Alison - Interview 25

Age at interview: 49

Brief Outline: Alison became involved with clinical trials when her son was six years old, he is now 22. Alison talks about consenting on behalf of her son to supporting her son in making his own decisions and the reasons for taking part in clinical trials.

Background: Alison, aged 49 years, put her career on hold to care for her son who was diagnosed with Cystic Fibrosis soon after birth; he is now 22. Alison now works part time in a School and lives with her husband and second son who is a carrier of CF.

More about me...

 Alison, put her career on hold to devote her time to care for her son who was diagnosed with Cystic Fibrosis (CF) soon after birth; he is now 22. Alison now works part time as a School Administrator and lives with her husband and second son who is a carrier of CF. Alison became involved with clinical trials when her son was six years old. 

 

When Robert was aged seven Alison transferred from a local hospital to a specialist hospital. It was at this time that she began to be invited to enroll Robert into various clinical trials. Because it was some years ago she cannot remember all the details. However, she does remember it was usually some extra test or investigation while they were waiting for their appointment at a routine clinic visit. They always said yes with exception of any that involved blood tests, as at the time her son had a phobia of needles. The trials were usually short one-day trials so didn’t require any extra travelling or appointments.

 

The reason they took part was to help research and enhance medical knowledge and treatment of Cystic Fibrosis. 

 

Now that Robert consents to take part in trials, Alison supports him from the “sidelines”. Robert continues to take part in research and Alison talks about his involvement in a Phase 1 gene therapy trial, and her feelings from watching and supporting him from the “sidelines”; it has been worrying at times for Alison.

 

Clinical trials have helped to improve the treatment for cystic fibrosis, but this often takes a...

 We had the approach of the only way to make progress it requires you to, for them to have, you know, live data to work on and you’ve got to have people take part in these things, if you don’t! And we’d also seen even up to that point because he was probably seven or eight by the time of his first, you know what he did. We’d already known by then that significant advances had been made in the treatment of CF [cystic fibrosis] and those don’t happen just totally remotely, you know, in the lab. When he was born they didn’t, they hadn’t discovered the gene, the actual gene defect, you know, they knew it was a genetic defect but not the actual location of it on the, you know, the DNA chain. And that happened very, very soon after he was born actually, I think. And then at the time they were saying “Oh in five years time we’ll have a cure”. Well here we are 22 years later. And you know you understand, from a scientific and medical point of view, that knowledge in one part in theory in a lab is a total, totally different prospect from actually implementing it and knowing how to deliver it. But we, nevertheless, we’d seen the improvements in drugs we’d seen various different approaches to treatment. I mean the most notable one that happened up to that point was the improvement in the digestive enzymes that they use because they can’t digest food so. When he was first born they were on a low fat diet although they need lots of calories because they couldn’t cope with digesting the fat. Well all of a sudden this miracle new enzyme, you know, drug came on and it totally transformed the eating lives for CF, you know sufferers. And so, and we’d seen all kinds of, you know, progress, you know, in that way.

 

Oh yes, yes but we also understand too that you don’t always know or see any of the benefits that might be, you know, derived. As I say the gene was found in ’87 and then, you know, you read in the paper and people come over “Oh I see they’ve found a cure for it so you’re alright now”. Yes actually they’ve found a cure but they know putting it into practice is something else. Even when they put it into practice those, it might not help those who are already of a certain age or living with it because of it’s too late basically. Because if they’ve already got lung damage then you can’t reverse it so and that’s I think Robert sort of touched on that. That a lot of the treatments, well they won’t necessarily help him the most, new babies born, it will give them the best, yes in the same way how he’s benefitted from changes in drugs. They were probably trialled on older people who probably are no longer with us but they did that and that’s helped the present, you know, generation so.

 

When her son was younger, Alison took responsibility to enrol him in trials, except when blood...

 And then, as I say we were at the [Hospital] and they were at the point of sort of saying “Well we’ve got this trial on the go, we need some, you know, would you mind a small test since your here”. And I would think yes that’s fine by me. Yes Robert, hearing what he said they would, we would give him minimal information because at that point, certainly you know, as an eight year old decisions about him were down to me really. And yes it was explained to him beforehand well this is just going to happen, you don’t mind do you and in the main he was fine. The only times that we refused it or listened to it and then said no were when it was going to involve blood tests because of his needle phobia. But otherwise pretty much in the main, he just saw it as oh that’s another thing to do, I’ll do it, we’ll do it, we saw it as a yes we know what the ultimate goal is. We know that along the way they have got to look into and collect data, try different things out and they need, you know, people to take part to do that. And it wasn’t intrusive on our lives particularly because usually we’re in clinic, it just happened as an add on maybe once or twice it delayed us leaving the hospital by maybe half an hour or an hour or something. But at that stage when he was a young child or as a child, it didn’t involve us having to make special trips, you know, up there to take part in the trials.

