Alex's Lemonade Stand Foundation for Childhood Cancer

Cancer is the leading cause of death by a disease in children ages 0-19 years old. Whereas cure rates for pediatric hematologic malignancies as a class can reach 90% or more, solid tumor cures have historically lagged and contribute disproportionately to the cancer death rate in children. A common mechanism among relapsed and treatment-resistant pediatric solid tumors is a combination of errant signaling that drives cancer cell proliferation and blocks cancer cell death. Thus, multiagent treatment that addresses multiple cancer-causing pathways is required.

Mentor: Loren Walensky

Mentor: Dr. Alfred Thomas Look

Lay Summary: Children with metastatic osteosarcoma have an especially poor prognosis. Treatments for osteosarcoma have not changed in decades which highlights the need for new and novel therapies. Little is known about why tumors form metastases in some patients and remain localized in other patients. Cells from the tumor break free and circulate in the bloodstream of patients with osteosarcoma. These cells can be detected as circulating tumor cells (CTCs) and are thought to contribute to metastasis formation.

Neuroblastoma (NB) is the most common pediatric extracranial solid tumor. Although many high-risk neuroblastoma patients respond to conventional radiation and chemotherapy, only half survive and many continue to suffer from significant toxicities. Novel therapeutics are desperately needed for this disease, especially target specific therapies that will reduce off-target toxicity. Here, we plan to develop an effective strategy for NB by targeting a novel oncoprotein, EP300, which we identified as a dependency in NB.