Scleroderma and stem cells – Transplantation found to “significantly improve outcomes”

What is Scleroderma?

Scleroderma, which means “hard skin” is an uncommon condition that tissue to become hard and thick. Usually affecting the skin on the limbs and body, scleroderma can also cause swelling in joints, muscles and internal organs and is potentially fatal.

Doctors believe scleroderma is caused by the immune system attacking the connective tissue under the skin and around internal organs and blood vessels and manage symptoms with cyclophosphamide, an immune-suppressing drug.

How might stem cells help?

Blood stem cells, known as haematopoetic stem cells or HSCs have the capacity to become any other type of blood cell and are already used to treat a range of conditions threatening the human blood and immune systems. By using stem cells to replace the cells lost during radiation therapy or chemotherapy, doctors enable new blood cells to form, regenerate and repair the patient’s immune system and aiding recovery.

A research team led by Dr. Keith M. Sullivan at Duke University has been studying HSC transplantation as a treatment for scleroderma and comparing this regenerative approach to outcomes following regular doses of cyclophosphamide.

“While treatment decisions should always be made on an individual basis, we hope that our work will help define a new standard of care for this severe, life-threatening autoimmune disease.” Dr. Keith M. Sullivan, Duke University

The study was funded by NIH’s National Institute of Allergy and Infectious Diseases. Results appeared in the New England Journal of Medicine on January 4, 2018.

After being monitored for 54 months, people who received the transplant procedure had significantly better health outcomes overall than those who received cyclophosphamide.

“We need effective therapies for scleroderma and other severe autoimmune diseases, which can be not only debilitating to the patient but also difficult to treat. These results add to the growing evidence that stem cell transplants should be considered as a potential treatment option for people with poor-prognosis scleroderma.” NIAID Director Dr. Anthony S. Fauci

Can cord blood treat scleroderma?

Whilst Scleroderma News lists umbilical cord blood as a source of the HSCs used to treat patients, clinical trials have so far been restricted to autologous hematopoietic stem cells, HSCs from their own bone marrow.

“Clinical trials in the U.S. and Europe have found these types of stem cells have dramatically improved skin fibrosis in scleroderma patients.” Scleroderma News

We know from more extensive research into other blood and autoimmune conditions that cord blood stem cells are more ‘plastic’ than those found in bone marrow, and more closely resemble embryonic stem cells. This means they do not require a perfect match, they are potent and they are less likely to result in graft versus host disease. 

In the Duke University study, researchers at 26 U.S. and Canadian sites randomly assigned 36 people with severe scleroderma to receive the hematopoietic stem cell procedure and 39 to receive one year of cyclophosphamide. Both types of treatment caused side effects, such as infections. It is possible that these side effects would be reduced by using cord blood HSCs.

We will follow reports closely as investigators continue to study participants to assess long-term health outcomes and keep you updated.


Stem cell transplant helps people with severe scleroderma

Myeloablative Autologous Stem-Cell Transplantation for Severe Scleroderma

Hematopoietic stem cell transplantation improved outcomes for patients with systemic scleroderma

3 Stem Cells Which Could Be Used to Treat Scleroderma

stem cell preservation

BSc (Hons) Microbiology

Chief Executive Officer | Biovault Family

Biovault Family CEO, Kate Sneddon, joined Biovault in July 2009 and became Chief Executive Officer in 2016. As health industry professional her experience includes working as a microbiologist and leader at GSK for over 10 years. Her expertise in cord blood banking has been recognised in her awards, features in Parliamentary Review and Parents Guide to Cord Blood, as well as contributions to research with UCL and others.

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