Childhood Cancer Research

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Children’s Hospital of Philadelphia

3401 Civic Center Boulevard
Philadelphia, PA 19104
United States

Cancer therapies such as CAR T cells and targeted ALK drugs can be remarkably effective for some patients, but others do not respond or eventually relapse, and we often do not know why. Our project will develop a next-generation blood test to understand, in detail, how these novel treatments work—or fail to work—by reading signals from tiny fragments of DNA circulating in the bloodstream.

Nearly 40% of all childhood cancers contain abnormal gene fusions, yet targeted therapies exist for only a subset of these alterations. Fusions, formed when two separate genes are joined together, promote tumor growth and often drive disease, making them attractive therapeutic targets. However, the complicated structure of fusion proteins often impedes small molecule inhibitor development, and when drugs are available, resistance to therapy inevitably develops. Furthermore, the diversity of rare fusion partners hinders traditional drug development mechanisms.

Children’s Hospital of Philadelphia

As global temperatures rise, extreme heat is becoming more common. Under-resourced and historically minoritized communities are especially vulnerable to the strain of heat because their neighborhoods have more concrete, fewer green spaces, and worse air quality. The kidneys are particularly sensitive to heat stress and dehydration. Children with acute lymphoblastic leukemia (ALL) are already at high risk of kidney injury from their disease and the medications they receive for treatment. Kidney injury can lead to delays in chemotherapy, hospitalizations, and other serious complications.

Children’s Hospital of Philadelphia

Neuroblastoma is a cancer of infants and children that arises from the developing nervous system. Patients with aggressive forms of the disease, termed high-risk neuroblastoma, continue to face poor long-term survival and substantial treatment-related side effects from an intensive treatment regimen of chemotherapy, surgery, radiation, and immunotherapy. There is an urgent unmet need for more effective precision therapies that target cancer cells while sparing healthy cells to improve survival outcomes and reduce treatment-related side effects.

Children’s Hospital of Philadelphia

Mentor Name: Alix Seif

Children’s Hospital of Philadelphia

Mentor Name: Adam Wolpaw

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