Janssen Biotech, Inc., Pharmaceutical Companies of Johnson & Johnson, Raritan, NJ.
Janssen Biotech, Inc., Pharmaceutical Companies of Johnson & Johnson, Raritan, NJ.
Cytotherapy. 2022 Jul;24(7):750-753. doi: 10.1016/j.jcyt.2022.02.004. Epub 2022 Mar 15.
Over the last decade, cancer immunotherapy has progressed from an academically interesting field to one of the most promising forms of new treatments in which not the cancer but the immune system is treated. In particular, genetic modification for purposeful redirection of autologous T cells is providing hope to many treatment-resistant patients. This personalized form of medicine is radically different from more traditional oncologic drugs. With these evolving medical advancements and more cellular therapies becoming available, some regulatory agencies have created new regulatory requirements to manage the production of these types of products. The regulations are specifically suited for the manufacture of gene and cell therapy products, as they use a risk-based approach towards product development and manufacturing, when there is limited characterization available. The correct interpretation of how and when requirements apply is crucial, since theoretical approaches to implementing GMP can easily lead to disproportionate and unwarranted restrictions that may not address the specific risks that regulators were intending to control. This is especially relevant for cell collection and biopreservation preceding the manufacturing process for products manufactured from autologous T cells. Both the fresh and cryopreserved apheresis materials can be filed as minimally manipulated starting materials to the authorities. The preservation of such cellular material can then routinely be managed using the available regulations for tissues and cells, allowing for a more fit-for-purpose approach to the control measures implemented.
在过去的十年中，癌症免疫疗法已经从一个学术上有趣的领域发展成为最有前途的新型治疗方法之一，这种方法不是针对癌症，而是针对免疫系统。特别是，为了有目的地重新定向自体 T 细胞而进行的基因修饰，为许多耐药患者带来了希望。这种个性化的医学形式与更传统的肿瘤药物有根本的不同。随着这些不断发展的医学进步和更多细胞疗法的出现，一些监管机构已经制定了新的监管要求，以管理这些类型产品的生产。这些规定专门适用于基因和细胞治疗产品的制造，因为在可用的特性有限的情况下，它们采用基于风险的方法进行产品开发和制造。正确解释何时以及如何适用要求至关重要，因为实施 GMP 的理论方法很容易导致不成比例和不必要的限制，这些限制可能无法解决监管机构旨在控制的具体风险。对于从自体 T 细胞制造的产品的制造过程之前的细胞采集和生物保存，这一点尤其重要。新鲜和冷冻保存的单采材料都可以作为经过最低限度处理的起始材料提交给当局。然后可以使用现有的组织和细胞法规常规管理此类细胞材料的保存，从而对实施的控制措施采用更适合目的的方法。
