December 2, 2025 • 40 mins
Rockstar Casey McPherson and his family were shocked to the core when his daughter Rose was diagnosed with a rare genetic mutation that has no known treatment. Faced with the possibility of losing his daughter, the front man of the Flying Colours dropped everything to devote his life to trying to find a cure before time runs out.
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****** SPOILER ALERT BELOW ******
You can learn more about AlphaRose at https://www.alpharose.com/, To Cure A Rose Foundation at www.tocurearose.org and TCAR RareLabs at https://www.tcarrarelabs.org/.
You can follow Casey @casey.mcpherson on instagram.
See omnystudio.com/listener for privacy information.
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Transcript
Episode Transcript
Available transcripts are automatically generated. Complete accuracy is not guaranteed.
Speaker 1 (00:03):
Ruby. She was screaming uncontrollably, like she was in pain
and confused and angry. You could just see on her face.
It was very, very clear that something was going very wrong.
Speaker 2 (00:18):
When Rosie's thing was more of a new thing to
all of us, I kind of more focused on the
bad parts of it.
Speaker 3 (00:26):
I Am not going to be okay if we're too
late to help Rosie. It has to be Rosie. We
have to get it done in time. For Rosie.
Speaker 1 (00:32):
There's really nothing we can do casing, you know, rather
than make her comfortable good luck.
Speaker 4 (00:40):
How terrifying would it be to fight an unknown enemy,
one you didn't recognize and didn't see coming. What if
that enemy was coming from within a disease that even
doctors couldn't identify. Nearly half of Americans suffered from some
chronic illness, and many struggle for an accurate dify diagnosis.
(01:01):
These are their stories. I'm Lauren Bray Pacheco, and this
is symptomatic. Before Casey McPherson became a father, he was
a rock star as the front man of Indecentsation Flying Colors.
He was used to life on the road.
Speaker 1 (01:21):
I was touring all the time in a band. I
would be on tour for three weeks, six weeks, come
home for a few weeks. You know, you sort of
arrange your life however you saw fit.
Speaker 4 (01:34):
You found incredible success as a musician, but it was
really hard earned. Tell me a little bit about the
early days when you first started out.
Speaker 1 (01:45):
I'd spent over a decade living under the poverty line,
printing CDs in my house and playing to bartenders, you know,
touring in broken down vans. I can't tell you how
many times we broke down or the van caught on
fire or whatever. I remember one place we played said
we drank more than they were going to pay us,
so we owed them money. And I just remember thinking, like,
this is my life, you know. And so there was
(02:07):
a degree of grit. As you know, for artists, there's
an incredible amount of devotion you have to your craft.
I didn't think anything could ever take me out of
the music business. I had a songwriting out of making records.
I never thought I would be doing anything else besides that.
Speaker 4 (02:23):
And then you became a father. What was the experience
of becoming a parent for the first time, like.
Speaker 1 (02:30):
For you, I was in my mid thirties when I
had my first daughter, Weston, And I'll never forget that
day because I was already singing to her, you know,
in the womb, and when she came out, I knew
that there was nothing I wouldn't do for her, and
(02:52):
that my life was not my own anymore. I just knew.
I knew in that moment the meaning of unconditional love.
Speaker 4 (03:02):
And how long after Wes was born was your second
daughter born.
Speaker 1 (03:06):
I think a little under two years that the point
Rose was born, I had a major label record deal,
I had a couple of top ten hits on the radio.
I was touring the world. I'll never forget when Rose
was born. She had the umbilical cord wrapped around her neck,
and you know, it was very scary. I remember holding
this tiny, tiny little girl in my hands and thinking
(03:30):
that my charge now was to keep these little ladies alive,
help make them strong and independent, and you know, accomplish
their dreams.
Speaker 4 (03:41):
Very quickly, Casey and his family began to have concerns
about regression in Rose's development. When looking back, is the
first time that something stuck out to you as maybe
an issue in terms of Rose's health?
Speaker 1 (03:59):
About eighteen months I wasn't really convinced until she lost
her ability to speak, she was learning language. She first
learned a sign, which was normal at her age, and
then she started saying words like mom, dad, and outside.
She point to the door and say outside, and we'd
go outside. But when she stopped talking, it was that
(04:21):
sort of deterioration of language was when I knew this
was not okay. I have the girls, So she's in
(04:51):
the other room. You maybe can hear squeak and squeal
every now and then.
Speaker 4 (04:56):
Today Rosie is nonverbal through sounds and physical cues. Throughout
the episode, we will rely on those closest to her
to share her experience. Though Rose no longer communicates with words,
she is full of personality. Here's her older sister, Weston.
Speaker 1 (05:16):
She loves animals.
Speaker 2 (05:18):
She looks like super shy and like scared, but once
she gets to know her, she's a little bit like
spicy and like a little bit sassy, but she's super
happy all the time whenever she loves. It's one of
the strongest emotions I get from her because that's one
of the only times you know what she's feeling.
Speaker 4 (05:38):
What would you say is her absolute favorite food?
Speaker 2 (05:45):
I think her favorite food is probably pasta. Her favorite
drink is definitely doctor pepper. She's stolen my dad's Doctor
Pepper millions sometimes.
Speaker 4 (05:56):
What are some of your favorite things to do with Rosie?
Speaker 2 (06:00):
I love listening to music with her. I think she's
always been someone who loves music too, whether it's like
watching TV or like listening to my dad play. I
think she just really enjoys like the sound of music
and like the feelings that come with it. I'm so
lucky to have her as my sister, because even though
(06:21):
she can't talk, she's just one of the most amazing
people I've ever met. It beautiful.
Speaker 4 (06:43):
Did you notice any difference initially between Wes as a
baby and Rose as a baby.
Speaker 1 (06:50):
You know, other than personality, not really. You know, at
some point Rose was beginning to experience some issues. You know,
she was having trouble going to the bathroom so I'd
have to give her enemas, and she would struggle with
eating solids. But for the most part, doctors told us,
you know, that's normal. Kids kind of developed, their systems
(07:13):
are getting started in different ways in different times. She
didn't want to walk, and then when she would try
to walk instead of falling on her hands, she would
fall flat on her face without putting her hands in
front of her, So there was this kind of gross
motor lack of response. She was also screaming uncontrollably, like
(07:34):
she was in pain all the time and confused and angry.
You know, you could just see on her face. This
wasn't like I'm tired or I need milk or you know,
it was very very clear that something was going very wrong.
Speaker 4 (07:48):
What did our doctors say when you voiced your concerns?
Did they run any tests or give you any answers.
Speaker 1 (07:57):
So, because our healthcare is some runs off of what
insurance will cover, they have a process, and the process
maybe differs from hospital to hospital, but it's relatively the same.
So they'll say, you know what, We're going to run
a panel and it tests for diseases that currently have treatments.
It doesn't test for diseases that don't. And keep in mind,
(08:20):
there's over ten thousand rare diseases and only five percent
of them have any treatments, so you can imagine how
many are left out on these panels.
Speaker 4 (08:29):
The panel tests come back showing nothing to indicate an
abnormality that could be contributing to Rose's decline. With no
clear pathway laid out by Roses doctors, Casey takes it
upon himself to research what needs to be done next.
Speaker 1 (08:45):
Her mom and I had started kind of doing some research,
and we knew enough about neurodevelopment to know that it's
often caused by genetic mutations. It was clear that she
was having trouble with balance and losing her words. She
struggled with feeding herself. When you know kids at that
point were sort of getting better at that you know,
(09:07):
it was severely missing milestones at this point. In order
to find a genetic mutation, you needed genetic sequencing done,
and at the time, the only one we could afford
was a whole exome sequence because it was ten thousand dollars.
Keep in mind it's two hundred and fifty dollars now.
