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Children’s Hospital Los Angeles

4650 Sunset Blvd.
Los Angeles, CA 90027
United States

For those who survive brain tumors, neurocognitive deficits are common in the areas of memory, problem-solving and planning. These deficits have been linked not only to poor educational attainment, but also to behavioral and social difficulties, all of which may contribute to poor quality of life. There is some evidence that chemotherapy alone may contribute to cognitive effects in patients, but there is little research in the area of these effects in children treated in this manner for brain tumors.

Background

Neuroblastoma is the second most common solid cancer of children, and less than 50% of children survive who have the widespread (metastatic) form of the disease. The New Approaches to Neuroblastoma Therapy (NANT) consortium is a group of 13 university children’s cancer centers that are dedicated to finding new treatments that will cure more patients (see www.nant.org). The aim of our studies is to develop and test new therapeutic strategies that are developed in our laboratories and that may be effective against the most difficult neuroblastomas.

Neuroblastoma is the second most common solid cancer of children, and less than 50% of children survive who have the widespread (metastatic) form of the disease. The New Approaches to Neuroblastoma Therapy (NANT) consortium is a group of 13 university children’s cancer centers that are dedicated to finding cures for this disease.

Neuroblastoma is a solid malignant tumor of childhood that occurs along the spinal cord and adrenal gland and is often aggressive and difficult to cure. While most high-risk neuroblastoma patients initially respond well to chemotherapy, more than 50% of those patients ultimately die of neuroblastoma that recurs and becomes resistant to currently used chemotherapy.

Background


Primary brain tumors are the second most common type of cancer in children and adolescents and are the major cause of cancer related death in this population. Embryonal tumors, which include medulloblastomas and the other primitive neuroectodermal tumors (PNET), atypical teratoid/rhabdoid tumors (AT/RT) and ependymomas comprise the most common group of malignant brain tumors and are particularly prevalent in patients with these malignancies. This goal is summarized by our mission statement “Saving Lives, Saving Neurons.”

Background


Medulloblastoma is a type of brain tumor seen most often in children. Standard treatment for medulloblastoma includes radiation to the whole brain and spine, as well as surgery and chemotherapy. Unfortunately, radiation to the brain has significant long-term side effects in children. These side effects range from mild learning disabilities to severe mental retardation. The brain-damaging effects are most severe in young children.

Ewing’s Sarcoma Family Tumors (ESFT) are aggressive tumors of bone and soft tissue which are mostly confined to childhood and adolescence. Even with intensified chemotherapy, approximately 40% of those diagnosed with ESFT will die of the disease. Despite intensive study into the molecular-level mechanisms of these tumors, remarkably little of use to patients has resulted. My lab has just published findings that demonstrate that a mechanism common to many adult cancers, also plays an important role in ESFT. This mechanism is called the Hedgehog-Gli pathway.

ALSF-funded team uncovers how childhood leukemia cells become drug-resistant

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