Original subtitles

Uh-oh. Uh-oh. You better pull the tongue up, pull the tongue up.

- There you go. Is that better? - That... That's a lot...

- Yeah. - Okay.

- All right, I got this. - You guys going?

Very slowly but surely,

we're going somewhere.

He doesn't want people to say, you know, "Oh, he's the blind kid."

Because to him, he's... he's pretty much anything but the blind kid.

Hi, Jackson!

Hi!

He's the boy who can do the monkey bars at recess.

He's the boy on the wrestling team.

He's, you know... He is just Jackson.

- You okay? - Ow!

Sorry about that.

We've been hoping, since the day we found out what he had,

that something would come along and...

be able to help him.

- You all right, bud? - Yeah.

- You sure? - Yeah.

See you later!

And now there is an FDA-approved treatment.

There is some kind of hope.

We're on the verge of a technological breakthrough

that could change the future of mankind.

Most people have no idea how rapidly things are changing.

We're now changing the DNA, the code of life.

It's amazing, the sorts of things that we could do with this.

The question is, should we?

What happens if diseases were just eradicated?

All technology is inherently about changing away from what is natural.

A groundbreaking treatment

for people with a faulty gene that impairs vision.

It would be the first gene therapy in the US for an inherited disease.

How does gene therapy work? This treatment?

It's pretty amazing.

You know, you inherit two chromosomes: one from the mother, one from the father.

In this case,

they inherited a faulty gene from each parent.

Is this CRISPR?

No. So, CRISPR corrects something that's already there.

This is taking a normal copy of the gene

and putting it in to replace the function that was missing.

We're living in a 21st century

where we can create a world where no life is limited by genetic disease.

I mean, that's our vision as a company.

There is a lot that I think this genetic revolution can do

to transform the state of the human condition

throughout the world.

Building on the knowledge of the Human Genome Project,

which identified the 30,000 different genes, uh,

that are expressed in humans,

we knew that this piece of DNA, RPE65,

is very important in the visual cycle.

We demonstrated that if we can just give a copy of a normal gene

to the cells that are there,

then we have a possibility to restore vision.

Ultimately, you're transferring in a normal, functional copy,

a healthy copy of a gene

alongside the 23 pairs of chromosomes.

It would be fair to say

that DNA therapeutics

is one of the most complex types of therapies,

and it's taken a long time to develop.

We're designing a rocket ship, but someone's gotta get into it,

and those people ultimately shape history.

And it is like stepping into the proverbial void and staring into it

and saying, you know, "We're gonna hold hands together and jump."

♪ Bodies multiply ♪

♪ Too many fortunes ♪

♪ Too many lies ♪

♪ Where's your blood ♪

♪ Walk into the fire... ♪

Oh, come on.

Fuck me.

And there it is.

Which one are you?

Down here.

I like to see you lose when you get really mad.

I don't get mad, really.

I get mad about when some bullshit happens.

Language.

No, no filter.

No filter for me.

All right.

They'll need to beep out every word that I say.

I'll leave you to your game.

Finally.

"Finally."

I am a computer nerd.

Listen to music,

play games,

watch movies,

talk to friends online.

You know, it's something I can do independently...

in a world where I don't have much independence.

I remember saying, "I have so many cousins

who have so many children, and everyone is healthy. How...

- What is this? How can we... - Yeah.

And where does it come from in our genes?"

Yeah, and... and a good way to explain it to people,

when I explained it to some friends of mine, was,

"The husband can't blame the wife, and the wife can't blame the husband.

We each have to carry the gene

in order for this thing to be passed on to the next generation."

Right.

- Hello. - Hello.

How's that belly doing? How's the baby doing?

- Good. - Any kicking activity?

A little flutter, but not much.

Well, introduce him, Manny.

Well, this is the newest member to the Piazza family here.

His name is Nicholas Eric Piazza.

It's like, you know, he moves...

cries, and then he just, like, stops.

We were over the moon,

and then at a well-care visit, at seven months of age, the doctor...

lifted him to bear weight,

and he wasn't putting any weight on his legs.

And he basically then said, um...

"I suspect him to have Werdnig-Hoffmann disease,

- which is SMA type 1." - Right.

And he bluntly said to me, um...

"Go home and love your baby.

He's gonna die before the age of two.

And don't have any more kids."

♪ Happy birthday to you... ♪

- You want to blow? One... - ♪ Happy birthday ♪

- ♪ To you ♪ - ...two...

♪ Happy birthday... ♪

Yay.

Yay!

Yay!

SMA affects the cells that...

you control your legs with,

your arms with, your voluntary muscles.

