Unnatural Selection (2019) s01e02 Episode Script
The First to Try
1 - [low chatter.]
- [child speaks indistinctly.]
[up-tempo music playing.]
[inaudible.]
[people chatter indistinctly.]
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.
- [father.]
Okay.
- [Jackson.]
All right, I got this.
- [father.]
You guys going? Very slowly but surely, we're going somewhere.
[Jackson grunts.]
[Mary Ann.]
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.
[child's voice echoes.]
Hi, Jackson! [Jackson.]
Hi! [Mary Ann.]
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.
- [grunts.]
You okay? [giggles.]
- Ow! Sorry about that.
[Mary Ann.]
We've been hoping, since the day we found out what he had, that something would come along and be able to help him.
- [father.]
You all right, bud? - Yeah.
- You sure? - Yeah.
See you later! [Mary Ann.]
And now there is an FDA-approved treatment.
There is some kind of hope.
- [beeping.]
- [theme music plays.]
[man 1.]
We're on the verge of a technological breakthrough that could change the future of mankind.
[man 2.]
Most people have no idea how rapidly things are changing.
[woman.]
We're now changing the DNA, the code of life.
[man 3.]
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? [man 4.]
All technology is inherently about changing away from what is natural.
[voice echoing.]
[machine whirs, beeps.]
[reporter.]
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.
[reporter.]
Is this CRISPR? [man.]
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.
[man 2.]
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.
[High.]
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.
[Marrazzo.]
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.
" [speaks indistinctly.]
[heavy metal music plays over speakers.]
- [song continues loudly over headphones.]
- Bodies multiply Too many fortunes Too many lies Where's your blood Walk into the fire [Nick.]
Oh, come on.
Fuck me.
And there it is.
[chuckles.]
Which one are you? Down here.
[woman.]
I like to see you lose when you get really mad.
- [Nick.]
I don't get mad, really.
- [chuckles.]
I get mad about when some bullshit happens.
[whispers.]
Language.
No, no filter.
- [chuckles.]
- No filter for me.
All right.
They'll need to beep out every word that I say.
[woman.]
I'll leave you to your game.
Finally.
[woman.]
"Finally.
" [rock music playing.]
[Nick.]
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.
[indistinct dialogue.]
- [younger Manny.]
Hello.
- Hello.
[younger Manny.]
How's that belly doing? How's the baby doing? - [younger Kathleen.]
Good.
- [younger Manny.]
Any kicking activity? [younger Kathleen.]
A little flutter, but not much.
[younger Kathleen.]
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.
[Kathleen.]
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.
- [coughs.]
- 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.
" [group.]
Happy birthday to you - You want to blow? One - [group.]
Happy birthday - To you - two Happy birthday Yay.
- [woman.]
Yay! - [younger Manny chuckles.]
[blows.]
- [group.]
Yay! - [clapping.]
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.
[Manny.]
So, in one aspect, he's paralyzed, but in another aspect, he still has all feeling in his body.
[Nick.]
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.
" [reporter.]
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.
[woman 1 on video.]
Use your arms.
Push up.
[woman 2.]
There you go.
You're doing it.
- [woman 1.]
Push up.
- [man on video.]
Go.
Push, baby.
Push, baby.
[woman 2 exclaims.]
- [man.]
Yeah! Good! - [women cheer.]
- Wow.
- [woman 1 continues cheering on video.]
- I cannot believe that.
- I know.
- That kid was a type 1? - Mm-hmm.
That's amazing, man.
[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.
[Kathleen.]
I know.
[Manny.]
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 [stammers.]
Exactly, in Nick's lifetime.
My understanding is, for the first year, - it's $750,000.
- [Manny.]
Right.
- And then, it's $350,000 for life.
- [Manny.]
Every year after.
- Every year after.
- [Manny.]
Right.
And insurance has not approved us yet, - so we are still fighting.
- Yeah.
- [office phone ringing.]
- [low chatter.]
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.
[reporter.]
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.
