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♪ (QUIET MUSIC PLAYING) ♪

♪ (MUSIC CONTINUES) ♪

SAM BERNS: When I'm building
Legos, I feel like I can...

create my own world
and kind of go into that place

where, you know,
everything is based on my rules.

(LEGOS CLACKING)

There's really no way of telling
what I would be like

if I didn't have progeria.

I wish I could ride
any roller coaster

I wanted to ride, like, ever.

Like, I wish I could, like,
you know, do loop-de-loops

and corkscrews and stuff,
and barrel rolls.

Even though I'm, like,
terrified of them,

it would be, like, cool to know
that I could try them out.

(OBJECT CLACKING)

I didn't put myself
in front of you

to have you feel bad for me.

I put myself in front of you
to let you know

that you don't need
to feel bad for me,

because this is how-- this is--
I want you to get to know me.

This is my life,
and this progeria's part of it.

Not-- it's not a major part
of it, but it is part of it.

♪ (MUSIC CONTINUES) ♪

♪ (MUSIC FADES) ♪

(FOOTSTEPS)

(DISHES CLINKING)

(CABINET DOOR OPENS)

SAM: Bye, Dad.
Love you.

SCOTT BERNS: (SMOOCHES)
Love you, too. (SMOOCHES)

-You look great today, too.
-SAM: Thanks.

(FOOTSTEPS RECEDING)

(HEAVY BREATHING)

(DRAWER OPENS)

LESLIE GORDON: Your drugs
are in the bathroom.

Okay. Mom, can you hold my book

-while I brush my teeth?
-LESLIE: Yep.

-I got your socks, too.
-Thank you. Okay.

(EXHALES)

(SAM SIGHS)

I have a math final
next Wednesday.

He's giving us
the study guide today.

LESLIE: All right.
I didn't know you had finals.

People will say things like,

"I don't know
if I could do this."

And my answer to them is,
"Yes, you could.

You-- you-- you can."

Mothers can do anything.

You do what you need to do, um,
because you know

that you're not the most
important person in the room.

The most important person
in the room

is the child sitting
in front of you.

That's really the bottom line.

That doesn't come
from just my brain,

that comes from my heart.
That comes from my soul.

And that's what drives me.

-Love you, Momma.
-LESLIE: I love you, too.

-Have a great day, buddy.
-SAM: Thank you, so--

-LESLIE: See you after school.
-SAM: Yeah.

(BUS ALARM BEEPING)

SCOTT: I'm an optimistic person,
but if you were to say,

"What's the worst thing
that could happen to you?"

Certainly, as a parent, it's
to be told your son has a...

condition that there's nothing
you can do about.

I mean, isn't that
the worst thing

that could possibly happen
to a parent?

To be told that you have a child

for which there is nothing
that can be done.

That there is a certain outcome.

That he or she will live,
you know...

that he or she will live
13 or 14 years on average,

and that will be it.

So... You know, what do you do?
You-- We're well aware

of the prognosis,
but you know what?

We're gonna stay optimistic,
because why wouldn't we be?

I mean, what-- what are you--
what are we gonna do,

like, go into a shell and say,

"Well, we don't really know
what's gonna happen,

so we're not gonna live"?

(INDISTINCT CHATTER)

TEACHER 1: So, when X is two,
Y is negative three,

we put a dot there.

Do you understand that that--
Where that goes?

Now, you have two points,
and you can make a line.

I got it.

I'm just really confused.
This is my, uh...

SAM: Tell me your equation.

Like, if I were to do
negative three times...

-No, not negative.
-So would it start on the, uh...

-the X-- the Y-axis?
-Yeah.

Zero times negative three
is zero,

minus two is negative two.

One times negative three
is negative three,

minus two is negative five.

So just keep, um,
subtracting three,

just to make it easier
on yourself.

(INDISTINCT CHATTER CONTINUES)

That is...

Wait, Jake. I'm so confused.
Wait, you have to find X?

We're here to discuss
any concerns

of Sam adjusting to his new
eighth-grade situation.

Okay, so-- so--

Progeria is known
as a premature aging syndrome.

It doesn't have
all of the signs of aging,

because Sam's a 13-year-old
with a 13-year-old mind,

and he doesn't have cataracts,

and he doesn't have,
you know, other, uh,

cancer and other problems.

But from the cardiovascular
standpoint,

these kids all develop

progressive
cardiovascular disease...

without exception.

Sam's cardiovascular status
is not what it was a year ago.

He's had no strokes,
he's had no heart attacks,

but he does not have a normal
cardiovascular system.

And his bones
are a little weaker, I think.

So, we went to Disney World,

-and he went on a ride...
-SCOTT: Oh, yeah.

...and he cracked two ribs
just by going on a ride.

-It was one of these old--
-SCOTT:
It wasn't Space Mountain.

It wasn't. We kept him
off all roller coasters.

And he was mad,

and then he went
on this older ride,

-so it was
a little more jarring.
-Yeah. It was old.

LESLIE: And he must have gone
up against the bar.

-Yeah.
-LESLIE: (SNAPS FINGERS)
Two ribs.

-(INDISTINCT CHATTER)
-Yeah.

Thank you, Derrick.

(SPEAKING FRENCH)

-(INDISTINCT CHATTER)
-(SPEAKING FRENCH)

(TEACHER 2 SPEAKING FRENCH)

(SPEAKING FRENCH)

(SPEAKING FRENCH)

(INDISTINCT CHATTER)

SCOTT: The vigilance
is really important

'cause he is getting older now,

and, you know, we've always
been very cautious, but...

you know, we're very--

We wanna be careful.
I know you do, too.

But if he's having, you know,
an obvious breathing problem,

or chest pain,
or something like that,

then that's obviously
more serious.

Not to scare you too much,
but just being observant,

without, you know,
being like this,

would be probably a good idea.

(INDISTINCT CHATTER)

SAM: I kind of just want
my mom to be done with progeria.

For her sake, 'cause, uh...
she doesn't have a normal job.

I mean, she has a job,

but it's not the normal
kind of job

where you go in, you do
what you're supposed to do,

you get paid, and you can leave
whenever you want

at your own risk.

My mom will keep working forever

until progeria's cured
and she doesn't have to.

♪ (SOMBER MUSIC PLAYING) ♪

(INDISTINCT CHATTER)

The researchers involved

have found
the gene for progeria.

This discovery is likely
to shed light

on the normal process of aging,

and on heart attack,
and on stroke,

and, hence, it reverberates
well beyond its application

specifically to progeria.

LESLIE: When Sam was diagnosed
with progeria in 1998,

there was nothing out there.

There was no research going on.

No treatment, no cure,
no foundation to go to.

I was in the middle
of my first year

of medical residency,
and I stopped.

SCOTT: We came up with this idea
to form a foundation,

and Leslie's sister, Audrey,
was right there

right from the very beginning.
And Audrey, being a lawyer,

she was ready to drop
that career to help her nephew.

I say to Leslie,
"Just tell me what you need.

Tell me how much
it's gonna cost,

and don't worry about the money.

We'll find it
because we have to."

♪ (MUSIC CONTINUES) ♪

TECHNICIAN:
...technically somewhat limited,

-um, especially
when it's in such a short...

Looks good.

I scanned these
at Rhode Island Hospital.

That won't be
good enough quality?

-You wanna do it in-house?
-No, we also wanna take a...

LESLIE: Now, we have the team.
We've got the experts.

We've got people
who know more about progeria

than anybody else in the world.

So, this is what we found,
and this is the heart disease

of a 65-year-old.

And it looks very similar
to what you're seeing here.

His skin is a little bit like--
This is what I see

when I see the younger kids
with progeria.

-And look at her hands.
-(INDISTINCT CHATTER)

LESLIE:
But we're right in the center.

I wanna move around
a little bit and just check.

I'm impressed
because this is not...

-Right.
-I didn't expect it

to grow this well.
I would put these back, though.

♪ (QUIET MUSIC PLAYING) ♪

LESLIE: When we found
the gene for progeria...

hundreds of doors opened for us.

There was a drug, Lonafarnib,

that had already been developed
for over ten years.

It was developed
for other diseases

with protein abnormalities
that are similar

to what we see in progeria.

