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tv   National Institutes of Health 2019 Budget  CSPAN  May 18, 2018 4:19am-5:59am EDT

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>> the directors from the national institutes of health testified on the presidented 2019 budget request. senator roy blunt chairs the senate appropriations ske hearing. chairs the senate subcommittee hearing. >> [laughter] >> the appropriations subcommittee on labor, health and human services, education and related agencies will come together. glad to have our friends from nih here today, dr. collins and the institute directors. the recent support of medical research by our subcommittee and congress is clear. during the three years senator maria cantwell i worked together i workedor murray and
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together, $7 billion above what we were three years ago. we traveled the alzheimer's research amount, started the precision mesu medicine initiative and confident resources to such revolutionary projects as the universal flu like vaccine and a renewed canstment in nih has and provide millions of americans and their families without that they wouldn't otherwise have. ni funded researchh has raised life expectancy, vastly improved the quality of life for all americans, and in addition removal we can see ways to lower health care costs and to create economic growth by supporting the jobs. yjr research
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so far we have been able to maintain an important momentum that everybody on this subcommittee certainly sees at the moment we are in. things like the west africa challenge three years ago with the devastating outbreak of killed moreisease than 11,000 people in africa, became a major public health threat in the united states, and cdc were in important part of responding to that. another outbreak in the
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democratic republic of the congo. i think there was more news on that topic even this morning. we need to be well prepared to continue to enable to do what we need to do to respond to those kinds of challenges. earlier this month the nih launched enrollment in the "all of us" study which will collect health information from one million americans, an idea dr. collins mentioned tw years agoo as -- two years ago as an nih goal. we have the ability to unlock decision medicine for the diseases we suffer from such a. this initiative has the potential to change our health system from one-size-fits-all to understanding more about personalized medicine. we are at the point where we see drug reproducing happening. we are testing current drugs we
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know are safe to see what other uses we might find in those drugs. there is a current clinical trial targeting the most common adult leukemia, with a drug first used to test arthritis more than 25 years ago. that particular work is being done at the university of kansas. senator moran has been a candidate for nih research. we hope to visit the university of kansas medical center to look at the project along with others. the national academy of sciences published a report in february that shows nih funding contributed to everyone of the 2 fdaew drugs approved by the from 2010 to 2016. that is quite a record. thatpreciate the fact that
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work continues. we see increased grants among young researchers. anything you have to say about that would be welcome. senator murray has been a great as we hope to be appropriately involved in oversight responsibility as well. collins, itay: dr. is great to see you and all of your amazing team. thank you for being here today. she is a great credited agency. i wanted to recognize her back there as well. president trump's budget for fiscal year 20 1900 seeks deeps deepar 2019 again seeks cuts to the nih below the 2017
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enacted level. that request is out of step with the sentiments of congress and the country. less than two months ago president trump signed into law a bipartisan bill that increased funding by nih by $3 billion, the second largest increase in its history. those funds will be used to discover cures to alzheimer's, cancer, and other diseases, tackle the opioid addiction crisis, develop a universal flu vaccine, antibiotics, and more. president trump's request would undermine these efforts. by comparison, in just the past three years, congress has provided nih an overall increase of $7 billion, boosting its budget by almost 1/4. after adjusting for inflation, the nih budget still falls short of its peak in 2003 and grains remain highly competitive, especially for our early-stage investigators, but the numbers
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are trending in the right direction. i'm hopeful individual our subcommittee will assuredly begin writing. i know chairman blunt feels the same way. medical research with a goal of achieving breakthroughs that benefit all of us, including those who have been historically underrepresented in clinical research and preserve our nations leadership. we are in a time of event promise and challenge for the research communityl the promis of unprecedented new tools and computational power balanced by the challenge of making the most of the staggering and ever-growing amount of data that nih produces. the brain initiative efforts to cure alzheimer's disease, the cancer moonshot, and all of us initiative to advanced precision medicine pose
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extraordinary challenges how to manage and make sense of it all allow the, insurance community to leverage as much as possible is a needle in atech, while at the short time remaining each patient's data remains secure. i see the potential every time i visit the institute for brain science in seattle, but few organizations have their level of sophistication. two years ago the committee tasked nih for outlining how it would manage and make the most of the data it is producing. nih released that plan earlier this month on schedule, the real work lies ahead. i am concerned there remains no senior member of the leadership responsible for the portfolio. i see nih recently posted an of being for a chief data strategist, and you are actively reaching out to those in the private data sector in hopes of
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attracting the right person to public service. it is essential we find them quickly, bring them on board, and make sure you properly support them to implement your strategic plan. i look forward to hearing nih's claims in this area when we turn to the questions. i'm pleased the administration's fiscal year 2019 budget abandoned its ill-conceived proposal to cut great support funding -- cut grant support funding, but slashes researcher salary by 20%. like the previous proposal, it is blindly destructive and short on details, a gimmick not meant to be taken seriously. i see the budget follows congress's lead to provide funding for opioid addiction and spur the development of nonaddictive treatments. i think i can speak for all of toin saying we expect nih
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make the most of those resources entrusted to address this crisis. we and those we represent our accounting on you on this one. -- are counting on you on this one. it has been troubling questions have been raised about the impartiality of a study to assess the health benefits of alcohol consumption. i know you have been focused on determining the facts ensuring the integrity of nih's research practices, and hopefully he will provide us with an update on that. >> i'm going to local the institute directors here with us today. the director of the national can cer institute is here with us. we are glad you are part of this team. collins, glad to have
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your testimony. >> it is an honor to be at the table. to my right is the director of the national institute on drug abuse. you mentioned our newbie to my right. the director for the center of neurological diseases and stroke. on the far end, someone you know pretty well, at his 439th hearing or something like that, the director of the national institutes of allergy and infectious disease. i want to say to all of you, thank you. you have gone above and beyond in your support of nih research. on the half of all of them, i am immensely grateful for your support and the words you have spoken. it gave me a great confidence we
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are on a good and strong path toward the future. i spent a lot of time with early-stage researchers. wherever i go, i set aside time to hear about their dreams, their ideas, and yes their concerns. i know you too has met with many of them, both in your home districts and in your appreciated visits to nih. as we move forward with implementing the fiscal year 2018 budget and you begin considering the fy 19 request, it is a good time to think about the early-stage researchers to foster the next generation of discovery and help our nation to remain the world leader in biomedical innovation. i believe the answers could be said to lie in certain key areas. i would call them the five keys to success in science today. your first and foremost a stable trajectory of support, followed by a vibrant workforce,
