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So, embryonic stem cells

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are really incredible cells.

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They are our body's own repair kits,

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and they're pluripotent, which means they can morph into

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all of the cells in our bodies.

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Soon, we actually will be able to use stem cells

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to replace cells that are damaged or diseased.

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But that's not what I want to talk to you about,

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because right now there are some really

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extraordinary things that we are doing with stem cells

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that are completely changing

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the way we look and model disease,

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our ability to understand why we get sick,

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and even develop drugs.

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I truly believe that stem cell research is going to allow

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our children to look at Alzheimer's and diabetes

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and other major diseases the way we view polio today,

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which is as a preventable disease.

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So here we have this incredible field, which has

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enormous hope for humanity,

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but much like IVF over 35 years ago,

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until the birth of a healthy baby, Louise,

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this field has been under siege politically and financially.

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Critical research is being challenged instead of supported,

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and we saw that it was really essential to have

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private safe haven laboratories where this work

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could be advanced without interference.

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And so, in 2005,

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we started the New York Stem Cell Foundation Laboratory

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so that we would have a small organization that could

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do this work and support it.

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What we saw very quickly is the world of both medical

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research, but also developing drugs and treatments,

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is dominated by, as you would expect, large organizations,

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but in a new field, sometimes large organizations

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really have trouble getting out of their own way,

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and sometimes they can't ask the right questions,

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and there is an enormous gap that's just gotten larger

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between academic research on the one hand

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and pharmaceutical companies and biotechs

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that are responsible for delivering all of our drugs

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and many of our treatments, and so we knew that

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to really accelerate cures and therapies, we were going

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to have to address this with two things:

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new technologies and also a new research model.

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Because if you don't close that gap, you really are

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exactly where we are today.

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And that's what I want to focus on.

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We've spent the last couple of years pondering this,

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making a list of the different things that we had to do,

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and so we developed a new technology,

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It's software and hardware,

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that actually can generate thousands and thousands of

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genetically diverse stem cell lines to create

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a global array, essentially avatars of ourselves.

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And we did this because we think that it's actually going

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to allow us to realize the potential, the promise,

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of all of the sequencing of the human genome,

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but it's going to allow us, in doing that,

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to actually do clinical trials in a dish with human cells,

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not animal cells, to generate drugs and treatments

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that are much more effective, much safer,

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much faster, and at a much lower cost.

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So let me put that in perspective for you

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and give you some context.

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This is an extremely new field.

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In 1998, human embryonic stem cells

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were first identified, and just nine years later,

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a group of scientists in Japan were able to take skin cells

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and reprogram them with very powerful viruses

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to create a kind of pluripotent stem cell

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called an induced pluripotent stem cell,

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or what we refer to as an IPS cell.

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This was really an extraordinary advance, because

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although these cells are not human embryonic stem cells,

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which still remain the gold standard,

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they are terrific to use for modeling disease

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and potentially for drug discovery.

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So a few months later, in 2008, one of our scientists

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built on that research. He took skin biopsies,

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this time from people who had a disease,

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ALS, or as you call it in the U.K., motor neuron disease.

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He turned them into the IPS cells

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that I've just told you about, and then he turned those

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IPS cells into the motor neurons that actually

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were dying in the disease.

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So basically what he did was to take a healthy cell

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and turn it into a sick cell,

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and he recapitulated the disease over and over again

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in the dish, and this was extraordinary,

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because it was the first time that we had a model

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of a disease from a living patient in living human cells.

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And as he watched the disease unfold, he was able

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to discover that actually the motor neurons were dying

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in the disease in a different way than the field

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had previously thought. There was another kind of cell

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that actually was sending out a toxin

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and contributing to the death of these motor neurons,

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and you simply couldn't see it

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until you had the human model.

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So you could really say that

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researchers trying to understand the cause of disease

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without being able to have human stem cell models

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were much like investigators trying to figure out

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what had gone terribly wrong in a plane crash

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without having a black box, or a flight recorder.

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They could hypothesize about what had gone wrong,

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but they really had no way of knowing what led

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to the terrible events.

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And stem cells really have given us the black box

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for diseases, and it's an unprecedented window.

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It really is extraordinary, because you can recapitulate

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many, many diseases in a dish, you can see

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what begins to go wrong in the cellular conversation

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well before you would ever see

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symptoms appear in a patient.

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And this opens up the ability,

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which hopefully will become something that

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is routine in the near term,

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of using human cells to test for drugs.

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Right now, the way we test for drugs is pretty problematic.

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To bring a successful drug to market, it takes, on average,

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13 years — that's one drug —

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with a sunk cost of 4 billion dollars,

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and only one percent of the drugs that start down that road

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are actually going to get there.

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You can't imagine other businesses

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that you would think of going into

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that have these kind of numbers.

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It's a terrible business model.

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But it is really a worse social model because of

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what's involved and the cost to all of us.

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So the way we develop drugs now

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is by testing promising compounds on --

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We didn't have disease modeling with human cells,

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so we'd been testing them on cells of mice

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or other creatures or cells that we engineer,

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but they don't have the characteristics of the diseases

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that we're actually trying to cure.

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You know, we're not mice, and you can't go into

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a living person with an illness

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and just pull out a few brain cells or cardiac cells

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and then start fooling around in a lab to test

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for, you know, a promising drug.

