Mouse studies of a promising treatment for Rett syndrome, already in clinical trials, offer a note of caution about the drug’s potential. Preliminary findings from the research, presented Sunday at the Society for Neuroscience annual meeting in New Orleans, show why mouse work remains important even after clinical trials are underway.

Researchers are testing a wealth of new treatments for fragile X syndrome, an inherited intellectual disability often accompanied by autism. They presented preliminary results from animal studies of several drugs, as well as an automated approach for screening compounds, Sunday at the 2012 Society for Neuroscience annual meeting in New Orleans.

Deleting an enzyme that regulates protein synthesis reverses some of the molecular and behavioral deficits in a mouse model of fragile X syndrome, according to research published 2 October in Neuron.

A drug called arbaclofen improves behavioral problems in people with fragile X syndrome, an inherited condition that can lead to mental retardation and autism, according to the results of a clinical trial published today in Science Translational Medicine. A second study published in the same journal showed that the drug restores normal brain function in a mouse model of the disorder.

Despite the growing number of young adults with autism, few studies have looked at how best to support these teens as they transition into adulthood.

The first approved stem cell trial for autism will soon be under way. Is there a rationale for testing stem cells to improve symptoms of the disorder?
 

Losing one or both copies of TSC1, one of the two genes responsible for tuberous sclerosis complex, in specific cells of the cerebellum can trigger several autism-like behaviors in mice, according to research published 1 July in Nature.

A small clinical trial at the Seaver Autism Center at Mount Sinai aims to test whether an existing drug, the growth factor IGF-1, will help treat some of the core symptoms of autism in children with a specific genetic mutation.

A new initiative launched by the National Institute of Mental Health aims to redefine clinical trials for autism by funding short, biomarker-based studies that will allow investigators to quickly rule out ineffective compounds. 

A $38.7 million project in the European Union — the largest single grant for autism research in the world — aims to bring together academic labs and pharmaceutical companies to speed the move from basic to clinical research.