May 6, 2023 • 53 mins
Taylor and Peter chat with Luke Pembroke from Haemnet.
Luke is the Director of Community Engagement with Haemnet and spearheads their podcast, Haemcast. Haemnet is a specialist research and communications consultancy in the bleeding disorder community. You might be familiar with their name; they conducted the Glanzmann's 360 survey. It was during a podcast interview about the Glanzmann's 360 survey with Luke and their Director of Research, Kate Khair, that Peter and Taylor discovered that Luke was born with severe hemophilia B, and during the pandemic, he was a participant in a gene therapy clinical trial. With Dr. Wilcox's promising gene therapy research and a potential cure right around the corner, they knew they wanted to pick Luke's brain more on the process of his gene therapy.
This episode dives into the who and what of Haemnet as well as diving into Luke's own experiences growing up with a severe bleeding disorder and his journey with gene therapy.
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Episode Transcript
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Taylor Anne Burtz (00:00):
welcome back to Bruise, not Broken Life with
Glands Men's Thrombopenia.
For those of you just joining usglands, men's Thrombopenia is an
extremely rare autosomalrecessive disorder of the blood
in which the platelets lackglycoprotein two B three A.
Hence, no clotting can occur andbelieving time is significantly
prolonged.
GT can be life-threatening andis estimated to affect one in
(00:22):
every one.
This podcast is presented by theGlans Man's Research Foundation.
My name's Taylor, and I'm yourhost, and as always, I'm joined
by.
Today we are joined by LukePembroke.
Luke is the director ofCommunity Engagement for Hamnet.
It's a specialist research andcommunications consultancy
working in the bleeding disordercommunity.
(00:45):
Luke is uniquely qualified forhis position with Hamnet
because, well, Luke is ahemophilia.
Peter and I met Luke almost ayear ago when he and Dr.
Kate Care approached us aboutjoining them on their podcast
Hang and discussing Glancemeets.
Luke's personal journeyimmediately inspired and
intrigued us, and we knew weneeded to find a way to
(01:05):
introduce him to our GTcommunity.
So without further ado, Lukewelcome.
Luke Pembroke (01:12):
Thank you very much for having me.
And wow.
Flawless introduction there.
Honestly, I have to go back andrerecord mine on our podcast all
the time, but that was, that wassomething.
You're a natural.
Taylor Anne Burtz (01:22):
Thank you.
I worked hard on this, and Ihave a script now so that I
don't go too.
Off key.
Luke Pembroke (01:30):
It's all in the prep work.
Taylor Anne Burtz (01:32):
Absolutely.
Well, Luke, how are you doing?
You know, the last that thethree of us spoke, while we
spoke to Kate as well, y'allwere preparing to attend the E A
H A D, which first off, what isthat?
Luke Pembroke (01:48):
So it's the European Allied Health.
See, I can't remember the thingoff the topic.
I stumped you with the firstquestion.
Yeah.
So basically everyone justrefers to it as eha.
So you're like, oh, you're goingto eha this year.
And, and I can't remember whatthe exact acronym off the top of
my head is, but essentially it'sa primarily healthcare
professional conference forpeople in Europe who specialize.
(02:13):
Hemophilia and bleedingdisorders.
And to be fair, they also have afair few clinicians from the
states and other parts of theworld attend.
It really has sort of grown inreach over a number of years,
but it's sort of high science.
And the latest developments inbleeding disorders research are
presented through varioussessions and symposia.
And then they have, a big hallof posters and abstracts that
(02:36):
people submit.
And when we attended this year,We had a poster up and presented
the data from the Glands Man's360 study that we, as you
mentioned, sort of been going onnearly a year now in terms of
when we first got it allstarted.
And then Kate Care who workswith me was also presenting
(02:56):
information about the study inthe nurses' pre-conference day
as well.
So they had a lot of nursesattend there who had a
specialist interest in.
Ultra rare bleeding disorders
Taylor Anne Burtz (03:07):
That's really cool.
And especially, I guess thething I don't know if it would
be similar to like the nationalHemophilia's bleeding Disorder
conference.
Sounds like it's a little bitmore like focused on the
research aspect of it.
Which that's always exciting.
I nerd out over some of thatscience.
I may not, I may not understandit, but, but Google is my
(03:28):
friend.
Luke Pembroke (03:29):
Yeah.
The stuff they present is veryhigh science.
I mean, even a lot of thedoctors and researchers, they'll
be the first people to jump onTwitter and go, I.
This presentation I was just inwas amazing, but wow, it's
complicated.
So when you actually see, thescientists and researchers
admitting it's difficult, I, youknow, I'm very happy if I
understand at least sort of like20, 30% of the content.
(03:51):
Cuz some of it is, really highscience and I sat through some
of the platelet disorder and vonWillbrand stuff and that's when
the science gets supercomplicated.
And I always say hemophilia isso simple when you compare it to
all these other bleedingdisorders.
US hemophilias are basic.
Taylor Anne Burtz (04:06):
Well, I will say one thing that Peter and I
look for anytime that we speakwith someone from the bleeding
disorder world is their passion.
Are they passionate about theirwork?
Do they truly wanna see change?
We try not to be jaded orcynical when we meet with the
new people, but after doing thisfor three years, we do maintain
a certain level of wariness.
I will say, You and Kate kind ofbroke through that immediately.
(04:30):
You listened, you cared.
And it just really, it's notsomething that we necessarily
come across on a regular basiswhen we talk to people or when
people ask us about gt.
What feeds your drive to make achange in the bleeding disorder?
Luke Pembroke (04:47):
So I think even before I got involved in, in
Hemnet to begin with, it wasvery much sort of a, a humble
beginning situation.
Kate as a specialist nurse inbleeding disorders, practiced at
Great Ormond Street, had seen,Patients and families with every
bleeding disorder you couldimagine.
And she was attending a nurseround table where our current c
(05:08):
e o Mike was working as afreelance medical writer at the
time.
And Mike immediately noticedKate's passion and drive and the
way she would be verycomfortable in kind of, Calling
things out and, and really sortof maybe sort of upsetting the
apple cart and saying what needsto be done better?
And, and I think Mike sort ofwas immediately attracted to
(05:29):
that energy cap brought.
And then from there they startedon this half of setting up
Hemnet, which primarily was asort of community platform to
encourage nurses and otherallied healthcare professionals
to network share experience.
And then from there, a lot ofthe research grants.
He net managed to secureinvolved a lot of patient
(05:50):
perspective and the qualitativeresearch side brought in these
patient perspectives and werealized that there was so much
data and nuance out there in allof these stories that people had
about growing up with a bleedingdisorder that we realize were
really important to uncover and,and tell those stories through
(06:10):
doing research and, andcollecting the data.
And I think that's really,Drives us is that we know things
can be better for everyone witha bleeding disorder.
we honestly believe that andwith all these treatment
developments that have gone on,gone on in recent years, I think
we all recognize that there isthat risk that the gap continues
(06:32):
to get bigger.
Hemophilia for the smallpercentage in the developed
world who get access to goodtreatments are doing well and
things are positive and thingscan still do better.
But then there's all these othersubsets of this wider bleeding
disorders population that maybesort of get left behind or left
in the shadows a bit.
and we really feel stronglyabout shining a light on these
(06:53):
other areas of the community inthat if things improve, they
have gotta improve for everyonetogether.
And it shouldn't be that sort ofone group of the community gets
it better than others.
You know, whether it's womenwith bleeding disorders, those
people with ultra rare bleedingdisorder.
The priorities should be just ashigh for them as it is for the
plain old hemophilias like me,if you like.
Taylor Anne Burtz (07:13):
I mean your pat, patient-centric attitude,
honestly it's not somethingthat's seen very often,
especially in companies who dothese surveys for pharmaceutical
companies or even just forresearch in general.
The Glands Man's 360 survey isvery unique.
I love it.
(07:33):
I went through it.
I made my sister go through itand fill it out and everything.
Cause I was like, I think theseare really good questions.
Peter.
I think you've, you've taken itas well.
Yep.
Yep.
And just explain the survey andthe, and the thought process
behind the questions, I guess.
Luke Pembroke (07:51):
Well, I guess the biggest challenge to start with,
Glans Man's being a fairlyunrecognized disorder and
they're not really being muchout there in terms of
publications and knowledge inthe scientific community because
of how rare it is and thereforeit maybe doesn't get the
attention from companies,investors, that sort of thing.
(08:12):
So when we are approached by HeHema, about conducting this
natural history study, weimmediately were like, yes, this
is something we'd really love todo.
And it's in a air of bleedingdisorders that is, Pretty
underserved.
And there was so much we wantedto know.
And it was that kind of balanceof like, well, we need to do a
survey to collect, as much dataas possible, but we can't make a
(08:35):
survey that's gonna take peopleages.
Like I know as someone who'sdone surveys on hemophilia for
years, like if you don't get itright and you ask too much,
people switch off.
You don't get the completionrates.
And ultimately that kind ofweakens your data.
So we, we often try andstructure this where we have a
survey where we try and collect.
Sort of more top line data thatreally sort of gives an idea of
(08:56):
how people are impacted on adaily basis by their condition.
And then we, within the survey,give people the opportunity to
opt into a more in-depthinterview where Kate or one of
our other colleagues, Simon, theother research nurse, will, will
do those interviews with them toreally get into the, the nitty
gritty of what it's like to livewith a certain bleeding disorder
(09:16):
such as glands.
So the the survey really sort ofis kind of top line stuff, but
we, we kind of pulled togetherdifferent tools that exist out
there particularly forhemophilia in terms of like EQ
five D or Hemoco and pulled bitsand pieces from those to see how
we could sort of adapt those twobetter understanding glands
(09:37):
mens, for example, because werecognize it is still a rare
bleeding disorder.
There might be some similaritiesand overlap in terms of how it
impacts people.
How hemophilia or VonWillebrands does, but also
recognizing that some things aregoing to be different.
I think the initial thing thatreally stood out for us is the
sort of regular frequency ofexternal bleeding that people
(09:58):
with glands, mens just sort ofput up with on a weekly basis.
And, and the kind of attitudereally just seemed like, well,
you know, it is what it is.
We just have to deal with it andget on with it.
I think in hemophilia we'realways told, oh, you know, like
a nose bleed or a, or a cut orsomething is like treat it basic
first aid and it, and that'sassuming that someone with
hemophilia is on prophylaxisfactors.
(10:19):
So those sort of little externalbleeds don't cause them a
problem.
Whereas people with glands mens,it's like this is actually a
huge impact on people's day today.
But there is this thing with thedisability paradox where you
just kind of normalize somethingthat is abnormal and and you
know, it's just really shockingto.
Wow.
Like people are just having todeal with this and put up with
(10:40):
this, and it's something that'sdefinitely not given as much
recognition as it should be.
Because ultimately, if you'rehaving a nosebleed that lasts
for hours and it's impactinggoing to school or working that
isn't normal and something needsto be done about it.
So even though we can say, andit might seem obvious to you
guys in the Lansman community,like, yeah, of course this stuff
(11:01):
is going on.
It's important that we capturethose insights and that data and
actually put it out there intothe world and go, look, this is
a problem and we need to payattention to it because there
are people out there dealingwith things that they really
shouldn't have to be goingthrough on such a severe level.
Taylor Anne Burtz (11:18):
Yeah, I, I mean, I can guarantee you that.
GT patients aren't telling theirdoctors every bleed that they
have, they're probably not evenrecording every bleed that they
have.
Just knowing, the people that Iknow who I talk to on a regular
basis, cuz it, like you said, ifit's not creating a problem in
that moment, then, then it'sjust a normal day in their
(11:39):
lives.
It's only when it gets to thepoint where they're like, oh
well, okay.
Maybe this nose bleed is alittle too extreme.
Maybe I do need to go to thedoctor.
That's kind of when you know,that's when the medical
intervention happens, whichthere's nothing wrong with that.
You know, being able to takecare of a bleed on your own at
home, but when doctors aren'thearing about it, like you said,
(12:03):
then they don't know about itand they're just ends up being
this gap.
They don't realize how much ittruly is impacting the every
day.
So 10 years ago, would you haveseen yourself in this spot?
