You wake up, and its tough to get out of bed. Its not for the faint of heart. But once you are there, ive got a mission. Rose the future of medicine when we continue. Rose fudging for charlie rose is provided by rose funding for charlie rose has been provided by american express. Rose additional funding provided by and by bloomberg, a provider of multimedia news and Information Services worldwide. Captioning sponsored by Rose Communications from our studios in new york city, this is charlie rose. Rose dr. Francis collins is the direct are of the National Institutes of health. He has been described as quote the man who holds the most powerful job in american m with two projects, theasked Precision Medicine initiative and the Brain Initiative. The Precision Medicine initiative provides new approaches to Disease Treatment and prevention. It accounts for an individuals geng etics, environment and lifestyle to determine a diagnosis. The Initiative Aims to enroll one million volunteers. The Brain Initiative looks to increase Scientific Understanding of the brain and neurological diseases. Im pleased to have dr. Collins, our friend, back at this table. Welcome. Great to be back at this wonderful table with you, charlie. Weve talked about many things here. We have. Tell me about these new things. Im constantly, you know, curious about where the new is. Of course. What is Precision Medicine. Precision medicine is sort of the contrast to one size fits all medicine. Maybe its easy to say what it is by what its not. Of course weve been trying to make our medicine precise over time with variable success. But still its the case, charlie, if you go to the doctor and you need some kind of intervention or some recommendation about your health care, youre likely to get kind of a again erich recommendation based on the evidence that was built upon a again erich person. Youre not again erich. Im not either, nobody listening to this is either. So the idea of Precision Medicine is to try to combine what we are learning but with some pretty dizzeing new technologies about individual differences. Some of that is your dna, your genome some is your environmental exposure, some of it is your lifestyle, your exercise pattern, your health behaviors. If we had all of that data in one place on a very large cohort of americans and we could actually begin to find out what works for the individual, that would be a good thing. Thats one of the goals of this new initiative which is pretty breathe taking. What does that mean, what works for an individual. Well, if you are healthy, as most of us try to be, well, what should we be doing to maintain that healthiness . Pretty much now everybody gets told to do the same thing. Rose exercise, diet. Yeah, right. And dont smoke. And those are good things. But individual. Rose everything in moderation. Everything in moderation. But those things will perhaps not be the best fit for every person. There may be some people for whom its probably more important to Pay Attention to your diet than to your exercise. There may be other people whore whom smoking is like really high risk and should never have started if they do they should quit. Rose why is that . There is this thing called the genome. We all are born with a sort of deck of cards here. Its up to us how we play the cards. But that genome influences a lot of what happens to us in the course of our lifetime as far as health. An if we get sick, we also want to know how best to manage that illness, and right now a lot of that is one size fits all. You wouldnt go to the shoe store and just pick any old shoe off the rack without noticing the size. But when you go to your doctor, you are kind of getting that kind of a shoe. And it may not be right for you. If you have diabetes, what kind of diabetes do you have, you know, type 2 diabetes is probably not one disease, a paper published last week says it is at least four. Different interventions for different types would make a good outcome happen. Precision medicine is trying to take that apart. And it is particularly trying to take it apart rye now cor cancer which is something we could talk about, because that is where we are furtherrest along with this concept. Someone said to me in your profession, that in the next five to ten years well have significant curs to cancer. I totally agree with that. Rose totally agree with that. Totally agree with that. Significant cures, let me be cure. Im not going to say that every case of cancer will be cured in five to ten years. After all, cancer is probably a thousand diseases but we will have cured some that we cant cure now in the next five to ten years. Rose how many can we cure now . You know, we do really, really well with childhood leukemia. Rose that is. Hodgkins disease, we cure that almost all the time. We increasingly do well with mel noama even after had it has spread. We, of course, the death rate from cancer is actually dropping every year by about 1 percent, so we are, acrosstheboard, saving more lives from cancer. But theyre hard won those battles. To see that 1 percent per year actually drop even further, is going to take all the technologies that were talking about here and spring them bring them to bear on this with every bit of energy and determination that our nation and the world can mount. Rose so what do you do at the institutes of health . We are the largest supporter of Bio Medical Research in the world. The National Institutes of Health Thanks to the paks taxpayers what has a budget of about 30 billion a year. It goes out there to all of the finest institution, medical centers, universities, Small Businesses in this country and some outside the country that are doing this cutting edge research. So when you read about a breakthrough in cancer, immunotherapy, it is because nih paid for it it. So Francis Collins decides how to spend 30 billion in terms of pushing medical research. Not all by myself. Rose but the buck stops with you. Much of what happens, people send us, investigators send us their best ideas. And we dont tell them what those should be. Here is