we have guidelines if we have ethical standards we probably need to pay real close attention to them and i appreciate even in the face of some resistance you are you know you're holding that line i think the  future of this research depends on people believing that there are ethical boundaries and qualifications for this research and so i think it's it's to your credit to just switch topics fairly dramatically  wanted to go back to something i talked about in my opening statement about the intersection of private and public research i've got a really interesting company in cheshire connecticut alexian pharmaceuticals that is produces a drug for a rare blood disorder that i won't attempt to butcher the pronunciation of the drug is called solaris and it's an example frankly of a rare instance where a company has been able to commercialize a drug with a fairly limited application a couple thousand cases in the united states at a given time and i guess my question is this there are a number of different factors that go into your decision on what kind of where you place research clearly the top of the list is what's in the public interest what are the diseases that conditions that we have the greatest national interest in confronting but i would assume an element is also what kind of research is most likely to be done in the private sphere and what research is most likely to not be done there and has to be taken up by by by public funds and so in the context of rare diseases i guess i'd i'd ask for your your opinion as to how that calculation plays out with regard to rare to and whether we see more companies like this one in cheshire being able to do this on a private basis or whether we continue to see the need to have a larger public role in that space i think it's going to be a little different for each disease and i appreciate the question i think for many rare diseases the economics just aren't going to be sufficient to inspire a company to make the investment in taking something all the way to therapy if there's if there's not already a pretty good idea on the table and what academic investigators supported h can now do increasingly is to make that possible there's a new program called trend which stands for therapeutics for rare and neglected diseases funded by the congress twenty four million dollars this year fifty million it requested next year in the president's budget which is exactly to try to provide that kind of de risking opportunity for these rare disease projects not to take them away from a company's interest but actually to try to get them far enough along to inspire a company's interest and we're seeing that happen and now that program has already started five new pilot projects for rare diseases one is for a disease called hereditary inclusion body myopic the now who has heard of that relatively few people affected but there's a very promising therapy and it just needs a little push to get to the point where a company like the one in cheshire might well be willing to pick it up we're trying to do that and with rare diseases not only will they affect real people and you want to do something for those real people but we often learn things from rare diseases that  broader reach my own lab because i have a small research lab at a night studying an incredibly rare disease called progeria which is a form of most dramatic premature aging and we could only find thirty kids in the world to try on this clinical trial which is currently underway at an amazingly rapid pace because we just discovered the cause of this a few years ago but what we're learning from progeria is turning out to have pretty significant implications for normal aging and maybe that will turn out to be one of the most exciting things that's come neglected diseases for just a moment this past week in the new england journal of medicine they had an article about the curious case of cultural scene a drug that has been around for three thousand years and yet the f d a decided that since we've never stud this maybe someone ought to so someone did and they were given exclusive rights for cultural scene for three years an additional seven years under the orphan drug act so people would suffer with gout cult see the second line drug of  choice but went from pennies to dose to several dollars a dose but people with familial mediterranean fever were called seeing really can be a benefit are now left with facing a situation where they their costs are going to significantly go up so why would the what the n i m h be interested in proactively funding any of the research on some of these compounds that have been around for a long time to sort of preclude this problem from from happening again to some other illness again not many people with a meal or mediterranean fever of the figure they gave the new england journal medicine was one hundred thousand i don't know whether that's in the country or globally but it's it's not a large universe of people but the people who have it are now significantly affected by the activities  food and drug administration dr burgess that's a great question dr daniel kastner at n i is the world's expert on familiar mediterranean fever and i know there's a good deal of anxiety about what this is going to do in terms of the cost of taking care of these patients yeah exactly i think this is something that and i can do i mentioned this program called trans the therapeutics for rare neglected diseases and one of its explicit goals is to try to identify compounds that have been out there for a long time and repurpose  uses for rare diseases where they might actually have considerable benefit but nobody actually tried that because if you do that you've ended up with something which may be both inexpensive and actually you're pretty close to the point of being able to ask f d a to approve it without going through those long long steps of developing a totally new compound it's a great idea we now when we are searching for a new compound for a disease that there is no therapy we always make sure we check all the available ones that have already been given to patients that we know are safe and once in a while you turn up with something really exciting it's a really good player on the you famously