WOMAN: I was in dance all of my life. It was the thing I did.
Every year, we had the big spring recital and they record it.
And there was this one year, I was, maybe 14.
My mum, she played it
and for some reason, I asked my mum, I'm like, "Is that how I look?"
And my mum said, "Yes."
But, you know, I still obviously knew I was shorter
and that I had dwarfism and everything,
but for some reason in my head,
I looked as uniform as all of the other girls.
And after I saw that video,
I realised I wasn't going to be like everybody else.
I was going to look like that for the rest of my life.
MARC FENNELL: Around the world,
one in 25,000 children are born with achondroplasia,
a type of dwarfism which stunts growth
and can lead to dangerous health problems.
But now, scientists have developed a drug,
which they believe can treat the condition,
allowing patients to grow to an average height.
It's being hailed as a miracle,
but do those who are born small want to grow tall?
Da, da, da...
Ta. Thank you.
MARC: In the world of modern science
Casper and his mum Daisy are a pretty big deal.
14-month-old Casper has achondroplasia,
the most common form of dwarfism.
But thanks to a scientific break-through,
Casper may outgrow his condition.
- Hello. How are you? - Good Thank you. How are you?
- I'm good. Who's this? - This is Casper.
Hello, Casper. How are you?
Woo. I gotta wave.
Since he was five months old,
Casper has been part of a global trial for a drug called Vosoritide.
Here they are. Oh, here he is.
Hey, who's this guy? Who's this grown-up boy?
DAISY: Yeah, I know.
Doctors believe the drug will help Casper, and kids like him,
to grow at an average rate.
- And how are the injections going? - The injections are going well.
And who's doing them, mainly?
- Myself and Justin. - Yep.
- And my mum, sometimes. - That's good.
She got the hang of it.
It's good to have mum trained
because at least you can sort of have a day off.
Yeah. Absolutely. That's exactly right. Rest for mum.
Geneticist Dr Ravi Savariryan is leading the trial.
He's known Daisy since Casper was just two weeks old.
When I first met Daisy, she had all of those emotions.
Which you can imagine, she was delighted to be a mum.
But she was terrified that her child had achondroplasia.
Who's this? Who's this?
My job was to try and give her accurate, up-to-date information.
So then she could get some control back over
what was a very disempowering situation.
You're going to crawl right off the edge, aren't you?
DAISY: He's coming for you.
Achondroplasia affects bone growth.
In adulthood, patients typically develop large heads,
regular-sized torsos and very short arms and legs,
which limits their height.
Does he stand at all unsupported with a...holding on? No? Not yet.
- Not yet. - Yeah.
Ravi believes that starting a few months after birth,
a daily injection of Vosoritide,
will help a child's bones grow at an average rate.
With Vosoritide, how does it actually work?
Achondroplasia is like trying to drive your car
with the handbrake on.
This medication basically comes in and takes off that handbrake
so that bone growth can resume as it would have otherwise.
In babies with achondroplasia,
an important gene called FGFR3
is, medically speaking, considered faulty.
FGFR3 regulates bone development,
but a rare mutation in the gene
causes bones to grow slower than normal.
An average two-year-old grows six centimetres a year,
while a child with achondroplasia grows about four centimetres a year.
When bone growth stops in early adulthood,
the result is dramatic reduction in height and proportions.
Enter Vosoritide.
When administered as a daily injection,
a molecule within Vosoritide
interferes with the faulty gene pathway,
allowing bones to grow at a regular rate.
The drug assists the faulty gene,
minimising the effects of achondroplasia.
Or at least, that's the theory.
I guess what I'm interested in is the why?
Like, why does it matter to you?
There's actually 500 different types of dwarfism.
Most people think it's a condition that just affects stature
and they're totally unaware of the huge medical complications
that patients can have.
Alrighty, Mister, let's see if we can hook these up.
There's an increased risk of sleep-disordered breathing,
having to have an operation on your spine.
In the first five years,
the incidence of sudden infant death syndrome
is 50 times that of a child without achondroplasia.
So, Casper is a really healthy child right now
with a potential whole list of problems.
And I hope this medication will mean
that none of those problems will ever come to fruition.
Do you want to help me hold that?
The early data is promising.
Results suggest patients achieve average growth
after three to six months on the drug.
Good job. That's that one done. Good job.
