In her monthly column "Cell Notes," AABB's Christina Celluzzi, PhD, MS, CABP(H), shares insights, findings and commentary on emerging topics in biotherapies. Subscribe to CellSource, AABB's biotherapies newsletter, to receive "Cell Notes" and the latest news directly in your inbox.
AABB’s Cell Therapy (CT) member subsection meetings provide a platform for discussing emerging and impactful topics in the field. A recent discussion in the Cell Therapy Current and Emerging Topics Subsection focused on the use of cell-based therapies for autoimmune diseases. A particularly intriguing question arose: What role do mesenchymal stromal cells (MSCs) play in clinical therapies for autoimmune diseases? This question sparked a lively discussion, prompting further exploration of this topic. I’d love to share some findings—and I invite you to add your insights.
The Challenge of Autoimmune Diseases
Autoimmune diseases such as systemic lupus erythematosus (SLE), rheumatoid arthritis (RA) and multiple sclerosis (MS) arise when the immune system mistakenly attacks the body’s tissues, leading to chronic inflammation and progressive damage. Current treatments, including immunosuppressive drugs, help manage symptoms but come with drawbacks, including an increased risk of infection.
This is where MSC therapy offers a potentially groundbreaking alternative. MSCs are multipotent stem cells with powerful immunomodulatory and anti-inflammatory properties. They’ve shown promise in treating conditions such as neurological injuries, osteoarthritis, graft-versus-host disease (GVHD) and MS. In a major milestone, the FDA recently approved Ryoncil (remestemcel-L-rknd), an allogeneic bone marrow-derived MSC therapy, for steroid-refractory acute GVHD in pediatric patients—marking the first-ever FDA-approved MSC therapy. This paves the way for expanding MSC-based treatments to other immune-related disorders.
MSC Therapy for Autoimmune Diseases: Progress and Potential
Research into MSC therapy for autoimmune diseases is rapidly advancing. Studies have explored its potential in treating conditions like Crohn’s disease and MS, with encouraging results. A
meta-analysis of randomized controlled trials (RCTs) involving 2,183 patients demonstrated significant symptom improvements in RA, osteoarthritis and SLE. However, results for MS and systemic sclerosis remain mixed, highlighting the need for further investigation.
MSCs exert their effects through direct cell contact and paracrine signaling, offering a targeted, less toxic alternative to traditional immunosuppressants. By reducing inflammation and promoting tissue repair without significantly increasing adverse events, MSC therapy holds promise as a more sustainable treatment option.
Long-Term Benefits and Safety Considerations
Beyond symptom relief, MSC therapy may offer long-term benefits due to its regenerative properties. These include tissue repair and regeneration, sustained therapeutic effects, neuroprotection and trained immunity (involving immune system “reprogramming”).
Although generally considered safe, MSC therapy is not without risks. Potential adverse events include transient fever, localized reactions, fatigue, constipation, insomnia or immune rejection. There’s also a theoretical risk of tumorigenicity, which requires ongoing monitoring.
Clinical trials have shown significant improvements in patients with traumatic brain injury (TBI) and spinal cord injury (SCI) (improved motor function, reduced inflammation), severe COVID-19 (reduced mortality, improved clinical symptoms) and osteoarthritis (reduced pain, improved joint function).
These successes underscore MSC therapy’s broad potential, strengthening the case for its application in autoimmune diseases.
Future Challenges and Broader Applications
Despite its promise, MSC therapy for autoimmune diseases faces challenges, including:
- Patient selection criteria (which patients are most likely to benefit most?).
- Ensuring long-term safety and efficacy.
- Standardizing cell quality.
- Addressing cost and accessibility.
Beyond autoimmune diseases, MSCs are being investigated for cardiovascular repair, liver disease, amyotrophic lateral sclerosis (ALS) and acute respiratory distress syndrome (ARDS). As research continues, MSC-based therapies could redefine how we treat a wide range of chronic and inflammatory conditions.
Some Final Thoughts
MSC therapy is at the forefront of next-generation autoimmune disease treatments. While challenges remain, ongoing research is refining its applications and addressing safety concerns. Could MSC therapy be the future standard of care for autoimmune conditions?
I’d love to hear your thoughts. Let’s continue the conversation! And think about joining an AABB subsection to share with biotherapies professionals!
