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Few minutes now.
I get to say something that I don't get to say
often enough.
Good evening, Seattle.
I seem to recognize your face
Haunting, familiar
Yet I can't seem to place it
Cannot find the candle of thought
To light your name
Oh, lifetimes, they're catching up
With me
All these changes taking place
I wish I'd seen the place
But no one's ever taken me
Hearts and thoughts, they fade
Fade away
Oh, hearts and thoughts they fade
Fade away
I swear I recognize your breath
Oh, memories like fingerprints
Yeah, they're slowly raising
Me, you wouldn't recall
For I'm not my former
It's hard when You're stuck upon the shelf
I changed by not changin' at all
Small town predicts my fate
Perhaps that's what No one wants to see
Oh, I just want to scream
Hello
My God, it's been too long
Never dreamed you'd return
But now here you are
And here I am
Hearts and thoughts, they fade
Away
Hearts and thoughts, they fade
Away, away
Hearts and thoughts, they fade
Away, away
Hearts and thoughts, they fade
Away, away
Hearts and thoughts, they fade
Away, away, oh
Hearts and thoughts, they fade
Away
Hearts and thoughts, they fade
Fade away
This week, we have a particularly special event
happening in Seattle for a truly important cause.
You can feel the excitement building
and some hometown pride as Eddie Vedder is taking the stage
for a one-of-a-kind show at Benaroya Hall.
All proceeds from the concert will go to finding a cure for epidermolysis bullosa,
a rare genetic skin condition
that mostly affects children.
I'm falling harder Than I ever fell before…
We're bringing patients and researchers
and people from the biotech world all together
to talk about how we can cure EB,
which is totally happening.
Longing to belong
To you
The global community of scientists, researchers, patients
are focused on one big goal,
which is to cure EB by the end of this decade.
So when you have an Eddie Vedder that steps up to the plate and says,
"I'm gonna play massive shows for this cause,"
it means the world to these families,
it means the world to those that battle rare disease because they feel seen.
We've been involved in all sorts of things,
and haven't cured cancer yet, can't fix the planet.
I don't know, this is the one thing that seems like, "Oh, this one's gonna work."
Out of all the things we care about, this is gonna be the one.
-Hello. -Hello.
I think of things like curing a disease,
this is like an idea that's gonna take two or three lifetimes.
You know, maybe your kid's generation,
they'll get to see the end of something that you started.
But we're selfish, we want to see it in our lifetimes.
And I think it can happen.
So coming together in Seattle really is about hope.
Surely it will be an unforgettable night with families, researchers, and fans
knowing that every note played brings us closer to a world without EB.
Longing to belong to you
-Thanks for doing this. -Yeah.
We really appreciate it. What are you looking at?
These areas look like they would fill in the patient's wound sites well.
-This is essentially one week. -Wow.
Epidermolysis bullosa, or EB, is a terrible disease,
but it's simple to explain.
It's mutations in one gene,
and that gene encodes a protein that connects the top layer of your skin
to the bottom layer of your skin, called collagen VII,
to basically make a staple.
The staple is called an anchoring fibril,
and that's what keeps our skin intact.
And if any one of those links in the chain is broken, your skin doesn't stay on.
Any alteration in the DNA that affects that link
weakens the glue and the glue can fall apart.
And there's a wide range of, you know, the glue sticks a little bit,
the glue sticks a lot, the glue doesn't stick at all.
Epidermolysis means "breakage."
Bullosa is the Latin word for "blister,"
so epidermolysis bullosa basically means fragile, blistering skin.
It's actually called "butterfly disease,"
and kids with EB are called "butterfly children"
because their skin is as fragile as a butterfly's wings.
And you're born with EB.
Oftentimes, parents have never heard the words "EB"
until they have a child delivered in the emergency room
and they realize something is different.
Day-to-day life for a kid suffering with EB is brutal and traumatic.
They have to endure hours-long bandage changes,
bleach baths which help soak off the bandages, but also sterilize.
And parents actually have to inflict pain on their kids to keep them healthy.
The dressing and the gauze serves as a cocoon for them.
Preventing infection and hoping they don't scar,
trying to keep the itch and the pain to a minimum.
Nothing is treating the underlying disease.
All you're doing is wrapping up the patient,
trying to protect them from friction or trauma,
but you're doing nothing for the disease.
It's not just the wounds that you see.
Collagen is produced all over your body.
It makes your eyes work, your throat work, and your internal organs.
So EB is much larger than a problem of the skin.
Even in their stomach lining and intestinal lining,
they can't absorb proper nutrition.
It can take scientists lifetimes or generations
to decode the complex genomics of any particular disease.
What gives us hope for EB is that we have pinpointed the gene mutation
that causes EB down to one gene.
Now we're getting to the underlying problem,
the genetic cause, which is so exciting and important.
This is a situation, honestly,
where the science is relatively straightforward.
So how exciting is that?
Where we know the gene, we know the problem areas,
we have the scientists who are excited about it.
We're shrinking the timeline so it's faster,
and so we can bring the therapies and hopefully a cure much faster.
EB has often been referred to as, "The worst disease you've never heard of."
We like to think of it now as,
"The worst disease we will find a cure for."
The energy in the community is palpable.
And I think because there's a trajectory,
and because there is a target,
you just can't help but be moved and inspired.
This was an issue that we thought we could be involved for a year or two,
maybe three.
Raise some funds, get some people who really cared about it,
and then, you know, move on to the next, and…
But it's kept us right in the middle
because this is so important.
And, you know, the goal is treatments.
The goal, ultimately, is cure.
And we're all lifting each other up.
And to be part of that for the last ten years, it brings a sense of hope.
And it's not false hope.
What time do you want us at your house to load everything out of the cars?
What time do we have to be at the set? 5:00 a.m., right?
Is that, like, load-in is pretty much 5:00 a.m. at Benaroya?
Yeah, we have to start loading in, do sound checks,
get the speakers brief, test the slides.
We need to be ready for family arrivals.
We've got the Hollers coming in from Colorado
and Deanna's gonna arrive from Toronto shortly after.
And Eli and family will get in the next morning.
Please, I'm gonna ask the string quartet to come join me.
Ladies and gentlemen, on the violin, Miss Brianna Atwell.
Yeah.
Also on violin, Sada Doup.
My friend on the cello, Chris Worswick.