♪ Itsy-bitsy spider Went up the waterspout... ♪

But the strange thing about the disease is

is if you take your finger and you go like this to Nicholas,

- he'll feel that. - He can feel that.

So, in one aspect, he's paralyzed,

but in another aspect, he still has all feeling in his body.

My tattoo is a spine with chains wrapped around it

in the shape of DNA.

What I tend to tell people

when they ask me, you know,

"What is it like to have SMA?" um...

I say when you get home,

lie on your bed,

and get comfortable,

and don't move.

If you have an itch, don't move.

If you have to pee, don't move.

If you're in pain, don't move.

And tell me how long you last."

This drug called Spinraza was approved just before Christmas.

It's a really exciting drug.

It treats spinal muscular atrophy, which is a rare genetic disease.

It is just devastating.

There has been nothing to treat this disease until now.

And this drug has really shown, in clinical trials, to make a difference.

Use your arms. Push up.

There you go. You're doing it.

- Push up. - Go. Push, baby.

Push, baby.

Yeah! Good!

Wow.

- I cannot believe that. - I know.

- That kid was a type 1? - Mm-hmm.

That's amazing, man.

Look at you standing there all by yourself.

Get them up here?

Can you get them up there?

I couldn't even imagine if that happened... 24 years ago.

I know.

These people have no idea how lucky they are.

We knew that there were medicines coming. We knew that they were working on stuff.

Just never thought it'd be in Nick's lifetime.

We just never thought it was gonna happen in...

Exactly, in Nick's lifetime.

My understanding is, for the first year,

- it's $750,000. - Right.

- And then, it's $350,000 for life. - Every year after.

- Every year after. - Right.

And insurance has not approved us yet,

- so we are still fighting. - Yeah.

These new drugs that are coming to market,

they're gene therapies, they're miracle drugs,

but they have to be affordable for people.

We're gonna bankrupt families and cause our system to buckle

under the weight of those prices.

This drug, Spinraza, will be about $750,000 for the first year.

After that, about $375,000 a year for maintenance.

So, really effective, but really, really expensive.

Are we gonna be seeing more of these high-priced drugs?

Yeah, we are. There are dozens of them,

some expected to cost in the millions of dollars,

which impacts all of us and our insurance.

Big news in the drug pricing world.

Spark Therapeutics, of course, last month received approval

for the first gene therapy that treats a rare inherited form of vision loss

that can lead to blindness.

The company says it's pricing that one-time gene therapy

at a total of $850,000,

or $425,000 per eye.

Just like how NAS came out...

We're closing in on a million dollars when we talk about Luxturna.

And, oh, by the way, Luxturna

is based on basic science

paid for by taxpayers through the NIH.

A lot of people don't know, I think, a lot of patients don't even know

a lot of these drugs are funded with taxpayer dollars.

Pharma wants us to believe...

that if they can't make outrageous profits,

there will be no research and development done.

The pharmaceutical industry is the most profitable industry

in the country.

They have the highest-paid executives.

They spend more money on advertising and marketing

than they do on research and development.

Spark Therapeutics now have enough cash to keep running for years.

What do we do in the great ethos debate

about pricing for something that brings back sight?

The real question we focused in on was less about

what was the cost of investment,

what was the cost of manufacturing it, or cost of goods,

but what was the value of sight in a child?

It makes me really angry

when I hear drug companies ask the question,

"What is it worth to you to save your child's life?

What is it worth to you to cure blindness?"

Hell! It's worth whatever I have to spend. You know? I'll empty my bank accounts.

I'll... I'll mortgage my house. What do you want?

That is not the right question.

When insulin was invented in the 1920s,

the three inventors sold the patents

each for one dollar.

They said, "We're giving insulin to the world."

When Jonas Salk invented the polio vaccine,

he didn't patent it.

Who owns the patent on this vaccine?

Well, the people, I would say. There is no patent. This is...

Could you patent the sun?

I received that polio vaccine

when I was six years old at Cerveny School.

But if you would have priced that polio vaccine

the way drug corporations are pricing drugs now,

it probably would have cost a million dollars.

We're quickly coming to a point where the cost of these drugs,

for the numbers of people who could benefit from them,

will break the... the proverbial bank.

And so, I think every indication is

this will be an issue that only becomes much, much, much, much bigger.

What does it mean to grant patent protection,

intellectual property protection,

for genes or gene-editing tools?

Or are we granting intellectual property protection

for things that people shouldn't have the right to claim ownership over? Right?

It's sort of granting, in a way, a property protection

to something that isn't their property.

Reform to lower drug prices won't happen

without patients rising up and demanding change.