They sent some boxes to us of swabs and I
(09:28):
had to swab me, swab Mom, swab Weston, swab Rose
and send those back and I think that took a
few months or four months to get all of that.
Speaker 4 (09:39):
The stress of waiting for answers while watching Rose's health
get worse weigh heavily on Casey and his family as
they await a prognosis, Casey and his wife split up.
Can you give us an example of just how serious
things were getting?
Speaker 1 (09:58):
Yeah, you know, she died from her first seizure for
a couple of minutes when mom had to bring her back.
Speaker 4 (10:06):
That must have been terrifying for everyone, almost losing Rosie,
but also still having to wait for answers as to
what was causing all of this. What was that like
for you as a father? How did you handle that?
Speaker 1 (10:26):
I was trying to keep everybody calm, and as so
many ways, I was minimizing, like, hey, it's going to
be okay, She's going to get through this. And you know,
I think anytime you experience unimaginable loss or devastation, there's
sort of a level of unbelief or disbelief, and I
(10:48):
think I sort of had that. So I was doing
the work to try to get to an answer, but
also didn't really think it was going.
Speaker 3 (10:55):
To be this bad.
Speaker 1 (10:56):
And then they emailed us a report, and that's when
I had to schedule another pediatric neurologist appointment so that
he could tell me what I was looking at. I
remember I had both girls, I was alone with them.
I held both their hands, walked them across the parking
(11:17):
lot into the elevator. Rose cannot handle elevators. She was screaming,
makes west and cry, then finally calling my name, going
into the small little room that Rose also didn't like.
I remember the pediatric neurologist walking in with very little
empathy and saying this Jeneta report shows that she's got
(11:40):
a genetic disease, a mutation in the h and rmph
two gene.
Speaker 4 (11:44):
What the neurologist tells them shocks Casey and his family
to the core. Rose's results show a rare mutation on
the h n r n pH two gene of the
H chromosome. The condition is called h n r n
pH two related developmental disorder. At only three years old,
(12:07):
Rose is diagnosed with an untreatable disorder with a devastating prognosis.
Speaker 1 (12:13):
Because this is what happens with these kids. They never
live on their own, and they developed seizures around eight
Some of them have died. We don't know why. We
think it could be the seizures. Some of them have
a few words that they can say, most of them
can't talk. Some end up catatonic and wheelchairs. Some can walk.
So there's this huge variety of what could happen to Rosie,
(12:36):
but one thing was clear. She would never live on
her own, she would never go to college, she would
never get married, or have a job or go to
the movies with her friends. Like that, life was not
going to happen.
Speaker 4 (12:48):
Were you left with any advice? What did the doctors say?
Speaker 1 (12:55):
There's really nothing we can do casing, you know, other
than make her comfortable. Good luck, good luck, and those
words ringing in my ears. I remember being shocked leaving
there with both girls and getting in the van and
feeling super alone.
Speaker 4 (13:18):
Casey is left without answers to deal with a disorder
that is so rare that in twenty twenty one, only
thirty three cases had been documented worldwide. Here is Rodney Bowling, Junior,
an expert in the field of molecular genetics, to explain
the mutation causing Rosie's condition.
Speaker 3 (13:37):
Rosie has a single based permutation in her hn rn
pH two gene very niche. It is a member of
the spliceosome that removes entrons from the premature message to
allow it to be made into messenger RNA. So in
Rosie's case, h n rnph two, I'm just going to
(13:58):
call it H two. A H two reaches out with
one hand and grabs hold of an unprocessed RNA. The
other hand grabs the splicees ome and introduces the RNA
to the splice's ome for processing. In Rosie's case, it
reaches out and grabs the RNA, but there's a great
(14:19):
big trip to fan amino acid right in the middle
of the domain that should introduce and bind the splicesome
that blocks that interaction. It's like putting on a boxing
glove and trying to shake somebody's hand. It's not going
to happen very well. So the protein sequesters more than
four hundred different messages away from being processed, and this
(14:41):
is what causes her disease.
Speaker 1 (14:44):
Rosie definitely has this disease. So I have to accept
that right, and that took some time, Like there's a
grief that you go through when you know that your
child is not going to have the same freedoms and
hopes and dreams and cap abilities that you and I do,
or you know you want that for your children.
Speaker 4 (15:04):
Faced with a challenge she could not fix with the
tools available, Casey began to wonder if he could build
his own.
Speaker 1 (15:12):
Why doesn't she have a treatment. What's the problem. And
that's first sent me down a journey of research. Right,
the veil had sort of been lifted and said, oh,
the hospital can't help my daughter. So I remember the
first metric that really got me was that there were
(15:35):
over four hundred million people with diseases like this, half
of them are children. Thirty percent of those kids die
before their fifth birthday. That's in the tens of millions, right,
and that only five percent of these ten thousand plus
rare diseases actually have treatments. So I had to identify
the monster first, what was the actual problem? And I
(15:56):
realized it wasn't a technology issue for most these diseases,
it was a business model issue. We had created a
healthcare system where we developed drugs for large populations, but
most genetic disease is ulterriy even could be one person
similar to Rose. She didn't have a cure, she didn't
have a treatment because nobody felt like they could make
(16:17):
enough money off of it. Wow mm hmm.
Speaker 4 (16:21):
Just thinking of like sitting in that van with your
two girls, having just been blindsided by the brutal reality
of not only what she's dealing with, but the fact
that no one's offering you hope?
Speaker 2 (16:34):
How did you?
Speaker 4 (16:35):
How did you turn your pain into the path forward?
Speaker 1 (16:41):
Anyone who's experienced immense tragedy in their life or suffering
or loss, I'd lost both my brother and father to suicide,
I think understands you kind of have two options. You
have an option to continue your suffering by stuffing it
down or or hiding it away or running away, or
(17:06):
you find some way to create purpose out of it.
I had to figure out how to turn pain into purpose.
But I knew nothing about genetics. I certainly didn't know
anything about ner developmental disease, you know, being a rock
and roll guy, having fallen asleep in biology class in
high school. I'm like, am I smart enough to do
(17:27):
something about this?
Speaker 4 (17:29):
We'll be right back with Symptomatic, a Medical Mystery Podcast.
Now back to Symptomatic, a Medical Mystery Podcast. Casey McPherson
(17:50):
has been told by doctors that there is no treatment
for the rare condition that will continue to plague his
daughter Rose. Time is against him. Is Rosy's condition gains ground,
he feels that he must take matters into his own
hands and do something. Having lost both his brother and
father to suicide in the years prior. Casey was no
(18:12):
stranger to pain and suffering. But he will do what
he has always done, turn that pain into purpose. He
sets out to find a cure for Rose. So where
did you begin.
Speaker 1 (18:26):
There were these, you know, genetic technologies. Some people do
research with them, and you know have created treatments in
the past, but it was never like you, as a
parent had could do that. So I google parent cure's
child of rare disease, and I find a few parents
that actually did. They raised a couple of million dollars,
(18:46):
they hired some researchers, they built a genetic treatment, they
treated their kid in a clinical trial. I'm like, holy crap,
this is actually happening all over the world. And that
gave me a lot of hope that not only was
this possible, people were actually doing it, but not everybody
can raise that kind of money, and I didn't have
that kind of money. I particularly focused on learning from
(19:09):
a woman named Julia Viderello. Julia Viderello and doctor tim
U created the first ever genetic treatment for one person
in the world, for her daughter Mila. They went from
diagnosis to designing the drug and treating her in a
trial in less than a year, and it was the
first time in the world anyone had ever done that,
and so she really helped me get started on the
(19:29):
path of who to talk to and how to think
about it.
Speaker 4 (19:32):
Casey, a professional musician with no background in medicine or biology,
started reaching out to anyone and everyone he could find
to gain insight into the process of building a genetic treatment.
