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University of Wisconsin - Madison

Research and Sponsored Programs21 North Park Street Suite 6401
Madison, WI 53715-1218
United States

Diffuse midline glioma (DMG), previously defined as diffuse intrinsic pontine glioma (DIPG), is an aggressive brain cancer that appears in children between 4-9 years of age. Only a few hundred children are diagnosed with DIPG/DMG in the US each year, and it is still poorly understood. There is no cure for DIPG/DMG, and children do not live more than 1-2 years after diagnosis. DIPG/DMG is a formidable type of cancer because there are no good treatment options, and it is universally lethal.

Half of all neuroblastoma patients are diagnosed with high-risk neuroblastoma. Even with the recent addition of immunotherapy, which has been proven to help, only ~ 50% of children with high-risk disease are cured. Laboratory studies have identified three characteristics that make high-risk-neuroblastoma harder to cure using the current clinical regimen of chemotherapy, surgery, radiation and immunotherapy.

Posterior fossa group A (PFA) ependymomas are an especially clinically challenging type of brain tumor that occurs in young children. In order to develop new treatments to combat these tumors, we first need to understand the molecular pathways that drive the cells to grow. Recent work from our group and others found that nearly all PFA ependymoma tumors have abnormally high levels of a protein that we named EZHIP. Normally, the EZHIP gene is only turned on in cells that make up part of the testes and ovaries.

Background

Despite advances in pediatric oncology treatments and technology, some children with cancer may die while receiving oncology treatments or from their disease. It has been reported that many children who die a cancer-related death may die while experiencing two to eight poorly controlled symptoms. Prevention of suffering, including effective symptom management, in children dying of cancer is a central value for clinicians in pediatric oncology.

Background

Neuroblastoma is one of the most common forms of cancer in childhood, and approximately 700 young children are diagnosed with this tumor each year in the United States alone. The disease is very aggressive, and despite various forms of treatment such as chemotherapy, radiation and surgery, more than one third of all patients with advanced-stage disease will eventually die. The patients who survive are stricken with the long-term side effects of highly toxic treatments. Thus, more targeted therapies that kill the cancer but spare healthy tissue are urgently needed.