Research

In 2005, Michelle was diagnosed with aplastic anemia. Read more...

Research into causes, characteristics, treatments, responses, side effects and long-term outcomes is performed every day to enhance the ongoing health and quality of life of children.

Translational research is focused on using outcomes data to provide the most advanced medical technology and updated clinical protocols.

The collaboration between organizations such as Children's Research Institute, the Medical College of Wisconsin, Blood Research Institute and Midwest Athletes Against Childhood Cancer Fund ensures that the best resources come together to provide the best outcomes. Some of the current research highlights include:

• Researchers Mary Jo Kupst, PhD, and Kristin Bingen, PhD, are involved in a long-term follow-up study investigating the neurocognitive and psychosocial functioning of blood and marrow transplant patients from pretransplant to five years posttransplant. Children's Hospital of Wisconsin is one of three centers that have followed patients pre to posttransplant. This research will help physicians and families understand how a transplant affects a child's cognitive, academic and psychosocial functioning throughout his or her life. 
• Cheryl Hillery, MD, Nancy Wandersee, PhD, Kirkwood Pritchard, PhD, and Neil Hogg, PhD, are involved in nationally funded research programs to study the role of inflammation, clotting pathways and red cell destruction in blood vessel problems and organ damage found in sickle cell disease. This research will provide a better understanding of the mechanisms leading to complications of sickle cell disease, as well as providing the testing of potential novel therapies to prevent complications.
• Julie Panepinto, MD, MSPH, is leading the way in health-related quality of life evaluations for children with sickle cell disease. Accounting for environmental, family and socioeconomic factors, this research will evaluate quality of life issues in relation to treatments and medications.
• The Wisconsin Sickle Cell Center has been involved in a multicenter trial studying stem cell transplantation for sickle cell disease using matched sibling donors and unrelated cord and stem cell transplants. 
• To combat inherited bleeding disorders, researchers are developing gene therapy. The therapy, being investigated by David Wilcox, PhD, and Robert Montgomery, MD, involves taking out a patient's marrow, replacing the bad gene with a good gene and then giving the same marrow back to the patient. In this National Institutes of Health-funded study, researchers hope to develop replacement genes in blood-related diseases such as hereditary platelet disorders, hemophilia A and B and von Willebrand disease. Dr. Montgomery also is leading an international, multicenter, translational study of von Willebrand, investigating the relationship between genetic defects in the von Willebrand genes and the clinical diagnosis and management of von Willebrand.
• An interdisciplinary and multi-institutional study is looking at the increased incidence of hemangiomas in relation to the increase in low birth weight rates in the U.S. The study, under the direction of Beth Drolet, MD, and Michael Kelly, MD, PhD, is studying infants diagnosed with large, complicated hemangiomas to determine and compare the effectiveness and safety of steroids, the current standard of care, versus other drugs. Other research sites include the University of California-San Francisco and Indiana University.
• Neuroblastoma is the most common tumor found in infants and accounts for 15 percent of all childhood cancer deaths. Researchers Rimas Orentas, PhD, and Bryon Johnson, PhD, are developing a vaccine for neuroblastoma by genetically modifying cancer cells. The vaccine activates the immune system to combat cancer. The goal of this immunotherapeutic approach is to increase the cure rate for children by integrating vaccines with other methods of treatment.
• David Margolis, MD, and William Drobyski, MD, helped pioneer the use of donor leukocyte infusions as a treatment for leukemia relapse after a blood and marrow transplant. Based on this initial work, Dr. Drobyski is investigating a novel approach using gene therapy to control the immune reactivity of T-lymphocytes given for donor leukocyte infusion therapy.
• Robert Truitt, PhD, along with Jeffrey Woodliff, PhD, Carolyn Taylor, PhD, and Drs. Johnson, Drobyski and Margolis, are studying human regulatory T-cells in blood and marrow transplant patients and their donors. They are exploring ways to grow regulatory T-cells outside the body so they might be given back to patients to prevent graft-versus-host disease.
• Julie An Marie Talano, MD, and Drs. Margolis and Taylor are developing donor derived T-cells with the goal of preventing and treating severe infectious complications due to cytomegalovirus, Epstein-Barr virus and adenovirus.
• Researchers William Grossman, MD, PhD, and Jack Gorski, PhD, are investigating how T-cells impact the ability to control immune responses to infections like influenza and control graft-versus-host disease. Dr. Grossman and James Verbsky, MD, PhD, also were the first to describe a novel mechanism used by these T-cells to control immune cell responses. 
• Dr. Verbsky has described a new cause for IPEX syndrome due to mutations in the gene CD25. IPEX syndrome is a rare genetic disease of the immune system that is fatal in the first year of life if not treated. He is studying how this gene contributes to autoimmune diseases.
• Research by Jack Routes, MD, looks at viruses that contribute to cancer development. He is using models to study how these viruses contribute to that development. Dr. Routes also is actively studying viruses in patients with common variable immunodeficiency and how these viruses can lead to complications of this disease.