Arsenic and old lace: novel approaches in elderly patients with acute myeloid leukemia

Arsenic has been used for more than 2,000 years in the treatment of a variety of medical conditions, including plague, hysteria, syphilis, and cancer. Numerous potential mechanisms of action have been identified. Arsenic trioxide has remarkable efficacy in acute promyelocytic leukemia and is approved by the US Food and Drug Administration for this indication. It has also been studied in acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and multiple myeloma and has limited single-agent efficacy in these diseases. We have completed a phase I/II trial of arsenic trioxide combined with low-dose ara-C (LDAC) in 49 patients with Int-2/high-risk MDS and 64 patients age 60 years and older with untreated AML. The regimen was generally well tolerated and complete remissions were observed in both MDS and AML patients, including in patients with poor baseline performance status and unfavorable cytogenetics. Manuscript has been accepted for publication.