Breaking News:Stem Cell Eye Drops Improve Refractory Sjögren’s Dry Eye– What Just Happened

NEW early-phase clinical data suggest that eye drops derived from umbilical cord mesenchymal stem cells (MSCs) may offer a novel treatment option for patients with refractory dry eye disease (DED), including those with Sjögren’s syndrome who have limited responses to existing therapies.

In this first-in-human, prospective pilot study, researchers evaluated the safety and preliminary efficacy of topical MSC eye drops in patients with severe, treatment-resistant dry eye. While MSC-based therapies have previously been explored through ocular injections, this study is the first to assess MSCs delivered via eye drops, a route considered more practical and patient-friendly for chronic ocular conditions.

Testing a Novel Topical Therapy

The open-label, single-arm trial enrolled 16 patients, including 11 with non-Sjögren’s dry eye (NSDE) and five with Sjögren’s syndrome–associated dry eye (SSDE). All participants received MSC eye drops twice daily for two weeks and were followed at 4 weeks and again at 12 months after treatment.

Clinical efficacy was assessed using both patient-reported and objective measures, including the ocular surface disease index (OSDI), tear meniscus height, non-invasive tear break-up time, Schirmer I test scores, and corneal fluorescein staining. Additional assessments examined meibomian gland function, lipid layer quality, and conjunctival redness. Safety was monitored throughout the study period.

Improvements in Tear Production and Ocular Surface Health

Following treatment, patients in both NSDE and SSDE groups experienced significant improvements in several key clinical parameters. Notably, tear production increased, as reflected by higher Schirmer test values and tear meniscus height, alongside reduced meibomian gland obstruction. Improvements in symptoms and tear film stability were more pronounced in patients with non-Sjögren’s dry eye, although patients with Sjögren’s syndrome also demonstrated measurable benefit.

Importantly, no serious adverse events were reported during treatment or long-term follow-up, supporting a favourable initial safety profile for topical MSC administration.

Signals of Immune Modulation

Beyond clinical outcomes, the study explored potential biological mechanisms. Tear analysis revealed significant reductions in inflammatory cytokines interleukin-6 and interleukin-17A after treatment, alongside increased levels of MUC5AC, a mucin essential for tear film stability. Proteomic analyses further suggested that MSC eye drops may exert their effects by suppressing inflammation driven by T helper 17 cells, a pathway implicated in both Sjögren’s syndrome and severe dry eye.

Looking Ahead

The authors describe this study as an exploratory step rather than definitive evidence, emphasising the small sample size and lack of a control group. Nonetheless, the combination of symptomatic improvement, objective ocular surface changes, and favourable safety signals positions MSC eye drops as a potentially transformative approach for refractory dry eye disease.

Larger, randomised controlled trials will now be needed to confirm efficacy, compare outcomes with existing treatments, and define which patient subgroups may benefit most. If validated, MSC-based eye drops could represent a new therapeutic avenue for patients whose dry eye remains difficult to control with current options.

Reference

Zhang D et al. 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. Stem Cell Res Ther. 2025;16(1):202.