Autism Consortium members publish in PNAS: Mechanism, treatment for Rett syndrome -- top cause au...( Autism Consortium Scientists Publish...),Health

Autism Consortium Scientists Publish Study Defining Mechanism and Potential Treatment for Rett Syndrome, Leading Cause of Autism in Girls

- Clinical trial to test molecule in humans is being planned - - Data in PNAS reveals therapeutic that could apply to other forms of autism -

BOSTON JANUARY 9, 2009 The Autism Consortium, an innovative research, clinical and family collaboration dedicated to radically accelerating research and enhancing clinical care for autism spectrum disorders (ASDs), announced today that several Consortium members published a paper with significance for clinical trials in autism in the Proceedings of the National Academy of Sciences.

The research led by Autism Consortium members Mriganka Sur, PhD, Newton Professor of Neuroscience at the Picower Institute and Head of the MIT Department of Brain and Cognitive Sciences; and Rudolf Jaenisch, PhD, Founding Member, Whitehead Institute and Professor of Biology at MIT, demonstrates for the first time a mechanism for Rett Syndrome and a therapeutic that could be directly applicable to humans. As a result, a clinical trial in humans is in development.

This groundbreaking study demonstrated that by treating mice with a peptide fragment of IGF1, a molecule that is utilized by the brain for neuronal and synaptic development, the symptoms of Rett Syndrome in the mice were largely reversed.

"The next step is to test recombinant human IGF1 which is already available for pediatric use in humans with the hope of treating or reversing Rett Syndrome," said Omar Khwaja, MD, PhD, Director of the Rett Syndrome Program at Children's Hospital Boston and head of the clinical trial team for IGF1. "We are working as quickly possible to develop the protocol, secure funding, and initiate the trial."

"This new study presents promising novel data suggesting that targeting t
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