were interviewed on the colbert report and talked about attracting more young people to the to the study of science and so you could do to make to make science appealing to young people i think the term that was used to make science sexy how are you coming in that endeavor well maybe you're a better judge of that members than i am as you may recall that caldera suggested that if i really wanted to attract scientists into the field that i needed to sort of loosen up a little bit take your glasses off and shake out your hair against my better judgment i did that on national television but i'm not doing that this afternoon so we are really interested to get serious here in terms of trying to recruit the best and brightest of the next generation we were big participants in national lab day where we sent a lot of scientists out to high school classrooms to try to make science a little more real and hopefully less geeky than it often appears to be to the next generation we're hoping to play some role in whatever happens next in terms of beefing up science in k through twelve education because clearly the way science gets taught to a lot of young people is very exciting and it should be this is the century of science especially biology so many opportunities for people that we would love to see them just jump on  and join us because this is going to be an exciting time beyond any that anybody's experience before in the next few years we're going to learn all this stuff we're going to figure out how to apply it what a great career which i don't and i know this question has already been asked but i do i was out of the room and i want to ask you to address briefly the doctors or when he came to us right before he your predecessor right before he left the post and talked about the wondrous things that have happened with the human genome in cracking the code the single nucleotide polymorphisms where there were just three  two thousand and three when i started in congress there were no i don't remember how many he said last last fall and there were more being discovered literally every week but there was a reduced report that there will be there was some disappointment with the clinical applications of sequencing the human genome so where are we on that well with the brave new world that doctors are going to talk about or are we on a dead end street you know the first law of technology says that when you make a major discovery the anticipation of its immediate effects is always going to be overblown but the anticipation of the long term effects will generally be underestimated i think that's going to be true of genomic as well there were a lot of wild claims made in two thousand about how this was going to change ever and i knew that there was a lot more steps involved from understanding what the letters are in the d n a code to figuring out what to do with them but i think the advent of personalized medicine is certainly coming along now in a quite gratifying way in today's new england journal by the way and i put this at your place is an article by myself peggy hamburg called the path to personalized medicine going through some of these really exciting advances and also making it clear how f d a and i are working together to support them so i think any declaration that the outcome of the genome project has been disappointing is probably just not based upon realistic expectations i do think that revolution is going to be quite something when it takes full shape in the next ten years i want to come back to that partnership of an ag these tests in fact our committee our subcommittee on oversight investigations is going to be conducting or is conducting a bipartisan look into that and i guess at this point out what i would ask you is would  you be willing to work with our staffs on both the majority the minority side as we as we look into this issue obviously we want this done correctly we don't want people hurt or misled by the by the process and i think that's what what raised the the initial concerns when when one company was marketing in a commercial drugstore and then withdrew it and it raised a lot of questions but you know there's no question of the value of the of the procedure on the issue of the f mean you guys you guys got a ton of money in the stimulus bill i mean it was almost immeasurable the amount of money that you got and no one had ever seen that much money before even in government and so you produced you put it to work and you're producing all this stuff now the f d a is as we heard the other day to another subcommittee hearing kind of like a little bit like a bottleneck in the in the pipeline so how are you and dr hamburg working out these problems between your two institutions so two ways and probably many other smaller ones but two major ways one is the formation of this and i f d a leadership council which will have senior leadership from both agencies at the table on a regular basis with projects and ideas coming to us both from the industry and from the advocacy groups all the stakeholders to really try to say where are the bottlenecks what are we going to do about let's get serious here the second is to have a actual scientific organizational plan about how are we going to give the f d a some additional information about how to do regulatory science because many of the things that are coming at them are new if you have a clinical trial where everybody in the trial is on a slightly different combination of their puter as will happen soon for cancer work combination therapy is going to be the name of the game with all these various targeted therapies that are possible how do they review that we need science to understand that that particular regulatory science program is funded jointly by f d a but actually because f d a is a little strapped on cash has come up with a larger proportion of the funds and we're happy to do that and i'm happy to work with a scientist and a physician of the capabilities of dr hamburg i think we're actually forming a pretty good partnership and we expect a gentleman time's expired at her questioning five minutes and then we will recess for the votes we will return immediately upon the completion of the votes because their members want to question