The study is ongoing,
with 121 kids enrolled in seven countries around the world.
(CASPER CRIES) - I know. I know you don't like stretching.
For them, the cost of growing up is a daily injection at home,
followed by frequent hospital visits for intensive testing and study.
Good boy.
And there's an even tougher pill to swallow.
Being a controlled study,
one in two patients receive a placebo in their first year of the trial,
meaning Casper may not even be getting Vosoritide.
So, there's a 50/50 chance that he's on the inactive dose.
Yes.
How do you feel about that?
Initially, I was hesitant.
Because giving Casper the injections to start with
when he wasn't used to it was quite distressing.
But with time, Casper's getting used to it, I'm getting used to it.
And as you know, Daisy, the good thing is that whatever he's on,
inactive or active, after one year he'll have access to the active drug
until he finishes growing.
The good news for Casper,
is that his one-year anniversary is just a few weeks away.
But the reality is there is no guarantee
Casper, or any trial participant,
will experience enhanced growth, or safely finish the trial.
For all involved it is a giant leap of faith.
DR RAJIV: People like Daisy who, you know,
put their children up for these trials,
which are still in early stage, experiments,
it's a hugely courageous thing to do.
She's putting her trust in us, in me,
that this medication is going to benefit her child
and not harm her child.
So, they are the absolute heroes of this.
They're the pioneers of this
that's going to set the stage for the future.
DAISY: It's a lot of hard work. It's tough at times.
But he's the love of my life
and I just want to do as much as possible,
to give him the best possible start to life.
All over the world, unsuspecting home's like Daisy's and Justin's
are where the trial plays out every day.
(KNOCK ON DOOR)
- Hello. - Hi. How are you?
Hello. How are you?
It's Casper!
Say, hi, Marc.
- Good wave. Good wave. - Good boy.
- Come on in. - Ta. Thank you.
How you been?
So, we're just about to get Casper's Vosoritide injection ready for the day.
Essentially we just need to mix the water with the powder in the vial
to create the liquid.
Then we wait a couple of minutes and then get the injection ready.
(CASPER CRIES)
It's alright, darling. It will only take a second.
Shh. Shh.
Well done. Good boy.
There we go. It's OK.
Back to normal again.
- So, you do this every day. - Every day.
So, how long is he going to need to do these?
Until he reaches his final adult height.
So, until puberty finishes.
That's two decades.
- Yeah, about two decades, yeah. - Jeez. Right.
For Daisy, for Justin, this is all a huge commitment.
Life with little Casper is a fair bit different to what they were expecting
when they first got pregnant.
DAISY: It was my first little bub.
And I'd wanted to be a mum for a very long time.
So we were very excited,
until the point where we went for our 12-week scan,
and we were told that something was wrong.
But they weren't sure what.
JUSTIN: When he was born,
our doctor said he was pretty sure that he had achondroplasia.
But he couldn't confirm it.
And then we brought him home and when he was 10 days old,
they gave us a call and said, "Yeah, it is what we suspect."
You want that one?
I felt pretty ill-equipped at the time,
but our hospital referred us to Dr Ravi.
And he sort of just said, "It's going to be OK."
And we were told about the trial.
And so, how did you weigh up the decision
to put Casper through the trial?
I think I had anxieties about what his future would look like,
what his life would look like.
Worrying about him getting bullied at school for his appearance.
But, yeah, ultimately the decision to become a part of the trial
wasn't aesthetic or cosmetic for me.
A lot of my decision was rooted in health complications
and avoiding as much of that as possible.
Have either of you, before the birth of Casper,
ever had any dealing with short statured people, little people?
- Achondroplasia at all? - No.
I guess one of the complications is, you're two average height parents
making decisions for a really young child
that are gonna affect him for the rest of his life.
How do you feel like you're handling that pressure?
There are days where, you know, you still doubt yourself
and go, like, "Am I doing the right thing?
"Am I doing everything that I could?"
As his parents we have a duty of care as it were.
We want to give him the best start.
So, we are two average height parents,
but two average height parents doing the best we can.
Statistically, 80% of kids with dwarfism
are born to average height parents,
meaning the decision to put children into the Vosoritide trial
is often made by parents with little to no prior experience of dwarfism.
This disconnect has led many to question the ethics
of the Vosoritide trial.