Right now as the communicationsdirector of a research and
communications consultancy firmfor bleeding disorders,
Luke Pembroke (12:27):
No, I mean, I, I never really thought I'd end up
working within the hemophiliableeding disorder space.
Like, I mean, living with it 247, right?
You'd think it's enough.
I did biology at universitybecause the hemophilia and
science side of it really didinterest me, but I was more into
sort of like natural historyand, wildlife conservation, that
sort of thing, and went off touniversity and then very quickly
(12:50):
realized, without a sort of abank of mom and dad fallback
plan and not much time to sortof go around doing a bunch of
voluntary stuff that, okay, Ineed to get a job.
And, and I was pretty good atthe biotech side of things and
writing scientific stuff.
So I got a job in healthcarecommunications in, it was like
(13:10):
my first grad job in London andworked across loads of different
disease spaces.
And then had an opportunity togo freelance.
That's when I ended up getting achance to work for Hemnet in a
freelance capacity.
And then as we grew they made mean offer I couldn't refuse.
And I, joined up with Hemnetfull-time because, the work they
(13:30):
were doing felt really importantto me.
It was something I was genuinelyvery passionate about.
And both Mike and.
Treated me as a expert in my ownright and not the sort of like
token patient like, oh, youknow, he's per person with
hemophilia.
He is helpful to have around.
It's kind of like they see me aseverything but a person with
hemophilia.
And that kind of comes last, butthat obviously is helpful in C
(13:53):
in terms of helping me sort ofrelate to what's going on in the
community and.
Yeah, I didn't expect to sort ofbe living and working all
things, bleeding disorders andhemophilia.
But and people do, my friendsask like, doesn't it drive you
crazy?
Isn't it too much?
And I was like, yeah, it does attimes.
But you know, I, the job that Ihad before drove me crazy as
(14:14):
well and I was like, you know,at least at least I can really
be working in something I'mpassionate about in a community
that I really care.
It kind of makes dealing withthe, the stressful, crazy side
of it all worth it.
And, you know, the opportunityto meet so many people in the
community and work on somereally cool projects and, be
able to go to all theseconferences and hear about
really cool nerdy science, whichI already liked is just, it's a
(14:36):
bit of a treat and yeah, itdoes, it, it does make me kind
of chuckle that I've ended upbeing the, heading up community
engagement for Hemnet because.
I think anyone who knows mequite well will say that I'm,
I'm more of a classic introvertthan than, than someone super
extroverted, which for communityengagement doesn't kind of make
(14:57):
sense, but I think that kind ofhelps cuz I'm one of those
people who, who very much wouldrather kind of sit back and
listen to other people sharetheir stories and.
Expert insights rather than kindof like dominate the
conversation all the time, whichI, I think definitely helps in
the the role that I have now.
And yeah, so usually I'm not theone talking on podcasts.
I'm sort of sat there silentlyin the background helping Kate,
(15:19):
you know, produce them and thenI sit there and edit away.
So that's, I think that's what Iprefer.
You know, it's, it's nice to bea fly on the.
Taylor Anne Burtz (15:28):
Oh yeah, I, I work with someone very similar
to that,
Luke Pembroke (15:33):
and yet he still says nothing.
Taylor Anne Burtz (15:35):
He's the man behind the curtain.
Well, okay, so then this leadsstraight into my next question.
Your area of expertise iscommunications and social media.
Instead of just being the manbehind the camera and or behind
the keyboard, you do step out ofthat every once in a while and
(15:56):
you like just looking throughlike your YouTube and looking
through articles and stuff likethat.
You kind of lay it all out therefor everyone.
I guess my question is cuz youinclude people in as much of
your journey as you can, whichis awesome.
It can also, especially withthis day and age of social media
(16:18):
and everything, how do youachieve letting people in, being
vulnerable, but also stillmaintaining a semblance of
privacy, I
Luke Pembroke (16:28):
guess?
That's a really good question.
It's tricky and it's something Igenuinely have struggled with
and I mean, I don't make videospublicly sharing a lot as, as
often as I did.
Because it did become verydraining.
And I, you know, for example, Ihad to come off of Facebook
because I'd get a message everyday from either, you know, a mum
or someone with hemophiliaasking for medical advice and
(16:50):
you know, really worrying stuff.
And all I could do is be like,call your center.
Call your hemophilia center.
Like, I'm not a doctor, don'task me.
And then people from thedeveloping world, just asking if
I could get treatment to them.
And, you know, obviously it'snot really something I'm able to
do.
And so that, that sort of thingis, And trying to strike that
balance can be really tricky.
(17:11):
But I think, you know, it is,it's helpful.
And I've had some really, youknow, positive comments from
people with hemophilia andcarers parents who said that,
you know, they found my videosand they've really helped them.
So that's kind of what makes itworthwhile for me to do.
And yeah, but it, but it isn'teasy.
You do have to find that balanceand, and I think it's kind of
(17:33):
giving yourself permission to.
Share what you are willing toshare and set your own
boundaries.
And if, you know people arehounding after you after certain
bits of information and data,like just switch off from it
and, you know, be comfortable insharing what you wanna share.
So the big example of that is Imade the conscious decision and,
and I discussed it with myhemophilia team prior to
(17:54):
actually receiving gene therapythat I wanted to document.
It was a kind of personalexercise more than anything, but
I felt that there was this kindof iron curtain up between the
community and then those goingthrough the trials of gene
therapy.
And I kind of wanted to tearthat down a little bit and, you
know, sh show how the sausagewas being made, if you like.
(18:14):
And I said to my team, you know,within the first year I fully
intend to avoid mentioning whatmy levels were because in the
context of gene therapy in thefirst year of the trial, What my
levels would be week to week iswholly irrelevant, and it's not
really useful information forthe patient community to have at
(18:34):
that point.
I'm one person, I'm in the firstyear where the variability can
be up and down, and so I made adecision that, you know, I
wasn't gonna reveal that datauntil I at least hit the year
mark, but every week or everytime I put a new post out, it
was just comments and privatemessages from people.
Like, what are your levels?
What, what's going on?
And you know, They wanted everynitty gritty piece of detail,
(18:56):
and I just kind of had to say,look like I'm not willing to
disclose that.
That's kind of personalinformation for me right now,
and I have my reasons for notsharing it you know, for the
benefit of myself, but also forthe community.
And I was like, you know, but ifyou are interested, like, you
know, comment, like, subscribe,and maybe you'll find out in a
year, time, year's time.
(19:16):
But you know, there are peoplewhere if you put yourself out
there a bit, they kind of thenexpect everything from you.
And you know, if you're then notvery sort of Free with handing
out all the information aboutyourself.
People are kind of like, well,why not?
And it's, you know, there aresome things that you just wanna
keep to yourself and you know,that's why I kind of took a bit
of a step back and a break frommaking videos as often because I
(19:37):
found it, you know, quiteexhausting in a lot of ways.
And also having to like listenback to your own voice and edit
it is also drives you mad aswell.
Taylor Anne Burtz (19:46):
So this is gonna be a dual question for
both of you.
Uhoh.
Luke Pembroke (19:52):
Brace yourself, Peter.
Taylor Anne Burtz (19:54):
So we're kind of in a weird spot now with, and
this is still on the vein ofsocial media, and I know Peter's
like, oh, where's she going withthis?
My generation, millennials, youknow, we grew up with the
internet, right?
Like, not throughout our wholelives, but definitely, you know,
to the point where we feltcomfortable, we overshare all of
(20:17):
that, right?
Well now millennials are havingkids.
Some of those kids have thesebleeding disorders or have these
rare bleeding disorders or, youknow, going through any of this
type of stuff.
And so, That aspect of sharingon social media doesn't
necessarily switch off.
(20:37):
And, and then these kids aren'tgrown yet, so they can't tell
us, you know, the, the effectsthat this is having or anything.
Hmm.
But with your own personalexperiences with social media,
with both of y'all having, Iguess both of y'all being
patient advocates and beingapproached and.
Having control now over what youshare with the world and
(20:59):
everything, what do you think?
Like, I guess, not necessarilyput yourself in their shoes,
but, but what is, what do youthink when you see this, when
someone's journey is beingshared so in detail and you know
that they're not at the capacitywhere that they can give
permission for that or not?
Luke Pembroke (21:21):
Yeah, it's a, it is slippery slope.
Like I've seen stuff over theyears that's really quite
concerning in terms of just thelevel of detail that some
parents go to in terms ofsharing everything about what
their kid is going through.
And of course that's a weird onebecause like you say, the child
doesn't really have the capacityto sort of consent to that.
(21:42):
And I think there are somethings that need to be.
private I also do see the, thebenefits to it.
I think it does help for otherparents and other children to
see people like them.
And I think because of socialmedia, the visibility of of
other people who might havesomething like you is a a lot
more frequent.
So you always hear people withbleeding disorders, you know,
10, 20, 30 years ago, didn'treally know anyone else growing
(22:06):
up.
And wouldn't really come acrossanyone else.
And you know, when they finallydid, everyone always talks about
it being a, you know, reallycathartic and beneficial
experience.
I think with social media youkind of give people an
opportunity to go, look, thereare other people like you, you
aren't alone.
But then it is that slipperyslope where it's like, well, you
know, are you showing too much?
(22:27):
Like examples where I've seenlike parents showing like the
gruesome details of every bleedtheir child is having on social
media and I'm like, You know,oh, maybe a bit much.
Right?
Like yeah, you kind of arereliant on people kind of self
calibrating and, and some peopledo it better than others, I
think.
Yeah.
(22:47):
I think it's very reactionary inhow they post in really intense
moments.
And then maybe if they took abeat and, and in a couple of
weeks time they're actuallylike, oh, maybe I should have
like held back a bit there.
Taylor Anne Burtz (22:58):
I guess this is something that's so close to
my heart because, you know andI, I think I've told you this
before, Luke.
It's my little sister who hasglans bins and my mom was who
founded the Glans Men's ResearchFoundation In that effort, you
know, it was okay.
My sister became the face of.
(23:20):
The whole time growing up.
Mm-hmm.
And so I've kind of watched itas a sibling, not as a parent,
so, right.
Like it's a differentperspective when you're watching
your, your sibling go throughit.
And it's hard because you knowthat that parent is doing what
they think is best.
You know, that there'sabsolutely every great intention
(23:40):
to it, but I also feel likethere's also a fallout to it
because, Being not having anysense of personal privacy,
you're eventually going to pay acost for that.
Whether it be, you know, thatthat person no longer feels
comfortable talking about theirdisorder at all.
(24:01):
Yeah.
Or, you know, they just kind ofshut away the outside world or
they go into denial or anythinglike that.
Peter, I mean, what do you, whatdo you think.
Peter Zdziarski (24:11):
It's hard to not go with the 50 50 approach.
I'm trying to pick something alittle bit more definite.
I, I think there has to be anage limit, and I don't know what
that age limit is, but it needsto be the point where you're
actually asking them if theywant to be online and they need
to be able to understand thatthere's a lot of people that's
(24:32):
gonna be able to see this.
Taylor Anne Burtz (24:33):
Yeah, I agree with that.
I think that's good.
And to put it in perspectivethat, I mean, it's online.
Anybody can access it basically.
Luke Pembroke (24:42):
Yeah.
I've, I've seen like some of my,you know, favorite YouTubers I
follow who've, you know, gone tohave children, have made very
conscious decisions to likenever show their children's face
in their vlogs or whatever, andthey sort of edit it very
cleverly where you can't seethem and you know, they say, you
know, once they're at an agewhere I can ask them, do you
wanna be in this video?
And actually explain to themwhat that means for them to.
(25:07):
Have the autonomy and say, yeah,I'm okay with it.
But until that point, they'rekind of like, I'm not gonna
reveal my, you know, children'sface all over social media if
I've got a public profile, whichI totally get and I'm totally
behind.
And you know, I think if from avery, when it comes to a health
condition, if you're sort oflike bearing this child's soul
in terms of every detail aboutwhat it's like, mm-hmm.
(25:28):
For them to live with thiscondition, you know, all of a
sudden they grow up and they'rejust kind of like, well, no,
none of my life has felt likeit's.
Private and they haven't reallyhad control over their story.
And like you say, that couldthen lead to them shutting down
completely.
You know, even from my likeolder perspective, when I first
started making videos, I wasreally into it.
