December 2, 2022 • 18 mins
Join Peter and Taylor as they speak with Dr. David Wilcox, who has spent close to 30 years researching a cure and advocating in the medical research community for Glanzmann’s.
Tune in to hear how advances in the medical field have brought him close to a cure and how you can help him get even closer.
DISCLOSURE: Dr. David A. Wilcox, Ph.D., is the inventor, President, and Founder of Platelet Targeted Therapeutics, LLC.
He has an equity interest and intellectual property rights in the company.
Dr. Wilcox is an Associate Professor of The Department of Pediatrics HEM/ONC/BMT at the Medical College of Wisconsin. The Content of this PODCAST does not necessarily represent the official views of the Medical College of Wisconsin or its sponsors.
Mark as Played
Transcript
Episode Transcript
Available transcripts are automatically generated. Complete accuracy is not guaranteed.
Taylor Anne Burtz (00:00):
Peter and I have Dr.
David A.
Wilcox with us today.
He is a researcher at theMedical College of Wisconsin,
and he has been working towardsa cure for Glanzmann's for how
long now?
Dr. David Wilcox (00:12):
We were working on it since 1994.
Almost 30 years
Taylor Anne Burtz (00:18):
since this is our first episode and we have
quite a few people who have kindof joined our community, and
then we'll probably have somepeople listening.
Don't really know anything aboutGlanzmann's Thrombasthenia, can
you explain exactly what it is?
Back in 1918, a doctor Swisspediatrician his name was
Eduardo Glanzmann.
And, and he had a young childcome into his office whose blood
(00:40):
couldn't clot in the test.
This is right after World War I,when it was reported.
So he called it Thrombasthenia,which the platelets are
thrombocytes thrombo means weakplatelets, so they named it
after him.
Glanzmann's Thrombasthenia.
and basically what it is, isthat from an early onset in a
(01:01):
person's.
They experience prolongedbleeding, usually at the mucus
membranes.
It can be terrible if they havea gastrointestinal bleeding, the
molecular basis wasn'tdetermined until the 1970s when
they found out that people withdrugs and ths are missing one of
two proteins it's G P two Bthree A, or called Alpha two B
(01:24):
and beta.
And they form a, a complex onthe surface of platelets that
bind into sticky protein calledfibrinogen.
They help platelets to aggregatetogether and seal up a wound and
then re pull the strands offibrinogen together to stop the
bleeding.
So people who have grosman don'thave those proteins.
(01:45):
And so if you're missing one,neither of the proteins can get
to the surface it could be aqualitative defect, which means
that only a single change getsto the surface, but it's not
working.
Or it can be quantitative defectwhere you don't have any protein
on the surface.
So a type one is person that hasless than 5% of G P two B three
A on their platelet surface, hasno platelet aggregation, and
(02:10):
type two person has 10 to 20% ofG P two B three.
And a person who's called avariant can have where from 20
to a hundred percent of normallevels.
But what's all in common withall three subtypes is that
doesn't matter how much proteinyou have, it doesn't work that
well.
What sparked your interest inGlanzmann's?
Dr. David Wilcox (02:30):
In 1991, I started graduate school actually
at the Medical College ofWisconsin and I got in with Dr.
Peter Newman at the UniversityBlood Research Institute and he
was studying the very.
Gluzman patients to be analyzedby something called pcr, polymer
(02:51):
chain reaction and they foundout that they could use this
technique on gluzman platelets.
And they discovered two defectsand people from Israel and some
people from Iraq.
When I went to work for Dr.
Peter Newman, I was just agraduate student and I asked him
for a project and he said, okaya young boy from France who's
(03:13):
five years old, and we know hehas gone to him, but we don't
know how can you do this?
Technique and find out.
I really loved to solve themystery.
So that was my very first paperin 94, was solving that person's
defect.
And then in, in 19 94, I decidedinstead of re finding out how
(03:37):
these people's platelets aredefective Correct their
condition by getting their bonemarrow stem cells from them, and
then trying to put in thereplacement gene, one that works
perfectly fine into the stemcells and seeing if we could
restore the megasite plateletfunction.
And two people volunteered tosend.
(04:00):
They had their stem cellscollected.
So this was the early version ofgene therapy.
