I think a barrier might be, so as I said when I was younger we didn’t do anything where I had to have another blood test. So if there’s a particular procedure involved that you don’t really want to do then you’re very free to say no. But even if it’s something that only, you know, that only mildly inconveniences you, like I had to have these bronchoscopies, you know, it’s not exactly a fun thing to have but you think well it’s worth it to put myself through that for the benefit that the trial gains from it. Or I mean there was another, with the pilot study they had this worst test ever, they had like a wire pipe cleaner brush that they’d stick in your nose and go whoosh and take it out. And it’s really, really uncomfortable and oh it was horrible but in doing so they got a million epithelial cells from each nostril doing it so, and the doctor doing it said I know these are horrible we’ve done them on, we’ve done them on ourselves as wel but the amount of data we get from it is incredible. So I think perhaps sometimes maybe for young people to try and see past the, you know, slight uncomfort they may have to go through for the greater gain that it brings yes. And I think as, as a facilitator as I said being a young person on the gene therapy trial if I’ve got a chance of being one of the first people to have gene therapy and then to be on that drug then that’s a, a really great opportunity for my health. So even though that’s a yes it’s not a financial incentive it’s still a really good incentive to take part.

I was diagnosed with acute lymphoblastic leukaemia on the 21
August 2007, yes. And I mean as soon as I was diagnosed my parents were given
these papers to sign saying that they are willing to consider taking part in a
trial. I didn’t know anything about it at the time, so that’s basically when my
parents found out about it. And I found out about it when I was just about to
start my intensification blocks, so there’s two intensification blocks because.

What are those
what’s intensification?

it’s where, so basically the treatment is set out that you have,
you have sort of chemo every week for quite a while and then you have a bit of
a break and then you go back on and then I think about a quarter of a way into
your treatment you have two, I think it’s seven or nine neat blocks where it’s
a lot of chemo like you’ll be on weekly chemo, daily chemo then you’ll be on
six hour infusion chemo. So it’s a lot of chemo so that’s the main thing that
kills everything and basically the rest of the treatment after that is just to
prevent it coming back. So the study they were actually doing was because in
Europe they don’t actually do two blocks they do one, so they were running a
study where you were randomised, if you said yes, you were randomised to either
have one block or two.

Okay.

I, well yes, I decided against going in the trial. My dad thought
I should go in the trial but the way I saw it and the way my mum saw it is that
if I have two blocks it’s less chance of it coming back, it’s basically having
an extra nine weeks intense chemo or doing it for years later on, basically. I
didn’t get that much information on the trial, I went, because I was quite
mature as it was but once you get, you get an illness you sort of mature
straight away. So I pretty much read my parents information with a bit of help
but I didn’t really, as far as I remember I didn’t get any information.

Oh didn’t you?

I don’t really remember.

So it was your parents?

My parents got the information as soon as I was diagnosed and they
were told to read it which I think was a bit unfair. But then when, I mean it
was more my consultant explained it to me and my parents explained it to me and
what was at risk and what they were trying to do and then from that my parents
and I just sat down and discussed it and we made a decision, basically.

My consultant discussed it with me, so he gave me like quite a
while before my block and then like every time, we’d sort of discuss it every
time I went to see him and then I think we just sort of came to the conclusion.
And I think it was really good my parents, because my parents let me decide,
they didn’t see it as it was their decision not mine. They realised that I was
mature enough and it was my life I was playing with and that I should be able
to make that decision.

Do you think
that’s important as well?

Yes I mean parents as well as doctors have to understand that when
a child gets a serious illness they’re not children anymore. they’re almost
adults in the way they think and the way they do things. And they have to
understand that it’s their lives that they’re playing with not their parents,
not theirs but the child’s life and they have to be able to make the final call
no matter what age. It should be their decision, if they’re old enough to
understand then why should they not be allowed to make the decision. I agree
with things like consent, a parent to consent but I think that children should
also have to give consent as well.

And just going
back to the trial because I think you, you know you made a big decision it must
have been really hard at the time for you, did it take you a long time to make
that?

Yes it did, it definitely did it was one of those things were you
wouldn’t sleep at night thinking about it. It was one of those things that just
kept ticking over in my head for about three weeks.

And how did you
cope with that, with school and things like that?

I was pretty much ill and I wasn’t really in school so, I was
lying on the sofa for most of the time.

