CLINICAL TRANSLATION CORE

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In its current form, the Baylor College of Medicine (BCM) IDDRC’s Clinical Translational Core (CTC) has provided an outstanding platform to integrate basic science researchers, clinical investigators, and clinicians with infrastructure and resources to enable the translation of many mechanistic discoveries from our center into the clinical realm. In this proposal, we are excited to build upon the success of our CTC and expand on the services using a new administrative and novel core structure that will allow us to leverage our strengths for developing therapies for IDD. The new structure and services were born from our “evolving Core theme” in order to accommodate and translate the new and exciting discoveries from our center investigators. The CTC will be composed of three units: Study Design and Implementation Unit, Biospecimen and Data Unit, and Center Navigation Unit that will collaborate closely with other cores of the BCM IDDRC, national and international networks, patient registries, patients, and stakeholders to facilitate all aspects of transitional research in IDD. A vision that we have for our CTC is that it should have an impact beyond our local center and that our processes and activities should be of use to other centers and IDD researchers across the United States. In line with that vision, the CTC will establish a Clinical Outcome Assessment Panel that will leverage the significant preclinical and clinical expertise at our center to develop and validate endpoints for clinical trials which would be critical for clinical trial readiness. Additionally, the CTC will work towards having a biospecimen repository from which precious samples can be shared with other centers and researchers. Finally, as the future of IDD research is dependent on young clinicians and scientists, the CTC will focus on training the next- generation of clinical and translational researchers in IDD. We strongly believe that the CTC will be the nidus by which: 1) basic researchers will have access to clinical information and human samples and thus be integrated into the clinical enterprise, 2) basic research questions will be validated in a clinical arena, and 3) newer diagnostic modalities and therapies will be fostered. The structure and services of the CTC will not only facilitate the translation of findings from the “bench-to-the- bedside”, but also drive research in the laboratory based on phenotypic observations made in the clinics. Such bidirectional translation will not only advance the basic science research but also make a significant impact on the care of the patients with IDD.