it’s 1920 you find yourself desperately
thirsty you’re making frequent trips to
the bathroom and you’re basically
wasting away you go to your doctor and
he tells you that you have high levels
of sugar in your urine then he gives you
a dire diagnosis you have diabetes
mellitus or type 1 diabetes as we call
it today and Greek mellitus means sweet
but back then there is nothing sweet
about this diagnosis it was a death
sentence
type 1 diabetes is an autoimmune disease
that results in the destruction of a
cell called the beta cell that resides
in the pancreas the beta cell is part of
a cell structure called the islets of
langerhans and it is responsible for
producing insulin many of you have
probably heard about insulin it’s a very
critical hormone that is a response for
regulating your blood sugar levels and
those blood sugar levels must be kept
within a very narrow range in for good
health in life type 1 diabetes unlike
type 2 which you’ve probably heard about
is a there’s many more patients out
there with type 2 diabetes
but that’s tends to be sometimes a
lifestyle disease not always but tends
to be in contrast type 1 diabetes hits
basically without warning young and old
and once it’s established it’s a
lifelong disease a hundred years ago
that life was heartbreakingly short what
I show here is a child with type 1
diabetes back then a child usually died
within one year after diagnosis adults
maybe two years or a little bit longer
this all changed in 1921 when two
Canadian physician scientists by the
name of dr. Frederick Banting and his
Institute in cysts and Charles best
isolated insulin from the pancreas of
dogs and showed that that insulin was
able to control the blood sugar levels
this important groundbreaking discovery
led to a Nobel Prize for dr. Banting but
also quickly led to the use of insulin
in humans and this changed type 1
diabetes from a fatal disease to a
chronic disease here we now see that
same child just two months after
starting a therapy with insulin so you
can see a remarkable recovery the use of
insulin was historic and momentous
discovery in the use of it but it still
was not a cure not even close
it really changed a death sentence to a
life sentence patients with type 1
diabetes still had a chronic disease
that they had to continuously manage in
order to to control it to survive these
patients have to monitor and control
their diet track their physical activity
measure their blood sugar levels
throughout the day and then choose the
right amount of insulin to eject
throughout the day to maintain that
blood sugar level fast-forward to today
a hundred years later we really haven’t
changed the way we treat this disease
we’re still basically doing the same
thing we’ve gotten better we have better
ways of measuring the blood sugar levels
we have better insulins we have board of
methods to administer it more convenient
methods to administer it but the end of
the day we’re still doing exactly the
same thing we’re injecting insulin and
trying to get it just right what I’m
going to show you is is really kind of
give you a feel for what it means to be
a type 1 diabetic so what I have here
what I’m going to dump out here
represents all of the injections that a
type 1 diabetic has to take throughout a
year keep in mind this is a lifelong
disease so multiply this by decades add
to this the constant threat of getting
the insulin dosage wrong and that can
lead to life-threatening bouts of low
blood sugar and you can see that type 1
diabetes has a major impact on the
of life let’s watch a video of a
caregiver talk about that impact that
day-to-day struggle with type 1 diabetes
it’s a fatal disease
I call it spending your life not dying I
think that’s such a strong statement
spending your life not dying and that
really is kind of what it’s like
patients will tell you that barely a
moment passes by during the day that
they don’t think about this disease and
it’s especially hard on on loved ones
especially parents of children with type
1 diabetes you can imagine trying to
inject your child with this insulin on a
regular basis the day to day impact of
this disease is bad enough but to add
insult to injury or in this case injury
to insult even with very careful
management type 1 diabetes exacts a
heavy toll in the form of long term
complications this includes heart
disease stroke blindness reap kidney
disease foot officers and even
amputation due to vascular disease sadly
the life expectancy for a patient that
has type 1 diabetes is decreased by an
average of a decade or more patients
need a cure in fact they’re desperate
for cure let’s listen to a video of a
patient that’s enrolled in a clinical
trial my company is running right now
talk about what a cure means to him you
spend your entire life wishing for one
thing above all else if someone said
we’ll give you 50 million dollars or the
cure for diabetes I wouldn’t have to
think about it I can tell you right now
so the introduction of insulin was not
only important for managing disease it
also gave us critical insights into the
underlying cause this is critten this is
what combining this understanding the
