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Lifesaving cells: For 40 years‚ the U of Minnesota has led the way in treating childhood diseases with blood and marrow transplantation

The 4-month-old baby had no other hope. Nearly a dozen relatives had already died from the inherited disorder that prevented his body from making the blood cells needed to fight infections.

Four-year-old Alana Barner received a blood and marrow transplant from a University team including Jakub Tolar, M.D., Ph.D., that reversed her disease.

His doctor‚ Robert Good‚ M.D.‚ Ph.D.‚ had hope. After years of research‚ the University of Minnesota immunologist was ready to take the big step. In August 1968‚ Good made history by curing his tiny patient with the world’s first successful transplant of bone marrow-derived stem cells from a matched donor.

Since then‚ Good’s successors at the University have continued to lead the way in blood and marrow transplantation (BMT). In 1975‚ John Kersey, M.D., headed the team that performed the first BMT to treat lymphoma. In the 1980s‚ University physicians used a patient’s own marrow to treat chronic myelogenous leukemia. In ensuing decades‚ others pioneered the use of umbilical cord blood as a source of cells for transplant and the use of BMT to treat inherited diseases such as Tay-Sachs and osteopetrosis.

John Kersey, M.D., performed the first blood and marrow transplant for treating lymphoma in 1975.

And in just the last few months‚ professor and director of the Division of Pediatric Hematology-Oncology and Blood and Marrow Transplantation John Wagner‚ M.D.‚ and colleagues opened the doors to a new era when they used stem cells to repair a deadly skin condition.

“The University has made tremendous advances over the years in the field of blood and marrow transplantation‚” Wagner says. “Yet this is just the tip of the iceberg in terms of what we think this therapy can achieve.”

Finding a hot spot for treatment

A 40-year track record of famous “firsts” is nothing to shrug at‚ for sure. But when Patti Cuddihee and Bart Barner of Ashland‚ Missouri‚ think about BMT at the University of Minnesota‚ one image comes to mind: the bright-eyed 4-year-old who pads into their kitchen every morning.

Most blood and marrow transplants done here are performed to treat cancer. But in the 1980s‚ University physicians began using BMT to treat inherited metabolic and storage diseases‚ in which the body is unable to break down certain chemicals‚ leading to a disabling or deadly accumulation of toxic compounds.

In January 2005‚ the University’s national reputation as a leader in applying BMT to such diseases brought Cuddihee‚ Barner‚ and their daughter‚ Alana‚ north through ice and snow to Minnesota.

Just days before her first birthday‚ Alana had been diagnosed with Hurler syndrome‚ in which the body doesn’t break down a molecule that‚ as it accumulates‚ damages the brain and other organs. A feverish search of the Internet and medical literature—Hurler syndrome is progressive‚ so time is of the essence—turned up the University of Minnesota as a hot spot for treating the disorder with BMT.

In April of that year‚ Alana received a transfusion of umbilical cord stem cells under the direction of BMT physicians Paul Orchard, M.D., and Jakub Tolar‚ M.D.‚ Ph.D.

“She’s doing stupendously‚” Cuddihee says.Without treatment‚ Alana would be in a downward spiral of organ failure and neurological damage. Instead‚ she’s learning her colors and ABCs and helping Patti in her veterinary clinic.

“We’re beyond thrilled—we’re absolutely ecstatic‚” Cuddihee says. “We feel like the University of Minnesota played a huge‚ huge role in that.”

‘Hinge in history’

Today about 75 children each year receive a BMT at the University in hopes of curing cancer‚ bone marrow failure syndrome‚ immune deficiency‚ or an inherited metabolic storage disease.

Last fall‚ New Jersey toddler Nate Liao received a highly publicized transplant at the University for a revolutionary reason—to cure a life-threatening skin disease called epidermolysis bullosa. EB is a rare genetic disorder in which skin fails to produce a type of collagen that holds the layers of skin together. This means that the skin can slough off at the slightest touch and that a cough can cause tears in the lining of the esophagus and stomach.

Nate Liao continues to improve after his transplant

Alerted to the boy’s condition by his mother‚Tolar and professor of pediatrics Bruce Blazar, M.D., began laboratory studies to determine whether stem cells could supply the missing collagen. Armed with the lab team’s encouraging results‚ Wagner designed a transplant he hoped would meet Nate’s needs.

And it did. In June Wagner made international news when he announced that the transplanted stem cells were producing the missing collagen‚ and Nate’s skin was starting to mend.

“Our work on EB serves as a perfect example of translational bench-to-bedside research and what a team of dedicated researchers‚ physicians‚ and nurses can accomplish‚” Wagner says. “Stem cell therapies offer hope to so many patients with life-threatening disease. It is my belief that we are at a hinge in history when the practice of medicine will undergo a revolutionary transformation. And it’s a privilege to be part of it.”

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