The Human Trial (2022) - full transcript
Behind every breakthrough are the patients who risk everything for everybody else.
foodval.com - stop by if you're interested in the nutritional composition of food
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That is yuck, right?
What's yuck'?
-Blood.
-Yeah. It is what it is.
Okay, push hard.
-Oh!
-You see it's here!
I know and then
I have to squeeze it.
-Is it out?
-Look at how much blood there is
Mmm, what does that say? Do it?
-No? OK. Two, ni--, go ahead.
-...Two, nine, four.
Mmm, yeah.
Sucks, . Weird!
I don't know, Jack
Yeah, I know.
This is just not good.
I don't know why it's going up.
I have to talk to my doctor.
The average person has three
traumatic events in their life.
Waking up in the
ICU was one of mine.
Maybe we can test
daddy's blood sugar
-Let's see what daddy's is.
-Mhmm.
-There it is.
-There.
-Hey, look!
-Yeah. Okay.
-Nine, six.
-Wow!
I've spent the last 30 years
trying to outrun my disease,
but it's not working.
The irony is,
I look healthy but I'm not.
I always feel like I'm going to
the principal's office for
these calls.
I hate doing these calls.
My little stress ball, pancreas.
-Hi, Mary Rose.
-Hi Lisa.
You know, there's one
thing I want to say.
I wanted to tell you that
I have a little tingling in
my heel, in my left heel.
And it's only like
when I'm in the shower
and I'm shaving my leg
or whatever,
but I've been feeling
a bit of tingling and that's,
I'll be honest, it's kind of
freaking me out
but I don't know.
Like...
Well, it can. The diabetes can
be starting to affect
your nervous system.
This could be
the beginning of neuropathy.
So, the best thing to do
is get your blood
sugars under control.
I mean, you know, I've had
Type 1 now 31 years.
And I wasn't always in
really tight control.
Yeah. Oh my God.
-Okay.
-Well...
-I, yeah.
-I know, it's hard.
-Yeah.
-Well...
We've got today and we can
move forward from here.
You know, it's best to face this
before it gets real bad, Lisa.
I could never judge you for not
being perfect with this, honey
'cause I couldn't do it myself
and I don't, that's why I think
you should come see me more
because you can't do it
yourself either.
Yeah.
-Bye.
-Bye. Thank you.
Hmm.
It's just something
I hope that goes away,
if I take better care of myself.
It's become a joke
in the community
that the cure is
always five years away.
Stay strong, the cure
is on the horizon.
But each year more than 5
million people die from diabetes
waiting for that cure.
If it's so close,
why it's taking so long?
We started looking
for a biotech company
that was doing
something different,
something radical.
Then in 2014, my husband Guy
and I heard about a company
that was rethinking
how to cure diabetes.
They had engineered stem
cells to implant into humans.
We were skeptical but intrigued.
The first patient got
his insulin in 1922.
We cannot be a strong nation
unless we are a healthy nation.
And so we must recruit not
only men and materials,
but also knowledge and science.
Scientist Cutter in a new
medical age
with the monumental reports
that prove the Salk vaccine
against crippling polio
to be a sensational success.
Dr. Salk's own child was 1
of the 2 million children
involved in tests
of his vaccine.
Tests which have ended
for all time,
the threat of one of the
world's most vicious diseases.
Measles vaccines have
been successfully tested
by thousands of parents who
have permitted their children
to participate
in the field trial.
Behind it all are
tests and research
and years of
development and hope.
Do you think that
you'll live to see a cure?
No.
Science moves in a way
that's not linear.
It can be incremental,
punctuated by breakthroughs,
back to incremental.
Is there any guarantee
this will actually work?
There is never a guarantee when
you're doing a clinical trial.
If there was a guarantee, you
wouldn't have to do the trial.
We just submitted our first IND
to the FDA in the United States.
And that's an investigative
new drug application.
And altogether it's
over 8,500 pages.
I'm just going to
clear off some space
so that it doesn't
look so awful.
Why? No.
Perfect.
For me, it's only
the culmination
of a little over
three years of work.
But for some of my colleagues,
they've been involved in
ViaCyte for over a decade.
and their heart and soul
professionally
has gone into this
company and our product.
So, this is a mock up
and it doesn't contain
an actual device but
Actually let me just take
a look, maybe I'm lying to you.
it's easier if I take
it out of this package.
Oh, I lied.
So, you can see the
implant right in there.
And it contains cells.
That one contains cells
right now?
Yeah.
What we're developing
is a bio artificial pancreas
that uses stem cells
to potentially cure
Type 1 diabetes.
It sounds groundbreaking,
but diabetes is complicated.
Alright.
It all started with the
pancreas,
the ugliest organ in the body.
In Type 1 diabetes,
the body attacks itself
and destroys the cells
that produce insulin.
And when you don't have insulin,
sugar builds up in the blood
and it can't get into the cells.
It's as vital to
the body as oxygen.
While people
with Type 2 diabetes
have trouble absorbing insulin,
Type 1's don't produce
any insulin at all.
Either way, too much sugar
in your blood is toxic.
High blood sugars can lead
to blindness, kidney failure,
strokes and amputation.
Low blood sugars, and you risk
unconsciousness and death.
So, I'm constantly
injecting insulin
to control the amount
of sugar in my blood.
Normal blood sugars are
flat with the odd spike.
My blood sugars look
like the Himalayas.
Perfect.
ViaCyte wants to fix
this toxic rollercoaster,
program stem cells
to make insulin,
put them in a high tech teabag,
implant them into humans
and over time,
they'll replace
the damaged cells in the body.
Okay Jay, ready?
Seal.
We can do as much testing
on the bench
and in animals as we want.
And we still don't know
how it'll work in people.
And that's why we're all here
to make it work in people.
Okay, so today was our
30-day clock on the IND.
I was hoping to make this
a very suspenseful meeting,
but somehow having
bowels of champagne.
And I guess if
the answer was bad,
it would've been hard liquor.
There are no hold issues
and the clinical trial
may proceed.
The third or fourth
emotion I had,
It was like, "Oh yeah. Now,
I can be excited about this."
Been here 12 years.
This is my life's work
to see it come this far and
officially go into a patient.
It's amazing.
This is a new frontier.
The FDA is understandably
concerned
about the safety risks
associated with any kind
of cell therapy,
but they recognize that
patients need better treatments.
It takes 10 to 20 years
to develop a new drug,
but those are usually drugs
that are not that novel.
It's a pill, you eat it.
Everyone knows how to do that.
This is extremely novel.
Nightie Night.
I don't think anyone knew
how long this trial
was going to take.
We were still in Phase 1,
where scientists test
if their product is safe
and tolerated by humans.
I am sure you were hoping we
would have definitive answers
for you to film by now.
Welcome to the exciting,
but often frustrating
world of biotechnology.
It's a critical stage,
but it doesn't show if
the product is working.
Clinical studies like this
are set up in phases
for very good reason.
The first phase is
designed to determine
basic safety tolerance
and not put a whole
bunch of patients at risk
without understanding
that it's safe first.
The second phase is
the most exciting,
because that's where
you get the data.
The proof of concept.
Two, three.
Then the moment
we'd all been waiting for.
The FDA gave them
the green light
to move to Phase 2 of the trial.
It was finally time to see
if the cells could
work in people.
How much is riding on this?
Everything.
Everything, sorry.
ViaCyte opened 7 trial sites
in the US and Canada.
Including one at the
University of Minnesota.
And that's where
we met patient one,
Maren.
Want to come downstairs?
Come on.
When did you give me insulin?
3:30, more or less.
From the time I was very little,
I said that I would
always be first in line.
If something came up or there
was the opportunity for a cure.
Nervous?
There's a lot of unknowns
and I think it's the unknowns,
that are terrifying.
I'd like to think I'm a pioneer.
When you read in
textbooks 50 years later,
she was the first.
She was the first one
to try it and it worked.
This is you.
It's a huge deal.
This can impact
millions of diabetics.
And it's me, it's me on
that operating table.
This way.
We're the first door
on the left here.
Hi Maren, welcome.
-So, it's done.
-It's done.
It's great.
It went really, really well.
She's in the main
O.R. waking up area
where they really watch
airway and everything.
And then once they
deem her to be safe,
then she'll come here for
a little bit more time
and they'll be
checking her sugars
and doing all
those other things.
And then you guys
can be with her here.
-Perfect.
-Alright, thank you.
Take a deep breath. Huh?
32 years in the waiting.
It's been a long time.
Hoped and prayed for
this for a long time.
Oh yeah. These ones look great.
-They don't even hurt at all.
-Yeah.
They're barely even
bruised around there.
-Those don't hurt at all.
-Yeah.
Other than that, it's sore
down here.
-Yeah.
-Right in this area.
-You have such art on your arm.
-Still right now.
10 pods were implanted
into Maren's body.
Six in her arm
and four in her side.
Great.
Every few weeks,
one of her pods will be removed
to see if the cells are
surviving and producing insulin.
Yeah, I think sometimes
the hardest part
is getting used to
the medications.
I really do.
It's sore but the
pain isn't that bad.
It's sore but it's not the pain.
We'll be continuing
to monitor blood tests
for evidence of
insulin production.
And I'm sure Cathy
will text you or call you
to make sure that it got done.
So, yeah.
-All right. Good.
-Thank you.
All right, I'll let you go
do the hard work here, Cathy.
-Yeah.
-And I'll see you next week.
Alright, sounds good.
Thank you. Bye.
I was diagnosed with
diabetes at the age of two.
I don't know any other way.
Living a normal life to me
is living life as a diabetic.
I have some extreme lows which
caused me to have seizures.
You're so terrified that
you're going to go so low
or you're going to pass out
and your kids are going to find
you seizing on the floor.
Just the sweat,
just the dripping and the sweat
and the tremors
and the shaking and the fear.
I mean just the fear,
the fear of
the low blood sugars.
You can feel your
body shutting down
and you've got to stop it.
T, Z, Y, something, something L.
And then blink a couple times.
Can you pick out
anything below that?
T, Z V, E, C, L.
Go ahead and take
your glasses off.
I want you to look
straight ahead towards
the end of the room.
And if a light gets
bright, you can look up.
Okay.
When I was younger, my family
with all their good intentions
they'd tell me things like,
"If you don't check your
blood sugar
you're going to loose your foot"
And you know, I'm just like,
"Okay, I'm going to lose
my feet."
And that would cause
depression, fear,
and a lot of the times,
it made me feel like
I just want to give up.
And I have,
I've spent years not
taking care of my disease.
Just like,
"Fuck it."
My dad and me are
the only two people,
with diabetes that I ever knew.
He was legally blind
by the time he was 30.
I'm going blind.
It's an apple orchard.
Apple orchard.
What? Oh.
Come on ladies,
let's go find a pumpkin.
We're talking
about my life here.
And I'm not afraid of death,
but I'm here.
I need to be here
for my daughter.
That's what this is about.
Well, come here.
Let's see pumpkin.
Quick, quick, there.
And it's not just because
she's going to be
in this world alone.
I want to see her
fucking grow up.
I don't want to abandon her.
That's the worst feeling
on earth.
So, then this talks about
the number of participants here.
