>> the appropriations subcommittee on labor health and huge services and related agencies will come to order. i am certainly pleased we could have this opportunity this morning, dr. collins, to talk to you and the other institute directors about the work you're doing and the work you'd like to do. certainly every family faces health concerns during their lifetime and there are so many things that can be done by nih that i think can't be done anywhere else. a new drug, a new device, new treatment can take anywhere from a decade to longer to develop, can cost billions of dollars on occasion with a pretty high failure rate even when you think you're on the right path. certainly it's necessary for the federal government to invest in biomedical research. it represents the hopes of lots of people and lots of families

and particularly now as we see conditions growing as people survive heart problems and strock problems, we see more people with alzheimer's' and cancer challenges. we see the potential for designer medicine largely because of the great work that was done to figure out how to define and understand the human genome system in a better way. this year this committee -- subcommittee and full committee have placed a high priority on this research. we have planned for and have a bill that includes $2 billion of extra money for that research, increase of about 7% over current years spending. over the past decade, with not much new money going into nih, e purchasing power at nih is decreased by about 22%. we hope to see that reversed if we're successful with what we

are trying to do to provide the increase that we are looking at here. these are clearly difficult budgetary times and i am sure we could spend a lot of this hearing talking about how there should be more money for other things in this budget, and there's a disagreement in some cases and an agreement in some cases that if we had all the money in the world priorities might be a whole lot easier to achief. butic look forward to hearing from your team that you brought as we talked about this morning. i specifically said can you bring some of the people that we haven't seen lately who are focused on the individual areas of research so we can get a greater sense of understanding what the potential is, what the needs are, what is out there that you're seeing and begin to see. and also the challenge of young researchers having research grant approved are dramatically less than we were a decade ago.

and i'm sure that's a topic we want to discuss as well. how long do young researchers stay in research if they continue to have their ideas not allow them to move forward. so those are all things we are going to talk about here. i will turn to senator murray who is a big supporter of your work as well. >> thank you very much, mr. chairman, i think we all really appreciate it. thank you for being here. i am grateful as we all are to champion the critical work that nih does. it's great to see you here. and thank you to all of your team with us today. we look forward to hearing from all of you. all of us agree there's a lot more we need to do to keep our families healthy and continue investing in strengthening our economy frot the mittle out. the work of the national institutes of health is vitly

important to that effort. the nih supports basic research that makes medical advances possible, gives those living with diseases hope and competitive. in my own state of washington we have researchers who are working on ways to repair heart tissue that's been damaged. we have people working on decoding difficult to treat forms of breast cancer. we use precision medicine to tackle eye disease and alzheimer's' and the list goes on. these are just a few examples of the incredible work to improve health and well being across the country and around the globe. at the same time, the life sciences are helping to drive economic growth and job creation in my state the life sciences sector directly employs 34,000 people making it the fifth largest employment sector in my state. the investments that we make in others ducation and

that support the life sciences indirectly help create the jobs of the 21st century and ensure a workforce that can take them on. that is why like chairman blunt i see maintaining our country's central role as the top priority. and federal inswrestments could not be more important to the effort. pporting and making sure short-sighted budgeting doesn't get in the way. you, fact, i know that dr. collins, have said that increasingly the nih is having to turn promising projects away. for patients and family wloss are waiting and hoping for medical breakthroughs, that is unacceptable. i am very proud that in late 2013 democrats and republicans were able to reach a budget agreement to roll back sequestration for fiscal years

2014 and 2015 and as we all know that deal expired last week which means congress is going to once again have to come together and find a solution. as i have made clear, i believe that we need an agreement that bills on the bipartisan foundation set from the budget deal last congress rolls back the cuts to defense and nondefense investments equally and protects priorities that are essential to promoting a strong and growing middle class like research and education and infrastructure. i have been encouraging my colleagues on the other side of the aisle to work with us so we can reach another bipartisan budget deal and avoid those automatic cuts that impact these and other important investments in our country's future. i'm also currently working with chairman alexander, who is here today on the help committee on a bipartisan initiative to advance medical innovation that is an effort that is very much related to the conversation today. i see that initiative as an

opportunity to help patients get the best. most effective cures and treatments, as quickly as possible while upholding the highest standards. and to me a central part of accomplishing this goal and tackling the tough medical challenges our country faces is making sure that research and development can thrive. i am pleased that so far we have seen bipartisan interests in camping up investments in the nih and the f.d.a. and i am clear i will only support a bill that does just that. i am going to be focused on finding a path forward in the coming weeks because put simply stronger investments in medical research means a stronger healthier country. so i am hopeful that republicans and democrat ks come together to build on the bipartisan foundation we set in the budget deal last congress and make the investment wes need to seize these and other opportunities in a way that helps our economy and our country work better for our families.

>> we have a deep bench of remarkable scientists leaders, 27 institutes and centers. you will see five of those folks. starting to my left dr. john lor. dr. walter to him korshez. a distinguished neurologist and scientists. over here on my right dr. doug dowey.

next to him dr. griff rogers, director of the diabetes digestive diseases and kidney diseases institute. and also one of those folks whose being honored this evening because he is one of the nominees for this year's awards for public service. volkal r end dr. nora who serves as our able and highly recognized by the press because she is often in front of them talking about addiction. the national institute of drug abuse. that is my team. now, i will maybe let you start the clock and i would like to tell you a few things by way of an opening statement. it is a great honor to be here before you to discuss how nih is investing in a healthier future for all americans. longevity, you can see here what has happened. breakthroughs generated by

research are behind many of the gains our country has enjoyed. for example, cardiovascular diseases, death rates have fallen by more than 70% in the last 60 years. cancer death rates are now dropping by one to two percent annually. likewise hiv aids originally when first being written about as a death sentence now treatments greatly extend lives and prevention strategies and the increasing potential of an effective vaccine are enabling us to envision for the first time an aids-free generation. i want to thank all of you for your continuing support. we see in front of us a remarkable landscape of biomedical opportunities powered by exceptional advances in scientific knowledge and technological innovation. this morninging's announcement of those noble prizes is a compelling example of how these

investments have been paying off building upon work that has been going on for decades. but i would like to share with you this morning an inspiring has , another one that emerged from decades of research. this is the story of cancer immuno therapy, a treatment that involves harnessing the body's own immune system to fight this disease. i would like you to meet emily. back in 2010 when this photo was taken she was struggling with acute lymphoblastic leukemia, a disease that, thanks to advances made possible by nih, chemotherapy can cure it 90% of the time. unfortunately, emily was in the other 10%. her prognosis, after failed chemotherapy, was grim. but doctors at children's hospital philadelphia approached her parents about trying something radically different. a clinical trial of an experimental approach called immuno therapy. so i would really like to use this story to make a point

about the long arch of medical research involving many investigators and work leading to emily. let's take a brief journey back in time. the history of cancer immuno therapy can be dated bact to the 1890s. a new york surgeon reported success in treating a few inoperable cancers by stimulating patients' immune systems with back material toxins. but his results were highly variable, the treatment was very toxic, and this largely fell by the wayside until the mid 1980s. then, at the national cancer institute, steve explored the ability of certain immune cells to destroy tumor cells. he wondered why the immune system doesn't recognize cancer cells all the time and eliminate them and whether the immune system could be helped to do this by taking the t-cells out of the body, stimulating them with an

activating factor and then reinfusing them. it did not always work but there were some dramatic responses. steve was and is a true pioneer. and in a wonderful stroke of timing steve was named this morning as the federal employee of the year by the partnership for public service and will be recognized in the same ceremony this evening. meanwhile, the dna revolution was gathering momentum. basic research spear headed again in large part by nih led to methods to splice fragments together giving birth to the whole field of biotechnology. armed with this powerful set of tools and technologies, james alison you see here pioneered one particular form of cancer immuno therapy. he discovered that a particular protein on the surface of the t-cells actually acts as a braking system preventing the