We had evidence
in the laboratory, in mice,

that this drug might apply
to progeria as well.

But jumping
from a mouse to a human

when you're trying to ask
such an important question,
"Will this drug work?"

is like leaping over
a huge chasm.

SCOTT: The specialist who was
expert in this drug said,

"Cancer in mice has been cured
a thousand times,"

or something like that,

"but don't bank on the fact
that this is going to...

have an impact on humans."

That was really
a powerful reality check for us.

LESLIE: The only way
to really find out,

"Is this drug gonna be helpful
for children with progeria?"

is to give it to children
with progeria.

Time is... is passing.

We're just in a race.

We're just in a race
that we just need to win.

♪ (HOPEFUL MUSIC PLAYING) ♪

(SPEAKING URDU)

(BOTH SPEAKING HINDI)

(PRIYA SPEAKING HINDI)

LESLIE: Hey! Ciao.

-Ciao?
-NICCOLO'S MOTHER: Ciao.

LESLIE: Ciao? (LAUGHS)
Hey, slap me five.

Ouch, ouch!

-How are you?
-SAMMY: Hi.

-(CHUCKLING)
-LESLIE: Oh, it's great
to see you.

-You look fantastico.
-(CHUCKLING)

-LESLIE: Hey!
-SAMMY'S FATHER: Hi.

-SAMMY'S MOTHER: Hi.
-LESLIE: Hey! (LAUGHS)

How are you?

-(INDISTINCT CHATTER)
-(BOTH CHUCKLE)

MEGAN'S MOTHER:
Say, "Looking for..."

-(MEGAN LAUGHS)
-Okay. Brown?

Brown, white, black, or both?

-Both.
-Yeah.

What kind?

(INDISTINCT CHATTER)

DEVIN'S MOTHER: (WHISPERING)
Can I fall asleep on you?

-DEVIN: No.
-DEVIN'S MOTHER: Why?

-DEVIN: Because.
-DEVIN'S MOTHER: I'm tired.

You'll fall.

DEVIN'S MOTHER:
Well, you're strong enough.
You can hold me up.

(LAUGHS)

DEVIN'S MOTHER:
Eight years ago, he...

suffered two major strokes
back-to-back, three weeks apart.

Um, the first stroke
affected the left side,

the second stroke
affected the right side.

♪ (SOMBER MUSIC PLAYING) ♪

Doctors pretty much told me

there's really nothing
they can do for him.

"Just take him home and...

enjoy the rest of the time
you have with him."

MEGAN'S MOTHER:
We took Megan for--

MEGAN'S FATHER:
Over two years' worth
of different doctors,

different tests,
from genetics to--

Celiac disease,
to cystic fibrosis,

to thyroid issues.

And then finally, one day,
the doctor called,

and says, "I think it's
something called progeria."

And I said, "Oh.
Well, what's that?"

He says,
"Well, it's-- it's just--

You know how someone
at 60 looks great,

and somebody else
at 40 looks bad?"

He says, "That's all it is."
He says, "It's just

some people age
better than others."

♪ (SOMBER MUSIC CONTINUES) ♪

NICCOLO'S MOTHER: (IN ITALIAN)

(MACHINE BEEPING)

NURSE 1: (IN ENGLISH)
So, this is gonna be the med,

and we're gonna
connect that, okay?

Now, we're gonna make this run
over a half an hour, all right?

Yep.

NURSE 1: So,
I'm gonna program this machine.

Nice, big breath for me.

Thank you, sir.

LESLIE: A drug trial
is an experiment.

DR. MONICA KLEINMAN: Push this
so you're comfortable.

LESLIE: There's always a risk.

These kids have never
had any drug like this

given to them before.

I don't wanna hurt anybody.
I don't wanna hurt Sam.

I don't wanna hurt
any of the other kids.

'Cause, in the end,
a parent and their child

is gonna be
sitting in front of me,

putting their trust in me.

And that's a huge trust

because they can't possibly
really know what's going on.

I mean, who can?

I don't even know
what's going to happen.

NURSE 2: Try the other side?

(INDISTINCT CHATTER)

The gold standard
for clinical trials

is testing the drug

on hundreds and hundreds,
if not thousands, of people.

And half those people
get the drug,

and half those people
don't get the drug.

And it's the best way
to show you

whether a drug works or not.

DR. KLEINMAN: I don't think
that one will be very good.

LESLIE: Well, we have children
who are dying from a disease.

So how ethical is it
to give a child a placebo?

NURSE 1: Okay, ready to go.

LESLIE: We don't have
the luxury of doing that

with this tiny, tiny, tiny
population.

We chose to give the drug
to all of the kids.

(BOTH CHUCKLE)

-DEVIN'S FATHER: Pretty boring?
-DEVIN'S MOTHER:
Yeah, pretty boring.

(INDISTINCT CHATTER)

(OBJECTS CLATTERING)

TECHNICIAN: So, we're just
gonna check all these pictures,

but we'll be right back.
Okay, Sammy?

(MACHINE RATTLING, WHIRRING)

TECHNICIAN: I'm actually
gonna do a cross table

of the head, so...

NURSE 3: All right, and, Mom,
you're welcome to stay

in the room for both scans,

as long as you know
there's radiation.

LESLIE: The question is,
is this drug having an effect?

We don't know yet.

We don't know that
until the end of the trial.

And at the end of the trial,
you look at the data,

and you put together the paper,

and then you send it for review
by a scientific journal.

And that journal has people
who look very carefully at it

who are not involved
with the trial.

If they like it, it gets
published in an objective way,

that says, "Yes, this is a drug
that can treat progeria."

And that is the only way
to get this drug approved

through the FDA.

That's really the only way
we're gonna get to the end.

If the paper doesn't
get published, that's it.

No child with progeria is ever
gonna get this drug again.

(MACHINE WHIRRING)

MEGAN'S MOTHER: Yeah. I know.
She was in the study today.
We had some...

MEGAN'S FATHER:
They are doing all the testing,
and we haven't seen any results.

So we haven't heard,
you know, are the bones

better than they were
a couple years ago? Is--

-Yep.
-Are the arteries better?

(MEGAN BREATHING HEAVILY)

-She's warm.
-NURSE 1: It's a little warm.

She's warm, yeah.

LESLIE:
In your head, you're wondering,

"Okay, how old is she really?
How many years does she have?"

NURSE 1: You did
a good job, Megan, thank you.

LESLIE: I think parents...

of children who are sick,

they understand
that you cross a threshold

that you couldn't have imagined.

-(SPEAKING ITALIAN)
-(MACHINE WHIRRING)

LESLIE: It becomes
sort of ingrained

in your everyday thoughts,
in your dreams,

in what you think about at night
when you're sleeping.

(INDISTINCT CHATTER)

DR. KLEINMAN:
How you doin'? Yes? Okay.

(MACHINE WHIRRING)

As a mom, who thinks about
not only Sam

but all of the other kids...
all of the time...

it-- it brings the bar...

um, of excellence
very, very high.

(MACHINE WHIRRING)

LESLIE: There's no cloudiness.

There's no thought
about academic success

-because it all revolves
around what's safest...
-Here?

...but also, hopefully,
best for helping the children.

It's pure.

♪ (SOMBER MUSIC PLAYING) ♪

Progeria is one of the rarest
diseases in the world.

This is a disease that affects
one in four million children.

That means, in the entire world,

somewhere between 200
and 250 kids

with progeria are living today.

Progeria is caused by one single
tiny change in the DNA.

It's a C to a T. That's it.

And it causes an abnormal
protein called progerin

to be created.

Proteins need to fit
into each other correctly

in order to make
the right scaffolding

to build the human being.

Progerin acts as an imposter.

It tries to take the place
of the normal protein.

So it goes into the nucleus
of the cell...

and it sticks,
and it sticks in places

where the normal protein
would stick, only it's toxic.

And from there,
that's how it all begins.

That's how the dominoes
begin to fall.

So you get this sort of
downstream, huge effect

on cells and on organs
and on the whole body

that causes disease.

Progerin builds up over time.

It's in the organs
that are affected by aging.

PHOTOGRAPHER 1:
Over here a little bit more.
Turn a little bit more.

