A first-in-human, prospective pilot trial of umbilical cord-derived mesenchymal stem cell eye drops therapy for patients with refractory non-Sjögren's and Sjögren's syndrome dry eye disease

作者：Di Zhang, Taige Chen, Qi Liang, Xuebing Feng, Jiaxuan Jiang, Zeying Chen, Yun Tang, Yiran Chu, Bin Wang, Kai Hu · 发表于：Stem Cell Research & Therapy · 年份：2025 · DOI：10.1186/s13287-025-04292-8 · 被引用次数：15 · 研究领域：Ocular Surface and Contact Lens、Salivary Gland Disorders and Functions、Corneal Surgery and Treatments

BACKGROUND: Patients with refractory dry eye disease (DED) often face the threat of diminished visual quality and have limited responses to existing treatments. Ocular injection of Mesenchymal stem cells (MSCs) has recently emerged as a promising new therapeutic strategy for DED. Topical eye drops are the clinical favorable choice for drug administration in DED. To date, the clinical use of MSC eye drops has not been reported in settings. This clinical trial represents a groundbreaking exploration into the preliminary therapeutic potential and safety of umbilical cord MSC eye drops for patients with refractory DED, including both non-Sjögren's dry eye (NSDE) and Sjögren's syndrome dry eye (SSDE). The study also aimed to investigate the possible underlying mechanisms. METHODS: In this open-label, prospective, single-arm, self-controlled trial, 11 NSDE and 5 SSDE patients received twice-daily MSC eye drops for two weeks, subsequent follow-up visits were scheduled at 4 weeks and 12 months after treatment. The primary efficacy was evaluated using the ocular surface disease index (OSDI) score, tear meniscus height (TMH), non-invasive break-up time (NIBUT), Schirmer I test (SIT), and corneal fluorescein staining (CFS) score. Secondary assessments focused on the evaluation of lipid layer, meibomian gland function, and bulbar conjunctival redness. Safety was monitored by recording adverse events (AEs) throughout the study. Changes in tear levels of interleukin-6 (IL-6), IL-17A, Mucin...