And so there's a site in Canada,
and right now there's two sites
in the United States.
And there'll probably
be a few more
that are added over time.
This part talks about the cells
that are actually being tested
and what the cells are,
is they're human cells.
So, they're actually derived
from an embryonic
stem cell source.
So it's a embryo
that was going to be
disposed of from
in-vitro fertilization.
Okay.
We don't expect them in
that first month or two
to actually do anything
functionally yet,
because we do know that
they need time to mature
to make insulin.
-Sure.
-Okay?
Remind me, when
were you diagnosed?
-I was 11 years old.
-11 Years old?
-Yeah.
-Okay.
About how many times
in the past year
do you think you've been so out
of it, mentally or physically
that you wouldn't have been
able to treat yourself,
you need someone
to help treat you?
Gosh, I'd say at probably
about 10, at least.
-About 10 in the past years.
-At least, yeah.
I get like confusion.
I don't know what to do.
I get panicky and
then I'll over-treat.
So, then I have
to deal with that.
Well, and then when
he gets sick,
he ends up so much worse
because the cold
or the flu he has
is making his blood
sugar all over the place.
Yeah, he spent four
days in the hospital
and with DKA and pneumonia
-And pneumonia.
-Yeah.
I mean, I resent my disease.
I really want...
This to work.
I completely get that. Yep.
Do you want a minute?
or do you want to-
-No.
-Go through this still?
All right. You sure? Okay.
How you doing?
It's a lot.
Okay.
You okay?
Let me take a look at your
temperature under your tongue.
I think you're good.
Okay, you can step away.
Okay, you're going to
feel a little poke.
Hi, this is Maren.
-I'm Greg.
-Hi, nice to meet you.
-Maren?
-Maren, yep.
-Nice to meet you.
-I'm Greg.
Are you overwhelmed?
-A little bit.
-A little bit.
Well, they're awesome here.
They will take good
care of you, I promise.
So, you probably have
a ton of questions.
I had a ton of questions
and I was first.
-Yeah.
-So, ask away.
Well, so,
where did they put all that?
And those are the scars
that you got?
Yep, you can feel them,
you can feel them inside.
-Do you mind?
-Yeah, go for it.
-Oh. Okay!
-Interesting.
So there's one, two,
three, four, five, six,
and then I have four
more right here.
Hmm. Okay.
It's a very surreal feeling
that you can't describe
unless, you know.
This whole thing's been
a surreal ride for me.
I'm still just, ah.
And even the day of,
they're wheeling me
back to the O.R.,
I was laying there
and I'm going,
"Oh my God, I don't
know if I can do this,
I don't know if I can do this."
'Cause they haven't done it.
And I'm thinking to myself
going, "Can I do this?"
Wow.
Is it affecting you?
-You know, technically, no.
-Yeah.
But I think there's
definitely some changes
going on in my body.
I can't exactly
tell what they are.
And then at the
end of the study,
they take them out, it's like,
you know what I mean?
I don't know what that's going
to do to me psychologically.
Well, it is.
It'll mess with you.
I guarantee it'll mess with you.
And they're taking one out in
a week and a half already.
And I don't want them to.
- Right.
-You know what I mean?
'Cause I want to see
if it's going to work,
but yet part of science
and I'm trying to come
to grips with on my own is,
is it even working?
I've told people it's
like winning the lottery.
Because if it works, it's
like winning the lottery.
Exactly.
I'd rather have this
than win the lottery.
There are two worlds
in a clinical trial.
Inside the lives of the patients
who are putting themselves
on the line to be first
and inside the labs
of the researchers,
who want to transform
patient hope into reality.
The two worlds create
a dynamic universe
yet they aren't allowed to meet.
There's a firewall between
the patients and researchers
to ensure the study
remains objective.
We became the bridge
between these two worlds.
We learned that whatever
was revealed to us,
could never be
revealed to the other,
including the question
on everyone's minds.
Is it working?
Was that Tamara? Hi Tamara.
-Hi Lisa.
-How are you?
-Okay guys.
We get email all the time.
These come from all
around the world.
"Hi, I have diabetes type 1
and I would like to know
if there's any chance
to participate as a volunteer
living here in Brazil.
Thanks from Paolo."
Here's another one.
"Hi, our 15-year old son
was diagnosed with
type 1 diabetes this week."
Wow.
This is an email from Mexico.
"Hello, yesterday, my
six-years old Paulina
was diagnostic diabetes type 1,
insulin independent.
She had a perfect
health till yesterday
that my little girl was rushed
into the emergency room
with a 480 glucose level.
She's so cheerful and all day,
every day is singing
every song she hears and
know she is attached
to a machine
that has to introduce insulin to
her little hand and her body."
Capital letters, "I don't want
that life for her."
Yes, so these keep
keep things in perspective.
Just goes on and on.
I have two biological children
and four adopted children.
Is your phone working
after you spilled on it?
Yeah.
I'll try calling you quick
and see if it makes the noise.
-My phone's over here.
-No, don't answer.
When I gave birth to
Katelyn, I had a seizure.
That was the first
seizure I had had.
-Yeah, it worked.
-Okay.
Okay. Bye Kate.
-Bye.
-See ya.
We'll see you at church, okay?
Bye. Katelyn. Love you.
I almost died.
I almost left my
daughter without a mom
and my husband without a wife.
The second seizure I had was
after the birth of Andrew,
which was actually
in the hospital.
So, we made
the determination that
we were done having biological
children at that point.
Go pack lunch.
Cold lunch, you mean?
Yep. Cold lunch.
Get your shoes on.
You got backpack?
I want to go.
I think living with a disease,
it creates who you are
and it makes you more accepting
of other people in
their unique situations.
I don't know why
her tales moping.
Guess what's for dinner?
Pizza night.
Mom, are you going to come?
-To church?
-Yeah.
-I'll help Merlie again.
-Okay.
I do the arithmetic in my head.
I want to see them graduate.
I want to see them get married.
I set personal goals for myself.
What I want to see them reach.
I thought they were
going to get an IV in,
but I don't know.
I don't think so.
We're doing a blood draw today.
-Yeah, we got it.
-First try too.
Are you showing
them your sugars?
No,
I have no explanation.
I know.
The average glucose
dropped from 164 to 140
-164 to 140 was your average.
-To 140.
That's really good.
-Here was your surgery day.
-Yeah.
And I wasn't feeling real
great those couple days
-That is not a surprise, there.
-But--
But it really hasn't gone
much up since then
and it's actually gone down.
That's interesting.
Let's see if the doctor--
Okay, so here we go.
Look what happened on its own?
My blood sugars went up
and now it's plateauing.
That's, I think
probably 200 points less
than what it got to last time.
Well, I'm anxious to
get the results now
'cause I'm curious,
I'm very curious.
I think everybody is.
It's not really good
to speculate that much,
but it should be too early
for yourselves to be working.
I know it is.
That's what they said.
Whatever it is,
something's working.
No, no. 'Cause it had only
went up to like 260
and then it went
down on its own.
It's on its way
down right now.
I didn't dose.
Yeah. I mean, you can't really
argue it at this point.
I mean, it doesn't do that
on its own.
You know what I
mean? For a diabetic?
Right, so.
Right. Not fully.
Three weeks today.
Three weeks today. Yup.
You guys want to bite of this?
Andrew, you want to
share it with me?
-What is it?
-I don't like the texture.
I think I'm blind.
-Does it work?
-Yep.
Okay.
That's good.
Six weeks into the trial,
Maren was telling me
how good she felt
and that her blood sugars
had never looked better.
But, the researchers were
telling me something different.
That it was too early for
her cells to be working.
Gotta get them on the phone now.
I don't know your sign in.
I was now straddling
the firewall
that separated the patients
from the researchers.
So I called Howard Foyt,
who's in charge of
clinical trials at ViaCyte.
Hi Howard, it's Lisa.
He became my bridge to what
was happening in the lab.
So, can I ask you
as a patient with type 1, me?
If I was doing all the work
testing my sugars, uploading,
going in three times a week
to my uni to get tested,
all that jazz
and I was seeing
some good results,
do you think and
you weren't told anything,
do you think that emotionally
that would be hard?
Because they're seeking answers.
The patients are so desperate
to know what's happening.
I see way what you
are challenging.
because they have a lot
of hopes and aspirations
that this therapy is
going to help them.
And so,
yes, I can see where it
would be frustrating,
but at the same time,
they understand that this
is clinical research.
It's a convoluted protocol.
I mean, it's one of
the most complicated studies
I have ever been involved with.
I'm the first one to admit that.
Have you seen any
C-peptide levels yet?
I cannot comment on that
and you know that.
Oh, perfect timing.
-Bye.
C-peptide levels are
the holy grail of this study.
They mean the cells are
producing insulin in patients.
Greg, is it going to bother
you too much
if I turn lights on here?
-No.
-Okay.
There we go.
So, it sounds a lot worse today
than you feel over the weekend.
So like Saturday it was mostly
like sore throat, right?
Okay. So, one of those
infections that
could be something that
you would've gotten anyway
if you weren't on the drugs,
so it's tough.
Okay, that's fine.
What we're going to,
for just a couple days,
we're going to cut the dose of
the CellCept in half. Okay?
But you've had a headache,
in particular this morning.
-It was splitting.
-Yeah.
Hopefully by tomorrow,
it's already a better for you.
That would be wonderful. Yeah.
Oink. Oink.
Ooh, those got
chocolate ones too.
Those are my favorite
when I was a kid.
Will you put one in my hand?
Oh thank you.
Yeah, that's my favorite.
A lot of mail to go through.
The disease is expensive.
It's very expensive.
Thousands of dollars
just from the last couple years.
This is July.
I'm getting 125 bucks.
Then there's this bill.
What is this?
Oh, that's for you.
That doesn't even
have my name on it.
Diabetes supplies include
sensors for the Dexcom,
the CGM, the constant
glucose monitoring,
my insulin pump,
the insulin itself,
and all that stuff adds up.
I mean, I lived for years
working in restaurants,
so, I didn't have
insurance and stuff
and I didn't have the $100
to pay for the vials
that you get.
I'd get sick and
end up in the ER
for like eight hours getting
bags and bags of fluids.
And then they'd
eventually let me leave
with a bottle of insulin.
which is enough to last
me maybe about a month.
Oh, a card.
They're envelopes.
But cards do come in envelopes.
I wish they were cards.
Like all birthday cards.
Come on, pup.
Insulin is like one of
the most expensive liquids
on earth.
They're making a
boatload of money
from selling us all this crap.
So I mean, why would they
want to get rid of that?
I hate insulin needles.
I hate the smell of insulin.
I just want this
disease to go away.
This is the most important
room in our house.
This is where the world
changed on October 31st, 1920.
It was here at 2:00 AM,
after a night restless sleep.
Dr. Banting came
up with this idea,
which led to the
discovery of insulin.
Diabetes was a death sentence
before the discovery of insulin.
A hundred years ago,
insulin wasn't a business.
It was a medical breakthrough
that saved millions of lives.
When Frederick Banting
accepted the Nobel Prize
for Medicine,
he famously said,
"Insulin doesn't belong to me,
it belongs to the world."