And then it, you know, once youstart doing that and people
(25:50):
expect more of you, it doesbecome quite intense and getting
invited to talks all the time.
Eventually I was like, oh, I, Idunno if I want to end up being
like Mr.
Hemophilia, if you know what Imean.
Yeah.
It, it's draining.
And I've had other people outthere who are very, you know,
public and, and vocal abouttheir story over the years, and
they've all said the same thing.
(26:11):
Like, I, you know, I don't wannaend up being like Mr.
Hemophilia because, I, I don'tknow.
It's, it's not really a labelthat I think I, I think people
wanna be recognized for all theother things that they do and
bring to the world rather thanjust like, oh, my blood doesn't
work.
Like there is more to me.
You know, which it sounds ironicbecause my friends, like when I
(26:31):
was at school, like all mynicknames were like hemophilia
related, but I really sort oflent into it because that kind
of, Dark humor side of thingswas a coping mechanism for me.
It like helped me get throughtough times.
If, you know, I let my friendscall me thin blood or hemo or
whatever, and you know, it, itseems weird that I'm like, oh
yeah, I don't wanna be definedby my hemophilia.
But then on the back of mylever's, hoodie, when I finished
(26:52):
school, it said hemo.
And it was like, hmm, can't haveit both ways, Luke.
But you know, it's the way Ifound a balance, I guess.
Taylor Anne Burtz (27:00):
No, I get that.
I get that.
Especially, you know, my sisterused to be like She, yeah, she
used to always throw it back atus that, you know, she could
bruise easily or anything likethat.
Especially like, and that, I'mgonna let you know right now is
unfair in sibling rivalry typedeals.
Like when you're fighting andyour sibling can be like, oh
yeah, well you can't touch meright now.
(27:22):
Like, I'm a bruise, or I'm gonnatell mom you see this bruise
here, I'm gonna tell mom you didthat.
Like, yeah,
Luke Pembroke (27:29):
my brother didn't care.
He would, he would just straightup be.
Oh, I'm gonna, I'm gonna giveyou a bruise.
Don't be careful.
You know, we'll be on thetrampoline doing all the WWE
moves.
My mom would come running up,running up the garden like, no,
what are you doing?
You're gonna have a bleed?
I was like, yeah, but we justlearnt this cool new move.
And yeah, my brother, mybrother, you know, he credit to
him, you know, despite, youknow, being unaffected.
(27:51):
He was very good at sort of justtreating me like normal.
He said, you know, he's youngerthan me, but I think.
As, as it goes for sort of likehemophilia siblings.
He was very mature about it whenI actually reflect on it.
Taylor Anne Burtz (28:02):
Well, so let's dive into, get away from
the controversial part of this.
Mm-hmm.
Let's talk about your genetherapy journey.
So you had your infusion inFebruary of 2020.
Were you approached, did youfind the clinical trial?
How did that even, how did thatconversation even begin?
Luke Pembroke (28:23):
So it first came, to my knowledge, the, the trial
when I was in for one of mybiannual visits at my hemophilia
center, and I.
Went in and, and I actually sawmy doctor very quickly, which
is, you know, kind of a rarity.
And then it became clear why?
It was like, oh, we have thistrial that's recruiting at the
moment.
You know, here's some moreinformation.
(28:44):
Would you be interested?
And I was like, you know, I'mcurious.
They told me that other people,you know, the first cohort had
already been dosed and, youknow, it was looking promising.
And would I be, you know, opento.
Getting involved.
And, and I mean, as a firststep, it, I had to even see if I
was legible to take part byscreening for the neutralizing
(29:05):
antibodies against the, thevector they use to deliver the
gene therapy.
So my, my mindset was kind oflike, well, it can't hurt to
know, right?
Like, if I find out, if I'm notillegible, then at least I can
clear it out of my mind.
So I did the blood test and itcame back that I would be
suitable to take.
And that gave me a lot to thinkabout.
And so I went away over a coupleof weeks before I came back into
(29:28):
the center and ultimately saidat that point, That would've
been in 2018 that I justcouldn't really commit to it
because I had so much planned inthe diary for work travels
personal stuff throughout 2019and that, you know, it wasn't
the right time for me.
Maybe something to revisit inthe future.
And then role.
(29:48):
Roll around to the end of 2019.
The conversation came up again,and in my head I'm like, okay,
they're really you know,desperate for recruitment
numbers.
That's how trials work.
Like, I, I'm not silly to that.
When you're being approachedabout taking part in a trial you
know, obviously it's a, a uniqueopportunity and it could be
really, you know, life changing,but at the same time, like these
(30:10):
treatment centers do haverecruitment targets to meet,
right?
Like they do need trialparticipants.
So it was always bearing that inmind that I kind of had the
power, I guess.
You know, like the ball was inmy court, if you like.
Mm-hmm.
And.
And then, yeah, I, I wentthrough all the blood screening
again cuz it had been a year, sowe needed to make sure I hadn't
developed those antibodies inthat time.
(30:31):
And spoke to the hemophiliacenter that would actually be
doing the dosing and the teamthere, and then decided that
okay, yeah, I'm, I'm actuallygonna go for this.
I'm, I'm, I've made thedecision, I've given it a lot of
thought.
I've spoken to people in mylife, I've discussed it with
work to make sure it wouldactually be practical and doable
(30:52):
and looked at 2020 and was like,yeah, I can make this work.
You know, plan everything aroundit and try and, you know, fit
the trial into normal everydaylife as much as possible.
So yeah, I was dosed in Februaryof 2020, and then as part of the
trial I went on to.
High doses of steroids tosuppress my immune system to
give the gene therapy the bestchance of taking.
(31:15):
And yeah.
Then I think we all know whathappened in 2020 and so any sort
of planning or ideas I had aboutwhat was gonna happen, all went
out the window because thepandemic just.
Sent everything into absolutechaos.
And so it was a, a very uniqueclinical trial experience to say
the least.
Cuz when you're on high doses ofimmunosuppression in a global
(31:37):
pandemic, everyone startsgetting a little bit panicky.
So
Taylor Anne Burtz (31:41):
yes, I can only imagine.
Well, okay, so you.
An amazing video for the 2021Rare Reach Festival.
That's what I saw on YouTube andI we're gonna share it in the
show notes because I honestlythink that it's, great.
Our researchers, Dr.
Wilcox and everything, he'sworking on gene therapy.
(32:01):
And so having you be able toshare your experience and, and
be on this podcast today,honestly, I feel like it's
shining a light on somethingthat's been talked about for so
long within our community, butis such a big unknown.
So when you start to kind ofanswer those questions as to
what it's like and everything,it, it, it makes your comfort
(32:24):
level a little bit better,right?
It makes you not as panicky.
But there were two things thatreally stood out to me in this
video and it.
When you talked about when youwere going in and for your
infusion and how you couldn'tnecessarily say that you were
scared, but you also couldn'tsay you were excited that it was
(32:45):
like taking a test, like a, Acollege test.
Yeah.
Like you just kind of had thispit in your stomach, like of
nerves basically.
Yeah.
Yeah.
And then you talked about howwhen you were on the
immunosuppressants, You were byyourself and everything, and how
you had every side effectpossible, like anything that
they listed that you couldpotentially have.
(33:07):
You had it right, the migraines,the, the being sick, all of
that.
And you explained how livingwith hemophilia, yes, you're
living with something on aday-to-day basis, but you never
thought of yourself as beingsick because you weren't sick on
a day-to-day basis.
And that really struck mebecause.
(33:29):
I think so many people,especially within our community,
when they, when they watch thisvideo, it's, it'll ring true to
them.
Because I don't think, Peter, Imean, I don't know.
I don't have gt, I don't think alot of our community truly view
(33:49):
themselves as being sick becauseagain, they're not, you know,
dealing.
Vomiting or anything like thaton a day-to-day basis, they
would never compare it to, youknow, what somebody who has
cancer is going through, orsomebody who, you know,
something similar where it takessuch a physical toll on your
body.
If you were to ask somebody withgt, they're like, oh, well I
(34:10):
just bleed.
Like I don't do that.
And, and Peter let me know ifI'm overstepping.
But no, that sounds about it.
When you put that in perspectivelike that, I don't know.
It just, it ran, it hit me, ithit a cord.
And I just really liked thevideo.
Luke Pembroke (34:28):
That was to sort of summarize and give a sense of
what that first year of thetrial was like.
And yeah, it did make me reflecton, you know, having grown up
hemophilia and actually, youknow, for the most part I've
always, you know, been doingokay, like bleeds are painful
and can really wipe you out.
But I wasn't living with, youknow, some of these other rare
conditions out there where itjust seems like these, you know,
(34:49):
poor people are constantlyunwell and.
And you know, that's not reallyhow like hemophilia or sun
bleeding disorders manifest, butat the same time, I guess there
are certain things that you putup with regularly that you just
sort of normalize.
You know, I think of GT and youknow, when it comes to anemia,
like there some people couldvery easily sort of have like a
(35:09):
sort of sub you.
Suboptimal sort of level likebaseline of sort of mild anemia
where they've just sort of gotused to it and it just feels
normal to them.
But like if someone elsesuddenly, you know, was in their
shoes, they'd be like, God, I, Ido not feel well at all.
But it's just what we get usedto, right?
Like it is that normalization ofthe abnormal that I think people
(35:32):
with bleeding disorder.
Tend to do because we're sortof, we're born with these
conditions and, and that's thething I say about gene therapy
now is prior to the genetherapy, people would always
ask, what's it like to live withhemophilia?
And it was the most annoying,complicated question to answer
because I was like, I don'treally know what it's like to
not live with it, so I can'treally give you a really.
(35:53):
Authentic in-depth answer of,you know, what is it like to
live with hemophilia?
How is it different to beingnormal?
Because it was just kind of mynormal, and I would always
compare myself to other normalpeople, but I, I could never
really kind of co-opt thatexperience accurately.
And then since having genetherapy and being made a mild
(36:15):
person, someone with mildhemophilia is what my phenotype
would be classed as.
There are, you know, vastdifferences where I'm like, wow,
there was a lot of stuff I hadto do and put up with when I was
living with severe hemophiliathat I do not miss, that I would
hate to go back to.
Mm-hmm.
And I think it only, only hahaving gene therapy made me
(36:35):
realize just how much of animpact my condition did still
have on me without wanting tosound like.
Ungrateful for the, the goodcare I've had and how fortunate
I am to have grown up in the UKand have not been born during
the contaminated blood era, likerelative to a lot of the
community.
I've had it pretty good, but Ithink there are still some
(36:57):
serious, you know, issues ofgrowing up with hemophilia that
have an impact on you.
And it was only since I had genetherapy that I, I actually
recognized those and gave themmore credit.
Taylor Anne Burtz (37:07):
So what does life look like for you post gene
therapy?
I mean, you've told us, and now,now you're technically a mild
hemophilia.
But like, what is that, youknow, first year, like do you
have to go in every so often?
What, and, and now that you'reat three years, what does that
look like for you?
Luke Pembroke (37:28):
So the, the, the first year.
Massively intense.
In particular, those first sortof six, six to nine months were
very regular hospital visits.
So, you know, started off at thepost dosing going in three times
a week for several months.
That then came down to twice aweek.
Then it became weekly for acouple of months, and then it
(37:49):
went to biweekly.
And, and so the gaps startgetting bigger and bigger.
Then by the, the second yearvisits went down to monthly, and
so I had monthly visitsthroughout year.
Occasionally I'd have to go inan additional time for maybe
some follow up bloods or to havesome scans done on my liver,
whatever it may be.
But my trial team were very goodat trying to get all the stuff
(38:09):
to land on the same day ifpossible.
And because it was still kind ofCovid era a lot of people
weren't able to get.
Appointments at hospital.
So for those tho for thoseyears, even though you know, it
was a really tough time, Iactually kind of had like v i p
access to the hospital, if youlike.
So everything was made asconvenient as it could be.
And then once I got past theyear two mark, so going into
(38:31):
2022 that's when I, my visitswent down to, for the trial
purpose, I only needed to go intwice a year to get my levels
check.
And, you know, have other bloodtests run.
And so that's when I really sortof had the opportunity to kind
of experience.
Life post gene therapy withoutthe trial sort of dominating
(38:53):
day-to-day life.
