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University of Colorado Denver

12605 E 16th Ave
Aurora, CO 80045
United States

Background

The primary objective of this study is to better understand how to combine a treatment that blocks a survival mechanism in pediatric brain tumors with other cancer therapies to improve survival. The survival mechanism, autophagy, is a way for cells to recycle things inside the cell and use them for energy when under stress. Cancer cells also use this to find ways to survive treatments we use to try and kill them.

Overall survival for children diagnosed with acute lymphoblastic leukemia (ALL) has dramatically improved over recent years due to optimization of dosing and timing of standard chemotherapeutics. However, this intensified treatment is associated with increased short-term side effects and long-term, irreversible toxicities, which occur in up to 25% of pediatric patients and include cognitive and developmental disabilities, infertility, damage to organs, and secondary cancers. In addition, subsets of patients are refractory to therapy or have disease relapse.

Ewing sarcoma is a common cancer of bone and soft tissue affecting children and young adults. It is a very aggressive cancer with a poor long-term outcome. Even with the best chemotherapy, long-term survival for Ewing sarcoma patients is about 50%, drops to 25% for those presenting with metastatic disease, and plummets to 10% for patients with recurrent disease. Thus, there is a great need for new therapies for this disease. MicroRNAs (miRs) are recently discovered molecules normally occurring in cells, which have critical functions in normal physiology and in disease.

Background


Brain tumors are the leading cause of death from cancer in children under the age of 15 years. Medulloblastoma, the most common pediatric brain tumor, continues to have 5-year overall survival rates of 60%. Furthermore disease and treatment related morbidity is a major problem. Developing disease risk assessment tools for targeting current therapeutics is vital. Developing novel therapies that are less toxic is also crucial.

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