Table 3 Deterrents for gene therapy

Deterrents

 Chemotherapy

‘Um, I don’t know, but, I – I – I don’t know. I just think to put them through something like the chemotherapy and everything involved right now, I don’t know that I would do it right now.’ [parent]

‘I guess I am a bit concerned about the uh, the uh, process of going through uh, any of the chemotherapy. Um I’ve done my best to keep myself in good shape and I think I’ll be fine, um, but it’s just going through that, I know, it’s an ordeal, so, I mean that’s worrying, but it’s not going to put me off of anything that uh,, you know, could potentially fix my thalassemia’ [patient]

 Genetic manipulation

‘it’s growing in your body and it’s your genes that are being edited, maybe there’s some long-term effects, right, maybe your offspring, um, would get you know, would inherit something that’s maybe coming to your gene sequence that wasn’t there before’ [Parent]

‘I don’t even know if we would know that for a generation. You know, of having done this and as those patients have children is when we would really start to find out what’s happening. So um, but I would, I wouldn’t um, I wouldn’t not pursue it because of that you know, if the success rates are great, I wouldn’t not pursue it because there’s a chance that, the editing of the gene can impact the next generation’ [Parent]

 Trading one disease for another

‘could there be a side effect that uh, he currently does not have to deal with….., could the gene therapy introduce a new challenge that he currently doesn’t face… and uh, maybe the answer is no but we would want to become slowly informed about the after effects and side effects of gene therapy before we make any kind of decision’ [Parent]

 Keeping the status quo ‘not rocking the boat’

‘chronic transfusions, while they are a uh, disturbance in quality of life, it is a, it’s a known uh, quantity, it’s a, like we know, it’s easy, it’s not simple, but, uh, we know what that probably looks like and we know that there’s little risk associated with it, and that’s just because we did on it for 33 times now I think’ [Parent]

‘it’s just that they’re very, very hard questions and you know why they’re hard? Because she’s stable right now. If you asked me this four years ago, I would say yes to everything. Cause she was in critical condition, um but now she’s living her normal or normal um, you know transfusions every three weeks is really not that bad for us, and she has a pretty healthy quality of life, so that’s what makes it really hard’ [Parent]

 Possibility of failure

‘you have something that is working and it’s not the most ideal but it’s gotten you to this point which is a pretty far point, getting to.., getting to my age, um, and then you’re asked to kind of take a leap of faith that hey, this could work and you could eliminate this great need for this uh, treatment, uh, but then on the other end you know if it doesn’t, do you find yourself in a worse situation’ [Patient]

Socio-economic

‘I guess my biggest concern about gene therapy would be, be err probably the cost ……, but how are patients going to be expected to pay for that. What’s that going to look like?’ [Patient]

‘I know that it can be a long process, it takes a – at least, um, a few months, um, so I’d be concerned about being out of work for that long. Um, and then if the insurance would cover it, or if I would have to pay all of it out of pocket’. [Patient]