And so in 2022, I went away forthree months.
I did two months in Peru and amonth in Canada.
And I didn't have to, I tooksome emergency treatment, a
factor with me but I didn't haveto use it once.
It was a good job because by thetime I took out the storage at
the, the camp in Peru, in thejungle, Answer just obliterated
(39:14):
the whole kit.
Thankfully the factor vials werelike sealed, but like all the
other bits were just likecompletely demolished.
I was like, oh dear, that's notgood.
But you know, it was a niceproblem to have that I, I just
didn't have to touch my factoronce and I was, you know, super
active, long hikes, long days,and I didn't have a bleed.
Yeah.
Which is just mind bogglingbecause I wasn't treating with
(39:36):
factor and so I.
Gave me a chance to road testthe gene therapy which is what
I'd kind of been yearning to do.
And Covid had put a lot of thaton hold.
So it was it was nice toactually get out there, put it
to the test, and yeah, when Ithink about when I used to
inject intravenously three timesa week with Factor, I used to
(39:59):
plan so many things around myhemophilia and.
Schedule things around treatmentdays and you know, going on a
work trip or a holiday, havingto account to that extra bag
full of all the treatment andall of a sudden all these little
things I just don't have toworry about.
And I still, now, it happensless and less as time goes on,
(40:20):
but there are those momentswhere I'm having to rush out the
door cause I'm late forsomething or I'm gonna meet
friends at the pub and my brainkind of goes, oh, have you taken
your factor?
And then I'm like, no, wait.
I don't have to do that now.
And it does take time to adjustto and I think that's only
normal, right?
You grow up your whole life witha condition where you're
(40:40):
required to treat constantly andthen all of a sudden you don't
have to do that.
I think there is still that sortof muscle memory or that sort of
trigger built into your brainwhere something happens and your
immediate reaction is like, ah,gotta think about treatment,
gotta think about my hemophiliaand.
It's, you know, it's a niceproblem to have to adjust to
(41:00):
such a different way of life.
And, you know, fingers crossed,long may it continue, but I
understand that I did sign up toa clinical trial.
There are no guarantees.
And, you know, we hope that genetherapy will last a significant
amount of time.
But we're only gonna know.
If that's the case, if we getthe data and people sign up to
trials and I mean, it'sobviously crazy to see within
(41:22):
this past year now approval forhe hemophilia gene therapy for
hemophilia A and hemophilia B.
You know, it all seems to havejust suddenly arrived in, in one
go.
And I'm just baffled that it'sthree years ago I was sort of
embarking on the trial and I'mlike, wow, like.
You know, really do seem to bemoving at this increasingly
(41:43):
right.
Financial rate in development.
But it, but again, it all seemsto be happening in hemophilia.
Yeah.
And that gap between hemophiliaand all the other bleeding
disorders just feels like it'sgetting bigger.
Taylor Anne Burtz (41:55):
Well, I know, and this is something that we
hear all the time and it'ssomething that, you know, we've
talked about with Dr.
Wilcox and stuff.
The money is in hemophilia, it'seasier to get approved for
something for a clinical trialwith hemophilia than it is with
one of these, you know ultrarares or something, because it's
(42:18):
not necessarily, you know, that,that risk factor, right?
Like, okay, if we're gonna throwmillions of dollars into this
and it doesn't work, like ookay, but.
What do, what did we learn fromthis really?
So the fact that, you know, youdid your trial or you're in the
middle of your trial, but youstarted your trial three years
(42:38):
ago, and now just within thepast three years, there's so
many things that have beenapproved.
I mean, you can, you come from avery similar world as we do to
see that many things getapproved in such a short amount
of time is.
The speed of light in thisworld.
Mm-hmm.
(42:58):
Honestly, because we, I mean wehave, we have a running a joke.
Oh, you know, in just 10 yearswe could have a cure in just 10
years.
We could have a cure.
Like it's, I looked at one of mymom's old interviews and she was
like, I'm hoping that by thetime Julia's 2021, that we will
have cured this thing.
(43:19):
And you know, Julia's now 24and.
And it really is just kind ofthe approvals, the getting
everything set and, and now thatthe legwork has been done,
hopefully that means everythingelse will speed up.
And so, yes, it may seem weirdthat we're talking to you, a
hemophilia who went through genetherapy, but honestly, in order
(43:43):
for gene therapy for Glands,mens to happen.
There has to be success in genetherapy for hemophilia.
Yeah.
And so that's, that's why it'sso important to have these
conversations and also to get toknow the rest of the bleeding
disorder world as well, becauseit may not be the exact same
thing, but your experiences arevery similar to experiences
(44:06):
within our own community.
And I'm, I'm all for bringingnew ideas, brainstorming and
meeting new people just to.
Increase awareness for us andget, and get the word out I
guess.
Yeah.
So with all of that said aboutgene therapy and how quickly
(44:26):
it's moved, just within the pastcouple of years, what do you
think the future of bleedingdisorders looks like?
Luke Pembroke (44:33):
I think it's gonna be a lot more options.
I think treatment options will,will increase massively.
I think the main challenge ismaking sure that there are
options for people.
Who don't just have hemophiliaor, you know, standard
hemophilia A without inhibitors.
I think, you know, we've seen alot of development come along
recently, and now people arealmost somewhat spoiled for
(44:55):
choice.
I mean, we, I won't get into itnow in terms of what patient
choice really is.
And it varies between countriesand rules and insurers and
payers in terms of what youactually get.
But I think the community is ina good place to have more
treatment options movingforward.
I, you know, from my sciencebackgrounds and also personally,
(45:15):
I, I, you know, I'm reallyhopeful about the idea of
genetic medicines and genetherapies and, you know, people
would consider the gene therapyI had as sort of first
generation gene therapy.
And the technology's only gonnaimprove and get better as as
time goes on.
So I am hopeful that geneticmedicines will offer.
Treatment options to peoplewhere, you know, maybe it won't
(45:39):
technically be considered acure, but you'll pretty much be
able to live a life free of yoursymptoms for the most part.
And you'll just have to havesort of treatment plans in place
for those unexpected things in,in your life, whether it's
surgery or you know, badaccident, for example.
And, and I really do hope thatgenetic medicines, gene
(46:01):
therapies, Made available topeople with other rare bleeding
disorders and just other rarediseases in general.
That's something that I sort ofhighlighted in the video that
ultimately I think gene therapycan have a much more meaningful
difference and impact on peopleoutside of hemophilia and I, and
I really do hope to see that.
(46:23):
But as you say, hemophilia,multi-billion pound industry and
you know, you've got investorsleft and right, all these
companies and, and it is wherethe money is and, and that's why
the research tends to get donethere first.
But hemophilia offers itself asa really useful Petri dish if
you like, to kind of developthese medicines and hopefully
then expand them out.
(46:45):
Other conditions that I would,you know, hazard to say are
perhaps more in need and moredeserving than hemophilia.
Not to say that people withhemophilia don't deserve the
best treatments, but you know,if you've got access to all
these other great treatmentsoutside of gene therapy that
exist and you're, you know,fortunate enough to be in a
country that has access to them,you can live a pretty much near
(47:05):
to normal life now.
And you know, I come acrossparents and younger people with
bleeding disorder.
Youth camps and you know, justseeing people run around and do
things that I was tolddefinitely not to do when I was
younger.
And it just makes me realizethat, wow, you know, even, even
in sort of 20 years, so much haschanged and I think.
(47:28):
Things can only continue toimprove as, as we move forwards.
Provided we all do our part aspatient advocates and we make
sure our voice is continuouslyheard throughout the development
process.
You know, we have our say inthose, those clinical programs
and, and that that voice isn'tlost.
And we gotta avoid that slipperyslope of kind of becoming
victims of our own success.
(47:50):
You know, if treatments get sogood that it kind of normalizes
people, there are other thingsthat can kind of slip off the
agenda that we need to make surewe, you know, we catch those and
that, you know, we have goodtreatments.
But that, you know, it canalways be better.
You know, I think we, you know,we, we always strive for
excellence as much as wepossibly can, and I think that's
why things have.
(48:11):
Gotten so good for people in thecommunity over tho those last
couple of decades
Taylor Anne Burtz (48:17):
we're, we're huge advocates for that.
Like, yes, you may have onemedication or whatever, but like
that works for the majority ofpeople within the community, but
we're the options.
What happens if it stopsworking?
You know, so we're, we're hugeadvocates of that.
Like, we want options.
Give us all of the options.
Luke Pembroke (48:36):
Yeah, exactly.
Taylor Anne Burtz (48:38):
Well, You're joining us for our conference in
July, right?
I know Kate is, but are youcoming as well?
Yes.
Luke Pembroke (48:44):
Yes.
Perfect.
I'll be there.
I will be there.
So if anyone listening in tothis podcast is also going to be
attending, Then yeah, just lookfor me, and I know I've had gene
therapy, but I don't have anylike extra arms or, you know,
third eye that's developed.
So it might not be obvious thatI'm a genetically modified
individual now.
Taylor Anne Burtz (49:04):
Oh, this is where like my dark humor comes
in and be like, let's get a nametag calling you specimen number,
whatever.
Luke Pembroke (49:10):
Yeah, exactly.
I mean, I like the worst memberof the X-Men possible.
Right?
Like it's your superpower.
Oh I bleed a lot, so,
Taylor Anne Burtz (49:20):
Well, last but not least, I asked this
pretty much of everybody that wetalked to.
What would you say to youryounger self, to the boy who was
fighting the debilitating painof a bleeding disorder every
day?
What would you say now to him
Luke Pembroke (49:35):
to stick with it and you know, don't let it
define you like your hemophiliadoesn't have to be.
It's the dominating thing inyour life.
There are so many things to lookforward to.
When I was younger, I didn'tthink I'd have so many
connections in the community andthat, you know, you are gonna
(49:55):
find people and connect withpeople that become lifelong
friends.
That, you know, the reasonyou're friends with them isn't,
you know, down to you havinghemophilia.
But it, it started off as that,that initial connection and you
know that it's an amazingcommunity that.
Actually lucky to be a part of.
And that whilst there's a, youknow, a little kid who was being
(50:15):
told he couldn't do things washeartbreaking at the time that
ultimately it's kind of wateroff the duck's back as we say
now as I'm moving into being anadult.
And I'd also say to him,Football isn't the be all and
end all.
If I had a chance to go back tomy younger self and, and tell
him to get involved in moreunique like niche sports, that's
(50:36):
what I would do.
Because now I'm like too old anduncoordinated to figure out all
these new hobbies.
And I'm always like, ah, I wishI'd have done this sport when I
was younger instead of justhounding after football, you
know, desperate to be agoalkeeper, a, a mighty five
foot, 10 thinking, you know,it's not the hemophilia that
held me back.
It was my lack of talent andheight.
So, you know, can't blame it onthe hemophilia younger Luke, you
(50:58):
know, get over it and focus onsome other sports, and you might
have actually made it as a, youknow, a sportsman somewhere
else.
Taylor Anne Burtz (51:05):
Well, if you are interested in Hume's Glands
Man's 360 survey and would liketo participate, we are going to
link it in the show notes.
Luke, I am going to do a coupleof news items and then we will
say goodbye to you.
But first, I do wanna thank youfor doing this.
Like I've mentioned multipletimes throughout this, you stuck
(51:25):
out to Peter and I, you're veryeasy to talk to.
You're open.
And it was something that wewanted to share with our
community, so we're, we're verylucky to have met you.
So for our news items,registration is open for the
2023 G R F EducationalConference in Boston for this
upcoming July.
(51:46):
Applications for lodging grantsare open until May 15th, and you
can find more information.
We've been updating it on thewebsite www.curegt.org.
Also, if you or a family memberhas gt, definitely check out our
support group on Facebook.
(52:06):
It is a space for people with GTand caregivers or family
members.
It's, it's a pretty.
That's pretty much all who's inthat group.
We try to keep it as private aspossible, so that is a safe
space for everybody.
Luke, again, thank you.
Luke Pembroke (52:27):
No problem.
I really enjoyed it.
And yeah, hopefully I explainedthe sort of gene therapy stuff
in an accessible way, cuzsometimes I, I look back over
all those years and I go, youknow, actually what did happen?
