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Grief leads to hope for others

Nine-month-old Sarah Mooreland died before she could receive a University-developed experimental treatment for her rare skin disease. Today her parents, Jay and Lonni, have created a fund in her name to help advance the breakthrough research.

Family’s research fund helps U physicians hone in on a cure for a devasting skin disease

The minute Sarah Mooreland was born, her parents knew that caring for her would be different from caring for their two other children. Clearly, something was wrong with Sarah’s skin.

Jay and Lonni Mooreland, of Folsom, California, soon learned that the problem was serious; Sarah had a life-threatening skin disease called recessive dystrophic epidermolysis bullosa (EB).

“We were totally shocked,” Lonni Mooreland says. “We had never even heard of EB before.”

But what they learned was frightening. In people with EB, the body lacks a type of collagen that helps skin attach to the body. That makes their skin extremely delicate—even the slightest touch can cause the skin to tear or blister.

In severe cases, EB also affects the lining of the gastrointestinal tract. Coughing and swallowing can tear the lining of the esophagus and stomach.

As a result, people with EB are very susceptible to infection and can easily become malnourished. If they live long enough, they will likely acquire a fatal, aggressive skin cancer in young adulthood.

And, devastating to the Moorelands, there was no cure.

A glimmer of hope

The Moorelands kept searching. Through an online EB network, Lonni learned that a University of Minnesota team led by blood and marrow transplantation (BMT) pioneer John Wagner, M.D., had developed an experimental therapy aimed at curing EB and had just given it to a child as part of a clinical trial.

The trial marked the first time physicians had approached EB treatment systemically, using marrow-derived stem cells as a way to replace the missing collagen. In theory, with the help of an infusion of stem cells from a healthy donor, the body would start producing the collagen and correct the underlying genetic defect.

“This work can’t be done just anywhere,” Wagner says. “It’s being done at the University of Minnesota for a reason: We have all of the elements required to make this research happen.”

Those elements, he says, include a first-of-its-kind Stem Cell Institute, a Center for Translational Medicine that helps move new therapies from the laboratory to patients faster, and a group of world-class investigators who are thinking about how the immune system will respond to new stem cells.

John Wagner, M.D., made news last year when Nate became the first person to undergo an umbilical cord blood and bone marrow transplant aimed at curing epidermolysis bullosa. Nate is now thriving, and his body is producing the collagen it was once missing.

Worth the chance

They knew the treatment would be risky. Studies in mouse models looked promising, but only two other humans—2-year-old Nate Liao and his 5-year-old brother, Jacob—had undergone the experimental therapy.

For Jacob, it was too soon to tell whether the transplant was working. But for Nate, who’d had the therapy seven months earlier, it appeared that the transplant was beginning to take. Lab tests showed that his body was producing the missing collagen. He wasn’t blistering as much as before, his face plumped up, and he was asking for potato chips, a food he was now able to eat for the first time.

On top of the risk involved in any experimental therapy, Sarah had had some kidney and heart problems shortly after she was born.

But Jay and Lonni felt that the potential benefit was worth the risk. Without the transplant, Sarah likely would live much of her life in pain and die in her teens or early 20s of skin cancer.

“We had to try—for her,” says Jay. “If people don’t step forward and take the risk, doctors will never be able to improve upon their strategies.”

Tragically, Sarah died from complications of the chemotherapy that preceded the BMT. Doctors believe that her heart—while it appeared to be functioning well during tests—may have been predisposed to weakness because of her previous heart problems, her parents say.

Bringing hope to other Sarahs

Today the Moorelands are taking another step to help University physician-researchers improve the EB therapy. They’ve created the Sarah Mooreland EB Fund to provide Wagner and colleague Jakub Tolar, M.D., Ph.D., with funding to continue their innovative work.

“We have spent hours with Dr. Wagner and Dr. Tolar,” says Jay Mooreland. “We truly believe they have a gift from God to see things down to the cellular level, something that separates them from others.”

Thanks to the Moorelands, their friends and relatives, and others around the country, the fund total has now surpassed $100,000.

“We know Sarah didn’t die in vain, but it helps to see how her death can influence others to donate,” says Lonni. “Those donations help the doctors, which ultimately brings hope to other families where there wasn’t any before.”

To make a gift to the Sarah Mooreland EB Fund or to learn about other ways to support EB research at the University of Minnesota, contact Elizabeth Patty at 612-273-8638 or e.patty@mmf.umn.edu.

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