They just can't afford to keep providing it for those of us on the extension.
It's not, necessarily, going away forever. It's going away for an undetermined length of time, and may or may not come back, depending on what they decide after analyzing the data.
Doesn't that just fill you with confidence that it will one day, be approved and on the market? Yeah, me neither.
That said - a good friend and I were discussing the recent termination of the STX209 extension, and the different ways people are responding to its cancellation. Lots of people are saying the drug made a significant difference in how their child's speech developed, and how they socialized.
And my friend said something that I found very true: "Fragile X seems to have plateaus and then jumps." (In development. Some that sometimes seem quite dramatic.)
One of the ways I check to see how my boys with Fragile X are doing, developmentally, is to compare them to other children their age with Fragile X. Everyone says you can't compare them to a typical child. So I compare them to the closest thing to them, developmentally. There are a couple of people who live not too far away who we've become friends with, who have sons with Fragile X nearly the same age as AJ and Zack. Who are not taking part in the trial. And when we get together, I'm able to observe their children with mine, side by side.
I'll take Trial Drugs for $200, Alex.
The answer is no.
The question is, Do I see a discernible difference in language ability, socialization skills, and general maturity between my boys, who've been on the trial drug for sure for 16 months, and these other kids with Fragile X, who have not?
My friend, whose son is not on the trial, has observed plateaus and jumps in her son's development. In fact, to my knowledge, he's not on any medications at all, and is just gluten and dairy free. I have of course seen jumps and plateaus in my own sons, too. Zack, especially, seems to experience phases of behavior changes and severe sensory difficulties, and then improvements.
I just read over everything I've written on this blog about the trial. A few observations jumped out at me.
1. I noticed changes in them when we came off the trial drug - assuming we were on it - during the actual trial part. I noticed behaviors that had gone away, return.
2. At every point I was sure to point out all the other things that could be affecting the boys changes - weather, weight gains, changes in routine, illnesses, etc.
3. Finally, just a few months ago in January, I posted this one where I decided once and for all that they WERE in fact benefitting from Arbaclofen. Even though Zack was still having some troubles, I recognized that overall both boys showed quite a bit of measureable progress in the 12 months of being on the extension.
But of course the big question is this - were the changes due to Arbaclofen in their systems - or would they have changed anyway, just from growing up and receiving tons of therapy?
Is there any way to get an answer to that?
It's going to be interesting to see the changes in the boys as they come off the Arbaclofen. They are going to have a tough time, I already know, because it will be summer. And summer is always harder for us. A looser schedule and humidity are tough on them.
Honestly, after this experience, I don't know how any mediation ever gets to the market. I don't know how any test results can ever be relied on. Aren't there infinite, indeterminable factors that could affect whatever changes are observed?
In an article I can't seem to find right now to link here (if someone knows what I'm referring to, please put it in the comments), it's said that the trial drug DID in fact help kids with social anxiety - it's just that the placebo helped almost just as much.
Wait a second. Doesn't that prove that it DIDN'T help? If nothing helped as much as the drug itself did?
I don't understand science.
|Why yes, I am wearing my sister's sandals. I can't find that wonderful pink shirt of hers anywhere, so I have to move on.|