full system. by designing an immuno body to block that, brakes can be released, dramatic responses to previously untreatable cancers began to appear. another award. alison just rereceived america's nobel prize for this work last month. building on this growing momentum, other scientists like lab uhn, and whose chairman blunt has visited have been developing even more precise therapies. t cells are collected from cancer patients and engineered in the lab using dna so that they can produce special proteins on their surface. when those modified cells are infused back foot patients they multiply. and with guidance from their newly engineered receptors they seek and destroy the tumor

cells. let me show you how these seek and destroy cancer cells with a quick video. this is pretty dynamic and the results can be dramatic. so that's a t cell that you see there lit up in red busy migrating around. it's looking for foreign invaders. you can see, when it finds a cancer cell it's going to get really excited. the cancer cells in blue the t-cell going after it. i'm going to change the colors. the t cells are now in green. the cancer cells are red. watch for the red flash. that is where the t cells just ruptured the membrane of the cancer cell and sent it off to the cancer cell graveyard. you can watch this happening repeatedly with different cancer cells being targeted by these t-cells that go after them and figure out how to do away with them. you can see why one of these recent patients refers to those t-cells as little ninja

warriors. they do their job. this isn't just the future of cancer treatment. it's the present. again, note this is built on decades of work. in fact, going back to jim, a recent analso sis shows that the pathway that led to his award include it had contributions of 7,000 scientists over more than a century with many of those scientists pursuing basic questions that had no apparent connection to cancer. so i tell you this story to emphasize critical need for federal investment. if we do that we can realize accelerating discovery across this vast landscape of biomedicine and ultimately save lives. remember emily? here she is today. a junior bridesmade, the picture of health. this happy picture made possible by her parents' decision to go ahead and enroll her in that pioneering cancer immuno therapy trial.

28 days after that treatment emily was cancer free and more than 5 years later she remains cancer free. emily is just one success story. i could tell you many more including all these folks across the entire nih portfolio about how this is leading to a healthier future for americans. from the development of neuro technologies to the brain initiative to the million or more cohorts person in the precision medicine initiative that will generate knowledge applicable to an entire range of health disease. you would say our future has never been brighter. to realize that future nih needs your sustained report. so thank you, mr. chairman. my colleagues and i very much welcome your questions. >> well, thank you. i'm certainly glad you're here. let me ask a couple of questions. i did visit with senator too manyy and i went to see what dr. carl juhn was doing.

that effort which very much -- and you can correct me where i'm wrong here. very much focused on what the individual patients needs, dozens of individual patients at all age groups have been seeing success in that particular effort. but two thoughts about that. one is what -- how does this -- is this likely in cases like this to go beyond treatment to the level of where in this particular case this particular fighting agent is always there so you're talking about cure instead of treatment? and i would be interested what discussion is going on, how we look at the world where cure is one of the options as opposed to a health care world that is largely been defined by treatment up until now. and i was going to ask my second question at the same time, which is on these individual cases, i would

assume at some point one of the challenges are what do we do that makes that most likely to be scaleable? so that every patient doesn't have all of the expense of a unique treatment baw scaleable effort made that will -- i think will come naturally? but talk to me about those two things and whoever you would like to answer those questions. >> those are great questions, mr. chairman. and i think i will turn to dr. loy who is investing in big ways in cancer immuno therapy. >> thank you, senator blunt. this is really a critical juncture right now because we have opportunities for long-term responses. and what you're asking is are a subset of those responses going to lead to cure? and we certainly are optimistic and hopeful that this will happen at least in some cases.

but we need to understand better as you point out what the mechanisms are that drive the important clinical responses to immuno therapy. and if we can understand them better, we may be able to devise even more effective immuno therapies that also will have fewer side effects. and that's an area that we really are actively supporting investigations in because we do hope eventually that we can get to the area of precision and predictive oncology where patients we know what treatment to give to them and we know what kinds of immuno therapy to give to them in addition to targeted treatments. thank you. >> in terms of the scaleability, which is a tough question for some of the very personalized immuno therapy that you saw in carl juhn's

lab, there are strong interests in companies and figuring out how to do this where you can make this available to thousands of patients instead of in small trials. we would think that is a very appropriate kind of place for public-private partnerships to spring up so that this idea of engineering your own t-cells to go after your cancer could be done for more and more individuals. >> i think this is a topic that even i'm having some discussions with people representing health insurance companies. are you thinking about a future where traditional treatment may be less expensive initially but a long-term potential cure, more expensive initially but less expensive over time? what are your standards going to be? are you thinking what we can do now, which really the whole concept i'm sure will get to of designer medicine, what can happen with the efforts on the

brain and how that impacts. and i think we will have time for more than one round of questions. we have a couple of people on a time frame. we will try to get to them quickly and we will do this by order of both appearance and if we're here on time seniority on the committee. but the first person is senator murray. >> thank you. as you know we are working on a continuing resolution until december 11. i wanted to ask you what effect would a year lonk cr have at the current rate, have on the nrn ih research? >> we're thinking and worrying a lot about that. here we are in a circumstance where perhaps emboldened by the enthusiasm we have seen in both the senate and the house in the fy 16 budget process have a number of very exciting initiatives that we would like to launch in fy 2016. the precision medicine

initiative, the brain initiative under way for two years but at a critical point to ramp up and build on what has already been done, the ability to be able to push our vaccine strategies for influenza, for hiv aids. all of those are at a critical point where more investment is needed and we have been heartened greatly by the actions of this committee and in the house ee to combleeve we might have a chance to do those things. a year-long cr would be simply devastating. the precision medicine initiative for instance would basically have to go into the freezer orp moth balls or whatever the appropriate discouraging metaphor would be. we would just be at the point of starting to enroll a million americans in this study and carry out exciting study and those would basically have to go on hold. that would be enormously disappointing. similarly imagine the brain

initiative, it would have to basically take a pause for a year just at the point when the momentum is building. so i can't emphasize enough how much we are worried about this. we can struggle along with the cr until december 11. but if it is a year long cr it is going to be a dark year, indeed a dark year. >> more than 20 million people in the united states have a substance abuse problem. and we know that only a small percent of that possible lation will get help and those looking for treatment often can't find it because of long waiting times for cure or limited in shurens coverage. the work that nida is critical but i worry that cutting funds would make it hard for addicts to get the help they need, especially at a time when 10,000 more americans now die annually from overdoses than

they do in car crashes. the substance abuse block grant that represented 42% of state spending on substance abuse as recently as 2007, that sure would likely drop to 32% under the subcommittee's bill. i wanted to ask you and take advantage of your being here today. are you seeing shortages in treatment services around the country for addict that is want help? and if so does that concern you? >> unfortunately, the answer is yes. and of course it concerns us. because the problem of drug addiction is actually one that has been increasing in our country and we've known all along that only 15% of those addicted have access to treatment. >> 15%? >> 15%. only 15% of the people who ask for help? >> no. 15% of individual that is have addiction to have access. they know all of them search for treatment.

