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OK, with that out of the way let's get started i'm very happy to introduce
doctor Glenn Furuta Dr. Furuta is professor of pediatrics at University of Colorado School of Medicine
he serves as the director of the gastrointestinal eosinohilic dieases program
which is a multidisciplinary program that dedicated to providing care
for children with eosinophilic diseases
his research focuses on the timing of our audio samples and gastrointestinal
function
he was also speaker at the APFED conference
conference over the summer
without further ado i'm going to turn this over to doctor Furuta
you can turn on your microphone and we'll get started
the
who curriculum when you're knee
super sold well good evening everyone and it's really a pleasure
to be uh...
on this call tonight i am grateful for this opportunity for both at the
as well as the university of michigan jillian and julie you have done a great
job putting this together tonight
and i appreciate certainly all of you out there
across the
unit of drawing tonight are taking time away from her
family and uh...
are very busy time i know of every evening
the uh... at but i'd like to do before we get started is just to talk a little
bit about
research and wind theirs is so important
and what what we really
think about me
are concerned with research in and
when you go to any positions often as you know that they've gone to medical
school
done much training to go take care your child or
of of u_n_ one of the questions that always comes up is how you keep up and
how do you really know
what to do next and and
what to do with respect to decision making in
*** positions go through
certainly or burgers training
initially and then when you
when things develop
how do you really keep track of that and we go to meetings
we uh... see a member of patients and have a lot of clinical experiences
we speak with our colleagues and then finally we do
what you are but here is go to the literature
what does that mean moments ago the letter triple it means that we read
research
and we try to understand how that research may pertain
specifically to our patients and so
they will hold those will speak a little bit about
global aspects of research
and then i'll
specifically address some of the research has been done recently some of
these things are presented to
uh... at the conference recently
and then finally all go back to answer many of the questions are trying to
answer many questions that at that have come up that you guys have centimeter
wide assortment reading so let me just start by examines some of the slides
that on your screen now and i think there are four questions
that i wanted to address upfront and
to try for research
into perspective and these are questions that come up often
went on my speaking with families and colleagues and i think the first
question is
uh... what is searching what his research
and i think all of you uh... certainly appreciate the fact that from the
questions that you have written and i think it's
lately at obvious that
many of the answers
we don't have to
the questions you that's because haven't even been asked yet
and so in many regards but i like to say is that we're doing search
very often now as opposed to research so we're really looking to try to
understand
some of the very basic questions and answers to some of those basic questions
both well you can't be able to go back to make sure that were correct in
understanding that sells
what will i mean by that
while having one of them
needed to most prominent samples
to me that the first is how do you make the diagnosis of this disease and
we
didn't know how to do that decade ago we've become much better at that now
since two thousand seven or those publication identified
to the diagnostic
features of this disease
and
these have been republished now in two thousand love and that have been a
refined
with input from all of our colleagues this was a multi-disciplinary project
but it really wasn't there before spot was calm but but the search
the second and probably one of the most interesting and important areas right
now is that
we need to think about
uh... wet how to make the deaf
diagnosis of
usability i diseases
in the small intestine
colon
and uh... and stomach where we don't have as much diagnostic thirty so that's
an area clearly of searching we need to try to understand that uh...
the second questioned her bra top is how does this happen
and how do we
uh... do research in
research always
starts with the question
and as physicians scientists which are
all of uh... all the researchers work in this area of work
physicians first and scientists were tremendously and things
second so it when we meet with you and see you on that
hospital or she was and and
outpatient clinic we wonder how we can try to help make your lives by rome
where those very important questions that come up
then take those questions and develop
hypotheses to try to understand
those baron
and with those hypotheses then
revolver research protocol
to go for an answer that so we may say something as simple and going back again
to the diagnostic criteria how do you make the diagnosis
and and
uh... we move may make a hypothesis that we think that clinical
symptoms
tissue abnormalities with increased numbers of your sniffles
or crippled making the diagnosis of the isn't going to stop the darkness and on
the basis of our hypothesis the model for doing research study
to try to understand that and we need to
uh...
develop a protocol
and then we need to added by exactly how that's gonna happen
and that we need to do that important in particular very regularly on truman your
discussion
how do you support that when we talk about support we talk about that
money that's needed to to this study itself matter
important that we all russell weapons like understand really how you
contribute to
the research mission by your support of appetite
many other organizations that go forward part so
uh... we have an idea we develop our offices who devote protocol we look for
money to support that protocol
and then we do the studies so you can see right there
that uh... pop up for a while
uh... to happen we do the study
uh... it takes a while anywhere for months the years do that study and then
re-write those results basically take those interpret them usar statisticians
expertise that
try to understand those what those results network about a manuscript in
them
that sentiment withdrawal
to have them publish
and then they come in the literature
so that's the the process and how does that happen
and moments and we're going to look for that's what it means by
trying to understand uh... with their questions why is it so expensive and and
at the candidates appreciate the fact that when you go through and you
here that process and it's one of those points
need to pay for hand two different
uh... possibly uh... so at the personnel
at the laboratory equipment
all the different features that go into that
long-standing process and
one of things that uh... uh... comes up a few especially when we think about
their apprx
and when we think about new kinds of treatments that go through exactly that
same process more appropriate
a new drug to our patients or new treatment live takes up hundreds
thousands uh... millions
of dollars and if it's a completely new drugs sometimes
at tens of hundreds of millions of dollars to really bring to a patient
because it takes so long and and some indications that this happened
so uh...