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computational power, new technologies and facilities, and perhaps most important, scientific information. -- scientific inspiration. early-stage researchers are seeing a stable trajectory of support. your work over the last three years is beginning to help us reverse a decline in nih's purchasing power for research carried out throughout the nation. strong public support lies at the heart of nih science. that is vital to our second key of success, a vibrant workforce. success can't lie simply in boosting the number of grants made, it must be in increasing the number of creative minds receiving those grants. a new metric we are using to evaluate success uses the number of printable investigators supported by nih over the past 15 years. that number is once again growing nicely. note especially the surge around
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2016, a surge that reflects when you show nih support and shows that are starting to pay off. the third key to success is computational power. like so much else, biomedical research has been transformed by the recent explosion in computing, and the big data it is generating. for example, the brain initiative has created new imaging tools that are churning out troubles of amazing data. data. troves of amazing there is biomedical research supported by your appropriation. in a major national launch event, all of us begin the full scale of enrolling one million americans, building on their pilot phase that already involved 25,000, aiming to
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determine how differences in lifestyle and biological makeup can influence health. we also must develop new technologies and facilities. quite often it is the technology itself driving the need for equally innovative facilities. take the case of the new cell-based treatment, immunotherapy, gene-based therapy. many involved removing cells from the patient's body, reengineering those cells and returning them to the patient. the challenges many labs -- is many times are not set up for this process, so it is crucial we keep pace. the president's proposal includes a much-needed increase in building facilities to assist. now on to my favorite of these topics, scientific inspiration. i can assure you nih funded
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researchers come to work every day full of innovative ideas and the wherewithal to see those through. i can sen talk about this all day, mindful of the clock, with me share one example. a decade ago nih launched a special project on spinal ascular atrophy, or sma, uniformly fatal inherited disease. you can see it here. in its most severe form, beliefs ba -- form, it leaves babies floppy, unable to breathe. 10 years ago there was no treatment for sma, but researchers discovered dna mutations and caused it. nih supported more research to move promising leads into therapeutic development. one of the most exciting comes from jerry mandel's team in columbus, ohio, which recently tested gene therapy in 15
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infants with severe sma. they infused the viral vector designed to deliver the normal gene to the spinal cord, and held their breath. nths,the next few mo something dramatic, almost ridiculous happened. little mateo, you see in this video -- 100% of the infants who got the highest dose of the gene therapy were alive at nine months. all could talk and eat on their own. mateo, like some here standing on their tiptoes, were able to walk. this was a standing -- outstanding. as a direct result of an nih inspired effort, we are seeing the emergence of life-saving gene therapy for sma. in closing, i am proud to lead nih with such strong
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congressional support. the resources you have entrusted to us will be used to bring hope to untold numbers of patients and their families. in that spirit, i would like to finish with a quote from the poet peter levi. life in every sphere of is a privilege that attaches to action. no action, no hope." to apply theope is best investigators to the best science to the best for those whose hopes can be realized. .> thank you there will still be time for a second round. if we are still having the hearing at 11:15, i think it is one vote, so we will work around that and continue the hearing. one of the things we put in the
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appropriations bill this year was an effort that frankly we were asked to put in where there would be partnership money from pharma into opioid research. the idea being like other partnerships between nih and other biopharmaceutical companies, we could deal with this quicker. you decided, and frankly without consultation of the committee, not to do that. it may very well relate to the alcohol study senator murray brought up. i would like you to respond to both of those topics, starting with why you decided not to do what you have asked us to put in the budget to allow you to do. >> i would be glad to explain that. this is a topic many have been wrestling with a bit, as we have. we have been in deep conversations with industry
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partners about ways in which we might develop a partnership to come up with better ways to treat addiction, overdose, and treatments for chronic pain. working with companies over the course of many months, we identified a number of opportunities which we could you effectively in a private-public partnership in which neither sector could do alone. that includes sharing data, sharing assets, sharing compounds that could be abandoned and could prove to be valuable. the good news is that partnership is very much alive and well in fact be going forward. we are close to having the full plan laid out, and i would expect in a few weeks to say about how we would conduct this. the controversy, senator, was whether in fact given the circumstances over the opioid crisis, and there are lawsuits
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filed against no less than five of these companies claiming they may have played some role in the opioid crisis in the first place by marketing such drugs as oxycontin, whether it is a good idea or potentially carries a reputational risk for nih to receive funds from the company. i convened an expert group of advisors who has lots of experience in both sectors, and who were finely tuned to the questions of reputational risk and ethics. to my surprise, they made a strong recommendation we go forward with the partnership, but not have actual cash contributions from the companies involved. their concern was that would create at least the impression, if not the reality, that the projects going forward would be a conflict of interest, driven by something other than the best needs of the public. i had to accept that strong recommendation coming from those
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groups. the foundation for nih, our partner, convened their board and came up with the same recommendation. with apologies for not having consulted as i should have with members of this subcommittee, we felt we had to make the decision that we did. the good news is the partnership will go forward as planned, but we are not asking the companies to contribute money, but expertise data and assess with our clinical trials network. yes, the alcohol issue was also in this particular discussion, because we're in the midst of another place i wish we are not, where there are deep concerns about whether a study that got launched which aims to look at whether there might be medical benefit of modest doses of alcohol in humans. this particular study was set up where the funding is largely
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coming from the beverage industry. there is evidence nih employees recruited funding in ways that violated our policies. we are investigating that through a working group i have convened. there are sufficient concerns about the study i would like to tell you that one week ago. we decided to suspend enrollment in this study of the moderate effects of alcohol. while they make a decision over the fact whether the studies still worth pursuing. all of those are complicated issues and have caused considerable pain and stress among the people involved. for nih, our reputation is so critical. if that could be called into question, i felt we had to look at that very seriously and come up with another strategy. >> i can probably ask another question quickly and get beyond my five minutes, but i will not
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do that. if you want to stay for second questions, we can certainly do that. senator murray. >> i want to go back to the big data. one of the biggest challenges facing science is to effectively use this data research is producing. without the tools to efficiently manage and manipulate data, the value of it is significantly reduced. likewise we don't want scientists spending time re-creating data that exists from other research products. the committee directed nih in the 2017 to develop a plan to address these issues. lan, can you have the you tell us the timeline for implementation? >> the timeline is ambitious. deliberations with many experts within nih an respondingd two comments from