and if dr collins agrees if people have additional that they're popular because they include training funding and there's a concern about where training funding will come from the t s a grant applications are fine and will applicants still be eligible for training dollars from  the national center for research resources or other institutions and i appreciate your question because the c t s say there are very important part of a night clinical research network in our plans for   in all kinds of new and innovative approaches to clinical applications therapeutics diagnostics and so on we have currently funded forty six c t s aids around the country it's a very competitive process there is another round of competition that has just gone on and there will be a few more announced as a result of that and then there's one more competition coming next year but the goal was to find a total of sixty that pretty much will exhaust the dollars that are possible to put into this which is now coming close to half a billion dollars for these sixty centers we do very much expect that they are going to link up in interesting networks both regionally and nationally so that the whole will be greater than the sum of the parts and every one of the c t s days has been encouraged as your question pointed to focus on training to focus on bringing together disciplines that may be in the past had not talked to each other even within an institution but also to building networks around each of the c t other local institutions including community hospitals and community outreach is a big part of this as well i wish we could come up with the dollars to fund one hundred instead of sixty but sixty is probably as far as we can go we do expect that out of this will arise a whole new way of approaching clinical research i've been to visit now four of these and i'll go see another one actually tomorrow the one in st louis it's very interesting they're all those different and they're taking advantage of what they have locally in terms of special expertise to make themselves different and it's pretty exciting when you see the ways in which they have people talking to each other who never would have been in the same room before and they are certainly encouraging training in clinical research which has been a big concern because many physicians have been sort of staying away in droves when it came to research because they had big debts to pay it wasn't clear what the pathway was and wasn't clear where they were going to get men toward the c p s a's are really helping to turn that around it's going to be a tough process of course to turn that around because there's so many other things pulling on physicians but i think this is going to be really interesting few years to watch the first seat the essays are just now coming up for their first renewal so we certainly believe the training is critical for our future and every one of the institutes has a special a way of trying to support that the national institute of general medical sciences and i g m s is a particularly important part of n i s for training organizational efforts and they fund for instance the mt ph d training programs the medical sciences training programs which are a way in which we are hopefully nurturing a particularly energetic and inspired group of future leaders who get d and the ph d degree we have been both concerned about whether we're training enough individuals and whether we're actually supporting them adequately one of the things that we are trying to do is to catch up on the stipend for trainees which have been flat for a long time and we are clearly asking people to remain in training positions many of them were already with doctoral degrees at really quite quite low salaries and that's beginning to be a problem in terms of recruiting people to do this we're going to try to pick up a little bit on that again resources i keep coming back to that with many of the questions with the fact that we are facing potentially a very difficult time in f y eleven and perhaps beyond we would love to expand training programs but where where would we take the dollars from in order to do this with so many other needs for disease research for grants and so on we are every day trying to figure out how to get that balance right committee will be in recess tell the completion of the third thank you committee will come to order and the chair recognizes mr space i wanted to focus my questions today however on one specific disease and there's diabetes as you probably no recently conducted a comprehensive study in which it was determined that these are two thousand and seven numbers by the way which on that italy are significantly lower than they would be today but that determine that we're spending as a nation about one hundred seventy four billion dollars every year most of that money represents costs associated with health care for those suffering from diabetes and particularly those in the chronic stages but a significant portion also represents lost productivity and this is one disease in one year we spend more as a nation than we did for example in just about any war year during the war in iraq obviously if we were to be able to cure this  was diagnosed soon six years of age and after his diagnosis my wife and i we knew very little about diabetes at the time but we began to educate ourselves on this impression that we were ten years away from a cure in this was based on exhaustive studies and research that i could commit myself because of an obvious personal interest and here we are  fourteen years later and i'm still hearing the same thing maybe ten years away from a cure and i'm familiar with the closed loop system and the concept of an artificial chris that  every type one diabetic in the country had it would eliminate countless episodes of suffering premature death and disability but would also end up saving a lot of money in the long run and my question is to you is regarding the special that bts program and stand that this has been funded it on a hundred fifty million dollars per year incidentally sounds like a big number it is a big number but if we're spending one hundred seventy four billion dollars a year combating this disease and what it takes ten percent