U researchers use stem cells to cure rare skin disease

The University’s Stem Cell Institute looks at all different uses for stem cells.

The University announced last week the discovery of a presumed cure for a young child with a rare disease, called RDEB for short, which came from what some believe to be the likeliest of sources: stem cells.

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To learn more about stem cells and the ongoing research on campus, go to www.stemcell.umn.edu

John Wagner , professor of pediatrics, was the leader of the group that conducted the research and performed the stem cell transplant that cured the skin disease.

Wagner called RDEB a “painful, debilitating, scar-forming” skin disease in which the skin of the patient does not properly attach to the body.

Those who suffer from it get tears and blisters on their skin very easily – patients rarely live into their 20s, Wagner said, but those that do are at a high risk for cancer. Two thousand to 3,000 people in the United States have some form of the disease, he said.

The University has performed two transplants for the disease: one in October on 2-year-old Nate Liao from New Jersey, and one on May 30 for his older brother, Jacob.

The process was an example of quick scientific research, Wagner said.

In 2004, Wagner said the children’s mother approached him, “desperately seeking some option” for her children. Wagner began researching the disease and performing tests on mice to see if testing made an impact on the disease.

When a certain type yielded a result – three surviving mice out of 20 – Wagner said he was ready to justify a human clinical trial.

“By selecting out this population of cells that no one had done before, we were able to show that it gave rapid recovery,” he said of the chosen bone marrow stem cells.

Now, months after the stem cell transplant, Nate’s skin appears to be attaching properly, Wagner said.

The Liao family’s case shows a clinical application for stem cells. Jonathan Slack , director of the University’s Stem Cell Institute , said the process could have big implications for stem cell research.

“The scientist in me, and others, is really interested to know why it works,” he said.

The Stem Cell Institute is currently researching many possible implications for stem cells, including areas such as diabetes, and cardiac and muscle problems.

There are two main types of stem cells studied, Slack said – the controversial embryonic stem cells, which come from the embryos of animals and humans, and adult stem cells, which come from many areas of an adult body, such as bone marrow.

“Any disease where cells are dying or could possibly be replaced by transplantation is a potential indication for stem cell treatment,” he said.

Slack said there is a paradox in stem cell use; despite knowing less about adult stem cells than the embryonic variety, most clinical cases use them, he said.

Students have also gotten involved in stem cell research on campus.

Neuroscience sophomore Jenny Zick is about to begin researching stem cells and their effect on spinal cord injuries.

Zick said she is likely to continue researching stem cells in the future.

“Assuming that we are able to use embryonic stem cells and get funding for more different types, then it will help cure a lot of diseases, it will help research in all areas of medicine,” she said.

Rep. Phyllis Kahn , DFL-Minneapolis, co-authored a bill that would allow the University to use state funds to research embryonic stem cells. The bill was passed by the Legislature, but Gov. Tim Pawlenty vetoed it May 23.

Kahn said the bill became known as the “Kahn Cloning Bill,” because it allowed the controversial use of therapeutic cloning, in which a nucleus from a person’s cells are inserted into an egg with no prior nucleus to create a new, matching embryo.

“We can’t really maximize the potential of adult stem cells without the parallel embryonic stem cell science,” she said.

The federal government currently doesn’t fund embryonic research, Kahn said.

Matt Hanzlik , co-chair of the University’s Student Society for Stem Cell Research , said he predicts stem cells will yield a cure for a major disease, such as diabetes, within five years.

“I think after that first cure or treatment is developed, I see the flood gates opening,” he said.

Slack said he thought stem cell research would bring out similar results.

“In the long term,” he said, “this technology has to be fabulously important. It’s got to make an enormous difference to everybody’s lives.”