So yeah, if anyone is curious,like you said, you, you'll put
links to the video in the, inthe show notes.
So, questions, comments thatpeople might have, like if they
leave them on YouTube orInstagram or whatever it may be.
(52:49):
I always do my best to get backto people and answer as well.
So, you know, don't be afraidto, to reach out if you do have
any other questions.
Taylor Anne Burtz (52:57):
And as always, please like and
subscribe to this podcast.
Leave a review if you feel soinclined.
And remember that you may be onein a million, but you are not
alone.
welcome back to Bruise, not Broken Life with
Glands Men's Thrombopenia.
For those of you just joining usglands, men's Thrombopenia is an
extremely rare autosomalrecessive disorder of the blood
in which the platelets lackglycoprotein two B three A.
Hence, no clotting can occur andbelieving time is significantly
prolonged.
GT can be life-threatening andis estimated to affect one in
(00:22):
every one.
This podcast is presented by theGlans Man's Research Foundation.
My name's Taylor, and I'm yourhost, and as always, I'm joined
by.
Today we are joined by LukePembroke.
Luke is the director ofCommunity Engagement for Hamnet.
It's a specialist research andcommunications consultancy
working in the bleeding disordercommunity.
(00:45):
Luke is uniquely qualified forhis position with Hamnet
because, well, Luke is ahemophilia.
Peter and I met Luke almost ayear ago when he and Dr.
Kate Care approached us aboutjoining them on their podcast
Hang and discussing Glancemeets.
Luke's personal journeyimmediately inspired and
intrigued us, and we knew weneeded to find a way to
(01:05):
introduce him to our GTcommunity.
So without further ado, Lukewelcome.
Luke Pembroke (01:12):
Thank you very much for having me.
And wow.
Flawless introduction there.
Honestly, I have to go back andrerecord mine on our podcast all
the time, but that was, that wassomething.
You're a natural.
Taylor Anne Burtz (01:22):
Thank you.
I worked hard on this, and Ihave a script now so that I
don't go too.
Off key.
Luke Pembroke (01:30):
It's all in the prep work.
Taylor Anne Burtz (01:32):
Absolutely.
Well, Luke, how are you doing?
You know, the last that thethree of us spoke, while we
spoke to Kate as well, y'allwere preparing to attend the E A
H A D, which first off, what isthat?
Luke Pembroke (01:48):
So it's the European Allied Health.
See, I can't remember the thingoff the topic.
I stumped you with the firstquestion.
Yeah.
So basically everyone justrefers to it as eha.
So you're like, oh, you're goingto eha this year.
And, and I can't remember whatthe exact acronym off the top of
my head is, but essentially it'sa primarily healthcare
professional conference forpeople in Europe who specialize.
(02:13):
Hemophilia and bleedingdisorders.
And to be fair, they also have afair few clinicians from the
states and other parts of theworld attend.
It really has sort of grown inreach over a number of years,
but it's sort of high science.
And the latest developments inbleeding disorders research are
presented through varioussessions and symposia.
And then they have, a big hallof posters and abstracts that
(02:36):
people submit.
And when we attended this year,We had a poster up and presented
the data from the Glands Man's360 study that we, as you
mentioned, sort of been going onnearly a year now in terms of
when we first got it allstarted.
And then Kate Care who workswith me was also presenting
(02:56):
information about the study inthe nurses' pre-conference day
as well.
So they had a lot of nursesattend there who had a
specialist interest in.
Ultra rare bleeding disorders
Taylor Anne Burtz (03:07):
That's really cool.
And especially, I guess thething I don't know if it would
be similar to like the nationalHemophilia's bleeding Disorder
conference.
Sounds like it's a little bitmore like focused on the
research aspect of it.
Which that's always exciting.
I nerd out over some of thatscience.
I may not, I may not understandit, but, but Google is my
(03:28):
friend.
Luke Pembroke (03:29):
Yeah.
The stuff they present is veryhigh science.
I mean, even a lot of thedoctors and researchers, they'll
be the first people to jump onTwitter and go, I.
This presentation I was just inwas amazing, but wow, it's
complicated.
So when you actually see, thescientists and researchers
admitting it's difficult, I, youknow, I'm very happy if I
understand at least sort of like20, 30% of the content.
(03:51):
Cuz some of it is, really highscience and I sat through some
of the platelet disorder and vonWillbrand stuff and that's when
the science gets supercomplicated.
And I always say hemophilia isso simple when you compare it to
all these other bleedingdisorders.
US hemophilias are basic.
Taylor Anne Burtz (04:06):
Well, I will say one thing that Peter and I
look for anytime that we speakwith someone from the bleeding
disorder world is their passion.
Are they passionate about theirwork?
Do they truly wanna see change?
We try not to be jaded orcynical when we meet with the
new people, but after doing thisfor three years, we do maintain
a certain level of wariness.
I will say, You and Kate kind ofbroke through that immediately.
(04:30):
You listened, you cared.
And it just really, it's notsomething that we necessarily
come across on a regular basiswhen we talk to people or when
people ask us about gt.
What feeds your drive to make achange in the bleeding disorder?
Luke Pembroke (04:47):
So I think even before I got involved in, in
Hemnet to begin with, it wasvery much sort of a, a humble
beginning situation.
Kate as a specialist nurse inbleeding disorders, practiced at
Great Ormond Street, had seen,Patients and families with every
bleeding disorder you couldimagine.
And she was attending a nurseround table where our current c
(05:08):
e o Mike was working as afreelance medical writer at the
time.
And Mike immediately noticedKate's passion and drive and the
way she would be verycomfortable in kind of, Calling
things out and, and really sortof maybe sort of upsetting the
apple cart and saying what needsto be done better?
And, and I think Mike sort ofwas immediately attracted to
(05:29):
that energy cap brought.
And then from there they startedon this half of setting up
Hemnet, which primarily was asort of community platform to
encourage nurses and otherallied healthcare professionals
to network share experience.
And then from there, a lot ofthe research grants.
He net managed to secureinvolved a lot of patient
(05:50):
perspective and the qualitativeresearch side brought in these
patient perspectives and werealized that there was so much
data and nuance out there in allof these stories that people had
about growing up with a bleedingdisorder that we realize were
really important to uncover and,and tell those stories through
(06:10):
doing research and, andcollecting the data.
And I think that's really,Drives us is that we know things
can be better for everyone witha bleeding disorder.
we honestly believe that andwith all these treatment
developments that have gone on,gone on in recent years, I think
we all recognize that there isthat risk that the gap continues
(06:32):
to get bigger.
Hemophilia for the smallpercentage in the developed
world who get access to goodtreatments are doing well and
things are positive and thingscan still do better.
But then there's all these othersubsets of this wider bleeding
disorders population that maybesort of get left behind or left
in the shadows a bit.
and we really feel stronglyabout shining a light on these
(06:53):
other areas of the community inthat if things improve, they
have gotta improve for everyonetogether.
And it shouldn't be that sort ofone group of the community gets
it better than others.
You know, whether it's womenwith bleeding disorders, those
people with ultra rare bleedingdisorder.
The priorities should be just ashigh for them as it is for the
plain old hemophilias like me,if you like.
Taylor Anne Burtz (07:13):
I mean your pat, patient-centric attitude,
honestly it's not somethingthat's seen very often,
especially in companies who dothese surveys for pharmaceutical
companies or even just forresearch in general.
The Glands Man's 360 survey isvery unique.
I love it.
(07:33):
I went through it.
I made my sister go through itand fill it out and everything.
Cause I was like, I think theseare really good questions.
Peter.
I think you've, you've taken itas well.
Yep.
Yep.
And just explain the survey andthe, and the thought process
behind the questions, I guess.
Luke Pembroke (07:51):
Well, I guess the biggest challenge to start with,
Glans Man's being a fairlyunrecognized disorder and
they're not really being muchout there in terms of
publications and knowledge inthe scientific community because
of how rare it is and thereforeit maybe doesn't get the
attention from companies,investors, that sort of thing.
(08:12):
So when we are approached by HeHema, about conducting this
natural history study, weimmediately were like, yes, this
is something we'd really love todo.
And it's in a air of bleedingdisorders that is, Pretty
underserved.
And there was so much we wantedto know.
And it was that kind of balanceof like, well, we need to do a
survey to collect, as much dataas possible, but we can't make a
(08:35):
survey that's gonna take peopleages.
Like I know as someone who'sdone surveys on hemophilia for
years, like if you don't get itright and you ask too much,
people switch off.
You don't get the completionrates.
And ultimately that kind ofweakens your data.
So we, we often try andstructure this where we have a
survey where we try and collect.
Sort of more top line data thatreally sort of gives an idea of
(08:56):
how people are impacted on adaily basis by their condition.
And then we, within the survey,give people the opportunity to
opt into a more in-depthinterview where Kate or one of
our other colleagues, Simon, theother research nurse, will, will
do those interviews with them toreally get into the, the nitty
gritty of what it's like to livewith a certain bleeding disorder
(09:16):
such as glands.
So the the survey really sort ofis kind of top line stuff, but
we, we kind of pulled togetherdifferent tools that exist out
there particularly forhemophilia in terms of like EQ
five D or Hemoco and pulled bitsand pieces from those to see how
we could sort of adapt those twobetter understanding glands
(09:37):
mens, for example, because werecognize it is still a rare
bleeding disorder.
There might be some similaritiesand overlap in terms of how it
impacts people.
How hemophilia or VonWillebrands does, but also
recognizing that some things aregoing to be different.
I think the initial thing thatreally stood out for us is the
sort of regular frequency ofexternal bleeding that people
(09:58):
with glands, mens just sort ofput up with on a weekly basis.
And, and the kind of attitudereally just seemed like, well,
you know, it is what it is.
We just have to deal with it andget on with it.
I think in hemophilia we'realways told, oh, you know, like
a nose bleed or a, or a cut orsomething is like treat it basic
first aid and it, and that'sassuming that someone with
hemophilia is on prophylaxisfactors.
(10:19):
So those sort of little externalbleeds don't cause them a
problem.
Whereas people with glands mens,it's like this is actually a
huge impact on people's day today.
But there is this thing with thedisability paradox where you
just kind of normalize somethingthat is abnormal and and you
know, it's just really shockingto.
Wow.
Like people are just having todeal with this and put up with
(10:40):
this, and it's something that'sdefinitely not given as much
recognition as it should be.
Because ultimately, if you'rehaving a nosebleed that lasts
for hours and it's impactinggoing to school or working that
isn't normal and something needsto be done about it.
So even though we can say, andit might seem obvious to you
guys in the Lansman community,like, yeah, of course this stuff
(11:01):
is going on.
It's important that we capturethose insights and that data and
actually put it out there intothe world and go, look, this is
a problem and we need to payattention to it because there
are people out there dealingwith things that they really
shouldn't have to be goingthrough on such a severe level.
Taylor Anne Burtz (11:18):
Yeah, I, I mean, I can guarantee you that.
GT patients aren't telling theirdoctors every bleed that they
have, they're probably not evenrecording every bleed that they
have.
Just knowing, the people that Iknow who I talk to on a regular
basis, cuz it, like you said, ifit's not creating a problem in
that moment, then, then it'sjust a normal day in their
(11:39):
lives.
It's only when it gets to thepoint where they're like, oh
well, okay.
Maybe this nose bleed is alittle too extreme.
Maybe I do need to go to thedoctor.
That's kind of when you know,that's when the medical
intervention happens, whichthere's nothing wrong with that.
You know, being able to takecare of a bleed on your own at
home, but when doctors aren'thearing about it, like you said,
(12:03):
then they don't know about itand they're just ends up being
this gap.
They don't realize how much ittruly is impacting the every
day.
So 10 years ago, would you haveseen yourself in this spot?
Right now as the communicationsdirector of a research and
communications consultancy firmfor bleeding disorders,
Luke Pembroke (12:27):
No, I mean, I, I never really thought I'd end up
working within the hemophiliableeding disorder space.
Like, I mean, living with it 247, right?
You'd think it's enough.
I did biology at universitybecause the hemophilia and
science side of it really didinterest me, but I was more into
sort of like natural historyand, wildlife conservation, that
sort of thing, and went off touniversity and then very quickly
(12:50):
realized, without a sort of abank of mom and dad fallback
plan and not much time to sortof go around doing a bunch of
voluntary stuff that, okay, Ineed to get a job.
