Stem Cells Helping Children With Life Threatening, Blistering Skin Disease Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Writer: Robert Valenzuela on Aug 16 2010.

University of Minnesota researchers use stem cells to treat children with life-threatening, blistering skin disease

Breakthrough is the first to push stem cell therapies beyond treatment of blood and marrow disorders

University of Minnesota Physician-researchers have been triumphant in finding a way to treat a deadly skin disorder using stem cell therapy.

Bone marrow stem cells were employed by medical school researchers for the first time, from John E. Wagner, M.D., and Jakub Tolar, M.D., Ph.D., in collaboration with researchers in Portland, Oregon, the United Kingdom, and Japan, in restoring the skin of patients with a deadly skin disease called recessive dystrophic epidermolysis bullosa, or RDEB.

Bone marrow stem cells, researchers revealed for the first time can dwell in the skin and upper GI tract and change the progress of the disease.

“Whether stem cells from marrow could repair tissues other than itself has been quite controversial,” said Wagner, director of pediatric blood and marrow transplantation and clinical director of the Stem Cell Institute. “But in 2007 we found a rare subpopulation of marrow stem cells that could repair the skin in laboratory models. This astounding finding compelled us to test these stem cells in humans. This has never been done before.”

“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,” said Tolar, associate professor of pediatric transplantation.

“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.”

Epidermolysis bullosa (EB), are inherited skin disorders that are as rare as they come.

A trifling contact will lead to skin sore and peeling off.

It involves the skin and lining of the mouth and esophagus.

It was incurable before and for babies born with the most serious type, mercy killing was considered as the first recourse in some countries.

Many EB victims will eventually develop skin cancer, if they donít die right after birth, and may live up to their 20′s or 30′s.

Not many will survive to live long enough and death is mostly the outcome for the most serious types.

“Bone marrow transplantation is one of the riskiest procedures in medicine, yet it is also one of the most successful,” said Tolar. “Patients who otherwise would have died from their disease can often now be cured. It’s a serious treatment for a serious disease.”

Since Wagner and Tolar started the research in the fall of 2007, ten children diagnosed with the most serious kind of EB have undergone transplants at the University of Minnesota Amplatz Children’s Hospital. All the children have shown some varying results as a consequence of the operations.

“To understand this achievement, you have to understand how horrible this disease actually is,” said Wagner. “From the moment of birth, these children develop blisters from the slightest trauma which eventually scar. They live lives of chronic pain, preventing any chance for a normal life. My hope is to do something that might change the natural history of this disease and enhance the quality of life of these kids.”

There are several ways that Wagner and Tolar monitor the improvements that each child experiences. The doctors use clinical means in observing the progress of the skinís stability after the transplant. The lab quantifies how the donated cells are adapting to the skin and appraise the presence of collagen 7, the protein absentee in patients with RDEB, which does the job of sticking the skins together on top of each other and to the flesh.

“What we now know is that after this treatment, healthy donor cells reside in the skin, collagen 7 consistently increases over time and the skin gradually becomes more resistant to blister formation.” said Wagner. “This discovery expands the scope of marrow transplantation and serves as an example of the power of stem cells in the treatment of disease.”

“While the treatment offers a chance for a better life, it comes with significant risk,” said Tolar. “Two children have died from complications related to the treatment, so refinements are needed.”

In fact, earlier this year Wagner and Tolar launched a new generation of the study by combining different stem cell populations. “We are fully conscious of what we have accomplished so far and the enormity of what else needs to be done,” said Tolar. “But we have one goal – to take EB off the incurable list.”

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