July 1, 2026 • 8 mins
Do you know the story of Kendra Riley? She joined Preston to share and discuss important legislation.
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Speaker 1 (00:00):
As promised, we have with us Kendra Riley. Kendra is
a business owner. She's the mom of three girls, two
of whom were diagnosed with a condition called MLD. She'll
explain in a few moments back in twenty twenty. See
she has since become an advocate for other rare disease
families like hers across the nation. Kendra, welcome to the program.
(00:23):
Tell us about MLD and your daughters.
Speaker 2 (00:27):
Yeah, thanks for having me. So, MLD quite a rare
terminal illness. And in twenty twenty when we got our
first daughter's diagnosis, no treatment, no cure, average lifespan of
age six, and we found out when she was a
year and a half. It's very aggressive, so within the
(00:48):
first ninety days of diagnosis she lost the ability to walk,
then talk, then tube fed. Fast forward to today, she's
now eight years old and in hospice, whereas our second
daughter that got diagnosed that same year, had a very
different outcome.
Speaker 1 (01:06):
Tell us why.
Speaker 2 (01:08):
We actually found out that there was a experimental gene
therapy treatment only available in Milan, Italy, that could potentially
save her life. It couldn't work for our first daughter
because she was symptomatic and they've already tried in clinical
trials and it didn't work. But our other daughter was
a newborn at the time and not yet symptomatic. Come
(01:30):
to find out, under the original Right to Try law,
we could not get this treatment in the US, and
we had one month's time to raise half a million
dollars and move our family to Italy during the pandemic
to get her this treatment. So we did just that
and she became the thirty second child in the world
to have it, and she's now a happy, healthy six
(01:52):
year old.
Speaker 1 (01:54):
That is amazing news. You are right now living the
example though, of a before and and after and a
difference that a treatment can make. Why is it that
a treatment like that is not available in the United States?
Speaker 2 (02:15):
Great question, And you know, science and technology is moving
so much faster, much faster than the speed of bureaucracy
would but it was questionable. Why you know, in America
we can't have access to something that could potentially save
our life. Still that question baffles me. But I know
so many more gene therapies are coming out that are
(02:37):
tailored to people's DNA like this one. So hopefully with
Right to Try two point zero, this will change all
of that and any rare disease patient can get access
to a potentially life saving treatment.
Speaker 1 (02:48):
Before we get to that, let let's talk about right
to try the first version. Where did it fall short?
What was the intent and what ended up happening and
why did it fall short?
Speaker 2 (03:02):
Yeah, that was great, and it was to help families
like ours and patients like my daughters who can have
a chance and a little bit of hope instead of
heartbreak when it comes to a fatal diagnosis and having
an opportunity to live a little bit longer, if not,
you know, live a full life. But unfortunately, it just
didn't cover these very specific treatments tailored to someone's DNA
(03:27):
because I mean really, they weren't around at that at
that time. So fast forward to twenty twenty six and they,
you know, they've realized, Wow, we really do need to
include something like this because it's becoming more and more
common for rare to these patients to be able to
have these treatments.
Speaker 1 (03:44):
If you're a parent, a grandparent, this has to touch
your heart. Before we get to some of the politics
involved here and some of the questions I asked before
going to the break, Kendra tell Us tell us, how
care is processing the fact that she has been ostensibly
cured and her older sister is not.
Speaker 2 (04:06):
You know, she's come to realize that she's gotten older,
kind of the process that we had to go through,
and so we've had to answer over time, you know,
why did I get the treatment and not Livy? You know,
what does every kid have MLD? Why did that kid
not get a treatment? So there's a lot of things,
you know, as kids grow up and they start realizing
(04:27):
what's going on and why do I have to go
back to Italy for checkups? So over time we've had
to kind of explain why Livy wasn't able to get
the treatment, why she was you know, and so often
she says, you know, I wish Livy got the treatment too,
so she could like play with us and go to school, YadA, YadA.
So you know, she's she's realizing more and more the
(04:49):
ramifications of what's happened to our family and what she
sees every day and why she's seeing it, and coming
to realize that her situation is very rare and not
every kid is like her. But to a lot of
her friends at school, you know, they have no clue.
They just think she's a normal six year old girls
because she has zero symptoms. So it'll be interesting as
she continues to develop and how that impacts you know,
(05:13):
how that will be impacting her and maybe even what
she does in the future.