Speaker 1 (19:47):
I probably talked to two or three hundred CEOs of
biotech companies, drug developers, scientists and developed a network of
people that wanted to help. And even Ultrogenics let me
attend their drug development boot camp for four days, which
showed me how to manage a drug development program. I
started taking genetics classes online and I started learning how
(20:10):
people raise money for this.
Speaker 4 (20:13):
Somewhere along as outreach journey, Casey comes across the expert
in molecular genetics and drug development that you heard from earlier,
Rodney Bowling Junior. So how do you explain what you
do to everyday people?
Speaker 3 (20:28):
There are ten thousand different rare diseases. There's another child
born with a new rare disease that we've never seen before.
Every day, a rare disease that will not be addressed
by big pharma population medicine. A child that is as
loved as any other, but with no option for treatment.
(20:52):
So I work to make a treatment for that child.
Speaker 4 (20:56):
So you are a problem solver by nature.
Speaker 3 (20:59):
Well, I am an engineer. I approach medicine with those
engineering practices I learned in school.
Speaker 1 (21:07):
If you're looking for a drug developing scientist, one of
the best folks to look for are these drug hunter
guys that have put these drugs into the clinic, and
they're a different breed. They know biology like you might
know your backyard or your bedroom, like where you could
navigate it in the dark. That's how these guys know biology,
(21:28):
in how cells work and how genetics work.
Speaker 4 (21:31):
When Casey found him, Rodney was the director of molecular
genetics at a biotech company and had been working on
gene targeting projects and drug development for decades. Shooting his
shot as is consistent with Casey's approach to life, Casey
sent him a message.
Speaker 1 (21:49):
I reached out to him on LinkedIn and said.
Speaker 3 (21:51):
My daughter has an alphabet disease. The doctor doesn't know
what it is. I sure as hell don't know what
it is. I see from your credentials that you should
be to help. I worked with a team of scientists
and spoke almost exclusively to them, and you develop a
language that's unique to the sciences, particularly to antibody science.
(22:12):
And here a father called, and I didn't know how
to speak his language, and it just was miserable.
Speaker 1 (22:18):
I wanted to help, so they decided to meet.
Speaker 4 (22:22):
It was a day that would go down in history
for both of them.
Speaker 3 (22:27):
The man I met over lunch is a father who
would do anything and has done everything to help his daughter.
He is charismatic and on a mission. And it is
so easy to fall in behind him and help him
carry that mission. Very quickly, you become a brother. You
(22:47):
want to see him succeed. You need to see him succeed.
That lunch turned into dinner and we started to cure
rose that day.
Speaker 4 (22:56):
Did it surprise you in retrospect that you were so
in a media.
Speaker 3 (23:00):
Honestly, I have six healthy children, and I feel that
I owe a debt, an unpayable debt, to the fact
that I have those six healthy kids.
Speaker 1 (23:11):
I'll remember he had all these papers, you know, about
the disease, and said, oh my god, this is so serious. Casey.
We have to do something about this. He left his
career at ex Biotech. He was just super successful drug developer.
He was offered a ton of money to go to Murk.
He had like over fifteen drugs he had put in
clinical trials. He would tell me, Casey, I'm tired of
(23:33):
making rich people richer. I want to do something that
matters to me, and I want to see these kids
get treated. I'll never forget that day.
Speaker 4 (23:42):
And from that day, Casey and Rodney would work as
a team with a shared mission find a cure for Rose.
With a problem this big to solve. Where do you
even start in science? There's always someone in front of you.
So there was an academic center that had already demonstrated
(24:04):
significant progress and understood a lot more about.
Speaker 3 (24:07):
Rosy's disease than I did. So I read those papers
and went and connected Casey to those people. The scientists
that we were able to work with were incredible and amazing.
The Technology Transfer Office made it impossible to work with them,
so we ended up having to leave that academic center.
(24:27):
From there, I went to a contract research organization. And
this contract research organization had always done excellent work. Their
average contract was probably more than two million dollars.
Speaker 1 (24:39):
But you know, again, not everybody can raise that kind
of money, and I didn't have that kind of money.
Speaker 3 (24:45):
I came to them with a little penny. Any sixty
thousand dollars contract nothing to them, but for Casey that's
all the money he had and asked them to make
me seven cell lines. They told me, okay, we'll have
those for you in six months.
Speaker 4 (24:59):
These cell lines, produced from Rose's own genetics, were vital
to developing a treatment to treat the disease they first
needed to understand it. But six months come and go
and Rodney has still not heard back about the cell
lines that are necessary to progress their research on finding
a treatment. Battling intensifying gastro intestinal issues, looming growth challenges,
(25:24):
and potentially life threatening seizures, they are fighting the clock,
and for Rosie, every moment without a cure counts.
Speaker 3 (25:33):
More than a year later, I called up and was
just screaming at them, where are these what is going on?
And they came back and said, well, honestly, we've lost
half the cell lines and we can't locate them. We
paid thirty thousand dollars up front. I got three of
the seven. I was Okay, well then we've paid you
were done. No, they sent a lawyer after us and
(25:55):
sent us to collections to collect the other thirty thousand
dollars off the cell lines if they never could find.
So I was literally sitting on Casey's front porch crying
into a glass of whiskey that I just I couldn't
make it happen. And Casey said, Rodney, we just need
to start our own lab.
Speaker 1 (26:14):
When I realized that private contract research organizations or academic centers,
none of those were going to have the same urgency
that we had to make these treatments, and other families
needed a place to go, so we spent out this lab.
Speaker 4 (26:29):
Starting their own lab from scratch would be no easy feat.
With their funds already depleted from their unsuccessful partnership with
the contract research organization, Casey and Rodney needed to raise money,
and lots of it. So Casey, the ultimate creative, got resourceful.
Speaker 1 (26:49):
So you know, I started with forming a foundation, Alpharo's Therapeutics,
to initially try to fund that work. The foundation's basically
funded by fans of my band, people from my church,
people that latched onto Rose's story. We launched Rosefest and
so we would raise money with rock and roll shows
(27:09):
and then eventually a golf tournament at Pinehurst, eventually a
Rose Gala at the Barton Creek. And that money went
towards paying for the science to develop a treatment for Rose.
It also went to, you know, gathering that community up
so that we could figure out how to create a
(27:30):
sustainable model.
Speaker 4 (27:33):
The further Casey got down the path to funding a
treatment for Rosie, the more he recognized that his family
was not alone in their desperate need for ultra rare
condition treatments.
Speaker 1 (27:45):
So what I realized was that it'll cost me a
million bucks of safety tests and things to treat Rose.
But to commercialize this medicine will cost between ten and
twenty million because it has to go through a proper
commercial LEFTI a clinical trial. But if I only treat Rose,
what about the kids in Australia, What about the kids
(28:06):
in France? What about the kids in South America that
have this disease? I either could raise just enough money
to treat Rose and it would be stuck in a
perpetual clinical trial forever, or create a system that once
we treated Rose, we would have some sustainable way of
doing this. So parents didn't have to pay for this
crap anymore.
Speaker 4 (28:27):
It was then that Casey and Rodney's mission grew beyond
just saving Rose, but expanded to building a future model
for all families needing a treatment to save their loved one.
Speaker 1 (28:40):
It iterated from can I save my daughter? To now
it's can I fix the system? So now you know,
the families get to own all their intellectual property. We
have a lab we can build these treatments in.
Speaker 4 (28:52):
Now that you have your lab set up, how do
you approach finding a treatment?
Speaker 1 (28:57):
We start with blood.
Speaker 3 (29:00):
The blood is a mixture of a thousand different cell lines,
and we pull out a monocyte. A monocyte is an
immune cell that retains the ability to do multiple things,
and to that we add four genes that create a
stem cell. We finally grew up and differentiated some neurons
(29:22):
from rosy stem cells and added ASOS antisense. Oligos started
with fifty five and ran attack Man assay. Attack Man
assay allows you to measure specifically an RNA that you're
looking for, and in seven of our fifty five asos
we saw it significant progress and that was that was
(29:43):
an amazing moment. We had an embarrassment of riches to
have seven. I couldn't wait to tell Casey.