Was you?

So that was sort of like a thing then, I wouldn’t say I was, it
was in the back of my mind all the time but I did give it a lot of thought.
Because it is a big decision and I had to make.

Just explain to
me, you know, your, again why you decided you didn’t want to take part?

I didn’t want to take part because I saw it as two blocks now of
intense chemo now or what happens if fifteen years down the line I get it again
and then I will regret not having that second block because obviously it was
the wrong decision. So it’s like you do it now, you do it once and it won’t
come back, most likely, it won’t come back or you do a bit now and you might
end up, there’s more chance of having to have it later on.

So is that what the two blocks mean?

No it’s not
exactly what it means but that’s, they’re saying that because in the,
they didn’t understand whether like if you have one block there’s more risk of
it coming back, that was why they were doing the trial. I think that’s just how
I interpreted it and I think, my mum did influence quite, influence quite a bit
my decision. But I sort of understood what she was saying more than the logic
behind my dad saying go into the trial.

So your dad
wanted you to?

My dad wanted me to go in the trial and my mum wanted me to have
two blocks but they let me make the final decision and they basically said
we’re happy either way but you have to understand that this is a life changing
decision that is basically kind of decide what happens in the future. Either
way we will support you.

How did you make
that decision?

I just sat with my parents and we talked about it one Saturday, I
think for a like a few weeks the topic just kept coming up like briefly and
then like one day I said right I know what I want to do.

What was the, you
know the real reason for not wanting?

The real reason for not going into the trial was it was loads of
chemo now or more when I’m older, to put it in a sentence. it’s do it properly
now or take the risk of getting it years along the line when I was full steam
ahead with my life, I’m somewhere I want to be and then there’s this bomb shell
being thrown once again.

And was it good
to be able to talk to your parents?

I think, it means it’s like yes they had a big influence on my
decision and they have the right to decide for me but they didn’t which I think
was really good.

You obviously
appreciated that?

Yes I definitely appreciated that.

Even though if
you’re still young relatively?

Yes I was ten, wait no, was I even ten, no I might have been nine,
yes no, yes I was nearly ten, I was still nine. it’s a big decision.

And how did you
sort of overcome the differences of opinion with your parents, how did you
overcome that?

They basically said here’s my point of view and here’s my point of
view and we’ll let you make the decision. It was like we sort of debated it out
but they did just say you make the decision we don’t mind either way.

And who did, when
you told them how did they feel?

I think they were okay about it I think they understood the
reasons, and they just respected that I was mature enough to make that decision
myself.

And once you’d
made that decision and you realised then that you wasn’t
going into the trial that you were just going to have the statutory thing that
was going to be the best that you were going to get anyway, how did that feel afterwards?

I mean it was a reassurance to know that I was having it all and
having it once. It was almost a security blanket knowing that yes I’m having
loads but it’s going to happen in the future. But sods law now it’s only one
block and I had so many complications in block two that I wouldn’t say I regret
it, I don’t regret it not one bit but I think now I look back I might have been
better going into the trial. But what’s done is done and as long as you don’t
regret it it’s fine.

I wouldn’t
say there is any regret I would say that if I did it again I would do it
differently but I don’t regret doing the second block. I mean now that I know
that they only do one block I would have chosen differently if I had known that
information but I don’t regret it no. Because I did what I thought was best in
the situation I was in.

Ryan’ But when I was getting it they kept having to take blood tests to see
if I was able to get on the drug.

So you had to sort of
meet, they call it, it’s like meeting the criteria, don’t they? So what sort of
things did you have to do to, before you could go on the trial?

Ryan’ I had like quite a few blood tests and I had to, they said like if only
like one or two joints were swollen, I won’t be able to get on it. But I had
like my knees and my feet and my hips and my elbows and that were sore.

Mum’ He had eleven joints that were swollen, and you had to have more than
four.

So you had way more
then, didn’t you really?

Ryan’ Yes.

Mum’ And you had, he had to have had all of his MMR injections and all of his
vaccinations up to date to get on it.

So there were quite a
few things beforehand, weren’t there?

Ryan’ Yes.

And were you pleased
that you could take part, when they said, Yes, you ca?

Ryan’ Yes, because I thought, I think, I was thinking, A drip’s better than
a needle every week Because the needle just made me sick and sometimes I just
didn’t want it.

