I'm going to talk to you today about hopefully converting fear into
hope. When we go to the physician today, when we go to the doctor's
office and we walk in, there are words that we just don't want to
hear. There are words that we're truly afraid of. Diabetes, cancer,
Parkinson's, Alzheimer's, heart failure, lung failure. Things that
we know are debilitating diseases, for which there's relatively
little that can be done.
And what I want to lay out for you
today is a different way of thinking about how to treat debilitating
disease, why it's important. Why without it, perhaps, our health
care system will melt down, if you think it already hasn't. And
where we are clinically today, and where we might go tomorrow, and
what some of the hurdles are. And we're going to do all of that in
18 minutes, I promise.
I want to start with this slide,
because this slide sort of tells the story the way Science Magazine
thinks of it. This was an issue from 2002 that they published with a
lot of different articles on the bionic human. It was basically a
regenerative medicine issue. Regenerative medicine is an
extraordinarily simple concept that everybody can understand. It's
simply accelerating the pace at which the body heals itself to a
clinically relevant timescale. So we know how to do this in many of
the ways that are up there. We know that if we have a damaged hip,
you can put an artificial hip in. And this is the idea that Science
Magazine used on their front cover.
This is the complete
antithesis of regenerative medicine. This is not regenerative
medicine. Regenerative medicine is what Business Week put up when
they did a story about regenerative medicine not too long ago. The
idea is that instead of figuring out how to ameliorate symptoms with
devices and drugs and the like -- and I'll come back to that theme a
few times -- instead of doing that, we will regenerate lost function
of the body by regenerating the function of organs and damaged
tissue. So that at the end of the treatment, you are the same as you
were at the beginning of the treatment.
Very few good ideas
-- if you agree that this is a good idea -- very few good ideas are
truly novel. And this is just the same. If you look back in history,
Charles Lindbergh -- who was better known for flying airplanes --
was actually one of the first people, along with Alexis Carrel, one
of the Nobel Laureates from Rockefeller, to begin to think about,
could you culture organs? And they published this book in 1937,
where they actually began to think about, what could you do in
bio-reactors to grow whole organs? We've come a long way since then.
I'm going to share with you some of the exciting work that's going
on.
But before doing that, what I'd like to do is share my
depression about the health care system and the need for this with
you. Many of the talks yesterday talked about improving the quality
of life and reducing poverty. And essentially increasing life
expectancy all around the globe. One of the challenges is that the
richer we are, the longer we live. And the longer we live, the more
expensive it is to take care of our diseases as we get older.
This is simply the wealth of a country versus the percent of
population over the age of 65. And you can basically see that the
richer a country is, the older the people are within it. Why is this
important? And why is this a particularly dramatic challenge right
now? If the average age of your population is 30, then the average
kind of disease that you have to treat is maybe a broken ankle every
now and again, maybe a little bit of asthma. If the average age in
your country is 45 to 55, now the average person is looking at
diabetes, early-onset diabetes, heart failure, coronary artery
disease. Things that are inherently more difficult to treat, and
much more expensive to treat.
Just have a look at the
demographics in the U.S. here. This is from "The Untied States of
America." In 1930, there were 41 workers per retiree. 41 people who
were basically outside of being really sick, paying for the one
retiree who was experiencing debilitating disease. In 2010, two
workers per retiree in the U.S. And this is matched in every
industrialized, wealthy country in the world. How can you actually
afford to treat patients, when the reality of getting old looks like
this?
This is age versus cost of health care. And you can see
that right around age 45, 40 to 45, there's a sudden spike in the
cost of health care. It's actually quite interesting -- if you do
the right studies, you can look at how much you as an individual
spend on your own health care, plotted over your lifetime. And about
seven years before you're about to die, there's a spike. And you can
actually -- (Laughter) -- we won't get into that. (Laughter)
There are very few things, very few things that you can really do
that will change the way that you can treat these kinds of diseases
and experience what I would call healthy aging. I'd suggest there
are four things. And none of these things include an insurance
system or a legal system. All those things do is change who pays.
They don't actually change what the actual cost of the treatment is.
One thing you can do is not treat. You can ration health care.
We won't talk about that anymore. It's too depressing. You can
prevent. Obviously a lot of monies should be put into prevention.
But perhaps most interesting, to me anyway, and most important,
is the idea of diagnosing a disease much earlier on in the
progression, and then treating the disease to cure the disease
instead of treating a symptom. Think of it in terms of diabetes, for
instance. Today, with diabetes, what do we do? We diagnose the
disease eventually, once it becomes symptomatic, and then we treat
the symptom for 10, 20, 30, 40 years. And we do OK. Insulin's a
pretty good therapy. But eventually it stops working, and diabetes
leads to a predictable onset of debilitating disease.
Why
couldn't we just inject the pancreas with something to regenerate
the pancreas early on in the disease, perhaps even before it was
symptomatic? And it might be a little bit expensive at the time that
we did it, but if it worked, we would truly be able to do something
different.
