Alex's Lemonade Stand Foundation for Childhood Cancer

Acute myeloid leukemia (AML) is an aggressive cancer of the blood and bone marrow. It starts in stem cells that should normally become healthy white or red blood cells, but instead turn into leukemia cells that grow uncontrollably. AML can affect people of all ages, but some of the most dangerous forms occur in children and teenagers. These patients often require intense chemotherapy, hospitalization, and sometimes bone marrow transplants. Even with these treatments, some children relapse, and options become limited.

Pediatric acute myeloid leukemia (AML) is a life-threatening blood cancer that interferes with the normal formation of blood cells, leaving children vulnerable to infection, bleeding, and relapse after treatment. Among the genetic changes linked to poor outcomes in pediatric AML, mutations in the gene ASXL1 are especially concerning, as children whose leukemia carries these mutations often have more aggressive disease and fewer effective treatment options.

Mentor Name: Bill Carroll

The cure rate for childhood acute lymphoblastic leukemia (ALL) has improved remarkably over the last four decades. However in spite of this improvement 10% to 15% of children will suffer a recurrence and their prognosis is dismal making ALL one of the leading causes of death due to cancer in children. We and others have shown that relapse is due to the outgrowth of a resistant subclone and we have discovered that these subclones contain mutations in genes that regulate response to the drugs used in therapy. This is true for both conventional, as well as, new immunotherapeutic agents.