Well, I don’t really know what to say. The UNC Institutional Review Board has put the trial on hold. Kids who currently have implanted devices and are receiving drug, will continue, but everyone else (which means Trey and everyone after him), are on hold. And it’s not even a drug thing. The drug is making miracles happen. It’s the device/port used to get the drug into the brain and central nervous system that is the problem. A number of the boys in the trial have had to have their ports replaced for various reasons, which means more surgeries, which means increased risk.
However, when Dr. Muenzer asked the parents whose children needed to have their ports replaced if they wanted to remove the device and stop the trial, the parents looked at him as if he were crazy. I would have done the same. I paged him twice yesterday (and he amazingly- he is my hero- called me back both times, even though he wasn’t ‘working’) to figure out a way to keep our trip going, for some way to convince the IRB to remove the hold quickly, so that we can still come down for our trip.
Each time we have gone down for assessment, Dr. Muenzer discusses with us that the worst scenario of this trial is death. Each time. I imagine that for Melissa Thomas, the mum of the first child in the trial, this consent may have been a little harder to sign than for the rest of us who now know what brilliant things this drug is doing for our kids. However, I would have done it (I went down to do it, only Trey’s IQ was too high at the time) and I know that many other parents would have done it as well.
You see, in the severe form of Hunter Syndrome, where the brain is affected, we know the outcome. Loss of skills, loss of personality, no sleep along the way, vegetative state, death. So when I found out that the IRB had put a hold on the trial, and although I understand that from a removed, medical board kind of place, why and how they would make such a decision, from my perspective, I am shocked and blown away. This drug is fixing hearts, fixing hearing, bringing IQ’s back up, to name only a few. As a mum whose kid is on a path to you know where, the idea to stop a trial that is fixing at the very least hearts, hearing and brains, not to mention the possibilities for spinal cord compression, eyes, hydrocephalus and the list goes on and on and on, in which the only problem is the device, is crazy. Why they could not discuss concerns, discuss remedies and change criteria all while continuing the trial is beyond me.
I know the port issue needs to be resolved. Long term I would not want Trey to have multiple surgeries either. But if it meant Trey got life saving drug in his brain each month, I would still do it. Which is what the parents of the kids currently in the trial are doing. They know how lucky they are to be getting this drug. There are so so many remedies to be tried that could all be done while the trial continues. It is my hope that the IRB and any other governing boards who are concerned, will realize how immensely the benefit outweighs the risk and will lift the hold, so the trial can continue, while working to find a port/device solution. Knowing and seeing what this drug is doing for the boys in the trial so far, I don’t want to have to watch Trey progress anymore.
Here is a link to a mum’s blog, who has become a good friend, whose son is in the trial: http://networkedblogs.com/ja7UP
I’ll update as I know more.
PS. The picture above is one of my favourites of Trey & I. We were dancing and having such a blast. One of those moments when all the stuff I’m talking about above, doesn’t even register. All that mattered was that moment. The photo was also taken before Trey started ERT and his ‘features’ were more prominent. It’s raw and real & I just love it.