[reporter.]
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? - [interviewer chuckles.]
[Mitchell.]
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.
[Zayner.]
I think things don't change unless the medical establishment has no choice but to change.
Two big bags of moss.
- [woman.]
Two big bags of moss.
- [Zayner.]
You get that.
[woman.]
We need some containers.
[Zayner.]
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? [frog croaks.]
[Zayner.]
We're gonna pretend like somebody just received a kit in the mail, right? [woman.]
Okay.
[Zayner.]
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.
" [Zayner laughs.]
[Zayner.]
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! - [woman cheers.]
[laughs.]
Hi! I'm here today to show you how to inject a frog safely and effectively.
[clears throat.]
We are going to inject this frog with the IGF1 gene.
IGF1, what's that? IGF1 makes you grow bigger.
[woman.]
Syringes.
[Zayner.]
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.
[indistinct nearby chatter.]
[dog barks.]
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.
[Manny.]
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.
[suction whirring.]
[line ringing.]
- [man.]
You good? - Yeah.
- [phone beeps.]
- [automated.]
Please stay on the line while we connect you to our associate.
[woman.]
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.
[Brian.]
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.
- [Caroline.]
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.
[Brian.]
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.
[Caroline.]
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 - [Caroline stammers.]
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.
- [line disconnects.]
- It's ridiculous.
It's just, everywhere we go, there's, like, another roadblock for this freaking thing.
[Manny.]
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.
- [line ringing.]
[automated.]
Your call has been forwarded to an automatic voice message system.
- [voicemail beeps.]
- [Manny.]
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.
- [line disconnects.]
[Nick.]
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.
[automated voice.]
If you have a medical emergency which needs to have a doctor contacted immediately, - please press one.
- [phone beeps.]
[base rhythm thumping.]
[overlapping low chatter.]
[inaudible dialogue.]
So, we do have a test, which Yeah, yeah, so I have been optimizing.
Yeah.
Oh, yeah, I saw that picture.
[Ishee.]
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" - [man.]
Now you got that gene, you can - [Ishee.]
Yeah.
- [man.]
That's great.
- [Ishee.]
Technically [overlapping chatter.]
[Zayner over microphone.]
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.
[audience applauds.]
[Zayner.]
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.
[scattered cheers.]
[Zayner.]
Science is your hope.
Knowledge is your hope.
- I say, be your own fucking hope.
- [inaudible.]
[man.]
Roberts? - How you doing today? - Mmm, I'm doing all right.
- Deep breath.
- [inhales deeply.]
That's it.
[exhaling.]
[Roberts.]
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.
[low chatter.]
We got Aaron.
We're already on a live stream.
- Can we have you - [man.]
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.
[Traywick.]
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.
[chuckles.]
[dogs barking.]
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.
- [dogs barking.]
- Come here, girl.
[whistles.]
Pele.
[printer whirring.]
[Ishee.]
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.
[Traywick.]
Excellent.
[dogs barking.]
[Ishee.]
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 [engine starts.]
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.
[Kahn.]
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.
[stammers.]
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.
" - [Jackson.]
It's a lot of casserole.
- [father.]
I think you can manage.
[Mary Ann.]
I kind of flattened it out so it'd cool down faster.
I gave you a spoon so it might be a little easier.
- [phone ringing.]
- [father.]
You need a spoon? - [Mary Ann.]
I got him a spoon.
- All right.
I'm good.
How are you? [woman.]
So what I have so far, it looks like, is that Jackson could have surgery on the 16th of July.
[Mary Ann.]
Okay.
And then the second eye would be the 31st.
[Mary Ann.]
Okay.
Now, they're actively looking at your insurance for the drug itself.
[Mary Ann.]
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? - [Mary Ann.]
Sounds good.
Thank you.
- [father.]
That's awesome.
Oh, you're quite welcome.
- Are you excited? - [Jackson.]
Yeah.
- Yeah.
- That was the phone call.
[Mary Ann.]
