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Boy gets an almost completely new skin transplanted
For the first time in the world, a team of doctors successfully treated a patient with transplants from genetically modified stem cells. Due to a rare disease, the boy lost about 80 percent of his epidermis. The condition of the child was critical. The physicians had to act quickly, because already failed all conventional therapies.
For the first time, a treatment team from the burn injury center of the Ruhr University Bochum and the Center for Regenerative Medicine of the University of Modena (Italy) successfully treated a boy with major skin damage with transplants from genetically modified stem cells. The boy suffers from the so-called butterfly disease, a genetic skin disease that has destroyed about 80 percent of his epidermis.
For the first time skin transplantation from genetically modified stem cells succeeded. (Image: edwardolive / fotolia.com) After all established therapies had failed, the Bochum team of physicians opted for an experimental approach: they transplanted skin from genetically modified stem cells to the wound surfaces. The treatment was successful, so that today, around two years later, the boy can return to family and social life. The doctors report in "Nature".
Life-threatening condition
The butterfly disease, scientifically Epidermolysis bullosa, refers to a congenital skin disease that is considered incurable. The cause is damage to protein-forming genes, which are essential for the skin structure. Even the slightest impact or impact on the skin surface can lead to the formation of blisters, wounds and skin loss with scarring. Depending on the severity of the disease, internal organs may also be affected or severe functional disorders may be caused.
The disease severely limits the quality of life of those affected; Often it is life-threatening. As in the case of the then seven-year-old Hassan: When he was admitted to the pediatric intensive care unit of the Catholic Hospital in Bochum in June 2015, 60 percent of his epidermis was lost. "He suffered from severe sepsis with a high fever and weighed only 17 kilograms - a life-threatening condition," Dr. Tobias Rothoeft, Senior Physician at the Department of Pediatric and Adolescent Medicine at the Catholic Hospital Bochum. All conservative and surgical therapy attempts failed.
Worldwide new therapy concept for major skin defects
Due to the poor prognosis, the Bochum team of Pediatricians and Plastic Surgeons decided in cooperation with Prof. Dr. med. Michele De Luca from the Center for Regenerative Medicine at the University of Modena for an experimental therapy: the transplantation of genetically modified epidermal stem cells. These stem cells of the patient were obtained by skin biopsy and further processed in Modena. To do this, the researchers inject the intact gene into the stem cells. In the process, so-called retroviral vectors are used, ie virus particles specifically modified for gene delivery.
The genmodified stem cells were grown in a cleanroom laboratory and then processed into transgenic skin grafts. After approval of the parents, the approval of the competent authorities and the state of North Rhine-Westphalia and the certification of the surgical center of the University Hospital Bergmannsheil as a genetic engineering device, the transplant could be carried out.
80 percent of the body surface transplanted
The transplants were transplanted to the arms and legs, entire back, flanks, and parts of the abdomen, neck, and face at Bergmannsheil University Department of Plastic Surgery. "Overall, the small patient was transplanted 0.94 square meters of transgenic epidermis to cover all defects and thus 80 percent of his body surface," said Privatdozent Dr. med. Tobias Hirsch, Senior Consultant at the Department of Plastic Surgery and Heavy Burned Persons at Bergmannsheil.
After the first transplant in October 2015, the condition of the patient began to improve. The transgenic stem cells formed a new epidermis with intact binding protein in the area of all transplanted areas. The integration of the intact gene by the retroviral gene transfer into the genome of the epidermal stem cells had worked and could be proven to be stable.
Very good treatment result
In February 2016, Hassan was released from inpatient treatment. Today, almost two years after the start of the treatment, the transplant areas show a high-quality, stress-resistant skin with intact refatting, beginning hair development and no scar contractures. Hassan returns to primary school and participates in the social life of his family.
According to the international treatment team, Hassan is the first patient in the world to receive skin grafts from transgenic epidermal stem cells. "This approach offers significant potential for researching and developing new therapies for the treatment of epidermolysis bullosa and patients with major skin damage," says Tobias Hirsch.
The case is considered unique in this dimension worldwide. "Transplanting 80 percent of the skin and monitoring the patient intensively for eight months was an extreme challenge," emphasize Tobias Rothoeft and Tobias Hirsch. "The close cooperation between the Bochum hospitals and the expertise of the University of Modena has led to success. We are very proud of this."
From Bochum involved in the therapy
Bochum University Hospital Privatdozent dr. Tobias Hirsch, Senior Physician of the Clinic for Plastic Surgery and Heavy Burned Persons at Bergmannsheil (Director: Prof. Dr. Marcus Lehnhardt) and Dr. med. Tobias Rothoeft and dr. Norbert Teig, Senior Physicians of the Department of Paediatrics at the Catholic Clinic Bochum (Director: Prof. Dr. Thomas Lücke).