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.

Ewing sarcoma is a group of bone and soft tissue cancers that can occur in children, adolescents, and young adults. The high response rates to DNA-damaging chemotherapy suggest that there may be defects in DNA damage repair pathways that make Ewing sarcoma cells susceptible to chemotherapy-induced DNA damage. However, despite these high response rates, there is still an urgent need for new treatment options for Ewing sarcoma, particularly for patients with relapsed and/or refractory disease.

Osteosarcoma is a tumor of the bone that typically occurs in adolescents and young adults. Many patients with osteosarcoma are cured of their disease, but a significant proportion of patients are not. The primary approach to treating osteosarcoma includes surgery and chemotherapy. Unfortunately, this approach has not improved for several decades. One of the reasons why it has been difficult to develop new treatment approaches for osteosarcoma patients is that we don’t fully understand how these tumors develop.

Pediatric cancer remains the second leading cause of death in children. One type of cancer that is particularly difficult to treat is FLT3 mutant acute myeloid leukemia (AML). AML cells that have a FLT3 mutation grow at a rapid rate and develop strategies to escape monitoring by the immune system, including blocking natural killer (NK) cell anti-tumor activity. We also know that many cancer patients have low NK cell numbers and decreased NK cell activity as a result of chemotherapy.

Desmoplastic small round cell tumor is a rare cancer that affects children and young adults. Most patients have very advanced disease already when they are diagnosed, and the long-term survival rate is low. Current treatments can't cure DSRCT and are very toxic. Patients who do survive are often left with debilitating side effects that last the rest of their lives. We have developed a new type of radiation therapy that targets the tumor cells specifically without harming normal tissue. In mice, this type of therapy can cure tumors without causing toxicity.