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over 800 organizations. need to dot what we in the area of infrastructure, in the data ecosystem, in software and tools. we realize our bench is not as deep as it should realized our bench is not as deep as it should be and also sustainability. do we put something together today and into tomorrow. ll of this is laid out in a broad and bold way. the way in which we're going to implement this certainly of things thater are already under way because we've been working on this issue working 2 when the group of my advisory committee made the first set of recommendations. some of our largest data sets into the cloud in a fashion that protects the privacy of the participants but makes that data accessible to researchers all
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big step hat's a forward and most of our big data sets will need to be handled way going forward. as you mentioned in your opening comments we're actively a chief data strategist, someone we ideally hope to recruit from industry silicon valley with deep experience in how to handle ig data sets, machine learning and after the official intelligence. >> are there barriers, cases researchers are facing challenges sharing their data, daviesing their data until they publish their findings, things like that? >> we're certainly attuned to the risks of people hoarding data. i'm happy to say the 21st daviesing their data until bil authority about requiring data from ourd data release grand -- which i used to have to do some con jolg but now i have to have more clout and that's helped in us that regard. i think the general ethic of the has shiftedommunity much more in the direction of it is your responsibility to make your data available as soon as
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might be able to use it. lot.e encouraged that a >> good. doctor, given the scale of investments that we're baking in disease, the data we're generating may be used to scientificsome major breakthroughs, has nia looked at xisting data sharing policies to determine how they are working? do you know what percentage of fulfilling ents are obligations aring on this project? >> i can echo the same. we've had a history in the alzheimer's researchers ommunity of a strong willingness to share and that's increased over past years. when k we're at a stage we're dealing in part with technical feasibilities of cross, ata interpretable, shareable and compatible and are working very collaboration across federal agencies and with
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outside groups to make that effective.e we have the culture as well as ie arm to enforce sharing and think we're on an excellent sharing trajectory in that regard. >> doctor, one more question on the moderate drinking study. huge enterprise and if this was happening in one it could be happening in others. are you doing anything to make compromised n't studies elsewhere in nih? >> i'm very concerned that this of a larger tip iceberg and the reason i put together this distinguished group of experts. closely to see if there are other examples of this very because that would be much against the principles that we stand for which is separation from outside rces with decisions about science. and also, of course, our peer process ought to be absolutely above any reproach as conflicts. i would be glad to report back to you after we've done a bit
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more digging. of my roles as the director. when we find something that's don't just assume it's a little thing that you can put a band-aid on. it's not reflective of larger issues and we aim to fix those, too. >> thank you. senator alexander? >> thank you, mr. chairman. chairman me say to blount and senator mur ri, senator durbin, all of the xhebs committee, how much i appreciate their leadership and funding for the national institutes of health. second, it's good to see a piece of legislation that had such bipartisan support. senator mcconnell said it was the most important law of 2016. part of that is because of the talent of the team we see before gotlieffthers, like dr. who know what they are doing and
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are taking advantage of the new authority. third, we've developed quite a onsensus on science and research, as sandra blount said, biomedical e in innovation. i would add to that, the subcommittee which senator einstein and i chair, we increased funding for the physical -- for the office of 16%.nces that was the third straight year of record funding for the office of science that supports the national laboratories so when you get three straight years for national institutes of health, three straight years of record funding for the office of that, in d you had to the obama administration and in he first two years of this administration, we've funded uper computing, at impressive levels to keep us first in the world in that, the only people not on-board with the president's america first comments are the office of management and budget. so i'm going to try to talk to at the ident and others white house and say why don't you include this in your america
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first agenda. why not buy medical innovation. why not national laboratories. something er compute we need to lead the world in that congress wants to do that both republicans and democrats. to get the omb on board. let me use the rest of my time you about one area. nonaddictive pain strategies. whoave 25 million americans hurt badly, chronic pain. a hundred million who hurt some. nonaddictive treatment for opioid abuse. for opioidsreatment is more opioids. abuse dicated assisted and sometimes they say you don't ever get off opioids. so my question is, in the 2:30, progress are you making? senator murray and i have included the transactional to ority that you asked for give you even more flexibility, what progress are you making on pain, new pain strategies, and why are 95% of the treatments
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opioids more opioids? why don't we have more vivitrol or some other treatment that helps away from opioids? >> it's unfortunate to have at the table two experts who can of that th parts question. maybe we'll go first to dr. talk about the treatments for addiction, and then i'll ask dr. koroshetz to to nonaddictive pain.ments for >> this is one of our priorities. how do we develop alternative edications for opioid addiction? we currently only have three and are what we call opioid additives. are i right, that two about 95% of the treatment, right? >> that's correct. those are the numbers. problem is not all f the patients respond to the
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vivivitrol treatment. expand the to alternative medications that we can give to patients and thating also on strategies are not involved in the opioid system. o that we can help those individuals be able to recover and be at one point able to stay opioid medications. you're absolutely right. one thing that could really help n the long term to stem the opioid problem is developing nonaddictive pain medicines to opioids in the prescription box for patients, and -- we funded $500 million in esearch, $500 million proposed in the budget for this coming year. >> that's right. we have working with our plan to partners a really accelerate the development of new medications of the basic science point view we have a number of different targets that look very
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to develop medicines that are not interacting with the opioid pathway. testing s in current now is what's called anti-nerve therapy, which came from really very important asic science years ago on the intersection of that growth factor and the pain system, and now have developed antagonists to that which look promising in early results. example but we think there are many more. >> thank you, mr. chairman. reid?nator >> thank you very many, mr. chairman. i particularly want to thank dr. sharpless for giving me an opportunity to look at the program and ology meeting all their tremendous women doctors and ph.d.s who that effort. thank you. of another port tremendous woman, senator we were able to pass the we were able to pass the
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starr act and can you give us an idea of how you will use this? it will pass the house very soon and become law. >> sure. i can directly comment on legislation. first-off, thank you for the visit. a lot to the pediatric doctors. wonderful s really and thank you for your support of senator capito's support cancer research. the starr act, the intent of it, it's very laudable to address issues in pediatric oncology and hildhood cancers that are important. one issue is survivorship the good news about pediatric we're curing more and more kids but the two problems s, we're not curing everyone, and a lot of the kids we cure are left with lifelong toxicities. results from therapy can be quite debilitating, translating disfiguring ity, surgery, cognitive dysfunction decades.s for so survivorship has become an
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issue. pafl a million pediatric cancer survivors. we can do is an important area in pediatric oncology research. aggregateds issue of data in pediatric cancer. areof the shortages of that samples. biospecimens that can be cataloged analyzed, and presented to the research community in a format that's research. usable for those two addressing survivor biospecimens are really we rtant and something that are behind. >> you have recently announced trans-nih on of a pediatric consortium. can you give us an idea of what to accomplish? >> i'll be very happy to. we have the national institute development, which sounds like the place where pediatric research gets done but about 18% of only pediatric research across nih.
