In this new, recurring feature, we’ll explore a disease area that is currently being transformed by cell therapy. This month? Genetic skin diseases.
Two months ago, a research team in Italy announced they had successfully used gene and stem cell therapy techniques to almost entirely reconstruct and replace the skin of a 7-year-old boy afflicted with Epidermolysis bullosa (EB) a rare and often-fatal genetic skin disease.
EB causes skin to blister and tear off at the slightest touch. With no treatments available, patients must avoid even minor skin damage, and often require regular medical attention. The boy in this case had lost more than 80% of his skin to the disease and was confined to a hospital bed.
The combination technique began with stem cells grown from a 4-square centimeter of undamaged skin taken from the boy’s groin. Using gene therapy, the team was able to correct the mutation and use those corrected stem cells to grow sheets of new skin in the lab. These sheets were then grafted to the patient in three surgical sessions.
As the team revealed in a Nature story detailing the research and clinical findings, during the culturing process they were also able to identify the specific stem cells that produce skin cells.
“This is a significant advance in the field of autologous regenerative medicine, one that has been decades in the making. These therapeutic successes give hope to people suffering from diseases for which there are no effective treatments. The next step will be to prove that the same or similar approaches can be effectively applied to broader patient populations,” said Chris McClain, Head of Business Development and Sales, at Be The Match BioTherapies.
Skin diseases have long been seen as potential targets for gene therapy—but complexity of the tissue has been an obstacle. Skin cells regenerate every 30 days, with three different cells types contributing to the process. But researchers have long puzzled over the individual roles played by each specific cell type. This new finding could have major implications for biotech and pharma companies, such as Abeona Therapeutics, Fibrocell Science and Krystal Biotech, already pursuing cell therapy programs in EB and other genetic skin diseases.
Although long-term follow-up is still needed to ensure the boy suffers no adverse effects from the procedure, many are already hailing it as a major development in the use of stem cell therapies. And best of all, two years after the procedure, the boy is living the normal life of a 9-year-old.