Long-lasting wounds from a painful genetic skin disease can be healed with skin grafts genetically engineered from a patient's own cells, researchers reported in The Lancet.
In severe dystrophic epidermolysis bullosa, or EB, the skin is so fragile the slightest touch – even from clothing - causes blistering and wounds, eventually leading to large, open lesions that never heal.
"With our novel gene therapy technique, we successfully treated the hardest-to-heal wounds, which were usually also the most painful ones for these patients," study leader Dr. Jean Tang of Lucile Packard Children's Hospital Stanford in Palo Alto, California said in a statement.
Severe dystrophic EB is caused by a defect in the gene for collagen VII, a protein that normally holds the skin together. As a result, the layers of the skin separate in response to even slight friction.
To create the personalized skin grafts, doctors take a small biopsy sample of the patient's un-wounded skin and introduce a corrected version of the collagen VII gene to the skin cells. These cells are then grown into sheets of healthy skin.
For the late-stage study, 11 patients with recessive dystrophic EB had a total of 43 wounds treated with grafts. For each treated wound, the researchers also identified a comparable "control" wound on the same patient that was managed with traditional measures.
Six months later, 81% of treated wounds were at least half healed, compared with 16% of control wounds. Roughly two-thirds of treated wounds were at least three-quarters healed, compared with 7% of control wounds, and 16% of treated wounds had completely healed, compared with none of the control wounds.
In addition, grafted areas had less pain, itching and blistering.
The same research team had previously developed a gene therapy gel for treating smaller EB wounds.
"I hope that if these patients are diagnosed as infants and start the gene therapy gel, maybe they won't develop big wounds," Tang said. "But if the gels don't work and a wound does expand, the skin graft therapy is the right treatment. The life arc of their disease will, I hope, be modified, with less suffering."
An editorial published with the study notes that EB patients who participated in early-stage trials of the skin grafts still had decreased blistering and wounding, pain, and itch at grafted sites five years later.
In April, the U.S. Food and Drug Administration granted Abeona Therapeutics approval for the skin grafts as an EB therapy.
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