A recent clinical trial has shown encouraging results for the use of human bone marrow-derived clonal mesenchymal stem cells (hcMSCs) in treating moderate to severe atopic dermatitis (AD). The study indicated that patients receiving hcMSCs experienced a significantly higher rate of achieving a 50% reduction in the Eczema Area and Severity Index (EASI50) at 12 weeks compared to the control group, prompting researchers to call for further large-scale studies.
Background
Previous research has established the safety and immunomodulatory potential of hcMSCs in various conditions. For instance, hcMSCs have been shown to reduce inflammatory markers in acute pancreatitis patients. In preclinical studies involving a mouse model of AD, hcMSCs were found to regulate key biomarkers responsible for immune inflammatory responses. This recent study is the first human trial to assess the safety, tolerability, and efficacy of hcMSCs in patients with moderate to severe AD.
Methods
The trial (NCT04179760) was conducted in two phases. Phase 1 was a randomized, open-label trial involving 20 patients across six sites in Korea. Participants received either a high dose (1×10^6 cells/kg) or a low dose (5×10^5 cells/kg) of hcMSCs via three intravenous infusions every two weeks. Safety was closely monitored, and upon confirmation of no serious adverse effects, additional patients were enrolled.
Phase 2 expanded to 72 patients with moderate to severe AD from 15 sites, using only the low-dose hcMSCs in a double-blind, placebo-controlled setup. Infusions were administered over three weeks, with follow-ups extending over 24 weeks. Rescue therapy was allowed for patients with intolerable AD symptoms, beginning with topical medications and escalating to stronger treatments under medical supervision, excluding oral corticosteroids.
Results
The primary goal in Phase 1 was to assess EASI50 response rates, which were 70% for the high-dose group and 67% for the low-dose group, with no significant difference between the two. In Phase 2, the low-dose hcMSCs group demonstrated a higher EASI50 response rate (58%) compared to the placebo group (32%) at week 12, a statistically significant difference.
Secondary endpoints in Phase 2 included higher rates of EASI90 responses (24% vs. 6%), quicker achievement of EASI75, and improvements in Investigator Global Assessment (IGA) scores and affected Body Surface Area (BSA) with hcMSCs compared to placebo. However, changes in pruritus and quality of life scores did not significantly differ between groups, nor did serum biomarker levels.
The safety profile indicated that adverse events (AEs) were generally mild to moderate, with infections being the most common. In Phase 1, AEs affected 10% of patients in the high-dose arm and 30% in the low-dose arm. In Phase 2, AEs affected 28% of the hcMSCs group and 22% of the placebo group, with most resolving by the end of the study.
Conclusion
The study demonstrated that low-dose hcMSCs effectively improved AD symptoms and had a manageable safety profile compared to placebo, supporting their potential as a treatment option for moderate to severe AD. Researchers suggest that a larger-scale, more comprehensive evaluation is necessary to determine the long-term sustainability of these effects.
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