Alex's Lemonade Stand Foundation for Childhood Cancer

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.

Lay Summary: Wilms tumor is the most common pediatric kidney cancer. The best predictor of clinical outcome for Wilms tumor patients is how their tumor looks under the microscope (histology). The majority of Wilms tumor patients have favorable histology tumors that respond to surgery, chemotherapy, and radiation. These tumors are usually called triphasic tumors because they contain three main cell types--epithelial cells, blastemal cells, and stromal cells.

Background

Pediatric solid tumors arise during the development of diverse tissues such as bone, muscle and adrenal gland. The tumor cells maintain many features of the normal tissue where they develop including the developmental hierarchy. As a result, cells within an individual patient's tumor has features of different developmental stages. This tumor heterogeneity is important because, in some patients, a subset of those cells survive treatment and contribute to disease relapse which is often fatal.