Extraordinary Measures (2010) - full transcript

A Portland couple have two children with Pompe disease, a genetic anomaly that kills most before a child's tenth birthday. The husband, John, an advertising executive, contacts Robert Stonehill, a researcher in Nebraska who has done innovative research for an enzyme treatment. He has little money to fund his laboratory, and a thorny personality that drives away colleagues and funders. John and his wife Aileen raise money to help Stonehill's research and the required clinical trials. John takes on the task full time, working with venture capitalists and then rival teams of researchers. Time is running short, Stonehill's angry outburst hinder the company's faith in him, and the profit motive may upend John's hopes. The researchers race against time for the children who have the disease.

- Hi there.
- Hi.

So glad to see you.

Kathy, could you take that?

Thank you.

It's my job to market this drug for
Bristol-Myers, and I'm telling you,

kids won't go for it
unless it tastes like bubble gum.

- Mr Crowley, you're late.
- All right. Thank you. Bye.

"Everyone just walked by.

"'Why won't anyone buy our newspaper? '
SpongeBob wondered aloud.

"'They must be rock haters,' said Patrick."

- John! Give it back to me!
- She's mine now!

- Give me a minute, okay?
- Give it back to me, John!

You save our spot. Got it?

You better give me back my Fiona!

Help, Mom!

- Help me! She's trying to kill me!
- I'm gonna get you!

- You can't escape!
- No! Mom, help me!

Just give it back to me.

- Mom! She's trying to kill me!
- Megan Kathryn Crowley.

If you kill your brother,
you cannot have your party.

Why not? He stole Fiona.

I didn't steal her, I kidnapped her.
I'm holding her for ransom.

Release the victim.

Time to get ready for your party.

We've got to get moving on
the product launch.

I'm just waiting on legal, John.

Damn it!

I just missed my train.
I'm gonna have to call you...

I agree. How soon do you need it?

As soon as possible.
I'm presenting to my boss next week.

John, we're loading up the van right now.
Come on!

- Okay, we ready to roll, Kate?
- My name is Jane.

- Kate was yesterday, Mom.
- I am so sorry.

The way the agency shuffles
day nurses on us,

you'd think they'd, you know,
give you a number instead of a name.

Okay, John.

John, come on, get your butt in the car.


- Are you bringing your RipStik?
- Yes.

- Do you have to?
- Yes.

...a pain, that's why.
Wait, I gotta catch a cab.

Hi. Come here. Hey, hey. Hello?

- Daddy's meeting us there, right?
- Absolutely.

I am sure he has everything under control.


You need a hand?

Great. I'm sorry, I'm sorry.
I'm kind of in a hurry.


Come on, come on, come on,
come on, come on, come on.



He totally promised he'd be here by now.

He's on his way, baby. He's on his way.

Hi, guys.

- Get in there and give her a kiss.
- I know.

- I don't wanna hear it. I don't wanna hear it.
- Go, go, go.

Happy birthday. I made it.

- Yay!
- Yay!

Make a wish, Megan.

Who wants cake?

- I do!
- I do!

Line up single file, or no cake for you.

- All right, how many do we have?
- I'm first! Me first!

- No, I'm the big guy.
- Back to the end of the line.

So, where is Fiona going, anyway?

Lollipop Land?

I'm eight, Daddy. I'm not a baby any more.

She's driving to Antarctica
to see the penguins.

Really? That's pretty far away.

Why doesn't she just go
to the Portland Zoo?

They have a lot of penguins there, too.

- Haven't you ever heard of adventure?
- Yeah.

It's gonna take her years.

Won't she need a suitcase?

It's an adventure.
You don't need a suitcase on an adventure.

Well, I don't know about you,
but if I was going on an adventure,

I would take a suitcase.

- That's 'cause you're a businessman.
- That's right. I am.

And you're eight years old.
And happy birthday. And I love you.

Love you, too, Daddy.

- Good night. See you in the morning.
- Good night.


Sweet dreams.

Good night, Dad.

- Good night, Daddy.
- Night, buddy.

- Hello?
- Hello, Dr Stonehill?

- Are you there? Hello?
- Yeah.

This is John Crowley. I don't know
if you've gotten any of my messages.

- Who?
- John...


I'm interested in speaking with you.
I understand from many researchers...

...having a conversation with you
if that's convenient.

Is this a good time to talk?

Dr Stonehill? Hello?

- Asshole.
- "Asshole?"

Yeah, let's up
her breathing treatments today.

- What's going on?
- Just a cold. She's awfully congested.

- Call the doc just to be on the safe side?
- Yeah, I left word.

Sweetie? I'm gonna go to work, okay?

Feel better.


We're already doing great
marketing the drug to physicians.

It's our direct marketing to patients
we need to beef up.

Mr Crowley, I'm really sorry.

Aileen's on the line,
she really needs to speak to you.

Okay, thank you. I'm just gonna... Hey.

We're at the hospital.
The doc wants Megan under observation.

- Is she all right?
- Just keep your cell with you.

- Okay. Can I do something?
- I'll call you when I know more.

I should really get back to Megan, so I'll call
you as soon as I know anything, okay?

Bye. Love you.

- Excuse me, please.
- Sir?

My daughter, Megan Crowley,
was brought in for observation.

Actually, they're moving her to the ICU.

- Where?
- That way.

- Aileen.
- John.

Has she told you about the trip that Fiona's
taking with her remote-controlled car?

Yeah, yes, she did. You're right.
That's right, sweetie.

You're gonna see
all the penguins, aren't you?

She might send some postcards
or something like that.

- Lots of good stuff to see along the way...
- It's okay.

...like icebergs and maybe a polar bear.

How about some seals?

As you know, Pompe patients suffer muscle
deterioration in every part of the body,

so this sort of respiratory complication, well,
that's just an inevitable fact of the disease.

Mr Crowley, Mrs Crowley,

Megan is not responding well.

Okay? Now, we've done everything we can,
but she's just...