And then we put the gene in andthen we groom in the, in the
tissue culture, and we saw thatwe could get the receptor to the
surface.
And then your mom called meHelen Smith.
She called me about in the year2000.
I got a call in in Milwaukee oneday from her and I was surprised
(04:24):
and she was very determined.
Back then the internet wasn'tused as much as it is today, but
she was very determined to helpher daughter and.
People and children like herdaughter Julia to find a cure
for Gosman.
I wish I could have heard thatphone call I know.
I was just thinking the samething.
Well, she said, okay, so we wereworking with mice then that had
(04:50):
S'S and and she said, wellDavid, how much money do you.
And I set up, you know, maybe wespent like we probably need
about, I don't know, we needed$3,000 for the dog, so we
needed, I don't know how much,thousands of dollars.
And she said, wow, that's a lotof money.
I said, well, it's only like adollar a day for mouse cages.
(05:11):
And she said, oh, I, I think Ican do that.
So I kind of broke it down forher.
And then she started having thefundraisers.
So from that fundraising, wewere able to then treat my
bondsman and try put the cellsback in.
And we showed in around 2000,was in, I wanna say five, that
(05:32):
we could cure mice with.
And then in 2011 we moved on totreat dogs that had with Thro
Taylor Anne Burtz (05:40):
I will tell you, your passion for what you
do is very evident in when youspeak about it.
And we are extremely lucky tohave you dr.
Wilcox, you mentioned about thedogs.
So you did, you did cure it inmice and you had dogs as well.
How did you come across dogswith glands?
Dr. David Wilcox (05:59):
Mens?
Once we cured the mice, then Igot a phone call from Auburn in
Alabama from Mary Boudreau.
She said David, I have dogs herewith Gosman Thro.
Beautiful, great purist dogs.
And you know, I understand thatpeople are a little bit hesitant
when you talk about working withdogs.
(06:21):
But I would never feelcomfortable going to a human
from a mouse.
We were happy because we coulddo a protocol where you would do
it just like the peripheralblood stem cell pheresis.
You give'em a shot.
We had to give'em a little bitof sedation because they would
jump off the table and run awayif we didn't.
But other than that, it was oncewe collect the cells from them.
(06:41):
They wake up and they eat withinabout a half an hour and they're
fine.
So we did pretty much theprotocol.
On dogs that we're proposing todo in people.
Taylor Anne Burtz (06:52):
That actually segues perfect into, you know,
the whole gene therapy thing.
You have explained a little bitwithin your answers what gene
therapy is, but it's pretty, newto the us.
And I know that it's very, verynew within the hemophilia
community.
They are starting to do genetherapy for hemophilia over in
(07:13):
the UK right now.
But what exactly is gene therapyand why, does the medical
community think that it might bethis great big next step?
Dr. David Wilcox (07:23):
If you have glos men, either you not missing
but defective, I guess I wouldsay, or doesn't work properly
and we can put in our genetransfer vector.
A good copy of the normal geneand it will go into the person's
dna, into their parent cells andwill make the proper protein
(07:44):
hopefully for the rest of theperson's life.
We treated three dogs, and aboutsix months after we, gave them
back their own cells we stoppedtheir bleeding at I think it was
from three minutes to sevenminutes.
Which is near normal or normalbleeding types.
And when these are big, hairydogs and when we shave their
(08:06):
legs normally they had petechiaor big bruising all over their
legs, and so we didn't see thatanymore.
Taylor Anne Burtz (08:12):
Gene therapy is way less invasive than bone
marrow transplants.
Correct.
Cause you're not having to goin, kill all of the current,
stem cells.
You are actually using the stemcells that were already in that
person.
Dr. David Wilcox (08:25):
We are doing a bone marrow transplant, but it's
of the person's own cells thathave been genetically modified
and therefore they shouldn'tmake an immune response to their
own cells, which would be hostversus graft effect.
And graft versus host diseaseswhere if they didn't get a
perfect match, there have beengosman patients treated with
(08:48):
bone marrow transplants and theydo.
Lethal, or we call it totalablated, a mylo ablation, which
means you wipe out the entirebone marrow and it can be
dangerous because if the graphdoesn't take, then you need a
backup.
