Institutes of health in bethesda, maryland is dr. Francis collins, National Institutes of health director. Thank you very much for joining us here on the newsmakers program. Dr. Collins glad to be with you, and great to have a chance to chat at the beginning of a new decade. Steve joining us with the question is Kimberly Leonard, who covers Health Policy issues for the washington examiner, and Health ReporterJayne Odonnell of usa today. I want to begin because this june marks the 20th anniversary of decoding the genes that make up the human body. Two decades later, where are we . Dr. Collins my goodness. Where do i begin . It has been an amazing couple decades of taking that own insight into our human instruction book and beginning to figure out how to read it, written in this strange language with just four letters in the alphabet, and how to apply that to some basic lessons about how life works and how we can use that information to advance medical care. If you look at the place where genomics has become central to biomedical 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. What the micro biome is all about, the micro biomes that live in and on us. In terms of clinical applications, the most obvious one is in cancer. Now because we know cancer is a disease of the genome, it comes about because of misspellings in dna. We have the ability for individuals to say, why exactly are those cells doing what they shouldnt, and how could we choose the optimum therapy . That has been transformative for the management of cancer. In the longer term, we are getting closer to this era of Precision Medicine, where we can use this information to optimize individual plans for staying healthy, so you know what to watch out for in terms of your inheritance. As well as a whole host of ways we can use this to identify the right drug for the right person at the right dose when you need a treatment. We recognize we are all different and dna holds the clues as to why. So it has been a wild ride. Its going to be wild for a while. Genomics has become the center of an awful lot of what we do in medical research, and increasingly in medicine. Steve with more on this is kimberly from the washington examiner. Kimberly dr. Collins, the National Institute of health has secured several funding increases over several years and another heading into the following year. Obviously, you testified before appropriations committees, but also met privately with lawmakers to talk about why investing in medical research is an important thing to do. I was hoping you could take us behind the scenes of those conversations. How do you convince lawmakers it is important for Public Health . Particularly those who might be a little bit averse to increasing government funding . Dr. Collins well, we have been very fortunate that over the last five years, congress has seen fit to increase the budget for nih. Each one of those years, by 2 billion or 3 billion. For fy 20, another 2. 6 billion is being added to our base, which makes it possible to do a vast array of science that otherwise would have to wait or be done at a smaller scale. It is part of my job to explain that. Im not allowed to lobby the congress, so i cannot go and knock on some congressmans door and say, do you have a minute . I have to wait to be invited. But i am always glad to be invited. And i enjoy every one of those meetings, because what i have to tell them about is not a special interest. It is not pleading for some handouts, this is in fact one of the most critically exciting and important things the government does. It both advances human health and is also a really good investment in the economy. Every dollar that nih gives out in grants to institutions all over the country has a return of about over the course of the 8. 33 next five years. There arent too many things where you can point to that kind of roi for our economy. But people do care about health. Ive yet to meet anybody who doesnt have a concern about cancer or cancer alzheimers disease, or diabetes, or some rare disease in their family or a constituent has approached us about. They want to see advances happen here. They are excited when i can tell them what a remarkable time we are in right now with the acceleration of discoveries leading to cures for diseases, cancers, cystic fibrosis, that we dared not even hope for a few decades ago. So its not a hard conversation to have. I can be somebody who brings the information to them, answer their questions, and i always go away feeling like it was time well spent. Steve Jayne Odonnell. Jayne thank you. Dr. Collins, i would love to hear more about your thoughts on the kind of research that is not medical. I personally am not surprised that medical research is not well supported, given the wellknown importance but also influence of the pharmaceutical industry on capitol hill, but i wonder what you would say to members of congress about the importance to look at prevention, to throw more money, to put it bluntly, behind research into things such as hunger, poverty, the kind of things that are leading to the addiction and poor health so many people have. Mentioning quickly the socalled depths of despair, alcohol, drugs, and suicide. What about these underlying problems that often get ignored . Dr. Collins they should not get ignored, they are critically important, and i am with you on that. The opioid crisis, which is an example of one of the depths of despair that you refer to. We have another institute focus on alcoholism, the National Institute of alcohol abuse and alcoholism, and another that focuses heavily on suicide. The National Institute on mental health. Each one of those has a bold, vigorous agenda to identify what are the factors that play out in those outcomes and not just to name them, but to do something about them by initiating interventions to see what we can do to reduce the risks that happen to people in those circumstances. Yeah, that is in the prevention agenda. Let me