of that figure and spread it out over five years say seventeen billion is just around the downwards to fifteen billion spent three billion dollars a year or five years in my opinion is we could give every type one diabetic ng ited states a closed loop system that worked and it was reliable and would more than pay for itself within a few short years  and possible cure when it comes to type one diabetes services space i appreciate your question especially the personal experience you've had with your son nick and i think parents who have had that experience must be like you frustrated that we don't yet have a better solution to this this clearly important and very serious disease of type one diabetes the special diabetes program which as you point out is one hundred fifty million dollars a year it is set to expire at the end of fiscal year eleven and i know there are some anxieties amongst the investigators working in those projects about they're going to have to phase out their efforts at that point it certainly includes a number of i think rather ambitious efforts and certainly the artificial pancreas is one of those dreams that we all hope will come true in a broader sense in the not too distant future but obviously many challenges there in terms of how you get the engineering and the biology right so that you have a system that is reliable because obviously any system that has the capability of delivering insulin needs to be fail safe because overdoses would be potentially very serious i think there's real progress being made there but i'm sure from your perspective and from mine it's lower than we wish it could be in terms of type one diabetes and what causes it in the first place we have i think a much better idea of what the genetic factors are we now have accounted for more than half of the heritability of type one diabetes and there's this program called which is now following several thousand children who are at high genetic risk to see whether in fact they begin to show some signs of the auto immune component which is a sign that the disease is getting started because as you probably know by the time a young person shows up with actual symptoms of diabetes most of those beta cells in their pancreas have already been destroyed by the automated process and the best hope is to catch that early and then treat it with appropriate drug therapies including a new therapy so i would say there is reason to be optimistic that the steps are moving forward but i hear what you're saying that you heard that in one thousand nine hundred six i mean patient to i want to see this disease conquered i want to see the best idea supported to make that come true it is challenging to figure out for us exactly how to take the resources we've got and apply them in the best way we do what we can in terms of estimating what's going to work and what's not going to work there's steps forward sometimes are setbacks especially in clinical research where you know you have to come up with something that is both effective and safe but i think with the way that the field is moving it's hard for me to see that that dream you were promised in one thousand nine hundred six is not going to happen it's just very hard to put timetables on these kinds of trajectories i wish i could apart from the obvious benefits associated with the alleviation of human suffering and saving lives is that your assessment that that would also save a lot of my a lot of money absolutely and i certainly hear what you're saying about both for type one and for type two diabetes or the huge sums of money that our medical care system are going to take care of individuals we also have very vigorous research programs on type two diabetes particularly now and prevention where we have the ability perhaps for people who are starting to tip over into that to be able to prevent the full onset of the disease following up on the results of the diabetes prevention program which has taught us how to do that with simple things like diet and exercise and lifestyle changes that can be successful if properly managed and coached and just finally in the event that the special diabetes program were not to be reauthorized with that have a deleterious or negative effect on advances in the field it certainly would mean with all the momentum that's been generated because of that we'll see how far i get into this first i want to just make a comment i was here present when dr christianson was talking asking questions and bringing all of our attention to the issue of health disparities of minority populations and i just wanted to share with you my interest in expanding the way we think of that to include also the lesbian gay bisexual and transgender population in years past  information and i'm sad to say that the answer was you know either we'll get back to you or we're not aware of any even asking questions related to sexual orientation or gender identity to be able to gather the evidence and the data i don't know what role you might have personally played in the focus that the i o m is now putting on this question but i'm delighted and want to commend any role that you did play and bringing that together because i think it will examine the current state of knowledge and hopefully produce recommendations of where we can obtain even greater knowledge that's just a comment really with the rest of my time i wanted to get as much of a status update  on some new programs and etc if you don't have the information available today please feel free to respond at a later point but last year the christer christopher and dana reeve paralysis act was signed into law i had authored the free nd the other would grant funds to maintain a clinical trial network with sites focusing on people living with paralysis wondering if you can give me any update on the implementation of this rather new legislation happy to  very quickly in terms of l g b t we are in fact supporting and we've done an inventory here some two hundred seventy two projects through an i t research costing over two hundred thirty nine million dollars so there is a research portfolio there but we are very much looking forward the results of this i o m study which i strongly support to try to learn more about the