There's nothing more we can do. I'm sorry.

As you know, she was already well past the
life expectancy, for, you know, Pompe kids.

After all, it's not just her lungs, okay?

Her heart, her liver,
these organs have been compromised

and would've become fatally enlarged.

Now, I wish that we had a drug
to treat Pompe, but we simply don't.

I'm so very sorry.

Look, maybe...

Maybe you can find some small comfort in
knowing that Megan's suffering will be over.

Maybe you can see this as a blessing.

- Megan?
- Help!

- Help! Help!
- Megan?

Help! Help!

Baby girl, can you hear me?

- Please, someone come help!
- 321!

- Megan Crowley, room 321, please!
- Code Blue!

- Baby girl, Mama's here, all right?
- Let's go! Page Dr Heder.

- Asystole!
- Yeah, right through here.

Can you hear my voice?
Can you hear Mama?

- Starting compressions.
- I'll switch her out.

- Over here, please. Quickly.
- Hang in there, Megan.

- Hang in there, baby, okay?
- I need to bag her.

- Aileen?
- Some room right there.

Let us in, all right?

- Please, Mrs Crowley, let...
- Stop it!

Let us help her!

- Please... Megan!
- Aileen.

- Epinephrine, 0.1 mils per kig.
- Got it.

Still no pulse. No reading.

- Forget the weight, come on, get it in there!
- Aileen, Aileen.

- 0.1 epi.
- It's all right.

- Come on, Megs. Come on. Come on.
- IV push.

- Okay, just give the epi a couple seconds.
- Still non-responsive.

- All right, charge the defib.
- Charging.

Your daughter is some fighter.

- She's gonna be okay.
- Oh, my God.

Now, look,
she's still a very sick girl, obviously,

but her vital signs have improved
and I'm cautiously optimistic.

So, I guess you could say
we dodged that blessing, huh?

- Mrs Crowley, these came for Megan.
- Thanks so much. Thank you.

Hey, John?

Any time you're ready, man.

You gonna tell us
about the product launch?

John, you okay?

I have to go.


- Where you going?
- Nebraska.

Excuse me, please.
There's a gentleman here to see you.

Best make yourself comfortable, hon.

Oh, dear.

Sir, sir.
That's Dr Stonehill. He just left, I'm sorry.

- Thank you.
- Yeah.

Dr Stonehill!

Hey, Dr Stonehill!

Dr Stonehill?

- Yeah?
- I'm John Crowley.

- Okay.
- Didn't you get any of my messages?

What messages?

I left you three or four messages
in the last month,

and you even hung up on me
the night before last.

What the hell are you talking about?
I never hung up on you.

Dr Stonehill, two of my kids have Pompe.

Oh, Jesus.

Well, I'm sorry.

A terrible disease,

but, hell, you shouldn't have come
all this way without talking to me.

I do research, son. I don't see patients.

Well, it's your research
that I'm interested in.

I've read all the journal articles
on Pompe disease,

and all the researchers out there say
that your work holds the most promise.

Get to the point, son.
What do you want from me?

We almost lost my daughter last week.

I need to hear about your research.

Your kids have a genetic disease.

They're missing an enzyme

which metabolises
a certain kind of sugar, glycogen.

It's defective in Pompe patients.

So, this sugar builds up in the muscle cells,

especially in the heart, the skeletal muscles,
the diaphragm,

which is why they have
a hard time breathing.

- Yeah, I know. I know a lot about that.
- Everybody's making an enzyme,

but you can infuse kids all day long
with their enzyme,

and it's not gonna do any good
'cause it's not gonna get into their cells.

Understand what I'm saying?

Yeah, so that means
that if you put the sugar...

- Don't interrupt.
- Sorry.

I'm making a different version
of this enzyme.

My enzyme has a biological marker,
mannose 6-phosphate.

I'm the only one that's cloned the gene
for phosphotransferase.

I'm the only one who's cloned the gene
for uncovering enzyme,

so I can get more mannose 6-phosphate
onto the lysosomal protein,

and I can deliver
a more effective amount of enzyme

into the cells.

That's why people are citing my work.

That's why you read my name
in the literature.

I can get more enzyme into the cells.

What's the matter, Sal?
Not up on your glycobiology?

I thought Doc was just your nickname.

No, that man's a genius. He's on the verge
of a scientific breakthrough.

Wow. Who knew?

I'm not on the verge of anything, kid.
This is a theory, not a therapy.

I'm just an academic.

- You got any idea how shitty my funding is?
- No.

University of Nebraska
pays their football coach

more money in a year
than my entire science budget.

Nobody's paying attention to this work.

Half my grants don't get approved.

I don't have the money to make my theory
into a useable medicine.

How old are your kids?

Six and eight.

Look, do yourself a favour.
Go home and spend time with your kids.

Enjoy 'em while they're still here.

- How much?
- How much what?

How much money would it take
to prove your theory,

to create your version of the medicine?

I'd need half a million bucks
just to fund the lab work.

- That's why you need me.
- I need you? Why?

Because I'm the founder of
the Pompe Foundation for Children.

I've been working on this disease
for 10 years.

I have never heard of you guys.

That's because we're relatively new.
We're just in the gearing-up phase.

- How soon do you need a grant?
- Now would be good.

Well, not all the money's in place,
but it will be, and soon.

This is a very exciting time.

- Thanks.
- Thank you, sir. Have a good day.


You okay?

- Did you get my messages?
- Your messages?

You suddenly walk out of the meeting

and fly to Nebraska
without talking to me about it,

and you think messages make it okay?

- I'm sorry.
- My God, John.

I wanna find a miracle
as much as you do, okay?

But how could you put your job in jeopardy?

- Aileen.
- How exactly

do we pay $40,000 a month in healthcare
if you lose your insurance?

Honey, come on.
Look, I'm not gonna lose my job.

Oh, really?

Pete called here twice last night
to ask if you were okay.