Speaker 4 (29:50):
So of course, if this were a Hollywood movie, he
would come in, you would tell him the good news,
and then you guys would run and get rosy. But
what's the reality.
Speaker 3 (30:00):
The reality is, we did a pre clinical rat study.
This rat study will ultimately go to the FDA as
proof of safety lack of toxicity of our drug for rosie.
In parallel, we have an h n R NPH two
natural history study going on looking for scientifically measurable ways
(30:22):
to demonstrate that the ASO is in fact working. Does
this affect her walking gate? Is the child seeing any
benefit of the drug? Next we write up an investigational
new drug and hopefully in the coming year, began a
clinical trial to see this drug released into the US
for five hundred give or take patients in the US
(30:45):
and likely five thousand in the world.
Speaker 1 (30:47):
We're very excited. We found a hospital that we can
treat her and some other children in and we're setting
up a proper clinical trial to attempt to get it
approved by the FDA so we can create you know,
gloe Moble access for the drug.
Speaker 4 (31:02):
You have gone from record label rock star to running
a biotech company to being a thought leader in the
drug development space.
Speaker 1 (31:11):
How have people in.
Speaker 4 (31:12):
Your life reacted to that.
Speaker 1 (31:14):
There's a lot of folks that have said, stay in
your lane. You know, you're just a parent, be a
patient advocate. Why do you have to be screwing around
with this stuff? This is our job? And my answer
has been simple, is like I would have loved it
to be your job. I never wanted to quit my
music career, but here we are because someone didn't do
their job, you know, and I'm going to do it
until somebody else does it better than me. I would
(31:36):
be more than happy for someone to take over and
fix this problem. I'm not trying to fix it because
I think I'm some special person. I'm just trying to
fix it because no one has yet. I had to
go learn science. I had to go learn fundraising. I
had to go learn drug development. I had to go
learn self discipline. I had to go learn networking. I
(31:57):
had to go learn how to project estimates and revenue
and expenses. We've worked with over twenty two families. We're
running like eighteen programs. We've made eight new drugs already.
Some of those patients are already their doctors are prescribing
those medicines that we've discovered. I did every single one
of those things for Rose.
Speaker 3 (32:18):
The Casey that I met in twenty nineteen had never
passed high school biology, and now he knows the language.
He's running a pharmaceutical company and is using those connections
and that charisma to cure not only his daughter, but
create a pathway for everyone with rare disease.
Speaker 4 (32:41):
That compassionate, forward thinking leadership harnessed by Casey has resulted
in incredible strides, and only a few years they've already
created a pre clinical treatment for Rose.
Speaker 1 (32:54):
You know, I hope Rose will outlive me, but she
may not. At any case, even as late as we are.
She's nine. Now when I treat her, she's not going
to gain all of her abilities back. She might be
able to talk again, you know, if we're treating her
at eighteen months, potentially she could grow completely normal with
a drug like this, but at this point she won't.
(33:15):
And so you know, we're not going to stop doing
all of this, Rosie and I aren't because we're always
going to be in it. We're always going to be
fighting for this, and I just think if we can
save the next family that comes along. You know that
it's going to make roses suffering and my family's suffering
have that much more value. We can almost wear it
(33:37):
with some pride in that way.
Speaker 4 (33:40):
What do you think actually drives your father at the
end of the day.
Speaker 2 (33:46):
I think what pushes my dad the most to do
all this is being able to first help these kids
and do these amazing things, but also being able to
hear Rosie speak for the first time and knowing the
emotion she's felt. I feel like it's going to be
so happy whenever I get to hear a voice for
the first time.
Speaker 1 (34:08):
I would like to be able to treat her next year,
and I would like to see her be able to
say daddy again. I would like to see her run
in the park and maybe learn how to play with
a friend, you know, maybe learn how to acquire a
skill or a hobby or something that's fun for her
to do.
Speaker 2 (34:28):
People like sometimes they're like, oh, well, if Rosie didn't
have this condition, you'd be able to talk to her.
But sometimes it helps me remember that, like, if she
didn't have the condition, then we wouldn't have helped all
these kids.
Speaker 1 (34:55):
Rose.
Speaker 4 (34:56):
The reason for all of this the daughter that inspired
Casey to just rucked an industry and create positive change.
Rosie touches their lives every single day and reminds them
why they are doing it.
Speaker 1 (35:09):
How is Rose doing today? She is happy. We've got
her seizures under control. We worry about that, but she
is in a pretty happy space in her life, which
is great. After this podcast, I'll go snuggle with her
in bed and I can tickle her and she'll giggle,
and she'll let me kiss her on the cheek, and
(35:30):
you know, I can interact with her. She'll let me
snuggle with her, which is just amazing. Like it's my
favorite part of the day.
Speaker 2 (35:38):
I'm just so proud of my parents and my sister
for pushing through all of this. And even though it
has taken its toll on all of us and like
been a really hard obstacle, I think it's kind of
shaped us into the people we want to be or
the people we are now.
Speaker 4 (35:57):
Is there anything else you want to add, just in
terms of what message you want listeners to take away.
Speaker 3 (36:04):
How easy would it have been for Casey to claim
victimhood and crawled under a rock and hold his daughter
and cry, And that's not what he did. He has fought,
and as we've struggled, he's continued to fight, and he's invented,
literally invented new ways to fight. And his fight has
(36:25):
already helped others, and the team that he's building around
him will continue to find ways invent ways to help others.
This is the way we're going to beat rare disease.
Speaker 1 (36:41):
Cuzy New.
Speaker 2 (36:48):
Sweet can switch subrain on.
Speaker 1 (36:58):
We'll talk well, key walk oh. Because you know, there
was sort of a death of my career and one
of the things that I noticed was that Rose really
could regulate her mood with music, and so every night,
(37:22):
I would, you know, sing to her. Well, I sing
to her every day when she gets really irritated, because
it makes it calms her down. You know, I don't
really play publicly much anymore unless it's for fundraisers and stuff,
but I do for Rosie, you know, and that's strangely
totally enough for me. Last thing I would say is
(37:43):
that we live in this incredible time of technology. This
is more of a civil rights issue than anything. We
have the technology to treat many of these diseases, but
we don't have the systems and business models and support
to do it at any sort of scale yet, And
as we're building that, I think it's important that families
take courage, have the couraged hope, because to have hope
(38:07):
means you think that maybe something could change, and that's
a scary thought when you're trying to accept the reality
of what you're in. And I think it's okay to
completely love your child and accept the reality you're in
and at the same time not stand for it. I
think that's okay.
Speaker 4 (38:40):
You can learn more about Alpha Rose at www dot
Alpha Rose dot com, to Curos Foundation at www dot
Decurros dot org and TCR Rare Labs at tcrrare Labs
dot org, and you can follow Casey at k see
McPherson on Instagram. Coming up on next week's episode of Symptomatic,
(39:07):
what started as intermittent rashes that popped up years apart
turned into excruciating, long term flare ups with no reprieve.
Speaker 5 (39:16):
For Kristin Willard, it feels to me as if someone
has removed all my skin down to like the muscle
and then covered me in a bull blanket.
Speaker 4 (39:27):
Unable to sleep, shower, or even put on pants. Kristen's
quality of life had plummeted, and she could find no
common trigger to explain the debilitating flares she was forced
to endure for months on end. As always, we would
love to hear from you. Send us your thoughts on
(39:47):
this episode, or share a medical mystery of your own
at Symptomatic at iHeartMedia dot com, and please rate and
review Symptomatic wherever you get your podcasts. We'll see you
next time, and until then, be well. Symptomatic a medical
mystery podcast, is a production of iHeartMedia's Ruby Studio. Our
(40:11):
show is hosted by me Lauren Bright Pacheco. Our executive
producers are James Foster, Matt Romano, and myself. Our supervising
producers are Ryan Ovadia, Haley Aliah Ericsson, and Daniel Ainsworth.
