Alex's Lemonade Stand Foundation for Childhood Cancer

H3.3K27M-altered pediatric diffuse midline glioma (DMG) are fatal brain tumors, accounting for 10% of pediatric brain tumors and affecting young children between the median ages of 5-10 years old. Despite advances in surgical resection, radiation, and chemotherapy, there remains no effective curative option. There is an unmet clinical need to develop new and effective therapies for DMG. The majority of DMGs are driven by mutated histone proteins known as H3.3K27M oncohistones.

Medulloblastoma, a tumor of the cerebellum, is the most common malignant brain tumor in children. Although a combination of surgery, radiation and chemotherapy can cure many medulloblastoma patients, 25-30% of patients still die from their disease. Moreover, those who survive suffer devastating side effects from the treatment, including cognitive deficits an increased susceptibility to other cancers later in life. More effective strategies for treating MB depend on development of animal models that can be used to study the causes of the disease and to test new approaches to therapy.