May 2013 left many in the Fragile X community devastated when financial pressures led Seaside Therapeutics to stop developing an experimental drug, arbaclofen, for Fragile X syndrome (FXS).
Today arbaclofen rises again! Allos Pharma Inc, a late-stage pharmaceutical company that concentrates on developing effective therapeutics for neurodevelopmental disorders, is launching a new development program for arbaclofen in the treatment of Fragile X syndrome. To dive deeper into this exciting development, explore our official press release titled 'Allos Pharma secures exclusive license on arbaclofen in Fragile X syndrome.'
Allos Pharma was recently cofounded by Mark Bear, PhD, MIT Picower Professor of Neuroscience, and Randy Carpenter, MD. Dr. Bear has been a FRAXA Investigator since 1999 and is a member of FRAXA’s Scientific Advisory Board.
Many people in the Fragile X community felt that arbaclofen helped their family members, and a small group created a website, STX209Stories, which featured nearly 50 stories from families who had experienced positive results from the experimental drug. Parents printed out the stories and delivered them to Members of Congress with the hope of continued access the medication. One of them was Holly Usrey-Roos, whose experience was featured in the New York Times’ An Experimental Drug’s Bitter End. We spoke with Holly today:
I never expected to get a phone call telling me that arbaclofen, the drug that changed my son’s life in the best ways, would be back. Losing arbaclofen seven years ago was devastating. With it, we were able to do things just like everyone else: we could go to the public pool, we could go to the movies, and best of all, it led to my son talking. We fought to keep it but we couldn’t. We were forced to move on.
I’m sure I am not alone in believing everyone had moved on and we would never hear about arbaclofen again. But we were wrong! Dr. Mark Bear never gave up on it. He never gave up on our children, on finding a way to make arbaclofen available again.
I’ve cried a lot during this pandemic, but these are the first tears of pure happiness. This news not only brings back hope for my son and the Fragile X community but reminds me that the amazing people who are dedicated to Fragile X will never give up on us.
Holly Usrey-Roos