mention another major effort in prevention which is underway right now that perhaps your listeners might want to learn more about, because they could join and be part of. We really want to understand, what are all the factors that play out in an individuals ability to stay healthy or put them at risk of falling ill . We would like to know that not just about a few people, but a very large number of people who are very diverse in their background. That is the motivation behind the program called all of us. If you want to learn more about it, go to joinallofus. Org. Americans are being invited to be a part of this. We aim to sign up one million americans who will take part in this project. Very diverse in their backgrounds geographically, economically, socially, collect every kind of data we can about their medical experiences, behaviors and life experiences. What is in their medical records, dna. All of this which people agree to take part in as part of this national adventure. We have already signed up over 300,000 people. On the way to one million. This is an opportunity to learn a lot about yourself but also to provide anonymized information very carefully kept in a Data Security system for researchers to learn about what are the factors that help people stay healthy. Not just studying disease, but studying wellness as well. Your question is highly appropriate. I dont think people necessarily realize how much of nihs interests are in this space. Im glad you brought it up so that i could Say Something about it. Steve let me follow up on the question regarding gene therapy. What role does that play with regard to mental or physical conditions or illnesses . Dr. Collins gene therapy has been a dream of many of us for 35 years. It has had a very upanddown course with the things that seem to be working and then they werent working, and a bad outcome was happening. Gene therapy has really come into its own in the last 45 years. As an example, look at sickle cell disease, the first disease described, affecting about 100,000, mostly africanamericans in the u. S. , comes about because of a single misspelled letter for a gene in the codes for human hemoglobin. And yet, we have not had much to offer. Now, as you might have seen very recently, we are at the point using gene therapy not just to help people with that disease, but it appears we are actually curing them, using the delivery of a normal copy of the gene into their bone marrow cells. Which is where red blood is made. Basically, correcting the problem they have lived with with extreme pain all their lives and now bringing back them into an essentially almost normal state. That is pretty dramatic to be able to say. I think, given that success and other conditions like spinal muscular atrophy, another dramatic story in the past year, were at the point of beginning to recognize that maybe the 7000 diseases where we know the dna misspelling might now be ones we could tackle using the same approach and do so in a fashion that is actually scalable. So a lot of discussions about we how we could take the successes of the last couple of years and really move that forward, to accelerate the pace of application, to more and more diseases for families that are still waiting. Jayne just back to congress in prevention, and im glad steve mentioned that. That and so many things we are talking about are incredibly expensive and also have wealthy lobbyists pushing it on capitol hill. Prevention is not something that costs a lot of money and you are going to be coming up with some great conclusions. Presumably soon. We already know a lot of things that work. Just in a nutshell, quickly, before we get back to drug addiction, what would you say to congress about the need to Fund Prevention . As well as cures . Dr. Collins i think they get it. I think they want to know specifically what do you mean by that. I think they have gotten excited about this all of us program i just mentioned. If you look at the budget, they upped the support to 500 million as a concrete indication million as a concrete indication of their enthusiasm for this kind of very coordinated, largescale prospective study. Jayne good to hear. Conveniently, id a, the Drug Abuse Institute and of course the alcohol institute. I dont think anyone would disagree the federal government does not have a coherent marijuana policy. It is illegal federally, it is becoming increasingly legal medically and recreationally in states, and researchers say this hampers their ability to study it. Recently, nih awarded an academic who is funded in part by the marijuana industry, a multimillion dollar grant on the study of the benefits of marijuana affecting ptsd. He has told me about the very grave concerns of marijuanas effects on mental illnesses. The link to psychosis, schizophrenia, and other illnesses. Given that, and the fact that nih had to shut down and alcohol nih had to shut down and alcohol benefit study because of industry involvement, how is nih going to avoid industry influence and satisfy the worried parents of those with psychosis and other conditions and marijuana supporters who hope the drug is considered objectively . Dr. Collins so i share with the doctor deep concerns about the potential harms that widespread use of marijuana can induce, particularly on the developing brain of adolescents. And also on pregnant women, because it may affect the fetal developing brain. Frankly, we know far too little about the benefits and risks of smoked marijuana, there have been very few studies that have actually rigorously tested that. We are in a funny place, because the way that this currently works in the united states, if nih is approached about funding a Research Project on smoked marijuana, there is only one allowed source, which happens to be a farm in mississippi that we run. People dont realize that i run a farm in mississippi that grows marijuana, because i am required to do so. That is the only source investigators can use, and it