Positive signs for potential autism treatment

A potential drug therapy for neurodevelopmental disorders discovered at the University of Auckland has shown positive results in pre-clinical trials for Phelan-McDermid syndrome. This rare genetic condition involves the same gene thought to be associated with autism.

Dame Professor Margaret Brimble

Neuren Pharmaceuticals, which develops new therapies for neurodevelopmental and  neurodegenerative disorders, says there have been positive effects in early laboratory testing of NNZ-2591 for Phelan-McDermid syndrome, a rare genetic disorder characterised by intellectual disability, delayed or absent speech, motor delays and epilepsy along with symptoms associated with autism.

NNZ-2591 is a compound developed as a potential drug target by Dame Professor Margaret Brimble of the University of Auckland and Maurice Wilkins Centre for Molecular Biodiscovery. Early work by Dr Jian Guan and Sir Peter Gluckman identified the neuroprotective properties of the peptides involved. Peptides are fundamental components of cells that carry out important biological functions.

The testing was done with mice, targeting a gene called SHANK3 which codes for the shank3 protein which supports the structure of synapses between nerve cells and the brain.

The gene is thought to be associated with some cases of autism spectrum disorder. NNZ-2591 has also demonstrated efficacy in pre-clinical models of Parkinson’s disease, stroke, traumatic brain injury and memory impairment.

“The latest pre-clinical tests for McPhelan-McDermid are a very positive sign and hopefully further research results will mean that testing can be taken the next step,” Dame Professor Brimble says.


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