Fragile X and Autism Families for STX209

Hey everyone, if you’ve missed it, the fragile X parents have been blowing up social and traditional media talking about the sudden termination of the STX209 extension trial we are participating in. We have our date to visit our clinic, turn over our supply of medication and get a titration package…it’s so nice to be able to watch the ax descend in slow motion right after they pull the rug out from under your feet. Yep, that was sarcasm…it’s back…but leaning a bit towards the mean side due to that simmering pile of rage sitting in my stomach.

I’ve had a few people ask me why we are fighting for a drug that failed the latest trial. *sigh* It sounds perfectly reasonable to stop spending money on a drug that failed doesn’t it? Even I would agree that when you have limited resources, you shouldn’t keep throwing them at a solution that does not work.

But here’s the thing…the drug didn’t fail. It didn’t. And, no, I’m not calling the headline writers liars. They have limited space to work with and “Autism drug fails” fits a lot better than, say…

“Autism drug fails to meet the primary endpoint chosen for this study but met several secondary endpoints which the FDA completely disregards because they are an archaic, lumbering beast of an organization that doesn’t know how to handle drugs for such complex, spectrum disorders.”

So…they aren’t liars…but the headlines don’t tell the whole story. Just as the “primary endpoint” doesn’t tell the whole story of a drug’s effectiveness.

The drug failed to meet the primary endpoint of the study. The primary endpoint which must be chosen by the company, per the current FDA rules, becomes the BE ALL and END ALL for a drug. So, if you pick social anxiety and not enough participants who received the drug have a statistically relevant improvement in social anxiety…you fail. EVEN IF, you saw statistically relevant improvements in the secondary endpoints. You have to pick a NEW endpoint and spend millions and millions of dollars more to see if you can meet that new primary endpoint. Wash, rinse, repeat.

The drug “failed” but the process was heavily stacked against it. The process should be protecting the sick and vulnerable, not standing between them and the drugs they thrive on.

So, that’s why we are fighting so hard for a drug that “failed.”

On the fragile X side…it has already cleared this efficacy phase (Phase II) and had moved on to the dosage efficacy phase (Phase III) which is all about figuring out how much to give each patient to treat their symptoms. So on the fragile X side…where I am sitting…this is even more of an awful development because we are *THIS* close to getting through. And it might still, if they got lucky and get the data they need they’ll go to the FDA and try to finish the process. In the meantime, kids like mine, who are proof that this does work, are left hanging…fingers crossed that they did get lucky and fingers crossed that the rest of the process will be done at some point and fingers crossed we don’t experience a total regression in his developmental and educational process.

That’s some tough crap to ask a mom to tolerate…our future relying on *fingers crossed* when there is something, right now, that works.

Which is why a few parents have decided that we want the world to stop and rewind. No? Fine, we want Roche Pharmaceuticals to rewind. We want them to revisit their business decision and PUT THE KIDS FIRST…put them before the dollars…where they belong.

And thus, Fragile X and Autism Families for STX209 was born. Please read our stories and share them…please help us get the word out so we can convince Roche that pulling their support was not the right decision…at least not for the many, many families who’s lives are being turned upside down and inside out by that decision.

Please click on the image and like the page on Facebook. Then read stories of kids who need this medication!

There are two petitions circulating as well to get out the story…please sign the petitions if you haven’t already!

Change.org (click on the image to go to the petition):

The White House (click on the image to go to the petition):

Thank you for all of the love and support, it’s what is keeping us focused and moving!