You know, mentally.

I'll just...
I'll call, and I'll smooth that one over.

Yeah, no shit.

I'm sorry.

Something in me just kind of snapped.

That night at the hospital,
when we thought that we were losing her,

I prayed that if it was her time,

she'd go quickly, without so much suffering.

But morning came, and she pulled through.

Her eyes,

so determined, so defiant.

The fight in her.

Was he worth it? This Stonehill guy?

He's really eccentric, but his science
is way ahead of everybody else's.

- He's definitely the one to put our money on.
- Lf we had any.

I promised him that we'd raise some,

because he's got this conference up here
next month,

and I said I'd have a cheque ready for him.

- How much?
- Five hundred.

- That's all?
- Thousand.

Is it $500 or $1,000?


By next month.

- Sweetheart, are you totally insane?
- Apparently.

We're holding our first fundraiser
on the 23rd at the school,

and we would love
if you and Frank could join us.

- Ready for your spelling test?
- I'm gonna nail it.

Okay. Good luck.

My name's John Crowley.
I'm calling from Oregon,

and I got your name from
the Atlanta-area Pompe parents' group.


And I was so sorry
not to see you at the reunion

because you were one of
my most favourite people in high school.

Anyway, John and I
are starting this foundation.

- Hey, Dad? Dad.
- No, you know what I mean.

Just make it right.

- Dad?
- Can't you see I'm on the phone?

- Okay, well, tell me what he told you.
- Dad!

No, I'm gonna have to call you back.
I can't talk. All right, bye.

- Yeah, what is it?
- I sold my RipStik.


Is it enough to give to
Megan and Patrick's foundation?

Yeah, it's more than enough.

- Are you John?
- Yes.

- Marcus Temple. We spoke on the phone.
- Right. Absolutely. Good to see you.

- All the way in from Atlanta.
- Yes.

Well, this is for your foundation,
from my relatives, friends and church group.

My goodness.

Thank you very much.


- Hi, Dr Stonehill. I'm Megan.
- Hi, Megan. I'm Dr Stonehill.

Yeah, that's why I said, "Hi, Dr Stonehill."

- Are you coming in or not?
- Yeah, thanks.

Where's Mom and Dad?

They'll be down in a minute.

Patrick's vent alarm is going off,
so they need to fix it.

His alarm goes off even more than mine.

So, what's your deal? You married?

Do you have a wife?

Ex-wives. Two of 'em.

- Yeah? How come?
- 'Cause I'm so easy to get along with.

- Any other questions?
- No. Your turn to ask one.

You're probably wondering
what grade I'm in.

- What grade are you in?
- I'm in third.

My hobbies are video games and penguins.
How about you?

- I already graduated.
- No, I mean hobbies.

Mostly I just work.

Little bass fishing
every once in a while, but...

What's your favourite subject?

I'm good at reading, but I like P.E. Best,
especially the sprint races.

Sprint races?

- Wanna see?
- Sure.

Come on.

- Is he here yet?
- I don't know.

I'm gonna win!

Come on, slowpoke.

Yeah, I won!

I won.

Oh, yeah.

Told you I'm gonna beat you.


Dr Stonehill has two ex-wives.

- I see that Megan's introduced herself.
- Yeah.

- How are you, Dr Stonehill?
- I'm okay, thank you.

- This is Aileen, my wife.
- So nice to meet you.

- And John Jr.
- Hello.

- Here's Patrick.
- Hi.

- Hey, Patrick.
- I think we should probably go inside.

Yeah, come on inside.

Megan, we'll see you inside. Come on.

- Night, Patrick.
- Good night.

Night, SpongeBob.

- You gotta say good night to Dr Stonehill.
- Kiss SpongeBob.

- Good night.
- Good night, son.

Good night, Patrick.

I'm not gonna say it again. It is bedtime now.

It's for him.

- Me?
- For good luck. For bass fishing.

Thank you.

You like it?

Well, sure. A happy-go-lucky little fellow,
lots of personality, huh?

- He's a plastic toy.
- I know.

Here's the father-daughter dance.

You haven't lived until you've seen
Megan do the Wheelchair Watusi.

Here you go.

- Go ahead and eat the cobbler while it's hot.
- Smells great, honey.

This was hilarious.

Bob, we went to this dude ranch.
All right if I call you Bob?

You got a cheque for a half-million dollars,
hell, you can call me Peggy Sue.

I'll just...

Well, Bob.

This is the first instalment
against the promised half a million.

Tell you the truth,
that's more than I expected.

Dr Stonehill, we promise that...

- Yeah, I know.
- Lf... In enough time, then...

I know. I know.

You can learn a lot about a person
on the Internet these days.

Learn about a working-class kid
from New Jersey,

worked his way through
Harvard Business School,

clawing his way up the ladder
at Bristol-Myers.

But what Google can't tell you

is that this scared, desperate, bullshitting
son of a bitch...

- Wait just a minute...
- Don't interrupt.

Will do and say whatever it takes
to get results.

Which is why you and me are going
into business together, Jersey.

Excuse me?

I'm tired of begging breadcrumbs
from the university

and giving them the patents to my ideas.

They don't value my work. You do,

which is why I'm setting up my own shop.

Figure any dude in a business suit

can help me raise venture capital
and run the company,

but who's gonna be half as motivated as
a dad who's trying to save his own kids?

So, this is...

So this is the shittiest offer
you're ever gonna get.

I can promise you less money, longer hours,
lousy working conditions,

plus, if we raise the money,
you're gonna have to relocate to Nebraska.

And with the right business plan,

I can also promise you
a working enzyme for Pompe disease.

I can't cure your kids, you know that.
They're always gonna be in wheelchairs,

but I think I can save their lives.

Outstanding cobbler.

Okay, okay.
So, we agree we can't uproot the kids.

What if Stonehill fails?

Or what if he succeeds too late
to help Megs and Patrick?

- Then what?
- I know.