might be rather different than what you can get in the states where marijuana is approved in terms of its constituents. In order to do that research, the researcher has to get approved by dea as a schedule one user and get fda to approve their research as well. That is a big deterrent for a lot of research to go on. Frankly from our perspective as scientists, it will be hard to interpret data about smoked marijuana when the actual nature of the product is vastly different depending on where you got it, in terms of how much thc, the active hallucinogenic ingredient is in there, how much cbd is in, the other ingredient that doesnt make you high but might have other benefits we would really like to have those studies where we are studying studies where we are studying those compounds in pure form so they can see what they are doing. Again, because of various limitations of schedule one limits, we are not able to do as much of that as we would like. We are spending about 150 million a year on research on marijuana, cannabis, and thc and cbd. It is still pretty limited in terms of clinical application for the list of things i mentioned. I would not say at the present time that industry is attempting to influence a lot of what we are doing in the marijuana area. You mentioned the alcohol study. That was a very unfortunate circumstance. We have been very clear about that, we will not be making that mistake again. People got fired about that at nih. And should have been. A deal was made potentially, but it never happened, a study that would have documented that small doses of alcohol are good for your health, something the industry would very much want to be true. We will never do that again. Steve let me turn to Kimberly Leonard. Kimberly dr. Collins, we will have more questions about marijuana, but i want 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 all kinds of governmentfunded research not just mice, but dogs, cats, primates. I know at the nah, you worked to end Research Using chimps. I do you also get a lot of backlash from Animal Rights groups who say the research is unnecessary and cruel. I am wondering, do you see a future in science where Animal Research might be more rare. There have been so many technological developments. Have you already made progress in this area . Do you think there could be a time when animals are used in used rarely in conducting medical research . Dr. Collins we are working very hard to develop alternative ways to test drugs to see if they are safe and effective that dont involve animals, such as putting human cells on chips where you can assess the effect of the drugs without putting humans at risk, or using animals. And we are seeing real progress, but its far from what it would need to be to substitute for the current expectations about how you demonstrate whether something works and whether it is safe and effective. We will i hope someday get there, but it would be premature at the present time to say we have the information we need to be able to do research that ultimately will lead to outcomes for humans that are what we need them to be, which is safe and effective. When it comes to animal models, we certainly primarily use mice and rats for that. We are engaged right now in a high level study at nih trying to understand what we might want to do in terms of looking at the reliability of those studies, because there are certainly instances where the animal models do not turn out to be predictable for humans, and we need to understand that and stop doing things that are not helping us. For me, i would say if we are not able at the present time to do research on animals, we would lose a great deal of information that we need to try to help those people who are waiting for answers. I mentioned the gene therapy as an area of great advancement right now. I do not know if you are somebody who has a child with a rare genetic disease, but you want the child to be the first animal to be able to receive gene therapy treatment, which has never been tried previously on a mouse or rat and even a nonhuman primate, you would like to know that data was there. I would like to provide it, to ensure people, with their lives at risk, that we know what we need to know. At the present time, that means we need to do research on animals. We have the most careful oversight of this, which i am not sure everyone is aware of, where every animal study gets reviewed by a rigorous group, including public citizens to , determine whether or not it is justifiable on an ethical basis and whether the pain and suffering has been thought about in every imaginable way. If people are worried about that, they can learn more about it by going to our website. But i do think all in all, animals in research are critical for our research at , least for the present time. Steve did you want to follow up . Kimberly i was going to ask, can you help viewers who are troubled by experimentations done on animals, can you help them understand why keeping the Animal Research is so important . There are some instances where it does not translate very well to humans and that has raised concerns among animalrights groups and even government waste groups. Dr. Collins sure. Take the example of chimpanzees, where i did ask for a careful review by the National Academy of medicine about whether research on chimpanzees was still justifiable. They looked at the ethical issues and looked at what we were learning medically from studying our closest relatives, and concluded that the balance was in favor of saying, lets stop doing this. I agreed with that, and chimpanzee research is no longer allowed within nih funding. We are listening carefully to those arguments. If on the other hand, if you , wanted to say, how did we get to the point right now, we announced a month or two ago that we had an effective treatment for ebola virus, a terrible, scary disease that came to the u. S. Only in a few people, but could still come back someday. We now have