He and his team,
sold their patents
to the University of Toronto
for a dollar, each.
Now our illness fuels
a $245 billion industry
designed to manage our disease.
Here, I'll show you this.
So, before they
discovered insulin,
the only way you could treat
a person who has diabetes,
like your mom, was to put him
on a really horrible diet.
And so this child is 3 years old
and weighs 15 pounds.
Do you think you
weigh more than him?
-No.
-Oh you do.
- You weigh a lot more.
- You weigh 50 pounds.
50 pounds.
But there's a catch 22.
Biotech needs big pharma's
profits to fund clinical trials.
That without its support,
the researchers wouldn't
have gotten this far.
Like most relationships,
it's complicated.
It's the Flame of Hope,
it was lit 30 years ago
and it stays lit until a
cure for diabetes is found.
So, it burns for everyone around
the world who has diabetes.
Jack, you know what this means?
If the light goes
out for your mom?
It means you don't have
to get insulin no more.
-No more scary nights.
-Yeah.
-Hi. How are you? I'm Mike.
-I'm Michelle.
Michelle, good to meet
you in person.
Sure.
As a biotech startup company,
there are a lot of expectations
around what are we going
to achieve and how quickly
are we going to achieve it.
Good to meet you, Mike Scott.
There's something that's
called the valley of death.
When a startup company
gets through
an initially successful period
and then gets to the point
where some of the initial
excitement has died,
we're faced with
trying to raise money
without convincing
clinical data yet.
We're still making progress,
but not to that critical
juncture where you have that
data that's going to get
people over the hump of saying,
"Yes, we're going
to write a check."
The other really key part is,
is vascularization
around the outside.
You can see that at one end,
there's a piece of tubing.
And it's not that there
isn't a lot of interest.
It's just that the nature
of investors at this stage
is that, "Well, just show
me a little nibble,
give me a taste of
the success there,
and then I'll dump a
whole ton of money in."
And from our perspective it's,
"Well, that's what we need
the investment for,
to collect that data.
So, we can prove to you
that this works."
It's not going to work.
That's the challenge right now.
So what tugs at my
heartstrings too is, I mean...
First thing to keep in mind is,
we're down to about $13 million
and change at this point,
including the $10 million loan.
So, we're getting down
to the nitty gritty here.
We also have the CIRM
and JDRF fundings,
but we still need to
raise some equity capital.
Yeah, we have 180 days of cash.
Till that, $40 million
round is closed.
We are on life support.
In this company,
that discontinuous
development might be terminal.
If we have to cut back
on some of that core,
the thought leaders,
you lose those folks.
There's no guarantee you're
going to get them back.
So, what you're talking about
is not just a delay in
getting back rolling again.
You may not be able to rebuild,
as effective a team.
The cost of developing
a drug is staggering.
On average, $3 billion.
That's a lot to raise
from bike rides and walks.
And while government and
nonprofit funding is critical,
it alone can't cover
the cost of a clinical trial.
So, we followed Paul,
following the money.
And that took us to Riyadh.
It turns out that Saudi Arabia
has one of the highest rates of
type 1 diabetes in the world.
One thing about running
a biotech company is,
you never stop raising money.
You have to turn
over a lot of rocks.
And you don't know where
the investment might come from.
This really represents
potentially a functional cure.
If this works as
well in humans,
as it has in the
animal models we study,
it would essentially
take it to a disease
where patients no longer have
to think about it any further.
Does the American
public understand
the cost of developing a cure?
I don't think they really
have much of a clue
as to what goes into
getting that funding
and how much you have to raise.
-Hi, Paul Laikind.
-Hi, nice to meet you.
We've raised about $70 million
in venture financing.
In total, it's about
$150 million
that's been invested
in a company to date
to get us to where we are.
That's the billion
dollar question.
What happens if you
don't raise the money?
Shut the doors.
We're going to meet with
a large pharmaceutical firm.
It's been doing due
diligence on ViaCyte.
So, what we're looking for
is that they would
be able to take
a position in the company,
invest in the company
to the tune
of about $15 million or so.
All right, so tomorrow
we start at 9:00 AM.
ViaCyte is a small fish.
We're good at inventing.
We're good at innovating,
but we don't have deep pockets.
And that's where big pharma
can play a critical role
in taking this new technology
to the many, many
patients who need it.
-Exactly.
It's not that big pharma
companies don't take risks,
they take a wait
and see attitude
until it's proven that
the benefit outweighs the risk.
This is a byproduct
of our profit driven society,
where we have to
answer to shareholders.
That's the reality of national
and international business.
Ladies and gentlemen,
welcome to the Shinkansen.
This is the Hikari
Super Express.
We will be stopping at
Start running and
then jump on it.
I'm serious. Run, run, run.
What you go over there for?
Someone will be taken
out before the end of this.
I just hope it's not me.
It's fun. It's exciting.
It's scary but I can't
explain what is happening.
I've had two explants.
I had two taken out of my side
and one taken out of my arm.
One in October and
one in December.
It's exhausting.
I'm tired.
It's a lot physically.
It's a lot mentally,
it's a lot emotionally.
It has been more
than I ever expected.
Okay. So now Amber, don't squish
them but shake it.
Shake it.
That's the last 24 hours.
My insulin dose is about half,
but 50% overall.
I feel good, down
about 20 pounds
without even trying.
I haven't had any extreme lows.
Have I Case?
No extreme lows,
but you've had lows.
Lows, but not extreme lows.
No extreme highs.
So, I think it's definitely
narrowed the gap.
Sounds like the cells
are working.
Something's working.
I don't know what's working,
something's working.
What does Dr. Bellin say?
She said something
that have pointed me
in the direction that,
"Oh, they know something,
but they're not filling
me in on everything,
'cause I think they're
cautiously optimistic as well."
I never had super high
expectations to begin with,
but this is better
than I ever expected.
Even where I'm at right now.
So, would you agree
with that Case?
- I wasn't listening.
-Okay.
Hi Howard.
I don't believe it, necessarily.
Why not? You've been telling us
that the C-peptide is
the gold standard test.
It's the gold standard.
I'm not convinced given
that level of C-peptide,
given that it's a solitary
measurement.
And given that it's
a single patient.
I don't get it.
You guys would
get desensitized
if you were following
each and every patient
of the 11 we're doing right now,
but you're focusing
in on two patients,
the two patients at
the Minnesota site.
Everybody's hopeful.
-Yeah, well.
-Even the PI.
Yeah, I know.
That's why they pay me
to be skeptical.
Deep breath.
Good. Did you check your
wristband to make sure
that everything's
right on there?
-Sure.
-What's your birthday?
-Today.
-It's your birthday today?
-Yes. Happy birthday.
-Yeah.
I would say this cell
survival is moderate.
It just looks like, to me,
they might be fewer
than we would hope for at...
12 weeks.
...Which is a pretty
definitive time point.
It can keep going up after that,
but I wish it were
more right now.
Or you would
expect it to continue
to increase after 12 weeks.
Yes.
It's been 12 weeks since
Maren got her implant
and still no C-peptide levels
have shown up in her blood.
The researchers think her cells
should have matured by now.
They should be
producing insulin.
Do you think it's fair to say
that the patient shouldn't
give up hope just yet,
because as you always
tell me, Howard.
Rats are not humans
and this is an
experimental study.
Sure. Absolutely.
I mean, you have to
consider the outcome.
In rodent, the earliest
that we would anticipate
seeing C-peptide is on the
order of two to three months,
again though, that is depending
upon the assumption
that the time course in man,
is the same as that in rodent.
And we do not know
that for sure.
So, we're still in the
game with these two patients?
We're still in the game
with these two patients
and we haven't ruled them out.
Just want to know,
we've been working
together for a long time
and want to know like,
"Hey, I should still have
hope, me.
Not the filmmaker, but
me Lisa, the patient."
I mean, everything
is getting better.
You just have to be patient.
All right, Howard.
Thanks for your time.
Okay. Bye-bye.
Hi, I'm just going to
complain the whole time.
I'm a mess of a wreck.
I don't know if I can
keep doing this anymore.
I might have to go
back in today again,
which would be
surgery number five
in four months, and so tired.
Tired doing this and
so much physically
and emotionally on me.
All this in hopes of the cure.
I can't compare this experience
to anything in my life, ever.
It has been an
emotional rollercoaster,
is the only way to explain it.
I would give anything.
If I could sit down with
the people doing the study
out in San Diego and say,
"This is what it's
like on our end.
You can go ahead
and see my labs.
You can go ahead
and see the blood work.
But, talk to Greg and me.
Talk to us about
how we're doing."
Nice and close.
But I'm going to
have to stick it out
until someone tells me to stop.
I don't know what else to do.
I mean, I'm knee
deep in horse shit.
So, I don't know
what I'm supposed to do.
I mean, I've already
got eight incisions
all over my body
and been a lab rat.
I'm this far into it.
I might as well keep going.
Me and the other patients
are experiencing
better glucose numbers.
And I think that
could be attributed
to a placebo effect because
we're under a microscope.
Humans are self-conscious.
And they wanna leave
a good impression
So, looking at this
at the broad overview,
I'd say you're doing
reasonably well.
I mean, you're in
target 70% of the time.
Yeah.
You obviously have some
highs, but we expect that.
You have some lows,
but they seem like
they're pretty brief in nature.
You have a little more
highs maybe after lunchtime,
but nothing that I'm concerned
about for you.
Do you think it's working?
I do.
I think it's doing something
because my disease
seems easier to manage,
but it could just be me.
Oh, I like the socks today.
Yeah. Striped.
Used to play a lot of
craps on the streets.
I should probably paint some
dots on this thing.
Make him feel at home.
-What is that, Greg?
- This is my father.
He passed away about a year ago
from complications
of Pneumonia and diabetes.
-Is there an urn in it?
-This is an urn.
-Oh, that's the urn.
-Yeah.
For a Mexican family,
this is it, man.
This is what we got.
Yeah,
this is him.
He was in a nursing home
due to a hypoglycemic episode
that left him
mentally handicapped.
He couldn't live on his own.
He couldn't function.
He had to relearn how to talk
and he... but he
relearned, barely.
So yeah, this is:
Gregory Romero Senior.
Gracious God, we thank you
for Maren.
And for the love that she has,
that has been poured
out into so many lives.
And especially of all her kids,
who are gathered around her now.
Heavenly father,
we pray for Maren
and for the doctors who
will be looking after her,
we ask that your presence
will be with Maren
and be with the
rest of her family.
Lord, we lift her up to you
and know that she is in your
careful and loving arms.
In Jesus' name, we pray.
-Amen.
-Amen.
Katelyn, can stand up.
So, you're getting
some results today?
Hopefully I find something out,
either yes or no.
Are the cell's dead
or are they alive?
Okay, so they did
the explants, right?
Yep. So this is the January one.
-This is the most recent one.
-Okay. A couple weeks ago.
Remember they were looking
at the T cells before
that cause rejection.
So, they still see those cells.
Around the implant
and inside of it.
They see very, very few
from what they told me,
actual donor cells anymore.
Interesting, okay. Yep.
They don't think
that there's any chance
that the remaining ones
are going to make insulin.