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But what you can do with human stem cells, now,

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is actually create avatars, and you can create the cells,

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whether it's the live motor neurons

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or the beating cardiac cells or liver cells

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or other kinds of cells, and you can test for drugs,

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promising compounds, on the actual cells

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that you're trying to affect, and this is now,

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and it's absolutely extraordinary,

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and you're going to know at the beginning,

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the very early stages of doing your assay development

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and your testing, you're not going to have to wait 13 years

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until you've brought a drug to market, only to find out

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that actually it doesn't work, or even worse, harms people.

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But it isn't really enough just to look at

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the cells from a few people or a small group of people,

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because we have to step back.

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We've got to look at the big picture.

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Look around this room. We are all different,

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and a disease that I might have,

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if I had Alzheimer's disease or Parkinson's disease,

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it probably would affect me differently than if

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one of you had that disease,

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and if we both had Parkinson's disease,

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and we took the same medication,

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but we had different genetic makeup,

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we probably would have a different result,

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and it could well be that a drug that worked wonderfully

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for me was actually ineffective for you,

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and similarly, it could be that a drug that is harmful for you

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is safe for me, and, you know, this seems totally obvious,

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but unfortunately it is not the way

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that the pharmaceutical industry has been developing drugs

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because, until now, it hasn't had the tools.

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And so we need to move away

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from this one-size-fits-all model.

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The way we've been developing drugs is essentially

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like going into a shoe store,

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no one asks you what size you are, or

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if you're going dancing or hiking.

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They just say, "Well, you have feet, here are your shoes."

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It doesn't work with shoes, and our bodies are

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many times more complicated than just our feet.

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So we really have to change this.

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There was a very sad example of this in the last decade.

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There's a wonderful drug, and a class of drugs actually,

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but the particular drug was Vioxx, and

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for people who were suffering from severe arthritis pain,

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the drug was an absolute lifesaver,

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but unfortunately, for another subset of those people,

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they suffered pretty severe heart side effects,

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and for a subset of those people, the side effects were

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so severe, the cardiac side effects, that they were fatal.

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But imagine a different scenario,

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where we could have had an array, a genetically diverse array,

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of cardiac cells, and we could have actually tested

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that drug, Vioxx, in petri dishes, and figured out,

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well, okay, people with this genetic type are going to have

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cardiac side effects, people with these genetic subgroups

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or genetic shoes sizes, about 25,000 of them,

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are not going to have any problems.

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The people for whom it was a lifesaver

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could have still taken their medicine.

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The people for whom it was a disaster, or fatal,

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would never have been given it, and

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you can imagine a very different outcome for the company,

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who had to withdraw the drug.

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So that is terrific,

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and we thought, all right,

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as we're trying to solve this problem,

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clearly we have to think about genetics,

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we have to think about human testing,

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but there's a fundamental problem,

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because right now, stem cell lines,

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as extraordinary as they are,

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and lines are just groups of cells,

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they are made by hand, one at a time,

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and it takes a couple of months.

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This is not scalable, and also when you do things by hand,

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even in the best laboratories,

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you have variations in techniques,

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and you need to know, if you're making a drug,

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that the Aspirin you're going to take out of the bottle

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on Monday is the same as the Aspirin

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that's going to come out of the bottle on Wednesday.

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So we looked at this, and we thought, okay,

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artisanal is wonderful in, you know, your clothing

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and your bread and crafts, but

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artisanal really isn't going to work in stem cells,

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so we have to deal with this.

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But even with that, there still was another big hurdle,

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and that actually brings us back to

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the mapping of the human genome, because

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we're all different.

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We know from the sequencing of the human genome

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that it's shown us all of the A's, C's, G's and T's

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that make up our genetic code,

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but that code, by itself, our DNA,

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is like looking at the ones and zeroes of the computer code

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without having a computer that can read it.

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It's like having an app without having a smartphone.

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We needed to have a way of bringing the biology

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to that incredible data,

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and the way to do that was to find

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a stand-in, a biological stand-in,

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that could contain all of the genetic information,

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but have it be arrayed in such a way

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as it could be read together

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and actually create this incredible avatar.

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We need to have stem cells from all the genetic sub-types

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that represent who we are.

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So this is what we've built.

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It's an automated robotic technology.

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It has the capacity to produce thousands and thousands

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of stem cell lines. It's genetically arrayed.

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It has massively parallel processing capability,

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and it's going to change the way drugs are discovered,

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we hope, and I think eventually what's going to happen

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is that we're going to want to re-screen drugs,

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on arrays like this, that already exist,

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all of the drugs that currently exist,

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and in the future, you're going to be taking drugs

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and treatments that have been tested for side effects

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on all of the relevant cells,

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on brain cells and heart cells and liver cells.

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It really has brought us to the threshold

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of personalized medicine.

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It's here now, and in our family,

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my son has type 1 diabetes,

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which is still an incurable disease,

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and I lost my parents to heart disease and cancer,

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but I think that my story probably sounds familiar to you,

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because probably a version of it is your story.

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At some point in our lives, all of us,

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or people we care about, become patients,

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and that's why I think that stem cell research

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is incredibly important for all of us.

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Thank you. (Applause)

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(Applause)