And one of the people on the planet I respect most,
my friend on viola, April Cameron.
We've never played this one.
Not practiced, not nothing.
Walk down the street Can't find the keys to my own home
I'll take a walk So I could curse my ass for bein' dumb
I make a right after the arches Stinking grease and bone
Up at the supermarket People stare like I'm a dog
I'm goin' to Lukin's
I got a spot at Lukin's
I knocked the door at Lukin's
Open the fridge Now I know life is worth
I found my key But I return to find an open door
Some fuckin' freak who claims I fathered By rape, her own son
I take a walk, I call the cops This days work's never done
The last I heard That freak was purchasin' a fuckin' gun
Guys, how's the packing going? You know we have to go in a couple hours.
Can you guys start packing? Please?
-Okay. -Okay, where's your list?
Two pencils and erasers, a sharpener,
and coloring kit, all so I can color on the plane.
There's a lot of work that goes into packing for Eli.
He has tons of supplies that have to go with us.
I always get nervous when we fly 'cause we have to go through security,
and they're gonna be like, "What's all this?"
"Why do you have needles and scissors in your bag?" All that.
All of his bandages that we use for his bath and bandage change.
G-tube supplies, like all his formula, and lots of gauze and needles.
-And then, like, toothbrush. -Yeah.
-Please don't forget your toothbrushes. -All right, fine, dental hygiene.
This will go in my carry-on in case Eli gets hurt on the plane
or at the airport.
He's always bleeding all the time everywhere, so it's good to have backup.
We are in Seattle,
and we traveled from Colorado.
Um, 20-minute drive to the airport,
and then a two-hour flight to Seattle.
Yeah.
I'm here with my mom and daddy, and my baby sister Celine.
And my mom's pregnant, so she's gonna have a little baby brother.
I mean, I'm just excited that we're here,
and all of our things have made it here.
So this is gonna be such a fun experience,
and meeting all of these really amazing people at the summit.
I'm super looking forward to people with EB,
but then also the doctors and researchers.
Let's do it.
-Okay, you good? -Yep.
I'm speaking at a summit this weekend.
We're going to Seattle for that. I'm excited.
-Do you need the cushions, Deanna? -Oh, I can sit. No, it's fine.
I'll put them under my feet.
That's good. Thank you.
We're good.
All right.
It's really easy to get sick, or even catch an infection from flying,
'cause it's recycled air.
That's something that I'm always cautious about.
I'm excited, though.
Excited to speak at the conference, and talk to doctors,
and meet everyone.
Backpack, backpack.
We always forget.
'Cause I'm not used to having this all the time.
Sometimes I get lifted out of the chair, and we forget, and we're like, "No!"
And like, I'm like, attached.
Why is it all…
I don't know, Dad. This is hot. Just throw it on.
-You good? -Yeah, I'm good. Just watch my head.
-I'll watch your head. -And yours.
Don't flash my ass. There's a camera.
I want
To be a good woman
And I want
For you to be a good man
And this is why
I'll be leaving
And this is why
I can't see you no more…
I was born in Taiwan,
and then my family immigrated to the US for a better life.
When I was a kid, I remember reading National Geographic,
learning about HIV and AIDS, sickle cell disease.
The other kids may have liked Britney Spears, Christina Aguilera.
And I really loved genetic disease and medicine.
Um, and I was a nerd all the way through,
so I haven't been to many rock concerts.
I got my talk ready. I got my slides.
Really the most important thing of my luggage is my phone,
my computer, and charger.
I just feel like, yeah, whatever I wear, it doesn't really matter,
'cause I'm always the scientist, and nobody really pays attention to that.
I love a party.
And I love celebrating the kids, and the families,
and everything they've done.
Um, you know, so much of what we do is clinical trials
or taking care of EB patients
when something's going wrong, to just hang out with them in a happy occasion.
I can't see you no more
And this is why
I am lying
When I say
That I don't love you no more
'Cause I want
To be a good woman
And I want
For you to be a good man
We have three biological children,
and a 17-year-old son, Charlie.
And then Meili, and she just turned 12.
Nobody can possibly understand
what it feels like to be a mother to a child with EB.
It's like watching a toddler just learning to walk,
and you're afraid they're gonna hit their face on the coffee table.
That's how it feels watching your child
with EB at any age.
EB families, we have our groups on Facebook,
and we support each other,
but meeting in person is important to me.
It's comforting to not feel alone.
I know, it feels good, 'cause I'm cold.
So, the patient panel, we've got Deanna, Hodges.
How's Deanna feeling about speaking?
She's ready. I think we just gotta get out of her way, let her do her thing.
Yeah.
I'm just hoping everything goes smooth and everyone arrives safely.
And I'm so excited just to… that this is happening.
How long have we been talking about this? We did it. We're making it happen.
You know, it's interesting.
There's a scientific theory called "the butterfly effect."
And what it means is that one small initiative of motion
can create a tidal wave of motion at a later state.
And in a lot of ways, I believe the progression
and transformation of the EB community and EBRP
has a lot to do with the butterfly effect.
Before this organization started,
no treatments, no science,
no options, little hope.
Ten short years later,
we've gone from zero to 40 clinical trials.
We've raised $60,000,000.
We've funded 140 projects.
And for the first time ever, we now have the first FDA-approved treatment.
Why is that significant?
There's 400,000,000 people on the planet with a rare disease,
one in ten people.
95% of people with a rare disease have zero approved treatments.
Because of the dedication and hard work of this organization,
we crossed into the 5%.
But we're just getting warmed up.
Doing things the old way is just not gonna cut it.
Rare disease needs bold moves to shake things up.
And I feel like we're doing that.
We're finding new ways to get results and we've been pushing boundaries,
and that's how you make real progress that can actually change lives.
We're hopeful that the model and approach that we're using to tackle EB, um,
could extend on to curing other rare diseases
like Huntington's and cystic fibrosis and Tay-Sachs.
After we cure EB,
which I'm super hopeful will happen, we can just move on to the next one.
The power of a community all focused on the same dream is powerful and moving.
We're super driven.
I think we're gonna put an end to it.
That's my hope, anyways.
When I first learned about EB, and when I first met my friend's…
my friend's child that's affected, I…
we were just like,
"This is the most crazy, intense, brutal thing I've ever seen."
And, "What can we do?"