i wanted to provide that to you a little bit as an overview because i think any
research project that we have
at these things all come up and and
as we go through
some of these days i'll take you through that's uh... that's one into them
so uh... without with gold going forward now what i'd like to do a little bit is
just go through some of the very basics about the diagnosis because
this does pertained to some of the questions that were raised and i think
is important as a baseline
when we need the hum
when we make the diagnosis of the s_ and i thought this out the door it is
you need to have symptoms
we need to have an abnormal tissue which has increased numbers of usa nipples
we exclude other causes of those symptoms
and that inflammation and at the end of all this process and what's going
through our minds mort
thing for you and think about this it's
roles try what what causes this
these things to happen
and uh... why do these things happen that exists in the laboratory to try to
understand
make things better
this is the publication that doctor lee chorus uh... really spearheaded and
doctor service that helped as a senior author on this busy can see it's a
composed by a number of different peoples all these different people from
different specialties adult
uh... physicians pediatricians technologist allergist
a gastroenterologist participated in in coming together and thinking about
what it means to make the diagnosis and and the things that just
riverview
uh... were included in their no symptoms that in are included
uh... for younger children dom open vomiting eating problems
or her children and adults food getting stuck problems swallowing tristan
reflux symptoms that they don't get better and i mention these first three
because
one of the challenges that we've had and i think some of you
have been following this to the internet or through that work with the f_d_a_
tappan
we've been doing is really how you measure these things
and one of the important parts of that is if we can't measure it with some
degree of uh... thirty
it's very difficult to say whether things are
getting better
and that's what the f_d_a_ would like to know their ways that you can measure
these symptoms
to help us know whether it proper treatment is working and so there's a
allot of work being done in this regard bye tc spectrosc roman doctor trooper
who were developing aids
a way to measure this adults
uh... doctor uh... bonino
french osian or converted developed this
in cincinnati with aids
uh... another type of scale too
really help us measure these and and if we can measure them then we can help to
use those and studies
about therapypets overtime so this is our research really gets
integrated into they did a quick here
we think about
the next
or blind coping behavior this is a
something i'm sure many of you in the audience of
have seen your experience in which your symptom and not be prominent because
you've developed some ways to cope with that simple
so you may have initially her problem was falling but you chew your food a lot
of you
pyrenees water wash the food down so it's not as much of a problem because
you cope with that
and and so on the research stamp one with things we do is how do you
first you need to identify that so we
so you know what this is developing unbelievably document that very clearly
enough
in in a in a manuscript we put that out their publications work always can read
about that
that's a form of research on what we've actually helped identify some
some symptoms that in fact maybe some present that bar
unnecessary helped make the diagnosis but again how you measure that in itself
can be quite a challenge
last line here talks about other symptoms that may be present including
and specifically lead pains
uh... t things like that then
you know were dress at the very early stages this again i think this is part
of the search
of what we do is to how
what those symptoms or how do you measure them what causes them
but certainly they may be president some entrance
when we think about
the tissue at this
these group of diseases were earlier one of the few that
if you're looking at textbook
uh... characterized by an increase
number of oneself and so that's quite unusual to have
uh... disease that's characterized by one cell number but we do that right now
and we can all the members of the ascent those uh... to make the diagnosis
i think what you'll see over to my news that we're going to get more
sophisticated than our ability to
document this type of information
uh... we're going to be held at
uh... i think about this in a more sophisticated way because
but you is that those can actually increase in
in the kitchen
uh... for a number of different ways sand and those include cromer suction
allergic reactions things like that so
we want to really be able to describe this information the better way
so won't i won't be
have done
uh... and this next slide here really do shows away inflammation looks like in
that panel a can see this is the normal appearing tissue
uh... it's uh... that top part of this is where the
uh... food comes through and then as you go down further on the slide
this is the layers of what we call the epithelium of the lining cells pinto
beans shows
uh... that he has some of those of the painting staining cells impanel cd can
see the easiest thing fills with arrows pointing or on the very end superficial
or that top surface of this element penalty here what we call micro
abscesses are collections of these celso you can see a day
they're very
uh... obvious and they're able to be seen but need it
that really
all we do is characterized the numbers and and where they are now but in one of
the important things to do is really how do you measure this now how do you
characterize that
a little bit better sweeping prayer document treatment responses
this next slide shows for the presence of mast cells and
in years and i thought the south of died as the brown staining cells amassed
cells and we wonder whether the cells may be other kinds of cells that are
actually present and may have some
that your diagnostic or for therapeutic influence on this uh... work that's been
done by doctor service
this is listed understand their increased numbers of these cells that
are present
uh... and and the finding that lola's is uh... yet to come
this bus like talks about the other causes of