And there have been people whohave been cured with, with bone
marrow transplants, but othershaven't worked as well.
(09:11):
They save that for the patientsthat it seems like no other
options.
We don't have to give a full.
Mylo ablative treatment where wewipe out your entire bone marrow
because it's your own self.
We learned with the dogs that ifyou get about 25, 10 to 20% of
the graft to take, it's called amini transplant you only have to
give a little bit of,chemotherapy just a minor amount
(09:34):
to get a, a little bit of theircells to en graft.
And then you really have moreplatelets in your body than you
need to stop bleeding.
So if we correct just a fractionof'em, I would say hopefully now
with this protocol, we're hopingto get around 25 to 30%.
Platelets that are, have the newgene in them while the rest are
(09:55):
your same old cells.
Taylor Anne Burtz (09:57):
In the human cell and everything, you are
able to correct it on that leveland so, You have been preparing
to potentially to go to the nextstep, which is getting the FDA
approval for a clinical trialWhat are you finding to be your
biggest challenges?
Dr. David Wilcox (10:12):
We take the challenges as they come.
We expect that there are gonnabe challenges.
We are making our vectorsacceptable for the fda.
We have to make a little minoradjustment.
And so one of the problems was,is that we always wonder, are we
going's one out of a millionpeople?
And that's predicted.
To find a person who wouldvolunteer for the study.
(10:36):
When it's only one out of amillion people you would say,
okay, maybe there's 300 peoplein America in USA that are, and
they have to be an adult too.
You have to get your vectorsready.
You have to test them in, thetissue culture environment.
We have our papers with the dogsand the mice to back us up as
preclinical data.
And then you have to apply tothe FDA for an application to do
(10:58):
a trial.
We probably would propose aphase one trial, which is a
safety study to see how safe itis on maybe two people but I
would say it's doable If we canget the funding to get a, we
call their investigational drugapplications, i n d.
If we get that approved, then wecould move forward The Gosman
(11:19):
Research Foundation got usthrough all the preclinical with
donations from people, frompatients from the foundation.
Got us through all that testingand we got some NIH grants to
help.
Taylor Anne Burtz (11:31):
The National Hemophilia Foundation's starting
to focus a little bit more onultra rares.
It seems like a couple of otheryou know, organizations are
starting to focus on ultra raresand then we're seeing a lot of
pharmaceutical companies kind offocusing and turning their heads
our way as well.
Dr. David Wilcox (11:47):
When you can see things working.
And even though I can tell you Igot many notebooks and I have a
few pages, I call'em the goldenPages where everything worked
after so many tries.
You just cherish those momentsso it does take a while.
You have to have a very You knowa lot of persistence and
patience.
So hang in there.
I do appreciate everyone who hassent me Christmas pictures over
(12:12):
the years, t-shirts, towels evenjust pictures or Christmas cards
and things like that.
And it makes me happy to, to seethese things, you know?
I mean, it just, I haven'tforgotten about anyone.
My SVA Kuku, who's also my wifeI asked her because she's better
off Facebook and she keeps trackof the database she keeps me
(12:33):
informed every now and then ofwhat's going on the good things
and the sad and the challengesand so I do know what's going
on.
So, Don't think that I forgotten
Taylor Anne Burtz (12:45):
I know you and mom kept in touch very much
so.
You were talking at least, oncea month and she would kind of
keep you up to date andeverything.
So really, even though.
You're this like myth and likelegend within the community.
You have, you have kept tabskind of on everybody and you do
(13:05):
you care.
I think that that's one of thosethings where not every community
has something like that wherethe people who are working
towards a cure, have become,cheerleaders for the community
and, we're very lucky to haveyou.
I did wanna update you, so Ithink we told you when we saw
you last that we were likearound 600 members for the
(13:29):
support group.
We're at 764.
Dr. David Wilcox (13:33):
That's amazing.
And, because I think Helenpictured that and, but you, we
couldn't grasp it.
You know, if you think about, Imean, maybe more people have
gomen than we know, and maybemore people are carriers Forman
than we know.
But for them to all be able toget together.
(13:54):
I know that I've heard so manytimes that when somebody felt so
isolated and alone that they hadadvancements and then they find
people just that have it also,and that they're not the only
one in the world dealing withthis.