LESLIE:
So when a child with progeria

is eight, nine, or ten,

they're gonna have
the blood vessels

that look very much
like somebody who's 70 years old

or maybe 80 years old
for the rest of us.

(INDISTINCT CHATTER)

And most importantly,
without treatment,

all of the children develop
similar heart disease,

and they die
of heart attacks and strokes.

We do not know if progerin
represents anything close to

a key to the Fountain of Youth,

but what we do know is that,
as we age, we all make progerin.

We make much less of it
than children with progeria do,

but we make it.

We only know this
because we cared enough

to try to find a cure
for progeria.

And now, it's transferring over
to the field of heart disease

and aging in general
for all of us.

The aging fields will take this
and run with it,

but my job, my passion,
is to stick with these kids

and figure out
how we can help them

by applying this to aging.

(DEVIN BREATHING SHAKILY)

-Okay. All right. Okay. Okay.
-(DEVIN SNIFFLES)

NURSE 1: Done? Okay. Good job.

Devin, thank you so much
for trying today.

If it wasn't for Leslie...

I don't think
I'd have him right now.

-(INDISTINCT CHATTER)
-You're basically done, right?

Um, Leslie, give this to Sam
for me. (SMOOCHES)

-Give that to Sam for me.
-(LESLIE SMOOCHES)

-Thank you. That's really nice.
-I will. Okay.

I will give that to him.

He talks about you
all the time...

(SIGHS) I think
she's the greatest thing

that's ever happened to us.

I mean, I'm sorry
it happened to her son...

but we kind of needed her.

Sorry.

(INDISTINCT CHATTER)

LESLIE: What's he doing?
'Cause he's like...

-SCOTT: He's looking around.
-LESLIE: Very sweet.

SCOTT:
He's checking out the world.

-(MOUSE CLICKING)
-LESLIE: Hi, Sam.

Look at that little nose.

-(CHUCKLES)
-(SCOTT CHUCKLING)

I can't believe
this is my child.

-AUDREY GORDON: It is.
-This is amazing.

-(CHUCKLES)
-(SCOTT CHUCKLING)

♪ (QUIET MUSIC PLAYING) ♪

PHOTOGRAPHER 2: Okay, stand
next to Leslie for a picture.

(INDISTINCT CHATTER)

LESLIE: Sammy.

Hi, Sammy.

Hi, buddy.

(SAM LAUGHING)

(LESLIE CHUCKLES)

(BOTH LAUGH)

(LAUGHING)

(LESLIE LAUGHS)

(SCOTT LAUGHS)

-(LESLIE CHUCKLES)
-(SCOTT BABBLES)

-(COOS)
-(EXCLAIMS)

SAM: It all started
with a cup of coffee.

Um, they were
both going to Brown,

uh, both medical students,
and they met.

They were doing
a project together.

And after the project,
my mom was sitting

in her little office thing
at her desk,

and my dad came in, and he goes,
"Hey, you want a cup of coffee?"

So they both
had coffee together.

And, uh, they got married
in 1993 on October tenth.

And they had me in 1996
on October 23rd.

And, yeah, that's pretty much
how it happened.

(CHUCKLES)

LESLIE: When Sam was born,
to us, he looked normal.

But even when we look back,

we can see that something
just wasn't right. And...

we went to the--
to our pediatrician,

and we kept saying,
"Something's wrong.
Something's wrong."

SCOTT:
He wasn't really gaining weight

over that first year and a half.

And we attributed that
initially to the fact

that he didn't eat as much.
He spit up.

And babies spit up, right?

And I noticed
there were things about his skin

that I would think, "Well, what,

this is a little bit different
around his stomach,

and what's that all about?"
And we're both physicians,

so it's like, "Oh, maybe
we're being too sensitive

about noticing
some of these things."

LESLIE:
It just came from my soul,

like, "Something
is going on here,"

and it was incredibly
frustrating.

Finally... (SIGHS)

...um, a brilliant friend
and colleague of ours

came up with this idea that Sam
might have progeria.

I immediately went over
to the medical library

and looked up progeria,
and saw pictures in there,

and these were pictures
of my son.

I mean, they weren't pictures
of my son,

but they could have been.

That's when it really struck me.

LESLIE: I didn't know
if I believed it,

but I also didn't know

how to necessarily
evaluate that.

So we made an appointment
to see a geneticist,

a local geneticist,
brought Sam there,

and decided to get x-rays.

So here we are, you know,
in the back room

with the radiologist,

and he put up an x-ray
of Sam's chest.

And it showed
the clavicles being short,

but I was just a resident,
I was just starting out,

so I asked the radiologist
to put up a comparison shot

of a normal chest x-ray...

and all of the sudden, I just...

It was just so dramatically
different because I could see

that things were very different
in his clavicles.

And this is exactly
what was in the paper.

And I said to the radiologist,
"Could this be anything else?"

And he said, "I don't think so.

I don't think
it could be anything else."

Nothing we could do.

I--
It just wasn't part of my...

psyche, or the way I think,
to accept that,

certainly not as a person,
certainly not as a clinician.

I was working
as a pediatric
emergency physician.

I was fixing things.
That was my job.

So a kid would come in,
have an ear infection,

and we treat that.

Uh, someone would come in
and have a cut on their arm

and needed stitches,
and I would fix that.

And here I was thinking,
"I can't fix this?

There's nothing I can do?"

I mean,
that just rips you apart.

♪ (SOMBER MUSIC PLAYING) ♪

LESLIE: I think my senses maybe
became hyperacute, in a way.

I-- I didn't need
much sleep at all.

I needed to know
that he was still breathing

all night long, every night,
for a really long time.

I could hear him breathing
from my bedroom to his room.

This was a couple of years
of just getting through this.

I didn't really have control
over it.

I just surrendered to it
and, uh...

just kept going.

SAM: Why is that book
your favorite?

LESLIE: Well...

it's about a seagull,
but it's kind of about life.

♪ (LIVELY MUSIC PLAYING) ♪

(INDISTINCT CHATTER)

LESLIE: The most important thing
that I needed...

(INDISTINCT CHATTER)

...wasn't necessarily
a scientist.

It was my family and friends.

(ALL LAUGHING, CHEERING)

SAM: Nice. Goodness.

LESLIE: I just needed the people
that were closest to me to say,

"Go for it. We believe in you.

Whatever you want, yes.
Just do it.

We're there for you."
And that's what I got.

-SAM: Oh! (LAUGHS)
-(CROWD CHEERING)

-(LAUGHTER
-(INDISTINCT CHATTER)

♪ (MUSIC FADES) ♪

-(INDISTINCT CHATTER)
-♪ (PIANO MUSIC PLAYING) ♪

Hello. How are you?

-Good to see you.
-(CHUCKLES)

-How have you been?
-Good.

LESLIE: Good? You look great.

I want to know how they got--

-See this scarf?
-DR. KLEINMAN: Yeah.

LESLIE: It's got the flowers
on the bottom.

It matches the dress.
Unbelievable.

She's got
the whole thing worked out.

-Yeah.
-LESLIE: So...

Very good. All right.

MR. VELURI: (IN HINDI)

DR. KLEINMAN:
You're looking pretty good.

PRIYA: (IN HINDI)

-(MACHINE WHIRRING)
-(OBJECT RATTLING)

(MACHINE BEEPING)

LESLIE: Priya has had
pretty advanced symptoms

of possible strokes

and chest pain
and high blood pressure.

And so the fact
that she's feeling well

is very encouraging.

I don't know exactly
what it means,

but she hasn't declined.

She hasn't gotten worse.
She's feeling very good.

This is the first time
that's happened,

and it's incredibly exciting

'cause, you know,
it means good things for her.

(MACHINE BEEPS)

-(CAMERA SHUTTER CLICKS)
-(MACHINE BEEPS)

DR. MARIE GERHARD-HERMAN:
So, her vessels look
actually better, if anything.

They definitely
don't look worse.

-The--
-So are they pulsating any?

-Yeah.
-Yeah.

-She looked good.
-LESLIE: Yeah.

-(CHUCKLES)
-I know.

She feels better, so maybe

she's in a nice
honeymoon period.

Do you know her triglycerides
are 1200, though?

-No.
-Yeah, yeah.

So, you know?