but one of the reasons why they don't search is they are discouraged by the lack of infrastructure to support their needs. as well as the issue of stigma. so those are two aspect that is have made it very, very difficult to provide treatment. what nida is doing is try to take advantage of infrastructure that we have in our country to maximize their involvement in substance abuse disorders. that includes the health, criminal justice systems. those two are structures that we are engaging to provide with evidence-based treatment that can provide outcomes. >> i wanted to ask you, i know the brain initiative recently released its second round of awards bringing nih investments to 2015. cults us about the progress that you've already made under the brain initiative? >> yes. so the brain initiative is incredibly exciting and off to a great start. the sfert of the brain

initiative develop new technologies to allow us to monitor interrogate and also mod late brain circuit activity. and if you think about it that's really what patients are suffering from is disorders in brain circuit tri activity. the problem is that we don't have the neck nolings to monitor or mod late those except in a very unsophisticated manner. so some things have already come out that are really exciting. a couple of examples. there's a new technology which you can put artificial geen into particular nureons in the brain and with a drug that has no other effects you can turn on or turn off precisely certain neuron types in the brain. this is really an amazing fete to be able to do that. so just to contrast that with treatment for parkinson's disease, where a wire is put into is the brain and electric

current is sent in and it goes willy nilly no one knows exactly what it's doing. it turned out to be quite effective. you can imagine how this technology can completely change how we can basically normalize or cause compensation in brain circuits for patients neurologic deficits. so it's really quite exciting. >> thank you. unfortunately, time is the out. i will wait for the second round. >> in my state of mississippi we have one of the highest rates of type two diabetes in the nation. we're told that over 12.5% of our state's adults have the disease and the problem is growing rampantly. are there any new approaches that you have in mind in dealing with hot spots,

outbreaks, whatever you want to call it, in areas like our state? >> that's a great question for dr. rogers since his institutes oversees diabetes research at nih. >> thank you for the question. tibe two diabetes is increasing at an alarming level there. currently about 29 million people in the u.s. with diabetes and 86 million who have pre-diabetes. and there are two things we're doing about that. number one, for people who are established diabetes there's a common drug that started on patients. but unfortunately, in the great majority of patients, that drug will no longer be effective. we started a trial in which we are characaterizing the combination of this with one or four different classes of drugs to see what the next effective drug would be for a given individuals. this is actually a trial that

involves 5 sthourks individuals in 45 centers around the country to determine the effectiveness. this really will eventually get to the area of precision medicine. the second thing for those people who are sort of underneets the iceberg, the 86 million americans who have a possibility of going on to develop diabetes, we have tried to translate a very effective diabetes prevention program to scale this up in a way to offer this lifestyle which was quite effective in these patients to prevent them or delay them from becoming a diabetic. these will have an important financial role in the future in erms of cutting costs. >> thank you, chairman. >> thank you so much for organizing this hearing. to our colleagues from nih, this is a committee and the members here today are the

pragmatists. when you look at it, we have a chairman that's very much dedicated to nih, certainly a vice chairman that is. we have the authorizers here in terms of murray and alexander. all of us here going around have a history of support for this. so you've got people who really want to be nonpartisan. so i want you to know that. but we are sometimied. we are sometimied by our own process. when i first arrived in the senate we had a triad that worked. authorizing. they would often create great policy on a bipartisan basis. kennedy-hatch, kennedy-cass belle, et cetera. we had appropriations that really could move the ball forward. and we had a budget process that gave us an orderly methodology process for doing that. so we've got problems here. so we've got big problems here. and i know that you live them

out every single day. colleagues, when i visit the national institute, which has been my great joy to represent for 28 years, i call it the national institutes of hope. this is what we just heard here. the national institutes of hope in both what they do on the campus in bethesda but also what they do through the great extra nurel research like in the university of maryland and johns hopkins. you're a hopkins guy. they're going to dedicate a room at the hopkins club, the faculty club two weeks from now. 38 people associated with hopkins have won the nobel prize. two thirds of that have been in life science. 38 people, one university. but because of the role that our government plays in doing that. but we're an economic engine. when you think about the jobs that are created because of that, in pharmaceuticals,

biomedicals, medical devices, you are a turbo engine. so rather than seeing a cost factor, we should see you as an economic generator, an economic generator. and i hope we can be able to do that. i am deeply concerned about the caps. i don't like budget caps. but most of all, i don't like the caps on inno vailings. we cannot continue to continue to cap innovation. we cannot cap breakthroughs. we cannot cap the opportunity for young people to train for these careers by dr. rosenberg and all of you have here. so here is my question to you. that when we look at both the research to be done and the workforce that needs to do it, i worry about the young investigator and the debt that they carry that's a deterrent to pursuing this. could you tell me if we could go down the table, if we lifted the caps and went to president

obama's budget -- nothing more. president obama's budget. what would be the three things each and every one of you could do, and how would it also impact young investigators? >> ok, folks. there's the challenge. maybe we should just quickly go own the table. >> if you just have one -- one would be enough. >> but number one is we would accelerate the development of medications for addiction. there are many very potential interesting targets. but pharmaceutical industry is not investing, the responsibility relies on federal government money predominantly through the nih. so that would be one. he second one would be to have , expand the story to study the human brain. that will inform prevention efforts. the third will make research

more accessible to young investigators so we don't lose talent. >> i would just expand upon that. the three areas i would focus on are the young investigators. we know that there are two critical points in which an investigator is likely to stay in research or exit. one is on their first application to get a grant. the second is getting that renewed. and they get that through that second hurdle it's likely that they will be with us a long time. so we would like to encourage them by making incentives for both the first application as well as the first renewal. the other point i would make is with expanded funds we would be able to allow for expansion of some of our existing clinical studies which are very expensive because of the curtail that we would have to do. and one way to amplify the investment in infrastructures in these clinical trials is by using -- by having answer larry

studies of these trials. so i would expand existing trials as well as answer larry studies of these trials. >> the question of young people e at nci are in the process of trying to develop new approaches to enhance their ability to move from being graduate students and post doctoral fellows to starting their own laboratories. the areas where we would invest would be in cancer prevention, cancer screening, and cancer treatment using molecular precision medicine approaches, which have enormous potential in those areas. and i would highlight the potential of immuno therapy as was discussed earlier because of the issue of its potential for improved responses, decreased side effects, and scaleability, as senator blunt

mentioned. >> in terms of research projects, we talked about the brain initiative. it's also the national action plan for alzheimer's' disease research. and all of those have milestones. it's all planned out. if we had funding we could really accelerate both of those major projects. in terms of the young people, i think it is incredibly important. the average age of becoming independent with an nih grant is now gunning towards the mid 40's. we have to move that down. i think we have a couple of thing that is we are playing with to just take a shot at somebody who is really young who really looks bright and just give them a chance much, much earlier in their career. >> so my institute mostly funds fundamental basic research. and the great ideas that lead to the discoveries that dr. collins told you about and basic research don't come from me. as much as my esteemed colleagues, don't come from them. they come from the great

brilliant minds in your districts across the country. so we would focus on supporting investigator research to promote these brilliant scientists to do their work. as a measure of that and the success of that my institute has funded a number of nobel prizes. i would said 81 yesterday but as of this morning it's 83. i think that's an indication of the power of investigative research. we would certainly have a focus on promoting the careers of the young scientists who will win the nobel prizes of the future. >> i will point out that only senator middle class ski gets 50% more time than she is allocated. that is totally fine with me. one of the things i did want to do today is get on the records the kinds of things you would do now. strictly speaking to senator my cullski's question senator asked for the increase.

but i'm going to look very carefully that you said you would do if you had the president's number and assume i could multily that by two. that you get at nih. >> thank you. dr. collins, picking up on what senator blunt says talking about money, funding, is important. say you had $3 billion more above. what could you do with it as far as investigating and hoping to turn a lot of -- you're just investigating results into better health and better treatment? what would $3 billion -- use that as -- i made it up. but i hope we could do something for you. what would it do for you? what would it do for us. >> for america, for the world. i appreciate the question.

