According to a story from the Dartmouth Geisel School of Medicine, a group of researchers associated with the school recently published a study that indicated that certain scleroderma patients that were not responding well to conventional treatments could benefit from stem cell transplantation. This discovery offers hope for this group of patients, who have few alternatives to fall back on.
About Scleroderma
Scleroderma, which is also referred to as systemic sclerosis, describes a group of autoimmune diseases that can cause system-wide effects in the most severe cases. The mechanism of this disease is believed to be an autoimmune response in which the immune system mistakenly attacks body tissue. Some factors that may contribute to triggering the autoimmune response include mutations of the HLA genes and exposure to certain materials, such as certain solvents, white spirits, ketones, and silica. Symptoms are broad ranging and systemic, including kidney failure, erectile dysfunction, fatigue, stroke, headaches, facial pain, congestive heart failure, skin abnormalities, high blood pressure, chest pain, indigestion, and many more. Treatments are varied and depend on the symptoms, but most patients take medications in an attempt to suppress the autoimmune response. In severe cases, life expectancy is around 11 years from onset. To learn more about scleroderma, click here.
New Treatments Needed
There are no FDA certified treatments specifically for scleroderma. The substantial variability of this disease means that it is difficult to predict how patients will respond to treatment; a drug that may work wonders for one patient may have minimal effect on another.
About The Study
In this study, the scientists decided to separate differing groups of patients based on their genetic characteristics. The researchers used data from a prior study, which showed that scleroderma patients often saw substantial changes in gene expression after stem cell transplant, in order to do this.
What they found was that patients of a certain genetic expression deemed ‘fibroproliferative’ saw much greater benefit from stem cells transplant than they did from treatment with the immune system suppressing drug cyclophosphasmide. While more research is needed in order to more fully understand the molecular differences between patients that contributed to this result, the scientists hope that this data can be developed into a diagnostic tool for scleroderma that can help doctors understand what treatment patients are going to experience the greatest benefit from.
Check out the abstract from this study here.
