Susan Solomon: The promise of research with stem cells

95,493 views ・ 2012-09-13

TED


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Translator: Joseph Geni Reviewer: Morton Bast
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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)
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