"It was the challenge of curing patients with life-threatening diseases that appealed to me."
- John E. Wagner, M.D., principal investigator of the umbilical cord blood transplant studies.

"Most recent results with cord blood transplantation have been nothing other than striking. Our results with children have been great — in adults, unprecedented. Our approach has been adopted worldwide and is now referred to as the 'Minnesota Regimen.' "

Dr. John E. Wagner, Scientific Director of Clinical Research of the Blood and Marrow Transplantation Program and Stem Cell Institute, was part of the medical team that made medical history in 1990 by performing the first umbilical cord blood transplant in the world for leukemia, and in 2000 by performing the first umbilical cord blood transplant from a sibling donor "created" after embryo selection. More recent results in adults have made cord blood transplantation increasingly the preferred transplant approach.

Noting the promise of cord blood stem cells to create healthy blood, Wagner and the team successfully treated a 4-year-old boy with a rare blood disease called juvenile chronic myelogenous leukemia in 1990.

Since then, the uses of umbilical cord blood have expanded remarkably. It is now used to treat a variety of childhood blood diseases and cancers. Today more than 6,000 cord blood transplants have been performed around the world. Wagner's own research and the clinical programs at the University of Minnesota continue to expand the applicability of cord blood transplants and improve success rates.

Even so, that's not enough for Wagner. "The results are spectacular, but not everyone is cured, so we're not satisfied. That's why our research program is so broad, deep and aggressive. We're always looking for better treatment tools, better ways to improve survival to the goal of 100 percent."

Recruited by the University of Minnesota Medical School from The Johns Hopkins University School of Medicine in 1991, Wagner has created one of the top umbilical cord blood research programs in the country that has received national and international attention. With multimillion-dollar grants from major government institutions and nonprofit disease societies, Wagner's research now focuses on several areas:

1. Umbilical Cord Blood Transplantation in children and adults with leukemia, lymphoma, and other blood disorders and cancer.

• double unit transplantation
• non-myeloablative preparative therapies
• preimplantation genetic diagnosis to select HLA identical embryo
• co-infusion of T-regulatory cells
• co-infusion of MSC
• intra bone marrow injection

2. Multipotent Adult Stem Cells (MAPC)

• translational development/large scale manufacuture of MAPC
• evaluation of MAPC therapeutic potential in congenital and acquired disorders

3. Fanconi anemia

• novel preparative therapies
• gene therapy — multipotent adult stem cell
• phenotype-genotype correlations (collaboration with Rockefeller University )
• pathophysiology

"My interest is in the treatment of patients with diseases previously considered to be 'incurable'. Through a series of carefully controlled studies, we hope to develop new treatments that will cure these patients," said Wagner. "It's the constant challenge. Although some will not survive at first, the clear message is 'Don't give up.' There's hope when basic research leads to novel ideas to be tested in a clinical trial. This strategy, along with our world class team, is time tested."

Though his research interests continue to branch out, it is all part of a pattern, a pattern begun in undergraduate days when a fascination with human biology overtook him. Born in Maryland, Wagner majored in biology at the University of Delaware before attending medical school in Philadelphia at Jefferson Medical College.

"In the beginning I was primarily interested in oncology and research in leukemia," Wagner says. "Leukemia is a challenging disease, and I wanted to get away from treatment issues related mainly to a patient's age. In children, there's never a question about whether to be aggressive. You are always aggressive."

An internship and residency in pediatrics at Duke University and subsequent fellowship in cancer treatment at Hopkins allowed him to pursue aggressive treatments in leukemia. Exposure to bone marrow transplantation techniques in 1985 at The Johns Hopkins University School of Medicine took this interest in aggressive treatments a step further.

"I had never done anything like this. At the time, few survived transplant. But it also had such an amazing potential for cure! It was the challenge of the treatment — curing patients with life-threatenting diseases — that appealed to me." Later faculty appointments in pediatrics and oncology at Johns Hopkins University School of Medicine furthered both clinical and research interests.

Working with cord blood is a natural extension of Wagner's work with bone marrow — to find ways to reduce the risk of graft-versus-host-disease (GVHD). Since his pioneering involvement with the first umbilical cord blood transplants more than 15 years ago, Wagner and his team at the University of Minnesota continue to lead the field.

For example, umbilical cord blood transplant survival rates continue to improve. New services have been initiated at the University of Minnesota to make the transplants faster and easier. In adults, survival has tripled just since 2000. Our results are the best reported in the U.S. and Europe.

"Ultimately, what we'd like to see," Wagner said, "is that cord blood can be a donor source of stem cells for everyone regardless of age, size, or ethnic and racial background. To do that, we need to figure out the mechanism, to figure out what makes it different from bone marrow, so we can make it both safer and more available."