Alarcan Hugo, Al Ojaimi Yara, Lanznaster Debora, Escoffre Jean-Michel, Corcia Philippe, Vourc'h Patrick, Andres Christian R, Veyrat-Durebex Charlotte, Blasco Hélène
Laboratoire de Biochimie et Biologie Moleculaire, CHRU Bretonneau, 2 Boulevard Tonnellé, 37000 Tours, France.
UMR 1253 iBrain, Université de Tours, Inserm, 10 Boulevard Tonnellé, 37000 Tours, France.
J Pers Med. 2022 Jun 29;12(7):1071. doi: 10.3390/jpm12071071.
Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disorder that still lacks an efficient therapy. The barriers between the central nervous system (CNS) and the blood represent a major limiting factor to the development of drugs for CNS diseases, including ALS. Alterations of the blood-brain barrier (BBB) or blood-spinal cord barrier (BSCB) have been reported in this disease but still require further investigations. Interestingly, these alterations might be involved in the complex etiology and pathogenesis of ALS. Moreover, they can have potential consequences on the diffusion of candidate drugs across the brain. The development of techniques to bypass these barriers is continuously evolving and might open the door for personalized medical approaches. Therefore, identifying robust and non-invasive markers of BBB and BSCB alterations can help distinguish different subgroups of patients, such as those in whom barrier disruption can negatively affect the delivery of drugs to their CNS targets. The restoration of CNS barriers using innovative therapies could consequently present the advantage of both alleviating the disease progression and optimizing the safety and efficiency of ALS-specific therapies.
肌萎缩侧索硬化症（ALS）是一种毁灭性的神经退行性疾病，目前仍缺乏有效的治疗方法。中枢神经系统（CNS）与血液之间的屏障是包括ALS在内的中枢神经系统疾病药物研发的主要限制因素。已有报道称，这种疾病存在血脑屏障（BBB）或血脊髓屏障（BSCB）的改变，但仍需进一步研究。有趣的是，这些改变可能与ALS复杂的病因和发病机制有关。此外，它们可能会对候选药物在大脑中的扩散产生潜在影响。绕过这些屏障的技术正在不断发展，可能为个性化医疗方法打开大门。因此，识别血脑屏障和血脊髓屏障改变的可靠且非侵入性标志物，有助于区分不同亚组的患者，例如那些屏障破坏会对药物向中枢神经系统靶点递送产生负面影响的患者。使用创新疗法恢复中枢神经系统屏障，可能会带来缓解疾病进展以及优化ALS特异性疗法安全性和有效性的双重优势。
