Sometimes I wish that I could say this clinical trial is all unicorns and roses. It IS wonderful, don’t get me wrong, but it is hard, and it has been harder on some than on others. There have been enough problems with this port-a-cath device to fill pages. There are several boys currently without devices, receiving doses by lumbar puncture under general anesthesia each month until their bodies are healed enough for a new device, but that certainly is not a long-term, lifelong solution. Some kids have shown dramatic responses, others not as much.
But really, all we’re all trying to do is just give our boys and our families a better life, a healthier life, a longer life. And that, I think and hope is happening.
So, the nitty gritty…..
Two significant developments last month. First was the result of the CSF white count. White counts are tested by taking CSF off the port (or from a lumbar puncture) before the dosing, but after dose #4, they don’t wait on the results before dosing. Well, Case got his dose, but his white count came back at 15. Under 5 is normal. So… we are guessing that his port is likely broken. I was heartbroken because the longest this port had lasted was 12 months, so we were hoping that it wasn’t a fluke. We are at 12 months, so we’ll see what it shows at the next dose and if port surgery is in our near future. Please pray that somehow the results are back to normal, although we’re not sure what we would then conclude caused the increase. Like I’ve said, a clinical trial is never a place to get too comfortable.
The second significant thing was that last month was Case’s 12-month cognitive testing. While I hoped for a huge jump, simply to substantiate the amazing changes we have seen, I know in my heart that it is just a number. The goal is, and always has been, stabilization. And frankly, since cognitive tests are normed by age, at this stage, even what shows as a loss in cognitive points could actually be a stable level of cognition and skills. On the flip side, a stable cognitive index is indicative of learning and improvement commensurate with their level.
I love graphs. I am a visual learner and I love the ability to visualize the positives, negatives, facets, and intricacies of data. If you could look at the graph of a child with MPS with cognitive affect, you would normally watch a steady or steep fall after the age of 3 or 4. There might be periods of stabilization, but the fall continues.
It is the fall that chills the hearts and haunts the dreams of MPS parents.
Numbers do not mean everything. Our kids are notoriously difficult to test because of their hyperactivity, motor challenges, and lack of attention span. But what these numbers represent are the minimum level of functioning, and they may be below their potential level.
So when you see that initial dramatic drop from 2010, visualize what WOULD HAVE BEEN had Case not entered this trial. THAT is the true difference this has made.
But frankly, these numbers don’t even tell the whole story. Here’s some examples:
76? Fabulous! But that’s only one tiny piece of the story.