 

As he got older I probably consulted with him marginally more but not a lot more because in the main he was very comfortable with relating to different people. That’s something that had been very apparent right from early on and I think that’s because of all the hospital treatment that he underwent right from day one. He’s always related very well to, you know, adults and he’s not been fazed by, as long as there’s not a needle attached to it [laughter]. Yes, you know, he’s been, you know, very compliant, you know and very good. So but as he got older we would probably look at each other, he would be there as they were explaining it to me and then I would sort of say to him well if you’ve got any questions, are you happy to do this because I’m happy for you to do it. And it would happen like that. 

 

Having a good relationship with her son has helped Alison in supporting him make an informed...

 When would you stop, would there come a time when you would stop him taking part?

 

No there wouldn’t because I wouldn’t be able to stop him taking part. I could have conversations with him but we’ve built up enough of a relationship with each other and enough knowledge about each other that I wouldn’t say you cannot, you know, I forbid you to do this. Because he, he could just turn around and say well, you know, quite rightly you don’t have the right to and I wouldn’t. I would have to approach it by taking the tack of talking him through on an informed basis, are you sure because of that and then if he wanted to do it then I would have to say, you know, of course I’ll always be there for you to do it. I suppose I’d probably say I have my doubts about it but if you’re sure to do it then, you know, I’ll support you.

 

Have an open communication?

 

Yes I mean I don’t, I don’t see and I wouldn’t want to have that kind of relationship where I say, you know, you’re not doing it, yes I don’t see how I could possibly do that anyway so yes.

 

Alison wanted her son to let the hospital know about a possible side effect of treatment; taking...

 Moving on to the Phase 1 trial which was, and this was the one that had all the information on it about, you know, some, some of the possible risks of it. And that’s what I did, you know, I have to say I do not sneak into his bedroom when he’s away or whatever, or see what’s lying around. [laughter] ”Oh just clearing up your room, oh I found this”, [laughter] so. And that, I remember having the conversation with him saying are you sure have you read it properly and, you know. And that and that one, that could be, because actually that had quite an involvement for me in the end as well which wasn’t clear at the start but, you know, he spoke about having these bronchoscopies and things. Well of course what he didn’t realise, and it was, which is good in a way for him because with all the hospital treatment he’s had over the years we’ve always just sort of made it as a normal part of life. You just get it over and done with, you do it, it doesn’t really interfere with your life or rather your life goes on around it. So he missed, even though he had hospital, hospitalisations and things, he didn’t miss that much school because to him it was normal to be ill. That was normal for him so we would just carry on and carry on and do things unless he was really, really unwell and really couldn’t get out of bed, life went on, doing it. So he had to go for these bronchoscopies and other things and he’s “Oh I’ve got to have a general anaesthetic”, you know, “But it’s only a day, so I’ve arranged for it this day”. And I would say “Well okay I’ll just check the calendar because I’ll just see if I’m around” you know. “Well it’s alright because, you know, I’ll just go up there and I’ll catch the train home and come back” and [laughter] and “Actually Robert if you’ve had a general anaesthetic you won’t be allowed to just come home, I will have to be here to make sure I can look after you”. And just things like that. Yes so we’ve got to work out how to get you back from the hospital, and “oh”. And yes so I had to be sure enough and they said “Yes, yes you’ve got to be looked after for 24 hours” you know afterwards and things. Yes I know I do work [laughter]. And so its, I mean it’s quite refreshing in a way to have that attitude towards it. But, so I had to, yes I had to be on hand, you know, for that and of course when his voice went and I’d say “Perhaps you should tell them at the hospital”. But well no he came out of hospital two days later, went off to Belgium, and we didn’t hear from him for a week. And then on the day he arrived back, I knew he was due home that night, we had a text, no we had a phone call from a friend of his and said “Oh Robert will be arriving on such a train he can’t talk to you because he hasn’t had any voice all week”. And I’m thinking what on earth is going on, and then you find out, you know, that oh maybe 10 days after he hadn’t had a voice, not sure that this is right I think you need to ring the hospital. So it from that point of view I’ve had to be on the sidelines but still have an active role just the same.