here's some of those again within the lining of the esophagus it's a list of
things that we think about
try to understand
uh... what what disease may be happening i think it really relates back to
many of the questions that came out in
that you guys have been sent to an end and i think it
makes us wonder about what the relationships are
between this information and other
diseases including sealy activities
plants has told this motel or the how does is that those caused some of these
things and
how do we think about that over time
well from having jones lab has helped us to
understand this veteran
means i said not to leave chris has been believers in this area
for quite a long time
well wanted to present to you a few
other items before we get to the questions about what's
new in this area and and uh... some of these areas are listed here
well there's there's you know and and when we think about
any disease we want to try to understand what the function of the esophagus
uh... function of any organism if you think about
it's a sunrise as well
if you go into the doctor's office they may have you do a
a peak flow you blow into a device and
that lets us know how pass that there is coming now
if you are planning to go to a had a heart problem get-up heart racing or
stress test them out lets us know about the function of a heart
wrote for the is the start of this right now we're somewhat limited
because we ask you questions about how the esophagus may be functioning
bugaboo x-ray test that may let us know about
whether the impact these
uh... that with the contours of the esophagus look like but we're not really
able to fit well function that well and and this is work that's been done by in
the chicago group by doctor consultancy collado group
use this toward hobby and a flip
to look at this opportunity function
well you can see is there is a
on the right-hand upper panel
but there is a up balloon that's placed in the esophagus
that balloon
uh... is goes down and then the balloon can
is expanding on the
is able to
um... measure
how
uh... the esophagus is able to expand or contract
sort of sense is assaulted real movement and that's quite an important test
uh... that may help us with the motility disorder so
it's not exactly the same as a man almond tree test because it senses how
use out of this many
expand or contract in a very dynamic way and that bottom panel at sea
gives you an idea about just how that looks
graphically so it simply it may be important essence a research tool right
now and
uh... the work they're doing is really helping us to understand that in the
future well one of the questions that came up previously about this mass elves
are there any way to master psychosis and i think that uh... and certainly we
don't know that you have that's one of the things that were understanding a and
think a little bit more and when we think about master psychosis that's
really a disorder in which
throughout the body there are too many mast cells and so i don't know it's
exactly related to delete
so going back again to the two shoes standing as i said were really somewhat
limited and in our ability right now too
to characterize the tuition helping us make the diagnosis so
this was one of the we're done with that doctor jimmy lee at the scottsdale
spares owner sharp wrote the robes to try to develop some new ways to actually
quantify that information away you can see on the left hand side is just a but
tissue study that shows
uh... and normal piece of the uh... inflame piece of tissue works the is the
bulls are present on the right hand side this is a
uh... the same piece of tissue discussing with an antibody specific for
uh... anarchy is an uphill so lets a c no sells a little bit more clearly and
then when you see on this light is on the left hand side they're actually or
any apparently has some of those there but
because they've all the granulated or let loose all their a specific proteins
and when you look at this slide on the right-hand side you can see the paint
staining shows that these samples of actually
released all of their but granules
and this is a study in which we
going back to just the thinking process we said fees may be some of these
tissues happiest in tocqueville's but it's not so obvious to me that is a
hypothesis we by enroll patients we gather to chew we stay in the tissue and
then we measure that over time in this is the result of that so we think that
this may be assist us in helping to make a sense of uh... some further diagnostic
test
now that the next level are they show you was fun
this in the slight that that looks of biomarkers are james and i think
usually sturm biomarker quite often in and what is a biomarker welts biomarkers
any molecule or protein or something that's measured up body sample
and the concentration or the amount of that
sample really thought reflects the severity
or the presence of some physiological state or disease so
uh... one way to look at this is that
if u
again going back to the analogy of asthma if you were to uh... cloth up
anne samples here sputum sample that you could look at that sputum sample
and identify hello active asthma he is and that's been done and many types of
studies looking at as well
and and measurements of biomarkers consistent helping to make a diagnosis
making a determined the course of treatment
look at the responsive g to treatment the likelihood of having a complication
or other types of things that go along with that so
uh... is as you are are likely where much of this work has been by dr arrived
from bergen instance marion his group and it's been working you've supported
you know through your contributions in on this letter shows
a picture of
uh... what appears to be quite
complicated
picture but i want to walk through this
this through you because it's important for us to try to understand this and
yeah goes back to the some of the complexities of
researching on that that'll left hand side you can see us go
says normal
number below that
each one of those rose identifies a patient
uh... sample are subject sample
and there was analyzed
uh... four
different kinds of genes that may be present so you see eight if you go
across
it looks it in a difficult patient horizontal and then is you go down
it looks at individual jeans on the normal side there's a series of uh...