Speaker 1 (05:17):
Kendra, I mentioned my visit with a regular doctor that
we have on the program who has been in the
cancer field for his entire career and is retired. But
he talked about how, you know, with the geoblastoma development,
and I asked him if that development, which was done
at Shan's Hospital at the University of Florida, if that
(05:38):
was going to be for example, shared with, say the
Saint Jude's Children's Hospital. He said, inside the United States,
those types of treatments are shared widely, that there's always collaboration.
Is the treatment for MLD is it being shared or
is it still only found in Italy?
Speaker 2 (05:59):
Yes, it is being shared. So it was first approved
in Europe, and then my family as well as other
families who have benefitted from this treatment did testify with
the SDA. We got it FDA approved a few years ago,
and then just in December twenty twenty five, we went
to DC and Secretary Kennedy did add MLD to nationwide
(06:23):
recommended newborn screening. So that'll make a huge difference for
families because now they'll know right when their child's born
that they have it and can immediately get them this
treatment which is now being given at multiple hospitals across
the nation.
Speaker 1 (06:37):
So let's talk about the unfortunate politics that's involved in
this journey here. You mentioned we're at the second effort
on the right to try. Who's behind the effort in Congress?
Speaker 2 (06:50):
Yeah, so it was introduced by Senator Ron Johnson from
Wisconsin and Representative Danna Harshburger from Tennessee.
Speaker 1 (06:56):
And what's the pathway? Where is it in the process?
What needs to happen? Is it getting bipartisan support? And
by bipartisan I live in that world every day with
doing this show. I'm not talking one person on the
other side of the aisle. Is this getting full support
on both sides of the aisle?
Speaker 2 (07:15):
Yes, so far. So when they first reformed the bill,
that was in twenty twenty one, when our family got
back from Italy and they said, you know, you've inspired
us to make some changes here because we realize where
science is moving. So I did share my story, our
girl's story at the Arizona State Capital first where it
was initially passed, and it's now passed in seventeen other states,
(07:38):
and then it's just got introduced to Congress earlier this month.
So far, in all the states that have passed it,
it's been a pretty unanimous yes. Most people don't see
why we wouldn't make this change and help people save
their own lives.
Speaker 1 (07:56):
What can people listening to the program do to help
this produces sess?
Speaker 2 (08:01):
I would say, you know, right to your state representatives
in Congress, let them know how important this is. Hearing
stories like ours that are first person from families and
kids that are dealing with this and could potentially have
their life saved from a treatment that they'll now be
able to get in the United States will make a
big difference. It's all about hearing those stories.
As promised, we have with us Kendra Riley. Kendra is
a business owner. She's the mom of three girls, two
of whom were diagnosed with a condition called MLD. She'll
explain in a few moments back in twenty twenty. See
she has since become an advocate for other rare disease
families like hers across the nation. Kendra, welcome to the program.
(00:23):
Tell us about MLD and your daughters.
Speaker 2 (00:27):
Yeah, thanks for having me. So, MLD quite a rare
terminal illness. And in twenty twenty when we got our
first daughter's diagnosis, no treatment, no cure, average lifespan of
age six, and we found out when she was a
year and a half. It's very aggressive, so within the
(00:48):
first ninety days of diagnosis she lost the ability to walk,
then talk, then tube fed. Fast forward to today, she's
now eight years old and in hospice, whereas our second
daughter that got diagnosed that same year, had a very
different outcome.
Speaker 1 (01:06):
Tell us why.
Speaker 2 (01:08):
We actually found out that there was a experimental gene
therapy treatment only available in Milan, Italy, that could potentially
save her life. It couldn't work for our first daughter
because she was symptomatic and they've already tried in clinical
trials and it didn't work. But our other daughter was
a newborn at the time and not yet symptomatic. Come
(01:30):
to find out, under the original Right to Try law,
we could not get this treatment in the US, and
we had one month's time to raise half a million
dollars and move our family to Italy during the pandemic
to get her this treatment. So we did just that
and she became the thirty second child in the world
to have it, and she's now a happy, healthy six
(01:52):
year old.
Speaker 1 (01:54):
That is amazing news. You are right now living the
example though, of a before and and after and a
difference that a treatment can make. Why is it that
a treatment like that is not available in the United States?
Speaker 2 (02:15):
Great question, And you know, science and technology is moving
so much faster, much faster than the speed of bureaucracy
would but it was questionable. Why you know, in America
we can't have access to something that could potentially save
our life. Still that question baffles me. But I know
so many more gene therapies are coming out that are
(02:37):
tailored to people's DNA like this one. So hopefully with
Right to Try two point zero, this will change all
of that and any rare disease patient can get access
to a potentially life saving treatment.
Speaker 1 (02:48):
Before we get to that, let let's talk about right
to try the first version. Where did it fall short?
