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Translational Research

Kids playing with a wagon


Osteosarcoma, the most common malignant bone tumor in children, is an aggressive malignancy. Although advances in chemotherapy have improved survival, patients with metastatic disease have only a 20% chance of surviving their disease; whereas patients without metastatic disease have about a 65% chance of surviving their disease. To date, the responsiveness of the primary tumor to presurgical chemotherapy is the most powerful predictor of outcome in both metastatic and non-metastatic osteosarcoma. There is a clinical need to identify as early as possible if a patient will fail standard therapy and would benefit from more intensive or alternative treatments or upfront surgery. Several biomarkers have been proposed to predict response of osteosarcoma. Despite all of these efforts, reliable markers that predict therapeutic response to chemotherapy have not yet been found.

To identify these biomarkers we are using serum samples from the Children's Oncology Group Osteosarcoma Biology Protocol P9754. In addition, clinical trials are in progress at Riley Hospital for Children that will allow us to follow these biomarkers through the treatment phase and for several months afterwards. We are collecting samples from osteosarcoma patients at multiple time points during and after treatment, as well as samples of healthy children which are used as controls.


Acute lymphoblastic leukemia (ALL) is the most commonly diagnosed cancer in children. Overall cure rates for children with ALL currently approach 80%. It is well recognized that ALL is a heterogeneous disease comprised of many different subtypes. Our goals are to identify subsets of patients at higher risk of relapse through proteomics and pharmacogenomics studies. In addition, we are optimizing information visualization tools to link high throughput datasets with clinical and laboratory data. This effort will greatly enhance data analysis by clinical experts in leukemia.

To identify these subsets of patients, a clinical trial at Riley Hospital for Children is being conducted, collecting a small amount of extra blood and bone marrow from each study participant at the time of diagnosis or relapse when bone marrow and blood examination are routinely done.

Wilms Tumor

Wilms tumor is the most common kidney cancer in children. Concerted efforts have dramatically improved the prognosis of most patients. The current focus is to reduce the morbidity of treatment for low risk patients and reserve more intensive treatments for high risk patients. Therefore, a major research focus is to find biomarkers that could be used to predict tumor behavior. To identify these biomarkers, we are using microarray data and serum samples from the national Children's Oncology Group's most recent National Wilms Tumor Study. In addition, a clinical trial at Riley Hospital for Children is being conducted, collecting samples from Wilms' patients as well as from healthy children, which are used as controls.

Heart Disease

Heart disease is the leading cause of death in the world. Available screening and diagnostic methods are often insufficient to determine the extent of heart disease. In addition, there are no prognostic markers for congestive heart failure that would aid in the management of patients. In the past, single biomarkers have been used to assess disease risk. However, in complex diseases, multiple biomarkers representing different pathophysiological processes will provide a much more complete picture. A proteomic and metabolomic approach enables the simultaneous measurement of a large number of proteins and metabolites to find the optimal biomarker combination for risk stratification of patients with coronary artery disease. This approach has been applied to the global protein profiling of 250 patients that underwent coronary angiography at the German Heart Center in Munich. Currently the Center is analyzing the metabolites in the serum of these patients.

Eye Diseases

Glaucoma and cataracts are the leading causes of blindness worldwide and have a major impact on quality of life. The scientific understanding of these and other eye diseases is severely limited by the lack of pathology specimens for study. Without tissue samples, the biochemical and signaling pathways involved in these diseases remain poorly characterized. Without good knowledge of how these diseases occur, progress in developing better treatments to prevent blindness is hindered. In lieu of tissue specimens, biofluids from eyes of patients are an important source of valuable information about the processes that occur within the eye. Because the fluids are in direct contact with the major structures with the eye, proteins and other metabolic products spill into the fluids and thus offer important clues about what happens in the eye. Hence, the discovery of biomarkers within ocular fluids would greatly enhance the understanding of eye diseases. We are prospectively collecting aqueous and vitreous humor from patients with eye diseases such as glaucoma and cataract.

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