And, like, "Okay, we can maybe talk about this and see…"
You know, "Just start small and see what happens."
And I meet people on the street, like…
You know, on the street, or when I go to shows,
when they're playing, and there's always an awesome Pearl Jam fan
that will come up to me and be like,
"God, I love what you guys are doing with…"
That's gonna make me cry. "…for the kids with EB."
The closer you get to this community,
and the more you understand it,
you cannot escape how difficult the challenges are for these families.
We've had to clear some hurdles already
that are examples of why you can have faith,
and why you can have hope.
But, you know, the tricky part is patience.
If raising funds and raising awareness…
if those things are able to fuel the process
on the scientific end of finding a cure and how to apply it,
it feels like just a matter of time.
Drifting, drifting, drifting away
I got myself a mansion Then I gave it away
It's not the world that's heavy It's just the things that you save
I'm drifting, drifting away
Drifting, drifting, drifting
I rid myself of worries And my worries were gone
I only run when I want to And I sleep like a dog
I'm drifting, drifting along…
Let's do… Uh, no, it's not in good.
-How do you want it? -Just a bit further in.
There we go.
Yeah. Yeah, we're good, okay.
My family and I, especially me and my mom,
we have a really close-knit relationship.
Having that is something that has made me so positive in life.
I'm fortunate to have people that care for me
and are willing to adapt their lives to help me live my own.
I don't use makeup to mask my EB,
but I use it to provide self-confidence,
and kind of give me that armor
that I need to stand up for myself.
I was born in '92.
As a child, I wasn't able to crawl because my knees would break down
and my hands would break down.
So I actually was able to learn how to walk quite quickly.
I did fairly well in school.
As a teenager, it was tough.
I definitely tried to protect myself.
I think the confidence that I had in my own self made a big difference.
I've always loved art.
I remember just drawing as a kid
because I couldn't build with Lego blocks.
I couldn't pull them apart. I couldn't stack them well.
So my parents giving me crayons and paper,
it was just something that I found I could occupy myself with for hours.
I tend to blend realism and surrealism in my work.
Just like crazy and like silly things
and things that make you think twice
about what you're looking at.
EB has taken a lot of things.
My ability to walk, my arm.
I tried different prosthesis,
and because of my skin fragility, it was a no-go, it didn't work out for me.
It was kind of the point where,
"Do I keep damaging my skin with this
or do I just learn to live without it?" kind of thing.
For me, my approach to life would be to live'
in each moment to the fullest
and not dwell on the things that you can't change.
-It'd be kind of cool if Charlie was here. -Yeah.
-What do you think he would be doing? -Chasing me, shooting me.
Shooting you. Yes, that's correct.
We used to do treatment foster care
for kids with medical needs.
My husband worked full-time and I was a stay-at-home mom.
I got a phone call
that there was a baby abandoned at the hospital in Milwaukee, Wisconsin.
He was born with a disease,
and he was so fragile you needed to hold him on a pillow.
And they wanted to know if I could come down,
and learn about him, and take him home, because he was gonna die.
-Doing awesome, buddy. -Just taking some pieces off.
They sent me home with a bottle of morphine
and said, "Keep him comfortable."
He didn't die.
Instead of getting worse, he thrived.
And there was no way in the world we were going to let him go back
into the foster care system.
We loved him, obviously, at this point.
And so we adopted him.
-Hi, Char. -Hi.
-What did you make for me? Mmm! -Look.
Charlie struggled a lot.
-No! -The yellow one…
He was in pain from the time he was born.
Tremendous pain.
His skin slid off just like the skin on a ripe peach.
I don't think that he could've gone through all this
unless he wasn't such a fighter.
Good one, Charlie!
Wow!
This is fun.
I really want to go again.
Meili, she was in an orphanage
in Beijing, China.
They sent us pictures.
And there was one picture of her
wrapped in gauze.
My husband, Kevin, and I, we looked at that, we're like,
"Oh gosh, we can't say no to that."
I can't ima… EB's hard enough.
But to be a child alone in an orphanage
with recessive dystrophic EB,
I… I…
I couldn't let that go.
So we adopted Meili.
She was six years old.
Hey, Mom.
Look! It's… Oh, she's scared.
It's okay, baby.
Meili was just so comforted
that now she was able to have bandages
that made her not hurt so much.
Rowan is just her own unique person.
Mom, I can carry this.
She's like the funniest person you'll meet, the wittiest.
And having been through as much as she has,
she's able to do it and still maintain all of her joyousness.
Okay.
I forgot to put shorts on.
The day Rowan was born was the best day of my life.
She had some small things on her body, like redness under her fingernails
and some what looked like blisters almost on the surface of her eyes,
but they were very small.
I was assured that they were just weird newborn things.
When she started having wounds appearing,
it quickly became basically like a nightmare
and the worst day of my life.
That's when a doctor came in
and said, "She either has a serious infection…
or she has something called 'epidermolysis bullosa.'"
And he said it so quietly.
And he said, "And it would be for life."
I would give anything for Rowan to be healthy.
Absolutely anything.
This disorder is so cruel.
And it doesn't care how sweet of a child you are.
It doesn't care that you're only a baby or you're only a three-year-old.
It just basically tears you apart as a child, as a family, and…
Yeah, I would give anything for her to not have EB.
All right. See, Dad's here for your bath.
-Dad's here. -That's cool.
I think as a parent,
you take comfort in knowing that there are people out there
who are trying their best
to work towards a cure.
I have a lot of hope for gene editing.
No, no, no, no, no!
It's okay. We're gonna fix you all up.
-We're gonna get it all fixed up, okay? -Big boo-boo!
Okay, we're gonna fix you all up, okay?
We'll make it better.
-Done! -Look. Careful, Rowan.
Well, what were we saying about you being like a mermaid, Rowie, yesterday?
Because I have EB, so it kind of makes me special.
-Mmm-hmm. -So…
Yeah, it definitely makes you special. And mer--
And like mermaids are special because they're half fish.
'Cause they're like a little bit different than everybody else.
Maybe they've gotta do things differently than everybody else.
Can I take these bandages off?
Come around…
You meet these young folks that are dealing with this.
It's the most extreme version of seeing something
as vulnerable
as anything you've ever witnessed
in your life on the planet.
