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Amonafide: a future in treatment of resistant and secondary acute myeloid leukemia?

Ciara L Freeman1, Ronan Swords, Francis J Giles

  • 1Mayo Clinic Phoenix Campus, Department of Hematology & Oncology, 5777 East Mayo Boulevard, Phoenix, AZ 85054, USA. freemanciara@yahoo.co.uk

Expert Review of Hematology
|January 26, 2012
PubMed

Insights

Amonafide, a topoisomerase II inhibitor, showed early promise for cancer treatment but ultimately failed to improve survival in acute myeloid leukemia (AML) patients when combined with cytarabine.

Area of Science:

  • Oncology
  • Pharmacology

Background:

  • Amonafide, a novel topoisomerase II inhibitor, was developed nearly 40 years ago.
  • Initial preclinical studies demonstrated significant antineoplastic efficacy.
  • Its development shifted towards acute myeloid leukemia (AML), particularly secondary and treatment-associated forms.

Purpose of the Study:

  • To review the development of amonafide from preclinical research to clinical application.
  • To discuss the potential role of amonafide in the current management of AML.

Main Methods:

  • Review of amonafide's development history.
  • Analysis of preclinical data and clinical trial outcomes (Phase I, II, and randomized studies).

Main Results:

  • Amonafide showed limited efficacy in solid tumors but demonstrated potential in AML.
  • Phase I/II studies suggested promise for older patients with multidrug-resistant AML.
  • Randomized studies combining amonafide with cytarabine did not show a survival advantage over standard care.

Conclusions:

  • Despite initial promise, amonafide did not demonstrate a survival benefit in randomized AML trials.
  • The article discusses amonafide's journey and its limited current role in AML management.

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