Borrás-Blasco J, Plaza-Macías I, Navarro-Ruiz A, Perís-Martí J, Antón-Cano A
Servicio de Farmacia, Hospital General Universitario de Elche, Alicante, España.
Rev Neurol. 1998 Dec;27(160):1021-7.
The Amyotrophic Lateral Sclerosis (ALS) is a disease characterized by the selective degeneracy of the superior motoneurons of the cortex motor and of the inferior motoneurons at level of the encephalic trunk and spinal marrow. Exist sporadic and familiar forms, being estimated an incidence of 1-2 cases by 100,000 inhabitants. The cause of the neuronal degeneracy is yet unknown, being implied, between other mechanisms, the glutamic exotoxicity is the responsible for the death neuronal. The riluzol is a benzothiazole derivative whose neuroprotector mechanism still it has not been totally clarified, though seems that reduces the neuroexcitatory action of the glutamic acid blocking his transmission.
Two clinical trials have been accomplished with similar characteristics: multicentre, randomized, double blind, and placebo-controlled. Between both studies have been included more than 1,100 patient, obtained significant statistic results in the prolongation of the survival time, however this effect was not going accompanied of an improvement in the muscular force neither of the pulmonary capacity, what is translated in which the riluzol does not modify the quality of life of the patient. The drug presents good tolerance and mild adverse effects and as consequence of this in 1996, the FDA approved his marketing and utilization in the treatment of the ALS. The approval of the riluzol as first agent for the treatment of the ALS has raised an important number of problems about the efficiency and cost of the treatment.
Though its benefits are modest, it is considered a starting point in the pharmacotherapy of the ALS.
肌萎缩侧索硬化症(ALS)是一种以大脑皮质运动区的上运动神经元以及脑干和脊髓水平的下运动神经元选择性退化为特征的疾病。存在散发性和家族性形式,估计每10万居民中有1 - 2例发病。神经元退化的原因尚不清楚,在其他机制中,谷氨酸的兴奋性毒性被认为是神经元死亡的原因。利鲁唑是一种苯并噻唑衍生物,其神经保护机制尚未完全阐明,尽管它似乎通过阻断谷氨酸的传递来降低其神经兴奋作用。
已完成两项具有相似特征的临床试验:多中心、随机、双盲和安慰剂对照。两项研究共纳入了1100多名患者,在延长生存时间方面取得了显著的统计学结果,然而这种效果并未伴随着肌肉力量或肺功能的改善,这意味着利鲁唑并未改善患者的生活质量。该药物耐受性良好且不良反应轻微,因此在1996年,美国食品药品监督管理局(FDA)批准其上市并用于治疗ALS。利鲁唑作为治疗ALS的首个药物的批准引发了关于治疗效果和成本的大量问题。
尽管其益处不大,但它被认为是ALS药物治疗的一个起点。