This episode was written by Haley Aliah Erickson and edited
by Samuel Richardson.
Ruby. She was screaming uncontrollably, like she was in pain
and confused and angry. You could just see on her face.
It was very, very clear that something was going very wrong.
Speaker 2 (00:18):
When Rosie's thing was more of a new thing to
all of us, I kind of more focused on the
bad parts of it.
Speaker 3 (00:26):
I Am not going to be okay if we're too
late to help Rosie. It has to be Rosie. We
have to get it done in time. For Rosie.
Speaker 1 (00:32):
There's really nothing we can do casing, you know, rather
than make her comfortable good luck.
Speaker 4 (00:40):
How terrifying would it be to fight an unknown enemy,
one you didn't recognize and didn't see coming. What if
that enemy was coming from within a disease that even
doctors couldn't identify. Nearly half of Americans suffered from some
chronic illness, and many struggle for an accurate dify diagnosis.
(01:01):
These are their stories. I'm Lauren Bray Pacheco, and this
is symptomatic. Before Casey McPherson became a father, he was
a rock star as the front man of Indecentsation Flying Colors.
He was used to life on the road.
Speaker 1 (01:21):
I was touring all the time in a band. I
would be on tour for three weeks, six weeks, come
home for a few weeks. You know, you sort of
arrange your life however you saw fit.
Speaker 4 (01:34):
You found incredible success as a musician, but it was
really hard earned. Tell me a little bit about the
early days when you first started out.
Speaker 1 (01:45):
I'd spent over a decade living under the poverty line,
printing CDs in my house and playing to bartenders, you know,
touring in broken down vans. I can't tell you how
many times we broke down or the van caught on
fire or whatever. I remember one place we played said
we drank more than they were going to pay us,
so we owed them money. And I just remember thinking, like,
this is my life, you know. And so there was
(02:07):
a degree of grit. As you know, for artists, there's
an incredible amount of devotion you have to your craft.
I didn't think anything could ever take me out of
the music business. I had a songwriting out of making records.
I never thought I would be doing anything else besides that.
Speaker 4 (02:23):
And then you became a father. What was the experience
of becoming a parent for the first time, like.
Speaker 1 (02:30):
For you, I was in my mid thirties when I
had my first daughter, Weston, And I'll never forget that
day because I was already singing to her, you know,
in the womb, and when she came out, I knew
that there was nothing I wouldn't do for her, and
(02:52):
that my life was not my own anymore. I just knew.
I knew in that moment the meaning of unconditional love.
Speaker 4 (03:02):
And how long after Wes was born was your second
daughter born.
Speaker 1 (03:06):
I think a little under two years that the point
Rose was born, I had a major label record deal,
I had a couple of top ten hits on the radio.
I was touring the world. I'll never forget when Rose
was born. She had the umbilical cord wrapped around her neck,
and you know, it was very scary. I remember holding
this tiny, tiny little girl in my hands and thinking
(03:30):
that my charge now was to keep these little ladies alive,
help make them strong and independent, and you know, accomplish
their dreams.
Speaker 4 (03:41):
Very quickly, Casey and his family began to have concerns
about regression in Rose's development. When looking back, is the
first time that something stuck out to you as maybe
an issue in terms of Rose's health?
Speaker 1 (03:59):
About eighteen months I wasn't really convinced until she lost
her ability to speak, she was learning language. She first
learned a sign, which was normal at her age, and
then she started saying words like mom, dad, and outside.
She point to the door and say outside, and we'd
go outside. But when she stopped talking, it was that
(04:21):
sort of deterioration of language was when I knew this
was not okay. I have the girls, So she's in
(04:51):
the other room. You maybe can hear squeak and squeal
every now and then.
Speaker 4 (04:56):
Today Rosie is nonverbal through sounds and physical cues. Throughout
the episode, we will rely on those closest to her
to share her experience. Though Rose no longer communicates with words,
she is full of personality. Here's her older sister, Weston.
Speaker 1 (05:16):
She loves animals.
Speaker 2 (05:18):
She looks like super shy and like scared, but once
she gets to know her, she's a little bit like
spicy and like a little bit sassy, but she's super
happy all the time whenever she loves. It's one of
the strongest emotions I get from her because that's one
of the only times you know what she's feeling.
Speaker 4 (05:38):
What would you say is her absolute favorite food?
Speaker 2 (05:45):
I think her favorite food is probably pasta. Her favorite
drink is definitely doctor pepper. She's stolen my dad's Doctor
Pepper millions sometimes.
Speaker 4 (05:56):
What are some of your favorite things to do with Rosie?
Speaker 2 (06:00):
I love listening to music with her. I think she's
always been someone who loves music too, whether it's like
watching TV or like listening to my dad play. I
think she just really enjoys like the sound of music
and like the feelings that come with it. I'm so
lucky to have her as my sister, because even though
(06:21):
she can't talk, she's just one of the most amazing
people I've ever met. It beautiful.
Speaker 4 (06:43):
Did you notice any difference initially between Wes as a
baby and Rose as a baby.
Speaker 1 (06:50):
You know, other than personality, not really. You know, at
some point Rose was beginning to experience some issues. You know,
she was having trouble going to the bathroom so I'd
have to give her enemas, and she would struggle with
eating solids. But for the most part, doctors told us,
you know, that's normal. Kids kind of developed, their systems
(07:13):
are getting started in different ways in different times. She
didn't want to walk, and then when she would try
to walk instead of falling on her hands, she would
fall flat on her face without putting her hands in
front of her, So there was this kind of gross
motor lack of response. She was also screaming uncontrollably, like
(07:34):
she was in pain all the time and confused and angry.
You know, you could just see on her face. This
wasn't like I'm tired or I need milk or you know,
it was very very clear that something was going very wrong.
Speaker 4 (07:48):
What did our doctors say when you voiced your concerns?
Did they run any tests or give you any answers.
Speaker 1 (07:57):
So, because our healthcare is some runs off of what
insurance will cover, they have a process, and the process
maybe differs from hospital to hospital, but it's relatively the same.
So they'll say, you know what, We're going to run
a panel and it tests for diseases that currently have treatments.
It doesn't test for diseases that don't. And keep in mind,
(08:20):
there's over ten thousand rare diseases and only five percent
of them have any treatments, so you can imagine how
many are left out on these panels.
Speaker 4 (08:29):
The panel tests come back showing nothing to indicate an
abnormality that could be contributing to Rose's decline. With no
clear pathway laid out by Roses doctors, Casey takes it
upon himself to research what needs to be done next.
Speaker 1 (08:45):
Her mom and I had started kind of doing some research,
and we knew enough about neurodevelopment to know that it's
often caused by genetic mutations. It was clear that she
was having trouble with balance and losing her words. She
struggled with feeding herself. When you know kids at that
point were sort of getting better at that you know,
(09:07):
it was severely missing milestones at this point. In order
to find a genetic mutation, you needed genetic sequencing done,
and at the time, the only one we could afford
was a whole exome sequence because it was ten thousand dollars.
Keep in mind it's two hundred and fifty dollars now.
They sent some boxes to us of swabs and I
(09:28):
had to swab me, swab Mom, swab Weston, swab Rose
and send those back and I think that took a
few months or four months to get all of that.
Speaker 4 (09:39):
The stress of waiting for answers while watching Rose's health
get worse weigh heavily on Casey and his family as
they await a prognosis, Casey and his wife split up.
Can you give us an example of just how serious
things were getting?
Speaker 1 (09:58):
Yeah, you know, she died from her first seizure for
a couple of minutes when mom had to bring her back.