Having a good relationship with her son has helped in supporting him through clinical trials.

 And in fact at the hospital they did start, I know certainly by 16 he, we signed some forms that he could then consent to things or had to and it wasn’t just sort of me. So we did start that process very early on. And he was he wasn’t fazed by having to move from the paediatric department to the adult department. And although I always sort of said, you know, of course I will come with you if you want me to; you’ve got just this [laughter]. And but in the main he sort of dealt with things on his own except for, there was a few occasions when I maybe stepped in and said you really need to go to the hospital, you really need to ring them up, [laughter] you need to do this.

 

Yes but in that he has assumed, he assumed responsibility, you know, quite early on. And then I realised that I had to back away from making the decisions, you know, for him, he would then have to make the decision for, you know, for himself. But what I would try and do is always strike up a conversation to say “Oh I see that, you know, you’ve been asked to do this” or “Something’s arrived in the post, oh what was that”, you know, “Can I see it or, would you like to talk about it”. And then, I’ve made sure that I’ve read up on things he’s been asked to do and gone back to him and said “You have read this properly haven’t you” and “Had you taken that into consideration”. And I always remember he turned round, and he said to me “Yes I’ve read it all I know what the risks are, I’ve read it and I’m willing to accept them”. 

 

As a parent you know, I’ve had, I’ve had to say “Okay fine good, then I support you in what you’re doing”, because he was very clear about “I’ve made my decision, still want to do it”, you know. One of the possible side effects in one of the papers was possible risk of cancer being induced by it or something. It was something like that and he was no “I’ve read it, considered it and I’m willing to accept it”. And we chose, we have no choice but to say well, you know, he’s an adult and it’s up to him.

 

But how do you feel?

 

You feel I think it’s, it’s one of the lessons I learnt and this was the hardest lesson to learn being a parent of a CF (Cystic Fibrosis) child. And it probably applies generally, you know, anyway, is that having being so involved in his life, and helping him, you know direct his life to get the best out of things. The hardest thing to learn is actually it’s their life. It’s not my life, you know, it’s their life to live you know, as they want to. And they are responsible at the end of the day for the decisions, you know, regarding their life. And if I’m not happy with it I can, I can’t change that for him. All I can do is make sure from my point of view that I’ve helped him to make a considered decision if that’s how it’s to be so. Sometimes you feel, when you see it in black and white there’s this, this and this risk and then and actually I’ll talk a bit more about this study and the problems he did have after it. 

 

When your child consents to a trial it is up to him or her, to report symptoms or possible side...

 Yes, yes it is and then he said I didn’t contact you from Belgium because I knew you’d just worry. So he, and then, but then you think well actually afterwards, you know, a month later when he was in, he later he went back to Uni and he’d ring me and I’d say “Your voice is still not better is it?” “Oh no it’s fine” “No I can tell it’s not better” [laughter]. I would have thought, he should have been thinking is this going to be long term damage, is that something, you know, extra, you know, that’s happened. So yes that.

 

Was that on the information sheet as well?

 

No. No not really I don’t recall that in there, I mean other than that I know that if you have a general anaesthetic, any general anaesthetic let alone one that’s had the camera down for your lungs anyway you’ve, aside from that you will have had a tube down your throat in any case because everyone does. And it’s normal after an anaesthetic to have a sore throat or something. But no that wasn’t expected and I don’t think that the hospital were that helpful about it afterwards. Sometimes you got the feeling, you know with that one that their ultimate goal was to get the mechanics of the trial done. [Yes.] And sometimes if there were any side effects they weren’t quite so interested to do those. [Really?] Yes, that’s my sort of view of it. His sounds slightly different but [laughter].

 

But did you question any of it with the hospital?

 

No because you see you don’t have the right to. Once they’re an adult, it’s his choice.

 

But when you can research things on the internet anyway and you see well one of the triggers for this vasculitis he had is if you’ve had some kind of respiratory disturbance and you’re thinking well he’s had various things shoved down his lungs, he’s had a dose of gene therapy trial. It’s possible even a lay person can say well surely you can link those together and make five, but, you know. So but, you know, I did actually take it up directly, you know, with him then. And I said “you know, actually he’s not doing so well generally all round because he’s got quite a burden to deal with now. And whether it’s linked of not to, to the gene therapy trial he’s left, been left with some issues that he’s got to deal with and I just want to make sure you’re actually aware of that”. And so I did feed it back to them, you know, that way. But that was, that was a very difficult time.

