Alex's Lemonade Stand Foundation for Childhood Cancer

Mentor Name: Ranjit Bindra

Childhood leukemia is a devastating cancer and many current treatments are not effective for certain aggressive forms of the disease. This project aims to uncover how specific cancer-causing genes alter normal cell processes to drive leukemia. In particular, we are investigating how these changes cause cells in very young infants to become cancerous. By understanding these mechanisms, we can identify new waysto block the cancer’s growth.

Background
Pediatric brain tumors often are treated with high doses of chemotherapy and radiation therapy. These treatments are effective but lead to significant late effects on long-term survivors.

Background

Diffuse Intrinsic Pontine Glioma (DIPG) is a rare but lethal childhood tumor with no known effective treatment. Given its location in the brainstem, surgical resection is not feasible, and the only available treatment is radiation therapy, which is palliative in nature. As such, better therapies are needed which can be combined with radiation therapy for durable disease control. Remarkably, there are limited platforms suitable for use in DIPG screening studies to identify such agents.

Primary brain tumors are one of the most common solid neoplasms in children, and they are a leading cause of cancer-related mortality. In particular, pediatric high grade gliomas are clinically devastating tumors, with associated 5-year survival rates of less than 20%. The current treatment for these cancers begins with surgical removal of the tumor, followed by radiation therapy (RT) and chemotherapy. RT consists of ionizing radiation (IR), which kills residual tumor cells by inducing DNA breaks in the genomes of these cells.