Wednesday, May 15, 2013

3 1/2 years... When a drug trial ends...

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 (who has Fragile X Syndrome) was 10 1/2.

3 1/2 years ago Parker could barely talk.
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 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  ... or 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 - 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?  Yes, 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 they 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 data 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.


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 possibly 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.  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 and fragile x family 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 stop this from happening.

I am so scared of what the future now holds for Parker.  I am so angry 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 in August.  A new school.  A new teaching team.  And no trial drug.

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. 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 to go on without it.  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, to guide us.

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 at keeping that promise.  I am so sorry.  So very sorry.


To learn more about Fragile X Syndrome visit the National Fragile X Foundation at www.fragilex.org
*Fragile X is the leading cause of inherited intellectual disability and the leading known genetic cause of Autism.



35 comments:

  1. Holly, I just want to give you a hug right now. I wish so much I could do more than that. This isn't fair to Parker, your family, or you. This isn't fair to so many that have seen such improvement and hope. Do me a favor, though. Don't blame yourself. You made that promise because you were given something that brought about great promise itself. While the promise of the med trial may be falling through, you are still there to love Parker through whatever happens. Your first promise was to love and support him and do what was best for him. You have done that, and are continuing to do that. You have not broken that promise, nor will you ever. I hope and pray that someone, anyone, will see that the benefits of this drug are far outweighing any risks, and that the demand is great. I hope this is only a bump in the road for something great to happen. I am going to keep that hope alive for you. My greatest amounts of prayer, hope, and love surround you tonight. Kimi has told me of his improvements. I just can't fathom this.

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  2. That is horrible. Do you know the reason they are doing it? Your above story is worth spreading. Is there someone important you can send it to? If anyone can change things it's you Holly, maybe you'll find a similar drug... I don't know what to say. I am so sorry.

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  3. They are broke. They no longer have the funds to make the drugs or continue the trial. I am hopeful we can figure out a solution..or that this has happened for a reason and that reason is there is something better.

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  4. Thank you both... I want so badly to make everything better for everyone, especially Parker. Thank you for helping to get the word out!

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  6. Holly, someone made my organization aware of your crisis and your blog. We are a social work run news organization and we try to help where we can. We can republish your blog on our site and it will it be indexed on Google news, or we can just create a story with a link to this page. We can also spread the word through social work circles, we have 21K followers on facebook. Email me , matt@socialjusticesolutions.org and we will do what we can. My heart goes out to you, but do not give up hope!

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  7. Thank you Matt! I will email you!

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  8. Holly, I, too was part of a drug trial that was halted for financial reasons. My neurologist told me first, as they knew I'm a social worker that would fight for myself and the other 13 patients. I did. We appealed being removed from being able to obtain Banzel. Our test study was with Banzel, which is a seizure medication approved only for Lepsten-Barr children. Our group, all 14 of us, began the study in February, 2008, and by June 20, 2008 my seizures had halted. Not slowed ... STOPPED. This was the same story for all of us.

    I wrote the appeal which the doctor's office posted to the manufacturer that day. It was denied. We then did a request for review, which was also denied. Then, we filed an exception with the FDA, stating information on all of us that were on Banzel. In that statement, I told them that I had been an uncontrolled epileptic for 32 years, and this was the only drug that had ever stopped my seizures. I emphasized that if Banzel were to be stopped, in six months my seizure would return and my social work degree would be useless as I would be unable to work. All the other participants posted similar statements.

    A ruling by the FDA was issued that all participants in the test study would be allowed to continue on the medication. It has now been 4 years and 10 months since I had a seizure. Please, see if you can appeal this medication being halted on your son. I can't promise that they will agree, but the worst thing that could happen is that they say no. The best thing ... could be amazing.

    If you want to talk ....

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    1. That is incredible! I would love to talk and find out how to appeal the medicine being halted. I know there are many families who would join me in the process. Please let me know the best way to reach you! Thank you!!

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  9. We are kinda in shock here too. I wish I could say I had the dramatic changes that you saw with Parker but I can tell you that I have a fear...a fear of if we are off the meds now what...How much was it helping in little ways or big ways...I can not believe it is just done. I have been doing this with skyler for about as long as you and it is a lot of effort, time and $ to get to these appointments...it is usually a 12 hour day for us. Now feels like we are just thrown back out to survive.

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  10. I also just took him off tons of his other meds cocktail...and he was doing ok...now one more to be gone...So scary

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    1. ((Hugs)) Elaine, it is scary. We are going to do everything that we can. We are all in this together.

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  11. Are you going to try the old product Baclofen which was FDA approved in 1977 and now available as generic? The dose will be higher that arbaclofen, but maybe it will work just as well.

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    1. Right now that will be our first step, unfortunately the results of those who have done this have not seen the same - or similar results. There is just enough of a difference in the 2 compounds that makes one effective and one not.
      That said - it's still something we will be trying with the hope that Parker is one that is works for.

