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在症状性婴儿痉挛的多次打击大鼠模型中甘丙肽类似物 NAX 5055 的疗效和耐受性。

Efficacy and tolerability of the galanin analog NAX 5055 in the multiple-hit rat model of symptomatic infantile spasms.

机构信息

Saul R. Korey Department of Neurology, Laboratory of Developmental Epilepsy, Albert Einstein College of Medicine, Bronx, NY, USA; Unité de Neurologie et Neuroréhabilitation Pédiatrique, Département Médico-Chirurgical de Pédiatrie, CHUV, Lausanne, Switzerland.

NeuroAdjuvants, Inc., Salt Lake City, UT, USA; Department of Pharmacology and Toxicology, University of Utah, Salt Lake City, UT, USA.

出版信息

Epilepsy Res. 2014 Jan;108(1):98-108. doi: 10.1016/j.eplepsyres.2013.10.015. Epub 2013 Oct 28.

Abstract

Infantile spasms are seizures manifesting in infantile epileptic encephalopathies that are associated with poor epilepsy and cognitive outcomes. The current therapies are not always effective or are associated with serious side effects. Early cessation of spasms has been proposed to improve long-term outcomes. To identify new therapies for infantile spasms with rapid suppression of spasms, we are using the multiple-hit rat model of infantile spasms, which is a model of refractory infantile spasms. Here, we are testing the efficacy and tolerability of a single dose of the galanin receptor 1 preferring analog, NAX 5055, in the multiple-hit model of spasms. To induce the model, postnatal day 3 (PN3) male Sprague-Dawley rats underwent right intracerebral infusions of doxorubicin and lipopolysaccharide; p-chlorophenylalanine was then injected intraperitoneally (i.p.) at PN5. After the onset of spasms at PN4, 11-14 rats/group were injected i.p. with either NAX 5055 (0.5, 1, 2, or 4mg/kg) or vehicle. Video monitoring for spasms included a 1h pre-injection period, followed by 5h of recording post-injection, and two 2h sessions on PN5. The study was conducted in a randomized, blinded manner. Neurodevelopmental reflexes were assessed daily as well as at 2h after injection. Respiratory function, heart rate, pulse distension, oximetry and blood glucose were measured 4h after injection. The relative expression of GalR1 and GalR2 mRNA over β-actin in the cerebral cortex and hippocampus was determined with real time reverse transcription polymerase chain reaction. There was no acute effect of NAX 5055 on spasm frequency after the single dose of NAX 5055 (n=11-13 rats/group, following exclusions). Neurodevelopmental reflexes, vital signs, blood glucose measured 4h post-injection, and survival were not affected. A reduction in pulse and breath distention of unclear clinical significance was observed with the 7mg/kg NAX 5055 dose. GalR1 mRNA was present in the cerebral cortex and hippocampus of PN4 and adult rats. The hippocampal - but not the cortical - GalR1 mRNA expression was significantly lower in PN4 pups than in adults. GalR1 mRNA was also at least 20 times less abundant in the PN4 cortex than GalR2 mRNA. In conclusion, a single dose of NAX 5055 has no acute efficacy on spasms or toxicity in the multiple hit rat model of medically refractory infantile spasms. Our findings cannot exclude the possibility that repetitive NAX 5055 administration may show efficacy on spasms. The higher expression of GalR2 in the PN4 cortex suggests that GalR2-preferring analogs may be of interest to test for efficacy on spasms.

摘要

婴儿痉挛症是婴儿癫痫性脑病中表现出来的癫痫发作,与癫痫和认知预后不良有关。目前的治疗方法并不总是有效,或者会伴有严重的副作用。早期停止痉挛发作被认为可以改善长期预后。为了寻找具有快速抑制痉挛作用的婴儿痉挛新疗法,我们使用了多打击型婴儿痉挛模型,该模型是一种难治性婴儿痉挛模型。在这里,我们正在测试甘丙肽受体 1 偏好类似物 NAX 5055 在痉挛多打击模型中的疗效和耐受性。为了诱导模型,出生后第 3 天(PN3)雄性 Sprague-Dawley 大鼠接受右颅内阿霉素和脂多糖输注;然后在 PN5 时经腹腔注射对氯苯丙氨酸。PN4 时出现痉挛后,每组 11-14 只大鼠经腹腔注射 NAX 5055(0.5、1、2 或 4mg/kg)或载体。痉挛的视频监测包括注射前 1 小时,注射后 5 小时记录,PN5 时进行两次 2 小时记录。研究采用随机、双盲的方法进行。神经发育反射在每天以及注射后 2 小时进行评估。在注射后 4 小时测量呼吸功能、心率、脉搏扩张、血氧饱和度和血糖。使用实时逆转录聚合酶链反应确定大脑皮层和海马中海马肽受体 1 和 2 mRNA 相对于 β-肌动蛋白的相对表达。单次剂量的 NAX 5055 后,NAX 5055 对痉挛频率没有急性影响(每组 11-13 只大鼠,排除后)。神经发育反射、生命体征、注射后 4 小时测量的血糖以及存活率均不受影响。7mg/kg NAX 5055 剂量观察到脉搏和呼吸扩张减少,但临床意义不明。PN4 天和成年大鼠的大脑皮层和海马中均存在 GalR1 mRNA。PN4 幼鼠海马中海马肽受体 1 mRNA 的表达明显低于成年鼠。PN4 皮层中海马肽受体 1 mRNA 的丰度也至少比 GalR2 mRNA 低 20 倍。总之,单次剂量的 NAX 5055 对多打击型难治性婴儿痉挛大鼠模型中的痉挛或毒性没有急性疗效。我们的研究结果不能排除重复给予 NAX 5055 可能对痉挛有疗效的可能性。PN4 皮层中 GalR2 的高表达表明 GalR2 偏好类似物可能对测试痉挛疗效具有重要意义。

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