Narrator: Coming up on Mayo Clinic Q&A,
Dr. Noelle Larson: My goal in life is to make new stuff for
patients to make the treatment less burdensome, and to make
their lives better. And so, if we really want to make new
drugs, new devices, new treatments, we all have to work
together.
Narrator: That collaboration often comes by working with
patient advocacy groups. The results can lead to new
advancements and treatments for patients.
Dr. Noelle Larson: I mean, there's no one that cares more
than the patient right and the patient's family. And so, to
bring the patients together allows them to work together and
let us as the researchers and the doctors serve them, is a
really powerful force.
DeeDee Stiepan: Welcome, everyone to the Mayo Clinic Q&A
podcast, I'm Deedee Stiepan sitting in for Dr. Halena
Gazelka. Patient advocacy and support organizations play an
important role for patients suffering with diseases or
conditions. The goal of these groups is to connect with others
who may be in similar situations and to help locate resources and
manage or treat their condition. But patients aren't the only
ones who benefit from these connections. Physicians and
researchers can benefit too from getting involved. Here to
discuss is Mayo Clinic orthopedic surgeon Dr. Noelle
Larson. Dr. Larson, welcome to the program. Thanks so much for
joining us.
Dr. Noelle Larson: Really a pleasure to be here with you
this morning, DeeDee.
DeeDee Stiepan: So, tell me how did you start becoming more
involved with groups for patients and their families?
Dr. Noelle Larson: From my standpoint, taking care of
patients is my passion. So, it really comes naturally to be
interested and involved in my patient's lives. My primary
clinical and research focus is scoliosis. So, I see a lot of
young teenagers, primarily girls and their parents and really
feel for them as they go through their walk in life. Oftentimes,
for non-operative management of scoliosis, children are being
asked to wear a brace to school, which is very difficult
particularly for a young teenage lady. And so, I think my first
introduction was really getting involved with Curvy Girls
Scoliosis Support Group, which is a state-based and national
organization, actually global organization, which helps
children with scoliosis develop supportive strategies as far as
how to wear a brace, how to wear clothes that fits over a brace,
how to prepare for surgery, how to go through the recovery
following surgery. And I saw that a lot of my patients were
benefiting from these networks. So, that was kind of the first
group that I have become involved with. I had the
pleasure of going to their national meeting a couple years
ago prior to COVID, and spent the weekend with over 100 lovely
young ladies and their mothers at a hotel in Long Island and
really got a chance to listen to our patients. So often, our
encounter in clinic is quite short, it's a 10 to 15 minute
encounter. And you don't get a sense of the impact of what our
treatment has on that child's overall life and well-being. So,
it was really a change in my practice and my change of how I
view each patient encounter to spend a couple of days on-site
with families and children affected by scoliosis.
DeeDee Stiepan: Well, that's wonderful. Would you say is it
common to have Mayo MD faculty such as yourself attending these
organization meetings for patients and their families?
Dr. Noelle Larson: I think it's becoming increasingly common,
and I would be a huge advocate for it. I would recommend it to
my colleagues, and then I'd recommend patients, if you're
struggling with a condition and you only get to see your
physician once or twice a year, yet you're having challenges in
your own life, that ability to reach out, and I think a lot of
that reaching out is now occurring through social media,
and through internet and through online connections. I mean,
there's a huge ability now to make contact with others. And
that gives power, and education, and networking to the families.
And I think physicians, if we're not involved then that's a real
shame. Because again, these patients know more about their
condition and living with their condition than we do at some
level.
DeeDee Stiepan: Oh, absolutely. And you touched on it a little
bit, but can you speak more about how patients and families
benefit from these groups?
Dr. Noelle Larson: I think there's a lot of word of mouth
happening nowadays where new treatments arise. And really the
poster child for all of this is a group that I'm not
particularly involved with at the moment that I admire, and
it's Cure SMA. So, spinal muscular atrophy is a relatively
common condition that can be life threatening and affects
children. And there was a parent who had a child with spinal
muscular atrophy and wished to do research. So, they set up a
large research fund and actually funded University of
Massachusetts and some other partners, and they actually
developed a drug that cures SMA, so spinal muscularatrophy, now
if the children get the drug prior to, you know, one year of
age, the children are now walking, they're not needing
ventilators, they're up living a much more normal life, because
of this patient organization that achieved the dedicated
funding that then was transferred into a
pharmaceutical product. So, I mean, that really is the gold
standard, right? And I think there hadn't, I mean there's no
one that cares more than the patient, right? There's no one
that cares more than the patient and the patient's family. And
so, to bring the patients together, allow them to work
together, and let us as the researchers and the doctors
serve them, is a really powerful force. So, I'm hoping we'll see
those same kind of developments for scoliosis. I would love a
medical cure for scoliosis where we don't have to put children in
a hard plastic brace, and we don't have to do surgery on
their spines. So, I think we should do it.
DeeDee Stiepan: I agree, it sounds incredible. Talk a little
bit more about how the medical community benefits from these
organizations.
Dr. Noelle Larson: I think at the end of the day, we want to
see fast, safe innovation. And so, from our standpoint if we
have patient organizations and patient networks to work with,
we can meet more patients who might be interested in enrolling
in a study. When we have patients that are enrolled in a
study, again if they're invested and interested or better, yet,
their support group is funding the study, you know, they're
going to do their best to make the best possible data that will
then help the FDA decide whether or not to approve that
treatment, which will then help insurance companies decide
whether or not they're going to approve it. So, again about five
years ago I decided I had published enough papers, I got
my little professorship, you know, I ticked all the boxes.