Is this happening with everyone?
Do you know?
So, you are the only person
in the study so far that has
had this type of rejection.
Interesting.
So from our standpoint,
if they're not going to work,
then we're putting medicine
that you don't need,
leaving them in when
you don't need it.
Truly, truly I'm relieved.
I feel like I can breathe.
It's been a long five months.
It's been a really
long five months.
Six months almost
since the start of it.
I feel like I can maybe go
back to normal for a bit,
My normal, whatever
my normal is.
Do you feel guilty that the cure
is always 5 years away?
I don't feel guilty.
I feel hopeful.
I feel really hopeful.
I'm not kidding.
-You do?
-Yes.
-Really, for real?
-That's so nice of you.
I'm working here.
I can see what's going on.
It's going to be good for you.
I know it's been
really hard as heck.
Thanks.
I'm not kidding.
It's true.
I mean, there's nobody,
no person with a Dexcom
or a pump or anything
works as well as the cells.
The cells have evolved
over millions of years.
They're the best glucose
sensors, the best insulin
pumps in the world.
That's what biology's all about.
And that's why
we're doing it this way.
It takes a ton of work
and a ton of money,
but it's worth it.
That's good.
We want them to
get blood vessels.
Oh, that's great.
The loading port, right?
-Do you see it, Greg?
-It's the top one.
And for the other
one, there it is.
And then the port, port, port.
So if I come up this way.
I used to have great vision.
I remember as a child,
my dad being diabetic would
ask me to look down the street
and see what bus is coming
and I'd have to tell him.
And I'm like,
"How do you not see that?"
Now I understand.
Mr. Romero.
-Hi.
-How are you doing?
-Good, how are you?
-Good, good.
Your left eye vision
seems a lot more blurry
than it was last time I saw you.
It is a little bit.
Let me take a look real quick.
I looked at your pictures
and I have a suspicion
as to what's going on.
Look up.
Up and left.
and down,
down and right.
And right.
And left.
So, it seems that
you've had some bleeding
inside your left eye
from the diabetes.
Yeah, I see a floater or
it looks like a drop of blood.
Yeah.
So, that's probably at
the bottom of my eye somewhere.
Yeah, there's quite a bit of
blood in there
that is blocking your vision.
And it's definitely worse than
it was last time we saw you.
If it fails to clear, we might
have to do surgery,
-but I'm hoping to avoid that.
-Okay.
-Thank you very much.
-Oh, sure.
Yeah, whatever.
It's all part of the experience,
I guess.
I can't fucking see.
The study is the positive
thing for me right now.
We're just waiting.
We're all waiting
to see if these things work.
If they don't,
we'll cross that bridge
when we get to it.
I have neuropathy, retinopathy
and other
complications due to
the high blood sugars
throughout the years.
A lot of nerve damage I have.
I'm sorry.
I'm not going to say what
I was just going to say.
But yeah.
You suffer from
the disease as well.
So, I'm sorry too.
It's something that
we're all going to have
to encounter and deal with,
every one of us.
Sorry.
But if these cells work,
I'll cry for a week,
like, out loud
and I'm not the type of
person to do that very much.
I'm tired.
You had the ViaCyte?
-Yeah.
-Are they all gone now?
-They're gone.
-Okay.
I assume they're gone.
I got a phone call
and there's
a pancreas available.
I thought about it for years,
for years.
And the trial gave me
the opportunity to
try the medicine
and see how I handled it.
And I handled it just fine.
Recent changes in your
vision or your hearing?
No.
So, it opened the door for me
to think more about
doing this transplant.
-Sore throat?
-No.
I would do the trial over again.
I would do everything
all over again.
'Cause, it got me to this point.
I have zero regrets.
Zero.
-Hi.
-Hi, come here.
-How are you?
-Good, how are you?
Okay.
I'm going to sneak
a hug in there.
See you. Wish me luck.
I'm going to see you next time.
We'll go out for cheesecake.
-Cheesecake Factory
-with sugar.
Okay. Take care.
Come visit me. Okay?
All right. We'll see you, Greg.
One, two, three.
A friend of mine that
had a pancreas transplant
a year and a half ago,
text me this afternoon.
She goes, "You'll wake up
tomorrow with a new life."
So, it was pretty cool.
So, fingers crossed.
I've been going with Paul
to meetings
with potential investors
for the last two years.
Every time you're like,
"Oh, this is it."
These guys get it.
They're excited about it.
And then
they just don't call you back.
Don't reply the email. Or
It just falls apart
one way or the other.
So, that's been really hard.
The pouch is
designed to fully contain
the implant itself,
but still allow vital
nutrients and proteins
such as oxygen,
glucose, insulin,
and other hormones to
travel between the cells.
Well, it's nice. It's a good
description of the product.
Well, it's nice.
Good to see you.
All right.
I'm really pleased to announce
that as of this morning,
the checks rolled in
and we completed an $80 million
financing for ViaCyte.
That's $105 million over
the last three months.
It's really exciting,
but boy, we're going to be
under pressure to perform.
I believe that everybody in here
will redouble their efforts
to really show this new group
of investors
that they made a great decision.
All right, let's do a toast
to the success of this team,
the success of this program and
to curing type 1 diabetes.
I feel like Paul has mastered
the game of selling risk.
He convinces venture capitalists
to embrace the long game,
that science takes time
and progress is incremental.
You got to be optimistic
and stress resistant.
Two criteria.
If it doesn't work in
patient two,
it's going to work in 22.
But what if Paul's
strategy hadn't worked?
I can't help but think
this promising trial
would've been cut short,
only to languish on a shelf
next to the thousands
of other patents
collecting dust.
ViaCyte believes it
has the key elements needed
to produce transformative
therapies to treat diabetes.
Hello? I'm Greg.
Good. How are you?
We're going to talk
about your results.
-Yeah.
-And when you had your surgery
to remove one of the
small cell pouches.
So, I had a really
good conversation
with a sponsor yesterday.
And so there are some
donor cells there,
but there are not many of them
and they don't expect them
to get any better
from this point on.
So, we look for
C-peptide as a marker
of the cells actually making
insulin in your bloodstream
and that's always been negative.
So, I don't know if we're not
going to get any more benefit
from you, that it's
worthwhile for you
to have to continue to
take these medications
and have potential side effects
-and risk from the medications.
-Yeah.
And I, had to...
-Yes, I feel the--
-You feel the same way.
Well, you've done a great job
of taking care of yourself
and staying on top of things
through this whole process.
So, well then that would
lead me to this question,
which is, so you guys
have a Eureka moment
and this is something
that's going to work
for a large majority
of the population.
How long would
it take for your guys,
the cure, the solution
that you found
to hit clinics where
I'd be able to actually go
and get the dang thing?
Yeah, right. Good question.
That probably is at least,
from the time they're starting
these phase one studies,
I'd say it's probably at least
a five to seven year process.
Okay.
All right.
Other questions you have?
I don't know.
The more silent I get outside,
the more loud it
gets inside my head.
So, even if the patients
we've been following, Howard
don't seem to be
producing C-peptides
or essentially
their own insulin,
that's not a failure?
It depends upon how
you define failure.
It's obviously a setback
for these patients
who have volunteered
their time and effort
to participate in
these clinical trials.
But we've learned
a lot from them
and we continue to tweak
the implantation procedure,
trying to improve the
engraftment process.
And we continue
to make gains there.
What's the mood like
at ViaCyte right now?
I think the mood is upbeat.
Everybody is excited.
I mean, everybody
here at ViaCyte
is convinced our cells
do a great job,
they work the way
they're supposed to
and behave very physiologically.
They will cure type 1 diabetes.
There is one thing that
you would like to know.
Yeah.
Remember the first
patient from Minnesota.
Yeah.
We early terminated that patient
because of lack of C-peptide,
lack of potential
efficacy and the like,
so we explained all of
the remaining units.
Would you like to know
what the histology showed?
Yes.
That were viable clumps
of viable cells there.
So Howard, what does that mean?
What's the takeaway?
What's my takeaway
as a layperson.
The takeaway is
that cells can survive
for long periods of time in
this encapsulated environment.
And that's important.
That's really good news.
It's great news.
Would've been even better
if we'd been seeing C-peptides,
but you know, you don't always
get exactly what you want.
If you actually could
talk to these patients,
the ones we've
gotten to know well,
is there anything that you would
tell them as a message?
We owe all of these subjects,
a tremendous debt of gratitude.
Thus far, we have been
able to give them a lot
in terms of effectively
treating their own disease.
But we have gained a
tremendous amount of knowledge,
which hopefully will help us
get across the finish line
in terms of future iterations.
What are you thinking?
If it hadn't been
for Greg and Maren,
for the other patients who
participated in this trial
and they're up to patient 19,
they wouldn't be
where they are today.
And it's been a huge
sacrifice for these patients.
It's just been
really hard on them.
And I wish it had
worked for them,
but they're paving
the way for others, so,
that's it.
I was trying to come
to terms with the fact,
this may not work.
The trial wasn't over but
it was for Greg and Maren.
Any of the negative crap
you hear coming from me,
that's subjective crap.
That's Greg.
I know that I've been part
of something very important
and very beneficial to us.
All of us with diabetes.
Officially, as of today,
I'm considered a failed
transplant patient.
I have an appointment at my
1-year mark, late July
to get relisted.
I'm young, I'm healthy.
So therefore I'm a priority.
I'm not ready physically or
emotionally yet to do it again.
In time, maybe I will.
I have shared only this
with a few people.
I will cross that
bridge if people ask,
but it's a tremendous loss,
a loss I thought I was
ready for, but I was not.
Thank you for listening.
-Hey,
-Good to see you.
-Good to see you.
-Yeah.
We have the data that
shows that the product
is producing insulin in the
patients for the first time.
It's the first patients ever
in the history of
diabetes cure research.
So, this is a big deal.
I mean, we know now
that the cells work,
we didn't know that
five years ago.
All the pieces are there.
It's just a matter now
of completing the puzzle.
We all want stories
with a beginning,
middle and end.
One where all the loose
ends fit together.
But clinical research
is messy and hard.
It doesn't fit into
a tidy headline,
no matter how much
you want it to.
The cells may not have
worked for Greg and Maren,
but they're producing
insulin in nine patients.
Nine patients.
Because of them,
we are one step
closer to a cure.
But science alone isn't enough.
It needs a spotlight,
a champion
and money.
If we rethink how
we cure disease,
then the breakthroughs
that we so desperately
need and want
will come.
Will we get there in five years?
I think we can.
♪ I met two angels
♪ But they were in disguise
♪ Took one look to realize
♪ Tell them anything
♪ And they will sympathize
♪ These arms hold me tight
♪ Old fears
♪ Why me
♪ How'd I get this hallelujah
♪ Hallelujah
♪ Been that way since '95
♪ Three roads, one light
♪ Why me
♪ How'd I get this hallelujah
♪ Hallelujah
♪ Hallelujah
♪ Why me
♪ How'd I get this hallelujah
♪ One I wish I could see now
♪ These arms reach out
♪ Why me
♪ How'd I get this hallelujah
♪ Why me
♪ How'd I get this hallelujah
♪ Why me
♪ How'd I get this hallelujah
♪ Hallelujah
---
That is yuck, right?
