tv Quantum Computing Research Applications CSPAN May 18, 2018 5:51pm-7:02pm EDT
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cross-interpret bl, shareable and compatible and are working very hard in collaborations across federal agencies and with outside groups to make that sharing more effective. but i think we have the culture as well as the arm tear yum to enforce sharing, and i think we're on an excellent trajectory in that regard. >> okay, thank you. dr. collins, i wanted to -- just one more question on the moderate drinking study. nih is a huge enterprise, and if this was happening in one institute, it could be happening in others. are you doing anything to make sure this is, hasn't compromise studies elsewhere in nih? >> i'm very concerned that this might be the tip of a larger iceberg, and that's part of the reason that i pulled together this very distinguished group of experts to look at it. we will look closely to see if there are other examples of this sort, because that would be very much against the principles that we stand for which is separation of funding sources from outside with decisions about science and
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also, of course, our peer review process ought to be above reproach as far as conflicts. i would be glad to report back to you, but this is one of my roles as the ning ih director. when we find something that has gone awry, we don't just assume it's a little thing that you can put a band-aid on. we make sure that we aim to fix those. >> thank you very much. and thank you, mr. chairman. >> senator alexander. >> thank you, mr. chairman. first, let me say to chairman blunt and to senator murray, senator durbin, all the members of the committee how much i appreciate their leadership in the increase in funding for the national institutes of health, and i've pledged my support to do that for the future. second, it's good to see a piece of legislation that had such broad bipartisan support. senator mcconnell said it was the most important law of 2016, actually be so useful in its implementation, a part of that
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is because of the talent of the team we see before us and others like dr. gottlieb who know what they're doing and leaning forward and taking advantage of the new authority. third, i -- we've developed quite a consensus on science and research. as senator blunt said, a 23% increase in biomedical innovation. i would add so that in the water and energy appropriations committee, we increased funding for the office of sciences 16%. that was the third straight year record funding for the office of science that supports the national laboratories. so when you get three straight years for the national students of health -- institutes of health, three state years of record funding for the office of science and you add to that in the obama administration and in the first two go years of this administration we've funded supercomputing at impolicive levels to keep us -- impressive levels to keep us first in the
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world in that. i think the people not onboard are the office of management and budget. so i'm going to try to talk to the president and others at the white house and say why don't you include this in your america first agenda. why not biomedical innovation, why not national laboratories, why not supercomputing? congress wants to do it, both republicans and democrats. we need to get the omb onboard. now, let me use the rest of my time to ask you about one area, non-addictive pape strategies. we have 25 million americans who hurt badly, chronic pain, 100 million who hurt some. and then non-addictive treatment for open op yoid abuse. 95% of the treatment for opioids is more opioids. it's medicated-assisted abuse, and sometimes they say you don't ever get off opioids. so my questions -- my question is, in two minutes and a half, what progress are you making? senator murray and i have
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included the transactional authority that you asked for, what progress are you making on pain, new pain strategies, and why are 95% of the treatments for opioids more opioids? why don't we have more treatments like vivitrol or some other treatment that helps people get away from opioids? >> so i'm fortunate to have at the table two experts who can handle both pa parts of that question. maybe first to dr. volkov to talk about the treatments for addiction, and then i'll ask nora to respond about non-addictive treatments for pain. we're deep into that as well. >> thanks, francis. we are -- this is one of our priorities, how do we develop alternative medications for the management of opioid addiction. we currently only have three of them, and two of what are -- [inaudible] and vivitrol is an antagonist. >> i'm right that 95% of the treatment, right? >> that's correct, those are the
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numbers. and part of the problem is that that not all of the patients' response to the vivitrol intervention and part of the problem is it's very difficult to induct someone that is addicted right away into vivitrol, and that's when we lose the patients. so we are trying to expand the alternative medications that we can give to patients and focusing also on strategies that are not involved in the opioid system so that we can help those individuals be able to recover and be at one point able to stay without opioid medications. >> so i think that you're absolutely right that one thing that could really help in the long term stepping the opioid problem is developing non-addictive pain medicines to replace opioids in the prescription box for patients. >> and we funded $500 million in research this past year, senator blunt and murray did, and 500 more proposed by the budget for this coming year. >> that's right. and so we have working with our
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industry partners, as francis mentioned, have a plan to really accelerate the development of new medications from the basic science point of view we have a number of different targets that look very promising to develop medicines that are not interacting with the opioid pathway. one that's in current testing now is what's called anti-nerve growth factor therapy which came from really very important basic science years ago on the intersection of that growth factor and the pain system. and companies now develop antagonists to that which look promising in early results. so just one example. but we think there's many more. >> thank you, mr. chairman. >> senator reed. >> well, thank you very much, mr. chairman. thank you, all. and i particularly want to thank dr. sharpless for giving me an opportunity to look at the pediatric oncology program and meeting all the tremendous women doctors and ph.d.s who lead that effort. so thank you. and with the support of another
