"The groundwork of all happiness is health." - Leigh Hunt

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

Follow me on Twitter. @RobShmerling

Some medical news doesn't get the eye it deserves. Perhaps it's because the disease is rare and plenty of people have never heard of it.

I believe this story a couple of latest treatment for scleroderma is a great example.

What is Scleroderma?

The term “scleroderma” means “hard skin”. The name comes from the way in which it thickens and tightens the skin. With “limited scleroderma,” the disease is generally limited to the skin. With the “systemic” type of the disease, other vital organs may also be affected, including the digestive system, lungs, heart, and kidneys. It is taken into account an “autoimmune disease”; There is evidence that the immune system of individuals with scleroderma is abnormal and appears to be attacking its host.

Better treatment is required.

Since the disease was first described by Hippocrates around 400 BC, no reliable effective treatment is accessible. Current treatments include moisturizers, medications to forestall heartburn and improve circulation, and immune-suppressing medications. However, despite treatment, many individuals proceed to suffer from distressing symptoms and life-threatening complications resembling severe scarring of the lungs or kidney failure.

A brand new study of stem cell transplantation for scleroderma

Researchers published in a recent edition of New England Journal of MedicineDescribe a new method. For the treatment of severe scleroderma: stem cell transplantation. With this treatment, stem cells (which may grow to be many differing types of cells) are removed and the body's immune system is destroyed, primarily through chemotherapy and radiation. The stem cells are then returned to the body where they rebuild the immune system – a type of “rebooting” of the immune system.

This is dangerous, especially right after treatment begins, because there may be a time when the immune system shouldn't be working well enough to guard the person from infection. In the study, 36 individuals with severe scleroderma received stem cell transplantation and were compared with 39 otherwise similar individuals who received a yr of ordinary immunosuppressive drugs.

After 4.5 years, they were assigned to receive stem cell transplantation.

  • improved overall event-free survival compared with standard treatment (79% vs. 50%); Event-free survival means survival without serious lung, kidney, or heart complications.
  • Less need for immunosuppressive drugs (9% vs 44%)
  • More treatment-related deaths (3% vs 0%).

These findings suggest that stem cell transplantation could also be higher than standard treatment for individuals with severe scleroderma, even though it is more dangerous within the short term.

what's next?

Despite these encouraging results, additional research is required to discover those with scleroderma who're the very best candidates for stem cell transplantation and to cut back the chance of this treatment. There continues to be loads of room for improvement: many study subjects treated with stem cell transplantation died inside 5 years of treatment. Therefore, you may expect to listen to about additional research that seeks to enhance stem cell transplantation and other treatments for scleroderma.

Despite many years of research, scleroderma stays a mysterious and sometimes fatal disease. So, you can too expect researchers to report latest findings about how and why it develops in the primary place. Also, stem cell transplantation is probably not appropriate for less severe cases of scleroderma. We need higher treatment for them too.

Such news represents progress for a situation that's sorely needed. While you could not hear much about this research within the news, it's definitely something that can get the eye of individuals with scleroderma, their family members, and their doctors.