Taylor Anne Burtz (14:10):
everyone.
You've mentioned a couple oftimes your team, now you've
actually had a couple of peopleon your team who have been there
from the very beginning
Dr. David Wilcox (14:19):
in the lab for 20 just reached 20 years.
SAVI came on a little bit after,but she's been there a long
time.
And I also have Lily in my labwho's working on the Factor
eight side.
For hemophilia.
Bone marrow, transplanters,hematologists that see patients
regularly.
Patient advocates.
We have a team in BostonChildren's we had to assemble a
(14:40):
team from Indiana University ofPennsylvania.
So I go to the best groups inthe world and ask them to work
with us so that the best groupsI can find to help.
To do this properly
Taylor Anne Burtz (14:54):
You mentioned in there that you know Boston is
kind of one of the places thathelps and gives a little bit of
feedback this is the first timethat we are announcing this.
But we are going to do our firstever annual Glassman's research
Foundation conference in Bostonof July, 2023.
It's something that mom had beenworking towards for a very long
(15:17):
time.
And, ever since you let me knowwhat your funding goals are now
and how large they are.
I.
Our consensus within the boardof the foundation was we need to
start thinking like what's goingto get potential funders
attention boston is what wedecided for our location.
(15:39):
It is a headquarter for a lot oflarge pharmaceutical companies.
And we wanted to kind of havesomething for people with
Glands, mens who could gettogether.
They can, present to each other.
We'll have some round tablethings.
We'll have some, providers givesome presentations and stuff as
well.
We just wanted something thatwas solely focused on GT and
(16:03):
really be able to get thesepeople together.
Because, like you had saidearlier, it's that moment when
you see somebody with Glands,men's meet somebody else.
Where they'd been told, youknow, they're one in a million,
they're probably never gonnacome across anybody else with
it.
To meet them in person andreally kind of get that sense of
there are other people out therelike me.
Dr. David Wilcox (16:24):
And that's wonderful.
A lot of clinicians like haveseen Gosman or heard of it or,
but you, know, there's still alot of clinicians that need to
be educated because they, knowabout gosman, but they haven't
seen a patient and they need toknow
what
to do and Specialty physicians as well as
(16:44):
the basic science community.
And believe me these rare, theycall'em orphan disorders or
ultra rare disorders.
There is interest in helpingthese people.
When the pharmaceuticalcompanies see that the patients
want this they start to gettingexcited about it.
So congratulations
Taylor Anne Burtz (17:04):
We're hoping.
With everything going, ifeverything goes well, we want
this to be something that's anannual thing, whether or not
it's in, in Boston or not.
But we'll you know, we wanna tryto get as many people there as
possible.
Dr. David Wilcox (17:18):
And a big thing just to throw out there is
we're gonna try and make itavailable for everyone
international as well.
So we're gonna try and dosomething so that you guys can
be there too.
Your mom would be so proud ofyou and so happy.
She made me she said to me oneday, David, I want you to
promise me right now that you'regonna see this through the end
and you're never gonna give up.
(17:40):
And I want you to promise meright now.
I said, Helen, are you serious?
You're gonna ask me to make,I've been working on this for 20
years.
You think I'm gonna give up now?
Just promise me.
And I said, okay, I promise you.
So now I gotta do it.
Taylor Anne Burtz (17:54):
Dr.
Wilcos, thank you so much fordoing this with us.
It was only right to have you asour first guest on the podcast.
You're kind of one of thereasons why the foundation
exists.
Dr. David Wilcox (18:05):
Well, thank you.
I appreciate it.
And we really made some greatfriends in the past and in
hopefully even more new ones inthe future.
It helps to see the people andhear the stories and it
motivates us to do.
To work harder, to work fasterto get things right to know that
(18:28):
you're behind us is so uplifting
Peter and I have Dr.
David A.
Wilcox with us today.
He is a researcher at theMedical College of Wisconsin,
and he has been working towardsa cure for Glanzmann's for how
long now?
Dr. David Wilcox (00:12):
We were working on it since 1994.
Almost 30 years
Taylor Anne Burtz (00:18):
since this is our first episode and we have
quite a few people who have kindof joined our community, and
then we'll probably have somepeople listening.