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tremendous woman, senator cap toe, we were able to pass the star act. can you give us an idea of how you will use this? it'll pass, we hope, the house very soon and become law. >> sure. i shouldn't directly comment on pending legislation, but i think -- and, first off, thank you for the visit. i think it really means a lot to have you, the senate come up and express an interest in what they're doing. and i think it is really, really wonderful, and thank you for your support of pediatric cancer research. i think -- the star act, the intent of it is very laudable to address issues, some issues in pediatric oncologying and childhood cancer that are important. one of these is survivorship where the good news is we're curing more and more kids. but the problem is, well, the two problems are we're not curing everyone, we still have kids lie of cancer. and a lot of the kids we cure are left with lifelong toxicities, so the results from therapy can be quite
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debilitating and transferring to infertility, disfiguring surgery, cognitive dysfunction that lasts for decades. so survivorship has become an issue, there are probably half a million survivors in the united states, and trying -- further research to understand the basic science and why these toxicities occur and what we can do is really important to the area of research. similarly, i think we have this issue of aggregated data in pediatric cancer, and one of the shortages of that are samples, biospecimens that can be sequenced, analyzed and cataloged and presented to the research community in an aggregated format that's usable for research. i think those two, addressing survivorship and biospecimens, are really important and something nci's behind. >> thank you very much. and, dr. collins, thank you again for your hospitality on my visit. you have recently announced support of a research consortium, can you give us an idea what yo want to accomplish by that, doctor? >> i'd be happy to. we have the national institute
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of child health and human development which sounds like the place where pediatric research gets done, and a lot does get done there, but it's actually only about 18% across all of nih. most of the institutes are also heavily invested in pediatric research. i've asked diana by yankee to convene -- by onchi to convene, a high level of players to see whether we could come up with a more coordinated strategic plan for defining where are the greatest priorities in pediatric research, and how can we work together whether it's cancer, whether it's autism, whether it's birth defects, whether it's development, whether it's behavioral issues. i believe with her leadership -- and she's a very strong leader, indeed -- that we have the chance to put forward a plan for pediatric research that will be quite exciting. that has just gotten going, and i'd be glad to report back to you in the coming months about how this is leading us in new
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directions. >> i would be remiss if i did not applaud the fogerty center named after my beloved predecessor in the united states congress, john fogerty. but this is the international arm, if you will, of nih, and we celebrated the 50th anniversary. and it seems particularly important today as we talk about ebola emerging and countries like in that we have this agency -- like this this that we this agency in very close rapport with cdc. can you just comment upon the next wonderful 50 years of the fogerty center? and tell peter kilmarks i said hello. [laughter] >> senator, appreciated you very much being there and speaking to the group. one of the things foagerty has done that has been most powerful, even though it's the smallest, is their training program for fellows. i'm just going to quickly ask tony fauci to ask you a question
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or two. >> thank you, francis. yes, it really has been very important and very much interding tated with what we do as an nih institution. but particularly in the area of infectious diseases within anaid. you recall that when we had the ebola outbreak in 2015 and early '16, that there were cases that -- as you would expect -- traveled not knowing they were infected to places like mali and places like nigeria. and those cases did not result in outbreaks in those countries. and the people who were taking care of those individuals were individuals almost all of whom were trained as fogerty fellows. so we had a pre-existing network of people there who had been trained according to the culture of the nih in science and public health. so that was just a dramatic
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public health example of how the fogerty center was a true partner not only on our campus, but foagerty in lag goes. it was really quite striking. >> you could be elected to congress in the second district. thank you. [laughter] >> senator capito. >> thank you, mr. chairman and the ranking member. dr. collins, it's great to see you again. my first question is on opioids and what you're doing, and we've talked numerous times about this. obviously, i live in an area that's highly affected here. two questions. you mentioned and you said in your -- that we had put $500 million investment into this, but you've mentioned in your response to questions that if you have private partners, could you elaborate on how that partnership works and what kind of commitment dollar wise that private partnership is committing to add to the $500 million we're doing in the research? >> happy to. we have worked intensively since
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your wonderful decision to make $500 million available in fy-18 and to have that in the base so it will be there in '19 and beyond. working with all of the students across nih, we have put forward an opportunity for new and bold ideas to come forward, and we are in a place now, i think, of having a remarkable portfolio that we're ready to launch. you also graciously gave us that first year of support as two-year money so that we could, in fact, carry over some of the fy-18 funds into '19 since it's late enough in the year that starting brand new things is a little challenging. but we have figured out how we're going to spend a significant fraction of that 500 right away. it does include a wide variety of applications including some of the things that have already been discussed by dr. volkow, but also such things about what to do about the neonatal abstinence syndrome and what is
