Acute Myeloid Leukemia (AML) is the most common acute blood cancer that primarily affects older adults, with a median age of 68 years at diagnosis. In AML, blood cells grow uncontrollably without maturing properly, filling up the bloodstream and suppressing healthy cell growth. Genetic changes in these cells influence their behavior, making the disease unique for each patient.

Standard curative treatment for AML consists of intensive induction chemotherapy followed by consolidation chemotherapy, allogeneic stem-cell transplantation, or both. However, because of advanced age, coexisting conditions, and a high incidence of unfavorable genomic features, older patients are frequently ineligible for or do not respond well to standard chemotherapy. Instead, such patients often receive less intensive regimens. One well-tolerated treatment approach that has yielded promising results is the combined utilization of Venetoclax plus Azacitidine. This combination is the standard of care for older adults and those ineligible for intensive chemotherapy. This therapeutic combination has exhibited enhanced response rates and overall survival compared to Azacitidine alone.

The synergistic effect of Venetoclax plus Azacitidine in treating AML stems from the distinct mechanisms of each. Venetoclax, a targeted therapy, inhibits the B-cell lymphoma 2 (BCL-2) protein, triggering apoptosis, or cell death, in malignant AML cells. Additionally, Azacitidine disrupts abnormal DNA methylation patterns in cancer cells, restoring normal cellular functions. This approach weakens leukemia cells' survival mechanisms and promotes apoptosis, yielding a more effective treatment strategy.

The University of Cincinnati Cancer Center Clinical Trials Office is opening a sub-study of the Beat AML Master Clinical Trial titled, A Randomized Phase 2 Trial of 28 Day (Arm A) versus 14 Day (Arm B) Schedule of Venetoclax (Ven) + Azacitidine (Aza) in newly diagnosed acute myeloid leukemia (AML) patients ≥ 60 years comparing a lower than standard dosage of this widely used treatment. This clinical trial is designed to evaluate the efficacy this dosage and treatment schedule of Venetoclax plus Azacitidine may have for newly diagnosed AML patients. This trial will inform optimal dosing and safety information for more tailored treatments in the future. The results of this study could potentially improve treatment outcomes and survival rates for patients well beyond the local area.

Enrollment will open to all AML patients, with a defined number of spots open per site.

To learn more about this clinical trial, contact the University of Cincinnati Cancer Center Clinical Trials Office at 513-584-7698 to reach our clinical research staff. You can also find the trial on the High Enroll app.

About Beat AML

According to the Leukemia & Lymphoma Society, the Beat AML Master Clinical Trial, “is the first collaborative precision medicine clinical trial in a blood cancer. The trial uses advanced genomic technology to identify each patient’s cancer-driving genetic mutations, and then matches patients to the most promising, targeted treatment.” Learn more about Beat AML on ClinicalTrials.gov.