Stem-cell transplant: A possible high-risk/high-reward treatment for scleroderma

Follow me on Twitter @RobShmerling

Some medical news stories don’t get the attention they deserve. Perhaps it’s because the disease is rare and not many people have ever heard of it.

I think this story about a new treatment for scleroderma is a good example.

The term “scleroderma” means “hard skin.” The name comes from the way it causes thickening and hardening of the skin. With “limited scleroderma,” the disease is mostly confined to the skin. With the “systemic” form of disease, other major organs, including the digestive tract, lungs, heart, and kidneys, may be affected as well. It is considered an “autoimmune disease”; there is evidence that the immune system of people with scleroderma is abnormal and appears to be attacking its host.

Since the disease was first described by Hippocrates around 400 bc, reliably effective treatments have been unavailable. Existing treatments include moisturizers, medications to prevent heartburn and to improve circulation, and immune-suppressing medications. However, despite treatment, many people continue to suffer with bothersome symptoms and life-threatening complications such as severe scarring of the lungs or kidney failure.

Researchers publishing in a recent edition of the New England Journal of Medicine describe a new approach to treating severe scleroderma: stem-cell transplantation. With this treatment, stem cells (which can develop into many different types of cells) are removed and the body’s immune system is essentially wiped out with chemotherapy and radiation. The stem cells are then returned to the body where they rebuild the immune system — a sort of “rebooting” of the immune system.

It’s risky, especially soon after treatment begins, because there is a period of time in which the immune system doesn’t function well enough to protect the person from infections. In this study, 36 people with severe scleroderma received stem-cell transplantation and were compared with 39 otherwise similar people who received a year of standard immune-suppressing medication.

After 4.5 years, those assigned to receive stem-cell transplantation had

• improved overall survival compared with standard treatment (79% vs. 50%)
• less need for immune-suppressing medication (9% vs. 44%)
• fewer deaths related to worsening scleroderma (11% vs. 28%)
• more deaths related to treatment — (3% vs. 0%).

These findings suggest that stem-cell transplantation may be much better than standard treatment for people with severe scleroderma even though it is riskier in the short run.

Despite these encouraging results, other researchers need to reproduce these findings with larger and longer-term trials. And even if confirmed, there’s still plenty of room for improvement. Twenty-one percent of study subjects treated with stem-cell transplantation died within 5 years of treatment and treatment-related deaths were more common in this group. So, you can expect to hear about additional research that seeks to refine stem-cell transplantation and other treatment approaches for scleroderma.

Scleroderma remains a mysterious and sometimes deadly disease despite decades of research.  So, you can also expect researchers to report on new findings regarding how and why it develops in the first place. In addition, stem-cell transplantation would not be appropriate for less severe cases of scleroderma; we need better treatments for them as well.

News such as this represents progress for a condition that badly needs it. While you may not have heard much about this study in the news, it’s certainly one that will catch the attention of people with scleroderma, their loved ones, and their doctors.