Genetic Engineering of T-cells for Adoptive Immunotherapy of Chronic Lymphocytic Leukemia

免疫系统在清除大量病毒感染的细胞而不损害正常的未感染组织方面非常有效，但它通常对癌细胞没有反应。在这个项目中，我们计划修改t细胞，免疫系统的关键组成部分，使它们杀死已形成的癌症。我们建议从患有慢性淋巴细胞性白血病（CLL）的患者身上提取正常的t细胞，在实验室里对它们进行基因改造，使它们能够杀死白血病细胞，以期最终将它们作为一种治疗方法交还给患者。这项工程利用了我们对免疫系统如何工作的详细了解。从某种意义上说，我们将利用大自然的工具包来创造一种杀死癌症的免疫反应。我们将在患有人类白血病的老鼠身上测试这些t细胞，以确定这种新疗法的安全性和有效性。我们计划利用从这个项目中收集的数据，为标准治疗失败的CLL患者的临床研究做好基础工作。这项工作代表了一个新的治疗领域的开始，在这个领域中，t细胞可以被设计成专门去除有害细胞群而不损害健康的正常组织

The immune system is highly effective at removing large-numbers of virally infected cells without damaging normal non-infected tissues, however it often remains unresponsive to cancer cells. In this project, we plan to modify T-cells, a key component of the immune system, so that they kill established cancers. We propose taking normal T-cells from patients with a form of cancer known as chronic lymphocytic leukaemia (CLL), and genetically engineer them in the laboratory so that they kill the leukaemia cells, with a view to ultimately giving them back to the patient as a treatment. This engineering exploits our detailed understanding of how the immune system works. In a sense we will use nature‘s toolkit to create a cancer killing immune response. We will test these T-cells in mice with human leukaemias, to establish safety and effectiveness of this new form of treatment. We plan to use the data amassed from this project to prepare the ground-work for a clinical study in patients with CLL that have failed standard treatment. This work represents the beginning of a new field of therapy, where T-cells can be engineered to specifically remove harmful cell populations without damaging healthy normal tissue