I am finally allowing myself to believe that this clinical trial is working for Pudding. Although I have seen the results that other boys have achieved, I have until now always had to remind myself that:
a) a trial is exactly that. A trial. What works for some may not work for everyone and that is why they have to test it out.
b) at the time of testing Pudding had almost the lowest possible score that would still allow him to go on the trial. With results that unpromising I had no idea whether this would effect what we could expect from the drug.
The natural course for those on the most severe end of Hunter Syndrome is for progression until some time around age 4 or 5, and then a time when skills plateau before being gradually lost. This is because the normal enzyme replacement therapy that Pudding now receives weekly doesn’t cross the blood brain barrier to work there. In the hope of stopping this regression, every four weeks we make a trip to Manchester for a concentrated form of this enzyme to be injected into his spinal fluid to break down waste products in his brain.
Since very early on, Pudding has always continued to develop and improve, albeit very slowly compared to others in his age group. Lately it feels like his rate of progress is making a little leap, and as he gets closer and closer to his fifth birthday we have to see that as a good sign.
Most of his achievements might not seem very much to another mother of a four and a half year old, but for us they are massive. As well as continuing to add to his vocabulary, his understanding is continuing to improve. For instance, when playing ball the other day I told him to ‘Stand further back’. I had to work SO hard not to accompany it with a gesture as I wanted to check whether he understood without. And he did.
He can now almost finish this set of jigsaws without any help; he picks out all the bits correctly and only struggles with putting together the three-piece police helicopter. (He is also often very insistent that the policeman drives another vehicle, but I’m not going to argue about that one!)
His ability to compromise is also improving. Rather than just stubbornly demanding TV he can now sometimes be encouraged to help tidy up first.
He has got used to lots of new routines at school and has even in the last few days been able to pick out his name from amongst all the other children’s. (Not something I’ve been able to recreate at home, but again we can’t have everything!)
So I should be feeling nothing but pride in his achievements and relief, right?
Part of me (a small part) doesn’t really want the trial to work. I can hardly believe I’m actually writing this. What an unnatural parent I must be to wish away my child’s chance at life. But much as I feel bad for this I can’t brush my emotions under the carpet and pretend I never have them.
The truth is, if the trial doesn’t work then I won’t have to live with the fact that we have this chance when others don’t. Others who didn’t pass the screening, or for whom it came too late. Others like Ethan who I was so happy to meet only a few weeks ago but who is currently having a hard time of it. Other mothers’ sons all around the world who deserve a chance too.
I know that allowing my child to suffer will not alter the suffering of others, so of course we will continue on this path. I hope it is proved to work. I hope the NHS will accept it as a treament. But I will continue to feel guilty about it.
So I ask of you, please celebrate with us when things are going well, but don’t forget the rest of our MPS family. I’m grateful for this trial, I truly am, but it is only a stop-gap. A far from ideal solution. We need a cure.