Wednesday, August 11, 2010

Stem Cells Used to Treat Children With Life-Threatening, Blistering Skin Disease

Source: University of Minnesota
Date: August 12, 2010

Summary:

University of Minnesota Physician-researchers have demonstrated that a lethal skin disease can be successfully treated with stem cell therapy. Medical School researchers John E. Wagner, M.D., and Jakub Tolar, M.D., Ph.D., in collaboration with researchers in Portland, Oregon, the United Kingdom, and Japan have for the first time used stem cells from bone marrow to repair the skin of patients with a fatal skin disease called recessive dystrophic epidermolysis bullosa, or RDEB. This is the first time researchers have shown that bone marrow stem cells can home to the skin and upper gastrointestinal tract and alter the natural course of the disease. The results are published in the New England Journal of Medicine.

Below is a summary of media coverage of about this development:

Los Angeles Times, August 11, 2010, 1:59 p.m. PDT: "Stem cell therapy appears successful in treating rare, deadly skin disease":

Stem cell therapies hold enormous promise. But, so far, there are few confirmed stem cell treatments beyond traditional bone marrow transplantation. Researchers reported Wednesday, however, that they have been able to use stem cells to treat a rare, often-fatal skin disease in children. The results of the experimental therapy suggest that stem cells from bone marrow can travel to injured skin cells and repair damage to those cells.


Agence France Presse (AFP), August 11, 2010, 5:04 pm ET: "Doctors use bone marrow stem cells to treat skin disorder":

In what is believed to be a medical first, researchers have used stem cells from bone marrow to repair the skin of young patients with a painful and usually deadly skin disease, a study published Wednesday says. Researchers led by University of Minnesota doctors John Wagner and Jakub Tolar in 2007 began treating children with a rare genetic skin disorder, called recessive dystrophic epidermolysis bullosa (RDEB), with bone marrow stem cells that had been found in lab tests to repair skin in mice.


Reuters, August 11, 2010 5:27 pm EDT: "Stem cells may hold key for fatal skin disease":

High-risk bone marrow transplants partially cured five children with a potentially deadly genetic defect in which proteins that hold layers of skin together are absent, U.S. researchers said Wednesday. But one other child died from side effects of a drug used to prepare for a transplant and a second died from a post-transplant infection.

People with recessive dystrophic epidermolysis bullosa, or RDEB, are plagued by painful blisters on the skin, mouth and throat, caused by the slightest trauma that can expose the body to infection and, in some cases, an aggressive form of cancer. With the new treatment, "there was improved healing, fewer blisters, and their quality of life was positively affected. They could do things they couldn't do before, like ride a bicycle or go on a trampoline," said Dr. John Wagner of the University of Minnesota, who worked on the study.

It was published in the New England Journal of Medicine.

Minneapolis Star-Tribune, August 11, 2010 - 8:37 PM CDT: "U doctors find treatment for painful, lethal skin disease":

Two years ago, doctors at the University of Minnesota took an enormous risk by putting a little boy with a terrible skin disease through a bone marrow transplant. For that boy, Nate Liao, it worked out, and he is healthier now. Thursday, in a study published in the New England Journal of Medicine, the researchers are for the first time making public their results treating seven other children with the same genetic disease. In it, they acknowledge just how risky the procedure is: Two of the seven, including the older brother of the first patient, died as a result of the treatment. But in the others it worked -- a leap forward for a devastating and painful genetic disease for which there is no other treatment and a potentially significant advance for the use of adult stem cells.


HealthDay News, August 11, 2010: "Stem Cell Treatment May Offer Hope Against Fatal Skin Disorder":

A debilitating and usually fatal skin disorder may be treated by bone marrow stem cell transplant, a new study finds. The results may have implications for the treatment of other skin diseases and also for the potential of stem cells in bone marrow to turn into other cell types, according to the study published in the Aug. 12 issue of the New England Journal of Medicine.