Stem cell-based therapies offer a new treatment alternative for individuals suffering from devastating disorders such as Parkinson's disease (PD) and Huntington's disease (HD). Derivation and transplantation of stem cells or their derivatives have shown significant therapeutic outcomes in animal model studies and clinical trials. However, commercialization of stem cell-based therapies and widespread access to these treatments are largely hampered by access to large quantities of high-quality products generated under standardized, robust, and controlled conditions. Over the last three decades, the Pharmaceutical Production Research Facility (PPRF) of the University of Calgary, founded by the late Professor Leo A. Behie, has carried out groundbreaking research focusing on the fundamentals of bioprocessing for various stem cells including human neural precursor cells (NPCs) and mesenchymal stem/stromal cells (MSCs). These efforts led to the development of serum-free growth media and expansion protocols for large-scale production of therapeutically-relevant NPCs for the treatment of neurodegenerative disorders. Similarly, high-performance, defined growth media and reliable, large-scale computer-controlled bioreactor protocols were developed for the expansion of MSCs derived from human bone marrow and other sources and the generation of a therapeutically-relevant secretome. Important bioprocessing considerations such as the hydrodynamics of the bioreactor, mass transfer, cellular microenvironment and synergistic impact of media components were evaluated in these studies. Undoubtedly, the diverse topics of research carried out at PPRF significantly contributed to the translation of the laboratory-based stem cell culture studies into production of clinical-grade materials and eventually commercialization of stem cell-based therapies through applying principles of bioengineering and bioprocessing.

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开发用于干细胞疗法的生物反应器方案

基于干细胞的疗法为患有帕金森氏病 (PD) 和亨廷顿氏病 (HD) 等毁灭性疾病的个体提供了一种新的治疗选择。干细胞或其衍生物的衍生和移植在动物模型研究和临床试验中显示出显着的治疗效果。然而，基于干细胞的疗法的商业化和这些疗法的广泛使用在很大程度上受到了在标准化、稳健和受控条件下产生的大量高质量产品的阻碍。在过去的三年中，由已故的 Leo A. Behie 教授创立的卡尔加里大学药物生产研究机构 (PPRF)，开展了开创性研究，重点研究各种干细胞的生物加工基础，包括人类神经前体细胞 (NPC) 和间充质干/基质细胞 (MSC)。这些努力导致了无血清生长培养基的开发和用于大规模生产用于治疗神经退行性疾病的治疗相关 NPC 的扩展方案。同样，开发了高性能、确定的生长培养基和可靠的、大规模计算机控制的生物反应器方案，用于扩增源自人骨髓和其他来源的 MSCs，并产生治疗相关的分泌组。重要的生物工艺考虑因素，例如生物反应器的流体动力学、传质、在这些研究中评估了细胞微环境和培养基成分的协同影响。毫无疑问，PPRF 开展的各种研究课题极大地促进了基于实验室的干细胞培养研究转化为临床级材料的生产，并最终通过应用生物工程和生物加工原理将基于干细胞的疗法商业化。