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their long-term future. the public/private partnership which you mentioned is actually ad modest part of this -- a modest part of this broad portfolio but an important one. again as we talked about earlier, the decision was made by me but based on strong recommendations that that partnership should involve assets that are contributed by industry that have value; data, compounds, scientific expertise but not a cash contribution. it is a little challenging right now to attach a dollar value to what their in-kind contributions are going to add up to, but it will be substantial. >> good. >> so putting that all together, we think we're in a good place to move both the public/private partnership and the rest of this broad portfolio together quite rapidly. >> good, thank you. dr. volkow, on the neonatal abstinence syndrome, we're the state most high layfected by that as -- highly affected by that as well, and this is an area of deep concern to everybody. we've got 1,000 more kids in foster care, a lot of these
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babies probably will be there, repeat mothers. they had repeat exposure -- i mean, they had numerous exposure to numerous drugs. and i believe you visited our lily's place down in huntington to see what kind of care they're getting there. the research that you're doing and the act now trials, are those being conducted across the nation, or how do you find the right place to conduct those kinds of -- and where are you doing that? >> well, the act now is one to have programs that will be funded through the new money that has come for the opioid crisis and will allow us to actually maximize protocols that will enable, for example, us to determine what are the optimal be interventions for the best outcomes on neonates. and that includes -- [inaudible] we have other research studies also ongoing, and i was at west virginia last week, and i was in huntington, and i was horrified to hear that one out of five
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newborns had opioids on them. and one of the points that is highlighted is that we need to address the -- [inaudible] but we need to address also the needs of the mother once the baby's born so that the outcome from that -- >> right. >> is addressed. so we're working with research of new medications and new non-medication interventions to improve the outcomes of those neonates. and the other aspect that francis mentioned is we're vup interested in understanding how the brain of these newborns is going to have been influenced by getting exposed to opioids as well as other drugs during fetal development when the brain is very, very vulnerable. >> right. and i don't think we -- i mean, i'm anxious to hear how we progress with that. i guess one of the points in asking my question is there's a wealth of data all around the country with people who are hands-on in these, in these scenarios dealing with this right now that i think can feed not only good information, but
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can help you conduct trials on the local level. sounds like that's what you're already -- >> correct. >> -- moving forward on. i am really pleased to be on the star act, the childhood cancer act. i'm glad to hear about the survivorship. that's something of great importance to me. a lot of this i'm very interested in, as dr. collins knows, but i do want to go to dr. hodes because alzheimer's is something i've personally are been touched with. doctor, one of the things i have heard is that it's hard to recruit patients for clinical trials for alz heym her's. is that the case? >> uh-huh. >> i know it's hard, because by the time you start exhibiting the symptoms, you're already in it. how do you, how are you developing all those trials with different -- >> well, senator, it's an excellent question, and it is a challenge to us for multiple reasons. as you've commented upon, for individuals who are already affected with symptoms, we carry out studies that are extremely important. but there's also a sense that in order to be most effective in --
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>> find aggravation cure. >> -- preventing the appearance of symptoms that we need to intervene earlier. >> right. >> this means we need to recruit people who are not coming through a clinician who suggests they participate in a clinical trial. these are people who are at high risk but show no symptoms. we need to develop and are developing strategies for screening those at high risk and then the charge of finding people who are committing, dedicated to participate in studies to see if we can make a difference, all of us present unique opportunity for this. a million people who have signed on with their interest, we can screen those who by a variety of metrics may be at high risk for developing symptoms years or decades later and take an opportunity to intervene with those now. so we're in the midst of a nationwide program which will be announced this summer to look at multiple modalities for recruiting patients into studies. >> good, thank you. we want to support that. thank you. >> senator shaheen. >> thank you, mr. chairman. and thank you to you and senator
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murray and senator alexander for your commitment to research and funding for nih and to you, dr. collins and everyone there, thank you so much for the work that you're doing. you give hope to so many people across this country. dr. volkow, thank you for coming to new hampshire. i especially appreciate the work that's being done to address the opioid epidemic, like my league clos on this -- colleagues on many committee, we have been very hard hit in new hampshire, as you know, from your visit to catholic medical center and other places in the state. i was interested in your discussion about new drugs that are in development. as you're look at what the potential is, are you separating out fentanyl as a particular opioid that has very deadly properties and, therefore, requires a different kind of response, or are you lumping that in with everything else?
