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Regenerative Medicine

Stem Cell Therapy

Stem Cell Therapy · Z79.899

Stem cell therapy overview: types of stem cells, mechanisms of action in regenerative medicine, current clinical applications, evidence base, and regulatory considerations.

2026-03-28

At a Glance

Stem cell therapy overview: types of stem cells, mechanisms of action in regenerative medicine, current clinical applications, evidence base, and regulatory considerations.

Definition and Overview

Stem cells are cells with the capacity for self-renewal and multilineage differentiation potential. Various types exist, ranging from embryonic stem cells in developmental stages to adult stem cells found in various tissues of the mature organism. Stem cell therapy is a core field of regenerative medicine that utilizes these cells to treat diseases.

The most extensively studied and clinically applied stem cells are mesenchymal stem/stromal cells (MSCs) [1]. MSCs can be harvested from various tissues including bone marrow, adipose tissue, umbilical cord (cord blood), dental pulp, and placenta, and possess the ability to differentiate into osteocytes, chondrocytes, and adipocytes, along with potent immunomodulatory and anti-inflammatory functions [1].

Therapeutic Mechanisms of Mesenchymal Stem Cells

MSCs are understood to exert their therapeutic effects primarily through the secretion of various factors rather than directly replenishing cells in damaged tissue [2][3].

Neurotrophic factor secretion: MSCs secrete BDNF (brain-derived neurotrophic factor), NGF (nerve growth factor), and NT-3 (neurotrophin-3) to promote neuronal survival, growth, and functional recovery [3].

Anti-inflammatory effects: MSCs secrete prostaglandin E2, IL-10, and TGF-beta to suppress inflammatory responses and induce regulatory T cells [2].

Angiogenesis promotion: MSCs secrete VEGF (vascular endothelial growth factor) and FGF to support vascular regeneration [3].

Secretome and exosomes: Exosomes secreted by MSCs contain growth factors, miRNA, and proteins that induce tissue regeneration through intercellular communication [3].

Neurological Applications

Clinical applications of MSCs are being investigated in spinal cord injury, ALS, Parkinson's disease, post-stroke rehabilitation, and autonomic dysfunction. Small-scale clinical studies have reported that MSC administration partially restores neurological function or slows disease progression; however, evidence from large-scale randomized controlled trials remains limited.

In autonomic dysfunction, MSCs may support functional recovery by distributing around damaged autonomic ganglia and peripheral autonomic nerve fibers, supplying neurotrophic factors and suppressing inflammation.

Adipose-Derived Regenerative Cells

Adipose tissue is an abundant source of MSCs and other regenerative cells. Adipose tissue obtained through liposuction or small-volume harvesting is enzymatically processed and centrifuged to yield the stromal vascular fraction (SVF). SVF contains MSCs, pericytes, endothelial progenitor cells, macrophages, and T lymphocytes.

Autologous SVF offers the advantage of same-day treatment without the risk of immune rejection. Clinical applications of SVF in arthritis, neuropathy, and autonomic dysfunction are ongoing.

Safety

Meta-analyses have confirmed that MSC-based therapy has a generally favorable safety profile with a low incidence of major adverse events [4]. Short-term side effects include injection site pain, mild fever, and headache. Although concerns about tumor development have been raised, a causal relationship between MSC administration and tumor development has not been established in clinical studies to date [4].

Since long-term safety data are not yet sufficient, it is important that treatment be performed at accredited medical institutions following established clinical protocols.

Frequently Asked Questions

Stem cells are cells capable of differentiating into various cell types and self-replicating. Stem cell therapy involves administering these cells into the body to regenerate damaged tissue, or to secrete substances that reduce inflammation and protect nerves, thereby treating diseases. Beyond direct regeneration through cell differentiation, the various factors secreted by the cells (the secretome) are increasingly recognized as central to their therapeutic effects.

Mesenchymal stem cells (MSCs) are the most widely used in clinical practice. They can be harvested from bone marrow, adipose tissue, umbilical cord (cord blood), and other sources. Neural stem cells and induced pluripotent stem cells (iPSCs, reprogrammed from adult cells) are also being investigated for nervous system regeneration. SVF (stromal vascular fraction) is a cell mixture extracted from adipose tissue that contains stem cells and is used in regenerative treatments. Either autologous or allogeneic cells are used.

Animal studies and early clinical research have reported that mesenchymal stem cells secrete neurotrophic factors and suppress inflammation at sites of nerve damage, promoting neurological function recovery. Clinical studies are underway for amyotrophic lateral sclerosis (ALS), spinal cord injury, post-stroke rehabilitation, Parkinson's disease, and autonomic dysfunction. Most neurological stem cell therapies are currently at the clinical research stage, with meaningful results emerging in some conditions.

Meta-analyses evaluating the safety of MSC-based therapy have reported a low risk of tumor development or serious adverse events. Short-term effects may include injection site pain or mild fever. However, long-term safety data are still insufficient, and stem cell therapy at unaccredited facilities may have uncertain efficacy and safety. Treatment should always be received at properly licensed medical institutions based on clinical evidence.

The stromal vascular fraction (SVF), obtained through minimal processing of adipose tissue, contains a variety of cells including mesenchymal stem cells, pericytes, endothelial progenitor cells, and immune cells. Because autologous fat is used, the risk of immune rejection is low. Clinical application studies are underway for arthritis, neuralgia, and autonomic dysfunction, and OSANG Neurosurgery also offers SVF-based regenerative treatments.

References

  1. [1] Pittenger MF, Mackay AM, Beck SC, Jaiswal RK, Douglas R, Mosca JD, et al. (1999). "Multilineage potential of adult human mesenchymal stem cells." Science, 284: 143-147. DOI PubMed
  2. [2] Caplan AI, Correa D (2011). "The MSC: an injury drugstore." Cell Stem Cell, 9: 11-15. DOI PubMed
  3. [3] Drago D, Cossetti C, Iraci N, Gaude E, Musco G, Bachi A, et al. (2013). "The stem cell secretome and its role in brain repair." Biochimie, 95: 2271-2285. DOI PubMed
  4. [4] Lalu MM, McIntyre L, Pugliese C, Fergusson D, Winston BW, Marshall JC, et al. (2012). "Safety of cell therapy with mesenchymal stromal cells (SafeCell): a systematic review and meta-analysis of clinical trials." PLOS ONE, 7: e47559. DOI PubMed
  5. [5] Zhao Y, Jiang Z, Zhao T, Ye M, Hu C, Yin Z, et al. (2012). "Reversal of type 1 diabetes via islet β cell regeneration following immune regulation by cord blood-derived multipotent stem cells." BMC Medicine, 10: 3. DOI PubMed

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