It’s been a while, but that’s a good sign.
Isaac’s most recent IV’s finished a week before Christmas, with him feeling much better. Since then, he’s been pretty good. We all enjoyed a festive break full of much needed sleep, family, friends, celebrations, and house de-cluttering. Before going back to work, I spent three feral days in PJ’s, working all day….moving round furniture, cleaning, packing up bags for charity, loud music blaring. Me in my element. I got to spend so much quality time with my Anouk and Rosa too, and a quick family trip to Center Parcs. My beautiful girls astound me everyday. I am one lucky Mama.
For these IVs, I successfully accessed his portacath myself, my third time. Today, the community nurses came round for his routine flush and despite my growing confidence, I frustratingly failed. His port site is very scarred now, and his port is very small (he had it implanted when he was 13, so he’s grown a lot since then). Not all nurses can access it either, first, second or third times, so I know it’s not just me, but still… sticking an inch long needle down into his chest is not fun. My being able to do his port flushes means one less trip to hospital a month (our paediatric community nurses leave us once he turns 18 in March, and after that our only other option is going into clinic). I am determined to keep trying, and on his part, he is a very patient patient.
I’ve enjoyed working more with the CF Trust of late, both in focus groups for them, and as a patient representative for scientists and pharmaceutical companies. This week I had the pleasure of hearing Dr Eric Alton talk again about gene therapy and up coming trials. Way before CFTR modulators became the buzz drugs in CF land, gene therapy seemed our best option of a cure (…of sorts. There is ultimately no cure of cures, but I believe there will be treatments that help halt or slow the progression of the disease).
When Isaac was first diagnosed we followed gene therapy news with eager anticipation, salivating at any news of progress. Basically, Dr Alton was our GOD. But then it stalled. It was funded, and then not. It was in trials, and then not. They tried a lipid vector to transport the corrected CFTR gene into the lungs, and then not. Then they teamed up with a much needed pharmaceutical to fund further research into using viral vectors, and it went a bit…. quiet. Or maybe it didn’t, maybe CFTR modulators just grabbed all the headlines. Anyway, the trials are now ON. I'm not sure how much I can say about these yet, only that Phase 1 and 2 are planned and likely to start, in the UK, later this year. As an adult (just) and one of the 10% of people with CF whom current CFTR modulators do not work, Isaac should be both eligible and prioritised for clinical trials.
This is heart bangingly exciting, scary and overwhelming all at once. For one, he may not be accepted in the trials. Or he may not want to commit (the trials mean stays in hospital, intensive treatment, and rigorous follow up over 15 years - in which time he cannot commit to any other similar trials). Or he does participate, and the treatment isn't tolerated or effective.
It’s also very new and despite the excellent results in non-human trials, the residual risks linger. Previous gene therapy trials resulted in cancer in patients.
What would you do? If your lung health was declining, and your quality of life was already impaired, not just symptomatically, but by the aggressive treatment regime? In the last year Isaac has spent three months on IV antibiotics. With every exacerbation he risks losing more lung function, and decreased QOL. Would you jump at the chance of something which could, ultimately, make a huge difference? Which may result in far fewer daily treatments, drugs, subsequent side effects, IVs, boring hospital stays, and invasive procedures? When you think of it like that, it seems like a no-brainwork right?
But it’s Isaac. Our brilliant, wonderful, gorgeous Isaac.
2023 is looking like a big year for us. A potential drug that would treat the underlying cause of his disease, for the very first time; that is seismic. Alongside this, we're heading into a very exciting weekend for the whole family, more news on that and pictures to follow. A new family member ❤️
Love x
Rosa teaching me selfie etiquette…