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>> we cannot lump it with everything else because what we are hearing from the field and being reported in -- [inaudible] is that when people overdose with fentanyl, the doses that we use of far can are not -- far can are not sufficient -- narcan. the other concern is you get exposed because of its potency, your risk of infection is much higher. as of now, we have not ever done a trial of how do you treat someone who's addicted to fentanyl, and that's one of the projects we would like to implement as soon as get the approvals. but also with respect to the medications, we are actually funding researchers who are partnering with pharmaceuticals to develop stronger antagonists. so this is the nasal narcan which we actually developed. >> right. >> but it's not sufficient for fentanyl. so we're developing with companies longer lasting, more potent or alternative medications that may not be based on the same mechanism as naloxone. because the other challenge is
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not just fentanyl, but what we're observing is people are overdosing with multiple medications; alcohol, fentanyl, so this is not sufficient. >> and we see so many people who have co-occurring mental health issues along with their substance abuse disorder. often they're using substances to address their mental health issues. so are you doing any research that looks at the two problems together that may give promise for, as we think about what the future holds? >> we cannot not do that because it is many times much more frequent than the isolation. and if you don't address, for example, the depression on someone that's addicted to opiates, you are not going to succeed. also a very important component to follow the overdose is we really do not know which ones are intentional. therefore, suicide. if you are reverting someone that has suicidal behavior
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behavior and you don't intervene, you're not going to succeed. we're recognizing the reality is comorbidity of addiction with substance abuse and with pain. and those are more challenging than when they are in isolation. >> so did you want to add anything to that, doctor? >> i think dr. volkow very accurately characterizes the situation. i think one of the things that we're most concerned about in terms of this crisis, just if i can show you this graph, is the way in which the epidemic in terms of the overdose death has shifted from being prescription opioids to now fentanyl just going straight off the chart as it gets into the heroin supply. as more individuals who have fallen into addiction cannot find sufficient access to prescriptions, then they shift over. >> right. absolutely, that's what we're seeing in new hampshire where we have the highest rate of overdose deaths from fentanyl. dr. fauci, senator blunt
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mentioned the ebola end themmic and the -- end demic and the news report this morning that said the first case had been found in an urban area, large city in the democratic republic of congo. and given drc's other challenges, how worried are we that that epidemic is going to get out of control again? >> well, obviously, given our prior experience we are on very high alert. there are some factors that mitigate against their having the same situation as we saw in west africa. but there are also factors that actually might favor that. as you mentioned, the first cases that were reported in the very early may, the first week in may, were in a place that is on a remote area called lake tumba, and it's -- the bad news is that it's very difficult to get help in. the good news is it's very difficult to get anybody out. but what you heard this morning in the report is that there is multiple different zones.
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bakarro is one zone, another zone is an area that has a city of 1.1 million people. and even though there's only one case there, there's a total now of 44 cases even though only 2 have been confirmed. there are 20 that are probable and 20 that are suspicious, so there are probably many more cases. what we're doing now is shipping in with the who helping, obviously, the kinds of things that were the fruits of the work that we did with the support of this committee and others to develop countermeasures. and i'll very briefly give you an example. you heard probably on the media that the who has authorized the shipping of the vsv vaccine that had its first phase one trial right at the clinical center in nih, and then we did it in africa, and that was the one that did the ring vaccination. we also have zmap that we
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published from the intramural program at nih that went over there in liberia. that's also being shipped. and we have some experimental drugs. one we're partnering with gilead and some antibodies that have been developed. so although in direct answer to your question, we are on high alert, we are always concerned when there's ebola, but we right now have a number of countermeasures that we're able to develop to go in and, hopefully, block that. so our hopes, our expectations are always cautious, but the hopes are that we will not have the kind of outbreak that we saw in west africa. >> thank you. thank you, mr. chair. >> senator durbin. >> thanks, mr. chairman. and let me say at the outset that in a world of frustration and partisanship, what is happening in this room this morning is a welcome exception. you will find more positive feelings, more achievement i hope and more bipartisanship than in almost any other room on capitol hill. and i want to salute the chairman of the subcommittee,
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senator blunt, his ranking member, senator murray are. i told her i was going to praise her before she left -- [laughter] and, certainly, senator alexander in his work and senator shaheen. this is, this is a great assignment because, with the help of the folks sitting at this table, we actually feel like we are taking steps forward. and to senator blunt for his leadership on this, we were -- we've established a standard, i think. i hope it's one that we can live by of sustainable, reliable increases in medical research funding in the united states of america. dr. collins told me several years ago that's what we need, and we're doing our best to meet that need. one of the areas that i've recently learned a little bit about was in foreign affairs magazine, of all places. and i happened to be reading it, and this liberal arts lawyer came to try to understand something called crisper which
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is included in your publication here of promising technologies. and ironically, i i talked to dr. collins about this earlier, a family from illinois came to see me last week, and the mother, wife has myotonic dystrophy, and it is a genetic disease she has unwittingly passed on to her children, a son and daughter, whose circumstances are even more challenging than her own. and, of course, their future is really unknown. i mentioned crisper to them, and they lit up. they said this is the one area where we feel like there's a chance. please, dr. collins or one of your colleagues, give us a moment about crisper and what we are doing. >> i'm happy to because i agree that this is one of the most exciting things that's happened in a long time in terms of providing tools both for basic science and for therapeutic
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applications that opens up entirely new vistas particularly for individuals with genetic disorders where we know there's a misspelling in the dna. this provides an opportunity to go in and fix that in a very precise fashion. so it's also a great story about basic science because this particular enzyme called cass 9 was discovered in a very obscure area. people trying to understand how bacteria can fight off their own viruses. bacteria have their own viruses, and somehow they manage to survive. and it's a very elegant system where the bacteria have an enzyme that basically recognizes a foreign dna sequence and goes and snips it and inactivates it. but it's programmable. so jennifer and her colleagues and another doctor and his colleagues and a few other people who are arguing about who gets the credit basically came up with a way to make that bacterial system work in all kinds of cells including human cells. it is very precise. you program it, it can find in a
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3-billion letter instruction book the one that you want to alter and zero in on it and very precisely make a cut or make a substitution. now, of course, in the basic science lab this is fantastic. every laboratory that's doing molecular biology is using crisper cause, including mine. but they are therapeutically the here is what, i think, has us particularly excited. as i mentioned earlier, sickle cell disease may well be the first success of this, because the problem is in the bone marrow stem cells. you can take them out, you can purify them, you can utilize this enzyme system to fix the sickle mutation and then put them back. and that should be not just a treatment, but a cure for people with this decide. 100,000 of them in the u.s. i think that's going to happen in the next five years. the first clinical trials are probably going to start this year. >> i hate to interrupt you, because this is important, but i have one other topic that i want
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to mention, and i hope the subcommittee can zero in on the crisper technology and what is happening there. i think there's so much promise. >> indeed. >> i wanted to mention one other -- >> please. >> -- that is important to most of us. 27% of the high school students in the state of illinois have something in common, 27% of them are now using e-cigarettes and vaping. it used to be 28% using tobacco cigarettes, now they're into e-cigarettes and vaping. and the people who are peddling these products that they inhale with nicotine are putting them in candy flavors. here's one, brand is lung candy, and the flavoring is cake batter. in this listing here, it's a long listing of flavors for children to see opportunities for vaping. we see exactly what is happening. can i get a comment from any of you about what you consider to be the perils or danger of this type of addiction?