And, and I was pretty good atthe biotech side of things and
writing scientific stuff.
So I got a job in healthcarecommunications in, it was like
(13:10):
my first grad job in London andworked across loads of different
disease spaces.
And then had an opportunity togo freelance.
That's when I ended up getting achance to work for Hemnet in a
freelance capacity.
And then as we grew they made mean offer I couldn't refuse.
And I, joined up with Hemnetfull-time because, the work they
(13:30):
were doing felt really importantto me.
It was something I was genuinelyvery passionate about.
And both Mike and.
Treated me as a expert in my ownright and not the sort of like
token patient like, oh, youknow, he's per person with
hemophilia.
He is helpful to have around.
It's kind of like they see me aseverything but a person with
hemophilia.
And that kind of comes last, butthat obviously is helpful in C
(13:53):
in terms of helping me sort ofrelate to what's going on in the
community and.
Yeah, I didn't expect to sort ofbe living and working all
things, bleeding disorders andhemophilia.
But and people do, my friendsask like, doesn't it drive you
crazy?
Isn't it too much?
And I was like, yeah, it does attimes.
But you know, I, the job that Ihad before drove me crazy as
(14:14):
well and I was like, you know,at least at least I can really
be working in something I'mpassionate about in a community
that I really care.
It kind of makes dealing withthe, the stressful, crazy side
of it all worth it.
And, you know, the opportunityto meet so many people in the
community and work on somereally cool projects and, be
able to go to all theseconferences and hear about
really cool nerdy science, whichI already liked is just, it's a
(14:36):
bit of a treat and yeah, itdoes, it, it does make me kind
of chuckle that I've ended upbeing the, heading up community
engagement for Hemnet because.
I think anyone who knows mequite well will say that I'm,
I'm more of a classic introvertthan than, than someone super
extroverted, which for communityengagement doesn't kind of make
(14:57):
sense, but I think that kind ofhelps cuz I'm one of those
people who, who very much wouldrather kind of sit back and
listen to other people sharetheir stories and.
Expert insights rather than kindof like dominate the
conversation all the time, whichI, I think definitely helps in
the the role that I have now.
And yeah, so usually I'm not theone talking on podcasts.
I'm sort of sat there silentlyin the background helping Kate,
(15:19):
you know, produce them and thenI sit there and edit away.
So that's, I think that's what Iprefer.
You know, it's, it's nice to bea fly on the.
Taylor Anne Burtz (15:28):
Oh yeah, I, I work with someone very similar
to that,
Luke Pembroke (15:33):
and yet he still says nothing.
Taylor Anne Burtz (15:35):
He's the man behind the curtain.
Well, okay, so then this leadsstraight into my next question.
Your area of expertise iscommunications and social media.
Instead of just being the manbehind the camera and or behind
the keyboard, you do step out ofthat every once in a while and
(15:56):
you like just looking throughlike your YouTube and looking
through articles and stuff likethat.
You kind of lay it all out therefor everyone.
I guess my question is cuz youinclude people in as much of
your journey as you can, whichis awesome.
It can also, especially withthis day and age of social media
(16:18):
and everything, how do youachieve letting people in, being
vulnerable, but also stillmaintaining a semblance of
privacy, I
Luke Pembroke (16:28):
guess?
That's a really good question.
It's tricky and it's something Igenuinely have struggled with
and I mean, I don't make videospublicly sharing a lot as, as
often as I did.
Because it did become verydraining.
And I, you know, for example, Ihad to come off of Facebook
because I'd get a message everyday from either, you know, a mum
or someone with hemophiliaasking for medical advice and
(16:50):
you know, really worrying stuff.
And all I could do is be like,call your center.
Call your hemophilia center.
Like, I'm not a doctor, don'task me.
And then people from thedeveloping world, just asking if
I could get treatment to them.
And, you know, obviously it'snot really something I'm able to
do.
And so that, that sort of thingis, And trying to strike that
balance can be really tricky.
(17:11):
But I think, you know, it is,it's helpful.
And I've had some really, youknow, positive comments from
people with hemophilia andcarers parents who said that,
you know, they found my videosand they've really helped them.
So that's kind of what makes itworthwhile for me to do.
And yeah, but it, but it isn'teasy.
You do have to find that balanceand, and I think it's kind of
(17:33):
giving yourself permission to.
Share what you are willing toshare and set your own
boundaries.
And if, you know people arehounding after you after certain
bits of information and data,like just switch off from it
and, you know, be comfortable insharing what you wanna share.
So the big example of that is Imade the conscious decision and,
and I discussed it with myhemophilia team prior to
(17:54):
actually receiving gene therapythat I wanted to document.
It was a kind of personalexercise more than anything, but
I felt that there was this kindof iron curtain up between the
community and then those goingthrough the trials of gene
therapy.
And I kind of wanted to tearthat down a little bit and, you
know, sh show how the sausagewas being made, if you like.
(18:14):
And I said to my team, you know,within the first year I fully
intend to avoid mentioning whatmy levels were because in the
context of gene therapy in thefirst year of the trial, What my
levels would be week to week iswholly irrelevant, and it's not
really useful information forthe patient community to have at
(18:34):
that point.
I'm one person, I'm in the firstyear where the variability can
be up and down, and so I made adecision that, you know, I
wasn't gonna reveal that datauntil I at least hit the year
mark, but every week or everytime I put a new post out, it
was just comments and privatemessages from people.
Like, what are your levels?
What, what's going on?
And you know, They wanted everynitty gritty piece of detail,
(18:56):
and I just kind of had to say,look like I'm not willing to
disclose that.
That's kind of personalinformation for me right now,
and I have my reasons for notsharing it you know, for the
benefit of myself, but also forthe community.
And I was like, you know, but ifyou are interested, like, you
know, comment, like, subscribe,and maybe you'll find out in a
year, time, year's time.
(19:16):
But you know, there are peoplewhere if you put yourself out
there a bit, they kind of thenexpect everything from you.
And you know, if you're then notvery sort of Free with handing
out all the information aboutyourself.
People are kind of like, well,why not?
And it's, you know, there aresome things that you just wanna
keep to yourself and you know,that's why I kind of took a bit
of a step back and a break frommaking videos as often because I
(19:37):
found it, you know, quiteexhausting in a lot of ways.
And also having to like listenback to your own voice and edit
it is also drives you mad aswell.
Taylor Anne Burtz (19:46):
So this is gonna be a dual question for
both of you.
Uhoh.
Luke Pembroke (19:52):
Brace yourself, Peter.
Taylor Anne Burtz (19:54):
So we're kind of in a weird spot now with, and
this is still on the vein ofsocial media, and I know Peter's
like, oh, where's she going withthis?
My generation, millennials, youknow, we grew up with the
internet, right?
Like, not throughout our wholelives, but definitely, you know,
to the point where we feltcomfortable, we overshare all of
(20:17):
that, right?
Well now millennials are havingkids.
Some of those kids have thesebleeding disorders or have these
rare bleeding disorders or, youknow, going through any of this
type of stuff.
And so, That aspect of sharingon social media doesn't
necessarily switch off.
(20:37):
And, and then these kids aren'tgrown yet, so they can't tell
us, you know, the, the effectsthat this is having or anything.
Hmm.
But with your own personalexperiences with social media,
with both of y'all having, Iguess both of y'all being
patient advocates and beingapproached and.
Having control now over what youshare with the world and
(20:59):
everything, what do you think?
Like, I guess, not necessarilyput yourself in their shoes,
but, but what is, what do youthink when you see this, when
someone's journey is beingshared so in detail and you know
that they're not at the capacitywhere that they can give
permission for that or not?
Luke Pembroke (21:21):
Yeah, it's a, it is slippery slope.
Like I've seen stuff over theyears that's really quite
concerning in terms of just thelevel of detail that some
parents go to in terms ofsharing everything about what
their kid is going through.
And of course that's a weird onebecause like you say, the child
doesn't really have the capacityto sort of consent to that.
(21:42):
And I think there are somethings that need to be.
private I also do see the, thebenefits to it.
I think it does help for otherparents and other children to
see people like them.
And I think because of socialmedia, the visibility of of
other people who might havesomething like you is a a lot
more frequent.
So you always hear people withbleeding disorders, you know,
10, 20, 30 years ago, didn'treally know anyone else growing
(22:06):
up.
And wouldn't really come acrossanyone else.
And you know, when they finallydid, everyone always talks about
it being a, you know, reallycathartic and beneficial
experience.
I think with social media youkind of give people an
opportunity to go, look, thereare other people like you, you
aren't alone.
But then it is that slipperyslope where it's like, well, you
know, are you showing too much?
(22:27):
Like examples where I've seenlike parents showing like the
gruesome details of every bleedtheir child is having on social
media and I'm like, You know,oh, maybe a bit much.
Right?
Like yeah, you kind of arereliant on people kind of self
calibrating and, and some peopledo it better than others, I
think.
Yeah.
(22:47):
I think it's very reactionary inhow they post in really intense
moments.
And then maybe if they took abeat and, and in a couple of
weeks time they're actuallylike, oh, maybe I should have
like held back a bit there.
Taylor Anne Burtz (22:58):
I guess this is something that's so close to
my heart because, you know andI, I think I've told you this
before, Luke.
It's my little sister who hasglans bins and my mom was who
founded the Glans Men's ResearchFoundation In that effort, you
know, it was okay.
My sister became the face of.
(23:20):
The whole time growing up.
Mm-hmm.
And so I've kind of watched itas a sibling, not as a parent,
so, right.
Like it's a differentperspective when you're watching
your, your sibling go throughit.
And it's hard because you knowthat that parent is doing what
they think is best.
You know, that there'sabsolutely every great intention
(23:40):
to it, but I also feel likethere's also a fallout to it
because, Being not having anysense of personal privacy,
you're eventually going to pay acost for that.
Whether it be, you know, thatthat person no longer feels
comfortable talking about theirdisorder at all.
(24:01):
Yeah.
Or, you know, they just kind ofshut away the outside world or
they go into denial or anythinglike that.
Peter, I mean, what do you, whatdo you think.
Peter Zdziarski (24:11):
It's hard to not go with the 50 50 approach.
I'm trying to pick something alittle bit more definite.
I, I think there has to be anage limit, and I don't know what
that age limit is, but it needsto be the point where you're
actually asking them if theywant to be online and they need
to be able to understand thatthere's a lot of people that's
(24:32):
gonna be able to see this.
Taylor Anne Burtz (24:33):
Yeah, I agree with that.
I think that's good.
And to put it in perspectivethat, I mean, it's online.
Anybody can access it basically.
Luke Pembroke (24:42):
Yeah.
I've, I've seen like some of my,you know, favorite YouTubers I
follow who've, you know, gone tohave children, have made very
conscious decisions to likenever show their children's face
in their vlogs or whatever, andthey sort of edit it very
cleverly where you can't seethem and you know, they say, you
know, once they're at an agewhere I can ask them, do you
wanna be in this video?
And actually explain to themwhat that means for them to.
(25:07):
Have the autonomy and say, yeah,I'm okay with it.
But until that point, they'rekind of like, I'm not gonna
reveal my, you know, children'sface all over social media if
I've got a public profile, whichI totally get and I'm totally
behind.
And you know, I think if from avery, when it comes to a health
condition, if you're sort oflike bearing this child's soul
in terms of every detail aboutwhat it's like, mm-hmm.
(25:28):
For them to live with thiscondition, you know, all of a
sudden they grow up and they'rejust kind of like, well, no,
none of my life has felt likeit's.
Private and they haven't reallyhad control over their story.
And like you say, that couldthen lead to them shutting down
completely.
You know, even from my likeolder perspective, when I first
started making videos, I wasreally into it.
And then it, you know, once youstart doing that and people
(25:50):
expect more of you, it doesbecome quite intense and getting
invited to talks all the time.
Eventually I was like, oh, I, Idunno if I want to end up being
like Mr.
Hemophilia, if you know what Imean.
Yeah.
It, it's draining.
And I've had other people outthere who are very, you know,
public and, and vocal abouttheir story over the years, and
they've all said the same thing.
(26:11):
Like, I, you know, I don't wannaend up being like Mr.