brought by eight samples in each one of those
as you go down
of the slide those are looking at different genes the next
uh... block over shows product this all the guys in again and individual patient
on each line in there as you go down
uh... this you're looking at the different genes
is that the look esophagitis patients and then it says food to eat his own
responders
meaning that the user patients that responded to a treatment
so
if you look just pull back a look at the global patterns on here what you can see
is theirs
a lot of yellow on
your side of the year simple consulted artists and then
if you look at the pattern ideas with a look at some progress on the top there's
blown apart from its talk there's a lot of red boxes on the bartenders below
and what that sternest is that they're paying since all of you sampled this
article is
have a
different set of genes and biomarkers
their associated with active disease
isd commentator dan rather and and
and blue so they looked up and the beige and saw the southard art is the brand
impatience of normal disease and then
if you look over to the following inside
when patients are treated these genes can revert back to normal
so i only think about this the this is an important concept to think about
because what it lets us know is that
come here biomarkers the potentially can allow us to think about how to make the
diagnosis
helpless to understand responsive treatment
and importantly
how the disease might happen so each of those genes as president
we may have bill that used to uh... dissect out exactly how this disease
happens and then
when you think about that the next thought is can we intervene and an
actually interrupt that gene
to to provide a cure
i wanted to just
let you know that when we think about this meeting too much about on this a
little bit more on that
we'll talk about treatments overall
but they're very few diseases that we actually
hero
in if you order it open up our big textbook of of medicine they're very few
that actually
procured shouldn't cancers are curator infection secure but we offer a lot of
the season that's why we always talk about creating a disease to help
information get better to help patients feel better but they're very few the
direction
soldiers to looking at this has been there since questions about uh...
uh... this with respect to how close his name when using biomarkers too
to using now and i think
and it taking it to me it makes that timeframe but
everything pass the process in this really applies to any kind of
treatments are devices and more trees
sand somewhat work we've been doing with devices
uh... in the next several slides but i think what happened there are only do
these kinds of studies is that
uh... we have any to validate them to make sure that this holds
true in a number of different
uh... studies that actually that was sold for singer actually
uh... flair and are able through this to school since with the large number of
patients and then
uh... if it is a drug that needs to be identified or devices that needs to be
validated and go to the f_d_a_ and it has to go to the whole process there
were a number of benchmarks uh... before that a for us to to to insure that these
things and i want to be safe but better than what is on the market late
seventies
a number of years too
that to go through
so
uh... let me go to through next does some work that's just for a current to
looking at the
for the micro biomass opportunities and i bring this up because
there's a lot of very important as won't now going on with the mic requirement
and other no
diseases including krone's disease
uh... specifically but it
these studies also just subtract
the bacteria that in your body and there are several pounds of activity with us
normally main contributor initiate
any inflammation enso worked
uh... work by sophie field is in our group that has been supported by that is
looking at the end we go back to think about a hypothesis in the esophageal
micro by may contribute to esophagitis
the aim of it
the studies that she's doing this to measure the michael bonnet health and
disease and the method she's using their
her shown here of looking at a south of chilled by axes and some more
strings samples
to see what that microbiol ms
at give you the slight additionally again some of the techniques they're
using this is very similar to the work that's
uh... how these slow uh... samples being collected and analyzed in
doctorow timber excel work for you
among the left hand side you can see
sample that's been collected
in the middle of this is d_n_a_
uh... that's been uh... harvested and then on the right hand slide you can see
where this
d_n_a_ is put into a tree all in this picture it could be put in it
me into a machine to be analyzed
uh... engine an episode of these work is now
uh... measuring shown in in one study what the normal assault the chilmark
provide looks like and then
and brother worked if she's preparing now how this may
the different or uh... change
and use it for this opportunity snouts were to come so so watch for that
bond so finally what i wanted to show you what is this
tests and it cannot answer both questions work we're doing here
to look at an assault the drill string test and and again we go back to
although it back in and just again to think about how research happens
we hear repeatedly in the office when do we do our next in dollars could be do we
need to do another and ask to be a and and are there ways that we may bogus
study
what's going on in the esophagus without having to do and i asked me
we thought back into the the literature we went back to that that uh... think
about that and and here's a test
and we're showing here in this
slide on panel is these the entra test in this is a a test that was develop
back in the nineteen seventies and bottom lefthand corner shows the capsule
