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.

Rhabdomyosarcoma is the most common soft-tissue cancer in children and adolescents and a highly aggressive from of cancer. Rhabdomyosarcoma resembles developing muscle tissue, and it is likely to arise as a 'developmental mistake' from progenitor cells that belong to the family of skeletal muscle cells. However, the precise cell type(s) from which rhabdomyosarcoma originates remain unclear. We argue here that it is important to understand the interplay between cancer-associated genes and the cell environment in which they take effect in order to develop novel, much needed treatments.

While much success in the treatment of pediatric cancers has been obtained over the past 20 years by optimizing the use of previously discovered chemotherapies, new agents are desperately needed to cure those patients who do not respond to current therapy. DNA mutations and structural abnormalities in chromosomes contribute to cancer formation by improperly regulating genes in the cell. Another mechanism by which genes become misregulated in cancer is through aberrant DNA methylation.

With chemotherapy treatment, 80-90% of children with acute myeloid leukemia (AML) achieve remission.  However, 30-40% of these patients subsequently suffer recurrence, and the long-term survival rate is only 50%.  Novel therapies are urgently needed to prevent or treat recurring AML.  Parthenolide, a naturally-occurring compound, induces robust AML cell killing and has the potential to reduce recurrence of AML by killing cells resistant to traditional chemotherapeutics and responsible for relapse.

Cure rates for T-cell Acute Lymphoblastic Leukemia and Lymphoma (T-ALL/LL) have gone from 0% to approximately 80% over the past 4 decades. However, this success comes with a price. The incidence of both acute and long-term toxicities of intensive therapy is becoming increasingly common as more children survive their initial cancers. Moreover, despite intensive therapy, a significant number of children still relapse. Unfortunately, virtually none of these children can be cured with current therapy.