Province of Ontario Neurodevelopmental Disorders (POND) Network

Evdokia Anagnostou, MD
Clinician Scientist, Holland Bloorview Kids Rehab Hospital
Associate Professor, Department of Pediatrics
University of Toronto
Canada Research Chair in Translational Therapeutics
The challenge
• > 300,000 children and youth in Ontario with
neurodevelopmental disorders (NDs):
– Autism Spectrum Disorders (ASD), Attention Deficit
Hyperactivity Disorder (ADHD), Obsessive
Compulsive Disorders (OCD) and Intellectual
Disability (ID).
• Lifetime cost per individual as high as $2 million
in Canada
• Ability to change long term outcomes remains
The challenge
• Few medications to treat these disorders
– Almost none have been discovered by translating the
findings from basic sciences such as genomics, animal
models, pathology etc.
• There are many reasons why development of novel treatments
for NDs has been limited:
– It is difficult to stratify patients into those most likely to
respond to the intervention
– Few molecular targets
– There are no established networks of excellence to
rapidly screen compounds and advance promising
• An integrated discovery system to expedite
– understanding biology
– Translation into novel therapeutics
• Across developmental disorders
• 4 research sites: HBKRH, SickKids, McMaster, Western
• >20 investigators, 40 clinicians
• >15 industry partners and advocacy organizations
The plan
• Create the first clinical trials network
dedicated to neurodevelopmental
disorders in Canada
– Existing expertise in NDs
– Enhancing regulatory expertise
– Go where the children are
The plan
• Embed the OCTN in a large biomarker core
– clinical database of children and youth with NDs
• properly characterize with behavior, cognition
• Characterize biology
– imaging and electrophysiology techniques
– Genomics (leveraged) and epigenomics
The plan
• In parallel:
– Develop animal and cellular models
• better understand disease mechanisms by crossanalyzing data from the human subjects with mouse and
cell modelling results
• enable testing of medications for response and safety
before they are used in children.
Database / Biomarker core
• Currently: >1000 children with ASD,
– Core technologies used for characterization:
• Detailed behavioural characterization
Stage 1: demographics
Stage 2 : cross disorder phenotyping
Stage 3: In depth diagnostic phenotyping
Stage 4: Cognitive phenotyping
• Genomics (leveraged)
• Imaging ~200 imaged children
• Electrophysiology (MEG, ERP/EEG, autonomic
nervous system markers)
Results: Electrodermal activity (EDA)
Results: Effect of anxiety
Future Directions
* As of summer 2014:
Specific commitment to:
Tourette Syndrome
Down syndrome
Fragile X
Rett syndrome
• NEW RFA for pilot studies, due Summer 2015
• New compound for next clinical trial secured: GSK3b
• Thank you

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