Department of Neurological Surgery, Emory University School of Medicine, Atlanta, GA 30322, USA.
Lancet Neurol. 2011 Jun;10(6):509-19. doi: 10.1016/S1474-4422(11)70097-7. Epub 2011 May 10.
Human retinal pigment epithelial (RPE) cells produce levodopa and their transplantation into the striatum might improve continuity of administration compared with that achieved with oral levodopa. We aimed to assess the safety, tolerability, and efficacy of transplantation of microcarrier-bound human RPE cells versus a sham surgery control in patients with advanced Parkinson's disease.
In this randomised, double-blind study eligible patients were aged 36-70 years, had been symptomatic for at least 5 years, were in Hoehn and Yahr stage 3-4 and had unified Parkinson's disease rating scale (UPDRS) motor scores of 38-70 when off medication (off state), and had symptoms that responded to oral levodopa but were insufficiently controlled by optimised pharmacotherapy. Randomisation was done in a 1:1 ratio. Only the neurosurgical team was aware of treatment assignments. During stereotactic transplantation around 325,000 cells per side were injected into the postcommissural putamen; sham surgery patients received partial burr holes. The primary efficacy endpoint was change in UPDRS off-state motor score at 12 months. This study is registered with ClinicalTrials.gov, number NCT00206687.
Of 71 enrolled patients, 35 underwent cell transplantation and 36 sham surgery. Change in mean motor scores did not differ significantly between groups (-10.5 [SD 10.26] for transplantation vs -10.1 [SD 12.26] for sham surgery, p=0.9). The overall rate of adverse events was similar in the two study groups, although the number attributable to surgery or RPE cells (mostly neurological or psychiatric) was higher in transplant recipients. Two and seven patients died in the sham surgery and transplantation group, respectively; one death in the latter group was possibly related to surgery or RPE cells.
Transplantation of human RPE cells provided no antiparkinsonian benefits compared with sham surgery.
Bayer HealthCare AG.
人视网膜色素上皮（RPE）细胞能够产生左旋多巴，与口服左旋多巴相比，将其移植到纹状体可能会提高给药的连续性。我们旨在评估微载体结合的人 RPE 细胞移植与假手术对照治疗晚期帕金森病患者的安全性、耐受性和疗效。
在这项随机、双盲研究中，合格的患者年龄在 36-70 岁之间，已经出现症状至少 5 年，处于 Hoehn 和 Yahr 分期 3-4 期，并且在停药状态下（OFF 状态）统一帕金森病评定量表（UPDRS）运动评分达到 38-70，并且症状对口服左旋多巴有反应，但通过优化药物治疗不能得到充分控制。随机分组比例为 1:1。只有神经外科团队知道治疗分配。在立体定向移植过程中，每侧注射约 325,000 个细胞；假手术患者接受部分颅骨钻孔。主要疗效终点是 12 个月时 UPDRS 停药状态运动评分的变化。这项研究在 ClinicalTrials.gov 注册，编号为 NCT00206687。
在 71 名入组患者中，35 名接受了细胞移植，36 名接受了假手术。两组间平均运动评分的变化无显著差异（移植组为-10.5[10.26SD]，假手术组为-10.1[12.26SD]，p=0.9）。两组的不良事件总发生率相似，但移植组归因于手术或 RPE 细胞（主要为神经或精神）的不良事件更多。假手术组和移植组分别有 2 例和 7 例患者死亡；后者组的 1 例死亡可能与手术或 RPE 细胞有关。
与假手术相比，人 RPE 细胞移植没有提供抗帕金森病的益处。
拜耳健康护理公司。
