University doctors used an experimental stem cell treatment for two brothers with a rare skin disease that most often ends in skin cancer and death before adulthood. The Liao boys, younger brother Nate and older brother Jake, suffered from a disease called recessive dystrophic epidermolysis bullosa (RDEB). People with this condition cannot produce a protein necessary to form one of the collagens that hold layers of skin together.
The disease has devastating effects. The slightest pressure or bump forms huge blisters on the skin. The blisters cause the skin to slough off. The same process affects the lining of the mouth, esophagus and intestinal tract, making it impossible for children to eat normally. There was no treatment for this disease. That is, there was no treatment until the experimental process used on Nate and Jake.
Doctors at the University of Minnesota provided this treatment through a bone marrow stem cell transplant on each brother. They collected cord blood and bone marrow stem cells from a healthy sibling of Nate and Jake. They first treated two-year-old Nate, despite that trying this experimental technique was a big medical risk. Until then, it had only been tested on mice. Theresa Liao’s two year-old had already lost fingers and bore numerous scars due to the disorder. Knowing there was no existing cure, she felt they had little to lose by trying.
To the great joy of the boys’ family, as well as the surgeons, the procedure worked. The boy’s doctors believe they have found a cure for the painful disease. He said, “It’s not often that it feels like you hit a home run in medical research, but this one feels like it.”
Nate’s brother Jake was the second to receive a bone marrow transplant. In California there is currently a clinical research trial that will include 30 patients.