What's yuck'?
-Blood.
-Yeah. It is what it is.
Okay, push hard.
-Oh!
-You see it's here!
I know and then
I have to squeeze it.
-Is it out?
-Look at how much blood there is
Mmm, what does that say? Do it?
-No? OK. Two, ni--, go ahead.
-...Two, nine, four.
Mmm, yeah.
Sucks, . Weird!
I don't know, Jack
Yeah, I know.
This is just not good.
I don't know why it's going up.
I have to talk to my doctor.
The average person has three
traumatic events in their life.
Waking up in the
ICU was one of mine.
Maybe we can test
daddy's blood sugar
-Let's see what daddy's is.
-Mhmm.
-There it is.
-There.
-Hey, look!
-Yeah. Okay.
-Nine, six.
-Wow!
I've spent the last 30 years
trying to outrun my disease,
but it's not working.
The irony is,
I look healthy but I'm not.
I always feel like I'm going to
the principal's office for
these calls.
I hate doing these calls.
My little stress ball, pancreas.
-Hi, Mary Rose.
-Hi Lisa.
You know, there's one
thing I want to say.
I wanted to tell you that
I have a little tingling in
my heel, in my left heel.
And it's only like
when I'm in the shower
and I'm shaving my leg
or whatever,
but I've been feeling
a bit of tingling and that's,
I'll be honest, it's kind of
freaking me out
but I don't know.
Like...
Well, it can. The diabetes can
be starting to affect
your nervous system.
This could be
the beginning of neuropathy.
So, the best thing to do
is get your blood
sugars under control.
I mean, you know, I've had
Type 1 now 31 years.
And I wasn't always in
really tight control.
Yeah. Oh my God.
-Okay.
-Well...
-I, yeah.
-I know, it's hard.
-Yeah.
-Well...
We've got today and we can
move forward from here.
You know, it's best to face this
before it gets real bad, Lisa.
I could never judge you for not
being perfect with this, honey
'cause I couldn't do it myself
and I don't, that's why I think
you should come see me more
because you can't do it
yourself either.
Yeah.
-Bye.
-Bye. Thank you.
Hmm.
It's just something
I hope that goes away,
if I take better care of myself.
It's become a joke
in the community
that the cure is
always five years away.
Stay strong, the cure
is on the horizon.
But each year more than 5
million people die from diabetes
waiting for that cure.
If it's so close,
why it's taking so long?
We started looking
for a biotech company
that was doing
something different,
something radical.
Then in 2014, my husband Guy
and I heard about a company
that was rethinking
how to cure diabetes.
They had engineered stem
cells to implant into humans.
We were skeptical but intrigued.
The first patient got
his insulin in 1922.
We cannot be a strong nation
unless we are a healthy nation.
And so we must recruit not
only men and materials,
but also knowledge and science.
Scientist Cutter in a new
medical age
with the monumental reports
that prove the Salk vaccine
against crippling polio
to be a sensational success.
Dr. Salk's own child was 1
of the 2 million children
involved in tests
of his vaccine.
Tests which have ended
for all time,
the threat of one of the
world's most vicious diseases.
Measles vaccines have
been successfully tested
by thousands of parents who
have permitted their children
to participate
in the field trial.
Behind it all are
tests and research
and years of
development and hope.
Do you think that
you'll live to see a cure?
No.
Science moves in a way
that's not linear.
It can be incremental,
punctuated by breakthroughs,
back to incremental.
Is there any guarantee
this will actually work?
There is never a guarantee when
you're doing a clinical trial.
If there was a guarantee, you
wouldn't have to do the trial.
We just submitted our first IND
to the FDA in the United States.
And that's an investigative
new drug application.
And altogether it's
over 8,500 pages.
I'm just going to
clear off some space
so that it doesn't
look so awful.
Why? No.
Perfect.
For me, it's only
the culmination
of a little over
three years of work.
But for some of my colleagues,
they've been involved in
ViaCyte for over a decade.
and their heart and soul
professionally
has gone into this
company and our product.
So, this is a mock up
and it doesn't contain
an actual device but
Actually let me just take
a look, maybe I'm lying to you.
it's easier if I take
it out of this package.
Oh, I lied.
So, you can see the
implant right in there.
And it contains cells.
That one contains cells
right now?
Yeah.
What we're developing
is a bio artificial pancreas
that uses stem cells
to potentially cure
Type 1 diabetes.
It sounds groundbreaking,
but diabetes is complicated.
Alright.
It all started with the
pancreas,
the ugliest organ in the body.
In Type 1 diabetes,
the body attacks itself
and destroys the cells
that produce insulin.
And when you don't have insulin,
sugar builds up in the blood
and it can't get into the cells.
It's as vital to
the body as oxygen.
While people
with Type 2 diabetes
have trouble absorbing insulin,
Type 1's don't produce
any insulin at all.
Either way, too much sugar
in your blood is toxic.
High blood sugars can lead
to blindness, kidney failure,
strokes and amputation.
Low blood sugars, and you risk
unconsciousness and death.
So, I'm constantly
injecting insulin
to control the amount
of sugar in my blood.
Normal blood sugars are
flat with the odd spike.
My blood sugars look
like the Himalayas.
Perfect.
ViaCyte wants to fix
this toxic rollercoaster,
program stem cells
to make insulin,
put them in a high tech teabag,
implant them into humans
and over time,
they'll replace
the damaged cells in the body.
Okay Jay, ready?
Seal.
We can do as much testing
on the bench
and in animals as we want.
And we still don't know
how it'll work in people.
And that's why we're all here
to make it work in people.
Okay, so today was our
30-day clock on the IND.
I was hoping to make this
a very suspenseful meeting,
but somehow having
bowels of champagne.
And I guess if
the answer was bad,
it would've been hard liquor.
There are no hold issues
and the clinical trial
may proceed.
The third or fourth
emotion I had,
It was like, "Oh yeah. Now,
I can be excited about this."
Been here 12 years.
This is my life's work
to see it come this far and
officially go into a patient.
It's amazing.
This is a new frontier.
The FDA is understandably
concerned
about the safety risks
associated with any kind
of cell therapy,
but they recognize that
patients need better treatments.
It takes 10 to 20 years
to develop a new drug,
but those are usually drugs
that are not that novel.
It's a pill, you eat it.
Everyone knows how to do that.
This is extremely novel.
Nightie Night.
I don't think anyone knew
how long this trial
was going to take.
We were still in Phase 1,
where scientists test
if their product is safe
and tolerated by humans.
I am sure you were hoping we
would have definitive answers
for you to film by now.
Welcome to the exciting,
but often frustrating
world of biotechnology.
It's a critical stage,
but it doesn't show if
the product is working.
Clinical studies like this
are set up in phases
for very good reason.
The first phase is
designed to determine
basic safety tolerance
and not put a whole
bunch of patients at risk
without understanding
that it's safe first.
The second phase is
the most exciting,
because that's where
you get the data.
The proof of concept.
Two, three.
Then the moment
we'd all been waiting for.
The FDA gave them
the green light
to move to Phase 2 of the trial.
It was finally time to see
if the cells could
work in people.
How much is riding on this?
Everything.
Everything, sorry.
ViaCyte opened 7 trial sites
in the US and Canada.
Including one at the
University of Minnesota.
And that's where
we met patient one,
Maren.
Want to come downstairs?
Come on.
When did you give me insulin?
3:30, more or less.
From the time I was very little,
I said that I would
always be first in line.
If something came up or there
was the opportunity for a cure.
Nervous?
There's a lot of unknowns
and I think it's the unknowns,
that are terrifying.
I'd like to think I'm a pioneer.
When you read in
textbooks 50 years later,
she was the first.
She was the first one
to try it and it worked.
This is you.
It's a huge deal.
This can impact
millions of diabetics.
And it's me, it's me on
that operating table.
This way.
We're the first door
on the left here.
Hi Maren, welcome.
-So, it's done.
-It's done.
It's great.
It went really, really well.
She's in the main
O.R. waking up area
where they really watch
airway and everything.
And then once they
deem her to be safe,
then she'll come here for
a little bit more time
and they'll be
checking her sugars
and doing all
those other things.
And then you guys
can be with her here.
-Perfect.
-Alright, thank you.
Take a deep breath. Huh?
32 years in the waiting.
It's been a long time.
Hoped and prayed for
this for a long time.
Oh yeah. These ones look great.
-They don't even hurt at all.
-Yeah.
They're barely even
bruised around there.
-Those don't hurt at all.
-Yeah.
Other than that, it's sore
down here.
-Yeah.
-Right in this area.
-You have such art on your arm.
-Still right now.
10 pods were implanted
into Maren's body.
Six in her arm
and four in her side.
Great.
Every few weeks,
one of her pods will be removed
to see if the cells are
surviving and producing insulin.
Yeah, I think sometimes
the hardest part
is getting used to
the medications.
I really do.
It's sore but the
pain isn't that bad.
It's sore but it's not the pain.
We'll be continuing
to monitor blood tests
for evidence of
insulin production.
And I'm sure Cathy
will text you or call you
to make sure that it got done.
So, yeah.
-All right. Good.
-Thank you.
All right, I'll let you go
do the hard work here, Cathy.
-Yeah.
-And I'll see you next week.
Alright, sounds good.
Thank you. Bye.
I was diagnosed with
diabetes at the age of two.
I don't know any other way.
Living a normal life to me
is living life as a diabetic.
I have some extreme lows which
caused me to have seizures.
You're so terrified that
you're going to go so low
or you're going to pass out
and your kids are going to find
you seizing on the floor.
Just the sweat,
just the dripping and the sweat
and the tremors
and the shaking and the fear.
I mean just the fear,
the fear of
the low blood sugars.
You can feel your
body shutting down
and you've got to stop it.
T, Z, Y, something, something L.
And then blink a couple times.
Can you pick out
anything below that?
T, Z V, E, C, L.
Go ahead and take
your glasses off.
I want you to look
straight ahead towards
the end of the room.
And if a light gets
bright, you can look up.
Okay.
When I was younger, my family
with all their good intentions
they'd tell me things like,
"If you don't check your
blood sugar
you're going to loose your foot"
And you know, I'm just like,
"Okay, I'm going to lose
my feet."
And that would cause
depression, fear,
and a lot of the times,
it made me feel like
I just want to give up.
And I have,
I've spent years not
taking care of my disease.
Just like,
"Fuck it."
My dad and me are
the only two people,
with diabetes that I ever knew.
He was legally blind
by the time he was 30.
I'm going blind.
It's an apple orchard.
Apple orchard.
What? Oh.
Come on ladies,
let's go find a pumpkin.
We're talking
about my life here.
And I'm not afraid of death,
but I'm here.
I need to be here
for my daughter.
That's what this is about.
Well, come here.
Let's see pumpkin.
Quick, quick, there.
And it's not just because
she's going to be
in this world alone.
I want to see her
fucking grow up.
I don't want to abandon her.
That's the worst feeling
on earth.
So, then this talks about
the number of participants here.
And so there's a site in Canada,
and right now there's two sites
in the United States.
