Clouds, May 2010

Clouds, May 2010

Wednesday, August 14, 2013

guilty blessing

Remember the clinical trial I was posting up the you-know-what about, a couple of months ago?

At the time my social media feeds were completely filled with one heartbreaking story after the next... due to the abrupt end to the experimental study, of the drug Arbaclofen / STX209.

There were online petitions, numerous articles (including Holly's story in The New York Times), & even more coverage as many families also contacted their local news stations. Although these very vulnerable families were only seeing the beginning of a very difficult time... they did not hesitate to put themselves out there in front of the media... to share their story... & to fight for their children.

The most recent media coverage of the Welin family's story is one which I strongly encourage you to watch.
It was a sudden, fresh reminder of a complete range of emotions for me.

In the words of Caleb's incredible mom, Melissa:

As much as it hurts my heart to know what Caleb will be missing out on there are families who are facing much, much more devastating consequences. There are moms being slapped, pinched, bitten, having their hair pulled and their hearts *broken* into thousands of pieces because their kids cannot handle the assault of every day life without this medication. Don't tell us it didn't work. IT WORKED...and there are moms bearing the physical and emotional scars to prove it..."

Their son Caleb is about a year older than Hayden, & definitely has personality traits which constantly remind me of H. Incidentally, Hayden was supposed to be a part of this trial too-- at least that was the plan.

Over the years we have been to clinics on both coasts & seen countless specialists. So let me be blunt: everyone said Hayden would eventually need drugs. Well at first it was therapy, therapy, therapy... & it still is... but then your child approaches that age when you're strongly encouraged to consider treatment medications, in addition to the therapies.

Our little kid was growing into a bid kid, his behaviors were getting tougher, our challenges were increasing, expectations were changing... & just when I thought we had this figured out, I think that's also when we began to realize just how much help Hayden really needs.

Very long story short: instead of trying something already on the market which can treat symptoms of fragile x (such as anxiety, attention defecit, & so forth), we educated ourselves about the various clinical trial options.

There was one study drug in particular which seemed the most appropriate for Hayden: STX209, also known as Arbaclofen. The hiccup, however, was that the study center closest to us was still awaiting FDA approval to lower the age inclusion. The approval was delayed, & delayed again, & then delayed some more.

Finally we had the green light. I scheduled a parent intake over the phone to confirm that H would qualify, & soon enough we would have access to this particularly promising treatment we'd been hearing so much about.

After years & years of all the clinic visits & all the doctors... after years & years of all the conversations with specialists, & those private ones amongst ourselves... after years & years of weighing the options & all the pros & cons... this is what it came down to. My hope was so strong I felt like I could see it.

But on the evening of the parent intake following a series of questions, the reality was instead the most unexpected, hard-hitting sucker punch: Hayden did not qualify. The words of the researcher on the other end of the phone seemed to hit me in my gut, "he does not even come close," he said.

The FDA mandates one target symptom. Of course in terms of helping someone with fragile x or autism, honing in on a single target is nearly impossible. That aside, pharmaceutical companies obviously must play by the rules & the target symptom of this clinical trial was social withdrawal.

Hayden just happens to be very social, & engaging. These are some of the reasons that no one ever suspected the specific type of developmental disability he has... which typically exhibits autistic-like characteristics.

I thought this news was the worst of it. I thought the worst part of this situation was the fact that Hayden did not qualify for this clinical trial...

But at least we had hope from the other participants. At least they were seeing continued improvement, various promising results, & not just in terms of the ONE target symptom. Many times over it was explained to us that just because H did not qualify for the trial, is not to say that he would not benefit from this drug... & we should expect that he will. Soon.

One day soon. Out of all the ongoing trials, this particular study medication was closest to FDA approval.

But then came the second blow which hit everyone very hard. Seaside Therapeutics-- the company which owns the patent on Arbaclofen-- sent a communication to all of the participating study centers. After learning they would be unable to secure needed financing, they were announcing the end of the extension trial medication known as STX209.

We had been patiently waiting our turn... it was as if a carnival operator shut down the ride right in front of us. But the worst emotion was yet to come.

More than being upset that H did not qualify for the trial in the first place... more than being upset that the study came to such an abrupt halt... & more than being devastated for the families mourning the loss of the person that this treatment had allowed their children to be... I felt an eery sense of relief. And with that, a tremendous amount of guilt.

We could have been one more among the numerous families affected by this. We were supposed to be, but we weren't.

I encourage you to please read their stories and then share them with others. For the more than 40 families on that site alone, & all the other people within the fragile x community who are affected by this... share one, share some, or share all... but know that your support does make a difference.