One Year Later - Reflections of STX209

It was a year ago today that we made our first trip to the Fragile X clinic at Rush for the beginning of the arbaclofen trial.  I have such mixed emotions today.  I can't forget the amount of hope I had for those little pills.  I remember how I stared at Ayden each day watching for anything different.  Then I slowly saw my baby emerge from the fog of anxiety he was living in.  One year ago my baby was barely saying fifteen words.  They were never spontaneous.

Today, he greeted me by name.  Told me what cereal he wanted for breakfast.  Asked for a different shirt.  Told me he didn't want to wear his hat to school.  (which at this moment I realize he actually managed to pull off!).  He also gave me a hug and then told me bye before running to get on the bus - where of course he greeted his bus driver.

I of course wonder now if the arbaclofen jump started his language or if it was just coincidence that we started around the same time he had a developmental leap.  My gut tells me it was a little of both, but in the long run it doesn't really matter.  We have been off arbaclofen since June 9.  In late July we started on sertraline and while it isn't as good as arbaclofen it is an acceptable alternative.  He still has a lot of anxiety and it doesn't really help his sensory issues, but that is okay.  For now, it's the best we have.  Had we not seen what arbaclofen could do for him already, I'm sure I would be totally happy with this.  But I have.  So it is hard to be happy with a 'second best' and why I feel like my emotions are so jumbled.  I want it back for him.  It all just feels so unfair still.  Even with his progress and how amazing he is doing - I wonder how much better it would be.

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Why This All Matters

There are those moments in life where it feels like a band aid has been ripped away and an open wound left exposed.  Those moments make me stop and wonder how I couldn't have realized what was under that band aid in the first place.  The ending of the STX209 trial was exactly like that for me in many ways.

I work tirelessly to be sure that my children have the best lives they can.  For Ayden, that work is just...more.  Our goal for him has always been to give him the happiest, loving, most independant life he is capable of.  I don't know what that entails, but I do know that I will move mountains to see that it happens.  STX209 was a step in that direction.  It helped him to function better with the high amounts of anxiety he has every day.  I believe that lessening his anxiety was what was primarily responsible for almost every other development we saw.  It just made it so much easier for him.  It should be fairly clear why it being ripped away in the fashion it was has been such an emotional roller coaster.  It left me feeling vulnerable and exposed - I highly dislike both of these feelings.  The only logical step was to take action, and thankfully a group from my X-family felt the same way - thus was born STX209 Stories.

In the midst of this, I did something I now realize should have been done sooner.  I looked into the FDA approval process for medications.  As with most government programs, it's a mess to navigate.  There aren't really any clear processes that aren't just broad blanket statements meant for basic understanding.  In the process of all this I came to this conclusion.

The FDA process is not setup to accomodate a drug that would target a core cause of a spectrum disorder.

Let me explain this more.  A spectrum disorder is a term most people are familiar with but maybe don't understand exactly.  Let's take autism for example.  Say you have twin boys and both are diagnosed with autism.  Each boy can be affected very differently.

 Boy A may be non-verbal, have a difficult time playing with toys correctly, and crave sensory input in the form of being hung upside down or spinning in circles.  

Boy B may have no problems speaking, but is unable to function socially and shies away from eye contact, peers, and any loving gestures.  Boy B also has stim behaviors like flapping and humming and can also been found rocking in a dark place quite often.

These boys both have autism, yet it is presented very differently.  Thus why it is called a "spectrum" disorder.      You may have mild issues on that spectrum, or more severe such as you usually see displayed in the entertainment industry.

Let us take the same twins from above ...

When introduced to a drug like STX209, these boys will both react differently.  You aren't attempting to help their specific symptoms of autism, you are helping the core of it to correct symptoms.  As such, Boy A may stop craving sensory input and begin speaking more while Boy B may stop using stim behaviors as much and begin to interact socially.

Is this medication then helping the twins?  Absolutely.  Anyone that interacts with them can see it certainly is helping them.  However, if they were enrolled in the drug trial  for STX209 that just failed on it's primary endpoint it would not show this.  The primary endpoint the FDA looks for in drug trials says that ONE thing will be improved and that one thing will determine the success or failure of the trial.   Other secondary endpoints may show the improvements that the twins are experiencing showing up in the data, but because that primary endpoint does not it is considered a failed trial.

THIS scenario that I just played out is why Roche pulled their funding from STX209.  They believe now that their own formula will be more successful in meeting a primary endpoint for FDA approval.  I disagree.  Their formula (now in Phase II clinical trials) is built on the same science and research as STX209.  It is also a NEW drug meaning long term use and affects are not known.  STX209 is farther in trials, shown to be working, and becaues it is a reformulated version of an already FDA approved drug we do know the long term affects.  Baclofen has been around since the 1920s I beleive.

And this is why I believe this group of other parents and myself are doing something important.  I believe all other medications that uses this same science will eventually hit the EXACT SAME roadblocks as STX209.    I also believe with the right backing we can get the FDA to approve a medication like this - it will be groundbreaking when it happens - but it will be a long road. A long road made much much longer by choosing to push a drug that isn't as far along in the process already as STX209.

So I'm asking.  I'm begging.  SIGN THIS LETTER.

http://stx209stories.com/an-open-letter-regarding-stx209-to-dr-severin-schwan-roche-pharmaceuticals/

Did you click that link?  Did you sign it?

http://stx209stories.com/an-open-letter-regarding-stx209-to-dr-severin-schwan-roche-pharmaceuticals/

What?  You still haven't signed it?  Look what you did.....



Sign the letter.  Please.  Be a part of the change for Ayden, Emmalina, and every other individual living with fragile x syndrome or autism as well as those many, many more who have yet to be diagnosed.

Here's that link again (in case you missed it)

http://stx209stories.com/an-open-letter-regarding-stx209-to-dr-severin-schwan-roche-pharmaceuticals/

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Fragile X and Autism Families for STX209

Been working on something....

I've been working with an amazing group of women, other fragile x parents.  We're hoping it will be big.  We need it to be big.

You can help by reading our stories on Facebook.  Liking them.  SHARING them.  Each person that shares  one of our children's stories radiates those stories to their entire group of friends.  Who in turn share.  Which makes our prescence much bigger.  So please, won't you help?

Visit this facebook page:  http://goo.gl/RAUGj

(Did you click that link?  What are you waiting for? ....)

(No, really, click it.)

.......

.......

.......

That wasn't difficult, was it?  See all those adorable faces?  Remember them as you continue on.

Like us.

Read the "about" section to see exactly what we're all about.

Read the links in our media section and see the coverage of how this drug is helping.

Read our stories.  Children, families, changed forever.

Then share with your friends, family, and whomever else you can.

PLEASE help us.  We need to make an impact.  For Ayden and for the many other children affected by this.  This sets us back YEARS in getting a theraputic intervention for fragile x syndrome AND autism.

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