Don't really know anything aboutGlanzmann's Thrombasthenia, can
you explain exactly what it is?
Back in 1918, a doctor Swisspediatrician his name was
Eduardo Glanzmann.
And, and he had a young childcome into his office whose blood
(00:40):
couldn't clot in the test.
This is right after World War I,when it was reported.
So he called it Thrombasthenia,which the platelets are
thrombocytes thrombo means weakplatelets, so they named it
after him.
Glanzmann's Thrombasthenia.
and basically what it is, isthat from an early onset in a
(01:01):
person's.
They experience prolongedbleeding, usually at the mucus
membranes.
It can be terrible if they havea gastrointestinal bleeding, the
molecular basis wasn'tdetermined until the 1970s when
they found out that people withdrugs and ths are missing one of
two proteins it's G P two Bthree A, or called Alpha two B
(01:24):
and beta.
And they form a, a complex onthe surface of platelets that
bind into sticky protein calledfibrinogen.
They help platelets to aggregatetogether and seal up a wound and
then re pull the strands offibrinogen together to stop the
bleeding.
So people who have grosman don'thave those proteins.
(01:45):
And so if you're missing one,neither of the proteins can get
to the surface it could be aqualitative defect, which means
that only a single change getsto the surface, but it's not
working.
Or it can be quantitative defectwhere you don't have any protein
on the surface.
So a type one is person that hasless than 5% of G P two B three
A on their platelet surface, hasno platelet aggregation, and
(02:10):
type two person has 10 to 20% ofG P two B three.
And a person who's called avariant can have where from 20
to a hundred percent of normallevels.
But what's all in common withall three subtypes is that
doesn't matter how much proteinyou have, it doesn't work that
well.
What sparked your interest inGlanzmann's?
Dr. David Wilcox (02:30):
In 1991, I started graduate school actually
at the Medical College ofWisconsin and I got in with Dr.
Peter Newman at the UniversityBlood Research Institute and he
was studying the very.
Gluzman patients to be analyzedby something called pcr, polymer
(02:51):
chain reaction and they foundout that they could use this
technique on gluzman platelets.
And they discovered two defectsand people from Israel and some
people from Iraq.
When I went to work for Dr.
Peter Newman, I was just agraduate student and I asked him
for a project and he said, okaya young boy from France who's
(03:13):
five years old, and we know hehas gone to him, but we don't
know how can you do this?
Technique and find out.
I really loved to solve themystery.
So that was my very first paperin 94, was solving that person's
defect.
And then in, in 19 94, I decidedinstead of re finding out how
(03:37):
these people's platelets aredefective Correct their
condition by getting their bonemarrow stem cells from them, and
then trying to put in thereplacement gene, one that works
perfectly fine into the stemcells and seeing if we could
restore the megasite plateletfunction.
And two people volunteered tosend.
(04:00):
They had their stem cellscollected.
So this was the early version ofgene therapy.
And then we put the gene in andthen we groom in the, in the
tissue culture, and we saw thatwe could get the receptor to the
surface.
And then your mom called meHelen Smith.
She called me about in the year2000.
I got a call in in Milwaukee oneday from her and I was surprised
(04:24):
and she was very determined.
Back then the internet wasn'tused as much as it is today, but
she was very determined to helpher daughter and.
People and children like herdaughter Julia to find a cure
for Gosman.
I wish I could have heard thatphone call I know.
I was just thinking the samething.
Well, she said, okay, so we wereworking with mice then that had
(04:50):
S'S and and she said, wellDavid, how much money do you.
And I set up, you know, maybe wespent like we probably need
about, I don't know, we needed$3,000 for the dog, so we
needed, I don't know how much,thousands of dollars.
And she said, wow, that's a lotof money.
I said, well, it's only like adollar a day for mouse cages.
(05:11):
And she said, oh, I, I think Ican do that.
So I kind of broke it down forher.
And then she started having thefundraisers.
So from that fundraising, wewere able to then treat my
bondsman and try put the cellsback in.
And we showed in around 2000,was in, I wanna say five, that
(05:32):
we could cure mice with.
And then in 2011 we moved on totreat dogs that had with Thro
Taylor Anne Burtz (05:40):
I will tell you, your passion for what you
do is very evident in when youspeak about it.
