New autism drug shows early promise in trials

A new drug called NitroSynapsin has shown to be effective against autism in mouse models, according to a report published in the journal Nature Communications.

NitroSynapsin, which is intended to restore an electrical signalling imbalance in the brain found in virtually all forms of autism spectrum disorder, largely corrected electrical, behavioural and brain abnormalities in the mice.

The researchers had previously identified a gene called MEF2C as a potentially important factor in brain development.

They discovered that disrupting the mouse version of MEF2C in the brain, early in foetal development, causes mice to be born with severe, autism-like abnormalities.

Since that discovery in mice in 2008, other researchers have reported many cases of children who have a very similar disorder, resulting from a mutation to one copy of MEF2C (human DNA normally contains two copies of every gene, one copy inherited from the father and one from the mother). The condition is now called MEF2C Haploinsufficiency Syndrome (MHS).

The study’s senior investigator, Stuart Lipton, said: ‘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.’

‘This drug candidate is poised to go into clinical trials, and we think it might be effective against multiple forms of autism.’

The drug could have other uses, too. The researchers found that NitroSynapsin compound improves synapse function, meaning that the ability of the drug to improve network communication in the brain may eventually lead to its use in other neurological diseases such as Alzheimer’s.