Multiple sclerosis (MS) is a chronic condition that affects the central nervous system, where the immune system mistakenly attacks the protective covering of nerve fibers, called myelin. This damage interrupts communication between the brain and the body, leading to symptoms such as muscle weakness, difficulty walking, fatigue, and loss of coordination. MS is a complex disease with varying patterns. Some people experience relapsing and remitting symptoms, while others develop progressive forms that steadily worsen over time.

Current treatments for MS focus on reducing the frequency of relapses, managing symptoms, and slowing disease progression. However, these treatments are often limited in their effectiveness, especially in severe or progressive forms of the disease. Some medications can also cause significant side effects, including flu-like symptoms, skin irritation, or increased risk of infections. This has led researchers to explore new therapeutic strategies, including the use of mesenchymal stem cells (MSCs).

In this review, Islam et al. assess the effectiveness and safety of MSC therapy in individuals diagnosed with MS.

Understanding Mesenchymal Stem Cells

MSCs are a type of adult stem cell found in multiple tissues, such as bone marrow, adipose, and umbilical cord tissue. They are known for their ability to grow and differentiate into various cell types, including bone, cartilage, and nerve cells. MSCs also produce molecules that help regulate inflammation and support tissue repair.

Because of these properties, MSCs have been investigated as a potential therapy for many conditions, including heart disease, spinal cord injury, and autoimmune disorders. In MS, researchers believe MSCs could help repair damaged nerve cells, reduce inflammation, and potentially slow or even reverse disease progression.

Clinical Evaluation of MSC Therapy for MS

This systematic review and meta-analysis by Islam et al. examined the effectiveness and safety of MSC therapy in patients with MS. This study pooled data from multiple clinical trials, looking at how patients’ conditions changed after receiving MSC treatment. The main measure used to track improvement was the Expanded Disability Status Scale (EDSS), a standard tool used in MS research to evaluate mobility, coordination, and overall neurological function.

The analysis found that approximately 40% of patients experienced improvements after MSC therapy. Another 33% remained stable, while about 18% saw a worsening of their condition. According to the authors, these results suggest that MSC therapy could have a meaningful impact on disease progression for a significant proportion of MS patients.

Safety Profile of MSC Therapy

Safety is a critical consideration for any new treatment. In this meta-analysis, no major complications were reported. Some minor side effects, including headaches, fever, urinary tract infections, and respiratory infections, were observed. Most of these were mild and manageable, indicating that MSC therapy is generally well-tolerated.

Interestingly, the source of the MSCs appeared to influence the therapy’s effectiveness. MSCs derived from umbilical cord or placental tissue were associated with higher improvement rates (57%) compared to MSCs derived from bone marrow (38%). According to the authors, these differences may be related to factors such as lower immunogenicity, higher cell proliferation capacity, and non-invasive collection methods for umbilical cord or placental MSCs.

Routes of MSC Administration and Effectiveness

MSCs can be delivered intravenously or directly into the cerebrospinal fluid through intrathecal injection. The study found that intravenous administration resulted in better outcomes, with 58% of patients showing improvement, compared to 33% for intrathecal administration. This information may guide future treatment protocols and clinical decisions.

Mechanisms of MSC Therapy in MS

The therapeutic effects of MSCs in MS are thought to be driven by their ability to modulate the immune system and promote nerve repair. In MS, immune cells such as T helper cells and microglia contribute to inflammation and nerve damage. MSCs can shift the balance of these immune cells, reducing harmful inflammation while encouraging protective and repair-oriented responses.

Additionally, MSCs may directly support the regeneration of neurons and glial cells, which are essential for maintaining the structure and function of the nervous system. By promoting a healthier environment for nerve cells, MSC therapy has the potential to improve neurological function and slow disease progression.

Insights from Clinical Trials

Several clinical trials have evaluated MSC therapy for MS, both as randomized controlled studies and observational research. The pooled data from these trials support the therapy’s potential to improve or stabilize neurological function. Early reports also confirm its safety, with minimal serious adverse events.

Studies suggest that factors such as patient age, disease severity, and the origin of MSCs influence outcomes. For example, younger donor cells and MSCs from umbilical cord or placental tissue appear to have higher efficacy. Intravenous administration also seems more effective than intrathecal delivery.

Comparison with Conventional MS Treatments

Existing MS treatments, such as disease-modifying drugs like Ocrelizumab, Fingolimod, and Teriflunomide, are effective for some patients but often fall short in severe or progressive cases. Side effects and long-term risks can also limit their use. MSC therapy offers a novel approach by potentially repairing nerve damage rather than simply managing symptoms or suppressing the immune system. For patients who do not respond well to conventional treatments, MSC therapy may provide a new option.

Current Limitations and Future Research Directions

While MSC therapy shows promise, there are still unanswered questions. Clinical trials vary in terms of the number of patients, dosage, source of MSCs, and methods of administration, which can make it challenging to compare results. There is also a need for larger, long-term studies to determine the most effective protocols and confirm the durability of treatment benefits.

Future research will likely focus on optimizing MSC doses, identifying the best cell sources, and refining delivery methods. Researchers also aim to better understand the mechanisms by which MSCs promote repair and reduce inflammation in the nervous system.

Future Outlook for MSC Therapy in Multiple Sclerosis

Mesenchymal stem cell therapy represents a promising new approach for treating multiple sclerosis. Many patients experience improvements or maintain stability after receiving MSCs, and serious adverse events are rare. The therapy’s ability to modulate the immune system, support nerve repair, and promote tissue regeneration sets it apart from conventional treatments.

Ongoing research is focused on refining MSC therapy protocols, determining optimal dosages, and assessing long-term outcomes. Larger, high-quality clinical trials will be essential to establish MSC therapy as a reliable and effective option for people living with MS.

For patients exploring new treatment possibilities, MSC therapy offers hope for improved neurological function, better quality of life, and potential disease stabilization. 

Source: Islam MA, Alam SS, Kundu S, Ahmed S, Sultana S, Patar A, Hossan T. Mesenchymal Stem Cell Therapy in Multiple Sclerosis: A Systematic Review and Meta-Analysis. J Clin Med. 2023 Sep 30;12(19):6311. doi: 10.3390/jcm12196311. PMID: 37834955; PMCID: PMC10573670.

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