Some in the short statured community are going so far
as accusing scientists of trying to eradicate little people.
Did you realise early on
that the trial was controversial amongst the little people community?
- Not at the beginning. - OK.
But, you know, we've been a part of it for a while now
and I'm aware of the controversy surrounding it.
Do you understand why there is controversy around it, in that community?
Yeah. Yeah, I believe I do.
Right now, around the world,
many adults with achondroplasia are angered
by the development of Vosoritide.
And the strongest opposition is in the United States,
where an estimated 30,000 people have a form of dwarfism.
WOMAN: I'm fearful of the drug Vosoritide.
I think it could lead to a lot more bad than good.
MAN: So, do you want to go get some food from the store?
And then we'll make lunch here?
- Sounds good. - Alright.
Becky, a motivational speaker, and her husband Ryan
belong to Little People of America,
a support organisation for short statured people.
Should I wear my Packers hat or my Patriots hat?
Oh, man. I think the Packers may match the green.
Perfect.
The organisation does not endorse the use of Vosoritide.
As a little person I'm super passionate about this space
because I just don't think it matters what your height is.
We just want a chance to participate in a meaningful way in society.
- We got good weather today, Becky. - Gorgeous.
Just like Casper back in Melbourne,
Becky and Ryan were born to average height parents
and into average height families.
Becky and Ryan both attended regular high school,
graduated college
and until recently, Becky worked as a casting agent in Hollywood.
You look like a Christmas tree.
BECKY: (LAUGHS)
True.
MARC: Apart from their height,
there's nothing much that distinguishes them
from your average American.
BECKY: There is kind of a stigma I think
that we can't do this, we can't do that.
But I think we just find ways to figure it out.
MARC: In terms of day-to-day living
that can be as easy as asking for the odd hand.
Excuse me. Do you mind helping us reach the tartar sauce?
- Thank you so much. - OK.
Yeah, just the fat free.
- This one? - Yep. Thank you. - Thank you very much.
MARC: Making simple modifications...
They think you just have to be tall to drive.
I have a little accessory called pedal extensions
that pushes the original pedal.
..or occasionally shopping in the children's section.
- Actually it is kind of nice. - Yeah.
I do kind of want it.
MARC: The way Becky sees it, there just aren't enough situations
that require little people to change physically,
a fact that she says empowers their community.
BECKY: I don't think me being a foot taller
would add any additional value to my life.
I'm who I am and I think the level of pride just continues to increase
the more bias and stigmas that come our way.
And, yes, there are so many challenges that I've had to face
to get to where I am today,
but I think it just makes us stronger and it adds more to our characters.
- Do it in the oven? - Yeah.
People often ask me, if I could wave a magic wand
would I choose to be a little person in my next life.
And I always respond by saying yes.
But I would change the way the outside world reacts to me
being a little person.
The first cocksucker to nail a bullseye...
(CHEERING)
MARC: According to Becky, the biggest challenge facing little people
is the prejudice of the average height world.
CROWD: One, two, three...
That happened to me when I was living in Los Angeles,
where someone tried to pick me up
and I ended up hitting my head on the cement.
People assume that it's socially acceptable to pick us up
and throw us across a room.
People look at people with dwarfism as non-humans.
MARC: Becky and many others feel there's a simple explanation
for these attitudes.
# We represent the lollipop guild
# The lollipop guild
# The lollipop guild. #
BECKY: Historically, people with dwarfism
have been portrayed negatively in the media
as elves and leprechauns.
And it just affects how each of us are treated in everyday life.
MARC: Films like The Wizard of Oz, Willy Wonka,
The Wolf of Wall Street, and Austin Powers, are often criticised
for their harmful depictions of dwarfism.
Many also argue that these negative portrayals
lead parents to enrol children in trials for drugs like Vosoritide.
Down, Mini Me.
..O Lord, these gifts which we are about to receive...
MARC: Becky fears the consequences for her people.
..through Christ our Lord. Amen.
MARC: If this drug is approved and does become popular,
what do you think will happen to that community?
I hate to say this, but I'm almost glad
that I may not be alive during that time,
because there may be no community if it becomes so popular.
So, when people say terms like 'genocide' and 'elimination',
it's accurate.
Absolutely.
People are talking about
how it's not meant to make our population extinct,
but it's meant to take away the painful parts of what we go through.
I feel that you can't have one or the other.
