Transplantation of autologous neural stem cells for treatment of Parkinson's disease: study protocol for a non-randomized controlled trial

Background and objectives: As degenerative changes in dopaminergic neurons occur only in the substantia nigra and striatum of patients with Parkinson's disease (PD), cellular therapy is suitable for this condition. However, previous clinical studies using stem cells for treatment of PD have short follow-ups. Thus, studies with longer follow-ups are required to further investigate the efficacy of stem cell-based therapy. Design: A non-randomized controlled trial. Methods: Ninety eligible PD patients will be recruited from the Department of Neurology at the Affiliated Hospital of Xuzhou Medical University, China. These patients will be assigned to three groups according to each patient's or their legal guardians’ wishes (n = 30 per group). In the control group, 12-week routine drug treatment will be performed. In intrathecal administration and intravenous administration groups, intrathecal administration of autologous neural stem cells (NSCs) into the subarachnoid space or intravenous administration of autologous NSCs will be performed once a week for 4 successive weeks in addition to the 12-week routine drug treatment. Outcome measures: The primary outcome measure is symptom improvement rate at 36 months post-treatment. Secondary outcome measures are nigrostriatal dopamine content, α-synuclein content in blood and cerebrospinal fluid, Barthel index, and safety indicators. Discussion: This study will be performed to demonstrate efficacy of autologous NSC transplantation as a prospective treatment for PD and investigate differences between intrathecal and intravenous transplantation routes, thus providing objective quantitative evidence for clinical applications. Ethics and dissemination: This study was designed in July 2017. Ethics approval from Medical Ethics Committee of the Affiliated Hospital of Xuzhou Medical University of China was achieved on December 22, 2017 (approval No. XYFY2017-KL052-01). The study protocol was registered on December 25, 2017. Patient recruitment began in January 2018 and will end in January 2019. Each patient will be followed up for 36 months. Follow-ups will be completed in January 2022. Data analysis will be performed in July 2022. Results will be disseminated by publication in a peer-reviewed journal. Trial registration: This trial was registered with the Chinese Clinical Trial Registry (registration number: ChiCTR-ONC-17014141). Protocol version (1.0).