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Nate Liao with his mother

Theresa Liao, Nate's mother, relentlessly raised funds and sought a cure for her son's disease. She and her husband have another child with EB.

U offers unique chance

U physicians try to save a young boy's life

By Molly Portz

From M, winter 2008

"This magic moment...," Theresa Liao sang to her 18-month-old son Nate, holding him as potentially life-saving bone marrow stem cells were infused into his small body. Nate suffers from the most severe type of epidermolysis bullosa (EB), a genetic disease that causes skin to slough off with the slightest friction or movement. To protect their fragile skin, children with EB must be bandaged from head to toe and often suffer painful wounds that are slow to heal. EB even effects skin on the interior of the body and children need to eat soft foods to protect their intestines. The disease has no treatment and no cure and people with EB die young from skin cancer. Doctors at the University of Minnesota Children's Hospital, Fairview are hopeful that will soon change. On October 19, a team led by John Wagner, head of pediatric blood and marrow transplantation, transplanted bone marrow and cord blood cells into Nate from his brother Julian, who does not have EB. Julian is a 100 percent match for Nate. Wagner anticipates that Julian's bone marrow and umbilical cord blood will result in a new healthy blood system that produces type VII collagen, the protein missing in children with EB. The physicians don't know for sure if the treatment will work since it has never been tried before.

"It has been a long road to get here," says Theresa Liao. "If there is a chance my kids can be better, we have to try."

The Liao family raised funds to support the lab research. The U was the perfect place to do this work because of the close ties between researchers and clinicians, and the atmosphere of collaboration where competition bows to the common good.

University researchers were able to correct the disease in mice through bone marrow transplant (BMT) with a 25 percent success rate. They tested various types of stem cells to determine which would give rise to the development of type VII collagen and produce the anchoring fibrils that bind the skin to the body. The Liao family raised funds to support the lab research. They have four boys--two suffer from EB. If all goes well with Nate, his five-year-old brother Jake will receive a transplant in several months.

More on the story

To read more about Nate Liao's experimental treatment and to watch a video, see the November 1 issue of USA Today.

"It has been a long road to get here," says Theresa Liao. "We uprooted our lives and we aren't leaving Minnesota until both boys are transplanted. If there is a chance my kids can be better, we have to try. We have the chance to give hope to all the other kids and families who live with EB." This is the first time doctors have approached EB from a systemic perspective, using transplant as a means to rid the body of its defective blood system and replace it with a healthy blood system. "Our goal is to determine the usefulness of stem cells whether from the umbilical cord blood or adult tissues like bone marrow in the treatment of human disease," says Wagner. "There are hundreds of thousands of children and adults waiting for new breakthroughs in stem cell research. In two years, the team was able to move this project forward remarkably fast--from testing in animal models to treating patients. Time will tell whether this risky treatment will work as effectively in humans." Doctors anticipate that at 100 days post-transplant they will be able to judge whether Nate has benefited from the experimental treatment. The pediatric BMT program at University of Minnesota Children's Hospital, Fairview is internationally recognized for its pioneering work in umbilical cord blood and bone marrow transplantation, including the world's first successful BMT in 1968. In 2000, Wagner and his team performed the first umbilical cord blood transplant from a sibling whose embryo was selected for implantation because of its match as a donor. The program leads the nation in the use of umbilical cord blood in the treatment of adults and children and in development of innovative treatments of various rare genetic diseases such as adrenoleukodystrophy (ALD) and Fanconi's anemia.