3 1/2 years ago really wasn’t all that long ago, yet it feels like a life time.
3 1/2 years ago Parker left massive bruises on my body daily.
3 1/2 years ago Parker could rarely make it through a day of school without a complete meltdown.
3 1/2 years ago Parker couldn’t go to the movies.
3 1/2 years ago Parker couldn’t go to the pool.
3 1/2 years ago Parker couldn’t tell me about his day, his wants, his needs.
3 1/2 years ago Parker couldn’t tell me he loved me.3 1/2 years ago, I took a chance. I enrolled Parker in a drug trial by Seaside Therapeutics. A drug called STX209, later to be named “Arbaclofen”
While it may seem easy to make the decision to put Parker into a drug trial he clearly qualified for, it wasn’t. It’s scary as hell to make that decision. With this one, the decision was made a little easier because this drug was a tweaked drug of Baclofen, something that had been on the market for years. I knew the long term side effects. I knew, long and short term, it would not hurt him. So, we did it.
Every 2 weeks for 16 weeks we drove to and from Chicago, 4 hours each way, 8 hours round trip. Usually for a 15 minute check up. Sometimes for longer for cognitive testing, paperwork (that damn Vineland takes forever), blood draws, etc.
The first visit it took almost a full hour to get Parker from the van in the parking deck into the Fragile X clinic. I was not not only exhausted but covered in bruises when we finally made it in the door. While many people walked by as we sat on the hall floor together either in tears or pep talking or me trying to remove my arm from his mouth… this was normal to us. Nothing about this frustrated me or was out of the norm. This was just our life, this was the life we knew but were hoping to change.
The drug trial proved to be life changing. It was a double blind trial, nothing changed during the placebo time… everything changed when he went on the real thing. For the first time ever, my son said, unprompted, not mimicking but on his own “I love you mom” (you can read about it here). I held him so tight and cried. 10 1/2 years and worth the wait to hear it repeated over and over since that night.
The drug had received approval to go into an extension - because so many patients had the outcome of such incredible improvement. The extension was to last until we had FDA approval and the drug was available to everyone with the hopes of FDA approval in 2013 or 2014.
During this time, I traveled to Massachusetts and met the incredible and dedicated staff at Seaside Therapeutics. I worked with the press to show the incredible changes this drug made in Parker, tv, newspaper, magazines… all of it. If a phone call, letter or email was needed to the FDA or government, I was on speed dial. I made it happen. Parker was improving every day, even today… he still continues to improve. I often gave the example that Fragile X had left Parker living life in a box with the lid tightly sealed on. This drug took off that lid. It let him out of the box and no matter it took, no matter what I had to do, no matter who I had to call, email, travel to see – even if it meant stealing – I would never make him live life without this drug again. I would never let anyone put that lid back on the box with him inside. I made this promise to Parker.
I was asked during an interview, “Does Parker know that these pills help him?” Every morning and every night, the boy who used to fight taking his medicine asked for his pills. Did he know? Clearly. He knew.
In the past 3 1/2 years, I have watched my son blossom into an incredible young man who is now 14. A young man who still faces the struggles of Fragile X but to a much lesser degree. A young man who rarely ever physically attacks me and when he does, in moments of sheer despair, it is nothing compared to the aggression of his past. A young man who is incredibly active in school, significantly increasing his workload. A young man who can go to the movies, to the pool, to the store, out to dinner. A young man who has become part of our community in many of the same way his friends are. A young man who can tell me when he’s hurt, sick, scared, happy, excited, what he wants to eat, what he wants to do, when he is cold, when he is hot, who is friends are… a young man who can tell me he loves me.
I knew that Seaside was having trouble financially. We had seen staff go. We had been told there was financial difficulties. We were told if the study needed to end we would get 4-6 months notice. We never stopped believing we could make something work. Seaside never stopped believing they could make something work.