basic research the basic knowledge of
the disease with breakthrough technology
is what will get us to a cure and that
that combination is often called
translational research or translational
medicine the promise of translational
research has been a real driving force
in my career as a scientist it prompted
me to leave academia about thirty years
ago to become a one of the pioneering
people in the biotechnology industry and
founding one of the early biotechnology
companies here in San Diego those were
really exciting times we were working on
the forefront of new fields interacting
with the best and brightest throughout
the areas and I really found it
intoxicating and I went on to become a
founder of a number of other
biotechnology companies here in town
however it’s not always easy being on
the cutting edge seldom is I can give
you an example one company I was a
founder of which was called vying vie
gene was one of the early first dream
therapy companies gene therapy is a
tremendously exciting technology but we
were way way ahead of our time
gene therapy today was just got to its
first product approval last year by
another company a full 25 years after we
started buying six years ago I was
presented another opportunity to push
the boundaries of science and medicine a
recruiter called me and told me told me
a compelling story about a local company
called via site via Seitz mission was
nothing short of delivering a cure for
type 1 diabetes using a pioneering
regenerative medicine approach I was
intrigued this was an opportunity to not
just work on a treatment which is what
we often do in my industry but actually
to deliver a cure something that’s
pretty rare after meeting with the
dedicated and talented team at via site
I was I found a work that they were
doing the decade or more that they had
spent on the pioneering work was very
convincing and it led me to join for the
first time a company and run a company
that I haven’t actually started so how
are we going to deliver cure so if your
call I said that the problem with type 1
diabetes is a destruction of an
autoimmune destruction of the beta cell
so if the problem is a loss of a beta
cell then maybe we can really can cure
that problem with a replacement cell as
it turns out about 18 years ago
another amazing Canadian physician
scientist by the name of dr. James
Shapiro showed the world that a cure for
type 1 diabetes is indeed possible by
replacing that missing cell what dr.
Shapiro and his team did was they
isolated islet issue from pancreases
including beta cells from pancreases
taken from cadavers human cadavers and
they infuse that islet tissue into the
liver of patients with type 1 diabetes
excitingly many of these cells would
take up residence and be able to produce
insulin in normal fashion and correct a
disease in fact these patients who
receive cadaver islet transplants are
now most of them will go for 5 years or
longer completely insulin independent
essentially functionally cured so that
was very exciting
however cadaver islet transplant has its
own set of severe limitations the
biggest limitation is just a shortage of
cells this has to come from cadaver from
pay courses taken from cadavers in the
u.s. there’s only about 1,500 believe it
or not only about 1,500 pancreas is
available each year and that it would be
suitable also these patients have to
take continuous immuno suppression and
it’s very costly as a result of these
limitation while there are millions of
people out there that have type 1
diabetes only approximately 2000
patients have been treated with cadaver
so our ambitious goal is to overcome all
of these limitations and make a cure
available for all patients we have a lot
of help on this we work with the
California Institute regenitive medicine
we work with JDRF which was formerly
known as joyed Juvenile Diabetes
Research Foundation clinical
investigators and importantly with
amazing patient volunteers and we’re on
a path to make this cure happen so
tackle the issue is supply we are using
another emerging exciting technology
that you actually heard a little bit
about earlier about earlier which is the
use of an embryonic stem cell line
embryonic stem cells have the unique
capability of being able to transform
into any cell in the body moreover these
cells are essentially immortal meaning
that a single cell line that we have
banked gives us sufficient material to
produce replacement cells for
essentially all patients so by
discovering how a embryonic stem cell
naturally becomes a beta cell our
scientists developed methods to drive
these cells to become pancreatic
progenitor cells that we then implant
into a subject and those cells further
mature to produce insulin and other
factors from the normal human islet and
correct correct a disease that may sound
relatively straightforward but this
slide one slide here represents about
ten years of work and a couple hundred
issued patents that came as a result of
it so having the cells is important but
it’s only part of the challenge we also
need a way to conveniently safely and
effectively deliver these cells to the
patient so for that we developed a
device technology basically a device