Like, it's so much a part of...
..our build and who we are and, like, if you're taking a drug
to then eliminate the painful parts,
but still keeping the character traits,
I don't know if you can have both.
Becky, Vosoritide is being given to babies
who really have no agency in this process at all.
What do you think of that?
If it was my baby, I wouldn't do it.
I think a lot of average height parents are fearful
because they don't have enough evidence
about what the future can be like for their child.
This is who we are and we can be fully capable and independent
just as who we are.
And, yes, there may be some medical procedures
that come up along the way
but it would just be so nice
if people didn't just think that we needed to be fixed
and embraced who we were.
In the US, this trial has faced pretty significant opposition
from certain quarters.
Why do you think that is?
There's a big belief that this medication will inherently
take away the identity of those people being treated.
It's no more changing their identity
than if they have pneumonia and I treat them with penicillin.
If you've got a community that their bonds are built on height
some of that fear must be well founded, though.
Whenever something new comes out it's controversial.
When the treatment for leukaemia came out in the '40s and '50s,
people said it's unethical to treat children with leukaemia.
And now we wouldn't think twice
about our children being treated for leukaemia
because we have almost 100% improvement rate.
And I guess the other thing, these treatments are not compulsory.
What they are, is an option for families.
And when families hear about this option, almost always,
they want an option that might improve the health of their child.
But in the end, we're not trying to change anyone's identity,
we're trying to keep children healthy.
Becky, what would you like those parents to know,
to be thinking about as they make those choices?
What is it that you're trying to run away from or hide from?
And what do you think this can fix?
Your child can be anything,
give them the chance to spread their wings
and be the best versions of themselves.
And don't let your doubts or limitations get in their way.
Although Vosoritide is on the cutting edge,
it's not the only time science has tried to help people
overcome the physical limitations of dwarfism.
I wish they would let you try clothes on.
It's just the little things in life
like being able to pick a pair of pants at the store
try them on and have them fit.
27-year-old Chandler was born with achondroplasia.
Her parents are average height so are her brother and sister.
Actually really nice.
But when she was a teenager,
she chose to undergo a surgical procedure called 'limb lengthening'.
CHANDLER: Limb lengthening involves the cutting of the bone.
And they put screws or rods in your legs or arms
and pull the bone apart to let new bone form,
which makes you achieve more height.
So, this was probably the highest shelf I could reach
before any of my lengthenings.
But, you know, now I can just grab a jar of sauce
off of a shelf like that.
Chandler is now 4 foot 11.
That's over 30cm taller than she was expected to grow naturally.
Now, I'm able to just get in a car
drive, and not have to worry about pedal or hand adaptations.
I mean, it just gives me
another sense of independence.
Chandler had her first lengthening at 16,
a few years after seeing this home video.
To gain her extra height she had three procedures,
each taking between six and eight months to complete.
Oh, yeah. I forgot. You wanted to watch.
And, being a good millennial,
she documented all of it on social media.
I always heard opinions from people with dwarfism about lengthening
that, it was very excruciating and you're bedridden
and you're just doing all of this just to be taller.
And that's not it at all.
This was, like, my 'I'm going to wear eyeliner' phase.
Chandler now works as a patient advocate,
helping families understand the medical treatments available
to children with dwarfism.
But the reaction from the short statured community
hasn't always been good.
Chandler, when you decided to get limb lengthening,
what were you hoping for?
What did you hope it would achieve?
The first thing in my mind that I was hoping for
was that I would look like everybody else.
It wasn't really a matter of my height.
It was more of a matter of I wanted my arms and legs to be proportionate
Because, you know, I just... I wanted to look normal.
You've been pretty up-front talking about this experience.
Have you had any pushback from the rest of the community?
I have received feedback from those in the dwarfism community
who would private message me
and tell me that my family never loved me
and never accepted me.
I'm going to cry.
They told my mum, "Your child is not one of you.
"She is one of us."
And there are days when those words do get to me,
but at the end of it,
why does the choice that I made for myself affect you?
If a drug like Vosoritide had been available to you
and you had had the choice when you were younger,
do you think it's something you would have taken up?
I know had I had an option like Vosoritide,
it is something that in a heartbeat I would have wanted to be on
Vosoritide, it treats and prevents
a lot of the risks and complications
that come with having achondroplasia,
but Achondroplasia itself will not go away.
