I want to start with a poll how many of
you here have heard of gene therapy
before raising him okay keep your hands
up for a second and if you haven’t
raised your hand yet you probably should
but how many of you with your hands up
have been treated with or know someone
else who’s been treated with gene
therapy pretty much every hand just went
down okay regardless of how you answered
those two questions I want you to look
at this list of diseases on the screen
behind me please raise your hand if you
or someone you know has been diagnosed
with any of these diseases would it
surprise you to know that this is a list
of diseases which gene therapy is
currently being used to treat and
successfully so why don’t more people
have access to gene therapy because it’s
complicated and expensive I’ve been
doing gene therapy for nine years in
October and I’m gonna tell you two
stories the first patient I ever gave
gene therapy to and another patient who
changed my mindset about how we need to
be thinking about how to make treatments
available to everyone this is Charlie
the first patient that I gave gene
therapy to in 2009 Charlie was in his
early 50s living with his wife and his
daughter in Homer Alaska practicing as a
psychiatrist doing what most of us would
do on the fourth of July celebrating
with friends and family on a lake about
midway through the day Charlie came in
from a little boat ride and his wife
noticed that a speech was slurring she
thought at first have you been drinking
but he hadn’t in fact it was something
more serious than that a trip to the ER
and head scan showed that Charlie had
Stage four brain cancer glioblastoma
multiforme II a few months before
Charlie’s diagnosis in 2009 this disease
killed senator Ted Kennedy two weeks ago
Senator John McCain was
most in fact about a hundred and
ninety-two thousand people around the
world will be diagnosed with this
disease in the next year and seventeen
thousand of them will lose their battle
with it
it’s a highly aggressive form of cancer
most patients including Charlie only
have a 50% chance of surviving one year
and that’s with intensive therapy that
includes surgery chemotherapy radiation
more chemo therapy Charlie felt like he
needed to try something more and he took
the chance on a clinical trial and it
happened to be our clinical trial for
gene therapy to treat his disease now in
2009 this map is showing you the number
of centers around the world who were
doing some form of gene therapy now I
told you Charlie lived in Alaska so it
may seem like he came to Seattle because
it was the closest to him but in fact in
2009 we were the only Center doing gene
therapy for glioblastoma while each of
these centers was capable they were all
studying different diseases different
clinical trials why did Charlie even
have to come to Seattle well the type of
gene therapy we were giving him was
called blood cell gene therapy we have
to first bring him into the clinic take
blood cells out of him then we have to
move those to a clean room facility
because all of the manipulation we’re
gonna do is gonna happen outside of the
body this is an example of what our
clean room facility in Seattle looks
like controlled airflow highly skilled
staff equipment monitoring regulation of
every material and reagent that comes in
and out multi-million dollar
infrastructure once we have the cells
there maybe we don’t want all the blood
cells maybe we only need T cells or
maybe we only need stem cells so now we
need to separate those out that takes
more complex technology and people who
know how to use it after that we have to
get the DNA into those cells that we
want to target this is a snapshot of
what part of that process looks like it
can take anywhere from 2 to 14 days
depending on the cell and the type of
the DNA that you’re trying to get in you
see 3 people in this photo one person
who’s reading directions line by line
one person who’s actually performing
those instructions one at a time and a
third person who’s observing to make
sure that they all get done
correctly and in the right order again
multi-million dollar infrastructure
after all that manipulation we have to
make sure those cells are ready to go
back into the patient and that they’re
safe that we haven’t introduced a
contaminant that they’re functioning the
way that they should be and then and
only then can they go back to the
hospital and get infused into the
patient that is of course unless your
patient shows up wearing a Michigan
shirt when he knows that you are a rival
of hi-oh State University fan so I told
him I would only let him take this
picture if I could cover it up not only
to Charlie have to come to Seattle for
two weeks to receive this gene therapy
had to stay for another two weeks to be
monitored after that and then he had to
come back to Seattle every three to four
weeks for nine months for additional
treatment and research so was all that
bang worth its buck well in addition to
Charlie we cheated treated ten more
patients and we changed the survival
from less than 50% at one year to 100%
