Scientists have grown a replacement, genetically modified skin to cover nearly the entire body of a seven-year-old Syrian boy who was struggling with a devastating genetic disorder.
The treatment scars a rare and striking success for the field of regenerative medicine, which has been struggling to transform futuristic-sounding science in to therapies that make a difference to patients. In the latest demo, the life of the young boy – whose illness got come close to killing him – was transformed.
Before undergoing surgery, the boy had lost 80 percent of his skin, leaving him covered in untreatable, infected wounds. He was given morphine to cope with the pain great doctors were preparing to start palliative treatment after all typical therapies had failed.
Prof Cé dric Blanpain, a stem cell scientist at the Free of charge University of Brussels, described the work as one of the most amazing examples to date of the use of stem cells in human beings. “ There are very few diseases that have benefitted so far, ” he said. “ This is a beautiful example of something that has been unthinkable before the study. To replace and gene-correct the whole epidermis of a patient is just amazing. ”
Claire Higgins, a lecturer of bioengineering at Imperial University London, described the trial as “ a huge accomplishment and quite remarkable”.
The boy, that arrived in Germany in 2013 after his family fled Syria as refugees, was suffering from a genetic illness called junctional epidermolysis bullosa, which causes the skin to become sensitive and blister. By the time he came to be treated, he had dropped the surface layer of skin, called the epidermis, from nearly his entire body, with only the skin on his head as well as a patch on his left leg remaining intact.
His doctors, based at University Children’ s Medical center, Ruhr University Bochum, had attempted to graft skin through his father, but the transplant had been rejected. As a final resort, the team sought the help of Italian scientists whom had pioneered a technique to regenerate healthy skin within the laboratory – but had never attempted to use it to get such an ambitious case.
The Italian group, led by Michele De Luca at the University associated with Modena, had successfully grafted laboratory-grown, genetically modified epidermis onto small areas of the body, such as part of a leg. “ This is the first time that such an amount of body has been transplanted, ” said De Luca. “ He basically dropped almost completely his epidermis. ”
The particular boy’ s disease was caused by a mutation in a gene, called LAMB3, that produces a protein that anchors the epidermis to the deeper layers of skin beneath. With out this protein the skin blisters easily, causing chronic injuries and ulcers to form.
The treatment, outlined within a paper in the journal Nature, involved first taking a trial from the patient’ s remaining healthy skin. The researchers then genetically modified these skin cells, using a computer virus to deliver a healthy version of the LAMB3 gene into the nuclei.
The skin contains its own supply of specialised come cells, which allows the epidermis to be constantly renewed throughout existence, with cells turning over roughly every month. This also enables scientists to grow grafts in culture, simply by taking a little sample.
In this case, the team grew sufficient skin to cover almost the entire body of the boy. Throughout two operations in autumn 2015, the new epidermis has been attached like a patchwork quilt, covering almost his overall body. Within a month, the graft had integrated into the lower levels of skin.
The genetically modified cellular material in the graft include specialised skin stem cells that will meant once the transplant was integrated it was able to restore and sustain the healthy skin.
“ Once you have regenerated the epidermis, the stem cells keep producing the renewal of the epidermis as in a normal [healthy person], ” said De Luca. “ All the data we now have … are telling us that this is going to be a stable circumstance. ”
Two years on the boy is doing properly, his doctors said. His skin is healthy, he or she doesn’ t need to take medication or use ointments, they are back at school, plays football and when he will get a cut it heals normally. A potential risk from the treatment is that the introduction of genetic changes could boost the chances of skin cancer – although the study found simply no evidence that dangerous mutations had been caused.
In the future, if the treatment is shown to be safe in the long term, researchers believe the approach could be used to treat less serious skin disorders.
“ What is nice about this research is the combination of gene and cell therapy together, ” said Higgins, whose own work focuses on skin revitalization. “ The success of this combined cell and gene treatment will have huge implications for the field of regenerative medication and the treatment of genetic diseases. ”