Stem cells used to treat kids with lethal skin disease

Washington: Scientists at the University of Minnesota Physician have shown that a lethal skin disease can be successfully treated with stem cell therapy.

John E. Wagner and Jakub Tolar, 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.

This is the first time researchers have shown that bone marrow stem cells can home to the skin and upper gastrointestinal tract and alter the natural course of the disease.

"Whether stem cells from marrow could repair tissues other than itself has been quite controversial," said Wagner.

"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," Wagner added.

Tolar said, "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," Tolar added.

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. Previously, there was no treatment and no chance for cure.

In some countries, even euthanasia has been considered for newborns with the severest forms. If children with EB do not die of infection in their early life, many with the disease do not live beyond their 20s or 30s because they develop an aggressive form of skin cancer. While a few will live long term, the severest forms of EB are generally lethal.

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


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