needs and the research opportunities and i should be addressing in terms of the christopher and dana reeve paralysis act yes there certainly has been as a consequence of that the formation of a network here which is funded by the national center for medical rehabilitation research which is within the child health institute traditionally that's where rehabilitation research has been located but together with the neurology institute and the national institute for bio imaging in biotechnology that resulted in solicitation of a whole bunch of interesting proposals and in fact seven sites that received the best peer review recommendations are now being funded by those three institutes and they're going to look at biological medical behavioral social aspects of paralysis and very much try to make this a consortium effort as opposed to a collection of disconnected research projects great thank you  recovery and reinvestment act funds were awarded through the national center for research resources to address the critical research infrastructure needs across the nation but i would say that the funds  were inadequate to meet all the critical needs that are out there and so i'm wondering moving forward assuming once the recover three dollars are expended will funds for construction or remodelling of facilities be a priority and be allocated to and c r so yes where we were delighted in the recovery act to have a billion dollars to put forward for construction and renovation and those grants were rigorously reviewed and fairly recently decisions made and announcements put forward and those one billion dollars are going to support grants and new construction one hundred forty six projects in forty four states and the district of columbia so this was clearly one of those cajuns where dollars from the recovery act we're going to support both medical institutions and kaname in terms of the reconstruction efforts future funding is not included in the president's budget for f y eleven and i must tell you that when budgets are tight as we fear they may be in f y eleven and beyond dollars for this kind of application is often hard to come by the and that's something the congress decides we don't decide if we don't have an allocation in that part of our budget we're not allowed to spend the money on buildings and facilities so we wait to see what the appropriators tell us i see i've ran out of time so i will conclude thank you thank you if the general idea has a couple more questions we had said we would be willing to do a second round oh well i would i would be delighted to a couple more minutes and then i will need a second round the university of wisconsin has long been responsible for running the national stem cell bank this year we've been informed that i would like to take this process through a competitive rebid and i wonder if you could tell me a little bit about what that timeline and the selection criteria will be for the rebuilding of the national stem cell bank so the university of wisconsin has been such an important leader in this whole field i think i mentioned earlier the contributions of jamie to and the way in which the wisconsin effort led to the formation of this bank was certainly an important resource for individuals who would want to get access to these stem cell lines and knew that they had been properly maintained and they could count on their quality and now that there are as i mentioned earlier in this hearing seventy three such lines that have been approved and going up we are going into the hundreds before long there is a big question mark in many people's minds about exactly what's the right way to make sure the lines are accessible to investigators who want to use them some will probably be used more than others  will be some favorites no doubt as has been the case in the past what we are trying to ascertain is whether it is actually time for this kind of banking activity to move into a competitive private sector enterprise because certainly in other circumstances where there are biological resources that many individuals are interested in the time sometimes arrives where it is more efficient and more cost effective to have this conducted by a small company who would then be basically recovering their cost of distribution by a modest charge what we don't want to do however is to have that end up resulting in excessive costs for and i jest a gator's that would sort of defeat the purpose so we are still exploring what the right pathway might be but it seems likely that we will want to set up a circumstance where there is more than one bank because obviously you wouldn't want to have the entire field resting upon a single resource and it will be i think a few more months before we have a clear sense of what's the best strategy for making sure that we have cell lines that are maintained at high quality and made available to investigators at reasonable cost i have two more but if to our topics in the fiscal year two thousand and ten appropriations bill congress called on the office of the director to engage in specific efforts to improve research on women's pain including coordination of a trance institute research initiative in fiscal year twenty ten that will support studies  identifying common logical pathways and with the goal of identifying potential therapeutic targets and secondly a conference to be held along the same lines could you could you tell me about the status of your efforts in this area so we certainly read that part of the health care reform bill carefully and we're happy to be called upon to work in this area of pain which we agree is extremely important there is a pain consortium at n i s which has been in place  several years which involves quite a number of the institutes that have an investment in an interest in this area and this is another sort of encouragement for that effort to get even more involved we are in fact planning the conference that is being asked for which i think is to be hosted by the secretary in the not too distant future because i think there is a timeline on that and there is also an ex or in the affordable care act bill about coming up with some new common fund ideas for pain our research which is certainly something that is being