And there'll probably
be a few more
that are added over time.
This part talks about the cells
that are actually being tested
and what the cells are,
is they're human cells.
So, they're actually derived
from an embryonic
stem cell source.
So it's a embryo
that was going to be
disposed of from
in-vitro fertilization.
Okay.
We don't expect them in
that first month or two
to actually do anything
functionally yet,
because we do know that
they need time to mature
to make insulin.
-Sure.
-Okay?
Remind me, when
were you diagnosed?
-I was 11 years old.
-11 Years old?
-Yeah.
-Okay.
About how many times
in the past year
do you think you've been so out
of it, mentally or physically
that you wouldn't have been
able to treat yourself,
you need someone
to help treat you?
Gosh, I'd say at probably
about 10, at least.
-About 10 in the past years.
-At least, yeah.
I get like confusion.
I don't know what to do.
I get panicky and
then I'll over-treat.
So, then I have
to deal with that.
Well, and then when
he gets sick,
he ends up so much worse
because the cold
or the flu he has
is making his blood
sugar all over the place.
Yeah, he spent four
days in the hospital
and with DKA and pneumonia
-And pneumonia.
-Yeah.
I mean, I resent my disease.
I really want...
This to work.
I completely get that. Yep.
Do you want a minute?
or do you want to-
-No.
-Go through this still?
All right. You sure? Okay.
How you doing?
It's a lot.
Okay.
You okay?
Let me take a look at your
temperature under your tongue.
I think you're good.
Okay, you can step away.
Okay, you're going to
feel a little poke.
Hi, this is Maren.
-I'm Greg.
-Hi, nice to meet you.
-Maren?
-Maren, yep.
-Nice to meet you.
-I'm Greg.
Are you overwhelmed?
-A little bit.
-A little bit.
Well, they're awesome here.
They will take good
care of you, I promise.
So, you probably have
a ton of questions.
I had a ton of questions
and I was first.
-Yeah.
-So, ask away.
Well, so,
where did they put all that?
And those are the scars
that you got?
Yep, you can feel them,
you can feel them inside.
-Do you mind?
-Yeah, go for it.
-Oh. Okay!
-Interesting.
So there's one, two,
three, four, five, six,
and then I have four
more right here.
Hmm. Okay.
It's a very surreal feeling
that you can't describe
unless, you know.
This whole thing's been
a surreal ride for me.
I'm still just, ah.
And even the day of,
they're wheeling me
back to the O.R.,
I was laying there
and I'm going,
"Oh my God, I don't
know if I can do this,
I don't know if I can do this."
'Cause they haven't done it.
And I'm thinking to myself
going, "Can I do this?"
Wow.
Is it affecting you?
-You know, technically, no.
-Yeah.
But I think there's
definitely some changes
going on in my body.
I can't exactly
tell what they are.
And then at the
end of the study,
they take them out, it's like,
you know what I mean?
I don't know what that's going
to do to me psychologically.
Well, it is.
It'll mess with you.
I guarantee it'll mess with you.
And they're taking one out in
a week and a half already.
And I don't want them to.
- Right.
-You know what I mean?
'Cause I want to see
if it's going to work,
but yet part of science
and I'm trying to come
to grips with on my own is,
is it even working?
I've told people it's
like winning the lottery.
Because if it works, it's
like winning the lottery.
Exactly.
I'd rather have this
than win the lottery.
There are two worlds
in a clinical trial.
Inside the lives of the patients
who are putting themselves
on the line to be first
and inside the labs
of the researchers,
who want to transform
patient hope into reality.
The two worlds create
a dynamic universe
yet they aren't allowed to meet.
There's a firewall between
the patients and researchers
to ensure the study
remains objective.
We became the bridge
between these two worlds.
We learned that whatever
was revealed to us,
could never be
revealed to the other,
including the question
on everyone's minds.
Is it working?
Was that Tamara? Hi Tamara.
-Hi Lisa.
-How are you?
-Okay guys.
We get email all the time.
These come from all
around the world.
"Hi, I have diabetes type 1
and I would like to know
if there's any chance
to participate as a volunteer
living here in Brazil.
Thanks from Paolo."
Here's another one.
"Hi, our 15-year old son
was diagnosed with
type 1 diabetes this week."
Wow.
This is an email from Mexico.
"Hello, yesterday, my
six-years old Paulina
was diagnostic diabetes type 1,
insulin independent.
She had a perfect
health till yesterday
that my little girl was rushed
into the emergency room
with a 480 glucose level.
She's so cheerful and all day,
every day is singing
every song she hears and
know she is attached
to a machine
that has to introduce insulin to
her little hand and her body."
Capital letters, "I don't want
that life for her."
Yes, so these keep
keep things in perspective.
Just goes on and on.
I have two biological children
and four adopted children.
Is your phone working
after you spilled on it?
Yeah.
I'll try calling you quick
and see if it makes the noise.
-My phone's over here.
-No, don't answer.
When I gave birth to
Katelyn, I had a seizure.
That was the first
seizure I had had.
-Yeah, it worked.
-Okay.
Okay. Bye Kate.
-Bye.
-See ya.
We'll see you at church, okay?
Bye. Katelyn. Love you.
I almost died.
I almost left my
daughter without a mom
and my husband without a wife.
The second seizure I had was
after the birth of Andrew,
which was actually
in the hospital.
So, we made
the determination that
we were done having biological
children at that point.
Go pack lunch.
Cold lunch, you mean?
Yep. Cold lunch.
Get your shoes on.
You got backpack?
I want to go.
I think living with a disease,
it creates who you are
and it makes you more accepting
of other people in
their unique situations.
I don't know why
her tales moping.
Guess what's for dinner?
Pizza night.
Mom, are you going to come?
-To church?
-Yeah.
-I'll help Merlie again.
-Okay.
I do the arithmetic in my head.
I want to see them graduate.
I want to see them get married.
I set personal goals for myself.
What I want to see them reach.
I thought they were
going to get an IV in,
but I don't know.
I don't think so.
We're doing a blood draw today.
-Yeah, we got it.
-First try too.
Are you showing
them your sugars?
No,
I have no explanation.
I know.
The average glucose
dropped from 164 to 140
-164 to 140 was your average.
-To 140.
That's really good.
-Here was your surgery day.
-Yeah.
And I wasn't feeling real
great those couple days
-That is not a surprise, there.
-But--
But it really hasn't gone
much up since then
and it's actually gone down.
That's interesting.
Let's see if the doctor--
Okay, so here we go.
Look what happened on its own?
My blood sugars went up
and now it's plateauing.
That's, I think
probably 200 points less
than what it got to last time.
Well, I'm anxious to
get the results now
'cause I'm curious,
I'm very curious.
I think everybody is.
It's not really good
to speculate that much,
but it should be too early
for yourselves to be working.
I know it is.
That's what they said.
Whatever it is,
something's working.
No, no. 'Cause it had only
went up to like 260
and then it went
down on its own.
It's on its way
down right now.
I didn't dose.
Yeah. I mean, you can't really
argue it at this point.
I mean, it doesn't do that
on its own.
You know what I
mean? For a diabetic?
Right, so.
Right. Not fully.
Three weeks today.
Three weeks today. Yup.
You guys want to bite of this?
Andrew, you want to
share it with me?
-What is it?
-I don't like the texture.
I think I'm blind.
-Does it work?
-Yep.
Okay.
That's good.
Six weeks into the trial,
Maren was telling me
how good she felt
and that her blood sugars
had never looked better.
But, the researchers were
telling me something different.
That it was too early for
her cells to be working.
Gotta get them on the phone now.
I don't know your sign in.
I was now straddling
the firewall
that separated the patients
from the researchers.
So I called Howard Foyt,
who's in charge of
clinical trials at ViaCyte.
Hi Howard, it's Lisa.
He became my bridge to what
was happening in the lab.
So, can I ask you
as a patient with type 1, me?
If I was doing all the work
testing my sugars, uploading,
going in three times a week
to my uni to get tested,
all that jazz
and I was seeing
some good results,
do you think and
you weren't told anything,
do you think that emotionally
that would be hard?
Because they're seeking answers.
The patients are so desperate
to know what's happening.
I see way what you
are challenging.
because they have a lot
of hopes and aspirations
that this therapy is
going to help them.
And so,
yes, I can see where it
would be frustrating,
but at the same time,
they understand that this
is clinical research.
It's a convoluted protocol.
I mean, it's one of
the most complicated studies
I have ever been involved with.
I'm the first one to admit that.
Have you seen any
C-peptide levels yet?
I cannot comment on that
and you know that.
Oh, perfect timing.
-Bye.
C-peptide levels are
the holy grail of this study.
They mean the cells are
producing insulin in patients.
Greg, is it going to bother
you too much
if I turn lights on here?
-No.
-Okay.
There we go.
So, it sounds a lot worse today
than you feel over the weekend.
So like Saturday it was mostly
like sore throat, right?
Okay. So, one of those
infections that
could be something that
you would've gotten anyway
if you weren't on the drugs,
so it's tough.
Okay, that's fine.
What we're going to,
for just a couple days,
we're going to cut the dose of
the CellCept in half. Okay?
But you've had a headache,
in particular this morning.
-It was splitting.
-Yeah.
Hopefully by tomorrow,
it's already a better for you.
That would be wonderful. Yeah.
Oink. Oink.
Ooh, those got
chocolate ones too.
Those are my favorite
when I was a kid.
Will you put one in my hand?
Oh thank you.
Yeah, that's my favorite.
A lot of mail to go through.
The disease is expensive.
It's very expensive.
Thousands of dollars
just from the last couple years.
This is July.
I'm getting 125 bucks.
Then there's this bill.
What is this?
Oh, that's for you.
That doesn't even
have my name on it.
Diabetes supplies include
sensors for the Dexcom,
the CGM, the constant
glucose monitoring,
my insulin pump,
the insulin itself,
and all that stuff adds up.
I mean, I lived for years
working in restaurants,
so, I didn't have
insurance and stuff
and I didn't have the $100
to pay for the vials
that you get.
I'd get sick and
end up in the ER
for like eight hours getting
bags and bags of fluids.
And then they'd
eventually let me leave
with a bottle of insulin.
which is enough to last
me maybe about a month.
Oh, a card.
They're envelopes.
But cards do come in envelopes.
I wish they were cards.
Like all birthday cards.
Come on, pup.
Insulin is like one of
the most expensive liquids
on earth.
They're making a
boatload of money
from selling us all this crap.
So I mean, why would they
want to get rid of that?
I hate insulin needles.
I hate the smell of insulin.
I just want this
disease to go away.
This is the most important
room in our house.
This is where the world
changed on October 31st, 1920.
It was here at 2:00 AM,
after a night restless sleep.
Dr. Banting came
up with this idea,
which led to the
discovery of insulin.
Diabetes was a death sentence
before the discovery of insulin.
A hundred years ago,
insulin wasn't a business.
It was a medical breakthrough
that saved millions of lives.
When Frederick Banting
accepted the Nobel Prize
for Medicine,
he famously said,
"Insulin doesn't belong to me,
it belongs to the world."
He and his team,
sold their patents
to the University of Toronto
for a dollar, each.