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most of the institutes, you've heard a great example from the cancer institute, are also pediatric ested in research. i've asked the new director to group at a ansnih high level from all the institutes that are players, which is essentially all of we could ee whether come up with a more coordinated trategic plan for defining where are the greatest priorities in pediatric research and how can we work together hether it's cancer or whether it's autism, whether it's birth defects. whether it's development, it's behavioral issues. i believe with her leadership, nd she's a very strong leader that we have the chance to put forward a plan for pediatric esearch that will be quite exciting. that's just gotten going, and i would be glad to report back to in the coming months about how this is leading us in new directions. remiss if i did ot applaud the fogert center, amed after my beloved
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predecessor, john fogert. 50th t celebrated the anniversary and it seems particularly important today as emerging.t eboli can you just comment upon the 150 years for the fogerty said and tell peter i hello. >> appreciate you very much being there at that 50th nniversary celebration and speaking to the group. one of the things that fogerty has done even though it's one of our institutes, is the training program for our program. story k -- to tell you a about how those fogertiy fellows ave played a significant role zeka.boli and
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>> it's been very important and of what we do at nih, articularly in the area of infectious diseases. ou recall that when we had the eboli outbreak, that there were you would , as expect, traveled, not knowing to places nfected like maui and nigeria. in e cases did not result outbreaks in those countries and care ople who were taking of those individuals, were individuals, almost all of whom were trained as fogerty fellows, we had a preexisting network had been there who trained according to the culture of the nih in science and public health. so that was just the dramatic public health example of how the fogerty center was a true partner, not only fogerty on our fogerty in lagos.
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striking.te >> with a speech like that, you ould be elected to congress from the second district. >> ms. capito. see ank you, it's great to you again. my first question is on opioids we've t you're doing and talked numerous times about this. obviously, i live in an area affected here. two questions. ou mentioned and you said that we had put a $500 million investment into this but you in your response to questions that you have private partners. ould you elaborate on how the private partnership works and what kind of commitment dollar-wise that private is committing to add to the $500 million that we're doing in the research? happy to. be we have worked intensively since our wonderful decision to make $500 million available in fy-18 nd to have that in the base so it will be there in 2019 and beyond. working with all the institutes
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put forward an opportunity for new and bold ideas to come forward, and we're now, i think, of having a remarkable portfolio launch.'re ready to you also graciously gave us that first year of support as we could, in s so fact, carry over some of the fy-18 funds into 2019 since it's late enough in the year that starting brand-new things is a little challenging but we've how we're going to spend a significant fraction of away.$500 right it does include a wide variety of applications including some of the things that have already by dr. volkow and dr. koroshetz but also such hings as what to do about the neonatal abstinence syndrome which is of such deep donner all us. how best to manage what happens to those babies and what's their long term future? public-private partnership is actually a modest part of this broad portfolio but an important one. again, as we talked about earlier, the decision was made
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strong ased on recommendations that that should involve assets that are credited by industry that have value, data, compounds, scientific expertise but not a cash contribution. it is a little challenging right attach a dollar value to what their in kind contributions are going to add up to but it substantial. so putting that altogether, we think we're in a good place to public-private partnership and the rest of this broad portfolio together quite rapidly. >> good. you. dr. volkow, on the neonatal syndrome we have obviously, we're the state most highly affected by that as well, an area of deep concern to everybody. you see a lot, we've got a more kids in foster care. a lot of these babies probably will be there, repeat mothers. repeat exposure, i mean, they have numerous exposure to numerous drugs, not particularly one. i believe you visited our place own in huntington to see what
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kind of care they are getting. the research that you're doing are he act now trials, those being conducted across the nation or how do you find the place to conduct those kinds -- and where are you doing that? >> well, the acts is one of the rograms that will be funded through the new money that's come for the opioid crisis and us to actually maximize products that will enable us, for example, to etermine what are the optimal interventions for the best outcomes on neonates. includes products that may not require the administration of medications to neonates with abstinence. we have all the research that's also ongoing. week in west virginia last and in huntington and i was horrified to hear that one out had opioids on them. and one of the points that is we need to is that address the needs of the neonate but also increase the needs of
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the baby is ce born. outcome from that is addressed so we're working with research of new medications and nonmedication interventions to improve the outcomes of those neonates, and the other aspect is we're very interested in understanding how the brain of going to have is been influenced by getting opioids as well as other drugs during fetal development when the brain is very vulnerable. mean, i'm anxious to hear how we progress with that. one of the point in -- the points in asking my question, there is a wealth of data all around the country with people who are hands on in these cenarios dealing with this right now that i think can feed not only good information but can help you conduct trials on local level. it sounds like that's what you're already moving forward on. pleased to be on the starr act, the childhood cancer
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ct and i'm glad to hear about the survivorship. that's something of great importance to me. a lot of this i'm very collins d in as dr. knows but i want to go over to end of s, on the other the spectrum, alzheimer's is omething i've personally been touched with. one of the things i have heard is that it's hard to recruit trials for clinical alzheimer's. is that the case? what are you finding? start by the time you exhibiting the symptoms, you're already in it. -- how are you developing all of those trials with different --? >> it's an excellent question a challenge to us. for multiple reasons. you've talked about, for individuals who are already affected with symptoms, we carry extremely important but there is also a sense in order to be most effective in the appearance of symptoms that we need to intervene earlier before done.rable damage is we need to recruit people who are not coming through a normal
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rocess where they see a clinician who suggests they participate in a clinical trial. these are people at high-risk show no symptoms so we need to develop strategies to screen to e at high-risk and then find people who are committed, dedicated, to participate in make a to see if we can difference with earlier intervention. all of us present a unique opportunity. a million people have signed on in participating research, we of screen those by a variety metrics, may be at high-risk for developing symptoms years or ecades later and take an opportunity to intervene with those now so we're in the midst nationwide program which will be announced this summer to ook at multiple modalities for recruiting patients into studies. >> good. thank you, we want to support that. thank you. shaheen?r >> thank you, mr. chairman and senator m you, murray, you, dr. collins, everyone, thank you so much for
