本文转自【央视新闻微信公众号】； "渐冻症必然被攻克！这只是时间问题。" 1月1日，渐冻症患者蔡磊借助眼控仪"写"下了一封致渐冻症病友的公开信，同时附上了数位渐冻症患者 的自拍视频，其中有人的健康状态已经得到改善。 △渐冻症患者的自拍视频（来源：蔡磊微博） 蔡磊介绍，这是渐愈互助之家平台数年来与中国科学家合作推进SOD1等基因类型渐冻症的治疗药物， 实现了领先世界的临床前和临床疗效。这些患者只需要几次使用，每次几分钟，就可能产生改写生命的 颠覆性效果。 "对他们来说，渐冻症这个200年的恶魔已被'秒杀'！几年前，这一切不可能，甚至不敢想，现在已是现 实。渐冻症的历史其实已被改写。"蔡磊写道。 根据身体功能评分（ALSFRS-R），与渐冻症抗争6年的蔡磊已从总分48分降至个位数，进入疾病的终 末期。蔡磊描述，自己的大脑无比清醒看着身体像磁铁一样被禁锢的动弹不得，时刻疼痛酸楚、麻木难 忍、窒息憋闷，却无法呼喊表达，比植物人还要残酷，连呼吸都成为一种奢望。 "目前全世界依然没有看到可以阻止病情或逆转改善的药物和办法（基因类型除外），我们似乎有痛苦 和悲观的充分理由。"但蔡磊表示了充分的自信，"这是一个科技爆发的时代 ...

1月1日，渐冻症患者、京东前副总裁蔡磊发布致所有渐冻症病友的公开信。 00:33 蔡磊表示，近期自己的身体状况受到了很多关注，身体功能评分（ALSFRS-R）已从总分48分降至个位 数，进入疾病的终末期，非常艰难，以至于很多病友甚至身边团队也丧失了很大信心，不少同事陷入悲 观情绪。 "我的家人也更加痛苦，不知道哪天就会失去我。确实，渐冻症无比残酷，四肢瘫痪也只是噩梦的开始 而已，当它侵蚀到颈部、喉部、头部，带来语言丧失、吞咽困难、呼吸衰竭，那才是真正的恐怖。大脑 无比清醒看着身体像磁铁一样被禁锢的动弹不得，时刻疼痛酸楚、麻木难忍、窒息憋闷，却无法呼喊表 达，比植物人还要残酷，连呼吸都成为一种奢望。"蔡磊表示，目前全世界依然没有看到可以阻止病情 或逆转改善的药物和办法。 尽管如此，对于攻克渐冻症，蔡磊表示充分的信心和希望，他以渐冻症患者生命被拯救的真实记录举 例，目前渐愈互助之家平台数年来与中国科学家合作推进SOD1等基因类型渐冻症的治疗药物已经实现 领先世界的临床前和临床疗效，归功于全世界科学家、药企、医生、患者的共同努力，也离不开国家开 明政策和社会爱心人士的支持。 "我依然坚持每天阅读数十个病友群的信息， ...

专题：2026新年致辞 企业家展望2026新趋势 "没有遇到蔡总，我老婆早就不在这个世界上了。NFL（神经丝轻链蛋白）从刚开始的218.4直接降到了 现在的10，差不多降了95%以上。" "医生都不相信，我们在（完全依赖）呼吸机辅助的情况下，现在最长时间可以脱机3-4分钟。" "他是我的救命恩人，因为有了蔡总才有我的今天。用药之前（NFL）接近80，现在只有9点多了。" "以前没用药的时候下滑比较厉害，走路拖地了，脚也就抬个10公分。用过药现在抬腿能抬个三四十公 分。而且庆幸它没有继续发展，有往好的地方发展。肺功能慢慢回升了，接近90了。" "用药以前他讲话只有我们家里人能猜出来，到现在基本上口齿伶俐。最让我们家人惊讶的是，他腿部 重新有力气了，在搀扶下可以走5-10分钟，这是三年来第一次重新站起来。如果不是遇到蔡总，自己的 坟头草可能长得都已经很高了。感谢蔡总和科学家推进的这款药物，真的把我父亲从鬼门关里拉回 来！" 炒股就看金麒麟分析师研报，权威，专业，及时，全面，助您挖掘潜力主题机会！ 来源：渐愈互助之家 原创：蔡磊 "200年的罕见病，医学都没有办法。用了八九个月，我就开始不发展，就把我高兴的呀，后面 ...

Core Viewpoint - The innovative drug Tofersen injection for treating SOD1-ALS has officially launched in China, marking a significant advancement in the treatment of this rare and fatal neurological disease [1][2]. Group 1: Drug and Treatment Details - Tofersen injection is the first disease-modifying treatment for SOD1-ALS, providing a new hope for patients suffering from this condition [1][2]. - The drug is an antisense oligonucleotide (ASO) that reduces the synthesis of the toxic SOD1 protein, thereby alleviating damage to motor neurons and slowing disease progression [2][3]. - The drug was approved in September 2022 for adult patients with SOD1 gene mutations, addressing a significant unmet need in the treatment of ALS [2][3]. Group 2: Patient and Clinical Insights - The average onset age for SOD1-ALS patients is around 50 years, with a typical presentation of lower limb onset and simultaneous damage to upper and lower motor neurons [3]. - There is an urgent demand for targeted therapies among patients due to the fatal nature of the disease [3]. - The importance of genetic testing for all ALS patients, including those with SOD1 mutations, is emphasized for accurate diagnosis and treatment [2][3]. Group 3: Advocacy and Future Directions - Experts call for the standardization of genetic testing and treatment protocols to ensure timely access to this breakthrough therapy for eligible patients [3]. - There is a collective appeal from advocacy groups to raise awareness and improve access to innovative therapies for rare disease populations [3].

Core Viewpoint - The article discusses the promising results of a clinical trial that tested low-dose IL-2 as an add-on therapy to Riluzole for treating Amyotrophic Lateral Sclerosis (ALS), indicating that modifying the immune system may effectively slow disease progression and reduce mortality risk [2][4][9]. Group 1: Disease Overview - Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig's disease, is a rare neurodegenerative disorder characterized by the progressive loss of motor neurons, leading to severe disability and a median survival time of only 2-3 years from symptom onset [1]. - Current treatment options are limited, with Riluzole being the only FDA-approved drug that can extend survival by only a few months, highlighting the urgent need for more effective therapies [1]. Group 2: Clinical Trial Details - A phase 2b, double-blind, randomized, placebo-controlled trial was conducted to evaluate the efficacy and safety of low-dose IL-2 as an adjunct therapy to Riluzole in ALS patients [2][4][5]. - The trial included participants aged 18-76 with a symptom duration of ≤24 months and a forced vital capacity of ≥70%, who had not previously received Riluzole treatment [5]. Group 3: Trial Results - Out of 304 screened ALS patients, 220 met the randomization criteria after a 12-18 week Riluzole monotherapy period, with a 62% male and 38% female distribution [6]. - The primary endpoint analysis indicated a 19% reduction in mortality risk for the IL-2 LD group, although this was not statistically significant; however, a subsequent adjusted analysis showed a significant 68% reduction in mortality risk [6][7]. - The treatment was found to be safe, with significant increases in Treg cells and decreases in plasma CCL2 levels observed at all time points [6][8]. Group 4: Implications for Treatment - The findings suggest that low-dose IL-2 could be considered a safe and well-tolerated treatment option for ALS, enhancing the effects of Riluzole and potentially improving patient survival rates [2][9].