Dr. Mannis on Targeted Agents in the Treatment of Patients With AML

Gabriel Mannis, MD, assistant professor, University of California, San Francisco Helen Diller Family Comprehensive Cancer Center, discusses the FDA approval and potential approval of 2 new agents that will provide a more nuanced treatment approach for patients with acute myeloid leukemia (AML).

The success of recent drug development is due, in part, to the realization that not all AML cases are the same. Every patient presents a unique form of AML and should be treated accordingly. By understanding the molecular heterogeneity of the disease, physicians are better able to target each case of AML.

IDH1/2 mutations are present in 6% to 13% of patients. The 2017 approval of the IDH2 inhibitor enasidenib (Idhifa) in the setting of relapsed/refractory AML shows a 20% remission rate and 40% overall response rate (ORR). It is very well tolerated, so older patients can receive it.

Mannis and colleagues at UCSF participated in the phase I study of the IDH1 inhibitor ivosidenib (AG-120). Ivosidenib is projected to be approved early in 2018.