Results and insights from a phase I clinical trial of Lomecel‐B for Alzheimer's disease. Issue 1 (31st March 2022)

• Record Type: Journal Article
• Authors: Brody, Mark; Agronin, Marc; Herskowitz, Brad J.; Bookheimer, Susan Y.; Small, Gary W.; Hitchinson, Benjamin; Ramdas, Kevin; Wishard, Tyler; McInerney, Katalina Fernández; Vellas, Bruno; Sierra, Felipe; Jiang, Zhijie; Mcclain‐Moss, Lisa; Perez, Carmen; Fuquay, Ana; Rodriguez, Savannah; Hare, Joshua M.; Oliva, Anthony A.; Baumel, Bernard
• Is Part Of: Alzheimer's & dementia. Volume 19:Issue 1(2023)
• Page Start: 261
• Page End: 273
• Publication Date: 2022-03-31
• Subjects: Alzheimer disease -- anti‐inflammatory agents -- biological therapy -- bone marrow mesenchymal stem cell -- clinical trial -- cytokines -- hippocampus -- human bone marrow -- inflammation -- inflammation mediators -- interleukins -- Lomecel‐B -- 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; Alzheimer's disease -- Periodicals; Alzheimer Disease -- Periodicals; Dementia -- Periodicals; Démence; Maladie d'Alzheimer; Périodique électronique (Descripteur de forme); Ressource Internet (Descripteur de forme); 616.83
• DOI: 10.1002/alz.12651
• Languages: English
• ISSNs: 1552-5260

Abstract

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.