Exploring the State of the Science in the Field of Regenerative Medicine Challenges of and Opportunities for Cellular Therapies: Proceedings of a Workshop (2017) / Chapter Skim
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1 Introduction
1 Introduction
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The potential applications of cellular therapies are broad, ranging from the use of islet cell transplantation and regeneration to cure type 1 diabetes, to regenerative neurobiology approaches to treat injuries and degenerative diseases like spinal cord injury and amyotrophic lateral sclerosis (Feldman et al., 2014; Shapiro et al., 2000)
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Such knowledge may help researchers address the scientific and technical hurdles related to assessing and ensuring successful long-term outcomes of cell therapies, controlling cell differentiation, and refining processes for production on a scale that is commercially sustainable and yields quantities of product that have the potential to be clinically effective. Some areas of regenerative medicine, such as hematopoietic stem cell transplants for the treatment of blood cancers, have experienced success in treating patients, and lessons and best practices could potentially be applied to other areas of research (Chhabra et al., 2016)
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The forum members spent time considering many of the challenges facing the field. On October 13, 2016, the Forum on Regenerative Medicine hosted its first public workshop with the goal of developing a broad understanding of the opportunities and challenges associated with regenerative medicine cellular therapies and related technologies.
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Cynthia Dunbar, the workshop co-chair and president of the American Society of Gene and Cell Therapy, asked presenters and participants to focus on describing the gaps in basic scientific knowledge, identifying resources that did or would help move regenerative medicine forward, and discussing common challenges such as reproducibility and standardization. There are a number of factors that are coming together to increase the power and success of regenerative medicine success, said Lorenz Studer, the keynote speaker and director of the Center for Stem Cell Biology at the Memorial Sloan Kettering Cancer Center.
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In many cases, introduced pluripotent cells mature at a slower rate, which can not only delay the point at which they become clinically effective, but potentially create safety issues as well. Developing accurate models for testing therapies is critical; however, Studer said, current animal models often do not match the necessary physiology well enough to predict the effects of a therapy in the human body.
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As of 2016, Studer's lab had produced approximately 1,000 doses of human pluripotent stem cells using good manufacturing practice (GMP) standards, keeping them in frozen storage for later use in humans.
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Speakers on the hematology and immunity panel shared their views on the state of the science in hematopoietic stem cell transplantation, gene editing, and T cell therapies. Chapter 4 delves into the scientific and clinical advances in regenerative medicine for neurological and ophthalmological tissues.
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Chapter 6 explores the state of the science and the potential applications of regenerative medicine for renal tissues. The panelists discussed the prevalence of renal failure, polycystic kidney disease, and emerging technologies related to organoids.
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