effective treatments for that. We know initially they are going to work because we were able to try them out on nonhuman primates, on monkeys who get this disease, and nknow we were on the right track. If we did not have that, we would not be where we are right kimberly talk about alzheimers, because i know that has great interest. I have more questions on the ethics of testing on animals and humans when it comes to cannabis, but lets turn to alzheimers for now, and we will come back to it if there is would not be where we are right now. Think about the consequences. They would be major. Steve let me turn back to Jayne Odonnell of usa today. Jayne i think we should let time. That would be fun. Steve we have about five minutes. Kimberly sure, sure. Thanks, steve. Obviously, there has been an increase for alzheimers funding. A big concern for a lot of our viewers. I know the numbers are frightening. 14 million predicted to have alzheimers by 2050, it is the sixth leading cause of death. There is still no cure or treatment. Do you think we will find a cure and how much funding will it take . Dr. Collins you are right, we are fortunate to have another 350 million added to nihs budget for this, bringing us to 2. 8 billion. That is more than four times the budget we had a six years ago and we are putting every dime of , that to good use. We have more than two dozen new drug targets that are vigorously being pursued, going beyond where most of the focus has been on the protein called amyloid. 2020 will be really interesting, because as you know, biogen has announced there might be a signal in their most recent trial. It was not there when they first analyzed the data, but they reanalyzed it and thought maybe in a subset something was working, and asked the fda to look again, and fda are going to. That will make a huge difference, because the difference between having 40 failed trials and 39 failed trials and one that looks like it might have worked for a subset of patients is enormous, because that tells us we are on the right track. But i do not know where the outcome is going to be. We have hedged our bets at nih over the course of the last five or six years to look at every nook and cranny, and working with industry and the accelerated medicines partnership, in an open access way, to further expanded the list of new drug targets that can be vigorously pursued. Will we have a cure this year . Probably not. Will we have a way to slow the disease in the next five years . I believe we will, but it will take every bit of energy, creativity, and determination and resources possible to get there. Jayne if we could come back to the cost of drugs, i know you do not want to talk about legislation on the hill and you do not lobby, but so many of the drugs that are developed, they are developed with so much taxpayer money, critics would argue that a lot of the benefit goes to the drug companies. More importantly, so many taxpayers cannot afford these drugs. There is an example, you mentioned sickle cell, which is a great example of how there is a program where you folks are working with the Gates Foundation to make these drugs more affordable to people in developing countries, but is there anything we can learn from that and ways to make these drugs more affordable to people in this country, those drugs developed with taxpayer money . Dr. Collins it is a critical issue, and of issue, and of course, we are deeply sympathetic with the need for something to happen here, because this great increase in the cost of drugs is unsustainable and makes them unreachable for many people who need them. We at nih play a major role in the early stages of almost every drug that gets developed and approved by fda. It was 100 of the drugs between 2010 and 2016 that were reviewed in a careful analysis, all of Research Involved in the early stages of getting that to that success. We do not have levers to pull here, however, because once the process has happened, a license is made by a nih university grantee, and then it gets way out of our hands. Our efforts in the past to try to involve ourselves in that were not successful. They basically discourage collaborations. So we are cheering for those who have good ideas on how to deal with this. We are probably not in the best position to change that dynamic, but we hope that others will succeed in doing so. We need that now. Steve final question, Kimberly Leonard . Kimberly i know this will be kind of hard to narrow down, but here we are. , we this heirs, airs will be in 2020. You have been overseeing the agency for the entire last decade and i am wondering, if there is one nih funded discovery that you would say particularly stands out to you, that you would highlight as one of the biggest achievements of the last 10 years . Dr. Collins it is very hard to pick one. I have touched on a few that i am excited about. I will mention what i have not talked about, cancer immunotherapy. The ability to harness the immune system to go after those lingering cancer cells when all else has failed, it is amazing to see the progress we have made in that 10 year period, and there is more coming in that space. Steve but in regards to cancer, and we have all been touched by cancer directly or indirectly. Medically speaking, what is the leading question you have in your mind to find more cures . Dr. Collins again, i guess it comes to immunotherapy. How do we activate the immune system to recognize cancer cells for solid tumors like aggressive cancer, pancreatic cancer, the ones that do not respond very well. They must have cells there that do not look like cells, but the immune system does not see them. How do we take the immune system to graduate school and teach them what to watch for . Steve you think we will get there when . Dr. Collins we are already seeing that happen in isolated cases. The next five years, you just watch. This is going to be a dramatic series of events. And the idea of having stage four, when it is usually