That was the phone call we've been waiting for.
[father.]
Looks like you got something on your mind.
[Jackson.]
What if it doesn't work? - [father.]
If the surgery doesn't work? - Yeah.
[father.]
Well, I think we should still try it, right? But we know that it might not work, right? [Jackson.]
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.
- [father.]
I think so, too.
- Yeah.
- [father.]
We'll stay positive, right? - Mm-hmm.
Yeah.
[Mary Ann.]
I know they have to detach his retina.
- And then they inject - [Jackson.]
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! - [father laughs.]
No! They're not gonna remove your eye.
[grunts.]
Ava! - I think I got it.
- [Ava.]
Let me try it.
They're trying to still navigate who's paying what, how much are they going to accept.
[father.]
Are we gonna be on the hook for anything? [Mary Ann.]
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.
- [Mary Ann.]
Are we eating ramen noodles - Exactly, yeah.
Can we at least have stuffed pepper casserole? - [chuckles.]
- [father.]
Yeah.
It would be nice to know.
But I don't know, you know We were denied for genetic testing.
[father.]
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.
[Ava.]
It's not comfortable.
[Jackson.]
Well, that's what it feels like when you're on a rocket, silly.
[Ava.]
It's not comfortable! [Mary Ann, voice quavering.]
To think that, you know, to put him through all of this, and then it not work [sniffles.]
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.
[sighs.]
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.
[Kathleen.]
Hi, Caroline.
[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? [Caroline laughs.]
Will do.
[door opens.]
[Manny.]
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.
- [Manny laughs.]
Absolutely.
There's gotta be a high-low, right? Exactly.
There's got to be a high and low.
[Manny.]
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.
[stammers.]
I will.
I'll see you later, alligator.
Adios.
[Nick.]
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.
[reporter.]
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.
[woman.]
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.
[crowd chanting.]
[woman in Croatian.]
We will not give up.
I will not give up because I want to live.
[chanting.]
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! [reporter.]
"We need this drug or people will die.
" That's the message from families of those living with spinal muscular atrophy.
[voice quavering.]
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.
[texting.]
[Traywick.]
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.
[Roberts.]
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.
[chuckles.]
Just to play devil's advocate, though, there is some risks associated to this, right? [Roberts.]
This is an update on Ascendance Biomedical and the experimental HIV therapy for the N6 antibody.
[Roberts over video.]
I am excited and um [stammers.]
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.
[Kahn.]
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.
[helicopter whirring.]
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.
" [chuckles.]
[Zayner.]
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.
[Zayner.]
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.
[man chuckles.]
"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.
[man.]
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? [taps echoing.]
[ringtone plays.]
[Roberts.]
Can you hear me? [Traywick.]
So, how are you feeling? Like, you know, just generally about going through the treatment? [Roberts.]
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.
[chuckles.]
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.
[chuckles.]
Okay? Um First responders always get hurt.
They don't always get hurt, but a certain amount of first responders [clicks tongue, takes a deep breath.]
The first one through the fence always gets cut up a little bit, but they still keep running.
- [Manny.]
Okay, you want a B? - [Kathleen.]
B-11.
- [Manny.]
How about a B-1? - [multiple voices.]
Yeah! - That's good.
Give me another one.
- [balls clattering.]
N 33, Who was 33? Was that, uh - [Kathleen.]
Jesus Christ.
- [indistinct chatter.]
Jesus Christ died when he was 33.
That's a very true statement.
- [woman.]
Patrick Ewing.
- Patrick Ewing's number, too? [Kathleen.]
Oh, they're probably gonna get Twenty-four years, Nick, I waited for this.
[Nick.]
Yep.
[Manny.]
Yeah, no.
I I honestly thought that this day was never ever gonna come.
[Kathleen.]
Right.
[Nick.]
Me, too.
- It's just a shot.
- [Kathleen.]
It's this little vial.
They draw it out, stick it in, and off you go.
[man.]
It's amazing.
I tell you, it's one expensive shot, my friend.
