Alex's Lemonade Stand Foundation for Childhood Cancer

Ependymoma is a severe childhood brain tumor that often cannot be cured with current treatments. Children typically undergo surgery and radiation, which can help manage the cancer but often cause long-term side effects. Unfortunately, there are no medicines that specifically target the biology of this cancer, and many children experience tumor regrowth even after aggressive treatment. Most ependymomas in the upper part of the brain are caused by a genetic error where two genes are abnormally fused together, creating what is called a fusion oncoprotein.

Neuroblastoma is the most common solid tumor found outside the brain in children and accounts for nearly 10% of all childhood cancer diagnoses, while causing almost 15% of deaths from childhood cancers. Despite the use of very aggressive and toxic combination treatments, children with high-risk neuroblastoma still have poor survival rates, and there are currently no cures for children whose disease comes back after treatment. High-risk neuroblastoma includes several genetic subtypes.

Patients with metastatic solid tumors, such as rhabdomyosarcoma (RMS), continue to suffer from dismal patient outcomes. Innovation for treating these patients is hampered by our limited understanding of how RMS cells detach from tumors and seed metastatic sites. Our laboratory are leaders in the study of the complex cellular architecture of RMS. In prior work, we identified the broad range of cell types within RMS and are now in a position to translate those results. In preliminary work, we have developed an easy-to-perform and replicable system to model the process of cell detachment.

Safe and effective treatment options are desperately needed for pediatric brain tumor patients. Specifically, I am focused on group 3 medulloblastoma, a lethal brain tumor that often affects infants and has very poor prognosis. The Krenciute lab at St. Jude, in which I do my research, is dedicated to developing immunotherapy treatment options for pediatric brain tumor patients in which a patient’s own immune system is re-engineered to fight their cancer in a way that is effective and safe for all ages.

Children with solid tumors have poor overall survival. Further, the treatments used for these diseases are very toxic. One particularly difficult to treat pediatric solid tumor is called neuroblastoma (NB). Children with neuroblastoma are treated with very toxic therapies, and even so, many of them will not be cured. In addition, while large-scale studies have identified the specific proteins that NB cells depend on for growth, these proteins are especially hard to target with traditional drugs. This limits our ability to treat NB effectively.