Alex's Lemonade Stand Foundation for Childhood Cancer

Background

The aggressive and unpredictable behavior of relapsed T-cell acute lymphoblastic leukemia (T-ALL) presents a major clinical challenge, as relapsed disease continues to have a dismal prognosis in both children and adults. A major hurdle in the development of new chemotherapies is identifying drugs that specifically kill malignant T cells, while leaving normal cells untouched.

Background

Each year, hundreds of children undergo radiation therapy, in order to treat their primary cancer. Due to radiation's potency and in the absence of highly effective protection systems, these children may suffer brain damage and experience developmental problems. Also, radiation can affect their skin and bone marrow, causing skin cancer and leukemia.

Infant acute leukemias (arising in children less than one year old) makes up a unique subset of leukemias that are particularly lethal and that are characterized by recurrent mutations in one gene. This gene has been termed the mixed lineage leukemia (or MLL) gene, and mutations in MLL are found in the majority of infant leukemias as well as in smaller proportions of childhood and adult leukemias. The mutations in MLL are intriguing, because they are the result of chromosomal breakage which abnormally joins the MLL gene to a variety of partner genes.

Research in pediatric cancer over last 30 years has led to improvements in the treatment and cure rate for most of the pediatric cancer. Despite improvement in the pediatric patient survival over the last decades, still about 30-40% of children die of cancer and also many treatments affects the quality of the life. There is a need for identification of new targets and anti-cancer agents to improve the survival and quality of life. TLE1 gene is a novel tumor suppressor and co-repressor gene that is deleted or methylated in acute myeloid leukemia (AML) and other hematological malignancies.

Background

The pediatric kidney cancer, Wilms tumor, is the fourth most common type of cancer in children. Wilms tumor seems to arise because of abnormalities in the formation of the kidneys from primitive cells in the embryo. The most studied genes mutated in Wilms tumor include WT1 and beta-catenin, which are essential for controlling the development of normal kidneys and in controlling how cells divide. However, these genes are only mutated in a small percentage of Wilms tumors.