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Two decades later, where arby are we . It is about beginning to figure out how to read it with just this strangely which how to apply that to some basic lessons about how life works and how we can use that information to advance medical care. Where look at the places this has become central to research, you cannot find a place where that is not the case. Everyone is using a variety of approaches to understand. Phenomenal questions about what cells do and how the brain works. In terms of clinical applications, the most obvious one is in cancer. We know cancer is a disease of the genome and comes about from misspellings in dna. We have the ability for individuals to say why cells are doing what they shouldnt how we choose therapy. Transformative for the management of cancer. We are getting closer to this era of Precision Medicine and can use this information to optimize individual plans to stay healthy. As well as a whole host of ways we could use this to identify the right drug for the right person when you need treatment. Its going to be wild for a while. This has become the center of an awful lot of what we do in medical research and medicine. Dr. Collins, the National Institute of health has secured several funding increases. You testified before appropriations committees but also meet privately with lawmakers to talk about why investing in medical research is urgent. Usas hoping you could take behind the scenes of those conversations. Lawmakers itnvince is important for Public Health . We have been fortunate the Congress Sees fit to increase the budget. Another 2. 6 billion is being added, which makes it possible to a vast array of science that otherwise would have to wait. It is part of my job to explain that. I cannot go and knock on some congressmans door, i have to be invited. And i enjoy every one of those meetings. Them about is top not a special interest. Most, in fact one of the critically exciting things the government does it advances human health and is a really good investment the economy. Out has aar we give return of about eight dollars and . 33. Arent too many things where you can point to that kind of roi. But people do care about health. Ive yet to meet anybody who doesnt have a concern about cancer or alzheimers disease. They family or a constituent has approached us about. I want to tell them what a remarkable time we are in right now with the discoveries leading to cures for cancers we dare not even hope for a few decades ago. Its not a hard conversation to have. Feeling always go away that was time well spent. Thank you. Dr. Collins, i would love to hear more about your thoughts on the kind of research that is not. Personally am not surprised medical research is not well say about congress throwing more money behind research into things such as , thingsnd poverty leading to the addiction and poor health so many have. Mentioning alcohol, drugs, and suicide. What about these underlying problems that often ignored get ignored . They should not get ignored and i am with you on that. We have an entire institute on drug abuse which is the lead in our major efforts on the opioid crisis. Have another Institute Focused on alcohol and alcoholism in another that focuses heavily on suicide. Agendae has a bold, big to identify the factors that play out in those outcomes. Them, but toame initiate interventions to reduce the risk to people in those circumstances. That is in the prevention agenda. Let me mention another major effort in prevention your listeners might want to learn more about. Understandants to all of the factors that play out in individual abilities to stay healthy. We would like to know that not just about a few, but a large number of people that are diverse in their background. Behind the motivation the program called all of us. Toyou want to learn more, go dachshund go online. We aim to sign up one million americans who will take part in this project. Very diverse in their backgrounds racially and socioeconomically. Dataollect every kind of we can about their behaviors and life experiences. Dna, medical records and all of this which people agree to take part in. Have already signed up over 300,000 people. To learnn opportunity about yourself but also to provide anonymized information kept for researchers to learn about the factors that help people stay healthy. Not just studying disease, but wellness as well. I dont think people necessarily realize how much interest is in the space. Let me follow up on the question what role does gene therapy play with regard to mental or physical conditions . Has been a dream of many of us for 35 years. It has had an upanddown course which with the things that seems not to be working. It has come into its own in the last 45 years. , theat Sickle Cell Disease first disease described affecting 100,000 africanamericans coming about because of a single misspelled ter for jean in hemoglobin a gene in hemoglobin. Now, if we are using gene therapy not just to help people but to cure them, using the delivery of a normal copy of the gene into their bone marrow cells. Problemy correcting the they have lived with and bringing them back into an essentially almost normal state. That is pretty dramatic to be able to say. And other success conditions, we are beginning to recognize that may be the 7000 diseases where we know the misspelling might now be ones we could tackle using the same approach and do so in a fashion that is scalable. So a lot of discussions about taking the successes of the last couple of years and moving it forward. Just back to congress in prevention, and im glad steve mentioned that. So many things we are talking about are incredibly expensive. Prevention is not something that costs a lot of money and you are going to be coming up with some great conclusions. Just internet show in a nutshell, what would you say to congress about the need to Fund Prevention . I think they have gotten excited about the program i just mentioned. If you look at the budget, they up the support to 500 million as a concrete indication of their enthusiasm for this kind of coordinated, largescale effort. Mentioned the drug abuse heads andthe doctor the alcohol institute. I dont think anyone would disagree the federal government does not have a coherent marijuana policy. Researchers say this hampers