looked at by the pain consortium to see if there are opportunities there in addition there have been a number of specific workshops focused on particular subsets of paid to try to bring investigators together with new ideas and i must say there are some exciting things coming along when just mention one which is another example of how rare disease can really lead you to a very interesting new hypothesis there are rare individuals who are born with what's called congenital insensitivity to pain particularly dramatic publication a year or so ago studying pakistani family where the number of the siblings were completely unable to feel pain which is not actually good for you these siblings ended up involved in street theater where they would walk on hot coals or they would actually pass knives through their arms obviously amazing people going by not feeling pain but of course putting themselves in gr cause for that has been discovered and they are basically missing one of these sodium channels that normally transmits the pain signal in the nervous system and they're otherwise entirely well that's a really interesting finding that says if you could  y a drug a small molecule that blocked that activity in the rest of us transiently that would be an incredibly powerful pain reliever and analgesics and many companies have now jumped on that description of rare family thinking this might be the best clue we've had in a long time for a totally new approach to pain management and my last question and you've been very tolerant of my battery of a different updates  curious about the national research service award funding program are there any efforts underway to reframe that change the amount or the allocation of training funds under that program so we are aware that stipends for an r s a trainees the truth kirstine in r s a awards have fallen behind over the course of many years without having a substantive increase in quite a long time and so we have supported a six percent increase in the current circumstance to try to make up some part of that and there has been a sense of the congress and we agree with this that we should continue to try to catch up a bit here we depend on these trainees to do a lot of the frontline research it's part of their training but they're also really important for the productivity of the enterprise we certainly wouldn't want talented mines to go elsewhere because they just couldn't figure out how they're going to buy the groceries so we are doing what we can to try to catch up on that recognising that we have fallen behind it of course comes at a tough time if we're seeing a more stringent budget looming in the future if we are going to increase stipends we may have to decrease the number of train s because there's got to be an ultimate reckoning and we would hate to do that too so it's just one more example of the tough decisions that may be facing us at a time where the economy is struggling  recognize thank you thank you madam chair doctor you know before i came here before i was at my other subcommittee on the engine commerce committee which is the the energy subcommittee and they we were questioning the the b p executives and the other executives and i can tell you this subcommittee is much more friendly today yeah i'm glad of at this table it's not out of the room but i want to first of all personally thank you for the good work that you do i followed your work and i'm very impressed  spoke at the new dams several weeks ago i was there and very very impressed with what you have to say so thank you first of all for all the the good work that you do at that meeting with the new dams you mentioned a drug called i think it was a risk ressa yes about breast cancer i'm wondering if you could just again tell us about about that and and if somebody would want to sign up for it  or anything like that is that possible there are certainly trials going on to so i rest for which the generic name is just fit and the ebb is one of these new targeted therapies for cancer about which there is a great deal of excitement so basically works by blocking the action of a particular kinases which is an enzyme that is normally presidents cells but in cancer cells from some tumors gets overly activated by a mutation and if you are somebody who has a cancer that has that specific mutation i rest can be extremely effective i think i told the story of a woman whose cancer was diagnosed when she had lung cancer which was already medicine added to her brain in two thousand and two and expected to live for a few months she's doing fine today i'm happy to tell you because this drug was the perfect antidote to her particular tumor mutation now that's the good news the troubling part of this is that i ressa doesn't work for about eighty five percent of people with lung cancer because they don't have that mutation they have some other way that their cells have gone haywire and are causing the cancer in their system but it does say that we are reaching the point with i wrestle with the drug i talked about in my opening statement which is another example of the sort of targeted therapy for cancer where you can sample a tumor and say ok what's here and then look at your list of drugs that are available and do the match and as that match gets more and more possible because the list of drugs and the list of targets gets longer we're going to see a real transformation in the way that cancer is approached and that's a great thing that it's personalize their p we're talking about now instead of giving everybody the same dose of chemotherapy thank you i'd like to ask you a question on just a general diabetes according to the american diabetes association just shell diabetes affects two to five percent of all pregnant women and that's one hundred thirty five thousand cases every year in the united states and it occurs i'm told in pregnant women who've never had diabetes before but develop it between the twenty fourth and twenty eighth weeks of of pregnancy and while it generally goes away after pregnancy you can have health impacts for both the mother and the baby i'm told that there's currently an insufficient system for monitoring discuss some of the research currently underway to address the need for