Rare Disease Highlight: Acute Myeloid Leukemia

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Rare Disease Highlight

Acute Myeloid Leukemia (AML) is a blood cancer in which blast cells (immature myeloid precursor cells) multiply uncontrollably within the bone marrow and blood.1 The accumulation of blast cells disrupts normal blood cell production in the bone marrow as well as blood distribution to the tissues. This disruption leads to symptoms such as chronic fatigue, anemia, a compromised immune system, and high risk of hemorrhage.4,5 In addition, the current treatments for AML can cause many additional side effects due to drug toxicity.6-8 As these combined effects are severe, AML patients have low overall survival despite treatment and can still relapse after recovery is observed.2,3 According to the Surveillance, Epidemiology, and End Results (SEER) program, there were 19,520 new cases of AML in the US in 2018, with a reported 5-year survival rate of 27.4%.9  Risk factors associated with AML include age, history of blood disease, and/or genetic disorders.2,3 Thus, an unmet need exists for AML therapies which are non-invasive, non-toxic, and do not result in relapse.

References used
  1. Spivak JL. Acute Myelogenous Leukemia (AML). Merck Manual 2017; http://www.merckmanuals.com/professional/hematology-and-oncology/leukemias/acute-myelogenous-leukemia-aml. Accessed November 20, 2017, 2017.
  2. Pui C-H, Carroll WL, Meshinchi S, Arceci RJ. Biology, Risk Stratification, and Therapy of Pediatric Acute Leukemias: An Update. J Clin Oncol. 2011;29(5):551-565.
  3. PDQ PTEB. Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®). 2017.
  4. Cheng MJ, Hourigan CS, Smith TJ. Adult Acute Myeloid Leukemia Long-term Survivors. Journal of leukemia (Los Angeles, Calif). 2014;2(2):26855.
  5. Dohner H, Estey EH, Amadori S, et al. Diagnosis and management of acute myeloid leukemia in adults: recommendations from an international expert panel, on behalf of the European LeukemiaNet. Blood. 2010;115(3):453-474.
  6. Lipshultz SE, Sallan SE. Cardiovascular abnormalities in long-term survivors of childhood malignancy. J Clin Oncol. 1993;11(7):1199-1203.
  7. Volkova M, Russell R. Anthracycline Cardiotoxicity: Prevalence, Pathogenesis and Treatment. Curr Cardiol Rev. 2011;7(4):214-220.
  8. Von Hoff DD, Layard MW, Basa P, et al. Risk factors for doxorubicin-induced congestive heart failure. Ann Intern Med. 1979;91(5):710-717.
  9. SEER. Cancer Stat Facts: Leukemia – Acute Myeloid Leukemia (AML). 2018; https://seer.cancer.gov/statfacts/html/amyl.html.

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