So we need to lower
her triglycerides.

LESLIE: Cholestyramine,
or, I mean, what would you add?

DR. GERHARD-HERMAN:
We have a couple options,

what would work
in that small person.

♪ (MUSIC PLAYING) ♪

(MACHINE BEEPS)

MR. VELURI: (IN HINDI)

LESLIE: Where's his nose?
Is that his nose?

Is that his nose?

ZOEY'S MOTHER: Zoey, say hi.

-(COOS)
-(ZOEY'S MOTHER CHUCKLES)

-(LESLIE LAUGHING)
-ZOEY'S MOTHER: Thank you.

ZOEY'S FATHER:
Where you going, Zoey?

ZOEY'S MOTHER:
Say, Pooh Pooh is nice.

(CHUCKLES)

(CHUCKLING) Hi.
That was really good.

-Want your bear?
-(GASPS)

-(SMOOCHES)
-(SMOOCHES) Kiss, Zoey.

LESLIE: Right now,
I have 17 children

who are not in this trial
because I discovered them

after the trial was full.

Seventeen children...
who could be taking drug, if...

we could just
ship it out to them.

-ZOEY'S FATHER: Yes.
-(FOOTSTEPS)

LESLIE: Zoey,
who is the cutest little thing

you've ever seen in your life,
is one of those children.

Step.

PEDIATRICIAN: For us,
as pediatricians...

LESLIE: Zoey has to wait,
and Zoey's parents have to wait.

They have to watch her decline.

PEDIATRICIAN ...theoretical
benefits to children, so...

And that's not easy.

It's not easy to look at Zoey,
and know that she has to wait.

Where's your nose?

Is your nose right here?

LESLIE:
I think it must be torture...

for parents to know
that there's a drug out there

that's being tested
that their child isn't getting.

Everything is at a standstill
until this trial is over.

Until we show everybody
the statistics.

It just seems like
it would take a lot longer

-than it seems you'd wanna wait
if you could do something.
-(ZOEY COOING)

I mean, to get it so that
it would be approved
for everyone,

couldn't that take a lot--
a lot of years?

(CHUCKLES) Everybody's waiting
and watching

and wanting to spring
into action

if this-- if this is
a good thing for the kids.

What I want you to know is,

although it's torture
for you to wait

for the answers,
that she will be--

she's-- she's, like,
on the top of my mind.

She's in the top of our minds,
and nobody wants

her to have to wait
or you to have to wait...

-Okay.
-...any longer than possible.

So, basically,
what you're saying is

we don't have to worry that--
I mean,

as soon as it is possible
for something to be done,

something will be done
to get her on...

LESLIE: I'm on it.

-Okay, I know. Okay.
-I promise.

And-- and-- that doesn't mean

you can't check in with me.
You can, I mean...

It's a cruel balance
between making sure

that everything is done right

and that it's as safe
as it can possibly be,

and the fact that children
are not getting drug,

and their disease is taking
its toll every single day.

I mean, obviously,
the skin's thick, so--

So you worry
about everything, yeah.

A year from now,
you'll come back,

and you'll see how it compares.

And that's the most important
information.

-ZOEY'S MOTHER:
Right, the change.
-Right? Yeah.

We've got no time to waste.

A lot of children
are continuing to get sick.

In my office, I have pictures
of children who are living

and children who have died.

There's one picture
in particular...

from a long, long time ago...

where almost all of the children
have passed away.

Um, I think there are two
who are alive.

One of them is Sam.
And they're the only ones left.

It's a very interesting question
because, you know,

are they gonna wanna just--

How easy is it to run this?
Do you really wanna run both...

Time is-- is passing.

-(INDISTINCT CHATTER)
-LESLIE: We can't reverse time.

But every day,
what I'm thinking about,

above all else,
is trying to save the lives

of these kids
and save Sam's life.

(INDISTINCT CHATTER)

♪ (MUSIC PLAYING) ♪

SCOTT: There's a lot of candles.
That's a good thing.

-Yay!
-(ALL CHEERING)

SAM: I feel like this is kind of
a serious, uh, thing.

I kind of deal with death
a lot different than...

than, like, other people might.

Kids with progeria, sometimes,
they-- they can't--

they can't fight it out,

and, uh, you know,
they pass away, and it's just...

it's-- it's hard
to think about it.

Well, there was this time period
when I asked my mom

about one of my friends, Ory.

And my mom said,
"Oh, he, uh, passed away."

I'm like,
"Well, what about Stuart?"

She's like,
"Well, he passed away."

"What about, um,
you know, Ronnie?"

"He died, and Mierko died."

My friend Andrew, that--
that was hard. Um...

You know, we were good friends,
and he could have been,

you know, such a great part
of my life,

and, uh, you know,
it's just too bad

that I didn't get to know him
for longer.

And so I feel like I deal
with death in a different--

in a lot more constructed way,
or, you know, structured way,

than other people would.

♪ (SOMBER MUSIC PLAYING) ♪

♪ (MUSIC FADES) ♪

♪ (PIANO MUSIC PLAYING) ♪

LESLIE:
What I need to do is just

understand
that it is what it is.

Sam has progeria.
The other kids have progeria.

There isn't a God anywhere

that would've done that
to a child on purpose.

You know, subject a child
to that kind of pain,

that kind of disease.

We're just here.

We're here
in a position where...

we've gotta do everything we can
to help them.

That's the position I'm in.
It is what it is.

And, um, I feel...

impassioned by it.
I feel like I'm guided by it.

And I don't get angry
about this.

I don't wallow in,
"This isn't fair."

It's a nonproductive
thing to do,

and I think what is driving this
is love for Sam.

Love for the other children.

And anything
that is gonna delay that

is just not worth
thinking about.

So I just pass it by.

SAM: Good boy.

-(INDISTINCT CHATTER)
-SCOTT: We've been
to Fenway twice,

-or three times actually, right?
-SAM: Uh-huh.

-♪ (HUMMING) ♪
-(SCOTT CHUCKLES)

SCOTT: My perspective on life
changed with Sam's diagnosis.

I try to take things in more.

Um, I make myself take account
of the present more.

SAM: I think he's eating
so much, he's like...

SCOTT: And maybe that's part
of just growing up in general

and becoming more mature
as a human being.

I think that this experience

has perhaps accelerated
that realization.

And I hope everyone
comes to that realization

as soon as they can, which is,

there are things happening
all around you right now

that you really should
take account of and enjoy

'cause, you know, we don't know
if we're gonna be here tomorrow.

We just don't. None of us do.

-(INDISTINCT CHATTER)
-Here you go, buddy.

We've been to so many shows
every summer

for the last eight
or nine years,

I mean,
it's gotta be well over 30,

probably 30 or more shows.

It's one of my favorite ways
to spend time with Sam, I think.

It makes me feel like a dad.

-Hello.
-USHER 1: Hi.

-SCOTT: Hi.
-USHER 2: Hi there.

SCOTT: Tonight we actually
are gonna have the opportunity

to meet the band.

I mean, I don't know exactly
what Sam would say,

but these sorts of experiences
are bittersweet

because Sam is having
that experience

in part because, you know,
he does have progeria,

and we're trying to find a cure,

and they wanna show
their support.

How are you? Nice to see you.

-All right, all right.
-Scott.

Hey, Scott, how you doing?
All right.

What's going on? Oh,
I don't wanna see the set list.

-I don't wanna see it.
-Oh, there's no set list there.

SAM: I was gonna say,
"Big Eyed Fish"

and "The Bartender"
for the opener, anyway.

All right. Don't tell me
anything else. I'm good.

JEFF COFFIN:
I didn't tell you anything.

-You just guessed the first two.
-(CHUCKLES)

-SCOTT: Hey, guys.
-Hey.

-What's up, man?
-Good to see you.

Good to see you again, man.

-Hey, Stefan.
-Hey, how you doing?

-How you guys doing?
-Good to see you, Carter.

-How are you?
-I'm doing great.

I'm gonna have some more sticks
for you tonight.

-Thank you.
-Yeah.

-How are you doing?
It's good to see you again.
-Good to see you, too.

-You doing good?
-Yeah, I'm doing really well.

-Thank you for coming.
-SAM: Oh, no problem.