[man.]
Yeah Yeah, well - we are talking about drug companies.
- [Manny.]
Yeah.
- [all chuckling.]
- [Manny.]
It's one expensive shot.
[radio coverage of baseball game plays indistinctly.]
Ah.
[Kathleen.]
Once the needle is in you, he's gonna draw it back.
If he gets fluid [Nick.]
That's what I'm saying, yeah.
[Kathleen.]
He then will disconnect [Manny.]
This shot's actually going into his cord? [Nick.]
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 [Manny.]
Aren't they worried about damage? [Nick.]
No, because that is how they do a A spinal tap.
[Nick.]
Yeah, same thing.
Right.
[radio coverage of baseball game plays indistinctly.]
[Kathleen and Nick speak indistinctly.]
[car beeps.]
[Kathleen.]
Here we go, Nick.
[Traywick.]
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 - [Traywick.]
Yeah.
- [Davis.]
Yeah, just pinch and pull.
- [Roberts.]
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.
" [Davis.]
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? - [inhales sharply.]
- [Davis.]
Just don't don't, uh Don't - Don't - [Davis.]
Try not to touch the inside.
Okay.
Tristan Roberts is self-experimenting Oh, it's, like, stuck at the bottom.
[Davis.]
Okay, so you'll use the, uh, needle.
You'll use the syringe to suck it up.
[Traywick.]
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.
" - [laughs.]
All right.
- Got it.
- [Davis.]
Okay.
[Roberts.]
Guard that.
[man.]
Okay.
Ready? [Manny.]
He, uh, he got the dose and then started to develop a headache.
[Kathleen.]
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.
[Kathleen.]
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.
[quietly.]
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.
[air hissing.]
[machine whirring.]
[water running.]
[Nick grunts.]
[vibrating groans.]
[machine beeps.]
[children shouting, chattering.]
[woman.]
I am so happy for you guys.
He's determined.
[man.]
Is everything good with the insurance now? [sighs.]
They're not gonna let us know until, they're saying, like, this coming week.
[children chatting.]
- [Mary Ann.]
It's very surreal.
- [woman.]
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 [Mary Ann.]
They expect it to last forever.
Okay.
That's very cool.
So [Mary Ann.]
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.
[cheering.]
[woman.]
We love you, Jackson! [Mary Ann and father.]
Do you want to say anything? - Thanks.
- [applause.]
[father chuckling.]
[Mary Ann.]
"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.
[speaks indistinctly.]
- Ahh.
- [Manny.]
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.
[sighs.]
Ma, there's a bee around me.
[grunts.]
Being outside is more a pain in the ass than anything else.
Unfortunately, because I do enjoy it.
[grunting breath.]
I have one of two choices.
SMA will kill me.
These new drugs might [stammers.]
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.
[Mary Ann.]
Oh boy.
This tells you, like, before surgery and then day of surgery.
There's a sample packing list.
[Jackson.]
Try to squeeze this in here.
[Mary Ann.]
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.
[grunts.]
Ah.
[Jackson.]
We should make a story.
[father.]
How does the story start? - [Jackson.]
Uh - [Mary Ann.]
Watch your head.
[father.]
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? [Ava giggles.]
[Jackson.]
He wants to be Rocky.
[father.]
He's Rocky, right? [Jackson.]
Yeah.
[father.]
And he's about to have surgery.
[Jackson.]
Yeah, he's going to.
- [father.]
He's nervous, and he's scared.
- [Jackson.]
Yeah.
[father.]
Yeah, but he climbs.
He runs up the stairs.
We're gonna figure out the ending, right? [Jackson.]
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.
[man.]
What is the biggest risk with this? What What risks do you see, uh, with what you're doing? [Roberts stammers.]
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 - [Roberts.]
Okay.
- Yeah.
Oof.
All right.
Got some air bubbles in there.
Are they at the bottom? [Roberts.]
I want to dedicate this to all the people who have died while not being able to access treatment.
