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 firstname.lastname@example.org.
We’ve got this. We’re in good hands with ACADIA and Neuren.
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.
This Blog is hosted on behalf of Mel Lancaster, whose original blog was silenced.
I like to end this month on an up note and that presented itself when I was invited to speak at Acadia in San Diego. Bill Keller, Executive Director; Patient Advocacy and Industry Relations, of Acadia wanted to do a Rett syndrome Awareness event for the employees of Acadia… because the EMPLOYEES wanted to know more about it and to get a sense of the families and children they are working so hard to help.
Now, had Bill told me the scope of the audience I still would have showed up but probably with a liiittttle bit more trepidation. And, I wouldn’t bring it up except that I want you, the reader, to understand the interest and to know that this was a voluntary event. There were so many people who wanted to be there that our interview had to be broadcast to another room, there weren’t enough chairs for everyone and people were standing AND it was being broadcast as a webinar to Princeton.
Katelin, Flynn and I were met warmly by so many of the executive staff and made to feel so very welcome. Bill had asked what kind of food Katelin liked and damn! did they get it right. My girl feasted on Chicken Alfredo and then feasted on my Chicken Alfredo, lol. and, get this… Katelin’s wheelchair was in need of some minor repair but it was making it difficult to use and Bill promptly called the maintenance dept and it was zoomed away and came back as good as new! Thank you, Juan! There was a photographer snapping away. I’ve been sent four pictures, two of them have Flynn in them. I have to wonder if the other two are the only ones that didn’t have Flynn, lol, she was VERY taken with our boy. Needless to say, we were treated like very honored guests.
It was a big deal. Probably one of the biggest deals I’ve ever participated in. There was a briefing, a debriefing, sound checks, I almost felt like I was on a News talk show. I had a portable microphone. It was an experience I can hardly believe Katelin and I were invited to. I can only hope that I represented our community as well as it deserves.
The first thing they showed was a video of Katelin of her initial journey with Rett syndrome that I made as a NNZ-2566 trial fundraising video (I edited the very end out that was asking for donations ) Luckily, Bill had forwarded the questions to me in advance so I was prepared and I will share some of them with you.
Can you share with the ACADIA team some of the activities that are going on in the local community around Rett Syndrome? :
I, of course, brought up the strollathons, and golf fundraisers, the LA Feast, but other events that I feel are important for the community are events like the Paint a Purple Pumpkin, and the Paint Your Nails Purple and post a picture. These are important for our community to feel connected to each other. One of my FAVORITE events is Blue Sky Day and the message it sends- so symbolic of our climb as parents to help our children, find a cure/treatment and symbolic for our children- the effort to achieve every little advancement in their own personal lives.
I recently participated in a podcast series and shared the link with Bill- To listen to the episodes, please visit anchor.fm/remarkablepodcast or search for “Remarkable Rett” wherever you listen to your favourite podcasts.
What about other clinical trials and areas of research?
I mentioned GP2C here. They did a great article about the different trials going on this year. The ones specifically I mentioned were the Sarizotan trial; Anavex 2-73 we discussed the Epidiolex trial in another question. I know there are two that I missed- the Ketamine trial and Triheptanoin UX007. The Ketamine trial is something I’m excited about. I haven’t yet researched Triheptanoin UX007.
I know that you have been following the Rett Syndrome research space for years. What is going on that excites you about future developments?
Well, of course, I said the Trofinetide Phase III excites me, lol. But, there are other developments as well. Anavex 2-73 is an exciting possibility. One area that is intriguing is the use of stem cells and one recent practice is feeding a fresh placenta to children, you can find out more about it here- Placenta Preservation. But, what excites me the most is the sheer number of trials in some stage of development or another.
Can you share your opinion about a topic we hear a lot about in California which is medical marijuana? Does it have a place?
I freely admitted this is not an area that I have expertise in. Mostly of what I shared came from reading accounts of other parents. I said it definitely has a place, whether it’s Charlotte’s Web or the like or other systems of delivery, like Epidiolex. I also brought up other alternative therapies such as essential oils and other naturopathic therapies. But, because this is not an area I can really give in depth insight on, I referred to Syndi Knowlton of Utah Kids.
Can you share with the ACADIA team your recent introduction to Dame Margaret Brimble and her role in the development of Trofinetide?
How is Katelin doing today? What are you excited about for her future?
What is your greatest hope?
These two go together. First, Katelin is doing very well. She’s healthy. Her spinal rod surgery went as well as could ever be expected- her back is straight, she grew almost 6 inches and she hardly ever chokes in her sleep anymore.
My greatest hope and what I am excited about in her future are intricately linked. Trofinetide is my greatest hope. *disclaimer: her trial has not been unblinded, this is my personal opinion* I’ve seen it at work. My daughter has felt it at work. Katelin has only love and admiration for Trofinetide and the people who work on it; it’s obvious every single time we do something like this, meet people involved in the trials, when we met Margaret, when we meet James Shaw and Dr. Nancy Jones of Neuren. She KNOWS it works and she’s waiting very patiently for it. Sometimes when she’s very upset, I tell her…when Trofinetide comes, this will be alright… and she calms right down.
What am I excited about her future??? College. I fully expect that Trofinetide will enable her to communicate/speak well enough to attend classes. She is going to get to LEARN what SHE wants to learn-space, biology, maybe even writing! (be still my heart )
After our interview there was a short Q & A; the one question that struck me the most was- We’ve had several parents here now and you all seem to have such joy, why is that? Well, I did joke that we were when we were there! (In my mind I was thinking- we get a great lunch and don’t have to clean up, it’s like a vacation!) However, for me, this is why I think we are able to find joy in the face of everything we deal with- we learn that the little things are the big things and the big things that most people worry about are nothing compared to those little things. You learn that showing your child a butterfly or smelling all the different flowers while you’re out walking keeps you young and reminds you of the miracles all around you. When a butterfly lands on you it can sustain you, because really, isn’t that a small miracle? Simply put we learn to be present in the moment and find the joy.
Recently, there’s been a disconnect in our community and I want to say, this experience proves to me that our hope is dependent upon all the different facets that parents promote: fundraising, research, awareness; even if it doesn’t always look like we think it will.
Without funding there is no research or trials, without trials there is no hope, and awareness isn’t needed just for those things to occur; it’s for people like the employees of Acadia- people who are going to work every day to save our children, who want to know why they should, who deserve to be told that we are grateful; people who deserve to see the children they are going to give a voice to; people who deserve to know that we are committed right alongside them to help our children, that we are not just waiting for others to be our saviors. Neuren and Acadia, among other companies, deserve to be made aware that yes they get a paycheck, but there’s a reward of a lifetime waiting at the end of trials- saving thousands of lives. Not just, hopefully, that they survive better but that they truly get to live.
Watch for: A Day with Acadia- Part 2; Assurance! Coming next week!~