-You wanna ask Dave
to sign your picture?
-Yeah. Do you mind signing

-a couple things?
-Sure.

Any songs
you're hoping for tonight?

Well, I'm hoping
for "Kit Kat Jam,"

-but whatever you wanna play.
-Okay.

We'll see. You know, we've got
some different ones in there.

-Thanks. I hope
we get it right again tonight.
-Thank you so much.

-Yeah, that was awesome.
-Wow.

SAM: (SINGING)
♪ I'm so excited ♪

♪ And I just can't hide it ♪

(CROWD CHEERING, APPLAUDING)

SCOTT: When we go
to see concerts,

the experience for us is,
it's many things.

It's... a bonding experience.
It's almost spiritual in a way.

♪ ("BIG EYED FISH"
BY DAVE MATTHEWS BAND PLAYING) ♪

(SINGING)
♪ Decided to climb down ♪

♪ And to run off to the city
But look at him now ♪

♪ Tired and drunk
He's livin' in the street ♪

♪ As good as dead, you see ♪

♪ The monkey man should know
You got to breathe ♪

♪ But, oh, God
Under the weight of life ♪

♪ (VOCALIZING) ♪

♪ ("BARTENDER"
BY DAVE MATTHEWS BAND PLAYING) ♪

♪ Bartender, please... ♪

SCOTT: We don't have to think
about Sam's condition,

or the fact that he needs to go
to the hospital soon, or...

We don't think
about those things.

That doesn't have to be said
between us,

but I think we both
have that feeling.

It's therapy, in a way.

♪ (DAVE MATTHEWS VOCALIZING) ♪

-(CROWD CHEERING, APPLAUDING)
-(INDISTINCT CHATTER)

-DAVE: Thank you very much.
-(CROWD CHEERING)

DAVE:
We're going to play a song

for a good friend of ours.
He's been a fan for a long time.

He's a young man. He's only 13.
He's been to about 550 shows.

(CROWD CHEERING)

So I wanna send this--
this out to our good friend Sam.

He asked for it,
and so we said, "Hell, yes.

We will do it for you,
young fella."

(CROWD CHEERING, APPLAUDING)

♪ ("KIT KAT JAM"
BY DAVE MATTHEWS BAND PLAYING) ♪

SCOTT: For me, I had to tease
out my feelings

as a dad and as a fan

because, you know,
I'm a fan of the band too,

and I was excited as a fan,
but more excited as a dad.

Can I just put my arms
around you?

-Woo-hoo!
-SAM: Yeah!

♪ (MUSIC CONCLUDES) ♪

(CROWD CHEERING, APPLAUDING)

-(LAUGHS)
-Great job.

All right,
we did that one just for you.

Great job. Thank you so much.
That was amazing.

CARTER BEAUFORD: We'll see you
next time, all right?

Yeah, I guess so. Yeah.

CARTER: Anywhere you wanna come,
you just let us know.

-All right, cool. All right.
-So good seeing you, bro.

-Great to see you, too.
-All righty, practice, practice.

-I will, I will.
-CARTER: All right.

SCOTT: I think, at some level,
I-- I wish every fan

could have
that sort of an experience.

It was amazing.
It was great to see the guys.

It was great to talk to 'em.
The show was amazing.

The set list was,
like, really, really good.

And, uh, they played
"Kit Kat Jam" for me,

and that was just amazing.
So it was a great experience.

Couldn't have been better.

-Couldn't have been better.
-Really.

I guess, what I'm trying
to say is that...

we would have been very happy
to be at that show,

and to go to these shows
all the time,

and to never meet the band,

and to have Sam
not have progeria,

and for him to be cured.

♪ (QUIET MUSIC PLAYING) ♪

♪ (MUSIC FADES) ♪

-(KNOCKING ON DOOR)
-(DOOR OPENS)

-SCOTT: Okay.
-Thank you.

♪ (SOMBER MUSIC PLAYING) ♪

(DOOR CREAKS, CLOSES)

SCOTT: Thanks.

♪ (MUSIC CONTINUES) ♪

All right.

Okay.

Gotcha.

I got you.

Scottie, I got these two things.

-SCOTT: Got the hat?
-LESLIE: Got the hat.

♪ (MUSIC FADES) ♪

SAM: When I'm in the hospital,

I'm isolated from my friends,
I'm isolated from television,

I'm isolated from my home,
I'm isolated from my cats.

(HEARTBEAT THUMPING)

SAM: It's like...

I lose...

all of the things
that make me, me,

except for progeria.

-Can I stretch out your arm?
-So once it's in, it's in.

(GASPS)

NURSE 2: Okay.
Relax your arm, okay?

I wanna play some--

another person on Xbox Live
tonight. That was so fun.

SCOTT: By the way,
I did play at my first game

of the season,
and I won, three to one.

-Good job, Daddy.
-Thanks. You proud of me?

-Yes.
-You taught me.

-You trained me well.
-I trained you well, rookie.

(SIGHS) Okay.

-NURSE 2: Breathe, Sam, okay?
-SAM: Okay.

-NURSE 2: One, two, three.
-Okay. Ow.

LESLIE: She's got it,
she's got it, she's got it.

-(SOFTLY) Okay, are we good?
-NURSE 2: Sorry, honey.

It's in. You just
have to relax for me, okay?

-And it works well now.
-Okay?

-NURSE 2: Take a deep breath.
-(SAM EXHALES DEEPLY)

NURSE 2: Good, Sam.

There. It's okay. Good job.

DR. MARILYN LIANG: Okay.

Okay. Let's see what this says.

SAM: It's on the left foot.

DR. LIANG: It's okay.
I'm gonna look at all of them.

SAM: Yeah, I know,
I was just saying.

Great.

Let's see.

-I know, it's bright.
-(CHUCKLES)

-DR. LIANG: Okay.
-(OBJECT RUSTLING)

Okay.

(CAMERA SHUTTER CLICKS)

DR. LIANG: Okay, I got 12
that time. Let's try that again.

Count again.

-NURSE 2: Is that uncomfortable?
-Oh, yeah. It always is.

NURSE 2: You can step away
from it, though.

-SAM: Let's just get the height.
-Okay. Can you
relax your arms at all?

Okay, good. And your shoulders.

-Hard to keep balance.
-(RATTLING)

And I'm gonna adjust
your head, okay?

-Okay, nice, big inhale.
-(INHALES)

One twenty-nine point two.

Good. Last time, okay?

All right, buddy, don't move.

(MACHINE BEEPS, WHIRS)

-LESLIE: I mean...
-That's good, right?

I mean, we'd have to have 'em
side by side,

so I don't really know.

(INDISTINCT CHATTER)

All right.

-SAM: I'm nervous.
-LESLIE: About the side effects?

-SAM: Mm-hmm.
-How can I help you with that?

You can't.

Just take Tylenol all the time.

You know what? Let's make sure
you stay hydrated.

We'll measure you if you think
you have a fever,

-or you feel funny...
-Mm-hmm.

...immediately,
and we'll take care of it.

-PHOTOGRAPHER 3: Sam.
-LESLIE: Last one, Sammy.

(MACHINE WHIRRING)

PHOTOGRAPHER 4:
A little bit lower this time.
Yeah, that's good.

-(CAMERA SHUTTER CLICKS)
-(CAMERA BEEPS)

Good, okay. And then...

SAM: My least favorite part
is the medical photography

because I feel
like a specimen being examined.

PHOTOGRAPHER 4: Other way.

-(CAMERA SHUTTER CLICKS)
-(CAMERA BEEPS)

PHOTOGRAPHER 4: Okay.

SAM: I don't even feel
like a person.

I don't even feel like I'm me.

I feel like that's all
that I'm good for.

-(SIGHS)
-(CAMERA SHUTTER CLICKS)

(CAMERA BEEPS)

-(CAMERA SHUTTER CLICKS)
-NURSE 3: Below the elbow?

SAM:
And not too close to the hand.

NURSE 3: So somewhere
around here is okay?

SAM: Yeah, it's fine.

If you feel, it's kind of thin
there for some--

NURSE 3: It is kind of thin,
but the vein...

-(GROANS)
-...is better right here.

(CHATTER ON TV)

NURSE 3: It's no fun, huh?