I think things don't change

unless the medical establishment has no choice but to change.

Two big bags of moss.

- Two big bags of moss. - You get that.

We need some containers.

Where are the frog boxes?

I used to get, like, maybe one email a month,

and now I get, like, ten a day.

Generally, it's people who are, you know, seriously sick.

Uh...

A lot of muscular dystrophy people because...

I... I did that CRISPR experiment on my muscle.

All these people need help,

but nobody has the money or knowledge to help them.

What happens if we can double or triple the scientific population of the world?

We're gonna pretend like...

somebody just received a kit in the mail, right?

Okay.

So, like... we'll start off with, like...

"When you first receive your kit from The Odin,

it's very important that you follow these instructions."

This frog right here has not received the gene therapy.

He is at the exact same weight when he started.

This frog, who we have named Thick Boy, received the gene therapy.

He started out also at eight grams of weight.

And he now weighs...

1.6 grams more than he did at the beginning.

Yeah!

Hi! I'm here today

to show you how to inject a frog safely and effectively.

We are going to inject this frog with the IGF1 gene.

IGF1, what's that?

IGF1 makes you grow bigger.

Syringes.

This gene therapy will teach you

not only how to do gene therapy on animals,

but how to be an all-around good scientist.

I want scientists to be badasses.

I want people to actually do science.

All right, is this one ready to go out?

- Ready. - Ready to go out.

What now?

I might wanna say... say something.

I miss being able to, like...

just... drive down a hallway,

on my own,

without needing someone to push me.

Would be amazing to have that.

Because I don't think I realized how much

I valued moving on my own

until I lost it.

He's never walked,

so the very first time he ever got into a powered wheelchair, we... we cried.

Over the years, he started to lose that function,

and if he could get back into a powered chair...

- That would be... - ...just being able to sit up

a little bit more...

- That would be unbelievable. - ...and get that thumb to drive.

But even... even at a lower level,

- even, like, just being able to swallow. - Right.

He drools on himself,

and we have to go back there

and wipe him, and clean him up.

If it was to help him to stop doing that, to just make him swallow better,

and give him some dignity.

- You good? - Yeah.

Please stay on the line

while we connect you to our associate.

Hello, this is Caroline.

Hey, Caroline, how you doing?

Good. How are you?

Good, good. My name's Manny Piazza.

I'm calling you in regards to my son, Nicholas Piazza.

Yes.

The question that we have is what would the dose actually cost?

That's a hard thing to say because it depends on the insurance.

I'm talking about if I wanted to pay for it.

Uh, so, our... our, um, wholesale at physician cost

for the drug is $125,000 a dose.

So I wanted to get my son a treatment, it would be $125,000 for that treatment?

That's obscene.

Yeah, it's those pharmaceutical companies for you.

Fucking Christ.

Yeah, that's crazy.

Is there any negotiation with that? Let's say I came up with...

If I tried to do a GoFundMe page.

- I'm just... I'm just asking a question. - Yeah, no...

How much money would I need to raise

in order to give my son that first treatment?

Well, so, I mean, we don't want us to have to come to that stage at all.

It's just sad,

and it's... it's...

It's... You don't notice it

until it's, like, affecting your family,

until it's, like, your son who needs this medicine,

your father who needs this,

your brother who needs this treatment

just to maybe have a better quality of life.

In the United States,

the number one cause of bankruptcy is medical bills.

Exactly, yeah.

You should not have to go bankrupt

to not die.

Yeah.

That's crazy.

Yeah.

It took us almost eight months to find a doctor to even talk to.

You know, I even called up Spinraza

to ask them how much it cost to make the drug.

I mean, I... I don't even know if this price is even a legit drug

or if they're ripping me off, to be quite honest with you.

I'm wondering why it's so expensive in the first place.

Sure. Absolutely. Let me look into...

um, let me look into that

and see what information I can get for you.

All I can say is that, for adults...

Yeah, Caroline...

What I think I don't want to do is I don't want to tell my son no anymore.

- I'm a little tired of doing that. - I... I don't...

No, no, no, I absolutely understand that. I don't wanna do that either.

I'm really trying to keep my emotions in check here,

but to be quite honest with you,

the frustration's getting to be a little bit ridiculous at this point.

It's ridiculous.

It's just, everywhere we go,

there's, like, another roadblock for this freaking thing.

When I think about

how hard we worked to get to this point,

and...

now we gotta deal with some kind of bureaucratic nonsense.

They're saying this life...

is worth a million dollars.

Yeah.

Pay up or fuck you.

Yeah, pretty much.

That's what they're saying.

Your call has been forwarded to an automatic voice message system.

