On Wednesday, November 8, 2017, Medical News Today published a 7-year old’s true story. We would like to share that story with you.
After an un-common genetic condition destroyed nearly 80 percent of a child’s skin, doctors believed the boy would die. Fortunately, stem cell treatment saved his life. Do you want to know how stem cell and gene therapy saved this little boy’s life?
The disease is Epidermolysis Bullosa (EB), a severe and often lethal genetic condition, caused by mutations in the genes responsible for encoding proteins that hold the epidermis and dermis together.
Dr. Bruckner, from the Department of Dermatology at the University Medical Center, University of Freiburg and the EB Center Freiburg, in Germany, explained that people with EB are constantly in pain, and that skin layers can separate easily from minor mechanical stress or friction. Currently no cure exists for EB and patients require frequent hospital visits and medical treatment.
The patient’s family was sure that he would die, but then experts in the field of regenerative medicine gave them hope.
Dr. Tobias Rothoeft, a physician at the Department of Neonatology and Pediatric Intensive Care at the University Children’s Hospital, Ruhr University Bochum in Germany, co-authored a review of the treatment. The article was published in the journal Nature.
The patient’s story began in the summer of 2015, when the boy was admitted to the hospital, because he had developed an infection that caused him to lose nearly two thirds of his body surface area. He was admitted to the burns center in a septic state; so, the first challenge was to keep him alive. After trying several different treatments over the span of two months, physicians in the hospital believed there was nothing more they could do for the child. Desperate, the parents asked if there was anything else that could be done. The physicians decided to try using stem cells to treat the child.
Dr. Michele De Luca, director of the Centre for Regenerative Medicine, Stefano Ferrari, at the University of Modena and Reggio Emilia in Italy, has dedicated a big part of his life to developing therapies for skin and eye conditions.
While Dr. Rothoeft, and his team, were trying to keep the boy alive; Dr. De Luca and his colleagues were preparing the cells in their laboratories in Modena.
By the time the boy under-went his first operation; he had lost nearly 80 percent of his skin. The surgical team applied genetically modified skin, made from stem cells, to his arms and legs. Following surgery, the child was kept in an artificial coma for 12 days to keep him immobile, which allowed the cells to attach. The procedure was successful and the patient showed the first signs of progress!
The infinite supply of cells that Dr. De Luca was able to grow through regenerative medicine, and the use of stem cells, is a clear advantage for EB treatment. Physicians now had access to as many cells as they needed, more than double the number of cells needed to cover the entire surface of the body, enabling them to cover all open wounds. Stem cell treatment was a wonderful option for this child.
The patient under-went a second and third operation to cover the rest of the body. Following the third operation, the team of treating physicians, could stop his pain medication. After nearly 8 months in the intensive care unit, the young boy was able to leave the hospital and return home.
After 21 months, the boy’s skin is healthy (smooth, stable and able to heal), and he is doing great! He never has blisters where the transplantation was done. Typically, patients with EB can’t take part in outdoor activities because of the risk of damage to their skin; but for this patient the story is different, he’s happily playing soccer with his friends and siblings
The improvement to the patient’s quality of life is unbelievable! He went from being on morphine the whole day to no drugs at all; in addition, he now has the freedom to enjoy outdoor activities.
For stem cell and gene therapy, this is clearly a success story. Scientists were able to show, using only a small number of stem cells, they were able to regrow skin. Dr. De Luca speculates that stem cells exist in the skin for our entire lifetime. The researchers believe that once you’ve regenerated the epidermis with stem cells, these cells behave as normal epidermis.
The significance of these findings however, goes beyond the basic science of how our skin works; it also gives researchers such as Dr. De Luca the necessary information to grow just the right mix of cells in the laboratory for stem cell therapy.
The development of therapies like this, gives us hope that curative treatments will become possible, and clinically real, for many patients with different diseases.
The Stem Cells Transplant Institute in Costa Rica is a pioneer in regenerative medicine. Using advanced technology, the experts at our clinic can help you write your story of hope. Contact Us.