Clinical Programs

The University of Minnesota Division of Pediatric Hematology/Oncology and Bone Marrow Transplantation has several clinical programs which our fellows gain experience from. The Bone Marrow Transplant, Fanconi Anemia and Leukemia Programs each offer unique opportunities for our fellows to gain expertise in these areas. Each of these programs are listed below in more detail.

BMT 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.

Fanconi Anemia Program

The Fanconi Anemia Comprehensive Care Program at the University of Minnesota 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 bone marrow transplant (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.

We have treated more FA patients with BMT than any other center in North America.   In 2000, we broke ground by performing the world’s first cord blood transplant on a Fanconi Anemia patient using pre-implantation genetic testing to ensure a perfect 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.  Our patients are treated by a team of experts specializing in gastroenterology, nephrology, cardiology, orthopedic surgery, endocrinology, infectious diseases, nutrition, neurology, general surgery, otolaryngology and hand surgery.

Dr. John E. Wagner, Professor, University of Minnesota Medical School, and Scientific Director of Clinical Research, Blood and Marrow Transplant Program and Stem Cell Institute, and Dr. Margaret MacMillan, Assistant Professor, University of Minnesota Medical School, are world-renowned experts in the field of BMT and Fanconi Anemia, in particular.  Their research at the University of Minnesota’s Stem Cell Institute involves exploring novel ways to increase cure rates and improve quality of life for patients with FA.

This expertise in FA translates into a unique opportunity for our Pediatric Hematology/Oncology/BMT fellows to learn about a disease that was once uniformally fatal and now has a greater than 80% cure rate.  Our fellows spend time in the FA clinic during their BMT outpatient months in their first year.

Leukemia Program

The Pediatric Leukemia Program here at the University of Minnesota Amplatz Children’s Hospital began in the summer of 2006 and has become an integral part of the Divisions of Pediatric Hematology/Oncology and Blood and Marrow Transplantation. Although the cure rates for childhood leukemia continue to rise, over 500 children will relapse each year with ALL, AML, chronic leukemias or lymphoma and are in desperate need for further therapy. One of our Program missions is to develop new therapies or novel treatment combinations to cure these children of their relapsed disease. At the University of Minnesota Amplatz Children’s Hospital our patients have the opportunity to enroll onto novel and cutting edge treatment protocols that are available through the Children’s Oncology Group (COG), Treatment Advances in Childhood Leukemia and Lymphoma Consortium (TACL) or Phase I/II studies that were developed here and available only at the University of Minnesota.

Our Leukemia Program is a comprehensive program designed to deliver state of the art and novel treatment strategies for high-risk or relapsed patients with leukemia or lymphoma. It is comprised of members from both Pediatric Hematology/Oncology and Pediatric Blood and Marrow Transplantation. The group meets on a weekly basis to evaluate all new patients diagnosed with leukemia or lymphoma here at Amplatz, along with any relapsed patients, referrals for BMT and/or second opinions of difficult cases that may be occurring at centers across the country or abroad. The program has developed treatment algorithms to use as a guideline for the treatment of newly diagnosed and relapsed patients. These algorithms incorporate standard chemotherapy approaches, phase I and II investigational trials, and other novel therapies and protocols that are being developed and investigated here at the University of Minnesota. Each week the Program typically reviews 4-6 new patients and is updated on the patients who are currently being treated at the University.

University of Minnesota Leukemia Program Members:

Oncology

  • Michael Burke, M.D.
  • Brenda Weigel, M.D.
  • Emily Greengard, M.D.
  • Lexi Maciej, P.N.P.
  • Melissa Claar, P.N.P.
  • Program Nurse Coordinator: Barb Trotz, R.N.

BMT

  • Michael Verneris, M.D.
  • Heather Stefanski, M.D., Ph.D.
  • Angela Smith, M.D.
  • Margy MacMillan, M.D.

Neuro-oncology/Neural Tumors Program

Central nervous system tumors constitute the second most common pediatric cancer diagnosed in the United States each year. The care of patients with these neoplasms requires specific knowledge and expertise. Each patient benefits from the attention of a multidisciplinary team, the hallmark of excellent pediatric neuro-oncologic care. We are proud of our group of dedicated providers, including pediatric neuro-surgeons, geneticists, and neuropsychologists.

The Pediatric neuro-oncology program, led by Dr. Christopher Moertel, encompasses care for children and young adults with brain and spinal cord tumors, peripheral nerve tumors, and retinoblastoma.

Dr. Moertel is also the director of the comprehensive neurofibromatosis clinic, which provides care for those affected by neurofibromatosis-related neoplasia, including optic pathway tumors, plexiform neurofibromas and malignant peripheral nerve sheath tumors.

The neuro-oncology program benefits from its close relationship with Dr. Brenda Weigel and the University’s designation as a COG phase I institution. The neuro-oncology team also works closely with the bone marrow transplant program as we develop new therapies and explore high dose chemotherapy options for our patients.

Understanding the late effects of brain tumor therapy is key to our patients’ long term success and Dr. Neglia is an international leader in studying the late effects of childhood cancer.

The neuro-oncology translational research program is lead by David Largaespada, PhD.  This program includes several labs who are exploring the science of brain tumor stem celle, neuro-immunology, new agent development and neurofibromatosis-related neoplasia.  Research opportunities are available in these labs.

The University of Minnesota is the home of the Center for Magnetic Resonance Research, a participating member of the MIND Institute Consortium along with Harvard and the National Institutes of Health, and the home of some humongous magnets that reveal never-before-seen details of the human brain and its function.

Neuro-oncology clinics are held twice weekly and pediatric neuro-oncology care conferences are held on a monthly basis. These conferences include representatives of neurosurgery, neuro-radiology, neuro-pathology, neuro-oncology, endocrinology, radiation therapy and neurology, among others.

All pediatric hematology/oncology fellows will do a neuro-oncology rotation during their first year. This rotation will provide focused experience with clinical brain tumor management, neuro-radiology, therapeutic radiology, and neuropathology. Each fellow will have the opportunity to lead a neuro-oncology care conference during this rotation and will have opportunities for research laboratory exposure.

The neuro-oncology team includes the following members:

  • Christopher L. Moertel, MD - Clinical Director
  • Joseph P. Neglia, MD, MPH
  • Tambra Dahleimer, RN, CNP
  • Jane Torkelson, RN