at one year
Charlie got six and a half years enough
time to see his daughter graduate from
that horrid University Michigan the
ability to go back to work full-time
until his retirement but he didn’t know
that in 2009 I didn’t know that in 2009
none of us could have predicted that
that would be the outcome so I want to
ask you a question
you don’t need to raise your hand this
time if you’d been given a diagnosis
with a prognosis of 11 months would you
be willing to leave your community your
family your friends your co-workers and
take a chance on something like this
nine out of those eleven months he had
to be back and forth in Seattle so when
I treated Charlie along with the team at
Fred hutch in 2009 this is what I was
thinking about thank goodness he was
willing to take this chance what if we
could move this therapy to where he is
how can we move gene therapy to where
the 200,000 people that are gonna be
diagnosed this year are going to be that
was the first time I thought about how
can we simplify this think about how to
get more clinics doing this that same
year I was going to be confronted with a
problem in gene therapy of much larger
scale in proportion and it was all
started by this man Timothy Ray Brown
he’s also known as the Berlin patient or
the only man on the planet right now
who’s been cured of an HIV infection for
more than 10 years now Timothy didn’t
get gene therapy what he got was a blood
cell therapy from a person whose DNA
made their blood cells resistant to HIV
infection while that was a
one-in-a-million treatment for Timothy
it paved the way for gene therapy to
become a treatment option for HIV
positive persons around the world and
affect the same type of outcome that
would mean not having to take
antiretroviral drug drugs every day for
the rest of their lives in 2013 dr. Hans
Peter King showed here along with the
American Foundation for AIDS research
the Timothy Ray Brown Foundation and the
National Institute of Health received a
grant along with other colleagues that
Fred
it’s part of a large collaboratory to
study how we could make gene therapy to
treat HIV a reality as part of this
funding source we in Seattle get to host
an HIV cure meeting once a year it’s
actually going to be in the next few
weeks and that meeting not only includes
researchers who are working on how to
make gene therapy for HIV a possibility
it also includes persons living with HIV
community advocate members like Josh and
others that was in 2014 the first time I
saw this map this is a map of HIV
prevalence in the in the world darker
blue means more HIV positive persons now
between 2009 and 2013 the number of
clinics capable of doing gene therapy
definitely increased but you can see
that there’s still a very large problem
with distribution and we’re not talking
about 200,000 people a year we’re
talking about more than 36 million
people living with HIV right now another
2.1 million every year 150,000 of which
will be children who primarily live in
sub-saharan Africa so same meeting
sitting here thinking about all of this
stuff prominent researcher comes up on
the stage talking about gene therapy and
how we’re making it a possibility person
living with HIV sitting in the audience
raises their hand and says the hope is
amazing how do you envision getting this
complex therapy to the millions of
patients who need it and the researchers
response was well first we have to show
it can work and then we’ll figure out
how to get it there
what would you want a treatment that had
been developed someplace else on people
who didn’t share your genetics didn’t
share your culture didn’t share your
environment didn’t share your lifestyle
we know that those things all impact how
treatment outcomes are effective
so I’m sitting there going I don’t think
that’s okay there has to be a way for us
to do this now and if none of these
people are gonna stand up and do it then
I guess I’ll try so I thought about this
process all this complex infrastructure
and technology that’s required well
obviously if we could just put the DNA
in a syringe and take that around the
world and inject it into a person that
would be great
and I can tell you that we’re working on
that for some of those diseases on that
list we’re gonna get there sooner than
others but for glioblastoma and HIV not
so quickly the reason is that the blood
cell we need to hit with our DNA is a
stem cell that sits in the bone marrow
and they’re very few so we don’t yet
have the technology to inject DNA and
have it get to enough of those very few
cells in the marrow and not get to a
bunch of other cells that we don’t want
it to go to so we still need to do this
outside of the body okay so I know what
we mean I have all these things right we
got to be able to process the blood
product we’ve got to be able to get to
the cells we want we need to be able to
keep those cells happy outside the body
for a period of time we need to be able
to add the DNA in a specific order and
sequence so I just started looking what
technologies are already out there there
