Can Stem Cell Therapy Help With Autoimmune Diseases?

Some stem cell-based approaches may improve certain outcomes in selected autoimmune diseases, but the evidence varies by condition, treatment type, and patient. HSCT has established clinical use for selected severe autoimmune diseases, while many MSC-based applications remain investigational.

Key Takeaways

• Researchers are studying whether certain stem cell-based approaches can modify immune activity and improve specific clinical outcomes in autoimmune diseases.
• HSCT and mesenchymal stem cell (MSC) therapies work differently and should not be considered interchangeable.
• Clinical studies have reported improvements in selected outcomes for some autoimmune diseases, but findings vary by condition and treatment approach.
• Research findings cannot be applied equally to every autoimmune disease or every patient.
• Many stem cell-based applications remain investigational and require further evidence on safety, effectiveness, and long-term outcomes.

Can Stem Cells Improve Autoimmune Disease Outcomes?

Dealing with autoimmune conditions can be unpredictable. A patient may have times when their condition seems relatively stable and then suddenly experience flare-ups that interfere with everyday activities, work, and quality of life. Although existing standard treatment may help control disease progression and symptoms, some patients still experience significant complications. 

This has led researchers around the world to investigate whether certain stem cell-based approaches may help influence abnormal immune activity in ways that may improve specific clinical outcomes. 

However, researchers are studying different cell types and treatment protocols across autoimmune diseases, including systemic lupus erythematosus, systemic sclerosis, multiple sclerosis, and rheumatoid arthritis. Because the evidence differs between conditions and approaches, findings from one disease should not automatically be applied to another.

Which Stem Cell Approaches Are Being Studied for Autoimmune Diseases?

Two major areas of research are hematopoietic stem cell transplantation (HSCT) and mesenchymal stem cell (MSC) therapies. Although both involve stem cells, they differ substantially in their purpose, procedures, evidence base, and safety considerations.

Hematopoietic Stem Cell Transplantation (HSCT)

HSCT is under investigation for severe autoimmune disorders where conventional therapies have not been effective. HSCT is being studied for severe autoimmune diseases, particularly in patients whose disease remains difficult to control with conventional treatment. The approach uses conditioning therapy followed by hematopoietic stem cell transplantation to rebuild the immune system.

Mesenchymal Stem Cells (MSCs)

The role of MSCs is under investigation based on their immune-modulating properties. They are being studied to see if they can affect inflammatory and immune responses associated with autoimmune disorders, but most of the applications using MSCs are investigational.

A systematic review and meta-analysis in 2022 of 18 randomised controlled trials for five autoimmune diseases reported potential benefits in some autoimmune diseases; however, the authors suggested that more trials are required to verify their results.

Which Autoimmune Diseases Are Being Studied With Stem Cells?

Current research is not limited to one autoimmune condition. Scientists are investigating different stem cell approaches across different conditions where abnormal immune activity can cause persistent inflammation, tissue damage, or disability. 

Research has included conditions such as:

• Systemic Lupus Erythematosus (SLE): Studies are looking at HSCT as well as MSCs in terms of disease activity and immune modulation.

• Systemic Sclerosis (SSc): Studies have looked into HSCT in patients who have severe disease, especially the role of HSCT in terms of disease progression.

• Multiple Sclerosis (MS): Studies are looking at HSCT and other cell therapies in terms of disease activity and neurological effects.

• Rheumatoid Arthritis (RA): The therapeutic potential of MSCs and other cell-based treatments in immunomodulation is under study.

However, the quantity and quality of the evidence base vary greatly between these diseases. Results obtained in the context of one autoimmune disease cannot be generalised to another.

What Outcomes Are Researchers Measuring?

Researchers are focusing on the various aspects of the autoimmune condition rather than the symptoms alone. Depending on the autoimmune disease and study design, researchers may examine:

• Clinical Outcomes: disease activity, disability, relapse rates, or organ function

• Safety Outcomes: adverse events, infections, and treatment-related complications

• Durability: whether any improvement continues during longer-term follow-up

The study published in 2022 consisted of a systematic review and meta-analysis comprising 18 randomised controlled trials in relation to five autoimmune diseases. There was an observed benefit of MSC treatment on the SLEDAI scores and some lab parameters in the case of systemic lupus erythematosus, but the findings in MS were contradictory.

Improvement in the activity of the disease in one clinical trial cannot automatically mean that the stem cell treatment is effective in all patients suffering from that disease.

They are also considering long-term effects since any improvement seen immediately after the treatment may not indicate that the patient will continue to have good control of the disease.

Why Can Stem Cell Outcomes Differ Between Patients?

Stem cell research often does not produce the same results for every patient, even with the same condition. Outcomes can vary because the autoimmune disease, its severity, previous treatments, and the specific cellular approach that are being studied may differ.

• The nature and progression of the autoimmune disorder
• The degree of organ involvement
• Prior response to conventional therapy
• The general health of the patient
• Cell source and manufacturing process 
• Duration and completeness of follow-up 

These variations indicate why the outcomes from a clinical trial can never be automatically generalised to all patients or autoimmune disorders.

What Are the Risks and Limitations of Stem Cell Therapy for Autoimmune Diseases?

