Skin Disease Cured by Adult Stem Cells -- Part Two of Two

Once suffering from a fatal skin disease, Nate Liao, 2, of Minneapolis, has been successfully treated using stem cells from umbilical cord blood and bone marrow. This was the first time such a treatment was attempted in humans.

“Nate’s quality of life is forever changed,” Dr. John Wagner of the University of Minnesota Medical School, told the Los Angeles Times. “Maybe we can take one more disorder off the incurable list.”

Dr. Wagner, who performed the treatment, added, “It’s not often that it feels like you hit a home run in medical research, but this one feels like it.”
After Nate’s success, his mother decided to have her older son, five-year-old Jake, receive the treatment as well. The transplant took place May 30.

The umbilical cord blood for Jake was not a perfect match since it did not come from a family member, so he will have to take anti-rejection medication. The University of Minnesota team is preparing to treat 9-month-old Sarah Rose Mooreland of Folsom, California as well.

The disease, recessive dystrophic epidermolysis bullosa, or EB, affects one in every 100,000 children. These children lack a critical protein called collagen type VII “that anchors the skin and lining of the gastrointestinal system to the body,” according to Thomas Maugh of the Los Angeles Times.

“Their skin is extraordinarily fragile,” he explained. “Tearing and blistering occur with minimal friction, leading to painful wounds and scarring. Solid food produces erosion of the esophagus. Death usually results from malnutrition, infections or aggressive skin cancer.”

Nate had spent his life before treatment eating only pureed foods to protect his esophagus and intestinal tract, and was constantly covered with bandages over his blistered skin, according to the Star Tribune.

Doctors at the University of Minnesota gave Nate a transplant of umbilical cord blood in October. Donated by Nate’s brother Julian, who does not have EB, the cord blood contained the protein missing in Nate’s body.

Nate’s condition has improved markedly since the treatment, and many of his blisters are gone. Skin has grown on his right leg, which previously only had a “translucent purple-and-red coating over his internal tissues and veins,” according to the Pioneer Press. He can also eat solid food, including his new favorite, spicy Doritos chips.

Wagner said that Lab tests show that Nate’s body is now making collagen type VII.

“I have watched Nate improve every day,” Theresa Liao, Nate’s mother, told UMN News. “It has been slow, but I believe we are on the road to him getting better “[His improvement to date] has made a tremendous impact on our lives.”

Wagner was equally enthusiastic. “We have established a new standard of care for these EB patients, beginning with Nate,” he told the Pioneer Press. “It’s now allowing this kid for the first time to take bandages off for part of the day, to be able to eat or drink [everyday foods]. We have significantly impacted his quality of life. What we believe is going to happen is it will get even better than it is today.”