Research suggests that a new targeted therapy, quizartinib, may be a safe and effective option to treat a subset of patients with treatment-resistant acute myeloid leukaemia (AML), whose leukaemia has the FLT3-ITD mutation.

Quizartinib is the first and only single-agent drug that has produced a clinical benefit in AML patients with this deadly mutation who have failed previous therapy.

The number of patients bridged to a stem cell transplant was very significant. Dr Levis and his colleagues plan on using these encouraging results to design and conduct additional randomized trials that will hopefully lead to the approval of quizartinib to make it accessible to those patients who previously had no hope for a cure.

From an official Press Conference at ASH 2012.