Now our illness fuels
a $245 billion industry
designed to manage our disease.
Here, I'll show you this.
So, before they
discovered insulin,
the only way you could treat
a person who has diabetes,
like your mom, was to put him
on a really horrible diet.
And so this child is 3 years old
and weighs 15 pounds.
Do you think you
weigh more than him?
-No.
-Oh you do.
- You weigh a lot more.
- You weigh 50 pounds.
50 pounds.
But there's a catch 22.
Biotech needs big pharma's
profits to fund clinical trials.
That without its support,
the researchers wouldn't
have gotten this far.
Like most relationships,
it's complicated.
It's the Flame of Hope,
it was lit 30 years ago
and it stays lit until a
cure for diabetes is found.
So, it burns for everyone around
the world who has diabetes.
Jack, you know what this means?
If the light goes
out for your mom?
It means you don't have
to get insulin no more.
-No more scary nights.
-Yeah.
-Hi. How are you? I'm Mike.
-I'm Michelle.
Michelle, good to meet
you in person.
Sure.
As a biotech startup company,
there are a lot of expectations
around what are we going
to achieve and how quickly
are we going to achieve it.
Good to meet you, Mike Scott.
There's something that's
called the valley of death.
When a startup company
gets through
an initially successful period
and then gets to the point
where some of the initial
excitement has died,
we're faced with
trying to raise money
without convincing
clinical data yet.
We're still making progress,
but not to that critical
juncture where you have that
data that's going to get
people over the hump of saying,
"Yes, we're going
to write a check."
The other really key part is,
is vascularization
around the outside.
You can see that at one end,
there's a piece of tubing.
And it's not that there
isn't a lot of interest.
It's just that the nature
of investors at this stage
is that, "Well, just show
me a little nibble,
give me a taste of
the success there,
and then I'll dump a
whole ton of money in."
And from our perspective it's,
"Well, that's what we need
the investment for,
to collect that data.
So, we can prove to you
that this works."
It's not going to work.
That's the challenge right now.
So what tugs at my
heartstrings too is, I mean...
First thing to keep in mind is,
we're down to about $13 million
and change at this point,
including the $10 million loan.
So, we're getting down
to the nitty gritty here.
We also have the CIRM
and JDRF fundings,
but we still need to
raise some equity capital.
Yeah, we have 180 days of cash.
Till that, $40 million
round is closed.
We are on life support.
In this company,
that discontinuous
development might be terminal.
If we have to cut back
on some of that core,
the thought leaders,
you lose those folks.
There's no guarantee you're
going to get them back.
So, what you're talking about
is not just a delay in
getting back rolling again.
You may not be able to rebuild,
as effective a team.
The cost of developing
a drug is staggering.
On average, $3 billion.
That's a lot to raise
from bike rides and walks.
And while government and
nonprofit funding is critical,
it alone can't cover
the cost of a clinical trial.
So, we followed Paul,
following the money.
And that took us to Riyadh.
It turns out that Saudi Arabia
has one of the highest rates of
type 1 diabetes in the world.
One thing about running
a biotech company is,
you never stop raising money.
You have to turn
over a lot of rocks.
And you don't know where
the investment might come from.
This really represents
potentially a functional cure.
If this works as
well in humans,
as it has in the
animal models we study,
it would essentially
take it to a disease
where patients no longer have
to think about it any further.
Does the American
public understand
the cost of developing a cure?
I don't think they really
have much of a clue
as to what goes into
getting that funding
and how much you have to raise.
-Hi, Paul Laikind.
-Hi, nice to meet you.
We've raised about $70 million
in venture financing.
In total, it's about
$150 million
that's been invested
in a company to date
to get us to where we are.
That's the billion
dollar question.
What happens if you
don't raise the money?
Shut the doors.
We're going to meet with
a large pharmaceutical firm.
It's been doing due
diligence on ViaCyte.
So, what we're looking for
is that they would
be able to take
a position in the company,
invest in the company
to the tune
of about $15 million or so.
All right, so tomorrow
we start at 9:00 AM.
ViaCyte is a small fish.
We're good at inventing.
We're good at innovating,
but we don't have deep pockets.
And that's where big pharma
can play a critical role
in taking this new technology
to the many, many
patients who need it.
-Exactly.
It's not that big pharma
companies don't take risks,
they take a wait
and see attitude
until it's proven that
the benefit outweighs the risk.
This is a byproduct
of our profit driven society,
where we have to
answer to shareholders.
That's the reality of national
and international business.
Ladies and gentlemen,
welcome to the Shinkansen.
This is the Hikari
Super Express.
We will be stopping at
Start running and
then jump on it.
I'm serious. Run, run, run.
What you go over there for?
Someone will be taken
out before the end of this.
I just hope it's not me.
It's fun. It's exciting.
It's scary but I can't
explain what is happening.
I've had two explants.
I had two taken out of my side
and one taken out of my arm.
One in October and
one in December.
It's exhausting.
I'm tired.
It's a lot physically.
It's a lot mentally,
it's a lot emotionally.
It has been more
than I ever expected.
Okay. So now Amber, don't squish
them but shake it.
Shake it.
That's the last 24 hours.
My insulin dose is about half,
but 50% overall.
I feel good, down
about 20 pounds
without even trying.
I haven't had any extreme lows.
Have I Case?
No extreme lows,
but you've had lows.
Lows, but not extreme lows.
No extreme highs.
So, I think it's definitely
narrowed the gap.
Sounds like the cells
are working.
Something's working.
I don't know what's working,
something's working.
What does Dr. Bellin say?
She said something
that have pointed me
in the direction that,
"Oh, they know something,
but they're not filling
me in on everything,
'cause I think they're
cautiously optimistic as well."
I never had super high
expectations to begin with,
but this is better
than I ever expected.
Even where I'm at right now.
So, would you agree
with that Case?
- I wasn't listening.
-Okay.
Hi Howard.
I don't believe it, necessarily.
Why not? You've been telling us
that the C-peptide is
the gold standard test.
It's the gold standard.
I'm not convinced given
that level of C-peptide,
given that it's a solitary
measurement.
And given that it's
a single patient.
I don't get it.
You guys would
get desensitized
if you were following
each and every patient
of the 11 we're doing right now,
but you're focusing
in on two patients,
the two patients at
the Minnesota site.
Everybody's hopeful.
-Yeah, well.
-Even the PI.
Yeah, I know.
That's why they pay me
to be skeptical.
Deep breath.
Good. Did you check your
wristband to make sure
that everything's
right on there?
-Sure.
-What's your birthday?
-Today.
-It's your birthday today?
-Yes. Happy birthday.
-Yeah.
I would say this cell
survival is moderate.
It just looks like, to me,
they might be fewer
than we would hope for at...
12 weeks.
...Which is a pretty
definitive time point.
It can keep going up after that,
but I wish it were
more right now.
Or you would
expect it to continue
to increase after 12 weeks.
Yes.
It's been 12 weeks since
Maren got her implant
and still no C-peptide levels
have shown up in her blood.
The researchers think her cells
should have matured by now.
They should be
producing insulin.
Do you think it's fair to say
that the patient shouldn't
give up hope just yet,
because as you always
tell me, Howard.
Rats are not humans
and this is an
experimental study.
Sure. Absolutely.
I mean, you have to
consider the outcome.
In rodent, the earliest
that we would anticipate
seeing C-peptide is on the
order of two to three months,
again though, that is depending
upon the assumption
that the time course in man,
is the same as that in rodent.
And we do not know
that for sure.
So, we're still in the
game with these two patients?
We're still in the game
with these two patients
and we haven't ruled them out.
Just want to know,
we've been working
together for a long time
and want to know like,
"Hey, I should still have
hope, me.
Not the filmmaker, but
me Lisa, the patient."
I mean, everything
is getting better.
You just have to be patient.
All right, Howard.
Thanks for your time.
Okay. Bye-bye.
Hi, I'm just going to
complain the whole time.
I'm a mess of a wreck.
I don't know if I can
keep doing this anymore.
I might have to go
back in today again,
which would be
surgery number five
in four months, and so tired.
Tired doing this and
so much physically
and emotionally on me.
All this in hopes of the cure.
I can't compare this experience
to anything in my life, ever.
It has been an
emotional rollercoaster,
is the only way to explain it.
I would give anything.
If I could sit down with
the people doing the study
out in San Diego and say,
"This is what it's
like on our end.
You can go ahead
and see my labs.
You can go ahead
and see the blood work.
But, talk to Greg and me.
Talk to us about
how we're doing."
Nice and close.
But I'm going to
have to stick it out
until someone tells me to stop.
I don't know what else to do.
I mean, I'm knee
deep in horse shit.
So, I don't know
what I'm supposed to do.
I mean, I've already
got eight incisions
all over my body
and been a lab rat.
I'm this far into it.
I might as well keep going.
Me and the other patients
are experiencing
better glucose numbers.
And I think that
could be attributed
to a placebo effect because
we're under a microscope.
Humans are self-conscious.
And they wanna leave
a good impression
So, looking at this
at the broad overview,
I'd say you're doing
reasonably well.
I mean, you're in
target 70% of the time.
Yeah.
You obviously have some
highs, but we expect that.
You have some lows,
but they seem like
they're pretty brief in nature.
You have a little more
highs maybe after lunchtime,
but nothing that I'm concerned
about for you.
Do you think it's working?
I do.
I think it's doing something
because my disease
seems easier to manage,
but it could just be me.
Oh, I like the socks today.
Yeah. Striped.
Used to play a lot of
craps on the streets.
I should probably paint some
dots on this thing.
Make him feel at home.
-What is that, Greg?
- This is my father.
He passed away about a year ago
from complications
of Pneumonia and diabetes.
-Is there an urn in it?
-This is an urn.
-Oh, that's the urn.
-Yeah.
For a Mexican family,
this is it, man.
This is what we got.
Yeah,
this is him.
He was in a nursing home
due to a hypoglycemic episode
that left him
mentally handicapped.
He couldn't live on his own.
He couldn't function.
He had to relearn how to talk
and he... but he
relearned, barely.
So yeah, this is:
Gregory Romero Senior.
Gracious God, we thank you
for Maren.
And for the love that she has,
that has been poured
out into so many lives.
And especially of all her kids,
who are gathered around her now.
Heavenly father,
we pray for Maren
and for the doctors who
will be looking after her,
we ask that your presence
will be with Maren
and be with the
rest of her family.
Lord, we lift her up to you
and know that she is in your
careful and loving arms.
In Jesus' name, we pray.
-Amen.
-Amen.
Katelyn, can stand up.
So, you're getting
some results today?
Hopefully I find something out,
either yes or no.
Are the cell's dead
or are they alive?
Okay, so they did
the explants, right?
Yep. So this is the January one.
-This is the most recent one.
-Okay. A couple weeks ago.
Remember they were looking
at the T cells before
that cause rejection.
So, they still see those cells.
Around the implant
and inside of it.
They see very, very few
from what they told me,
actual donor cells anymore.
Interesting, okay. Yep.
They don't think
that there's any chance
that the remaining ones
are going to make insulin.
Is this happening with everyone?
Do you know?
