There is currently no single drug treatment for autism. Many doctors treat autistic patients with a variety of psychotropic drugs geared at managing their perceived antisocial symptoms, but this is reasonably controversial, especially in children. A new drug targeted at restoring an electrical signaling imbalance in the brain is showing exciting success in mice and researchers hope to move into human clinical trials soon.

Autism spectrum disorder (ASD) is an extremely complex brain disorder affecting, in the United States, an estimated 1 out of every 68 children. The new research centers on a form of autism called MHS (MEF2C Haploinsufficiency Syndrome).

Back in the 1990s, scientists discovered that when there was a mutation in a gene called MEF2C a child went on to display significant autism symptoms. In mouse models the researchers identified the mutated gene as causing an excess of excitatory brain signaling, and this was thought to explain many of the cognitive and behavioral features of autism.

The latest study deployed a new drug called NitroSynapsin to reduce this excess brain signaling. The drug was tested on MHS mouse models for three months and the results were incredibly encouraging. The previously identified electrical signaling imbalance was improved and abnormal autism-like behaviors in the mice were reduced.

Signs of anxiety, abnormal repetitive movements, and impaired spatial memory, were all characteristics that were somewhat repaired in the mouse model experiments. Despite MHS specifically only accounting for a minor volume of overall autism cases, the researchers are confident these results should translate to a broad spectrum of autism disorders.

"Because MEF2C is important in driving so many autism-linked genes, we're hopeful that a treatment that works for this MEF2C-haploinsufficiency syndrome will also be effective against other forms of autism, and in fact we already have preliminary evidence for this," says senior investigator on the study, Stuart Lipton.

The drug is currently being tested in mice models for other autism disorders and in vitro testing on human cells are showing positive results. The next step for the team is to start paving the way for human clinical trials, so the reality of a drug treatment for autism may still be some time in the future, but the path might suddenly have become a lot clearer.

The study was published in the journal Nature Communications.

Source: The Scripps Research Institute