When I was little my best friend Ashley and I used the same after school babysitter. In second and third grade we would wait in the elementary school circle driveway waiting for her to come get us. Sometimes she would be late. I would turn to Ashley and nervously ask, "what if she doesn't come?" Answer, "she will." I'd wait five more minutes and then ask, "but if she doesn't come, what will we do?" Answer, "she's going to come." This wasn't exactly highly reassuring to a second grader with anxiety and a chronically late babysitter. I'd say, "but how do you know?" And she'd say, "I just do. Stop asking me."
This is like that times a million. There are so many unknowns and uncertainties, but also some blind determination. Now that the treatment is growing closer to our desperate grasps, the reality of what this lifesaving and prolonging treatment means is settling in. These kids are undergoing this invasive treatment every two weeks, forever. Right now, there is no stopping point. We know the cyclodextrin works well for at least three years, because that's how long the kids in the trial have been on it. This is great news for us, as a brand new family stepping in: it is the hope. But as the families who are paving this road, in the trenches, I imagine it is also a sobering reality, two week terms of certainty knowing your child is safe, and only for now. And this is going to become our reality, too. It already is.
I don't have all the details of the treatment administration yet, but as I understand it, this is what it will entail for Marian. At 18 months old, she will be going to the hospital, being put to sleep, having a needle inserted into her spine, treatment injected into her spinal fluid, being held downwards at a 45 degree angle for forty minutes afterwards, hooked up to an IV, wake up groggy and scared, monitored for effects, and then see how she reacts. Every two weeks. Then, how is it working, is it too much or too little. Should we take a break, or keep going. I fear, how long will it take for her to start screaming when she sees us pulling up to treatment. I wonder, will she understand on a level that it is saving her life?
This will be our life every two weeks for the rest of her life, which I pray will be many many years, until further advances are made. And this is such a miracle and blessing to have. I feel ungrateful and selfish to even bat an eye at any of this because I know so many children with NPC and other disorders have lost their lives calling out in unanswered prayers for something like this to come along and save them. And it hasn't come in time. And for Marian it maybe has - but it is still absolutely terrifying. However, the alternative to not receiving the treatment is certain death. Probably in childhood, maybe as a teenager. The thought makes my hands shake as I try write this.
For now, we are waiting. Waiting for the treatment to begin, waiting to see how it works in the short and then long term. Waiting to see what will happen. If and when the babysitter's van will speed into the school circle, telling us to get in and that we are ok now.
#NiemannPickTypeC #treatment #VTS270 #cyclodextrin #parenting #raredisease #parentingfears