This blog post summarises the key insights from the latest Virtual Education Session (VES) led by Dr Marie Hudson, a rheumatologist and epidemiologist affiliated with the Jewish General Hospital and the Lady Davis Institute in Montreal. As an Associate Professor at McGill University, she leads research efforts that focus on innovative therapeutic approaches.
Dr Hudson is also a founding member of the Canadian Scleroderma Research Group (CSRG), which has been active since 2003. The group includes 15 sites across Canada, 20 clinicians, and 10 basic scientists, having recruited over 1,600 patients and secured over $4 million in funding. Their collaborative efforts have led to more than 100 peer-reviewed publications, establishing CSRG as a recognised leader in scleroderma research. If you want to learn more about this Canadian scleroderma organisation, click here to access their website.
This article shares what research is being done with stem cells in scleroderma treatment and management.
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Session Overview
In this VES, Dr Hudson shared the promising potential of stem cell transplants, emphasising their positive effects on lung function and the reduced risk of renal crises in patients. This form of treatment leverages the body’s own stem cells to regenerate a healthy immune system, effectively treating scleroderma, which is effectively the immune system attacking the body’s own tissues.
HSC Transplants
One cell therapy, hematopoietic stem cell (HSC) transplants, is particularly promising, demonstrating significant improvements in both early and delayed lung function compared to those undergoing standard treatments. Further, the risk of developing a scleroderma renal crisis was reduced by five times in transplant recipients, highlighting the therapy’s potential to enhance survival and organ function.
The process of a HSC transplant goes as follows:
- A patient’s stem cells are harvested, followed by chemotherapy to eliminate any abnormal immune cells.
- The stem cells are then reinfused in the patient, allowing for the development of a normal immune response.
So far, this treatment has been the only one associated with improved survival rates for scleroderma patients, with recent findings suggesting that HSC transplants not only enhance survival, but also improve various symptom manifestations, including skin and lung function, and overall quality of life. These results have been observed not only in clinical trial settings, but also in real-world applications, providing key proof about the efficacy of this treatment. However, while the benefits of HSC transplants are clear, Dr Hudson pointed out that they are suitable for a limited number of patients due to the treatment’s toxicity. As chemotherapy is involved in HSC transplants, there’s a 5-10% risk of early mortality. Of the 1,500 patients in her Canadian cohort, only about 5% were deemed suitable for the procedure.
There have also been some post-treatment discrepancies regarding mental health outcomes. While physical health improved, many transplant recipients did not report a corresponding enhancement in their mental health quality of life. Dr Hudson attributed this to potential misconceptions about the nature of the transplant, with patients entering with high expectations for a cure, when in reality, the transplant alleviates symptoms but does not eliminate the disease.
MSC Transplants
Another promising stem cell therapy is through mesenchymal stromal cells (MSCs). Unlike HSC transplants, MSCs can be harvested from umbilical cords or adipose tissue, allowing for administration without the associated chemotherapy. Initial studies have indicated that MSCs may help with fibrosis, vasculopathy, and immune activation—key issues in scleroderma.
Dr Hudson highlighted a recent French study that showed positive early outcomes with MSC infusions, leading to plans for further research in a controlled, blinded trial using umbilical cord-derived MSCs. This innovative approach aims to establish a more effective and less risky treatment for scleroderma, with the potential to recruit more patients for improved therapeutic insights. However, this stem cell therapy trial is still ongoing, so results are limited.
CAR T-Cell Transplants
The final stem cell therapy Dr Hudson shared was Chimeric Antigen Receptor T cell (CAR T) therapy, a form of immunotherapy that involves genetically modifying a patient’s T cells to better recognise and attack specific disease targets. While primarily developed for certain types of cancers, there is increasing interest in applying CAR T cell therapy to scleroderma.
The process of a CAR T-cell transplants goes as follows:
- A patient’s T cells are collected from their blood through leukapheresis.
- The collected T cells are genetically modified in a laboratory to express CARs that specifically target antigens associated with scleroderma. This enables the T cells to identify and destroy cells that contribute to the autoimmune response.
- The modified T cells are expanded in the lab and then reinfused back into the patient.
The specific targets for CAR T cells in scleroderma are still under investigation, with the aim of developing receptors that can effectively recognise and attack cells involved in the scleroderma development process. However, small studies indicate potential benefits such as improved scleroderma symptoms such as skin thickening and lung function, and a long-term defence that reduces the need for continuous medication.
As there is limited research for this therapy so far, there are concerns of significant side effects, such as cytokine release syndrome (CRS) and neurotoxicity. These risks necessitate careful monitoring during and after treatment.
Conclusion
As of this VES, HSCT is the established therapy, MSCs are being actively researched, and CAR T-cell therapies are emerging as a promising avenue. And while HSC transplants present substantial benefits for specific patients, there is an urgent need for safer treatment alternatives, like MSC and CAR T-cell therapies, that could potentially provide relief without the associated risks of traditional stem cell transplants.
What is promising is that this field is constantly evolving, with ongoing studies aimed at improving treatment outcomes for scleroderma. So keep an eye out as more information comes our way!
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