Arbaclofen shows promise as a treatment for Fragile X syndrome and autism.
An experimental drug called arbaclofen has helped improve outcomes in patients with Fragile X syndrome. Fragile X syndrome causes cognitive and social dysfunctions that are similar to autism. Researchers are hoping this treatment may have broader applicability, though that remains to be seen. From NPR:
An experimental drug that helps people who have Fragile X syndrome is raising hopes of a treatment for autism.
The drug, called arbaclofen, made people with Fragile X less likely to avoid social interactions, according to a study in Science Translational Medicine. Researchers suspect it might do the same for people with autism.
Arbaclofen appears to work by tamping down overactive brain signals that can make it hard to navigate social interactions.
There’s a good chance it will help people with autism unrelated to Fragile X because they have similar problems with social interactions, says Mark Bear, a researcher at MIT and a co-founder of Seaside Therapeutics, which makes arbaclofen.
“It would be, I think, unrealistic to expect that this drug would be uniformly beneficial to all people that have an autism diagnosis,” Bear says. “But I think we can still be quite optimistic that it can be beneficial to a subset of those patients.”