Phase 1 evaluation of CRISPR-CAR gene edited T cells in relapsed refractory B cell acute lymphoblastic leukaemia

CRISPR-CAR基因编辑T细胞治疗复发难治性B细胞急性淋巴细胞白血病的1期评估

基本信息

批准号：
MR/S019022/1
负责人：
Waseem Qasim
金额：
$ 166.03万
依托单位：
University College London
依托单位国家：
英国
项目类别：
Research Grant
财政年份：
2019
资助国家：
英国
起止时间：
2019 至无数据
项目状态：
已结题

来源：
https://gtr.ukri.org/projects?ref=MR%2FS019022%2F1
关键词：
Phase evaluation CRISPR CAR gene

项目摘要

New treatments for leukaemia using gene therapy to re-program white bloods cells against cancer cells are showing a great deal of promise. In some trials, over 8 out of every 10 patient treated had a good response. Most of the trials so far have relied in collecting a patients own white cells and then using a disabled virus to fit new genes into a group of cells called T cells. This process then arms the cells against leukaemia, so when they are given back to the patients they can attack the disease. We recently showed that its possible not only to arm cells against leukaemia but also disarm them to stop side effects if they are taken from a healthy donor and transplanted to a patient without any matching. A technique called gene editing was used to to achieve this, and we are currently running a clinical trial to test the approach in more patients. In the meantime, improvements in the way we modify cells have led to the development of new versions of the treatment that we think are going to be more widely used. This project will test the new versions, made using CRISPR gene editing, to see if we can treat leukaemia in children where all other treatments have failed. We aim to treat 10 children over two years, and if they can be cleared of leukaemia, they will receive a vote marrow transplant to improve their chances of cure.

使用基因疗法重新编程白细胞对抗癌细胞的白血病新疗法显示出巨大的希望。在一些试验中，每 10 名接受治疗的患者中就有超过 8 名获得了良好的反应。迄今为止，大多数试验都依赖于收集患者自己的白细胞，然后使用失活的病毒将新基因装入一组称为 T 细胞的细胞中。然后，这个过程使细胞能够抵抗白血病，因此当它们被回输给患者时，它们可以攻击这种疾病。我们最近表明，如果从健康的捐赠者身上取出细胞并在没有任何匹配的情况下移植到患者身上，不仅可以武装细胞对抗白血病，还可以解除它们的武装以阻止副作用。一种称为基因编辑的技术用于实现这一目标，我们目前正在进行一项临床试验，以在更多患者中测试该方法。与此同时，我们修改细胞方式的改进导致了新疗法的开发，我们认为这种疗法将得到更广泛的应用。该项目将测试使用 CRISPR 基因编辑制作的新版本，看看我们是否可以治疗所有其他治疗方法都失败的儿童白血病。我们的目标是在两年内治疗 10 名儿童，如果他们能够清除白血病，他们将接受骨髓移植，以提高治愈的机会。