their ability to study it. Recently, nih awarded an academic funded by the Marijuana Industry a multigrant. Comes as i have been told about the very grave concerns of effects on mental illnesses. And the fact that nih had to shut down and alcohol nihif it study, how is going to avoid industry satisfy thed worried parents of those with conditions and marijuana supporters who hope the drug is considered objectively . I share deep concerns about the potential harms that widespread use of marijuana can induce, particularly on the developing brain of adolescents. Little, we know far too about the benefits and risks of smoked marijuana. Place becausenny the way that this currently works, if nih is approached about funding a Research Project , there is only one allowed source which happens to be a farm in mississippi which we run. That is the only source investigators can use and it may be rather different than what you can get in a state where marijuana is approved. To do that research, the researcher has to be approved as a schedule one user and has to from the fda. That is a big deterrent for research. Frankly, its very hard to interpret data about smoked marijuana when the actual nature of the product is different depending on where you got it, how much thc, the active cbd,cinogenic ingredients, we would really like to have studies where you are studying those compounds in pure form. But because of various are not able to do as much of that as we would like. We are spending 150 million a year on research and marijuana. Limited in pretty terms of applications for the things i just mentioned. Industry isot say attempting to influence a lot of what we are doing. You mentioned the unfortunate alcohol study. We will not make that mistake again. People got fired about that and should have been. A deal was made to work out a study that might have documented that alcohol is good for your health, something the industry would want to be true. We will never do that again. We will have more questions about marijuana, i would like to turn quickly to Animal Research. I have done a lot of stories on this as part of my work for the washington examiner. Animals are used in a lot of research. I know you work and Research Using chimps. I do you also get a lot of backlash from Animal Rights groups say the research is unnecessary and cruel. Dca future where Animal Research might be more rare. Have you already made progress in this area . Do you think there could be a time when animals are used in medical Research Medical research . We are working hard to test drugs that dont involve animals. Putting human cells on chips where you can assess drugs without putting humans at risk. Butre seeing real progress, its far from what it would need to demonstrate whether something is safe and effective. I hope to get there, it would be premature to say we have the information we need to do research we need. Models,comes to animal we certainly primarily use mice and rats. Tryingengaged in study to understand what we might want to do in terms of looking at the reliability of those studies there are incidents where the animal models dont turn out that protectable. Predictable. For me, if we arent able to do research on animals, we would lose a great deal of the information we need to help those people. I mentioned gene therapy as an area of great advance. I dont know if you have a child with a genetic disease where you want that child to be the first. O receive treatment you would not like to know that data is there and i would like to provide it to ensure we know what we need to know. At the present time, that still s we need to do research. We have oversight where every animal study gets reviewed by a rigorous group to determine whether it is justifiable on every imaginable way. If people are worried, they can learn more about it. But i do think animals and research are critical for our future. Can you going to ask, help viewers who are troubled by experimentation understand why keeping Animal Research is so important . Where it instances does not translate well and raised concern among Animal Rights government waste groups. Take the examples of chimpanzees where i asked for careful review. They looked at the ethical issues and what we were learning medically and concluded that the balance was in favor of saying lets stop doing this and i agreed with that. Chimpanzee research is no longer allowed. So we are listening carefully. But on the other hand, if you wanted to say, how did we get to the point now that we have an effective treatment for ebola disease terrible, scary that could still come back. We now have effective treatment for that. We know it will work because we were able to try them out on nonhuman primates who get this disease. If we had not had that we would not be where we are. Major. Sequences would be i think we should let kimberly talk about alzheimers because i know that has great interest. I have questions on the ethics of testing and humans, but why dont we turn to alzheimers for now . We have about five minutes. Obviously, there has been an increase for alzheimers funding. The numbers are frightening. 14 million predicted 10 alzheimers, it is the sixth leading cause of death. Do you think we will find a care and how much funding will it take . We are fortunate to have another 350 million added to the budget. It is more than four times the budget we had years ago. Dozen newre than two targets that are being vigorously pursued, going beyond most of the focus. 