And we are extremely lucky tohave you dr.
Wilcox, you mentioned about thedogs.
So you did, you did cure it inmice and you had dogs as well.
How did you come across dogswith glands?
Dr. David Wilcox (05:59):
Mens?
Once we cured the mice, then Igot a phone call from Auburn in
Alabama from Mary Boudreau.
She said David, I have dogs herewith Gosman Thro.
Beautiful, great purist dogs.
And you know, I understand thatpeople are a little bit hesitant
when you talk about working withdogs.
(06:21):
But I would never feelcomfortable going to a human
from a mouse.
We were happy because we coulddo a protocol where you would do
it just like the peripheralblood stem cell pheresis.
You give'em a shot.
We had to give'em a little bitof sedation because they would
jump off the table and run awayif we didn't.
But other than that, it was oncewe collect the cells from them.
(06:41):
They wake up and they eat withinabout a half an hour and they're
fine.
So we did pretty much theprotocol.
On dogs that we're proposing todo in people.
Taylor Anne Burtz (06:52):
That actually segues perfect into, you know,
the whole gene therapy thing.
You have explained a little bitwithin your answers what gene
therapy is, but it's pretty, newto the us.
And I know that it's very, verynew within the hemophilia
community.
They are starting to do genetherapy for hemophilia over in
(07:13):
the UK right now.
But what exactly is gene therapyand why, does the medical
community think that it might bethis great big next step?
Dr. David Wilcox (07:23):
If you have glos men, either you not missing
but defective, I guess I wouldsay, or doesn't work properly
and we can put in our genetransfer vector.
A good copy of the normal geneand it will go into the person's
dna, into their parent cells andwill make the proper protein
(07:44):
hopefully for the rest of theperson's life.
We treated three dogs, and aboutsix months after we, gave them
back their own cells we stoppedtheir bleeding at I think it was
from three minutes to sevenminutes.
Which is near normal or normalbleeding types.
And when these are big, hairydogs and when we shave their
(08:06):
legs normally they had petechiaor big bruising all over their
legs, and so we didn't see thatanymore.
Taylor Anne Burtz (08:12):
Gene therapy is way less invasive than bone
marrow transplants.
Correct.
Cause you're not having to goin, kill all of the current,
stem cells.
You are actually using the stemcells that were already in that
person.
Dr. David Wilcox (08:25):
We are doing a bone marrow transplant, but it's
of the person's own cells thathave been genetically modified
and therefore they shouldn'tmake an immune response to their
own cells, which would be hostversus graft effect.
And graft versus host diseaseswhere if they didn't get a
perfect match, there have beengosman patients treated with
(08:48):
bone marrow transplants and theydo.
Lethal, or we call it totalablated, a mylo ablation, which
means you wipe out the entirebone marrow and it can be
dangerous because if the graphdoesn't take, then you need a
backup.
And there have been people whohave been cured with, with bone
marrow transplants, but othershaven't worked as well.
(09:11):
They save that for the patientsthat it seems like no other
options.
We don't have to give a full.
Mylo ablative treatment where wewipe out your entire bone marrow
because it's your own self.
We learned with the dogs that ifyou get about 25, 10 to 20% of
the graft to take, it's called amini transplant you only have to
give a little bit of,chemotherapy just a minor amount
(09:34):
to get a, a little bit of theircells to en graft.
And then you really have moreplatelets in your body than you
need to stop bleeding.
So if we correct just a fractionof'em, I would say hopefully now
with this protocol, we're hopingto get around 25 to 30%.
Platelets that are, have the newgene in them while the rest are
(09:55):
your same old cells.
Taylor Anne Burtz (09:57):
In the human cell and everything, you are
able to correct it on that leveland so, You have been preparing
to potentially to go to the nextstep, which is getting the FDA
approval for a clinical trialWhat are you finding to be your
biggest challenges?
Dr. David Wilcox (10:12):
We take the challenges as they come.
We expect that there are gonnabe challenges.
We are making our vectorsacceptable for the fda.
We have to make a little minoradjustment.
And so one of the problems was,is that we always wonder, are we
going's one out of a millionpeople?