Hi, Katherine.

Um, I am the father of Nicholas Piazza,

and, um, I just had a couple of questions I wanted to go over with you.

When you get a minute, give me a call.

SMA, if left untreated,

is a death sentence.

The disease is degenerative.

Every moment that goes past,

I'm losing more and more nerve cells.

If you have a medical emergency

which needs to have a doctor contacted immediately,

please press one.

So, we do have a test, which...

Yeah, yeah, so...

I have been optimizing.

Yeah. Oh, yeah, I saw that picture.

Yeah, the fluorescent DNA.

When I first started, it was like maybe one sperm has it, right?

But right now, there's, like, so many of them.

But that's just to test the protocol. And then, once we have that,

I'm gonna be using CRISPR to start repairing genetic diseases.

- Yeah. - So...

But right now it's still just like, "Let's get past that barrier..."

- Now you got that gene, you can... - Yeah.

- That's great. - Technically...

For all of history,

all of us, all organisms,

have been slaves to our genomes.

To see all these people talk about genetic engineering, CRISPR,

and... and say,

"I'm afraid

of somebody doing something bad."

We've all... Everybody's talked about this at this conference,

"I'm afraid of somebody doing bad."

You know who it doesn't scare?

The people who are suffering from genetic diseases,

the people who suffer every day

and just wish there was something to help them out.

If you're a scientist,

what are you doing

if you're not trying to fucking help people?

We don't have review boards.

There's nobody to tell us what to do. There's no committees

who can sit there and say you can and can't do this.

No. We make that choice.

The fucking thought

that somebody like David Ishee can contribute to scientific literature

is scoffed at by academics.

He doesn't have a PhD or a master's or a bachelor's,

but he's one of the most brilliant dudes I've met.

We buy our equipment on eBay.

We run it out of our garages, our kitchens, our sheds.

And because of this,

everybody thinks we're gonna destroy the world.

Well, fucking A.

The world's already destroyed,

and biohackers are the only ones who can motherfucking save it.

Science is your hope.

Knowledge is your hope.

I say, be your own fucking hope.

Roberts?

- How you doing today? - Mmm, I'm doing all right.

Deep breath.

That's it.

People who have HIV are consistently tested

for their viral load,

which is a measure of how much virus is actually in their bloodstream.

I was on the traditional, uh, medication.

When I left my job, though,

my insurance became a little bit more imperiled.

The ability to access treatment

really is a factor of your socioeconomic well-being.

The medicine, without insurance,

would run someone around $25,000 per year.

It doesn't make sense.

We put up with it because it seems like there's no better solution.

And I'm starting to see that... we're ready for that better solution.

Being HIV-positive,

the sort of distance that it places between you and everybody else,

especially your loved ones,

where, like, the very expression of your physical love

brings with it the possibility of death.

As a privileged white dude,

I would be just fine getting this medicine,

but I know that it's just not the reality

for millions of other people on this planet.

I'm interested in pursuing an alternative therapy

through this network of biohackers,

a way that...

transcends the... the need for the FDA to get involved.

We got Aaron. We're already on a live stream.

- Can we have you... - Absolutely.

I'm Aaron Traywick. I'm the CEO of Ascendance Biomedical.

We are developing gene therapy technologies

outside of the approval process.

Our first application will be the, uh, N6 HIV therapy for Tristan.

We're just gonna have to show the world

what radical gene therapy technology can do

to help people take the health of their lives into their own hands.

We're now ready to move forward with live testing with Tristan for HIV.

It's currently the wild, wild west.

Here we are, with minimal resources.

We don't have a lot of money, but we've got...

everything that it takes to do this,

and we do this largely as a self-funded entity.

Individual entrepreneurs banded together to deliver these treatments.

The goal is identify the need,

deliver a quality product,

self-experiment.

There is some cause for concern, for certain.

Like, I'm not gonna say that I'm not at all, like, bothered

by the possibilities of this causing some sort of allergic reaction

or of this ending up becoming, like, a virus in its own way,

where it's reproducing, but it's irritating my immune system.

But these are risks that I'm willing to take,

not just because of the potential impact,

but because I trust the researchers

to put together something that will probably not kill me.

I'm hoping the trust is not misplaced.

So, I've got a really large amount of DNA prepped.

I have everything I need to do this.

It's just a matter of doing the work.

So, in order to get Pele pregnant,

I'm mixing the DNA solution that carries bacterial genes and allows them to glow.

And now I'm adding it to the dog sperm cells here.

So now, hopefully, her eggs will be fertilized by those sperm...

and, uh, we will make a transgenic puppy or two.

Come here, girl.

Pele.

