FLT3 inhibitors: A story of the old and the new

Amir Fathi, Mark Levis

Research output: Contribution to journalArticlepeer-review

38 Scopus citations


Purpose of Review: Ever since the recognition that FMS-like tyrosine kinase 3 (FLT3) mutations exert a profound negative prognostic impact on the clinical outcome of patients with acute myeloid leukemia (AML), researchers have sought to find effective small-molecule inhibitors of this receptor tyrosine kinase. This review will attempt to provide a survey of the FLT3 inhibitors currently under investigation and provide a discussion on their current status in clinical trials. Recent Findings: Over the past 10 years, a number of different compounds have been studied in vitro and clinically as FLT3 inhibitors. The first inhibitors studied were hampered by cumbersome pharmacokinetics and a general lack of potency. However, some agents have shown promise in clinical trials with transient responses in AML. Newer compounds, such as AC220, have demonstrated profound selectivity and potency against the FLT3 target, and are currently being investigated in clinical trials. Summary: Clinical trials have so far demonstrated that inhibitors of FLT3 do have clinical activity in patients with FLT3-mutant AML, although this activity is often transient and correlates with effective in-vivo suppression of the FLT3 target. As newer, more potent agents are now entering advanced clinical trials, opportunities will emerge for real progress against this grim disease.

Original languageEnglish (US)
Pages (from-to)71-76
Number of pages6
JournalCurrent opinion in hematology
Issue number2
StatePublished - Mar 2011


  • FLT3
  • acute myeloid leukemia
  • internal tandem duplication
  • tyrosine kinase inhibitors

ASJC Scopus subject areas

  • Hematology


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