新京报记者 郭懿萌 编辑 陈晓舒 校对 杨利 从能爬楼梯到被困到轮椅上，在渐冻症患者小刘身上只花了一年时间。至今，她还记得三年前摔倒后趴 在地板上挪动的样子——狼狈、无助，尊严散落一地。 病情的急剧恶化，冻结了她对未来的期待，直到父亲在渐愈互助之家病友群里发现一则试药招募信息。 在北京，小刘第一次见到蔡磊，听到他说："你可能有救了。" 7针药物注入体内。试药结束后几个月，她竟试着站稳了几秒。如今，身体依旧止不住晃动，但她能在 屋子里扶着东西慢慢走上几步。 她是蔡磊口中"200年来最幸运的一批人"，在被称为"五大绝症之首"的渐冻症面前，药物让她的病情停 止恶化，甚至出现了些许逆转。 这场由蔡磊驱动，科学家、医生与药企汇聚的"破冰"战役，取得了阶段性的曙光。更多被"冰封"的生 命，也在漫长的寒冬里，看见了春天的可能。 "如果不试，就一点希望都没有" 一根针刺入腿部肌肉，医生来回调整针在肌肉内的位置，随后接通电流进行刺激。双腿、双臂、后背、 下巴、舌头……全身要扎十几处肌肉，以检测各区域肌肉的功能状态。 从2023年夏天开始试药的半年多时间里，小刘做了7次肌电图检查。一次完整的检查要持续三四个小 时，每次针扎进去， ...

Core Viewpoint - Haisheng Pharmaceutical has signed an innovative drug cooperation agreement with Wanbangde Pharmaceutical to collaborate on the development of treatments for amyotrophic lateral sclerosis (ALS) [1] Group 1: Agreement Details - The agreement was signed on January 12, 2026, and focuses initially on the WP205 product, which has already received orphan drug designation [1] - Haisheng Pharmaceutical will invest 150 million yuan, which will be paid in phases according to the agreement [1] - Upon successful collaboration, Haisheng Pharmaceutical will receive 15% of the global commercialization revenue from the WP205 product [1] Group 2: Future Collaboration - The partnership will later expand to include the development of small molecule cyclic peptide agonists, involving both raw materials and formulations [1] - The agreement is subject to approval by the shareholders' meeting of Wanbangde Pharmaceutical Holdings Group Co., Ltd. [1]

Core Insights - The article highlights the personal and professional journey of Duan Rui, who transitioned from an accounting career to become a live-streaming entrepreneur to fund research for her husband Cai Lei, a well-known ALS (Amyotrophic Lateral Sclerosis) patient in China [2][4][16] - It emphasizes the challenges faced in the live-streaming e-commerce industry, where success is heavily reliant on data and performance metrics, and the pressure to generate revenue for ongoing research is immense [5][6][12] Group 1: Personal Journey - Duan Rui's husband, Cai Lei, was diagnosed with ALS six years ago, prompting her to invest all resources into combating the disease, which she views as her "last entrepreneurial venture" [2][14] - Duan Rui's transition to live-streaming was driven by the need to raise funds for ALS research, as traditional funding avenues were insufficient [4][16] - The emotional toll of Cai Lei's deteriorating health is evident, as Duan Rui balances her roles as a caregiver, entrepreneur, and mother [10][21] Group 2: Industry Challenges - The live-streaming e-commerce sector is characterized by high pressure and a lack of safety nets, where success is binary, and brands often overlook smaller players due to performance metrics [5][6] - Duan Rui faces significant challenges in attracting brand partnerships, as many brands prioritize data-driven results over social causes [5][12] - The funding landscape for rare disease research, particularly ALS, is difficult, with many investors hesitant to support projects due to the high failure rates associated with drug development [16][14] Group 3: Research and Development - Cai Lei's efforts have significantly accelerated ALS drug development in China, with over 27 clinical pipelines initiated from late 2020 to early 2023, representing a 20-fold increase in research activity [14][16] - Despite the progress, the future of ALS drug development remains uncertain, heavily reliant on continuous funding and the ability to sustain research efforts [16][21] - The article underscores the importance of perseverance in the face of adversity, as both Duan Rui and Cai Lei continue to push for advancements in ALS treatment despite the challenges [14][21]

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].