The Blood and Marrow Transplant (BMT) Program at University of Minnesota Amplatz Children's Hospital is currently the only program in the world providing groundbreaking treatment for epidermolysis bullosa (EB), using a mixture of stem cells. EB is a condition that doctors previously considered untreatable, except for providing wound care and making the child as comfortable as possible.
A child born with EB has a deficiency of one of several proteins that anchor the skin to the body. One such protein is C7—an important form of collagen. Without enough of these proteins, the skin tears and pulls away from body easily, causing serious wounds and recurrent blistering. In severe cases of EB, the soft tissues inside the body are often also affected, such as the lining of the mouth, esophagus, stomach and intestines. Many children with the severest forms of EB often do not reach their teenage years, and some die in infancy.
Researchers at the University of Minnesota have been studying the use of stem cells to treat children with severe EB — in particular the recessive dystrophic and junctional forms of the disease. So far, the results have clearly demonstrated that stem cells from a healthy donor find their way (home) to the skin and secrete the protein that is missing in EB. For the patient, this can mean a substantial reduction in blistering and enhanced healing of the skin.
The EB program at University of Minnesota Amplatz Children's Hospital includes expert health care providers from a variety of specialties. These include dermatology, infectious diseases, anesthesiology, pediatric surgery, gastroenterology, nephrology, nutrition, and pain management. Each of these University of Minnesota Physicians' specialists is now experienced in the complex care required for patients with severe EB. Our expert multidisciplinary team makes every effort to ensure that the care we provide meets the unique needs of each patient and family.
Our physician specialists will evaluate your child's organ systems and dermatologic condition, including a detailed assessment of the skin and specific protein abnormalities. This helps us determine the type and severity of EB. Once we know the type of EB, members of our team will work with you to provide education and instruction in wound care, nutrition and other important aspects of day-to-day care. In addition, we will discuss opportunities for your child to participate in research studies and clinical trials. Through these trials, we are striving to enhance the benefits and minimize the risks to the patient.
Physicians and researchers at the University of Minnesota are already exploring newer and safer ways to treat EB and improve the quality of life for those who have the disease. We were the first to successfully use stem cells to treat EB. We are also actively involved in collaborating with researchers around the world in this process. Treating EB with stem cells, however, involves difficult decisions and extraordinary complex patient care. Our experienced, compassionate team is here to help you with the decision-making process and with the care of your child in any way possible.
On your one-day initial visit, you will meet with Dr. Wagner or Dr. Tolar; Heather Zierhut, M.S., C.G.C., our genetic counselor; one of our social workers; a nurse coordinator; and other specialists, depending upon your child's needs. The doctors will provide you with:
The genetic counselor will discuss:
The initial results of using stem cells for the treatment of EB have been very encouraging. We continue to develop new treatments for EB using different kinds of stem cells. Ongoing research includes:
We also are involved in new research using additional stem cells, called mesenchymal stem cells, for tissue repairs. Other research includes using the patient's own skin to create genetically corrected stem cells that are capable of making different kinds of tissues.