Alex's Lemonade Stand Foundation for Childhood Cancer

Mentor Name: Elvin Wagenblast

Clonal hematopoiesis (CH) occurs when certain blood cells begin to grow abnormally and dominate over others, even in people who are otherwise healthy. This condition becomes more common with age and increases the risk of developing blood cancers. However, for individuals with a rare inherited disorder called RUNX1 familial platelet disorder (RUNX1-FPD), CH can appear at a much younger age. While these early clones are often unstable and less likely to lead to cancer, stable clones become more frequent as patients grow older, increasing their cancer risk.

Leukemia is the most common cancer in children, accounting for almost 1 out of 3 cancers. Acute myeloid leukemia (AML) is a cancer of the myeloid line of blood cells and is the leading cause of childhood leukemic mortality. There is a great need to improve the outcome of children who are diagnosed with AML by developing better-targeted treatment options. Although both children and adults are affected by AML, studies indicate that the specific genetic mutations present in childhood and adult leukemia are different.

Mentor Name: Elvin Wagenblast

Hepatoblastoma (HB) is the most frequent pediatric form of liver cancer, generally arising in young children (<3yo). Despite being a rare tumor (1.8 in 1,000,000 children/year), HB rates are on the rise and therapeutic options are limited to chemotherapy. Chemotherapy (i.e. cisplatin) is given to patients without any consideration to a specific mutation within tumors and toxicity is significant. Here we seek to advance a novel therapeutic candidate for HB that has been optimized to selectively kill cancer cells with specific mutations, while sparing normal hepatocites.