Alex's Lemonade Stand Foundation for Childhood Cancer

Despite various treatment options, cure rates for sarcoma patients continue to be very poor. Many researchers have developed new therapies that harness the immune system to cure cancers; however, most of these treatments (called immunotherapies) are not effective in solid tumors. One of the most promising therapies involves T cells expressing artificial receptors that allow them to recognize and kill cancer cells.

Medulloblastoma (MB) is the most common malignant pediatric tumor, accounting for 15-20% of childhood brain tumors. Current multimodal therapies--including surgery and radiochemotherapy--increase long-term survival to 60-80%, but 33% of children diagnosed die in 5 years; the median survival for recurrent MB patients being less than 12 months. Molecular profiling and genetic analysis have categorized MB into 4 subgroups, each associated with distinct genetic alterations, age at onset, and prognosis.

Lay Summary: Low-grade gliomas (LGG) are the most common brain tumors diagnosed in children, and high-grade gliomas (HGG) are the most common cause of death. While many children can be treated with surgery alone for LGG, a large number of patients are unable to undergo surgery due to the location of the tumor, while others carry higher risk factors due to underlying mutations in the tumor.

Background

Medulloblastoma is a pediatric cancer that quickly grows in the cerebellum. Although, there are many different types of medulloblastoma, patients are uniformly treated with therapies including surgery, radiation and chemotherapy. In order to find better treatments with less severe side effects it is important to understand the different types of medulloblastoma so we can tailor the treatment to each type of medulloblastoma.

Background

ASH1L is an epigenetic regulator that is thought to act in gene expression through its activity as a histone H3, lysine 36 methylase. It has also been shown to act synergistically with the MLL1 proto-oncoprotein to regulate important pro-leukemogenic targets.