it's a lovely thing to contemplate. because as you've heard we have lost over the last 12 years about 22% of our purchasing power. this would be about a 10% increase. it wouldn't get us back to 2003 but it would be an enormous shot in the arm to a community that has such talent and such energy and is basically being squeezed at the point where a lot of innovation is just not happening. you heard from my colleagues the areas that they would pick and i'm sitting here thinking about the other institute directors, i will mention a couple others that i would want to put on that short list. vaccines. i mean, we are on the brink of being able to develop a vaccine that would work against all influenza strains. if dr. fauci was here he would tell you about that. we have a path towards something that would be able to then result in not needing your yearly flu shot that has to be reengineered and sometimes it works sometimes it doesn't. but more importantly,

protecting against that worldwide pandemic which is overdue. we aren't pushing that because the resources aren't there. we do see a path to make that happen after 30 very frustrating years but yet we can't do on hiv aides. i think has a lot of bipartisan support which the scientific community after many workshops and working group that debated about this is very jazzed about -- we can't start that if we have a year lonk cr. in answer to senator murrifment but we could start it and ramp it up much faster if we have this kind of a curve to work with as far as research. and then there's this whole area that we call high risk, high reward research. we just announced a couple days ago the funding that 78 of these new awards, these are pioneer awards, new innovators, new awards transformative

awards. these are ones you can't apply unless u you have an idea out of the box. but if your idea is exciting we want to see what you can do. give the person the money and let them run with it. many of the institutes are taking that path. but we could go faster and inspire people to be more risk taking if we had that kind of opportunity. put all that together and with $3 billion, let's try it. let's try the experiment and see how that turns out. i ploms you, it would be amazing. >> not only health. it would be a good economic investment. >> you repeat an economic analysis demonstrates the return is about 2.2 fold in the first year to the local community and of course our dollars go out to all 50 states. >> i would like to touch on quickly, where are we today as far as cutting-edge research on sistic fibrosis and also a lot of the auto immune disease such

as lupus? >> thank you again. great question. and great progress being made. i have a personal deep and longstanding interest as my own laboratory found the cause of that back in 1989 when i was at the university of michigan. and this is a very exciting time for that disorder. because after all those years figuring out how that small glitch in the genome was capable of causing this disease, we now know a great deal about the protein that's normally made and why it doesn't do what it's supposed to. and just in the last few months we have now seen the second drug strategy approved by the f.d.a. for the treatment of more than half of those. based upon a small mol cue, a drug that is based upon molecular understanding of the disease. very exciting times indeed. lupus, rheumatoid arthritis, i want to mention one partnership, the accelerating medicine's partnership which

several have been working to bring together. one of the targets is in fact lupus and rheumatoid arthritis trying to figure out again i showed that picture about t cells. are they overactive? are they going after normal tissues when they shouldn't? what could we do with the new technologies to understand how single cells are behaving in order to come up with strategies? so all of these areas are just full of potential. >> thank you. >> thanks, mr. chairman. let me just say at the outset to address this side of the room for a moment. gathered in this room at this moment in the united states senate are the people, 11 or 12 people who could literally make a difference for generations in medical research. milk noted by senator ski we have the committee in the senate. if we took a bipartisan stand

and said come hell or high water we are not going to tolerate a shutdown, we are going to increase the funding for nih and related medical research agencies, we can make a difference. we can just make it clear. don't try to get through the senate because you're going to touch it. and i want to commend the chairman because i bothered, begged, challenged him for a long long time based on dr. collins' admobition to me. give me 5% real growth for 10 years and i will light up the scoreboard. and we've done it in this bill. i might add partner thetically at the expense of some other things. that are equally or i should say important as well. but i want to commend the chairman for making this commitment for 7% growth at nih which includes 5% real growth at nih. as you said, and was asked by senator shelby, one time infusion is a good thing.

but constancey, predictability, is what leads to researcher commitments and long-term success in what we achieve. i would like to just throw out this possibility that we rally around one particular person who is up here. for 28 years barbara has been the strongest voice for the national institutes of health on capitol hill. and she is leaving soon. unfortunately for all of us. but i hope we can make a promise that we are not going to forget the commitment of this budget and the commitment of years to come. and in the time i've been in the senate you don't want to break a promise to barbara. something you will hear about. >> well said. >> so i hope we can be inspired by that. let me try to bring this down to ground level, if i can. i have two questions if i can get to them. the first is when we talk about $2 billion in growth, $2 billion in growth, in this

coming year, i need to ask you, when it comes to areas like alzheimer's', we node that we pent $226 billion on alzheimer's' treatment just medicare and medicaid. we estimate that the private contribution of families is almost equal to that in value. so we are talking about one half of 1% of what we are spending as a nation on alzheimer's' is the delta, the $2 billion that we are looking for here. and when it comes to brain research, we now have reached i think a point -- and please confirm if i'm right -- where we can start to visualize the development of alzheimer's' in the brain and know many years before the obvious onset that a person is moving in that direction. what do you see, dr. collins, or those who are with you, in terms of what we could do if we knew 15 years in advance that alzheimer's' was likely to

occur? what could we look forward to do soon to delay it or, god willing, find a cure? >> so i'm just showing you a picture that outlines the statistics. i didn't know you were going to ask the question. you can see what the relative numbers here in terms of what's currently being spent on alzheimer's' disease. this is 2013 numbers. obviously that number is going up. and we need it to. but comparing that to what we're spending, $203 billion in 13 and an estimated $1.2 trillion if nothing gets done. this is a matter of great urgency not just a matter of economics of course but the enormous human tragedies. as a neurologist, can tell you something about where we are on this and why we are optimistic that we can actually get to a place that doesn't result in that enormous blue arrow. >> thanks, senator. i think you made a really interesting point that for the

first time we can actually see what is going on in the brain and people with alzheimer's' disease. so in the past, we knew what happened when people died but we couldn't see that happening in the living people. but now we have markers scanning markers for anlloyd and tal, which are the major cullprits. we can see that in the brain now of living people. as you said, the anlloyd we can see developing years before that starts to hit in and that seems to be the thing that kills the cells. so alzheimer's' is like the gun, tal is like the bullet. so the vision is that we develop a screening tool for people who are developing the anlloyd know that they are going to develop alzheimer'ses and then come in with a drug to block that process. in actual fact those are being testd in clinical trials. so we could get lucky. this looks very, very promising at this point in time. >> let me add, because

secretary moniz would hope that i would add that this technology which allows us to visualize our department of energy and office of science had a lot to do with it. so looking at medical research the technology side of this equation looks like other agencies. do i have ten seconds? darn it. i will try to be here for the second round. >> i would like to quickly say that is a very good point and we have a joint meeting about the brain between the department of energy and nih coming up in two weeks in chicago. >> senator alexander. >> thanks, mr. chairman. i'm sorry barbara left because i would like for her to know that dr. collins played the guitar and sang at a place senator durbin has been and barbara likes to go, blue bird cafe in nashville. it was quite a show. what is that song, knockout disease? >> that was it. i'm surprised you remember. >> it's a great hit.

i want to thank -- we all admir mire your work but we admire the work of your team. we know they could be making more money some other place. but the fact that they are here and working to help other people is something we all respect and appreciate. i would ask you, dr. collins, earlier, for the bill that senator murray mentioned. she and i are working on. we are trying to create environments. precision medicine can succeed where we get discoveries through the process more rapidly. one of the problems we have is that the national academies groups have identified that investigators these ones that are wanting to get more money for, spend 42% of their time on administrative tasks. now, if we're talking about millions more for investigators, shouldn't with be spending an equal amount of time getting that 42% down so we create more dollars there? there's a new report headed by the former chancellor of the

university of texas which makes a number of serious specific recommendations about how to deal with that. one includes a research board that would coordinate an approach toward the spending of the $42 billion we put out. not all that through nih. the colleges and universities. to try to eliminate duplication to make it more efficient. my question is, would you -- a lot of their recommendations have to do with nih. would you review that report and over the next year set up a systemic way to consider making the changes that it makes? and if you have impediments either within the administration or the law that would keep you from doing that, if you let us know we might be able to include them in the legislation senator murray and i are working on. specifically the research policy board. >> senator, i appreciate very much the role you played in bringing this important issue to the attention of the

academic community and other constituents as well as including the government. >> thank you. i'm going to ask you to leave me about two minutes because i have another question i want to ask. >> we will take with great seriousness this report. we've looked at it in a preliminary way. we will look at it much more deeply. i think we do have a number of ideas and responses i will be glad to share about how to reduce that 42%. >> i want to ask you questionings about funding but i don't need the answers today but all of us need the answers in the next few weeks. the house included something called mandatory funding as well as the discretionary funding. for many of us mandatory funding is the villain. the reason you don't have mun is because that part of the budget has gone up like this and the discretionary side is like this and you're in the discretionary side. so our visceral reaction is against new mandatory funding. but i'm convinced that this is a critical time in science and a critical time of opportunity. so i'm willing to think about that. and i have these questions as i

think about that. and these are the questions i would like to talk with you about sometime. what happens at tend of the 5 years that the house proposed? there's a cliff and you lose $2 billion. what happens then? what's the purpose of the mandatory funding? if there's a difference between discretionary funding or mandatory funding you just mix it all up or is there some distinct purpose that would justify a steadier stream of money toward mandatory funding? and what would that be? should there be a focus for the mandatory funding on preventive medicine, for example, or on precision medicine, for example, or on investigators or -- for example? and what about oversight? we had an embarrassing thing happen at the nih about its manufacturing of sterile drugs recently.