There's a company called Ascendance.

We kind of got connected through a biohacker buddy of mine in Austria.

So, he read a study about, uh, an antibody that kills...

lots of different strains of HIV.

And so they wanted to, um,

see if they could turn that into a therapy

and try to make it work with my cheaper gene therapy platform.

Tristan wanted to make sure that there was, um,

a little bit of tamper-proofing,

and this needs to not be frozen, so it just needs to be cool.

So honestly, it would be reasonably stable at room temperature.

You know, we ship blood samples back to the lab,

and as long as it gets there within a day and the temperature is still safe,

then it should be just fine.

Right, right.

The way I've got it, there's four syringes.

So, all he'll need to do, uh, when he gets it

is just stick it in the fridge till you're ready to use it.

Excellent.

This is N6.

This is a rare HIV antibody.

It's a new approach to attacking HIV.

So, the idea of the whole project is to...

uh, take this antibody that occurs in some people with the right genes

and transfer that to people it wouldn't normally occur in.

So, basically, you're transporting the immunity

from one person to another

by taking this... this DNA sequence from one person to another.

I want to participate

in the biohacker community of spreading this around,

because I want to see working-class people be able to fix themselves.

If a handful of people control the technology

and a handful of people have that power,

then those handful of people are holding the reins on the world

and they can say, "Okay, well, we're gonna cure all the diseases,

but you can't have it," or, "You can't have it,"

or, "You just don't have the money,"

or, "You're not the right class or race or sex," or whatever.

But if everybody has it, if we all can do it...

Everything is... is legal under US law, the way we're doing it.

You know, what you're really talking about is your own ownership of yourself.

You can do whatever genetics you want to do on yourself.

And that's something that we all should really work to protect.

There's... There is a long history of people experimenting on themselves.

It's not permitted, at least, in regulated environments

like universities,

but people outside of that environment

is something we don't have a lot of control over.

And I have to believe, at some point,

the FDA would intervene and say, "You're effect...

You're selling a drug, a device, whatever, without license.

Stop, cease and desist."

- It's a lot of casserole. - I think you can manage.

I kind of flattened it out so it'd cool down faster.

I gave you a spoon so it might be a little easier.

You need a spoon?

- I got him a spoon. - All right.

I'm good. How are you?

So what I have so far, it looks like,

is that Jackson could have surgery on...

the 16th of July.

Okay.

And then the second eye would be the 31st.

Okay.

Now, they're actively looking at your insurance for the drug itself.

Okay, good.

And I don't know how much they cover.

I'm kind of caught in the middle of it

because Spark is doing the money part of it for the drug.

They're trying to work all that out with people, so...

But at least we'll be in the loop here to get moving. So, okay?

- Sounds good. Thank you. - That's awesome.

Oh, you're quite welcome.

- Are you excited? - Yeah.

- Yeah. - That was the phone call.

That was the phone call we've been waiting for.

Looks like you got something on your mind.

What if it doesn't work?

- If the surgery doesn't work? - Yeah.

Well, I think we should still try it, right?

But we know that it might not work, right?

And if it doesn't, what happens?

Well...

We just... We wait.

'Cause they're doing good stuff every day.

But I have a good feeling. I think it's gonna work.

- You think it's gonna work? - Yeah.

- I think so, too. - Yeah.

- We'll stay positive, right? - Mm-hmm.

Yeah.

I know they have to detach his retina.

- And then they inject... - Eww!

...the Luxturna underneath...

the retina. They said it's...

like, literally just, like, a few drops.

They're going to remove my eye?

- No! - No!

They're not gonna remove your eye.

Ava!

- I think I got it. - Let me try it.

They're trying to still navigate

who's paying what, how much are they going to accept.

Are we gonna be on the hook for anything?

I don't know.

- I hope not. - Does it matter?

- Eh, no, but... - We're doing it no matter what.

Yeah, I know. But it's a matter of...

You know, it would be nice to know.

- Are we eating ramen noodles... - Exactly, yeah.

Can we at least have stuffed pepper casserole?

Yeah.

It would be nice to know.

But I don't know, you know...

We were denied for genetic testing.

Right.

So, who knows. You know, it's so new. Like...

For a lot of the insurance companies, it's their... their first couple patients.

You know, he could very well be this insurance company's...

Right, this isn't... Right, this isn't getting your tonsils out.

- This is a brand-new... Right. - Right.

- He could be their first patient. - Right, yeah.

Which, that in of itself is scary.

It's not comfortable.

Well, that's what it feels like when you're on a rocket, silly.

It's not comfortable!

To think that, you know,

to put him through all of this, and then it not work...

