第一章 “美女病”与奥巴捷

因为患有多发性硬化,天才大提琴家杰奎琳·杜普蕾(Jacqueline du Pré)28岁便不得不离开心爱的音乐舞台,42岁溘然辞世,她的故事后来被改编成电影《她比烟花还寂寞》(Hilary and Jackie)。多发性硬化是一种因自身免疫系统病变引起的大脑神经髓鞘的破损和剥落,患者表现出多种神经性疾病的症状,如肢体麻木、肌肉痉挛和抽搐等,严重影响正常的生活与工作。因为患者人群中20~40岁年轻女性的人数远远高于男性,所以它又被称为“美女病”。小分子口服药物奥巴捷(Aubagio)于2012年获得FDA(美国食品药品监督管理局)批准,2013年获得欧盟批准,它通过对患者免疫系统的调控,延缓和阻止神经髓鞘的进一步衰变。

Because of Multiple Sclerosis (MS), virtuoso Cellist Jacqueline du Pré left her beloved music performance stage at the young age of 28, and passed away at the age of 42. Her sad story was adapted to an Oscar nominated movie Hilary and Jacky. MS is a seriously debilitating autoimmune disease, which causes damages to myelin sheath of nerve fibers in the patients' brain, and has typical symptoms of neurological diseases, such as numbness of limbs, muscle twitching and spasms, etc. Since MS affects woman aged 20—40 much more than any other age and sex groups, it is often referred to as “pretty women's disease.” Luckily, a small molecule oral drug Aubagio is now available to MS patients. Through down regulation of related immuno-reactivities, Aubagio inhibits the further damage to myelin sheath of nerve fibers.