And that's predicted.
To find a person who wouldvolunteer for the study.
(10:36):
When it's only one out of amillion people you would say,
okay, maybe there's 300 peoplein America in USA that are, and
they have to be an adult too.
You have to get your vectorsready.
You have to test them in, thetissue culture environment.
We have our papers with the dogsand the mice to back us up as
preclinical data.
And then you have to apply tothe FDA for an application to do
(10:58):
a trial.
We probably would propose aphase one trial, which is a
safety study to see how safe itis on maybe two people but I
would say it's doable If we canget the funding to get a, we
call their investigational drugapplications, i n d.
If we get that approved, then wecould move forward The Gosman
(11:19):
Research Foundation got usthrough all the preclinical with
donations from people, frompatients from the foundation.
Got us through all that testingand we got some NIH grants to
help.
Taylor Anne Burtz (11:31):
The National Hemophilia Foundation's starting
to focus a little bit more onultra rares.
It seems like a couple of otheryou know, organizations are
starting to focus on ultra raresand then we're seeing a lot of
pharmaceutical companies kind offocusing and turning their heads
our way as well.
Dr. David Wilcox (11:47):
When you can see things working.
And even though I can tell you Igot many notebooks and I have a
few pages, I call'em the goldenPages where everything worked
after so many tries.
You just cherish those momentsso it does take a while.
You have to have a very You knowa lot of persistence and
patience.
So hang in there.
I do appreciate everyone who hassent me Christmas pictures over
(12:12):
the years, t-shirts, towels evenjust pictures or Christmas cards
and things like that.
And it makes me happy to, to seethese things, you know?
I mean, it just, I haven'tforgotten about anyone.
My SVA Kuku, who's also my wifeI asked her because she's better
off Facebook and she keeps trackof the database she keeps me
(12:33):
informed every now and then ofwhat's going on the good things
and the sad and the challengesand so I do know what's going
on.
So, Don't think that I forgotten
Taylor Anne Burtz (12:45):
I know you and mom kept in touch very much
so.
You were talking at least, oncea month and she would kind of
keep you up to date andeverything.
So really, even though.
You're this like myth and likelegend within the community.
You have, you have kept tabskind of on everybody and you do
(13:05):
you care.
I think that that's one of thosethings where not every community
has something like that wherethe people who are working
towards a cure, have become,cheerleaders for the community
and, we're very lucky to haveyou.
I did wanna update you, so Ithink we told you when we saw
you last that we were likearound 600 members for the
(13:29):
support group.
We're at 764.
Dr. David Wilcox (13:33):
That's amazing.
And, because I think Helenpictured that and, but you, we
couldn't grasp it.
You know, if you think about, Imean, maybe more people have
gomen than we know, and maybemore people are carriers Forman
than we know.
But for them to all be able toget together.
(13:54):
I know that I've heard so manytimes that when somebody felt so
isolated and alone that they hadadvancements and then they find
people just that have it also,and that they're not the only
one in the world dealing withthis.
Taylor Anne Burtz (14:10):
everyone.
You've mentioned a couple oftimes your team, now you've
actually had a couple of peopleon your team who have been there
from the very beginning
Dr. David Wilcox (14:19):
in the lab for 20 just reached 20 years.
SAVI came on a little bit after,but she's been there a long
time.
And I also have Lily in my labwho's working on the Factor
eight side.
For hemophilia.
Bone marrow, transplanters,hematologists that see patients
regularly.
Patient advocates.
We have a team in BostonChildren's we had to assemble a
(14:40):
team from Indiana University ofPennsylvania.
So I go to the best groups inthe world and ask them to work
with us so that the best groupsI can find to help.
To do this properly
Taylor Anne Burtz (14:54):
You mentioned in there that you know Boston is
kind of one of the places thathelps and gives a little bit of
feedback this is the first timethat we are announcing this.
But we are going to do our firstever annual Glassman's research
Foundation conference in Bostonof July, 2023.
It's something that mom had beenworking towards for a very long
(15:17):
time.
And, ever since you let me knowwhat your funding goals are now
and how large they are.
I.
Our consensus within the boardof the foundation was we need to
start thinking like what's goingto get potential funders
attention boston is what wedecided for our location.
