We recently reported on a drug trial that had been discontinued do to lack of funding when Roche Pharmaceuticals terminated its agreement with Seaside Therapeutics. The STX209 families who were in the trial published an open letter to the CEO of Roche and received a response via Twitter. The trial was for a drug known as STX209/Arbaclofen. The study was to include both Fragile X Syndrome and autism. We realize that Fragile X is a very different diagnosis from autism in that it is testable and confirmable. There is some overlap in presentation and there were autism families in the study. While we tend to be leery of pharmaceutical options, for those families for whom this drug was improving their children's lives in the short term - we hope they find a way to continue the study.
Here is an excerpt of the letter:
An Open Letter Regarding STX209 to Dr. Severin Schwan, Roche Pharmaceuticals
Dr. Severin Schwan
Chief Executive Officer
F. Hoffmann-La Roche AG
June 10, 2013
Dear Dr. Schwan:
Behind every drug Roche Pharmaceuticals makes are the personal stories of patients and their families. You must hear these stories everyday, and we cannot imagine the pressures you face determining where to put your resources and whose lives – whose children’s lives – you will try to save. We understand you cannot support every drug and every trial, but you are a father, so surely you will understand our desperation and need to reach out to you directly to help our children. Like most parents, we are certain you would go to any lengths – contact any person – if your children needed help; so you’ll understand why we are writing you this letter.
Recently, Roche Pharmaceuticals was involved with a Cambridge, Massachusetts-based pharmaceutical company called Seaside Therapeutics, Inc., which has been developing a drug called STX209 (Arbaclofen). This compound has been in Phase III trials for children with fragile X syndrome.
When Roche pulled back on its R&D, it ended its involvement and financial support for STX209. We understand there have been issues with the therapy’s trial results, but it had statistically relevant improvements to secondary outcome measures. Luca Santarelli, head of neuroscience research at Roche has been quoted as saying “We concluded that arbaclofen wasn’t going to provide that real difference for patients.” Dr. Schwan, we respectfully disagree, the improvements we have seen in our children have changed the lives of our families.
Without Roche’s backing, on May 15, 2013, Seaside Therapeutics was forced to issue the following statement to the clinics involved in the administration of Arbaclofen:
We regret to inform you that Study 209FX303 [An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of STX209 (Arbaclofen) in Subjects with Fragile X Syndrome] is being terminated immediately. The closure of the study is due to resource limitations at Seaside Therapeutics, Inc., and is not related to any known safety issues in patients dosed with STX209.
As of July 1, 2013, none of our children will have access to STX209. The abrupt end of the study have left some of our children to suffer withdrawal symptoms. But that is hardly a consideration compared to what is about to happen to us all. We’d like to share with you the thoughts of just a few of the families in the trial.
From Cortney AbouElSeoud, mother of Ayden, age 5:
“A little background first. Ayden had a 5-word vocabulary, but seldom spoke. Within one week of treatment with Arbaclofen, his tantrums diminished noticeably. Within a month, he started using words spontaneously. Now seven months into treatment, he has surpassed development goals in every area. He speaks in small sentences, can isolate the sounds of the alphabet and can trace all his upper and lowercase letters. On Arbaclofen, he could enter first grade on par with his peers.”
“I’ve often heard fragile X described as having the sound of a vacuum in your head. Consider going through life daily with that sound and attempting to speak or to walk into a large room full of people. Imagine how difficult it would be and how easily you could become frustrated. Imagine then that there was something you could do to shut that noise off. That is how I look at Arbaclofen…. Now we have to turn that switch back on. Devastated does not even begin to describe the emotions I am feeling. Terrified. Worried. Overwhelmed…. And the tears. Because they can’t find enough money to finish the trial, I am facing a reality where I may never again hear my son say “mommy.”
Read the full letter and learn more at STX209 Fragile X and Autism Families. The treatment category is sponsored by Lee Silsby, the leader in quality compounded medications for autism.