£40m boost for research into neurodevelopmental disorders
A major US philanthropic foundation has announced a multi-million-pound investment in the University of Edinburgh to better understand autism and associated neurodevelopmental disorders.
The Simons Foundation has pledged up to £40 million over the next 10 years for researchers at the Simons Initiative for the Developing Brain (SIDB) to continue pioneering studies into the biological mechanisms that underpin changes in brain development associated with neurodevelopmental disorders, including autism.
Researchers say the investment could eventually lead to new treatments for people with severe neurodevelopmental disorders, such as Fragile X syndrome (FXS), SYNGAP1 haploinsufficiency and CDKL5 deficiency disorder.
Brain research
Autism affects approximately 75 million people worldwide. People with single gene causes of autism often have co-occurring conditions, including epilepsy, sleep disorder, mental health problems and intellectual disability.
The SIDB was founded in Edinburgh in 2017 following a generous donation from the Simons Foundation.
Since then, more than 40 principal investigators across several research sites have used advanced techniques to investigate how DNA changes known to cause autism affect brain development.
Experts have investigated how variations in the brain’s wiring can affect how it processes information, which ultimately determines our intellectual and social abilities.
The SIDB's fundamental research is guided by the philosophy that improving our understanding of the brain will lead to better therapeutic options for people with these neurodevelopmental disorders. We are extremely grateful for this support from the Simons Foundation, which will have a significant and lasting impact on our fundamental and clinical research programmes.
Professor Peter Kind
Director of the SIDB
Previous success
The initiative’s commitment to basic research led to a promising treatment for Rett syndrome – a rare neurological disorder similar to autism that causes loss of motor and language skills. Groundbreaking work showed that even advanced symptoms in mice could be improved by restoring a key gene, MECP2, which challenged the idea that effective treatment is only possible during early brain development.
Building on this discovery, researchers developed a gene therapy for Rett syndrome that is now showing promising results at low doses in an ongoing clinical trial.
Scientists are also studying how SYNGAP1 gene mutations affect how the brain processes visual information. Those with SYNGAP1 mutations can experience epilepsy, developmental delays, movement disorders and autism.
In mouse models, they found that the mutation makes it harder to distinguish visual patterns and requires more repetition to learn. This research identified guanfacine – an existing ADHD medication – as a potential treatment. A clinical trial is now being developed to test its effectiveness in people with SYNGAP1-related conditions.
We are deeply grateful to all at the Simons Foundation for this vote of confidence in the work of Peter Kind and his team. This project area is emblematic of the University’s over-arching goal to make the world a better place. Our partnership with the Foundation is a shining example of the way in which far-sighted philanthropy accelerates scientific progress and deepens long term impact for local and global patient communities.
Professor Peter Mathieson
Principal and Vice-Chancellor of the University of Edinburgh
We are proud and delighted to support Peter Kind and his colleagues at SIDB. Their dedication to advancing the basic science of autism and neurodevelopmental disorders, their strong emphasis on collaboration, and their leadership in open science embody the core values the Simons Foundation aims to support in its interdisciplinary collaborations.
Kelsey Martin
Executive Vice President, Autism and Neuroscience at the Simons Foundation
