Doctors in Bochum, Germany and scientists at a research laboratory in Modena, Italy managed
When the boy was an infant, the doctors found that his LAMB3 gene was dysfunctional and since then he had started developing nasty ballooning blisters on his body along with creases in his face. The doctors treated the child for preventing the blisters for seven long year. Though, by 2015, the condition had destroyed 60 per cent of his skin. The child was taken to the burn unit of the Ruhr University Children’s Hospital in Bochum, Germany in June. He had contracted septic from a certain strain of staph combined with high fever. Despite heavy doses of antibiotics, painkillers, iodine baths, wound dressing, the treatments did not help. When the boy’s father gave some of the skin from his own body, the child’s body rejected it and after a five-week-long struggle in the intensive care unit, the child had reached a life threatening stage.
Desperate times called for desperate measures and the doctors turned to researchers in neighbouring Italy for a skin culture procedure. Doctors snipped a small square portion of the boy’s skin and sent it to the Modena-based lab. The researchers injected the LAMB3 gene into the cells of the skin patch including some stem cells using a virus. Then they started the culture process. The cells grew till they could seed them onto a nine sq ft gauze and protein gel which was just enough for the child.
The newly grown patch of skin was shipped to Germany from Modena in October which was then grafted onto certain areas on the arms and legs of the boy. A second patch was also sent a month later and that was grafted onto the chest and back portion. By January, they were finishing up the spots which were missed.
It took seven-and-a-half months for the child to be treated since he was admitted at the hospital but after the completion, he walked out without a wound on his body. The doctors and scientists had performed the largest-ever infusion of transgenic stem cells. The procedure was so effective that the child returned to normal routine quickly. Within a few week, he was back in elementary school and there has not been any blister on his skin since after the treatment.
The story of the child told in journal Nature and reported by Wired shows the immense potential and promise of stem cell and gene therapies. To treat an incurable disease via genetically engineered stem cells tells the story of progress of medical research in the area.
“For the first time outside of the hematopoietic system we’ve been able to show that a transgenic stem cell can permanently regenerate an entire tissue,” the report quoted Michele De Luca, director of the Centre for Regenerative Medicine at University of Modena and Reggio Emilia, and first author of the paper as saying.
However, a point of caution was highlighted b Paul Knoepfler, biomedical researcher at University of California, Davis who said that the faulty gene is still present in the child’s body like in the heart, lungs, esophagus etc. However, the non-mutated LAMB3 genes in the transgenic skin cells that cover the boy’s body seem to be enough to make them functionally healthy.
In the paper, the Italian researchers themselves pointed out that the intervention was incredibly aggressive and that such a treatment should only be used in life threatening situations.