Ill boy gets brand new skin with gene therapy
November 10, 2017 00:00:00
LONDON, Nov 09 (AP): Doctors treating a critically ill boy with a devastating skin disease used experimental gene therapy to create an entirely new skin for most of his body in a desperate attempt to save his life.
Two years later, the doctors report the boy is doing so well that he doesn't need any medication, is back in school and even playing soccer.
"We were forced to do something dramatic because this kid was dying," said Dr. Michele De Luca of the University of Modena in Italy, who got a call for help from the German doctors treating the boy.
The boy, then 7, was hospitalized in June 2015 with blisters on his limbs, back and elsewhere. He quickly lost about 60 percent of the outer layer of his skin and was put into an induced coma to spare him further suffering. Doctors at Children's Hospital at Ruhr University in Bochum, Germany, tried skin grafts from his father and donor skin, but all failed.
"He was in severe pain and asking a lot of questions," the boy's father said in a video provided by the hospital "Why do I suffer from this disease? Why do I have to live this life? All children can run around and play, why am I not allowed to play soccer? I couldn't answer these questions."
The boy's parents asked about experimental treatments, and De Luca and his colleagues were contacted. They had previously used gene therapy to produce a small piece of skin in a similar case. They told the family that the boy's precarious state meant that he might not survive the complicated surgeries needed to save him.