if you're not accountable to us for what happens there, then you're not accountable to anybody, really. and that's our job as appropriators. so as a republican, when i read ben bernanke's column that says that the fed can't create a growth economy, it takes education, capital formation, infrastructure, and research and technology, i agree with that. i'm all for more research. i think we should be doubling energy research rather than subsidizing wind mills and putting money in the pockets of rich investors somewhere for after 22 or 23 years. i think we should be setting priorities. my priorities very well do include your work and dr. moniz's work. but i would like you to think about the questions i ask about that type of funding and maybe one of the days we will have a chance that discuss it. >> thank you. >> senator mercury. >> thank you very much. and thank you for your team for the vision the video of the t

destroying the t cells is inspiring. we hope that over the years ahead every possible type of receptor on every possible type of cancer cell, the ability to program t cells to attack them will continue to develop. i think that's the vision that we're anticipating. one of the concerns that i've heard often -- and oregon health sciences university is a major research partner with nih. a lot of grant funding goes there. is the stranding of young researchers. i believe dr. rogers mentioned the young investigators, folks who were partway into their career. they've gone through their post graduate work, they're in a laboratory. and then the grants don't come through. and then they have this

incredible specialty about some form of nerve communication or chemistry deep within the cell that may be the key but who knows. but about suddenly they're going, what do i do now? does this continue to be a problem? and to what degree should we be deeply concerned about the loss of this? we expend a huge amount of resources to develop that talent and then suddenly its ability to be applied is cut short. >> we should be deeply concerned. we do put a great deal of resources into the training of this generation of young scientists. the talents and the skills that they possess are incredibly possessive. and yet if you, as i often do, go and visit universities across this country and meet with graduate students, post doctoral fellows, it used to be when i made those visits they want to tell me about the science they're doing. now they want to tell me about their anxiety about whether

there's a career path for them or not or whether they ought to be doing something else. and some of them have decided to do other things or have gone to other countries where biomedical research continues to grow. every institute at nih thinks about this worries about this and we sit around and try to figure out strategies. r. lors' institute is deeply involved. although i will tell you, there's no magic here without seeing some relief from the budget squeeze in terms of what we can do. we can try to make every dollar count. >> thank you. i will ask you to be very brief. >> sure. we are starting a new pilot program to explore a new grant mechanism that would be a single grant per researchers. i would wo address some of senator alexander's issues. but it would be more stable because it would be a single grant. that would, as dr. rogers alluded to, help carry them through that sort of valley of

death after that first grant when it's very hard. that's something we're focusing on. >> i want to go back to where i started in terms of the receptors and the t-cells. early in the zna world that you were centrally positioned in, it took a long time. curve ine a similar terms of the time and effort to identify key receptors and be able to read -- to identify t cells? >> you can now get yours sequenced in a day or less. proteins,f looking at they are encoded by jean so we have a connection there were we can take full advantage of what we have learned through the last several decades.

we have a good senses of what are the proteins on the surface of various cells including cancer. the trick is that every cancer is slightly different and that's where the precision medicine fits in.i think that's at the cutting-edge of trying to bring andnology and genomics cancer biology together is to figure out how to make that strategy work not in a one-size-fits-all because it won't work that way but in a precision individualized way. >> shifting topics -- -cigarettes -- we have seen a tremendous growth and there have been studies at nih about high school students tripling their use in a single year and so forth. what do you see as the role of nih in terms of this new form of tobacco and tobacco addiction? >> thank you, senator. the nih is concerned about tobacco consumption because it

has such an impact on disease. in addition, the issue of the e-cigarettes where we do not know with the short-term or long-term impact is from the cigarettes themselves nor do we know what the implications are for behavior. therefore, the nih in conjunction with the fda is conducting research to investigate these critically important areas. >> thank you very much. >> senator cassidy. >> as a practicing physician, sometimes i am so aware of your good work and i think we should double your budget. i understand the impact it would have upon my patients. when i did my residency in 1983 los angeles, i am very aware of the hiv epidemic.

dr. collins, i have been concerned that 20 years ago it was suggested that nih rebalance its hiv spending from the 10% it had become to diseases like alzheimer's/dementia which are more important now. if you receive the 7% increase the chair and ranking member aspire to, will 10% of that budget continue to go to hiv research? and i have you discussed this on occasion and i think you raise a good point about whether it makes sense to have a formula driven way in which we define how resources we to be spent or should focus that entirely on what the public health needs are in the scientific opportunities, the things that nih usually does. i do not think that if we had the good fortune to receive this kind of increase that there should be a lockstep 10% formula driven based upon which we would define the hiv-aids research budget. i think we should not take our

foot off the accelerator at a for when hiv-aids is poised some major advances including the potential development of a vaccine. i think we should step away from the formula. , i don'tr directive have it in front of me, but you mention among the focal points in terms of the hiv research would be an emphasis c ono-abilities. one way for your about young researchers not having dollars, this is a concern for we pull the minutes from the 2013 national institute of heart, lung, blood and they speak about how the success rate of application, non-aids applications are 10% but for aids applications, 42% meaning that it took a less quality project to be approved and they hope to encourage more submissions of a project -- of aids projects. then i see that the project currently being done is looking orbilities.

that thee found out 610,000 people die every year from heart disease, only roughly them die fromf aids. we spend 21% of their budget. we are spending 21% of a budget of those who die from hiv. if we are going to focus co-morbidities, spending 21% of , it seems like% we're going in the wrong direction. what are your thoughts? >> i am not totally familiar with the numbers but i will look at those. we are in the process of trying

to right size the way in which our hiv research budget is being allocated. the office of aids research has the potential to move dollars around between institutes and between programs. >> can we move it out of hiv? is $1is a study here that menion to have chinese having sex with other chinese men in china. it would have been great to put that to alzheimer's in oregon. one of your predecessors said we are not the industry -- we're not the international institute of health, we are the national institute of health. why are we spending this on china? 4> we have identified the areas of high priority and i don't think that study would fit those rarities. oppositentioned the aids research moving dollars between institutes but if this if thekind of study --

national heart lung is 42% approval rate for the hiv-aids study, frankly, they are getting too much money for hiv-aids. toy have to find people apply for something that is 21% of the budget. can we move money out of that area into other diseases like alzheimer's, parkinson's, als? we should be making decisions across nih on the basis of public health needs and signs of priorities. i would say there are scientific priorities emerging and hiv i would not want to see neglected particularly the opportunity to end this epidemic and the investment in the vaccine which is likely to be quite expensive. taking your point, i don't think in the process of rethinking this portfolio which we do actively now, we should not neglect the potential of investing in different ways in hiv-aids that will bring an end to this epidemic. >> there is something else i read -- i have read so much

about this. if you decide to focus exclusively on the cure one youase, and inevitably, ignore other more pressing needs for it where hundred million dollars right now on aids vaccines domestically. i think $24 million on the international aids vaccine initiative. not that we cannot spend more but to justify 10% of the budget based on that looks like it will neglect other diseases. record, dr.the collins, you may have mentioned it but i don't know if i heard target areas in hiv-aids, when did you announce that? that was a recent announcement? >> it was in august. i can quickly say those 4 priorities -- we see the incidence of hiv-aids and research toward a cure who are