my grant. Here is what i could do if you give me money for five years. We put that through the peer review process, the toughest one in the world. And their peers look at their proposal and try to decide whether they think did will work or not. Then they assign it a priority score. If its in the good range, then they get funded and we say go to it and lets see what you can do in a period of four or five years which is the usual length of a grant. That is, i think, the best system in the world. Right now i have to tell you, it is a system under great stress. And the chance that that grant, that that person sent in is actually going to get funded is down to about one in six. Where it used to be one in three. You see six good projects and you will fund one of them. Yeah. And i know im not just throwing away things that dont matter. Rose you are throwing it away because we are not funding it, things that could save lives, is what you are saying. Im afraid i am saying that. Nih has lost over the last 12 years 25 of its purchasing power by research, by flat budgets that have been eroded by inflation, complicated by the is he quester that took away a billion and a half dollars on one day in march 20136789 we have not recovered in that. Our deficit in this nation dealing with our fiscal situation has had a consequence for medical research and has slowed us down for things we could have been doing by you no. We would be further along with Cancer Research than we are rose who funds it if youd we dont . Foundations . There are lots of foundations very invested in this. Rose universities. Universities, of course lets be clear. Were in an ecosystem with the private sector. And they spend twice as much in r d as nih does getting those great ideas all the way out the door to an fda approved therapeutic. And we need them and they need us. But theyre not actually in a position now to ramp up their funding either. Rose tell me about the Brain Initiative. I would love to. Rose so would i. So the brain, well, gosh, this three pounds between our ears here, made up of some 86 billion neurons, each of which has maybe a thousand connections to ear neurons. Rose how many. 86 billion. So that would be 86 trillion connections. Rose right. The most complicated structure in the known universe. And we are audacious enough to say its time to figure out how it works. Not just in a sort of general idea, but in a detailed idea. How do those skirts in the brain do what they do. How is it that im looking at you, my brain is processing your image and i know youre charlie. And how is it that you are liss egg listening to my words and hopefully theyre making sense because are you processing them. And how is it that you are tree treefing memories as are you talking about other things you heard about. Rose or the things you told me or whatever. We dont know how that works. And boy do we need to know how that works if were really going to get to the bottom of Alzheimer Disease or autism or epilepsy or traumatic brain injury or als. All of these incredibly important frustrating neurological conditions. Our problem is, we are making progress on those but not at the level we could if we really understood the basic foundations of how the brain works. And we can do things pretty well when you look at the whole brain. We have these amazing scans now whether they are mris or pet scans. And we can look at individual neurons and say whats that cell going to do if i tweak it with this neurotransmitter. T there is this huge space in between the single cell and the whole brain that we dont have much nrvetion about. So the brain Initiative Aims in a ten year period and were in the third year now, to go after that and figure out the kinds of technologies that need to be invented to be able to measure maybe tens of millions of neurons at once. Acting under some interesting stimulus, to see what they are doing. Learn the language of how the brain accomplishes these amazing things. And i think its probably the most audacious program that you could imagine. Some people have argued our brains arent complicated enough to understand our brains. Rose our brains are not complicated were not smart enough to understand our brains. Somebody who said that, but maybe computers will help us there am because we will have to model it. Rose you are funded for how many years, five. Of course at nih everything is funded for one year because the congress has to decide what are they going to do the year after. The plan for this scien tiffically which is a very bold plan put together by some extremely visionary neuroscientists including corrie bardman from right here in new york at rockefeller, lays out a plan to 2025. And we were now into that just starting the third year. And much of it in the first few years is building technologies. And then it moves into applications and ultimately to understanding disease. Rose when you look at some of the more remarkable things like gene editing. Yes. Rose what is happening there . This is a great story. I know. And it comes out of the most obscure area of basic science that you can imagine. People studying bacteria, and the viruses that infect bacteria, now who would care about that, but its sort of interesting basic science. They realize that bacteria have their own way of fighting off viruses and try to figure out how that works. Basically the bacteria have figured out how to damage the viral dna by editing it. When you learn that system and you see how incredibly elegant it is, you can just pick it up by recome by nant dna out of that bacteria system and put it almost anywhere else you want to it is a revolution. Rose what happens when you put it wherever you want to. If you are a researcher and you want to make a mouse that has a mutation in a very particular spot, that used to take you years and it was frawt with errors. Now it is a week or two of using so the called crisper. Rose what is that. It is basically a very elegant molecular surgery that because of the ability of these bacterial enzymes to home in on a genome of three billion letters and find