and the capsule is filled with the string that shown as you go up on the
slide and so if you order take this diet or by this test you'd see a capsule
which is that a little bit of the screen they're shown here in the white arrow
coming out of the top of the capsule and and the string is actually fills the
capsule
so when you swallow that capsule the strangest largest within uh... made
within the intestinal tract in that little pieces taped to the treat
wet inside you can see that there is a an indicator state that you can't touch
to restrain their shows an acid
part so that's my stomach and alcorn pardon that's in the esophagus
when you look at
the next apart of this
a slide what you can see is that uh... made it does not require sedation to
swell that's for you the impact you need to be a way to swallow the string in a
dislike trees
and when you can see here's the orange
piece of the orange line is this spring it courses through the esophagus
the stomach and then sits in the small intestine
inaction in the idea behind this is that
now here's the string within the esophagus itself only but we think
happens as it were absurd against that inflammation that i showed you earlier
and by rubbing against that inflammation then weekend sample the contents that
are within the
sa fitness itself
and so that's the study that we did was that the east wall this training left in
place well for a number of hours
and then we pull the string out
and we collected the liquid that was attached to the
bus train
we sought this pavilion suction
liquid that was attached there
uh... endoscopy was poor performed at the same time where we are
obtained by axes samples and then we took
the samples from the string we took the samples from the biopsy and we put them
through some chemical processing and we looked at the contents of of looked at
those samples for he has some thoughts contents
and so
well we there was an this is the results of that study major basic protein is one
of the
proteins that were present within
anne s n_f_l_ on the y_ axis is the amount of major basic protein its
present within the and strengths sample
and where you can see is this for are on the left hand side shows the amount of
major basic protein present
inactive infomation
on the right hand side shows from patients who don't have inflammation and
there is a significant increase in that quantity
uh... present with and uh... assessment within the stream itself and and when
you go through additional analyses doing
we i were able to confirm that in fact
this amount of protein was consistent with what we see in the biopsy samples
themselves
so it will working on this test now we've done the first
setup test
and to show that this uh... actually works
uh... on an overnight sample the nex set of studies that were doing cooperation
with doctor recommended
papua work rule
and colorado it's it's made out there we are clever in scottsdale is is now going
on to it to look at this time shorter time frame that may be more applicable
to day
uh... clinical setting
the studies are being done in patients or within seven years of age uh... it we
think that this will be better than an endoscopy because it won't require you
undergoing sedation
uh... it we think that it will be no allow us to collect
the same kind of them from information that you may be able to
acquire the time of
an endoscopy and help us to monitor
treatment over time
so um... i think just to summarize your there's a lot of research is going on
now from a number of different investigators across the country
and anything there directly addressing many of the patients need and invite
your involvement without that
a number of different organizations across this
the united states you're you're contributing to this work so that i want
to spend the first half the time talking about that
uh... just a looking at some of the questions are coming up
tom we are trying to quantify the amout and and i think the last question here
really indicates that the level of knowledge in sophisticated out there the
next challenge for us is really saying how what there's uh... a number of
derived from a string test what
how does that correlate with
the amount of information in its present invitational itself and so that is the
next level studies that are actually being done
so
uh... what i thought i do now in the next uh... for this is really try to go
through some of the questions it
that you have uh... absent in an address some of those overall x but
as was brought up by truly enjoy him in the beginning that v
it's very difficult for me too
and speak specifically about uh... patient or at specific circumstance will
try to keep some of these
brought in
and uh...
uh... in my answers
uh... and and dreaded groupies according to a different categories
many questions focused on our other associated
associations with other diseases
and so when we think about that
other certainly remember different diseases now that may be
associated with
uh... inflammation of the esophagus including sealy active season test
oldest motility
by things like that are associated with air just in the stage is now trying to
report what those associations are there have not been any clear
uh... clear
categorization serve them today
when we think about the pathogenesis of this disease work from doctors for
rickles group and again the cincinnati group of companies
formally uh...
document rec at cincinnati now case western
knuckle rothenberg looking at mouse models of this
disease were understanding that indeed allergies
likely contributed as their number of ways that they make
contribute to this both from food
uh... that may spark this information the evidence for that is that if you you
food
you may have the symptoms of victims taken away at the symptoms may get
better
there are
uh...
