Abstract Details

Title Mesenchymal Stem-cell Therapy in Mild-to-moderate Alzheimer’s Disease: Safety, Cognitive Efficacy, and Biomarker Signals from Controlled Human Trials

Topic Aging, Dementia, and Behavioral Neurology

Presentation(s) P9 - Poster Session 9 (5:00 PM-6:00 PM)

Poster/Presentation
Number 13-001

Objective To evaluate the safety, cognitive efficacy, and biomarker effects of mesenchymal stem-cell (MSC) therapy in adults with mild-to-moderate Alzheimer’s disease by synthesizing evidence from randomized and controlled human trials, providing an updated appraisal of therapeutic potential and guiding design priorities for future biomarker-driven phase II/III studies.

Background

Mesenchymal stem cell (MSC) therapy is an emerging disease-modifying strategy for Alzheimer’s disease (AD). We synthesized controlled and randomized human trials to appraise safety, cognitive efficacy, and biomarker effects in adults with mild-to-moderate AD.

Design/Methods

We integrated two independent evidence pulls (ClinicalTrials.gov–based and Elicit/OpenAlex–based). Eligible studies were randomized controlled trials (RCTs) or controlled clinical trials (CCTs) of MSCs (any source/route) in adults with mild-to-moderate AD that reported safety, cognition, or biomarkers. Data elements covered trial design, population, MSC product/source/route, follow-up, adverse events (AEs), cognitive outcomes (ADAS-Cog, MMSE), and biomarkers (neuroimaging, CSF/blood).

Results Eight phase I–IIa controlled trials evaluated umbilical-cord, bone-marrow, or adipose-derived MSCs delivered intravenously, intraventricularly, or intracerebrally, with follow-up of 12 weeks–12 months. Safety was primary in most. One double-blind RCT reported more AEs with MSCs (6/11 vs 3/10), including three serious events but no treatment-related deaths. Other studies (Lomecel-B/LMSCs) found MSCs safe without dose-limiting toxicity. Cognitive results were mixed: low-dose Lomecel-B improved MMSE by 2.69 points (P = 0.018) with QoL gains; laromestrocel showed a nonsignificant change (0.38; 95% CI −0.06 to 0.82) and MMSE–hippocampal correlation (r = 0.41). Biomarker data were incomplete but suggested slowed brain/hippocampal atrophy (~48–62%) and transient vascular-inflammatory effects; amyloid/tau findings were inconsistent.

Conclusions MSC therapy for mild-to-moderate AD shows an overall acceptable short-term safety profile, with one study noting higher AEs in MSC recipients. Cognitive outcomes were heterogeneous with isolated short-term gains, and imaging/biomarker signals suggest slower atrophy and vascular modulation. Evidence remains limited; larger, biomarker-driven phase II/III RCTs are needed to confirm efficacy.

Authors/Disclosures
Aditi Narsinghpura, MBBS PRESENTER	Dr. Narsinghpura has nothing to disclose.
Salma Younas, PhD, PharmD	Miss Younas has nothing to disclose.
Abhigna Mallepally, MBBS	Abhigna Mallepally, MBBS has nothing to disclose.
SANKRANTHI SARATH CHANDRA, MBBS	Dr. CHANDRA has nothing to disclose.
Aditya Jain, MBBS	Dr. Jain has nothing to disclose.
Sweta Sahu	Dr. Sahu has nothing to disclose.

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