Childhood Cancer Research
Children’s Hospital of Philadelphia
3401 Civic Center Boulevard
Philadelphia, PA 19104
United States
Background
Neuroblastoma remains a devastating clinical problem and continues to be a leading cause of childhood cancer morbidity and mortality despite dramatic increases in therapy. Recent developments in cancer research are changing our understanding of neuroblastoma biology and treatment on the basis of the molecular underpinnings of individual tumors, and are challenging us to design more rational and less toxic therapies.
Neuroblastoma remains a devastating clinical problem and continues to be a leading cause of childhood cancer morbidity and mortality despite dramatic increases in therapy. We made the discovery that the Anaplastic Lymphoma Kinase (ALK) oncogene is a promising therapeutic target in neuroblastoma and translated this seminal finding into a clinical trial.
Neuroblastoma remains the deadliest solid tumor of childhood. A genetic alteration found in a large number of high-risk cases makes it particularly challenging, and existing therapies still fail to cure over half of these patients. New treatment strategies, both safe and effective against this aggressive form of the disease are, therefore, urgently needed. A drug delivery strategy using tumor targeted nanocarriers that will be evaluated and optimized by our team in this project is designed to address this need.
Acute myeloid leukemia (AML) is a rare but very serious type of blood cancer in children. While treatments for childhood cancer have improved, 20-40% of children of AML still do not survive. Black and Hispanic children are less likely to survive than White children, which may be due to differences in access to care, delays in getting diagnosed, or other barriers that disproportionately affect families from minority communities. Black and Hispanic children are also less likely to be enrolled on clinical trials, but children on clinical trials do better. We don’t know why.
Brain tumors pose a significant challenge in pediatric oncology and are among the leading causes of cancer-related deaths in children in the United States. Among these, Pediatric High-Grade Gliomas (pHGG) make up about 10% of all brain and central nervous system tumors. These tumors are particularly aggressive and have a very poor prognosis. Patients with pHGG tumors like diffuse intrinsic pontine gliomas (DIPG) typically do not survive more than two years, and there are not many treatment options available for them.
Retinoblastoma is the most common childhood cancer of the eye. With early detection and access to current optimal clinical management, retinoblastoma can be controlled in some children, although treatment-related toxicities remain including potential vision loss and development of secondary cancers. In cases where retinoblastoma has gone untreated or comes back after treatment, removal of the eye is still the only option available to prevent the cancer from spreading to the brain, where it is incurable.
Mentor Name: Timothy Spear
Mentor Name: Caitlin Elgarten
Mentor Name: Richard Aplenc
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