Improving Cure Rates in AML with Targeted Combinations

Naval G. Daver, MD, professor, Department of Leukemia, Division of Cancer Medicine, and director, Department of Leukemia, Division of Leukemia Research Alliance Program, The University of Texas MD Anderson Cancer Center, reviews the rapid advances in the treatment of acute myeloid leukemia (AML) over the past decade. He notes that 11 new FDA drug approvals in the last 8–10 years have transformed the field, shifting the treatment paradigm from a uniform chemotherapy-based approach to a precision medicine strategy guided by molecular and genetic profiling.

For younger, fit patients, intensive chemotherapy remains the backbone of induction therapy. However, the integration of targeted agents, such as FLT3 inhibitors for FLT3-mutated disease, IDH inhibitors for IDH1/2-mutated disease, and the emerging menin inhibitors has significantly improved outcomes. Dr. Daver reports that in appropriate molecularly defined subgroups, these combinations are yielding cure rates approaching 70%–75%.

For older patients (typically >70–75 years) or those unfit for intensive chemotherapy, progress has been more incremental. The current standard in this population often combines hypomethylating agents (HMAs) with venetoclax. Ongoing clinical trials are now evaluating the addition of targeted therapies—including FLT3 inhibitors, IDH inhibitors, and menin inhibitors to HMA–venetoclax backbones. Early data suggests these triplet regimens may further improve remission rates and overall survival in this historically high-risk group.

Dr. Daver underscores that while treatment advances have markedly improved prognosis for younger, fit patients with AML, ongoing research is critical to closing the survival gap for older or frail individuals. With continued drug development and optimized combinations, the goal is to achieve durable remissions across all patient subsets.