(SAM GROANS)

(SIGHS)

(GROANS)

(EXHALES)

We just turned up
the heat, too, okay?

Just make sure you don't rub
any of the IV spots.

NURSE 3:
I'm going right between 'em.

There.

SCOTT: Anything we can get you,
besides the television?

-SAM: Muzzy would be good now.
-Muzzy?

(SIGHS)

♪ (MUSIC FADES) ♪

(INDISTINCT CHATTER)

♪ (SUSPENSEFUL MUSIC PLAYING) ♪

LESLIE: You actually
cannot even ask the question,

"Is there evidence
that this drug is working?"

until the last child
has had their last visit.

(SPEAKING HINDI)

LESLIE: (IN ENGLISH)
That's when we start
to look and ask the question

in a very solid,
scientific manner,

"Is this drug having an effect?"

DR. GERHARD-HERMAN: ...children
with progeria as well.

And it's starting to develop
over here.

All right. That's-- that's
exactly what my question is.

-How is this part of this to--
-DR. GERHARD-HERMAN: No, no, no.

-Unless that's muscle. Okay.
-DR. GERHARD-HERMAN:
That's muscle.

Hutchinson-Gilford kids
don't splice progerin.

And we specify how VH, RVH,
plus, minus--

Some things did sort of pop up
and go away.

But then, it is every
four months,

-which is really nice to track.
-Okay.

-That one's the better one,
I think, don't you?
-LESLIE: Okay.

-Shows it more clearly.
-I like it.

I would take this out.

DR. KLEINMAN:
It didn't just happen.

DR. MARK KIERAN: ...just reached
your threshold of...

DR. KLEINMAN: something that
we could see, but if we try
to reason out of it...

LESLIE:
...be very careful about that

is because once you send a file,

the potential for that file
is lost to us.

-♪ (BAND MUSIC PLAYING) ♪
-(CROWD CHEERS, APPLAUDS)

(SAM CHEERING)

PHYSIOTHERAPIST: You getting
a good stretch? I know, I know.

Heel cords
and hamstrings are the worst.

And you can do side leg lifts,
and then one-leg bridges,

and then we'll do some sit-ups.

-Seven, eight.
-(GRUNTS)

TECHNICIAN:
Technically, the cardiac...

We need to know
that this child is this child,

and this child is another child.

And hopefully decreased a little
bit more with the Lonafarnib.

That might be a little tricky
to show with western.

-Let's win by two.
-CHILD 1: It's mine.

CHILD 2: I got it, I got it.

-Oh!
-Oh! (CHUCKLES)

(INDISTINCT CHATTER)

-Strike.
-(CROWD APPLAUDING)

COACH: There you go!
Hustle in, guys, hustle in!

Don't be last! Let's go!

LESLIE: ...gonna make
some statements, and then...

Generalized osteopenia
nine to 20.

AVN went from six to 17 kids.

-Yeah. And--
-All right. So we've got...

RESEARCHER:
...simplifying because
if you actually start giving...

LESLIE: ...most of the things
that we found here, okay?

RESEARCHER: ...reporting
in the Journal of Biomechanics.

-MAN: Whoa!
-(CROWD CHEERING)

(CROWD APPLAUDING, CHEERING)

Now, I'll get the one.

Sorry. That's my sorry.

LESLIE: Oh! (LAUGHS)

...change in the course
of a disease that otherwise
would not change spontaneously.

I think, to the extent
that that's all we can say,
that's all we can say.

DR. KLEINMAN:
You know, our other paper
argued that hiding...

LESLIE: Hello.

-Okay.
-DR. KIERAN: Ready?

Yes.

-This will submit.
-Are you sure?

(INDISTINCT CHATTER)

(MOUSE CLICKING)

-DR. KIERAN: We're done.
-All right. Congratulations.

-Thank you.
-Cross your fingers now.

-Now, the hard part starts.
-Yeah.

♪ (BAND MUSIC PLAYING) ♪

(CROWD CHEERS, APPLAUDS)

TEACHER 3:
This is the leadership award.

It's given to students
who are able to inspire others.

And the recipients
are as follows...

Sampson Berns.

(CROWD APPLAUDS, CHEERS)

-Well done, Sam!
-Thank you so much. Yeah.

TEACHER 4: These are
the presidential awards

for a strong commitment
to high academic achievement.

The first recipient,
Brittney Anderson, for the arts.

-(CROWD CHEERING)
-TEACHER 4: Sam Berns.

(CROWD HOOTING)

-Thank you again. (CHUCKLES)
-TEACHER 4: Dan Berr.

-SAM: Whoo!
-TEACHER 4: Lindsey Cundiff.

-Go, Lindsey!
-(CROWD CHEERS, APPLAUDS)

This award is given
to students

who have a grade point average
of 90 percent or more

for their four years
at the Ahern.

-Sam Berns.
-(CROWD CHEERING)

♪ (LIVELY MUSIC PLAYING) ♪

Thank you. (CHUCKLES)

The winner

of the Francis T. Chimney Award
for this year is Sam Berns.

(CROWD CHEERS, APPLAUDS)

-SCOTT: Way to go, man!
-Thank you very much.

(CHEERING CONTINUES)

TEACHER 5: Let's stand.

♪ (MUSIC PLAYING) ♪

(INDISTINCT CHATTER)

(MOUTHING) Oh, my God.

-(INDISTINCT CHATTER)
-SAM: Yeah. (CHUCKLES)

Thank you.
Thank you so much. Yeah.

I can't see anything. Yeah.

Yeah, I know, really.
Thanks, Kyle.

-Man, you're awesome!
-Thank you so much. Thank you.

Hey, thank you.

-Thank you so much.
-Congratulations.

-I am so proud of you.
-Thank you.

Thanks a lot. Thank you.
Thank you, Joe.

-Congratulations.
-Thank you. Thank you so much.

(INDISTINCT CHATTER)

Go, Bruins!

-Hey.
-(CHUCKLES)

PHOTOGRAPHER 5:
Oh, that was a good one!

(SEAGULLS SQUAWKING))

♪ (MUSIC FADES) ♪

♪ (SOMBER MUSIC PLAYING) ♪

(KEYBOARD CLACKING)

LESLIE: It's frustrating.

For something like progeria
and other very rare diseases,

a lot of times, a journal says
something like, "You know what?

This is great, but usually
half of the people

get a placebo and half
of the people don't."

And that's what people
are used to publishing.

We didn't have that.
We have a tiny population.

And we gave everybody drug.

They said, "You know,

you really haven't shown
a clinical difference

in your patient population.

Even though
you've shown a change,

we just aren't interested
in this

because it's not enough for us."

But we still had the statistics
to back up our findings.

And so we have to do the work
and submit again.

CROWD:
...seven, eight, nine, ten,

11, 12, 13, 14, 15, 16...

-Sorry, Sam.
-It's okay.

...18, 19, 20...

SAM: The main thing that
I'm excited for in high school

is marching band,
because I've always known

that I wanted
to be on a drumline.

I had already, you know, known
that I wanted to be a drummer,

but, like, the drumline
just looked so cool.

BANDLEADER: T-ten, a-ten-hut!

BAND MEMBERS: Play!

SAM: 'Cause the snare drum
sounded really awesome,

and you could really
show yourself off

and be part of something
really, really cool.

♪ (DRUMS PLAYING) ♪

SAM: This marching band
is outrageously good.

You have to try out,

and you just have to do
really well.

...and it should be a vessel
with progeria...

and either showing progerin
in that vessel or...

♪ (DRUMS CONTINUE PLAYING) ♪

ALL: One, two.

MARCHING BAND MEMBER:
How you doing, Sam?

-SAM: It's hard to keep balance.
-MARCHING BAND MEMBER:
Yes, but you got it.

(SAM CHUCKLES)

Everything in drumline
is challenging

because the mechanism
to carry the drums is, like,

a metal vest-type thing
that adds weight,

and the drum
is pretty heavy anyway.

I can't march
with this thing on.

LESLIE: Do the motions
without the drum. Go ahead.

(INDISTINCT CHATTER)

Yeah, it's all right... yeah.

-COACH: Left, Joseph. Left...
-(CHUCKLES)

...left, left.