If we're gonna lose them young, you want
to spend every minute you can with them.

John, that day that you flew to Nebraska,

when you suddenly just ditched work and...

My God, I thought you'd lost it.
You scared me.

But once we rolled our sleeves up,

and we started fundraising
and meeting other Pompe families,

it made me feel like...

I mean, do we just accept our fate

and do what we're told
by all the well-meaning doctors

and wait for the worst to happen,
or do we fight it?

I think you're making a mistake. I really do.

Even if you were credible as a CEO,
which you are not,

the odds against you are crushing.
Nine out of 10 biotechs crash and burn.

So where does that leave your kids
when their dad is flat broke

with no job and no health insurance?

Now, if you stay at Bristol,

the guys upstairs are ready to put you in
the VP slot at the end of the third quarter

with a 40% salary bump.


Your family's gonna need that money,
aren't they?

Pete, you're right. This is crazy.

I'm chasing the wind.

But I can't just

sit around and wait for my kids to die.

I can't do it.

You have arrived at your destination.

Come on in. It's open.

- Hey, there, Bob.
- Hey, John.

How are you?

- You have trouble finding me?
- No problem.

- Sit down.
- Thanks.

Ex-wife. She loved cats.

Well, say, what do we...

What are we gonna do?
How about we work up a strategy

for the meeting at Renzler next week?

We don't need to show those guys anything.

The meeting's a formality.

George Renzler and I have known each other
since med school.

He's been telling me for years that
he'd give me the seed money

if I ever decide to make the big move
and set up my own shop.

Well, that's good. That's good,
but, still, all the same, you know,

if we're gonna go in to pitch 'em,
we should have something tangible.

Those guys understand me.
They're real scientists.

They're not like a lot of
these big biotech moneymen.

Okay. All right, tell you what,
how about if we just talk it through,

and then that way we can just sketch up
something rough on paper?

You give it to me,
I'll make up a business plan.

We need to rough out a real strategy.
These guys are venture capitalists.

That rough enough for you?

Bob? Look, Renzler's crucial to us. Without
them onboard, we got zero credibility

- with any of our investors.
- That's why you're here, Jersey!

Whip up some business bullshit
by tomorrow.

- I'm going fishing.
- This is... This is gibberish!

Then you better get busy.

See you.

As you know, the market potential
for an orphan drug is enormous.

If you were still in business school,
I'd give you an A

for all these lovely charts and graphs and...

But school is out.

Most of us here are scientists.
We need to see the science.

Bob, make us believe.

Love to, George.

...the phosphotransferase
and the second uncovering enzyme...

At this point, the challenge
is to scale up this process

to produce kilogram quantities of HPGAA.

And the question is
whether or not it's cost-effective

to contract this work out
or to build our own manufacturing facility.

Either way, we're gonna eventually need
an investment on the order of $10 million.

It's a lot of information
in a short period of time.

Does anybody have any questions?

- This is astonishing.
- Well, thank you, George.

This man is light years ahead of the field.


Well, why don't you have your money guys
get together with John

- and work out the business...
- Sounds great.

Of course, we'll need you to explain
some of the mundane stuff, Bob,

the nuts and bolts.

- Nuts and bolts? What...
- Yeah.

You're a brilliant theoretician,

but you've never actually brought
a new drug to market before, right?

No, I've never brought a drug to market.
I haven't.

I mean, for example, how are you getting
the phosphotransferase?

I'm purifying it
out of a lactating bovine udder.

I see. And where are you getting the udders?

From the stockyards.

- Pretty straightforward stuff.
- Not really.

- You can't inject cow protein into people.
- I know that.

The bovine version of the enzyme
is for lab work, for proof of concept.

I'll make a copy of the human enzyme
for actual drug trials.

- What about uncovering enzyme?
- What about it?

How exactly do you plan on making

- an exact replica?
- I'm making it in T-293,

out of human kidney cells.

- Oh, my goodness. No.
- "Oh, my goodness. No?"

No. The FDA will never allow that.

- Hey, Bob, just...
- Wait a second.

For Christ's sake, we will change
the cell line before clinical trials.

You've gotta have that process locked down
long, long before you think about...

Bob, why don't we just take a coffee break...

Why are you talking to me this way?
I don't want any goddamn coffee.

Bob, can you just tell us
how you're planning to make

the three enzymes
under FDA-approved GMP in...

I'm not here to cross every "t"
and dot every "i", George.

And I'm not here to be poked and prodded
like a first-year med student.

- This is ridiculous.
- Bob.

This is bullshit.


Bob, if you can't answer a question,
you say you're working out the details.

You don't storm out on these guys
like some sort of spoiled child, okay?

- Now, we're gonna go back in there.
- No, I'm not going back in there.

We're going back in there.
This is business, Bob. It's not personal.

That was personal.

Stonehill goes and completely sabotages
the meeting.

Thank you.

I mean, he completely blew my plan,
right out of the gate.

Okay, I get that he's a loose cannon,
but do you still believe in his science?

Honey, if I don't raise $10 million in capital,

his science doesn't matter. At all.

What's going on with his arm?

Hey, buddy, you okay?

- You all right?
- I can't throw them any more.

- When did this happen?
- I don't know.

- You should help him, Daddy.
- That's a good idea.

Okay, here we go. You ready?

- One, two...
- Into the water.

That was a good shot.
We can do better than that.

Try again.
Let's make, like, a little pellet.

Here, ducky, ducky.

Let's do it again.

Ready? One, two, three.

Give me another piece, John.

I can just throw them.

And you do all the laughing,
and I'll do all the throwing.

That's good, the ducky shared.

- Did you see that?
- Yeah.


- We're out of time.
- I know.

He doesn't have the strength
to throw a piece of bread to a duck.

- It scares the hell out of me.
- Me, too.

John, what are you doing?

I'm offering Renzler a deal
he can't turn down.

Excuse me. Morning. Dr Renzler?

Dr Renzler?

