The Medical School, University of Edinburgh, Edinburgh BioQuarter, UK.
Unit of Functional Neurosurgery, National Hospital for Neurology and Neurosurgery, University College London Hospitals NHS Trust, London, UK.
J Parkinsons Dis. 2024;14(3):415-435. doi: 10.3233/JPD-230328.
Parkinson's disease (PD) is an increasingly prevalent neurological disorder, affecting more than 8.5 million individuals worldwide. α-Synucleinopathy in PD is considered to cause dopaminergic neuronal loss in the substantia nigra, resulting in characteristic motor dysfunction that is the target for current medical and surgical therapies. Standard treatment for PD has remained unchanged for several decades and does not alter disease progression. Furthermore, symptomatic therapies for PD are limited by issues surrounding long-term efficacy and side effects. Cell replacement therapy (CRT) presents an alternative approach that has the potential to restore striatal dopaminergic input and ameliorate debilitating motor symptoms in PD. Despite promising pre-clinical data, CRT has demonstrated mixed success clinically. Recent advances in graft biology have renewed interest in the field, resulting in several worldwide ongoing clinical trials. However, factors surrounding the effective neurosurgical delivery of cell grafts have remained under-studied, despite their significant potential to influence therapeutic outcomes. Here, we focus on the key neurosurgical factors to consider for the clinical translation of CRT. We review the instruments that have been used for cell graft delivery, highlighting current features and limitations, while discussing how future devices could address these challenges. Finally, we review other novel developments that may enhance graft accessibility, delivery, and efficacy. Challenges surrounding neurosurgical delivery may critically contribute to the success of CRT, so it is crucial that we address these issues to ensure that CRT does not falter at the final hurdle.
帕金森病(PD)是一种日益流行的神经退行性疾病,影响着全球超过 850 万人。PD 中的α-突触核蛋白病被认为会导致黑质中多巴胺能神经元的丧失,从而导致特征性运动功能障碍,这是当前医学和手术治疗的目标。PD 的标准治疗方法几十年来一直没有改变,也不能改变疾病的进展。此外,PD 的症状治疗受到长期疗效和副作用问题的限制。细胞替代疗法(CRT)提供了一种替代方法,有潜力恢复纹状体多巴胺能输入,并改善 PD 中使人衰弱的运动症状。尽管有有前景的临床前数据,但 CRT 在临床上的效果喜忧参半。最近在移植物生物学方面的进展重新引起了人们对该领域的兴趣,导致了几个全球范围内正在进行的临床试验。然而,尽管细胞移植物的有效神经外科输送具有重要的潜在影响治疗效果,但围绕其的因素仍未得到充分研究。在这里,我们重点关注 CRT 临床转化中需要考虑的关键神经外科因素。我们回顾了用于细胞移植的仪器,突出了当前的特点和局限性,同时讨论了未来的设备如何解决这些挑战。最后,我们还回顾了其他可能增强移植物可及性、输送和疗效的新进展。神经外科输送方面的挑战可能对 CRT 的成功至关重要,因此我们必须解决这些问题,以确保 CRT 不会在最后关头失败。