LESLIE: All of a sudden,
Sam was told,

"No, no, you're not
gonna be on the field."

-One, two, three.
-LESLIE: And he was shocked

and really, really upset.

COACH:
There is an intensity to it.

We occasionally have kids
bumping into each other.

I mean, it's-- when you're--

in the rehearsal process,
it's pretty ragged and sloppy.

That's why I have some concerns

about just his physical safety,
being in the midst of that.

The snare drum parts
are, without question,

the hardest parts
in the percussion line.

Combining these very complex
parts, carrying a drum,

and moving, that could be really
pushing the envelope.

You're right.
There is no way he's gonna carry

whatever that structure is
that they carry.

Not only is the drum itself
too heavy, but that--

-the contraption where--
that holds it onto--
-COACH: Right.

There is zero, zero chance.

He doesn't even
have those shoulders.

So we-- we did find some things,

um, that we-- we think
might work. They're lighter.

-SCOTT: This is very thin.
-COACH: Yeah.

-LESLIE: Yeah.
-COACH: No, I'm happy
to look that over.

LESLIE: But it's a way
to not knock out that position

right now.

All right.
Want me to hold that for you?

Thanks, buddy.

COACH: Your mom talked
to me about the fact

that you were interested
in playing snare drum.

That may or may not happen.

We're still trying to figure out
skill sets of all the kids

and get the people
in the right place

to produce the best band.

-That fair?
-All right.

Well, thank you very much,
both of you.

COACH: All right.

(DRUMSTICKS TAPPING)

LESLIE: It was difficult
to see Sam so stressed...

because he just kept thinking,
"I've wanted this for so long.

It's so cool, I love the band,

I love being with
all these kids and playing.

Can we make this work?"

♪ (QUIET MUSIC PLAYING) ♪

-So, this is the new frame.
-SAM: Oh, that's cool.

TECHNICIAN: So that, um,
the middle hole
is where you said you wanted it,

-and I put two more holes...
-LESLIE: Yeah, sure.

TECHNICIAN:
It's less than a pound.
(GRUNTS SOFTLY) Great.

-Yeah.
-Very light.

♪ (MUSIC FADES) ♪

♪ (SOMBER MUSIC PLAYING) ♪

LESLIE: I think, sometimes,
we take time for granted.

Time is running out
for every kid with progeria,

and it's a horrible feeling.

Every child deserves
everything we can give them,

everything I can give them,
every day.

Mr. Veluri, can you tell me
in your own words

what-- what happened
on that final day?

I know it's very difficult,
and I thank you.

(MR. VELURI SPEAKING HINDI
OVER THE PHONE)

SCOTT: One of the hardest things
that we have to face

is when another child
with progeria passes away.

And I can tell you that...

uh,
when we do see those families...

after their child passes away,

that nothing needs to be said.

That... the hug
really says it all, and...

there's an instant
communication that happens,

which... is the feeling of,
"I'm sorry

that you lost your child,
from me.

I wish
that we could have done...

more to help, sooner."

And then that feeling
right back, which is...

"We know that you tried,

and that you're doing
everything that you can,

and you concentrate on Sam
and all the other kids now,

and we're gonna
be right behind you."

And those are the feelings
that don't...

and the thoughts that don't
have to be actually said,

because they're just there.

LESLIE: You should know
that you and your wife did

absolutely everything
possible for Priya.

She was incredibly
well taken care of.

(MR. VELURI SPEAKING HINDI)

INTERPRETER: (IN ENGLISH)
He's, uh-- he's trying to say
thank you to you

because you have provided
her medicines,

and, uh, so, actually,
he's grateful to you.

Thank you, Mr. Veluri.

Um, I want you to know that
please contact me if you ever--

or your wife or family ever
has any questions or needs,

I will always be here for you,

and so will the rest
of our team.

(MR. VELURI SPEAKING HINDI)

(BIRDS CHIRPING)

SAM: Something happened, like,
right around my birthday.

I woke up, and everything
kind of sounded weird,

'cause I had just
woken up and...

um, my mom was...

I was basically listening
to my mom

filling out a death certificate.

Um,
I didn't know who it was for,

but she was just talking
about... a patient,

time of death,
all that stuff, so...

When I woke up,
I was kind of shocked.

I asked my mom, I said, um...

"You know, I-- I heard
somebody died." Um...

And... she asked me, like,
how I'd heard that,

not like-- she just asked me
how I'd heard that,

and I said I overheard her
talking about it,

and, um, she said it was Priya.

I was sad, obviously.
I was kind of shocked 'cause...

I had just seen her...

a couple years before,
and she seemed--

she seemed good, and...

So, um... that was it.

And so, Priya--
Priya was dead, and...

I just kind of had
to keep going, and...

she knew that,
and I knew that, and...

I've never really learned
about a child

that had passed away
in that sort of way.

My mom had always told me.

So it was just
one of those things.

But it happened.

♪ (SOMBER MUSIC CONTINUES) ♪

♪ (MUSIC FADES) ♪

♪ (QUIET MUSIC PLAYING) ♪

(LEGOS CLACKING)

SAM: My hope for the future...

I would like to become
an inventor when I grow up.

I, uh, want to go to
a really good technology school,

preferably MIT.

You know, I think that's really
where I can get my start

to achieve my goal in life
to be an inventor.

Kind of like Albert Einstein
and Steve Jobs combined.

Uh, you know, kind of, like,
invent things

to help people out
with their daily lives.

And then, hopefully, I'll get
to win, you know, a Nobel Prize.

Um, but if I don't, that's okay,
as long as I,

you know, I'm happy
with what I'm doing.

Maybe retire early, spend time
with my family, I don't know.

I just would like to live
a happy, happy life.

That's pretty much it.

♪ (MUSIC CONTINUES) ♪

♪ (MUSIC FADES) ♪

-(LESLIE SIGHS)
-(MOUSE CLICKING)

-Ugh.
-(MOUSE CLICKING)

Okay.

They want a revision.

Oh, there's another revision.

Oh, my.

There's another revision.

Okay.

-(MOUSE CLICKING)
-Okay. Let's see where we are.

We have to revise again
and submit again.

This is gonna be
our last shot at it,

so crossing my fingers.

We'll just--
we just need to do the work.

So, that's it.

♪ (UPLIFTING MUSIC PLAYING) ♪

♪ (MUSIC CONTINUES) ♪

(KEYBOARD CLACKING)

♪ (MUSIC FADES) ♪

(KEYBOARD CLACKING)

-(SIGHS) All right.
-(MOUSE CLICKING)

Oh, my goodness. Okay.

It's in. (EXHALES)

It's in.

It's in!

-Paper's in, dude.
-Yay!

-(LESLIE GIGGLES)
-SAM: Finally.

Hi! (LAUGHS)

I-- I just-- I just opened it.

I just opened it. Yahoo!

-Audrey Gordon.
-AUDREY GORDON: Yes?

"We are pleased to inform you
that the PNAS editorial board

has given final approval
for your article...

-for publication."
-AUDREY: Oh, my God! (LAUGHING)

(CHUCKLES)

-AUDREY: I think I'm gonna cry.
-Don't cry now!

AUDREY: What am I supposed
to do right now?

(LAUGHS)

REPORTER 1:
Big news coming out of

the Boston Children's Hospital
at this hour.

The first-ever treatment
for a rare childhood disease...

REPORTER 2: Researchers
at Children's Hospital have made

a significant breakthrough
on an extremely rare...

REPORTER 3:
...for children suffering
from a rare disease, progeria,

-which causes rapid aging.
-REPORTER 4: Most of the time,

medical research
is more like a marathon

than a 100-meter dash.

But today, researchers
are publishing

the result
of a scientific sprint.

LESLIE:
We had never studied progeria.

We didn't know anything
about progeria.

It's an extremely rare disease.

Finally, I can say,

"Yes, something
is really happening here."

(CHUCKLES)

I'm here to talk to you
about the results

of the first treatment trial
for children with progeria.

No family knows exactly
what's gone on.

No family knows the results.
They don't know.

Um, they don't know until
the rest of the world knows.

What you see here

are the children who succeeded
in rate of weight gain.

All of that effort
and all of that faith

and all of that waiting

sort of culminates
in this moment

for those families.

