Managing Editor's Note: The drug in question called arbaclofen, was in clinical trials for both Fragile X and autism. To learn more and to read about family stories, please visit stx209.com
A Plea for Help: Fragile X Stories
By Melissa Welin
Most pharmaceuticals represent years of research, millions of dollars of investment, and the tacit promise made by the government that what is in the bottle is safe and effective.
Most pharmaceuticals also represent the personal stories of patients and their families; stories that are often filled with suffering, struggle, but most importantly hope.
Of course, we understand the responsibility of the FDA to protect the public.
Of course, we understand that pharmaceutical companies are responsible to shareholders and have a complex formula to determine how they allocate resources.
But sometimes, amidst shareholder meetings, bottom lines and risk aversion, our personal stories are lost. Sometimes, we have to remind those who control access to promising treatments that there are lives at stake… that balanced against their ledger is our precious, fragile hope.
Recently, Roche Pharmaceuticals was involved with a Cambridge, Massachusetts-based 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 (the most common known single gene cause of autism) and Phase II trials for children with ASD (which has had positive results).
When Roche pulled back on R&D investment, it ended its involvement and financial support for STX209. Without Roche’s backing, Seaside Therapeutics was forced to issue the following statement on May 15, 2013 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 June 7, 2013, Phase III trial participants will no longer have access to STX209. The abrupt end of the study will leave our children in danger of significant regression.
For many of us in the study, arbaclofen has been that miracle for which we had hardly dared hope. A child, who had never wanted to be touched, opened his arms for a hug. For the first time, a five-year-old said, “Mommy”; a ten-year-old said, “I love you.” Children were making friends, learning to read and write. We started to believe in the possibility of a “normal” normal.