University of Minnesota Pediatric Bone Marrow Transplant Program

Our Pediatric Hematology/Oncology/BMT fellows have the amazing opportunity to learn from world leaders in the field of Bone Marrow Transplant.  During their training here they will rotate both on the inpatient BMT floor and the outpatient BMT clinic, where they will not only be exposed to a wide variety of diseases but also multiple conditioning regimens and bone marrow sources including double and triple umbilical cord blood transplants. 

Our program is the oldest and one of the largest and most respected pediatric blood and marrow transplant (BMT) research programs in the world.  Children and adolescents come from around the world to be treated at the University of Minnesota.  Caring, dedicated University of Minnesota doctors and nurses, pharmacists, social workers, child life and Care Partners are focused on delivering of state of the art care to patients undergoing BMT. 

Driven to finding new treatments and safer transplant therapies for children with life threatening malignant and non malignant diseases, University of Minnesota investigators have received millions of dollars in NIH and industry support to manipulate the immune system to kill cancer cells and harness the power of stem cells to repair diseased or chemotherapy and radiation damaged tissues.   Treatments are available for leukemia, lymphoma, neuroblastoma and other solid tumors, aplastic anemia, Fanconi anemia, sickle cell disease, thalassemia, metabolic syndromes and immunodeficiency states.

The world’s first successful bone marrow transplant was performed in 1968 at the University of Minnesota in a child with severe combined immunodeficiency syndrome. Since that time, the Blood and Marrow Program at the University of Minnesota has been a leader in the field of blood and marrow transplantation for children with primary immunodeficiencies, such as Wiskott-Aldrich syndrome and SCID syndrome, and histiocytic disorders, such as Hemophagocytic Lymphohistiocytosis (HLH) and Langerhans’ Cell Histiocytosis (LCH).

The Immune Deficiency and Histiocytic Disorders Program continues to explore innovative strategies in transplantation in order to make them safer, with higher success rates, and more widely available.

The principal focus of our current research efforts include:

• Use of umbilical cord blood stem cells form unrelated donors to expand availability of BMT for all patients with immune deficiencies, HLH and LCH
• New methods of T cell depletion of related and unrelated donor stem cells in order to reduce the risk of transplant therapy
• Novel--reduced intensity--preparative regimens designed to reduce risks and complications in children
• Gene therapy clinical trial for treatment of immune deficiency

The first bone marrow transplant for osteopetrosis was performed at the University of Minnesota.  Currently Dr. Paul Orchard is leading the research and treatment investigations at the University of Minnesota.  Dr. Orchard has an interest in the use of transplantation for osteopetrosis, as well as the biology and genetics of this disorder.  As the primary organizer of the “International Symposium on Osteopetrosis: Biology and Therapy” at the National Institutes of Health on October 24, 2003, Dr. Orchard brought researchers together in the first meeting of its kind to discuss what is currently known regarding this disorder and what has yet to be done to improve overall chances of long term survival.

At the University of Minnesota, Dr. Orchard and his team developed transplant protocols using intense and less toxic therapy to improve overall outcomes.   In addition, he developed a research protocol to investigate the function of osteoclasts from individuals with osteopetrosis with collaborators in Pittsburgh and in St. Louis.  Dr. Orchard is also a world expert in the genetics of this disorder.

Currently at the University of Minnesota the ability to test for the two most common genes causing osteopetrosis is in development, and should be available for patient testing in early 2007.  This testing will allow evaluation of other family members to determine carrier status, and will be invaluable as a research tool to determine the implications of genetic findings in the prognosis and treatment of these patients.

The Fanconi Anemia Comprehensive Care Program at the University of Minnesota Medical Center is the single largest treatment center for patients with Fanconi Anemia (FA) in the United States.  The care provided is individually tailored to meet the unique needs of each patient and family.  This program uses a team of expert health care providers who specialize in FA, applying leading-edge research developed by University of Minnesota scientists.  The University of Minnesota has set the standard for BMT as a treatment for Fanconi Anemia and other diseases since we performed the world's first successful BMT in 1968.  University of Minnesota physicians have performed sibling donor transplants for FA since 1976 and unrelated donor transplants for FA since 1987. 

In 2000, we broke ground by performing the world's first umbilical cord blood transplant on a Fanconi Anemia patient using pre-implantation genetic testing to ensure a perfect HLA tissue match.  As a result of this work, Fanconi Anemia patients have been referred at diagnosis or shortly thereafter to assist in the care of FA patients well in advance of BMT.

In total, our fellows spend half of their first year divided among BMT inpatient and outpatient rotations with the remaining 6 months rotating through HemOnc inpatient and outpatient settings which include Blood Banking/Coagulation, Hematopathology, Radiation Oncology, and Neuro-Oncology.

University of Minnesota Blood and Marrow Transplantation Program Milestones

1968: World’s first successful human bone marrow transplant, performed by University of Minnesota Physicians in an infant with an immune deficiency syndrome.

1975: World’s first successful transplant in a patient with lymphoma.

1982: First transplant for an inherited metabolic disease.

1980s: Development of autologous marrow transplantation (using the patient’s own marrow) for chronic myelogenous leukemia

1991: First cord blood transplant performed with a related donor for a child with leukemia.

1994: Unrelated cord blood transplant program established

2000: The first umbilical cord blood transplant is performed on a Fanconi Anemia patient using pre-implantation genetic testing to ensure the donor’s umbilical cord blood would provide a perfect tissue match

2000: First successful double-cord blood transplant

2004: Initial publication of experience with alternative donor, reduced intensity cord blood grafts for adult recipients who are too advanced in age or whose condition does not allow conventional allogeneic transplantation

2004: Successful early experience of leukemia reinduction using immunoablative therapy along with infusions of haploidentical donor natural killer cells

2007: The 5,000th BMT is performed

Other Program Highlights

• The University of Minnesota BMT program is one of the largest unrelated donor transplant programs in nation and is one of the largest umbilical cord blood transplant centers for adults worldwide. 

• The University of Minnesota has treated more Fanconi Anemia and metabolic disease patients than any other center in North America. 

• The University of Minnesota leads the field in innovative research and exceptional patient care along with leading the nation in long term patient follow-up and research into late medical effects of BMT.