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  12. https://www.change.org/petitions/pharmaceutical-companies-sponsor-arbaclofen-stx-209-for-chidren-with-autism-and-fragile-x

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  13. You are welcome! Holly is there a picture that we can use for this petition? I'd love to associate a pic of you and your son as I included your blog post in the petition which has almost 150 signatures. If it gets enough signatures and attention, it gets high placement on change.org and will include the visual... If you have anything, can you link it up here and I can post it w/ the petition.

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    2. I just signed the petition on change.org. But if you're petitioning the president, have you considered a petition directly to the president? https://wwws.whitehouse.gov/petitions Every petition that gets enough signatures, the Administration guarantees a response. Also, have you involved the executives at Seaside Therapeutics in your petition or your cause? You'll need them on board.

      Seaside received $30 million from an anonymous donor a year ago, so they have support, if the issue is purely financial.

      I have a PR background and am happy to help, if I can. I've worked with companies that have appealed to the FDA. You should go that route, but you should do it by getting as much publicity for your issue as possible. Start by talking to your local papers, but as this story has national implications, I suspect you could find national media who would cover it. Your story really touched me. meghanthompson@me.com

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    3. Thank you so much - we are all trying to figure out what to do - I'll email you asap!! Thank you!

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  14. We just got the news, too. Our 7 year old son has made tremendous strides in language and ability to calm. I'm not sure what to do now, but I think we need to appeal directly to the FDA. There is a tradition of patient rights activism that goes back to the early days of AIDS drug trials that we might want to look at for ideas. This is horrible! I suspect it results from an overreaction to the negative trial results for the autism study, which probably led to venture capital being withdrawn (that's just a guess). That is something that should be targeted as well. Please keep us posted and let's see what we can come up with.

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    1. I will Bill. I am sorry you are going through this too. It is gut wrenching. I am hoping to have more information on the appeal process soon! I will definitely share, it will take ALL of us together to make this happen.

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  15. So sorry to hear your news. Not sure if it is an option (or if it is different from the appeal process other users mentioned), but perhaps try for applying for Expanded Access (aka Compassionate Use) through the FDA:

    http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/AccesstoInvestigationalDrugs/ucm176098.htm

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  16. PJ - THANK YOU!!! I will look into this asap!!

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    1. HOlly, this is exactly what i was going to suggest. Learned about it on NBC Rockcenter. Here is a general quote: Dr. Timothy Cote, former director of the FDA’s division for drugs that treat rare or so-called orphan diseases, explained the FDA’s policies in general.

      Cote says this is a process for patients who will die without a medication. It’s called “compassionate use,” and requires doctors to get special approval from the FDA on behalf of their patients.

      given at hospitals with special dispensation from the FDA, a provision called “compassionate use.”

      I sure hope they will listen to you and the other families with this disorder!!
      Good Luck!!

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  17. Holly thank you for sharing this. I'm crying along with you. I pray that Parker and all of the others are not silenced. You have not failed him. You gave him a gift. Praying and holding you from afar.

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    1. Thank you, so much. We are going to fight, I refuse to let my promise be broken. Thank you so much for the support and prayers!

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  19. Holly, I know a someone earlier suggested to try generic baclofen as an alternative. Perhaps you are already aware, but baclofen is just the racemic mixture of equal parts R-baclofen and S-baclofen (the two different enantiomers or mirror images of the molecule). Arbaclofen is just the pure R enantiomer version of baclofen. Theoretically, taking an approximate 2-fold higher dose of baclofen would be comparable to a dose of arbaclofen given (you'll have to talk to your doctor). Side-effects and safety issues may be a concern with having to using higher doses of baclofen to achieve therapeutic effects. You mentioned others have not had similar successes with baclofen compared to arbaclofen, at least anecdotally, but I'm not aware of any controlled studies to indicate as much.

    Basically, I think this is an option definitely worth looking into, especially given your son's positive response to arbaclofen. Best of luck!

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    1. I agree, it will be our first option. It wasn't a controlled study of our subgroups, it was the recommendation for those who couldn't get into the trial. It could have been many things - too low of dose, frustration from not being in the trial, etc that left them feeling that this did not work.
      Even if it has a small improvement - it's still an improvement and something I feel we have to try.
      Thanks for sharing that!

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  20. Hi again, Holly. A good friend of mine is a prominent advocate in the autism community (and author of Cutting Edge Therapies for Autism - in which I wrote about this drug and Seaside Therapeutics). Ken Siri recommends you contact The Canary Party, National Autism Association, Autism One. They are apparently excellent advocates in situations like yours. Very best wishes, Meghan

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    1. Thank you very much! We will do that!

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  21. I just read your story... tears came to my eyes.

    I'm an organic chemist myself and work on drug development. I've never considered focusing on fragile X before, but hearing your story... well it has made me want to see if I can help

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  22. Nick - Thank you so much - that means so much to me and the fragile x community. If there is anything I can do to help, please let me know.

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