[Roberts exhales.]
[theme music playing.]
- [child speaks indistinctly.]
[up-tempo music playing.]
[inaudible.]
[people chatter indistinctly.]
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.
- [father.]
Okay.
- [Jackson.]
All right, I got this.
- [father.]
You guys going? Very slowly but surely, we're going somewhere.
[Jackson grunts.]
[Mary Ann.]
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.
[child's voice echoes.]
Hi, Jackson! [Jackson.]
Hi! [Mary Ann.]
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.
- [grunts.]
You okay? [giggles.]
- Ow! Sorry about that.
[Mary Ann.]
We've been hoping, since the day we found out what he had, that something would come along and be able to help him.
- [father.]
You all right, bud? - Yeah.
- You sure? - Yeah.
See you later! [Mary Ann.]
And now there is an FDA-approved treatment.
There is some kind of hope.
- [beeping.]
- [theme music plays.]
[man 1.]
We're on the verge of a technological breakthrough that could change the future of mankind.
[man 2.]
Most people have no idea how rapidly things are changing.
[woman.]
We're now changing the DNA, the code of life.
[man 3.]
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? [man 4.]
All technology is inherently about changing away from what is natural.
[voice echoing.]
[machine whirs, beeps.]
[reporter.]
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.
[reporter.]
Is this CRISPR? [man.]
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.
[man 2.]
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.
[High.]
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.
[Marrazzo.]
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.
" [speaks indistinctly.]
[heavy metal music plays over speakers.]
- [song continues loudly over headphones.]
- Bodies multiply Too many fortunes Too many lies Where's your blood Walk into the fire [Nick.]
Oh, come on.
Fuck me.
And there it is.
[chuckles.]
Which one are you? Down here.
[woman.]
I like to see you lose when you get really mad.
- [Nick.]
I don't get mad, really.
- [chuckles.]
I get mad about when some bullshit happens.
[whispers.]
Language.
No, no filter.
- [chuckles.]
- No filter for me.
All right.
They'll need to beep out every word that I say.
[woman.]
I'll leave you to your game.
Finally.
[woman.]
"Finally.
" [rock music playing.]
[Nick.]
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.
[indistinct dialogue.]
- [younger Manny.]
Hello.
- Hello.
[younger Manny.]
How's that belly doing? How's the baby doing? - [younger Kathleen.]
Good.
- [younger Manny.]
Any kicking activity? [younger Kathleen.]
A little flutter, but not much.
[younger Kathleen.]
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.
[Kathleen.]
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.
- [coughs.]
- 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.
" [group.]
Happy birthday to you - You want to blow? One - [group.]
Happy birthday - To you - two Happy birthday Yay.
- [woman.]
Yay! - [younger Manny chuckles.]
[blows.]
- [group.]
Yay! - [clapping.]
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.
[Manny.]
So, in one aspect, he's paralyzed, but in another aspect, he still has all feeling in his body.
[Nick.]
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.
" [reporter.]
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.
[woman 1 on video.]
Use your arms.
Push up.
[woman 2.]
There you go.
You're doing it.
- [woman 1.]
Push up.
- [man on video.]
Go.
Push, baby.
Push, baby.
[woman 2 exclaims.]
- [man.]
Yeah! Good! - [women cheer.]
- Wow.
- [woman 1 continues cheering on video.]
- I cannot believe that.
- I know.
- That kid was a type 1? - Mm-hmm.
That's amazing, man.
[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.
[Kathleen.]
I know.
[Manny.]
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 [stammers.]
Exactly, in Nick's lifetime.
My understanding is, for the first year, - it's $750,000.
- [Manny.]
Right.
- And then, it's $350,000 for life.
- [Manny.]
Every year after.
- Every year after.
- [Manny.]
Right.
And insurance has not approved us yet, - so we are still fighting.
- Yeah.
- [office phone ringing.]
- [low chatter.]
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.
[reporter.]
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.
[reporter.]
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? - [interviewer chuckles.]