Speaker 4 (10:06):
That must have been terrifying for everyone, almost losing Rosie,
but also still having to wait for answers as to
what was causing all of this. What was that like
for you as a father? How did you handle that?
Speaker 1 (10:26):
I was trying to keep everybody calm, and as so
many ways, I was minimizing, like, hey, it's going to
be okay, She's going to get through this. And you know,
I think anytime you experience unimaginable loss or devastation, there's
sort of a level of unbelief or disbelief, and I
(10:48):
think I sort of had that. So I was doing
the work to try to get to an answer, but
also didn't really think it was going.
Speaker 3 (10:55):
To be this bad.
Speaker 1 (10:56):
And then they emailed us a report, and that's when
I had to schedule another pediatric neurologist appointment so that
he could tell me what I was looking at. I
remember I had both girls, I was alone with them.
I held both their hands, walked them across the parking
(11:17):
lot into the elevator. Rose cannot handle elevators. She was screaming,
makes west and cry, then finally calling my name, going
into the small little room that Rose also didn't like.
I remember the pediatric neurologist walking in with very little
empathy and saying this Jeneta report shows that she's got
(11:40):
a genetic disease, a mutation in the h and rmph
two gene.
Speaker 4 (11:44):
What the neurologist tells them shocks Casey and his family
to the core. Rose's results show a rare mutation on
the h n r n pH two gene of the
H chromosome. The condition is called h n r n
pH two related developmental disorder. At only three years old,
(12:07):
Rose is diagnosed with an untreatable disorder with a devastating prognosis.
Speaker 1 (12:13):
Because this is what happens with these kids. They never
live on their own, and they developed seizures around eight
Some of them have died. We don't know why. We
think it could be the seizures. Some of them have
a few words that they can say, most of them
can't talk. Some end up catatonic and wheelchairs. Some can walk.
So there's this huge variety of what could happen to Rosie,
(12:36):
but one thing was clear. She would never live on
her own, she would never go to college, she would
never get married, or have a job or go to
the movies with her friends. Like that, life was not
going to happen.
Speaker 4 (12:48):
Were you left with any advice? What did the doctors say?
Speaker 1 (12:55):
There's really nothing we can do casing, you know, other
than make her comfortable. Good luck, good luck, and those
words ringing in my ears. I remember being shocked leaving
there with both girls and getting in the van and
feeling super alone.
Speaker 4 (13:18):
Casey is left without answers to deal with a disorder
that is so rare that in twenty twenty one, only
thirty three cases had been documented worldwide. Here is Rodney Bowling, Junior,
an expert in the field of molecular genetics, to explain
the mutation causing Rosie's condition.
Speaker 3 (13:37):
Rosie has a single based permutation in her hn rn
pH two gene very niche. It is a member of
the spliceosome that removes entrons from the premature message to
allow it to be made into messenger RNA. So in
Rosie's case, h n rnph two, I'm just going to
(13:58):
call it H two. A H two reaches out with
one hand and grabs hold of an unprocessed RNA. The
other hand grabs the splicees ome and introduces the RNA
to the splice's ome for processing. In Rosie's case, it
reaches out and grabs the RNA, but there's a great
(14:19):
big trip to fan amino acid right in the middle
of the domain that should introduce and bind the splicesome
that blocks that interaction. It's like putting on a boxing
glove and trying to shake somebody's hand. It's not going
to happen very well. So the protein sequesters more than
four hundred different messages away from being processed, and this
(14:41):
is what causes her disease.
Speaker 1 (14:44):
Rosie definitely has this disease. So I have to accept
that right, and that took some time, Like there's a
grief that you go through when you know that your
child is not going to have the same freedoms and
hopes and dreams and cap abilities that you and I do,
or you know you want that for your children.
Speaker 4 (15:04):
Faced with a challenge she could not fix with the
tools available, Casey began to wonder if he could build
his own.
Speaker 1 (15:12):
Why doesn't she have a treatment. What's the problem. And
that's first sent me down a journey of research. Right,
the veil had sort of been lifted and said, oh,
the hospital can't help my daughter. So I remember the
first metric that really got me was that there were
(15:35):
over four hundred million people with diseases like this, half
of them are children. Thirty percent of those kids die
before their fifth birthday. That's in the tens of millions, right,
and that only five percent of these ten thousand plus
rare diseases actually have treatments. So I had to identify
the monster first, what was the actual problem? And I
(15:56):
realized it wasn't a technology issue for most these diseases,
it was a business model issue. We had created a
healthcare system where we developed drugs for large populations, but
most genetic disease is ulterriy even could be one person
similar to Rose. She didn't have a cure, she didn't
have a treatment because nobody felt like they could make
(16:17):
enough money off of it. Wow mm hmm.
Speaker 4 (16:21):
Just thinking of like sitting in that van with your
two girls, having just been blindsided by the brutal reality
of not only what she's dealing with, but the fact
that no one's offering you hope?
Speaker 2 (16:34):
How did you?
Speaker 4 (16:35):
How did you turn your pain into the path forward?
Speaker 1 (16:41):
Anyone who's experienced immense tragedy in their life or suffering
or loss, I'd lost both my brother and father to suicide,
I think understands you kind of have two options. You
have an option to continue your suffering by stuffing it
down or or hiding it away or running away, or
(17:06):
you find some way to create purpose out of it.
I had to figure out how to turn pain into purpose.
But I knew nothing about genetics. I certainly didn't know
anything about ner developmental disease, you know, being a rock
and roll guy, having fallen asleep in biology class in
high school. I'm like, am I smart enough to do
(17:27):
something about this?
Speaker 4 (17:29):
We'll be right back with Symptomatic, a Medical Mystery Podcast.
Now back to Symptomatic, a Medical Mystery Podcast. Casey McPherson
(17:50):
has been told by doctors that there is no treatment
for the rare condition that will continue to plague his
daughter Rose. Time is against him. Is Rosy's condition gains ground,
he feels that he must take matters into his own
hands and do something. Having lost both his brother and
father to suicide in the years prior. Casey was no
(18:12):
stranger to pain and suffering. But he will do what
he has always done, turn that pain into purpose. He
sets out to find a cure for Rose. So where
did you begin.
Speaker 1 (18:26):
There were these, you know, genetic technologies. Some people do
research with them, and you know have created treatments in
the past, but it was never like you, as a
parent had could do that. So I google parent cure's
child of rare disease, and I find a few parents
that actually did. They raised a couple of million dollars,
(18:46):
they hired some researchers, they built a genetic treatment, they
treated their kid in a clinical trial. I'm like, holy crap,
this is actually happening all over the world. And that
gave me a lot of hope that not only was
this possible, people were actually doing it, but not everybody
can raise that kind of money, and I didn't have
that kind of money. I particularly focused on learning from
(19:09):
a woman named Julia Viderello. Julia Viderello and doctor tim
U created the first ever genetic treatment for one person
in the world, for her daughter Mila. They went from
diagnosis to designing the drug and treating her in a
trial in less than a year, and it was the
first time in the world anyone had ever done that,
and so she really helped me get started on the
(19:29):
path of who to talk to and how to think
about it.
Speaker 4 (19:32):
Casey, a professional musician with no background in medicine or biology,
started reaching out to anyone and everyone he could find
to gain insight into the process of building a genetic treatment.
Speaker 1 (19:47):
I probably talked to two or three hundred CEOs of
biotech companies, drug developers, scientists and developed a network of
people that wanted to help. And even Ultrogenics let me
attend their drug development boot camp for four days, which
showed me how to manage a drug development program. I
started taking genetics classes online and I started learning how
(20:10):
people raise money for this.
Speaker 4 (20:13):
Somewhere along as outreach journey, Casey comes across the expert
in molecular genetics and drug development that you heard from earlier,
Rodney Bowling Junior. So how do you explain what you
do to everyday people?
