A new day dawning? First signs of promise for a drug that treats the core symptoms of autism
As many parents know, there currently are no available medical treatments for ASD targeting core autism symptoms. Available medications target symptoms associated with ASD, such as hyperactivity, irritability, anxiety, or depression. Although the available medicines have helped many who struggle with the challenge of these symptoms, these drugs do not address the difficulties in the areas of social and communication deficits or repetitive behaviors and restricted interests.
Recently, hope has recently been kindled in a new drug for ASD that developed out of basic research on the neural mechanisms of Fragile X syndrome. Back in 2005 research in Dr. Mark Bear’s lab at Harvard showed the Fragile X mutation affects communication between neurons. Specifically, the mutation results in an excess of an excitatory neurotransmitter called glutamate, which impairs communication between neurons by making them over-stimulated. Seeing the potential to help families, a small company called Seaside Therapeutics was started to see if certain drugs could help reduce the level of excitability of neurons.
The drug, arbaclofen, is the first drug being tested. Arbaclofen works by increasing GABA, an inhibitory transmitter, which counteracts the over-excitability of cells. The preliminary results of a trial conducted with children with Fragile X syndrome looked so promising that Seaside Therapeutics announced the results on this year’s meeting of the International Society for Autism Research (read a review of that meeting’s highlights). More recently, arbaclofen has been tested in children with ASD without Fragile X. The results of this trial have been reported in the news. The trial treated 25 children and adolescents with autism for 8 weeks and the preliminary data revealed that arbaclofen was not only well-tolerated but also increase sociability and eye contact, and reduced tantrums and anxiety.
Of course, the testing of this drug continues and a review of the data by independent scientists is essential for evaluating the true benefit of this new drug, however these preliminary results offer good reason for hope and that news is always worth sharing.