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the work you're doing. give hope to so many people across this country. for coming to new hampshire. i especially appreciate the work that's being done to address the opioid epidemic like my colleagues on this committee. hard hit in new hampshire as you know from your visit to catholic medical center other places in the state. in your erested discussion about new drugs that are in development. at what the king potential is, are you separating out fentanyl as a particular that has very deadly properties, and therefore, of ires a different kind response, or are you lumping everything else? >> you can't lump it in with everything else because what we're hearing from the field and that's being reported is that when people overdose with
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doses we use of narcane to reverse the overdose. of risk is much higher so as now we, for example, we have not of how do you treat someone that's addicted to fint tinel. would like hing we to implement as soon as we get the approvals. lso, with respect to medications we're actually unding researchers, who we're partnering with, to develop a narcane antagonist for ut it's not sufficient for fentanyl. so we're funding all-star team that may not be -- people are oversewing ith multiple medications, lcohol, fentanyl, so this is
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not sufficient. >> and we see so many people who ave co-occurring mental health issues along with their substance abuse disorder. substances are using to address their mental health doing any are you research that looks at the two give ms together that may promise, as we think about what the future holds? cannot not do that because it's much more frequent than the isolation. if you don't address, for example, the depression on someone that's addicted to pioids you're not going to succeed. also, very important component, which oneso not know are intentional. therefore, suicide. reversing someone on overdose who has suicidal to vior, you aren't going succeed. prioritizing. mental addiction and illness and
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with pain. challenging than when they are in isolation. add anything t to to that, doctor? > i think dr. volkow very accurately characterizes the situation. i think one of the things that in e most concerned about terms of this crisis, if i can show you this graph, is the way epidemic, in terms of the overdose deaths has prescription eing opioids to now fentanyl, just going straight off the chart as the heroin supply and as more individuals who have cannot nto addiction find sufficient access to prescriptions, then they shift over. we're seeing in new hampshire where we have the overdose deaths fentanyl. >> doctor, senator blount mentioned the eboal epidemic and reports this morning the first case had been found in city in area, a large
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the democratic republic of congo challengesrc's other how worried are we that that epidemic is going to get out of control again? given our prior experience we're on high alert. there are factors mitigating ens the same situation as we saw in west africa, but there are also factors that actually might favor that. mentioned, the first cases that were reported in the ery early may, the first week in may, were in a place remote area aro, a called lake tumba. the bad news is that it's very difficult to get help in. news is, it's very difficult to get anybody out, but what you heard this morning report is that there is cairo le different zones, is one zone, another is an area that has a city of 1.1 million and even though there is only one case there, there is a
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cases, even 44 though only two have been confirmed, there are 20 that are that are nd 20 suspicious. so there are probably many more cases. we're doing now is shipping helping, he who, obviously, the kinds of things that were the fruits of the work did with the support of this committee and others, to countermeasures. i'll very briefly give you an example. you probably heard in the media who has authorized the shipping of the vaccine that had right st phase one trial at the clinical center at the nih and then we did it in africa that was the one that did the ring vaccination. we also have zip map which has triple combination of anybody's that we published in the "new england journal of from the intramural program at nih that went over there in liberia. and wealso being shipped have some experimental drugs e drugs, one we're partnering with
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and some mono -- antibodies that have been developed. so in direct answer to your we're on high alert, we're always concerned with ebla, but we have a number of to block that. our hopes and expectations are always cautious but the hopes are that we'll not have the kind outbreak that we saw in west africa. >> thank you. chairman., mr. >> senator durbin. thanks, mr. chairman. in a say at the outset, world of frustration and partisanship what's happening in room this morning is a welcomed exception. you will find more positive more achievement, i hope, and more bipartisanship other room on any capitol hill, and i want to alute the chairman of the subcommittee, senator blount, inking member senator murray, told her i was going to praise her before she left and
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and inly senator alexander his work and senator shaheen. this is a great assignment because with the help of the this table, we actually feel like we're taking steps forward. for his r blount leadership on this, we've established a standard, i think. one that we can live y, of sustainable, reliable increases in medical research funding, in the united states of america. several ns told me years ago, that's what we need, and we're doing our best to meet that need. recentlye areas that i learned a little bit about was in foreign affairs magazine of and, i happen to be eading it, and this liberal arts lawyer came to try to understand something called chris- crisp per, which is included in your publication
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technologies.g family from illinois came to see the mother, and he wife, has myo-tonic dystrophy. it's a genetic disease which she unwittingly passed on to her children, son and daughter, whose circumstances are even than her of course, their future is unknown. i mentioned crisper to them and they lit up. the one area is where we feel like there is a chance. please, dr. collins or one of give us a gues, moment about crisper and what we're doing? i'm happy to because i agree this is one of the most exciting things that's happened in a long of providing tools both for basic science and for therapeutic applications. hopes up entirely new vistas, particularly for individuals with genetic disorder where we misspelling in
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the dna. provides an opportunity to go in there and fix that in a fashion.ise it's also a basic story, this particular it is a very elegant system where bacteria have an enzyme that recognizes a foreign dna sequence and fixes it. jennifer doudner and her herseagues, and a few ot arguing about who gets credit, found a way to take that bacteria system and make it work and all kinds of cells. it can find in a 3 billion letter instruction book the one that you want to alter and zero in on and make a cut or substitution. in basic science, this is fantastic. every lab doing biology is using
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it, but therapeutically, the promise has us excited. may be the disease first success. the problem is in the bone marrow stem cells. you can take them out or utilize this enzyme system. that should be not just a treatment but a cure. i think that will happen in the next five years. you, but to interrupt i hope that the subcommittee can zero in on the technology that is happening there. i wanted to mention one other thing that is important to most of us. 27% are using e-cigarettes and developing.
