Children’s Hospital of Philadelphia

Children’s Hospital of Philadelphia

3401 Civic Center Boulevard

Philadelphia, PA 19104

United States

Development of Immunostimulatory DNA as a Novel Therapy in Pediatric Acute Lymphoblastic Leukemia

Leukemia, Acute Lymphoblastic Leukemia (ALL)

Acute lymphoblastic leukemia (ALL) is the most common childhood cancer. While current therapies have resulted in significant improvements in survival, relapsed ALL does not usually respond to chemotherapy. If a child does eventually respond to more intensive chemotherapy, bone marrow transplant offers the best chance for cure, although with significant risks of severe illness or death.

Biology-based clinical trials for children with cancer.

General Pediatric Cancer

Disease-specific research teams at the Children's Hospital of Philadelphia (CHOP) have been successful in bench to bedside work, having identified key molecules involved in the genesis of neuroblastoma, and subsequently having studied agents that target these molecules in preclinical and clinical settings. From a programmatic point of view, it is logical to build upon this success and apply the bench to bedside paradigm beyond the disease-specific setting.

BH3 profiling to define therapy resistance classes in neuroblastoma.

Neuroblastoma

The failure of cancer therapy to control tumor growth results from alterations in the pathways that control cell death decisions within tumors. Although the alterations cancer cells develop to gain a survival advantage are diverse, most ultimately lead to the disruption of death signals that should be Òturned onÓ when lethal stress is encountered. A family of Bcl2-homology (or BH) proteins are responsible for responding to these stressors to initiate a death signal when sufficient stress is present.

Pharmacogenomics of childhood AML susceptibility and treatment response.

Acute Myeloid Leukemia (AML)

Although acute myeloid leukemia (AML) causes a substantial disease and treatment burden in both children and adults, the causes of AML and unsuccessful treatment are poorly understood. Currently, many scientists believe that naturally occurring genetic variation changes AML susceptibility and relapse risks. This application seeks to identify these specific genetic variations by searching throughout the genome with a genome-wide association study (GWAS). This research seeks to find these genetic changes with three specific aims.