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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.