Speaker 3 (20:28):
There are ten thousand different rare diseases. There's another child
born with a new rare disease that we've never seen before.
Every day, a rare disease that will not be addressed
by big pharma population medicine. A child that is as
loved as any other, but with no option for treatment.
(20:52):
So I work to make a treatment for that child.
Speaker 4 (20:56):
So you are a problem solver by nature.
Speaker 3 (20:59):
Well, I am an engineer. I approach medicine with those
engineering practices I learned in school.
Speaker 1 (21:07):
If you're looking for a drug developing scientist, one of
the best folks to look for are these drug hunter
guys that have put these drugs into the clinic, and
they're a different breed. They know biology like you might
know your backyard or your bedroom, like where you could
navigate it in the dark. That's how these guys know biology,
(21:28):
in how cells work and how genetics work.
Speaker 4 (21:31):
When Casey found him, Rodney was the director of molecular
genetics at a biotech company and had been working on
gene targeting projects and drug development for decades. Shooting his
shot as is consistent with Casey's approach to life, Casey
sent him a message.
Speaker 1 (21:49):
I reached out to him on LinkedIn and said.
Speaker 3 (21:51):
My daughter has an alphabet disease. The doctor doesn't know
what it is. I sure as hell don't know what
it is. I see from your credentials that you should
be to help. I worked with a team of scientists
and spoke almost exclusively to them, and you develop a
language that's unique to the sciences, particularly to antibody science.
(22:12):
And here a father called, and I didn't know how
to speak his language, and it just was miserable.
Speaker 1 (22:18):
I wanted to help, so they decided to meet.
Speaker 4 (22:22):
It was a day that would go down in history
for both of them.
Speaker 3 (22:27):
The man I met over lunch is a father who
would do anything and has done everything to help his daughter.
He is charismatic and on a mission. And it is
so easy to fall in behind him and help him
carry that mission. Very quickly, you become a brother. You
(22:47):
want to see him succeed. You need to see him succeed.
That lunch turned into dinner and we started to cure
rose that day.
Speaker 4 (22:56):
Did it surprise you in retrospect that you were so
in a media.
Speaker 3 (23:00):
Honestly, I have six healthy children, and I feel that
I owe a debt, an unpayable debt, to the fact
that I have those six healthy kids.
Speaker 1 (23:11):
I'll remember he had all these papers, you know, about
the disease, and said, oh my god, this is so serious. Casey.
We have to do something about this. He left his
career at ex Biotech. He was just super successful drug developer.
He was offered a ton of money to go to Murk.
He had like over fifteen drugs he had put in
clinical trials. He would tell me, Casey, I'm tired of
(23:33):
making rich people richer. I want to do something that
matters to me, and I want to see these kids
get treated. I'll never forget that day.
Speaker 4 (23:42):
And from that day, Casey and Rodney would work as
a team with a shared mission find a cure for Rose.
With a problem this big to solve. Where do you
even start in science? There's always someone in front of you.
So there was an academic center that had already demonstrated
(24:04):
significant progress and understood a lot more about.
Speaker 3 (24:07):
Rosy's disease than I did. So I read those papers
and went and connected Casey to those people. The scientists
that we were able to work with were incredible and amazing.
The Technology Transfer Office made it impossible to work with them,
so we ended up having to leave that academic center.
(24:27):
From there, I went to a contract research organization. And
this contract research organization had always done excellent work. Their
average contract was probably more than two million dollars.
Speaker 1 (24:39):
But you know, again, not everybody can raise that kind
of money, and I didn't have that kind of money.
Speaker 3 (24:45):
I came to them with a little penny. Any sixty
thousand dollars contract nothing to them, but for Casey that's
all the money he had and asked them to make
me seven cell lines. They told me, okay, we'll have
those for you in six months.
Speaker 4 (24:59):
These cell lines, produced from Rose's own genetics, were vital
to developing a treatment to treat the disease they first
needed to understand it. But six months come and go
and Rodney has still not heard back about the cell
lines that are necessary to progress their research on finding
a treatment. Battling intensifying gastro intestinal issues, looming growth challenges,
(25:24):
and potentially life threatening seizures, they are fighting the clock,
and for Rosie, every moment without a cure counts.
Speaker 3 (25:33):
More than a year later, I called up and was
just screaming at them, where are these what is going on?
And they came back and said, well, honestly, we've lost
half the cell lines and we can't locate them. We
paid thirty thousand dollars up front. I got three of
the seven. I was Okay, well then we've paid you
were done. No, they sent a lawyer after us and
(25:55):
sent us to collections to collect the other thirty thousand
dollars off the cell lines if they never could find.
So I was literally sitting on Casey's front porch crying
into a glass of whiskey that I just I couldn't
make it happen. And Casey said, Rodney, we just need
to start our own lab.
Speaker 1 (26:14):
When I realized that private contract research organizations or academic centers,
none of those were going to have the same urgency
that we had to make these treatments, and other families
needed a place to go, so we spent out this lab.
Speaker 4 (26:29):
Starting their own lab from scratch would be no easy feat.
With their funds already depleted from their unsuccessful partnership with
the contract research organization, Casey and Rodney needed to raise money,
and lots of it. So Casey, the ultimate creative, got resourceful.
Speaker 1 (26:49):
So you know, I started with forming a foundation, Alpharo's Therapeutics,
to initially try to fund that work. The foundation's basically
funded by fans of my band, people from my church,
people that latched onto Rose's story. We launched Rosefest and
so we would raise money with rock and roll shows
(27:09):
and then eventually a golf tournament at Pinehurst, eventually a
Rose Gala at the Barton Creek. And that money went
towards paying for the science to develop a treatment for Rose.
It also went to, you know, gathering that community up
so that we could figure out how to create a
(27:30):
sustainable model.
Speaker 4 (27:33):
The further Casey got down the path to funding a
treatment for Rosie, the more he recognized that his family
was not alone in their desperate need for ultra rare
condition treatments.
Speaker 1 (27:45):
So what I realized was that it'll cost me a
million bucks of safety tests and things to treat Rose.
But to commercialize this medicine will cost between ten and
twenty million because it has to go through a proper
commercial LEFTI a clinical trial. But if I only treat Rose,
what about the kids in Australia, What about the kids
(28:06):
in France? What about the kids in South America that
have this disease? I either could raise just enough money
to treat Rose and it would be stuck in a
perpetual clinical trial forever, or create a system that once
we treated Rose, we would have some sustainable way of
doing this. So parents didn't have to pay for this
crap anymore.
Speaker 4 (28:27):
It was then that Casey and Rodney's mission grew beyond
just saving Rose, but expanded to building a future model
for all families needing a treatment to save their loved one.
Speaker 1 (28:40):
It iterated from can I save my daughter? To now
it's can I fix the system? So now you know,
the families get to own all their intellectual property. We
have a lab we can build these treatments in.
Speaker 4 (28:52):
Now that you have your lab set up, how do
you approach finding a treatment?
Speaker 1 (28:57):
We start with blood.
Speaker 3 (29:00):
The blood is a mixture of a thousand different cell lines,
and we pull out a monocyte. A monocyte is an
immune cell that retains the ability to do multiple things,
and to that we add four genes that create a
stem cell. We finally grew up and differentiated some neurons
(29:22):
from rosy stem cells and added ASOS antisense. Oligos started
with fifty five and ran attack Man assay. Attack Man
assay allows you to measure specifically an RNA that you're
looking for, and in seven of our fifty five asos
we saw it significant progress and that was that was
(29:43):
an amazing moment. We had an embarrassment of riches to
have seven. I couldn't wait to tell Casey.
Speaker 4 (29:50):
So of course, if this were a Hollywood movie, he
would come in, you would tell him the good news,
and then you guys would run and get rosy. But
what's the reality.