Sir, good morning.

- John Crowley.
- Crowley?

- Yes, sir.
- What are you doing here?

- Good morning.
- Good morning.

I understand from your secretary that
you're going to be leaving for France

- for a month.
- That's right.

I have a proposal for you,
and I was hopeful

- that you could take a look at it.
- We're running late for a flight.

I appreciate that. It'll only take a second,
just to have a quick read.

- Fine. I'll read it in the car.
- It might interest you.

Good, thank you.

- Does Stonehill know about this?
- Of course.

And he's okay with these terms?

All I need is your signature.





Over here.



Hi. Bob.

What'd you do to yourself?

- I got the investment from Renzler.
- You what?

Listen, I want you to

think about something before you read that.

What the hell is this?

Under the circumstances,
this is the best deal that we could ever get.

This isn't a deal.
This is the terms of our surrender, for...

Renzler comes in
for a couple of million now,

and you give him the option
of jumping in as a full partner later?

Well, after sabotaging our meeting,
I had to do something dramatic.

And you promise him
we'll be in clinical trials in a year?

We can do this, Bob. All right?

We work harder, we push ourselves,
we work around the clock.

I already work around the clock!

Why don't you just give 'em
my balls in a jelly jar?

There's no other way I could get them
to come back to the table.

Who needs the bastards anyway!

Without Renzler, there's no reason for us
to even think about other venture groups.

Fine! Then I won't think
about other venture groups!


Spend the rest of your life dreaming up
great ideas that don't get funded.

Draw brilliant diagrams on the wall
that cure diseases in theory,

but never help a single human being
in reality.

What'd you do? Let me see it.

- It's just barely...
- Let me see.

Put pressure on it.

Got your blood all over this thing.

Want me to add a drop of mine,
make it official?

Or would a signature suffice?


I'm not paying a dollar more
than 22,000 a unit, okay?

I'm not doing it.

Hi, Bob.

These guys make me feel old.

Scientists get all sensible and careful
when they get old.

Young ones like risk,

not afraid of new ideas,

and you can pay 'em less.

We all believe in Bob Stonehill's
bold vision, otherwise you wouldn't be here.

But to reach clinical trials inside of a year,

I'm gonna have to ask you
to commit to a brutal schedule.

Ladies and gentlemen,
reset your watches to Priozyme Time.

- How long?
- Three, four hours.

Well, if you can get it any quicker,
that'd be good.

I'll try.

Come on.

- Vinh, check the breakers.
- Got it.

- No, the whole building's out.
- Well, get the backup generator going.

- We...
- Wait, wait, wait.

You never bothered
to buy a backup generator?

We're in the middle of tornado country,

- for Christ's sake!
- Yeah, so I'm a meteorologist?

If I don't have my refrigeration back
in 45 minutes, I lose the whole cell line.

Four months of work pissed away!

It was never in the budget.
We never had the money for it.

- I'm sorry, sir. Your card, it's been denied.
- What?

It got maxed out.
I bought the roller bottle unit for the lab.

Shit, I...

Just pay me back by the end of the month,
or I lose my apartment.

Hi, here you go. Thanks.




Thank you.

Fiona's in Mexico this week.
They've got pyramids there.

She's still got lots of things to see
before she gets to Antarctica.


She's gonna chop up
all the penguins with an axe.

And feed their bloody
remains to the walruses.

I see.


Every time you come home for the weekend,
you don't even listen to me.


You know what, you're right. I'm sorry.

It's just that I've been so busy

trying to make the special medicine
for you and Patrick.

- I want it pink.
- You want what?

The special medicine. I want it to be pink.

Okay. I guess I can ask Dr Stonehill.

Tell him dark pink, like this.
Not light pink. That's for babies.


Here you go.

Dr Renzler, be reasonable.
The company is only six months old,

and we are making great progress
towards in vivo testing.

I don't know.
The way the company is burning money...

Well, we've got to get a leg up
on the competition.

You know that Zymagen has
far more spending power than we do.

So, what you're saying,
I should have invested in Zymagen.

No, what I'm saying is,
is that we've got to beat 'em to the punch.

Either that, or scare them into buying us out.

Well, you're scaring us investors a hell of
a lot more than you're scaring Zymagen.

You sell the company to Zymagen,

you're in clinical trials
by the end of the fiscal year,

or we pull the plug and cut our losses.


This construct's looking pretty good,

but we gotta get better cleavage
between the alpha and the beta subunits.

Well, what if we add a furin site
like the one in uncovering enzyme?

Why would you wanna do that?
Doesn't it have its own...

We need to talk.

- I'm busy.
- You're always busy.

You always wanna talk.

Yeah, we could add furin
and try and cleave it before purification.

What are you doing?

- Giving you a preview...
- Guys, would you excuse us for a minute?

...of what's going to happen
if we are not in clinical trials in four months.

Our investors will turn out the lights.

Science takes time, Jersey.
Don't they understand?

Yeah, they do.
They can read the Wall Street Journal.

They see that Zymagen
is testing three different Pompe drugs.

They're testing three 'cause they don't know
what the hell they're doing.

- I'm testing one 'cause it's the right one!
- I know. I believe you, Bob.

Why else do you think
I put up with all your shit?


I want you to go toe-to-toe
with Zymagen's scientists.

- Prove to them that your enzyme is best.
- I can do that.

That's the reason I've entered
into conversations with them

to buy our company.

You're telling me? You're not asking me?

Come on, Bob.
I am just being fiscally responsible.

Nobody is gonna tell me how to run my lab!

If I can engineer a deal,
and that is a really big "if,"

you're going to have to forgive me
for all the money I'm gonna make you.

I don't care about money.

I'm a scientist.
I care about more important things than that.

Don't you tell me about
more important things to care about.

Do the math.

Either we sell the company
and get a huge cash infusion,

or the investors will shut us down.

Where you going?

I'm gonna go take a crap,
if that meets with your fiscal approval.