For 3 days this week, Parker is at Outward Ingersoll with his 8th grade class. He has been doing everything from canoeing, to learning how to tie knots, to first aid, to tracking to climbing walls and walking rope bridges. Today was day 2. For the 2nd day in a row, the young man who hasn’t been able to wear jeans since kindergarten has worn jeans to school (long pants were required for this trip). He has successfully with the help of his friends, been a part of every activity and successful. This is something he could not have done 3 1/2 years ago.
Today, as I am getting FB messages and texts with parents who are there witnessing his accomplishments, updates filled with pride, I never saw the email coming that said, “They are ending the STX209 trial immediately.”
That’s it. It’s over. Seaside will be analyzing the date from the placebo-controlled trials and will continue to work for FDA approval if these trial show benefit that is sufficient for approval. So, yes a tiny slimmer of a glimmer of hope but nothing that will happen right now. Nothing that will stop the medicine from ending in the next couple of weeks and in all honesty, from what I’ve learned of FDA drug trials/approval… I’m not holding my breath.
I am thankful to Seaside, the doctors at RUSH, the study coordinators -they have given me a real look inside my son for 3 1/2 years and I love what I see.
My heart is so broken, hope has been replaced with fear and pain. There is NO WAY I can explain to him why the pills will be gone. There is no way for me to stop what is about to happen. There is no way for me to prepare him, myself, his sister, his friends, our family for what could happen next.
I have to do what I swore I would never do again… put him back in the fragile x box and close the lid. I am so angry that I have dedicated so much time and heart into this… I have convinced other people to start the trial. I talked my best friend into putting her son on it. And now, not only do I have to see the pain of my family, the confusion of my son, the lid go back on his box… but I have to watch my friends go through it too. I have to watch an entire community of people I love and would do anything to make their lives better for hurt. All I can do is watch. I can’t fix it. I can’t take away the pain. I can’t do a damn thing. Nothing.
I am so scared of what the future now holds for Parker. I am pissed off that I know I will have to watch him regress. I will have to watch him live with the confusion of why I am not fixing this.
Parker starts high school next year. A new school. A new teaching team. And no drugs.
There are other clinical trials and I will look into them. That means driving back to Chicago every 2 weeks, 8 hours round trip, $100 in gas each time and a day off work. Not exactly in the budget of a single mom but something I will figure out if the right trial is available. A new trial with an outcome I cannot guarantee to be better. A trail of a newly formulated drug that I will have to go into not knowing how it will affect my son long term.
Tomorrow, tomorrow I will pull myself together and start discussing options. Tomorrow I will focus on hope again for the families impacted by this and a way to help them cope with the – what I can only foresee as devastation – that awaits us as we are titrated off the current drug and left with nothing. We have amazing teams at the Fragile X clinics who will work with us, help us find the next step in the path for our children. They are dedicated and want the very best. I believe in them to help guide me.
But today, today I cry and I can’t stop. I have tried. I need to cry. I hurt more than I can put into words. I need to cry. The fear of the pain of watching the regression that could take place is just overwhelming. The fear of losing the young man I’ve enjoyed so much for the past 3 1/2 years.
For the next few weeks, I will do my best to fight back my tears while Parker is awake. To treasure every single second with him where he can talk to me. To tell as many jokes with him as he can tolerate because he loves jokes more than anything. To talk about the Bulls, the Cubs and whatever sport team he wants until we’ve covered everything about them. Record every word in my memory and pray to God he doesn’t lose his ability to communicate.
My hope right now is that he has matured enough in 3 1/2 years and made progress that was credited to the drug but was really him. Time will tell.
All I can do now is wait… and pray that with this change I don’t lose the young man who was let out of that box. He shouldn’t ever have to have the lid put back on.
I promised him. I promised him I would never let that happen. I promised him.
I failed. I am so sorry. So very sorry.
Written By Holly Usrey-Roos
To learn more about Fragile X Syndrome visit the National Fragile X Foundation at www.fragilex.org