10 Feb 2009 07:00 AM
Autism Consortium Members Publish In PNAS: Mechanism, Treatment For Rett Syndrome -- Top Cause Autism Girls
Clinical trial to test molecule in humans is being planned
The Autism Consortium, an innovative research, clinical and family collaboration dedicated to radically accelerating research and enhancing clinical care for autism spectrum disorders (ASDs), has announced 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.
IGF1 Reverses Rett Symptoms in Mice; Clinical Trial Planned
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 the IGF1 signaling axis may present a useful therapeutic strategy that could ultimately be translated to humans," said Dr. Antony Horton, Chief Scientific Officer at the International Rett Syndrome Foundation. "We are encouraged by this collaboration between scientists and clinicians which is yielding valuable insights into potential new treatments for Rett syndrome."
About Rett Syndrome and the Findings
Rett Syndrome, a neurodevelopmental disorder mainly affecting girls and also the most common basis of autism in girls, is primarily caused by a sporadic mutation in the MECP2 gene on the X chromosome…
The Autism Consortium, an innovative research, clinical and family collaboration dedicated to radically accelerating research and enhancing clinical care for autism spectrum disorders (ASDs), has announced 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.
IGF1 Reverses Rett Symptoms in Mice; Clinical Trial Planned
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 the IGF1 signaling axis may present a useful therapeutic strategy that could ultimately be translated to humans," said Dr. Antony Horton, Chief Scientific Officer at the International Rett Syndrome Foundation. "We are encouraged by this collaboration between scientists and clinicians which is yielding valuable insights into potential new treatments for Rett syndrome."
About Rett Syndrome and the Findings
Rett Syndrome, a neurodevelopmental disorder mainly affecting girls and also the most common basis of autism in girls, is primarily caused by a sporadic mutation in the MECP2 gene on the X chromosome…