The potential outcomes of stem cell therapy need to be considered alongside its risks and the limitations of current evidence.

HSCT Risks: HSCT involves conditioning therapy and can carry significant risks, including infections and other treatment-related complications. Because of these risks, it requires careful patient selection and specialist monitoring.

MSC-related Uncertainties: MSC therapies are being studied across different autoimmune diseases, but studies vary in cell source, manufacturing methods, dosing, treatment protocols, study size, and follow-up duration.

Limited Evidence: Early-stage or small studies may not provide enough evidence to establish the long-term safety or effectiveness of a treatment.

Different Results: Findings of one study or one treatment protocol of one autoimmune disease cannot be automatically extrapolated to other conditions or protocols.

Individual Response: Positive results found during research cannot be considered as an indication of positive outcomes in all patients.

All those limitations have to be taken into account during the evaluation of possible involvement of stem cells in the treatment of a particular autoimmune disease.

What Is the Clinical Status of Stem Cell Treatments for Autoimmune Diseases?

The clinical status depends on the specific condition, cell type, treatment protocol, and indication. HSCT has established clinical use for selected autoimmune diseases, while many MSC-based applications remain investigational.

Some important factors to consider include:

• Specific Condition: The evidence and status of treatments may vary greatly for different autoimmune conditions.

• Cell Type: HSCT and MSC therapies have different clinical indications, procedures, evidence bases, and safety considerations.

• Treatment Status: Some applications have established clinical use, while others remain under investigation in clinical trials or other regulated research settings.

• Clinical Evidence: Findings should be evaluated separately for each condition and treatment strategy rather than treating “stem cell therapy” as one standard intervention.

• Regulatory Requirements: When assessing stem cell-based treatments in India, the relevant ICMR guidelines need to be taken into account.

In India, patients considering stem cell-based approaches should also consider applicable guidance from the Indian Council of Medical Research (ICMR) and the clinical setting in which the treatment is offered. The evidence and regulatory status should be assessed for the specific condition and treatment approach rather than for “stem cell therapy” as a broad category.

Looking Ahead

Stem cell-based approaches are still being studied for autoimmune conditions. While some areas of research show encouraging findings, others remain under investigation. Although the evidence varies by condition and treatment approach, understanding disease-specific research and appropriate clinical evaluation is important before considering regenerative medicine. 

Frequently Asked Questions

Q1. Can stem cells help autoimmune diseases?

Some stem cell-based approaches may improve specific outcomes in selected autoimmune diseases, but evidence varies by condition, cell type, treatment protocol, and patient.

Q2. Which autoimmune diseases are being studied with stem cell therapy?

Research includes conditions such as systemic lupus erythematosus, multiple sclerosis, systemic sclerosis, rheumatoid arthritis, inflammatory bowel disease, and ankylosing spondylitis.

Q3. Is HSCT the same as MSC therapy for autoimmune diseases?

No. HSCT and MSC treatment are distinct modalities with different methods, protocols, evidence base, and side effects. They cannot be regarded as equivalent treatment options.

Are MSC treatments for autoimmune diseases approved? 

Many MSC-based applications for autoimmune diseases remain investigational. Their clinical and regulatory status depends on the specific condition, cell product, treatment protocol, and applicable regulations.

Q5. What are the risks of stem cell therapy for autoimmune diseases?

Risks depend on the specific approach. HSCT can involve significant risks associated with conditioning, infection, and other complications, while risks and uncertainties associated with MSC-based therapies vary between protocols and studies.

Q6. Why does stem cell therapy affect autoimmune patients differently? 

Outcomes can vary because of differences in disease severity, organ involvement, previous treatments, overall health, cell source, treatment protocol, and follow-up duration.

Reference Links

(1) Indian Council of Medical Research (ICMR). (2021). Evidence-Based Status of Stem Cell Therapy for Human Diseases.

This is particularly relevant for discussing the evidence status of stem cell-based interventions in India.

ICMR – Evidence-Based Status of Stem Cell Therapy for Human Diseases

(2) Snowden, J. A., et al. (2022). Hematopoietic stem cell transplantation and cellular therapies for autoimmune diseases: overview and future considerations from the Autoimmune Diseases Working Party (ADWP) of the European Society for Blood and Marrow Transplantation (EBMT).

https://pubmed.ncbi.nlm.nih.gov/35578014/

Zeng, L., Yu, G., Yang, K., et al. (2022). Efficacy and Safety of Mesenchymal Stem Cell Transplantation in the Treatment of Autoimmune Diseases: A Systematic Review and Meta-Analysis of Randomised Controlled Trials. Stem Cells International, 2022, 9463314. DOI: 10.1155/2022/9463314.

https://pubmed.ncbi.nlm.nih.gov/35371265/

(4) Swain, H. N., et al. (2024). Mesenchymal stem cells in autoimmune disease: A systematic review and meta-analysis of pre-clinical studies. Biochimie.

https://pubmed.ncbi.nlm.nih.gov/38657832/

(5) Indian Council of Medical Research (ICMR). (2021). National Guidelines for Hematopoietic Cell Transplantation.

https://www.icmr.gov.in/guidelines