Michael Andreeff, MD, PhD, chair of genetics, professor of medicine, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, discusses the promise of venetoclax in acute myeloid leukemia.
Michael Andreeff, MD, PhD, chair of genetics, professor of medicine, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, discusses the promise of venetoclax (Venclexta) in the treatment of patients with acute myeloid leukemia (AML).
BCL2 is a survival factor for AML cells, particularly stem cells, Andreef says. For decades, this molecule was considered untargetable, but research over the last few years has proven otherwise. Recent data has suggested that the best established BCL2 inhibitor is venetoclax. In June 2018, venetoclax was granted full approval by the FDA for the treatment of patients with chronic lymphocytic leukemia or small lymphocytic leukemia, regardless of 17p deletion. The drug has shown promise even in patients with p53-mutant CLL, who are known to have a particularly poor prognosis.
Despite this success, when used in patients with AML, the agent had been largely ineffective and trials examining the use of venetoclax monotherapy had been negative. Combining the BCL2 inhibitor with chemotherapy agents like decitabine, however, has shown tremendous efficacy in elderly patients who previously had no effective options available to them. For example, results from a study conducted by The University of Texas MD Anderson Cancer Center and presented at the 2018 ASH Annual Meeting showed that the use of venetoclax in combination with demethylating agents resulted in response rates of up to 100% in this patient population.