Stem Cell Treatment Allows Skin Regeneration for Young Patient with Severe EB
Orange County, CA - November 10th 2017 - After a rare skin disease hospitalized 7 year old Hassan, doctors were forced to resort to experimental medicine to treat the blisters forming on his body including limbs, back, and numerous other places. Suffering from a genetic disease called epidermolysis bullosa, 60 percent of Hassan’s skin was covered in open sores and blisters. Patients with EB are occasionally called "butterfly children" because their skin is as delicate as a butterfly's wings; tearing and blistering at the slightest disturbance. Complications seen in these patients include skin cancer and deadly infections.
EB is a mutation of any of several genes that encode proteins vital for anchoring the epidermis to the tissue below. The missing or defective protein can lead skin to shed from the smallest of injuries, creating chronic injuries prone to infection. Some forms of EB are fatal in infancy, while others expose patients to an aggressive and deadly skin cancer. "The current treatment involves painfully dressing and redressing wounds on a daily basis adding up to costs of $100,000 a year in bandage," said Peter Marinkovich, a dermatologist at Stanford University in Palo Alto, California, who treats EB patients. “They’re like walking burn victims.”
Originally from Syria, Hassan’s family moved to Germany in hopes of finding a doctor that could cure his disease which had progressively worsened since infancy. Finally, in 2015, Hassan was admitted to the Children’s Hospital at Ruhr University in Bochum, Germany. In the failure of available conventional therapies, the medical team decided to try an experimental approach: they transplanted skin derived from genetically modified stem cells onto the wounds’ surfaces. Similar to established treatments of burn victims, this endeavor takes sheets of healthy skin grown from an individual’s own cells and grafts them over wounds.
The team of doctors in Germany reached out to stem cell biologist and physician Michele De Luca and his colleagues at the University of Modena and Reggio Emilia in Italy for collaboration on a treatment plan. De Luca’s team had previously treated two EB patients with the skin growth method with promising results published in 2006. The strain of EB that Hassan suffered from was congruent with the studied strain that De Luca’s team targeted in their ongoing clinical trial, however Hassan’s condition required immediate attention. The emergency treatment would be the first test of the gene therapy to cover such a large and severely damaged area.
Using a patch of unblistered skin no larger than a postage stamp, the team fostered the growth of epidermal cells and included stem cells periodically used in skin regeneration. Healthy copies of the needed gene were then injected and grew into sheets ranging from 50 to 150 square centimeters. In two surgeries the team at Ruhr University encompassed Hassan’s arms, legs, back, and some of his chest in the new skin. After a month, most of the new skin had begun to regenerate and covered 80% of his body in strong, elastic epidermis. The researchers reported their findings in the November copy of the online journal Nature. In the 2 years since his surgeries, Hassan has been able to live a normal lifestyle and hasn’t developed any blisters in the grafted areas.
According to the international medical team, Hassan is the first patient in the world who has been treated with skin transplants from transgenic epidermal stem cells to this extent. Dr. Tobias Hirsch, one of the doctors from Ruhr University said, "This approach has enormous potential for research into development of new therapies for thetreatment of epidermolysis bullosa as well as other diseases and trauma causing large skin defects.”
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Stem Cell Treatment Allows Skin Regeneration for Young Patient with Severe EB Orange County, CA – November 10th 2017 – After a rare skin disease hospitalized 7 year old Hassan, doctors were forced to resort to experimental medicine to treat the blisters forming on his body including limbs, back, and numerous other places. Suffering from a genetic […]