mouse models of suggests likewise that desensitizing bows
that these uh... both either to aero-allergens our food
uh... that this may spark the disease and interruption of that pathway to a
specific things such as
uh... antibodies may
information get better
well we think about
uh... that curing this disease as i talked about for we're not to the point
yet where we have a cure
we're certainly to a stage where we have a number of treatments including but
things like nutritional that's clusion says well asteroids that can diminish
the information help patients feel better combo unit and really uh... there
are new studies that are going to getting better before reaching a point
that we think
uh... will be to a patient's and in the future but it's still going to be a lot
because these are at the early stages of time
um... there are not monoclonal antibodies that we've seen as a as
treatments yet these are things that are still in the going
investigation we think that hopefully these are gonna be
to u_n_ in
years' time certainly not months time
uh... but the critical question i think this came up and amp number of different
ways in in questions that were submitted
i think go back to the natural history of this disease and this is a really
important question
what we mean when we say natural history
well we mean what happens
if someone is left
untreated over time what happens to that information
so uh... we think of that in the course of years
units the exact question that the f_d_a_ has posed to us
when we we're presenting their in september and discussing her we get new
treatments to u into your patient into your family members
so the natural history some is a question of how do you get what happens
to tissue over time and and and to the patient over time
and all speak from a practical standpoint first just to say that we
think there are three things that can happen the patients over time what is
that they may have problems leading and they may have problems uh... swallowing
that can lead to
malnutrition or problems with growth
the second is that a patient may get a piece of food stuck in their esophagus
and need to have an das could be to have that institute a canal
and that can either because of das office squeezes
or because there is
narrowing present
mess that really the third
kinda complication that relates to assault that john stricture
or scar
or narrowing some in that
regarding that there is difficulty with the food going down into this office
problem is that we don't know how often this happens
we don't know who
that develops this
uh... yet in in large studies and the reason we don't know the reason we make
be very difficult for us to know that is that none of us or are actually go
uh... advocate for patients to go on treated so
uh... the f_d_a_ asked us at the meeting more what happens after decades worth of
information and we responded we don't know who may never know because our
patients
or not
going to us
uh... i'd be going treated over time
uh... likely
more we can say is that
uh... we know that those dreams three things develop
and and we'd know that uh... work
part of a efforts that we have with respect to our tigers consortium and
in a number of other investigators crandall
document what happens
uh... overtime in this disease
uh... we don't see cancer as a part of over the long-term outcomes of this
and uh... overtime now ought
in regards to the natural history in this came up in some of the questions
also
is that there probably different sub-types of this disease and there may
be patients that don't want to astrakhan ollinat marilyn there may be patients
that respond very well to dietary therapy in there may be patients who
have um...
uh... and although onto that respond therapy including stairway to things
like that so
amidst the global oppressed know exactly crew all those subtypes or at different
groups of patients are the best part of the research it's going on over time
along with that i think
um...
well we have done any studies to identify the fact that steroids
are helpful
uh... and dietary exclusion zone helpful
one of the things that we haven't identify kids has come up as a part
questions is
is what
all the long-term complications of these different kinds of treatment
now speak to that first with regard to
nutritional management i think that these are
in large part preventable as long as we have patients ah...
who are undergoing treatment with
uh... the advice of dietitians and we make sure that
these patients undergo there
proper nutrition rehabilitation
uh... work from
uh... or group here with my shop henry has shown that
and patients up to sixty seven percent of patients may in fact
not even get at the adequate
uh... information about how to undergo truthful restrictions so it's going to
be very important
uh... to make sure that
the proper information skip patients and their restrictions of the day
like my calories marker nutrients
and uh... and nutrition when some foods are eliminated
when we think about the issue of of
long-term treatment with
uh... steroids
again
or literature is based on our experiences are based in into regards
the first is
uh...
the literature on using topical steroids for
the treatment has
and what we know
maddie's there can be a slight diminish growth
uh... that uh... uh... and that is part of the main
quietly probably or
we think of all the different kinds of symptoms maybe
or problems that may happen topical steroids
uh... they include both the mineralization cataracts
uh... or local infections with
uh... on this virus
uh... those will be the main types of things will think about
refused to talk steroids
now that the second part of that is that the way that these spirits are used for
the treatment of annoucement bulletin southard right is is that day
artistry in the mouth and swallow two uh... or made into a story and swallowed
and when there's fault they immediately go to the liberal
where they're metabolized in
union and not
i thought to be terribly
uh... as strong uh... and happy effect to the rest of the bodies so in contrast
to the
whether used for has mine which they are handled
the stories that are used previously booked south guys we think that the
weapon metabolism so we think that
there is last side-effects overall
so
uh... well
are global at least
i think my global experience has been that the use of these over time has been
relatively safe that what we always been and one of the things that
comes up in in every
a situation when they get their your position is we want a way out the risks
and benefits
and make sure that uh...
the benefits of using something outweigh the risks so
uh... that's always a question that's asked that i think that
uh... buster a stray uncertain use it
this heart disease
windstar initial experience has been my experience has been uh... relatively
good
uh... so let me just go down in in
this in thinking about
these other questions that have been presented i can
tried to highlight some of these
uh... when we go back again to it
maybe we can talk about the lower intestinal tract pass the esophagus and
i think this would start by saying that again
the diagnosis of those diseases can be difficult because believe me there's not
always complete agreed in oz to what numbers
uh... you think about as being abnormal in normal and whether the cells are
beneficial uh... with this but let's say that the patient has a large increasing
use of the bills they have symptoms that are compatible
uh... with that work in name
inflamed
and and what i mean by that is that if someone were to have
bleeding
bloody diarrhea cramping
in slight that
increased numbers of the it's nichols in there
poland and that will be consistent with someone i just thought politeness and
that's part of things we need to think about as we perform research to
understand that
disease more clearly
thing the second part of that the questions that pertain to visit
minorities
diseases affect the stomach small intestine ***
and allergic disorder an autoimmune disorder
i think that uh...