Mum’ He was also getting very angry at me because I’d learnt to do them so
we could do it at home instead of travelling. And he was getting angry at me
because I was the one having to give him the needle. So he wasn’t coming home
to get it.

Mum’ Okay yes you had the bone scan and the die in your blood and everything.

Oh was that to see if you were eligible and?

Mum’ For the trial she had to have can you remember all that?

Lois’ Yes.

Mum’ And you had to have the die to see how, whether it was definitely that condition and the bone scan.

Oh was that eligibility or?

Mum’ Yes it was just very simply she had the, when, on the Monday we went to see the doctor didn’t we and he did all these tests on her and things like that but just to clarify it definitely was that condition. We went down to, he came in the room didn’t he and she had die put in her blood to see how much iodine I think it was, to see how much it evaporated and they couldn’t believe how quickly her thyroid soaked it up.

Oh right you need to tell me about that?

Mum’ And then you had the, the bone scan didn’t you, to see whether your bones had grown and you’ve got to have that done every twelve months for the trial, do you remember?

Lois’ Yes.

Mum’ Is it all coming back now?

Lois’ Yes, it’s like…

Yes the whole process like you know thinking the whole story. So before you went into, when you agreed to take part they had to do some tests to see if you were eligible as well?

Lois’ I had a bone scan where just they x-rayed my hand to check it was okay and like I was growing properly and stuff. Then I had ink put in my hand and a thing called a gamma scan and like when you have the die in, it like shows up on like the x-ray your thyroid and they couldn’t believe how quickly it came up and he was like quite amazed.

Really, so that was after they put the die in your hand?

Lois’ Yes and you had to wait like half an hour afterwards and then you went into this thing and you had to lie down on a bed and then this like, big like circle thing came with over like your neck.

Yes, was that like a big x-ray machine was it? A scanner?

Lois’ It was just like a little stem it wasn’t like going into the big like ones it was just like.

The MRI?

Lois’ Yes like the MRI, it was just like a little one that just came over.

Oh right, what was it like doing that?

Lois’ It was quite fun actually.

Was it?

Lois’ Yes, it was funny having something like there and stuff. Yes and there was like tiny babies in there having it done. Yes and like one baby got rushed in, it was like we got like put behind a bit in the doctors and the nurse said like, but I didn’t mind because it was like a little tiny baby.

Oh so you didn’t mind waiting a bit longer for your turn?

Lois’ No because it was really tiny.

And did you have any other tests done, before you started the trial?

Lois’ No I don’t think so; I just had the bone and the gamma scan.

So that was just an x-ray on your hands?

So the time I actually
went to see the doctor she said I had all of them. So I got given this book to
fill out my temperatures as I had the salmon-coloured rash and the, the sore
joints. And I, they felt, they said they think I’ve had the temperatures. If I
had the temperatures, I’m eligible to go on to the trial. This took over a week
and I had a temperature with them every day of this week. So as soon as I gave
back the book they said, We can throw you on the drug trial straight away So
that was a, kind of a couple of hours’ decision, to decide if I actually wanted
to go on to this trial.

So you kind of, there
was like an eligibility criteria that you had to meet?

Yes.

And that was the rash
–?

The rash, a number of
joints would have to be inflamed, and the temperatures. Well, the rash was
quite obvious to see. The joints were quite easily able to be touched and
shown. But it was the temperatures what I had to do myself, and that was very
quickly done.

How many joints was
it?

I think it was about
four or five. I can’t remember exactly.

So you had to have
five or more [yes] joints affected?

And the temperatures
over 38 degrees.

And you met all of
that?

I met all of that. I
met the joints straight away. I met the rash straight away. It was just the
temperatures.

And so you just had to
do that over a week?

Yes, it was, they said
it can take up to a month to get this done, but I did it within a week. So that
was quite quickly.

So then what happened when you went to the hospital did your mum ring the hospital to see if you could take part in the trial?

Yes then we went up for the test but then I was on a medicine and I had to be on a stable thing, I was only on the medicine for two weeks, so I had to be on a stable medicines for like a month, so we had to wait for a month and go back again. Then we had another burst, trial thing and then I got on because my liver function was back where it needed to be for the trial.

Okay, so you had to meet a certain criteria?

Yes. Because you had to, because your LCI; lung clearance index had to be in this range and lung function had to be in another range so.

