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.

Why The Wiggles Suck Now

The original Wiggles

Disclaimer:  I feel qualified to grouse about the Wiggles because I have been watching them almost daily for around nine years now.  (Aliza loved them before the boys did.)  I see them on You Tube. I've seen all their DVDs, including the bonus features. Some parts of some of their videos I've seen and heard hundreds of times. I bet I've seen "Do the Monkey" thousands of times, because AJ's been loving that video for 5 years now. Seen them in concert twice. I haven't read Anthony's book yet, but it's on my list.  (What?  You didn't know Anthony had written a book?  Then you aren't as into the Wiggles as we are.)

(Quick background:  Greg, Murray, Anthony and Jeff formed a children's song and dance group in 1991 in Australia.  Found early popularity worldwide.  In 2006 Greg retired due to health issues, and Sam took over as the 4th Wiggle.  Six years and countless DVDs and live shows later, in early 2012, they announced that Greg was returning to his role and Sam was out.  Audiences were disturbed about this, since they'd become accustomed to Sam and most of their followers, being young children, didn't even remember Greg.  It was controversial.)

At the end of last year, Greg, Murray and Jeff retired, which was probably a good thing because two summers ago when we saw them in concert, Jeff was absent because he'd just had a Pacemaker put into his chest, which would have told anyone else hey, you might be getting too old to be playing children's shows, but not these guys.



Greg is out, Sam is in

And frankly, to me, with Sam in the group, it became "three old guys and one polished, young, cute professional."  Sam looked out of place.  He was too much better than the other three.  But the kids and I got used to him, and began to like him and accept him as one of the group.

Which is why we were all mad about how, last year, they gave Sam the boot unceremoniously and under the table, and then expected cheers and joy for welcoming Greg back into the group.  Nothing against Greg, we liked him back in the day, but Sam was well liked, too, and we didn't like the way they treated him. 

And there was an interview with Anthony on some Australian news program where he was asked about Sam and how Sam was let go, and how he felt about it, and Anthony acted like it hadn't occurred to him to ask Sam how he felt, and he admitted he hadn't even talked to Sam.  I don't know how they announced to him that he was fired, maybe they left him a post-it in his dressing room.



Sam is out, Greg is back

After all those years of singing and dancing right alongside him, Anthony couldn't figure out why people would think the group should have shown a little respect and courtesy toward Sam.

And now that all of them but Anthony have retired, The Wiggles is an almost completely unrecognizable group of people.  Anthony has always been the "cute" one, the one all the moms like, but now that Jeff, Murray, and Greg have been replaced by three very cute, young, bouncy people, Anthony is now the "old" one. 

They used to be this neat group of very ordinary, regular guys who got together and thought, why don't we start a kids' band?  And they hit it big, and that's what was great about them, was their normalness.  The fact that they weren't "Hollywood," that they were just like all the dads of the kids who loved them.  They were gawky and nerdy and we loved them for it.  Now The Wiggles are group of three beautiful people who went through auditions and tryouts and are professional and amazing and polished and perfectly adorable.


The new Wiggles - and Anthony

And Anthony.  The old guy.

(By the way, at the moment I'm publishing this, AJ is behind me at the kitchen table, watching "Do the Monkey" on the iPad.  I swear I am not making this up.  It's 8:15 AM and I've heard it around 15 times just so far this morning.  Which is why I don't think my above estimation of thousands is off base.)

Teeth and Nails

I trimmed AJ's thumbnail this morning.  It took two hours.  Because he didn't want it done, and so we took the iPad away from him and said he could have it back after he let one of us trim that nail.  Because it was long and sharp enough to perform surgery with.  They all are, really, but we were trying to be reasonable.  He was never going to sit still for all 10 nails at once.

So finally, he decided he wanted that iPad bad enough that he was relatively cooperative for the nail trimming.  By "relatively cooperative" I mean, I had to hold his arm and hand in place, but I didn't have to apply so much force that I worried I'd break his little arm.

One nail.  Two hours.

Last week we took the boys to the dentist.  AJ has a cracked tooth and two cavities on one side of his mouth and a tooth that is decaying on the other side.  We were due to get the boys put under anesthesia for teeth cleaning and putting on some sealants, but now we really need to get it done.  Soon.  I'm waiting for the dentist to call and let me know we are on the schedule.  (This is what you do with kids with autism and autism like disorders, where their sensory issues are such that they cannot lie quietly and submit to teeth cleaning any more than they can sprout wings and fly.)

We are going to have echocardiograms done on them at that time, as well, to check their heart valves.  Yep.  Fragile X comes along with a possibility of a heart condition called mitral valve prolapse.  Every doctor we see listens to their hearts all the time and they haven't detected any sign of it, but an echo will help us get a good idea what kind of shape their heart valves are in.

I started thinking today about other things I'd like to do, while the boys are under anesthesia.

• Trim all of their finger and toe nails.  Actually give them nice mani/pedis.
• Trim their hair.  Short.  Heck, maybe now that summer is around the corner, we should just shave their heads.
• Wash their ears.  They never really let me clean their ears.
• And I'd like to have homing devices implanted in the both of them, similar to what you can get for your cat or dog.  Then, I want an app on my phone where I can track their exact physical locations at all times.

Does that sound unreasonable?