the one that you told it to go and look at, and it goes and it finds it and then it snips it. And it either just cuts it and basically knocks that gene out or it can even, if you tweak it right, replace that letter, lets say it was an a with a c. So suppose you want to cure single cell anaemia, something i really hope we will get to. People are thinking maybe we can do that. Rose you mean trying to cure sickle cell anaemia. Yeah, you take somebody that has the disease. You use this remarkable induced fleury potent stem cell approach, ips cells where you can take a skin buy op see, grow a few cells, convince them to go back in time and become plu ry potent, they can make any cell you wanted them too. They grow foa ever. You take the enzyme and the sickle cell and you fix it. It is still that persons letter, you just change that one letter. You then grow that up into blood forming cells, give it back to the center, there it is their cells no transplant rejection. You should be then in a situation to cure the disease. Rose is it possible now. It is possible now. Rose is it being. It is become actively pursued now. It looks like it works pretty well in animals. Rose if you do that, just think of what you can do. Yes. Just think what you can do now were actually seeing though people getting concerned about wait a minute, are there concerns about not using this in certain places. Rose like designer edits. We will have a meeting later this week about how to worry about using this if it got into the germ line, that is if actually you changed the human genome in a way that got passed to the next generation, that is if you modified an embryo. I think personally thats a bad idea. We ought not to go there. Rose but give me the horror story. Well, the horror story is that we think were so smart that we think we understand how to modify human biology and make it better. Something that evolution has been working on for 3. 85 billion years. And we think in the next two months were going to make it faster and figure out how to make a better human being. So some person somewhere decides i will take a human embryo and i will use this crisper cass thing and im going to sort of mess around and try to make that person stronger. I will reduce their chances of getk Alzheimer Disease or a heart attack by manipulating some genes. Rose what is wrong with it so far. Because we dont know what we are doing, you know. The law of unintended consequences applies to the genome it is such a complicated system. When you actually con tell plate doing that, you dont know what the consequences are. An experiment that has gone wrong, you have created a human become and a human being who may reproduce. Rose with a dna that could reproduce. And you have basically also turned a child into a commodity. Rose can you do this with you know, it works reallyth well. Rose can you do it with rats. And mice too. Rose works well. It works well. Rose why wouldnt it work with humans. I think were special, charlie. Rose i do too but. I think you are working with mice or rats in a laboratory, you end up. Do two, three generations later rats who have had this done, are they do they go crazy, are they awful. Too early to know. Rose even with rats. Yeah, because weve only really been doing this a short period of time. Rose i thought that was the reason you worked with rats, you could make it faster. You could. An even if the rats didnt act odd after three or four generations, rats and humans are very different. The other thing here. Rose when you find cures in rats it doesnt necessarily transmit to humans. And unfortunately thats often the case. People would say weve cured cancer in mice so many times now, its almost like an every day event but most of those have not transmitted to humans. Rose let me test this. Suppose there you were having a inherited disease. Uhhuh. Rose take any one of the number of them. And you had the capacity to go into an embryo, an embryo knowing that with what we know now we can go in age change whatever it is, breast cancer, okay, an inherited disease. And change it. Is that okay . Again, you dont know what other things you might be affecting. And that scenario, you can do that right now without having to use gene editing with preimpli taition, genetic diagnosis where people basically create embryos in in vitro, do a diagnostic buy op see and decides which to implant. I saw something with nih about chronic fatigue. Yeah. Rose did you write something . This just happened. I have been puzzled and frustrated about how little we understand about this condition. Now just here is the theme weve been talking about. Rose chronic fatigue. Chronic fatigue syndrome, people who have that diagnosis t is a very heterogeneous collection of individuals. But the steult of medicine has just sort of defined what we should sort of limit it too is people who are profoundly affected by fatigue, oftentimes coming on after an acute. Rose what do you mean. You cant get out of bed. You are disabled. You are utterly unable to carry out daily activities. You have other things which eer shun seems to make you worse instead of better. And you have sleep disorders, sleep is not refreshing as it should be. You may have postural hypertension where you stand up, your Blood Pressure drops and then you stand up it is serious stuff. And its particularly frustrating to see cases and there are hundreds of thousands of them, of people who were healthy and then have what appears to be just a flulike illness, but they go to bed and then at the cant get up for months. So we Just Announced we are going to make a big push to try to get the answer here. Bring some of these new technologies, of againomics and metanlommics and figure out what is going on in this decision, and if we understand that maybe we would understand. Why do people on chemotherapy get fatigue. We dont really know, wouldnt it be nice to have that answer. There are so many things you can start to ask about the technology we have in front of us now. You also said an aids Free Generation is possible in the next few decades. An aids Fr