time to make your plans, people, it will no longer be the death sentence that it currently is for far too many. Steve dr. Francis collins, director of the National Institutes of health joining us from bethesda, maryland on cspans newsmakers. Dr. Collins, thank you for being with us. Happy new year. Dr. Collins happy new year to you all. Steve we will turn to our reporters first. I want to go back to something he talked about earlier. Precision medicine. Where are we heading in that . Jayne we are headed to a point 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 actually is best for you as an individual rather than a group. But i want to bring it back to my personal interest. A couple of my personal interests, the Surgeon General says what about Precision Medicine, Public Health medicine, Precision Medicine that is going to come out of this massive study that looks at what really works in the communities . Because we cannot afford these medical treatments. We cannot continue to afford the medical treatments that are being encouraged and lobbied for so heavily because we need to know what works to make people do the right thing, change their behavior, and to make them feel like life is worth living. Worth living soberly, frankly. Steve you bring up prescription drugs. How do we bend the curve so prices come down . There are so many different layers. Nih research, pharmaceutical companies, advertising, and you have the drugs that are out of the price range of many americans, as you pointed out. Jayne there is some pretty important legislation pending on capitol hill that may or may not pass this year before an election. It could be pretty tough. But to actually bring down the outofpocket costs. Particularly for medicare patients, it is unbelievable what some of them have to pay. Changing the cap from 13,000 to 3000 or 2000 could make a world of difference for these people. Some of these drugs cost well into six figures a year and it is just not sustainable. Steve Kimberly Leonard, you talked about alzheimers, an insidious disease affecting so many americans. Where are we headed in this . Kimberly there is no current cure or treatment right now and , i know families get together and parents start to notice that their aging parents are dealing with these issues. They are starting to think about planning for endoflife care. Its not something that will only have to be dealt with medically, but it affects so many caregivers of parents with alzheimers. We are heading into a massive generation that will have alzheimers and parents who will need to care with them, and as a society, we will have to decide how we make sure caregivers dont also go broke when this happens, that they dont develop medical and physical issues of their own. It is really a bigger problem than alzheimers itself. Steve let me ask you both about dr. Collins, he is someone who is able to navigate through federal bureaucracy and congressional politics. His tenure at the nih and your interactions with him as a leading physician, a leading researcher. Kimberly its interesting, because he is a holdover from the Obama Administration who is leading the National Institutes of health. So he has had the thread some difficult needles in terms of understanding where the Trump Administration is coming from. He is able to secure higher budgets for his agency, despite the Trump Administration overall calling for cuts in a lot of different agencies. Calling for cuts in a lot of different agencies. As he said, he talks to members of congress about what is going on. It is an issue that i know is bipartisan to be able to raise more money for medical research. Hes been in this post a long time. I talked to lots of scientists, and hope he will be there through the end of the Trump Administration, at least. Jayne as long as he is alive. He is superhuman. People are so impressed with him. Kimberly i talked to some scientists who have had some issues with the way he has governed but with the Trump Administration, just feel a lot safer that there is someone who has scientific rigor behind his role and as a geneticist who is able to really 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. Steve i will give you the last word. Jayne nobody is without their critics and i know we talked about earlier offline that he has gotten some criticism over the concerns about fetal tissue, but to have such a brilliant scientist who does understand everything about health, able to navigate this massive bureaucracy in an administration that has had so much turnover is heartening in this day and age when there are so many Serious Health problems and health costs are so high. Steve we will follow your work online. Jayne odonnell of usa today, Kimberly Leonard of the washington examiner. To both of you, happy 2020. Thank you for joining us on newsmakers. We appreciate it. My pleasure, thank you. Our pleasure. Tonight, on the communicators, a group that advocates for democracy, talks about the organizations report on world internet freedom. What were generally sitting on the social media is it used to be a level Playing Field for Free Expression type activists and that is really being coopted by some of the more powerful, well resourced actors in our society. That is where social Media Companies need to root out the bad actors and to perhaps make certain policy changes within the algorithms to incentivize the type of productive, democratic discourse and conversation. Watch tonight at 8 00 eastern on cspan2. Campaign 2020. Watch our continuing coverage of the president ial candidates on the campaign trail and make up your own mind. Watch our live coverage of the Iowa Caucuses on monday, february 3. Cspans campaign 2020, your unfiltered view of politics. Now, remarks from 2020 president ial candidate joe biden. His campaign visited iowa where we spoke about making Climate Change a priority and investing workforce. N in the for this past from this past weekend, this is about an hour and 25 minutes