Speaker 3 (30:00):
The reality is, we did a pre clinical rat study.
This rat study will ultimately go to the FDA as
proof of safety lack of toxicity of our drug for rosie.
In parallel, we have an h n R NPH two
natural history study going on looking for scientifically measurable ways
(30:22):
to demonstrate that the ASO is in fact working. Does
this affect her walking gate? Is the child seeing any
benefit of the drug? Next we write up an investigational
new drug and hopefully in the coming year, began a
clinical trial to see this drug released into the US
for five hundred give or take patients in the US
(30:45):
and likely five thousand in the world.
Speaker 1 (30:47):
We're very excited. We found a hospital that we can
treat her and some other children in and we're setting
up a proper clinical trial to attempt to get it
approved by the FDA so we can create you know,
gloe Moble access for the drug.
Speaker 4 (31:02):
You have gone from record label rock star to running
a biotech company to being a thought leader in the
drug development space.
Speaker 1 (31:11):
How have people in.
Speaker 4 (31:12):
Your life reacted to that.
Speaker 1 (31:14):
There's a lot of folks that have said, stay in
your lane. You know, you're just a parent, be a
patient advocate. Why do you have to be screwing around
with this stuff? This is our job? And my answer
has been simple, is like I would have loved it
to be your job. I never wanted to quit my
music career, but here we are because someone didn't do
their job, you know, and I'm going to do it
until somebody else does it better than me. I would
(31:36):
be more than happy for someone to take over and
fix this problem. I'm not trying to fix it because
I think I'm some special person. I'm just trying to
fix it because no one has yet. I had to
go learn science. I had to go learn fundraising. I
had to go learn drug development. I had to go
learn self discipline. I had to go learn networking. I
(31:57):
had to go learn how to project estimates and revenue
and expenses. We've worked with over twenty two families. We're
running like eighteen programs. We've made eight new drugs already.
Some of those patients are already their doctors are prescribing
those medicines that we've discovered. I did every single one
of those things for Rose.
Speaker 3 (32:18):
The Casey that I met in twenty nineteen had never
passed high school biology, and now he knows the language.
He's running a pharmaceutical company and is using those connections
and that charisma to cure not only his daughter, but
create a pathway for everyone with rare disease.
Speaker 4 (32:41):
That compassionate, forward thinking leadership harnessed by Casey has resulted
in incredible strides, and only a few years they've already
created a pre clinical treatment for Rose.
Speaker 1 (32:54):
You know, I hope Rose will outlive me, but she
may not. At any case, even as late as we are.
She's nine. Now when I treat her, she's not going
to gain all of her abilities back. She might be
able to talk again, you know, if we're treating her
at eighteen months, potentially she could grow completely normal with
a drug like this, but at this point she won't.
(33:15):
And so you know, we're not going to stop doing
all of this, Rosie and I aren't because we're always
going to be in it. We're always going to be
fighting for this, and I just think if we can
save the next family that comes along. You know that
it's going to make roses suffering and my family's suffering
have that much more value. We can almost wear it
(33:37):
with some pride in that way.
Speaker 4 (33:40):
What do you think actually drives your father at the
end of the day.
Speaker 2 (33:46):
I think what pushes my dad the most to do
all this is being able to first help these kids
and do these amazing things, but also being able to
hear Rosie speak for the first time and knowing the
emotion she's felt. I feel like it's going to be
so happy whenever I get to hear a voice for
the first time.
Speaker 1 (34:08):
I would like to be able to treat her next year,
and I would like to see her be able to
say daddy again. I would like to see her run
in the park and maybe learn how to play with
a friend, you know, maybe learn how to acquire a
skill or a hobby or something that's fun for her
to do.
Speaker 2 (34:28):
People like sometimes they're like, oh, well, if Rosie didn't
have this condition, you'd be able to talk to her.
But sometimes it helps me remember that, like, if she
didn't have the condition, then we wouldn't have helped all
these kids.
Speaker 1 (34:55):
Rose.
Speaker 4 (34:56):
The reason for all of this the daughter that inspired
Casey to just rucked an industry and create positive change.
Rosie touches their lives every single day and reminds them
why they are doing it.
Speaker 1 (35:09):
How is Rose doing today? She is happy. We've got
her seizures under control. We worry about that, but she
is in a pretty happy space in her life, which
is great. After this podcast, I'll go snuggle with her
in bed and I can tickle her and she'll giggle,
and she'll let me kiss her on the cheek, and
(35:30):
you know, I can interact with her. She'll let me
snuggle with her, which is just amazing. Like it's my
favorite part of the day.
Speaker 2 (35:38):
I'm just so proud of my parents and my sister
for pushing through all of this. And even though it
has taken its toll on all of us and like
been a really hard obstacle, I think it's kind of
shaped us into the people we want to be or
the people we are now.
Speaker 4 (35:57):
Is there anything else you want to add, just in
terms of what message you want listeners to take away.
Speaker 3 (36:04):
How easy would it have been for Casey to claim
victimhood and crawled under a rock and hold his daughter
and cry, And that's not what he did. He has fought,
and as we've struggled, he's continued to fight, and he's invented,
literally invented new ways to fight. And his fight has
(36:25):
already helped others, and the team that he's building around
him will continue to find ways invent ways to help others.
This is the way we're going to beat rare disease.
Speaker 1 (36:41):
Cuzy New.
Speaker 2 (36:48):
Sweet can switch subrain on.
Speaker 1 (36:58):
We'll talk well, key walk oh. Because you know, there
was sort of a death of my career and one
of the things that I noticed was that Rose really
could regulate her mood with music, and so every night,
(37:22):
I would, you know, sing to her. Well, I sing
to her every day when she gets really irritated, because
it makes it calms her down. You know, I don't
really play publicly much anymore unless it's for fundraisers and stuff,
but I do for Rosie, you know, and that's strangely
totally enough for me. Last thing I would say is
(37:43):
that we live in this incredible time of technology. This
is more of a civil rights issue than anything. We
have the technology to treat many of these diseases, but
we don't have the systems and business models and support
to do it at any sort of scale yet, And
as we're building that, I think it's important that families
take courage, have the couraged hope, because to have hope
(38:07):
means you think that maybe something could change, and that's
a scary thought when you're trying to accept the reality
of what you're in. And I think it's okay to
completely love your child and accept the reality you're in
and at the same time not stand for it. I
think that's okay.
Speaker 4 (38:40):
You can learn more about Alpha Rose at www dot
Alpha Rose dot com, to Curos Foundation at www dot
Decurros dot org and TCR Rare Labs at tcrrare Labs
dot org, and you can follow Casey at k see
McPherson on Instagram. Coming up on next week's episode of Symptomatic,
(39:07):
what started as intermittent rashes that popped up years apart
turned into excruciating, long term flare ups with no reprieve.
Speaker 5 (39:16):
For Kristin Willard, it feels to me as if someone
has removed all my skin down to like the muscle
and then covered me in a bull blanket.
Speaker 4 (39:27):
Unable to sleep, shower, or even put on pants. Kristen's
quality of life had plummeted, and she could find no
common trigger to explain the debilitating flares she was forced
to endure for months on end. As always, we would
love to hear from you. Send us your thoughts on
(39:47):
this episode, or share a medical mystery of your own
at Symptomatic at iHeartMedia dot com, and please rate and
review Symptomatic wherever you get your podcasts. We'll see you
next time, and until then, be well. Symptomatic a medical
mystery podcast, is a production of iHeartMedia's Ruby Studio. Our
(40:11):
show is hosted by me Lauren Bright Pacheco. Our executive
producers are James Foster, Matt Romano, and myself. Our supervising
producers are Ryan Ovadia, Haley Aliah Ericsson, and Daniel Ainsworth.
This episode was written by Haley Aliah Erickson and edited
by Samuel Richardson.
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