2020 will be interesting. The signal was not there when data was first analyzed, they thought something was working and have asked the fda to look again and they are going to. That will make a huge difference. The difference between 40 failed trials and 40 failed trials and one that looks like it may have worked is enormous. It tells you we are on the right track, but i dont know what the outcome will be. We have hedged our vets over the course of the last five or six years. Working with industry has further expanded the list of new drug targets that can be pursued. Will we have a care this year . Probably not. Will be slow the disease . Will be slow the disease . Will take can, but it every bit of resources possible to get there. If we could go back to the , i know you dont want to talk about legislation on the hill and you do not lobby , but somebody drugs are developed with taxpayer money. Oftics would argue a lot that benefit goes to drug companies. But so many taxpayers cannot afford these drugs. You mentioned sickle cell, a great example of how there is a program working with the gates to make these available to folks in developing countries, is there anything we can do to make the drugs more available to drugs in these countries in this country . Hypocriticala issue, though we are deeply synthetic for the need for some it to happen. This increase in the cost of drugs is unsustainable and we at nih play at early role in the major stages of almost every drug that gets developed. All 210 of recently developed drugs had nih Research Involved the early stages of that success. We dont have levers full here to pull here. Once there is the process, there is often a license in the drug process gets way out of our hands. Our efforts in the past to involve ourselves was not successful and they discouraged collaboration, so we are cheering for good ideas of how to do with this. We are not in the best position to change that dynamic but we encourage others to do so. I know this will be hard to narrow down, but here we are. Overseeing the agency for the entire last decade. I am wondering if there is one discovery you would say particularly stands out to you that you would highlight is one of the biggest achievements of the last 10 years. It is hard to pick one. I have touched on a few i am excited about. I will mention cancer immunotherapy. The ability to harness the immune system to go after those lingering cancer cells is amazing to see. Theres a lot more coming in that space. We have all been touched by cancer directly or indirectly. What is theeaking, leading question you have in your mind . I guess it comes to immunotherapy how to reactivate the immune system to recognize cancer cells for solid tumors. The ones that still dont respond very well. They must have cells there that are not looking like cells but the immune system still does not see them. How do we take the immune system to graduate school and teach them what to watch for. You think we will get there when . We are already seeing some of that happen in isolated cases the next five years, just watch. Iv will of having stage no longer be what it currently is for far too many. Dr. Francis collins is the director of the National Institute of health joining us on these make a program. On the program. Happy new year. Happy new year to you all. I want to go back to something he talked about earlier. Precision medicine. Where are we headed . Pointare headed to a where they will be able to find what works for individuals as opposed to a large number of people. They will be able to decide whether a certain Cancer Therapy is actually best for you as an individual. I want to bring it back to my personal interest. What about Precision Medicine that is going to come out of ats massive study that looks what really works in the communities. We cannot afford these medical treatments. We cannot continue to afford the medical treatments that are being encouraged and log for so heavily because we need to know what works to make people do the right thing, change their behavior, or make them feel like life is work worth living. How do we bed to the curve so prices come down . There are so many different layers. There is some pretty important legislation pending on capitol hill that may or may not pass this year before an election. To actually bring down the outofpocket costs for medicare patients is just unbelievable. Changing the cap from 13,000 to 3000 or 2000 could make a world of difference. Some of these drugs cost well into six figures a year and it is just not sustainable. Alzheimers,bout an insidious disease affecting 70 americans. Where are we headed so Many Americans . I know families get together and parents start to notice that their aging parents are dealing with these issues. That willomething only have to be dealt with medically but affects so many caregivers of parents with alzheimers. Heading into a massive generation that wills have will have alzheimers and needs care we will have to decide how caregivers dont also go broke when this happens and develop issues of their own. It is really a bigger problem than alzheimers itself. Dr. Collins is able to navigate through federal dropsy and politics. His tenure at the nih in your interactions with him as a leading researcher. Its interesting, because he is a holdover from the obama administration. The threat some difficult needles in terms of understanding where the Trump Administration is coming from. He is able to secure higher budgets the trump he talks to members of congress about what is going on. That is issue bipartisan to raise more money for medical research and hes been in this post a long time hoax he will declare through the end of the Trump Administration people are so impressed with him. Have been some with issues the way he has governed but with the Trump Administration, someone who has so much scientific rigor as a geneticist who is able to push some of those big projects, so he has a lot of praise from both sides of the aisle and a lot of different communities. Nobody is without their critics and i know we talked about earlier offline that he has gotten criticism over the concerns about fetal tissue, but to have such a brilliant scientist who understands everything about health, able to navigate this massive bureaucracy in it administration with so much turnover is heartening in this day and age when there are so many Serious Health problems and health costs are so hard high. Of you, happy 2020. Thank you for joining us on newsmakers. Our pleasure. I very much understand the skepticism. 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It was much safer than cigarettes, the fda would approve any day. When he was done, caleb and his friends went to have a talk to him. He repeated that juul was for adults and not kids, and took , showedeek looking juul the boys how it worked, and call it the iphone of vapes. We look at the issue of teen vaping and regulation of cigarettes. The trump decisions to ban most products and we discussed the issue with health care reporter. That is at 8 30 p. M. Eastern. 2020 democratic president ial candidate senator Amy Klobuchar held a town hall

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