that we could fill with these cells
implant under the skin and then those
cells would mature in the device and
begin producing the insulin and other
factors now where we wanted more than
just delivery we also want to protect
these cells from the immune rejection so
that we can eliminate the need for
thus was born the encapture cell
delivery device the device has a
membrane a semipermeable membrane that
traps the cells inside the membrane
allows the passage of insulin and other
protein hormones in back into the
bloodstream you can think of this as
like a tea bag the cells we put inside
are like the tea leaves and the insulin
is the tea essence that is coming out
the membrane provides a barrier against
a patient’s immune system and therefore
protects these cells from immune
rejection and eliminates that need for
the immuno suppression the cells and the
device together make up a product
candidate that we call Peck and cap Peck
and cap has been tested in animal models
of type on diabetes and shown to be very
effective in fact when we take these
human cells and we put them into an
animal we essentially humanize that
animal in terms of its blood sugar
regulation meaning that that animal will
now regulate its blood sugar to the
human setpoint rather than the normal
set point of that animal so it’s very
effective in those models we’ve now
moved on to testing this product
candidate in patients with type 1
diabetes a big step forward for any
program it’s early days and it’s
certainly too soon for us to claim
victory over type 1 however should we be
successful and I believe we will be then
about a hundred years almost exactly a
hundred years after type 1 diabetes went
from a fatal disease to a chronic
disease we’ll deliver the next big step
a functional cure that can be used by
all patients with the disease what this
means is when we’re successful instead
of the daily grind of trying to inject
insulin and keep that blood sugar under
control and prevent those long term of
complications a patient will receive a
dose of stem cell derived islets in a
device placed under the skin that could
last for years
that means we go from this big pile of
syringes you see next to me here
– essentially a device that looks like
this this is actually the encapture cell
delivery device that would be filled
with the cells and implanted under the
skin a big difference the regenerative
medicine approach that we’re pioneering
at our company is has tremendous
potential not just for diabetes but for
many other diseases our goal right now
though is to prove that potential by
delivering a functional cure to all
patients with diabetes we shared a goal
of JDRF we want to live in a world where
type 1 goes to type none thank you [Applause]
thirsty you’re making frequent trips to
the bathroom and you’re basically
wasting away you go to your doctor and
he tells you that you have high levels
of sugar in your urine then he gives you
a dire diagnosis you have diabetes
mellitus or type 1 diabetes as we call
it today and Greek mellitus means sweet
but back then there is nothing sweet
about this diagnosis it was a death
sentence
type 1 diabetes is an autoimmune disease
that results in the destruction of a
cell called the beta cell that resides
in the pancreas the beta cell is part of
a cell structure called the islets of
langerhans and it is responsible for
producing insulin many of you have
probably heard about insulin it’s a very
critical hormone that is a response for
regulating your blood sugar levels and
those blood sugar levels must be kept
within a very narrow range in for good
health in life type 1 diabetes unlike
type 2 which you’ve probably heard about
is a there’s many more patients out
there with type 2 diabetes
but that’s tends to be sometimes a
lifestyle disease not always but tends
to be in contrast type 1 diabetes hits
basically without warning young and old
and once it’s established it’s a
lifelong disease a hundred years ago
that life was heartbreakingly short what
I show here is a child with type 1
diabetes back then a child usually died
within one year after diagnosis adults
maybe two years or a little bit longer
this all changed in 1921 when two
Canadian physician scientists by the
name of dr. Frederick Banting and his
Institute in cysts and Charles best
isolated insulin from the pancreas of
dogs and showed that that insulin was
able to control the blood sugar levels
this important groundbreaking discovery
led to a Nobel Prize for dr. Banting but
also quickly led to the use of insulin
in humans and this changed type 1
diabetes from a fatal disease to a
chronic disease here we now see that
same child just two months after
starting a therapy with insulin so you
can see a remarkable recovery the use of
insulin was historic and momentous
discovery in the use of it but it still
was not a cure not even close
it really changed a death sentence to a
life sentence patients with type 1
diabetes still had a chronic disease
that they had to continuously manage in
order to to control it to survive these
patients have to monitor and control
their diet track their physical activity
measure their blood sugar levels