This video, I think, gets across the concept that
I'm talking about quite dramatically. This is a newt, re-growing its
limb. If a newt can do this kind of thing, why can't we? I'll
actually show you some more important features about limb
regeneration in a moment. But what we're talking about in
regenerative medicine is doing this in every organ system of the
body, for tissues and for organs themselves. So today's reality is
that if we get sick, the message is we will treat your symptoms, and
you need to adjust to a new way of life.
I would pose to you
that tomorrow -- and when tomorrow is we could debate, but it's
within the foreseeable future -- we will talk about regenerative
rehabilitation. There's a limb prosthetic up here, similar actually
to the one that the soldier that's come back from Iraq ... there are
370 soldiers that have come back from Iraq that have lost limbs.
Imagine if instead of facing that, they could actually face the
regeneration of that limb. It's a wild concept. I'll show you where
we are at the moment in working towards that concept.
But
it's applicable, again, to every organ system. How can we do that?
The way to do that is to develop a conversation with the body. We
need to learn to speak the body's language. And to switch on
processes that we knew how to do when we were a fetus. A mammalian
fetus, if it loses a limb during the first trimester of pregnancy,
will re-grow that limb. So our DNA has the capacity to do these
kinds of wound-healing mechanisms. It's a natural process, but it is
lost as we age. In a child, before the age of about six months, if
they lose their fingertip in an accident, they'll re-grow their
fingertip. By the time they're five, they won't be able to do that
anymore.
So to engage in that conversation with the body, we
need to speak the body's language. And there are certain tools in
our toolbox that allow us to do this today. I'm going to give you an
example of three of these tools through which to converse with the
body.
The first is cellular therapies. Clearly, we heal
ourselves in a natural process, using cells to do most of the work.
Therefore, if we can find the right cells and implant them in the
body, they may do the healing. Secondly, we can use materials. We
heard yesterday about the importance of new materials. If we can
invent materials, design materials, or extract materials from a
natural environment, then we might be able to have those materials
induce the body to heal itself. And finally, we may be able to use
smart devices that will offload the work of the body and allow it to
heal.
I'm going to show you an example of each of these, and
I'm going to start with materials. Steve Badylak -- who's at the
University of Pittsburgh -- about a decade ago had a remarkable
idea. And that idea was that the small intestine of a pig, if you
threw away all the cells, and if you did that in a way that allowed
it to remain biologically active, may contain all of the necessary
factors and signals that would signal the body to heal itself. And
he asked a very important question. He asked the question, if I take
that material, which is a natural material that usually induces
healing in the small intestine, and I place it somewhere else on a
person's body, would it give a tissue-specific response, or would it
make small intestine if I tried to make a new ear?
I wouldn't
be telling you this story if it wasn't compelling. The picture I'm
about to show you -- (Laughter) -- is a compelling picture. However,
for those of you that are even the slightest bit squeamish -- even
though you may not like to admit it in front of your friends -- the
lights are down. This is a good time to look at your feet, check
your Blackberry, do anything other than look at the screen.
(Laughter)
What I'm about to show you is a diabetic ulcer.
And although -- it's good to laugh before we look at this. This is
the reality of diabetes. I think a lot of times we hear about
diabetics, diabetic ulcers, we just don't connect the ulcer with the
eventual treatment, which is amputation, if you can't heal it. So
I'm going to put the slide up now. It won't be up for long. This is
a diabetic ulcer. It's tragic. The treatment for this is amputation.
This is an older lady. She has cancer of the liver as well as
diabetes, and has decided to die with what' s left of her body
intact.
And this lady decided, after a year of attempted
treatment of that ulcer, that she would try this new therapy that
Steve invented. That's what the wound looked like 11 weeks later.
That material contained only natural signals. And that material
induced the body to switch back on a healing response that it didn't
have before.
There's going to be a couple more distressing
slides for those of you -- I'll let you know when you can look
again. This is a horse. The horse is not in pain. If the horse was
in pain, I wouldn't show you this slide. The horse just has another
nostril that's developed because of a riding accident. Just a few
weeks after treatment -- in this case, taking that material, turning
it into a gel, and packing that area, and then repeating the
treatment a few times -- and the horse heals up. And if you took an
ultrasound of that area, it would look great.
Here's a
dolphin where the fin's been re-attached. There are now 400,000
patients around the world who have used that material to heal their
wounds. Could you regenerate a limb? DARPA just gave Steve 15
million dollars to lead an eight-institution project to begin the
process of asking that question.
And I'll show you the 15
million dollar picture. This is a 78 year-old man who's lost the end
of his fingertip. Remember that I mentioned before the children who
lose their fingertips. After treatment that's what it looks like.
This is happening today. This is clinically relevant today. There
are materials which do this. There are the heart patches.
But
could you go a little further? Could you, say, instead of using
material, can I take some cells along with the material, and remove
a damaged piece of tissue, put a bio-degradable material on there?
You can see here a little bit of heart muscle beating in a dish.
