Are Cell-Based Therapies Safe and Effective in the Treatment of Neurodegenerative Diseases? A Systematic Review with Meta-Analysis

Biomolecules, 2022 · DOI: 10.3390/biom12020340 · Published: February 21, 2022

Simple Explanation

This study reviews cell-based therapies for neurodegenerative diseases like multiple sclerosis (MS), spinal cord injury (SCI), Parkinson’s disease (PD), and Alzheimer’s disease (AD). These diseases involve the loss of structure and function of neurons. The review examines the safety and effectiveness of using different cell types, such as mesenchymal stromal cells (MSCs), to regenerate or protect the central nervous system. It analyzes clinical trials that tested these cell therapies in patients to see if preclinical findings could be confirmed. The study concludes that cell-based therapies are generally safe and feasible for neurodegenerative diseases, showing some promising clinical improvements. However, due to the variability in study protocols, caution is advised, and harmonization of study protocols is needed.

Study Duration

Not specified

Participants

27,043 records reviewed, 71 included in final analysis

Evidence Level

Systematic Review with Meta-Analysis

Key Findings

1
The overall frequency of serious adverse events (SAEs) after cell-based treatment was low, approximately 0.03 (95% CI: 0.01–0.08).
2
Studies in MS and SCI patients demonstrated efficacy of cell-based therapies, evaluating clinical outcomes via EDSS and AIS grade, respectively. The proportion of MS patients that improved their EDSS status during one year of follow up was 0.30 (95% CI: 0.17–0.46).
3
The proportion of SCI patients with an AIS grade improvement was 0.35 (95% CI: 0.25–0.46) during a 6–12-month follow up, and a risk-ratio analysis showed significantly higher clinical improvement in the treatment group versus control.

Research Summary

This systematic review and meta-analysis assessed the safety and efficacy of cell-based therapies for neurodegenerative diseases including MS, SCI, PD, and AD. The study analyzed data from 71 records included in the final quantitative analysis. The analysis indicated that cell therapy is generally safe and feasible, with a low frequency of serious adverse events (SAEs). Furthermore, no significant differences in SAE occurrence were observed between control and treatment groups. Clinical improvements were observed in MS and SCI patients, measured by EDSS and AIS grade, respectively, though the results require cautious interpretation due to high heterogeneity among studies.

Practical Implications

Harmonization of Study Protocols

There is a need for standardized study protocols in trials investigating cell-based therapies for neurodegenerative diseases to reduce heterogeneity and allow for better comparison of results.

Optimizing Administration Regimen

Further research is necessary to determine the optimal administration route, dose, and frequency of cell-based therapies to maximize efficacy and minimize the risk of side effects.

Further Research into Mechanisms of Action

Extensive research is required to elucidate the exact mechanisms of action, potential for immune rejection, functionality, and survival of administered cells to draw adequate conclusions regarding the efficacy of cell-based therapies.

Study Limitations

1
Heterogeneity in study design, dose, administration route, and cell type
2
Small sample sizes and lack of blinding in the majority of studies
3
Potential publication bias due to unpublished results from clinical trials.gov

Your Feedback

Was this summary helpful?

Back to Regenerative Medicine