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New Drug Prove Effective in Treating Various Forms of Autism in Mice

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Published on : Nov 15, 2017

In recent years, autism spectrum disorder (ASD), commonly known as autism, has shown increased prevalence, notably in developed nations. A recent research into a widely available drug candidate in mice brings hope for patients with different forms of ASD. The new drug, identified as NitroSynapsin, a key compound of FDA-approved drug memantine for treating the Alzheimer's, could rectify the imbalance in excitatory brain signaling in mice, with varying degree of success.  A large international team of scientists collaborated for the three month-study on mice engineered in lab with a syndrome called MEF2C Haploinsufficiency Syndrome (MHS).

Previous genetic studies have confirmed that MEF2C acts as a key determinant in all autism-linked genes by switching on expression of genes responsible for mutations found in various types of autism-linked disorders in humans.

The researchers from various institutes include those from Center for Autism Research and Treatment (CART); the University of California San Diego School of Medicine; Scintillon Institute, San Diego; and Institute of New Drug Research and Jinan University College of Pharmacy, China. The study is published on November 14, 2017 in the journal Nature Communications.

Correcting MEF2C Protein Transcription Factor Responsible for Gene Mutation

The scientists found that mice with MHS showed impaired spatial memory, undue repetitive movements, and abnormal anxiety. In addition, they detected a host of problems in their brain signaling, which characterizes the excitatory/inhibitory signaling imbalance. The results showed that NitroSynapsin helps in correcting the imbalance and boosting the mice’s performance in relation to their cognition and behavior, with an effect on reducing epileptic seizures.

Further Clinical Trials Planned     

Some of the researchers are taking the study further by designing experiments for evaluating the efficacy of the drug candidate in mice with other forms of ASD. In addition, they are using stem cell research to create models of MHS using skin of affected children. Of note, clinical trials are being planned in collaboration with a biotechnology company.

The research results has expanded the potential of NitroSynapsin and the scientist believe that the drug in the not-so-distant future will be used for treating a number of neurological diseases.