Alex's Lemonade Stand Foundation for Childhood Cancer

Juvenile Myelomonocytic Leukemia (JMML) is a type of blood cancer that affects young children. Even with intensive treatment, the cancer often comes back. Mutations (changes) in a gene called SETBP1 increase the chances that this cancer will return. When there is a mutation in SETBP1 in the cancer, only 18% of children with JMML live for more than five years after treatment. Despite how important SETBP1 is in JMML, we have no drugs that target changes in this gene to improve survival for children with this cancer.

Background

More than half of people with familial platelet disorder (FPD) are at risk of developing the blood cancer acute myeloid leukemia (AML). Individuals with FPD are born with mutations in a gene called RUNX1 and their cells may develop additional mutations over time that can trigger the development of AML. By performing genetic sequencing, we identified 16 patients with AML who have inherited RUNX1 mutations as well as additional mutations in known cancer-causing genes.

Watch the video below to learn about Dr. Charles Keller and his research on "Therapeutics in Ovo" at the Oregon Health & Science University in Portland, OR.