Prevention of leukemia in cancer predisposition syndromes

Pediatric Cancers

Hojun Li, PhD

Nicholas Gloude, MD

Children with bone marrow failure syndromes are pre-disposed to developing leukemia. The only way to prevent leukemia in these children is a bone marrow transplant to remove the diseased cells that ultimately turn into leukemia. However, current methods for bone marrow transplant have high rates of complications and death due to toxicity of the chemotherapy used to prepare a patient for receiving transplanted cells and inappropriate immune system activation after transplant. We will address these complications by developing less toxic chemotherapy alternatives and developing ways to shield the immune system from inappropriate activation. These advances will overcome obstacles that limit performing bone marrow transplant to prevent leukemia in children with bone marrow failure syndromes.