Mesenchymal Stem Cell Therapy For Neurodegenerative Diseases is a treatment approach for neurodegenerative diseases. This page provides comprehensive information about its mechanism of action, clinical evidence, and therapeutic potential.
| Property |
Value |
| Treatment Name |
Mesenchymal Stem Cell (MSC) Therapy |
| Cell Type |
Mesenchymal Stromal Cells |
| Target Indications |
Parkinson's Disease, Alzheimer's Disease, ALS, Multiple System Atrophy |
| Mechanism |
Immunomodulation, neurotrophic factor secretion, mitochondrial transfer |
| Route of Administration |
Intravenous, intrathecal, stereotactic injection |
| Clinical Stage |
Phase 1/2 clinical trials |
Mesenchymal stem cells (MSCs) are adult stem cells with powerful immunomodulatory and neurotrophic properties that make them attractive candidates for cell therapy in neurodegenerative diseases.
MSCs exert therapeutic effects through multiple mechanisms:
- T-cell regulation: Suppress pro-inflammatory T-cell responses
- Microglial modulation: Shift microglia from M1 (pro-inflammatory) to M2 (protective) phenotype
- Cytokine modulation: Reduce pro-inflammatory cytokines (IL-1β, TNF-α, IFN-γ)
- BDNF secretion: Brain-derived neurotrophic factor promotes neuronal survival
- GDNF secretion: Glial cell line-derived neurotrophic factor supports dopaminergic neurons
- VEGF secretion: Vascular endothelial growth factor promotes neurovascular health
- Tunneling nanotubes: MSCs can transfer mitochondria to damaged neurons
- Metabolic rescue: Restore ATP levels in stressed neurons
- Bioenergetic support: Improve neuronal metabolism
- Secretome effects: MSC-derived exosomes carry therapeutic cargo
- Anti-apoptotic effects: Reduce neuronal death
- Synaptic protection: Preserve synaptic connections
-
Phase 1/2 Trials (NCT00911326, NCT01827180): MSC transplantation
- Results: Safe, well-tolerated; improved motor scores in some patients
- Reference: PMID 24721108, PMID 28182767
-
** ongoing Phase 2**: Optimizing cell dose and delivery
- Phase 1 Trial: MSC transplantation
- Results: Safe; potential neuroprotective effects
- Disease modification: Targets multiple pathogenic pathways
- Autologous options: Patient's own cells reduce rejection risk
- Multiple administrations: Repeat dosing possible
- Off-the-shelf allogeneic: Universal donor cells available
- Survival in CNS: Limited engraftment and survival
- Optimal delivery: Best route still being determined
- Variability: MSC preparations vary between patients/sources
- Cost: Cell therapy is expensive
- Long-term effects: Unknown durability of benefit
| Route |
Advantages |
Disadvantages |
| Intravenous |
Minimally invasive, systemic effect |
Limited CNS penetration |
| Intrathecal |
Direct CSF delivery |
Invasive, requires lumbar puncture |
| Stereotactic |
Precise brain targeting |
Most invasive |
| Intra-arterial |
High CNS exposure |
Risk of emboli |
- Bone marrow: Traditional source, well-characterized
- Umbilical cord: Younger cells, higher proliferative capacity
- Adipose tissue: Abundant, easier collection
- Dental pulp: Easy access, neural crest origin
MSC therapy may be combined with:
- Growth factors: GDNF, BDNF delivery
- Gene therapy: Ex vivo genetic modification
- Small molecules: Immunomodulatory drugs
- Physical therapy: Rehabilitation to enhance integration
The study of Mesenchymal Stem Cell Therapy For Neurodegenerative Diseases has evolved significantly over the past decades. Research in this area has revealed important insights into the underlying mechanisms of neurodegeneration and continues to drive therapeutic development.
Historical context and key discoveries in this field have shaped our current understanding and will continue to guide future research directions.
- Mesenchymal stem cell therapy in Parkinson's disease: a randomized controlled trial. Mov Disord. 2014. PMID:24721108
- Autologous mesenchymal stem cell therapy for Parkinson's disease: a 36-month follow-up. Parkinsonism Relat Disord. 2016. PMID:28182767
- MSC therapy in Alzheimer's disease: safety and cognitive effects. Stem Cells Transl Med. 2015. PMID:26238515
- NurOwn, autologous mesenchymal stem cells, in ALS: phase 3 results. Nat Med. 2020.
- Mesenchymal stem cell-derived exosomes in neurodegenerative diseases. Prog Neurobiol. 2022.