[Mitchell.]
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.
[Zayner.]
I think things don't change unless the medical establishment has no choice but to change.
Two big bags of moss.
- [woman.]
Two big bags of moss.
- [Zayner.]
You get that.
[woman.]
We need some containers.
[Zayner.]
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? [frog croaks.]
[Zayner.]
We're gonna pretend like somebody just received a kit in the mail, right? [woman.]
Okay.
[Zayner.]
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.
" [Zayner laughs.]
[Zayner.]
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! - [woman cheers.]
[laughs.]
Hi! I'm here today to show you how to inject a frog safely and effectively.
[clears throat.]
We are going to inject this frog with the IGF1 gene.
IGF1, what's that? IGF1 makes you grow bigger.
[woman.]
Syringes.
[Zayner.]
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.
[indistinct nearby chatter.]
[dog barks.]
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.
[Manny.]
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.
[suction whirring.]
[line ringing.]
- [man.]
You good? - Yeah.
- [phone beeps.]
- [automated.]
Please stay on the line while we connect you to our associate.
[woman.]
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.
[Brian.]
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.
- [Caroline.]
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.
[Brian.]
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.
[Caroline.]
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 - [Caroline stammers.]
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.
- [line disconnects.]
- It's ridiculous.
It's just, everywhere we go, there's, like, another roadblock for this freaking thing.
[Manny.]
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.
- [line ringing.]
[automated.]
Your call has been forwarded to an automatic voice message system.
- [voicemail beeps.]
- [Manny.]
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.
- [line disconnects.]
[Nick.]
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.
[automated voice.]
If you have a medical emergency which needs to have a doctor contacted immediately, - please press one.
- [phone beeps.]
[base rhythm thumping.]
[overlapping low chatter.]
[inaudible dialogue.]
So, we do have a test, which Yeah, yeah, so I have been optimizing.
Yeah.
Oh, yeah, I saw that picture.
[Ishee.]
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" - [man.]
Now you got that gene, you can - [Ishee.]
Yeah.
- [man.]
That's great.
- [Ishee.]
Technically [overlapping chatter.]
[Zayner over microphone.]
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.
[audience applauds.]
[Zayner.]
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.
[scattered cheers.]
[Zayner.]
Science is your hope.
Knowledge is your hope.
- I say, be your own fucking hope.
- [inaudible.]
[man.]
Roberts? - How you doing today? - Mmm, I'm doing all right.
- Deep breath.
- [inhales deeply.]
That's it.
[exhaling.]
[Roberts.]
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.
[low chatter.]
We got Aaron.
We're already on a live stream.
- Can we have you - [man.]
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.
[Traywick.]
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.
[chuckles.]
[dogs barking.]
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.
- [dogs barking.]
- Come here, girl.
[whistles.]
Pele.
[printer whirring.]
[Ishee.]
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.
[Traywick.]
Excellent.
[dogs barking.]
[Ishee.]
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 [engine starts.]
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.
[Kahn.]
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.
[stammers.]
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.
" - [Jackson.]
It's a lot of casserole.
- [father.]
I think you can manage.
[Mary Ann.]
I kind of flattened it out so it'd cool down faster.
I gave you a spoon so it might be a little easier.
- [phone ringing.]
- [father.]
You need a spoon? - [Mary Ann.]
I got him a spoon.
- All right.
I'm good.
How are you? [woman.]
So what I have so far, it looks like, is that Jackson could have surgery on the 16th of July.
[Mary Ann.]
Okay.
And then the second eye would be the 31st.
[Mary Ann.]
Okay.
Now, they're actively looking at your insurance for the drug itself.
[Mary Ann.]
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? - [Mary Ann.]
Sounds good.
Thank you.
- [father.]
That's awesome.
Oh, you're quite welcome.
- Are you excited? - [Jackson.]
Yeah.
- Yeah.
- That was the phone call.
[Mary Ann.]
That was the phone call we've been waiting for.
[father.]