 

 

The stringent guidelines for conducting clinical trials in children gave Alison the reassurance...

 I think it was really it was not so much the level of risk because I think as a child; I was making the decisions when he was a child. [Yes.] There’s not a lot that they can do for children because the guidelines are much stricter I think over involving children on clinical trials. So they tended to be if they did ask you to do anything the involvement was fairly small and fairly not risk free but limited, a limited risk. As I say he mentioned the Flixotide trial well they knew that that was an established drug so it was safe but they wanted to see the, you know, whether it was actually effective. So they knew that if you took that the risk of taking that had already been investigated I guess by whoever makes it prior to it being released as a licensed drug onto the market and they were just then looking into the effectiveness of it. So I think when they are involving a child in a trial it was on a more limited risk basis. So the only decision we really made then was what kind of involvement did it have in terms of how, what tests were they asking you to do. And it was only if they were going to stick a needle into him, when we said “well thanks very much for asking but no, we’ll pass this one by”. It was only when he got older really where he needed to give permission for doing it or consent that the risks probably seemed to become slightly more increased and the actual level of commitment became a bit more increased as well.

Alison's son has Cystic Fibrosis and she has given consent for him to take part in various...

 As he got into his teenage years, probably from the age of about 14, we started a process of transferring his sort of care so to speak for him to take some responsibility for it. And that would be sort of me withdrawing from the room if the doctor was in there, if he wanted me to. It also, we had a lengthy plan of how we actually got him to go to the hospital on his own, so I’d be training him from about 14 upwards of learning the journey up to [hospital]. Stage by stage of me withdrawing one bit further each time of, you know, well you catch the train I’ll follow you on the next one then wait for me I’ll meet you at the, it sort of, so that he, he would be ready to sort of move into taking his own, making his own decisions. And in fact at the hospital they did start, I know certainly by 16 he, we signed some forms that he could then consent to things or had to and it wasn’t just sort of me. So we did start that process very early on. And he was he wasn’t fazed by having to move from the paediatric department to the adult department. And although I always sort of said, you know, of course I will come with you if you want me to; you’ve got just this [laughter]. And but in the main he sort of dealt with things on his own except for, there was a few occasions when I maybe stepped in and said you really need to go to the hospital, you really need to ring them up, [laughter] you need to do this. Yes but in that he has assumed, he assumed responsibility, you know, quite early on. And then I realised that I had to back away from making the decisions, you know, for him, he would then have to make the decision for, you know, for himself. But what I would try and do is always strike up a conversation to say “Oh I see that, you know, you’ve been asked to do this” or “Something’s arrived in the post, oh what was that”, you know, “Can I see it or, would you like to talk about it”. And then, I’ve made sure that I’ve read up on things he’s been asked to do and gone back to him and said “You have read this properly haven’t you” and “Had you taken that into consideration”. And I always remember he turned round, and he said to me “Yes I’ve read it all I know what the risks are, I’ve read it and I’m willing to accept them”. 

 

As a parent you know, I’ve had, I’ve had to say “Okay fine good, then I support you in what you’re doing”, because he was very clear about “I’ve made my decision, still want to do it”, you know. One of the possible side effects in one of the papers was possible risk of cancer being induced by it or something. It was something like that and he was no “I’ve read it, considered it and I’m willing to accept it”. And we chose, we have no choice but to say well, you know, he’s an adult and it’s up to him.

 

But how do you feel?

 

You feel, I think it’s, it’s one of the lessons I learnt and this was the hardest lesson to learn being a parent of a CF child. And it probably applies generally, you know, anyway, is that having being so involved in his life, and helping him, you know direct his life to get the best out of things. The hardest thing to learn is actually it’s their life. It’s not my life, you know, it’s their life to live you know, as they want to. And they are responsible at the end of the day for the decisions, you know, regarding their life. And if I’m not happy with it I can, I can’t change that for him. All I can do is make sure from my point of view that I’ve helped him to make a considered decision if that’s how it’s to be so. Sometimes you feel, when you see it in black and white there’s this, this and this risk and then and actually I’ll talk a bit more about this study and the problems he did have after it. 

Alison's son has completed a Phase 1 trial on gene therapy for cystic fibrosis.

 And there’s like I was saying the Phase 1 study, it was a targeted Phase 1 study because they ethically they had to give it to patients who already had CF [cystic fibrosis] because they couldn’t possibly go treating normal members, ordinary members of the public, normal ones, unaffected of the population. Ethically they would not have got it agreed to have given it to unaffected members of the population. So that’s why they had the big run-in study to get their database and to carefully select some Phase 1 type sort of patients.