This was done by Teruo Okano at Tokyo Women's Hospital. He can
actually grow beating tissue in a dish. He chills the dish, it
changes its properties and he peels it right out of the dish. It's
the coolest stuff.
Now I'm going to show you cell-based
regeneration. And what I'm going to show you here is stem cells
being removed from the hip of a patient. Again, if you're squeamish,
you don't want to watch. But this one's kind of cool. So this is a
bypass operation, just like what Al Gore had, with a difference. In
this case, at the end of the bypass operation, you're going to see
the stem cells from the patient that were removed at the beginning
of the procedure being injected directly into the heart of the
patient. And I'm standing up here because at one point I'm going to
show you just how early this technology is. Here go the stem cells,
right into the beating heart of the patient. And if you look really
carefully, it's going to be right around this point you'll actually
see a back-flush. You see the cells coming back out. We need all
sorts of new technology, new devices, to get the cells to the right
place at the right time.
Just a little bit of data, a tiny
bit of data. This was a randomized trial. At this time this was an N
of 20. Now there's an N of about 100. Basically, if you take an
extremely sick patient and you give them a bypass, they get a little
bit better. If you give them stem cells as well as their bypass, for
these particular patients, they became asymptomatic. These are now
two years out. The coolest thing would be is if you could diagnose
the disease early, and prevent the onset of the disease to a bad
state.
This is the same procedure, but now done minimally
invasively, with only three holes in the body where they're taking
the heart and simply injecting stem cells through a laparoscopic
procedure. There go the cells. We don't have time to go into all of
those details, but basically, that works too. You can take patients
who are less sick, and bring them back to an almost asymptomatic
state through that kind of therapy.
Here's another example of
stem-cell therapy that isn't quite clinical yet, but I think very
soon will be. This is the work of Kacey Marra from Pittsburgh, along
with a number of colleagues around the world. They've decided that
liposuction fluid, which -- in the United States, we have a lot of
liposuction fluid. (Laughter) It's a great source of stem cells.
Stem cells are packed in that liposuction fluid. So you could go in,
you could get your tummy-tuck. Out comes the liposuction fluid, and
in this case, the stem cells are isolated and turned into neurons.
All done in the lab. And I think fairly soon, you will see patients
being treated with their own fat-derived, or adipose-derived, stem
cells.
I talked before about the use of devices to
dramatically change the way we treat disease. Here's just one
example before I close up. This is equally tragic. We have a very
abiding and heartbreaking partnership with our colleagues at the
Institute for Surgical Research in the US Army, who have to treat
the now 11,000 kids that have come back from Iraq. Many of those
patients are very severely burned.
And if there's anything
that's been learned about burn, it's that we don't know how to treat
it. Everything that is done to treat burn -- basically we do a
sodding approach. We make something over here, and then we
transplant it onto the site of the wound, and we try and get the two
to take. In this case here, a new, wearable bio-reactor has been
designed -- it should be tested clinically later this year at ISR --
by Joerg Gerlach in Pittsburgh. And that bio-reactor will lay down
in the wound bed. The gun that you see there sprays cells. That's
going to spray cells over that area. The reactor will serve to
fertilize the environment, deliver other things as well at the same
time, and therefore we will seed that lawn, as opposed to try the
sodding approach. It's a completely different way of doing it.
So my 18 minutes is up. So let me finish up with some good news,
and maybe a little bit of bad news. The good news is that this is
happening today. It's very powerful work. Clearly the images kind of
get that across. It's incredibly difficult because it's highly
inter-disciplinary. Almost every field of science engineering and
clinical practice is involved in trying to get this to happen.
A number of governments, and a number of regions, have
recognized that this is a new way to treat disease. The Japanese
government were perhaps the first, when they decided to invest first
3 billion, later another 2 billion in this field. It's no
coincidence. Japan is the oldest country on earth in terms of its
average age. They need this to work or their health system dies. So
they're putting a lot of strategic investment focused in this area.
The European Union, same thing. China, the same thing. China just
launched a national tissue-engineering center. The first year budget
was 250 million US dollars.
In the United States we've had a
somewhat different approach. We -- (Laughter) -- oh, for Al Gore to
come and be in the real world as president. We've had a different
approach. And the approach has basically been to just sort of fund
things as they come along. But there's been no strategic investment
to bring all of the necessary things to bear and focus them in a
careful way.
And I'm going to finish up with a quote, maybe a
little cheap shot, at the director of the NIH, who's a very charming
man. Myself and Jay Vacanti from Harvard went to visit with him and
a number of his directors of his institute just a few months ago, to
try and convince him that it was time to take just a little piece of
that 27.5 billion dollars that he's going to get next year and focus
it, in a strategic way, to make sure we can accelerate the pace at
which these things get to patients. And at the end of a very testy
meeting, what the NIH director said is, "Your vision is larger than
our appetite." I'd like to close by saying that no one's going to
change our vision, but together we can change his appetite. Thank
you.
