Safety and Exploratory Efficacy Study of NEUROSTEM® Versus Placebo in Patients With Alzheimer's Disease

Summary

This combined phase 1/2a clinical trial is to investigate the safety, dose limiting toxicity (DLT), and exploratory efficacy of three repeated intraventricular administrations of NEUROSTEM® (human umbilical cord blood-derived mesenchymal stem cells) versus placebo via an Ommaya reservoir at 4 week intervals in patients with Alzheimer's disease.

Timeline

Start

2014-03

Primary completion

2019-12

Completion

2019-12

Publications

• Myeong SH, Lee NK, Lee NH, Choi SJ, Son HJ, Chang JW, Kim HJ, Na DL. Intracerebroventricular human mesenchymal stem cells induce MMP9-driven transient inflammation in Alzheimer's disease. Stem Cell Res Ther. 2026 Mar 11;17(1):145. doi: 10.1186/s13287-026-04958-x.
• Lee NK, Jang H, Choi Y, Hwangbo S, Lee S, Lee JI, Kim YJ, Chin J, Chang JW, Seo SW, Son HJ, Choi SJ, Na DL, Kim HJ. Mesenchymal Stem Cells With Adjuvant Dexamethasone in Patients With Alzheimer's Disease: A Phase IIa Trial. Dement Neurocogn Disord. 2025 Oct;24(4):272-285. doi: 10.12779/dnd.2025.24.4.272. Epub 2025 Oct 24.
• Choi Y, Shin S, Son HJ, Lee NH, Myeong SH, Lee C, Jang H, Choi SJ, Kim HJ, Na DL. Identification of potential biomarkers related to mesenchymal stem cell response in patients with Alzheimer's disease. Stem Cell Res Ther. 2023 Jul 19;14(1):178. doi: 10.1186/s13287-023-03410-8.
• Kim HJ, Cho KR, Jang H, Lee NK, Jung YH, Kim JP, Lee JI, Chang JW, Park S, Kim ST, Moon SW, Seo SW, Choi SJ, Na DL. Intracerebroventricular injection of human umbilical cord blood mesenchymal stem cells in patients with Alzheimer's disease dementia: a phase I clinical trial. Alzheimers Res Ther. 2021 Sep 14;13(1):154. doi: 10.1186/s13195-021-00897-2.

Drugs

Indications