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有望恢复瘫痪者运动功能!国家中检院发布iPSC神经细胞新药治疗脊髓损伤突破性疗效及作用机制
生物世界· 2025-09-01 08:30
Core Viewpoint - The article discusses a groundbreaking study published in the journal Cell Death & Disease, which reveals the efficacy of iPSC-derived spinal neural progenitors (spNPG) in treating spinal cord injuries, highlighting a novel therapeutic mechanism that addresses both cell replacement and microenvironment regulation [2][5][20]. Group 1: Spinal Cord Injury Overview - Spinal cord injury (SCI) affects approximately 15 million people globally, with over 3 million cases in China and 100,000 new cases annually [7]. - The injury often leads to paralysis and loss of motor and sensory functions, severely impacting the quality of life, as current treatments primarily focus on rehabilitation and supportive care [6][8]. Group 2: iPSC and spNPG Development - The research utilizes iPSC-derived spNPG cells, developed by Shizai Biomedicine, which have received dual regulatory approval from both Chinese and U.S. drug authorities for clinical trials [5][23]. - iPSCs are reprogrammed from human somatic cells, avoiding ethical issues associated with embryonic stem cells, and can differentiate into various cell types with low immunogenicity [10][11]. Group 3: Animal Study and Efficacy - In animal models of spinal cord injury, spNPG transplantation significantly improved motor function compared to control groups receiving saline [12][14]. - The study employed various behavioral tests, showing that the treatment group had a Basso animal score of 4.5, indicating the ability to walk independently but with instability, compared to 1.2 in the control group [21]. Group 4: Mechanism of Action - The therapeutic effects of spNPG are attributed to a dual mechanism: replenishing lost neural cells and improving the damaged microenvironment [15][16]. - The spNPG cells differentiate into key neural cell types, including motor neurons and interneurons, which integrate into the host's neural circuitry, facilitating signal transmission [17][18]. Group 5: Future Implications - This research represents a significant milestone in the field of spinal cord injury treatment, offering hope for patients previously deemed incurable [20][23]. - The successful transition of this technology into clinical practice could transform the treatment landscape for spinal cord injuries, making recovery a tangible reality [20].