That's scary.

But then it's like, if I didn't do it, I'd kick myself in the butt, you know?

'Cause his life could be completely different

- in just a few short months. - Mm-hmm.

You know, it's like, as a parent, I'm making this decision for him.

I really want to, like...

I really want to... like...

I really want to, like...

um...

I really want him to have the surgery

because I really want him... to...

um, to see.

It... To me, it's scary.

I think it's scary.

Hi, Caroline.

Hi.

So, I have, um, good news.

Um, I just got off the phone with your insurance,

and it looks like it was the straw that broke the camel's back, so I think

we are... we're in good shape to have this kick off in March.

Okay, great.

So then I guess when we see the radiologist on Wednesday...

We can start to set up a date to get the Spinraza.

...then we'll set up a date. Okay.

Oh, Caroline, if you can, give me your boss's name and number.

I'll see if I can get you a raise, okay?

Will do.

So, what do you think of that, bro?

The same thing that I've always said.

Until I am on the table,

needle in my back,

I'm not there yet.

Okay. Well, I can tell you what, that's the best news I've heard.

I mean, hey,

if I'm the realist, you're the optimist.

Absolutely.

There's gotta be a high-low, right?

Exactly. There's got to be a high and low.

Okay, I'm with you, bro.

Without the high, there is no low.

So, I think you better get ready for the next few Wednesdays.

I will.

I'll see you later, alligator.

Adios.

My parents are fucking superheroes.

Like, I don't know how in the hell

they've done what they've done the past 24 years...

and stayed sane.

But they have.

And I, uh...

I would not trade them for the world.

Drug companies...

work to keep control of the price.

If they raise the price too high

and the pain for real people becomes too great,

they know that it could tip the balance against them.

It almost sounds like you're taking advantage of the insurance companies.

I mean, how do you justify it?

You're bringing up the concerns, uh, around access that we certainly had

as we were getting closer to the approval.

And so, we provide rebates back,

uh, if it's not showing continued efficacy out as far as three years.

The company also outlaid, uh, three sort of interesting payment models,

including a way to potentially allow payers to pay in installments.

It seems, to me, a simpler way to deal with it is just lower the price.

Bring the price down,

uh, so that we are not dealing with this complex system

where the company maintains control of the price

and maintains control of whether people are actually gonna get access to it.

This is currently the only treatment

for people with spinal muscular atrophy,

and the fact it's being denied to families based on cost is just devastating.

We will not give up.

I will not give up because I want to live.

Wake up! We want the drug fast! Wake up! We want the drug fast!

We're gonna scream! We're gonna shout!

What do we want? Spinraza now!

"We need this drug or people will die."

That's the message from families

of those living with spinal muscular atrophy.

As parents, not only are we fighting the disease,

we are now fighting for our child's right to life and right to health.

You know, for me, I think of it like in Blade Runner

where corporations are in control

of the way the world's business is conducted

and our lives are lived.

The right question we need to ask as a society

is what is the price that will maximize people's ability

to get access to and to afford these incredibly important drugs,

and companies should make a reasonable profit.

So, the N6 that you're getting now

is basically, like, phase one, in terms of our production.

It is not for human consumption, for research purposes only.

What you do with it is what you do with it.

We are dissolved of...

liability.

Makes sense.

Right now is ideal for us to move forward.

Everything that we're doing, in principle,

is kind of what the governments of the world are afraid

that people will do with gene therapy technology.

Find a way to, um...

to just modify yourself

or give access to it to other people, right?

'Cause that is the equalizer.

That's leveling the playing field for health.

That's the dream.

Just to play devil's advocate, though,

there is some risks associated to this, right?

This is an update on Ascendance Biomedical

and the experimental HIV therapy for the N6 antibody.

I am excited and...

um...

I'm personally quite all right with taking flak for being, uh...

I don't wanna say careless, but, you know...

going outside of the boundaries.

Individual liberty,

the importance of individuals to be able to make decisions for themselves,

is something that we value and we want to make sure people enjoy,

but only up to a point.

We want heroism, we want heroes,

but we really want to set up a system that doesn't demand heroism

for us to have advances.

And the system that has been set up feels too slow sometimes,

but the question is, what do you lose as you go faster?

When should the state be permitted to infringe on the liberty of individuals?

The boundary tends to be the welfare, health,

protection of others.

Oh, man, I'm exhausted.

Um...

It was a very crazy experience.

I spent all day,

nine hours,

stuck in a room with...

FBI agents...

um...

who are in the weapons of mass destruction directorate,

biological countermeasures.

And we talked about...

biohacking, and...

possible...

ramifications...

and how the government wants to treat it.

