Original subtitles

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