or clinical experience again goes back to suggesting that it may be
uh... not always an allergic disorders certain or some patience that made
i'll respond to taking food out but i think the further down within the
intestinal tract widow
uh... the less likely it is a larger know whether it's an autoimmune disorder
and uh... it's difficult to know that auto-immune disorders would imply that
there is some specific feature of
at the intestine that the
body is attacking and we don't know what those
different things might be a
*** certain com molecules that are present life may indicate that
why that information is happening in we really haven't reached that
point of understanding
all typed wrong also because this is come up
arby's mada climbed real disorders
and mark andrea
just to go back and review a little better part of the body that part of a
solid is actionable to making energy
and and every cell has mike andrea that are present
uh... those mike andrea
function to
we
energy more tools such as a_t_ yet hope this'll work properly
when this all works properly then the organ were
pop so there has been
uh... an association both
abnormalities but though might probably in some patients who have
uh... yes the book information and and other kinds of diseases and
this is work that's been done and
in cincinnati but uh... putnam who was
and identified some patients it may in fact have
disassociation
all say that this is a very young area of research in that
that certainly made barrow relevance and it goes back to a gambler
what we do with that information that we take care of those patients there is a
suggestion that some
uh... at the addition of some
coenzyme some barnett's name help one in improving the partial might andrea but
this is
in the search that you've been to a tornado by
you know who the patients are at the end
and what characterizes them ultrastructure degree so i don't think
we're at flight to the point yeah
umts
so all of leaders the container to try to address some of that the questions
that are on
and that that you've
uh... provided questions about probiotics
were complementary therapies that may be helpful with
he isn't going to ask the court is though the research focus in on that has
been
very limited and and
i think is limited
uh... as far as i know the case reports i'd go back again to think about what
the rationale is for why something like that might be helpful
we spoke about the microbiol we've spoken about the fact that bacteria may
in fact initiate inflammation and other kinds of diseases such as
from sissies
and so one word hypothesize that if you are or place
bacteria or add bacteria that may be helpful
and maybe and create a pattern
uh... microenvironment will play that could produce that information
or use complementary therapies that are
uh... perhaps herbs or other kinds of supplements to make that uh... local
microenvironment better
maybe a little prove any kind of inflammatory state
and so while these may in fact the very helpful
there's been very little
or research done in this area
three days and and uh... the
and the support for that it is somewhat limited
uh... just going back again to thinking about her to looking at the diseases
one of the questions and several of them actually were wondering about whether
there may be an association with developmental delay
uh... sensory integration processing disorders
uh... and and disorders
about like it and i think you can wear it in some of the search status for this
associations have been made
uh... in work that we've
preliminary working with biscuit doctors wearable
uh... i think there is
and association certainly an outpatient populations we've seen
patients who may have both of them
that's an association whether that's the whole truest too
uh... cause and effect i think this is not yet know
here again
as some of the questions also four although i think about how do you know
when to monitor patients and
anna and i think that can be with regard to several different thing
and certainly for the treatment
of with
the disease how often you margaret we don't know that you have
i think again
are our feeling is that there are
important types of uh... monitoring that we need to be got going on
i think what we were alive and in a certain senses are adult colleagues and
i'm letting us know that
uh... patients have not developed
cancer uh... today with this and i think that patients who develop strictures
arbonne at the patients who need to be the focus was to try to stay on the long
ten
for malcolm the monitoring for patients who wants to release
patients ron long-term proton pump innovation
we still don't know
uh... about what that long-term outcome is and i think is pediatrician certainly
we think about that because we don't want expose our patients
to those kinds of drugs unnecessarily began to balance of of trying to make
sure the benifits of doing that and arm thought that his maintaining growth and
development is gonna outweigh that potential long-term problems and
will monitor those patients with
uh... who we have on those with uh... taken for bone density as well as uh...
heart disease and these are certainly stories i would need to be
uh... reform in the future
c just currency here if there are other questions are coming up that
someone kind of school to quite quickly before
uh...