At the same time, they've shown resilience,
and fortitude more than, again, anything you've ever witnessed
or seen in your life.
They're already working so hard to survive.
They could be rewarded for their efforts with a cure.
They would have earned their right to be on this planet, that's for damn sure.
We didn't really have a good idea of what it was until we brought him home.
Let me see your back.
Turn around.
We started to really understand what EB was like and how serious it was,
and how time-consuming it was to care for his skin.
Ow, ow, ow!
Pretty much just turned our world upside down.
The worst bath and bandage we ever had.
We had just been home for about three months from China,
so Eli was pretty brand-new to us, and EB was new to us.
I stopped counting at 30 blisters that we had popped.
He was, like, screaming in pain, and it was getting late.
And I looked around, and all my other kids had gone to bed.
I just felt super guilty because I didn't read a story.
Didn't tuck anybody in bed.
So I just stopped what I was doing, and I ran to their rooms,
and I gave them a hug, and I went back…
and finished.
Rock, paper, scissors, shoot.
Ah-ha!
-Ah! -That's better.
You always keep saying that.
-There we go. -It's so sticky.
I know it's too late for a lot of our friends who have EB.
I'm hoping that there's something for Eli in his lifetime
that will be able to help him.
Guys, go get your T-shirts.
We're running a little bit late, so put 'em on quick, okay?
Time is so important
because the kids that have EB don't have a lot of time.
The faster we get a cure, the more kids that we can extend their lifetime.
Don't forget your picture that you drew for Ed.
All right.
She's on a mission.
Which room is it, Rowie?
Right here
You know which one?
Oh, okay. Do you want to ring the doorbell?
Did it work?
-Okay, it definitely did that time. -Definitely worked.
-Hi! Come on in! -Hi!
We're here for a makeup application, yes.
Okay, so we're gonna do a little sparkle in your eyes. Sound good?
-Yeah. -Okay.
Can we push your hair back?
-Hold on, sweetie. -Yeah.
Your hair is so cute!
-Oh, thanks. -It's so long!
I cut mine all off because it was driving me crazy.
Okay. I'm gonna tickle you now.
-Does it feel funny? -Yeah.
-Like a little bug? -Yeah.
Okay, I'm going back in.
There you go!
Okay, take a peek.
-Is she gonna get her glasses on? -What do you think?
-I like it. -Want to see it with your glasses on?
-Yeah. -Okay, let's see. Put them on.
Okay, we'll do the same for the other side.
Okay.
See? We made it work.
-Yeah. -Yeah.
Okay, props, girl.
-Yeah! We'll see you later at the concert? -Yeah.
-Okay. Have fun. -Yeah.
-You too. -Okay! Bye, cutie!
Bye.
We just need your shoes. Anything else?
My shoes, my hat, and we already glued my earrings on.
-Mmm-hmm. -'Cause I can't pierce my ears.
Eyelash glue.
I don't know if we have enough…
-Is that straight? -Can you pinch this?
Okay, I think we're good.
I made this painting. It's a triptych.
And I made it for Eddie and Jill just to kind of say thank you
for having us out here and just thank you for everything that they've done for EB
and just the amount of global awareness that they've created,
because they really did start the wave.
I'm hoping to give it to him at the show, so I think that'll be really cool.
Just a little thank you.
And they've done so much for us,
so why not give back from everyone in the community?
Are you guys ready? Let's go.
We're so close. Ahh, I'm so excited.
Getting involved with EB, and EB Research Partnership,
I don't think it was a choice.
It chose us.
This community,
you are immediately taken by the amount of strength
and dedication on a daily basis
to overcome obstacles
that we can't even imagine.
I drew the Space Needle for Eddie Vedder's gift
'cause he lives here in Seattle.
When you see the intense pain that these kids go through…
Lily, get your face in!
…and imagine what it must do to the hearts of the parents,
there does become a timeline
and it's hard not to want to dedicate
a large portion of your life to doing whatever you can to help.
I wish I was a neutron bomb
And I would never go on
I wish I were a sacrifice
But somehow still lived on
I wish I were a sentimental
Ornament that you hung on
The Christmas tree
I wish I were The star that went on top
I wish I was the evidence
I wish I were the sound
Of all my hometown's hands
Upraised and open towards the sky
I wish I were a sailor
With someone who waited for me
I wish I was as fortunate
As fortunate as me
I wish I was the messenger
And all the news was good
Oh, I wish I was the full moon shining
Off your Camaro's hood
I wish I was a living being
At home behind the sun
I wish I were the souvenir
You kept your house key on
I wish I was the pedal brake
That you depended on
I wish I were the verb "to trust"
And I'd never you let down
I always thought I'd cross that river
The other side, distant now
As I got close, it turned and widened
Horizon now, fading out
Drifting off in the undertow
Can't spot a figure on dry land
And afterthoughts of safety
When in truth, none to be had None to be had
I used to tell time by my shadow
Till the thunderclouds They took the stage
The days will end As do the light's rays
Another read of the same page
Wide awake through this deepest night
Still waiting on the sun
As the hours seem to multiply
Find a star to soldier on
Living beneath a lion's paw
Knowing nothing can be tamed
Can be tamed
The momentum, the enthusiasm,
the hope, the optimism, the joy
to have everybody in a room where a patient can sit there
and talk to a doctor that's working on their cure.
That that doctor can talk to a biotech company that wants to take their science
from a lab and advance it through a clinical trial.
To have everybody in one space, in one room
is the coolest, most joyous thing that we could ever imagine.
Myself and Hodges are speaking on a panel together.
She's my official secretary, so she…
She keeps everything organized for me, 'cause I'm a mess.
I keep him on track.
Yeah, she keeps me on track, and I definitely need that.
We're a really small community,
so anybody that does anything on the level that Deanna and I are doing it,
we kinda all know each other.
-And it's good to hang out and party. -Yes.
Here and now
Won't hold me down
Won't hold us down…
-How are you? -I'm good. How are you?
-It's good to see you. -You too.
How about a hug? Can I have a hug?
How are you?
When everybody gets together with the same condition,
something magic happens.
That's where we're all the same. You know, we're all the same.
Like, we're all doing the same thing.
You just want somebody that understands
what you're going through on a day-to-day basis.
Share the light
Share the light, won't hold us down
Share the light, won't hold us…
Hi.
I'm good.
