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So let me ask for a show of hands.

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How many people here are over the age of 48?

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Well, there do seem to be a few.

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Well, congratulations,

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because if you look at this particular slide of U.S. life expectancy,

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you are now in excess of the average life span

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of somebody who was born in 1900.

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But look what happened in the course of that century.

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If you follow that curve,

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you'll see that it starts way down there.

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There's that dip there for the 1918 flu.

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And here we are at 2010,

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average life expectancy of a child born today, age 79,

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and we are not done yet.

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Now, that's the good news.

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But there's still a lot of work to do.

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So, for instance, if you ask,

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how many diseases do we now know

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the exact molecular basis?

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Turns out it's about 4,000, which is pretty amazing,

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because most of those molecular discoveries

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have just happened in the last little while.

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It's exciting to see that in terms of what we've learned,

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but how many of those 4,000 diseases

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now have treatments available?

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Only about 250.

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So we have this huge challenge, this huge gap.

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You would think this wouldn't be too hard,

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that we would simply have the ability

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to take this fundamental information that we're learning

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about how it is that basic biology teaches us

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about the causes of disease

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and build a bridge across this yawning gap

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between what we've learned about basic science

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and its application,

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a bridge that would look maybe something like this,

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where you'd have to put together a nice shiny way

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to get from one side to the other.

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Well, wouldn't it be nice if it was that easy?

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Unfortunately, it's not.

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In reality, trying to go from fundamental knowledge

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to its application is more like this.

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There are no shiny bridges.

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You sort of place your bets.

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Maybe you've got a swimmer and a rowboat

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and a sailboat and a tugboat

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and you set them off on their way,

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and the rains come and the lightning flashes,

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and oh my gosh, there are sharks in the water

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and the swimmer gets into trouble,

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and, uh oh, the swimmer drowned

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and the sailboat capsized,

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and that tugboat, well, it hit the rocks,

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and maybe if you're lucky, somebody gets across.

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Well, what does this really look like?

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Well, what is it to make a therapeutic, anyway?

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What's a drug? A drug is made up

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of a small molecule of hydrogen, carbon,

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oxygen, nitrogen, and a few other atoms

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all cobbled together in a shape,

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and it's those shapes that determine whether, in fact,

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that particular drug is going to hit its target.

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Is it going to land where it's supposed to?

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So look at this picture here -- a lot of shapes dancing around for you.

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Now what you need to do, if you're trying to develop

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a new treatment for autism

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or Alzheimer's disease or cancer

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is to find the right shape in that mix

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that will ultimately provide benefit and will be safe.

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And when you look at what happens to that pipeline,

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you start out maybe with thousands,

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tens of thousands of compounds.

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You weed down through various steps

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that cause many of these to fail.

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Ultimately, maybe you can run a clinical trial with four or five of these,

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and if all goes well, 14 years after you started,

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you will get one approval.

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And it will cost you upwards of a billion dollars

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for that one success.

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So we have to look at this pipeline the way an engineer would,

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and say, "How can we do better?"

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And that's the main theme of what I want to say to you this morning.

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How can we make this go faster?

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How can we make it more successful?

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Well, let me tell you about a few examples

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where this has actually worked.

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One that has just happened in the last few months

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is the successful approval of a drug for cystic fibrosis.

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But it's taken a long time to get there.

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Cystic fibrosis had its molecular cause discovered in 1989

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by my group working with another group in Toronto,

92
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discovering what the mutation was in a particular gene

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on chromosome 7.

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That picture you see there?

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Here it is. That's the same kid.

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That's Danny Bessette, 23 years later,

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because this is the year,

98
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and it's also the year where Danny got married,

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where we have, for the first time, the approval by the FDA

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of a drug that precisely targets the defect in cystic fibrosis

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based upon all this molecular understanding.

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That's the good news.

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The bad news is, this drug doesn't actually treat all cases of cystic fibrosis,

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and it won't work for Danny, and we're still waiting

105
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for that next generation to help him.

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But it took 23 years to get this far. That's too long.

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How do we go faster?