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>> indeed, we are very concerned because one of the issues here why 50% of teenagers say that they are starting to vape only with flavors, 30% of them are starting to vape p with nicotine and are becoming addicted to nicotine. and in stories that have been -- studies that have been already published, it has been shown that if you start to vape, you're much more likely to go into nicotine vaping, and then to go into smoking tobacco. so a major concern is that we will be losing a lot of the advances that we did on prevention of smoking among teenagers and then going to -- [inaudible] who through these type of -- [inaudible] another aspect that people don't really recognize is that nicotine acts as a priming. anything that you take becomes much more reinforcing. and as a result of that, you get exposed to drugs while you're having nicotine onboard, you're much more likely to become addicted. so the concern is not only that these teenagers will become cigarette smokers as data has already shown, the risk goes up,
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but also by doing this their brain becomes primed to the addictiveness of other substances. >> thank you. thanks, mr. chairman. >> thank you, senator durbin. dr. fauci, let me give you a couple minutes to talk about two things, zika -- i was at st. louis university during the zika crisis where they were working and felt like they were coming to conclusions. so are we going to have something available next time and flu, universal flu or whatever you want to talk about on your research on those two topics. >> thank you, mr. chairman. first, we'll take zika. as you know, as i reported to this committee on more than one occasion, we have progressed in our vaccine trial from the phase one trial that we originally did at the nih to now deploying a phase 2b trial, being relatively advanced to not be only ask is it safe, but does it induce the kind of response that you would predict would be effective.
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and i'm pleased to report to you that today we have ongoing now a phase 2b trial in several countries in south america, the new york stock in new york stock exchange coe, the in -- in mexico, in texas and florida to determine if the vaccine is safe. it is scheduled to have between 2400 and 5000 people in the study. we're accruing or very rapidly. we anticipate and i'm cautiously optimistic about that, that we will have it fully accrued by the end of 2018. and then if, in fact, there is an outbreak, we could get an efficacy signal. and if there is not, then we are working very closely with the fda. if we can get enough what we call data that shows it doesn't induce the kind of response that you want and bridge that to the animal data which are very convincing that this is a vaccine that can induce a good response, we're working with the fda to determine if, in fact, we
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can get accelerated approval. you never anticipate what their decision will be. next, universal flu vaccine. we are very grateful to the committee for supporting the addition of the $40 million for a universal flu vaccine. we had a meeting in rockville in june of '17 in which we developed in association with experts from all over the world and in the united states in influential saw a strategic plan -- influenza and strategic plan which we parished in -- published in february of this year. we are immaterial policemenning that plan both from a basic science standpoint, but also with candidates that are at various stages of development; namely, preclinical in animal study or phase 1 or phase 2. and i might just close you may have read just a couple of days ago an nanaid phase 2 trial with
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a candidate in association with the company -- [inaudible] has been niche candidate -- initiated. and we started a new months ago one of the trials from our vaccine research center. things are on track. obviously, as i mentioned to the committee it's not going to be an overnight type of thing, but we're well on the road in various iterations of going towards a universal flu vaccine. >> i'm assuming what we're trying to get at is every year we wouldn't have to try to calculate what that year's flu was like and more often than not be slightly off target. >> you're absolutely correct, mr. chairman. the perfect -- and that's going to be difficult to do -- universal flu vaccine would be one that would cover all versions of seasonal and any potential pandemic. but the road we're taking now is to go step by step. i tend to refer to it as universal flu vaccine 1.0 which means that we won't have to worry about the h3n2 that we do
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each year, and we have to change it a little each season, the first version will cover all of the h3n2s. the next one will cover very likely an h1n1. there are two major groups of influenza vaccines, viruses that have multiple viruses in each group. getting one that covers both groups is getting close to the perfect one. so the answer is the end game is that you and i -- but most likely our children -- will be able to get a vaccine early on in life, a boost and then maybe every ten years or so and not have to worry about each year guessing what the next iteration is going to be. it's the guess that's the problem. because sometimes you don't get it right, and even when you do, it changes enough that it evades the vaccine. and that's what we're trying to avoid. >> good. well, i may have unlimited time here, and i may exceed that. who knows what --
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[laughter] what may happen. dr. sharpless. so i think in america today, in the united states today there are 15.5 million cancer survivors. in 1971 there were 3 million cancer survivors. incredibly exciting time. imknew know therapy -- imknew know therapy, the brain initiative that i assume will have a significant cancer-looking element to it, crisper, could you just take -- while you and i have some time here, could you take a couple of minutes maybe and talk about your vision of what you hope and believe can happen over the next handful of years in the cancer institute. based on what we see happening already. >> sure. i think you're really right about that. it's a very exciting time in cancer research. i recently had a colleague e-mail me that it's like i picked the perfect time to lead the national cancer constitute
