Monday and Tuesday we were in Manchester again. It is the end of Pudding’s first year on the clinical trial that aims to halt the progression of Hunter Syndrome in his brain. So he needed to go for an MRI scan and lumbar puncture under general anesthetic.
It’s been an emotional journey of course. First there was the decision to enter the trial when the potential good had to be weighed against the negative implications. There was the major surgery itself meaning the placement of a portacath into his spinal cord. The first few months when he reacted with sickness, and then the gradual easing of tension as the reactions were brought under control. The waiting game as we wondered whether it would help him at all. And the slowly dawning hope that as he approaches his fifth birthday there is no sign yet of him losing skills. Indeed he seems to be gaining them.
It is little things. Things that other parents would hardly notice. Like me asking him to turn on a light that was beside him and him doing it. Like me saying ‘good building!’ as he played with his blocks and him responding ‘da dyu!’ (his version of thank you).
So although the results of this end of year MRI scan won’t be available to us, I’m pretty sure it will be positive. Enough boys with Hunter Syndrome have now been recruited onto this study and while we wait for them all to complete the trial year, Pudding will join others on the extension study. They continue to receive the monthly doses but with less regular blood tests, ECGs and so on – something both Pudding and I will be very happy about.
At the end of this calendar year, Shire (the pharmaceutical company who developed this drug) will be putting the numbers together and starting the process of getting it approved as a treatment.
I should be feeling positive.
Yet my newsfeed has been filled this week with stories of an NHS in crisis. Not enough beds, not enough doctors, not enough money to treat an increasing number of patients. With these sort of pressures who is going to sign off an eye-wateringly expensive new treatment?
Right now I should be filled with campaigning zeal. I should be writing to my MP and demanding to know what can be done to save the organisation that could save my child’s life. But I know what the response will be. The NHS is ‘very important to us’. ‘We are doing all that is necessary’. Health funding is at ‘record levels’.
It shouldn’t stop me, I know. I shouldn’t let the sense of powerlessness take over. But I just…right now, I just…can’t…