What, they can't drive from the airport
like ordinary people?

Bob, we need to make this happen.

They're gonna ask us some tough questions.

Don't take 'em personally, all right?
All right?

These lab results confirm
what I've been preaching for years,

that phosphorylation is key
to enzyme absorption.

My theory works for Pompe,

and eventually it'll work for
enzyme replacement therapies

for other lysosomal storage disorders.

- Is it too complicated?
- Too complicated?

The number of variables built into
your approach are mind-boggling.

I mean, Jesus, if you get even
one piece wrong in the synthesis,

just one carbohydrate ends up
in the wrong position...

Well, it's gotta be done right,
like anything worth doing.

You've never actually gotten
a drug approved.

Isn't it naive to think you can solve
these kinds of manufacturing challenges?

Bob? Bob?

You're right, I'm a theory guy.

My head is in the clouds.

Which is why we need you.

Unless, of course, your reputation

for solving protein-manufacturing problems
is undeserved.

Nice one.

- I still need to hear about profitability.
- Certainly, certainly.

As you know, this is not
a one-time treatment. It's a lifelong therapy.

And these patients,

they are going to generate revenue
over the span of their lifetime that is...

But even if you make a usable enzyme,
clearly it won't work for all patients.

So, what survival rate do we need
to achieve a robust profit?

What rate of patient death
can be defined as acceptable loss?

Acceptable loss?

I understand the question.

Because the profit margins on this
orphan drug are so incredibly high, the...

Even with a mortality rate of,
say, 25% over five years still indicates

that there is a highly lucrative
revenue source to be realised.

Gentlemen, we have a deal.

Dr Robert Stonehill to see Barry Renee.

I have a Dr Robert Stonehill to see Dr Renee.

- Sir, you need to be cleared.
- I'm just gonna use the restroom.

- You need to be cleared to do that, sir.
- Just need to...

- You need a security badge.
- Jesus.

Well, can I have a security badge
so I can go take a pee?

Yes, sir. He will give that to you.

- Hey, Bob.
- Hey, John.

You getting comfortable?

Define "comfortable."

You haven't cashed it yet?

I haven't earned it yet.

I'll cash it when we have a usable enzyme.

Well, it sure is beautiful.

It sure is big.

The kids love it.

Seize the day.


I'm stuck on this call.

Just wanted to let you know how thrilled
we are to have you onboard, John.

It's an honour.

- You remember Dr Kent Webber.
- Of course.

- It's good to see you again.
- Good to see you, too.

I'll let the two of you get into it.

- I'm expecting great things.
- Yes, sir.

I've got some pretty good ideas
about how to help the flow of information

between the four core enzyme groups.

Whoa, John, slow down.
That's not our established procedure.

Pardon me?

The four core teams are essentially
in competition with each other.

It's an entrepreneurial model. It works.

I'm all for competition,
but surely there must be some channel

for allowing sharing scientific insights
that can help develop all four enzymes.

John, in the interest of saving us time,
let me be blunt.

Most of the scientists here don't like the idea

of having a non-scientist
as senior VP of the Pompe programme.

Especially one whose objectivity

might be clouded
by having children with the disease.

I'm telling you this
in your own best interests.

Erich wanted to buy Stonehill's ideas,

and he couldn't do that without also
swallowing you as part of the pill.

My advice to you, John,
is to keep your head down.

- Thank you for that wisdom.
- You're very welcome.

All of your lab notebooks must be submitted
to the review committee!

All of your lab notebooks must be submitted
to the review committee!

This is my lab now! Get out!

I'm just trying to explain the protocol!

- You're wasting my time.
- Dr Stonehill...


What do you want?

I just dropped by to say that I think
that we might be missing an opportunity

to open a dialogue
with the other three core teams.

The other core teams? Core teams.

Core bullshit.

I'll come back another time.

The decline in muscle strength
is very troubling, of course,

but it's really, in both kids,

the organ enlargement, the liver,
and especially the heart,

that's the real threat to their lives.

We'll continue to monitor
the rate of cardiac enlargement.

- We'll keep tabs on their other organs...
- How long?

- I don't like to predict.
- Please.

Dr Waldman, we won't hold you to it.
We just need to know something.


How much more time do we have?
How long?


if there's another respiratory crisis,
all bets are off,

but otherwise,

Megan, maybe a year.

Patrick, less.

Thank you.


- Get the phone.
- What?

- The phone.
- Who is it?

- Hello?
- Hi. Is that Marcus?

- Yeah, yeah. What time is it?
- Yeah, I'm sorry. It's late.

- It's John Crowley.
- John, what you doin'?

You okay?

Hey, look, I gotta ask you a favour.

- Morning, Gavin.
- Morning, sir.

Is my 11:00 with Henessey confirmed?

Yes, and there's the breakfast meeting
down in the cafeteria.

Breakfast meeting?

There was an e-mail that came in
over the weekend.

Everyone on the Pompe project was invited.

- Who called the meeting?
- Mr Crowley, I believe, sir.


Something that people ask me all the time
is how we have two kids with Pompe.

By the time we realised
that Megan had Pompe,

I was already pregnant with Patrick.

I mean, I can't tell you
how many doctors we saw,

and the message was always the same,

that there is no drug to treat Pompe.

But thanks to you, all of you,
that message is changing.

What you've given us,
and a lot of other families, is hope.

So, thank you.

Good job, Aileen.

Now I'd like to welcome the Temple family,

who've come all the way from Georgia
to be with us here today.

Thanks, John.


I'm Marcus.

This is my wife, Wendy.

Our daughter Lauren, our oldest daughter,

Lauren wanted to come today
and say hi to all you guys.

She's a little weak,

but she wanted us
to show you guys her picture

and to send you her love.

And this is our daughter, Megan.

The most beautiful girls are named Megan.

Megan is four months old,
and you can't see it yet,

but she has Pompe, too.


I can't tell you what it means to us

to have all of you working on a medicine
for our children.