Hemophilia because, I, I don'tknow.
It's, it's not really a labelthat I think I, I think people
wanna be recognized for all theother things that they do and
bring to the world rather thanjust like, oh, my blood doesn't
work.
Like there is more to me.
You know, which it sounds ironicbecause my friends, like when I
(26:31):
was at school, like all mynicknames were like hemophilia
related, but I really sort oflent into it because that kind
of, Dark humor side of thingswas a coping mechanism for me.
It like helped me get throughtough times.
If, you know, I let my friendscall me thin blood or hemo or
whatever, and you know, it, itseems weird that I'm like, oh
yeah, I don't wanna be definedby my hemophilia.
But then on the back of mylever's, hoodie, when I finished
(26:52):
school, it said hemo.
And it was like, hmm, can't haveit both ways, Luke.
But you know, it's the way Ifound a balance, I guess.
Taylor Anne Burtz (27:00):
No, I get that.
I get that.
Especially, you know, my sisterused to be like She, yeah, she
used to always throw it back atus that, you know, she could
bruise easily or anything likethat.
Especially like, and that, I'mgonna let you know right now is
unfair in sibling rivalry typedeals.
Like when you're fighting andyour sibling can be like, oh
yeah, well you can't touch meright now.
(27:22):
Like, I'm a bruise, or I'm gonnatell mom you see this bruise
here, I'm gonna tell mom you didthat.
Like, yeah,
Luke Pembroke (27:29):
my brother didn't care.
He would, he would just straightup be.
Oh, I'm gonna, I'm gonna giveyou a bruise.
Don't be careful.
You know, we'll be on thetrampoline doing all the WWE
moves.
My mom would come running up,running up the garden like, no,
what are you doing?
You're gonna have a bleed?
I was like, yeah, but we justlearnt this cool new move.
And yeah, my brother, mybrother, you know, he credit to
him, you know, despite, youknow, being unaffected.
(27:51):
He was very good at sort of justtreating me like normal.
He said, you know, he's youngerthan me, but I think.
As, as it goes for sort of likehemophilia siblings.
He was very mature about it whenI actually reflect on it.
Taylor Anne Burtz (28:02):
Well, so let's dive into, get away from
the controversial part of this.
Mm-hmm.
Let's talk about your genetherapy journey.
So you had your infusion inFebruary of 2020.
Were you approached, did youfind the clinical trial?
How did that even, how did thatconversation even begin?
Luke Pembroke (28:23):
So it first came, to my knowledge, the, the trial
when I was in for one of mybiannual visits at my hemophilia
center, and I.
Went in and, and I actually sawmy doctor very quickly, which
is, you know, kind of a rarity.
And then it became clear why?
It was like, oh, we have thistrial that's recruiting at the
moment.
You know, here's some moreinformation.
(28:44):
Would you be interested?
And I was like, you know, I'mcurious.
They told me that other people,you know, the first cohort had
already been dosed and, youknow, it was looking promising.
And would I be, you know, opento.
Getting involved.
And, and I mean, as a firststep, it, I had to even see if I
was legible to take part byscreening for the neutralizing
(29:05):
antibodies against the, thevector they use to deliver the
gene therapy.
So my, my mindset was kind oflike, well, it can't hurt to
know, right?
Like, if I find out, if I'm notillegible, then at least I can
clear it out of my mind.
So I did the blood test and itcame back that I would be
suitable to take.
And that gave me a lot to thinkabout.
And so I went away over a coupleof weeks before I came back into
(29:28):
the center and ultimately saidat that point, That would've
been in 2018 that I justcouldn't really commit to it
because I had so much planned inthe diary for work travels
personal stuff throughout 2019and that, you know, it wasn't
the right time for me.
Maybe something to revisit inthe future.
And then role.
(29:48):
Roll around to the end of 2019.
The conversation came up again,and in my head I'm like, okay,
they're really you know,desperate for recruitment
numbers.
That's how trials work.
Like, I, I'm not silly to that.
When you're being approachedabout taking part in a trial you
know, obviously it's a, a uniqueopportunity and it could be
really, you know, life changing,but at the same time, like these
(30:10):
treatment centers do haverecruitment targets to meet,
right?
Like they do need trialparticipants.
So it was always bearing that inmind that I kind of had the
power, I guess.
You know, like the ball was inmy court, if you like.
Mm-hmm.
And.
And then, yeah, I, I wentthrough all the blood screening
again cuz it had been a year, sowe needed to make sure I hadn't
developed those antibodies inthat time.
(30:31):
And spoke to the hemophiliacenter that would actually be
doing the dosing and the teamthere, and then decided that
okay, yeah, I'm, I'm actuallygonna go for this.
I'm, I'm, I've made thedecision, I've given it a lot of
thought.
I've spoken to people in mylife, I've discussed it with
work to make sure it wouldactually be practical and doable
(30:52):
and looked at 2020 and was like,yeah, I can make this work.
You know, plan everything aroundit and try and, you know, fit
the trial into normal everydaylife as much as possible.
So yeah, I was dosed in Februaryof 2020, and then as part of the
trial I went on to.
High doses of steroids tosuppress my immune system to
give the gene therapy the bestchance of taking.
(31:15):
And yeah.
Then I think we all know whathappened in 2020 and so any sort
of planning or ideas I had aboutwhat was gonna happen, all went
out the window because thepandemic just.
Sent everything into absolutechaos.
And so it was a, a very uniqueclinical trial experience to say
the least.
Cuz when you're on high doses ofimmunosuppression in a global
(31:37):
pandemic, everyone startsgetting a little bit panicky.
So
Taylor Anne Burtz (31:41):
yes, I can only imagine.
Well, okay, so you.
An amazing video for the 2021Rare Reach Festival.
That's what I saw on YouTube andI we're gonna share it in the
show notes because I honestlythink that it's, great.
Our researchers, Dr.
Wilcox and everything, he'sworking on gene therapy.
(32:01):
And so having you be able toshare your experience and, and
be on this podcast today,honestly, I feel like it's
shining a light on somethingthat's been talked about for so
long within our community, butis such a big unknown.
So when you start to kind ofanswer those questions as to
what it's like and everything,it, it, it makes your comfort
(32:24):
level a little bit better,right?
It makes you not as panicky.
But there were two things thatreally stood out to me in this
video and it.
When you talked about when youwere going in and for your
infusion and how you couldn'tnecessarily say that you were
scared, but you also couldn'tsay you were excited that it was
(32:45):
like taking a test, like a, Acollege test.
Yeah.
Like you just kind of had thispit in your stomach, like of
nerves basically.
Yeah.
Yeah.
And then you talked about howwhen you were on the
immunosuppressants, You were byyourself and everything, and how
you had every side effectpossible, like anything that
they listed that you couldpotentially have.
(33:07):
You had it right, the migraines,the, the being sick, all of
that.
And you explained how livingwith hemophilia, yes, you're
living with something on aday-to-day basis, but you never
thought of yourself as beingsick because you weren't sick on
a day-to-day basis.
And that really struck mebecause.
(33:29):
I think so many people,especially within our community,
when they, when they watch thisvideo, it's, it'll ring true to
them.
Because I don't think, Peter, Imean, I don't know.
I don't have gt, I don't think alot of our community truly view
(33:49):
themselves as being sick becauseagain, they're not, you know,
dealing.
Vomiting or anything like thaton a day-to-day basis, they
would never compare it to, youknow, what somebody who has
cancer is going through, orsomebody who, you know,
something similar where it takessuch a physical toll on your
body.
If you were to ask somebody withgt, they're like, oh, well I
(34:10):
just bleed.
Like I don't do that.
And, and Peter let me know ifI'm overstepping.
But no, that sounds about it.
When you put that in perspectivelike that, I don't know.
It just, it ran, it hit me, ithit a cord.
And I just really liked thevideo.
Luke Pembroke (34:28):
That was to sort of summarize and give a sense of
what that first year of thetrial was like.
And yeah, it did make me reflecton, you know, having grown up
hemophilia and actually, youknow, for the most part I've
always, you know, been doingokay, like bleeds are painful
and can really wipe you out.
But I wasn't living with, youknow, some of these other rare
conditions out there where itjust seems like these, you know,
(34:49):
poor people are constantlyunwell and.
And you know, that's not reallyhow like hemophilia or sun
bleeding disorders manifest, butat the same time, I guess there
are certain things that you putup with regularly that you just
sort of normalize.
You know, I think of GT and youknow, when it comes to anemia,
like there some people couldvery easily sort of have like a
(35:09):
sort of sub you.
Suboptimal sort of level likebaseline of sort of mild anemia
where they've just sort of gotused to it and it just feels
normal to them.
But like if someone elsesuddenly, you know, was in their
shoes, they'd be like, God, I, Ido not feel well at all.
But it's just what we get usedto, right?
Like it is that normalization ofthe abnormal that I think people
(35:32):
with bleeding disorder.
Tend to do because we're sortof, we're born with these
conditions and, and that's thething I say about gene therapy
now is prior to the genetherapy, people would always
ask, what's it like to live withhemophilia?
And it was the most annoying,complicated question to answer
because I was like, I don'treally know what it's like to
not live with it, so I can'treally give you a really.
(35:53):
Authentic in-depth answer of,you know, what is it like to
live with hemophilia?
How is it different to beingnormal?
Because it was just kind of mynormal, and I would always
compare myself to other normalpeople, but I, I could never
really kind of co-opt thatexperience accurately.
And then since having genetherapy and being made a mild
(36:15):
person, someone with mildhemophilia is what my phenotype
would be classed as.
There are, you know, vastdifferences where I'm like, wow,
there was a lot of stuff I hadto do and put up with when I was
living with severe hemophiliathat I do not miss, that I would
hate to go back to.
Mm-hmm.
And I think it only, only hahaving gene therapy made me
(36:35):
realize just how much of animpact my condition did still
have on me without wanting tosound like.
Ungrateful for the, the goodcare I've had and how fortunate
I am to have grown up in the UKand have not been born during
the contaminated blood era, likerelative to a lot of the
community.
I've had it pretty good, but Ithink there are still some
(36:57):
serious, you know, issues ofgrowing up with hemophilia that
have an impact on you.
And it was only since I had genetherapy that I, I actually
recognized those and gave themmore credit.
Taylor Anne Burtz (37:07):
So what does life look like for you post gene
therapy?
I mean, you've told us, and now,now you're technically a mild
hemophilia.
But like, what is that, youknow, first year, like do you
have to go in every so often?
What, and, and now that you'reat three years, what does that
look like for you?
Luke Pembroke (37:28):
So the, the, the first year.
Massively intense.
In particular, those first sortof six, six to nine months were
very regular hospital visits.
So, you know, started off at thepost dosing going in three times
a week for several months.
That then came down to twice aweek.
Then it became weekly for acouple of months, and then it
(37:49):
went to biweekly.
And, and so the gaps startgetting bigger and bigger.
Then by the, the second yearvisits went down to monthly, and
so I had monthly visitsthroughout year.
Occasionally I'd have to go inan additional time for maybe
some follow up bloods or to havesome scans done on my liver,
whatever it may be.
But my trial team were very goodat trying to get all the stuff
(38:09):
to land on the same day ifpossible.
And because it was still kind ofCovid era a lot of people
weren't able to get.
Appointments at hospital.
So for those tho for thoseyears, even though you know, it
was a really tough time, Iactually kind of had like v i p
access to the hospital, if youlike.
So everything was made asconvenient as it could be.
And then once I got past theyear two mark, so going into
(38:31):
2022 that's when I, my visitswent down to, for the trial
purpose, I only needed to go intwice a year to get my levels
check.
And, you know, have other bloodtests run.
And so that's when I really sortof had the opportunity to kind
of experience.
Life post gene therapy withoutthe trial sort of dominating
(38:53):
day-to-day life.
And so in 2022, I went away forthree months.
I did two months in Peru and amonth in Canada.
And I didn't have to, I tooksome emergency treatment, a
factor with me but I didn't haveto use it once.
It was a good job because by thetime I took out the storage at
the, the camp in Peru, in thejungle, Answer just obliterated
(39:14):
the whole kit.
Thankfully the factor vials werelike sealed, but like all the
other bits were just likecompletely demolished.
I was like, oh dear, that's notgood.