comprehensive information on the causes and best treatment of just facial diabetes it's a very important problem and one that we need to address both in terms of research and in terms of just the public health and trying to identify individuals who have risks of this sort gestational diabetes is in fact a risk to the pregnancy as well it is associated with the higher birth weight with  prematurity and certainly something that you want to try to control because the mother who has out of control gestational diabetes is also likely to end up with pre eclampsia but more than that if you get the mother through this experience and most do the risk that that woman will go on to a full blown diabetes are substantially increased and ultimately about half of women who have gestational diabetes will ultimately become diabetic so it is apparently one of those circumstances where pregnancy is kind of a stress on the system and somebody who may already be predisposed it becomes more clear in the course of a pregnancy that that's the case so we need both better methods to monitor during pregnancy which means our medical care system needs to be of course accessible to those who need those services it is not particularly difficult to assess this if that person is getting adequate prenatal care and we need of course methods to prevent it in the first place and it appears at the present time that the risk factors for gestational diabetes are pretty much the same as the risk factors for type two diabetes in general which is to say family history which is to say body mass index obesity and exercise and what we've learned in terms of how to take people who are predisposed to diabetes and prevent that from things like the diabetes prevention program are probably very applicable here as well but certainly women who have gone through this experience and had gestational diabetes ought to have particularly good opportunity to recognize the risk to their future health and to be given the same kinds of interventions that we now offer to people with pre diabetes to try to development so i'm wondering if you could describe the program and offer your thoughts on it i appreciate the question because i do agree this is very exciting program my father in law has shocker marie tooth disease this is a disorder that's not only something from my clinic but also from my family experience and this is an interesting disorder which causes a weakness of the legs particularly but also the hands over the course of time and can be quite debilitating but it is well understood now what the cause of that is the genetic abnormality which has been has been now laid out in great clarity but what could you do about it so working with the n i j chemical genomics center which is this remarkable facility that has been mentioned already at least once in this hearing it is an effort is being made to identify a small molecule which is sort of a drug that would basically compensate for the genetic problem that's found in individuals with this type of his disease so called c m t one egg and that is an early stage effort but it's a good example of this therapeutics for rare and neglected diseases effort that and i each is putting increasing effort into  rare for companies to generally see this as a good investment for them in terms of developing a therapeutic but with the chemical genomic center working with academic investigators who know a lot about the disease if they can push this forward to the point of identifying a promising compound then you could imagine a company be getting pretty interested in licensing that out and carrying it all the way through to a clinical trial thank you with with the chairs indulgence i'd like to ask you about mental health problems of children adolescents because in my district we have the nathan kline institute and it's an internationally known psychiatric research facility and they believe that the unmet mental health problems of children adolescents is one of the most important continuing problems that haven't yet been adequately addressed and the research funded by and i h establishes that fifty percent of all mental disorders begin the pediatric ages and seventy five percent onset by age twenty four and yet as of two years ago only about eight percent of the budget of the n i m h was dedicated to addressing the needs of children adolescents so so let me ask you this how does research of pediatric adolescent mental health issues fit into the themes of opportunity you've you've identified with in your first year well i agree with you those are critical issues because certainly the onset of a mental health problem in childhood or adolescence means that that individual if not properly diagnosed and treated is going to have a very long course one of the things that i think we're trying to understand is what are the advantages of early  intervention i think there's a lot of need also just to understand in terms of basic neuro science what is going on in the brain in bipolar disease in schizophrenia in major depression in a number of the other conditions that do tend to come on in adolescence the human connectome project is an effort to try to do that in a more says dramatic way putting together genetic variations with what the linkages are in the brain between various regions and how those neurological tracks are different in people who develop one of these diseases but i hear what you're saying about the need to focus particularly on early onset risks and here i think the national institute for child health and human development working with and i m h which has been the main source of mental health research and also with the neurology institute and i n t s is undertaking i think a pretty important effort to try to develop a  better understanding of normal brain development in young people and then also to apply that to trying to see what happens in the course of the development of one of these diseases certainly the national children's study when it gets fully up and going we'll also have a chance to look for very early influences on mental illness in terms of environmental exposures genetics at cetera because we've not had really had a very good database and that study which will study now one hundred