Autism drugs on the way, but be careful

Dr. Manny

According to recent news reports, pharmaceutical giants like Roche, Novartis, and Seaside Therapeutics are racing to make medications that will relieve some of the behavioral problems caused by autism and Fragile X syndrome.

The first thing that needs to be cleared up is that autism – or the autistic spectrum, which might include pervasive developmental disorders or Asperger syndrome – is different from Fragile X syndrome.  Fragile X is an inherited intellectual disability in boys that is caused by a change in the gene FMR1 on the X chromosome.  The condition results in the failure to express FMRP – a protein needed for normal neural development.  While Fragile X syndrome has similar symptoms to autism, it differs in that it has a very well described genetic underpinning.
The reason this is important is because medications for the treatment of autism symptoms need to be carefully studied and individualized to specific spectrum disorders. If not approached in the right way, we may end up with medications that are perhaps not indicated properly and abused down the line.
A specific medication that has gotten everyone excited is the drug arbaclofen.  This drug has already been tested on small clinical trials, mostly in patients with Fragile X syndrome.  Researchers have seen some improvement in the behavior of trial participants – in particular, a decrease in the anxiety that some of these patients have.