What was the intent and what ended up happening and
why did it fall short?
Speaker 2 (03:02):
Yeah, that was great, and it was to help families
like ours and patients like my daughters who can have
a chance and a little bit of hope instead of
heartbreak when it comes to a fatal diagnosis and having
an opportunity to live a little bit longer, if not,
you know, live a full life. But unfortunately, it just
didn't cover these very specific treatments tailored to someone's DNA
(03:27):
because I mean really, they weren't around at that at
that time. So fast forward to twenty twenty six and they,
you know, they've realized, Wow, we really do need to
include something like this because it's becoming more and more
common for rare to these patients to be able to
have these treatments.
Speaker 1 (03:44):
If you're a parent, a grandparent, this has to touch
your heart. Before we get to some of the politics
involved here and some of the questions I asked before
going to the break, Kendra tell Us tell us, how
care is processing the fact that she has been ostensibly
cured and her older sister is not.
Speaker 2 (04:06):
You know, she's come to realize that she's gotten older,
kind of the process that we had to go through,
and so we've had to answer over time, you know,
why did I get the treatment and not Livy? You know,
what does every kid have MLD? Why did that kid
not get a treatment? So there's a lot of things,
you know, as kids grow up and they start realizing
(04:27):
what's going on and why do I have to go
back to Italy for checkups? So over time we've had
to kind of explain why Livy wasn't able to get
the treatment, why she was you know, and so often
she says, you know, I wish Livy got the treatment too,
so she could like play with us and go to school, YadA, YadA.
So you know, she's she's realizing more and more the
(04:49):
ramifications of what's happened to our family and what she
sees every day and why she's seeing it, and coming
to realize that her situation is very rare and not
every kid is like her. But to a lot of
her friends at school, you know, they have no clue.
They just think she's a normal six year old girls
because she has zero symptoms. So it'll be interesting as
she continues to develop and how that impacts you know,
(05:13):
how that will be impacting her and maybe even what
she does in the future.
Speaker 1 (05:17):
Kendra, I mentioned my visit with a regular doctor that
we have on the program who has been in the
cancer field for his entire career and is retired. But
he talked about how, you know, with the geoblastoma development,
and I asked him if that development, which was done
at Shan's Hospital at the University of Florida, if that
(05:38):
was going to be for example, shared with, say the
Saint Jude's Children's Hospital. He said, inside the United States,
those types of treatments are shared widely, that there's always collaboration.
Is the treatment for MLD is it being shared or
is it still only found in Italy?
Speaker 2 (05:59):
Yes, it is being shared. So it was first approved
in Europe, and then my family as well as other
families who have benefitted from this treatment did testify with
the SDA. We got it FDA approved a few years ago,
and then just in December twenty twenty five, we went
to DC and Secretary Kennedy did add MLD to nationwide
(06:23):
recommended newborn screening. So that'll make a huge difference for
families because now they'll know right when their child's born
that they have it and can immediately get them this
treatment which is now being given at multiple hospitals across
the nation.
Speaker 1 (06:37):
So let's talk about the unfortunate politics that's involved in
this journey here. You mentioned we're at the second effort
on the right to try. Who's behind the effort in Congress?
Speaker 2 (06:50):
Yeah, so it was introduced by Senator Ron Johnson from
Wisconsin and Representative Danna Harshburger from Tennessee.
Speaker 1 (06:56):
And what's the pathway? Where is it in the process?
What needs to happen? Is it getting bipartisan support? And
by bipartisan I live in that world every day with
doing this show. I'm not talking one person on the
other side of the aisle. Is this getting full support
on both sides of the aisle?
Speaker 2 (07:15):
Yes, so far. So when they first reformed the bill,
that was in twenty twenty one, when our family got
back from Italy and they said, you know, you've inspired
us to make some changes here because we realize where
science is moving. So I did share my story, our
girl's story at the Arizona State Capital first where it
was initially passed, and it's now passed in seventeen other states,
(07:38):
and then it's just got introduced to Congress earlier this month.
So far, in all the states that have passed it,
it's been a pretty unanimous yes. Most people don't see
why we wouldn't make this change and help people save
their own lives.
Speaker 1 (07:56):
What can people listening to the program do to help
this produces sess?
Speaker 2 (08:01):
I would say, you know, right to your state representatives
in Congress, let them know how important this is. Hearing
stories like ours that are first person from families and
kids that are dealing with this and could potentially have
their life saved from a treatment that they'll now be
able to get in the United States will make a
big difference. It's all about hearing those stories.
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