Looks like you got something on your mind.
[Jackson.]
What if it doesn't work? - [father.]
If the surgery doesn't work? - Yeah.
[father.]
Well, I think we should still try it, right? But we know that it might not work, right? [Jackson.]
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.
- [father.]
I think so, too.
- Yeah.
- [father.]
We'll stay positive, right? - Mm-hmm.
Yeah.
[Mary Ann.]
I know they have to detach his retina.
- And then they inject - [Jackson.]
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! - [father laughs.]
No! They're not gonna remove your eye.
[grunts.]
Ava! - I think I got it.
- [Ava.]
Let me try it.
They're trying to still navigate who's paying what, how much are they going to accept.
[father.]
Are we gonna be on the hook for anything? [Mary Ann.]
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.
- [Mary Ann.]
Are we eating ramen noodles - Exactly, yeah.
Can we at least have stuffed pepper casserole? - [chuckles.]
- [father.]
Yeah.
It would be nice to know.
But I don't know, you know We were denied for genetic testing.
[father.]
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.
[Ava.]
It's not comfortable.
[Jackson.]
Well, that's what it feels like when you're on a rocket, silly.
[Ava.]
It's not comfortable! [Mary Ann, voice quavering.]
To think that, you know, to put him through all of this, and then it not work [sniffles.]
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.
[sighs.]
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.
[Kathleen.]
Hi, Caroline.
[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? [Caroline laughs.]
Will do.
[door opens.]
[Manny.]
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.
- [Manny laughs.]
Absolutely.
There's gotta be a high-low, right? Exactly.
There's got to be a high and low.
[Manny.]
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.
[stammers.]
I will.
I'll see you later, alligator.
Adios.
[Nick.]
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.
[reporter.]
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.
[woman.]
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.
[crowd chanting.]
[woman in Croatian.]
We will not give up.
I will not give up because I want to live.
[chanting.]
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! [reporter.]
"We need this drug or people will die.
" That's the message from families of those living with spinal muscular atrophy.
[voice quavering.]
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.
[texting.]
[Traywick.]
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.
[Roberts.]
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.
[chuckles.]
Just to play devil's advocate, though, there is some risks associated to this, right? [Roberts.]
This is an update on Ascendance Biomedical and the experimental HIV therapy for the N6 antibody.
[Roberts over video.]
I am excited and um [stammers.]
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.
[Kahn.]
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.
[helicopter whirring.]
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.
" [chuckles.]
[Zayner.]
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.
[Zayner.]
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.
[man chuckles.]
"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.
[man.]
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? [taps echoing.]
[ringtone plays.]
[Roberts.]
Can you hear me? [Traywick.]
So, how are you feeling? Like, you know, just generally about going through the treatment? [Roberts.]
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.
[chuckles.]
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.
[chuckles.]
Okay? Um First responders always get hurt.
They don't always get hurt, but a certain amount of first responders [clicks tongue, takes a deep breath.]
The first one through the fence always gets cut up a little bit, but they still keep running.
- [Manny.]
Okay, you want a B? - [Kathleen.]
B-11.
- [Manny.]
How about a B-1? - [multiple voices.]
Yeah! - That's good.
Give me another one.
- [balls clattering.]
N 33, Who was 33? Was that, uh - [Kathleen.]
Jesus Christ.
- [indistinct chatter.]
Jesus Christ died when he was 33.
That's a very true statement.
- [woman.]
Patrick Ewing.
- Patrick Ewing's number, too? [Kathleen.]
Oh, they're probably gonna get Twenty-four years, Nick, I waited for this.
[Nick.]
Yep.
[Manny.]
Yeah, no.
I I honestly thought that this day was never ever gonna come.
[Kathleen.]
Right.
[Nick.]
Me, too.
- It's just a shot.
- [Kathleen.]
It's this little vial.
They draw it out, stick it in, and off you go.
[man.]
It's amazing.
I tell you, it's one expensive shot, my friend.
[man.]
