I should have been more specific.

When I said I wanted the boys to be more verbal, I should have been more specific.

I should have said, I want them to be able to express their thoughts and feelings and desires to me, in first simple, then more complex terms and phrases.

They are verbal. Oh, they definitely talk. They talk all the time. But they don't tell me much. Mostly, they use their verbal skills for scripting. Starting at 4AM, sometimes.

This morning Zack woke up and started talking immediately. I could hear him from our bedroom. I could tell from the tone he used, the dramatic inflections and the ups and downs, that he was talking about Dora, or it could have been Mickey Mouse Clubhouse. He was reciting parts of his favorite shows.

Scripting and self talk is something a lot of Fragile X kids do. It's a self-calming technique.

The vocabulary is excellent. It's just that it's only used to repeat things. When communicating, they still often use only one word.

They are verbal, but they can't tell me when they are hurt, or sad, or nervous, or excited. They can tell me they want "chips" or "chicken" but they can't say they are hungry. AJ can tell me when he's hurt, actually. Whatever the hurt, he'll look sorrowful and say "bandaid?" Bruises need bandaids. A sore tushy needs a bandaid.

So they are verbal. It's not that they can't talk. They just don't use words for much meaningful purpose, yet.

I guess I'll take what I can get. Yesterday AJ peed in the potty, and he looked up at the aide who was helping him at the time and declared, "it works!" It does indeed, AJ... It works at home, too, kiddo!

More Arbaclofen News and Impressions

About this drug trial, and the cancellation of the extension we were in?  I don't know what I think.

I don't know if it was working.  I can't decide.  If AJ were my only son, I'd be singing the praises of it.  Because he's a superstar, lately.  He's got a wonderful sense of humor and he's bubbly and interested in people.  He's still shy, but his curiosity helps him overcome it.  He says things that I couldn't have imagined him saying, a couple of years ago.  He still has quite a bit of anxiety; his hands are in his mouth all the time, and he self-talks.

If Zack were my only son, I'd be really down in the dumps about the fact that everyone else is reporting great benefits from Arbaclofen that I don't see.  Zack talks more, yes.  He is maybe a little bit more social.  He still has lots of problems with groups of people, though, and he still need to retreat and spend quality alone time with the iPad, if things get too busy or loud or intense.  He bites himself and his brother and sometimes others.  Especially if he's stressed.  And it seems like he's stressed a lot of the time.

It isn't a miracle drug.

But the thing is, we were on the extension because I trusted the doctors and I trusted the researchers, and I believe that it HAS benefitted them.  More than any other drug that is on the market?  I don't know.  Definitely not in the same way as any other drug on the market.  It has potential that current drugs on the market today, do not have.

Part of the benefit of Arbaclofen was the fact that, long term, it is supposed to help create the connections in the brain that the missing Fragile X protein would have created.  And I really do believe that any real changes in our boys are going to have to come from something that will work. long term.  I don't think you can fix problems in the brain quickly.  I expected it to take time.

And we lose time, now that we have to come off the medication.

I don't know.  I feel like I don't have any right to ask for it back.  Why should they continue to give it to me for free?  Because I helped them by participating in the trial?  Yes, maybe.  We were told, at the time, if we wanted to continue taking the medication that we'd be able to.  Until it was approved.  I realize there were no guarantees.

But, like I said, changes take time.  Time I was willing to put in.  It's rather frightening to have the possibilities of benefits yanked away.

There is a large group of people working to get funding restored, so that we can continue on the drug until it is approved.  I haven't had time in the past two days to keep up with all that they are doing, but I am glad they are doing it.

If you could just go to this site:

 

We The People

Your Voice in Our Government

Help find immediate funds to keep Seaside Therapeutics STX209/Arbaclofen extension active while FDA approval is pursued

And sign it by just clicking, I'd be appreciative.

