Latest / Conversations: Special Guests with Preston Scott / The "Right To Try Act 2.0"

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The "Right To Try Act 2.0" · Conversations: Special Guests with Preston Scott

• 0:00
  Speaker 1: As promised, we have with us Kendra Riley. Kendra is
• 0:04
  Speaker 1: a business owner. She's the mom of three girls, two
• 0:06
  Speaker 1: of whom were diagnosed with a condition called MLD. She'll
• 0:11
  Speaker 1: explain in a few moments back in twenty twenty. See
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  Speaker 1: she has since become an advocate for other rare disease
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  Speaker 1: families like hers across the nation. Kendra, welcome to the program.
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  Speaker 1: Tell us about MLD and your daughters.
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  Speaker 2: Yeah, thanks for having me. So, MLD quite a rare
• 0:31
  Speaker 2: terminal illness. And in twenty twenty when we got our
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  Speaker 2: first daughter's diagnosis, no treatment, no cure, average lifespan of
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  Speaker 2: age six, and we found out when she was a
• 0:44
  Speaker 2: year and a half. It's very aggressive, so within the
• 0:48
  Speaker 2: first ninety days of diagnosis she lost the ability to walk,
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  Speaker 2: then talk, then tube fed. Fast forward to today, she's
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  Speaker 2: now eight years old and in hospice, whereas our second
• 1:01
  Speaker 2: daughter that got diagnosed that same year, had a very
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  Speaker 2: different outcome.
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  Speaker 1: Tell us why.
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  Speaker 2: We actually found out that there was a experimental gene
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  Speaker 2: therapy treatment only available in Milan, Italy, that could potentially
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  Speaker 2: save her life. It couldn't work for our first daughter
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  Speaker 2: because she was symptomatic and they've already tried in clinical
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  Speaker 2: trials and it didn't work. But our other daughter was
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  Speaker 2: a newborn at the time and not yet symptomatic. Come
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  Speaker 2: to find out, under the original Right to Try law,
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  Speaker 2: we could not get this treatment in the US, and
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  Speaker 2: we had one month's time to raise half a million
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  Speaker 2: dollars and move our family to Italy during the pandemic
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  Speaker 2: to get her this treatment. So we did just that
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  Speaker 2: and she became the thirty second child in the world
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  Speaker 2: to have it, and she's now a happy, healthy six
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  Speaker 2: year old.
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  Speaker 1: That is amazing news. You are right now living the
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  Speaker 1: example though, of a before and and after and a
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  Speaker 1: difference that a treatment can make. Why is it that
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  Speaker 1: a treatment like that is not available in the United States?
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  Speaker 2: Great question, And you know, science and technology is moving
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  Speaker 2: so much faster, much faster than the speed of bureaucracy
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  Speaker 2: would but it was questionable. Why you know, in America
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  Speaker 2: we can't have access to something that could potentially save
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  Speaker 2: our life. Still that question baffles me. But I know
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  Speaker 2: so many more gene therapies are coming out that are
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  Speaker 2: tailored to people's DNA like this one. So hopefully with
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  Speaker 2: Right to Try two point zero, this will change all
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  Speaker 2: of that and any rare disease patient can get access
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  Speaker 2: to a potentially life saving treatment.
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  Speaker 1: Before we get to that, let let's talk about right
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  Speaker 1: to try the first version. Where did it fall short?
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  Speaker 1: What was the intent and what ended up happening and
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  Speaker 1: why did it fall short?
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  Speaker 2: Yeah, that was great, and it was to help families
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  Speaker 2: like ours and patients like my daughters who can have
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  Speaker 2: a chance and a little bit of hope instead of
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  Speaker 2: heartbreak when it comes to a fatal diagnosis and having
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  Speaker 2: an opportunity to live a little bit longer, if not,
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  Speaker 2: you know, live a full life. But unfortunately, it just
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  Speaker 2: didn't cover these very specific treatments tailored to someone's DNA
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  Speaker 2: because I mean really, they weren't around at that at
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  Speaker 2: that time. So fast forward to twenty twenty six and they,
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  Speaker 2: you know, they've realized, Wow, we really do need to
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  Speaker 2: include something like this because it's becoming more and more
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  Speaker 2: common for rare to these patients to be able to
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  Speaker 2: have these treatments.
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  Speaker 1: If you're a parent, a grandparent, this has to touch
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  Speaker 1: your heart. Before we get to some of the politics
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  Speaker 1: involved here and some of the questions I asked before
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  Speaker 1: going to the break, Kendra tell Us tell us, how
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  Speaker 1: care is processing the fact that she has been ostensibly
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  Speaker 1: cured and her older sister is not.
