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首页> 外文期刊>Lancet Neurology >Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial.
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Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial.

机译：CERE-120（腺相关病毒血清型2-neurturin）对特发性帕金森氏病患者的腹腔内递送CERE-120（安全性和耐受性）：一项开放标签的I期试验。

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BACKGROUND: There is an urgent need for therapies that slow or reverse the progression of Parkinson's disease (PD). Neurotrophic factors can improve the function of degenerating neurons and protect against further neurodegeneration, and gene transfer might be a means to deliver effectively these factors to the brain. The aim of this study was to assess the safety, tolerability, and potential efficacy of gene delivery of the neurotrophic factor neurturin. METHODS: In this phase I, open-label clinical trial, 12 patients aged 35-75 years with a diagnosis of PD for at least 5 years in accordance with the UK Brain Bank Criteria received bilateral, stereotactic, intraputaminal injections of adeno-associated virus serotype 2-neurturin (CERE-120). The first six patients received doses of 1.3x10(11) vector genomes (vg)/patient, and the next six patients received 5.4x10(11) vg/patient. This trial is registered with ClinicalTrials.gov, number NCT00252850. FINDINGS: The procedure was well tolerated. Extensive safety monitoring in all patients revealed no clinically significant adverse events at 1 year. Several secondary measures of motor function showed improvement at 1 year; for example, a mean improvement in the off-medication motor subscore of the Unified Parkinson's Disease Rating Scale (UPDRS) of 14 points (SD 8; p=0.000121 [36% mean increase; p=0.000123]) and a mean increase of 2.3 h (2; 25% group mean increase; p=0.0250) in on time without troublesome dyskinesia were seen. Improvements in several secondary measures were not significant, including the timed walking test in the off condition (p=0.053), the Purdue pegboard test of hand dexterity (p=0.318), the reduction in off time (p=0.105), and the activities of daily living subscore (part II) of the UPDRS (p=0.080). (18)F-levodopa-uptake PET did not change after treatment with either dose of CERE-120. INTERPRETATION: The initial data support the safety, tolerability, and potential efficacy of CERE-120 as a possible treatment for PD; however, these results must be viewed as preliminary until data from blinded, controlled clinical trials are available. FUNDING: Ceregene; Michael J Fox Foundation for Parkinson's Research.
机译：背景：迫切需要能够减慢或逆转帕金森氏病（PD）进展的疗法。神经营养因子可以改善退化的神经元的功能并防止进一步的神经变性，而基因转移可能是将这些因子有效传递到大脑的一种手段。这项研究的目的是评估神经营养因子神经营养素基因传递的安全性，耐受性和潜在疗效。方法：在这一第一阶段的开放标签临床试验中，根据英国脑库标准，对12例年龄在35-75岁，诊断为PD且至少5年的患者进行了腺苷相关病毒的双侧，立体定向，腹膜内注射血清型2-neurturin（CERE-120）。前六名患者接受1.3x10（11）vg /患者的剂量，随后六名患者接受5.4x10（11）vg /患者。该试验已在ClinicalTrials.gov上注册，编号为NCT00252850。结果：该程序耐受良好。在所有患者中进行的广泛安全性监测显示，在1年时没有临床上的重大不良事件。运动功能的一些次要指标在1年时有所改善。例如，统一帕金森氏疾病评分量表（UPDRS）的药物外运动评分平均提高了14点（SD 8; p = 0.000121 [平均增长36％; p = 0.000123]），平均提高了2.3 h（2;组平均增加25％; p = 0.0250）未出现运动障碍困扰。多项辅助措施的改善并不显着，包括关闭状态下的定时步行测试（p = 0.053），手灵巧性的Purdue钉板测试（p = 0.318），关闭时间的减少（p = 0.105）和UPDRS的日常生活子项（第二部分）的活动（p = 0.080）。 （18）用任一剂量的CERE-120治疗后，F-左旋多巴摄取PET均未改变。解释：初步数据支持CERE-120作为PD治疗的可能的安全性，耐受性和潜在疗效。但是，在获得盲目对照临床试验的数据之前，必须将这些结果视为初步结果。资金来源：Ceregene;迈克尔·J·福克斯（Michael J Fox）帕金森研究基金会。

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