Seta Yasuhiro, Kimura Konomi, Masahiro Goto, Tatsumori Kimiko, Murakami Yasufumi
Hitonowa Medical, Chiyoda 102-0085, Japan.
Kokoro to Karada Clinic, Ichikawa 272-0133, Japan.
Biomedicines. 2024 Sep 26;12(10):2193. doi: 10.3390/biomedicines12102193.
BACKGROUND/OBJECTIVES: Amyotrophic lateral sclerosis (ALS) is a progressive and irreversible neurodegenerative disease with limited treatment options. Advances in regenerative medicine have opened up new treatment options. The primary and exploratory objectives of this retrospective cohort study were to evaluate the safety and efficacy of stem cells from human exfoliated deciduous teeth-conditioned media (SHED-CM).
Safety assessments included adverse events, vital signs, and laboratory test changes before and after administration, and efficacy was measured using the ALS Functional Rating Scale-Revised (ALSFRS-R), grip strength, and forced vital capacity in 24 patients with ALS treated at a single facility between 1 January 2022, and 30 November 2023.
While ALSFRS-R scores typically decline over time, the progression rate in this cohort was slower, suggesting a potential delay in disease progression. Alternatively, improvements in muscle strength and mobility were observed in some patients. Although adverse events were reported in only 3% of cases (no serious allergic reactions), the treatment-induced changes in vital signs and laboratory results were not clinically significant.
The SHED-CM treatment is a safe and potentially effective therapeutic option for patients with ALS. Further research is needed to optimize the SHED-CM treatment; however, this study lays the groundwork for future exploration of regenerative therapies for ALS.
背景/目的：肌萎缩侧索硬化症（ALS）是一种进行性且不可逆的神经退行性疾病，治疗选择有限。再生医学的进展开辟了新的治疗途径。这项回顾性队列研究的主要和探索性目标是评估人脱落乳牙干细胞条件培养基（SHED-CM）的安全性和有效性。
安全性评估包括不良事件、生命体征以及给药前后的实验室检查变化，并使用ALS功能评定量表修订版（ALSFRS-R）、握力和用力肺活量对2022年1月1日至2023年11月30日期间在单一机构接受治疗的24例ALS患者进行疗效评估。
虽然ALSFRS-R评分通常会随着时间下降，但该队列中的疾病进展速度较慢，表明疾病进展可能有所延迟。此外，在一些患者中观察到肌肉力量和活动能力有所改善。虽然仅3%的病例报告了不良事件（无严重过敏反应），但治疗引起的生命体征和实验室检查结果变化在临床上并不显著。
SHED-CM治疗对于ALS患者是一种安全且可能有效的治疗选择。需要进一步研究以优化SHED-CM治疗；然而，本研究为未来探索ALS的再生疗法奠定了基础。
