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Sackler Institute

The Dr. Mortimer and Theresa Sackler Foundation, supports the advancement of the public both in the UK and overseas in the fields of art, science and medical research. 

The Sackler Institute was launched in 2013 and carries out multidisciplinary ‘translational’ studies of normal development, and how abnormalities in that process lead to high cost neurodevelopmental disorders. Our studies link the ‘basic’ sciences (e.g. in cellular mechanisms and circuits studied using stem cell and animal models) to clinical sciences. 

This is achieved by linking the work of the Sackler Institute to the new MRC centre for Neurodevelopemental Disorders and to our Scientific Steering Committee.  This includes experts in cellular mechanisms (Prof Jack Price), circuit development (Profs Oscar Marin and Juan Burrone), neonatology (Prof David Edwards), paediatrics and epilepsy (Prof Deb Pal), paediatrics and sleep (Prof Paul Gringras), cognition (Prof Francesca Happe), and child psychiatry (Prof Emily Simonoff). 

Training 

The Sackler Institute currently has 22 PhD students who link with our scientific steering committee.  We run an annual competition for new positions, and some of these link to our new MRC Centre for Neurodevelopmental Disorders. 

In addition, we run an MSc in clinical neurodevelopmental sciences. This provides the basis for some of our students to ‘transition’ to clinical careers or to subsequently pursue more basic science options during later PhD positions. 

Funding 

We lead large-scale research initiatives that link our Institute to others across the UK, Europe, North America, and Australasia. For example, we lead the EU innovative medicines initiative on autism (EU AIMS) that brings together 17 academic and industrial partners across Europe and links to biomarker consortiums in the USA, Canada, and Australia. 

Our work is funded by the original generous donation of The Dr. Mortimer and Theresa Sackler Foundation together with funding from the Medical Research Council (UK), National Institute for Health Research (NIHR), The Wellcome Trust, The National Institutes of Health (USA), Autism Speaks, Autistica, The European Commission (the Innovative Medicines Initiative for EU AIMS, together with other funding for EU BRAINVIEW, EU TACTICS, and EU MATRICS), the Simons Foundation (USA) and Industry (Shire, and GW Pharma). 

The challenge 

There are no effective pharmacological treatments for the core symptoms of many neurodevelopmental disorders, and our understanding of the pathophysiology of most is poor. Research in many disorders is hampered by a lack of valid and reliable cellular assays and animal models; an absence of tests that demonstrate efficacy from childhood to adulthood; and the reliance of clinical trials on biologically heterogeneous groups of patients as operationally-defined by DSM/ICD10 categories.  

Despite these limitations, the recent identification of genetic risk factors (e.g. copy number variants in autism, ADHD and intellectual disability) and molecular mechanisms (e.g. abnormalities in mGluR5 signalling and glutamate metabolism in Fragile X syndrome; and abnormal myo-inositol in Down syndrome) provide unique opportunities to develop new personalised medicine approaches for individuals with neurodevelopmental disorders across the lifespan. 

Our aims 

The Sackler Institute for Translational Neurodevelopment aims to transform our understanding of how disorders such as autism spectrum disorder, epilepsy and Down syndrome develop over a lifespan, to improve diagnostic approaches and to develop innovative treatments for these disorders. 

  • Advance our understanding of the origin, biological mechanism and lifespan evolution of challenging neurodevelopmental disorders 
  • Develop and evaluate novel diagnostic and treatment biomarkers to create individualised medicine 
  • Discover and test novel psychopharmacologic, behavioural and bioengineering interventions 
  • Pioneer genetic and cell therapies and offer state-of-the-art genetic counselling 
  • Foster interdisciplinary research training of postdoctoral students and clinicians 

Solutions via the Sackler Chair 

We propose to: 

  • Develop novel cellular assays and animal models based on confirmed genetic risks, and utilise these as translational endophenotypes for facilitating new drug discovery; 
  • Validate biomarkers as treatment targets and stratification tools for clinical trials; and 
  • Develop a sustainable clinical research infrastructure to test new treatments.