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because there are all these -- cancer institute. because there are all these exciting things. it's sort of like christmas in our field where all the new advances come out, and a number of exciting advances in a variety of different cancers. so i think there really are a lot of opportunities that cash in on this observation that cancer's not only ones or tens of diseases, but hundreds of thousands of diseases, and each one needs its own specific treatment. the good news is there's a lot of excitement, a lot of gnu technologies and approaches, and a lot of that research has been empowered by the funding from this committee. but i think the bad news about it is that problem is somewhat different from what we imagined. we used to think of cancer as one disease, and we had the cardiology paradigm where we put 800 people on one arm of the trial and 800 people on the other, and that no longer works. the modern approach to cancer, i think, has to be somewhat different. the things i thought the nci should focus on is training the work force so they have the right -- so that we have the
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right kinds of researchers, cancer biologists who understand basic biologying and big data because we're aggregating data at such a furious pace. we need to recommit to basic science. it's not enough to make progress against some cancers, we really have to make progress against all cancers, and that requires a basic biologic understanding of all cancers. .. >> and lastly we need to get serious to aggregate our data to have the reality and
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histology and clinical information all available that is safe and secure so we can understand what mutations would cause patients to respond and get a handle on the answers that we have to treat. >> i was at fda not too long ago they put a cancer team together. the thought of that discussion was not foreseeable future there just may be something that each individual prescribed for them amps up whatever their unique fighting capacity needs to be that is likely to happen to them. the other book is the genetic effort if that works but it is
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an exciting time with immunology that five years ago an observation made in passing with not have been made at all. this is what we're doing and i'm sure how you will continue to work on why this doesn't work on some cancers and what you need to find and it may not be possible ever and it may not be possible ever appreciate you raising this why does it work on -- not work looking at a very successful public-private partnership for advancing cancer therapies there are 12 pharmaceutical companies have partnered with nih to ask the question what are those biomarkers that tell you even know therapy will work or not? we have dramatic success stories emily whitehead is the little girl who failed to respond to the traditional
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treatment for leukemia and very much near the end and with t-cell therapy not only treated but cured and five years out and looks wonderful but it doesn't work for solid tumors nearly as well. why? what about prostate and breast and brain tumors? that is what the partnership aims to do. it is all competitive head out there in the public. it is contributing expertise and resources is exciting to see that taking shape under dr. sharpless. >> that is one of the initiatives that each of these therapies requires a detailed understanding of how they work but it is leaps and bounds every day we see a new area of a drug that used to work for now better with the new class of therapy.
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lotta stuff is going on but important to note we do still have some cancers where we haven't made much progress like brain cancer is still a real problem and i do think we have to focus on the success but even more of those that have been recalcitrant and that is an important part of our mission as well. >> i think the area of greatest spending the quickest growing spending of federal health dollars would be alzheimer's and dementia and maybe if you both can talk about what we are finding because we have made big investments we could eliminate a lot of things we know that don't work but how are we doing on the other end with that dementia impact?
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and when it comes to you? >> first, thanks to the committee for the increased appropriation of resources toward alzheimer's disease and related dementia has had an enormous impact for example in 2015 we funded 152 award supporting research in two years later it was 442. importantly it is reflected with confidence and excitement with research community seeing these national commitments sustaining resources with renewed energy on the part of early-stage investigator so all of these awards more than a quarter, 27% that would not receive that was from previously in the larger number more than one third never had support so we bring
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that into the community not just four dollars opportunities but the energy that comes those that will commit their careers bringing new discipline into the field this is included in the expansion of clinical trials reported at 140 and 40 and deep will be important to using new techniques to look at gene expression patterns and an amazingly effective consortium around alzheimer's disease to bring public and private expertise and financial support to together to find new discoveries to identify those targets there has been a consensus with new molecular targets that could into entirely new approaches especially for that translation into clinical trial.
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so those numbers only illustrate the intensity of excitement of sustained funding and the pace has increased exponentially. >> i feel more and more positive about the blood test and things like that that will begin to early identify what was happening with employees in the brain right step in the right direction. >> thanks for the wisdom for including the alzheimer related dementias with the major effort to decrease the public health problem of dementia in the country and most patients who have dementia are diagnosed with alzheimer's but especially the elderly looking at the brain, more often than not we find different things are going on.
