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131 I-MIBG Imaging and Therapy for Neuroblastoma: Infrastructure Proposal

Neuroblastoma

Neuroblastoma is a highly malignant tumor of children that arises in the peripheral sympathetic nervous system, and spreads widely to bones and bone marrow. The survival of children with metastatic neuroblastoma is less than 40%, despite intensive chemotherapy, radiation and bone marrow transplant. Metaiodobenzylguanidine (MIBG) is a chemical similar in structure to noradrenaline and specifically taken up by neuroblastoma, thus providing a means to detect tumor metastases and to deliver intravenous tumor-targeted radiation by attaching a radioactive iodine molecule.

A Pilot Study of the Feasibility and Acceptability of an Interactive Computerized Symptom Assessment Tool to Assess Disease and Treatment-Related Symptoms Experienced by Children with Cancer

General Pediatric Cancer

The use of Patient Report Outcomes in research and clinical care has increased substantially in recent years. Multiple challenges exist when researchers and clinicians collect symptom data from children. One goal of this research is to determine the feasibility and usefulness of a novel interactive computerized symptom assessment tool (SiSom) to be used in both clinical practice and research. The number and type of symptoms reported by children and their parents using a standardized symptom checklist will be compared to the SiSom system.

Optimizing EGFR targeted therapy in pediatric malignant glioma.

Glioma

Malignant glioma is a particularly aggressive form of brain tumor found in children and adults. Children with brainstem glioma, for example, live an average of ten months. The Epidermal Growth Factor Receptor (EGFR) is an overactive protein in a large number of these tumors and is associated with decreased survival. Drugs that block the function of EGFR have been developed but are only marginally effective in patients.

Identifying novel biomarkers for risk stratification in t-cell acute lymphoblastic leukemia (ALL).

Acute Lymphoblastic Leukemia (ALL), Non-Hodgkin Lymphoma

Cure rates for T-cell Acute Lymphoblastic Leukemia and Lymphoma (T-ALL/LL) have gone from 0% to approximately 80% over the past 4 decades. However, this success comes with a price. The incidence of both acute and long-term toxicities of intensive therapy is becoming increasingly common as more children survive their initial cancers. Moreover, despite intensive therapy, a significant number of children still relapse. Unfortunately, virtually none of these children can be cured with current therapy.

Sheng Zhong, MD/PhD

University of California San Francisco

Application of Epigenetic Markers for Early Detection of Childhood Leukemia.

Leukemia

Childhood leukemia is the most common cancer in children, and a devastating illness to a child and family. Treatments have improved; however, still too many children die, and those that live face a lifetime of reduced health and uncertainty. Our work is focused on epidemiology and prevention of this disease. We ask questions: what are the aberrations that cause leukemia? How does our environment interact with our bodies to produce these aberrations? When do mutations occur, and in knowing this can we develop early detection tests and prevention strategies such as vaccines?

131-I-MIBG and Targeted Therapy for Neuroblastoma: Infrastructure Proposal.

Neuroblastoma

Blockade of  Mycn in neuroblastoma

Neuroblastoma

1. Layman's Title for your Research Project: 

Approaching neuroblastoma therapy by targeting the best characterized genetic abnormality associated with this disease, Mycn.

2.  Research Objectives

What do you hope to achieve through your research and what will it mean to the world of childhood cancer if your project is successful?

Natalia Jura, PhD

University of California San Francisco

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Sawyer Knoblock

Sawyer is a young man with a big personality who always shows so much love for others. When he was diagnosed with germinoma, treatment had to be tailored specifically to him since he also has Down syndrome. Luckily, it worked and today Sawyer is NED!
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