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Pediatric Hematology/Oncology in Minnesota

Research

Hematology

Hemophilia

Mayo Clinic investigators are studying the effectiveness of a medical product called rVIIA in preventing bleeding during major elective surgery in patients with hemophilia and related disorders. Mayo Clinic is one of four centers collaborating on this project. The therapy, rVIIa, is not derived from human blood or plasma, making it safer than traditional alternatives. Preliminary studies at Mayo Clinic indicate that it is highly effective and has the potential for widespread use in controlling bleeding during surgery.

Oncology

Genetic Instability and Cancer Development

The role of recurrent chromosomal translocations and chromosome number instabilities in cancer are currently being investigated. The chromosomal translocation research is focused on the molecular analysis of fusion genes with specific breakpoints in FG repeat-containing nuclear pore complex (NPC) genes that seem to be essential for leukemia development. The chromosome number instability research explores the role of mutations in mitotic checkpoint genes and how that affects the development of a variety of tumors.

Signal Transduction

Mayo's research interests focus on aspects of signal transduction (intracellular events that mediate internal responses to extracellular conditions) that participate in cellular transformation in cancer and on antibody responses of the immune system. We have identified a set of genes that encode novel signaling proteins. Current efforts include establishing the physiologic roles for these genes, and identifying their contributions to malignant transformation in childhood leukemias and solid tumors. The ultimate purpose of this work is to accelerate the development of improved treatments for metastatic cancer and humoral immunodeficiencies.

Children's Oncology Group (COG)

Mayo Clinic performs research and provides treatment as a member of the Children's Oncology Group (COG), a national children's cancer research group. Research focuses on therapeutic, epidemiological and biological studies for pediatric patients with cancers. During the last half of the 20th century, childhood cancer went from an almost universally fatal disease to one that is curable in most patients. The increasing survival of children with cancer has resulted in the recognition of the need to reduce long-term side effects of cancer and improve quality of life. Recently, the major pediatric clinical trials groups based in North America — the Children's Cancer Group, the Pediatric Oncology Group, the Intergroup Rhabdomyosarcoma Study Group, and the National Wilms' Tumor Study Group — combined efforts as part of the Children's Oncology Group, to accelerate the goal of curing and eventually preventing cancer in children and adolescents. The group will continue to perform clinical and research trials to define optimal treatment for children and adolescents with cancer. This research will bring about more effective treatments with reduced short- and long-term side effects. It will identify causes of childhood cancer, develop strategies for prevention, and improve the quality of life for children and adolescents with cancer. The work of the Children's Oncology Group will foster partnerships on behalf of children and adolescents throughout the world.

Rhabdomyosarcoma Research

As part of the Children's Oncology Group, Mayo Clinic Division of Pediatric Hematology and Oncology studies children affected by rhabdomyosarcoma (cancer of the skeletal muscle). This cancer is more common in children than adults. Largely due to the work of COG and others, the recovery rate for this disease is 80 percent, compared to 25 percent in 1972.

Medulloblastoma

Mayo is researching the molecular biology and genetics of the pediatric brain tumor medulloblastoma. Investigators are trying to determine what genetic events result in the transformed phenotype of the tumor cells and what mechanisms are caused by these events. The investigators are especially interested in identifying development pathways in the tumor. Currently Mayo Clinic researchers have identified alterations in two pathways. In one, a tumor suppressor gene is homozygously inactivated. In the other, an oncogenic mutation occurs in one gene. These two mutations account for about 15 percent of medulloblastomas. The research group is looking for additional genes that might be important for development and growth of this malignant brain tumor, hoping that a better understanding of the molecular basis for the tumor will lead to more effective therapies.

Developmental Therapeutics

As part of the Children's Oncology Group, Mayo Clinic is studying new chemotherapeutic and biologic agents to treat cancer in children.

Stem Cell Transplantation

New methods for stem cell selection (CD34 selection) are being used. The Miltanyi Clinimacs device provides CD34 selection and very little contamination of the graft with T cells. This device is being studied at Mayo Clinic as a means to provide haploidentical or other transplants to selected patients.

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