CART细胞是自体或同种异体人类淋巴细胞,其被基因工程改造以表达靶向在肿瘤细胞上表达的抗原(例如CD19)的嵌合抗原受体。CAR-T细胞代表了一类新的医药产品,属于高级治疗药物产品(ATMP)的广泛类别,根据EC法规2007-1394的定义。具体来说,它们被归类为基因治疗药物。虽然CAR-T细胞是细胞疗法,制造和交付的组织与用于交付造血细胞移植物的组织大不相同,出于不同的原因,包括将其分类为药用产品。目前可用的临床观察大多是在美国或中国进行的试验中产生的。它们对晚期或预后不良的血液系统恶性肿瘤患者具有显着的疗效,然而,在相当比例的患者中有严重的副作用。毒性可以而且必须在负责患者的临床细胞治疗病房之间进行充分协调的背景下进行预期和处理。以及邻近的重症监护室.本次研讨会旨在确定要满足的先决条件,以便法国医院在启动旨在研究CAR-T细胞的临床试验之前有效组织并满足赞助商的期望。
CAR T-cells are autologous or allogeneic human lymphocytes that are genetically engineered to express a chimeric antigen receptor targeting an antigen expressed on tumor cells such as CD19. CAR T-cells represent a new class of medicinal products, and belong to the broad category of Advanced Therapy Medicinal Products (ATMPs), as defined by EC Regulation 2007-1394. Specifically, they are categorized as gene therapy medicinal products. Although CAR T-cells are cellular therapies, the organization for manufacturing and delivery is far different from the one used to deliver hematopoietic cell grafts, for different reasons including their classification as medicinal products. Currently available clinical observations were mostly produced in the context of trials conducted either in the USA or in China. They demonstrate remarkable efficacy for patients presenting advanced or poor-prognosis hematological malignancies, however with severe side effects in a significant proportion of patients. Toxicities can and must be anticipated and dealt with in the context of a full coordination between the clinical cell therapy ward in charge of the patient, and the neighboring intensive care unit. The present workshop aimed at identifying prerequisites to be met in order for French hospitals to get efficiently organized and fulfill sponsors\' expectations before initiation of clinical trials designed to investigate CAR T-cells.