thousand individuals from  pre conception until age twenty one should provide some other important clues i mean yes we don't have a chair i have another question could i one more question let me let me ask you about  v and aids research because i think that's really important and fifty six thousand people every year living in the u s become million factored with hiv and in your opening statement you mentioned the many ways it and i h researches contributed to the information that we know and the increased life expectancy for a hiv infected people we had discussed previously an innovative program in the bronx in new york where i'm from can you discuss that program and also of the current state hiv aids research specifically at the community level and i and i each is conducting so the program you're referring to a innovative idea about testing and treating and then linking to i mean explain what this is and why it's seems to be potentially very beneficial you're right fifty six thousand new cases each year and that's been pretty flat for the past few years we are clearly not successful in reducing the new cases of this epidemic even though we've developed effective antiretroviral therapy and individuals who are hiv positive can now expect to live many decades still we should be doing better and terms of ending the epidemic the idea here is that most of the new cases of hiv many of them anyway are actually acquired from individuals who do not know that they're a hiv positive because at the present time many individuals do not get diagnosed until they've already developed some symptoms they're found to have their immune systems already compromised at least by mathematical modeling and by some small studies done in other parts of the world it looks as if you tested every one of us every year to see who has a hiv positive and as soon as somebody turned up positive began treating them then you would both improve their likelihood of a good outcome importantly you would drop their viral load substantially so that their likelihood of passing the virus on would go way down so the idea here is that us sensually reduce the viral load of the whole population by a den five infected individuals as early as time as you could and mathematically it looks as if that get actually end the epidemic without having come up with a vaccine which is obviously been an enormously frustrating circumstance to test that this test and treat link to cure a t l c effort linked to care has been piloted just recently in the bronx and here in washington d c with support from the national institute of allergy and infectious disease so that we are going to be able to see does this work not in a mathematical model but in the real world and we kind of thought washington d c was the real world we thought the bronx was too i do too favorite places thank you very much thank you ma hans i'd like to ask you one question on behalf of chairman waxman who had other obligations he's can he's concerned about the peer review process and also the cure acceleration network as you know the it's reform act of two thousand and six required that all research that the and i can dr supports has to go through a peer review process and you pointed out in your testimony that the process is critical to ensuring that federal dollars are spent on the highest quality research the health care reform legislation that the president recently signed into law established this terrorist acceleration network whose goal is to further research into treatments for certain kinds of conditions specifically the ones we talked about earlier that have few existing therapies legislation established a competitive process for reviewing these can proposals but it didn't specifically that grants go through the m i a peer review process and so it's its chairman waxman's understand that the authors of the provision do not intend for the grant proposals to be subject to the process what is your understanding of how can grant proposals are going to be reviewed by the end i h we depend on peer review for everything we do the way in which we would expect this program to go forward would be to invite  projects that seem right for investments of this sort which is moving them through the so called valley of death where you have a promising compound but you want to get it all the way to a clinical trial those would have to peer reviewed to see where in fact are the most promising projects the most promising opportunities this is high risk potentially high failure science but we had and i are very motivated to push this forward the cure is acceleration that work would provide us with some flexibility that we haven't really had before but it would be done with period you one thing i want to say is that the bill does offer some darpa flexibility to and i saw so called other transaction authorities which we would welcome in that once a project has been peer reviewed and initiated it gives the project manager some flexibility about how to move the project forward and to kill the project if it looks like it's failing and missing milestones so we do embrace that kind of flexibility but i want to d my opening questions with this driving research thing that you're doing two hundred fifty thousand dollars you said you'd come back in the summer mansion on that i would hope you wo because then it's tough for us to defend the good when we have a question of dollars going to question directions that we need more clarification on so thank you ma'am thank you and just to reiterate several of the members expressed to me during the votes that they will submit their questions in writing so if we can get responses to all these questions that would be great we're happy to do that and i want to remind all of the members that they may submit additional questions for the record to be answered by the by the witness the question should be submitted to the clerk within ten days and then the clerk will notify all of the offices of the proceed is yours and i would like to just thank you in particular collins you have a very full plate we're glad you gave us your afternoon it was the wide range of questions that members i think were pressed by the answers and we look forward to working with you and this meeting