The first gene therapy trial that Isaac took part in (in which he was the very first patient worldwide to receive multiple doses) has now been discontinued due to adverse reactions in patients. We couldn’t talk about the details at the time, but the latest press release can be found here. The fact that Isaac was the sentinel patient was, pretty frickin huge.
I couldn’t stay in the room during the treatment (via nebuliser, his team were masked up carefully) and stood shaking in the corridor watching through the gaps in the blind. Lately, one of the team told me that it was just as scary for them (they are always cool as cucumbers at the time). Hearing that reassured me somehow, knowing not only how much they care for their patients, for him, but also that it was as significant to them also. This is new, groundbreaking science, and with that comes risks.
Knowing that this drug will not be progressed further is a blow, but we go in knowing that very few phase 1/2 trials progress to the point where they are actually licensed for clinical use. The learning that is gained will benefit future developments, and every clinical trial participant has contributed to that important fact. Beyond proud of our boy.
Furthers news that the Lenticlair trial was halted after just five patients being dosed is a stab to this wound. We had talked to the Brompton hospital, London, about joining this trial some time ago. At that time, again, he had the possibility of being the first patient dosed in the trial. Only with a trial for gene replacement rather than gene editing, the effects could be much longer lasting. At that time he opted for gene editing instead, because of the potential risks, but also that he would likely be ineligible for further gene therapy trials if he took part. Now that the trial has ceased, at least for now, we are so glad of his decision. Gene replacement in CF has been the ultimate goal to hope for since the early 2000’s, and now, what?
CF is a rare disease, and Isaac is now in a rarer group still. Only 10% of people with CF are left ineligible for CFTR modulators which are transforming the life of so many patients for the better. It’s not a great club to be in, and with trials being pulled it can feel isolating even further. Hope is everything, and any knock to that, for me, is hard to bounce back from.
His lung function has been on a downward trajectory all year, he finished IV antibiotics recently, and is starting new nebuliser treatments. In a week, his lung function climbed 10% which is, epic! He is on an urgent list for sinus surgery once more, and the headaches have been much worse than normal. Pain, heatwave, and a needle in your chest don’t make for the best summer, and that’s hard to watch. Really fucking hard.
I always want to be able to tell friends and family something positive, problem is, chronic illness rarely gifts us that. So instead, here are some pictures which say a thousand words. Rosa’s birthday, hospital with my boy, dancing with the best of friends, and getting out to listen to my book in a park x