And what was the criteria, do you know?

I think it was between 90 and 100% for lung function, the LCI, can’t remember what it was; I remember only just getting the lung function by 1%, because I got 91.

Wow, yes right, so you met all that and did that, did the tests take, did you have them all done on the same day or did you have to keep coming back?

Yes I had the same tests each time but I didn’t have the same amount on my first visit, so just had lung function and LCI to see if I matched the criteria, that’s all I had.

Okay and how long did that sort of process take before you could take part in the trial?

It took about an hour and a half, two hours because of all the talk and explaining.

Yes. And then, so on that day sort of did you agree on the same day, yes and you were happy and your mum?

Yes, it was worth it though going up there and it wasn’t, it isn’t a wasted day either because you only spend an hour and a half, two hours at the hospital you can just go, because like seeing the place where it is and everything afterwards, so that’s nice.

Did you have to
stop the trial at all?

I had to stop the trial for a bit because the IV’s interfere with
how well I am.

Okay, what’s the
IV, do you want to explain?

it’s intravenous which is basically where they put a tube into
your vein and the medicine goes straight into your veins instead of having to
digest it all. So it goes straight into your system and its stuff that can’t be
taken orally.

So did you have
to stop, you had to stop all the other, the trial?

The trial medicine, yes. Because I was on wash out month and I
just went downhill a bit, I don’t know, I think it’s because of the summer all
the tree spores and hay fever and everything.

Oh yes, you think
that, do you normally get that?

Yes it’s usually in the summer.

Is it?

And worse.

So that, did that
bother you at all?

A bit but I can go back on it.

What did they say
at the hospital when that happened?

Which
hospital, the trial?

The trial hospital yes.

They just said that you can go on IV’s and you have to be clear of
everything for six weeks and then you can come back. But now I’ve got to have a
camera in my lungs, I’ve forgotten what it’s called.

Bronchoscopy is
it?

Yes I’ve got to have that. it’s either at the end of this week or
the beginning of next week. So that’s and then I have to go on IVs straight
after so that’s been put back again and IV’s every six months.

Yes, but have
they said that you can stay in the trial?

We’re just going to see how it goes.

Right okay, so at
the moment you’ve had to come off the trial?

For a bit.

For a bit yes,
how do you feel about that?

I feel alright, Ive just got used to being off the trial for a
bit now. So we’re just going to see how it goes and see if there’s any point
going back on it. I probably will but we don’t know yet.

And what have
they said at the trial hospital, did they say?

They said that if there’s a clinic, a clinical problem just stop
the trial, get better and in six weeks being clear of everything.

And then you can
re start?

Yes do the same thing.

Does that kind
of, does that feel good that you can?

Yes it feels nice like I can restart it at some point.

And will you do
you think, do you think you will if things are okay?

Yes if I can I will, definitely.

I was having quite a bad period of health I think. My lung function had gone down quite a lot and I think, although my lung function had gone down it was still in the relevant kind of ranges to be on a clinical trial, that this particular clinical trial of Mannitol. So I basically wanted to do anything I could to try and get my lung and help and I was invited by, we’ve got a research nurse at my hospital where they we have CF care, and she leads all the clinical trials and the consultant there he liaises with the research nurse to get all the ethics approved etcetera. And I think they just approach patients that they feel would be happy to be involved with the clinical trial or fit the medical criteria. Because obviously there might be loads of people that want to take part in the trial but if they don’t fit the medical criteria they can’t get involved. And I think because I’m quite a compliant patient as well I think they knew that they I could they could rely on me to kind of take the medication every day and record if I had, you know, any side effects [noise] or anything like that which, I think they could rely on me which is quite a big thing if you want to get good data so.

Well luckily in this case I think I was doing the one that I would rather have done. But I guess there could be situations where you are doing a trial and then another one comes up that you’re really interested in and you want to do that one but you can’t and then you think, Oh shall I pull out of the one I’m doing just so I can do this other on and then. But yes the only time I can remember being told I was ineligible was because I was on another trial and that, in fact there was a very weird situation because the trial I was on wasn’t giving any drugs, this was the run-in study they were just taking observations and measurements. And this, and then the other trial I was approached for was just collecting breath samples. So neither trial would have been administering any kind of drug. They both would have been just taking observations. But it was still, you weren’t allowed to do both, which I didn’t quite understand, but I did what I was told.