(15:39):
It is a headquarter for a lot oflarge pharmaceutical companies.
And we wanted to kind of havesomething for people with
Glands, mens who could gettogether.
They can, present to each other.
We'll have some round tablethings.
We'll have some, providers givesome presentations and stuff as
well.
We just wanted something thatwas solely focused on GT and
(16:03):
really be able to get thesepeople together.
Because, like you had saidearlier, it's that moment when
you see somebody with Glands,men's meet somebody else.
Where they'd been told, youknow, they're one in a million,
they're probably never gonnacome across anybody else with
it.
To meet them in person andreally kind of get that sense of
there are other people out therelike me.
Dr. David Wilcox (16:24):
And that's wonderful.
A lot of clinicians like haveseen Gosman or heard of it or,
but you, know, there's still alot of clinicians that need to
be educated because they, knowabout gosman, but they haven't
seen a patient and they need toknow
what
to do and Specialty physicians as well as
(16:44):
the basic science community.
And believe me these rare, theycall'em orphan disorders or
ultra rare disorders.
There is interest in helpingthese people.
When the pharmaceuticalcompanies see that the patients
want this they start to gettingexcited about it.
So congratulations
Taylor Anne Burtz (17:04):
We're hoping.
With everything going, ifeverything goes well, we want
this to be something that's anannual thing, whether or not
it's in, in Boston or not.
But we'll you know, we wanna tryto get as many people there as
possible.
Dr. David Wilcox (17:18):
And a big thing just to throw out there is
we're gonna try and make itavailable for everyone
international as well.
So we're gonna try and dosomething so that you guys can
be there too.
Your mom would be so proud ofyou and so happy.
She made me she said to me oneday, David, I want you to
promise me right now that you'regonna see this through the end
and you're never gonna give up.
(17:40):
And I want you to promise meright now.
I said, Helen, are you serious?
You're gonna ask me to make,I've been working on this for 20
years.
You think I'm gonna give up now?
Just promise me.
And I said, okay, I promise you.
So now I gotta do it.
Taylor Anne Burtz (17:54):
Dr.
Wilcos, thank you so much fordoing this with us.
It was only right to have you asour first guest on the podcast.
You're kind of one of thereasons why the foundation
exists.
Dr. David Wilcox (18:05):
Well, thank you.
I appreciate it.
And we really made some greatfriends in the past and in
hopefully even more new ones inthe future.
It helps to see the people andhear the stories and it
motivates us to do.
To work harder, to work fasterto get things right to know that
(18:28):
you're behind us is so uplifting
Popular Podcasts
On Purpose with Jay Shetty
I’m Jay Shetty host of On Purpose the worlds #1 Mental Health podcast and I’m so grateful you found us. I started this podcast 5 years ago to invite you into conversations and workshops that are designed to help make you happier, healthier and more healed. I believe that when you (yes you) feel seen, heard and understood you’re able to deal with relationship struggles, work challenges and life’s ups and downs with more ease and grace. I interview experts, celebrities, thought leaders and athletes so that we can grow our mindset, build better habits and uncover a side of them we’ve never seen before. New episodes every Monday and Friday. Your support means the world to me and I don’t take it for granted — click the follow button and leave a review to help us spread the love with On Purpose. I can’t wait for you to listen to your first or 500th episode!
Crime Junkie
Does hearing about a true crime case always leave you scouring the internet for the truth behind the story? Dive into your next mystery with Crime Junkie. Every Monday, join your host Ashley Flowers as she unravels all the details of infamous and underreported true crime cases with her best friend Brit Prawat. From cold cases to missing persons and heroes in our community who seek justice, Crime Junkie is your destination for theories and stories you won’t hear anywhere else. Whether you're a seasoned true crime enthusiast or new to the genre, you'll find yourself on the edge of your seat awaiting a new episode every Monday. If you can never get enough true crime... Congratulations, you’ve found your people. Follow to join a community of Crime Junkies! Crime Junkie is presented by audiochuck Media Company.
Ridiculous History
History is beautiful, brutal and, often, ridiculous. Join Ben Bowlin and Noel Brown as they dive into some of the weirdest stories from across the span of human civilization in Ridiculous History, a podcast by iHeartRadio.