University of California San Francisco

University of California San Francisco

745 Parnassus Ave

San Francisco, CA 94143

United States

Targeting inhibitory phosphatase signaling in high-risk childhood ALL

About 25% of childhood B cell lineage acute lymphoblastic leukemias (ALL) carry oncogenic tyrosine kinases (e.g. JAK2/CRLF2, BCR-ABL and FLT3). Tyrosine kinase driven ALL (TKD-ALL) collectively define the group of patients with the highest risk and a high frequency of drug-resistance and relapse in children.

Blockade of MYCN in neuroblastoma

Neuroblastoma

Neuroblastoma is the third most common pediatric cancer. Amplification of MYCN is a strong independent predictor of poor patient outcome. MYCN represents an attractive target for therapy, as it is serves as a genetic marker for subset of high-risk neuroblastoma and it is expressed at relatively low levels in normal tissues. The ability to inhibit MYCN in patients however, presents a formidable challenge. Many proteins active in cancer (kinases) act by adding a phosphate to a target, thereby altering the activity of the modified target.

Investigation of Genetic and Molecular Mechanisms of Second Malignant Neoplasms in a Mouse Model

General Pediatric Cancer, Hodgkin Lymphoma, Osteosarcoma

Second malignant neoplasms (SMNs) are late complications arising after exposure to chemotherapy and radiotherapy, accounting for most of the ~90,000 new cancers that are diagnosed annually in the United States in persons who previously had a histologically distinct malignancy. Unfortunately, the incidence of SMNs is expected to grow as the at-risk population of cancer survivors increases. This prediction has been borne out with the improved survival of pediatric cancer patients over the last few decades and the increasing numbers of SMNs observed in this population.

Long-Term Morbidity in Survivors of Childhood Cancer with Down Syndrome

General Pediatric Cancer

This project will help to identify late toxicities and long-term follow-up needs specific to childhood cancer survivors with Down Syndrome (DS). Children with DS have a higher risk of certain cancers, particularly childhood leukemias. In recent years modified treatment regimens for leukemia patients with DS have been used because of acute toxicities and differences in overall survival. Nurses at the bedside and nurses involved in the care of survivors rely on information about treatment toxicities to help in the care and education of patients with DS and to guide their follow-up care.

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.

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

Alex's Lemonade Stand Foundation for Childhood Cancer