University of California San Francisco

University of California San Francisco

745 Parnassus Ave

San Francisco, CA 94143

United States

Genetic Predisposition to Atypical Teratoid/Rhabdoid Tumor Caused by Mutations in SWI/SNF Complex Genes

Atypical Teratoid/Rhabdoid Tumor (AT/RT)

Background

Optimizing Conformation Disrupting Aurora Kinase Inhibitors to Treat MYCN Amplified Neuroblastoma

Neuroblastoma

Background

Establishment and Validation of an IPS-based Neuroblastoma Model

Neuroblastoma

Background

Neuroblastoma is the most common extra-cranial solid childhood cancer, accounting for 15% of all cancer deaths in children. Targeted therapies hold promise to improve the outcome for children with high-risk neuroblastoma, however this issue remains challenging. Amplification of the MYCN oncogene occurs in half of high-risk patients.

Investigation of Causal Germline Mutations in High Hyperdiploid ALL

Acute Lymphoblastic Leukemia (ALL)

Background

High-dimensional Biochemical CyTOF Analysis of Pediatric T cell Leukemias

Acute Lymphoblastic Leukemia (ALL)

Background

Pediatric T cell Acute Lymphoblastic Leukemia (T-ALL) is an aggressive blood cancer and children are currently treated with chemotherapy but its non-specific and toxic aspect is problematic. More specific therapies are desired but T-ALL's differ from patient to patient and even within one patient there are cell subsets within the leukemia (called heterogeneity). It is therefore challenging to implement or analyze the effects of precision medicine that inhibit abnormally functioning proteins in T-ALL.

Targeting Secondary Mutations in Juvenile Myelomonocytic Leukemia

Leukemia, Juvenile Myelomonocytic Leukemia (JMML)

Background

While most types of childhood leukemia are routinely cured with therapy, juvenile myelomonocytic leukemia (JMML), a type of blood cancer that affects young children remains exceedingly difficult to treat. Even when cured, the infants and toddlers affected by this disorder have difficulty tolerating the required treatment, which can be very intense. Equally frustrating, it is challenging to predict who will respond to the treatments we currently have versus those that will require experimental therapy in order to cure them of their cancer.

Chemical Genetics and CRISPRi/a Screen for Novel Therapeutic Targets in a Humanized Model for MYCN Associated High-Risk Pediatric Neuroblastoma

Neuroblastoma

Background

Neuroblastoma is the most common extracranial solid tumor in children. Amplification of the MYCN oncogene is identified in about half of high-risk patients and is associated with rapid tumor progression and poor prognosis irrespective of tumor stage. MYCN protein overexpression is sufficient to drive pathogenesis of neuroblastoma in vitro and in vivo. Extensive studies have been done on targeting known regulators of MYCN to decrease pathological MYCN protein level.

Understanding the Role of Translational Control in Humanized Mouse Models for Medulloblastoma

Brain Tumors, Medulloblastoma

Background

Medulloblastoma (MB) is the most common malignant pediatric brain tumor. While 70-80% of patients survive more than 5 years, survivors have significant neurocognitive and neuro-endocrine disabilities. Several recent studies have identified 4 distinct molecular subgroups (WNT, SHH, Group 3 and Group 4). These subgroups differ in their mutational spectra, gene expression signatures and clinical features including outcome.

Impact of Whole Body Radiation Dose After Therapy with 131I-Metaiodobenzylguanidine (MIBG)

Neuroblastoma

Investigating the Role of Genetic and Epigenetic Variation in Risk of Childhood Acute Lymphoblastic Leukemia in Down Syndrome

Acute Myeloid Leukemia (AML)

Background

Acute lymphoblastic leukemia (ALL) is the most common cancer in childhood. Down syndrome (DS) is a genetic disorder caused by an extra chromosome 21, and DS children have a 20-fold increased risk of ALL. Furthermore, children with ALL and DS ("DS-ALL") have increased treatment-related deaths and disease recurrence than children without DS. Determining the cause of DS-ALL is, therefore, of paramount importance. The additional chromosome 21 must play a role in increased leukemia risk, but not every child with DS develops ALL.

Alex's Lemonade Stand Foundation for Childhood Cancer