在蔡磊还没丧失语言能力时，他总是宽慰妻子，"不会失败""你太累了"。现在他病情恶化，不得不搬到医院附近被专人照护。渐冻症是世界五大绝症之 首，没有医生能提供蔡磊病情进展的时间表，包括直播间里的观众在内，所有人都知道他的情况只会更坏。 对于段睿，"救命"的念想早已渗进心里，"你们被困住了，但这个屋子里有一把钥匙，把它找到了就可以不死了，你会怎么样？你一定得去找钥匙啊！你 即使在死前找不到，也会让后面的人，快些找到"。 直播，没有绿灯的新世界 段睿身上有很多反差。 每经记者｜林姿辰 可杨 每经编辑｜陈俊杰 北京又一年的冬天开始了。2025年立冬第二天，段睿去了天坛医院附近，院门口两侧的树叶子还有秋天的感觉，红黄绿夹杂在一起，在风声呼啸中轻轻摇 曳，像丈夫蔡磊现在的样子。 6年前，蔡磊确诊渐冻症，次年决定倾尽所有资源投入攻克该病，将此视为"最后一场创业"。于是，"中国电子发票第一人"、京东集团前副总裁蔡磊，成 为中国最知名的渐冻症抗争者，有人称他为让中国渐冻症药物研发提速的"孙悟空"——与其等死，不如战斗，纵使不敌，也绝不屈服。 为了给科研团队提供巨额经费，2022年，段睿做出一个决定，她离开了自己的会计师事务所， ...

Core Insights - The ALS (Amyotrophic Lateral Sclerosis) drug development landscape in China is evolving, with significant advancements in research and clinical trials, particularly in the context of funding challenges faced by startups in this field [1][6][8] Group 1: Drug Development Progress - The approval of Tofersen, the first "cause-targeted" ALS drug, in China marks a significant milestone, filling a gap for SOD1-ALS patients and aligning domestic treatment options with international standards [2][3] - Multiple ASO therapies, including Ulefnersen and siRNA therapies like RAG-17 and RAG-21, are in early clinical stages, showcasing the potential of nucleic acid-based treatments for ALS [3][4] - Domestic companies are increasingly becoming the main drivers of innovation in ALS drug development, with 15 drug development projects reported since 2025, excluding IIT research [4] Group 2: Funding Challenges - The ALS drug development sector is primarily composed of startups, which face significant funding challenges due to the limited commercial value of rare diseases and the high risk of early-stage research failures [5][6] - Despite the overall interest in innovative drugs, the financing environment for ALS remains cold, with only a few companies achieving significant funding rounds [7][8] - Companies like Shenzhi Changhua and Zhongmei Ruikang, which have advanced drugs to clinical stages, still encounter financial pressures, highlighting the common funding challenges in rare disease drug development [7][8] Group 3: Research and Regulatory Recommendations - The complexity of ALS, including unclear etiology and heterogeneous patient populations, complicates drug development, necessitating a robust support system for rare disease research [9][10] - Recommendations for improving the regulatory landscape include enhancing orphan drug recognition processes, flexible clinical trial designs, and extending market exclusivity periods to ensure adequate returns on investment [10] - Collaborative efforts are suggested to establish national rare disease patient registries and biobanks, as well as integrating international resources to create a unified disease database for ALS research [10]

Core Insights - The ALS (Amyotrophic Lateral Sclerosis) drug development landscape in China is evolving, with significant advancements in research and clinical trials, but challenges remain in funding and commercialization [1][2][9] Group 1: Drug Development Progress - The first "cause-targeted" ALS drug, Tofersen, has been approved in China, filling a gap for SOD1-ALS patients and aligning domestic treatment options with international standards [2][3] - Multiple ASO (Antisense Oligonucleotide) therapies are in early clinical stages, including Ionis Pharmaceuticals' Ulefnersen, and domestic companies like Zhongmei Ruikang are developing siRNA therapies [3][4] - Since 2025, there have been numerous drug development advancements in the ALS field in China, with at least 15 new drug candidates reported [6][7] Group 2: Funding Challenges - The ALS drug development sector is primarily driven by small and medium-sized enterprises, facing significant funding challenges due to the high costs and risks associated with early-stage research [9][10] - Initial funding for ALS projects is often reliant on external investments, as many startups lack mature products to generate revenue [10][11] - Despite the overall interest in innovative drugs, the ALS sector has seen limited large-scale financing, with only a few companies achieving significant funding rounds [10][12] Group 3: Research and Regulatory Environment - The complexity of ALS, including unclear etiology and heterogeneous patient populations, complicates drug development and increases the risk of early-stage failures [11][12] - Companies are advocating for improved regulatory frameworks to expedite drug approvals and support for rare disease research, including tax incentives and the establishment of specialized funds [12][13] - Collaborative efforts are suggested to create national registries and databases to enhance research and data sharing among stakeholders [12][13]

Core Insights - The breakthrough in single-gene ALS (Amyotrophic Lateral Sclerosis) treatment has been achieved, allowing some patients who were previously considered hopeless to survive, showcasing the power of Chinese technology and societal support [1][6]. Group 1: Patient Advocacy and Research Progress - The individual, Cai Lei, has been fighting ALS for six years since his diagnosis in 2019, and he has taken on the challenge of overcoming this previously untreatable disease [2][3]. - Cai Lei has successfully mobilized funding and support from various sectors, leading to the advancement of 10 drug pipelines into clinical stages in 2024 [6]. - The establishment of the "Jianyu Mutual Aid Home" has reached 15,000 ALS patients, demonstrating a significant community impact [6]. Group 2: Clinical Developments and Innovations - A drug targeting specific genetic types of ALS has entered Phase I clinical trials, marking a significant step forward in treatment options [6]. - The use of AI technology has accelerated the identification of new treatment targets and potential drugs, indicating a shift towards innovative approaches in ALS research [10]. Group 3: Personal Impact and Recognition - Cai Lei received the title of "National Self-Reliance Model," which he views as encouragement not only for himself but also for the broader ALS community [8]. - Despite the advancements, Cai Lei's specific condition does not fall under the newly targeted genetic types, highlighting the ongoing challenges faced by many patients [6].