Gastineau D A
Division of Hematology, Mayo Clinic, Rochester, MN 55905, USA.
Bone Marrow Transplant. 2004 Apr;33(8):777-80. doi: 10.1038/sj.bmt.1704452.
Advanced cell-based therapies are rapidly evolving and there is apparent increasing tension between regulation and introduction of new scientific advances. The model and concept of product development has introduced new challenges for laboratories developing cell-based therapies. The scale-up of experimental processes to production processes is an expensive but poorly compensated activity. The regulatory issues including personnel and facility requirements for manufacturing creates significant economic barriers to academic centers entering the field. As yet the model of product development envisioned by regulatory agencies is a patented commercial product to be manufactured by a company or its licensed manufacturers. The alternative model of advanced blood banks producing products may provide wider dissemination of products to patients. Although regulations have sometimes been perceived to be delaying advances, the regulatory agencies are evolving into a cooperative, supportive position that will ultimately speed the introduction of safe and effective cell-based therapies to broad clinical use. The vision for cell-based therapies needs scientific breadth and regulatory understanding to benefit the greatest number of patients.
先进的细胞疗法正在迅速发展，在监管与新科学进展的引入之间，明显存在着日益加剧的紧张关系。产品开发的模式和概念给研发细胞疗法的实验室带来了新的挑战。将实验流程扩大到生产流程是一项成本高昂但回报微薄的活动。包括人员和生产设施要求在内的监管问题，给学术中心进入该领域造成了巨大的经济障碍。到目前为止，监管机构所设想的产品开发模式是一种由公司或其授权制造商生产的专利商业产品。先进血库生产产品的替代模式可能会使产品更广泛地惠及患者。尽管有时人们认为监管会延缓进展，但监管机构正逐渐转变为合作、支持的立场，这最终将加速安全有效的细胞疗法广泛应用于临床。细胞疗法的愿景需要科学的广度和对监管的理解，才能使最多的患者受益。