-So happy to be here. -Yeah, I'm happy you're here. This is…
Jill is doing everything she possibly can
to cure EB.
She's given me hope with her attitude of, "We're so close, we're so close."
All the messages before were, "This is awful."
"This is, you know, this is awful."
And they changed that, they changed dread into hope.
I'm so relieved that it's finally here.
It was just a tiny little idea we kind of tossed around
six months ago.
To see it all finally come together, and how powerful it is,
and how moved people feel, and the families are proud.
And that's what I cared most about.
Thank you, thank you, thank you, and welcome to the first ever
in-person Venture Into Cures.
Thank you all for coming.
The patient panel was awesome to see Deanna and Hodges together
talking about what their day-to-day is.
The biggest struggle in being so independent
is the mindset to know that it's okay to ask others for assistance.
And just saying like, "Oh, excuse me, can you hold the door?"
Because the world is not a very accessible place
when you look at it from the proper perspective.
A lot of people don't know what that even means.
Quite frankly, sometimes you have to be a bitch
or you're not gonna get anywhere.
So there's so many things…
Sorry.
…that people just don't understand.
And I'm so grateful to everyone at EBRP
for doing what they do, and that's why I'm here.
There's nothing like being together with the patients,
your friends,
and the supporters who have funded your research for so many years.
We're all here for a cure
and to help our EB patients now and in the future.
When you talk to these families, talk to these patients,
there's more hope than there's ever been.
There's patients sitting in this room
that are receiving the first-ever FDA-approved treatment.
And it's healing their wounds, it's making an impact.
Up until recently, we were just applying bandages.
That's all we had, was just covering the wounds, that's all we could do.
But now, for patients with recessive dystrophic EB and dystrophic EB,
we have this new treatment that we can apply every week,
and it actually treats the source of the genetic problem with her skin.
It's giving us this chance to get ahead of the condition
in that it's gene-correcting the skin,
so helping the skin to have actual durability more than it has,
to stop that process of the wounding.
Now that we have these treatments that will help these chronic wounds,
then I think we're just inevitably gonna see differences
in the long-term prognosis, hopefully,
with people with RDEB like Rowan has.
Because if you have these wounds that never heal,
that's a really high risk factor
for that specific type of aggressive cancer that they get.
Many people don't survive.
I'm gonna put this on here, okay? Just for--
What does this say?
It's really tough thinking about the other families
who don't have this treatment yet for their type of EB
because they're still waiting.
All the thoughts you never see
You're always thinking
Brain is wide, the brain is deep
Oh, how you're sinking
Feel the path of every day
Which road you taking?
Breathing hard and making hay
Yeah, this is living
Look for love in evidence
That you're worth keeping
Swallowed whole in negatives
It's so sad and sickening
Feel the air up above
Oh, pool of blue sky
Fill the air up above
All black with starlight
Feel the sky blanket you
With gems and rhinestones
Feel the path cut by the moon
For you to walk on
Nothing left, nothing left
Nothing there, nothing left
Nothing left, nothing left
Nothing there, nothing there
Nothing there, nothing left
Nothing there, nothing there
Nothing left
See the path cut by the moon
For you to walk on
See the waves on distant shores
Awaiting your arrival
Dream the dreams of other men
You'll be no one's rival
Dream the dreams of others then
You will be no one's rival
A distant time, a distant place
That's where we're living
A distant time, a distant space
So what ya giving?
What ya giving?
More acorns? Yeah, you can go get an acorn.
I have it…
Oh, that's a good one. All right, let's take it in.
Let's see.
My job as a pediatrician and as an EB doctor is to make sure
that kids can have childhood,
and they can experience life.
Those things that we take for granted
are the things that I have to help protect for that child and that family.
-Hi, guys. -Hi.
Hi, Rowan, how are you?
EB, it starts in the beginning of life
and is something that only tends to get worse
and to encroach on a child's life more and more
over time.
So my job, and my amazing team's job,
is to figure out ways to help that child be a child.
Okay, all done. And that ear looks nice and healthy too.
Okay?
We know the underlying genetics of EB,
so we have targets for therapies
and ways to think about curing EB.
Your turn.
Careful, Rowie.
What's the diagnosis, doc?
It's nutty.
So here we have the immunofluorescent validation
of our xenograft model.
We're basically just looking for the presence of human skin
on the back of a mouse, and you can see here,
similarly looking at both of the markers,
human loricrin and human vimentin
to validate the successful generation of human skin.
EB really provides an important model to test novel therapies.
A decade ago, the therapy for EB
was primarily just asymptomatic care.
Who would have thought ten years ago
that we can now easily manipulate stealth cells in the dish,
achieve very precise gene editing,
and potentially provide a curative treatment
for currently incurable conditions, such as EB.
We progressed a lot,
and the recently approved viral-based gene therapy for these patients,
yes, it's not a permanent cure,
but it's a treatment that's really very effective,
and gives these patients interim solution
while we're developing a more permanent therapy.
It's one step forward
toward finding like a permanent cure for EB patients.
Yeah.
It's like when it doesn't have the support of the bandage, it's kind of like…
Your skin looks really healthy around your G-tube site.
I think a decade ago it was hard for us to imagine
what this cure looks like,
and here we are, it's no longer in our imagination.
It's in the laboratory.
It's in the hospital setting.
You know, it's in patients' homes. We're there.
Like, we are on the edge of the cure for EB.
Oh!
-Looks like it's gonna crash into the… -Oh no!
Our Ferris wheel trip is canceled.
Did you see the candy in that cupboard over there?
-What? -If you open the doors, there's candy.
This is my candy now.
Yeah! Kit Kats!
This is luxury.
Yeah, I like it.
I don't really draw with pens.
Eli was adopted from China when he was four and a half.
I'm almost done.
-Lily, I'm making a rocket ship. -Yeah? What?
Oh, really?
And I'm going to try to make ten boosters.
One, two, three, four, five,
six, seven, eight, nine, ten, eleven, twelve.
When Eli was like five or so,
we'd be out in public and people would be staring at him.
Lily would get so annoyed, and start waving at them,
and put her hand on her hip, like, "We can see you."
There's the… There's the bathroom.
So then we made these little shirts that said, "Come say hi,"
to give people the invitation to not just stare
but approach somebody who looks kinda different.
Lily made a little video
saying, "If someone looks different, just come say hi, don't stare at them."
