Alex's Lemonade Stand Foundation for Childhood Cancer

Many pediatric blood cancers are cured by hematopoietic cell transplantation (HCT), whereby a donor’s blood- and immune-cell forming stem cells are transferred to a recipient. Unfortunately, the resultant donor-derived immune system sometimes erroneously attacks recipient tissue, called graft-versus-host disease (GVHD). The chronic GVHD (cGVHD) form can last for years post-HCT, affecting numerous organ systems. The most life-limiting cGVHD manifestations are fibrotic, including sclerosis of the skin and bronchiolitis obliterans syndrome (BOS) of the lung.

Acute myeloid leukemia (AML) is a difficult-to-treat blood cancer, and many children with AML will eventually die from intensive chemotherapies and bone marrow transplantation. Antibodies against CD33, a protein found on the surface of AML cells of most patients, have been developed hoping to improve these outcomes. Indeed, gemtuzumab ozogamicin (GO), an antibody with a cell toxin attached (“antibody-drug conjugate” [ADC]) that binds to the distal, V-set portion of CD33, has benefitted some children with AML; however, many do not benefit despite having CD33 present on their AML cells.

Mentor Name: Brandon Hadland

Mentor: Soheil Meshinchi

Co-investigator: Dr. Colin Correnti