Gene therapy for healing: Researchers replace the entire skin of a seriously ill child
It is a last attempt to save a little boy from dying: a gene therapy to cure his severe skin condition. The attempt succeeds. Clinical everyday life will not be such a gene therapy for the time being.
With gene therapy, doctors have cured a young boy of a life-threatening hereditary skin disease. They took some of the skin cells from the child, smelt a healthy variant of the faulty gene in the laboratory and then multiplied the cells. The regrown healthy skin was transplanted to almost the entire body surface of the boy. He is today, almost two years after the procedure, largely free of complaints. The researchers present their therapy, which was conducted under the direction of Bochum scientists, in the journal “Nature”.
The Freiburg dermatologist Leena Bruckner-Tuderman, who was not involved in the study, speaks of a very good work. The physician expects improvements in the methodology help for other patients with other variants of the disease. “But this is a dream of the future, we have not got that far yet,” says the medical director of the Department of Dermatology at the University of Freiburg.
What was the child suffering from?
The treated boy suffered from the hereditary disease Epidermolysis bullosa, also called butterfly disease. The upper skin layer is only insufficiently anchored in the underlying skin layer. Even the smallest mechanical loads lead to blistering and detachment of the skin. Massive chronic wounds are the result. This not only severely limits the quality of life, it also often leads to skin cancer. A cure is not possible so far.
When the boy arrived at the Bochum Children’s Hospital in 2015 at the age of seven, 60 percent of his skin surface had already been destroyed. He was completely emaciated by the severe chronic wounds and infections. The usual treatments did not work, so basically only a palliative medical treatment came into question. At the request of their parents, the doctors looked for experimental therapy options – and came across the hitherto barely proven gene therapy.
How did the researchers go?
The skin tissue, which had been corrected for the genetic defect, was cultivated in Italy and transplanted in Germany. Overall, the physicians replaced 80 percent of the skin of their little patient. “At the beginning of the treatment, the boy lay like a mummy in his bed, he was wrapped in bandages from head to toe,” says Tobias Rothoeft of the Children’s Hospital in Bochum, who has helped the boy during his approximately eight-month hospital stay. “Today, his skin is stable, he goes to school, plays football and can lead a largely normal life.” Injuries to the new skin healed in him like any other child.
“It’s the first person to be treated like this, we have to wait and see if everything will continue to go so well, and that will show the time,” says Tobias Hirsch from the Bergmannsheil University Hospital, which operated on the boy. For him as a plastic surgeon it is “a miracle and a blessing” that the boy is doing so well. According to Hirsch, around 35,000 children in Europe are affected by the butterfly disease.
Are there risks?
Basically, gene therapies like the one presented have the risk of integrating the new gene in an unfavorable part of the genome. As a result, regulatory processes in the cell can be disturbed, resulting in cancer. Whether the treated boy will ever get problems, will show – so far, the researchers found in him no tumor or other harmful developments.
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