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-- and vaping. it used to be 28% using tobacco and e-cigarettes. the people peddling these products are putting them in candy flavors. the brand is lung candy and the flavoring is cake batter. a long listing of flavors for children to see opportunities for vaping. can i get a comment from any of you about what you consider to be the perils or the danger of this type of addiction? 50% of teenagers say that flavors,starting with 20% are starting to vape only with nicotine and are becoming addicted.
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it has been shown if you start to vape, you're more likely to go to nicotine vapor in, and -- vaping and then to go into tobacco. the concern is that we will be losing a lot of the advances that we did. nicotine acts as a primer so anything you take on nicotine becomes more enforcing. if you get exposed to drugs you are more likely to become addicted. these teenagers will become cigarette smokers. this, your brain is primed to the addictiveness of other subjects. >> dr. pyeongchang, -- dr. fauci, i was at this university
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during the zika crisis where they felt they were coming to a conclusion. availableve something next time? >> we have progressed in our vaccine trial to deploying a meaning trial, relatively advanced, not only asking is it safe but does it induce the type of response that you would predict? i am pleased to support the today we have ongoing a phase 2b puertoin mexico, in rico, in texas, and in florida.
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betweenheduled to have 24000 and 25,000 people in the and 5000 people in the study. i am confident we will have it fully accrued by 2018. if there is an outbreak we can ife an efficacy signal and it is not we are working closely with the fda if we can get enough data to show that it does of -- does induce the type response that you want and a bridge that to the animal data that is very convincing. we are working with the fda to determine if we can get accelerated approval. you can never anticipate their decision but we are being cooperative. next is the flu vaccine. we appreciate the committee for supporting the addition of $40 million for the development of a
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universal influenza the next scene. -- vaccine. we had a meeting in rockville where we -- worked with experts from all over the world on influenza. we developed a strategic plan which we published in the journal of infectious diseases this year. we are implementing that plan from a basic science standpoint and with candidates and various stages of development. the preclinical and animal study or phase one and two. you may have read just a couple sponsored phase two trial. ago, oned a few months of the trials from the vaccine research center. things are on track. it is not going to be an overnight thing but we are well
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on the road. assuminggot there, i'm every year we would not have to try to calculate what that mores flu was like and often than not be slightly off target. >> the universal flu vaccine would be one that would cover all versions of seasonal and any potential pandemic. the road we are taking is to go step-by-step. i refer to it as universal flu vaccine 1.0. we won't have to worry about neh 3. neh2 we have to change a little from season to season. the first version will cover all of the h3 and h2. the the next version will cover h1n1.
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there are two true versions of the virus that have multiple viruses in each group. two versions of the virus that have multiple viruses in each group. you and i and most likely our children will be able to get a vaccine early on in life and every 10 years or so and not have to worry each year guessing about the next iteration. it is the guest that is the problem. sometimes you don't get it right and even when you do it changes enough that it is the vaccine -- that it eveades the next scene. >> i may have unlimited time here and i may exceed that. in 1971 there were 3 million cancer survivors. it is an incredibly exciting
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time. the brainapy, initiative that i assume will have a cancer-looking element, crisper. can you take a couple minutes and talk about your vision of what you hope and believe can happen over the next handful of years, based on what we see already? >> i think you are right about that. i recently had a colleague email me. there are all of these exciting things going on. -- it isthe release like christmas in our field where all of the new advances come out. i think there are a lot of opportunities.
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each one of them needs specific treatment. is a lotnews is there of excitement going on and a lot of new technologies and new approaches. the bad news about it is that problem is somewhat different from what we imagine. we used to think of cancer as one disease. the things that i thought nci should focus on is training the right workforce. we need cancer biologists who understand basic immunology. we're aggregating data at a furious pace. we need to recommit to basic science. we have to make progress against all cancer.
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that requires a basic biological understanding. we need to fix the problem of clinical trials. the therapy based on genetic drivers, the personal element that makes cancer treatment relevant. we need to get serious in cancer about organizing data so we have sets- all of the data available to the research community so we can understand what beauticians caused which patients to respond to this drug and to get a handle on these rare cancers. fda not long ago
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and they put a cancer team together. the thought of that discussion came up in the foreseeable future. would maybe be something that each individual prescribed for them that amps up whatever their unique fighting capacity needs to be. that is one look. sure how the crisper effort works, but it is an incredibly exciting time. even with immunology, five years ago an observation made in passing would not have been made at all. i'm sure you will continue to work on why this doesn't work on some cancers and what you need
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to find. >> we talked earlier about partnerships and i want to point to this as a successful public and private partnership. 12 biotechnology pharmaceutical companies have partnered with nih to ask that question. what are the biomarkers that tell you that immunotherapy will work or not. , this is thead little girl who failed to respond to the traditional wastment for leukemia and very much improved by t cell therapy and was not just a success but was treated. she is five years out now. it doesn't work for solid tumors, but why does it work for and bringbreast,
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cancers? we need to figure that out. -- and brain cancers? out.ed to figure that pharma is contributing expertise and resources and cash and it is exciting to see this taking shape. >> each of these therapies requires a detailed conversation. it is leaps and bounds. every year we are seeing some new area that gives a whole new class of therapies. glioma, glioblastoma, it is still a real problem. at nci we have to not only focus on the cancer, but even more so
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beene ones that have recalcitrant. greatest the areas of spending, the quickest growing spending of federal health dollars would be alzheimer's and dementia. i thought you both would talk about what we are finding there. we made big investments. we would probably eliminate a lot of things that we know don't work. how are we dealing on the other end of that? done we will go to senator smith when she settles in. >> first of all i would like to thank the committee and congress for the resources as it relates to alzheimer's and dementia. it had an enormous impact.