But you know, it was a niceproblem to have that I, I just
didn't have to touch my factoronce and I was, you know, super
active, long hikes, long days,and I didn't have a bleed.
Yeah.
Which is just mind bogglingbecause I wasn't treating with
(39:36):
factor and so I.
Gave me a chance to road testthe gene therapy which is what
I'd kind of been yearning to do.
And Covid had put a lot of thaton hold.
So it was it was nice toactually get out there, put it
to the test, and yeah, when Ithink about when I used to
inject intravenously three timesa week with Factor, I used to
(39:59):
plan so many things around myhemophilia and.
Schedule things around treatmentdays and you know, going on a
work trip or a holiday, havingto account to that extra bag
full of all the treatment andall of a sudden all these little
things I just don't have toworry about.
And I still, now, it happensless and less as time goes on,
(40:20):
but there are those momentswhere I'm having to rush out the
door cause I'm late forsomething or I'm gonna meet
friends at the pub and my brainkind of goes, oh, have you taken
your factor?
And then I'm like, no, wait.
I don't have to do that now.
And it does take time to adjustto and I think that's only
normal, right?
You grow up your whole life witha condition where you're
(40:40):
required to treat constantly andthen all of a sudden you don't
have to do that.
I think there is still that sortof muscle memory or that sort of
trigger built into your brainwhere something happens and your
immediate reaction is like, ah,gotta think about treatment,
gotta think about my hemophiliaand.
It's, you know, it's a niceproblem to have to adjust to
(41:00):
such a different way of life.
And, you know, fingers crossed,long may it continue, but I
understand that I did sign up toa clinical trial.
There are no guarantees.
And, you know, we hope that genetherapy will last a significant
amount of time.
But we're only gonna know.
If that's the case, if we getthe data and people sign up to
trials and I mean, it'sobviously crazy to see within
(41:22):
this past year now approval forhe hemophilia gene therapy for
hemophilia A and hemophilia B.
You know, it all seems to havejust suddenly arrived in, in one
go.
And I'm just baffled that it'sthree years ago I was sort of
embarking on the trial and I'mlike, wow, like.
You know, really do seem to bemoving at this increasingly
(41:43):
right.
Financial rate in development.
But it, but again, it all seemsto be happening in hemophilia.
Yeah.
And that gap between hemophiliaand all the other bleeding
disorders just feels like it'sgetting bigger.
Taylor Anne Burtz (41:55):
Well, I know, and this is something that we
hear all the time and it'ssomething that, you know, we've
talked about with Dr.
Wilcox and stuff.
The money is in hemophilia, it'seasier to get approved for
something for a clinical trialwith hemophilia than it is with
one of these, you know ultrarares or something, because it's
(42:18):
not necessarily, you know, that,that risk factor, right?
Like, okay, if we're gonna throwmillions of dollars into this
and it doesn't work, like ookay, but.
What do, what did we learn fromthis really?
So the fact that, you know, youdid your trial or you're in the
middle of your trial, but youstarted your trial three years
(42:38):
ago, and now just within thepast three years, there's so
many things that have beenapproved.
I mean, you can, you come from avery similar world as we do to
see that many things getapproved in such a short amount
of time is.
The speed of light in thisworld.
Mm-hmm.
(42:58):
Honestly, because we, I mean wehave, we have a running a joke.
Oh, you know, in just 10 yearswe could have a cure in just 10
years.
We could have a cure.
Like it's, I looked at one of mymom's old interviews and she was
like, I'm hoping that by thetime Julia's 2021, that we will
have cured this thing.
(43:19):
And you know, Julia's now 24and.
And it really is just kind ofthe approvals, the getting
everything set and, and now thatthe legwork has been done,
hopefully that means everythingelse will speed up.
And so, yes, it may seem weirdthat we're talking to you, a
hemophilia who went through genetherapy, but honestly, in order
(43:43):
for gene therapy for Glands,mens to happen.
There has to be success in genetherapy for hemophilia.
Yeah.
And so that's, that's why it'sso important to have these
conversations and also to get toknow the rest of the bleeding
disorder world as well, becauseit may not be the exact same
thing, but your experiences arevery similar to experiences
(44:06):
within our own community.
And I'm, I'm all for bringingnew ideas, brainstorming and
meeting new people just to.
Increase awareness for us andget, and get the word out I
guess.
Yeah.
So with all of that said aboutgene therapy and how quickly
(44:26):
it's moved, just within the pastcouple of years, what do you
think the future of bleedingdisorders looks like?
Luke Pembroke (44:33):
I think it's gonna be a lot more options.
I think treatment options will,will increase massively.
I think the main challenge ismaking sure that there are
options for people.
Who don't just have hemophiliaor, you know, standard
hemophilia A without inhibitors.
I think, you know, we've seen alot of development come along
recently, and now people arealmost somewhat spoiled for
(44:55):
choice.
I mean, we, I won't get into itnow in terms of what patient
choice really is.
And it varies between countriesand rules and insurers and
payers in terms of what youactually get.
But I think the community is ina good place to have more
treatment options movingforward.
I, you know, from my sciencebackgrounds and also personally,
(45:15):
I, I, you know, I'm reallyhopeful about the idea of
genetic medicines and genetherapies and, you know, people
would consider the gene therapyI had as sort of first
generation gene therapy.
And the technology's only gonnaimprove and get better as as
time goes on.
So I am hopeful that geneticmedicines will offer.
Treatment options to peoplewhere, you know, maybe it won't
(45:39):
technically be considered acure, but you'll pretty much be
able to live a life free of yoursymptoms for the most part.
And you'll just have to havesort of treatment plans in place
for those unexpected things in,in your life, whether it's
surgery or you know, badaccident, for example.
And, and I really do hope thatgenetic medicines, gene
(46:01):
therapies, Made available topeople with other rare bleeding
disorders and just other rarediseases in general.
That's something that I sort ofhighlighted in the video that
ultimately I think gene therapycan have a much more meaningful
difference and impact on peopleoutside of hemophilia and I, and
I really do hope to see that.
(46:23):
But as you say, hemophilia,multi-billion pound industry and
you know, you've got investorsleft and right, all these
companies and, and it is wherethe money is and, and that's why
the research tends to get donethere first.
But hemophilia offers itself asa really useful Petri dish if
you like, to kind of developthese medicines and hopefully
then expand them out.
(46:45):
Other conditions that I would,you know, hazard to say are
perhaps more in need and moredeserving than hemophilia.
Not to say that people withhemophilia don't deserve the
best treatments, but you know,if you've got access to all
these other great treatmentsoutside of gene therapy that
exist and you're, you know,fortunate enough to be in a
country that has access to them,you can live a pretty much near
(47:05):
to normal life now.
And you know, I come acrossparents and younger people with
bleeding disorder.
Youth camps and you know, justseeing people run around and do
things that I was tolddefinitely not to do when I was
younger.
And it just makes me realizethat, wow, you know, even, even
in sort of 20 years, so much haschanged and I think.
(47:28):
Things can only continue toimprove as, as we move forwards.
Provided we all do our part aspatient advocates and we make
sure our voice is continuouslyheard throughout the development
process.
You know, we have our say inthose, those clinical programs
and, and that that voice isn'tlost.
And we gotta avoid that slipperyslope of kind of becoming
victims of our own success.
(47:50):
You know, if treatments get sogood that it kind of normalizes
people, there are other thingsthat can kind of slip off the
agenda that we need to make surewe, you know, we catch those and
that, you know, we have goodtreatments.
But that, you know, it canalways be better.
You know, I think we, you know,we, we always strive for
excellence as much as wepossibly can, and I think that's
why things have.
(48:11):
Gotten so good for people in thecommunity over tho those last
couple of decades
Taylor Anne Burtz (48:17):
we're, we're huge advocates for that.
Like, yes, you may have onemedication or whatever, but like
that works for the majority ofpeople within the community, but
we're the options.
What happens if it stopsworking?
You know, so we're, we're hugeadvocates of that.
Like, we want options.
Give us all of the options.
Luke Pembroke (48:36):
Yeah, exactly.
Taylor Anne Burtz (48:38):
Well, You're joining us for our conference in
July, right?
I know Kate is, but are youcoming as well?
Yes.
Luke Pembroke (48:44):
Yes.
Perfect.
I'll be there.
I will be there.
So if anyone listening in tothis podcast is also going to be
attending, Then yeah, just lookfor me, and I know I've had gene
therapy, but I don't have anylike extra arms or, you know,
third eye that's developed.
So it might not be obvious thatI'm a genetically modified
individual now.
Taylor Anne Burtz (49:04):
Oh, this is where like my dark humor comes
in and be like, let's get a nametag calling you specimen number,
whatever.
Luke Pembroke (49:10):
Yeah, exactly.
I mean, I like the worst memberof the X-Men possible.
Right?
Like it's your superpower.
Oh I bleed a lot, so,
Taylor Anne Burtz (49:20):
Well, last but not least, I asked this
pretty much of everybody that wetalked to.
What would you say to youryounger self, to the boy who was
fighting the debilitating painof a bleeding disorder every
day?
What would you say now to him
Luke Pembroke (49:35):
to stick with it and you know, don't let it
define you like your hemophiliadoesn't have to be.
It's the dominating thing inyour life.
There are so many things to lookforward to.
When I was younger, I didn'tthink I'd have so many
connections in the community andthat, you know, you are gonna
(49:55):
find people and connect withpeople that become lifelong
friends.
That, you know, the reasonyou're friends with them isn't,
you know, down to you havinghemophilia.
But it, it started off as that,that initial connection and you
know that it's an amazingcommunity that.
Actually lucky to be a part of.
And that whilst there's a, youknow, a little kid who was being
(50:15):
told he couldn't do things washeartbreaking at the time that
ultimately it's kind of wateroff the duck's back as we say
now as I'm moving into being anadult.
And I'd also say to him,Football isn't the be all and
end all.
If I had a chance to go back tomy younger self and, and tell
him to get involved in moreunique like niche sports, that's
(50:36):
what I would do.
Because now I'm like too old anduncoordinated to figure out all
these new hobbies.
And I'm always like, ah, I wishI'd have done this sport when I
was younger instead of justhounding after football, you
know, desperate to be agoalkeeper, a, a mighty five
foot, 10 thinking, you know,it's not the hemophilia that
held me back.
It was my lack of talent andheight.
So, you know, can't blame it onthe hemophilia younger Luke, you
(50:58):
know, get over it and focus onsome other sports, and you might
have actually made it as a, youknow, a sportsman somewhere
else.
Taylor Anne Burtz (51:05):
Well, if you are interested in Hume's Glands
Man's 360 survey and would liketo participate, we are going to
link it in the show notes.
Luke, I am going to do a coupleof news items and then we will
say goodbye to you.
But first, I do wanna thank youfor doing this.
Like I've mentioned multipletimes throughout this, you stuck
(51:25):
out to Peter and I, you're veryeasy to talk to.
You're open.
And it was something that wewanted to share with our
community, so we're, we're verylucky to have met you.
So for our news items,registration is open for the
2023 G R F EducationalConference in Boston for this
upcoming July.
(51:46):
Applications for lodging grantsare open until May 15th, and you
can find more information.
We've been updating it on thewebsite www.curegt.org.
Also, if you or a family memberhas gt, definitely check out our
support group on Facebook.
(52:06):
It is a space for people with GTand caregivers or family
members.
It's, it's a pretty.
That's pretty much all who's inthat group.
We try to keep it as private aspossible, so that is a safe
space for everybody.
Luke, again, thank you.
Luke Pembroke (52:27):
No problem.
I really enjoyed it.
And yeah, hopefully I explainedthe sort of gene therapy stuff
in an accessible way, cuzsometimes I, I look back over
all those years and I go, youknow, actually what did happen?
So yeah, if anyone is curious,like you said, you, you'll put
links to the video in the, inthe show notes.
So, questions, comments thatpeople might have, like if they
leave them on YouTube orInstagram or whatever it may be.
(52:49):
I always do my best to get backto people and answer as well.
So, you know, don't be afraidto, to reach out if you do have
any other questions.
Taylor Anne Burtz (52:57):
And as always, please like and
subscribe to this podcast.
Leave a review if you feel soinclined.
And remember that you may be onein a million, but you are not
alone.
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