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Well, one way to go faster is to take advantage of technology,

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and a very important technology that we depend on

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for all of this is the human genome,

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the ability to be able to look at a chromosome,

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to unzip it, to pull out all the DNA,

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and to be able to then read out the letters in that DNA code,

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the A's, C's, G's and T's

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that are our instruction book and the instruction book for all living things,

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and the cost of doing this,

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which used to be in the hundreds of millions of dollars,

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has in the course of the last 10 years

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fallen faster than Moore's Law, down to the point

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where it is less than 10,000 dollars today to have your genome sequenced, or mine,

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and we're headed for the $1,000 genome fairly soon.

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Well, that's exciting.

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How does that play out in terms of application to a disease?

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I want to tell you about another disorder.

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This one is a disorder which is quite rare.

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It's called Hutchinson-Gilford progeria,

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and it is the most dramatic form of premature aging.

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Only about one in every four million kids has this disease,

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and in a simple way, what happens is,

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because of a mutation in a particular gene,

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a protein is made that's toxic to the cell

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and it causes these individuals to age

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at about seven times the normal rate.

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Let me show you a video of what that does to the cell.

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The normal cell, if you looked at it under the microscope,

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would have a nucleus sitting in the middle of the cell,

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which is nice and round and smooth in its boundaries

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and it looks kind of like that.

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A progeria cell, on the other hand,

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because of this toxic protein called progerin,

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has these lumps and bumps in it.

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So what we would like to do after discovering this

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back in 2003

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is to come up with a way to try to correct that.

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Well again, by knowing something about the molecular pathways,

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it was possible to pick

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one of those many, many compounds that might have been useful

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and try it out.

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In an experiment done in cell culture

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and shown here in a cartoon,

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if you take that particular compound

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and you add it to that cell that has progeria,

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and you watch to see what happened,

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in just 72 hours, that cell becomes,

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for all purposes that we can determine,

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almost like a normal cell.

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Well that was exciting, but would it actually work in a real human being?

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This has led, in the space of only four years

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from the time the gene was discovered to the start of a clinical trial,

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to a test of that very compound.

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And the kids that you see here

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all volunteered to be part of this,

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28 of them,

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and you can see as soon as the picture comes up

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that they are in fact a remarkable group of young people

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all afflicted by this disease,

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all looking quite similar to each other.

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And instead of telling you more about it,

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I'm going to invite one of them, Sam Berns from Boston,

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who's here this morning, to come up on the stage

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and tell us about his experience

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as a child affected with progeria.

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Sam is 15 years old. His parents, Scott Berns and Leslie Gordon,

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both physicians, are here with us this morning as well.

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Sam, please have a seat.

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(Applause)

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So Sam, why don't you tell these folks

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what it's like being affected with this condition called progeria?

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Sam Burns: Well, progeria limits me in some ways.

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I cannot play sports or do physical activities,

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but I have been able to take interest in things

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that progeria, luckily, does not limit.

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But when there is something that I really do want to do

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that progeria gets in the way of, like marching band

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or umpiring, we always find a way to do it,

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and that just shows that progeria isn't in control of my life.

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(Applause)

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Francis Collins: So what would you like to say to researchers

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here in the auditorium and others listening to this?

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What would you say to them both about research on progeria

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and maybe about other conditions as well?

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SB: Well, research on progeria has come so far

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in less than 15 years,

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and that just shows the drive that researchers can have

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to get this far, and it really means a lot

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to myself and other kids with progeria,

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and it shows that if that drive exists,

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anybody can cure any disease,

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and hopefully progeria can be cured in the near future,

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and so we can eliminate those 4,000 diseases

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that Francis was talking about.

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FC: Excellent. So Sam took the day off from school today

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to be here, and he is — (Applause) --

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He is, by the way, a straight-A+ student in the ninth grade

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in his school in Boston.

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Please join me in thanking and welcoming Sam.

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SB: Thank you very much. FC: Well done. Well done, buddy.

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(Applause)

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So I just want to say a couple more things

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about that particular story, and then try to generalize

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how could we have stories of success

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all over the place for these diseases, as Sam says,

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these 4,000 that are waiting for answers.

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You might have noticed that the drug

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that is now in clinical trial for progeria

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is not a drug that was designed for that.

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It's such a rare disease, it would be hard for a company

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to justify spending hundreds of millions of dollars to generate a drug.

