Targeted and Less Toxic: The Future of AML Treatment

In an interview with Targeted Oncology, Richard Stone, MD, Dana-Farber Cancer Institute and Harvard Medical School, discusses how evolving treatment strategies may further diminish the role of the traditional 7+3 chemotherapy regimen as the sole induction therapy for acute myeloid leukemia (AML).

Watch the first part of Dr Stone’s interview.

As targeted agents and combination regimens become more widely integrated into frontline treatment, the question of whether 7+3 alone remains relevant may soon become less central to clinical decision-making. Stone notes that even today, relatively few patients receive 7+3 by itself. Instead, most treatment approaches incorporate additional agents tailored to disease biology or patient characteristics.

Looking ahead, Stone anticipates that the number of patients receiving 7+3 alone will continue to decline and may eventually approach 0. Rather than abandoning chemotherapy entirely, clinicians may increasingly combine it with other active agents. For patients with chemotherapy-responsive disease, regimens such as 7+3 plus venetoclax could emerge as potential strategies.

At the same time, less intensive approaches are likely to expand further. Combinations of hypomethylating agents—such as azacitidine (Vidaza) or decitabine—with venetoclax (Venclexta) are already widely used, particularly in older or less fit patients. These regimens may also be paired with additional targeted therapies depending on molecular drivers.

Stone highlights the FLT3-mutated AML setting as an example of how treatment paradigms could evolve. In the future, patients with FLT3 mutations may receive combinations such as a hypomethylating agent, venetoclax, and a FLT3 inhibitor, potentially even as fully oral regimens. Such strategies could induce remission with less toxicity and may allow some patients to proceed directly to stem cell transplant—or possibly avoid transplant altogether.

Overall, Stone expresses optimism that continued therapeutic innovation will enable clinicians to treat most patients with AML using less toxic yet highly effective approaches.

Read the full interview here.