has to be some things that I can sort of
piecemeal together to make this go right
so maybe put it into a box that we can
just ship around the world in
coincidentally I found one device it’s
manufactured by a German company it
couldn’t do gene therapy but it had all
the components that we needed actually
already in one unit so we bought one we
said okay let’s see if we can
reconfigure this to do what we need to
do in the order we need to do it
sometimes that took hands-on effort I
like to tell people this bag hanger only
comes standard on devices purchased in
Hawaii in the Caribbean
I was just the only one tall enough to
reach the time
other times we had to blend technologies
really complex alignment to get what we
needed until we found that someone else
also already manufactured that and we
can just incorporate it into our system
it took two years and more than 20
healthy people donating their blood
products to us to practice on as if they
were gene therapy patients before we
came to this gene therapy in a box on a
portable table meets all the same
criteria that the multi-million dollar
infrastructure cleanroom needs can go
anywhere that comes with pre-programmed
interfaces in theory all the things that
you would need to add the DNA to add the
gene can be things that you could get
from a pharmacy provided in a kit
it’s a proof-of-concept hasn’t left Fred
hutch yet we’re working on that part
getting the word out there is part of
that battle we think that now there’s no
excuse why we can’t be treating close to
60 million people on the planet or at
least overcoming those last barriers to
getting that much gene therapy out into
the world we also think that the more
people have access to this type of
treatment the more diseases we’re going
to discover could be benefited by this
treatment approach that means that we
can treat patients like Charlie patients
like Timothy patients like everyone in
this room wherever they exist in their
own communities so if you think that you
might be a candidate for gene therapy
talk to your doctor about it they’re the
ones who are gonna have to let us in the
door if you’re a healthy person consider
donating blood or other tissues to
research so that more scientists like me
can develop new approaches like this I
want to say thank you especially to all
of the patients that we treated in
Seattle and around the world with gene
therapy because they did take a big risk
they did step outside their communities
to make this possible I thank all of you
for your attention
you
you here have heard of gene therapy
before raising him okay keep your hands
up for a second and if you haven’t
raised your hand yet you probably should
but how many of you with your hands up
have been treated with or know someone
else who’s been treated with gene
therapy pretty much every hand just went
down okay regardless of how you answered
those two questions I want you to look
at this list of diseases on the screen
behind me please raise your hand if you
or someone you know has been diagnosed
with any of these diseases would it
surprise you to know that this is a list
of diseases which gene therapy is
currently being used to treat and
successfully so why don’t more people
have access to gene therapy because it’s
complicated and expensive I’ve been
doing gene therapy for nine years in
October and I’m gonna tell you two
stories the first patient I ever gave
gene therapy to and another patient who
changed my mindset about how we need to
be thinking about how to make treatments
available to everyone this is Charlie
the first patient that I gave gene
therapy to in 2009 Charlie was in his
early 50s living with his wife and his
daughter in Homer Alaska practicing as a
psychiatrist doing what most of us would
do on the fourth of July celebrating
with friends and family on a lake about
midway through the day Charlie came in
from a little boat ride and his wife
noticed that a speech was slurring she
thought at first have you been drinking
but he hadn’t in fact it was something
more serious than that a trip to the ER
and head scan showed that Charlie had
Stage four brain cancer glioblastoma
multiforme II a few months before
Charlie’s diagnosis in 2009 this disease
killed senator Ted Kennedy two weeks ago
Senator John McCain was
most in fact about a hundred and
ninety-two thousand people around the
world will be diagnosed with this
disease in the next year and seventeen
thousand of them will lose their battle
with it
it’s a highly aggressive form of cancer
most patients including Charlie only
have a 50% chance of surviving one year
and that’s with intensive therapy that
includes surgery chemotherapy radiation
more chemo therapy Charlie felt like he
needed to try something more and he took
the chance on a clinical trial and it
happened to be our clinical trial for
gene therapy to treat his disease now in
2009 this map is showing you the number
of centers around the world who were
doing some form of gene therapy now I
told you Charlie lived in Alaska so it
may seem like he came to Seattle because
