Can stem cell transplants help with systemic inflammation?

From the Guidelines

Stem cell transplants can be an effective treatment for systemic inflammation, particularly in conditions like multiple sclerosis and systemic sclerosis, as evidenced by recent studies 1.

Key Points to Consider

• The use of autologous haematopoietic stem cell transplantation (AHSCT) has been recommended for the treatment of multiple sclerosis and neuromyelitis optica spectrum disorder by the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) and the European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) 1.
• AHSCT can improve survival in patients with early diffuse cutaneous systemic sclerosis who are at high risk of mortality, such as those with very high skin scores or moderate skin involvement and worsening interstitial lung disease (ILD) 1.
• High-intensity immunosuppression followed by autologous haematopoietic stem cell transplantation (HSCT) may be considered for the treatment of selected patients with early severe dcSSc and poor prognosis, in the absence of advanced cardiorespiratory involvement 1.

Treatment Approach

• The procedure generally requires pre-treatment evaluation, possible immunosuppression, the transplant itself, and follow-up care.
• Stem cells work by releasing cytokines that dampen inflammatory pathways, promoting regulatory T-cells that control immune responses, and supporting tissue regeneration.
• However, this treatment remains experimental for many inflammatory conditions, with varying protocols and success rates depending on the specific disease being treated.

Recent Evidence

• A recent study published in 2025 in the journal Nature Reviews Neurology found that AHSCT is highly effective at stopping inflammation in the brain and can stabilize or even improve function in relapsing–remitting MS 1.
• Another study published in 2023 in the journal Nature Reviews Rheumatology found that autologous haematopoietic stem cell transplantation can improve survival in patients with early diffuse cutaneous systemic sclerosis who are at high risk of mortality 1.
• The 2023 update of the EULAR recommendations for the treatment of systemic sclerosis also suggests that high-intensity immunosuppression followed by autologous haematopoietic stem cell transplantation (HSCT) may be considered for the treatment of selected patients with early severe dcSSc and poor prognosis 1.

From the Research

Stem Cell Transplants for Systemic Autoimmune Diseases

• Stem cell transplants have been used to treat various systemic autoimmune diseases, including multiple sclerosis, systemic lupus erythematosus, and rheumatoid arthritis 2, 3, 4, 5, 6
• Autologous hematopoietic stem cell transplantation (HSCT) has shown promise in inducing long-term remission in patients with severe autoimmune diseases refractory to conventional therapy 2, 4, 5
• The procedure involves collecting stem cells from the patient's bone marrow or peripheral blood, followed by high-dose immunosuppression and reinfusion of the stem cells 2, 6

Efficacy of Stem Cell Transplants

• Studies have reported improvement in disease activity and quality of life in patients with systemic autoimmune diseases after undergoing stem cell transplants 2, 3, 5
• Non-myeloablative selective HSCT has shown overall survival benefits and improvement in event-free survival and skin thickness compared to cyclophosphamide 3
• Myeloablative selective HSCT has also demonstrated improvement in event-free survival and skin thickness, although with a higher risk of treatment-related mortality 3

Safety and Adverse Effects

• Stem cell transplants can be associated with serious adverse events, including infection, organ damage, and treatment-related mortality 3, 5, 6
• The risk of adverse effects is higher in patients with significant comorbidities or complications from their underlying disease 3
• Careful patient selection and risk-benefit assessment are essential to minimize the risks associated with stem cell transplants 3, 5

Current Status and Future Directions

• Stem cell transplants are still considered an experimental treatment for systemic autoimmune diseases, and more research is needed to fully understand their efficacy and safety 3, 4, 5
• Ongoing and planned clinical trials aim to evaluate the role of stem cell transplants in the treatment of severe autoimmune diseases and to optimize treatment protocols 3, 5