Results and insights from a phase I clinical trial of Lomecel-B for Alzheimer's disease

Abstract

Hypothesis: We hypothesized that Lomecel-B, an allogeneic medicinal signaling cell (MSC) therapeutic candidate for Alzheimer's disease (AD), is safe and potentially disease-modifying via pleiotropic mechanisms of action.

Key predictions: We prospectively tested the predictions that Lomecel-B administration to mild AD patients is safe (primary endpoint) and would provide multiple exploratory indications of potential efficacy in clinical and biomarker domains (prespecified secondary/exploratory endpoints).

Strategy and key results: Mild AD patient received a single infusion of low- or high-dose Lomecel-B, or placebo, in a double-blind, randomized, phase I trial. The primary safety endpoint was met. Fluid-based and imaging biomarkers indicated significant improvement in the Lomecel-B arms versus placebo. The low-dose Lomecel-B arm showed significant improvements versus placebo on neurocognitive and other assessments.

Interpretation: Our results support the safety of Lomecel-B for AD, suggest clinical potential, and provide mechanistic insights. This early-stage study provides important exploratory information for larger efficacy-powered clinical trials.

Keywords: Alzheimer disease; Lomecel-B; anti-inflammatory agents; biological therapy; bone marrow mesenchymal stem cell; clinical trial; cytokines; hippocampus; human bone marrow; inflammation; inflammation mediators; interleukins; medicinal signaling cell; mesenchymal stem cell; mesenchymal stromal cell; multipotent stem cells; neuroimaging; neuroinflammatory diseases; randomized controlled trial; regenerative medicine; vascular; vascular endothelial cell growth factor.