READS: "We'll gather supplies and make them our own
"and prepare to take flight into the great unknown."
MARC: Back in Melbourne, now 17-month-old Casper and his family
continue to be part of the global trial for Vosoritide,
a controversial drug which may help children with dwarfism grow tall.
"You're one of a kind and it's so clear to see
"the world needs who you were made to be."
I first met the family three months ago.
And since then, parents Daisy and Justin
have given their son around 84 shots, of either Vosoritide
or a placebo.
Shoosh, it's alright, darling.
And while they don't know
if Casper has already been receiving the active drug,
there have been some big changes for this little man.
Stand for Mum.
Good boy.
We're very excited because Casper's just started
to pull himself up and stand.
Watching Casper grow and develop is amazing.
And fills me with that warm, sort of sense of pride.
It's just immensely rewarding.
It's not the only change.
- Hello. - How are you, Daisy?
I'm well. How are you? - Good to see you. - Likewise.
It's been a year since Casper joined the trial,
and after a series of tests, Dr Ravi has some news.
We're going to be consenting you into the next part of the study,
which is the long-term extension part,
which means he's 100% sure to be getting the active drug.
And that medication potentially, he'll be on till he finishes growing.
So, for the next 18 years.
So it's very exciting. Thanks for hanging in there for the whole year.
No. Thank you.
So, how are you feeling?
Yeah. I'm excited.
I'm really excited to be a part.
So, I'll just get you to write his name there.
That's you.
And your signature there.
Welcome to the next part of his journey.
Thank you. Thank you. - Very exiting.
Thumbs up, Casper.
The condition that Casper has
means that he has a much higher incidence of medical complications
functional limitations and other issues.
And I just want to remove all of those
so that he can live a life without any of the consequences of his medical condition.
- He's very proud of himself. - He's so proud of himself.
He's just given me a massive 'How good am I?'
He thinks he's doing an amazing job.
He's just given me a massive 'How good am I?' smile.
I think it's what we all want for our children.
So, pretty simple.
And if this trial and this medication can be part of that,
I think it's a great option for families.
But behind all the excitement
is the reality that Casper is a boy growing up between two worlds.
- Froggie goes ribbet. - Ribbet. Ribbet
Ribbet.
For Daisy, it's important for her son to know
that whoever he becomes, he won't be alone.
- Ta. - Ta.
Today, Casper's meeting members of Short Statured People of Australia.
He has four words and I'm very upset that 'Mum' is not one of them.
(LAUGHTER)
MAN: We're actually a support organisation
for people with dwarfism and their families.
But we're also trying to provide an opportunity
for people to meet people with similar conditions to themselves
and be around people that are like them.
Maybe you can teach Casper one day.
MARC: For Casper, today is a chance
to play with other short statured kids like 4-year-old Sienna.
I just told Casper I love him.
Aww... That's beautiful.
For Daisy and Justin it's a place to get answers
they won't find anywhere else.
It was a whole new world parenting a child with short stature
as opposed to being short statured myself.
I'm still learning about the questions I need to ask
and the things I need look out for and do for her.
But if Casper does outgrow this group,
will he still have a place in it?
The drug trial has kind of been a journey I suppose,
for the organisation.
We want people to know that whether they want to be part of it
or don't want to be part of it,
they're welcome in our organisation.
And being part of the community has nothing to do with height.
It has to do with the condition
and has to do with identifying with your peers.
MARC: Vosoritide has now been approved for use in Australia.
Even if it can treat achondroplasia,
the effect on the world's short-statured community,
that won't be known for quite some time.
And the question of whether kids like Casper should grow tall
will face families for years to come.
CHANDLER: I always say just let everyone make their own decision
and it's always best to just be educated
and to know what's out there.
BECKY: I wouldn't do it.
But at the end of the day
they're still a human being and they want to be loved and accepted.
I would welcome them with open arms.
DAISY: I would just hope that others would respect
that our intentions were pure and we did the best we could.
And we did what we thought was the right thing.
It came from love.
Yeah. Yeah. Absolutely.
For us, Casper is just a normal child.
He's no different to any other child, really.
VOICEOVER: Next week on Dateline,
as country's rally for the World Cup
we uncover football's struggle to shake its racist reputation.
Captions by Red Bee Media (c) SBS Australia 2022