Happy new year, all you lovely people. Ours has been an unusually quiet one (we usually host a party and dance the night away) but for very good reason this year. We’re in a semi lockdown, a pandemic throwback, trying to avoid bugs for fear of jeopardising our hopes of Isaac joining clinical trials this year.
For a far more thorough and scientific explanation, you can read all about current gene therapy hopes on the CF Trust website, including interview panels of medics and scientists discussing these with the CF community (on which I may or may not appear 🫣). Take a look here.
But for a far more nonsensical and blunter version, I’ll try and tackle some of the real life questions I have been asked here too. I’m answering these from my own point of view, as a parent of a son with CF, as I’m not medical and only have a partial insight from our own side.
Is gene therapy a cure for Cystic Fibrosis?
No, sadly not. The current trials are looking at the lungs only. Since 90% of morbidity in CF is caused by lung disease, it’s a fantastic place to start though. The edited or replaced gene will temporarily help his lungs function normally. This treatment will need to be repeated (frequency varies depending on the type of gene therapy we’re talking about). The treatment will not directly help the other organs affected by his CF (mostly his liver, pancreas and digestive organs) but as his overall health may be improved, it might have an indirect impact. The hope is that it would stabilise his lung health, but it will not undo the lung scarring that he already has. We also hope that with healthier lungs, his current bacterial campers will up and leave. PseudoA has colonised his lungs for 17 years now; an unwanted lodger we all detest.
When will the trials start?
We can’t say for sure. We have some anecdotal dates in mind, but we also cannot confirm as we have been asked not to disclose any specific information online regarding specific trials.
If you join a trial, will he get to continue on the treatment if it works for him?
Sadly not. At least, not immediately. Many of these trials are in their very early days. First in humans. As such, data will need to be analysed from the trials intensively, and if there are any adverse affects in any patients, they may be stopped all together. If they all go well, there will still be a time where dosing will stop, and we would need to wait for further trial stages. If we did participate in early stage trials, we would be prioritised for latter stages though, so would hope to gain access much earlier than non-trial participants (this is the biggest bonus of clinical trial involvement, for us). From the beginning of trials to actual licensing and general access is likely to be many years.
But he’s getting on the trial, right?
This is the worst bit for us right now, as nothing is guaranteed. He needs to pass the screening and stay well enough for the period between screening and the trial. Not easy with a progressive, chronic condition. Even when on the trial, any adverse affects, or an exacerbation of his live-in lung infections, trial related or not, he could be pulled out.
How are these gene therapies given?
The current trials are nebuliser therapies. So he would be isolated and breathe in the drug over a period of time, once a day. There would be a huge amount of protocol involved (as there is with all clinical trials) especially as we’d be looking at a new or edited gene being aerosolised in the room, which could affect others, so he would need to be isolated for a period of time after treatment too.
What if something goes wrong?
Not something we like to dwell on too much. We know that these drugs have been through many trials already, but almost none in humans by this point. We do know that he would be in the right place, and have two to one care (two research nurses plus doctors at hand) and will be monitored more than he ever has been. Those huge fold out information sheets you get in a pack of tablets? That’s what these kind of trials feed into. If he as much as gets an itchy big toe after dosing? That will be on the list of side effects when prescribing begins. That tickles me somewhat.
More soon x
Lollo - her eyes mesmerise me ❤️