under lifelong treatment. it's a next generation of therapies with fewer side effects and these hiv-associated co-morbidities with thousands of people infected or having some of those co-morbidities and we need to understand the better. >> i thank all of you on the panel. i would like to thank dr. lorsch for spending time in west virginia at west virginia university, talking about a program i learned so much about, the idea program just -- which is a smart and successful program. i thank you for that. there is research with stroke and brain and a collaborative effort with other universities like marshall emma west liberty and wheeling jesuit. i thank you for that. if you had any takeaways in the visit there, you might be able to address that. >> thank you again, it was a fantastic visit that energized

my staff and myself. one thing we notice about the idea program is the best practices. there.2 the first was sharing of resources to create economies of scale particularly in access to technologies which we so was very critical especially to the young researchers. i think that model of creating economies of scale through resources isology something we should move on nationally because it can get the taxpayers more for their money on research for the other is training young investigators. the centers of biomedical research trains people and there was a paper published that investigators who participated in the centers were three times more likely to succeed than investigators who did not participate in the cobry centers. it's a maximum in terms of

getting our one grant and publishing papers. given the importance of young investigators, taking that model on the idea program and thinking about how we can use that nationally is really important. i give senator cochran a lot of credit for developing the idea program in the first place so thank you again. >> i think the enthusiasm we saw with the young investigators and young researchers is something that was very inspiring for me. about therd a lot problems of them moving to the next step so hopefully we can determine that. z, the national institutes of aging is partnered with the civic centers for disease control and prevention and community living an initiative to create more older americans into research programs and the program has a focus in alzheimer's patients. both my parents really passed away from alzheimer's. can you talk to someone like me who is 60 years old how you get

into these programs and how expansive they are and what are your expectations? i went to an alzheimer's meeting just the other day and they were talking about the push for whereity in your research you are researching minorities and other ethnic groups, women, itselfause it manifests differently in different types of groups. >> they are working hard on the alzheimer project. as you mentioned, one of the stumbling blocks is the culture of research in this country. as we develop new therapies, our barrier is really the number of people we can enroll into the studies. the national action plan for

alzheimer's has a number of milestones which are trying to expand how that happens. percentage, a large of people with cancer will and roll into trial but the incidence of alzheimer's is lower. we have some good plans for that. >> i would like to help you with that because it goes undiagnosed while they are getting old and that's just the way it is. you excited to hear about talking about the possibility of a vaccination. volkov, i'm from appalachia and we have a high incidence of prescription drug on anand now heroin --ronomical rise, overdoses and deaths resulting from the use of heroin i'm glad to see in the huffington post

which i don't normally read to embrace the concept of addiction is a -- as a chronic disease. we are all with you there. i would like to ask what you are doing in terms of rural america which is suffering from this. some of the smaller states and lower socioeconomic strata's are going toward heroin. where do you see your role here? >> the urgency of what's going region isappalachian made this one of our priorities. it's also one of the priorities for hhs. we have been working with the sister and brother agencies to toegrate the projects increase success. has better prescription practices for the proper management of pain.

they are very invested in developing treatments for the management of pain. we are restricted by what we currently have with results on the reliance of certain drugs. item number two, access to a medication that -- >> that was just legalized in our state. >> we are partnering with pharmaceutical companies for different ways of administering these. anyone can administer them. the third one is to play medication that have been shown to prevent overdoses and hiv infections. we are developing medications that can increase compliance. we want to partner with cdc in order to develop a project that can target the appalachian region. by how minimal the structure was in those towns.

we have tools and we have to deploy them. >> thank you very much. >> senator moran. very much, dr. collins and crew, welcome and thank you for the opportunity to have a conversation today. dr. collins, nih recently released its professional judgment for alzheimer's. correct in assuming the president's budget request was the startingpoint that you were going to build upon that request? >> that is true, we were building for fy 17, what the budget would look like assuming the president's budget was the fy 60 number. want to give- i you the chance to respond to the president's request. would it give us a greater thertunity to advance

research necessary to address the issues of alzheimer's question mark >> it would because some of the things we imagined we would fund and fy 17 could be started earlier in 16 so we would want to revise the number for the fy 17 professional judgment that it on that basis. subcommittee and the inclusion of a $2 billion increase at nih plus specific issues related to brain in alzheimer's -- brain and alzheimer's would have a significant consequence to advance the cause of treatment and cure? >> i think we are not limited at the present time by ideas or talent. we are limited by resources. if it were possible to have more resources in 16, we can start project that otherwise would have to wait longer. yes, we could go faster. >> of course, i think you are a bright and intelligent person but i have discover that you also have the ability to say the same thing more than once. [laughter]

perhaps you should be a senator. [laughter] >> for the alzheimer's plan and for the brain as well, we have serial projects and one depends on the other. we don't know what fy 16 will do that we are ready to go. butave announcements ready we will not be able to fund them unless additional money comes or puts fingers off. . >> view our prepared to spend the dollars included in the senate appropriations committee recommendations, our appropriation bill? >> we are shovel ready. >> good to hear. the disease research summit occurred last february. nih is poised to revive the research milestones it created in that national plan for it when can we expect that? is actuallynal plan

a community plan that was developed with consultation from the scientific community and the advocate community and the caregiver community. we are holding annual revisits to the plan. it is revise an annual timeline. we alternate between nids which diseasesmentia related , parkinson's disease which causes dementia with the aging institute which runs the pure alzheimer's plan. every year, we're alternating between those 2 items and redoing the plan. >> thank you very much. in the budget hearing back in april, you and i had a conversation in which you testified that to achieve our mission, we must serve as stewards with the resources we have been given by the merck and

public. -- by the american public. overarchingoping an nih strategic plan and will link this with individual institute strategic plans that reflect the rapid, current progress in bioscience. my question is -- what are the details? about what spaces has transpired since that conversation? what are you doing that is new and will mean that we are going forward and we have the latest opportunities because of that efficiency to achieve more? >> we are working very hard on developing the strategic plan you mentioned. we are scheduled to the congress in mid december. it tries to lay out any more clear fashion across all of nih how it is we set priorities and make decisions about where the dollars are most efficiently

spent and how we are being good stewards in terms of how the process of peer review and counsel review and director actions on what we fund is carried out as well as the number of other deficiencies we are concerned about including with senator alexander discussed earlier in terms of the burden that is applied to investigators trying to get the research done as well as human subject oversight. there will be a lot in this document and it will layout in greater detail that has been possible before how we intend to use all the dollars we have to the best benefit. >> mr. chairman and dr. collins, let me repeat myself -- admonitionred this if that's a safe thing to say -- that we are often told as members of congress that we don't want you meddling in the politics of deciding where research dollars should be spent. but itshared that view means it's incumbent upon nih to make the decisions that are necessary as to where the

dollars spent are the most ,ikely to achieve the quickest fastest, the best, the necessary results. >> i welcome that responsibility as do all my colleagues. youpologize for offending by suggesting you can serve the united states senate. [laughter] was a time when that might be considered a compliment but not now. we will start with senator murray. to the 29to go back million americans who have diabetes and 86 million who are prediabetic. that sounds like we have a crisis on our hands. the number of americans with that disease continues to grow. imagine the work the institute has done on prevention programs that incorporate regular exercise and reduce fat intake and a huge difference it makes. the cdc chronic disease program which our bill has cut helped fund programs like the one in my

home state that supported community health efforts through the ymca and many other organizations that promote healthier living. what might taking that preventive program to scale mean for this country's diabetic epidemic? tothe ymca you are referring and their ability to develop what we did in the clinical which is a lifestyle intervention on an individual basis, they took it to scale by doing the same lifestyle intervention but providing it in group a group setting. their results after the first year or two are quite similar to what we achieved in the individual program in terms of weight loss, etc. cost perrestingly, the patient involved in the clinical trial with the initial

instructions and the follow-up was about $6,000 per patient. at thegroup setting ymca, that cost was cut down to about $400. in terms of scaling this, we could expand this. we have not done economic analysis on this but there is a private group called the urban institute that has recently looked at what happens if you scale this. given those numbers, they estimate $191 billion over 10 with if this -- conservative assessments or assumptions if this could go to scale. >> that's impressive. as dr. collins mentioned, we have seen significant progress in recent years in the use of immunotherapy to treat certain forms of melanoma and lymphoma and lung cancer. i believe there is universal support on the subcommittee for efforts to find similar

breakthroughs for other cancers. to make theseing happen? we are investing in a number of different areas throughout the cancer spectrum. i think there are opportunities in many different areas. for example, we are investing heavily in pancreatic cancer because this is a cancer where we have not had significant progress despite long-term recognition of how serious this cancer is. we also are investing research in pediatric interventions. research nci supported developing 2 interventions against pediatric leukemia and lymphoma.

