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.

The clinical outcome for many patients with solid tumors including bone tumors, called osteosarcoma, remains poor. This is particularly true for patients in whom the cancer has recurred or spread to the lungs. Immunotherapy has the potential to improve outcomes for these patients. We are interested in a form of immunotherapy called chimeric antigen receptor (CAR) T-cell therapy, which takes the patient’s own immune cells, modifies them in the laboratory to recognize and kill cancer cells, and puts them back into the patients.