They're definitely playing the part of the federal government pretty strong.

Like, "We're here to help.

We just want you to cooperate with us

because we're trying to make everybody's lives better."

Like, "Do your part."

We need to start watching the shit we post on Facebook.

Like, I'm not mentioning names.

I didn't mention, like, you or anybody else.

- Thank you. - Here you go, guys.

It was really, really, really, really strange.

This is the story that one of the agents told.

Somebody from Iran ordered some DNA from a company.

FBI shows up, freaked out,

and they basically steamrolled the company into not sending it to Iran,

even though there was nothing wrong with it.

How does that make sense?

And I'm like, "No, that's the kind of shit I'm scared about." Right?

If I tell them, "Hey, I have customers in X country,

which I'm not gonna say..."

There's no reason for you to divulge any information.

It's like they're trying to regulate an ideology, a movement.

What happens, especially with the stuff I'm doing,

when you start getting into, like, human genetic engineering,

from stopping that dying person with cancer

from, like, trying out

- this novel DNA vaccine? - Right.

"No! Just die in... die in peace" or whatever.

"Die in peace."

"Just die, motherfucker."

Yeah. Like, you know, if the tools are there, they're gonna use it

whether they like it or not.

Which side are you gonna be on? 'Cause I know what side I'm gonna be on,

and, like, it's not on the side of the government.

The human side.

Yes, exactly. The fucking motherfucking human side.

Right. You know, I'm just worried that everything's gonna get muddled up

if one person makes one really big mistake.

And then, the whole thing...

There are just no mistakes being made. That's the problem.

Like, what can somebody do?

Can you hear me?

So, how are you feeling?

Like, you know, just generally about going through the treatment?

I'm just worried about that edge case.

If, for some reason, it keeps reproducing, and my body can't kill it,

and it causing an immune response.

Beyond that, I'm... I'm like, uh...

There's a little bit of emotion.

- Yeah. - Uh...

I'm more or less optimistic.

That's good because the emotional experience,

I'm sure, is gonna be...

rigorous, very rigorous,

especially since you're gonna be, really, the public face...

of... of this project.

You know, particularly the testing and testing it yourself,

it's, like, they're gonna come to you,

and they're gonna want to hear from you

the confidence, the strength.

Yeah, I'm with ya. I'm with ya.

Tristan is the ideal person to try this.

Is... More than...

More than probably most of the patients with HIV around the world,

he chose to challenge what was being force-fed him.

I want to say this in just the right way.

Okay? Um...

First responders always get hurt.

They don't always get hurt,

but a certain amount of first responders...

The first one through the fence always gets cut up...

a little bit, but they still keep running.

- Okay, you want a B? - B-11.

- How about a B-1? - Yeah!

That's good. Give me another one.

N... 33,

Who was 33? Was that, uh...

Jesus Christ.

Jesus Christ died when he was 33. That's a very true statement.

- Patrick Ewing. - Patrick Ewing's number, too?

Oh, they're probably gonna get...

Twenty-four years, Nick, I waited for this.

Yep.

Yeah, no. I... I honestly thought that this day was never ever gonna come.

Right.

Me, too.

- It's just a shot. - It's this little vial.

They draw it out, stick it in, and off you go.

It's amazing.

I tell you, it's one expensive shot, my friend.

Yeah... Yeah, well...

- we are talking about drug companies. - Yeah.

It's one expensive shot.

Ah.

Once the needle is in you, he's gonna draw it back.

If he gets fluid...

That's what I'm saying, yeah.

He then will disconnect...

This shot's actually going into his cord?

Yeah.

He said that to you? That he's going to stick it into his spinal cord?

I always thought that it would be some kind of an IV drip or something.

Nope.

Because that is where...

Aren't they worried about damage?

No, because that is how they do a...

A spinal tap.

Yeah, same thing.

Right.

Here we go, Nick.

So, you don't go straight in. You go in from the side.

Once you're in there, you can let go of the skin.

Right?

Yeah, the purpose of the pinching is to cause tension on your skin.

So... Like, if you didn't pinch, I've actually tried this before,

and you try to put the needle in,

your skin will just flex in against the needle

for a really long time,

'cause your skin's really stretchy.

- So, you gotta pull... - Yeah.

- Yeah, just pinch and pull. - Pull, and right there.

I'll pull out all of this, and get it nice and ready.

Yes. Plenty of syringes.

"Mix A into B, shake."

So, this one has the circle.

This one has the transfection reagent.

And you're gonna mix them.

Do you have gloves? Do you have gloves?

Just don't... don't, uh... Don't...

- Don't... - Try not to touch the inside.