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152.15, we funded two years later that increased to 442. importantly, this is reflected with confidence, excitement that we heard about from the -- across the research community. we are seeing a renewed energy on the part of early-stage investigators. category of new investors who have not received any major support previously. had any support for alzheimer's-related dimensions. not just more dollars and opportunities but the energy that comes with new people willing to commit their careers and bring new discipline into the field. expansion,cluded the over 140 and equally important using new techniques of looking
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at gene expression patterns and an amazingly effective consortium and partnership around alzheimer's disease to bring public and private science, critical expertise and financial support together to find new discoveries that have targetsed a wall of that might lead to an entirely new approaches. it is an incredible excitement. the numbers are only meant to illustrate the intensity. sustained funding has convinced investigators and the pace has increased exponentially. think people are feeling more and more positive about a
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blood test that would begin to early identify what was happening with anna lloyds in the brain. thatully we can look at sometime. >> thank you for the wisdom of the congress for including what we call the alzheimer's-related dementia and with this major effort to decrease the public health problem of dementia in our country. most patients who have mentioned are diagnosed with alzheimer's disease. more often than not they will find multiple different things going on. the two areas that are most common are the inclusion of the louis body disease. signature ofe the parkinson's disease which is cynically and aggregates -- s
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ynuclean aggregates. this is a focus we have been able to pursue more aggressively. the other area which is more common is the combination of vascular disease and people diagnosed with vascular disease. we have been decreasing the risk of stroke. it is incredibly common in people who have dementia are diagnosed with alzheimer's. with the hope that the things we already know about may prevent people from going on because we
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can block this contribution of the vascular component to dementia. >> thank you chairman blunt and all of you to testifying today. i have been involved with the cancer society for many years so i have seen how new treatments and new therapies have transformed the prognosis for many different types of cancers. role thatul for the nih has played in so many of those discoveries. would you are all doing is so important and i have seen it changing lives and i'm grateful for that. despite these recent innovations, my state in mississippi continues to have one of the highest cancer mortality rates. for many mississippians receiving the cutting-edge
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treatment would mean traveling hundreds of miles from home to a cancer center in another state which is not possible for so many patients. cancerthe national institute working to make sure are ableents like mine to participate in clinical trials and benefit from the newest discoveries? >> senator, thank you for the question. in some ways it is great news that we are making all of this progress to have these new therapies and new approaches, but if we cannot disseminate them into the broader community, then are we helping to the extent we could? the national cancer institute is very concerned about how we assure that the benefits translate beyond these cancer centers. they are a great program but there are only 70 and their
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range is limited. there are 900 sites across the country and we reach virtually the entire country. areas that also have have patients that are more likely to be rural patients and more likely to be underserved minorities. it is a great program. we have just decided to expand its scope. note thao important to t we can do clinical trials. this match trial had 6000 patients at 1100 sites. in cancer need
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research because 5% of oncology patients go on clinical trials. it is one way that we are working to address this issue. i am proud that the university of mississippi medical center and the jackson hinds, rancid medical center were chosen as sites for the research program in mississippi. this would help doctors identify treatments and personalized to the patient's lifestyle individual environments and genetic biology. was one of only six committee health centers chosen to help pilot the project. the nih rolls this program out nationwide, how have you incorporated lessons learned through the pilot that jackson
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hinds participated in this? >> we did spend the last year up a pilot phase, ramping all of this recognizing that this is one of the most ambitious objects -- projects that nih has dealt with. attention paid to the participants and their security and privacy. we made a commitment that this would be a project that was truly national and would include people who were not invited to take part. community health centers have oftentimes not been so engaged. jackson hinds was a particularly powerful place to be one of the ones to try. i think we would be very encouraged. we and rolled over 26,000 people. that was just the pilot on the way to our million. we were gratified to hear a lot of interest on the part of
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the medicalame for care getting engaged for this kind of research. results it has been a type of outreach that we have not necessarily tried at this scale before. thef may 6, we have done full launch and anybody at the united states can join up. >> will we certainly appreciate it. >> thank you mr. chairman. i did notk because want to go home and tell my family that i have this whole panel from nih in front of me and i did not ask about the prospects of research into a qr for type 1 diabetes. i have a canned -- granddaughter ao has type one and a cochair
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committee with senator collins and i know how devastating the potential is in this country if we don't figure out a qr -- a cure. of the most expensive chronic diseases that we have. can you give me an update about where we are with research? >> senator i would be happy to and i appreciate your leadership. this is one of those very exciting areas where things are moving very quickly. much of the excitement surrounds the potential of developing an artificial pancreas that would take care of the production of insulin. approvingeen the fda the first example of an artificial pancreas in a closed loop where there is a sensor and it administers insulin accordingly.
