Your Donations at Work
See how your contributions are making a difference in the fight against childhood cancer. Accomplishments Achieved in 2007 & 2008.
With a long-standing history as leaders in pediatric cancer researcy, University of Minnesota investigators continue to make major discoveries impacting the future of children and adults with life-threatening illnesses. Support from Children's Cancer Research Fund empowers these scientists to aggressively meet milestones on the way toward the development of innovative research and treatments that can potentially prevent and cure childhood cancer, as well as improve the quality of life of a rapidly growing number of cancer survivors.
New Cures & Treatment Options
Conquer Childhood Cancer Act
The Conquer Childhood Cancer Act was signed into law which authorizes $30 million annually over five years for collaborative pediatric cancer clinical trials research, to create a nearly population-based national childhood cancer database, and to further improve public awareness and communication regarding available treatment and research for children with cancer and their families. Notably, Dr. Julie Ross, Children's Cancer Research Fund Chief Medical Advisor, leads the effort to create the childhood cancer research database nationally, the concept of which was partially supported by Children's Cancer Research Fund.
First Bone Marrow Transplant to Treat Recessive Dystrophic Epidermolysis Bullosa
Physicians used transplant as a means to rid the body of the defective blood system and replace it with a healthy blood system that produces type VII collagen, the protein people with RDEB lack. Results to date show success and the patient’s sibling, who is also affected by this disease, has now also undergone transplant.
Treatment of Hurler Syndrome
Drs. Paul Orchard and Jakub Tolar launched the first clinical trial world-wide to treat patients with Hurler Syndrome by combining BMT with injecting a manufactured enzyme, deficient in Hurler Syndrome patients, into the patient’s spinal fluid. This trial promises to deliver the missing enzyme quickly to the patient’s brain during the time it takes for the transplanted stem cells to engraft, decreasing the loss of cognitive function in the patient and improving transplant outcomes.
First to use Total Marrow Irradiation with Double Umbilical Cord Blood Transplant
The first two patients to receive total marrow irradiation with a double umbilical cord transplant (2 cord blood units combined) for refractory acute lymphoblastic leukemia (ALL) were successfully completed with engraftment achieved. These patients would before, have no option outside of palliative/hospice as refractory ALL patients would not be accepted for transplant.
Brain Tumor Program Established
The goals of the program are to:
- Enhance a translational research program that will produce novel therapies to treat brain and spinal cord tumors, retinoblastoma, and neurofibromatosis-related tumors.
- Assemble a team of laboratory scientists, physicians and advanced practice nurses who will provide research-based, multi-disciplinary, comprehensive care to patients.
- Develop neuro-oncology education programs for medical students, residents, and fellows as well as continuing education programs for practicing health care providers.
Development of Clinical Trial Utilizing Vaccine Approach for Brain Tumor Treatment
A clinical trial utilizing a vaccine approach to treating brain tumors, specifically gliomas, has been developed investigators in the Brain Tumor Program and has been submitted to the FDA for approval. Enrollment of the first patients is planned for early 2009.
Clinical Trial Opens for Patients Battling Recurring Leukemia
Researchers at the University opened a new clinical trial for patients with relapsed/refractory ALL
(ages >12yrs). This trial utilizes radioactive CD19 antibody (CD19 is a protein expressed on the surface of leukemia cells and very early stage white blood cells) developed at the University of Minnesota to deliver a targeted radioimmunotherapy to treat these refractory leukemia’s.
Minimizing Side-Effects of Treatments
Progress in Prevention of Graft vs. Host Disease
The first clinical trial worldwide opened this year at the University of Minnesota to test if regulatory T cells, isolated and grown from adult peripheral blood, infused at the time of BMT can prevent graft vs. host disease (GVHD), a complication of BMT where the transplanted cells attack the patient’s tissues which can often be fatal. Regulatory T cells are a specialized subset of white blood cells that act to suppress activation of the immune system. This first trial will establish safety and the optimal dose of cells to give, however, if this trial is ultimately successful, we may have a new way of preventing GVHD with cellular therapy thereby avoiding the need for intense drug therapy with immunosuppressive agents.
Using the Body's Immune System to Prevent Post-Treatment Complications
A method for converting non-regulatory T cells into regulatory suppressor T cells (Treg suppressor cells) was designed and patented by University of Minnesota investigators. Treg suppressor cells are potent modulators of the immune system, capable of preventing immune complications common after bone marrow transplantation. However the potential of this discovery has implications in other settings such as treatment of autoimmune disorders (e.g. diabetes) or in solid organ transplant.
Development of Brain Sparing Protocol for Inherited Degenerative Neurological Diseases
Analysis of data from Drs. Paul Orchard and Jakub Tolar’s brain sparing BMT protocol using novel antioxidant therapy, demonstrated that this approach appears to minimize disease progression in high risk patients transplanted for inherited degenerative neurological diseases thereby improving transplant outcomes.
Investigating Causes of Childhood Cancer
What is Causing Osteosarcoma?
Dr. Logan Spector began recruitment for the NIH-funded study "Genetic Epidemiology of Osteosarcoma". This will be the largest study of genetic risk factors for bone cancer in adolescents to date and also the first study to recruit patients through the Childhood Cancer Research Network.
Evaluating Birth Factors and the Risk of Childhood Cancer
A consortium to pool childhood cancer datasets from the states of California, Minnesota, New York, Texas and Washington was established by Dr. Logan Spector. The combined dataset includes over 17,000 cases of childhood cancer and 55,000 controls, making it the largest study of its kind. The study will examine in greater detail than in previous studies the role of birth factors in the risk for childhood cancer.
Understanding Risk Factors for Wilm's Tumor, Neuroblastoma and Rhabdomyosarcoma
Dr Simona Ognjanovic is leading the pilot study of embryonal tumors (ISET study) in two European countries (Serbia and Slovenia). This is a part of the multi-center worldwide study of embryonal tumors initiated by the International Agency for Research on Cancer (IARC). Pediatric cases of Wilms tumor, neuroblastoma and rhabdomyosarcoma are continuing to be recruited to elucidate genetic determinants of these rare childhood cancers. The results of the pilot study will be used as basis for a large 5-year international study of embryonal tumors to improve our understanding of genetic and environmental factors associated with risk for these types of childhood cancer.
Causes of Childhood Cancer - Neonatal Blood Spot Study
The Riverside Birth Study, which will enroll and gather biologic samples from 500 pregnant women and from their babies at birth, was initiated by Drs. Simona Ognjanovic and Logan Spector. This study will give great insight into the measurement of biomarkers in neonatal blood spots, which are stored by a number of states and are being incorporated into studies of the causes of childhood cancer.