This is what you do when you see someone who looks different than you.
-Hello. -Hi.
See? That was easy.
-I want to teach you about EB. -EB?
Here you go.
Thank you.
When I see them handing out cards and wearing the T-shirts,
I'm amazed at how there's a six and an eight-year-old
teaching this kind of simple but profound message to adults.
-I want to teach you about EB. -Sure. Thank you.
I hope that
more people being kind to other people will come out of it.
They say be careful, that maybe you don't want to meet your heroes.
But there's someone here tonight I would love for you now
to just take a moment and meet my friend, my teacher, my hero, Eli.
I'll come and say hi
When I see Eli I'll come and say hi
When he heard that there was a treatment, he was so excited.
He was like, "Yes! Can we go get that?" I keep telling him,
"It's not for your type of EB, but they're working on something for you."
Oh, as I dream Your wings glow in the dark
When he has some really tough days, he's like, "Man, where is that treatment?"
"I need that right now."
I'm like, "I know."
Coming from you
Oh, you make the connection
And I follow your suggestion
When you say
I'll come and say hi
We need to find a cure, like, now.
We don't have time to wait.
We've lost so many kids lately with EB,
and I just stopped telling Eli and Lily, like, you know…
Sorry.
'Cause the older he gets,
the closer he is to the age of these kids that we're losing.
And that's tough.
There he is. Hey, Eli.
His mom and his sister.
There you go, man.
I'll come and say hi
When I see Eli
I'll come and say hi
When I see Eli I'll come and say hi
When I see Eli
I'll come and say hi
Oh, Eli, I'll come and say hi
Thanks, Garrett, for coming in today.
We really appreciate it.
What we're doing here today is a clinical trial
where we're examining a gene therapy skin graft
to see if it can heal chronic wounds.
What the clinical trial does is take a small biopsy of your normal skin,
send it to the lab, and we grow up your normal skin,
but we use a virus to insert in the right collagen VII.
So we hope to make your skin cells strong now.
And hopefully they will heal up the chronic wounds.
-Does that sound okay to you? -Mmm-hmm.
For many of these EB clinical trials, the first patients can't be the children.
Because we don't know the safety profile.
So we rely on heroes like Garrett and others who are older
to take the first plunge.
-Oops, sorry, Garrett. -Yeah. Mmm.
-Ready? -Yeah.
Is that okay, Garrett?
Garrett is 27.
Whenever he can help, he will help.
When I was younger, I really dreamed about playing basketball.
'Cause all my siblings played basketball.
I was able to play a little bit, but…
it got to be a little bit too hard for me, eventually.
Now I'm just gonna take a small, small sample.
So this area just hurts a little bit for him.
-Okay, you're doing fantastic. -How much more?
-Ow! What's going on? -I think she got it, Garrett.
I'm just dabbing. I'm just cleaning the area, that's it.
We always say, "We don't know if it's gonna work on you,"
but the data, and you showing up and participating
is gonna help tons of people.
I do all of it for the younger generation,
hoping they can get those wounds healed.
Maybe they gain, you know, another 10, 20, 30 more years, you know?
Hopefully they don't have to go through everything I've gone through.
This is mission critical. And you can't think about it too much.
Because if you do, you will cry. And you can't cry in front of them.
They need you to be chipper, positive, confident.
And we get through it together.
Once you have the gene, you understand how to model it,
how to test, how to correct that gene.
Put simply, you've got a copy of the patient's DNA,
and there's a mutation.
Researchers have discovered you can use enzymes
that swap the bad for the good piece of DNA,
and it can be that specific.
CRISPR correction is like a Word document,
and you highlight the word, you press delete,
the word goes away, and then you put in a new word.
And that's basically what we're doing into the nucleus of the cells of this tissue.
So the hope today is that we can actually generate
genetically-corrected tissues
for the patient to basically replace their current skin
with normal skin.
Going from putting a Band-Aid on it to basically correcting it permanently.
Which is so exciting because now we have hope.
I envision that we can put an end to their suffering.
Really reduce the pain.
Go play soccer without fear.
Get to participate more in life,
whatever that means for them.
Not have their dreams be cut short
because of EB.
For so long we had no FDA-approved therapies.
Like, goose egg. Like, nothing.
By putting all this attention on EB
and curing this disease that has a single gene mutation,
it's a bigger picture.
There's so few people that have EB, but there's lots of people
that have a rare genetic disorder,
and we just feel like this could open the floodgates
to curing so many other diseases.
Parkinson's, Alzheimer's,
anything we learn from here,
our circle of influence will expand upon these other diseases.
The patient's timeline is torturous.
Science, traditionally, has not moved as fast
because we work on government grants, we write papers.
Can be infuriating.
And so with private sources of funding,
patient foundation groups, we're really able to shrink that timeline.
There's just this feeling of, like,
"I am so glad I'm on this ride, and let's keep on going and go faster."
Jean, where are you?
Aw!
I'll tell you more about Jean in a second,
Um, but what I was gonna say is that my phone, um,
it's sitting on the piano there, and I check it just to make sure…
If it's John Fogerty, I wanted to say, "Goodnight."
"I'm heading out to play, I'll talk to him after."
And, um…
But what I was gonna say is that the phone, it buzzes.
And a lot of times, and I hate to throw anybody under the bus,
but a lot of times you're kind of shocked and amazed what people will text you
five minutes before you're supposed to go on.
Like, "Hey, Ed, I'm here, but driving… Where do I park?"
"Where do I park?" Um…
"I'm thinking about getting a couple burritos,
I might be late, would you like one?"
"I could bring it up to the stage if you want."
"Um, would you like… There's a little bar across the street."
"Do you want to get a drink before the show?"
You fucking idiot.
I'm trying to…
Sorry, I shouldn't say that.
Sorry, kids, I just…
But you know what, Rowan? Sometimes people are fucking idiots, and…
But that was not the text.
Um…
For one of the first times ever,
it was some useful information
that I would not have known.
And information that I thought,
"Well, that would be something to mention."
This guy, he texted me, he says,
"You realize, don't you, that it was 33 years ago today
that the band Pearl Jam played their first ever show
at the Off Ramp here in Seattle?"
Thirty-three!
So this is all such a blessing.