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This is a drug that was developed for cancer.

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Turned out, it didn't work very well for cancer,

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but it has exactly the right properties, the right shape,

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to work for progeria, and that's what's happened.

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Wouldn't it be great if we could do that more systematically?

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Could we, in fact, encourage all the companies that are out there

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that have drugs in their freezers

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that are known to be safe in humans

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but have never actually succeeded in terms

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of being effective for the treatments they were tried for?

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Now we're learning about all these new molecular pathways --

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some of those could be repositioned or repurposed,

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or whatever word you want to use, for new applications,

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basically teaching old drugs new tricks.

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That could be a phenomenal, valuable activity.

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We have many discussions now between NIH and companies

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about doing this that are looking very promising.

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And you could expect quite a lot to come from this.

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There are quite a number of success stories one can point to

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about how this has led to major advances.

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The first drug for HIV/AIDS

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was not developed for HIV/AIDS.

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It was developed for cancer. It was AZT.

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It didn't work very well for cancer, but became

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the first successful antiretroviral,

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and you can see from the table there are others as well.

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So how do we actually make that a more generalizable effort?

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Well, we have to come up with a partnership

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between academia, government, the private sector,

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and patient organizations to make that so.

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At NIH, we have started this new

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National Center for Advancing Translational Sciences.

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It just started last December, and this is one of its goals.

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Let me tell you another thing we could do.

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Wouldn't it be nice to be able to a test a drug

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to see if it's effective and safe

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without having to put patients at risk,

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because that first time you're never quite sure?

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How do we know, for instance, whether drugs are safe

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before we give them to people? We test them on animals.

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And it's not all that reliable, and it's costly,

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and it's time-consuming.

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Suppose we could do this instead on human cells.

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You probably know, if you've been paying attention

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to some of the science literature

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that you can now take a skin cell

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and encourage it to become a liver cell

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or a heart cell or a kidney cell or a brain cell for any of us.

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So what if you used those cells as your test

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for whether a drug is going to work and whether it's going to be safe?

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Here you see a picture of a lung on a chip.

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This is something created by the Wyss Institute in Boston,

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and what they have done here, if we can run the little video,

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is to take cells from an individual,

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turn them into the kinds of cells that are present in the lung,

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and determine what would happen

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if you added to this various drug compounds

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to see if they are toxic or safe.

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You can see this chip even breathes.

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It has an air channel. It has a blood channel.

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And it has cells in between

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that allow you to see what happens when you add a compound.

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Are those cells happy or not?

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You can do this same kind of chip technology

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for kidneys, for hearts, for muscles,

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all the places where you want to see whether a drug

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is going to be a problem, for the liver.

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And ultimately, because you can do this for the individual,

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we could even see this moving to the point

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where the ability to develop and test medicines

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will be you on a chip, what we're trying to say here is

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the individualizing of the process of developing drugs

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and testing their safety.

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So let me sum up.

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We are in a remarkable moment here.

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For me, at NIH now for almost 20 years,

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there has never been a time where there was more excitement

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about the potential that lies in front of us.

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We have made all these discoveries

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pouring out of laboratories across the world.

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What do we need to capitalize on this? First of all, we need resources.

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This is research that's high-risk, sometimes high-cost.

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The payoff is enormous, both in terms of health

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and in terms of economic growth. We need to support that.

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Second, we need new kinds of partnerships

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between academia and government and the private sector

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and patient organizations, just like the one I've been describing here,

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in terms of the way in which we could go after repurposing new compounds.

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And third, and maybe most important, we need talent.

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We need the best and the brightest

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from many different disciplines to come and join this effort --

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all ages, all different groups --

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because this is the time, folks.

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This is the 21st-century biology that you've been waiting for,

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and we have the chance to take that

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and turn it into something which will, in fact,

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knock out disease. That's my goal.

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I hope that's your goal.

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I think it'll be the goal of the poets and the muppets

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and the surfers and the bankers

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and all the other people who join this stage

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and think about what we're trying to do here

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and why it matters.

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It matters for now. It matters as soon as possible.

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If you don't believe me, just ask Sam.

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Thank you all very much.

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(Applause)