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this is the beginning of what i think will be a remarkable series of advances. the stem cell area will be an example as well. people have figured out how you can take a skin cell, convince it to be -- stim cell, give -- convince it to be pluripo tent, and convince it to take a pathway down. that is your own cell. creating a pancreas doesn't have silicon that has your own cells that have been reprogrammed. on top of that, in terms of prevention, particularly type 2 diabetes, we know it is tightly connected to obesity and insulin thestance, trying at efforts to reduce weight and increase exercise is looking promising over the long term to
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keep people from tipping over into diabetes. there is a lot of information on gestational diabetes and what we can learn when this happens during pregnancy. it clearly indicates a risk. it is a very exciting time. reassure you that nih is deeply in the middle of all of this. heitkamp: -- sen. h yde-smith: i appreciate all of that. and we need to do more for prevention with type two and where it makes a difference. i think we are not providing the resources that we need. in the long term it will cost more money. one of the things i have heard from the diabetes community is their frustration with how long it has taken the fda to approve
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an artificial pancreas and further developments on that. we are seeing people in the community doing what they call bootleg. they are basically making their own artificial and chris. feel like that provides more relief than they are getting currently given the system we have and the dependence on insulin. can you speak to that? is that something you have heard about? >> there are reasons to be careful about putting in place and artificial pancreas system without being absolutely confident it has the appropriate safety mechanisms, the guys we all know an overdose of insulin can put you into a coma and ultimately to death -- ultimately lead to death. fda has tod why the
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be rigorous in terms of evaluation. i do think that in scott, you have an fda commissioner that is dedicated to moving things along. we were very closely with fda under his leadership. certainly, some of the things we can do on providing that the data, is a partnership we are pushing pretty hard. >> thank you all very much for the great work you are doing. doctor, do you have anybody looking at the socialization challenges of the constant exposure to social media or the screens as a -- as something the onact that that may have adolescence and others. >> the pros and cons have yet to
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established. i think it would probably be mental health which is looking at the effects on adolescence. can look at other exposures on the development of almost half she is finished. >> i had a 50-50 shot and i think i missed it. a very socialis behavior. one of the things we have tried to understand is how the dramatic changes have happened in the way that teenagers are interacting with one another. maybe implanting their behavior most importantly -- behavior. most important way, we look
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adolescence to maximize the ability to respond to your environment. we are interested to know that -- if you, do you get influenced by that, how does that affect your brain. we are looking at 10,000 got on thend have scheduled to finish in september. a next 10 years, we want to characterize them socially and measure how exposure to social media ultimately influences the development of the brain. this is probably the most notable study that we have, because it will allow us to the dramatic change in how people are interacting with one another.
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we have also researched understanding house of a media is used to change the perspective of teenagers to getting exposed to drugs. the likelihood they will take a drug when they see it in social media. the electronic cigarettes, to what extent are they being frequently mentioned in social media, and how is that influencing a teenager on using them. worlds a completely new for all of us and we don't actually, as of now, has an understanding -- have an understanding of the human brain and behavior. >> thank you. >> senator moran? sen. moran: thank you, chairman. i'm not sure what has
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been asked and entered but i want to pursue a couple of concepts developing in regards to alzheimer's. we have talked about detection and identifying biomarkers for someone at risk or has alzheimer's before they show symptoms. the national institute on aging just coordinated to propose a biological construct which looks at the use of measurable changes in the brain to better understand the earliest biologic signs.ns -- biological how will this research -- how will this drive research forward? does it give a new understanding of the disease? >> the importance to identify the changes of alzheimer disease and related dementia early, as you emphasize this critical. we think the best opportunity to intervene at an early point, and be able to track successful failure of interventions by
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measuring biomarkers is going to be absolutely critical. the definition you have referred to is the publication which suggested that for the pathological definition of alzheimer's disease, a son regeneration, there is a set of biomarkers that is standardized -. the rigorously harmonize results of multiple efforts. two more quickly understand which biomarkers are best addicted of the disease and important predictive of the disease and contraction or not. referred to, the biomarkers most steadily used his imaging, which works really well and is in cumbersome and expensive. or spinal fluid analysis, which works but the goal of many now is to establish a fluid biomarker that can take advantage of blood and a serum,
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and identify a single my google -- a single molecule or a pegida molecules to screen more people than we currently can't through longitudinal studies and interventions. sen. moran: let me address this to dr. collins. with the leadership of sen. blunt: that we have had success in large part, i would say because you all have made a case for additional resources within each of your institutes. in which the public good is demonstrated and the advancements being made are sufficient for american people to feel comfortable that progress has been happening and dilip -- the cusp of of developments that make a difference in the lives. in the last year since you were here, dr. collins, in this setting, what can i tell my constituents that has transpired to reassure them that their tax dollars are being widely spent
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-- wisely spent? i had down the track in a town hall meeting explaining there are things that we should spend no money on, things we should reduce spending on, and things we should spend money -- more money on. that sentence,ay i'm wondering what my constituents are thinking. then, i use nih as the example. one thing we have been able to do in the subcommittee and senate is to prioritize our spending. it is making decisions on where to spend money, not just always the thing that many kansans think, spending more money on everything. dr. collins give me the townhall description of how successful our spending has been in the year since we last met. dr. collins: i would love to give that description. they you for the opportunity. be thing i would say, may the most important discovery
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that happened in the last year, is one we do not know about yet. it was a basic science discovery time, at theat the time come interesting. but we did not realize until a euro to --a year or two, how profound that was. when i was discovered, nobody had it on the list. another big discovery that we could probably mention for anybody across the table, would be something that has just happened in the more therapeutic applications. of a little boy walking around hanging on the monkey bars after being diagnosed with spinal muscular after the -- atrophy. i could say, let's talk about drug abuse yesterday, approval of the fda of the first non-opioid way to help people from opioids, which is an effort put together with the advances in cancer. that was already pretty good
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stuff a year ago. it has gotten so much more exciting with the additional advances. in terms of neurological diseases, advances in aging, some of the basic science of aging has also had interesting findings in the last year about the normal process, not just when he goes awry. certainly the other doctor would we in termsere are asebola and where are we now far as a vaccine for the flu and to, and maybe a solution deal with other microbial problems. i think you get the sense there is a long list of things we can point to ingest this last year. in case anybody thinks, that is fine, but is it worth the money, it is the greatest return on investment that anything the government spends the money on. return from every
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$8.38 return from every dollar you put in. sen. moran: i have another few paragraphs as well when concluding. you have a better spiel and a great story to tell. i appreciate you telling us of those successes. thank you. >> thank you dr. collins and the directors with you today. the record will stay open for one week for additional questions. the committee stands in recess.
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