Thank you.

Thanks so much.

- Hey, Kent.
- John.

So what did you think of the event?

In medical research, John,
objectivity is key.

If researchers get all emotional,

all desperate to help suffering patients,
they'll cut corners.

It's counterproductive.

Counterproductive is having
four core teams of scientists

working together on the same disease,
but not talking to each other.

Most of these guys have never
even seen a kid with Pompe before.

I don't see how that's relevant.

Did you see Erich?
That's the kind of motivation that we need.

I'm gonna ask him to get the core team
to stop competing, start working together.

Create a leadership team.

Well, if you hope to sell
this leadership team idea,

there's only one way to convince Erich.

And what's that?

Keep your guy, Stonehill, off the team.

In his short tenure here, he's managed to
alienate a remarkable number of colleagues.

You can't put him on a team
that's supposed to build cooperation.

So, decide how badly
you want your leadership team,

then do what you have to do.

That's it.

Dr Stonehill, please.

Turn it down. Yes.

Yes, the music.

I said down!

This guy's impossible.

I like that song.


What's up, John?

- Everything all right at home?
- Yeah.

Have you heard that Erich Loring

has agreed to put together
a leadership team?

Yeah, I heard some rumour about it.
What bullshit.

Now I'm gonna have to spend a couple of
hours a week jawin' with Zymagen guys?


No. No, you won't,
'cause I'm not putting you on the team.

What'd you say?

I really wanted to.

You're not putting me on the team?
Is that what you said?

What are you, some varsity track coach?

They didn't buy our company 'cause
they like your lvy League charm, Jersey.

It's me they wanted.

You can't bench me.
It's against the laws of nature.

You're still gonna be able
to prove that your theory is right.

Yeah, but other scientists are gonna
evaluate my results, right? Not me.

Everyone here reveres your science.

This is about interpersonal issues.

- Who put you up to this?
- The decision was mine.

It's for the overall good of the programme.

"For the overall good of the programme."

Wow, John, you really got
the corporate lingo down good.

What's next, "Acceptable loss?"


Does anybody really know how to do this?

Except for us.

- But wait, who's going?
- It's John's turn still.

Nice one.

Okay. I'll get it.

Nice one, Megs.

Hello? Marcus, hi.

Oh, my God.

Man. I almost got 'em down.

You guys sit tight, okay?
Here, John, grab him.

If there is anything we can do,
anything at all, will you call?

We love you guys so much.

Okay, bye-bye.


That was Marcus Temple.



He said she went in her sleep,
that it was peaceful.

How old is she?

How old was Lauren?

Lauren was nine years old.

Come here.

That way, we can be certain
that we're developing the correct one.

Forget certainty, and let's try and figure out
which enzyme has the better odds.

If we develop only one enzyme,

what would happen
if we guessed the wrong one?

Mr Crowley, I've asked Kent
to set up a testing protocol

to help us guess right.

We'll do an exhaustive range of tests.

I'm calling it
"The Mother of All Experiments."

The four enzymes will be colour-coded,
yellow, blue, green and magenta,

and known only by those colour codes

so no researcher will know
which one he's testing.

Only after we pick a winner,
the secret identity will be revealed.

Come on, Megan!

But I want to!

- Just roll your wrist.
- I can't.

It's okay. It's all right. Let's try another one.

Okay. Let's try again. Ready?


One, two, three, go!

- Big winner!
- Yay!

Here you go, look at that. You got a penguin.

Hey, Bob. Bob.

How you doin'?

I brought you some ribs.

Not as good as the ones
from The Corner Saloon,

but if you're hungry...

What do you want?

The results.

"Mother of All Experiments."


which enzyme did they choose?

The leadership team
spent the whole afternoon arguing.

Two enzymes, coded yellow and green,
are ahead of the others,

but it's too close to call.

Tomorrow, we spend the day hashing it out
to decide which one to choose.

Bob. I'm asking you to read this.

I value your opinion
more than anyone else in the world.

Well, maybe you should have valued
my opinion a little sooner,

instead of playing hatchet man
for the bean counters.

- Get the hell out of my lab.
- It's in your interest to know about this, Bob.

Get out.

- Wouldn't it benefit you...
- Get out.

The green enzyme's the best.
Show 'em my analysis, they'll understand.

Hey, wait up, Bob.

Is the green enzyme yours?

I recognise patterns in the results.
I'd know my baby a mile away.

- So the green one is yours?
- No.

No, it's not.

My theory is still the best,
but it's not ready for manufacture.

Theirs is crude and uninspired,
but it's ready.

Bob, I don't even know the words
to say thank you.

Don't bother.

I didn't do it for you.

Yes, it is!

- Oh, my gosh. Do you see the kite up there?
- Yeah.

That's so cool!

Annie, careful!

- The bastion.
- Right there. Yeah.

- Three!
- Okay!

Dad, like... Ready?

- Dad, you promised no more phone calls.
- I know. I know I did, but...

What's in there?

We can take turns burying the phone
when I get back.

Okay! I'll start digging a hole.

- You hold that for a sec.
- John Crowley.

John, it's Kent Weber.

Kent, hey. Did you get
my suggested protocol for the clinical trials?


You made some
pretty optimistic assumptions

about where our enzyme supply will be.

I see.

Well, what do you think
that a more realistic...

John, I'll give it to you straight.
The clinical trial will be for infants only.

Infants need so much less enzyme,

and, as you know,
our initial supply will be so limited.

And the drug has much higher odds
of being effective

with infants than with older children.

We have to consult Erich on this.
We have to consult Erich.

I already did. He confirmed the decision.
Infants only.

I understand your personal reasons for...

These decisions have to be
made objectively, rationally.

I'm sorry. I...

I really am.

Hold it, pal.

- Mr Crowley, what are you doin' in here?
- I was just...

- You're not authorised.
- No... I can explain everything.

What's the matter, Chuck?
Is there a problem?

