Abstract
Although complete remissions can be achieved in most patients younger than 60 years of age with untreated acute myeloid leukemia (AML), only 30-40 % of patients remain long-term survivors. Furthermore, long-term survivors represent only 10-15 % of all AML patients older than 60 years of age and <10 % of all patients with relapsed AML. The development of new treatments for AML is therefore needed. Novel therapies should target specific mechanisms and pathways implicated in the development and maintenance of AML, should strive to have better tolerability than conventional combination chemotherapy, be associated with improved quality of life and minimize utilization of health care resources. In this manuscript, we discuss the role of epigenetic regulators and immunomodulatory agents in the treatment of AML. Also, we review the data on inhibitors of protein homeostasis and its synergistic effect to DNA methyltransferase inhibitors, the potential role for inhibitors of heat shock proteins and the mitotic machinery and a novel formulation of conventional chemotherapeutic agents given at a fixed molar concentration. Finally, we briefly share our views on optimal clinical trial design and patient selection for future studies in AML.
Highlights
It is estimated that nearly 14000 adults will be diagnosed with acute myeloid leukemia (AML) in the US in 2012, and about 10000 will die from leukemia (SEER database, NCI)
Adult patients with AML who are candidates for intensive therapy are treated with the combination of cytarabine and daunorubicin, a regimen that was first introduced in the 1970s and is typically followed by three to four cycles of consolidation with high dose cytarabine or allogeneic hematopoietic stem cell transplantation
We describe some of the novel agents currently being used and tested in the treatment of AML that we find promising (Fig. 1)
Summary
It is estimated that nearly 14000 adults will be diagnosed with acute myeloid leukemia (AML) in the US in 2012, and about 10000 will die from leukemia (SEER database, NCI). It should be noted that an important exception to the above-mentioned statistics is acute promyelocytic leukemia (APL) This type of AML, formerly designated as M3, has a much better prognosis due to the implementation of sensitive molecular diagnostic tools and introduction of all-trans retinoic acid and arsenic trioxide into clinical practice. Improved prognostic stratification based on molecular abnormalities and a detailed understanding of the mechanisms of leukemogenesis may allow hematologists to more precisely tailor available therapy to individual patients and lead to the identification of new therapeutic targets. The tremendous advances in the understanding of the molecular biology and biochemistry of hematopoiesis and transformation to leukemia have lead to the identification of numerous putative therapeutic targets and a growing number of promising investigational agents. The role of newer immunomodulating drugs such as pomalidomide in the treatment of AML has not been established but merits further investigation
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