And circling back to Dr. Jean,
you know, she said that in the '90s
when she was going through med school, and many, many years of med school,
she was being driven and listening to music from Seattle,
and our band in particular.
To hear that and know that she is one of the top researchers
that will help cure this,
that she's also a doctor that treats patients,
to hear that our music had anything to do with your success
is one of the greatest things that we've been able to accomplish.
Yeah!
So, Jean, I love ya.
What the fuck is this world?
Running to, you didn't
Leave a message, at least I
Could have learned your voice One last time
Daily minefield, this could
Be my time, 'bout you?
Would you hit me?
The EB community is a special group.
You experience their bravery, and that has made me
come out of my comfort zone
to take more chances.
These kids are such, pardon my French, but badasses.
There's just so many really awesome, smart people
who are dedicating their lives to helping our kids. It means a lot.
All the bills go by, and
Initiatives are taken up
By the middle There ain't gonna be any middle any more
And the cross I'm bearing home
Ain't indicative of my place Left the porch
Hear my name, take a good look
This could be the day
Hold my hand, walk beside me
I just need to say
Hear my name, take a good look
This could be the day
Hold my hand, walk beside me
I just need to say
I could not take just one day
I know when I would not ever touch you
Hold you Feel you in my arms ever again
Yeah, yeah, yeah
So this is the treatment.
And this is what it looks like. It's four of these syringes.
It's an actual live virus that's inside of the syringes,
so the virus has been modified
to go into the skin cells
and clip out the DNA that she has that's, um, faulty.
When we first had the opportunity to get this medication,
I think I just needed like an hour to just cry.
-I don't know if you remember that. -Yeah.
But, like, it was such a big day
that we had been looking forward to for eight years.
But to have a treatment now, the first time ever,
I mean, that's massive.
A lot of tears, tears of joy.
Um…
But at the end of the day, it's a treatment and not a cure, and…
we need a cure.
You're doing really good, Ro.
Sometimes I'll see places where the skin looks like it's not scarred,
but it's almost looking back like normal skin,
and that's from the drug.
It's not something her skin would have done by itself.
J, I need another syringe of medicine.
So then we put the drug on,
this holds the drug on,
and then tomorrow, we'll rinse it all off
and re-bandage her with new bandages.
The whole process, I would say, between four to five hours.
In some ways, I sleep somewhat better at night.
Like, I have less bad, super anxious,
super sad days as a parent, I feel like.
And then if you just want to hand me the scissors.
I mean, those days still happen,
but I can just look at this
and think how far things have come just since Rowan was born.
That just makes me feel really hopeful.
Take leave in conscious mind
Found myself to be so inclined
Why sleep in discontent
For the price of companionship
My shadow runs with me
Underneath the big white sun
My shadow comes with me As we leave it all
We leave it all far behind
Oh, empty pockets will
Allow a greater sense of wealth
Why contain oneself Like any other book on the shelf
My shadow runs with me
Underneath the big white sun
My shadow comes with me As we leave it all
We leave it all far behind
Subtle voices in the wind
And the truth they're telling
The world begins where the road ends
Watch me leave it all behind
Far behind
We begin with news that saddens all of us.
Charlie Knuth has died.
Charlie Knuth, the boy we all came to know as "Little Charlie"
was 17.
He captured our entire community with his personality.
He cussed a lot, he swore a lot,
and, you know, people loved it because he was just so…
He was Charlie.
The phantom wizard has found the gems.
He wanted to be a dad,
he wanted to get married, he wanted to have girlfriends,
he wanted to go to school.
No…
Stop it! Stop it!
It was a normal day.
Charlie was fine, he's walking, talking, and, um, I was gone the day,
I was running some errands and things,
and when I came home, he said he didn't feel good.
He went to the bathroom, and then he came and laid out by me.
I was sitting on the couch, and he laid out by me on the couch,
and he just fell asleep.
And so I just let him fall asleep, you know?
Thought, "If he doesn't feel good, let's let Charlie take a nap."
And then in the middle of the night, I heard him make this crazy sound
that, like, he yelled out, something I never had heard of before.
And I ran down the stairs, and he was, um…
just sitting on the couch,
not making sense, he was incoherent,
I couldn't get his attention, and I knew he was in septic shock.
So we called 911,
and we are immediately in a helicopter
heading down to Milwaukee Children's Hospital.
For me, the greatest gift as a mom is that I knew he wasn't afraid.
Uh, Char?
Let go of the…
He didn't have to lose anymore.
I'm so fortunate to have met Charlie.
No regrets, none.
We almost hit Dad in the face.
Hi, Trisha and Meili.
Incredible parents, and we got to know each other.
We met in Minnesota many, many years ago.
And we would have many…
Um…
Sorry.
We would have many great conversations with Charlie on the phone,
and he had an incredible light,
and clever, and funny.
He was like a mini Jeff Ross with EB.
He was like, "Fuck you," like…
But, uh, so we now live with Charlie's memory,
and think about him all the time,
and we're so grateful that you came and were part of this weekend.
Thank you.
And, uh…
Charlie,
the only part of this song I will dedicate to you is the line in the chorus.
Waitin'
Watchin' the clock It's four o'clock
It's got to stop
Tell him
Take no more She practices her speech
As he opens the door
She rolls over
Pretends to sleep
As he looks her over
She lies And says she's in love with him
Can't find a better man
She dreams in color She dreams in red
Can't find a better man
We're at the aquarium, and we're gonna see seals and otters.
Rowie, uh, she frequently says she wants to be a normal kid.
The thing that I think she struggles with…
-We can touch dolphins! -I know! So cool.
Crab!
Me want to touch crab.
The thing that's really hard for me is she'll look at my hands and say,
"They're so beautiful. I want to have hands like that."
And those are the moments where we just are so hopeful for a cure,
just for the wounds to just stop happening,
and for her quality of life just to be more like everybody else's.
Rowan, I've fallen in love with her voice over and over.
Anytime she… I just…
It's the sweetest thing ever.
And thank you for coming.
You want to stand up, Rowan? You can.
There we go!
Hey!
Yes, I understand That every life must end
As we sit alone I know someday we must go
Oh, I'm a lucky man To count on both hands
The ones I love
Some folks, they got one Yeah, others they got none
Stay with me
Let's just breathe
-What's that seal's name? -That seal's name is Chiidax.
And he was a rescued animal.
Open.
