Morning Briefing
Summaries of health policy coverage from major news organizations
Scientists Genetically Modify Stem Cells To Grow Skin For 7-Year-Old Boy With Fatal Disease
The Washington Post: Genetically Modified Skin Grown From Stem Cells Saved A 7-Year-Old Boy’s Life
Scientists reported Wednesday that they genetically modified stem cells to grow skin that they successfully grafted over nearly all of a child's body — a remarkable achievement that could revolutionize treatment of burn victims and people with skin diseases. The research, published in the journal Nature, involved a 7-year-old boy who suffers from a genetic disease known as junctional epidermolysis bullosa (JEB) that makes skin so fragile that minor friction such as rubbing causes the skin to blister or come apart. (Cha, 11/8)
Stat: ‘Extraordinary’ Tale: Stem Cells Heal A Young Boy’s Lethal Skin Disease
The doctors were ultimately able to reconstruct fully functional skin for 80 percent of the boy’s body by grafting on genetically modified cells taken from the boy’s healthy skin. The researchers say the results of this single-person clinical trial, published on Wednesday in Nature, show that transgenic stem cells can regenerate an entire tissue. De Luca told reporters the procedure not only offers hope to the 500,000 epidermolysis bullosa patients worldwide — but also could offer a blueprint for using genetically modified stem cells to treat a variety of other diseases. (Blau, 11/8)
The Wall Street Journal: New Skin for 7-Year-Old Boy Marks Advance In Gene Therapy
The new skin remains functional after 21 months, and doesn’t blister or require ointment or medication, according to the team of scientists and doctors from Austria, Germany and Italy who described the case in a paper in the journal Nature. The child in the study has Junctional Epidermolysis Bullosa (JEB), part of a family of rare, often lethal, skin-blistering diseases. Epidermolysis Bullosa affects only around 1 in every 20,000 births in the U.S.; JEB is a severe form of the disease, often leading to death in early childhood. (Dockser Marcus, 11/8)
Los Angeles Times: 9-Year-Old Boy With Rare Disease Now Has Engineered Skin Covering 80% Of His Body
Over a five-month period in the fall and winter of 2015, they harvested some of the boy's few remaining healthy skin cells. Using a virus to invade the cells, they introduced a corrected version of the mutated gene that had caused the catastrophic failure of his epidermis, the body's largest organ. They cultured the corrected cells and, on sheets backed with plastic and a naturally-occurring adhesive, used them to build many square meters of healthy new epidermis. Then, ever so delicately, they clothed his small body in a new set of skin. (Healy, 11/8)
NPR: Genetically Altered Skin Saves A Boy Dying Of A Rare Disease
After eight months in the intensive care unit, the boy was well enough to go home. And, two years later, he is in school, even playing soccer. "The kid is doing quite well," Rothoeft said. "The skin is of good quality, it doesn't need any ointments or stuff like that. It's perfectly smooth and it is quite stable. And if he gets any bruises, they just heal like bruises in every other kid." (Harris, 11/8)
