The Science
Stem Cell Treatment & The FDA
Like all cells in the body, brain and spinal cord cells suffer from damage and eventually die over time. At its extreme, the damage and death of brain and spinal cord cells can lead to a myriad of neurological conditions and diseases.
In the area of neurological diseases, there are few conditions where FDA-approved disease-modifying therapies exist.
Among those that do exist, all have limited efficacy to impact the disease trajectory of the conditions they are intended to treat.
Also, the current FDA-approved disease-modifying treatments for neurological disease are associated with varying degrees of toxicity and risk, which may, on an individual level, deter an affected person from accepting one of these treatments.
Stem cell treatments hold the promise of therapeutic benefit for neurological conditions based on the currently published literature.
At present, the only stem cell products that are FDA-approved for use in the United States consist of blood-forming stem cells derived from umbilical cord blood.
These products are approved for use in patients with disorders that affect blood production, but they are not approved for other conditions.
Here is a list of references grouped by disease type, highlighting the therapeutic potential of mesenchymal stem cells (MSCs) for Alzheimer's disease, Parkinson's disease, ALS, and multiple sclerosis:
Alzheimer's Disease
1. Mesenchymal stromal cells for the treatment of Alzheimer's disease
- This review discusses MSCs' immunomodulatory, anti-inflammatory, regenerative, antioxidant, anti-apoptotic, and neuroprotective effects in Alzheimer's disease. MSCs promote neuron survival, improve microglial clearance of protein aggregates, and enhance bioenergetic profiles[1].
2. Stem Cell Therapy for Alzheimer's: Findings and Treatment
- Research highlights include reduced brain abnormalities in mouse models and improved memory in AD cell models. Clinical trials with human umbilical cord-derived MSCs demonstrated safety and potential efficacy[2].
3. Clinical safety and efficacy of allogenic human adipose MSCs-Exos
- A phase I/II clinical trial showed that intranasal administration of MSC-secreted exosomes improved cognitive function in mild to moderate Alzheimer's patients without adverse events[3].
Parkinson's Disease
1. Effects of mesenchymal stem cells on dopaminergic neurons
- A meta-analysis revealed that MSCs improved motor function, preserved dopaminergic neurons, and showed neuroprotective effects in Parkinson's disease animal models. Bone marrow-derived MSCs demonstrated the largest effect size[4].
2. Phase II Trial of Bone Marrow-Derived Mesenchymal Stem Cells
- This trial explores intravenous MSC infusions to modulate immune response and promote brain cell regeneration, aiming to slow disease progression in Parkinson’s patients[5].
Amyotrophic Lateral Sclerosis (ALS)
1. A meta-analysis of stem cell therapy in ALS
- Preclinical studies indicated that MSC transplantation attenuates neuroinflammation, improves motor performance, and extends survival in ALS models. However, optimal protocols for clinical application remain unclear[6].
2. Stem Cells: ALS Treatment Breakthrough
- Clinical trials suggest that MSC transplantation may delay disease onset and progression while improving muscle strength and motor function in ALS patients. Mixed results highlight the need for further research[7].
Multiple Sclerosis (MS)
1. Recent advances in mesenchymal stem cell therapy for multiple sclerosis
- Studies demonstrated that menstrual blood-derived MSCs (MB-MSCs) and umbilical cord-derived MSCs (UC-MSCs) reduce MS-related inflammation and promote remyelination. Co-administration with immunomodulatory drugs like rapamycin enhanced therapeutic outcomes[8].
2. Systemic administration of MSCs in MS rodent models
- MSC treatments increased neural progenitor markers, induced neuroprotection, and enhanced myelination while suppressing immune cell activity in experimental autoimmune encephalomyelitis (EAE) models[8].
This compilation underscores the promising role of MSCs across neurodegenerative diseases, though further research is essential to optimize protocols for clinical use.
Sources
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https://pmc.ncbi.nlm.nih.gov/articles/PMC9584646/
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https://www.dvcstem.com/post/stem-cell-therapy-for-alzheimers
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https://gpsych.bmj.com/content/36/5/e101143
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https://pubmed.ncbi.nlm.nih.gov/38051903/
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https://www.michaeljfox.org/grant/phase-ii-trial-bone-marrow-derived-mesenchymal-stem-cells-disease-modifying-therapy
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https://www.nature.com/articles/s41536-021-00131-5
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https://www.dvcstem.com/post/stem-cells-als
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https://www.frontiersin.org/journals/cell-and-developmental-biology/articles/10.3389/fcell.2025.1517369/full
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https://www.msif.org/research/challenges-of-ms-research/stem-cell-therapy-for-ms/
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