In 1983, two boys accidentally set themselves on fire. More than 97 percent of their bodies were burned. Their lives were saved by the physician Howard Green, who grafted regenerated skin onto the burned parts. Green’s technique has since saved countless lives and fuelled the entire fields of stem-cell biology and regenerative medicine. De Luca studied with Green in the early part his early career, and this procedure is almost identical to the one his mentor developed, except for one crucial part. He added gene therapy to the mix, modifying the stem cells A sheet of the laboratory grown genetically modified skin Photograph CMR Unimore behind the regenerating skin to correct the mutations behind Hassan’s condition. “What is nice about this study is the combination of gene and cell The surgeons spent two months grafting the skin grown in therapy together,” said Higgins, whose own work focuses on their labs onto the boy’s body through three surgeries replacing skin regeneration. “The success of this combined cell and gene about 80 percent of the skin. He spent months recovering, therapy will have huge implications for the field of regenerative covered in bandages, before being discharged from the hospital medicine and the treatment of genetic diseases.” in February 2016. The new epidermis was attached like a patchwork quilt, covering almost his entire body. Within a month, the graft had integrated into the lower layers of skin. “Once the epidermis has regenerated, the stem cells keep making the renewal of the epidermis as in a normal (healthy person),” said De Luca. “All the data we have … are telling us that this is going to be a stable situation.” Two years on, the skin is healthy, he doesn’t need to take medication or use ointments and when he gets a cut it heals normally. A potential risk of the treatment is that © The Atlantic the introduction of genetic changes could increase the chances of skin cancer – although the study found no evidence that dangerous mutations had been caused.
An article in The Atlantic, dated Nov 8, 2017, revealed that De Luca’s team is now running two separate clinical trials to test their gene-corrected skin grafts on around two dozen children with EB. His ultimate goal is to develop an effective and standardised procedure that could be carried out during early childhood, to prevent the painful blisters before they happen, rather than restoring lost skin after the fact. “It will take years to get there but it’s clearly doable,” he says. “Maybe this will be the last thing I’ll do in my career.” l ©The Atlantic
Figure A shows Hassan before treatment. Images C to E show his extraordinary recovery T. Hirsch et al Nature ©
Dr Laleh Lohrasbi is a pharmacologist. She has worked as an editor for the medical section of “Hamshahri”, a daily newspaper in Tehran.