for patients who have one immune disease
at higher risk of having other diseases uh... in their associations made in this
regard but we don't have any data necessary at this is there a higher
incidence of people were really trying to do now is just identify kaal many
patients have this and how uh... with the prevalence of these diseases or
missus ongoing war
but
hopefully will be helped
were needed throughout that another organization still has snow
prevalence incidence of these diseases are in
uh... registries of quite important in that regard
and in looking at the time and things are
*** lying down one of the things i really wanted to try to emphasize
uh... to the group is that
well we hate for anyone to have two seasons
i think they're the one thing that is quite clear is that there is a very
strong collaborative spirit crossed that the landscaping
oppose research has lost patient care that because uh... of a number of
different organizations and and people that there is a
forever clever spirit
uh... for doing the research
and for exchanging ideas about how to take care of you as well as your
children
and and what i mean by that well
certainly the f_d_a_ has reached out to
threat think about how do we go
medications to patients a day
uh... a disease
erratic a conference in september or they brought together a number of
investigators to secure the
concerns that
that we haven't how we try to bridge goes together
number of industry representatives that were there and i think that they weren't
urge for that feinerman gauge but that may help us to bring new treatments that
to you
and secondly i think meetings have really focused on this disease and and
about a decade ago this disease really not discussed at all in the major
academic meetings
and fact it was thought about that this was not its these were not this is it's
at all
and what i can tell you how is that they're uh... and significant part of
diseases adult
and largest guest room draw up multiple
meeting up
here which is held in
may have every year
has a number of sorrow it's actually the most
active section
uh... with respect to growth and research
this fictional narrative with respect esophagus stomach that small tests and
so it's a very
important and exciting guard in the allergy meetings
uh... also or i would focus in on this so that
in march but there are in february of this year in san antonio in your allergy
meetings there will be a
day-long symposia
that will focus obviously thoughts opportunites well-adjusted memorable
holds up and uh...
to think about new research projects and how we can try to
improve the care patients overall
m uh... as i said with respect to research one of things that is correct
was trying to capture
our understanding about this and you are critical and hope it s understand that
and the region which is the registry
uh... developed at the uh... cincinnati children's hospital
direction of doctors about me and welcome burgers uh... launching an
effort to capture of the patients who
uh... have these disease this weekend
uh... begin to
understand who and with the characteristics of these patients are
and and help to enroll them in studies are going to be important
uh... for us to do over time
so uh... i guess i just close by saying that these further
uh... i just returned from
world commerce a pediatric gastroenterology
five years ago when i'd been in a trip these diseases without being seen
there clearly are being seen there now
uh... by a number of different investigators and i think we'll see is
more research done in and other studies over time
so uh... farther other i guess more
mobile questions that i could try to answer is that there are a number of
questions are coming on with respect to think specific patience but if there are
other global questions i will be
more than happy to try to
susi
mentions two other types of diseases such as
uh... you're down those which is a connective tissue disease there just
reports of those with respect to
uh... an association again we haven't said that there's a cause-and-effect
the reason the babbitt comes quite important is that what we want to make
sure
but a patients with a word ammo syndrome have that problems with the way that
uh... the tissues are held together and work
the reason that this is quite important is that
will lead us to be as one of the things that we do we're doing try to understand
disease
uh... activity in patients with a look at what's in their homes in connective
tissue diseases
are actually be increased risk of perforation soon
quite cautious at the should do that we have not insisting that the natural
and possible that
issue a passing on these diseases to to uh... subsequent children
you were really the state is now but in a foreign
the uh...
the jeans there is a list and we're not really
to appoint an unnatural one will be to the point of being able to estimate how
often
uh... with the like little passing these on to children news right now
bananas impose on the law be happening in september is
uh... that meeting itself as the american academy about he has many
immunology if you go to
everyday a case of forays
an empire dot order
the symposium as the tiger symposium there will be held their end
and and it just came on the boat
this train here so far they i dot org
seeing
news securely to diagnose and informed before they have that not sample food so
again i think uh... researchers has not
allowed us to really understand when the disease develops um... and are they're
prevented of ways to keep the disease from happening
uh... i don't think that were i don't know that yet and and waste actually
uh... perhaps modify died before him
the prospect for studies and treatments in the lower intestinal tract i think
it's is something that will see and and i i hope that we continue to
address that acts that were doing work and that is what's
i think others are the challenges then again by having at patients who want to
enroll in studies and having enough patience to
validate the results that are going on but i would encourage you to
and maintain
animal are in the ball and and hope him on those and certainly in one region
yet but if you're so label
them hope aisa fillets in our arms having ruled out an folks with
evites unhide blood counts usually it anyway
uh... i think were researchers call this again is that there is
a number of different causes for
having increased numbers of your samples mclaren
that in in and out patient serbia slobodan gpr diseases those numbers will
go down
uh... phone
so but this is uh... been great i thought i'd appreciate this opportunity
uh... you know what the julienne into linden tell you is that i'm
it will list c l_ eighty eight of you online that i will many portrait
non-destructive new they really didn't have that much it's been great to ah...
into
uh... be with you tonight i hope we have the opportunity do this
began in the future and um... we really are are doing everything we can do to
try to help you guys and and k carrier sales as well as your children so
tonight and thank you again
day
and they did for all of the new attended
uh... you will be receiving an email address or assessment about tonight's
events we despite your feedback to be really great
uh... and things like that but it was great questions and doctor freedom was
really nice to hear efficiency
how can anybody