Stem Cells Open the Door to New Treatment Possibilities for Multiple Sclerosis
Multiple sclerosis (MS) remains a daunting adversary for most patients. This disease, which affects the brain, spinal cord, and optic nerve, gradually damages the protective myelin sheath of neurons and, in many patients, leads to chronic neurological impairment. Although modern medications can slow the progression, a causal treatment is still lacking. One of the most promising avenues of the past decade is stem cell research — an approach considered experimental but opening the door to new, yet unexplored therapeutic possibilities.
Stem Cells Open the Door to New Treatment Possibilities for Multiple Sclerosis Multiple sclerosis (MS) remains a daunting adversary for most patients. This disease, which affects the brain, spinal cord, and optic nerve, gradually damages the protective myelin sheath of neurons and, in many patients, leads to chronic neurological impairment. Although modern medications can slow the progression, a causal treatment is still lacking. One of the most promising avenues of the past decade is stem cell research — an approach considered experimental but opening the door to new, yet unexplored therapeutic possibilities.
How Can They Help?
Stem cells are undifferentiated cells capable of transforming into specific cell types within the body. They can also regenerate, which plays a key role in repairing damaged tissues.
They naturally occur, for example, in bone marrow, blood, liver, brain, or skin. While stem cells are already used in the treatment of various hematologic, immunologic, and metabolic diseases, in neurology (and specifically in MS) they remain the focus of intensive research.
Autologous Transplantation
Stem cell therapy involves replacing a dysfunctional immune system with a “reset” system using the transplantation of autologous or allogeneic stem cells. In clinical practice, autologous transplantation is most commonly used and is currently the closest to broader clinical application. How is it performed?
First, the patient’s improperly functioning immune system must be “wiped out” using specialized treatment (chemotherapy and immunological antibodies) that selectively removes autoreactive cells attacking the body's own nervous tissues. This is followed by stem cell transplantation, which gives rise to a renewed immune system. The goal is not to create sterile immunity, but to “restart” it so that it tolerates the natural structures of the nervous system instead of attacking them.
Results from several international review studies show that autologous hematopoietic stem cell transplantation (aHSCT) can lead to NEDA remission (no evidence of disease activity) in up to 68% of patients, especially younger individuals with aggressive, relapsing forms of the disease. At the same time, the risk of severe complications has decreased — mortality dropped from 7.3% in 1995–2000 to 1.3% in 2001–2007.
Regulators of Immune Processes
Mesenchymal stem cells (MSCs) represent another chapter in MS cell therapy. These cells, which can be isolated for example from bone marrow or adipose tissue, act as natural regulators of immune processes. They also support the regeneration of nervous tissue and release growth factors that help repair damaged myelin.
In clinical studies, these cells are often harvested from the patient, modified in laboratory conditions, and then reintroduced into the body. Sometimes they are specially engineered to enhance their ability to suppress immune reactions and support regeneration.
Although the results have not yet demonstrated clear breakthrough improvements, systematic reviews indicate that MSC therapy has a solid safety profile and shows some potential as an adjunct treatment for MS.
Other Stem Cell Types Under Investigation
In addition to hematopoietic and mesenchymal stem cells, experimental studies are exploring:
- Neural stem cells (NSCs): Focused on myelin repair; they can also be derived from MSCs.
- Human embryonic stem cells (hESCs): Capable of developing into any cell type but carry a risk of tumor formation.
- Induced pluripotent stem cells (iPSCs): Generated by reprogramming adult cells, likewise associated with potential cancer risks.
These approaches, however, remain in very early stages of research.
Beware of Misinformation
Although aHSCT is the most advanced stem cell–based therapy for MS, no form of stem cell therapy for this disease is officially approved by the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA). All such treatments are still considered experimental and typically take place within clinical trials.
Both the FDA and EMA warn against “regenerative” products and promises of quick cures offered outside approved clinical trials, which often lack proven efficacy and safety. Therefore, it is essential that both patients and physicians carefully evaluate available information and avoid unnecessary risks.
Editorial Team, Medscope.pro
Sources:
1. Wu L., Lu J., Lan T. et al. Stem cell therapies: a new era in the treatment of multiple sclerosis. Front Neurol 2024; 15 : 1389697, doi: 10.3389/fneur.2024.1389697.
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