Handy Chalonda R, Krudy Christina, Boulis Nicholas, Federici Thais
Department of Neurosurgery, Emory University, 101 Woodruff Circle, Room 6339, Atlanta, GA 30322, USA.
Neurol Res Int. 2011;2011:403808. doi: 10.1155/2011/403808. Epub 2011 May 3.
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder marked by progressive loss of motor neurons, muscle wasting, and respiratory dysfunction. With disease progression, secondary symptoms arise creating new problematic conditions for ALS patients. Amongst these is pain. Although not a primary consequence of disease, pain occurs in a substantial number of individuals. Yet, studies investigating its pathomechanistic properties in the ALS patient are lacking. Therefore, more exploratory efforts into its scope, severity, impact, and treatment should be initiated. Several studies investigating the use of Clostridial neurotoxins for the reduction of pain in ALS patients suggest the potential for a neural specific approach involving focal drug delivery. Gene therapy represents a way to accomplish this. Therefore, the use of viral vectors to express transgenes that modulate the nociceptive cascade could prove to be an effective way to achieve meaningful benefit in conditions of pain in ALS.
肌萎缩侧索硬化症(ALS)是一种致命的神经退行性疾病,其特征是运动神经元逐渐丧失、肌肉萎缩和呼吸功能障碍。随着疾病进展,会出现继发性症状,给ALS患者带来新的问题。其中就包括疼痛。虽然疼痛并非该疾病的主要后果,但相当多的患者会出现疼痛症状。然而,目前缺乏对ALS患者疼痛发病机制的研究。因此,应进一步探索疼痛的范围、严重程度、影响及治疗方法。多项关于使用肉毒杆菌神经毒素减轻ALS患者疼痛的研究表明,采用局部给药的神经特异性方法具有潜力。基因治疗是实现这一目标的一种途径。因此,利用病毒载体表达调节伤害性感受级联反应的转基因,可能是在ALS疼痛状况下取得显著疗效的有效方法。