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in the two areas that are most common are the inclusion that people have dementia diagnosed with alzheimer's and also have the signature parkinson's which is the aggregate so to understand the contribution of those not just alzheimer's dementia but also those that have it for a long time is a focus we can like it much more aggressively. the other area is a combination of vascular disease in people diagnosed with alzheimer's. this is incredibly important to understand because we have made strong progress over the last 70 years in decreasing the risk of stroke which is the most common consequence of vascular disease in the brain but also incredibly common in
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people who have dementia diagnosis. so we were working with the national institute of aging on projects to track the health of the vascular system and contributions of dementia with the hope with things we already know about to be more aggressive and prevent people from going on because we can block this component of dementia. >> thank you chairman and dr. collins for testifying today in all of you for testifying. i have been involved as a volunteer with the american cancer society for many, many years in mississippi so treatments and therapy that have formed the prognosis for so many different types of cancers i am grateful for the role that you have played in so many of those discoveries. what you are doing is so
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important and i truly see it changing in saving lives. despite these recent innovations my state continues to have the highest of any state those receiving cutting-edge treatments or participating in a clinical trial means traveling hundreds of miles to the cancer center in another state which is not possible for so many patients. so dr. sharpless how is the national cancer institute working to ensure patients in states like mine that do not have the designated cancer center can participate in clinical trials and benefit from the newest discoveries? do you think the question you raise an important issue which is great news we make this progress with these therapies and new approaches but if we cannot disseminate into the broader community to implement that into the real world then
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are we helping? so the national cancer institute is very concerned about how we assure the advances are just beyond the cancer centers which are a great program but there are only 70 in the range is limited. one program we have is the national committee which those sites that have several satellite sites across the country between the cancer centers we reach virtually the entire country. the site in addition to do clinical trials also have areas that have patients that are more likely to be rural and minorities so to keep that in a graphic that looks more like united states in general but we just decided to expand
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that scope so it is successful to build on that. and we can do clinical trials with 6000 patients on 1100 sites through the national cancer institute which is a real need of cancer research because 5% go on clinical trials that number needs to be higher this is one way to work to address the important issue. >> in the center to be both chosen at night with the research program in mississippi to rule them on national 1 million american volunteers to help identify treatment to have the locations lifestyle and
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genetic biology. it was only one out of six community centers nationwide but as the nih was speaking out how have you incorporated lessons learned for those that participated? >> it is a great question we did spend the last year ramping up and recognizing this is one of the most ambitious projects we have ever mounted it we need to have great attention paid to the needs of the participants for security and privacy and made a commitment that was truly national for those not traditionally were not invited in community health centers were not so engaged and particularly powerful place to
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see how this would go we have been very encouraged with the pilot projects that is one of the biggest studies nih did in a long time and from the community health centers like jackson heights we were gratified to hear interest on the part to get engaged in this research and they wanted to be about them so i think it has been a wonderful experience we haven't tried this scale before but now as of ma may 6i have done the launch and anybody in the united states can join all of us that is all you need to know. >> we appreciate it. >> thank you mr. chairman and i didn't want to go home and
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tell my family have the whole panel from nih in front of me without asking about the prospects of research for type i diabetes. i have a granddaughter who has type one iso cochair the diabetes caucus in and we know how devastating the potential is in this country if we don't figure out a cure. one out of three will have diabetes and one of the most expensive if not the most expensive chronic disease that we have so can you give me an update where we are with research? dimmick i would be happy too and i appreciate your leadership this is another very exciting area where things are moving very quickly. for type one and type two in that excitement that surrounds the potential around the artificial pancreas taking
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care of insulation on --dash insulin and the fda approved the very first example in a closed loop whether it is a sensor those levels of glucose to administer insulin accordingly but this is just the beginning of what will be a remarkable series of events long -- advances but whole stem cell area will be critical as well. after much hard work we have figured out how to take us them so and convince it to become potent then give it the appropriate set of cocktails to convince it to be a cell that makes insulin but it is your own self. so that is where we are headed the opportunity to make the artificial pancreas that doesn't have silicon but your
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cells that are reprogrammed to make up for what the other cells should have been doing. on top of that in terms of prevention with type two we know this is connected to obesity and insulin resistance to try out those efforts to reduce rate and increase exercise are looking promising over the long term from those that are tipping over to diabetes are from getting there also just asian or and what information we can learn when this happens during pregnancy what is the follow-up that indicates the risk in the future? it is an exciting time but i want to reassure you we are deeply in the middle of all of this and the progress is quite gratifying. >> i appreciate that i'm excited about the new developments we need to think about how we can reverse the incentives in the healthcare system to do more for prevention with type two where
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it makes a difference and unfortunately i think we are not providing the resources that we need there and in the long term it will cost more money. i want to ask a follow-up because what i have heard from the diabetes community is the frustration with how long it is taking to approve the artificial pancreas and the further developments on that. we see people in the community are doing the bootleg and making their own artificial pancreas. and feel that provides more relief than what they are getting currently given the system that we have and the dependence on insulin and the way that works. can you speak to that? is that something you have heard about or are you concerned the risk and what that might be?
