Brain Neurotherapy Bio, Inc., 5815 Balmoral Dr, 94619 Oakland, United States.
Department of Neurological Surgery, The Ohio State University Wexner Medical Center, N1047 Doan Hall, 410 W. 10th Ave., 43210 Columbus, United States.
Neurol Neurochir Pol. 2020;54(3):220-231. doi: 10.5603/PJNNS.a2020.0046. Epub 2020 Jun 18.
Vector-based intracerebral gene therapies are being used to treat specific neurodegenerative conditions such as Parkinson's Disease (PD). This review presents a basis for central nervous system (CNS) gene therapy treatments of neurodegenerative diseases such as PD, as well as the need for novel skill sets and health delivery strategies within the clinical neurosciences (neurology and neurosurgery) to meet future demand for such therapies.
Preclinical vector-based gene therapy approaches have been translated into clinical trials for PD and other neurodegenerative conditions. Unfortunately, such trials, and parallel efforts using other therapeutics, have yet to provide a breakthrough. Image-guided convection enhanced delivery (CED) optimises the parenchymal distribution of gene therapies applied within the CNS, and may ultimately provide such a breakthrough.
Currently, image-guided CED and gene therapy are not part of training programmes for most neurosurgeons and neurologists. As a result, few medical centres and hospitals have sufficiently experienced teams to participate in gene transfer clinical trials for PD or other neurological conditions. If CNS gene therapies prove to be efficacious for PD and/or other conditions, the demand for such treatments will overwhelm the available number of experienced clinical neuroscience teams and treatment centres.
Expanded indications and demand for CNS gene therapies will require a worldwide educational effort to supplement the training of clinical neuroscience practitioners. Initially, a limited number of Centres of Excellence will need to establish relevant educational training requirements and best practice for such therapeutic approaches. Advanced technologies, including robotics and artificial intelligence, are especially germane in this regard, and will expand the treatment team's capabilities while assisting in the safe and timely care of those afflicted.
基于载体的脑内基因治疗正被用于治疗特定的神经退行性疾病，如帕金森病（PD）。本文为中枢神经系统（CNS）基因治疗治疗神经退行性疾病（如 PD）提供了基础，同时也需要在临床神经科学（神经病学和神经外科学）中建立新的技能体系和医疗服务策略，以满足未来对这些治疗方法的需求。
基于载体的基因治疗方法已从临床前转化为 PD 及其他神经退行性疾病的临床试验。不幸的是，这些试验以及使用其他疗法的平行努力尚未取得突破。影像引导的对流增强递送（CED）优化了基因疗法在中枢神经系统内的实质分布，最终可能提供这样的突破。
目前，影像引导 CED 和基因治疗并不是大多数神经外科医生和神经科医生培训计划的一部分。因此，很少有医疗中心和医院有足够经验的团队参与 PD 或其他神经疾病的基因转移临床试验。如果中枢神经系统基因治疗对 PD 和/或其他疾病有效，那么对这些治疗的需求将超过经验丰富的临床神经科学团队和治疗中心的数量。
中枢神经系统基因治疗的扩展适应证和需求将需要全球范围内的教育努力，以补充临床神经科学从业者的培训。最初，需要有限数量的卓越中心来建立相关的教育培训要求和最佳实践，以用于此类治疗方法。先进的技术，包括机器人技术和人工智能，在这方面尤为重要，它们将扩大治疗团队的能力，同时协助安全和及时地治疗受影响的患者。
