Alex's Lemonade Stand Foundation for Childhood Cancer

Mentor Name: Kristen Long

Alkylating agents are a class of chemotherapeutics important for children with high-risk or relapsed leukemias. These are given during what we call conditioning regimens just before bone marrow transplantation (BMT). The goal of conditioning regimens is to eliminate all leukemia cells, before the bone marrow is replaced by BMT. However, the failure of these conditioning regimens to ablate all leukemia cells is a major problem that leads to relapse after BMT. Leukemic relapse is the most common cause of treatment failure of BMT regimens, and these patients have a very poor prognosis.

Project Team
Ross Levine, MD, Memorial Sloan-Kettering Cancer Center
Scott A. Armstrong, MD/PhD, Dana-Farber/Boston Children's Cancer and Blood Disorders Center
Vijay G. Sankaran, MD/PhD, Boston Children's Hospital

Background

Familial RUNX1 disorders [familial platelet disorder (FPD)/ acute myeloid leukemia (AML)] are caused by inherited mutations in one of the two copies of the RUNX1 gene. The RUNX1 gene produces a protein that normally turns on and off other key genes required for normal blood cell development. Individuals with FPD/AML have low platelet counts and increased leukemia risk. Because the leukemia onset takes time, there is a window of opportunity to intervene to prevent leukemia development.