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.