Yeah Yeah, well - we are talking about drug companies.
- [Manny.]
Yeah.
- [all chuckling.]
- [Manny.]
It's one expensive shot.
[radio coverage of baseball game plays indistinctly.]
Ah.
[Kathleen.]
Once the needle is in you, he's gonna draw it back.
If he gets fluid [Nick.]
That's what I'm saying, yeah.
[Kathleen.]
He then will disconnect [Manny.]
This shot's actually going into his cord? [Nick.]
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 [Manny.]
Aren't they worried about damage? [Nick.]
No, because that is how they do a A spinal tap.
[Nick.]
Yeah, same thing.
Right.
[radio coverage of baseball game plays indistinctly.]
[Kathleen and Nick speak indistinctly.]
[car beeps.]
[Kathleen.]
Here we go, Nick.
[Traywick.]
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 - [Traywick.]
Yeah.
- [Davis.]
Yeah, just pinch and pull.
- [Roberts.]
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.
" [Davis.]
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? - [inhales sharply.]
- [Davis.]
Just don't don't, uh Don't - Don't - [Davis.]
Try not to touch the inside.
Okay.
Tristan Roberts is self-experimenting Oh, it's, like, stuck at the bottom.
[Davis.]
Okay, so you'll use the, uh, needle.
You'll use the syringe to suck it up.
[Traywick.]
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.
" - [laughs.]
All right.
- Got it.
- [Davis.]
Okay.
[Roberts.]
Guard that.
[man.]
Okay.
Ready? [Manny.]
He, uh, he got the dose and then started to develop a headache.
[Kathleen.]
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.
[Kathleen.]
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.
[quietly.]
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.
[air hissing.]
[machine whirring.]
[water running.]
[Nick grunts.]
[vibrating groans.]
[machine beeps.]
[children shouting, chattering.]
[woman.]
I am so happy for you guys.
He's determined.
[man.]
Is everything good with the insurance now? [sighs.]
They're not gonna let us know until, they're saying, like, this coming week.
[children chatting.]
- [Mary Ann.]
It's very surreal.
- [woman.]
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 [Mary Ann.]
They expect it to last forever.
Okay.
That's very cool.
So [Mary Ann.]
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.
[cheering.]
[woman.]
We love you, Jackson! [Mary Ann and father.]
Do you want to say anything? - Thanks.
- [applause.]
[father chuckling.]
[Mary Ann.]
"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.
[speaks indistinctly.]
- Ahh.
- [Manny.]
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.
[sighs.]
Ma, there's a bee around me.
[grunts.]
Being outside is more a pain in the ass than anything else.
Unfortunately, because I do enjoy it.
[grunting breath.]
I have one of two choices.
SMA will kill me.
These new drugs might [stammers.]
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.
[Mary Ann.]
Oh boy.
This tells you, like, before surgery and then day of surgery.
There's a sample packing list.
[Jackson.]
Try to squeeze this in here.
[Mary Ann.]
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.
[grunts.]
Ah.
[Jackson.]
We should make a story.
[father.]
How does the story start? - [Jackson.]
Uh - [Mary Ann.]
Watch your head.
[father.]
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? [Ava giggles.]
[Jackson.]
He wants to be Rocky.
[father.]
He's Rocky, right? [Jackson.]
Yeah.
[father.]
And he's about to have surgery.
[Jackson.]
Yeah, he's going to.
- [father.]
He's nervous, and he's scared.
- [Jackson.]
Yeah.
[father.]
Yeah, but he climbs.
He runs up the stairs.
We're gonna figure out the ending, right? [Jackson.]
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.
[man.]
What is the biggest risk with this? What What risks do you see, uh, with what you're doing? [Roberts stammers.]
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 - [Roberts.]
Okay.
- Yeah.
Oof.
All right.
Got some air bubbles in there.
Are they at the bottom? [Roberts.]
I want to dedicate this to all the people who have died while not being able to access treatment.
[Roberts exhales.]
[theme music playing.]