Stem cell transplants may be the next new therapy for scleroderma

Date: Tue 26th February 2019

The results of a study spanning several years indicate that there may be long-term benefits of stem cell transplant in those suffering from severe scleroderma.

A stem cell is an undifferentiated cell that has the ability to turn into a specialised cell with a distinct function. This multi-centre study by researchers at Dartmouth, alongside other institutions, and led by Dr Jennifer Franks focused on comparing improvements in those treated with cyclophosphamide, the more conventional drug therapy for scleroderma, to those who had undergone a stem cell transplant.

The team of researchers at Geisel School of Medicine at Dartmouth, led by Dr Michael Whitfield, conducted molecular analysis to determine which scleroderma patients would advantage the most from the procedure. Molecular analysis is a biological technique which involves scientists studying the tissues, cells and fluids in the human body by examining the genetic blueprint of individuals. Leading from this it was possible to divide the patients into three groups, based on their gene expression: normal-like, inflammatory and fibroproliferative. It was concluded that patients in the fibroproliferative subset, who tend to respond more poorly to immunosuppressive therapy such as cyclophosphamide, were the most likely to benefit from stem cell transplants.

The team treated half of the fibroproliferative subset with cyclophosphamide and performed stem cell transplant surgery on the other half. The stem cells used in this instance were taken from bone marrow and umbilical cord blood. It was observed that those with a transplant experienced significant improvement in their condition compared to those who only received the drug. The team noted that the transplants are dangerous, invasive and costly, so it is critical that patients who undergo the surgery reap significant benefits from the transplant.

The researchers are now working on building upon these findings to create a personalised medicine approach for scleroderma, as different patients may respond differently to the same treatment due to the heterogeneous nature of the disease.

Next steps also include attempting to understand why one group of patients may positively respond to the surgery whereas the others do not. The fundamental aim is to be able to predict patients' responses to therapy. By achieving this, the team believe that we can then give the most effective treatments, such as stem cell transplant therapy, to individual patients based on the severity of their condition.

For some people living with severe scleroderma, these treatments can represent one of the best chances at longer term survival. SRUK works with clinicians and researchers in the UK to accelerate the progress of research, so that the benefits reach people sooner rather than later. Your donations and support means that we can continue to support groundbreaking research and improve the lives of people living with Scleroderma and Raynaud's.

If you are interested in helping SRUK to fund more work like this, then please visit our donations page. We rely on the generosity of our community to continue to support groundbreaking research in both scleroderma and Raynaud's.

If you would like more information regarding other treatments for scleroderma, please go to: Scleroderma Treatments

Information on another piece of new research can be found here: Gastroesophageal Reflux Contributes to Lung Scarring