Yeah, there's a problem.
I got Mr Crowley in here without clearance.

That's my fault, now.

I got busy in the lab, I sent Jersey boy here
to get me a couple jugs of enzyme.


Thanks, Chuck.

Thanks for saving my ass.

It's really big of you,

especially after what went down between us.

You mean your ruthless,
cold-hearted betrayal?

Yeah, well, when I thought about it
from your point of view...

Shit, if it was my kids dying, I wouldn't
have hesitated to crush you like a bug.

Do you remember when we first met,

and you told me that
I should stop chasing miracles

and should go home and enjoy my kids,

while they're still here?

- Yeah.
- I made the wrong choice.

- Jersey, you know what a sibling study is?
- No.

It's a drug trial with just two patients
with the same genetic inheritance.

Siblings, with the same disease.

Megs and Patrick.

Well, would Zymagen
go for something like this?

There's real research value in it.

And with just two patients,
it wouldn't take much enzyme.

But Webber would have to sign off on it.

Well, let's not tell him
till we've got our ducks in a row.

I could draw up a protocol and see if I can
find a hospital willing to administer it.

- Bob.
- Well, don't get your hopes up, kid.

It's a Hail Mary.

Ducks in a row.

"Stonehill, careful review, protocol,
Sibling Trial,146,

"are pleased to inform you Megan Crowley
and Patrick

"have been accepted into the trial!"

- I just got it.
- Quack, quack, quack.

Have you talked to him yet?

No. I did the science. You make the sale.

Thank you.

- Good luck.
- Right.

- Does he have about five?
- He does, yeah.

- Thanks.
- No problem, Mr Crowley.

Hey, Kent? Knock-knock.

Got a minute?


- Do you have any idea what you've done?
- Excuse me?

We got a call this morning
from Portland Rose Hospital,

about sending them enzyme for your kids?

Right. Yeah, I know.
That's what I'm here to talk to you about.

You and Stonehill set this up behind
my back. Now you come to me.

It's all tentative.

You want this company
to sponsor a drug study,

for two children whose father
is an executive of this company?

Have you never heard the term
"conflict of interest?"

It has nothing to do with a...

Do you know what the FDA would do
if they found out?

There is strong scientific justification
for this study.

There is great research value.

I'm a doctor, John.
I know the research value!

But you guys just can't go off half-cocked
without consulting us!

And I'm sure that if we just go
and talk to Erich, we can...

Erich already knows.

- He's furious about being blindsided.
- I didn't try to blindside anyone.

This is the reason
why we have a reporting structure,

why we have established procedure,
why there is protocol.

You heartless, bloodless machine.

You just have to fight me
every step of the way! Don't you?

You just hated it when I brought
the kids with Pompe to the doorstep!

You weren't thinking about those kids when
you put your children into that programme

and jeopardised a decade's worth of
research investment made by this company!

This is not about a return on an investment!

It's about kids,

kids with names, dreams,

families that love them.

You've jeopardised your chances
of ever getting your kids treated.

I'm not arguing science with you!

On every level!
On every level, you've stood...

I was just drafting a letter of apology
for senior management,

but I guess we're past that point.

I just spoke to Erich,
and we are terminating your employment.

Okay, I'll pack up my stuff.

Just one thing, John.

I may well be
a tight-ass pencil-pushing company man,

but I resent being called heartless.

- Look...
- Just let me finish.

The reason we're terminating
your employment,

is to eliminate the conflict of interest.

This strategy was brought to my attention
by our colleague, Dr Stonehill,

in rather crude terms.

This will allow the sibling trial to proceed

for its purely scientific value.

Have your desk cleared out
by the end of the day.

Kent, what's with the...

I wanted to make sure you didn't
punch me out before I could tell you.


- thank you very much for firing me.
- My pleasure.

- I never liked you.
- Likewise.

Hey, you guys,
bring anything you want extra...

- Mom! Mom!
- What?

- Can I bring the bow and arrow?
- Yes, yes.


John, what...


We have a usable enzyme
that's ready for clinical trials.

When? When will it be ready?

Megs and Patrick start their infusions
before the end of this month.

- Oh, my God.
- Yeah.

Oh, my God.


Hey, John, Daddy's here.

- Oh, hi, guys.
- Hey, Dad.

Why is Mommy sad?

Not sad, baby, so happy.

So happy.

Why is Mommy being so weird?

Well, it's because
we got the special medicine

for you, Megs, and for you, Patrick,
and you're gonna get it really soon.

That's great!

So, who wants to push the button?

Megs, Patrick, you ready?

When will we know if it's working?

Lt'll be a while, honey.

It's not pink.

Excuse me?
Your Uncle Bobby is here.

I don't know.

- You can go back if you like.
- Okay.

- John.
- Uncle Bobby.

They wouldn't let me in unless I was family.

- Some things for the kids.
- Thanks.

Sleeping, huh?

Hey, thanks for coming.

I'm a scientist. Gotta track the experiment.

I wish your enzyme was the one
that passed the test.

But the one the kids are getting,
it's the right one, right? I mean...

It's gonna help them.

Yeah, you guys have done
everything humanly possible.

We just have to see what the tests show.

You and I have had our differences,

and I just want you to know something.

I want you to know that...

- Yeah.
- Don't interrupt.


I want you to know
that I appreciate everything

that you've done for Megs and Patrick,

and I'm never gonna forget it
for the rest of my life.

Not gonna kiss me, is he?

I will restrain him.

Don't tempt me.

Daddy loves SpongeBob,
Daddy loves SpongeBob.

- Stop!
- Daddy loves SpongeBob.


What's going on?

What's so funny?

What are you laughing at?

What are you laughing at?


It's a sugar high.

The enzyme's breaking down
the sugar in their muscles.

- The medicine's working?
- Yeah.




- John, drive safe!
- Enjoy the ride!

Rock and roll!

- Bye! Have fun.
- Bye!


Yeah! Yeah!

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