So, you are the only person
in the study so far that has
had this type of rejection.
Interesting.
So from our standpoint,
if they're not going to work,
then we're putting medicine
that you don't need,
leaving them in when
you don't need it.
Truly, truly I'm relieved.
I feel like I can breathe.
It's been a long five months.
It's been a really
long five months.
Six months almost
since the start of it.
I feel like I can maybe go
back to normal for a bit,
My normal, whatever
my normal is.
Do you feel guilty that the cure
is always 5 years away?
I don't feel guilty.
I feel hopeful.
I feel really hopeful.
I'm not kidding.
-You do?
-Yes.
-Really, for real?
-That's so nice of you.
I'm working here.
I can see what's going on.
It's going to be good for you.
I know it's been
really hard as heck.
Thanks.
I'm not kidding.
It's true.
I mean, there's nobody,
no person with a Dexcom
or a pump or anything
works as well as the cells.
The cells have evolved
over millions of years.
They're the best glucose
sensors, the best insulin
pumps in the world.
That's what biology's all about.
And that's why
we're doing it this way.
It takes a ton of work
and a ton of money,
but it's worth it.
That's good.
We want them to
get blood vessels.
Oh, that's great.
The loading port, right?
-Do you see it, Greg?
-It's the top one.
And for the other
one, there it is.
And then the port, port, port.
So if I come up this way.
I used to have great vision.
I remember as a child,
my dad being diabetic would
ask me to look down the street
and see what bus is coming
and I'd have to tell him.
And I'm like,
"How do you not see that?"
Now I understand.
Mr. Romero.
-Hi.
-How are you doing?
-Good, how are you?
-Good, good.
Your left eye vision
seems a lot more blurry
than it was last time I saw you.
It is a little bit.
Let me take a look real quick.
I looked at your pictures
and I have a suspicion
as to what's going on.
Look up.
Up and left.
and down,
down and right.
And right.
And left.
So, it seems that
you've had some bleeding
inside your left eye
from the diabetes.
Yeah, I see a floater or
it looks like a drop of blood.
Yeah.
So, that's probably at
the bottom of my eye somewhere.
Yeah, there's quite a bit of
blood in there
that is blocking your vision.
And it's definitely worse than
it was last time we saw you.
If it fails to clear, we might
have to do surgery,
-but I'm hoping to avoid that.
-Okay.
-Thank you very much.
-Oh, sure.
Yeah, whatever.
It's all part of the experience,
I guess.
I can't fucking see.
The study is the positive
thing for me right now.
We're just waiting.
We're all waiting
to see if these things work.
If they don't,
we'll cross that bridge
when we get to it.
I have neuropathy, retinopathy
and other
complications due to
the high blood sugars
throughout the years.
A lot of nerve damage I have.
I'm sorry.
I'm not going to say what
I was just going to say.
But yeah.
You suffer from
the disease as well.
So, I'm sorry too.
It's something that
we're all going to have
to encounter and deal with,
every one of us.
Sorry.
But if these cells work,
I'll cry for a week,
like, out loud
and I'm not the type of
person to do that very much.
I'm tired.
You had the ViaCyte?
-Yeah.
-Are they all gone now?
-They're gone.
-Okay.
I assume they're gone.
I got a phone call
and there's
a pancreas available.
I thought about it for years,
for years.
And the trial gave me
the opportunity to
try the medicine
and see how I handled it.
And I handled it just fine.
Recent changes in your
vision or your hearing?
No.
So, it opened the door for me
to think more about
doing this transplant.
-Sore throat?
-No.
I would do the trial over again.
I would do everything
all over again.
'Cause, it got me to this point.
I have zero regrets.
Zero.
-Hi.
-Hi, come here.
-How are you?
-Good, how are you?
Okay.
I'm going to sneak
a hug in there.
See you. Wish me luck.
I'm going to see you next time.
We'll go out for cheesecake.
-Cheesecake Factory
-with sugar.
Okay. Take care.
Come visit me. Okay?
All right. We'll see you, Greg.
One, two, three.
A friend of mine that
had a pancreas transplant
a year and a half ago,
text me this afternoon.
She goes, "You'll wake up
tomorrow with a new life."
So, it was pretty cool.
So, fingers crossed.
I've been going with Paul
to meetings
with potential investors
for the last two years.
Every time you're like,
"Oh, this is it."
These guys get it.
They're excited about it.
And then
they just don't call you back.
Don't reply the email. Or
It just falls apart
one way or the other.
So, that's been really hard.
The pouch is
designed to fully contain
the implant itself,
but still allow vital
nutrients and proteins
such as oxygen,
glucose, insulin,
and other hormones to
travel between the cells.
Well, it's nice. It's a good
description of the product.
Well, it's nice.
Good to see you.
All right.
I'm really pleased to announce
that as of this morning,
the checks rolled in
and we completed an $80 million
financing for ViaCyte.
That's $105 million over
the last three months.
It's really exciting,
but boy, we're going to be
under pressure to perform.
I believe that everybody in here
will redouble their efforts
to really show this new group
of investors
that they made a great decision.
All right, let's do a toast
to the success of this team,
the success of this program and
to curing type 1 diabetes.
I feel like Paul has mastered
the game of selling risk.
He convinces venture capitalists
to embrace the long game,
that science takes time
and progress is incremental.
You got to be optimistic
and stress resistant.
Two criteria.
If it doesn't work in
patient two,
it's going to work in 22.
But what if Paul's
strategy hadn't worked?
I can't help but think
this promising trial
would've been cut short,
only to languish on a shelf
next to the thousands
of other patents
collecting dust.
ViaCyte believes it
has the key elements needed
to produce transformative
therapies to treat diabetes.
Hello? I'm Greg.
Good. How are you?
We're going to talk
about your results.
-Yeah.
-And when you had your surgery
to remove one of the
small cell pouches.
So, I had a really
good conversation
with a sponsor yesterday.
And so there are some
donor cells there,
but there are not many of them
and they don't expect them
to get any better
from this point on.
So, we look for
C-peptide as a marker
of the cells actually making
insulin in your bloodstream
and that's always been negative.
So, I don't know if we're not
going to get any more benefit
from you, that it's
worthwhile for you
to have to continue to
take these medications
and have potential side effects
-and risk from the medications.
-Yeah.
And I, had to...
-Yes, I feel the--
-You feel the same way.
Well, you've done a great job
of taking care of yourself
and staying on top of things
through this whole process.
So, well then that would
lead me to this question,
which is, so you guys
have a Eureka moment
and this is something
that's going to work
for a large majority
of the population.
How long would
it take for your guys,
the cure, the solution
that you found
to hit clinics where
I'd be able to actually go
and get the dang thing?
Yeah, right. Good question.
That probably is at least,
from the time they're starting
these phase one studies,
I'd say it's probably at least
a five to seven year process.
Okay.
All right.
Other questions you have?
I don't know.
The more silent I get outside,
the more loud it
gets inside my head.
So, even if the patients
we've been following, Howard
don't seem to be
producing C-peptides
or essentially
their own insulin,
that's not a failure?
It depends upon how
you define failure.
It's obviously a setback
for these patients
who have volunteered
their time and effort
to participate in
these clinical trials.
But we've learned
a lot from them
and we continue to tweak
the implantation procedure,
trying to improve the
engraftment process.
And we continue
to make gains there.
What's the mood like
at ViaCyte right now?
I think the mood is upbeat.
Everybody is excited.
I mean, everybody
here at ViaCyte
is convinced our cells
do a great job,
they work the way
they're supposed to
and behave very physiologically.
They will cure type 1 diabetes.
There is one thing that
you would like to know.
Yeah.
Remember the first
patient from Minnesota.
Yeah.
We early terminated that patient
because of lack of C-peptide,
lack of potential
efficacy and the like,
so we explained all of
the remaining units.
Would you like to know
what the histology showed?
Yes.
That were viable clumps
of viable cells there.
So Howard, what does that mean?
What's the takeaway?
What's my takeaway
as a layperson.
The takeaway is
that cells can survive
for long periods of time in
this encapsulated environment.
And that's important.
That's really good news.
It's great news.
Would've been even better
if we'd been seeing C-peptides,
but you know, you don't always
get exactly what you want.
If you actually could
talk to these patients,
the ones we've
gotten to know well,
is there anything that you would
tell them as a message?
We owe all of these subjects,
a tremendous debt of gratitude.
Thus far, we have been
able to give them a lot
in terms of effectively
treating their own disease.
But we have gained a
tremendous amount of knowledge,
which hopefully will help us
get across the finish line
in terms of future iterations.
What are you thinking?
If it hadn't been
for Greg and Maren,
for the other patients who
participated in this trial
and they're up to patient 19,
they wouldn't be
where they are today.
And it's been a huge
sacrifice for these patients.
It's just been
really hard on them.
And I wish it had
worked for them,
but they're paving
the way for others, so,
that's it.
I was trying to come
to terms with the fact,
this may not work.
The trial wasn't over but
it was for Greg and Maren.
Any of the negative crap
you hear coming from me,
that's subjective crap.
That's Greg.
I know that I've been part
of something very important
and very beneficial to us.
All of us with diabetes.
Officially, as of today,
I'm considered a failed
transplant patient.
I have an appointment at my
1-year mark, late July
to get relisted.
I'm young, I'm healthy.
So therefore I'm a priority.
I'm not ready physically or
emotionally yet to do it again.
In time, maybe I will.
I have shared only this
with a few people.
I will cross that
bridge if people ask,
but it's a tremendous loss,
a loss I thought I was
ready for, but I was not.
Thank you for listening.
-Hey,
-Good to see you.
-Good to see you.
-Yeah.
We have the data that
shows that the product
is producing insulin in the
patients for the first time.
It's the first patients ever
in the history of
diabetes cure research.
So, this is a big deal.
I mean, we know now
that the cells work,
we didn't know that
five years ago.
All the pieces are there.
It's just a matter now
of completing the puzzle.
We all want stories
with a beginning,
middle and end.
One where all the loose
ends fit together.
But clinical research
is messy and hard.
It doesn't fit into
a tidy headline,
no matter how much
you want it to.
The cells may not have
worked for Greg and Maren,
but they're producing
insulin in nine patients.
Nine patients.
Because of them,
we are one step
closer to a cure.
But science alone isn't enough.
It needs a spotlight,
a champion
and money.
If we rethink how
we cure disease,
then the breakthroughs
that we so desperately
need and want
will come.
Will we get there in five years?
I think we can.
♪ I met two angels
♪ But they were in disguise
♪ Took one look to realize
♪ Tell them anything
♪ And they will sympathize
♪ These arms hold me tight
♪ Old fears
♪ Why me
♪ How'd I get this hallelujah
♪ Hallelujah
♪ Been that way since '95
♪ Three roads, one light
♪ Why me
♪ How'd I get this hallelujah
♪ Hallelujah
♪ Hallelujah
♪ Why me
♪ How'd I get this hallelujah
♪ One I wish I could see now
♪ These arms reach out
♪ Why me
♪ How'd I get this hallelujah
♪ Why me
♪ How'd I get this hallelujah
♪ Why me
♪ How'd I get this hallelujah
♪ Hallelujah