A Day with ACADIA- Part 2: Assurance

Some of the ACADIA employees at the Rett syndrome Awareness event.

Katelin and I had the great pleasure of being invited to ACADIA a couple of weeks ago for Rett syndrome Awareness Month. Last week I posted Part 1: Awareness; I had PLANNED to follow that closely with Part 2, but a little thing called the “Official initiation of Phase III” interrupted that plan! But, this part is especially important now that the trial has officially started.

I have held ACADIA and Bill Keller, Executive Director; Patient Advocacy and Industry Relations, in high esteem since I first met them over a year ago. I was struck by their commitment to the families- how can we make this process easier for families. I was reassured by how important they believed US to be in this process. They interviewed multiple families, had an in-person roundtable with families in Princeton, and reached out to people and organizations that could give insight as to how to best run the trial in a way that supported families. I was reassured then, now I was given assurance.

One thing Bill Keller said that really resonated with me because of the sincerity and sense of duty that he said it with- (paraphrasing here) “I met Steve Kaminsky and he told me that since this drug (Trofinetide) existed it was our responsibility to get it to families.”

And that sense of responsibility is clear in everything ACADIA does-from listening to their employees’ desire to learn more about Rett syndrome from families to setting up a website weeks ago that put families in touch with sites. Despite all that, the one thing that sealed the deal for me was this-

ACADIA wants to hear from US, the Rett community-

  • Can you please let me know, so that I can share with ACADIA, the resources and support that you value most in caring for your Rett child by way of getting new information?
  • We also talked about community support programs such as Strollathons etc. but it would be great to know, if ACADIA attended these events, what information would you value the most. Keeping in mind they aren’t able to divulge anything about how the trial is going, but should they be a presence at these events ACADIA wants to make sure that they are providing something of value and not information we already know, which would cause frustration.

Let’s pull together and give them options to choose from. This is not a company that is saying… we’re the scientists, you’re just going to have to wait until it’s all over and deal with it… they’re saying, “We are in this together.” Something along the lines of “It takes a village” to do this Phase III and all its aspects right.

What assurances did we get?:

That they are systematically having sites inspected and rolling them out as soon as possible.

That all issues with manufacturing-such as multidose storage etc. have been resolved.

The mere fact that the trial is a go means they have enough Trofinetide in four different tiered doses for every child in the trial and for Lilac 2-an extension to (hopefully) cover from the end of the trial to approval.

That ACADIA has a plan in place to assist families with the costs of trial participation.

That ALL the families matter, those in the trial and those that don’t qualify because we’re all going to get it if (when) it gets approved.

I will forward suggestions to ACADIA in a couple of weeks, giving YOU-parents/grandparents/caregivers a chance to participate in a real way. ACADIA feels that trial participants are not the only ones who can make a difference. Here is a chance for those with children/adults that don’t qualify for the trial itself to contribute in a positive and meaningful way.

You can leave a comment here, on my main blog: Trail to a Texas Trial, on Facebook or email me at m3ml@hotmail.com. 

We’ve got this. We’re in good hands with ACADIA and Neuren.

A Day with ACADIA- Part 2: Assurance

Hot off the Presses- The Horses are Outta the Gate!

Our jockey wears a purple hat!

Well yesterday’s post WAS going to be the last one this month but then I got an email from Acadia- “Hot off the Presses” it said and then this announcement- ACADIA Pharmaceuticals Initiates Phase 3 Pivotal Study of Trofinetide in Rett Syndrome, a Rare Neurodevelopmental Congenital CNS Disorder  The tears started around the word “initiates” and by the time I got to “Trofinetide” I was a blubbery mess, snot and all. Within minutes of posting it to Facebook, I got a call from another mom and there we were the two of us crying, swearing, cheering, making no sense and so much sense all at the same time. I count that as one of the precious memories I hope to carry all the way to my grave.

Following quickly on the heels of that was this announcement from Acadia- Trofinetide Phase-3 Clinical Study Update There’s a lot of pertinent information in there, so please take a look. The thing that jumped out at me are three sites- Phoenix, AZ; Houston, TX; Chicago, IL- have already started screening. Now, I KNOW some parents are getting responses along the lines of… we already have so many interested families… do not be discouraged, reach out to sites further down the line and also remember unfortunately not every child screened will pass the process, so we NEED more interested families than spots in order for them all to get filled.

And lest we forget where it all began- Neuren released its own announcement . So, in case you all couldn’t guess, this is BIG, BIG NEWS.

I know that as we each have read these announcements the possibilities that come to us are all different. In a moment the “never gonna’s” have become “maybe will’s”, “could it really be possible’s” and while I am totally on the side of Trofinetide making a difference, I can’t possibly guess if all girls will be affected eventually in the same way, but I believe with all my heart and soul that at the very least some of your “never gonna’s” are going to go away. I believe you will hear the lilt of your child’s voice in some way that everyone will hear and understand. I believe hands will do things that may be very small or huge and it will seem like a miracle.

There are still things we need to know- is there a cap on improvement, meaning eventually is there a place where our children can’t improve anymore when we see there is still room? Are there side effects with long term use? Each and every family that partakes are heroes to all of us; even those who don’t end up qualifying, you tried and that’s everything in a world where trying can be so hard and defeating, it’s so very brave to take that chance.

The race has begun, the horses are out of the gate; I’m betting on ours, the one with the purple-headed jockey; I’m betting on us.

Hot off the Presses- The Horses are Outta the Gate!