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>> i do think there are we to be very careful about putting in place the artificial pancreas without being confident it has the appropriate safety mechanisms because an overdose of insulin can put you into a coma or lead to death. this needs to be a system well worked out. i understand why the fda has to be rigorous with evaluation but with the fda commissioner really is dedicated as soon as possible and works very closely with fda and his leadership and certainly some of the things we can do to provide that data is the partnership we are pushing pretty hard right now. >> thank you very much for your great work you are doing. >> dr. do you have anybody looking at the socialization challenges to social media
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exposure or the screens or the impact that could have on adolescents or others? >> social media is taking over the country and the consequences the pros and cons are yet to be determined at nih a lot are using social media to help research think the national institutes of mental health looks at the effects on adolescents but the doctor can mention an innovative project looking at this and other exposures on the development of children that she recently, you can finish. >> i had a 5050 shot. [laughter] >> it is important because drug taking and experimentation is a very
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social behavior and one of the things we try to understand is that dramatic change happens in the way that they interact with one another could influence their behavior with drug taking but they are developing during childhood and adolescence and with that ability to respond to the environment so as a child if they are exposed and how does that influence the development of the brain? so we started a study with our partners at the nih where we aim to recruit children we have already recruited 9900 finishing in september but are the next ten years to do brain imaging and to measure their
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exposure to social media and that influence with the development of the brain this will allow us to understand this dramatic change of how people are interacting with one another to influence the brain also research to understand how social media issues to change the perspectives and the likelihood from what we see in social media so just like electronic cigarette on -- cigarettes in those the likelihood that a teenager will use them the same with marijuana and alcohol it is a completely new world and as of now we don't have that understanding on the human brain and behavior.
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>> chairman thank you i'm glad the meeting is still going i don't know if you are pleased with my arrival but i am honored to be here. [laughter] >> dr. hodes i'm not sure what has been asked and answered but i have some concepts that are now developing regarding alzheimer's. we have talked about the detection and efforts to use biomarkers from someone at risk or has alzheimer's before they begin showing symptoms. the national institute on aging just coordinated to propose a biological construct looking at the use of measurable changes in the brain to better understand the earliest biological signs that lead to symptoms so how will this framework help to drive research forward?
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does it represent a new understanding of the beginning of the disease? >> that importance to identify those changes in the related dementia early is critical with the best opportunity to intervene at an early point to track success or failure to measure biomarkers is critical. the definition is a publication that suggested for the pathological definition based on plaques and tangles there is a set of biomarkers that are standardized can assess the cross study and in doing so vigorously colonize the results of multiple efforts and more quickly understand which biomarkers are the best predictor and out of a response to treatment or not. those studies that senator
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blunt referred to right now those biomarkers that are most successful are either imaging working extremely well that is cumbersome and expensive for cerebral spinal analysis which works but now the goal is to have fluid biomarkers to take the advantage of blood and serum or a pattern of molecules allowing us to screen many people through those longitudinal studies. >> doctor collins, with the leadership of senator blunt and others we have had success in large part because you have made a case for additional resources with each of your institute in which the public good it is demonstrated and the advancements being made are sufficient for american
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people to feel comfortable that progress is on the cusp of those in our lives. in the last year since you were here doctor collins and setting what can i tell my constituents to reassure them their tax dollars are wisely spent? sometimes i go down that track explained we should spend no money on or reduce spending or spend more money. by the time i say that sentence i wonder what they are thinking about a senator from their stay interested in spending more money on anything but then i use nih as the example one of the things we could do in the subcommittee is prioritize to make decisions where to spend more just not but spending more on everything.
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doctor collins, give me that how called description of how successful our spending has been in the years since we last met. >> i love to give that description thank you for the opportunity. one thing i would say is maybe the most important discovery is one we don't know about yet because of the basic science discovery at the time it seemed interesting but we did not realize just how profound it might be when it was discovered nobody had it on their list but another big discovery we could mention for anybody would be something that just happened in a more therapeutic application i told the story of a little boy who should not be alive who was walking around hanging on monkey bars after being diagnosed with spinal atrophy and now has had a dramatic
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response. i could say talk about drug abuse just yesterday talking about the first non- opioid way to help people withdraw from opioids which is an effort put together within industry. the advances of cancer therapy already was pretty good one year ago all my gosh is now so much more exciting with the -- additional advances with advances of aging even basic science has interesting findings of the process and certainly dr. fauci would say where were we one year ago with ebola and now even with things such as a vaccine for the flu or even hiv or ways to deal with a antimicrobial
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existent mom -- resistance problem that has already gone on too long but you can see there is a long list of things to point to and just the last year if anybody thinks is it worth the money is also the greatest return on investment anything the government spends money on every dollar it returns 38 cents and within five years because of what happens with the economic impact. >> i have another few paragraphs after i concludes. >> you have a great story to remind us of those successes. >> thank you doctor collins the record will stay open for one week for additional questions the committee stands in recess speefive.
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