David Loeb, MD, PhD

Funded by the Dick Vitale Pediatric Cancer Research Fund

Ewing sarcoma is the second most common bone tumor in children, adolescents, and young adults.  Patients who are diagnosed with a tumor that has not spread are usually cured.  Those who are diagnosed with metastases (the tumor has spread from its initial location) are rarely cured despite decades of clinical trials and intensifying treatment regimens aimed at improving their survival.  In preliminary animal experiments, we found that a drug called DFMO, already approved by the FDA for the treatment of African Sleeping Sickness, can inhibit Ewing sarcoma metastasis.  We will test the hypothesis that DFMO acts by interfering with critical metabolic pathways in tumor cells, that it is safe to combine DFMO with chemotherapy, and that the combination of DFMO and chemotherapy will work better than chemotherapy alone in prolonging the lives of mice with Ewing sarcoma.  Assuming we can show that the combination of DFMO and chemotherapy is better than chemotherapy alone in our mouse model, this will provide the rationale for future clinical trials testing the effectiveness of adding DFMO to standard chemotherapy regimens for Ewing sarcoma patients. 

Location: Albert Einstein Cancer Center - New York
Proposal: Targeting Urea Cycle Dysfunction to Prevent and Treat Ewing Sarcoma Metastasis
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