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  Speaker 2: You know, she's come to realize that she's gotten older,
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  Speaker 2: kind of the process that we had to go through,
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  Speaker 2: and so we've had to answer over time, you know,
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  Speaker 2: why did I get the treatment and not Livy? You know,
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  Speaker 2: what does every kid have MLD? Why did that kid
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  Speaker 2: not get a treatment? So there's a lot of things,
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  Speaker 2: you know, as kids grow up and they start realizing
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  Speaker 2: what's going on and why do I have to go
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  Speaker 2: back to Italy for checkups? So over time we've had
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  Speaker 2: to kind of explain why Livy wasn't able to get
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  Speaker 2: the treatment, why she was you know, and so often
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  Speaker 2: she says, you know, I wish Livy got the treatment too,
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  Speaker 2: so she could like play with us and go to school, YadA, YadA.
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  Speaker 2: So you know, she's she's realizing more and more the
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  Speaker 2: ramifications of what's happened to our family and what she
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  Speaker 2: sees every day and why she's seeing it, and coming
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  Speaker 2: to realize that her situation is very rare and not
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  Speaker 2: every kid is like her. But to a lot of
• 5:00
  Speaker 2: her friends at school, you know, they have no clue.
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  Speaker 2: They just think she's a normal six year old girls
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  Speaker 2: because she has zero symptoms. So it'll be interesting as
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  Speaker 2: she continues to develop and how that impacts you know,
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  Speaker 2: how that will be impacting her and maybe even what
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  Speaker 2: she does in the future.
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  Speaker 1: Kendra, I mentioned my visit with a regular doctor that
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  Speaker 1: we have on the program who has been in the
• 5:24
  Speaker 1: cancer field for his entire career and is retired. But
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  Speaker 1: he talked about how, you know, with the geoblastoma development,
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  Speaker 1: and I asked him if that development, which was done
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  Speaker 1: at Shan's Hospital at the University of Florida, if that
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  Speaker 1: was going to be for example, shared with, say the
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  Speaker 1: Saint Jude's Children's Hospital. He said, inside the United States,
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  Speaker 1: those types of treatments are shared widely, that there's always collaboration.
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  Speaker 1: Is the treatment for MLD is it being shared or
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  Speaker 1: is it still only found in Italy?
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  Speaker 2: Yes, it is being shared. So it was first approved
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  Speaker 2: in Europe, and then my family as well as other
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  Speaker 2: families who have benefitted from this treatment did testify with
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  Speaker 2: the SDA. We got it FDA approved a few years ago,
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  Speaker 2: and then just in December twenty twenty five, we went
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  Speaker 2: to DC and Secretary Kennedy did add MLD to nationwide
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  Speaker 2: recommended newborn screening. So that'll make a huge difference for
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  Speaker 2: families because now they'll know right when their child's born
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  Speaker 2: that they have it and can immediately get them this
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  Speaker 2: treatment which is now being given at multiple hospitals across
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  Speaker 2: the nation.
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  Speaker 1: So let's talk about the unfortunate politics that's involved in
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  Speaker 1: this journey here. You mentioned we're at the second effort
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  Speaker 1: on the right to try. Who's behind the effort in Congress?
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  Speaker 2: Yeah, so it was introduced by Senator Ron Johnson from
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  Speaker 2: Wisconsin and Representative Danna Harshburger from Tennessee.
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  Speaker 1: And what's the pathway? Where is it in the process?
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  Speaker 1: What needs to happen? Is it getting bipartisan support? And
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  Speaker 1: by bipartisan I live in that world every day with
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  Speaker 1: doing this show. I'm not talking one person on the
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  Speaker 1: other side of the aisle. Is this getting full support
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  Speaker 1: on both sides of the aisle?
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  Speaker 2: Yes, so far. So when they first reformed the bill,
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  Speaker 2: that was in twenty twenty one, when our family got
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  Speaker 2: back from Italy and they said, you know, you've inspired
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  Speaker 2: us to make some changes here because we realize where
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  Speaker 2: science is moving. So I did share my story, our
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  Speaker 2: girl's story at the Arizona State Capital first where it
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  Speaker 2: was initially passed, and it's now passed in seventeen other states,
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  Speaker 2: and then it's just got introduced to Congress earlier this month.
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  Speaker 2: So far, in all the states that have passed it,
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  Speaker 2: it's been a pretty unanimous yes. Most people don't see
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  Speaker 2: why we wouldn't make this change and help people save
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  Speaker 2: their own lives.
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  Speaker 1: What can people listening to the program do to help
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  Speaker 1: this produces sess?
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  Speaker 2: I would say, you know, right to your state representatives
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  Speaker 2: in Congress, let them know how important this is. Hearing
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  Speaker 2: stories like ours that are first person from families and
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  Speaker 2: kids that are dealing with this and could potentially have
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  Speaker 2: their life saved from a treatment that they'll now be
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  Speaker 2: able to get in the United States will make a
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  Speaker 2: big difference. It's all about hearing those stories.