it was the closest to him but in fact in
2009 we were the only Center doing gene
therapy for glioblastoma while each of
these centers was capable they were all
studying different diseases different
clinical trials why did Charlie even
have to come to Seattle well the type of
gene therapy we were giving him was
called blood cell gene therapy we have
to first bring him into the clinic take
blood cells out of him then we have to
move those to a clean room facility
because all of the manipulation we’re
gonna do is gonna happen outside of the
body this is an example of what our
clean room facility in Seattle looks
like controlled airflow highly skilled
staff equipment monitoring regulation of
every material and reagent that comes in
and out multi-million dollar
infrastructure once we have the cells
there maybe we don’t want all the blood
cells maybe we only need T cells or
maybe we only need stem cells so now we
need to separate those out that takes
more complex technology and people who
know how to use it after that we have to
get the DNA into those cells that we
want to target this is a snapshot of
what part of that process looks like it
can take anywhere from 2 to 14 days
depending on the cell and the type of
the DNA that you’re trying to get in you
see 3 people in this photo one person
who’s reading directions line by line
one person who’s actually performing
those instructions one at a time and a
third person who’s observing to make
sure that they all get done
correctly and in the right order again
multi-million dollar infrastructure
after all that manipulation we have to
make sure those cells are ready to go
back into the patient and that they’re
safe that we haven’t introduced a
contaminant that they’re functioning the
way that they should be and then and
only then can they go back to the
hospital and get infused into the
patient that is of course unless your
patient shows up wearing a Michigan
shirt when he knows that you are a rival
of hi-oh State University fan so I told
him I would only let him take this
picture if I could cover it up not only
to Charlie have to come to Seattle for
two weeks to receive this gene therapy
had to stay for another two weeks to be
monitored after that and then he had to
come back to Seattle every three to four
weeks for nine months for additional
treatment and research so was all that
bang worth its buck well in addition to
Charlie we cheated treated ten more
patients and we changed the survival
from less than 50% at one year to 100%
at one year
Charlie got six and a half years enough
time to see his daughter graduate from
that horrid University Michigan the
ability to go back to work full-time
until his retirement but he didn’t know
that in 2009 I didn’t know that in 2009
none of us could have predicted that
that would be the outcome so I want to
ask you a question
you don’t need to raise your hand this
time if you’d been given a diagnosis
with a prognosis of 11 months would you
be willing to leave your community your
family your friends your co-workers and
take a chance on something like this
nine out of those eleven months he had
to be back and forth in Seattle so when
I treated Charlie along with the team at
Fred hutch in 2009 this is what I was
thinking about thank goodness he was
willing to take this chance what if we
could move this therapy to where he is
how can we move gene therapy to where
the 200,000 people that are gonna be
diagnosed this year are going to be that
was the first time I thought about how
can we simplify this think about how to
get more clinics doing this that same
year I was going to be confronted with a
problem in gene therapy of much larger
scale in proportion and it was all
started by this man Timothy Ray Brown
he’s also known as the Berlin patient or
the only man on the planet right now
who’s been cured of an HIV infection for
more than 10 years now Timothy didn’t
get gene therapy what he got was a blood
cell therapy from a person whose DNA
made their blood cells resistant to HIV
infection while that was a
one-in-a-million treatment for Timothy
it paved the way for gene therapy to
become a treatment option for HIV
positive persons around the world and
affect the same type of outcome that
would mean not having to take
antiretroviral drug drugs every day for
the rest of their lives in 2013 dr. Hans
Peter King showed here along with the
American Foundation for AIDS research
the Timothy Ray Brown Foundation and the
National Institute of Health received a
grant along with other colleagues that
Fred
it’s part of a large collaboratory to
study how we could make gene therapy to
treat HIV a reality as part of this
funding source we in Seattle get to host
an HIV cure meeting once a year it’s
actually going to be in the next few
weeks and that meeting not only includes
researchers who are working on how to
make gene therapy for HIV a possibility
it also includes persons living with HIV
community advocate members like Josh and
others that was in 2014 the first time I
saw this map this is a map of HIV
prevalence in the in the world darker