 

Twenty-seven and they were actually given this single dose of, you know, gene therapy and that’s been done now and then they’re and they’re now ready to move onto the next stage of giving [a multi-dose].

 

So do they monitor that, are they monitoring that?

 

For the twenty-seven I mean, well yes they were monitored within the month period. Because if you give a single, single dose of it was, the effects of it were going to last for a certain number of days. But because the, the lungs always renew themselves it’s not going to be, you know a permanent change in there so. So that they just wanted to see, you know, test it in, on a single delivery and then, but then they then move on to do the multi dose trial where it is done over a sustained period of time to see whether it would, any benefit had been maintained over the time.

 

Having a good relationship with her son has really helped Alison in supporting him through...

 Moving on to the Phase 1 trial which was, and this was the one that had all the information on it about, you know, some, some of the possible risks of it. And that’s what I did, you know, I have to say I do not sneak into his bedroom when he’s away or whatever, or see what’s lying around [laughter]. ”Oh just clearing up your room, oh I found this”, [laughter] so. And that, I remember having the conversation with him saying are you sure have you read it properly and, you know. And that and that one, that could be, because actually that had quite an involvement for me in the end as well which wasn’t clear at the start but, you know, he spoke about having these bronchoscopies and things. Well of course what he didn’t realise, and it was, which is good in a way for him because with all the hospital treatment he’s had over the years we’ve always just sort of made it as a normal part of life. You just get it over and done with, you do it, it doesn’t really interfere with your life or rather your life goes on around it. So he missed, even though he had hospital, hospitalisations and things, he didn’t miss that much school because to him it was normal to be ill. That was normal for him so we would just carry on and carry on and do things unless he was really, really unwell and really couldn’t get out of bed, life went on, doing it. So he had to go for these bronchoscopies and other things and he’s “Oh I’ve got to have a general anaesthetic”, you know, “But it’s only a day, so I’ve arranged for it this day”. And I would say “Well okay I’ll just check the calendar because I’ll just see if I’m around” you know. “Well it’s alright because, you know, I’ll just go up there and I’ll catch the train home and come back” and [laughter] and “Actually Robert if you’ve had a general anaesthetic you won’t be allowed to just come home, I will have to be here to make sure I can look after you”. And just things like that. Yes so we’ve got to work out how to get you back from the hospital, and “oh”. And yes so I had to be sure enough and they said “Yes, yes you’ve got to be looked after for 24 hours” you know afterwards and things. Yes I know I do work [laughter]. And so its, I mean it’s quite refreshing in a way to have that attitude towards it. But, so I had to, yes I had to be on hand, you know, for that and of course when his voice went and I’d say “Perhaps you should tell them at the hospital”. But well no he came out of hospital two days later, went off to Belgium, and we didn’t hear from him for a week. And then on the day he arrived back, I knew he was due home that night, we had a text, no we had a phone call from a friend of his and said “Oh Robert will be arriving on such a train he can’t talk to you because he hasn’t had any voice all week”. And I’m thinking what on earth is going on, and then you find out, you know, that oh maybe 10 days after he hadn’t had a voice, not sure that this is right I think you need to ring the hospital. So it from that point of view I’ve had to be on the sidelines but still have an active role just the same.

Making an informed decision whether or not to enrol your child in a clinical trial is essential...

 Yes it’s making informed decisions really on that so. It very much depends on what kind of outlook and philosophy about life that the parents have. You know in their attitude in their family life, you know, really, it’s as I say we’ve always striven to do the best we can with the circumstances that we have and to that extent if you, if you can get better ways of treating and also better ways of understanding really, you know, about the condition so that that helps. In knowing what’s good or what’s bad or what could be you know, helpful to do. So it does really come down to whether, you know, the parents understanding I think really or what they want to do and what their child is willing to do as well.

Alison attended a conference providing feedback on a gene therapy trial. However, she felt her...

 Yes which we did have from the, they did this conference, you know, the one they did that all parents could go to and we went with some kind of inside knowledge of it and that’s where it was interesting to see “Oh yes all 24 of them were fine with no ill effects afterwards”. And you’re thinking mmmm okay my son’s experience was slightly different to that and that’s why I said afterwards, you know, are you sure it’s not connected but I didn’t feel it was appropriate to announce that to a conference hall.