Stem Cells Used to Cure Deadly Skin Disease in Children

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It was believed that stem cells from bone marrow could only be used in bone marrow transplants to replace diseased or damaged bone marrow, but a novel use of those cells has proved that they can also cure the deadly skin disease epidermolysis bullosa.

ScienceDaily reports how medical school researchers John E. Wagner, M.D., and Jakub Tolar, M.D., Ph.D., in collaboration with researchers in Portland, Oregon, the United Kingdom, and Japan have for the first time used stem cells from bone marrow to repair the skin of patients with a fatal skin disease called recessive dystrophic epidermolysis bullosa, or RDEB.

Epidermolysis bullosa (EB), is a rare, genetic skin disease that causes skin to blister and scrape off with the slightest friction or trauma. It affects the skin and lining of the mouth and esophagus.

Before their work, there was no treatment and no chance for a cure. Euthanasia has been considered for newborns with severe conditions in some countries. Children with EB who survive do not live beyond their 20s or 30s because they develop an aggressive form of skin cancer.

Dr Jakub Tolar, associate professor of pediatric transplantation, described their remarkable breakthrough:

This discovery is more unique and more remarkable than it may first sound because until now, bone marrow has only been used to replace diseased or damaged marrow — which makes sense. But what we have found is that stem cells contained in bone marrow can travel to sites of injured skin, leading to increased production of collagen which is deficient in patients with RDEB.

They started studies in 2007 when 10 children with the most aggressive forms of EB have been transplanted at the University of Minnesota Amplatz Children’s Hospital. All ten responded well to the treatment in varying degrees. A significant step nonetheless, one that has placed them securely on the path of removing EB off the list of diseases with no cure.

Dr Tolar and his team are continuing the study with a refined approach to further improve the treatment.

Image credit: National Health Institute

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