throughout the day and then choose the
right amount of insulin to eject
throughout the day to maintain that
blood sugar level fast-forward to today
a hundred years later we really haven’t
changed the way we treat this disease
we’re still basically doing the same
thing we’ve gotten better we have better
ways of measuring the blood sugar levels
we have better insulins we have board of
methods to administer it more convenient
methods to administer it but the end of
the day we’re still doing exactly the
same thing we’re injecting insulin and
trying to get it just right what I’m
going to show you is is really kind of
give you a feel for what it means to be
a type 1 diabetic so what I have here
what I’m going to dump out here
represents all of the injections that a
type 1 diabetic has to take throughout a
year keep in mind this is a lifelong
disease so multiply this by decades add
to this the constant threat of getting
the insulin dosage wrong and that can
lead to life-threatening bouts of low
blood sugar and you can see that type 1
diabetes has a major impact on the
of life let’s watch a video of a
caregiver talk about that impact that
day-to-day struggle with type 1 diabetes
it’s a fatal disease
I call it spending your life not dying I
think that’s such a strong statement
spending your life not dying and that
really is kind of what it’s like
patients will tell you that barely a
moment passes by during the day that
they don’t think about this disease and
it’s especially hard on on loved ones
especially parents of children with type
1 diabetes you can imagine trying to
inject your child with this insulin on a
regular basis the day to day impact of
this disease is bad enough but to add
insult to injury or in this case injury
to insult even with very careful
management type 1 diabetes exacts a
heavy toll in the form of long term
complications this includes heart
disease stroke blindness reap kidney
disease foot officers and even
amputation due to vascular disease sadly
the life expectancy for a patient that
has type 1 diabetes is decreased by an
average of a decade or more patients
need a cure in fact they’re desperate
for cure let’s listen to a video of a
patient that’s enrolled in a clinical
trial my company is running right now
talk about what a cure means to him you
spend your entire life wishing for one
thing above all else if someone said
we’ll give you 50 million dollars or the
cure for diabetes I wouldn’t have to
think about it I can tell you right now
so the introduction of insulin was not
only important for managing disease it
also gave us critical insights into the
underlying cause this is critten this is
what combining this understanding the
basic research the basic knowledge of
the disease with breakthrough technology
is what will get us to a cure and that
that combination is often called
translational research or translational
medicine the promise of translational
research has been a real driving force
in my career as a scientist it prompted
me to leave academia about thirty years
ago to become a one of the pioneering
people in the biotechnology industry and
founding one of the early biotechnology
companies here in San Diego those were
really exciting times we were working on
the forefront of new fields interacting
with the best and brightest throughout
the areas and I really found it
intoxicating and I went on to become a
founder of a number of other
biotechnology companies here in town
however it’s not always easy being on
the cutting edge seldom is I can give
you an example one company I was a
founder of which was called vying vie
gene was one of the early first dream
therapy companies gene therapy is a
tremendously exciting technology but we
were way way ahead of our time
gene therapy today was just got to its
first product approval last year by
another company a full 25 years after we
started buying six years ago I was
presented another opportunity to push
the boundaries of science and medicine a
recruiter called me and told me told me
a compelling story about a local company
called via site via Seitz mission was
nothing short of delivering a cure for
type 1 diabetes using a pioneering
regenerative medicine approach I was
intrigued this was an opportunity to not
just work on a treatment which is what
we often do in my industry but actually
to deliver a cure something that’s
pretty rare after meeting with the
dedicated and talented team at via site
I was I found a work that they were
doing the decade or more that they had
spent on the pioneering work was very
convincing and it led me to join for the
first time a company and run a company
that I haven’t actually started so how
are we going to deliver cure so if your
call I said that the problem with type 1
diabetes is a destruction of an
autoimmune destruction of the beta cell
so if the problem is a loss of a beta
cell then maybe we can really can cure
that problem with a replacement cell as
it turns out about 18 years ago
another amazing Canadian physician
scientist by the name of dr. James
Shapiro showed the world that a cure for
type 1 diabetes is indeed possible by
replacing that missing cell what dr.
