Training Program in Stem Cell Translational Medicine for Neurological Disorders

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In 2013, we launched our Training Program in Stem Cell Translational Medicine for Neurological Disorders (“training program”) at the University of California, Irvine (UCI) with the premise that such training is essential if stem cells are to be used successfully to treat neurological disorders. Nine years later, we believe that this premise is even more true, as many more academic groups and biotechnology companies explore the potential of stem cells to directly treat, or lead to treatments for, human disease and injury. Three main area of translational research are being conducted: the use of stem cells or their derivatives for transplantation to replace those cells damaged or destroyed by injury, the use of stem cells, including patient derived cells, to study disease processes and mechanisms, and the use of differentiated products of stem cells to screen for drugs that could alleviate disease or damage. In recent years the addition of CRISPR-mediated gene editing and the development of “brain organoids” have added new tools to the arsenal of researchers in the field. For the promise of stem cell treatments to become a reality, not only must basic research advance in this rapidly evolving field, but these advances must also be translated through preclinical and clinical development into clinical practice. Translational approaches must be taught directly. The goal of this renewal is to provide an even better environment to train a new generation of scientists in the translational application of stem cell biology to neuroscience. Building upon key successes and feedback from the trainees over the course of this T32 since its inception, our updated program will even better serve this purpose and includes (1) state-of-the-art rigorous and reproducible wet lab science employing training in the fundamental principles of rigor and reproducibility, as well as quantitative and statistical methods; (2) clinical experiences to understand and plan research efforts in the context of clinical translation; (3) knowledge regarding practical aspects of moving discoveries to clinical trials and therapies through industry internships and Sue and Bill Gross Stem Cell Research Center resources; (4) communication, StrengthsFinder and personal interaction skills; (5) Familiarity with the Food and Drug Administration (FDA) approval processes for clinical trials; and (6) an increased commitment to promoting diversity, equity and inclusion (DEI). These workshops/elements of training, which were universally praised, represent skills that are critical for success in interdisciplinary science and enhance success for all students, particularly underrepresented students because of the focus on and positive impact of networking. These six elements represent unique aspects of training designed for this training program based on UCI’s experience of successful clinical translation.