And really my goal in life is to make new stuff for patients to
make the treatment less burdensome, and to make their
lives better. And so, if we really want to make new drugs,
new devices, new treatments, we all have to work together.
Because what I think might be a good idea, a patient might say,
No way, I will never wear a brace that looks like that. I
would never want to go through a surgery that involve that. So,
that partnership between the families, and the patients, and
the researchers and the doctors all working together is really
critical. Same thing for an engineer. An engineer might
have a great idea for a new spine device, but as a
physician, if I come in there and say, well that means the
family has to travel here four times a year, and we have to do
surgery, you know, eight times, I can say hey, no that's not
feasible. You know that that's not a feasible treatment, go
back to the drawing board. So, at the end of the day any one
group alone is not going to make as much progress as if we all
can work together, and again make lives better for kids.
DeeDee Stiepan: Absolutely. Well said. Can you share any
other examples of how these collaborations lead to research
discoveries and medical advances?
Dr. Noelle Larson: Well, my main journey over the last six years
has been on fusion scoliosis treatment. And fusion surgery
has been around for 40 years. It's very predictable. All the
pediatric orthopedic spine surgeons, we know how to do the
procedure very well. There are 15,000 papers published in
medical literature talking about fusion surgery. But what I
started hearing from my patients five to seven years ago was that
they don't want a fusion, and that they don't want to have
rods in their back. And I can, again have this long discussion
with families, and say it's very reliable, it's a great solution.
But they want to preserve their child's growth and mobility. So,
there was a procedure that was developed in the U.S. that was
not approved by the FDA. So, we did a handful of those
procedures at Mayo, and at that point I ethically didn't feel
comfortable continuing doing non-fusion scoliosis surgery
without a study. So, I went and talked to my first handful of
patients, and I said, Hey, would you be willing to be in a study
if we are able to get FDA approval for it. So, we did a
surgeon sponsored investigational device exemption
study at Mayo, and I really have to thank all my patients for
being willing to come and do the follow up, and do the patient
reported outcomes. And it was really an outgrowth of hearing
from my families that they want an option other than fusion. And
then right now we're really kind of at an ethical standpoint,
because the non-fusion surgeries have a higher failure rate. So,
there's a higher chance of needing a second surgery with
non-fusion. But like the fusion, you know, you put a rod,
right. You put a rod and the kids back, and you pull the
spine straight, and it's great. And then the non-fusion devices
are like a plastic cord. So, you have like a plastic cord, you
know, where the child's back still bends, and moves, and
twists. But as you'd imagine, the rod, it's going to be quite
hard to break it right? But a little plastic cord, this is
going to brake at some rate, the device is going to fail, and are
families willing to accept that higher failure rate for the cost
of motion and growth? So, really, as a surgeon, I find
that a very hard decision to make. Most surgeons are very
conservative, we want to do a procedure that's going to work,
and the patient doesn't ever have to come back with more
problems. So, again, this is another threshold in time where
we need that feedback from families about what do they
value and care about. So, right now we're doing a FDA sponsored
study with Setting Scoliosis Straight Foundation. So, the
first aim of the study was to survey 300 patients and families
about their values regarding fusion versus non-fusion
surgeries. So, we've just completed aim one of the study.
And then the second aim is actually through the Mayo
Knowledge Education Research Unit, with Dr. Juan Brito. And
basically, we're developing a shared decision aid that doctors
can use when they're sitting with their families talking
about fusion versus non-fusion, and use that aid to help inform
patients. But I'd say for right now there are these very
involved social media groups that are interested in fusion
versus non-fusion, and they really would benefit from some
of the data. So, like tomorrow night, I'm doing, with Setting
Scoliosis Straight, we're doing a webinar for patients and
families with kind of the latest updates on non-fusion scoliosis
surgery. And again, trying to work with patient groups so that
they have an informed ability to decide what's best for their
child.
DeeDee Stiepan: That's a great example. Thank you for sharing
that. And I love a visual aid. So, I appreciate that. Dr.
Larson, are there any recent activities that you've been
involved with, with these organizations and support
groups?
Dr. Noelle Larson: This fall, I had an amazing opportunity to go
to the Children's Tumor Foundation Neurofibromatosis
Walk, and I actually ended up going to the one in Iowa and
also to the one in Minneapolis. And I had a couple of my
patients that I had treated with a newer procedure on the shin
bone on the tibia. One was age three, the other is about age
five, and both the little boys were there at the walk, and they
were running around together and meeting each other. And one
little boy was in a brace, and the other boy due to our
interventions is now out of the brace. So, it was really sweet
to see the two of them, you know, share bracing tips, and
meet their families, and see them playing together at this
walk. It was really like one of the highlights of my life, I
have to say, to see these two children living and playing
together. And I think neurofibromatosis is another
very common genetic condition, and Children's Tumor Foundation
is very active both as far as research, developing research
standards. So, I sit on a couple of committees where we have
patients with neurofibromatosis, and PhD's who research
neurofibromatosis, and clinicians who treat patients
with neurofibromatosis, all working together to figure like,
if we do a study what kind of outcomes should we look at?
What's important to families? What makes a difference in their
lives? Rather than just me as a doctor, as the orthopedic
surgeon, I care about the x-ray. That's our typical study. We
care what the x-ay looks like. We don't necessarily know how to
study the function, which at the end of the day is what's
important, right. The function is important, not the x-ray.
DeeDee Stiepan: Our thanks to Mayo Clinic orthopedic surgeon,
Dr. Noelle Larson, for joining us today. Dr. Larson, thank you
so much.
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