Shapiro and his team did was they
isolated islet issue from pancreases
including beta cells from pancreases
taken from cadavers human cadavers and
they infuse that islet tissue into the
liver of patients with type 1 diabetes
excitingly many of these cells would
take up residence and be able to produce
insulin in normal fashion and correct a
disease in fact these patients who
receive cadaver islet transplants are
now most of them will go for 5 years or
longer completely insulin independent
essentially functionally cured so that
was very exciting
however cadaver islet transplant has its
own set of severe limitations the
biggest limitation is just a shortage of
cells this has to come from cadaver from
pay courses taken from cadavers in the
u.s. there’s only about 1,500 believe it
or not only about 1,500 pancreas is
available each year and that it would be
suitable also these patients have to
take continuous immuno suppression and
it’s very costly as a result of these
limitation while there are millions of
people out there that have type 1
diabetes only approximately 2000
patients have been treated with cadaver
so our ambitious goal is to overcome all
of these limitations and make a cure
available for all patients we have a lot
of help on this we work with the
California Institute regenitive medicine
we work with JDRF which was formerly
known as joyed Juvenile Diabetes
Research Foundation clinical
investigators and importantly with
amazing patient volunteers and we’re on
a path to make this cure happen so
tackle the issue is supply we are using
another emerging exciting technology
that you actually heard a little bit
about earlier about earlier which is the
use of an embryonic stem cell line
embryonic stem cells have the unique
capability of being able to transform
into any cell in the body moreover these
cells are essentially immortal meaning
that a single cell line that we have
banked gives us sufficient material to
produce replacement cells for
essentially all patients so by
discovering how a embryonic stem cell
naturally becomes a beta cell our
scientists developed methods to drive
these cells to become pancreatic
progenitor cells that we then implant
into a subject and those cells further
mature to produce insulin and other
factors from the normal human islet and
correct correct a disease that may sound
relatively straightforward but this
slide one slide here represents about
ten years of work and a couple hundred
issued patents that came as a result of
it so having the cells is important but
it’s only part of the challenge we also
need a way to conveniently safely and
effectively deliver these cells to the
patient so for that we developed a
device technology basically a device
that we could fill with these cells
implant under the skin and then those
cells would mature in the device and
begin producing the insulin and other
factors now where we wanted more than
just delivery we also want to protect
these cells from the immune rejection so
that we can eliminate the need for
thus was born the encapture cell
delivery device the device has a
membrane a semipermeable membrane that
traps the cells inside the membrane
allows the passage of insulin and other
protein hormones in back into the
bloodstream you can think of this as
like a tea bag the cells we put inside
are like the tea leaves and the insulin
is the tea essence that is coming out
the membrane provides a barrier against
a patient’s immune system and therefore
protects these cells from immune
rejection and eliminates that need for
the immuno suppression the cells and the
device together make up a product
candidate that we call Peck and cap Peck
and cap has been tested in animal models
of type on diabetes and shown to be very
effective in fact when we take these
human cells and we put them into an
animal we essentially humanize that
animal in terms of its blood sugar
regulation meaning that that animal will
now regulate its blood sugar to the
human setpoint rather than the normal
set point of that animal so it’s very
effective in those models we’ve now
moved on to testing this product
candidate in patients with type 1
diabetes a big step forward for any
program it’s early days and it’s
certainly too soon for us to claim
victory over type 1 however should we be
successful and I believe we will be then
about a hundred years almost exactly a
hundred years after type 1 diabetes went
from a fatal disease to a chronic
disease we’ll deliver the next big step
a functional cure that can be used by
all patients with the disease what this
means is when we’re successful instead
of the daily grind of trying to inject
insulin and keep that blood sugar under
control and prevent those long term of
complications a patient will receive a
dose of stem cell derived islets in a
device placed under the skin that could
last for years
that means we go from this big pile of
syringes you see next to me here
– essentially a device that looks like
this this is actually the encapture cell
delivery device that would be filled
with the cells and implanted under the
skin a big difference the regenerative
medicine approach that we’re pioneering
at our company is has tremendous
potential not just for diabetes but for
many other diseases our goal right now
though is to prove that potential by
delivering a functional cure to all
patients with diabetes we shared a goal
of JDRF we want to live in a world where
type 1 goes to type none thank you [Applause]
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