Adult stem cells have been used successfully to treat children with a deadly skin disease known as recessive dystrophic epidermolysis bullosa (RDEB; one of the most severe forms of epidermolysis bullosa, a set of genetic skin diseases.) EB affects the skin and lining of the mouth and esophagus. It causes skin to blister and scrape off with the slightest friction. The blistering, peeling skin also leads to recurrent infections, and an aggressive form of skin cancer. Most children with EB do not live past their 20's. Previously, there was no treatment and it was considered incurable.

Now University of Minnesota researchers led by Dr. John E. Wagner and Dr. Jakub Tolar, along with international colleagues, have used adult stem cells from donor bone marrow or donor umbilical cord blood to treat EB successfully. Since 2007, they have transplanted a total of ten children with the most aggressive forms of EB; all of the children have responded to the therapy to varying degrees. Wagner said:

"To understand this achievement, you have to understand how horrible this disease actually is. 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."

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.

Tolar said:

"This discovery is more unique and more remarkable than it may first sound... 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.

"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."

Added 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."


The paper is published in the New England Journal of Medicine