Because I don't trust my own observations.  I think this drug is worth taking, and I think it should be approved.  The doctor we worked with certainly thinks so, and I trust her opinion vastly more than anyone elses' opinions.

I don't know what is going to happen.  I don't know if we'll get Arbaclofen back, or not.  But I can't not try.

More Clarity on the (Not Quite as Shocking as I Initially Reported) Drug Trial

It's been a couple of days.  The dust has settled, and I have a better grasp on what is actually happening now.  They aren't dropping STX209 and all the results of all the trials in the garbage and giving up.  They are still studying the data they have been collecting for the past several years.  If the data results look good (a pretty huge IF) they will submit it for approval to the FDA.

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.

The Shocking End of the Drug Trial

It's a sad day in the Fragile X world today.

Yesterday afternoon, I saw something on Facebook.  Something that shook me to my core.   So I did what any normal person does when shaken to their core.

I called my parents.

Later I got an email from the doctor, confirming the news I'd read online.

"Seaside Therapeutics has terminated the extension study for arbaclofen (STX209) in which you are currently participating, as of today."

And even though we were warned this could happen, I am shocked.   We've been in this study for 21 months now.  A lot of families I know have been on the medication for years.  I knew it could happen, but I really believed it would not.

I was concerned that the trial could be extended, and not approved by next spring, as had been projected.  I was concerned that I'd have to continue making these trips to Chicago that were starting to wear on us.

I wasn't concerned they'd terminate the trial.

Terminate.  What a terrible word.  Once when I was in high school and an early, inexperienced driver, I had to come up to the Twin Cities to pick up my mom at the airport.  It was easy to drive down the highway and follow the signs to the airport itself, but when I got closer, suddenly the choices weren't as obvious.  I had to decide whether to take an exit to the "TERMINAL" or go back on the highway.  I didn't know for sure what the TERMINAL was, but it didn't sound like a place I wanted to go.  It sounded very bad.  Very bad and very final.  (Okay, I was in high school, maybe I should have known what a terminal was, but I was from a small town and had never driven to the international airport before.  I panicked.)  So I went back to the highway.

This was before the Star Trek technology of cell phones, so my mom had to just stand there and wait and hope I'd figure out how to get there eventually, and I had to figure out for myself that the TERMINAL was actually the airport.

Getting the news yesterday felt a little like when we got the Fragile X diagnosis . I knew the boys were being tested for some genetic abnormalities, but I truly believed that nothing would be found.  Of course they wouldn't find anything, because that's logical, and the logical almost always happens, right?

This is what thinking positively gets you.  Blown away, when the positive thing doesn't work out.

How many times have we driven to Chicago?  I think we are at 13, in 21 months.

It's hard not to feel like it was all for nothing.  A lot of parents are grieving.  Sad.  Disappointed. Angry.  Heartbroken.  Devastated.  Crushed.  My word was crushed.  Because even though we hadn't experienced the miracles that other families were reporting, the fact that other kids with Fragile X DID show life-alerting responses meant that this drug was hope for us all.

For years now I've been hearing about a cure for Fragile X.  A treatment that eliminates the symptoms.  Hearing that reversing intellectual disability, once something of science fiction, is entirely possible, and within my boys' lifetimes.

This feels like a pinprick into our balloon of hope.  Not just a slow leak.

I know there are other medications being studied that show lots of promise.  I know this was just one of many.  It's just that it takes SO LONG to get a medication approved by the FDA in the US, and this one was SO CLOSE.  And a lifetime is so short, it seems.

A lot of us whose children are in the study keep our families and friends (and ourselves) updated with blogs.  Here are some of the posts others have written about their feelings on the termination of the Arbaclofen study.

Fragile X: One Day at a Time

It's Who I Am

Basically FX

The Reluctant Adventurer

It's hard to get up and be strong today, and continue the fight.  My spirit feels crushed.  We were given a torchlight of hope with STX209, and yesterday it was ripped from our hands and doused.