-Whoa! -Good. Nice one.
-You're a natural! -Thanks.
Nice! Good! That's good work.
Aw, he's so cute. He has big eyes like my dog.
Under everything Just another human being
It's just wonderful to be together
and to be doing something that's just kid stuff,
that's not having to do something painful.
Stay with me
You're all I see
Did I say that I need you?
Oh, did I say that I want you?
Hey, Ro, did you see these here?
-Are you gonna bandage the sea turtle? -Yeah.
-We're gonna help you feel better. -Very good, Rowie.
Very good.
So, yeah, this is like the best feeling to be here playing right now.
As I look upon your face
Everything you gave And nothing you would take
Nothing you would take
Everything you gave
Did I say that I need you?
Oh, did I say that I want you?
Oh, if I didn't, I'm a fool, you see
As I come clean
Nothing you would take
Everything you gave
Hold me till I die
I'll meet you on the other side
-So there's fantasy… -Are we doing the horror?
Yeah, I want to do that one too, but if we have time, okay.
I love reading, especially fantasy and fiction,
so this is just right in my wheelhouse,
and everything is art-based.
It's amazing.
These are incredible.
Holy shit.
I might geek out a bit.
I want to see these.
-I want these shorts. I'd wear those. -Yeah. Silver too?
Yeah.
Being an artist, you can really appreciate all the work
that goes into these creations.
The overall just atmosphere of it is overwhelming,
and it's inspiring to see
how incredible
just the simple application process can make something so beautiful.
Wow! That's amazing.
It's a banjo.
Art is absolutely an escape.
You kind of get lost in the moment when you're creating something,
and I like to put on the music and just kind of lose yourself.
And sometimes you've been sitting there for
three, four hours, and you didn't even know it.
It really makes you appreciate
the things that are right in front of you and tangible.
-Hi, Ed. -How are you?
-It's so nice to officially meet you. -I know! It's official now!
-Yeah. -Oh, my pal.
I brought this for you guys.
-Oh, yeah? -Well, I made it, actually.
I was hoping you'd say "made."
It's a little something to say thank you for all the work that you guys have done.
-You know that's not necessary. -For EBRP.
I know it's not necessary, but it's what I do.
Oh man.
Oh, nice.
-Aww! Oh, is that us? -Yeah.
Look at your detail, everything.
I'll kind of give you, like, my inspo.
Oh, and there we go.
You're welcome.
-We love you. -Thanks for what you did. I love you too.
It's been fun. I'm so glad you've been here.
-We've had a good time. -It was so fun.
The community has grown exponentially across the world.
And it's so comforting to know that other people can understand
what you're going through, and you're not alone.
Researchers today are saying a cure is achievable within a decade.
So if we rise up today, it will be possible.
All right, I have the privilege
to introduce you, in person,
um, to my great friend, Deanna.
Seattle, this is Deanna.
Deanna, this is Seattle.
And you said that we will be doing
at least one shot of tequila after…
Absolutely.
Let's get on with the show. We got some fun to be had.
Love you, Deanna.
One more time.
Such is the way of the world
You can never know
Just where to put all your faith
And how will it grow?
Gonna rise up Burning black holes in dark memories
Gonna rise up
Turning mistakes into gold
Such is the passage of time
Too fast to fold
Oh, yeah, suddenly swallowed by signs
Lo and behold
Gonna rise up
Burning black holes in dark memories
In general, cancer is something that EB patients are more susceptible to
because our skin is more vulnerable.
For me, personally, I've had five bouts of squamous cell carcinoma,
which is basically a stage-four skin cancer.
Try to live your best life for as long as you possibly can.
It's basically, for me, I've learned to look at it
as quality versus quantity.
Having EB has made me the person I am today.
I wouldn't wish it on anyone,
but I know that I'm better for it…
because it's made me the strong advocate
and independent person that I am today.
You know, it's so special to be playing for a purpose,
and the purpose being this incredible mission,
and to have the doctors, and researchers, and scientists,
and activists, and donors,
and the families that have shown us all such courage.
So to have everybody here, um, and…
I'm gonna get choked up. Give me a second.
-Whoo! -We love you, Eddie!
Get your shit together, tough guy.
Well, I won't back down
No, I won't back down
You can stand me up At the gates of Hell
But I won't back down
I will stand my ground
I won't be turned around
Gonna keep this world From dragging me down
Gonna stand my ground
And I won't back down
Hey, baby
There ain't no easy way out
We're putting out in the universe that we want a cure
because that's what the parents want, and what the kids want.
All the skeptics that think, "Oh, why are you trying to cure this?" Or…
You know.
Uh, why wouldn't we?
It's like, "No, gonna cure it. Gonna cure it. Gonna cure it. Watch us."
I got just one life
In a world that keeps on…
The timeline is there, it's realistic, and it's doable.
You don't want to give up on them.
You want something better for them.
Won't back down
I think we can cure EB.
And all of us in this community that have the profound honor
of serving those that battle EB,
our duty is to give them something to march to
that illuminates an endgame.
Stand my ground…
You know, the people who are part of the community
are very strong, and they make you stronger.
I just, I don't know how to put words to it.
There ain't no easy way out
Hey, now
Stand my ground
And I won't back down
Oh, no, I won't back down
Hey!
This is the drawing that Eli gave me.
It's so detailed, just incredible.
It stays in the studio here.
My hearing is so bad…
Oh, hi.
My hearing is so bad, you know, I'm easily crept up on.
And…
You can… I can… Of all these people… My eyesight's good, though.
I can see my wife nodding her head.
"Yes, Ed, you cannot hear shit."
When your time is limited
Well, nothing happens too soon
Navigate, come find a way
Our differences be damned
Somebody asked me to sign some baseball last night.
I agreed to do it.
I don't know why I had it, but this could be your birthday present.
You got the cure, I'll fight the fight
It's just a matter of time
Come here, come out here. Get in the light.
-How you doing? What's your name? -Deanna.
This is a long fucking way to go
to get free tickets to an Eddie Vedder show, I gotta tell you.
What else you got for me? Who do I look like?
Maybe Howie Mandel, I don't know.
Shrink the space between us
A reaching of a hand
Stakes are raised to elevate
Just a second to understand
What you give, this will to live
You got the cure, I got the fight
It's just a matter of time
A matter of time
Just a matter of time…