Clinical Translational Core (CTC)

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CLINICAL TRANSLATIONAL CORE ABSTRACT The objectives of the Clinical Translational Core (CTC) of our IDDRC are to accelerate the translation of research discoveries into new treatments for neurodevelopmental disorders, through collaboration with basic scientists and clinicians, as well as to train future leaders in translational neuroscience. Services offered by the Clinical Translational Core to support translational work include three sub-cores: 1) Patient-based Preclinical Services, comprised of a Biorepository and the induced pluripotent stem cell (iPSC) based Human Neuron Unit, 2) Clinical Phenotyping Services, consisting of the Neurobehavioral Unit and the Neurophysiology Unit, and 3) Clinical Trial and Regulatory Services, containing the Regulatory Unit and Research Participant Registry. The suite of services offered by the CTC enables investigators to pursue an integrated approach to therapeutic discovery for neurodevelopmental disorders, from patient derived samples in preclinical research to biomarker development to natural history and interventional clinical trials. Sub-core 1 - Patient Based Preclinical Services - maintains the regulatory approval to collect patient samples that can be used for primary research or for the generation of iPSC lines and neuronal differentiation within the Human Neuron Unit. Sub- core 2 - Clinical Phenotyping Services - provides expertise and access to high density EEG equipment as well as direct phenotyping through neuropsychological assessment and neurophysiological recording. Sub-core 3 - Clinical Trial and Regulatory Services - provides expert consultation in the regulatory framework supporting clinical research which enables both the services provided within the Core and IDDRC investigators of all backgrounds. To facilitate engagement in clinical research by basic science researchers and young investigators, the sub-core assists in the preparation of regulatory documents, the training of study staff, patient recruitment and data analysis. Patient recruitment is supported by the Research Participant Registry, a collection of over 30,000 individuals willing to participate in clinical research, including patients with a myriad of neurodevelopmental diagnoses, their parents and siblings, and typically developing controls. To ensure that the efforts of the Core are focused in areas that will produce meaningful benefits for the intellectual and developmental disability population, the Core is strongly connected to clinical care centers and patient advocacy groups, both of which advise the activity and development of the group. Through these efforts, the Core has a proven record of success in designing and launching preclinical translational and clinical projects for IDDRC investigators.