Translating Precision Medicine for Neurodevelopmental Disorders - Lynn Durham @Synchrony2022 |
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Neurodevelopmental disorders (NDDs) are a group of prevalent and highly heterogeneous conditions characterized by impairment in “personal, social, academic, or occupational functioning” with onset early in development. NDDs include Autism Spectrum Disorder (ASD), Intellectual Disability (ID), Attention Deficit Hyperactivity Disorder (ADHD), communication disorders, specific learning disorders, and motor disorders; moreover, the definition can also include some neuropsychiatric disorders such as schizophrenia and bipolar disorder, and other neurological disorders such as cerebral palsy or epilepsy.
The recent advances in genotyping and sequencing technologies have propelled the identification of risk/causal genes, which have pointed to remarkable genetic heterogeneity among and within specific NDDs, including ASD. According to the current diagnostic criteria based, an individual with ASD must show deficits in social interaction and communication combined with at least two of four subdomains of restricted or repetitive behaviors. However, ASD remains characterized by high heterogeneity in its behavioral manifestations and very complex genetic underpinnings, furthermore and despite significant progress in genomic, the molecular neuropathology in ASD overlaps with different neuropsychiatric disorders. All in one these data suggest the existence of subtypes of ASD. Therefore, efforts to categorize ASD and NDDS, must rely on defining a relationship between clinical symptoms and biological mechanisms to enable the emergence of biologically driven drug development and improve the outcome of clinical trials in the space. Along these lines, Database Endophenotyping Patient Identification (DEPI) technology was developed to enable the matching biologically defined populations of patients with neurodevelopmental disorders (NDDs) with tailored treatments, with a first application to ASD. DEPI uses systems biology and artificial intelligence to identify clinical and molecular characteristics underlying specific subgroups of patients. Application of DEPI led to the identification and clinical validation of two biologically defined subgroups of patients with ASD. This talk by LYNN DURHAM, CEO, and BALTAZAR GOMEZ-MANCILLA, CMO at Stalicla, was part of was part of Synchrony 2022 Online Symposium - 'From Bench to Biopharma', organised by the The BRAIN Foundation 🎥 For more Synchrony 2022 talks and highlights: https://www.youtube.com/playlist?list=PLDtO9h17tcWeoPMXLeBwJt106CncTePTj 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 Synchrony https://synchronysymposium.com/ is the first and only international symposium on translational research in #autism, that brings together academia, #biotech, pharmaceutical companies and #venture partners from around the world with the mission to improve health and quality of life of people with #autismspectrumdisorder. 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 🧠 The BRAIN Foundation https://brainfoundation.org/ is a 501c(3) non-profit. The founders of BRAIN envision a world where every child and adult on the autism spectrum is healthy, participates fully in education and employment, and has a better quality of life. It aims to catalyze research that results in evidence-based interventions for the disabilities associated with autism, and also results in better medical standard of care. To accomplish this, it funds impactful research through #philanthropy and our network of partners in the venture, corporate, and grassroots community. |