Ludolph Albert C, Bendotti Caterina, Blaugrund Eran, Chio Adriano, Greensmith Linda, Loeffler Jean-Philippe, Mead Richard, Niessen Heiko G, Petri Susanne, Pradat Pierre-Francois, Robberecht Wim, Ruegg Markus, Schwalenstöcker Birgit, Stiller Detlev, van den Berg Leonard, Vieira Fernando, von Horsten Stephan
Department of Neurology, University of Ulm, Germany.
Amyotroph Lateral Scler. 2010;11(1-2):38-45. doi: 10.3109/17482960903545334.
The development of therapeutics for ALS/MND is largely based on work in experimental animals carrying human SOD mutations. However, translation of apparent therapeutic successes from in vivo to the human disease has proven difficult and a considerable amount of financial resources has been apparently wasted. Standard operating procedures (SOPs) for preclinical animal research in ALS/MND are urgently required. Such SOPs will help to establish SOPs for translational research for other neurological diseases within the next few years. To identify the challenges and to improve the research methodology, the European ALS/MND group held a meeting in 2006 and published guidelines in 2007 (1). A second international conference to improve the guidelines was held in 2009. These second and improved guidelines are dedicated to the memory of Sean F. Scott.
肌萎缩侧索硬化症/运动神经元病(ALS/MND)治疗方法的研发很大程度上基于对携带人类超氧化物歧化酶(SOD)突变的实验动物的研究。然而,将在体内取得的明显治疗成功转化到人类疾病上已被证明是困难的,而且大量财政资源显然已被浪费。迫切需要针对ALS/MND临床前动物研究的标准操作程序(SOPs)。这样的SOPs将有助于在未来几年内为其他神经系统疾病建立转化研究的SOPs。为了识别挑战并改进研究方法,欧洲ALS/MND研究小组于2006年召开了一次会议,并于2007年发布了指南(1)。2009年召开了第二次国际会议以完善这些指南。这些第二次完善后的指南是为纪念肖恩·F·斯科特而制定的。