blue means more HIV positive persons now
between 2009 and 2013 the number of
clinics capable of doing gene therapy
definitely increased but you can see
that there’s still a very large problem
with distribution and we’re not talking
about 200,000 people a year we’re
talking about more than 36 million
people living with HIV right now another
2.1 million every year 150,000 of which
will be children who primarily live in
sub-saharan Africa so same meeting
sitting here thinking about all of this
stuff prominent researcher comes up on
the stage talking about gene therapy and
how we’re making it a possibility person
living with HIV sitting in the audience
raises their hand and says the hope is
amazing how do you envision getting this
complex therapy to the millions of
patients who need it and the researchers
response was well first we have to show
it can work and then we’ll figure out
how to get it there
what would you want a treatment that had
been developed someplace else on people
who didn’t share your genetics didn’t
share your culture didn’t share your
environment didn’t share your lifestyle
we know that those things all impact how
treatment outcomes are effective
so I’m sitting there going I don’t think
that’s okay there has to be a way for us
to do this now and if none of these
people are gonna stand up and do it then
I guess I’ll try so I thought about this
process all this complex infrastructure
and technology that’s required well
obviously if we could just put the DNA
in a syringe and take that around the
world and inject it into a person that
would be great
and I can tell you that we’re working on
that for some of those diseases on that
list we’re gonna get there sooner than
others but for glioblastoma and HIV not
so quickly the reason is that the blood
cell we need to hit with our DNA is a
stem cell that sits in the bone marrow
and they’re very few so we don’t yet
have the technology to inject DNA and
have it get to enough of those very few
cells in the marrow and not get to a
bunch of other cells that we don’t want
it to go to so we still need to do this
outside of the body okay so I know what
we mean I have all these things right we
got to be able to process the blood
product we’ve got to be able to get to
the cells we want we need to be able to
keep those cells happy outside the body
for a period of time we need to be able
to add the DNA in a specific order and
sequence so I just started looking what
technologies are already out there there
has to be some things that I can sort of
piecemeal together to make this go right
so maybe put it into a box that we can
just ship around the world in
coincidentally I found one device it’s
manufactured by a German company it
couldn’t do gene therapy but it had all
the components that we needed actually
already in one unit so we bought one we
said okay let’s see if we can
reconfigure this to do what we need to
do in the order we need to do it
sometimes that took hands-on effort I
like to tell people this bag hanger only
comes standard on devices purchased in
Hawaii in the Caribbean
I was just the only one tall enough to
reach the time
other times we had to blend technologies
really complex alignment to get what we
needed until we found that someone else
also already manufactured that and we
can just incorporate it into our system
it took two years and more than 20
healthy people donating their blood
products to us to practice on as if they
were gene therapy patients before we
came to this gene therapy in a box on a
portable table meets all the same
criteria that the multi-million dollar
infrastructure cleanroom needs can go
anywhere that comes with pre-programmed
interfaces in theory all the things that
you would need to add the DNA to add the
gene can be things that you could get
from a pharmacy provided in a kit
it’s a proof-of-concept hasn’t left Fred
hutch yet we’re working on that part
getting the word out there is part of
that battle we think that now there’s no
excuse why we can’t be treating close to
60 million people on the planet or at
least overcoming those last barriers to
getting that much gene therapy out into
the world we also think that the more
people have access to this type of
treatment the more diseases we’re going
to discover could be benefited by this
treatment approach that means that we
can treat patients like Charlie patients
like Timothy patients like everyone in
this room wherever they exist in their
own communities so if you think that you
might be a candidate for gene therapy
talk to your doctor about it they’re the
ones who are gonna have to let us in the
door if you’re a healthy person consider
donating blood or other tissues to
research so that more scientists like me
can develop new approaches like this I
want to say thank you especially to all
of the patients that we treated in
Seattle and around the world with gene
therapy because they did take a big risk
they did step outside their communities
to make this possible I thank all of you
for your attention
you
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