Okay.

Tristan Roberts is...

self-experimenting...

Oh, it's, like, stuck at the bottom.

Okay, so you'll use the, uh, needle.

You'll use the syringe to suck it up.

...a new and unapproved treatment.

Oh, that's just a guard.

Do we need to say "unapproved"? We don't need to say "unapproved."

We'll say "experimental."

All right.

- Got it. - Okay.

Guard that.

Okay.

Ready?

He, uh, he got the dose

and then started to develop a headache.

And he was nauseous. And then he had muscular pain,

and he couldn't describe it.

And then he said, "I think my body is shutting down."

They're putting the needle into his side,

so just think about all the stuff they gotta go through

to get to that spinal cord.

It's, uh, you know, it's just...

Scary, really scary.

Yeah. Yeah.

Here we are thinking there's this great hope

and there's this medication for him.

But maybe... maybe he... maybe his body can't do it.

- He's SMA type 1. - I know.

He's already on borrowed time. He's 24.

He's not trached. He doesn't have a lot of reserve.

I don't know if we can go forward,

because if this is gonna happen again, I don't know if he can take it again.

I don't know.

I just, uh...

But hopefully we'll get out of here soon.

- I think we're gonna be out of here soon. - I hope so.

I am so happy for you guys.

He's determined.

Is everything good with the insurance now?

They're not gonna let us know until,

they're saying, like, this coming week.

- It's very surreal. - Yeah.

You know, it doesn't feel like it's actually gonna happen.

And this will be it for him? He doesn't have to do another surgery...

They expect it to last forever.

Okay. That's very cool.

So...

It really means a lot that we had all you guys

here with us and going through this entire journey with us.

Thank you for supporting us and for supporting him.

We love you, Jackson!

Do you want to say anything?

Thanks.

"Here's hoping you bounce back quickly.

Good luck, Jackson. Love, Cody and Deanna."

"King Jack, you will win this match. Danielle, Gary, and Mimi."

"Jackson, continue your quest for the stars.

Love you, Mimi and Pop-Pop.

Good luck with the surgery."

That's Jackson on the moon.

I got you.

Okay.

- Ahh. - Okay.

That's it.

That's it.

Mm-hmm.

Oh, man.

You know, it all went...

It all went bad so fast.

You know what the worst part is?

It's a very rare side effect.

I believe only a handful...

of other patients had this reaction.

Because the swelling was in my brain,

I had a very severe headache.

And I had double vision, as well.

Ma, there's a bee around me.

Being outside is more a pain in the ass than anything else.

Unfortunately, because I do enjoy it.

I have one of two choices.

SMA will kill me.

- These new drugs might... - might help me.

So, it's either lose...

or throw a Hail Mary.

I'll throw a Hail Mary every time.

There's always something new on the horizon.

Eventually something will be right for me.

I just need to be patient.

Oh boy.

This tells you, like, before surgery and then day of surgery.

There's a sample packing list.

Try to squeeze this in here.

Um, "Luxturna will be surgically injected beneath your retina

by an experienced surgical team.

The entire procedure usually takes about 90 minutes.

After surgery, a patch will be placed over your eye,

and you may be taken to a post-operative area

where you will lie on your back, facing up,

as directed by your health-care professional."

I thought he was gonna be facedown for a while.

Putting four stuffed animals in there.

Then this just goes over the scary stuff, the side effects.

We'll skip that.

Oh, God, it's heavy.

Ah.

We should make a story.

How does the story start?

- Uh... - Watch your head.

There's a little boy...

and the little boy goes to sleep and he has a dream, right?

What's he have a dream about?

He wants to be Rocky.

He's Rocky, right?

Yeah.

And he's about to have surgery.

Yeah, he's going to.

- He's nervous, and he's scared. - Yeah.

Yeah, but he climbs. He runs up the stairs.

We're gonna figure out the ending, right?

Yeah.

And we're live.

My name is Aaron Traywick.

I am the CEO of Ascendance Biomedical.

We are here with Tristan Roberts.

Um... We do not advise that anyone watching this video...

um, do what is about to be done here.

These compounds are not for human consumption.

They are for research purposes only.

Tristan Roberts is completely within his rights to, uh, self-experiment

in any way that he deems medically appropriate.

What is the biggest risk with this?

What... What risks do you see, uh, with what you're doing?

The risk is that...

it does cause some sort of immune response in my body.

But there's always risk in making progress.

You should put it down the side of the...

- Okay. - Yeah.

Oof. All right.

Got some air bubbles in there.

Are they at the bottom?

I want to dedicate this to all the people who have died

while not being able to access treatment.