this was initially developed in the academic sector and has been picked up by venture capital and is rapidly going forward for clinical trials. interacting with the pharmaceutical industry to try to identify new and important uses for drugs that are havehe-shelf because they been approved for one intervention but not for another. inhibitors and melanoma, trying them and other diseases. this is potentially a very important innovation because we recognize that a percentage of patients who have different kinds of cancers may have the same molecular abnormality and therefore may benefit from targeted treatments initially developed in other areas. i could go on but i think given the time -- me whyyou tell

immunotherapy is effective in some patients but not in others who have the same cancer? >> yes, i think this is a critically important issue. doing to support research to understand mechanisms are critically important because if we could understand why some patients are benefiting whereas others are to, it should enable us whichse understanding should lead to better interventions for the people who or,ently are not responding at the very least, we would not be giving them treatment for which they are not going to benefit. >> it's a question we need to answer with more research? >> yes. >> thank you very much. >> i will go last in this second round. >> i will be as fast as i can. collins, there has been wherel articles regarding

biomedical studies results including those funded by nih that appear in top peer-reviewed journals cannot be replicated or reproduced. ae art -- one article cited haltedescribing how it 64% of its early drug target projects because in house experiments failed. you have done a study on this. what is the problem here? why cannot they replicate? to prince too fast? >> senator, it was you who brought this issue to attention in a hearing like this about three years ago. it was just beginning to appear and i appreciate very much your having shined a light on the

situation we are taking with great seriousness. this is a complicated multifactor situation. i am showing up on the screen what nih's -- has now posted as for is a summary of all the things we are engaged in to try to address this and to do things to improve the training of the next generation of scientists about these issues in terms of rigor and reproducibility. involved,tors certainly, the hyper competitive atmosphere that currently exist, much of it on the basis of the fact that funding is so tight, causes people to try to get publications out as quickly as possible3+ fact result in circumstances where the replication study did not quite get done and therefore some deals find out later that it would not have worked. we have an issue in terms of journals. in many instances, not being thorough in evaluating manuscripts. through the leadership of dr. aybac he convened the

thisals tok get together on and they want to make sure the statistical methods are described and so on. it clearly is something that touches many areas of science. we are looking at projects on cell lines because sometimes people publish papers about work and i cell line and it turns out it's not what they thought it was. these things get passed around and training is critical. we have training videos on this site if you want to see what we are asking mentors to use in lab meetings another group meetings to try to bring to the attention of trainees these critical issues about studies and how you set up an experiment where you know you have done it rigorously. where all over this and we are pushing pretty hard to see this problem addressed. it will always be the case that science gives you results that later on you cannot make sense out of.

if that happens, one it to happen in a way that was unavoidable, not because people were cutting corners. i think we've got the attention of the community now to this and you will see the problem get less than it had been, i hope much less. >> this is very important because it goes right to the essence of investigation and replicating what you have and what we benefit from, is that correct? have looked at this at our institute and there is another side to this. not that you cannot reproduce them. we have interesting technologies and when you try to reproduce them you cannot do it but the people who develop the technologies open up their labs so people can go in and learn how to do it and then it works. >> so some of them were on the right road but did not have the means to finish it? >> that's right.

>> thank you, mr. chairman. >> let's go to senator cassidy and then senator moran. >> i misspoke last time, safety $6 million is not 21% of the national heart, lung, and blood institute. hivt 1800 people died of and cardiovascular disease. that theard people say basic science of addiction and mental health is a barren field. i'm not a mental health provincial -- professional. do you feel -- if you had significant more resources, would you be able to the clinical signs to mix mix -- significant advances? >> definitely we could accelerate less of the discoveries. i apologize but i'm going to

disagree. i would not say that the research on mental health has been barren. over the past 10 years or 15 understandingded about what are the abnormalities in the brain of people who suffer from mental illness. >> i accept that. you would say there is great academic progress? >> that has enabled us to identify potential targets for treatment. the issue on alzheimer's/dementia and als and parkinson's, people say that the promise is not there as it may be in other fields. i have parents with dementias/alzheimer's and i want to give you a lot of money. i hope to convince my colleagues have more swag been made to do so as well. would that be a worthy

investment? could you do the basic clinical research? the neurodegeneration field in general is bringing in lots of smart people to solve these problems. we have the workforce. with the resources, we can .eally make hay there are a couple of things that are tantalizing. all of these degenerative diseases have one common feature. the cells that guy have proteins that aggregate and get stuck in those cells. the ideae really on that maybe there is a unified theory and if we can stop this process for one disease, we can stop it for all of them. it is a leap of faith right now but there is evidence that this is not impossible and we can make a big breakthrough. >> dr. collins? basically, you're never quite

sure where the brakes will come from and we have to be careful not to overly target research a specific direction of a specific disease because the answer might come out of a different investigation or from basic science. the biggestple -- breakthrough i have heard about my last month for als -- >> i've got one minute, 45 seconds. you have a bunch of bright, aggressive people. they would not be who they are were it not for you being at the pinnacle. you are a pinnacle of the doctors. you mentioned earlier the academic promise, the research promise of hiv aids as a rationale to continue their. re. is it lacking elsewhere? one of the reasons to continue the funding in one area as

opposed to others is the apparent academic prize -- promise in the one as opposed to the other. how do you allen's these folks who have such promise in their fields as you make that decision? you balance these folks who have such promise in their fields as you make that decision? >> there are areas up and coming in others that are perhaps not moving his rapidly and we adjust the decision-making but also not be overly top-down making decisions. a lot of the great ideas come from our wonderful scientific community out there we cannot anticipate where they are going. it's a constant revision date by day, week by week, where we want to put the emphasis. my wish were fulfilled and dollars were redirected from hiv aids into into some of these other areas, it sounds like there is fertile field that the andy would indeed fertilize so with great benefits. >> there are fertile fields all

across our landscape. >> i yield back. >> senator moran. >> the most significant development in the last 30 days is --? >> ok, thank you. [laughter] it fits with the conversation we're having. it was an investigator who is studying hiv-aids and trying to understand one of the co- morbidities which resembles als in a modest way. it's an intramural investigator and he discover there's an activation of a retrovirus that we all carry. it starts making copies of itself and causes this damage to the neurologic system particularly those anterior cells. theooks like als and publication was just cannot suggested this might be one of those missing clues. not for people who have hiv but --e similar symptoms to als

i don't know where that's going to go but it was an interesting example. you study one disease and you learn more about another one and you don't expect that to happen. >> dr. lowery, welcome to this panel. i want to give you the opportunity to express as the relatively new acting director your vision for the national cancer institute. i want to highlight a program that is being launched in pediatric clinical trials call pediatric match trials. could you tell us about that? isthe pediatric match trial a child that is currently under development. what it does is essentially does for pediatric cancer research what the adult match trial that started two months ago is doing

for adults who have advanced cancer for which there is no standard treatment. it puts the molecular abnormality of the patient front and center rather than the origin in the body of where it occurs. drugs that are off-the-shelf either experimental drugs or have been approved for other uses and it tests them in these other ways for cancers where they are not yet approved. the goal is to improve the outlook for these patients. this is a trial that is under development. uses for the precision medicine initiative that people have been talking about.

