Arbaclofen is a drug that has not yet been approved by the FDA. It appears to treat social withdrawal symptoms in people who have Fragile X Syndrome. There is hope that perhaps this drug could also help people who have autism with social difficulties.
Arbaclofen is also called STX209. It is the first drug that is designed to treat one of the symptoms of Fragile X Syndrome. More specifically, arbaclofen helps with the social withdrawal that many children who have Fragile X Syndrome experience.
Fragile X Syndrome is a genetic condition resulting from a chromosomal abnormality. It results in an X chromosome that is not properly formed. The syndrome affects more males than females.
A faulty X chromosome is passed from mother to child. Often, a person has no idea that she is carrying a defective X chromosome until after her child has been diagnosed with Fragile X Syndrome.
Symptoms of Fragile X Syndrome include intellectual and social difficulties that are similar to some of the symptoms of autism. Arbaclofen is an experimental drug that has been tested in a small number of patients. It has not yet been approved by the Food and Drug Administration (FDA). So far, the results of the testing show that the drug makes it easier for people who have Fragile X Syndrome to connect with other people in social situations.
Right now, there are no FDA approved medications that treat help children who have Fragile X Syndrome to improve their social skills. Treatment currently consists of intensive behavioral therapy, (similar to what children with an autism spectrum disorder may receive). There is hope that someday arbaclofen might be prescribed to children who have autism to help with social difficulties.
The trial was funded by Seaside Therapeutics. They are the company that is developing arbaclofen. There were 27 people in the study who had what is described as severe “social avoidance” problems. Out of that group, about 40% were “much improved” or “very much improved”, according to standardized tests. A larger, more definitive trial, is currently enrolling patients who have Fragile X Syndrome.
It is worth noting that arbaclofen was developed by scientists who were studying the fundamental genetic problems in Fragile X Syndrome. The drug is designed to restore a balance of neurotransmitters in the brain. The neurotransmitters are chemical messengers that assist in the communication between cells.
Image by Purple Penning on Flickr