Alex's Lemonade Stand Foundation for Childhood Cancer

This study is about finding new ways to fight cancer, especially in children with a cancer called neuroblastoma. Neuroblastoma is tricky to treat because the cancer cells can become resistant to chemotherapy, meaning the drugs don't work as well over time. The cancer cells can switch between two different states: one where they are sensitive to drugs and one where usual cancer treatments do not work well. Here, I am proposing to understand how these cells switch between these states, which allows them to survive and avoid our best treatments.

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.

Medulloblastoma (MB) is a deadly form of childhood brain cancer. Recent studies performed on tumor samples obtained from MB patients have discovered that MB is not a single disease, but best characterized as multiple clinically distinct "subgroups". Different MB subgroups are believed to begin in distinct cell types of the developing brain, in a region known as the cerebellum. Current treatments for MB are non-specific and cause significant lifelong side effects that prevent an independent life after cancer.

Mentor Name: Stephen Mack

While autologous chimeric antigen receptor (CAR) T cell therapy has proven efficacious for many patients with acute lymphoblastic leukemia, CD19-positive relapse often occurs after CD19-CAR T cell therapy indicating that the CAR T cells either lack persistence or functionality.