Alex's Lemonade Stand Foundation for Childhood Cancer

Background

Background

More children die from acute lymphoblastic leukemia (ALL) than from any other cancer. NOTCH1 was found to be the most frequent cancer-causing gene in T-ALL. However, in clinical trials anti-NOTCH drugs had too much toxicity and even promoted cancers. The reason for these side effects is that Notch is an important protein that keeps normal tissues healthy. How can we knock out Notch in cancer, but preserve Notch in normal cells? We believe that the answer may be two proteins called Zmiz1 and Ets1.

Background

Pediatric acute myeloid leukemia (AML) is a type of blood cancer that is in need of new therapies, as children suffering from high risk disease have little chance of cure. Aggressive treatment with traditional chemotherapy drugs can lead to complications and severe toxicities. Targeted therapy may have fewer side effects, and immune therapies in particular are a new class of treatments that offers great promise.

Background

Brain tumors are the leading cause of death among childhood cancers. Despite recent data showing differences in mutated genes in childhood and adult glioblastoma, the treatment is the same for both patient populations. The ATRX gene is mutated primarily in adolescent patients with glioblastoma, a devastating and highly malignant brain tumor. Treatments for human cancer are becoming increasingly personalized, and ATRX loss allows for a promising target for individualized treatment for patients with glioblastoma.