the overall vision for nci is to research as we have done historically to invest in precision medicine, not just in the areas of cancer treatment as is occurring with the precision medicine initiative and the oncology portion but also to emphasize precision medicine in the area of cancer ,revention and cancer screening understanding better the causes of cancer and understanding how cancer comes about. in addition, to put a focus on health disparities in cancer. unfortunately, there are many wererent kinds of cancer certain underrepresented minorities have much higher incidence of mortality. we need to treat these populations as we would any high risk population to understand the biology, the lifestyle

oftors, and the utilization medical utility. in addition, to try to mitigate these factors just as we do for any high-risk population. these are some of the important areas that we are looking forward to making progress in. >> thank you very much, i wish you well. thank you very much for this hearing. you, when senator moran was the ranking member on this committee, they started an effort with the defense advanced research project on high risk i think in fy 2014. i'm not sure whether we develop the same kind of category with nih yet. you mentioned high risk, high reward couple of times. give a couple of examples of things that worked out or things

tot did not that you want see as examples, the kinds of things we should think about when we think about going to a high risk area as opposed to something that is more likely to produce results but not nearly as big. >> recent developments in gene editing are an example of something that was an out-of-the-box idea. could we use the bacterial system that allows rearrangements of genes and use it to edit jeans and a mammalian cell to possibly repair those genes? that's something that has recently worked and has taken revolution izing medicine and a variety of ways. that's a great example of that. >> i might mention the common fund that nih with the congress made into a permanent part of 2006 is a back in place that specifically aims to try to support these high risk,

high report projects that no single institute would probably be able to invest in but collectively we can. micro-biome, the microbes that live within us, how do they play role in our health? that has been a revolutionary set of insights coming about because of new technologies that allow us to find out what's there and how it changes over time. that was one of the high risk -high reward projects that has now changed the landscape of how all the institutes are doing research. you don't want to think of a human is an organism, you want to think of us as super organisms. it's us and the microbes in the interaction between the two that makes a big difference. another example which is closer to clinical medicine is to try to come up with a standardized, reliable way of patients reporting outcomes from their perspectives. so much of clinical research is the researchers and the doctors

saying this is what we think happened to this patient who was given this treatment. you want to hear what the patient thought, too, and it's not quite the same thing. a program called promise which many people thought would be hard has actually transformed that process. it makes it possible now for us to run more clinical trials where the patients are not really patients, they are partners, full participants. their input is guiding our decision making about what works and what doesn't. >> i think washington university is doing some of microbial work? are, they are one of the main leaders in the world. the microbial structure is changing all the time and have you impacted that? >> that's right, as we announce this decision initiative and following one million americans to know what's happening to -biome as a

consequence of diet and exercise and not taking antibiotics, we could find this out a scale not previously imaginable once we get this up and going. >> senator murray mentioned we the block grant fund, in fact, we cut lots of programs in the budget to reorient toward other priorities. i think we eliminated totally the funding for 43 programs. spending $1.25e billion which had very good titles. there is not a single title that was not meritorious. but the process of prioritizing is exactly that. if all yourioritize do is get more money and spend it on something more important than you thought you were spending on last year. that is not really prioritizing, this is adding more money for good things. what brought me to that when i

thought about cutting those block grants by 3% but the majority of that money come in my view, when into increasing the money to combat opioid abuse. i have full of years ago, i had not heard of it at all. in committee hearings this year, we are your about all the time -- we hear about all the time. do you want to talk about opioid abuse and what research may be going on to come up with pain medicines that are less easily tosed, less easily converted other drugs, to be used in other ways? unfortunately, you're hearing about opioids because of the devastating consequences. on the one hand, we have basically seen 16,000 people dying from prescription opioid overdoses. over the past five years, we have seen a fourfold increase in

people dying from heroin. andas stable for many years in the past five years, it's 8000. we are seeing a steep increase from prescription opioids and now from heroin. that has led us to realize what is the nature of the problem. on the one hand, we have the reality that there are many patients suffering from chronic pain but we don't have sufficient opioids out there. nih, we arentrol at trying to develop their good medications that are effective for severe pain that are not addictive. we are trying to partner with the pharmaceutical industry to develop opioid medication that will not be divergent. other partnering with agencies and the fda created a new indication that in opioid

medication should not be divergent. we are also encouraging the better education of the health care providers on how to screen properly pain and manage opioids and how to screen for substantial disorders. multipronged approach from the perspective of nih with various institutes working together at the same time, with their sister agencies. >> i would think one of those may be was ever happening in defense department research on this topic. people in the service and propensityve a high to find themselves in that trap of becoming addicted to the pain medicines they are given often because of their service related injuries and that cannot be a good thing. >> this is something that not many people are aware of. the prevalence of pain and military people is much higher. as a result, they're are more

likely to be given prescription opioids, much higher than the rest of the public. peoplere, the number of that are dying from prescription opioids among the military is higher just by the fact of what you are saying. they are suffering from pain and we don't have many pain medications out there so they receive opioid medications. >> thank you for your help on this. >> thank you. up on theke to follow concerns you raise about opioid abuse and heroin. the fact that it has led to a heroin epidemic is a huge challenge that we are facing in the northeast and across the country. dover withe town of a population of 20,000 people, they had 2 recent deaths in one

day from drug overdoses. i think it should rise to the crossof the kind of -- it is a crisis and i appreciate that you all have a different mission in terms of research but this is something the medical community, the law enforcement community, the treatment community all need to be working on together. until that happens, we will continue to see this crisis escalates. it is already out of control and it's only going to get worse. you can buy a small bag of heroin for less than you can get your prescription filled, then we got a real problem. i want to make that point because i am not sure if there

are other ways in which you all are looking at addressing this issue beyond just the challenge and the extent to which that gets people addicted. farther other things you are looking at with respect to this epidemic? i'm happy to direct that to whomever would like to take that. again, it's a, devastating situation but one of the reinforcing things has been how integrated the agencies have been in working together to come up with solutions and how these solutions are coming into specific action items. the fda has a new indication for effort toand the bring back the medications that are not being used in their resulting in effective interventions. there is a strong concerted effort. there areews is that

medications we currently have that are effective for the treatment of people who become addicted to the opioid prescriptions. are notlenge is they being implement its aware of working with agencies to develop implementation and provide medication that will be easier to take. >> sorry to interrupt -- does nih have a view on whether narcan should be available over-the-counter to families, not just law enforcement or other people who do integrations but to families or concerned about drug overdoses in their families? is that something you think should be readily available? >> i think we are externally lucky to have narcan and we should make it as widely available as possible. nih tould just urge think about all of the ways in

which you can engage on this issue. it is out of control. , not better worse despite all of coordinate efforts we see in new hampshire and other states that are dealing with this issue, we made it the kind of all hands on deck priority that it should be. i would just add that there is a lot of agency work on this. there is inter-agency pain coordinating and i think they are doing good work. we have education programs -- >> how are they coordinating the work they are doing at the state level? with states dealing with this issue? >> i would have to get back to you on that. that's something we have not approached. there is a national pain strategy we are working on through hhs that addresses some of these problems.

there are education programs that nih funds. one of the issues is educating the practitioners on the actual and proper use. we have centers of excellence for education how to manage pain. as a tremendous video for education of practitioners on the use of narcotics and best management of pain and trying to reverse this problem. >> i don't know how we make sure that states are aware of the work that's going on but certainly, that seems to be one ints tocoordinating po better address the crisis. we have to make sure that kind of information is available. congressman hal rogers has been an effective leader in terms of bringing attention to those runs a summit every april and brings in the state.

we have been at that summit each time for the last couple of years. it is an opportunity for states to really hear what the opportunities are that are being thought about across the nation. it is probably not enough. we are still in the thick of a very serious epidemic. those connections are trying to be made and i have to give congressman rogers a lot of credit for being a convener. thank you, dr. collins. as you suggested, them are on the bench you can call in at some future time and we might ask you to do that. over the next week, the record will stay open for questions. i know senator alexander and others have already put some questions out there. andsure you'll get some thank you for being here and the subcommittee stands in recess. host[captions copyright national cable satellite corp. 2015] [captioning performed by the national captioning institute, which is responsible for its

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