Viability, specificity and yields of clinical grade primary and expanded human fetal cells for neural transplantation

MRC赞助了一项多中心临床试验，研究胎儿神经组织移植治疗严重进行性脑退行性疾病亨廷顿病的安全性和可行性。然而，由于欧盟新法规的实施，该试验在完成前5例移植后暂停，该法规要求对患者使用的任何细胞或组织进行制药级处理。虽然英国的试验已经充分评估了细胞的安全性，但我们无法符合认证所需的非常严格的环境控制条件。因此，我们对实验室进行了全面重建，以符合新的规定。在目前的拨款中，我们将进行所需的详细研究，以验证细胞制备和细胞培养扩增的新实验室程序，并确认细胞在动物模型中的功效。这将使我们能够完成暂停的HD试验，并将英国多中心细胞移植项目扩展到帕金森病和亨廷顿病的试验。

The MRC has sponsored a multicentre clinical trial on the safety and fesibility of fetal neural tissue transplantation in a severe progressive degenerative disease of the brain, Huntington s disease. However, this trial has been suspended after completion of the first 5 transplants because of the implementation of new EU regulations that require pharmaceutical grade handling of any cells or tissues used in patients. Although the UK trial had fully assessed the safety of the cells, we could not comply with the very stringent environmental control conditions required for accreditation. We have therefore undertaken a full rebuild of the laboratories to comply with the new regulations. In the present grant, we will undertake the detailed studies required to validate new laboratory procedures for cell preparation and expansion in cell culture and confirm the efficacy of the cells in animal models. This will allow us to complete the suspended HD trial, and to extend the multicentre UK cell transplant programme to trials in Parkinson s as well as Huntington s disease.