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?

Has anyone seen my focus?

If you have, please give it my phone number or address.  I haven't seen it in weeks.

Stuff happens but I can't seem to get it together enough to sit and write it down.

If I could, I'd write about how yesterday morning, Zack wanted to go outside so he went over and opened the front door.  By himself for the first time, as far as I'm aware.  Our front door is big and solid and sticks in warm weather, and historically, young children can't open it.

It was before 7 AM, so I told him no, we weren't going outside yet.  He tried for the back door, then.  So I told him to get dressed and then he could go out in the backyard.

A few minutes later AJ took one look at Zack climbing up the slide, and tried to have a little backyard play time himself, also in his PJs.

I brought him upstairs to get him dressed, waking Mark and telling him to go watch Zack out in the backyard.  He came right back, and said "Zack's not outside, he's in the living room."

"Oh, he must have come back in," I replied.  AJ was dressed now, so we went downstairs, and two things registered immediately, one after the other.  The front door was wide open.  And Zack was nowhere to be seen.

I went straight to a hopeful state of denial and thought no, he couldn't have gone out the front door, and I checked the backyard and the basement before admitting that Zack had to have gone out the front door, and into our wide open, unfenced, unaware neighborhood.  I stepped out onto the front stoop and saw him right away - he was bouncing around in our next door neighbor's front yard.  He wasn't far away, he wasn't in danger, and I wasn't dressed for public viewing, so I called Mark and he threw on some jeans and ran over to fetch Zack.  Whew.

After seeking advice from my Fragile X Family friends and parents, I am in the market for a product called the Door Guardian.  So that my two monkeys won't open the door one day and decide to run away to Australia and start a Wiggles tribute band.

If I had any focus, I might also talk about how we were at Grandma & Grandpa's yesterday and Zack showed us his inability to transition from wintertime-stay-in-the-house to springtime-go-outside-and-frolic.  We tried to make him, but he's bigger and stronger than he used to be and cannot be made to do things he doesn't want to do.

Beautiful warm weather - stand aside, I've got an iPad and I'm not afraid to use it.