Targeting Mitochondrial Pathways in Pediatric AML
Dr. Sakamoto sends her most heartfelt and sincerest “thank you” to all the members of the Pediatric Cancer Research Foundation for their dedication and commitment to children with cancer.
Leukemia is the most common form of childhood cancer. Acute myeloid leukemia (AML) is associated with a survival rate of 60-70%. The treatment is intensive chemotherapy and stem cell transplantation, which are associated with significant side effects and long-term complications. Therefore, more effective and less toxic drugs are needed. There are also very few treatment options for patients who relapse or have disease that becomes resistant to treatment. Our project proposes to repurpose an FDA approved drug known as niclosamide that has been used to treat children with tapeworm. Niclosamide blocks mitochondria, which produce energy in the body’s cells. This drug is well-tolerated and was shown to be effective in blocking the growth of pediatric AML cells in pre-clinical testing. We are also examining the possibility of other drugs that block the mitochondria that might act together with niclosamide for future clinical trials in children with relapsed or resistant AML.