Survival rates for children with AML are greater than 60%; however, the outcome for patients with relapsed or refractory disease remains poor with less than 40% becoming long term survivors. Treatment regimens for relapsed disease are conventional chemotherapeutic agents and hematopoietic stem cell transplant (HSCT). The investigators in this phase I study suggest utilizing two targeted therapies to combat AML. The first is selinexor, a drug that targets and inhibits exportin 1, a protein responsible for exporting oncoproteins (proteins that promote tumor growth and proliferation) out of cells. The second is venetoclax a drug that targets and inhibits a family of proteins called BCL2. Overexpression of BCL2 prevents cancer cells from being killed by immune cells as they inhibit a process called apoptosis (or programmed cell death) and this is associated with resistance to chemotherapy and worsened clinical outcomes. Thus, this phase I study will determine the recommended phase 2 dosing of the combination of selinexor and venetoclax in pediatric patients with relapsed or refractory (resistant) AML or ALAL.
Trial Registration: ClinicalTrials.gov Identifier: NCT04898894