Every parent
is gonna wanna know,

"Exactly what does this mean
for my child?"

What I'd like to go through
with all of you

is what we found for you.

-(INTERPRETER SPEAKING ITALIAN)
-LESLIE: (IN ENGLISH) Okay.

The next thing
I'm gonna show you

is about your blood vessels,
your cardiovascular system.

It's a measure of how stiff
your blood vessels are.

(INTERPRETER SPEAKING ITALIAN)

(IN ENGLISH)
You started at 18.3...

-(INTERPRETER SPEAKING ITALIAN)
-...which is up here.

-And you ended at 11.7.
-Wow. (CHUCKLES)

Okay?
It's a big, big, big change.

(INTERPRETER SPEAKING ITALIAN)

LESLIE: (IN ENGLISH)
This means we now know

that there are things
in progeria

that can change with treatment.

(INTERPRETER SPEAKING ITALIAN)

(IN ENGLISH) And that we have
the first drug for progeria now.

(INTERPRETER SPEAKING ITALIAN)

-NICCOLO'S MOTHER: Wow.
-LESLIE: (IN ENGLISH)
Are you okay?

-(CHUCKLES) Yes.
-(ALL CHUCKLE)

-LESLIE: Okay?
-NICCOLO'S FATHER: Okay.

-Mamma mia.
-(LESLIE SPEAKING ITALIAN)

-(IN ENGLISH)
Thank you. Thank you.
-LESLIE: You're very welcome.

So, what happened to you?

Well, after you took
the medicine for two years,

you were all the way
over here.

You did extremely well.
It's a very good thing to see.

We've got 55 kids in this box...

In one area or another,

all of the children
have benefited.

This is the big,
unbelievable result.

Completely unexpected.

Had no idea that we were gonna
find all of these things.

Improvements in weight,
improvements in bone,

and even improvements
in hearing.

But I don't think
I could have imagined

being able to see something good

happening
for the cardiovascular system,

because, of course,
that's a dream.

The stiffer the blood vessel is,

the harder
the heart has to work.

LESLIE: Niccolo's results
is a success.

-(CHUCKLES)
-LESLIE: He did really well.

And for you,
you started out at 10.1...

-DEVIN: Okay.
-...and where did you end up?

Oh, my gosh.

You came all the way down here.

-DEVIN: Six, four?
-(SOBS)

LESLIE:
Six point four, so that means...

-Oh. Are you okay?
-I'm sorry. (SNIFFLES)

-Okay.
-(SNIFFLES)

(VOICE BREAKING) Thank you.

(SNIFFLES)

Yeah.

-SCOTT: You okay?
-SAM: Hmm.

-DR. KLEINMAN: This okay?
-LESLIE: It's perfect.

Okay. All right.

So, you've probably heard
that the paper

that has everybody's results
all together

is published
to the rest of the world.

My question is,
what happened to Sam Berns?

So, this is your results,
right here, okay?

This is what exactly happened
with you in the study.

So, let me start with weight.

At the time that you came
into the study,

you were actually losing
a little bit of weight.

Something changed
between here and here.

And it was the treatment
that you were receiving.

Boom. You started growing.

That's a pretty
substantial increase.

DR. KLEINMAN:
That's, like, three pounds.

-LESLIE: Per year?
-DR. KLEINMAN: Per year.

LESLIE: So that's, like,
six pounds in two years.

-Yeah.
-DR. KLEINMAN: Exactly.

-SCOTT: Yeah.
-DR. KLEINMAN:
Those numbers are phenomenal.

Those chocolate shakes
helped a little.

-SAM: Yeah, yeah.
-DR. KLEINMAN: Wow. okay.

Let me also talk
about your bone density.

This is related to
risk of breaking a bone,

-risk of a fracture.
-SAM: Yes.

DR. KLEINMAN: After treatment,

your bone density
actually went down a little bit.

You know, I think
it would be safe to say

that Sam's bone density

is probably lower
than we'd want it to be.

So, of course,
we'll all be interested

to see what happens
to your bone density.

All right. As you know,

in progeria,
one of the biggest concerns

is the disease
in the blood vessels, okay?

And so we were measuring
something called

pulse wave velocity.

And a pulse wave velocity
that is fast...

indicates
the blood vessel disease

where arteries get stiffer
and more narrow.

Prior to treatment,

your pulse wave velocity
was 13.7,

and after the treatment,
your pulse wave velocity

was 10.8.

That means
that the arteries in the neck,

the carotid arteries,
which take blood to your brain,

are wider and more relaxed.

And that reduces--
Or at least what I can,
you know, infer that it reduces

the stroke risk
to a certain extent, which is...

DR. KLEINMAN:
That's our hope, exactly.

-SAM: Which is very good.
-DR. KLEINMAN: Right.

That's pretty-- that's insane.
I just-- That's amazing. Sorry.

-DR. KLEINMAN:
It is quite amazing.
-Yeah.

Wow.

SCOTT: It was an incredibly
momentous day.

There's just
no question about it.

I mean, we don't know
what the real long-term impact

is gonna be,
because we just don't know.

But, I mean, thinking back
to when he was diagnosed,

we were given a certain
prognosis and a certain outcome.

You know, I don't think
that's where we are anymore.

We're in a different place.

-Okay?
-All right.

-LESLIE: Thank you so much.
-Awesome.

Great. Good job, everybody.

(LAUGHS)

SAM: When I saw those results
on paper, I saw the numbers,

it was--
it was irrefutable for me.

I mean, I was--
I was skeptical going in

about whether the results
would show

a significant change at all,

and when I saw that,
I really believed it.

It's just irrefutable
in that aspect. It was just...

just -- it was just
so certain, for once,

that something good
was happening.

We're all in a really
good mood right now,

-so, um, I was wondering, um...
-(SCOTT CHUCKLES)

SAM: So, like, Gus and Varoon,
could they--

-could they maybe sleep over?
-(LESLIE GASPS)

Gee, I didn't anticipate
that question coming.

-SAM: Well, they were--
they were wondering--
-LESLIE: I had no--

Did you have any idea

he was gonna ask
for a sleepover tonight?

SAM:
And-- and, like, we could...

LESLIE: For the first time,
we have hope.

The reality of a treatment
is here,

and that makes everything else
seem possible.

It makes anything seem possible.
It makes cure seem possible.

This is not a cure.
This is a treatment.

But it feels
like a real beginning.

(INDISTINCT CHATTER)

(CROWD APPLAUDS, CHEERS)

-(CROWD CHEERING, APPLAUDING)
-BANDLEADER: A one, a two,

a one, two, ready, and...

♪ (DRUMS PLAYING) ♪

♪ (MARCHING BAND
MUSIC PLAYING) ♪

-ALL: Yeah!
-(CROWD CHEERING, APPLAUDING)

♪ (QUIET MUSIC PLAYING) ♪

SAM: I feel like now,
progeria is more known.

I know
more about it genetically...

so it's less
of an embodiment now.

It used to be, like, this thing
that prevents me

from doing all this stuff,
that causes other kids to die,

that causes everybody
to be stressed.

And now, it's, uh...

you know,
a protein that is abnormal,

that weakens
the structure of cells.

♪ (MUSIC FADES) ♪

So...

And it takes a burden off of me,

because now,
I don't have to think about...

progeria as...

an entity.

♪ (MUSIC PICKS UP) ♪

SAM: Uh, as for my life,
I just feel insanely lucky.

I have great friends and...

I am pretty happy
with my personality,

for the most part.

I always like to let
my friends and family know

how much I care about them,

even if it's not, like,
saying, "I love you,"

'cause that would be
a little awkward with my bros,

but, you know, just...

(SIGHS) I think
it's just all about...

being happy and loving
and accepting your emotions

so that you can make it better,
I guess.

I mean, there's always--

there are always things
to look forward to,

and I'm just happy that...

I get to experience them,
I guess.

♪ (MUSIC CONTINUES) ♪

♪ (MUSIC CONTINUES) ♪

♪ (MUSIC CONTINUES) ♪

♪ (MUSIC CONCLUDES) ♪

♪ (QUIET MUSIC PLAYING) ♪

♪ (MUSIC CONCLUDES) ♪